SWTX SpringWorks Therapeutics Inc.

85.7
-2.56  -3%
Previous Close 88.26
Open 87.85
52 Week Low 36.71
52 Week High 96.48
Market Cap $4,204,538,413
Shares 49,061,125
Float 22,911,031
Enterprise Value $3,729,402,412
Volume 214,198
Av. Daily Volume 228,074
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Upcoming Catalysts

Drug Stage Catalyst Date
Mirdametinib and lifirafenib
Solid tumors
Phase 1b
Phase 1b
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Nirogacestat and BLENREP (Belantamab)
Multiple Myeloma
Phase 1b
Phase 1b
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Mirdametinib - ReNeu
Neurofibromas
Phase 2b
Phase 2b
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Nirogacestat (DeFi)
Desmoid tumors
Phase 3
Phase 3
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BGB-3245
Solid tumors
Phase 1/2
Phase 1/2
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Drug Pipeline

Drug Stage Notes
Mirdametinib
Low-Grade Glioma
Phase 1/2
Phase 1/2
Phase 1/2 trial initiation announced June 15, 2021.
Nirogacestat and Teclistamab
Multiple myeloma
Phase 1b
Phase 1b
Phase 1b initiation of dosing announced April 1, 2021.
Nirogacestat
Desmoid tumors - children
Phase 2
Phase 2
Phase 2 trial commenced recruitment in September 2020.

Latest News

  1. 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:



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  2. 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

    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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  3. 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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  4. 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

    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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  5. 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

    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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