1. STAMFORD, Conn., Sept. 07, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that management will participate in the H.C. Wainwright 23rd Annual Global Investment Conference. A webcast of the company presentation will be available on-demand starting Monday, September 13, 2021 at 7:00 am ET.

    To access the webcast, please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the conference.

    About

    STAMFORD, Conn., Sept. 07, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that management will participate in the H.C. Wainwright 23rd Annual Global Investment Conference. A webcast of the company presentation will be available on-demand starting Monday, September 13, 2021 at 7:00 am ET.

    To access the webcast, please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the conference.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for patients living with severe rare diseases and cancer. SpringWorks has a differentiated targeted oncology portfolio of small molecule product candidates and is advancing 15 development programs, including two potentially registrational clinical trials in rare tumor types as well as several programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with innovators in industry and academia to expand its portfolio and create more solutions for patients with cancer. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn

    Contact:

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com 



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  2. STAMFORD, Conn., Aug. 30, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that it has entered into a sponsored research collaboration with Dana-Farber Cancer Institute (Dana-Farber). The collaboration is designed to further investigate nirogacestat, SpringWorks' investigational gamma secretase inhibitor (GSI), with anti-B-cell maturation antigen (BCMA) agents in a variety of preclinical multiple myeloma models. Constantine Mitsiades, M.D., Ph.D., Assistant Professor of Medicine at Dana-Farber and Harvard Medical School, will serve as the Principal Investigator for…

    STAMFORD, Conn., Aug. 30, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that it has entered into a sponsored research collaboration with Dana-Farber Cancer Institute (Dana-Farber). The collaboration is designed to further investigate nirogacestat, SpringWorks' investigational gamma secretase inhibitor (GSI), with anti-B-cell maturation antigen (BCMA) agents in a variety of preclinical multiple myeloma models. Constantine Mitsiades, M.D., Ph.D., Assistant Professor of Medicine at Dana-Farber and Harvard Medical School, will serve as the Principal Investigator for the research, which will utilize preclinical in vivo models from the Mitsiades Lab to simulate the multiple myeloma tumor microenvironment. In addition, the research will also deploy functional genomics approaches to explore determinants of response and mechanisms of resistance to GSI + BCMA combination therapy.

    Gamma secretase inhibition prevents the cleavage and shedding of BCMA from the surface of multiple myeloma cells. In preclinical models, nirogacestat has been shown to increase the cell surface density of BCMA and reduce levels of soluble BCMA, thereby enhancing the activity of BCMA-targeted therapies.1 To date, SpringWorks has entered into clinical collaborations with six industry partners to evaluate nirogacestat in combination with BCMA therapies across modalities.

    "SpringWorks has demonstrated its commitment to understanding the science and advancing the clinical exploration of GSI + BCMA combination therapies," said Dr. Mitsiades. "I am pleased to collaborate with them towards the goal of improving clinical outcomes for patients with multiple myeloma while simultaneously enabling the advancement of the underlying science."

    "We are delighted to be collaborating with the Mitsiades Lab at Dana-Farber to expand our understanding of nirogacestat's mechanism of action when combined with BCMA therapies," said Badreddin Edris, Ph.D., Chief Operating Officer of SpringWorks. "Dr. Mitsiades and his colleagues have been at the forefront of developing cutting-edge translational models of multiple myeloma and we believe that this collaboration has the potential to yield novel insights that can help us to further refine our approach to improving outcomes for these patients."

    Under the terms of the sponsored research agreement, SpringWorks will be responsible for funding the work and will retain an option to exclusively license any new intellectual property emerging from the research collaboration.

    About Nirogacestat

    Nirogacestat is an investigational, oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has six collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities, including with an antibody-drug conjugate, two CAR T cell therapies, two bispecific antibodies and a monoclonal antibody. SpringWorks has also formed research collaborations with Fred Hutchinson Cancer Research Center and Dana-Farber Cancer Institute to further characterize the ability of nirogacestat to modulate BCMA and potentiate BCMA therapies using a variety of preclinical multiple myeloma models.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors and from the European Commission for the treatment of soft tissue sarcoma. The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for patients living with severe rare diseases and cancer. SpringWorks has a differentiated targeted oncology portfolio of small molecule product candidates and is advancing 15 development programs, including two potentially registrational clinical trials in rare tumor types as well as several programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with innovators in industry and academia to expand its portfolio and create more solutions for patients with cancer. For more information, please visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success and timing of our product development activities, including the initiation and completion of SpringWorks' clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our collaboration partners' ongoing and planned clinical trials, (iv) our ability to obtain and maintain regulatory approval of any of our product candidates, (v) our plans to research, discover and develop additional product candidates, (vi) our ability to enter into collaborations for the development of new product candidates, (vii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (viii) our ability to meet any specific milestones set forth herein, and (ix) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines.

    Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements.

    For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" in Item 1A of Part I of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings.

    SpringWorks Media/Investor Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    203-561-1646

    kdiamond@springworkstx.com

    References

    1Eastman S, Shelton C, Gupta I, Krueger J, Blackwell C, Bojczuk P. Synergistic Activity of Belantamab Mafodotin (anti-BCMA immuno-conjugate) with PF-03084014 (gamma-secretase inhibitor) in Bcma-Expressing Cancer Cell Lines. Blood. 2019;134(supplement_1):4401. doi:10.1182/blood-2019-123705.



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  3. Entered into Clinical Collaboration with Seagen to Evaluate Nirogacestat in Combination with SEA-BCMA and Dosed First Patients in Three Phase 1 Clinical Trials Evaluating Nirogacestat in Combination with BCMA Therapies Across Modalities in Patients with Relapsed or Refractory Multiple Myeloma

    Announced Issuance of New U.S. Composition of Matter Patent to Polymorphic Form of Mirdametinib, Extending Patent Protection into 2041

    Announced Several New Clinical Trials for Mirdametinib, Including a Phase 1/2 Trial in Children and Young Adults with Low-Grade Glioma and a Phase 1b/2a Trial in Patients with Advanced Solid Cancers Harboring Selected MAPK-Activating Mutations

    Expanded Targeted Oncology Pipeline with Exclusive Worldwide

    Entered into Clinical Collaboration with Seagen to Evaluate Nirogacestat in Combination with SEA-BCMA and Dosed First Patients in Three Phase 1 Clinical Trials Evaluating Nirogacestat in Combination with BCMA Therapies Across Modalities in Patients with Relapsed or Refractory Multiple Myeloma

    Announced Issuance of New U.S. Composition of Matter Patent to Polymorphic Form of Mirdametinib, Extending Patent Protection into 2041

    Announced Several New Clinical Trials for Mirdametinib, Including a Phase 1/2 Trial in Children and Young Adults with Low-Grade Glioma and a Phase 1b/2a Trial in Patients with Advanced Solid Cancers Harboring Selected MAPK-Activating Mutations

    Expanded Targeted Oncology Pipeline with Exclusive Worldwide License to TEA Domain (TEAD) Inhibitor Portfolio

    Strengthened Leadership Team with Appointments of Dr. Mike Burgess as Head of Research and Development and Dr. Jim Cassidy as Chief Medical Officer

    STAMFORD, Conn., Aug. 04, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported financial results for the second quarter and year-to-date periods ended June 30, 2021 and provided an update on recent company developments.

    "We are very pleased with our execution in the second quarter of 2021, which included continuing to advance our strategy to evaluate nirogacestat as a BCMA potentiator across modalities, building upon our intellectual property portfolio by extending the patent protection for mirdametinib into 2041, and expanding our targeted oncology pipeline through collaborations with leading cancer centers as well as the in-license of a TEAD inhibitor portfolio," said Saqib Islam, Chief Executive Officer of SpringWorks. "In the second half of the year we expect important data readouts across our late-stage rare oncology, BCMA combinations in multiple myeloma and metastatic solid tumor programs, including topline data from our Phase 3 DeFi study. In parallel, we will continue to advance our 15 development programs on behalf of oncology patients."

    Recent Business Highlights and Upcoming Milestones

    Late-Stage Rare Oncology

    • SpringWorks expects to report topline data from the Phase 3 DeFi trial evaluating nirogacestat in adult patients with progressing desmoid tumors in the second half of 2021, as previously disclosed.
    • Recruitment is ongoing in a Phase 2 study sponsored by the Children's Oncology Group evaluating nirogacestat in pediatric patients with desmoid tumors.
    • Updated interim data from the adult stratum of the ongoing potentially registrational Phase 2b ReNeu trial evaluating mirdametinib in pediatric and adult patients with NF1-associated plexiform neurofibromas were presented at the 2021 Children's Tumor Foundation NF Conference in June 2021. These updated data were based on a cut-off date of March 23, 2021 and showed durable efficacy in the same 20 patients reported in February 2021, with the median time on treatment now having reached 13 cycles (approximately 12 months); the objective response rate remained at 50%, and 80% of these patients remained on study at the data cutoff. In addition, mirdametinib remained generally well tolerated; the majority of treatment-related adverse events (TRAE) in these patients were Grade 1 or 2, with only one Grade 3 TRAE observed and no Grade 4 or 5 adverse events (AE) reported in these 20 patients as of the data cutoff. The Company expects to complete enrollment of the trial in the second half of 2021.
    • In June 2021, SpringWorks entered into a collaboration to conduct a Phase 1/2 clinical trial evaluating mirdametinib in children and young adults with low-grade glioma. Patients are being dosed in this study, which is sponsored by St. Jude Children's Research Hospital.

    B-cell Maturation Antigen (BCMA) Combinations in Multiple Myeloma

    • In June 2021, SpringWorks entered into a clinical trial collaboration agreement with Seagen Inc. to evaluate nirogacestat in combination with SEA-BCMA, Seagen's investigational monoclonal antibody targeting B-cell maturation antigen (BCMA) in patients with relapsed or refractory multiple myeloma.
    • Enrollment is ongoing in four clinical trials evaluating nirogacestat in combination with BCMA therapies in adult patients with relapsed or refractory multiple myeloma: a Phase 1b trial sponsored by GSK evaluating nirogacestat in combination with BLENREP (belantamab mafodotin-blmf), a Phase 1 study sponsored by Allogene evaluating nirogacestat in combination with ALLO-715, a Phase 1 study sponsored by Janssen evaluating nirogacestat in combination with teclistamab, and a Phase 1/2a study sponsored by Precision BioSciences evaluating nirogacestat in combination with PBCAR269A. Initial data from the GSK-sponsored study are expected in 2021. SpringWorks also expects that two additional collaborator-sponsored trials will initiate enrollment in the second half of 2021, as previously disclosed: nirogacestat + Pfizer's elranatamab and nirogacestat + Seagen's SEA-BCMA.

    Biomarker-Defined Metastatic Solid Tumors

    • In May 2021, SpringWorks entered into an exclusive worldwide license agreement with Katholieke Universiteit Leuven (KU Leuven) and the Flanders Institute for Biotechnology (VIB) for the in-license of a portfolio of novel small molecule inhibitors of the TEAD family of transcription factors, designed for the potential treatment of biomarker-defined solid tumors driven by aberrant Hippo pathway signaling. The licensed portfolio includes advanced lead compounds and multiple backup compounds from diverse chemical series, which were discovered at KU Leuven's Center for Drug Design and Discovery (CD3) in collaboration with Professor Georg Halder of the VIB-KU Leuven Center for Cancer Biology. SpringWorks expects to nominate a development candidate from this portfolio and commence IND-enabling studies in 2022.
    • Enrollment is ongoing in a Phase 1b/2 trial evaluating mirdametinib with BeiGene's RAF dimer inhibitor, lifirafenib, in adult patients with RAS/RAF mutant and other MAPK pathway aberrant solid tumors. Initial clinical data from the BeiGene-sponsored trial are expected in 2021, as previously disclosed.
    • Enrollment is ongoing in a Phase 1 trial of BGB-3245 in adult patients with RAF mutant solid tumors. BGB-3245 is a selective RAF dimer inhibitor being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene. Initial clinical data from the MapKure-sponsored trial are expected in 2021, as previously disclosed.
    • In August 2021, SpringWorks announced a platform study sponsored by Memorial Sloan Kettering Cancer Center to evaluate mirdametinib both as a monotherapy and as a combination therapy in advanced solid tumors harboring selected MAPK-activating mutations. This trial is planned to initially explore mirdametinib in two patient cohorts: the first in combination with fulvestrant, a selective estrogen receptor degrader, in patients with estrogen receptor-positive metastatic breast cancer with MAPK alterations (particularly inactivating mutations in NF1) and the second as a monotherapy in advanced solid tumors harboring oncogenic MEK1 or MEK2 mutations.
    • In May 2021, SpringWorks entered into a research collaboration with the University of Zurich to further explore the activity of mirdametinib and lifirafenib combination therapy in preclinical patient-derived NRAS-mutant solid tumors models. The research, which is being led by Reinhard Dummer, M.D., builds upon previous translational work presented by SpringWorks and BeiGene and is intended to support ongoing and future patient selection strategies and to further define the competitive differentiation of the MAPK pathway vertical inhibition strategy supported by the combination of mirdametinib and lifirafenib.

    General Corporate

    • In July 2021, the United States Patent and Trademark Office issued a new composition of matter patent that covers the polymorphic form of mirdametinib that is currently in clinical development. This patent expires in 2041.
    • SpringWorks appointed Mike Burgess, M.B.Ch.B., Ph.D. as Head of Research and Development and Jim Cassidy, M.D., Ph.D. as Chief Medical Officer. Drs. Burgess and Cassidy each bring decades of experience in oncology drug discovery and development as physician-scientists and R&D leaders at leading biotech and pharmaceutical companies.

    Second Quarter 2021 Financial Results

    • Research and Development (R&D) Expenses: R&D expenses were $32.1 million for the second quarter, compared to $12.9 million for the comparable period of 2020. The increases in R&D expenses were primarily attributable to a nonrefundable upfront payment to KU Leuven and VIB for the in-licensing of the TEAD inhibitor program, an increase in external costs related to drug manufacturing and trial costs, and an increase in internal costs driven by the growth in employee costs associated with increases in the number of R&D personnel and an increase in stock-based compensation expense.
    • General and Administrative (G&A) Expenses: G&A expenses were $14.9 million for the second quarter, compared to $6.9 million for the comparable period of 2020. The increases in G&A expenses were primarily attributable to the hiring of additional personnel in G&A functions supporting the growth of the organization, as well as an increase in stock-based compensation expense.
    • Net Loss Attributable to Common Stockholders: SpringWorks reported net loss of $47.0 million, or $0.97 per share, for the second quarter of 2021. This compares to a net loss of $19.9 million, or $0.47 per share, for the comparable period of 2020.
    • Cash Position: Cash, cash equivalents and marketable securities were $507.5 million as of June 30, 2021.

    COVID-19 Update

    To date, the COVID-19 pandemic has had a relatively modest impact on SpringWorks' business operations, in particular on SpringWorks' clinical trial programs, and SpringWorks is undertaking considerable efforts to mitigate the various challenges presented by this crisis. For further details and descriptions of the risks associated with the COVID-19 pandemic, please see the Risk Factors in SpringWorks' periodic filings with the Securities and Exchange Commission and refer to the Forward-Looking Statements section in this press release.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for patients living with severe rare diseases and cancer. SpringWorks has a differentiated targeted oncology portfolio of small molecule product candidates and is advancing 15 development programs, including two potentially registrational clinical trials in rare tumor types as well as several programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with innovators in industry and academia to expand its portfolio and create more solutions for patients with cancer. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    SpringWorks uses its website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the Company's website in the ‘Investors & Media' section. Accordingly, investors should monitor such portions of the SpringWorks website, in addition to following press releases, SEC filings and public conference calls and webcasts.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for initiation, progress and completion of SpringWorks' clinical trials or third-party clinical trials of its product candidates, the timing for expected data readouts from partners and partners' clinical trials, the expected benefits of collaborations, the fact that interim results from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials involving its product candidates, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in SpringWorks' SEC filings. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.



    SpringWorks Therapeutics, Inc.

    Condensed Consolidated Statements of Operations

    (Unaudited)



    Three Months Ended

    June 30,
     Six Months Ended

    June 30,
    (in thousands, except share and per-share data)2021 2020 2021 2020
    Operating expenses:       
    Research and development$32,091   $12,947   $49,466   $22,674  
    General and administrative14,930   6,874   27,311   13,277  
    Total operating expenses47,021   19,821   76,777   35,951  
           
    Loss from operations(47,021)  (19,821)  (76,777)  (35,951) 
    Interest and other income:       
    Other income(41)     (38)    
    Interest income, net211   157   438   1,093  
    Total interest and other income170   157   400   1,093  
    Equity investment loss(159)  (229)  (420)  (329) 
    Net loss$(47,010)  $(19,893)  $(76,797)  $(35,187) 
           
    Net loss per share, basic and diluted$(0.97)  $(0.47)  $(1.59)  $(0.84) 
    Weighted average common shares outstanding, basic and diluted48,422,921   41,945,058   48,326,764   41,867,089  
                    

    SpringWorks Therapeutics, Inc.

    Selected Balance Sheet Data

    (Unaudited)



     June 30, 2021 December 31, 2020
    (in thousands)   
    Cash, cash equivalents and marketable securities$507,483   $561,820  
    Working capital (1)443,978   495,788  
    Total assets520,428   576,191  
    Total liabilities23,845   19,133  
    Accumulated deficit(195,400)  (118,603) 
    Total stockholders' equity496,583   557,058  

    (1) We define working capital as current assets less current liabilities.





    Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com

     



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  4. STAMFORD, Conn., Aug. 03, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that management will participate in a Fireside Chat at the 12th Annual Wedbush PacGrow Healthcare Conference on Wednesday, August 11th at 9:45AM ET.

    To access the live webcast please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the conference.

    About SpringWorks Therapeutics
    SpringWorks is a clinical-stage…

    STAMFORD, Conn., Aug. 03, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that management will participate in a Fireside Chat at the 12th Annual Wedbush PacGrow Healthcare Conference on Wednesday, August 11th at 9:45AM ET.

    To access the live webcast please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the conference.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for patients living with severe rare diseases and cancer. SpringWorks has a differentiated targeted oncology portfolio of small molecule product candidates and is advancing 15 development programs, including two potentially registrational clinical trials in rare tumor types as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with innovators in industry and academia to expand its portfolio to create more solutions for patients with cancer. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Contact:

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com

     



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  5. STAMFORD, Conn., Aug. 03, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the Company will be evaluating mirdametinib, an investigational MEK inhibitor, in a platform study sponsored by Memorial Sloan Kettering Cancer Center (MSK) and supported by SpringWorks exploring the compound both as a monotherapy and as a combination therapy in advanced solid tumors harboring MAPK-activating mutations. The trial, which is expected to begin recruiting patients during the third quarter of 2021, will initially explore mirdametinib in two patient cohorts: the first in combination…

    STAMFORD, Conn., Aug. 03, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the Company will be evaluating mirdametinib, an investigational MEK inhibitor, in a platform study sponsored by Memorial Sloan Kettering Cancer Center (MSK) and supported by SpringWorks exploring the compound both as a monotherapy and as a combination therapy in advanced solid tumors harboring MAPK-activating mutations. The trial, which is expected to begin recruiting patients during the third quarter of 2021, will initially explore mirdametinib in two patient cohorts: the first in combination with fulvestrant, a selective estrogen receptor degrader (SERD) in patients with estrogen receptor positive (ER+) metastatic breast cancer (mBC) with MAPK alterations (particularly inactivating mutations in NF1), and as a monotherapy in advanced solid tumors harboring oncogenic MEK1 or MEK2 mutations.

    "Emerging evidence points to alterations in the MAPK pathway playing a key role in mediating resistance to hormone therapy in ER+ mBC, which represents a significant unmet medical need," explained Ezra Rosen, M.D., Ph.D., Medical Oncologist, Assistant Member of MSK's Early Drug Development Service, and the study's principal investigator. "Based on emerging preclinical data, combinations of MAPK pathway inhibitors with ER-targeted therapy could potentially address this resistance mechanism and we look forward to studying mirdametinib to evaluate whether this MEK inhibitor can provide a clinical benefit. Separately, given the preclinical evidence that activating mutations in MEK1 and MEK2 can also act as oncogenic drivers in cancer, we're looking to explore a potential role for mirdametinib monotherapy in solid tumors harboring these driver mutations."

    Approximately 70% of breast cancers are ER+. Hormonal therapies targeting ER, such as SERDs, can be effective in treating ER+ mBC, but over 90% of patients eventually develop resistance to ER-targeted therapy. Loss of NF1 function has been shown to be responsible for enhanced ER transcriptional activity and reduced sensitivity to fulvestrant in preclinical models, with up to 6% of ER+ mBC patients harboring a detectable NF1 mutation.1 Combinations of MAPK pathway inhibitors and ER-targeted therapy could potentially address this resistance, as demonstrated by a combination of a MEK inhibitor and fulvestrant showing anti-tumor activity in fulvestrant-refractory NF1-deficient ER+ mBC preclinical models.1,2

    In addition, MEK1 and MEK2 mutations are present in up to 2% of solid tumors and have been validated as oncogenic drivers. Recent publications demonstrate the activity of MEK inhibitors, including mirdametinib, in preclinical models driven by a subset of these MEK mutations.3,4

    "This biomarker-driven platform study will enable us to evaluate mirdametinib's ability to address subsets of patients with solid tumors that harbor specific MAPK pathway mutations," said Mike Burgess, M.B.Ch.B., Ph.D., Head of Research and Development at SpringWorks. "We are committed to exploring the full potential of mirdametinib on behalf of patients with devastating cancers and look forward to collaborating with Dr. Rosen and his colleagues at MSK on this important trial."

    About the MSK-Sponsored Phase 1b/2a Trial

    The open-label Phase 1b/2a parallel design, platform study will evaluate the safety and tolerability, efficacy, and pharmacokinetics of mirdametinib in two study arms: (1) in combination with fulvestrant in postmenopausal patients with ER+ mBC harboring NF1 loss of function or other alterations of the MAPK pathway and (2) as a monotherapy in adult patients with advanced solid cancers driven by the alterations of the MAPK pathway, including MEK1 or MEK2 mutations.

    The primary objectives of the trial will be to evaluate the safety and tolerability and anti-tumor efficacy of mirdametinib in combination with fulvestrant and as a single agent. The efficacy endpoints will include best objective response by RECIST 1.1, disease control rate, duration of response, progression-free survival, and pharmacokinetic endpoints. Biomarker analyses will also be conducted to evaluate the changes from baseline in the biomarkers of tumor biology and anti-tumor activity and characterize potential mechanisms of resistance.

    About Mirdametinib

    Mirdametinib is an oral, potent, allosteric, brain-penetrant small molecule designed to inhibit MEK1 and MEK2, which are proteins that occupy pivotal positions in the MAPK pathway and that play a central role in multiple oncology and rare disease indications. To date, over 250 subjects have been exposed to treatment with mirdametinib across clinical trials, with preliminary evidence of clinical activity against tumors driven by over-activated MAPK signaling.5

    Mirdametinib is being evaluated as a monotherapy in a Phase 2b trial for pediatric and adult patients with NF1-associated plexiform neurofibromas (NF1-PN), and in a Phase 1/2 trial for patients with pediatric low-grade gliomas. In addition, mirdametinib is being evaluated in a Phase 1b/2 trial in combination with BeiGene's RAF dimer inhibitor, lifirafenib, in patients with advanced or refractory solid tumors harboring RAS mutations, RAF mutations, and other MAPK pathway aberrations.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for patients living with severe rare diseases and cancer. SpringWorks has a differentiated targeted oncology portfolio of small molecule product candidates and is advancing 15 development programs, including two potentially registrational clinical trials in rare tumor types as well as several programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with innovators in industry and academia to expand its portfolio and create more solutions for patients with cancer. For more information, please visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success and timing of our product development activities, including the initiation and completion of SpringWorks' clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our collaboration partners' ongoing and planned clinical trials, (iv) our ability to obtain and maintain regulatory approval of any of our product candidates, (v) our plans to research, discover and develop additional product candidates, (vi) our ability to enter into collaborations for the development of new product candidates, (vii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (viii) our ability to meet any specific milestones set forth herein, and (ix) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" in Item 1A of Part I of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings.

    SpringWorks Media/Investor Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    203-561-1646

    kdiamond@springworkstx.com

    References

    1 Razavi P, Chang M, Xu G, et al. The Genomic Landscape of Endocrine-Resistant Advanced Breast Cancers. Cancer Cell. 2018;34(3):427-438.e6. doi:10.1016/j.ccell.2018.08.008.

    2 Pearson A, Proszek P, Pascual J, et al. Inactivating NF1 Mutations Are Enriched in Advanced Breast Cancer and Contribute to Endocrine Therapy Resistance. Clinical Cancer Research. 2019;26(3):608-622. doi:10.1158/1078-0432.ccr-18-4044.

    3 Hanrahan A, Sylvester B, Chang M, et al. Leveraging Systematic Functional Analysis to Benchmark an In Silico Framework Distinguishes Driver from Passenger MEK Mutants in Cancer. Cancer Research. 2020;80(19):4233-4243. doi:10.1158/0008-5472.can-20-0865.

    4 Gao Y, Chang M, McKay D, et al. Allele-Specific Mechanisms of Activation of MEK1 Mutants Determine Their Properties. Cancer Discovery. 2018;8(5):648-661. doi:10.1158/2159-8290.cd-17-1452.

    5 Weiss B, Wolters P, Plotkin S, et al. NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults With NF1-Related Plexiform Neurofibromas. Journal of Clinical Oncology. 2021;39(7):797-806. doi:10.1200/jco.20.02220.



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  6. STAMFORD, Conn., July 27, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today that James (Jim) Cassidy, M.D., Ph.D., has been appointed Chief Medical Officer. Dr. Cassidy brings over 30 years of experience in oncology as an academic physician-scientist and a drug development leader in both biotechnology and pharmaceutical companies, with experience spanning from early-stage research to translational and clinical development to post-marketing medical affairs strategy and lifecycle management. Dr. Cassidy succeeds Jens Renstrup, M.D., MBA, who will be leaving the company…

    STAMFORD, Conn., July 27, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today that James (Jim) Cassidy, M.D., Ph.D., has been appointed Chief Medical Officer. Dr. Cassidy brings over 30 years of experience in oncology as an academic physician-scientist and a drug development leader in both biotechnology and pharmaceutical companies, with experience spanning from early-stage research to translational and clinical development to post-marketing medical affairs strategy and lifecycle management. Dr. Cassidy succeeds Jens Renstrup, M.D., MBA, who will be leaving the company.

    "Jim brings significant oncology experience as a physician-scientist and industry leader to our efforts at SpringWorks, where we remain intensely focused on continuing to build a leading targeted oncology company with a diversified portfolio of differentiated programs," said Saqib Islam, Chief Executive Officer of SpringWorks. "I am delighted to welcome Jim to SpringWorks. I would also like to thank Jens for his contributions to SpringWorks and wish him well in his future endeavors."

    "Having had the pleasure of working closely with Jim several times throughout our careers, I am confident that the energy and expertise that he brings to developing drugs on behalf of cancer patients will prove exceptionally valuable to our efforts at SpringWorks," added Mike Burgess, M.B.Ch.B., Ph.D., Head of Research and Development at SpringWorks.

    Dr. Cassidy joins SpringWorks from Regeneron Pharmaceuticals, where he was Vice President of Oncology Strategic Program Direction. Prior to Regeneron, Dr. Cassidy was Corporate Vice President of Translational Development at Celgene, where he oversaw translational science efforts for the company's entire portfolio of programs addressing both hematological malignancies and solid tumors. Before that, he was Vice President of Oncology at Bristol-Myers Squibb, where he was responsible for all oncology assets from development candidate nomination through clinical proof-of-concept studies, including biomarkers and translational research, and was closely involved with late-stage development, commercial, and business development efforts as well. Prior to Bristol-Myers Squibb, Dr. Cassidy held several roles of increasing responsibility at Hoffmann La-Roche, including Global Head of Translational Research for Oncology and Acting Head of the Oncology Therapy Area. Before joining Roche, Dr. Cassidy had been a leading academic physician-scientist, most recently having served as Professor of Oncology, Head of the Department of Cancer Research and Head of the Division of Cancer Sciences and Molecular Pathology at the University of Glasgow in Scotland. Dr. Cassidy received his medical degree and doctorate from the University of Glasgow.

    "I am very pleased to be joining SpringWorks during this important time of growth and evolution for the company and am excited by the breadth of oncology opportunities being advanced on behalf of patients with solid tumors and hematologic malignancies," said Dr. Cassidy. "I look forward to working with this talented team to continue accelerating the development of our pipeline with the goal of bringing innovative new medicines to cancer patients."

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for patients living with severe rare diseases and cancer. SpringWorks has a differentiated targeted oncology portfolio of small molecule product candidates and is advancing two potentially registrational clinical trials in rare tumor types as well as eight programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with innovators in industry and academia to expand its portfolio and create more solutions for patients with cancer. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success and timing of our product development activities, including the initiation and completion of SpringWorks' clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our collaboration partners' ongoing and planned clinical trials, (iv) our ability to obtain and maintain regulatory approval of any of our product candidates, (v) our plans to research, discover and develop additional product candidates, (vi) our ability to enter into collaborations for the development of new product candidates, (vii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (viii) our ability to meet any specific milestones set forth herein, and (ix) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" in Item 1A of Part I of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings.

    Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com



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  7. STAMFORD, Conn., July 20, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX) announced today that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 11,066,358 (the ‘358 patent), directed to mirdametinib, the Company's product candidate in development for several oncology indications, including as a monotherapy for patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). The ‘358 patent, assigned to Warner-Lambert Company LLC (a subsidiary of Pfizer), expires in 2041. The ‘358 patent is a composition of matter patent that covers the polymorphic form of mirdametinib that is currently in clinical development. SpringWorks has exclusive rights to the ‘358 patent pursuant…

    STAMFORD, Conn., July 20, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX) announced today that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 11,066,358 (the ‘358 patent), directed to mirdametinib, the Company's product candidate in development for several oncology indications, including as a monotherapy for patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). The ‘358 patent, assigned to Warner-Lambert Company LLC (a subsidiary of Pfizer), expires in 2041. The ‘358 patent is a composition of matter patent that covers the polymorphic form of mirdametinib that is currently in clinical development. SpringWorks has exclusive rights to the ‘358 patent pursuant to an existing worldwide license with Pfizer.

    "We are very pleased that the USPTO has issued this patent, which extends patent protection for mirdametinib into 2041," said Saqib Islam, Chief Executive Officer of SpringWorks. "This new patent is part of our ongoing intellectual property strategy to expand protections across our portfolio of targeted oncology product candidates as we seek to provide new advances and better outcomes for patients with devastating cancers."

    About Mirdametinib

    Mirdametinib is an oral, potent, allosteric, brain-penetrant small molecule designed to inhibit MEK1 and MEK2, which are proteins that occupy pivotal positions in the MAPK pathway and that play a central role in multiple oncology indications. To date, over 250 subjects have been exposed to treatment with mirdametinib across clinical trials, with preliminary evidence of clinical activity against tumors driven by overactivated MAPK signaling.1

    Mirdametinib is being evaluated as a monotherapy in a Phase 2b trial for pediatric and adult patients with NF1-associated plexiform neurofibromas (NF1-PN), and in a Phase 1/2 trial for patients with pediatric low-grade gliomas. In addition, mirdametinib is being evaluated in a Phase 1b/2 trial in combination with BeiGene's RAF dimer inhibitor, lifirafenib, in patients with advanced or refractory solid tumors harboring RAS mutations, RAF mutations, and other MAPK pathway aberrations.

    About SpringWorks Therapeutics 

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for patients living with severe rare diseases and cancer. SpringWorks has a differentiated targeted oncology portfolio of small molecule product candidates and is advancing two potentially registrational clinical trials in rare tumor types as well as eight programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with innovators in industry and academia to expand its portfolio and create more solutions for patients with cancer. For more information, visit http://www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn

    SpringWorks Forward-Looking Statements 

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success and timing of our product development activities, including the initiation and completion of SpringWorks' clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our collaboration partners' ongoing and planned clinical trials, (iv) our ability to obtain and maintain regulatory approval of any of our product candidates, (v) our plans to research, discover and develop additional product candidates, (vi) our ability to enter into collaborations for the development of new product candidates, (vii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (viii) our ability to meet any specific milestones set forth herein, and (ix) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" in Item 1A of Part I of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings. 

    Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    203-561-1646

    kdiamond@springworkstx.com

    References:

    Weiss B, Wolters P, Plotkin S et al. NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults With NF1-Related Plexiform Neurofibromas. Journal of Clinical Oncology. 2021;39(7):797-806. doi:10.1200/jco.20.02220. 



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  8. Precision BioSciences, Inc. (NASDAQ:DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies with its ARCUS® genome editing platform, and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the first patient has been dosed in the combination arm of Precision's Phase 1/2a trial evaluating PBCAR269A. In the study, Precision's investigational allogeneic BCMA-targeted CAR T cell therapy will be combined with nirogacestat, SpringWorks' investigational gamma secretase inhibitor (GSI), in patients with relapsed/refractory (R/R) multiple myeloma…

    Precision BioSciences, Inc. (NASDAQ:DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies with its ARCUS® genome editing platform, and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the first patient has been dosed in the combination arm of Precision's Phase 1/2a trial evaluating PBCAR269A. In the study, Precision's investigational allogeneic BCMA-targeted CAR T cell therapy will be combined with nirogacestat, SpringWorks' investigational gamma secretase inhibitor (GSI), in patients with relapsed/refractory (R/R) multiple myeloma.

    "We are pleased to begin dosing patients in the combination arm of our ongoing Phase 1/2a study evaluating PBCAR269A, our first-generation allogeneic CAR T candidate targeting BCMA in patients with R/R multiple myeloma. BCMA is a well-established therapeutic target for multiple myeloma and this arm of the study pairs PBCAR269A with SpringWorks' nirogacestat, a gamma secretase inhibitor, a combination intended to offer strong mechanistic rationale for clinical benefit," said Alan List, M.D., Chief Medical Officer at Precision BioSciences. "As we look forward to sharing interim monotherapy data for PBCAR269A later this year, we are also conducting IND enabling studies to advance PBCAR269B, an immune-evading, stealth cell formulation into the clinic in 2022. We have high conviction in both our technology and BCMA as a target and we are pursuing a broad, data-driven strategy to inform our future development plans for this indication."

    "We are pleased to advance this combination into the clinic so we can evaluate if nirogacestat paired with PBCAR269A offers a safe and efficacious treatment option for patients with multiple myeloma," said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics. "We have made significant progress in developing nirogacestat as a cornerstone of BCMA combination therapy across modalities and look forward to generating clinical data with all of our partners."

    In September 2020, Precision and SpringWorks entered into a clinical collaboration in which Precision is sponsoring the expanded Phase 1/2a study of PBCAR269A to include nirogacestat and evaluate the safety and preliminary clinical activity of the combination therapy. Simultaneously, Precision continues to enroll patients in the highest dose cohort (Dose Level 3 at 6.0 × 106 cells/kg) in its monotherapy study with PBCAR269A.

    About PBCAR269A (Clinical Trials Study Identifier: NCT04171843)

    PBCAR269A is an allogeneic BCMA-targeted CAR T cell therapy candidate being evaluated for safety and preliminary clinical activity in a Phase 1/2a multicenter, nonrandomized, open-label, parallel assignment, single-dose, dose-escalation, and dose-expansion study of adults with relapsed/refractory (R/R) multiple myeloma. The starting dose of PBCAR269A is 6 x 105 CAR T cells/kg body weight with subsequent cohorts receiving escalating doses to a maximum dose of 6 x 106 CAR T cells/kg body weight.

    PBCAR269A is part of a pipeline of cell-phenotype optimized allogeneic CAR T therapies derived from healthy donors and then modified via a simultaneous TCR knock-out and CAR T knock-in step with the Company's proprietary ARCUS® genome editing technology. Precision BioSciences optimizes its CAR T therapy candidates for immune cell expansion in the body by maintaining a high proportion of naïve and central memory CAR T cells.

    The U.S. Food and Drug Administration (FDA) granted Fast Track Designation to PBCAR269A for the treatment of R/R multiple myeloma for which the FDA previously granted Orphan Drug Designation. The PBCAR269A clinical trial is being conducted at multiple U.S. sites.

    About Nirogacestat

    Nirogacestat is an investigational, oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has six collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities, including with an antibody-drug conjugate, two CAR T cell therapies and two bispecific antibodies. In addition, SpringWorks and Fred Hutchinson Cancer Research Center have entered into a sponsored research agreement to further characterize the ability of nirogacestat to modulate BCMA and potentiate BCMA directed therapies using a variety of preclinical and patient-derived multiple myeloma models developed by researchers at Fred Hutch.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors and from the European Commission for the treatment of soft tissue sarcoma. The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.

    About Precision BioSciences, Inc.

    Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its wholly proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Company's pipeline consists of multiple "off-the-shelf" CAR T immunotherapy clinical candidates and several in vivo gene correction therapy candidates designed to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for patients living with severe rare diseases and cancer. SpringWorks has a differentiated targeted oncology portfolio of small molecule product candidates and is advancing two potentially registrational clinical trials in rare tumor types as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with innovators in industry and academia to expand its portfolio to create more solutions for patients with cancer. For more information, please visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Precision BioSciences Forward Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the expected timing of clinical updates and interim data reports related to PBCAR269A in combination with nirogacestat, statements regarding our clinical development and research pipeline and the potential clinical benefit of our product candidates. In some cases, you can identify forward-looking statements by terms such as "aim," "anticipate," "believe," "could," "expect," "should," "plan," "intend," "estimate," "target," "mission," "goal," "may," "will," "would," "should," "could," "target," "potential," "project," "predict," "contemplate," "potential," or the negative thereof and similar words and expressions.

    Forward-looking statements are based on management's current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical or greenhouse studies and clinical or field trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, biotechnology and agricultural biotechnology fields; our or our collaborators' ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators' development of product candidates; our or our collaborators' ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; our or our collaborators' ability to advance product candidates into, and successfully design, implement and complete, clinical or field trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; our ability to obtain an adequate supply of T cells from qualified donors; our ability to achieve our anticipated operating efficiencies at our manufacturing facility; delays or difficulties in our and our collaborators' ability to enroll patients; changes in interim "top-line" and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; the rate and degree of market acceptance of any of our product candidates; the success of our existing collaboration agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate key executives and personnel; market and economic conditions; effects of system failures and security breaches; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events effects of the outbreak of COVID-19, or any pandemic, epidemic or outbreak of an infectious disease; insurance expenses and exposure to uninsured liabilities; effects of tax rules; risks related to ownership of our common stock and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2021, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC's website at www.sec.gov and the Investors & Media page of our website at investor.precisionbiosciences.com. All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

    SpringWorks Therapeutics Forward Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success and timing of our product development activities, including the initiation and completion of SpringWorks' clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our collaboration partners' ongoing and planned clinical trials, (iv) our ability to obtain and maintain regulatory approval of any of our product candidates, (v) our plans to research, discover and develop additional product candidates, (vi) our ability to enter into collaborations for the development of new product candidates, (vii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (viii) our ability to meet any specific milestones set forth herein, and (ix) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" in Item 1A of Part I of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings.

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  9. STAMFORD, Conn., June 16, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported the initiation of a Phase 1/2 clinical trial to evaluate mirdametinib, an investigational MEK inhibitor, for the treatment of children, adolescents, and young adults with low-grade glioma (LGG). The study is sponsored by St. Jude Children's Research Hospital. More information on the trial can be found on www.clinicaltrials.gov under the identifier NCT04923126.

    Pediatric LGG can harbor genetic alterations that upregulate the MAPK pathway and promote tumor growth. Prior studies of therapeutic…

    STAMFORD, Conn., June 16, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported the initiation of a Phase 1/2 clinical trial to evaluate mirdametinib, an investigational MEK inhibitor, for the treatment of children, adolescents, and young adults with low-grade glioma (LGG). The study is sponsored by St. Jude Children's Research Hospital. More information on the trial can be found on www.clinicaltrials.gov under the identifier NCT04923126.

    Pediatric LGG can harbor genetic alterations that upregulate the MAPK pathway and promote tumor growth. Prior studies of therapeutic agents that target the MAPK pathway have demonstrated promising results in patients with pediatric LGG.1 Mirdametinib is an oral, allosteric, brain-penetrant small molecule designed to inhibit MEK1 and MEK2, which are proteins that occupy pivotal positions in the MAPK pathway. To date, over 250 subjects have been exposed to treatment with mirdametinib across clinical trials, with preliminary evidence of clinical activity against tumors driven by over-activated MAPK signaling.2

    "Children with LGG who do not achieve a cure following surgery can face years of increasingly aggressive chemotherapy, which can have lasting negative effects on learning, cognition and quality of life," said L. Mary Smith, Ph.D., Chief Development Officer of SpringWorks. "Recently, it has been recognized that most cases of pediatric LGG have genetic alterations that upregulate the MAPK pathway. Given its brain-penetrant properties, we look forward to studying mirdametinib to evaluate whether our MEK inhibitor can provide meaningful antitumor activity and clinical benefit in patients with LGG."

    This trial is being conducted pursuant to a research agreement that SpringWorks entered into with St. Jude Children's Research Hospital whereby St. Jude sponsors the trial and SpringWorks provides partial funding, study drug and other non-financial support.

    About the Phase 1/2 LGG Trial

    The open-label, multi-center Phase 1/2 trial will evaluate the safety, tolerability and pharmacokinetics of mirdametinib in children, adolescents and young adults with LGG. The study plans to enroll up to 130 patients between the ages of 2 years and 24 years of age. Patients will receive mirdametinib twice daily on a continuous schedule for up to two years.

    The primary objective of the Phase 1 portion of the trial will be to evaluate the safety, tolerability, and pharmacokinetics of mirdametinib. The Phase 2 portion of the trial is designed to measure the objective response rate and duration of response to mirdametinib. Patients in the Phase 2 portion will be stratified into one of three treatment cohorts based on tumor status and history of MEK inhibitor exposure: Cohort 1: Patients with newly diagnosed LGG; Cohort 2: Patients with progressive or recurrent LGG without previous exposure to a MEK inhibitor; Cohort 3: Patients with progressive or recurrent LGG with previous exposure to a MEK inhibitor.

    About Low-Grade Glioma

    Pediatric LGG is a type of tumor that affects the brain and spinal cord. LGG is the most common central nervous system (CNS) tumor in children, accounting for approximately 30% of all childhood CNS tumors.3 LGG can arise in several different areas of the CNS including the cerebellum, cerebrum, brainstem, hypothalamus, visual pathway, optic nerve, and spinal cord.3 Low-grade gliomas are associated with significant morbidity including seizures and cognitive decline, and recurrent gliomas often undergo malignant transformation.4

    Optimal management of LGG remains uncertain.3 While most children with LGG survive their cancer, children with progressive residual disease following surgery and those not amenable to surgery can oftentimes face years of increasingly aggressive cytotoxic therapies that can have lasting effects on learning, cognition, and quality of life.3 There are no FDA approved therapies for pediatric LGG and current treatments being used off-label can be associated with significant acute and life-long adverse effects.3 As a result, the unmet medical need for these patients remains high.

    About Mirdametinib

    Mirdametinib is an investigational, oral, potent, allosteric small molecule MEK1/2 inhibitor with clinical validation and over 250 subjects exposed to date. It is designed to inhibit MEK1 and MEK2. MEK proteins occupy a pivotal position in the MAPK pathway, a key signaling network that regulates cell growth and survival, and that plays a central role in multiple oncology and rare disease indications. Mirdametinib is being evaluated in a Phase 2b trial for pediatric and adult patients with NF1-associated plexiform neurofibromas (NF1-PN), which are tumors that grow in an infiltrative pattern along the peripheral nerve sheath.

    In addition to the monotherapy trials in NF1-PN and LGG, and given the critical role that the MAPK pathway plays in the growth and proliferation of a large number of tumor types, SpringWorks is also advancing mirdametinib in combination with other rational anti-cancer agents across a range of solid tumors.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for patients living with severe rare diseases and cancer. SpringWorks has a differentiated targeted oncology portfolio of small molecule product candidates and is advancing two potentially registrational clinical trials in rare tumor types as well as eight programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with innovators in industry and academia to expand its portfolio and create more solutions for patients with cancer. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success and timing of our product development activities, including the initiation and completion of SpringWorks' clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our collaboration partners' ongoing and planned clinical trials, (iv) our ability to obtain and maintain regulatory approval of any of our product candidates, (v) our plans to research, discover and develop additional product candidates, (vi) our ability to enter into collaborations for the development of new product candidates, (vii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (viii) our ability to meet any specific milestones set forth herein, and (ix) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" in Item 1A of Part I of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings.

    SpringWorks Media/Investor Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    203-561-1646

    kdiamond@springworkstx.com

    References 

    1 de Blank P, Bandopadhayay P, Haas-Kogan D, Fouladi M, Fangusaro J. Management of pediatric low-grade glioma. Curr Opin Pediatr. 2019;31(1):21-27. doi:10.1097/MOP.0000000000000717.

    2 Weiss BD, Wolters PL, Plotkin SR, et al. NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults With NF1-Related Plexiform Neurofibromas. Journal of Clinical Oncology. 2021;JCO.20.02220. doi.org/10. 1200/JCO.20.02220.

    3 Ryall S, Tabori U, Hawkins C. Pediatric low-grade glioma in the era of molecular diagnostics. Acta Neuropathol Commun. 2020;8(1):30. doi.org/10.1186/s40478-020-00902-z.

    4 Schiff D. Low-grade gliomas. Continuum (Minneap Minn). 2017;23(6):1564-1579. doi:10.1212/con.0000000000000537.



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  10. STAMFORD, Conn., June 15, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that an update on the previously reported interim data from the first 20 adult patients enrolled in the ongoing Phase 2b ReNeu trial evaluating mirdametinib, an investigational MEK inhibitor being studied in adult and pediatric patients with NF1-associated plexiform neurofibromas (NF1-PN), was presented at the 2021 Children's Tumor Foundation NF Conference being held June 14-16, 2021. The data were presented by Christopher Moertel, M.D., Professor and Medical Director of the Pediatric Neuro-Oncology…

    STAMFORD, Conn., June 15, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that an update on the previously reported interim data from the first 20 adult patients enrolled in the ongoing Phase 2b ReNeu trial evaluating mirdametinib, an investigational MEK inhibitor being studied in adult and pediatric patients with NF1-associated plexiform neurofibromas (NF1-PN), was presented at the 2021 Children's Tumor Foundation NF Conference being held June 14-16, 2021. The data were presented by Christopher Moertel, M.D., Professor and Medical Director of the Pediatric Neuro-Oncology and Neurofibromatosis Programs at the University of Minnesota Medical School and Principal Investigator of the ReNeu trial.

    In February 2021, SpringWorks reported results from the first 20 adult patients enrolled utilizing a January 22, 2021 data cutoff date and showed that 50% of these patients had achieved an objective response as assessed by blinded independent central review (BICR), that 80% remained on study, and that the median time on treatment for these patients was 10.1 cycles (approximately 10 months). The NF Conference presentation utilized a March 23, 2021 data cutoff date and showed durable efficacy in these same 20 patients, with the median time on treatment now having reached 13 cycles (approximately 12 months), the objective response rate remaining at 50%, and 80% of these patients still remain on study. Among these patients, there had been no further dose reductions as of the later data cutoff date and mirdametinib continued to be generally well tolerated, with the majority of treatment-related adverse events (TRAE) being Grade 1 or 2, with only one Grade 3 TRAE and no Grade 4 or 5 adverse events (AE) reported in these 20 patients.

    "Plexiform neurofibromas are tumors that can cause serious disfigurement and significant pain," said Dr. Moertel. "These interim data from the ReNeu trial are promising for patients who are in need of new treatment options and the availability of a pediatric formulation of mirdametinib, along with a dosing schedule that does not require fasting, may also be meaningful for patients, many of whom are children and young adults that require long-term treatment in order to control tumor growth and its associated morbidities."

    "We remain encouraged by these interim data, which reaffirm our belief that mirdametinib has the potential to be a best-in-class treatment for patients with NF1-PN," said Saqib Islam, Chief Executive Officer of SpringWorks. "The ReNeu trial is on track to complete enrollment in the second half of 2021 and we look forward to sharing additional updates as the trial progresses."

    Interim Phase 2b Data from ReNeu Trial:

    The interim Phase 2b ReNeu data set presented at the Children's Tumor Foundation NF Conference included the first 20 adult patients enrolled and utilized a March 23, 2021 data cutoff date. Objective responses were defined as a ≥ 20% reduction in target tumor volume measured by MRI and were assessed by BICR. Patients received mirdametinib at a dose of 2 mg/m2 twice daily (maximum dose: 4mg twice daily) without regard to food on a 3 weeks-on, 1 week-off intermittent schedule, with patients being allowed to stay on therapy for up to 24 cycles (approximately two years). The median time on treatment for the 20 adult patients evaluated for this analysis was 13 cycles (approximately 12 months), with an initial efficacy assessment performed following cycle five and then every four cycles thereafter.

    The preliminary efficacy and safety analysis presented showed:

    • 10/20 (50%) of patients had achieved an objective response by BICR, seven of which have been confirmed at a subsequent scan.
    • Responders had a median tumor volume reduction of 45%.
    • 16/20 (80%) of these patients remained on study as of the data cutoff and only one patient required a dose reduction due to an AE. Reasons for discontinuation included one each of progressive disease, participant decision, AE (Grade 1 diarrhea), and a patient being unable to undergo required MRI imaging due to a titanium rod implant from non-treatment-related worsening of scoliosis.
    • A generally well-tolerated safety profile. The majority of TRAEs were Grade 1 or 2; there was only one Grade 3 TRAE reported and no Grade 4 or 5 AEs reported in these 20 patients. The most common TRAEs were rash, nausea and diarrhea.

    About the ReNeu Trial

    The ReNeu trial is a multi-center, open-label Phase 2b trial evaluating the efficacy, safety, and tolerability of mirdametinib in patients two years of age and older with an inoperable NF1-associated PN causing significant morbidity. The study will enroll approximately 100 patients in the United States. Patients receive mirdametinib at a dose of 2 mg/m2 twice daily (maximum dose of 4 mg twice daily, calculated based on body surface area) without regard to food. Mirdametinib is administered in a 3-week on, 1-week off dosing schedule.

    The primary endpoint is objective response rate, defined as ≥ 20% reduction in target tumor volume as measured by MRI and assessed by BICR. Secondary endpoints include safety and tolerability measures, duration of response, and changes from baseline in patient reported outcomes.

    More information about the ReNeu trial is available at www.clinicaltrials.gov under the identifier NCT03962543.

    About NF1-PN

    Neurofibromatosis type 1 (NF1) is a rare genetic disorder that arises from mutations in the NF1 gene, which encodes for neurofibromin, a key suppressor of the MAPK pathway.1,2 NF1 is the most common form of neurofibromatosis, with an estimated global birth incidence of approximately 1 in 3,000 individuals, and approximately 100,000 patients living with NF1 in the United States.3,4 The clinical course of NF1 is heterogeneous and manifests in a variety of symptoms across numerous organ systems, including abnormal pigmentation, skeletal deformities, tumor growth and neurological complications, such as cognitive impairment.5 Patients with NF1 have an eight to 15-year mean reduction in their life expectancy compared to the general population.2

    NF1 patients have approximately a 30-50% lifetime risk of developing plexiform neurofibromas, or PN, which are tumors that grow in an infiltrative pattern along the peripheral nerve sheath and that can cause severe disfigurement, pain and functional impairment; in rare cases, NF1-PN may be fatal.3,4,6 Patients with NF1 can also experience additional manifestations, including neurocognitive deficits and developmental delays.4 NF1-PNs are most often diagnosed in the first two decades of life.3 These tumors can be aggressive and are associated with clinically significant morbidities; typically, they grow more rapidly during childhood.7,8

    Surgical removal of these tumors is challenging due to the infiltrative tumor growth pattern along nerves and can lead to permanent nerve damage and disfigurement.9 MEK inhibitors have emerged as a validated class of treatment for NF1-PN.4

    About Mirdametinib

    Mirdametinib is an investigational, oral, potent, allosteric small molecule MEK1/2 inhibitor designed to inhibit MEK1 and MEK2. MEK proteins occupy a pivotal position in the MAPK pathway, a key signaling network that regulates cell growth and survival, and that plays a central role in multiple oncology and rare disease indications.

    Mirdametinib has been evaluated in several Phase 1 and Phase 2 clinical trials, with over 250 subjects having been exposed to treatment. A Phase 2 trial was conducted by the Neurofibromatosis Clinical Trial Consortium and evaluated mirdametinib in 19 adolescent and adult patients with inoperable and symptomatic or growing plexiform neurofibromas. Patients received an oral dose of 2 mg/m2 BID with a maximum dose of 4 mg BID on a four-week cycle of three weeks-on, one week-off. Eight patients (42%) achieved an objective response by cycle 12, prospectively defined as volumetric reduction in their target PN of at least 20%. Mirdametinib was generally well-tolerated in this trial. The most commonly reported treatment-emergent Grade 2 or higher AEs were acneiform rash, fatigue and nausea.

    In addition, and given the critical role that the MAPK pathway plays in the growth and proliferation of a large number of tumor types, SpringWorks is also pursuing mirdametinib in combination with other rational anti-cancer agents across a range of solid tumors.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for patients living with severe rare diseases and cancer. SpringWorks has a differentiated targeted oncology portfolio of small molecule product candidates and is advancing two potentially registrational clinical trials in rare tumor types as well as eight programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with innovators in industry and academia to expand its portfolio and create more solutions for patients with cancer. For more information, please visit www.springworkstx.com, and follow @SpringWorksTx on Twitter and LinkedIn.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success and timing of our product development activities, including the initiation and completion of SpringWorks' clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our collaboration partners' ongoing and planned clinical trials, (iv) our ability to obtain and maintain regulatory approval of any of our product candidates, (v) our plans to research, discover and develop additional product candidates, (vi) our ability to enter into collaborations for the development of new product candidates, (vii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (viii) our ability to meet any specific milestones set forth herein, and (ix) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" in Item 1A of Part I of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings.

    Contact

    SpringWorks Media/Investor Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    203-561-1646

    kdiamond@springworkstx.com

    References

    1. Yap YS, McPherson JR, Ong CK, et al. The NF1 gene revisited - from bench to bedside. Oncotarget. 2014;5(15):5873-5892. doi:10.18632/oncotarget.2194.
    2. Rasmussen S, Friedman J. NF1 Gene and Neurofibromatosis 1. Am J Epidemiol. 2000;151(1):33-40. doi:10.1093/oxfordjournals.aje.a010118.
    3. Prada C, Rangwala F, Martin L, et al. Pediatric Plexiform Neurofibromas: Impact on Morbidity and Mortality in Neurofibromatosis Type 1. J Pediatr. 2012;160(3):461-467. doi:10.1016/j.jpeds.2011.08.051.
    4. Ferner R. Neurofibromatosis 1 and neurofibromatosis 2: a twenty first century perspective. The Lancet Neurology. 2007;6(4):340-351. doi:10.1016/s1474-4422(07)70075-3.
    5. Weiss BD, Wolters PL, Plotkin SR, et al. NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults With NF1-Related Plexiform Neurofibromas. Journal of Clinical Oncology. 2021;JCO.20.02220.doi.org/10. 1200/JCO.20.02220.
    6. Hirbe A, Gutmann D. Neurofibromatosis type 1: a multidisciplinary approach to care. The Lancet Neurology. 2014;13(8):834-843. doi:10.1016/s1474-4422(14)70063-8.
    7. Gross A, Singh G, Akshintala S, et al. Association of plexiform neurofibroma volume changes and development of clinical morbidities in neurofibromatosis 1. Neuro Oncol. 2018;20(12):1643-1651. doi:10.1093/neuonc/noy067.
    8. Nguyen R, Dombi E, Widemann B, et al. Growth dynamics of plexiform neurofibromas: a retrospective cohort study of 201 patients with neurofibromatosis 1. Orphanet J Rare Dis. 2012;7(1):75. doi:10.1186/1750-1172-7-75.
    9. Needle M, Cnaan A, Dattilo J, et al. Prognostic signs in the surgical management of plexiform neurofibroma: The Children's Hospital of Philadelphia experience, 1974-1994. J Pediatr. 1997;131(5):678-682. doi:10.1016/s0022-3476(97)70092-1.


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  11. STAMFORD, Conn., June 07, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the company has entered into a clinical trial collaboration agreement with Seagen Inc. to evaluate nirogacestat, SpringWorks' investigational gamma secretase inhibitor, in combination with SEA-BCMA, Seagen's investigational monoclonal antibody targeting B-cell maturation agent (BCMA), in patients with relapsed or refractory multiple myeloma.

    Gamma secretase inhibition prevents the cleavage and shedding of BCMA from the surface of myeloma cells. In preclinical models, nirogacestat has been…

    STAMFORD, Conn., June 07, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the company has entered into a clinical trial collaboration agreement with Seagen Inc. to evaluate nirogacestat, SpringWorks' investigational gamma secretase inhibitor, in combination with SEA-BCMA, Seagen's investigational monoclonal antibody targeting B-cell maturation agent (BCMA), in patients with relapsed or refractory multiple myeloma.

    Gamma secretase inhibition prevents the cleavage and shedding of BCMA from the surface of myeloma cells. In preclinical models, nirogacestat has been shown to increase levels of membrane-bound BCMA and reduce levels of soluble BCMA, thereby enhancing the activity of BCMA-targeted therapies.

    "We continue to make significant progress advancing nirogacestat as a potential best-in-class cornerstone of BCMA combination therapy for patients with multiple myeloma and are delighted to work with Seagen to study nirogacestat in combination with SEA-BCMA, our first collaboration with a monoclonal antibody targeting BCMA," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our goal is to meaningfully improve clinical outcomes for patients with multiple myeloma and we look forward to generating clinical data using nirogacestat in combination with BCMA-directed therapies across modalities."

    Under the terms of the agreement, Seagen will sponsor and conduct the Phase 1 study to evaluate the safety, tolerability and preliminary efficacy of the combination, and will assume all costs associated with the study, other than expenses related to the manufacturing of nirogacestat and certain expenses related to intellectual property rights. Seagen and SpringWorks will also form a joint development committee to manage the clinical study, which is expected to commence in the second half of 2021.

    In addition to its ongoing clinical collaborations with BCMA-directed therapies, SpringWorks is conducting a global Phase 3, double-blind, randomized, placebo-controlled clinical trial (the DeFi Trial) to evaluate nirogacestat as a monotherapy in adults with progressing desmoid tumors.

    About Nirogacestat

    Nirogacestat is an investigational, oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has six collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities, including with an antibody-drug conjugate, two CAR T cell therapies, two bispecific antibodies and a monoclonal antibody. In addition, SpringWorks and Fred Hutchinson Cancer Research Center have entered into a sponsored research agreement to further characterize the ability of nirogacestat to modulate BCMA and potentiate BCMA directed therapies using a variety of preclinical and patient-derived multiple myeloma models developed by researchers at Fred Hutch.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors and from the European Commission for the treatment of soft tissue sarcoma. The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for patients living with severe rare diseases and cancer. SpringWorks has a differentiated targeted oncology portfolio of small molecule product candidates and is advancing two potentially registrational clinical trials in rare tumor types as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with innovators in industry and academia to expand its portfolio to create more solutions for patients with cancer. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    SpringWorks Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success and timing of our product development activities, including the initiation and completion of SpringWorks' clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our collaboration partners' ongoing and planned clinical trials, (iv) our ability to obtain and maintain regulatory approval of any of our product candidates, (v) our plans to research, discover and develop additional product candidates, (vi) our ability to enter into collaborations for the development of new product candidates, (vii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (viii) our ability to meet any specific milestones set forth herein, and (ix) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" in Item 1A of Part I of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings.

    SpringWorks Media/Investor Contact:

    Kim Diamond

    203-561-1646

    kdiamond@springworkstx.com

     



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  12. STAMFORD, Conn., June 01, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that management will participate in a Fireside Chat at the Goldman Sachs 42nd Annual Global Healthcare Conference on Tuesday, June 8th at 3:00PM ET.

    To access the live webcast please visit the Events & Presentations page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the conference.

    About SpringWorks Therapeutics
    SpringWorks is a clinical-stage biopharmaceutical…

    STAMFORD, Conn., June 01, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that management will participate in a Fireside Chat at the Goldman Sachs 42nd Annual Global Healthcare Conference on Tuesday, June 8th at 3:00PM ET.

    To access the live webcast please visit the Events & Presentations page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the conference.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for patients living with severe rare diseases and cancer. SpringWorks has a differentiated targeted oncology portfolio of small molecule product candidates and is advancing two potentially registrational clinical trials in rare tumor types as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with innovators in industry and academia to expand its portfolio to create more solutions for patients with cancer. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn

    Contact:

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com



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  13. STAMFORD, Conn., May 14, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that management will participate in a Fireside Chat at the Cowen 2nd Annual Oncology Innovation Summit on Friday, May 21st at 10:00AM ET.

    To access the live webcast please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the summit.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical…

    STAMFORD, Conn., May 14, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that management will participate in a Fireside Chat at the Cowen 2nd Annual Oncology Innovation Summit on Friday, May 21st at 10:00AM ET.

    To access the live webcast please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the summit.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn

    Contact:

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com



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  14. STAMFORD, Conn., May 10, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today that Mike Burgess, M.B.Ch.B., Ph.D. has been appointed Head of Research and Development (R&D). Dr. Burgess is a physician-scientist with clinical experience in pediatrics and pediatric oncology and over 20 years of pharmaceutical industry R&D leadership experience across several therapeutic areas, including oncology. Dr. Burgess will lead the R&D organization as the company continues to advance its diversified targeted oncology pipeline and to expand its portfolio with additional oncology programs…

    STAMFORD, Conn., May 10, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today that Mike Burgess, M.B.Ch.B., Ph.D. has been appointed Head of Research and Development (R&D). Dr. Burgess is a physician-scientist with clinical experience in pediatrics and pediatric oncology and over 20 years of pharmaceutical industry R&D leadership experience across several therapeutic areas, including oncology. Dr. Burgess will lead the R&D organization as the company continues to advance its diversified targeted oncology pipeline and to expand its portfolio with additional oncology programs. He succeeds Stephen Squinto, Ph.D., who served as Acting Head of R&D since the company's launch in 2017. Dr. Squinto will continue to serve on the SpringWorks Board of Directors.

    "At SpringWorks, we are driven to execute on our ambitious plans to provide oncology patients with transformative new therapies, and our portfolio of programs is continuing to mature and expand in service of that goal," said Saqib Islam, Chief Executive Officer. "Mike is a strong R&D leader with deep experience in oncology and a track record of excellence across all stages of preclinical and clinical development, culminating in multiple approvals of new cancer medicines under his leadership. I am delighted to welcome Mike to SpringWorks and look forward to the scientific, clinical and leadership excellence he will bring to our already strong team as we continue to build a world-class oncology company on behalf of a broad range of cancer patients."

    "I am excited to be joining SpringWorks at such a seminal time as the company advances a diversified pipeline that is maturing across rare oncology indications, multiple myeloma and metastatic solid tumors," said Dr. Burgess. "There is a significant opportunity ahead of us to accelerate and expand these efforts, including through the buildout of additional preclinical and clinical R&D capabilities within the organization. I look forward to working with the team to further our mission of developing life-changing medicines for people with cancer."

    Dr. Burgess brings to SpringWorks over 20 years of experience building and leading R&D teams in oncology. He joins SpringWorks from Turnstone Biologics, where he was President of R&D and led the buildout of a leading cancer immunotherapy R&D organization. Prior to Turnstone, Dr. Burgess led the strategy and execution of translational medicine, early-stage clinical trials and clinical pharmacology across all therapeutic areas, including oncology, at Bristol-Myers Squibb. Before that, he held several different senior leadership positions at Hoffmann La-Roche, including Acting Global Head of Roche Pharma Research and Early Development (pRED) and Senior Vice President and Global Head of Oncology Research and Early Development. Prior to Roche, Dr. Burgess was with Eli Lilly and Company, where he worked on early-stage oncology trials. Dr. Burgess received his medical degree (M.B.Ch.B.) and Ph.D. in molecular biology from the University of Bristol, UK and spent 10 years as a practicing physician in pediatrics and pediatric oncology.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success and timing of our product development activities, including the initiation and completion of SpringWorks' clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our collaboration partners' ongoing and planned clinical trials, (iv) our ability to obtain and maintain regulatory approval of any of our product candidates, (v) our plans to research, discover and develop additional product candidates, (vi) our ability to enter into collaborations for the development of new product candidates, (vii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (viii) our ability to meet any specific milestones set forth herein, and (ix) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" in Item 1A of Part I of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings.

    Contact

    Kim Diamond

    Vice President Communications and Investor Relations

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com



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  15. – Reported Interim Data from Phase 2b ReNeu Trial of Mirdametinib in NF1-PN Demonstrating Encouraging Clinical Activity and Tolerability in the First 20 Adult Patients Enrolled –

    – Announced Achievement of First Patient Dosed in Allogene and Janssen Phase 1 Trials Evaluating Nirogacestat in Combination with BCMA Therapies –

    – Expanded Targeted Oncology Pipeline with Exclusive Worldwide License to TEAD Inhibitor Portfolio –

    STAMFORD, Conn., May 06, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported first quarter financial results for the period ended March 31, 2021…

    – Reported Interim Data from Phase 2b ReNeu Trial of Mirdametinib in NF1-PN Demonstrating Encouraging Clinical Activity and Tolerability in the First 20 Adult Patients Enrolled –

    – Announced Achievement of First Patient Dosed in Allogene and Janssen Phase 1 Trials Evaluating Nirogacestat in Combination with BCMA Therapies –

    – Expanded Targeted Oncology Pipeline with Exclusive Worldwide License to TEAD Inhibitor Portfolio –

    STAMFORD, Conn., May 06, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported first quarter financial results for the period ended March 31, 2021 and provided an update on recent company developments.

    "In the first quarter of 2021 we continued to execute on our 10 clinical programs and grew our portfolio of opportunities aimed at serving a broad group of patients across our three distinct oncology segments: late-stage rare oncology, hematological cancers and biomarker-defined metastatic solid tumors," said Saqib Islam, Chief Executive Officer of SpringWorks. "We are poised for multiple important data readouts this year, including topline data from our Phase 3 DeFi trial, initial clinical data from our BCMA combination trial of nirogacestat with GSK's BLENREP in multiple myeloma and initial clinical data from our metastatic solid tumor trials being conducted in collaboration with BeiGene. I look forward to reporting on our continued progress throughout 2021."

    Recent Business Highlights and Upcoming Milestones

    Late-Stage Rare Oncology

    • In February 2021, SpringWorks reported interim data from the adult stratum of the ongoing potentially registrational Phase 2b ReNeu trial evaluating mirdametinib in pediatric and adult patients with NF1-associated plexiform neurofibromas. Of the first 20 adult patients enrolled, 50% had achieved an objective response, the primary endpoint of the study, as assessed by blinded independent central review, and 16 of these 20 patients (80%) remained on study as of the January 22, 2021 data cutoff. In addition, mirdametinib was generally well tolerated, with the majority of treatment-related adverse events (TRAE) being Grade 1 or 2 and only one Grade 3 TRAE reported; there have been no Grade 4 or 5 TRAEs reported. The Company expects to report additional clinical data from the ReNeu trial at a medical conference this year and to complete enrollment of the trial in the second half of 2021.
    • SpringWorks expects to report topline data from the Phase 3 DeFi trial in the second half of 2021, as previously disclosed.
    • Recruitment is ongoing in a Phase 2 study sponsored by the Children's Oncology Group evaluating nirogacestat in pediatric patients with desmoid tumors.

    B-cell Maturation Antigen (BCMA) Combinations in Multiple Myeloma

    • Enrollment is ongoing in three Phase 1 studies evaluating nirogacestat in combination with anti-B-cell maturation antigen (BCMA) therapies in adult patients with relapsed or refractory multiple myeloma: a Phase 1b trial sponsored by GSK evaluating nirogacestat in combination with BLENREP (belantamab mafodotin-blmf), a Phase 1 study sponsored by Allogene evaluating nirogacestat in combination with ALLO-715, and a Phase 1 study sponsored by Janssen Research & Development, LLC (Janssen) evaluating nirogacestat in combination with teclistamab. Initial data from the GSK-sponsored study are expected in 2021. SpringWorks also expects that two additional collaborator-sponsored trials will initiate in the first half of 2021, as previously disclosed: nirogacestat + Pfizer's elranatamab and nirogacestat + Precision Biosciences' PBCAR269A.
    • In March 2021, SpringWorks entered into a services agreement with ONCOtracker, Inc., a leading innovation center for novel cancer treatment and testing with a focus on the diagnosis and treatment of multiple myeloma, other B-cell malignancies, and related disorders, to explore the effect of nirogacestat on soluble BCMA release and membrane-bound BCMA receptor density in primary bone marrow samples collected from multiple myeloma patients. This research is intended to further confirm nirogacestat's ability to potentiate BCMA by using ex vivo patient-derived models with associated longitudinal clinical characterization for each sample.

    Biomarker-Defined Metastatic Solid Tumors

    • In May 2021, SpringWorks entered into an exclusive worldwide license agreement with Katholieke Universiteit Leuven (KU Leuven) and the Flanders Institute for Biotechnology (VIB) for the in-license of a portfolio of novel small molecule inhibitors of the TEA Domain (TEAD) family of transcription factors, designed for the potential treatment of biomarker-defined solid tumors driven by aberrant Hippo pathway signaling. The licensed portfolio includes advanced lead compounds and multiple backup compounds from diverse chemical series, which were discovered at KU Leuven's Center for Drug Design and Discovery (CD3) in collaboration with Professor Georg Halder of the VIB-KU Leuven Center for Cancer Biology. SpringWorks expects to nominate a development candidate from this portfolio and move into IND-enabling studies in 2022.
    • Enrollment is ongoing in a Phase 1b/2 trial evaluating mirdametinib with BeiGene's RAF dimer inhibitor, lifirafenib, in adult patients with RAS/RAF mutant and other MAPK pathway aberrant solid tumors. BeiGene is sponsoring this trial and SpringWorks and BeiGene expect to report initial clinical data in 2021, as previously disclosed.
    • Enrollment is ongoing in a Phase 1 trial of BGB-3245 in adult patients with RAF mutant solid tumors. BGB-3245 is a selective RAF dimer inhibitor being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene. Initial clinical data from the MapKure-sponsored Phase 1 trial are expected in 2021, as previously disclosed.
    • In March 2021, SpringWorks entered into a research collaboration agreement with Inserm Transfert, the private subsidiary of Inserm, acting as a delegatee of the French National Institute of Health and Medical Research (Inserm) to explore the ability of nirogacestat to delay the development of resistance to the EGFR inhibitor osimertinib in preclinical models of non-small cell lung cancer (NSCLC) driven by the EGFR C797S gatekeeper mutation. This research, which will be led by Antonio Maraver, Ph.D. at Montpellier Cancer Research Institute (IRCM U1194), is intended to build on previous in vivo work from Dr. Maraver's lab demonstrating the potential for gamma secretase inhibition to enhance the activity of osimertinib in EGFR-driven models of NSCLC.1

    General Corporate

    • In March 2021, Bhavesh Ashar was appointed Chief Commercial Officer of SpringWorks. Mr. Ashar has more than 20 years of global pharmaceutical and biotechnology experience, most recently having served as Senior Vice President, General Manager of U.S. Oncology at Bayer Healthcare where he was responsible for a broad portfolio in prostate, liver, colorectal, GIST, hematologic and tumor-agnostic biomarker driven cancers.

    First Quarter 2021 Financial Results

    • Research and Development (R&D) Expenses: R&D expenses were $17.4 million for the first quarter, compared to $9.7 million for the comparable period of 2020. The increases in R&D expenses were primarily attributable to an increase in external costs related to drug manufacturing and trial costs, and an increase in internal costs driven by the growth in employee costs associated with increases in the number of R&D personnel and an increase in stock-based compensation expense.
    • General and Administrative (G&A) Expenses: G&A expenses were $12.4 million for the first quarter, compared to $6.4 million for the comparable period of 2020. The increases in G&A expenses were primarily attributable to the hiring of additional personnel in G&A functions supporting the growth of the organization, as well as an increase in stock-based compensation expense.
    • Net Loss Attributable to Common Stockholders: SpringWorks reported net loss of $29.8 million, or $0.62 per share, for the first quarter of 2021. This compares to a net loss of $15.3 million, or $0.37 per share, for the comparable period of 2020.
    • Cash Position: Cash, cash equivalents and marketable securities were $541.0 million as of March 31, 2021.

    COVID-19 Update

    To date, the COVID-19 pandemic has had a relatively modest impact on SpringWorks' business operations, in particular on SpringWorks' clinical trial programs, and SpringWorks is undertaking considerable efforts to mitigate the various challenges presented by this crisis. For further details and descriptions of the risks associated with the COVID-19 pandemic, please see the Risk Factors in SpringWorks' periodic filings with the Securities and Exchange Commission and refer to the Forward-Looking Statements section in this press release.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    SpringWorks uses its website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the Company's website in the ‘Investors & Media' section. Accordingly, investors should monitor such portions of the SpringWorks website, in addition to following press releases, SEC filings and public conference calls and webcasts.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for initiation, progress and completion of SpringWorks' clinical trials or third-party clinical trials of its product candidates, the timing for expected data readouts from partners and partners' clinical trials, the fact that interim results from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials involving its product candidates, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in SpringWorks' SEC filings. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    SpringWorks Therapeutics, Inc.

    Condensed Consolidated Statements of Operations


    (Unaudited)
      
    Three Months Ended March 31,
    (in thousands, except share and per-share data)2021 2020
    Operating expenses:   
    Research and development$17,375  $9,727 
    General and administrative12,381  6,403 
    Total operating expenses29,756  16,130 
       
    Loss from operations(29,756) (16,130)
    Interest and other income:   
    Other income3   
    Interest income, net227  936 
    Total interest and other income230  936 
    Equity investment loss(261) (100)
    Net loss$(29,787) $(15,294)
       
    Net loss per share, basic and diluted$(0.62) $(0.37)
    Weighted average common shares outstanding, basic and diluted48,229,539  41,789,120 



     
    SpringWorks Therapeutics, Inc.

    Selected Balance Sheet Data


    (Unaudited)
        
     March 31, 2021 December 31, 2020
    (in thousands)   
    Cash, cash equivalents and marketable securities$541,042  $561,820 
    Working Capital (1)462,785  495,788 
    Total assets554,588  576,191 
    Total liabilities19,891  19,133 
    Accumulated deficit(148,390) (118,603)
    Total stockholders' equity534,697  557,058 



    (1)We define working capital as current assets less current liabilities.
      

    Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com

    References

    1 Bousquet E, Quantin X, Pujol J, et al. Notch Inhibition Overcomes Resistance to Tyrosine Kinase Inhibitors Promoted by Gate-Keeper Mutations in EGFR-Driven Lung Adenocarcinoma. J Clin Invest. 2020;130(2):612-624. //doi.org/10.1172/JCI126896.



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  16. - TEAD Inhibition Represents an Emerging Approach for Addressing Multiple Biomarker-Defined Cancers Characterized by Aberrant Hippo Pathway Signaling, which is Genetically Altered in up to 10% of Cancers -

    - SpringWorks Expects to Nominate a Development Candidate and Commence IND-Enabling Studies in 2022 -

    STAMFORD, Conn., May 06, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that it has entered into an exclusive worldwide license agreement with Katholieke Universiteit Leuven (KU Leuven) and the Flanders Institute for Biotechnology (VIB) for the in-license of…

    - TEAD Inhibition Represents an Emerging Approach for Addressing Multiple Biomarker-Defined Cancers Characterized by Aberrant Hippo Pathway Signaling, which is Genetically Altered in up to 10% of Cancers -

    - SpringWorks Expects to Nominate a Development Candidate and Commence IND-Enabling Studies in 2022 -

    STAMFORD, Conn., May 06, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that it has entered into an exclusive worldwide license agreement with Katholieke Universiteit Leuven (KU Leuven) and the Flanders Institute for Biotechnology (VIB) for the in-license of a portfolio of novel small molecule inhibitors of the TEA Domain (TEAD) family of transcription factors, designed for the potential treatment of biomarker-defined solid tumors driven by aberrant Hippo pathway signaling.

    "We are very pleased to expand our portfolio with the in-licensing of these selective and potent TEAD inhibitors, which are highly complementary to our strategy of developing novel targeted therapies for patients with biomarker-defined tumors," said Saqib Islam, Chief Executive Officer of SpringWorks. "Based on the compelling in vitro and in vivo data generated to date, we believe that the CD3 and VIB team has created unique and differentiated chemical matter that lays the foundation for SpringWorks to advance a potentially best-in-class targeted oncology opportunity on behalf of patients with devastating cancers. We look forward to nominating a development candidate from this portfolio and commencing IND-enabling activities next year."

    "Since first identified by our group and others in the fruit fly Drosophila almost 20 years ago, the Hippo pathway has come to be recognized as a central regulator of cell proliferation and organ growth. Given that the Hippo pathway is genetically altered in up to 10% of cancers, TEAD inhibition holds great promise as a therapeutic approach," said Professor Halder. Patrick Chaltin, Ph.D., Managing Director of CD3 at KU Leuven, added: "This agreement demonstrates CD3's strong drive to translate promising scientific insights into novel therapies that improve patients' lives, and we are very proud and excited to collaborate with SpringWorks given their success in rapidly advancing new targeted oncology treatments on behalf of patients."

    Under the terms of the agreement, SpringWorks will pay KU Leuven and VIB an upfront payment of $11 million. KU Leuven and VIB are also eligible to receive up to $285 million in development, regulatory and commercial milestones from SpringWorks and tiered single-digit percentage royalties based on any future net sales.

    About the Hippo Pathway and TEAD

    The Hippo pathway is an evolutionarily conserved signaling cascade that regulates organ development and tissue growth and regeneration. The TEA Domain (TEAD) family is a group of four homologous transcription factors whose activity is regulated by the Hippo pathway. The TEAD family is the terminal component of the Hippo pathway and transcriptional activity of this family has been implicated in a variety of tumors characterized by aberrant Hippo signaling. Transcription of TEAD target genes requires TEAD to form a transcriptional complex with the co-activators YAP or TAZ. Activation of the Hippo pathway prevents YAP and TAZ from translocating to the nucleus, preventing TEAD-dependent transcription. Aberrations and genetic mutations that limit Hippo pathway activation can result in significant increases in TEAD dependent transcription and have been identified as key drivers in several tumor types, including subsets of mesothelioma, non-small cell lung cancer, head and neck cancer, and kidney cancer.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, the interim data of the ReNeu clinical trial, including its interim primary efficacy, safety and tolerability data, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this presentation are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this presentation, including, without limitation, risks relating to: (i) the success and timing of our ongoing DeFi and ReNeu clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our product development activities and initiating clinical trials, (iv) the success and timing of our collaboration partners' ongoing and planned clinical trials, (v) our ability to obtain and maintain regulatory approval of any of our product candidates, (vi) our plans to research, discover and develop additional product candidates, (vii) our ability to enter into collaborations for the development of new product candidates, (viii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (ix) our ability to meet any specific milestones set forth herein, and (x) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines.​

    Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. ​

    For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" section(s) of our filings with the Securities and Exchange Commission. ​

    SpringWorks Media/Investor Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com



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  17. SOUTH SAN FRANCISCO, Calif. and STAMFORD, Conn., April 12, 2021 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (NASDAQ:ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor T cell (AlloCAR T™) therapies for cancer, and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the first patient has been dosed in a Phase 1 trial evaluating ALLO-715, Allogene's investigational anti-B-cell maturation antigen (BCMA) AlloCAR T therapy, in combination with nirogacestat, SpringWorks' investigational gamma secretase inhibitor (GSI), in patients…

    SOUTH SAN FRANCISCO, Calif. and STAMFORD, Conn., April 12, 2021 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (NASDAQ:ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor T cell (AlloCAR T™) therapies for cancer, and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the first patient has been dosed in a Phase 1 trial evaluating ALLO-715, Allogene's investigational anti-B-cell maturation antigen (BCMA) AlloCAR T therapy, in combination with nirogacestat, SpringWorks' investigational gamma secretase inhibitor (GSI), in patients with relapsed or refractory multiple myeloma.

    "We are pleased to have initiated this portion of the UNIVERSAL trial and look forward to investigating ALLO-715 in combination with nirogacestat as part of our comprehensive anti-BCMA strategy aimed at bringing an off-the-shelf cell therapy to patients with relapsed or refractory multiple myeloma," said David Chang, M.D., Ph.D., President, CEO and Co-Founder of Allogene Therapeutics.

    Gamma secretase inhibition prevents the cleavage and shedding of BCMA from the surface of multiple myeloma cells. In preclinical models, nirogacestat has been shown to increase the cell surface density of BCMA and reduce levels of soluble BCMA, which may enhance the activity of BCMA-targeted therapies.1 In addition, emerging clinical data suggest that a GSI may increase antitumor efficacy of BCMA-targeted autologous CAR T therapy in patients with relapsed and refractory multiple myeloma.2,3

    "Multiple myeloma is incurable for the overwhelming majority of patients diagnosed with this cancer and those who have relapsed or are refractory to current treatments have very limited therapeutic options available to them," said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics. "SpringWorks is advancing nirogacestat as a cornerstone of BCMA combination therapy across modalities, with the goal of potentiating BCMA therapies to provide better outcomes for patients. This study represents the first clinical trial combining nirogacestat with a CAR T therapy and we are delighted that the milestone of dosing the first patient has been achieved."

    The Phase 1 trial (NCT04093596), which is part of the ongoing UNIVERSAL trial being conducted by Allogene, is an open-label study evaluating the safety, tolerability and preliminary efficacy of ALLO-715 in combination with nirogacestat in patients with relapsed or refractory multiple myeloma. The trial is being advanced pursuant to a clinical trial collaboration agreement between Allogene and SpringWorks. Under the terms of the agreement, Allogene is sponsoring and conducting the Phase 1 study. Allogene and SpringWorks have formed a joint development committee to oversee this portion of the clinical study.

    About ALLO-715

    ALLO-715, an AlloCAR T therapy targeting B-cell maturation antigen (BCMA), is a potential novel treatment for multiple myeloma and other BCMA-positive malignancies. Multiple myeloma originates in the bone marrow and it is characterized by abnormalities in plasma cells that reproduce uncontrollably in the bone marrow and other disease sites.4 Multiple myeloma is incurable for most patients, as relapses occur despite most treatments available.5 Initial results from the Phase 1 UNIVERSAL study of ALLO-715 in relapsed/refractory multiple myeloma were presented at an oral session of the American Society of Hematology (ASH) annual meeting in December 2020. This study also uses ALLO-647, Allogene's anti-CD52 monoclonal antibody (mAb), as a part of its differentiated lymphodepletion regimen.

    ALLO-715 utilizes the TALEN® gene-editing technology pioneered and owned by Cellectis. Allogene has an exclusive license to the Cellectis technology for allogeneic products directed at the BCMA target. Allogene holds the global development and commercial rights for this investigational candidate.

    About Nirogacestat

    Nirogacestat is an investigational, oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has five collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities, including with an antibody-drug conjugate, two CAR T cell therapies and two bispecific antibodies. In addition, SpringWorks and Fred Hutchinson Cancer Research Center have entered into a sponsored research agreement to further characterize the ability of nirogacestat to modulate BCMA and potentiate BCMA directed therapies using a variety of preclinical and patient-derived multiple myeloma models developed by researchers at Fred Hutch.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors and from the European Commission for the treatment of soft tissue sarcoma. The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.

    About Allogene Therapeutics

    Allogene Therapeutics, with headquarters in South San Francisco, is a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor T cell (AlloCAR T™) therapies for cancer. Led by a management team with significant experience in cell therapy, Allogene is developing a pipeline of "off-the-shelf" CAR T cell therapy candidates with the goal of delivering readily available cell therapy on-demand, more reliably, and at greater scale to more patients. For more information, please visit www.allogene.com, and follow @AllogeneTx on Twitter and LinkedIn.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com, and follow @SpringWorksTx on Twitter and LinkedIn.

    Allogene Forward-Looking Statements

    This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The press release may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the ability to progress the UNIVERSAL trial and combination of ALLO-715 with nirogacestat; the ability of ALLO-715 in combination with nirogacestat to provide better outcomes for patients; the ability to develop allogeneic CAR T therapies for cancer and the potential benefits of AlloCAR T therapy. Various factors may cause differences between Allogene's expectations and actual results as discussed in greater detail in Allogene's filings with the Securities and Exchange Commission (SEC), including without limitation in its Form 10-K for the year ended December 31, 2020. Any forward-looking statements that are made in this press release speak only as of the date of this press release. Allogene assumes no obligation to update the forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

    AlloCAR T™ is a trademark of Allogene Therapeutics, Inc.

    SpringWorks Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success and timing of our product development activities, including the initiation and completion of SpringWorks' clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our collaboration partners' ongoing and planned clinical trials, (iv) our ability to obtain and maintain regulatory approval of any of our product candidates, (v) our plans to research, discover and develop additional product candidates, (vi) our ability to enter into collaborations for the development of new product candidates, (vii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (viii) our ability to meet any specific milestones set forth herein, and (ix) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" in Item 1A of Part I of SpringWorks' Annual Report on Form 10-K for the year ended December 31, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings.

    Allogene Media/Investor Contact:

    Christine Cassiano

    Chief Communications Officer

    (714) 552-0326

    Christine.Cassiano@allogene.com

    SpringWorks Media/Investor Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    203-561-1646

    kdiamond@springworkstx.com

    References:

    1 Eastman et al., Abstract #4401 "Synergistic Activity of Belantamab Mafodotin (anti-BCMA immuno-conjugate) with Nirogacestat (PF-03084014, gamma-secretase inhibitor) in BCMA-Expressing Cancer Cell Lines", ASH 2019.

    2 Cowan et al., Abstract #204 "Efficacy and Safety of Fully Human BCMA CAR T Cells in Combination with a Gamma Secretase Inhibitor to Increase Bcma Surface Expression in Patients with Relapsed or Refractory Multiple Myeloma", ASH 2019.

    3 Blood. 2019 Nov 7;134(19):1585-1597. doi: 10.1182/blood.2019000050

    4 Multiple myeloma - Genetics Home Reference - NIH. Retrieved from https://ghr.nlm.nih.gov/condition/multiple-myeloma#

    5 Sonneveld P, Broijl A. Treatment of relapsed and refractory multiple myeloma. Haematologica. 2016;101(4):396-406



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  18. STAMFORD, Conn., April 01, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the first patient has been dosed in a Phase 1b clinical trial evaluating nirogacestat, an investigational gamma secretase inhibitor (GSI) being developed by SpringWorks, in combination with teclistamab, an investigational bispecific antibody targeting B-cell maturation antigen (BCMA) and CD3 being developed by Janssen Research & Development, LLC (Janssen), in patients with relapsed or refractory multiple myeloma.

    Gamma secretase inhibition prevents the cleavage and shedding of BCMA from…

    STAMFORD, Conn., April 01, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the first patient has been dosed in a Phase 1b clinical trial evaluating nirogacestat, an investigational gamma secretase inhibitor (GSI) being developed by SpringWorks, in combination with teclistamab, an investigational bispecific antibody targeting B-cell maturation antigen (BCMA) and CD3 being developed by Janssen Research & Development, LLC (Janssen), in patients with relapsed or refractory multiple myeloma.

    Gamma secretase inhibition prevents the cleavage and shedding of BCMA from the surface of myeloma cells. In preclinical models, nirogacestat has been shown to increase the cell surface density of BCMA and reduce levels of soluble BCMA, which may enhance the activity of BCMA-targeted therapies.

    "We are very pleased that patients have been dosed in this study, which represents the first clinical study combining nirogacestat with a bispecific antibody in patients with multiple myeloma," said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics. "We are advancing nirogacestat as a cornerstone of BCMA combination therapy across modalities and look forward to the enrollment of patients into multiple Phase 1 studies and generating data with our BCMA collaborators in 2021."

    The Phase 1b trial (NCT04722146), which is part of a multi-arm trial being conducted by Janssen, is an open-label study to evaluate the safety, tolerability and preliminary efficacy of nirogacestat in combination with teclistamab in patients with relapsed or refractory multiple myeloma. The trial is being advanced pursuant to a clinical collaboration and supply agreement that SpringWorks entered into with Janssen Biotech, Inc. in September 2020. Under the terms of the agreement, Janssen is assuming all costs associated with the Phase 1b study, other than expenses related to the supply of nirogacestat. SpringWorks has formed a joint oversight committee with Janssen to coordinate the sharing of study results, regulatory and other activities in connection with the study.

    In addition to its ongoing clinical collaborations with BCMA-directed therapies, SpringWorks is conducting a global Phase 3, double-blind, randomized, placebo-controlled clinical trial (the DeFi Trial) to evaluate nirogacestat as a monotherapy in adults with progressing desmoid tumors.

    About Nirogacestat

    Nirogacestat is an investigational, oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has five collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities, including with an antibody-drug conjugate, two CAR T cell therapies and two bispecific antibodies. In addition, SpringWorks and Fred Hutchinson Cancer Research Center have entered into a sponsored research agreement to further characterize the ability of nirogacestat to modulate BCMA and potentiate BCMA directed therapies using a variety of preclinical and patient-derived multiple myeloma models developed by researchers at Fred Hutch.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors and from the European Commission for the treatment of soft tissue sarcoma. The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com, and follow @SpringWorksTx on Twitter and LinkedIn.

    SpringWorks Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success and timing of our product development activities, including the initiation and completion of SpringWorks' clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our collaboration partners' ongoing and planned clinical trials, (iv) our ability to obtain and maintain regulatory approval of any of our product candidates, (v) our plans to research, discover and develop additional product candidates, (vi) our ability to enter into collaborations for the development of new product candidates, (vii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (viii) our ability to meet any specific milestones set forth herein, and (ix) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" in Item 1A of Part I of SpringWorks' Annual Report on Form 10-K for the year ended December 31, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings.

    SpringWorks Media/Investor Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    203-561-1646

    kdiamond@springworkstx.com



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  19. STAMFORD, Conn., March 22, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today that Bhavesh Ashar has been appointed Chief Commercial Officer. Mr. Ashar has more than 20 years of global pharmaceutical and biotechnology experience, most recently having served as Senior Vice President, General Manager of U.S. Oncology at Bayer Healthcare. Mr. Ashar will build and lead commercial operations at SpringWorks as the company continues to advance its diversified targeted oncology portfolio, which includes two potentially registrational rare oncology programs.

    "We are very happy…

    STAMFORD, Conn., March 22, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today that Bhavesh Ashar has been appointed Chief Commercial Officer. Mr. Ashar has more than 20 years of global pharmaceutical and biotechnology experience, most recently having served as Senior Vice President, General Manager of U.S. Oncology at Bayer Healthcare. Mr. Ashar will build and lead commercial operations at SpringWorks as the company continues to advance its diversified targeted oncology portfolio, which includes two potentially registrational rare oncology programs.

    "We are very happy to be welcoming Bhavesh to the SpringWorks team. His deep knowledge and experience in oncology product launches and proven record of building high-performing commercial teams will be instrumental as we prepare to serve patients with various forms of cancer," said Saqib Islam, Chief Executive Officer of SpringWorks. "SpringWorks has made great progress in developing nirogacestat for patients with desmoid tumors and mirdametinib for patients with NF1-associated plexiform neurofibromas. We look forward to reporting data from our two registrational trials in these devastating tumors later this year, including topline data from our Phase 3 DeFi trial and additional data from our Phase 2b ReNeu trial."

    "What attracted me to SpringWorks was the breadth of opportunities across the company's rare oncology programs and larger opportunities in genetically defined cancers," said Mr. Ashar. "I look forward to joining this team of passionate and driven people who share a deep commitment to bringing life-changing medicines to patients and families suffering from severe rare diseases and cancer."

    As Senior Vice President, General Manager of U.S. Oncology at Bayer, Mr. Ashar served as Business Unit head for the U.S. Oncology franchise and was responsible for a broad portfolio in prostate, liver, colorectal, GIST, hematologic and tumor-agnostic biomarker driven cancers. Before joining Bayer in 2017, he held roles of increasing responsibility at Sanofi Genzyme over a 15-year tenure, including Vice President, General Manager of U.S. Oncology and Vice President, Global Head of Transplant. Prior to Sanofi Genzyme, Mr. Ashar was a consultant with McKinsey & Company where he served clients in the biopharmaceutical space. Mr. Ashar received an MBA from the University of Chicago and a BS in Mathematics from the Imperial College in London.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success and timing of our product development activities, including the initiation and completion of SpringWorks' clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our collaboration partners' ongoing and planned clinical trials, (iv) our ability to obtain and maintain regulatory approval of any of our product candidates, (v) our plans to research, discover and develop additional product candidates, (vi) our ability to enter into collaborations for the development of new product candidates, (vii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (viii) our ability to meet any specific milestones set forth herein, and (ix) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" in Item 1A of Part I of SpringWorks' Annual Report on Form 10-K for the year ended December 31, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings.

    Contact

    Kim Diamond

    Vice President Communications and Investor Relations

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com



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  20. - Reported Interim Data from Phase 2b ReNeu Trial of Mirdametinib in NF1-PN Demonstrating Encouraging Clinical Activity and Tolerability in the First 20 Adult Patients Enrolled -

    Allogene- and Janssen-Sponsored Phase 1 Trials Evaluating Nirogacestat in Combination with BCMA Therapies Have Initiated -

    - Continued to Strengthen Nirogacestat Patent Portfolio -

    - Multiple Data Readouts Expected in 2021 Across Pipeline, Including Topline Data from Phase 3 DeFi Trial in Desmoid Tumors and Initial Nirogacestat BCMA Combination Data -

    - Ended 2020 with $561.8 Million in Cash, Cash Equivalents and Marketable Securities -

    STAMFORD, Conn., Feb. 25, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical…

    - Reported Interim Data from Phase 2b ReNeu Trial of Mirdametinib in NF1-PN Demonstrating Encouraging Clinical Activity and Tolerability in the First 20 Adult Patients Enrolled -



    Allogene- and Janssen-Sponsored Phase 1 Trials Evaluating Nirogacestat in Combination with BCMA Therapies Have Initiated -

    - Continued to Strengthen Nirogacestat Patent Portfolio -

    - Multiple Data Readouts Expected in 2021 Across Pipeline, Including Topline Data from Phase 3 DeFi Trial in Desmoid Tumors and Initial Nirogacestat BCMA Combination Data -

    - Ended 2020 with $561.8 Million in Cash, Cash Equivalents and Marketable Securities -

    STAMFORD, Conn., Feb. 25, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported fourth quarter and full-year financial results for the period ended December 31, 2020 and provided an update on recent company developments.

    "2020 was a year characterized by strong performance for SpringWorks as we advanced our diversified targeted oncology portfolio, which currently spans 10 development programs across our three core focus areas: late-stage rare oncology, BCMA combinations in multiple myeloma and biomarker-defined metastatic solid tumors," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our clinical execution has set the stage for multiple important data readouts in 2021, including the interim ReNeu data announced this morning and topline data from our Phase 3 DeFi trial later this year, and our business development efforts have allowed us to pursue additional combination therapy programs, continuing to grow our portfolio of opportunities across a broad range of potentially high-value and high-unmet need oncology patient settings. We look forward to reporting on our progress throughout 2021."

    Recent Business Highlights and Upcoming Milestones

    Late-Stage Rare Oncology

    • In February 2021, SpringWorks reported interim data from the adult stratum of the ongoing potentially registrational Phase 2b ReNeu trial evaluating mirdametinib in pediatric and adult patients with NF1-associated plexiform neurofibromas. Of the first 20 adult patients enrolled, 50% had achieved an objective response, the primary endpoint of the study, as assessed by blinded independent central review, and 16 of these 20 patients (80%) remained on study as of the January 22, 2021 data cutoff. In addition, mirdametinib was generally well tolerated, with the majority of treatment-related adverse events (TRAE) being Grade 1 or 2 and only one Grade 3 TRAE reported; there have been no Grade 4 or 5 AEs reported. SpringWorks reported that the ReNeu trial has reached approximately 70% of its final enrollment target of 100 patients and the Company expects to complete enrollment of the ReNeu trial in the second half of 2021.
    • SpringWorks expects to report topline data from the Phase 3 DeFi trial in the second half of 2021.
    • Recruitment is ongoing in a Phase 2 study sponsored by the Children's Oncology Group evaluating nirogacestat in pediatric patients with desmoid tumors.

    B-cell Maturation Antigen (BCMA) Combinations in Multiple Myeloma

    • Enrollment is ongoing in a Phase 1b trial sponsored by GSK evaluating nirogacestat in combination with BLENREP (belantamab mafodotin-blmf), GSK's anti-B-cell maturation antigen (BCMA) antibody-drug conjugate, in adult patients with relapsed or refractory multiple myeloma. Initial clinical data from this study are expected in 2021.
    • Two collaborator-sponsored Phase 1 studies were initiated evaluating nirogacestat in combination with BCMA therapies: nirogacestat + Allogene's ALLO-715 and nirogacestat + Janssen's teclistamab. SpringWorks also expects that two additional collaborator-sponsored trials will initiate in the first half of 2021, as previously disclosed: nirogacestat + Pfizer's elranatamab (PF‐06863135) and nirogacestat + Precision's PBCAR269A.

    Biomarker-Defined Metastatic Solid Tumors

    • Enrollment is ongoing in a Phase 1b/2 trial evaluating mirdametinib with BeiGene's RAF dimer inhibitor, lifirafenib, in adult patients with RAS/RAF mutant and other MAPK pathway aberrant solid tumors. BeiGene is sponsoring this trial and SpringWorks and BeiGene expect to report initial clinical data in 2021.
    • Enrollment is ongoing in a Phase 1 trial of BGB-3245 in adult patients with RAF mutant solid tumors. BGB-3245 is a selective RAF dimer inhibitor being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene. Initial clinical data from the MapKure-sponsored Phase 1 trial are expected in 2021.
    • In December 2020, SpringWorks entered into a research collaboration agreement with Dr. Tatu Pantsar and Dr. Antti Poso at the University of Eastern Finland to conduct novel computational biology research to characterize the effector protein dynamics of different RAS mutations. The objective of the collaboration is to understand the biological consequences of KRAS-effector protein interactions and downstream oncogenic signaling with the goal of guiding patient selection strategies and exploring novel targets for future targeted therapy development.

    General Corporate

    • In February 2021, a Notice of Allowance was received from the United States Patent and Trademark Office for Patent Application No. 16/886,622 (the ‘622 application) with composition of matter claims covering several polymorphic forms of nirogacestat, including the polymorphic form that is currently in clinical development, which is also covered by U.S. Patent 10,590,087 (the ‘087 patent, issued in March 2020). Pursuant to an existing worldwide license agreement with Pfizer, SpringWorks has exclusive rights to the patent issuing from the ‘622 application. The patent granting from the ‘622 application, as well as the ‘087 patent, will both expire in August 2039.
    • In October 2020, SpringWorks completed a follow-on public offering that raised $269.5 million in net proceeds, which included the underwriters' full exercise of their option to purchase additional shares of common stock.
    • In October 2020, SpringWorks and Jazz Pharmaceuticals entered into an asset purchase and exclusive license agreement under which Jazz acquired SpringWorks' fatty acid amide hydrolase (FAAH) inhibitor program. Under the terms of the agreement, Jazz made an upfront payment of $35 million to SpringWorks, with potential future milestone payments of up to $375 million payable to SpringWorks based upon the achievement of certain clinical development, regulatory and commercial milestones. In addition, SpringWorks will receive incremental tiered royalties on future net sales of JZP-150 (formerly PF-04457845) in the mid- to high-single digit percentages.

    Fourth Quarter and Full Year 2020 Financial Results

    • Revenue: Revenue of $35.0 million for the fourth quarter and year ended December 31, 2020 was attributable to the nonrefundable upfront payment from Jazz in October 2020, related to the asset purchase and exclusive license agreement between SpringWorks and Jazz.
    • Research and Development (R&D) Expenses: R&D expenses were $15.3 million and $51.9 million for the fourth quarter and year-to-date periods, respectively, compared to $12.2 million and $42.5 million for the comparable periods of 2019. The increases in R&D expenses in 2020 were primarily attributable to growth in employee costs associated with increases in the number of R&D personnel, including non-cash share-based compensation expenses, and increases in external costs related to drug manufacturing and clinical trial costs.
    • General and Administrative (G&A) Expenses: G&A expenses were $8.5 million and $29.5 million for the fourth quarter and year-to-date periods, respectively, compared to $5.2 million and $16.7 million for the comparable periods of 2019. The increases in G&A expenses in 2020 were primarily attributable to growth in employee costs associated with increases in the number of G&A personnel supporting the growth of the organization, including non-cash share-based compensation expenses, as well as increases in expenses related to the expansion of business activities.
    • Net Loss Attributable to Common Stockholders: SpringWorks reported net income of $11.3 million, or $0.24 per share, for the fourth quarter, and a net loss of $45.6 million, or $1.05 loss per share, for the year ended December 31, 2020. This compares to net losses of $16.2 million, or $0.39 loss per share, and $50.6 million, or $3.81 loss per share, for the comparable periods of 2019, respectively.
    • Cash Position: Cash, cash equivalents and marketable securities were $561.8 million as of December 31, 2020. This includes the net proceeds of $269.5 million from the Company's follow-on public offering completed in October 2020, and the $35.0 million upfront payment from Jazz Pharmaceuticals in October 2020, related to the asset purchase and exclusive license agreement between SpringWorks and Jazz.

    COVID-19 Update

    To date, the COVID-19 pandemic has had a relatively modest impact on SpringWorks' business operations, in particular on SpringWorks' clinical trial programs, and SpringWorks is undertaking considerable efforts to mitigate the various challenges presented by this crisis. For further details and descriptions of the risks associated with the COVID-19 pandemic, please see the Risk Factors in SpringWorks' periodic filings with the Securities and Exchange Commission and refer to the Forward-Looking Statements section in this press release.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    SpringWorks uses its website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the Company's website in the ‘Investors & Media' section. Accordingly, investors should monitor such portions of the SpringWorks website, in addition to following press releases, SEC filings and public conference calls and webcasts.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials or third-party clinical trials of its product candidates, the fact that interim results from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials involving its product candidates, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in SpringWorks' SEC filings. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    SpringWorks Therapeutics, Inc.

    Consolidated Statements of Operations

    (Unaudited)

            
      Year Ended December 31, 
    (in thousands, except share, unit, per-unit and per-share data)    2020  2019     2018 
              
    Licensing revenue $35,000  $  $ 
    Operating expenses:         
    Research and development  51,859   42,545   9,898 
    General and administrative  29,465   16,694   8,593 
    Total operating expenses  81,324   59,239   18,491 
    Loss from operations  (46,324)  (59,239)  (18,491)
    Other income:         
    Other income  25       
    Interest income, net  1,330   3,547   678 
    Total other income  1,355   3,547   678 
    Equity investment loss  (605)  (2,614)   
    Net loss $(45,574) $(58,306) $(17,813)
    Reconciliation of net loss to net loss attributable to common stockholders and unit holders:         
    Net loss $(45,574) $(58,306) $(17,813)
    Net gain attributable to extinguishment of Series A convertible preferred and Junior Series A convertible preferred units     7,729    
    Net loss attributable to common stockholders and unit holders, basic and diluted $(45,574) $(50,577) $(17,813)
    Net loss per unit, basic and diluted $  $  $(52.24)
    Net loss per share, basic and diluted $(1.05) $(3.81) $ 
    Weighted average common units outstanding, basic and diluted        341,014 
    Weighted average common shares outstanding, basic and diluted  43,300,063   13,274,836    





    SpringWorks Therapeutics, Inc.


    Selected Balance Sheet Data

    (Unaudited)

      As of December 31,
    (in thousands)  2020    2019 
           
    Cash, cash equivalents and marketable securities $561,820  $ 327,652 
    Working Capital (1)  495,788    319,391 
    Total Assets  576,191    334,831 
    Total liabilities  19,133    12,759 
    Accumulated deficit  (118,603)   (73,029)
    Total stockholders' (deficit) equity  557,058    322,072 

    (1) We define working capital as current assets less current liabilities.

    Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com



    Primary Logo

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  21. - Interim Data Reported from First 20 Adult Patients Enrolled with January 22, 2021 Data Cutoff -

    - 10/20 Patients (50%) Had Achieved an Objective Response by Blinded Independent Central Review (BICR) and 16/20 (80%) Remain on Study -

    - Mirdametinib Continues to Show a Potentially Differentiated Safety and Tolerability Profile -

    - Trial is Approximately 70% Enrolled and With Full Enrollment Expected in 2H2021 -

    - Conference Call and Webcast Scheduled for Today at 8:30 a.m. Eastern Time -

    STAMFORD, Conn., Feb. 25, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported…

    - Interim Data Reported from First 20 Adult Patients Enrolled with January 22, 2021 Data Cutoff -

    - 10/20 Patients (50%) Had Achieved an Objective Response by Blinded Independent Central Review (BICR) and 16/20 (80%) Remain on Study -

    - Mirdametinib Continues to Show a Potentially Differentiated Safety and Tolerability Profile -

    - Trial is Approximately 70% Enrolled and With Full Enrollment Expected in 2H2021 -

    - Conference Call and Webcast Scheduled for Today at 8:30 a.m. Eastern Time -

    STAMFORD, Conn., Feb. 25, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported interim data from the first 20 adult patients enrolled in the ongoing Phase 2b ReNeu trial evaluating mirdametinib, an investigational MEK inhibitor, in adult and pediatric patients with NF1-associated plexiform neurofibromas (NF1-PN). As of the January 22nd data cutoff date, 10/20 (50%) of these patients had achieved an objective response, as assessed by blinded independent central review (BICR), 16/20 (80%) remained on study, and the median time on treatment was 10.1 cycles (approximately 10 months). Mirdametinib was also generally well tolerated, with the majority of treatment related adverse events (TRAE) being Grade 1 or 2 and only one Grade 3 TRAE; there have been no Grade 4 or 5 adverse events (AE). SpringWorks also provided an update on the enrollment status of ReNeu, highlighting that the trial has reached approximately 70% of its target enrollment of 100 patients and that full enrollment is expected in the second half of 2021.

    "We are very encouraged by these emerging data from our ongoing ReNeu trial, as they reaffirm our belief that mirdametinib has the potential to be a best-in-class treatment for patients with NF1-PN," said Saqib Islam, Chief Executive Officer of SpringWorks. "The robust response rate, which was assessed by blinded independent central review, and the very encouraging tolerability profile observed in these interim data are particularly compelling given the unmet need among NF1-PN patients for a therapy that can provide durable efficacy while maintaining a safety profile that is suitable for long-term dosing. We look forward to completing enrollment in the ReNeu trial in the second half of this year and sharing additional data from the study at a future medical conference in 2021."

    Interim Phase 2b Data from ReNeu Trial:

    The interim Phase 2b ReNeu data set for the first 20 adult patients enrolled utilized a January 22, 2021 data cutoff. Objective responses were defined as a ≥ 20% reduction in target tumor volume measured by MRI and were assessed by BICR. Patients received mirdametinib at a dose of 2 mg/m2 twice daily (maximum dose: 4 mg twice daily) without regard to food on a three weeks-on, one week-off intermittent schedule, with patients being allowed to stay on treatment for up to 24 cycles (approximately two years). The median time on treatment for the 20 adult patients evaluated for this analysis was 10.1 cycles (approximately 10 months), with an initial efficacy assessment performed following cycle five and then every four cycles thereafter.

    The preliminary efficacy and safety analysis showed:

    • 10/20 (50%) of patients had achieved an objective response by BICR.
    • For seven of the 10 patients who achieved an initial objective response, subsequent scheduled scans were available, and six of these seven patients had confirmed responses.
    • 16/20 (80%) of these patients remain on study and only one patient required a dose reduction due to an AE. Reasons for discontinuation included one each of progressive disease, participant decision, AE (Grade 1 diarrhea), and a patient being unable to undergo required MRI imaging due to a titanium rod implant from non-treatment-related worsening of scoliosis.
    • A generally well-tolerated safety profile. The majority of TRAEs were Grade 1 or 2 with only one Grade 3 TRAE reported. No Grade 4 or 5 AEs have been reported. The most common TRAEs were rash, nausea and diarrhea.

    Conference Call and Webcast:

    SpringWorks will host a conference call and webcast today, Thursday, February 25, 2021, at 8:30 a.m. Eastern Time to discuss the ReNeu trial data and program update. Participants can listen to the call by dialing +1 (800) 708-4539 (domestic) or +1 (847) 619-6396 (international) and providing the confirmation number 50110177. A live webcast presentation can be accessed through the "Investors & Media" section of the Company's website at https://ir.springworkstx.com/. A replay of the webcast will be available on the SpringWorks website for a limited time following the event.

    About the ReNeu Trial

    The ReNeu trial is a multi-center, open-label Phase 2b trial evaluating the efficacy, safety, and tolerability of mirdametinib in patients two years of age and older with an inoperable NF1-associated PN causing significant morbidity. The study will enroll approximately 100 patients in the United States. Patients receive mirdametinib at a dose of 2 mg/m2 twice daily (maximum dose of 4 mg twice daily, calculated based on body surface area) without regard to food. Mirdametinib is administered in a three-weeks on, one-week off dosing schedule.

    The primary endpoint is objective response rate, defined as ≥ 20% reduction in target tumor volume as measured by MRI and assessed by BICR. Secondary endpoints include safety and tolerability measures, duration of response, and changes from baseline in patient reported outcomes.

    More information about the ReNeu trial is available at www.clinicaltrials.gov under the identifier NCT03962543.

    About NF1-PN

    Neurofibromatosis type 1 (NF1) is a rare genetic disorder that arises from mutations in the NF1 gene, which encodes for neurofibromin, a key suppressor of the MAPK pathway.1,2 NF1 is the most common form of neurofibromatosis, with an estimated global birth incidence of approximately 1 in 3,000 individuals, and approximately 100,000 patients living with NF1 in the United States.3,4 The clinical course of NF1 is heterogeneous and manifests in a variety of symptoms across numerous organ systems, including abnormal pigmentation, skeletal deformities, tumor growth and neurological complications, such as cognitive impairment.5 Patients with NF1 have an eight to 15-year mean reduction in their life expectancy compared to the general population.2

    NF1 patients have approximately a 30-50% lifetime risk of developing plexiform neurofibromas, or PN, which are tumors that grow in an infiltrative pattern along the peripheral nerve sheath and that can cause severe disfigurement, pain and functional impairment; in rare cases, NF1-PN may be fatal.3,4,6 Patients with NF1 can also experience additional manifestations, including neurocognitive deficits and developmental delays.4 NF1-PNs are most often diagnosed in the first two decades of life.3 These tumors can be aggressive and are associated with clinically significant morbidities; typically, they grow more rapidly during childhood.7,8

    Surgical removal of these tumors is challenging due to the infiltrative tumor growth pattern along nerves and can lead to permanent nerve damage and disfigurement.9 MEK inhibitors have emerged as a validated class of treatment for NF1-PN.4

    About Mirdametinib

    Mirdametinib is an oral small molecule designed to inhibit MEK1 and MEK2. MEK proteins occupy a pivotal position in the MAPK pathway, a key signaling network that regulates cell growth and survival, and that plays a central role in multiple oncology and rare disease indications.

    Mirdametinib has been evaluated in several Phase 1 and Phase 2 clinical trials, with over 200 subjects having been exposed to treatment. A Phase 2 trial was conducted by the Neurofibromatosis Clinical Trial Consortium and evaluated mirdametinib in 19 adolescent and adult patients with inoperable and symptomatic or growing plexiform neurofibromas. Patients received an oral dose of 2 mg/m2 BID with a maximum dose of 4 mg BID on a four-week cycle of three weeks-on, one week-off. Eight patients (42%) achieved an objective response by cycle 12, prospectively defined as volumetric reduction in their target PN of at least 20%. Mirdametinib was generally well-tolerated in this trial. The most commonly reported treatment-emergent Grade 2 or higher AEs were acneiform rash, fatigue and nausea.  

    In addition to the Phase 2b monotherapy trial in NF1- PN, and given the critical role that the MAPK pathway plays in the growth and proliferation of a large number of tumor types, SpringWorks is also pursuing mirdametinib in combination with other rational anti-cancer agents across a range of solid tumors.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com, and follow @SpringWorksTx on Twitter and LinkedIn.

    Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, the interim data of the ReNeu clinical trial, including its interim primary efficacy, safety and tolerability data, and other future conditions. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "would," "should" and "could," and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this presentation are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this presentation, including, without limitation, risks relating to: (i) the success and timing of our ongoing DeFi and ReNeu clinical trials, (ii) the fact that interim data from a clinical study may not be predictive of the final results of such study or the results of other ongoing or future studies, (iii) the success and timing of our product development activities and initiating clinical trials, (iv) the success and timing of our collaboration partners' ongoing and planned clinical trials, (v) our ability to obtain and maintain regulatory approval of any of our product candidates, (vi) our plans to research, discover and develop additional product candidates, (vii) our ability to enter into collaborations for the development of new product candidates, (viii) our ability to establish manufacturing capabilities, and our and our collaboration partners' abilities to manufacture our product candidates and scale production, (ix) our ability to meet any specific milestones set forth herein, and (x) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines.

    Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements.

    For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks' expectations and actual results, you should review the "Risk Factors" section(s) of our filings with the Securities and Exchange Commission.

    SpringWorks Media/Investor Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    203-561-1646

    kdiamond@springworkstx.com

    References

    1. Yap YS, McPherson JR, Ong CK, et al. The NF1 gene revisited - from bench to bedside. Oncotarget. 2014;5(15):5873-5892. doi:10.18632/oncotarget.2194.
    2. Rasmussen S, Friedman J. NF1 Gene and Neurofibromatosis 1. Am J Epidemiol. 2000;151(1):33-40. doi:10.1093/oxfordjournals.aje.a010118.
    3. Prada C, Rangwala F, Martin L et al. Pediatric Plexiform Neurofibromas: Impact on Morbidity and Mortality in Neurofibromatosis Type 1. J Pediatr. 2012;160(3):461-467. doi:10.1016/j.jpeds.2011.08.051.
    4. Ferner R. Neurofibromatosis 1 and neurofibromatosis 2: a twenty first century perspective. The Lancet Neurology. 2007;6(4):340-351. doi:10.1016/s1474-4422(07)70075-3.
    5. Weiss BD, Wolters PL, Plotkin SR, et al. NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults With NF1-Related Plexiform Neurofibromas. Journal of Clinical Oncology. 2021;JCO.20.02220.doi.org/10. 1200/JCO.20.02220.
    6. Hirbe A, Gutmann D. Neurofibromatosis type 1: a multidisciplinary approach to care. The Lancet Neurology. 2014;13(8):834-843. doi:10.1016/s1474-4422(14)70063-8.
    7. Gross A, Singh G, Akshintala S et al. Association of plexiform neurofibroma volume changes and development of clinical morbidities in neurofibromatosis 1. Neuro Oncol. 2018;20(12):1643-1651. doi:10.1093/neuonc/noy067.
    8. Nguyen R, Dombi E, Widemann B et al. Growth dynamics of plexiform neurofibromas: a retrospective cohort study of 201 patients with neurofibromatosis 1. Orphanet J Rare Dis. 2012;7(1):75. doi:10.1186/1750-1172-7-75.
    9. Needle M, Cnaan A, Dattilo J et al. Prognostic signs in the surgical management of plexiform neurofibroma: The Children's Hospital of Philadelphia experience, 1974-1994. J Pediatr. 1997;131(5):678-682. doi:10.1016/s0022-3476(97)70092-1.


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  22. STAMFORD, Conn., Feb. 22, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that management will participate in the following upcoming investor conferences:

    • Cowen 41st Annual Health Care Conference on Wednesday, March 3 from 11:00-11:30 a.m. ET
    • H.C. Wainwright Global Life Sciences Conference on Tuesday, March 9 (presentation available on-demand)
    • Barclays Global Healthcare Conference on Wednesday, March 10 from 3:35-4:00 p.m. ET

    Webcasts of these events can be accessed by visiting the "Events & Presentations" page within the Investors & Media section of the SpringWorks…

    STAMFORD, Conn., Feb. 22, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that management will participate in the following upcoming investor conferences:

    • Cowen 41st Annual Health Care Conference on Wednesday, March 3 from 11:00-11:30 a.m. ET
    • H.C. Wainwright Global Life Sciences Conference on Tuesday, March 9 (presentation available on-demand)
    • Barclays Global Healthcare Conference on Wednesday, March 10 from 3:35-4:00 p.m. ET



    Webcasts of these events can be accessed by visiting the "Events & Presentations" page within the Investors & Media section of the SpringWorks website at https://ir.springworkstx.com. A replay of the webcasts will be available on the Company's website for a limited time following the conferences.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Contact:

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com





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  23. STAMFORD, Conn., Feb. 04, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will participate in a fireside chat at the Guggenheim Healthcare Talks 2021 Oncology Day on Friday, February 12, 2021 at 3:00 p.m. Eastern Standard Time.

    To access the live webcast of SpringWorks' presentation, please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the…

    STAMFORD, Conn., Feb. 04, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will participate in a fireside chat at the Guggenheim Healthcare Talks 2021 Oncology Day on Friday, February 12, 2021 at 3:00 p.m. Eastern Standard Time.

    To access the live webcast of SpringWorks' presentation, please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the conference.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Contact:

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com



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  24. STAMFORD, Conn., Jan. 05, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will present at the 39th Annual J.P. Morgan Healthcare Conference on Tuesday, January 12, 2021 at 7:30 a.m. Eastern Standard Time.

    To access the live webcast of SpringWorks' presentation, please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the conference.

    About SpringWorks

    STAMFORD, Conn., Jan. 05, 2021 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will present at the 39th Annual J.P. Morgan Healthcare Conference on Tuesday, January 12, 2021 at 7:30 a.m. Eastern Standard Time.

    To access the live webcast of SpringWorks' presentation, please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the conference.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Contact:

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com



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  25. STAMFORD, Conn., Dec. 15, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today that Badreddin Edris, Ph.D., currently Chief Business Officer, has been promoted to Chief Operating Officer, and L. Mary Smith, Ph.D., currently Senior Vice President of Clinical Research and Development, has been promoted to Chief Development Officer.

    "Badreddin and Mary have demonstrated exceptional leadership and execution in their respective roles, which have been critical to the growth of SpringWorks, and it is my great pleasure to announce their promotions," said Saqib Islam, Chief Executive…

    STAMFORD, Conn., Dec. 15, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today that Badreddin Edris, Ph.D., currently Chief Business Officer, has been promoted to Chief Operating Officer, and L. Mary Smith, Ph.D., currently Senior Vice President of Clinical Research and Development, has been promoted to Chief Development Officer.

    "Badreddin and Mary have demonstrated exceptional leadership and execution in their respective roles, which have been critical to the growth of SpringWorks, and it is my great pleasure to announce their promotions," said Saqib Islam, Chief Executive Officer of SpringWorks. "They both embody SpringWorks' core values, continuously looking for opportunities on behalf of the patients we ultimately work for. I look forward to partnering with them and the rest of our experienced leadership team as we advance our ten clinical development programs for patients with severe rare diseases and cancer."

    Dr. Edris joined SpringWorks in 2018 as Chief Business Officer to lead the company's corporate strategy, business development and intellectual property efforts. During his tenure, he has played a pivotal role in advancing the company's strategy to develop nirogacestat as a cornerstone of BCMA combination therapy for multiple myeloma as well as in the company's expansion into biomarker-defined metastatic solid tumors. Dr. Edris' professional experience spans private and public equity investing, company formation and operations, corporate and business development, and strategic and product planning in the biotechnology industry. Prior to joining SpringWorks, Dr. Edris was an investment and operating professional on the private equity team at OrbiMed. Dr. Edris also co-founded and held operating roles at two OrbiMed portfolio companies, Silverback Therapeutics, where he was Chief Business Officer, and Edgewise Therapeutics, where he was Chief Operating Officer. Before OrbiMed, Dr. Edris was a management consultant at Bain & Company, where he collaborated with global pharmaceutical and biotechnology companies on a range of strategic and operational projects. Dr. Edris received his Ph.D. in Genetics from Stanford University, where he was an NSF research fellow.

    Dr. Smith joined SpringWorks at the company's inception in 2017 and was a founding member of the management team. During her tenure, she has designed and executed SpringWorks' clinical development programs, including advancing the company's lead programs into potentially registrational trials, led the regulatory strategy for the company's lead assets, and built a leading clinical development, manufacturing, and quality organization. Prior to joining SpringWorks, Dr. Smith was the Executive Vice President of Gene Therapy at Bamboo Therapeutics, a wholly owned subsidiary of Pfizer, where she led several key gene transfer programs for rare genetic diseases. Prior to joining Bamboo, Dr. Smith was the Vice President of Product Development at United Therapeutics, where she was responsible for development programs in oncology, regenerative medicine and virology. She also held other positions of increasing responsibility at several biotech companies throughout her 20+ year career. Dr. Smith holds a Ph.D. in Microbiology/Immunology from the University of New Hampshire and received her postdoctoral training at Emory University under a NIH postdoctorate research fellowship.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    Contact

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com 

     



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  26. STAMFORD, Conn., Nov. 24, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will participate in a Fireside Chat at the 3rd Annual Evercore ISI HealthCONx Conference, taking place virtually on Wednesday, December 2, 2020 at 12:35 p.m. ET.

    A video webcast will be available on the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the conference.

    About SpringWorks

    STAMFORD, Conn., Nov. 24, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will participate in a Fireside Chat at the 3rd Annual Evercore ISI HealthCONx Conference, taking place virtually on Wednesday, December 2, 2020 at 12:35 p.m. ET.

    A video webcast will be available on the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for a limited time following the conference.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Contact:

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com



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  27. - Enrollment Complete in the Phase 3 DeFi Trial of Nirogacestat in Adult Patients with Desmoid Tumors -

    - Clinical Collaborations Signed with Janssen, Pfizer and Precision Biosciences to Evaluate Nirogacestat in Combination with BCMA-Directed Therapies Across Modalities for the Treatment of Patients with Relapsed or Refractory Multiple Myeloma -

    - Entered into Sponsored Research Agreement with Fred Hutchinson Cancer Research Center to Further Evaluate Nirogacestat as a BCMA Potentiator in Multiple Myeloma -

    - Completed an Asset Purchase and Exclusive License Agreement of Global Rights to FAAH Inhibitor PF-04457845 to Jazz Pharmaceuticals, Receiving $35 Million Upfront Payment and Potential Future Milestone Payments

    - Enrollment Complete in the Phase 3 DeFi Trial of Nirogacestat in Adult Patients with Desmoid Tumors -

    - Clinical Collaborations Signed with Janssen, Pfizer and Precision Biosciences to Evaluate Nirogacestat in Combination with BCMA-Directed Therapies Across Modalities for the Treatment of Patients with Relapsed or Refractory Multiple Myeloma -

    - Entered into Sponsored Research Agreement with Fred Hutchinson Cancer Research Center to Further Evaluate Nirogacestat as a BCMA Potentiator in Multiple Myeloma -

    - Completed an Asset Purchase and Exclusive License Agreement of Global Rights to FAAH Inhibitor PF-04457845 to Jazz Pharmaceuticals, Receiving $35 Million Upfront Payment and Potential Future Milestone Payments of Up to $375 Million -

    - Raised $269.5 Million in Net Proceeds Through Follow-On Public Offering -

    STAMFORD, Conn., Nov. 12, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported third quarter financial results for the period ended September 30, 2020 and provided an update on recent company developments.

    "We are very pleased with our achievements in the third quarter of 2020 across our clinical development, business development and corporate initiatives. We completed enrollment in the Phase 3 DeFi trial, advanced our strategy to develop nirogacestat as a cornerstone of BCMA combination therapy for patients with multiple myeloma by signing additional collaborations with industry and academic leaders, and continued to progress our trials in biomarker defined metastatic solid tumors," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our ten clinical development programs are progressing as planned and we look forward to providing further updates as we advance our pipeline and continue to execute on our strategy to build a leading targeted oncology company."

    Recent Business Highlights and Upcoming Milestones

    Late-Stage Rare Oncology

    • Completed enrollment in the Phase 3 DeFi trial evaluating nirogacestat in adult patients with progressing desmoid tumors. SpringWorks expects to report topline data from the DeFi study in the second or third quarter of 2021.
    • Recruiting patients in a Phase 2 study sponsored by the Children's Oncology Group evaluating nirogacestat in pediatric patients with desmoid tumors.
    • Highlighted a publication by investigators at the University of Minnesota and the Dana-Farber Cancer Institute in the Pediatric Cancer & Blood journal reporting data from four pediatric and young adult desmoid tumor patients who received nirogacestat under the SpringWorks Expanded Access Program. In these four patients, the investigators reported one complete response, two partial responses, and one stable disease, with no grade 3 or 4 adverse events.
    • Enrollment is ongoing in the Phase 2b ReNeu trial evaluating mirdametinib in pediatric and adult patients with NF1-associated plexiform neurofibromas. SpringWorks expects to provide an update on the ReNeu trial in the fourth quarter of 2020 or first quarter of 2021.

    B-cell Maturation Antigen (BCMA) Combinations in Multiple Myeloma

    • Enrollment is ongoing in a Phase 1b trial evaluating nirogacestat in combination with BLENREP (belantamab mafodotin-blmf), GSK's anti-B-cell maturation antigen (BCMA) antibody-drug conjugate, in patients with relapsed or refractory multiple myeloma.
    • Entered into a clinical collaboration with Janssen Biotech, Inc. (Janssen) to evaluate nirogacestat in combination with Janssen's BCMA CD3 bispecific antibody, teclistamab, in patients with relapsed or refractory multiple myeloma. A Phase 1 study is expected to commence by early 2021, pending discussions with regulators.
    • Entered into a clinical collaboration with Pfizer Inc. (Pfizer) to evaluate nirogacestat in combination with Pfizer's BCMA CD3 bispecific antibody, PF‐06863135, in patients with relapsed or refractory multiple myeloma. A Phase 1b/2 study is expected to commence in the first half of 2021.
    • An IND application is expected to be filed by the end of 2020 to evaluate nirogacestat in combination with Allogene's BCMA AlloCAR T therapy, ALLO-715, in patients with relapsed or refractory multiple myeloma.
    • Entered into a clinical collaboration with Precision BioSciences, Inc. (Precision) to evaluate nirogacestat in combination with Precision's allogeneic CAR T candidate targeting BCMA, PBCAR269A, in patients with relapsed or refractory multiple myeloma. A Phase 1/2a clinical study is expected to commence in the first half of 2021, pending discussions with regulators.
    • Entered into a sponsored research agreement with Fred Hutchinson Cancer Research Center (Fred Hutch) to further explore the ability of nirogacestat to modulate BCMA and potentiate BCMA-targeting therapies in a variety of preclinical and patient-derived multiple myeloma models developed by researchers at Fred Hutch.

    Biomarker-Defined Metastatic Solid Tumors

    • Enrollment is ongoing in a Phase 1b/2 trial evaluating mirdametinib with BeiGene's RAF dimer inhibitor, lifirafenib, in patients with RAS/RAF mutant and other MAPK pathway aberrant solid tumors. SpringWorks and BeiGene expect to report initial clinical data in 2021.
    • Enrollment is ongoing in a Phase 1 trial of BGB-3245 in patients with RAF mutant solid tumors. BGB-3245 is a selective RAF dimer inhibitor being developed by MapKure, LLC, an entity that is jointly owned by SpringWorks and BeiGene, Ltd. Initial clinical data from the Phase 1 study are expected in 2021.

    General Corporate

    • In October 2020, SpringWorks completed a follow-on public offering that raised $269.5 million in net proceeds, which included the underwriters' full exercise of their option to purchase additional shares of common stock.
    • SpringWorks and Jazz Pharmaceuticals entered into an asset purchase and exclusive license agreement under which Jazz acquired SpringWorks' fatty acid amide hydrolase (FAAH) inhibitor program, PF-04457845. Under the terms of the agreement, Jazz assumed all milestone and royalty obligations owned by SpringWorks to Pfizer under the 2017 license agreement pursuant to which SpringWorks obtained rights to this asset. In addition, Jazz made an upfront payment of $35 million to SpringWorks, with potential future milestone payments of up to $375 million payable to SpringWorks based upon the achievement of certain clinical development, regulatory and commercial milestones. SpringWorks is also entitled to receive tiered royalties on future net sales of PF-04457845.

    Third Quarter and Year to Date 2020 Financial Results

    • Research and Development (R&D) Expenses: R&D expenses were $13.9 million and $36.6 million for the third quarter and year-to-date periods, respectively, compared to $10.7 million and $30.4 million for the comparable periods of 2019, respectively. The increases in R&D expenses in 2020 were primarily attributable to growth in employee costs associated with increases in the number of R&D personnel, an increase in non-cash share-based compensation expense and increases in external costs related to drug manufacturing and clinical trial costs.
    • General and Administrative (G&A) Expenses: G&A expenses were $7.7 million and $20.9 million for the third quarter and year-to-date periods, respectively, compared to $4.6 million and $11.5 million for the comparable periods of 2019, respectively. The increases in G&A expenses in 2020 were primarily attributable to growth in employee costs associated with increases in the number of G&A personnel supporting the growth of the organization, and an increase in non-cash share-based compensation expenses, as well as increases in expenses related to the expansion of business activities.
    • Net Loss Attributable to Common Stockholders: SpringWorks reported net losses of $21.7 million, or $0.51 loss per share, and $56.8 million, or $1.35 loss per share, for the third quarter and year-to-date periods ended September 30, 2020, respectively. This compares to net losses of $16.8 million, or $1.77 loss per share, and $34.4 million, or $9.24 loss per share, for the comparable periods of 2019, respectively.
    • Cash Position: Cash, cash equivalents and marketable securities were $276.8 million as of September 30, 2020. This does not include the net proceeds of $269.5 million from the Company's follow-on public offering completed in October 2020 or the $35 million upfront payment Jazz Pharmaceuticals made to SpringWorks in October 2020.

    COVID-19 Update

    To date, the COVID-19 pandemic has had a relatively modest impact on SpringWorks' business operations, in particular on SpringWorks' clinical trial programs, and SpringWorks is undertaking considerable efforts to mitigate the various challenges presented by this crisis. For further details and descriptions of the risks associated with the COVID-19 pandemic, please see the Risk Factors in SpringWorks' Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 12, 2020 and refer to the Forward-Looking Statements section in this press release.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    SpringWorks uses its website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the Company's website in the ‘Investors & Media' section. Accordingly, investors should monitor such portions of the SpringWorks website, in addition to following press releases, SEC filings and public conference calls and webcasts.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in SpringWorks' SEC filings. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.



    SpringWorks Therapeutics, Inc.

    Condensed Consolidated Statements of Operations

    (Unaudited)

                 
      Three Months Ended September 30,  Nine Months Ended September 30, 
    (in thousands, except share and per-share data) 2020  2019  2020  2019 
    Operating expenses:            
    Research and development $13,923  $10,745  $36,597  $30,373 
    General and administrative  7,669   4,584   20,946   11,495 
    Total operating expenses  21,592   15,329   57,543   41,868 
                 
    Loss from operations  (21,592)  (15,329)  (57,543)  (41,868)
    Other income:            
    Interest income, net  63   997   1,156   2,280 
    Total other income  63   997   1,156   2,280 
    Equity investment loss  (130)  (2,501)  (459)  (2,501)
    Net loss $(21,659) $(16,833) $(56,846) $(42,089)
                 
    Reconciliation of net loss to net loss attributable to common stockholders:            
    Net loss $(21,659) $(16,833) $(56,846) $(42,089)
    Net gain attributable to extinguishment of Series A convertible preferred and Junior Series A convertible preferred units           7,729 
    Net loss attributable to common stockholders $(21,659) $(16,833) $(56,846) $(34,360)
                 
                 
    Net loss per share, basic and diluted $(0.51) $(1.77) $(1.35) $(9.24)
    Weighted average common shares outstanding, basic and diluted  42,148,837   9,487,329   41,961,691   3,716,877 





    SpringWorks Therapeutics, Inc.

    Selected Balance Sheet Data

    (Unaudited)

     
      September 30, 2020 December 31, 2019
         
    (In thousands)  
    Cash, cash equivalents and marketable securities $276,819  $327,652 
    Working Capital (1)  266,150   319,391 
    Total Assets  286,100   334,831 
    Total liabilities  13,345   12,759 
    Accumulated deficit  (129,875)  (73,029)
    Total stockholders' (deficit) equity  272,755   322,072 
           
           

    (1) We define working capital as current assets less current liabilities.



    Contact:

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com

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  28. DUBLIN and STAMFORD, Conn., Oct. 26, 2020 /PRNewswire/ -- Jazz Pharmaceuticals plc (NASDAQ:JAZZ) and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX) ("SpringWorks") today announced that Jazz Pharmaceuticals Ireland Limited ("Jazz") and SpringWorks have entered into an asset purchase and exclusive license agreement under which Jazz has acquired SpringWorks' fatty acid amide hydrolase ("FAAH") inhibitor program including PF-04457845. Jazz will initially focus on developing PF-04457845 for the potential treatment of post-traumatic stress disorder ("PTSD") and associated symptoms. 

    "Adding to our robust and productive pipeline, the acquisition of SpringWorks' FAAH inhibitor program is a strong fit for Jazz as we continue to build out our mid-stage…

    DUBLIN and STAMFORD, Conn., Oct. 26, 2020 /PRNewswire/ -- Jazz Pharmaceuticals plc (NASDAQ:JAZZ) and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX) ("SpringWorks") today announced that Jazz Pharmaceuticals Ireland Limited ("Jazz") and SpringWorks have entered into an asset purchase and exclusive license agreement under which Jazz has acquired SpringWorks' fatty acid amide hydrolase ("FAAH") inhibitor program including PF-04457845. Jazz will initially focus on developing PF-04457845 for the potential treatment of post-traumatic stress disorder ("PTSD") and associated symptoms. 

    "Adding to our robust and productive pipeline, the acquisition of SpringWorks' FAAH inhibitor program is a strong fit for Jazz as we continue to build out our mid-stage neuroscience portfolio," said Robert Iannone, M.D., M.S.C.E., Executive Vice President, Research and Development of Jazz Pharmaceuticals. "This acquisition reinforces our unwavering commitment to developing and commercializing innovative medicines in areas of high unmet need, and, given there has been no new medication approved to treat PTSD for nearly two decades, we believe there is a significant patient need."

    "We believe that Jazz's track record of developing and commercializing neuropsychiatric medicines will help ensure that PF-04457845 reaches its full therapeutic potential on behalf of patients suffering from PTSD," said Saqib Islam, Chief Executive Officer of SpringWorks. "This transaction enables the continued development of the FAAH inhibitor program while simultaneously allowing SpringWorks to further optimize our growing targeted oncology portfolio across our three core focus areas of late-stage rare oncology, BCMA combinations in multiple myeloma and biomarker-defined metastatic solid tumors."

    PF-04457845 represents an innovative approach to treating PTSD with a novel mechanism of action that has the potential to address multiple core symptoms of the disease, including fear extinction, anxiety and disrupted sleep architecture. Clinical experience to date includes over 100 healthy volunteers and approximately 80 patients and PF-04457845 has demonstrated promising activity when studied in populations of relevance to PTSD, including fear extinction in healthy volunteers and anxiety symptoms in cannabis use disorder. Consistent with Jazz's strategy to focus on life-changing medicines for people affected by conditions for which current therapies are lacking, Jazz will initially focus on developing PF-04457845 as a potential treatment in PTSD for patients with inadequate response to currently approved therapies.

    Under the terms of the agreement, SpringWorks has assigned and exclusively licensed all assets relating to its FAAH inhibitor program to Jazz, including SpringWorks' proprietary FAAH inhibitor PF-04457845 and its license agreement with Pfizer, Inc. ("Pfizer"), under which Pfizer licensed PF-04457845 to SpringWorks ("Pfizer Agreement") in 2017. 

    In addition to assuming all milestone and royalty obligations owed by SpringWorks to Pfizer, Jazz will also make an upfront payment of $35 million to SpringWorks, with potential milestone payments to SpringWorks of up to $375 million upon the achievement of certain clinical, regulatory and commercial milestones. In addition, SpringWorks will receive incremental tiered royalties on future net sales of PF-04457845 in the mid- to high-single digit percentages.

    About Jazz Pharmaceuticals

    Jazz Pharmaceuticals plc (NASDAQ:JAZZ) is a global biopharmaceutical company dedicated to developing and commercializing life-changing medicines that transform the lives of patients with serious diseases — often with limited or no options. We have a diverse portfolio of marketed medicines and novel product candidates, from early- to late-stage development, in key therapeutic areas. Our focus is in neuroscience, including sleep medicine and movement disorders, and in oncology, including hematologic malignancies and solid tumors. We actively explore new options for patients including novel compounds, small molecule advancements, biologics and innovative delivery technologies. Jazz is headquartered in Dublin, Ireland and has employees around the globe, serving patients in more than 90 countries. For more information, please visit www.jazzpharmaceuticals.com and follow @JazzPharma on Twitter.

    About SpringWorks

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Jazz Pharmaceuticals Forward-Looking Statements 

    This press release contains forward-looking statements, including, but not limited to, Jazz Pharmaceuticals' development plans for PF-04457845 and the FAAH inhibitor program; potential clinical, regulatory, and commercial milestones under the agreement between Jazz and SpringWorks and potential future milestone, royalty or license payments by Jazz Pharmaceuticals to SpringWorks or Pfizer; the potential for PF-04457845 to transform the lives of patients with PTSD; and other statements that are not historical facts. These forward-looking statements are based on Jazz Pharmaceuticals' current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with pharmaceutical product development and clinical success thereof; the regulatory approval process; effective commercialization of any product candidates; and other risks and uncertainties affecting Jazz Pharmaceuticals, including those described from time to time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange Commission filings and reports (Commission File No. 001-33500), including the company's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 and future filings and reports by the company. Other risks and uncertainties of which Jazz Pharmaceuticals is not currently aware may also affect the company's forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof or as of the dates indicated in the forward-looking statements, even if they are subsequently made available by Jazz Pharmaceuticals on its website or otherwise. Jazz Pharmaceuticals undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made. 

    SpringWorks Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    Jazz Pharmaceuticals Contacts:



    Investors:

    Media:

    Kathee Littrell

    Jacqueline Kirby

    Vice President, Investor Relations

    Vice President, Corporate Affairs & Government Relations

    Ireland +353 1 634 7887

    Ireland +353 1 697 2141

    U.S. +1 650 496 2717

    U.S. +1 215 867 4910

    SpringWorks Contact:

    Kim Diamond

    Vice President, Communications and Investor Relations

    (203) 561-1646

    kdiamond@springworkstx.com

    Jazz Pharmaceuticals Logo (PRNewsFoto/Jazz Pharmaceuticals plc) (PRNewsFoto/Jazz Pharmaceuticals plc)

     

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  29. STAMFORD, Conn., Oct. 13, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today the closing of its underwritten public offering of 5,637,254 shares of its common stock at a public offering price of $51.00 per share, before underwriting discounts and commissions, which includes the exercise in full by the underwriters of their option to purchase up to an additional 735,294 shares of common stock in this offering. The gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, were approximately $287.5 million…

    STAMFORD, Conn., Oct. 13, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today the closing of its underwritten public offering of 5,637,254 shares of its common stock at a public offering price of $51.00 per share, before underwriting discounts and commissions, which includes the exercise in full by the underwriters of their option to purchase up to an additional 735,294 shares of common stock in this offering. The gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, were approximately $287.5 million.

    J.P. Morgan Securities LLC, Goldman Sachs & Co. LLC and Cowen and Company, LLC acted as joint book-running managers for the offering. Wedbush Securities Inc. acted as co-manager for the offering.

    An automatic shelf registration statement on Form S-3ASR (including a prospectus) relating to these securities was filed with the Securities and Exchange Commission (SEC) and has become effective. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    The offering was made only by means of a prospectus and prospectus supplement that form part of the automatic shelf registration statement. A copy of the final prospectus supplement and accompanying prospectus relating to the offering has been filed with the SEC and may be obtained from: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1115 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204, or by email at prospectus-eq_fi@jpmchase.com; Goldman Sachs & Co., Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526 or by emailing prospectus-ny@ny.email.gs.com or Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY,11717, Attn: Prospectus Department, by telephone at (833) 297-2926 or by emailing PostSaleManualRequests@broadridge.com.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' expectations with respect to the completion of the proposed public offering. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation risks and uncertainties associated with market conditions and the satisfaction of customary closing conditions related to the proposed offering, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    Contact

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com 

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  30. STAMFORD, Conn., Oct. 07, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today the pricing of an underwritten public offering of 4,901,960 shares of its common stock at a public offering price of $51.00 per share, before underwriting discounts and commissions. All of the shares in the offering are being sold by SpringWorks. In addition, SpringWorks has granted the underwriters a 30-day option to purchase up to an additional 735,294 shares of its common stock at the public offering price, less underwriting discounts and commissions. The gross proceeds from the offering…

    STAMFORD, Conn., Oct. 07, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today the pricing of an underwritten public offering of 4,901,960 shares of its common stock at a public offering price of $51.00 per share, before underwriting discounts and commissions. All of the shares in the offering are being sold by SpringWorks. In addition, SpringWorks has granted the underwriters a 30-day option to purchase up to an additional 735,294 shares of its common stock at the public offering price, less underwriting discounts and commissions. The gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, are expected to be approximately $250 million, excluding any exercise of the underwriters' option to purchase additional shares. The offering is expected to close on or about October 13, 2020, subject to the satisfaction of customary closing conditions.

    J.P. Morgan Securities LLC, Goldman Sachs & Co. LLC and Cowen and Company, LLC are acting as joint book-running managers for the offering. Wedbush Securities Inc. is acting as co-manager for the offering.

    An automatic shelf registration statement on Form S-3ASR (including a prospectus) relating to these securities has been filed with the Securities and Exchange Commission (SEC) and has become effective. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    The offering is being made only by means of a prospectus and prospectus supplement that form part of the automatic shelf registration statement. A preliminary prospectus supplement describing the terms of the offering has been filed with the SEC and forms a part of the effective registration statement. A copy of the final prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and may be obtained, when available, from: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1115 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204, or by email at prospectus-eq_fi@jpmchase.com; Goldman Sachs & Co., Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526 or by emailing prospectus-ny@ny.email.gs.com or Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY,11717, Attn: Prospectus Department, by telephone at (833) 297-2926 or by emailing PostSaleManualRequests@broadridge.com.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' expectations with respect to the respect to the completion and timing of the proposed public offering. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation risks and uncertainties associated with market conditions and the satisfaction of customary closing conditions related to the proposed offering, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    Contact

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com

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  31. STAMFORD, Conn., Oct. 06, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today that it has commenced an underwritten public offering of $150,000,000 of shares of its common stock. All of the shares in the offering will be sold by SpringWorks. In addition, SpringWorks expects to grant the underwriters a 30-day option to purchase up to an additional $22,500,000 of shares of its common stock in the offering. The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual…

    STAMFORD, Conn., Oct. 06, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced today that it has commenced an underwritten public offering of $150,000,000 of shares of its common stock. All of the shares in the offering will be sold by SpringWorks. In addition, SpringWorks expects to grant the underwriters a 30-day option to purchase up to an additional $22,500,000 of shares of its common stock in the offering. The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    J.P. Morgan Securities LLC, Goldman Sachs & Co. LLC and Cowen and Company, LLC are acting as joint book-running managers for the offering. Wedbush Securities Inc. is acting as co-manager for the offering.

    An automatic shelf registration statement on Form S-3ASR relating to these securities has been filed with the Securities and Exchange Commission (SEC) and has become effective. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    The offering will be made only by means of a prospectus and prospectus supplement that form part of the automatic shelf registration statement. A copy of the preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and may be obtained, when available, from: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1115 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204, or by email at prospectus-eq_fi@jpmchase.com; Goldman Sachs & Co., Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526 or by emailing prospectus-ny@ny.email.gs.com or Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY,11717, Attn: Prospectus Department, by telephone at (833) 297-2926 or by emailing PostSaleManualRequests@broadridge.com.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' expectations with respect to the completion, timing and size of the proposed public offering and granting the underwriters a 30-day option to purchase additional shares. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation risks and uncertainties associated with market conditions and the satisfaction of customary closing conditions related to the proposed offering, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    Contact

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com 

    Primary Logo

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  32. STAMFORD, Conn., Oct. 05, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the company has entered into a clinical trial collaboration agreement with Pfizer Inc. (NYSE:PFE) to evaluate SpringWorks Therapeutics' investigational gamma secretase inhibitor (GSI), nirogacestat, in combination with Pfizer's anti-B-cell maturation antigen (BCMA) CD3 bispecific antibody, PF‐06863135, in patients with relapsed or refractory multiple myeloma.

    Gamma secretase inhibition prevents the cleavage and shedding of BCMA from the surface of myeloma cells. In preclinical models…

    STAMFORD, Conn., Oct. 05, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the company has entered into a clinical trial collaboration agreement with Pfizer Inc. (NYSE:PFE) to evaluate SpringWorks Therapeutics' investigational gamma secretase inhibitor (GSI), nirogacestat, in combination with Pfizer's anti-B-cell maturation antigen (BCMA) CD3 bispecific antibody, PF‐06863135, in patients with relapsed or refractory multiple myeloma.

    Gamma secretase inhibition prevents the cleavage and shedding of BCMA from the surface of myeloma cells. In preclinical models, nirogacestat has been shown to increase the cell surface density of BCMA and reduce levels of soluble BCMA, thereby enhancing the activity of BCMA-targeted therapies, including CD3 bispecific antibodies.

    "This collaboration is another important step in continuing to advance our goal of developing nirogacestat as a best-in-class BCMA potentiator, and we are pleased to work with Pfizer to study nirogacestat in combination with PF‐06863135, which has recently demonstrated promising monotherapy clinical data," said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics. "We now have five collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities. We look forward to generating clinical data with our collaborators to further evaluate the ability of nirogacestat to improve outcomes for patients with multiple myeloma."

    Under the terms of the agreement, Pfizer will sponsor and conduct the Phase 1b/2 study to evaluate the safety, tolerability and preliminary efficacy of the combination, and will assume all costs associated with the study, other than expenses related to the manufacturing of nirogacestat and certain expenses related to intellectual property rights. Pfizer and SpringWorks Therapeutics will also form a joint development committee to manage the clinical study, which is expected to commence in the first half of 2021.

    "Entering into this clinical collaboration is a proud milestone in our strong relationship with SpringWorks," said Chris Boshoff, MD, PhD, Chief Development Officer for Pfizer Oncology at Pfizer. "We believe that studying nirogacestat in combination with PF-06863135 could hold significant therapeutic promise for patients with relapsed or refractory multiple myeloma, and we look forward to working together to advance this important area of research."

    In addition to its ongoing clinical collaborations with BCMA-directed therapies, SpringWorks is also currently conducting a global Phase 3, double-blind, randomized, placebo-controlled clinical trial (the DeFi Trial) to evaluate nirogacestat in adults with progressing desmoid tumors.

    About Nirogacestat

    Nirogacestat is an investigational, oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has five collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities, including with an antibody-drug conjugate, two CAR T cell therapies and two bispecific antibodies. In addition, SpringWorks and Fred Hutchinson Cancer Research Center have entered into a sponsored research agreement to further characterize the ability of nirogacestat to modulate BCMA and potentiate BCMA directed therapies using a variety of preclinical and patient-derived multiple myeloma models developed by researchers at Fred Hutch.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors (June 2018) and from the European Commission for the treatment of soft tissue sarcoma (September 2019). The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis (November 2018 and August 2019).

    About PF06863135

    PF‐06863135 is an anti-B-cell maturation antigen (BCMA) CD3 bispecific antibody being investigated in a Phase 1 clinical study to treat relapsed or refractory multiple myeloma. This bispecific antibody can be administered subcutaneously and has been optimized for binding affinity to both BCMA and CD3, enabling more potent T-cell-mediated tumor cell toxicity.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    SpringWorks Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for initiation or completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    SpringWorks Media/Investor Contact:

    Kim Diamond

    203-561-1646

    kdiamond@springworkstx.com

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  33. STAMFORD, Conn., Sept. 22, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will participate in a Fireside Chat at the H.C. Wainwright Precision Oncology Conference on Thursday, September 24, 2020 at 9:30 a.m. Eastern Standard Time.

    A video webcast will be available on the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for approximately 30 days following the conference.

    About SpringWorks

    STAMFORD, Conn., Sept. 22, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will participate in a Fireside Chat at the H.C. Wainwright Precision Oncology Conference on Thursday, September 24, 2020 at 9:30 a.m. Eastern Standard Time.

    A video webcast will be available on the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for approximately 30 days following the conference.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Contact:

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com

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  34. DURHAM, N.C. and STAMFORD, Conn., Sept. 21, 2020 (GLOBE NEWSWIRE) -- Precision BioSciences, Inc. (NASDAQ:DTIL), a clinical stage biotechnology developing allogeneic CAR T and in vivo gene correction therapies with its ARCUS® genome editing platform, and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced they have entered into a clinical trial collaboration agreement. Per the agreement, PBCAR269A, Precision BioSciences' wholly-owned investigational allogeneic chimeric antigen receptor (CAR) T cell therapy candidate targeting B-cell maturation antigen (BCMA), will be evaluated in combination with…

    DURHAM, N.C. and STAMFORD, Conn., Sept. 21, 2020 (GLOBE NEWSWIRE) -- Precision BioSciences, Inc. (NASDAQ:DTIL), a clinical stage biotechnology developing allogeneic CAR T and in vivo gene correction therapies with its ARCUS® genome editing platform, and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced they have entered into a clinical trial collaboration agreement. Per the agreement, PBCAR269A, Precision BioSciences' wholly-owned investigational allogeneic chimeric antigen receptor (CAR) T cell therapy candidate targeting B-cell maturation antigen (BCMA), will be evaluated in combination with nirogacestat, SpringWorks' investigational gamma secretase inhibitor (GSI), in patients with relapsed or refractory multiple myeloma.

    "Based on recent clinical data using GSIs in combination with BCMA-targeted therapies, it is exciting to combine these agents in patients who have a need for better therapies," said Nina Shah, M.D., study Principal Investigator and Associate Professor in the Department of Medicine at the University of California, San Francisco. "I look forward to the data generated from this investigational study to see if PBCAR269A plus nirogacestat improves clinical outcomes in patients with multiple myeloma."

    Gamma secretase inhibition has been shown preclinically to enhance the activity of BCMA-targeted therapies by preventing the cleavage and shedding of BCMA from the surface of myeloma cells, which increases the cell surface density of BCMA and reduces levels of soluble BCMA. Via this mechanism, nirogacestat may enhance the activity of BCMA-targeted therapies.1 Emerging clinical data also suggest that a GSI may increase antitumor efficacy of BCMA-targeted autologous CAR T therapy in patients with relapsed or refractory multiple myeloma.2,3

    "In June, we initiated our Phase 1/2a clinical trial of PBCAR269A, which targets BCMA for the treatment of relapsed or refractory multiple myeloma and has demonstrated anti-tumor activity in preclinical disease models," said Chris Heery, M.D., Chief Medical Officer at Precision BioSciences. "Preclinical data from our own program as well as others have suggested the importance of gamma secretase inhibition to unlock the full potential of BCMA targeted therapies. We look forward to further evaluating this in the clinic."

    Under the terms of the agreement, Precision BioSciences will assume all development costs of the expanded Phase 1/2a study of PBCAR269A to include nirogacestat and evaluate the safety and preliminary clinical activity of the combination therapy. Precision BioSciences and SpringWorks will form a joint development committee to oversee the clinical study, which is expected to commence in the first half of 2021, pending discussions with regulators.

    "Patients with multiple myeloma are in great need of treatment advances," said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics. "We continue to believe that nirogacestat has the potential to become a cornerstone of BCMA combination therapy for these patients and are pleased to work with Precision BioSciences and their leading group of scientific advisors and clinical investigators to evaluate the combination of our gamma secretase inhibitor with their ‘off-the-shelf' CAR T therapy."

    About PBCAR269A

    PBCAR269A is an allogeneic BCMA-targeted CAR T cell therapy candidate being evaluated for the safety and preliminary clinical activity in a Phase 1/2a multicenter, nonrandomized, open-label, parallel assignment, single-dose, dose-escalation, and dose-expansion study of adults with relapsed or refractory multiple myeloma. The starting dose of PBCAR269A is 6 x 105 CAR T cells/kg body weight with subsequent cohorts receiving escalating doses to a maximum dose of 6 x 106 CAR T cells/kg body weight.

    PBCAR269A is the company's third CAR T candidate to advance to the clinic and is part of a pipeline of cell-phenotype optimized allogeneic CAR T therapies derived from healthy donors and then modified via a simultaneous TCR knock-out and CAR T knock-in step with the Company's proprietary ARCUS® genome editing technology. Precision BioSciences optimizes its CAR T therapy candidates for immune cell expansion in the body by maintaining a high proportion of naïve and central memory CAR T cells.

    The U.S. Food and Drug Administration (FDA) recently granted Fast Track Designation to PBCAR269A for the treatment of relapsed or refractory multiple myeloma for which the FDA previously granted Orphan Drug Designation. The PBCAR269A clinical trial will be conducted at multiple U.S. sites. For more information, visit www.clinicaltrials.gov, study identifier number NCT04171843.

    About Nirogacestat

    Nirogacestat is an investigational, oral, selective, small molecule, gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has four collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities, including with an antibody-drug conjugate, two CAR T cell therapies and a bispecific antibody. In addition, SpringWorks and Fred Hutchinson Cancer Research Center have entered into a sponsored research agreement to further characterize the ability of nirogacestat to modulate BCMA and potentiate BCMA directed therapies using a variety of preclinical and patient-derived multiple myeloma models developed by researchers at Fred Hutch.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors (June 2018) and from the European Commission for the treatment of soft tissue sarcoma (September 2019). The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis (November 2018 and August 2019).

    About Precision BioSciences, Inc.

    Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Company's pipeline consists of multiple "off-the-shelf" CAR T immunotherapy clinical candidates and several in vivo gene correction therapy candidates to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences please visit www.precisionbiosciences.com.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Precision Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the Company's timing of clinical trials and results therefrom involving PBCAR269A and the expected benefits of producing clinical trial material at the Company's in-house manufacturing facility. In some cases, you can identify forward-looking statements by terms such as "anticipate," "believe," "could," "expect," "should," "plan," "intend," "estimate," "target," "mission," "may," "will," "would," "should," "could," "target," "project," "predict," "contemplate," "potential," or the negative thereof and similar words and expressions.

    Forward-looking statements are based on management's current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical or greenhouse studies and clinical or field trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, biotechnology and agricultural biotechnology fields; our or our collaborators' ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators' development of product candidates; our or our collaborators' ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; our or our collaborators' ability to advance product candidates into, and successfully design, implement and complete, clinical or field trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; our ability to achieve our anticipated operating efficiencies at our manufacturing facility; delays or difficulties in our and our collaborators' ability to enroll patients; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data privacy and security regulations and our compliance therewith; the rate and degree of market acceptance of any of our product candidates; the success of our existing collaboration agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate key scientific and management personnel; market and economic conditions; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events effects of the outbreak of COVID-19, or any pandemic, epidemic or outbreak of an infectious disease; insurance expenses and exposure to uninsured liabilities; and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2020, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC's website at www.sec.gov and the Investors & Media page of our website at investor.precisionbiosciences.com.

    All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

    SpringWorks Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    Contact:

    Maurissa Messier

    Senior Director, Corporate Communications

    Maurissa.messier@precisionbiosciences.com

    Kim Diamond

    Vice President, Communications and Investor Relations

    (203) 561-1646

    kdiamond@springworkstx.com

    References

    1 Eastman S, Shelton C, Gupta I, Krueger J, Blackwell C, Bojczuk. Synergistic activity of belantamab mafodotin (anti-BCMA immuno-conjugate) with PF-03084014 (gamma-secretase inhibitor) in Bcma-expressing cancer cell lines. Blood. 2019;134(supplement_1):4401. doi.org/10.1182/blood-2019-123705.



    2
    Cowan AJ, Pont M, Sather BD, et al. Efficacy and safety of fully human Bcma CAR T cells in combination with a gamma secretase inhibitor to increase Bcma surface expression in patients with relapsed or refractory multiple myeloma. Blood. 2019;134(supplement_1):204. doi.org/10.1182/blood-2019-129405.



    3
    Pont MJ, Hill T, Cole GO, et al. γ-Secretase inhibition increases efficacy of BCMA-specific chimeric antigen receptor T cells in multiple myeloma. Blood. 2019;134(19):1585-1597. doi:10.1182/blood.2019000050

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  35. STAMFORD, Conn., Sept. 18, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that it has entered into a sponsored research agreement with Fred Hutchinson Cancer Research Center (Fred Hutch) to further explore the ability of SpringWorks' investigational gamma secretase inhibitor (GSI), nirogacestat, to modulate B-cell maturation antigen (BCMA) and potentiate BCMA-targeting therapies, including radioimmunotherapies, in a variety of preclinical and patient-derived multiple myeloma models developed by researchers at Fred Hutch. Damian Green, M.D., Associate Member of the…

    STAMFORD, Conn., Sept. 18, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that it has entered into a sponsored research agreement with Fred Hutchinson Cancer Research Center (Fred Hutch) to further explore the ability of SpringWorks' investigational gamma secretase inhibitor (GSI), nirogacestat, to modulate B-cell maturation antigen (BCMA) and potentiate BCMA-targeting therapies, including radioimmunotherapies, in a variety of preclinical and patient-derived multiple myeloma models developed by researchers at Fred Hutch. Damian Green, M.D., Associate Member of the Clinical Research Division at Fred Hutch and Associate Professor at the University of Washington School of Medicine, will serve as the Principal Investigator for this research.

    Gamma secretase inhibition disrupts the cleavage and shedding of BCMA from the surface of myeloma cells. In preclinical models, the team at Fred Hutch was the first to show that gamma secretase inhibition can increase BCMA surface density on malignant plasma cells and concurrently decrease soluble BCMA to enhance the activity of BCMA-targeted therapies.1,2 Nirogacestat has demonstrated similar results.3 In addition, emerging and published clinical data from Fred Hutch suggest that a GSI may increase antitumor efficacy of BCMA-targeted autologous CAR T cell therapy in patients with relapsed and refractory multiple myeloma.1,4

    "We have already begun to see multiple myeloma patients appear to benefit from the ability of a GSI to potentiate BCMA-directed therapies," said Dr. Green. "Given the clinical experience with nirogacestat and the commitment that SpringWorks has made to combining their GSI with BCMA-directed therapies, I look forward to expanding on the important work that the Fred Hutch has already done in this area and collaborating with SpringWorks to explore this therapeutic hypothesis further."

    "Fred Hutch scientists have been leaders in developing novel treatment approaches for multiple myeloma and have presented promising preclinical and clinical data combining GSIs with BCMA-directed therapies," said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics. "We believe that nirogacestat has the potential to become a cornerstone of BCMA combination therapies across modalities and are delighted to be working with Dr. Green to build upon the encouraging results demonstrated to date. Our ultimate goal is to develop nirogacestat in such a way as to improve clinical outcomes for patients with multiple myeloma."

    About Nirogacestat

    Nirogacestat is an investigational, oral, selective, small molecule, gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has three collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities, including with an antibody-drug conjugate, a CAR T cell therapy and a bispecific antibody. In addition, SpringWorks and Fred Hutchinson Cancer Research Center have entered into a sponsored research agreement to further characterize the ability of nirogacestat to modulate BCMA and potentiate BCMA directed therapies using a variety of preclinical and patient-derived multiple myeloma models developed by researchers at Fred Hutch.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors (June 2018) and from the European Commission for the treatment of soft tissue sarcoma (September 2019). The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis (November 2018 and August 2019).

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    SpringWorks Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    SpringWorks Media/Investor Contact

    Kim Diamond

    203-561-1646

    kdiamond@springworkstx.com

    References

    1 Pont MJ, Hill T, Cole GO, et al. γ-Secretase inhibition increases efficacy of BCMA-specific chimeric antigen receptor T cells in multiple myeloma. Blood. 2019;134(19):1585-1597. doi:10.1182/blood.2019000050.

    2 Hill T, Pont MJ, Abbott J, et al. Gamma secretase inhibition increases recognition of multiple myeloma by BCMA-specific chimeric antigen receptor modified T cells, Presented at: Annual Meeting of the Society for Immunotherapy of Cancer (SITC). November 8-12, 2017. National Harbor, MD.

    3 Eastman S, Shelton C, Gupta I, Krueger J, Blackwell C, Bojczuk. Synergistic activity of belantamab mafodotin (anti-BCMA immuno-conjugate) with PF-03084014 (gamma-secretase inhibitor) in Bcma-expressing cancer cell lines. Blood. 2019;134(supplement_1):4401. doi.org/10.1182/blood-2019-123705.

    4 Cowen AJ, Pont M, Sather BD, et al. Efficacy and safety of fully human Bcma CAR T cells in combination with a gamma secretase inhibitor to increase Bcma surface expression in patients with relapsed or refractory multiple myeloma. Blood. 2019;134(supplement_1):204. doi.org/10.1182/blood-2019-129405.

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  36. - Expansion of Nirogacestat Clinical Program to Include Pediatric Patients Supported by Recent Publication of Nirogacestat Case Series in Four Children/Young Adults with Desmoid Tumors Treated under the SpringWorks Expanded Access Program -

    - Investigators Reported Clinical Responses in All Four Patients Treated with Nirogacestat, including One Complete Response, Two Partial Responses, and One Stable Disease, with No Grade 3 or 4 Adverse Events Reported - 

    STAMFORD, Conn., Sept. 16, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced a collaboration with the Children's…

    - Expansion of Nirogacestat Clinical Program to Include Pediatric Patients Supported by Recent Publication of Nirogacestat Case Series in Four Children/Young Adults with Desmoid Tumors Treated under the SpringWorks Expanded Access Program -

    - Investigators Reported Clinical Responses in All Four Patients Treated with Nirogacestat, including One Complete Response, Two Partial Responses, and One Stable Disease, with No Grade 3 or 4 Adverse Events Reported - 

    STAMFORD, Conn., Sept. 16, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced a collaboration with the Children's Oncology Group (COG) to evaluate nirogacestat, an investigational gamma secretase inhibitor, in a Phase 2 clinical trial for the treatment of children and adolescents with progressive, surgically unresectable desmoid tumors. The study, which will be sponsored by COG, a National Cancer Institute-supported clinical trials group, and supported by SpringWorks, will aim to enroll approximately 30 patients with a primary endpoint of 2-year progression-free survival. Fariba Navid, M.D., Associate Professor of Clinical Pediatrics, Keck School of Medicine of USC at the Children's Hospital Los Angeles' Cancer and Blood Disease Institute, will serve as the Study Chair for the trial, which is expected to begin recruiting patients this month.

    "For many children diagnosed with a desmoid tumor, surgery is not an option due to high tumor recurrence rates and the potential for other morbidities and disabilities that can occur following surgery," said Dr. Navid. "These patients often require medical treatment and we look forward to working with SpringWorks to study nirogacestat in a pediatric population."

    In addition, SpringWorks also reported a recent publication by investigators at the University of Minnesota and the Dana-Farber Cancer Institute in the Pediatric Cancer & Blood journal reporting data from four pediatric and young adult desmoid tumor patients who received nirogacestat under the SpringWorks Expanded Access Program. Following treatment with nirogacestat, the investigators reported a clinical response in all four patients. Specifically, after a median of 13.5 months on treatment, three patients experienced a durable benefit (one complete response, one partial response, and one stable disease) and one patient had an initial partial response followed by disease progression. These four patients had received between one and eight prior lines of therapy, including surgery, before being treated with nirogacestat. Furthermore, the investigators reported that no patient experienced a grade 3 or 4 adverse event while receiving nirogacestat.1

    "Desmoid tumors can cause significant morbidities, including severe pain, internal bleeding, incapacitating loss of range of motion, and, in rare cases, death, and there are currently no approved therapies for patients suffering from this rare tumor," said Saqib Islam, Chief Executive Officer of SpringWorks. "We are encouraged by the case reports published on the pediatric patients treated with nirogacestat under our Expanded Access Program and are pleased to be collaborating with COG to further explore the clinical benefit of nirogacestat in pediatric and adolescent patients with desmoid tumors. In addition to our ongoing Phase 3 DeFi trial evaluating nirogacestat in adults with progressing desmoid tumors, which is now fully enrolled, this new Phase 2 trial provides an opportunity to further evaluate the potential benefit of nirogacestat for desmoid tumors."

    About the COG-Sponsored Phase 2 Study

    The open-label, single arm Phase 2 study will evaluate the safety, pharmacokinetic and efficacy of nirogacestat in children and adolescents with progressive, surgically unresectable desmoid tumors. The study plans to enroll approximately 30 participants. Patients will receive nirogacestat at a dose of 90 mg/m2 twice daily. The study is expected to begin recruiting patients in September 2020.

    The primary objective of the study is to estimate the 2-year progression-free survival rate in patients and to characterize the safety, tolerability, and pharmacokinetics of nirogacestat in children and adolescents. A secondary objective is to determine the objective tumor response rate of nirogacestat in this patient population. 

    More information about the study is available at www.clinicaltrials.gov under the identifier NCT04195399.

    About Desmoid Tumors

    Desmoid tumors, also referred to as aggressive fibromatosis or desmoid-type fibromatosis, are rare and often debilitating and disfiguring soft tissue tumors characterized by a growth pattern that can invade surrounding healthy tissues, including joints, muscle and viscera. While they can arise in any part of the body, the most common sites are the upper and lower extremities, abdominal wall, thoracic areas, and the head and neck. The severity of a desmoid tumor can vary based on the location of the tumor and the aggressiveness of its growth pattern. Desmoid tumors can cause significant morbidities, including severe pain, internal bleeding, incapacitating loss of range of motion, and, in rare cases, death.2

    Desmoid tumors typically occur in patients between the ages of 15 to 60 years, and are more commonly diagnosed in young adults between 30-40 years of age, with a two-to-three times higher prevalence in females.2,3 It is estimated that there are 1,000 to 1,500 new cases diagnosed per year in the United States.4,5

    Historically, desmoid tumors were treated with surgical resection, but this approach has become less favored due to a high recurrence rate after surgery.6 There are currently no FDA-approved therapies for the treatment of desmoid tumors.

    About Nirogacestat

    Nirogacestat is an investigational, oral, selective, small molecule, gamma secretase inhibitor in Phase 3 clinical development for adult patients with progressing desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors (June 2018) and from the European Commission for the treatment of soft tissue sarcoma (September 2019). The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis (November 2018 and August 2019).

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the soluble BCMA from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA, which may serve as a decoy receptor for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has three collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities, including with an antibody-drug conjugate, a CAR T cell therapy and a bispecific antibody.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    About the Children's Oncology Group (COG)

    COG (childrensoncologygroup.org), a member of the NCI National Clinical Trials Network (NCTN), is the world's largest organization devoted exclusively to childhood and adolescent cancer research. COG unites over 10,000 experts in childhood cancer at more than 200 leading children's hospitals, universities, and cancer centers across North America, Australia, and New Zealand in the fight against childhood cancer. Today, more than 90% of the 14,000 children and adolescents diagnosed with cancer each year in the United States are cared for at COG member institutions. Research performed by COG institutions over the past 50 years has transformed childhood cancer from a virtually incurable disease to one with a combined 5-year survival rate of 80%. COG's mission is to improve the cure rate and outcomes for all children with cancer.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    Contact

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com

    References

    1 Takahashi, T., Presner, J. R., Robson, C. D., Janeway, K. A., & Weigel, B. J. Safety and Efficacy of Gamma Secretase Inhibitor Nirogacestat (PF_03084014) in Desmoid Tumor: Report of Four Pediatric/Young Adult Cases. Pediatric Blood & Cancer. 2020 Jul 22. doi:10.1002/pbc.28636.

    2 Gounder, M. M., Thomas, D. M., & Tap, W. D. Locally Aggressive Connective Tissue Tumors. Journal of Clinical Oncology. 2017; 36(2): 202-209. doi:10.1200/JCO.2017.75.8482.

    3 Skubitz, K. M. Biology and Treatment of Aggressive Fibromatosis or Desmoid Tumor. Mayo Clinic Proceedings. 2017; 92(6): 947–964. doi:10.1016/j.mayocp.2017.02.012.

    4 Reitamo, J. J., Häyry, P; Nykyri, E, & Saxén, E. The Desmoid Tumor. I.: Incidence, Sex-, Age- and Anatomical Distribution in the Finnish Population. American Journal of Clinical Pathology. 1982; 77(6): 665–673. doi:10.1093/ajcp/77.6.665.

    5 van Broekhoven, D. L., Grünhagen, D. J., den Bakker, M. A., van Dalen, T., & Verhoef, C. Time Trends in the Incidence and Treatment of Extra-Abdominal and Abdominal Aggressive Fibromatosis: A Population-Based Study. Annals of Surgical Oncology. 2015; 22(9): 2817–2823. doi:10.1245/s10434-015-4632-y.

    6 Scaramussa, F.S. & Castro, U. B. Desmoid Tumor in Hand: A Case Report. SM Journal of Orthopedics. 2016; 2(3): 1–3. doi:10.36876/smjo.1036.

     

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  37. STAMFORD, Conn., Sept. 14, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the company has entered into a clinical collaboration and supply agreement with Janssen Biotech, Inc. (Janssen) to evaluate SpringWorks Therapeutics' investigational gamma secretase inhibitor (GSI), nirogacestat, in combination with Janssen's bispecific antibody targeting B-cell maturation antigen (BCMA) and CD3, teclistamab, in patients with relapsed or refractory multiple myeloma.

    Gamma secretase inhibition prevents the cleavage and shedding of BCMA from the surface of myeloma cells…

    STAMFORD, Conn., Sept. 14, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the company has entered into a clinical collaboration and supply agreement with Janssen Biotech, Inc. (Janssen) to evaluate SpringWorks Therapeutics' investigational gamma secretase inhibitor (GSI), nirogacestat, in combination with Janssen's bispecific antibody targeting B-cell maturation antigen (BCMA) and CD3, teclistamab, in patients with relapsed or refractory multiple myeloma.

    Gamma secretase inhibition prevents the cleavage and shedding of BCMA from the surface of myeloma cells. In preclinical models, nirogacestat has been shown to increase the cell surface density of BCMA and reduce levels of soluble BCMA, which may enhance the activity of BCMA-targeted therapies, including CD3 bispecific antibodies.

    "We are delighted to enter into this collaboration with Janssen to study nirogacestat in combination with teclistamab," said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics. "We now have three collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities, including with an antibody-drug conjugate, an allogeneic CAR-T cell therapy and now a bispecific antibody. This collaboration is an important step in continuing to advance our goal of developing nirogacestat as a best-in-class BCMA potentiator."

    Under the terms of the agreement, Janssen will sponsor and conduct the Phase 1 study to evaluate the safety, tolerability and preliminary efficacy of the combination, and will assume all costs associated with the study, other than expenses related to the manufacturing of nirogacestat. SpringWorks Therapeutics will also form a joint oversight committee with Janssen. Pending discussions with regulators, the study is anticipated to commence by early 2021.

    In addition to its ongoing clinical collaborations with BCMA-directed therapies, SpringWorks is also currently conducting a global Phase 3, double-blind, randomized, placebo-controlled clinical trial (the DeFi Trial) to evaluate nirogacestat in adults with progressing desmoid tumors.

    About Nirogacestat

    Nirogacestat is an investigational, oral, selective, small molecule, gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is pursuing a combination therapy approach to evaluate nirogacestat as a BCMA potentiator across modalities by collaborating with industry leaders. In addition to today's announcement, SpringWorks has entered into two other clinical collaborations to evaluate nirogacestat in combination with GlaxoSmithKline's BCMA antibody-drug conjugate BLENREP (belantamab mafodotin-blmf) and with Allogene's allogeneic BCMA CAR-T cell therapy ALLO-715.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors (June 2018) and from the European Commission for the treatment of soft tissue sarcoma (September 2019). The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis (November 2018 and August 2019).

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com, and follow @SpringWorksTx on Twitter and LinkedIn.

    SpringWorks Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    SpringWorks Media/Investor Contact:

    Kim Diamond

    203-561-1646

    kdiamond@springworkstx.com

     

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  38. – Enrollment Complete in the Phase 3 DeFi Trial of Nirogacestat in Adult Patients with Desmoid Tumors

    - First Patient Dosed in Phase 1b Study Evaluating Nirogacestat in Combination with GlaxoSmithKline's Anti-B-Cell Maturation Antigen (BCMA) Antibody-Drug Conjugate, BLENREP (belantamab mafodotin-blmf), for the Treatment of Relapsed or Refractory Multiple Myeloma -

    STAMFORD, Conn., Aug. 12, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported second quarter financial results for the period ended June 30, 2020 and provided an update on recent company developments…

    – Enrollment Complete in the Phase 3 DeFi Trial of Nirogacestat in Adult Patients with Desmoid Tumors

    - First Patient Dosed in Phase 1b Study Evaluating Nirogacestat in Combination with GlaxoSmithKline's Anti-B-Cell Maturation Antigen (BCMA) Antibody-Drug Conjugate, BLENREP (belantamab mafodotin-blmf), for the Treatment of Relapsed or Refractory Multiple Myeloma -

    STAMFORD, Conn., Aug. 12, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported second quarter financial results for the period ended June 30, 2020 and provided an update on recent company developments.

    "We continued to advance our six clinical programs during the second quarter of 2020, most notably completing enrollment in the Phase 3 DeFi trial of nirogacestat in patients with desmoid tumors and initiating, through our collaboration with GSK, the first Phase 1 trial evaluating nirogacestat in combination with a BCMA therapy for patients with multiple myeloma," said Saqib Islam, Chief Executive Officer of SpringWorks. "During the second half of the year, we will remain focused on executing against our objectives and believe we are well-positioned to progress our pipeline across our three distinct oncology focus areas of late-stage rare oncology, BCMA combinations in multiple myeloma and biomarker-defined metastatic solid tumors."

    Recent Business Highlights

    • Achieved full enrollment in the Phase 3 DeFi trial evaluating nirogacestat in adult patients with progressing desmoid tumors.
    • Announced that the first patient was dosed in a Phase 1b trial evaluating nirogacestat in combination with BLENREP (belantamab mafodotin-blmf), GSK's anti-B-cell maturation antigen (BCMA) antibody-drug conjugate, in patients with relapsed or refractory multiple myeloma.
    • Received a new patent (U.S. Patent No. 10,710,966 [the ‘966 patent]) with method of use claims covering the treatment of desmoid tumors with nirogacestat. Like the recently granted composition of matter patent covering the polymorphic form of nirogacestat that is currently in clinical development, this method of use patent expires in 2039 and further enhances the intellectual property position of nirogacestat. The ‘966 patent is assigned to Pfizer and SpringWorks has exclusive rights to it pursuant to an existing worldwide license with Pfizer.
    • Presented preclinical data from studies of mirdametinib in combination with BeiGene's RAF dimer inhibitor, lifirafenib, at the 2020 American Association for Cancer Research (AACR) Virtual Annual Meeting II. Results demonstrated potent and synergistic activity in vitro and in vivo across a panel of cancer models harboring a variety of RAS mutations.
    • Expanded the ongoing Phase 1 trial of BGB-3245 to the United States and began dosing patients at Memorial Sloan Kettering Cancer Center. BGB-3245 is a selective RAF dimer inhibitor being developed by MapKure, LLC, a clinical-stage company that is jointly owned by SpringWorks and BeiGene, Ltd.
    • Continued to strengthen the Company's Board of Directors with the appointment of Julie Hambleton, M.D., an experienced oncology drug developer and pharmaceutical executive.

    COVID-19 Update

    To date, the COVID-19 pandemic has had a relatively modest impact on SpringWorks' business operations, in particular on SpringWorks' clinical trial programs, and the company is undertaking considerable efforts to mitigate the various challenges presented by this crisis. For further details and descriptions of the risks associated with the COVID-19 pandemic, please see the Risk Factors in SpringWorks' Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 12, 2020 and refer to the Forward-Looking Statements section in this press release.

    Second Quarter and Year to Date 2020 Financial Results

    • Research and Development (R&D) Expenses: R&D expenses were $12.9 million and $22.7 million for the second quarter and year-to-date periods, respectively, compared to $11.2 million and $19.6 million for the comparable periods of 2019, respectively. The increases in R&D expenses in 2020 were primarily attributable to growth in employee costs associated with increases in the number of R&D personnel, and an increase in non-cash share-based compensation expense.
    • General and Administrative (G&A) Expenses: G&A expenses were $6.9 million and $13.3 million for the second quarter and year-to-date periods, respectively, compared to $3.6 million and $6.9 million for the comparable periods of 2019, respectively. The increases in G&A expenses in 2020 were primarily attributable to growth in employee costs associated with increases in the number of G&A personnel supporting the growth of the organization, and an increase in non-cash share-based compensation expenses, as well as increases in expenses related to the expansion of business activities.
    • Net Loss Attributable to Common Stockholders: SpringWorks reported net losses of $19.9 million, or $0.47 loss per share, and $35.2 million, or $0.84 loss per share, for the second quarter and year-to-date periods ended June 30, 2020, respectively. This compares to net losses of $13.8 million, or $15.75 loss per share, and $17.5 million, or $22.47 loss per share, for the comparable periods of 2019, respectively.  
    • Cash Position: Cash, cash equivalents and marketable securities were $291.2 million as of June 30, 2020. 

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    SpringWorks uses its website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the Company's website in the ‘Investors & Media' section. Accordingly, investors should monitor such portions of the SpringWorks website, in addition to following press releases, SEC filings and public conference calls and webcasts.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in SpringWorks' SEC filings. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

     
    SpringWorks Therapeutics, Inc.

    Condensed Consolidated Statements of Operations

    (Unaudited)
               
      Three Months Ended June 30,  Six Months Ended June 30, 
    (in thousands, except share and per-share data) 2020  2019  2020  2019 
    Operating expenses:            
    Research and development $12,947  $11,205  $22,674  $19,628 
    General and administrative  6,874   3,646   13,277   6,911 
    Total operating expenses  19,821   14,851   35,951   26,539 
                 
    Loss from operations  (19,821)  (14,851)  (35,951)  (26,539)
    Other income:            
    Interest income, net  157   1,004   1,093   1,283 
    Total other income  157   1,004   1,093   1,283 
    Equity investment loss  (229)     (329)   
    Net loss $(19,893) $(13,847) $(35,187) $(25,256)
                 
    Reconciliation of net loss to net loss attributable to common stockholders:            
    Net loss $(19,893) $(13,847) $(35,187) $(25,256)
    Net gain attributable to extinguishment of Series A

    convertible preferred and Junior Series A convertible

    preferred units
               7,729 
    Net loss attributable to common stockholders $(19,893) $(13,847) $(35,187) $(17,527)
                 
                 
    Net loss per share, basic and diluted $(0.47) $(15.75) $(0.84) $(22.47)
    Weighted average common shares outstanding, basic, and

    diluted
      41,945,058   879,018   41,867,089   780,066 



    SpringWorks Therapeutics, Inc.

    Selected Balance Sheet Data

    (Unaudited)
     
      June 30, 2020

     December 31, 2019

    (In thousands)        
    Cash, cash equivalents and marketable securities $  291,233  $  327,652 
    Working Capital (1)    284,245     319,391 
    Total Assets    301,020     334,831 
    Total liabilities    10,146     12,759 
    Accumulated deficit    (108,216)    (73,029)
    Total stockholders' equity    290,874     322,072 
             
     (1)  We define working capital as current assets less current liabilities. 
     

    Contact:

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com

     

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  39. STAMFORD, Conn., Aug. 04, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will present at the 2020 Wedbush PacGrow Healthcare Conference on Tuesday, August 11, 2020 at 10:20 a.m. Eastern Standard Time.

    A video webcast will be available live on the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for approximately 30 days following the conference.

    About SpringWorks Therapeutics

    STAMFORD, Conn., Aug. 04, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will present at the 2020 Wedbush PacGrow Healthcare Conference on Tuesday, August 11, 2020 at 10:20 a.m. Eastern Standard Time.

    A video webcast will be available live on the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for approximately 30 days following the conference.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    Contact:

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com

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  40. STAMFORD, Conn., July 22, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that it has achieved full enrollment in its Phase 3 DeFi trial evaluating nirogacestat, an investigational gamma secretase inhibitor, in adult patients with progressing desmoid tumors. As previously disclosed, SpringWorks expects to report topline results from the DeFi trial in the second or third quarter of 2021.

    "Desmoid tumors are debilitating and disfiguring soft-tissue tumors and there is a significant need for an FDA-approved therapy for patients living with this devastating disease. We…

    STAMFORD, Conn., July 22, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that it has achieved full enrollment in its Phase 3 DeFi trial evaluating nirogacestat, an investigational gamma secretase inhibitor, in adult patients with progressing desmoid tumors. As previously disclosed, SpringWorks expects to report topline results from the DeFi trial in the second or third quarter of 2021.

    "Desmoid tumors are debilitating and disfiguring soft-tissue tumors and there is a significant need for an FDA-approved therapy for patients living with this devastating disease. We are delighted to have reached another milestone for the nirogacestat program by completing enrollment of the DeFi trial," said Saqib Islam, Chief Executive Officer of SpringWorks. "This achievement represents an important step toward our goal of bringing this new potential therapy to patients with desmoid tumors. We look forward to completing the trial and reporting data from the study next year."

    The DeFi trial is a global, randomized, double-blind, placebo-controlled Phase 3 trial to evaluate the efficacy, safety and tolerability of nirogacestat in adult patients with progressing desmoid tumors. The study was designed to enroll 118 patients across approximately 50 sites in the United States and Europe. Patients have been randomized 1:1 to receive 150 mg of nirogacestat or placebo twice daily. Key eligibility criteria included tumor progression by >20% as measured by Response Evaluation Criteria in Solid Tumors (RECIST 1.1) within 12 months prior to the first dose of study treatment. The primary endpoint is progression-free survival and secondary endpoints include safety and tolerability measures, as well as objective response rate, changes in tumor volume assessed by MRI, and changes in patient-reported outcomes. More information about the DeFi trial is available at www.clinicaltrials.gov under the identifier NCT03785964.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors (June 2018) and from the European Commission for the treatment of soft tissue sarcoma (September 2019). The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis (November 2018 and August 2019).

    About Desmoid Tumors

    Desmoid tumors, also referred to as aggressive fibromatosis or desmoid-type fibromatosis, are rare and often debilitating and disfiguring soft tissue tumors characterized by a growth pattern that can invade surrounding healthy tissues, including joints, muscle and viscera. While they can arise in any part of the body, the most common sites are the upper and lower extremities, abdominal wall, thoracic areas, and the head and neck. The severity of a desmoid tumor can vary based on the location of the tumor and the aggressiveness of its growth pattern. Desmoid tumors can cause significant morbidities, including severe pain, internal bleeding, incapacitating loss of range of motion, and, in rare cases, death.1

    Desmoid tumors typically occur in patients between the ages of 15 to 60 years, and are more commonly diagnosed in young adults between 30-40 years of age, with a two-to-three times higher prevalence in females.1,2 It is estimated that there are 1,000 to 1,500 new cases diagnosed per year in the United States.3,4

    Historically, desmoid tumors were treated with surgical resection, but this approach has become less favored due to a high recurrence rate after surgery.5 There are currently no FDA-approved therapies for the treatment of desmoid tumors.

    About Nirogacestat

    Nirogacestat is an investigational, oral, selective, small molecule gamma-secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the soluble BCMA from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA, which may serve as a decoy receptor for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is pursuing a combination therapy approach to evaluate nirogacestat as a BCMA potentiator across modalities by collaborating with industry leaders. To date, SpringWorks has entered into two clinical collaborations to evaluate nirogacestat in combination with GSK's BCMA antibody-drug conjugate belantamab mafodotin and with Allogene's allogeneic BCMA CAR-T cell therapy ALLO-715.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, visit www.springworkstx.com and follow @SpringWorksTx on Twitter and LinkedIn.

    SpringWorks Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    SpringWorks Media/Investor Contact:

    Kim Diamond

    203-561-1646

    kdiamond@springworkstx.com

    References:

    1 Gounder, M. M., Thomas, D. M., & Tap, W. D. (2017). Locally Aggressive Connective Tissue Tumors. Journal of Clinical Oncology, 36(2), 202-209. doi:10.1200/JCO.2017.75.8482.

    2 Skubitz, K. M. (2017). Biology and Treatment of Aggressive Fibromatosis or Desmoid Tumor. Mayo Clinic Proceedings, 92(6), 947-964. doi:10.1016/j.mayocp.2017.02.012

    3 Reitamo, J J; Häyry, P; Nykyri, E; Saxén, E. (1982). The desmoid tumor. I. Incidence, sex-, age- and anatomical distribution in the Finnish population. American Journal of Clinical Pathology, 77(6), 665-673. doi: 10.1093/AJCP.77.6.665

    4 van Broekhoven, D. L., Grünhagen, D. J., den Bakker, M. A., van Dalen, T., & Verhoef, C. (2015). Time trends in the incidence and treatment of extra-abdominal and abdominal aggressive fibromatosis: a population-based study. Annals of surgical oncology, 22(9), 2817–2823. doi:10.1245/s10434-015-4632-y

    5 Scaramussa, F.S. & Castro, U. B. (2016). Desmoid Tumor in Hand: A Case Report. SM Journal of Orthopedics, 2(3),1036.

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  41. Preclinical data presented at the American Association for Cancer Research 2020 Virtual Annual Meeting II

    Ongoing Phase 1b/2 clinical trial on track to commence dose expansion cohorts in KRAS mutant solid tumors by the end of 2020

    CAMBRIDGE, Mass. and BEIJING and STAMFORD, Conn., June 22, 2020 (GLOBE NEWSWIRE) -- BeiGene, Ltd. (NASDAQ:BGNE, HKEX: 06160))), a commercial-stage biotechnology company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced the presentation…

    Preclinical data presented at the American Association for Cancer Research 2020 Virtual Annual Meeting II

    Ongoing Phase 1b/2 clinical trial on track to commence dose expansion cohorts in KRAS mutant solid tumors by the end of 2020

    CAMBRIDGE, Mass. and BEIJING and STAMFORD, Conn., June 22, 2020 (GLOBE NEWSWIRE) -- BeiGene, Ltd. (NASDAQ:BGNE, HKEX: 06160))), a commercial-stage biotechnology company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced the presentation of preclinical data and provided a program update on their ongoing Phase 1b/2 study evaluating the combination of BeiGene's investigational RAF dimer inhibitor, lifirafenib, with SpringWorks' investigational MEK inhibitor, mirdametinib, in patients with advanced or refractory solid tumors harboring mutations in the MAPK pathway, including those with RAS mutations.

    The preclinical data were presented today in a poster at the American Association for Cancer Research (AACR) 2020 Virtual Annual Meeting II by researchers from BeiGene, SpringWorks, and Memorial Sloan Kettering Cancer Center. Among the findings presented, lifirafenib and mirdametinib demonstrated potent and synergistic activity in vitro and in vivo across a panel of RAS-mutated cancer models harboring a variety of mutations using clinically relevant concentrations of the compounds. Furthermore, in KRAS Q61K and KRAS G12C xenograft models, the combination demonstrated synergy as validated by pharmacodynamic pathway inhibition and tumor regressions. These preclinical findings provided further support for the ongoing Phase 1b/2 combination study of lifirafenib and mirdametinib, which is evaluating this "vertical inhibition" strategy for treating patients whose cancers harbor RAS mutations and other MAPK pathway aberrations.

    Based on the progress of the program and the clinical data observed to date, the companies reiterated their previous guidance to complete the dose escalation portion of the trial by the end of 2020 and to commence dose expansion cohorts in tumor types of interest thereafter, including KRAS-mutated non-small cell lung cancer and KRAS-mutated endometrial cancer. Initial clinical data from the trial is expected to be presented at a medical conference in 2021.

    "We have been pleased with the progress of the lifirafenib and mirdametinib combination therapy trial to date, and we look forward to commencing dose expansion cohorts in the coming months," said Ben Solomon, M.B.B.S., Ph.D., Senior Faculty Member and Medical Oncologist in the Lung and Head & Neck Service at the Peter MacCallum Cancer Centre, who is the principal investigator of the study. "There remains a significant unmet need for the nearly one-third of solid tumor patients whose cancers are driven by RAS mutations, RAF mutations, and other MAPK pathway aberrations, and this combination therapy represents a novel targeted approach for these tumors."

    More information about the ongoing trial of lifirafenib and mirdametinib may be found by searching clinical trial identifier NCT03905148 at https://clinicaltrials.gov.

    Details for the AACR 2020 Virtual Meeting II presentation are as follows:

    Title: RAF dimer inhibitor lifirafenib enhances the antitumor activity of MEK inhibitor mirdametinib in RAS mutant tumors

    Authors: Xi Yuan, Xinwen Zhang, Rong Du, Shing-Hu Cheung, Xikun Wu, Beibei Jiang, Zhiyu Tang, Min Wei, Changyou Zhou, Lai Wang, Todd Shearer, Badreddin Edris, L. Mary Smith, Claire N. Thant, Zhan Yao, Neal Rosen, Lusong Luo

    Poster #: 6415

    Session: Novel Therapeutic Approaches

    Date and Time: June 22, 2020; 9:00 a.m. ET

    About Lifirafenib

    Lifirafenib was discovered in BeiGene's research facilities in Beijing, China, and is an investigational small molecule kinase inhibitor with RAF monomer and dimer inhibition activities. Lifirafenib has shown antitumor activities in preclinical models and in cancer patients with tumors harboring BRAF V600E mutations, non-V600E BRAF mutations, non-small cell lung cancer and endometrial cancer harboring KRAS mutations. To date, lifirafenib has been dosed in more than 150 patients globally.

    About Mirdametinib

    Mirdametinib is an investigational, selective, orally bioavailable small molecule inhibitor of MEK1 and MEK2, proteins that play key roles in the MAPK pathway. The MAPK pathway is critical for cell survival and proliferation, and overactivation of this pathway has been shown to help enable tumor growth. By blocking activity of the MAPK pathway, mirdametinib may help arrest uncontrolled cellular growth associated with many types of tumors.

    Mirdametinib has been tested in several Phase 1 and Phase 2 clinical trials, and approximately 260 subjects have been exposed to treatment. SpringWorks is evaluating mirdametinib as a monotherapy for the treatment of patients with neurofibromatosis type 1-associated plexiform neurofibromas (the ReNeu trial) and is also pursuing mirdametinib in combination with other rational anti-cancer agents across a range of solid tumors.

    About BeiGene

    BeiGene is a global, commercial-stage biotechnology company focused on discovering, developing, manufacturing, and commercializing innovative medicines to improve treatment outcomes and access for patients worldwide. Our 3,800+ employees in China, the United States, Australia, and Europe are committed to expediting the development of a diverse pipeline of novel therapeutics for cancer. We currently market two internally-discovered oncology products: BTK inhibitor BRUKINSA® (zanubrutinib) in the United States and China and anti-PD-1 antibody tislelizumab in China. We also market or plan to market in China additional oncology products licensed from Amgen Inc., Celgene Logistics Sàrl, a Bristol Myers Squibb (BMS) company, and EUSA Pharma. To learn more about BeiGene, please visit www.beigene.com and follow us on Twitter at @BeiGeneUSA.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com.

    Follow SpringWorks Therapeutics on social media: @SpringWorksTx and LinkedIn.

    BeiGene Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the encouraging pre-clinical data for the combination of lifirafenib and mirdametinib, planned milestones for the Phase 1b/2 clinical trial and presentation of data, and the potential for the combination to be a novel targeted approach to treat solid tumor patients whose cancers are driven by RAS mutations, RAF mutations, and other MAPK pathway aberrations. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed products and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its technology and drugs; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGene's limited operating history and BeiGene's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates; the impact of the COVID-19 pandemic on the Company's clinical development, commercial and other operations, as well as those risks more fully discussed in the section entitled "Risk Factors" in BeiGene's most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

    SpringWorks Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    BeiGene Investor ContactBeiGene Media Contact
    Craig WestLiza Heapes
    +1 857-302-5189+1 857-302-5663
    ir@beigene.commedia@beigene.com

    SpringWorks Investor and Media Contact

    Kim Diamond

    Phone: 203-561-1646

    Email: kdiamond@springworkstx.com

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  42. STAMFORD, Conn., June 22, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the first patient has been dosed in a Phase 1b clinical trial evaluating SpringWorks Therapeutics' investigational gamma secretase inhibitor, nirogacestat, in combination with GlaxoSmithKline's (GSK) investigational anti-B-cell maturation antigen (BCMA) antibody-drug conjugate, belantamab mafodotin, in patients with relapsed or refractory multiple myeloma. The nirogacestat and belantamab mafodotin combination is being evaluated as a sub-study in GSK's ongoing DREAMM-5 platform trial.

    STAMFORD, Conn., June 22, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that the first patient has been dosed in a Phase 1b clinical trial evaluating SpringWorks Therapeutics' investigational gamma secretase inhibitor, nirogacestat, in combination with GlaxoSmithKline's (GSK) investigational anti-B-cell maturation antigen (BCMA) antibody-drug conjugate, belantamab mafodotin, in patients with relapsed or refractory multiple myeloma. The nirogacestat and belantamab mafodotin combination is being evaluated as a sub-study in GSK's ongoing DREAMM-5 platform trial.

    Gamma secretase inhibition prevents the cleavage and shedding of BCMA from the surface of myeloma cells. In preclinical models, nirogacestat has been shown to increase the cell surface density of BCMA and to reduce levels of soluble BCMA, thereby enhancing the activity of BCMA-targeted therapies. Further, preclinical data evaluating the combination of nirogacestat and belantamab mafodotin demonstrated synergistic increases in human multiple myeloma cell killing as compared to belantamab mafodotin alone, with an up to ~3,000-fold improvement in cytotoxicity with the combination.1

    "SpringWorks is focused on advancing a comprehensive strategy to evaluate the ability of nirogacestat to potentiate BCMA-directed therapies across modalities with the goal of making nirogacestat a cornerstone of BCMA combination therapy for patients with multiple myeloma," said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics. "We are delighted to have reached this important milestone of dosing the first patient in this collaborative trial with GSK, an industry leader committed to advancing BCMA therapies. We look forward to generating clinical data to test the hypothesis that adding nirogacestat to belantamab mafodotin could lead to better clinical outcomes for patients."

    The Phase 1b combination trial is being advanced pursuant to a global clinical trial collaboration agreement that SpringWorks and GSK entered into in June 2019. Under the terms of the agreement, GSK is sponsoring and conducting the Phase 1b study to evaluate the safety, tolerability and preliminary efficacy of the combination, and is assuming all development costs associated with the study, other than expenses related to the manufacturing of nirogacestat and certain expenses related to intellectual property rights. SpringWorks and GlaxoSmithKline have formed a joint development committee to help manage and oversee the clinical study.

    About the Phase 1b Trial

    The Phase 1b trial (NCT04126200), which is a sub-study of GSK's DREAMM-5 Phase 1/2 platform study, is an open-label study to evaluate the safety, tolerability and preliminary efficacy of nirogacestat in combination with belantamab mafodotin in patients with relapsed or refractory multiple myeloma. The study will include two parts, a dose exploration phase and a cohort expansion phase. The dose exploration phase will evaluate the safety and tolerability profile of GSK's belantamab mafodotin when administered in combination with nirogacestat and will identify a recommended Phase 2 dose for each drug in combination. The cohort expansion phase will evaluate the clinical activity of the combination in comparison to belantamab mafodotin monotherapy in additional patients with relapsed or refractory multiple myeloma.

    About Nirogacestat

    Nirogacestat is an investigational, oral, selective, small molecule gamma-secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the soluble BCMA from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA, which may serve as a decoy receptor for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is pursuing a combination therapy approach to evaluate nirogacestat as a BCMA potentiator across modalities by collaborating with industry leaders. To date, SpringWorks has entered into two clinical collaborations to evaluate nirogacestat in combination with GSK's BCMA antibody-drug conjugate belantamab mafodotin and with Allogene's allogeneic BCMA CAR-T cell therapy ALLO-715.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors (June 2018) and from the European Commission for the treatment of soft tissue sarcoma (September 2019). The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis in November 2018 and August 2019, respectively.

    About belantamab mafodotin (GSK2857916)

    Belantamab mafodotin is an investigational antibody drug conjugate comprising a humanized anti-B cell maturation antigen (BCMA) monoclonal antibody conjugated to the cytotoxic agent auristatin F via non-cleavable linker.2 The drug linker technology is licensed from Seattle Genetics; monoclonal antibody is produced using POTELLIGENT Technology licensed from BioWa.

    Belantamab mafodotin is not currently approved for use anywhere in the world.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com.

    Follow SpringWorks Therapeutics on social media: @SpringWorksTx and LinkedIn.

    SpringWorks Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    SpringWorks Media/Investor Contact:

    Kim Diamond

    203-561-1646

    kdiamond@springworkstx.com

    References:

    Eastman, S., Shelton, C., Gupta, I., Krueger, J., Blackwell, C., & Bojczuk, P. M. (2019, December). 4401 Synergistic Activity of Belantamab Mafodotin (anti-BCMA immuno-conjugate) with PF-03084014 (gamma-secretase inhibitor) in Bcma-Expressing Cancer Cell Lines. Poster session presented at the 61st American Society of Hematology Annual Meeting & Exposition, Orlando, FL.

    2 NCI Drug Dictionary - Anti-BCMA Antibody-Drug Conjugate GSK2857916. National Cancer Institute. https://www.cancer.gov/publications/dictionaries/cancer-drug/def/anti-bcma-antibody-drug-conjugate-gsk2857916. Accessed May 2020.

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  43. STAMFORD, Conn., June 02, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will present at the Goldman Sachs 41st Annual Global Healthcare Conference on Tuesday, June 9, 2020 at 8:50 a.m. Eastern Standard Time.

    A video webcast will be available live on the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for approximately 30 days following the conference.

    About SpringWorks Therapeutics

    STAMFORD, Conn., June 02, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will present at the Goldman Sachs 41st Annual Global Healthcare Conference on Tuesday, June 9, 2020 at 8:50 a.m. Eastern Standard Time.

    A video webcast will be available live on the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for approximately 30 days following the conference.

    About SpringWorks Therapeutics
    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com. Follow SpringWorks Therapeutics on social media: @SpringWorksTx and LinkedIn.

    Contact:
    Kim Diamond
    Phone: 203-561-1646
    Email: kdiamond@springworkstx.com

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  44. STAMFORD, Conn., May 26, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced the appointment of Julie Hambleton, M.D. to the Company's Board of Directors.

    "We are delighted to welcome Julie to the SpringWorks Board of Directors. She is a leading oncology drug developer whose experience will further strengthen our board and provide valuable insight as we advance our diversified pipeline of targeted oncology programs in rare oncology indications, multiple myeloma and metastatic solid tumors," said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics.

    Dr. Hambleton…

    STAMFORD, Conn., May 26, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced the appointment of Julie Hambleton, M.D. to the Company's Board of Directors.

    "We are delighted to welcome Julie to the SpringWorks Board of Directors. She is a leading oncology drug developer whose experience will further strengthen our board and provide valuable insight as we advance our diversified pipeline of targeted oncology programs in rare oncology indications, multiple myeloma and metastatic solid tumors," said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics.

    Dr. Hambleton, M.D. added, "I am impressed by the breadth and quality of targeted oncology programs that SpringWorks is advancing both as a monotherapy for patients with rare diseases, and as combination therapies for patients with highly prevalent cancers. SpringWorks has built leading drug development capabilities and has already demonstrated strong execution across their portfolio. I look forward to working with the team and sharing my experience to guide the current and future development programs."

    Julie Hambleton, M.D. is a senior biotechnology executive with over 20 years of experience in clinical drug development from pre-clinical through Phase 4 and post-marketing studies. She has extensive experience working with regulatory agencies, including the U.S. FDA and the European Medicines Agency (EMA), and in filings of Investigational New Drug Applications (INDs), Biologics License Applications (BLAs), and Special Protocol Assessments (SPAs). Dr. Hambleton served as Senior Vice President, Chief Medical Officer, Head of Development at IDEAYA Biosciences, an oncology medicine company, until her retirement on April 30, 2020. Previously, she was Vice President, Head of U.S. Medical at Bristol-Myers Squibb, overseeing Medical & Health Economic and Outcomes Research activities in support of the Oncology, Immuno-Oncology, Specialty and Cardiovascular marketed portfolios. Previously, she served as Executive Vice President and Chief Medical Officer at Five Prime Therapeutics and Vice President, Clinical Development, at Clovis Oncology. Dr. Hambleton began her industry career at Genentech, most recently as Group Medical Director, Global Clinical Development, leading a cross-functional group conducting Phase 2 and 3 trials of Avastin®. Dr. Hambleton also serves on the board of directors of IGM Biosciences, Inc., a publicly traded biotechnology company, and of Arch Oncology, a privately-held biotechnology company.

    Dr. Hambleton completed her medical and hematology-oncology training at the University California, San Francisco, where she then served on faculty from 1993 to 2003. She received a B.S. from Duke University, and M.D. from Case Western Reserve University School of Medicine and was Board-certified in Hematology and Internal Medicine.

    About SpringWorks Therapeutics
    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com. Follow SpringWorks Therapeutics on social media: @SpringWorksTx and LinkedIn.

    SpringWorks Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks' business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    SpringWorks Media/Investor Contact:
    Kim Diamond
    203-561-1646
    kdiamond@springworkstx.com

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  45. STAMFORD, Conn., March 18, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX) announced today that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 10,590,087 (the ‘087 patent), directed to nirogacestat, the Company's lead product candidate in development for patients with desmoid tumors and as a combination therapy for patients with multiple myeloma. The ‘087 patent, assigned to Pfizer, Inc., expires in 2039. The ‘087 patent is a composition of matter patent that covers the polymorphic form of nirogacestat that is currently in clinical development. SpringWorks has exclusive rights to the ‘087 patent pursuant to an existing worldwide license with Pfizer.

    "We are very pleased that the USPTO…

    STAMFORD, Conn., March 18, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX) announced today that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 10,590,087 (the ‘087 patent), directed to nirogacestat, the Company's lead product candidate in development for patients with desmoid tumors and as a combination therapy for patients with multiple myeloma. The ‘087 patent, assigned to Pfizer, Inc., expires in 2039. The ‘087 patent is a composition of matter patent that covers the polymorphic form of nirogacestat that is currently in clinical development. SpringWorks has exclusive rights to the ‘087 patent pursuant to an existing worldwide license with Pfizer.

    "We are very pleased that the USPTO has issued this patent, which builds upon our existing intellectual property portfolio and extends patent protection for nirogacestat to 2039," said Saqib Islam, Chief Executive Officer of SpringWorks. "The granting of this patent represents an important development towards our objective of expanding intellectual property protections for our portfolio of targeted oncology product candidates as we seek to advance promising science for underserved patient communities."

    SpringWorks is currently enrolling patients in a global Phase 3, double-blind, randomized, placebo-controlled clinical trial (the DeFi Trial) to evaluate nirogacestat in adults with progressing desmoid tumors/aggressive fibromatosis. In addition, SpringWorks is seeking to advance nirogacestat as a cornerstone of B-cell maturation antigen (BCMA) combination therapy in multiple myeloma. To date, SpringWorks has entered into two clinical collaborations to evaluate nirogacestat in combination with GlaxoSmithKline's BCMA antibody-drug conjugate belantamab mafodotin and with Allogene's allogeneic BCMA CAR-T cell therapy ALLO-715.

    About Nirogacestat

    Nirogacestat is an investigational, oral, selective, small molecule gamma-secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is pursuing a combination therapy approach to evaluate nirogacestat as a BCMA potentiator across modalities by collaborating with industry leaders. To date, SpringWorks has entered into two clinical collaborations to evaluate nirogacestat in combination with GlaxoSmithKline's BCMA antibody-drug conjugate belantamab mafodotin and with Allogene's allogeneic BCMA CAR-T cell therapy ALLO-715.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors (June 2018) and from the European Commission for the treatment of soft tissue sarcoma (September 2019). The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis (November 2018 and August 2019).

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com.

    Follow SpringWorks Therapeutics on social media: @SpringWorksTx and LinkedIn.

    SpringWorks Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, statements regarding SpringWorks' intellectual property, its future research and development activities, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-K for the quarter ended December 31, 2019, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    Contact:
    Kim Diamond
    Phone: 646-661-1255
    Email: kdiamond@springworkstx.com

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  46. - Dosed the First Patients in the Phase 2b ReNeu Clinical Trial of Mirdametinib for Patients with Neurofibromatosis Type 1-Associated Plexiform Neurofibromas (NF1-PN)

    - Signed Clinical Collaboration with Allogene to Evaluate Nirogacestat in Combination with Allogeneic CAR T Cell Therapy in Patients with Relapsed or Refractory Multiple Myeloma - 

    - Initiated a Phase 1 Clinical Trial of BGB-3245, a Selective Next-Generation B-RAF Inhibitor, in Adult Patients with Advanced or Refractory Solid Tumors - 

    - Ended 2019 with $328 Million in Cash and Cash Equivalents -             

    STAMFORD, Conn., March 12, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing…

    - Dosed the First Patients in the Phase 2b ReNeu Clinical Trial of Mirdametinib for Patients with Neurofibromatosis Type 1-Associated Plexiform Neurofibromas (NF1-PN)

    - Signed Clinical Collaboration with Allogene to Evaluate Nirogacestat in Combination with Allogeneic CAR T Cell Therapy in Patients with Relapsed or Refractory Multiple Myeloma - 

    - Initiated a Phase 1 Clinical Trial of BGB-3245, a Selective Next-Generation B-RAF Inhibitor, in Adult Patients with Advanced or Refractory Solid Tumors - 

    - Ended 2019 with $328 Million in Cash and Cash Equivalents -             

    STAMFORD, Conn., March 12, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today provided an update on recent company developments and reported fourth quarter and full-year financial results for the period ended December 31, 2019.

    "2019 was a very successful year for SpringWorks. We initiated three clinical trials, including two potentially registrational trials in rare oncology indications, formed MapKure with BeiGene to advance a next-generation B-RAF inhibitor, and signed our first collaboration with GlaxoSmithKline to evaluate nirogacestat in combination with BCMA-directed therapies in patients with relapsed and refractory multiple myeloma," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our strong execution in 2019 positions us to deliver on our planned strategy in 2020 and beyond. We are pursuing a broad effort with industry-leading BCMA developers to advance potentially best-in-class multiple myeloma combination therapies using nirogacestat, are focused on continuing to enroll our ongoing clinical trials, and further expanding our portfolio through additional in-licenses and collaborations."

    Recent Business Highlights

    • Initiated a Phase 1 clinical trial of BGB-3245, a selective next-generation B-RAF inhibitor, in adult patients with biomarker-defined advanced or refractory solid tumors. BGB-3245 is being advanced by MapKure LLC, a clinical-stage company that is jointly owned by SpringWorks and BeiGene, Ltd.
    • Entered into a clinical collaboration agreement with Allogene Therapeutics to evaluate nirogacestat in combination with ALLO-715, an investigational anti-B-cell maturation antigen (BCMA) allogeneic CAR T cell therapy, in patients with relapsed or refractory multiple myeloma.
    • Presented data at the American Society of Hematology meeting showing activity of nirogacestat alone and in combination with GlaxoSmithKline's investigational BCMA antibody-drug conjugate (ADC), belantamab mafodotin, in preclinical models of human multiple myeloma.
    • Dosed the first patients in the Phase 2b ReNeu clinical trial evaluating mirdametinib in children and adults with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN).             

    Fourth Quarter and Full Year 2019 Financial Results

    • Research and Development (R&D) Expenses: R&D expenses were $12.2 million and $42.5 million for the fourth quarter and year-to-date periods, respectively, compared to $3.7 million and $9.9 million for the comparable periods of 2018, respectively. The increases in R&D expenses in 2019 were primarily attributable to increased clinical study and drug supply costs related to the ongoing Phase 3 DeFi and Phase 2b ReNeu trials, as well as growth in employee costs, including non-cash share-based compensation associated with increases in the number of R&D personnel.
    • General and Administrative (G&A) Expenses: G&A expenses were $5.2 million and $16.7 million for the fourth quarter and year-to-date periods, respectively, compared to $2.7 million and $8.6 million for the comparable periods of 2018, respectively. The increases in G&A expenses in 2019 were primarily attributable to growth in employee costs, including non-cash share-based compensation associated with increases in the number of G&A personnel, and increases in consulting and professional services related to the expansion of our business activities.
    • Net Loss Attributable to Common Stockholders: SpringWorks reported net losses of $16.2 million, or $0.39 loss per share, and $50.6 million, or $3.81 loss per share, for the fourth quarter and year-to-date periods ended December 31, 2019, respectively. This compares to net losses of $6.2 million, or $10.82 loss per unit, and $17.8 million, or $52.24 loss per unit, for the comparable periods of 2018, respectively.
    • Cash Position: Cash and cash equivalents were $327.7 million as of December 31, 2019. 

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com.

    Follow SpringWorks Therapeutics on social media: @SpringWorksTx and LinkedIn.

    SpringWorks uses its website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the Company's website in the ‘Investors & Media' section. Accordingly, investors should monitor such portions of the SpringWorks website, in addition to following press releases, SEC filings and public conference calls and webcasts.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to SpringWorks' financial results, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, competition from other biopharmaceutical companies, and other risks identified in SpringWorks' SEC filings. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.


    SpringWorks Therapeutics, Inc. and Subsidiaries
    (formerly SpringWorks Therapeutics, LLC) 
    Consolidated Statements of Operations and Comprehensive Loss
    (Unaudited)

        Year Ended December 31,       
    (In thousands, except share, unit, per-share and per-unit data)   2019     2018       Period from
    August 18, 2017
    (Inception) to
    December 31, 2017
     
    Operating expenses:                  
    Research and development   $  42,545     $  9,898     $  2,799  
    General and administrative      16,694        8,593        1,861  
    Total operating expenses      59,239        18,491        4,660  
    Loss from operations      (59,239 )      (18,491 )      (4,660 )
    Other income:                  
    Interest income, net      3,547        678        21  
    Total other income      3,547        678        21  
    Equity investment loss      (2,614 )      —        —  
    Net loss   $  (58,306 )   $  (17,813 )   $  (4,639 )
    Reconciliation of net loss to net loss attributable to common stockholders:                  
    Net loss   $  (58,306 )   $  (17,813 )   $  (4,639 )
    Net gain attributable to extinguishment of Series A convertible preferred and Junior Series A convertible preferred units      7,729        —        —  
    Net loss attributable to common stockholders, basic and diluted   $  (50,577 )   $  (17,813 )   $  (4,639 )
    Net loss per unit, basic and diluted         $  (52.24 )   $  
    Net loss per share, basic and diluted   $  (3.81 )      —        —  
    Weighted average common units outstanding, basic and diluted      —        341,014        
    Weighted average common shares outstanding, basic and diluted      13,274,836        —        —  


    SpringWorks Therapeutics, Inc. and Subsidiaries
    (formerly SpringWorks Therapeutics, LLC)
    Selected Balance Sheet Data
    (Unaudited)

        December 31, 
        2019     2018  
    (In thousands)    
    Cash and cash equivalents   $  327,652     $  45,648  
    Working Capital (1)      319,391        43,353  
    Total Assets      334,831        48,390  
    Total liabilities      12,759        4,829  
    Accumulated deficit      (73,029 )      (22,452 )
    Total stockholders' (deficit) equity      322,072        (19,369 )

    (1)  SpringWorks defines working capital as current assets less current liabilities.


    Contact:
    Kim Diamond
    Phone: 646-661-1255
    Email: kdiamond@springworkstx.com

     

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  47. -- Phase 1 Study Initiated in Australia with IND Cleared to Expand Study to the U.S.--

    -- Industry-Leading Scientific Advisory Board Formed to Support Advancement of BGB-3245 --

    CAMBRIDGE, Mass. and BEIJING and STAMFORD, Conn., Feb. 27, 2020 (GLOBE NEWSWIRE) -- MapKure, LLC, a clinical-stage company developing precision medicines for patients with life-threatening diseases, together with BeiGene, Ltd. (NASDAQ:BGNE, HKEX: 06160)) and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), who are joint owners of MapKure, announced today that the first patient has been dosed in Australia in a Phase 1 clinical trial to evaluate BGB-3245, an investigational, next-generation B-RAF inhibitor, in patients with advanced or refractory solid tumors. The companies…

    -- Phase 1 Study Initiated in Australia with IND Cleared to Expand Study to the U.S.--

    -- Industry-Leading Scientific Advisory Board Formed to Support Advancement of BGB-3245 --

    CAMBRIDGE, Mass. and BEIJING and STAMFORD, Conn., Feb. 27, 2020 (GLOBE NEWSWIRE) -- MapKure, LLC, a clinical-stage company developing precision medicines for patients with life-threatening diseases, together with BeiGene, Ltd. (NASDAQ:BGNE, HKEX: 06160)) and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), who are joint owners of MapKure, announced today that the first patient has been dosed in Australia in a Phase 1 clinical trial to evaluate BGB-3245, an investigational, next-generation B-RAF inhibitor, in patients with advanced or refractory solid tumors. The companies also announced that the U.S. Food and Drug Administration (FDA) has allowed the Investigational New Drug (IND) application submitted for BGB-3245 to proceed, which will enable study expansion to U.S. sites.

    B-RAF gene mutations and fusions have been shown to play a key role in the development of certain cancers. BGB-3245 is designed to inhibit both monomeric and dimeric forms of activating B-RAF mutations including V600 and non-V600 mutations, and RAF fusions, and has shown potent preclinical activity against a range of B-RAF gene alterations, including those for which there are no approved targeted therapies and those associated with resistance to currently approved therapies.

    "Preclinical data suggest that BGB-3245 could potentially address a range of B-RAF driven tumors, which represent a significant need for patients who currently lack targeted therapeutic options, as well as patients who have developed resistance to first generation B-RAF inhibitors," said Neal Rosen, M.D., Ph.D., the Enid A. Haupt Chair in Medical Oncology at Memorial Sloan-Kettering Cancer Center and Chair of the MapKure Scientific Advisory Board. "If our hypothesis is correct, we believe that BGB-3245 could have meaningful, single agent antitumor activity in B-RAF-altered cancers through its ability to address key primary and resistance gene alterations that are currently unaddressed by approved therapies."

    The Phase 1 clinical trial is a first-in-human, open-label, dose escalation and expansion study to investigate the safety, pharmacokinetics (PK) and antitumor activity of BGB-3245 in adult patients with solid tumors, including those harboring specific B-RAF driver mutations and fusions that are likely to respond to a RAF dimer inhibitor, as well as in certain adult patients who have developed resistance to first-generation B-RAF V600 inhibitors. The trial is designed to define the dose and assess the tolerability of BGB-3245 and will capture early antitumor activity signals to allow for potential cohort expansion.

    In addition, MapKure has completed the formation of its Scientific Advisory Board (SAB), comprised of renowned leaders in MAPK pathway biology and targeted oncology clinical development. The SAB will continue to collaborate with MapKure's joint steering committee, consisting of representatives from BeiGene and SpringWorks, to support the advancement of BGB-3245. The composition of the SAB is as follows:

    • Neal Rosen, M.D., Ph.D., the Enid A. Haupt Chair in Medical Oncology at Memorial Sloan-Kettering Cancer Center.  Dr. Rosen is the founding member and Chair of the MapKure SAB.
    • Antoni Ribas, M.D., Professor of Medicine, Professor of Surgery, and Professor of Molecular and Medical Pharmacology at the University of California Los Angeles.
    • Kevin Koch, Ph.D., President and CEO of Edgewise Therapeutics; formerly Founder, President, and Chief Scientific Officer of Array BioPharma.
    • Zhan Yao, Ph.D., Assistant Research Professor, Molecular Pharmacology Program, Memorial Sloan-Kettering Cancer Center.
    • Dejan Juric, M.D., Program Director, Investigational Cancer Therapeutics Program and Attending Physician in Medical Oncology, Massachusetts General Hospital.

    About the Phase 1 Trial

    The Phase 1 trial (NCT04249843) of BGB-3245 is an open-label, dose-escalation trial of BGB-3245 in adult patients with advanced or refractory solid tumors, including those with B-RAF driver mutations and fusions that are likely to respond to a RAF dimer inhibitor. The study will enroll patients who have experienced disease progression during or after at least one prior line of systemic anticancer therapy or for which treatment is not tolerated or acceptable to the participants.

    The trial is designed in two parts: the Phase 1a portion will consist of a dose-escalation and dose-finding component to establish the maximum tolerated dose and/or the recommended Phase 2 dose and to evaluate the pharmacokinetics of BGB-3245 in patients with MAPK pathway aberrations. The Phase 1b portion will consist of one or more expansion cohorts to further evaluate the pharmacokinetics, safety, and tolerability of BGB-3245 at the recommended Phase 2 dose and to assess the preliminary antitumor activity of the compound in patients with select tumor types and B-RAF status (B-RAF point mutations and gene fusions).

    About BGB-3245

    BGB-3245 is an investigational, oral, selective small molecule inhibitor of monomer and dimer forms of activating B-RAF mutations including V600 B-RAF mutations, non-V600 B-RAF mutations, and RAF fusions. These mutations and fusions have been identified in a number of solid tumors to be drivers of cancer growth, including in non-small cell lung cancer, colorectal cancer, thyroid cancer, and brain tumors. Preclinical data have demonstrated that BGB-3245 has activity in patient-derived xenografts driven by B-RAF fusions and non-V600 mutations for which approved B-RAF inhibitors are ineffective. In addition, BGB-3245's preclinical activity in cancer models driven by V600 B-RAF mutations has suggested that it could provide an additional therapeutic option for patients with the potential to reduce dimer-driven resistance.

    In addition to its intended use as a monotherapy in several genetically defined solid tumor types, BGB-3245 also has the potential to be used in rational combination therapies in the future.

    About MapKure

    MapKure is a clinical-stage company created in 2019 to develop precision medicines for patients with life-threatening diseases, with an initial focus on cancer. By focusing on genetically defined disease drivers, MapKure is positioned to advance the development of transformative medicines to patients whose unmet medical needs are largely unaddressed. MapKure is jointly owned by BeiGene, Ltd. and SpringWorks, and is currently developing BGB-3245 under exclusive license from BeiGene in solid tumor patients harboring specific B-RAF driver mutations and RAF fusions, as well as in patients who have developed resistance to first-generation B-RAF inhibitors.

    About BeiGene

    BeiGene is a global, commercial-stage research-based biotechnology company focused on molecularly-targeted and immuno-oncology cancer therapeutics. With a team of over 3,500 employees in the United States, China, Australia, and Europe, BeiGene is advancing a pipeline consisting of novel oral small molecules and monoclonal antibodies for cancer. BeiGene is also working to create combination solutions aimed to have both a meaningful and lasting impact on cancer patients. In the United States, BeiGene markets and distributes BRUKINSA™ (zanubrutinib) and in China, the Company has received approval to market its anti-PD-1 antibody tislelizumab and markets ABRAXANE® (nanoparticle albumin-bound paclitaxel), REVLIMID® (lenalidomide), and VIDAZA® (azacitidine) under a license from Celgene Logistics Sarl, a Bristol-Myers Squibb companyi, and plans to market XGEVA® (denosumab) under a license from Amgenii. For more information please visit www.beigene.com.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com.

    Follow SpringWorks Therapeutics on social media: @SpringWorksTx and LinkedIn.

    BeiGene Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the encouraging pre-clinical data and therapeutic potential of BGB-3245 and plans for the operations of MapKure and clinical development of BGB-3245. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed products and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its technology and drugs; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGene's limited operating history and BeiGene's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates, as well as those risks more fully discussed in the section entitled "Risk Factors" in BeiGene's most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

    SpringWorks Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, statements regarding the development of BGB-3245 or other future research and development activities conducted by MapKure, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    BeiGene Investor Contact   BeiGene Media Contact
    Craig West   Liza Heapes
    +1 857-302-5189   +1 857-302-5663
    ir@beigene.com   media@beigene.com
         
    SpringWorks Investor and Media Contact
    Kim Diamond    
    Phone: 646-661-1255    
    Email: kdiamond@springworkstx.com    
         

    i ABRAXANE® is a registered trademark of Abraxis Bioscience LLC, a Bristol-Myers Squibb company; REVLIMID® and VIDAZA® are registered trademarks of Celgene Corporation, a Bristol-Myers Squibb company
    ii XGEVA® is a registered trademark of Amgen


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  48. STAMFORD, Conn., Feb. 18, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will present at the Cowen and Company 40th Annual Health Care Conference in Boston on Tuesday, March 3, 2020 at 8:40 a.m. Eastern Standard Time. A breakout question and answer session will immediately follow the presentation.

    To access the live webcast of SpringWorks' presentation, please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will…

    STAMFORD, Conn., Feb. 18, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will present at the Cowen and Company 40th Annual Health Care Conference in Boston on Tuesday, March 3, 2020 at 8:40 a.m. Eastern Standard Time. A breakout question and answer session will immediately follow the presentation.

    To access the live webcast of SpringWorks' presentation, please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for approximately 30 days following the conference.

    About SpringWorks Therapeutics
    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com.

    Follow SpringWorks Therapeutics on social media: @SpringWorksTx and LinkedIn.

    Contact:
    Kim Diamond
    Phone: 646-661-1255
    Email: kdiamond@springworkstx.com

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    • Increasing BCMA Surface Expression with Gamma Secretase Inhibitor Nirogacestat May Enable Deep and Durable Responses to ALLO-715, an Investigational Anti-BCMA Allogeneic CAR T Therapy

    SOUTH SAN FRANCISCO, Calif. and STAMFORD, Conn., Jan. 13, 2020 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (NASDAQ:ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor T cell (AlloCAR T™) therapies for cancer and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that they have entered into a clinical trial collaboration agreement. This agreement will…

    • Increasing BCMA Surface Expression with Gamma Secretase Inhibitor Nirogacestat May Enable Deep and Durable Responses to ALLO-715, an Investigational Anti-BCMA Allogeneic CAR T Therapy

    SOUTH SAN FRANCISCO, Calif. and STAMFORD, Conn., Jan. 13, 2020 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (NASDAQ:ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor T cell (AlloCAR T™) therapies for cancer and SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that they have entered into a clinical trial collaboration agreement. This agreement will evaluate ALLO-715, Allogene's investigational anti-B-cell maturation antigen (BCMA) AlloCAR T therapy in combination with SpringWorks' investigational gamma secretase inhibitor (GSI), nirogacestat, in patients with relapsed or refractory multiple myeloma.

    Gamma secretase inhibition prevents the cleavage and shedding of BCMA from the surface of myeloma cells. In preclinical models, nirogacestat has been shown to increase the cell surface density of BCMA and reduce levels of soluble BCMA, thereby enhancing the activity of BCMA-targeted therapies.1 In addition, emerging clinical data suggest that a GSI may increase antitumor efficacy of BCMA-targeted autologous CAR T therapy in patients with relapsed and refractory multiple myeloma.2 3

    "Autologous CAR T therapy has shown the potential for engineered cell therapy to treat multiple myeloma and provide significant benefits to patients. We believe allogeneic CAR T therapy is the next frontier in genetically engineered cell therapy for the treatment of cancer," said David Chang, M.D., Ph.D., President, CEO and Co-Founder of Allogene Therapeutics. "The search to find long-lasting and potentially curative therapies for patients with multiple myeloma continues to evolve. We are pleased with the progress of our ALLO-715 Phase 1 UNIVERSAL trial and are excited to explore the combination of ALLO-715 with nirogacestat as a means to further unlock the potential of allogeneic CAR T therapy in this disease."

    Under the terms of the agreement, Allogene will sponsor and conduct the Phase 1 study to evaluate the safety, tolerability and preliminary efficacy of the combination, and will assume all development costs associated with the study. Allogene and SpringWorks will form a joint development committee to oversee the clinical study, which is expected to commence in the second half of 2020 pending discussions with regulators.

    "Gamma secretase inhibition has emerged as a clinically validated mechanism to potentiate BCMA therapies and we believe that nirogacestat has the potential to become a cornerstone of BCMA combination therapy for patients with multiple myeloma," said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics. "We are delighted to partner with Allogene, a pioneer in the allogenic cell therapy field, to further explore nirogacestat in combination with an ‘off-the-shelf' CAR T therapy for these patients where the need for treatment options remains great."

    SpringWorks is currently enrolling patients in a global Phase 3, double-blind, randomized, placebo-controlled clinical trial (the DeFi Trial) to evaluate nirogacestat in adults with progressing desmoid tumors.

    About ALLO-715
    ALLO-715, an AlloCAR T therapy targeting B-cell maturation antigen (BCMA), is currently in Phase 1 development as a potential novel treatment for multiple myeloma. Multiple myeloma is characterized by abnormalities in plasma cells that reproduce uncontrollably in the bone marrow of people with the disease,4 multiple myeloma is incurable for most patients, and most patients relapse despite the treatments available.5 Preclinical study results for ALLO-715 were published in the journal Molecular Therapy validating the potential for an AlloCAR T to treat multiple myeloma and demonstrating the ability for ALLO-715 to sustain potent anti-tumor responses in pre-clinical models. Allogene initiated the UNIVERSAL study in the third quarter of 2019.

    ALLO-715 utilizes TALEN® gene-editing technology pioneered and owned by Cellectis. Allogene has an exclusive license to the Cellectis technology for allogeneic products directed at the BCMA target. Allogene holds global development and commercial rights for this investigational candidate.

    About Nirogacestat
    Nirogacestat is an investigational, oral, selective, small molecule gamma-secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is pursuing a combination therapy approach to evaluate nirogacestat as a BCMA potentiator across modalities by collaborating with industry leaders. To date, SpringWorks has entered into two clinical collaborations to evaluate nirogacestat in combination with GlaxoSmithKline's BCMA antibody-drug conjugate belantamab mafodotin and with Allogene's allogeneic BCMA CAR-T cell therapy ALLO-715.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors (June 2018) and from the European Commission for the treatment of soft tissue sarcoma (September 2019). The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis (November 2018 and August 2019).

    About Allogene Therapeutics
    Allogene Therapeutics, with headquarters in South San Francisco, is a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor T cell (AlloCAR T™) therapies for cancer. Led by a world-class management team with significant experience in cell therapy, Allogene is developing a pipeline of "off-the-shelf" CAR T cell therapy candidates with the goal of delivering readily available cell therapy on-demand, more reliably, and at greater scale to more patients. For more information, please visit www.allogene.com, and follow @AllogeneTx on Twitter and LinkedIn.

    About SpringWorks Therapeutics
    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com. Follow SpringWorks Therapeutics on social media: @SpringWorksTx and LinkedIn.

    Allogene Forward-Looking Statements
    This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The press release may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the timing and ability to initiate and advance a combination trial for nirogacestat and ALLO-715, the potential benefits of the combination trial, including the ability of nirogacestat to increase BCMA surface expression to enable a deep and durable response of ALLO-715, the ability to develop allogeneic CAR T therapies for cancer and the potential benefits of AlloCAR T therapy. Various factors may cause differences between Allogene's expectations and actual results as discussed in greater detail in Allogene's filings with the Securities and Exchange Commission (SEC), including without limitation in its Form 10-Q for the quarter ended September 30, 2019. Any forward-looking statements that are made in this press release speak only as of the date of this press release. Allogene assumes no obligation to update the forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

    SpringWorks Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, statements regarding the future research and development activities under the agreement with Allogene, the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    Allogene Media/Investor Contact:
    Christine Cassiano
    Chief Communications Officer
    (714) 552-0326
    Christine.Cassiano@allogene.com

    SpringWorks Media/Investor Contact:
    Kim Diamond
    Vice President, Communications and Investor Relations
    (646) 661-1255
    kdiamond@springworkstx.com

    _________________________

    1 Eastman et al., Abstract #4401 "Synergistic Activity of Belantamab Mafodotin (anti-BCMA immuno-conjugate) with Nirogacestat (PF-03084014, gamma-secretase inhibitor) in BCMA-Expressing Cancer Cell Lines", ASH 2019.

    2 Cowan et al., Abstract #204 "Efficacy and Safety of Fully Human BCMA CAR T Cells in Combination with a Gamma Secretase Inhibitor to Increase Bcma Surface Expression in Patients with Relapsed or Refractory Multiple Myeloma", ASH 2019.

    3 Blood. 2019 Nov 7;134(19):1585-1597. doi: 10.1182/blood.2019000050

    4 Multiple myeloma - Genetics Home Reference - NIH. Retrieved from https://ghr.nlm.nih.gov/condition/multiple-myeloma#

    5 Sonneveld P, Broijl A. Treatment of relapsed and refractory multiple myeloma. Haematologica. 2016;101(4):396-406

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  49. STAMFORD, Conn., Jan. 06, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will present at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco on Tuesday, January 14, 2020 at 1:30 p.m. Pacific Standard Time. A breakout question and answer session will immediately follow the presentation.

    To access the live webcast of SpringWorks' presentation, please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will…

    STAMFORD, Conn., Jan. 06, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that Saqib Islam, Chief Executive Officer, will present at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco on Tuesday, January 14, 2020 at 1:30 p.m. Pacific Standard Time. A breakout question and answer session will immediately follow the presentation.

    To access the live webcast of SpringWorks' presentation, please visit the "Events & Presentations" page within the Investors & Media section of the company's website at https://ir.springworkstx.com. A replay of the webcast will be available on SpringWorks' website for approximately 30 days following the conference.

    About SpringWorks Therapeutics
    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com.

    Follow SpringWorks Therapeutics on social media: @SpringWorksTx and LinkedIn.

    Contact:
    Kim Diamond
    Phone: 646-661-1255
    Email: kdiamond@springworkstx.com

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  50. STAMFORD, Conn., Dec. 09, 2019 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that its collaborator, GlaxoSmithKline, presented data evaluating SpringWorks' investigational gamma secretase inhibitor (GSI), nirogacestat (formerly PF-03084014), alone and in combination with GlaxoSmithKline's investigational anti-B-cell maturation antigen (BCMA) antibody-drug conjugate (ADC), belantamab mafodotin (formerly GSK2857916), in preclinical cell line models of human multiple myeloma and other lymphomas. The data demonstrated that treatment of BCMA-expressing cancer cell lines with…

    STAMFORD, Conn., Dec. 09, 2019 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced that its collaborator, GlaxoSmithKline, presented data evaluating SpringWorks' investigational gamma secretase inhibitor (GSI), nirogacestat (formerly PF-03084014), alone and in combination with GlaxoSmithKline's investigational anti-B-cell maturation antigen (BCMA) antibody-drug conjugate (ADC), belantamab mafodotin (formerly GSK2857916), in preclinical cell line models of human multiple myeloma and other lymphomas. The data demonstrated that treatment of BCMA-expressing cancer cell lines with nirogacestat led to significantly increased levels of cell surface expression of BCMA and corresponding decreases in shedding of BCMA, as measured by levels of soluble BCMA. Further, the combination of nirogacestat and belantamab mafodotin resulted in synergistic increases in cancer cell killing as compared to belantamab mafodotin alone, with an up to ~3,000-fold improvement in cytotoxicity. The data were presented in a poster presentation at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition.1

    "The data presented at ASH support the potential of nirogacestat to serve as a meaningful potentiator of BCMA-directed therapies," said Saqib Islam, Chief Executive Officer of SpringWorks. "We look forward to further evaluating the combination of nirogacestat and belantamab mafodotin in the clinic as we work to fully explore the role of nirogacestat in multiple myeloma and other BCMA-expressing cancer types."

    As previously announced, GlaxoSmithKline will sponsor and conduct a Phase 1b clinical trial to evaluate the safety, tolerability and preliminary efficacy of nirogacestat in combination with belantamab mafodotin in patients with relapsed or refractory multiple myeloma. This combination will be part of DREAMM-5, a platform trial being conducted by GlaxoSmithKline that is evaluating multiple belantamab mafodotin-containing combinations in separate sub-studies.2 SpringWorks expects the Phase 1b clinical trial of nirogacestat in combination with belantamab mafodotin to be initiated in the first quarter of 2020.

    Synergistic Activity of Belantamab Mafodotin (anti-BCMA immuno-conjugate) with Nirogacestat (PF-03084014, gamma-secretase inhibitor) in BCMA-Expressing Cancer Cell Lines: Poster No. 4401

    In a poster presentation at ASH, GlaxoSmithKline scientists presented findings from a preclinical study aimed at further enhancing belantamab mafodotin activity in BCMA-expressing cancer cell lines. The researchers sought to increase cell surface levels of BCMA by blocking shedding of BCMA using nirogacestat and to evaluate this effect on the activity of belantamab mafodotin by combining it with nirogacestat across a panel of human cancer cell lines.

    A 3-day proliferation assay on a panel of multiple myeloma and lymphoma cell lines with varying levels of BCMA expression was conducted. Results showed that adding nirogacestat enhanced multiple myeloma cell killing activity of belantamab mafodotin up to 3,000-fold and enabled sensitivity in belantamab mafodotin resistant cell lines outside of multiple myeloma. In a 24-hour assay to measure antibody-dependent cellular cytotoxicity (ADCC) activity, the study also showed increased effector function when combining nirogacestat with a BCMA monoclonal antibody.

    A Phase 1b clinical trial evaluating belantamab mafodotin in combination with nirogacestat in patients with relapsed or refractory multiple myeloma will be examined as a sub-study in GlaxoSmithKline's DREAMM-5 platform trial.

    About Nirogacestat

    Nirogacestat is an oral, selective, small molecule gamma-secretase inhibitor in Phase 3 clinical development for the treatment of desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

    In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat's ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma.

    Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors (June 2018) and from the European Commission for the treatment of soft tissue sarcoma (September 2019).  The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis (November 2018 and August 2019).

    About belantamab mafodotin (GSK2857916)
    Belantamab mafodotin is an investigational anti-B-cell maturation antigen (BCMA) antibody-drug conjugate in clinical development for patients with relapsed/refractory multiple myeloma and other advanced hematologic malignancies expressing BCMA.

    In 2017, belantamab mafodotin was awarded Breakthrough Therapy designation from the U.S. Food and Drug Administration and PRIME designation from the European Medicines Agency; these designations are intended to facilitate development of investigational medicines that have shown clinical promise for conditions where there is significant unmet need.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com.

    Follow SpringWorks Therapeutics on social media: @SpringWorksTx and LinkedIn.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, competition from other biopharmaceutical companies, and other risks identified in the section entitled "Risk Factors" in Item 1A of Part II of SpringWorks' Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks' subsequent filings with the Securities and Exchange Commission.. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    Kim Diamond
    Phone: 646-661-1255
    Email: kdiamond@springworkstx.com

    References:

    1 Eastman, S., Shelton, C., Gupta, I., Krueger, J., Blackwell, C., & Bojczuk, P. M. (2019, December). 4401 Synergistic Activity of Belantamab Mafodotin (anti-BCMA immuno-conjugate) with PF-03084014 (gamma-secretase inhibitor) in Bcma-Expressing Cancer Cell Lines. Poster session presented at the 61st American Society of Hematology Annual Meeting & Exposition, Orlando, FL.

    2 Richardson, P.G., Biswas, S., Holkova, B., Jackson, N., Netherway, T., Bao, W., …Opalinksa, J. (2019, December). 1857 Dreamm-5: Platform Trial Evaluating Belantamab Mafodotin (a BCMA-directed Immuno-conjugate) in Combination with Novel Agents in Relapsed or Refractory Multiple Myeloma (RRMM). Poster session presented at the 61st American Society of Hematology Annual Meeting & Exposition, Orlando, FL.

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  51. – Commenced Phase 2b ReNeu Clinical Trial for Patients with Neurofibromatosis Type 1-Associated Plexiform Neurofibromas (NF1-PN) –

    – Received Important Regulatory Designations for Nirogacestat and Mirdametinib – 

    – Completed $186 Million Initial Public Offering–

    STAMFORD, Conn., Nov. 12, 2019 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced financial results for the third quarter ended September 30, 2019, and provided an update on recent company developments.

    "The third quarter of 2019 was a productive period for SpringWorks as we completed a successful initial public offering…

    – Commenced Phase 2b ReNeu Clinical Trial for Patients with Neurofibromatosis Type 1-Associated Plexiform Neurofibromas (NF1-PN) –

    – Received Important Regulatory Designations for Nirogacestat and Mirdametinib – 

    – Completed $186 Million Initial Public Offering–

    STAMFORD, Conn., Nov. 12, 2019 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced financial results for the third quarter ended September 30, 2019, and provided an update on recent company developments.

    "The third quarter of 2019 was a productive period for SpringWorks as we completed a successful initial public offering while simultaneously achieving important regulatory and clinical development milestones, including dosing the first patient in our Phase 2b ReNeu trial of mirdametinib for patients with plexiform neurofibromas," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our team is focused on enrolling patients in our two potentially registrational trials in rare oncology indications and our Phase 1b trial in patients with RAS and RAF mutated solid tumors, and collaborating with our partners to initiate two additional Phase 1 studies in genetically defined cancers by early next year. We also continue to evaluate new business development opportunities to expand our pipeline and maximize the value of our portfolio."

    Third Quarter 2019 Business Highlights

    • Initiated Potentially Registrational ReNeu Clinical Trial: In October 2019, SpringWorks announced the initiation of the Phase 2b ReNeu clinical trial evaluating mirdametinib in children and adults with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN).
    • Completed Successful Initial Public Offering: In September 2019, SpringWorks completed its initial public offering of 10,350,000 shares of common stock, including the exercise in full by the underwriters of their option to purchase up to 1,350,000 additional shares of its common stock, at the public offering price of $18.00 per share. The Company raised $186.3 million in aggregate gross proceeds.
    • Received EU Orphan Drug Designation for Nirogacestat: In September 2019, the European Commission granted Orphan Drug Designation for nirogacestat for the treatment of soft tissue sarcoma.
    • Received Breakthrough Therapy Designation for Nirogacestat: In August 2019, the U.S. Food and Drug Administration granted Breakthrough Therapy Designation to nirogacestat for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.
    • Strengthened Leadership Team and Board of Directors: In August 2019, SpringWorks appointed Francis (Frank) I. Perier, Jr. as Chief Financial Officer and Alan Fuhrman to its Board of Directors and as Audit Committee Chair.
    • Received EU Orphan Drug Designation for Mirdametinib: In July 2019, the European Commission granted Orphan Drug Designation for mirdametinib for the treatment of neurofibromatosis type 1 (NF1).

    Third Quarter and Year to Date 2019 Financial Results

    • Research and Development (R&D) Expenses: R&D expenses as of September 30, 2019 were $10.7 million and $30.4 million for the third quarter and year-to-date periods, respectively, compared to $3.4 million and $6.2 million for the comparable periods of 2018, respectively. The increases in R&D expenses in 2019 are primarily attributable to increased clinical study and drug supply costs related to the ongoing Phase 3 DeFi and Phase 2b ReNeu trials, as well as growth in employee costs, including non-cash share-based compensation associated with increases in the number of R&D personnel.
    • General and Administrative (G&A) Expenses: G&A expenses as of September 30, 2019 were $4.6 million and $11.5 million for the third quarter and year-to-date periods, respectively, compared to $1.8 million and $5.9 million for the comparable periods of 2018, respectively. The increases in G&A expenses in 2019 are primarily attributable to growth in employee costs, including non-cash share-based compensation associated with increases in the number of G&A personnel, and increases in consulting and professional services related to the expansion of our business activities.
    • Net Loss Attributable to Common Stockholders: As of September 30, 2019, SpringWorks reported net losses of $16.8 million, or $1.77 loss per share, and $34.4 million, or $9.24 loss per share, for the third quarter and year-to-date periods respectively. This compares to net losses of $5.0 million, or $11.55 loss per share, and $11.6 million, or $44.20 loss per share, for the comparable periods of 2018, respectively.
    • Cash Position: Cash and cash equivalents were $343.8 million as of September 30, 2019. This includes net proceeds of $169.7 million from SpringWorks' initial public offering completed in September 2019.

    About SpringWorks Therapeutics

    SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks' strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio. For more information, please visit www.springworkstx.com.

    Follow SpringWorks Therapeutics on social media: @SpringWorksTx and LinkedIn.

    SpringWorks uses its website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the Company's website in the ‘Investors & Media' section. Accordingly, investors should monitor such portions of the SpringWorks website, in addition to following press releases, SEC filings and public conference calls and webcasts.

    Forward-Looking Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding SpringWorks' clinical trials and its strategy, business plans and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the timing for completion of SpringWorks' clinical trials of its product candidates, whether and when, if at all, SpringWorks' product candidates will receive approval from the U.S. Food and Drug Administration, or FDA, or other foreign regulatory authorities, competition from other biopharmaceutical companies, and other risks identified in SpringWorks' SEC filings, including its final prospectus for its initial public offering filed on September 13, 2019 and subsequent filings with the SEC. SpringWorks cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. SpringWorks disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent SpringWorks' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date.

    SpringWorks Therapeutics, Inc.
    Consolidated Balance Sheet
     (in thousands, except share, unit per-share and per-unit amounts)

      September 30, December 31,
        2019     2018  
      (unaudited)  
    Assets    
    Current assets:    
      Cash and cash equivalents $   343,825   $   45,648  
      Prepaid expenses and other current assets     689       1,382  
    Total current assets     344,514       47,030  
    Property and equipment, net     820       317  
    Investment in VIE (MapKure)    
    999
          -  
    Other assets     2,062       1,043  
    Total Assets $   348,395   $   48,390  
         
    Liabilities, Convertible Preferred Stock and Stockholders' (Deficit) Equity    
    Current liabilities:    
      Accounts payable $   2,157   $   774  
      Accrued expenses     7,682       2,568  
      Deferred rent     356       335  
    Total current liabilities     10,195       3,677  
    Long-term portion of deferred rent     881       1,152  
    Total liabilities     11,076       4,829  
         
    Commitments and contingencies    
    Convertible Preferred Stock:    
    Series A convertible preferred stock, $0.0001 par value; no shares authorized,    
    issued or outstanding at September 30, 2019 and December 31, 2018,    
    respectively.      -