VSTM Verastem Inc.

1.39
+0.03  (+2%)
Previous Close 1.36
Open 1.37
52 Week Low 0.83
52 Week High 4.6699
Market Cap $235,649,876
Shares 169,532,285
Float 150,642,162
Enterprise Value $164,834,907
Volume 4,936,479
Av. Daily Volume 4,495,030
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Upcoming Catalysts

Drug Stage Catalyst Date
Duvelisib (PRIMO)
Peripheral T-Cell Lymphoma (PTCL)
Phase 2
Phase 2
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Drug Pipeline

Drug Stage Notes
VS-6766 and Defactinib - FRAME
KRAS mutant advanced solid tumors
Phase 1
Phase 1
Phase 1/2 updated data noted 56% overall response rate (ORR) in patients with KRAS-G12 mutant low-grade serous ovarian cancer (LGSOC) and a 41% ORR in the overall LGSOC population - September 16, 2020.
Duvelisib - DUO
Chronic lymphocytic leukemia (CLL)//Small Lymphocytic Lymphoma (SLL)/Follicular Lymphoma
Approved
Approved
FDA Approval announced September 24, 2018.
Duvelisib (IPI-145)
Rheumatoid arthritis
Phase 2
Phase 2
Phase 2 failed to meet endpoint - Jan 2015
VS-6063
Mesothelioma prior to surgery
Phase 2
Phase 2
Phase 2 interim analysis showed lack of efficacy. Trial stopped. September 2015
Duvelisib (IPI-145) DYNAMO
Indolent non-Hodgkin lymphoma
Phase 2
Phase 2
Phase 2 data released June 2016 - short of expectations

Latest News

  1. Preliminary Data on VS-6766 and Defactinib Combination Continues to Show Encouraging Response Rates, Durability and a Favorable Safety Profile in KRAS Mutant Low-Grade Serous Ovarian Cancer in Investigator-Initiated Trial

    New Preclinical Data Demonstrating Synergy and Tumor Regression with G12C Inhibitors in Combination with VS-6766 and FAK Inhibitor In Vitro and In Vivo Also Presented

    Management to Host Investor Conference Call Today at 8:00 AM ET

    Verastem, Inc. (NASDAQ:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to advancing new medicines for patients battling cancer, today announced updated results from the ongoing investigator-initiated Phase 1/2 FRAME study evaluating VS-6766, its RAF/MEK inhibitor…

    Preliminary Data on VS-6766 and Defactinib Combination Continues to Show Encouraging Response Rates, Durability and a Favorable Safety Profile in KRAS Mutant Low-Grade Serous Ovarian Cancer in Investigator-Initiated Trial

    New Preclinical Data Demonstrating Synergy and Tumor Regression with G12C Inhibitors in Combination with VS-6766 and FAK Inhibitor In Vitro and In Vivo Also Presented

    Management to Host Investor Conference Call Today at 8:00 AM ET

    Verastem, Inc. (NASDAQ:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to advancing new medicines for patients battling cancer, today announced updated results from the ongoing investigator-initiated Phase 1/2 FRAME study evaluating VS-6766, its RAF/MEK inhibitor, in combination with defactinib, its FAK inhibitor, which demonstrated robust response rates, duration of response and a favorable safety profile in patients with low-grade serous ovarian cancer (LGSOC). These data will be presented in a virtual oral presentation today by Dr. Udai Banerji from The Institute of Cancer Research and The Royal Marsden at the 2nd Annual RAS-Targeted Drug Development Summit.

    "Existing treatments for patients with LGSOC are limited by either 10-25% response rates and/or increased toxicities, leading to high discontinuation rates. The FRAME data being presented today continue to demonstrate that RAF/MEK inhibition combined with FAK inhibition is well tolerated with a 56% overall response rate (ORR) in patients with KRAS-G12 mutant LGSOC and a 41% ORR in the overall LGSOC population. These data are still actively maturing with more than half of the patients still on treatment as of the data cutoff date, and responses in this patient population tend to deepen over time," said Dan Paterson, President and Chief Operating Officer of Verastem Oncology. "The response rates from this expanded data set are highly encouraging, consistent with the prior positive data from this study, and continue to speak to the significant potential of the VS-6766/defactinib combination for patients battling LGSOC."

    Verastem recently met with the Food and Drug Administration (FDA), and the FDA is supportive of the Company's adaptive study design for the planned Phase 2 registration-directed trials evaluating VS-6766 and defactinib in patients with recurrent LGSOC. Verastem expects to commence registration-directed clinical trials in both recurrent LGSOC and KRAS mutant non-small cell lung cancer by the end of 2020. Assuming a positive outcome from these registration-directed trials, Verastem expects to submit New Drug Applications to the FDA requesting accelerated approval for the VS-6766/defactinib combination in both LGSOC and KRAS mutant NSCLC.

    Updated Phase 1/2 FRAME Study Results in Patients with LGSOC

    Among the patients with LGSOC (n=17), the overall response rate (ORR) was 41% (7 of 17 patients), all partial responses (PRs). Among the patients with KRAS-G12 mutant LGSOC (n=9), the ORR was 56% (5 of 9 patients). Of the seven patients who responded, five had received one or more prior MEK inhibitors. In patients with KRAS mutant LGSOC receiving the recommended Phase 2 dosing (RP2D) regimen, the ORR was 50% (3 of 6 patients). The LGSOC cohort of the FRAME study remains ongoing, with 53% (9 of 17 patients) still on study as of the data cutoff date of August 17, 2020, with three patients on treatment for two years or more.

    The most common Grade ≥3 treatment-related adverse events (TEAEs) observed for the recommended Phase 2 dosing regimen were rash (4%) and elevated creatine kinase (4%). No patients discontinued from the FRAME study due to TEAEs.

    The novel, intermittent, combination dosing schedule used in the FRAME study continues to show encouraging clinical activity in patients with KRAS mutant LGSOC, including in patients who had previously progressed following treatment with a MEK inhibitor.

    "These updated safety and efficacy results in both KRAS mutant LGSOC as well as the overall LGSOC population are highly encouraging. Of particular note in this early look at the data, is the strong, 50% response rate, durability, and tumor reduction seen in patients with KRAS mutant LGSOC receiving the recommended Phase 2 dosing (RP2D) regimen, which is the regimen we will be taking into our upcoming registration-directed study," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "With nine out of 11 patients at RP2D active in the study and responses still developing, we look forward to continued data outputs from this study and we remain on track to commence Phase 2 registration-directed trials in both LGSOC and KRAS mutant NSCLC by the end of this year."

    Preclinical Results from Studies Investigating VS-6766 and Defactinib in Combination with G12C Inhibitors

    KRAS-G12C inhibitors may benefit from novel combination approaches to enhance their inhibition of the ERK signaling pathway. In the preclinical results that will be presented today at the meeting, VS-6766 showed synergy with KRAS-G12C inhibitors in reducing cancer cell viability across a panel of KRAS-G12C mutant NSCLC and colorectal cancer (CRC) cell lines. This enhanced cellular anti-cancer activity of the combination correlated with deeper and more durable inhibition of ERK pathway signaling relative to G12C inhibition alone. In KRAS-G12C mutant NSCLC models in mice, the RAF/MEK dual inhibitor VS-6766 was more effective than trametinib when compared at equal dose level both alone and in combination with a G12C inhibitor. In the KRAS-G12C NSCLC models tested, the combination of G12C inhibitor with VS-6766 and FAK inhibitor induced tumor regressions of ≥30% in all mice.

    "The anti-tumor effects of VS-6766 were generally comparable to those of KRAS-G12C inhibitors in KRAS-G12C NSCLC models in mice and were stronger than the effects of trametinib at a comparable dose," said Jonathan Pachter, Ph.D., Chief Scientific Officer of Verastem Oncology. "The tumor regressions observed with the triple combination of VS-6766, FAK inhibitor and G12C inhibitor were particularly striking. These data support clinical evaluation of VS-6766 and defactinib with G12C inhibitors in patients with KRAS-G12C mutant tumors."

    About the Phase 1/2 FRAME Study

    The FRAME study is an open-label, investigator-initiated study that is designed to assess safety, dose response and preliminary efficacy of the VS-6766/defactinib combination in patients with KRAS mutant solid tumors, including LGSOC, non-small cell lung cancer (NSCLC) and colorectal cancer (CRC). The FRAME study is being led by Dr. Banerji and is being conducted in the United Kingdom. In this study, VS-6766 was administered using a twice-weekly dose escalation schedule and was administered three out of every four weeks. Defactinib was administered using a twice-daily dose escalation schedule, also three out of every four weeks. Dose levels were assessed in three cohorts: cohort 1 (VS-6766 3.2mg, defactinib 200mg); cohort 2a (VS-6766 4mg, defactinib 200mg); and cohort 2b (VS-6766 3.2mg, defactinib 400mg). The recommended Phase 2 dose was determined to be VS-6766 3.2mg, defactinib 200mg. The FRAME study is now expanding to include new cohorts in pancreatic cancer, KRAS mutant endometrial cancer and KRAS-G12V mutant NSCLC.

    Details for the RAS-Targeted Drug Development Summit oral presentation are as follows:

    Title: Clinical Combinations: Dual RAF-MEK Inhibitor & FAK for Treatment of KRAS Mutant Cancers With a Focus on Low Grade Ovarian Cancer

    Lead author: Udai Banerji, The Institute of Cancer Research and The Royal Marsden

    Date and Time: Wednesday, September 16, 2020; 3:35 p.m. ET (12:35 p.m. PT)

    Title: Synergistic Combinations with the Dual RAF/MEK Inhibitor VS-6766 to Overcome Resistance Mechanisms

    Lead author: Jonathan Pachter, Verastem Oncology

    Date and Time: Wednesday, September 16, 2020; 12:10 p.m. ET (9:10 a.m. PT)

    Conference Call and Webcast Information

    The Verastem Oncology management team will host a conference call and webcast on Wednesday, September 16, 2020, at 8:00 AM ET to discuss the updated Phase 1/2 FRAME study data. The call can be accessed by dialing (877) 341-5660 (US and Canada) or (315) 625-3226 (international), five minutes prior to the start of the call and providing the passcode 5278200.

    The live, listen-only webcast of the conference call can be accessed by visiting the investors section of the Company's website at www.verastem.com. A replay of the webcast will be archived on the Company's website for 90 days following the call.

    About VS-6766

    VS-6766 (formerly known as CH5126766, CKI27 and RO5126766) is a unique inhibitor of the RAF/MEK signaling pathway. In contrast to other MEK inhibitors in development, VS-6766 blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows VS-6766 to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors.

    About Defactinib

    Defactinib (VS-6063) is an oral small molecule inhibitor of FAK and PYK2 that is currently being evaluated as a potential combination therapy for various solid tumors. The Company has received Orphan Drug designation for defactinib in ovarian cancer and mesothelioma in the US, EU and Australia. Preclinical research by Verastem Oncology scientists and collaborators at world-renowned research institutions has described the effect of FAK inhibition to enhance immune response by decreasing immuno-suppressive cells, increasing cytotoxic T cells, and reducing stromal density, which allows tumor-killing immune cells to enter the tumor.1,2

    About the VS-6766/Defactinib Combination

    RAS mutant tumors are present in ~30% of all human cancers, have historically presented a difficult treatment challenge and are often associated with significantly worse prognosis. Challenges associated with identifying new treatment options for these types of cancers include resistance to single agents, identifying tolerable combination regimens with MEK inhibitors and new RAS inhibitors in development addressing only a minority of all RAS mutated cancers.

    The combination of VS-6766 and defactinib has been found to be clinically active in patients with KRAS mt tumors. In an ongoing investigator-initiated Phase 1/2 FRAME study, the combination of VS-6766 and defactinib is being evaluated in patients with LGSOC, KRASmt NSCLC and colorectal cancer (CRC). Updated data from this study presented at the 2nd Annual RAS-Targeted Drug Development Summit in September 2020 demonstrated a 56% overall response rate and long duration of therapy among patients with KRAS-G12 mt LGSOC. Based on an observation of higher response rates seen in NSCLC patients with KRAS-G12V mutations in the study, Verastem will also be further exploring the role of VS-6766 and defactinib in KRAS-G12V NSCLC. The FRAME study was expanded in August 2020 to include new cohorts in pancreatic cancer, KRASmt endometrial cancer and KRAS-G12V NSCLC.

    About Verastem Oncology

    Verastem Oncology (NASDAQ:VSTM) is a development-stage biopharmaceutical company committed to the development and commercialization of new medicines to improve the lives of patients diagnosed with cancer. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition and focal adhesion kinase (FAK) inhibition. For more information, please visit www.verastem.com.

    Forward-Looking Statements Notice

    This press release includes forward-looking statements about Verastem Oncology's strategy, future plans and prospects, including statements related to the potential clinical value of the RAF/MEK/FAK combination and the timing of commencing a registration-directed trial for the RAF/MEK/FAK combination. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," "can," "promising" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement.

    Applicable risks and uncertainties include the risks and uncertainties, among other things, regarding: the success in the development and potential commercialization of our product candidates, including defactinib in combination with VS-6766; the occurrence of adverse safety events and/or unexpected concerns that may arise from additional data or analysis or result in unmanageable safety profiles as compared to their levels of efficacy; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the scope, timing, and outcome of any legal proceedings; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of our product candidates; whether preclinical testing of our product candidates and preliminary or interim data from clinical trials will be predictive of the results or success of ongoing or later clinical trials; that the timing, scope and rate of reimbursement for our product candidates is uncertain; that third-party payors (including government agencies) may not reimburse; that there may be competitive developments affecting our product candidates; that data may not be available when expected; that enrollment of clinical trials may take longer than expected; that our product candidates will experience manufacturing or supply interruptions or failures; that we will be unable to successfully initiate or complete the clinical development and eventual commercialization of our product candidates; that the development and commercialization of our product candidates will take longer or cost more than planned; that we or Chugai Pharmaceutical Co., Ltd. will fail to fully perform under the VS-6766 license agreement; that we may not have sufficient cash to fund our contemplated operations; that we may be unable to make additional draws under our debt facility or obtain adequate financing in the future through product licensing, co-promotional arrangements, public or private equity, debt financing or otherwise; that we will be unable to execute on our partnering strategies for defactinib in combination with VS-6766; that we will not pursue or submit regulatory filings for our product candidates; and that our product candidates will not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients.

    Other risks and uncertainties include those identified under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2019 as filed with the Securities and Exchange Commission (SEC) on March 11, 2020 and in any subsequent filings with the SEC. The forward-looking statements contained in this press release reflect Verastem Oncology's views as of the date hereof, and the Company does not assume and specifically disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law.

    References

    1 Chénard-Poirier, M. et al. Results from the biomarker-driven basket trial of RO5126766 (CH5127566), a potent RAF/MEK inhibitor, in RAS- or RAF-mutated malignancies including multiple myeloma. Journal of Clinical Oncology 2017: 35. 10.1200/JCO.2017.35.15_suppl.2506.

    2 https://clinicaltrials.gov, NCT03875820

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  2. New Results Evaluating Combination of VS-6766 and Defactinib to be Presented at the 2nd Annual RAS-Targeted Drug Development Summit

    Management to Host Investor Conference Call and Webcast on Wednesday, September 16 at 8:00 AM ET

    Verastem, Inc. (NASDAQ:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to advancing new medicines for patients battling cancer, today announced that management will host an investor conference call to discuss the updated clinical data from the low-grade serous ovarian cancer (LGSOC) cohort of the ongoing investigator-initiated Phase 1/2 FRAME study. The ongoing study is evaluating VS-6766, Verastem's RAF/MEK inhibitor, in combination with defactinib, its FAK inhibitor.

    The investor…

    New Results Evaluating Combination of VS-6766 and Defactinib to be Presented at the 2nd Annual RAS-Targeted Drug Development Summit

    Management to Host Investor Conference Call and Webcast on Wednesday, September 16 at 8:00 AM ET

    Verastem, Inc. (NASDAQ:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to advancing new medicines for patients battling cancer, today announced that management will host an investor conference call to discuss the updated clinical data from the low-grade serous ovarian cancer (LGSOC) cohort of the ongoing investigator-initiated Phase 1/2 FRAME study. The ongoing study is evaluating VS-6766, Verastem's RAF/MEK inhibitor, in combination with defactinib, its FAK inhibitor.

    The investor conference call is scheduled for Wednesday, September 16, 2020 at 8:00 a.m. ET. The conference call coincides with the oral presentation of this data at the 2nd Annual RAS-Targeted Drug Development Summit.

    The call will feature members of the Company's management team and Rachel Grisham, MD, Memorial Sloan Kettering Cancer Center, a medical oncologist and an expert in LGSOC.

    Verastem Oncology plans to commence a Phase 2 registration-directed trial investigating the VS-6766/defactinib combination in patients with recurrent LGSOC, as well as patients with KRAS-mutant NSCLC, by the end of 2020.

    Details for the RAS-Targeted Drug Development Summit oral presentation are as follows:

    Title: Clinical Combinations: Dual RAF-MEK Inhibitor & FAK for Treatment of KRAS Mutant Cancers With a Focus Low Grade Ovarian Cancer

    Lead author: Udai Banerji, Professor of Molecular Cancer Pharmacology at The Institute of Cancer Research, London, and Honorary Consultant in Medical Oncology, MBBS, MD, DNB, PhD, FRCP at The Royal Marsden NHS Foundation Trust, London.

    Date and Time: Wednesday, September 16, 2020; 3:35 p.m. ET (12:35 p.m. PT)

    Conference Call and Webcast Information

    The Verastem Oncology management team will host a conference call and webcast on Wednesday, September 16, 2020, at 8:00 AM ET to discuss the updated Phase 1/2 FRAME study data. The call can be accessed by dialing (877) 341-5660 (U.S. and Canada) or (315) 625-3226 (international), five minutes prior to the start of the call and providing the passcode 5278200.

    The live, listen-only webcast of the conference call can be accessed by visiting the investors section of the Company's website at www.verastem.com. A replay of the webcast will be archived on the Company's website for 90 days following the call.

    About VS-6766

    VS-6766 (formerly known as CH5126766, CKI27 and RO5126766) is a unique inhibitor of the RAF/MEK signaling pathway. In contrast to other MEK inhibitors in development, VS-6766 blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows VS-6766 to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors.

    About Defactinib

    Defactinib (VS-6063) is an oral small molecule inhibitor of FAK and PYK2 that is currently being evaluated as a potential combination therapy for various solid tumors. The Company has received Orphan Drug designation for defactinib in ovarian cancer and mesothelioma in the US, EU and Australia. Preclinical research by Verastem Oncology scientists and collaborators at world-renowned research institutions has described the effect of FAK inhibition to enhance immune response by decreasing immuno-suppressive cells, increasing cytotoxic T cells, and reducing stromal density, which allows tumor-killing immune cells to enter the tumor.1,2

    About the VS-6766/Defactinib Combination

    RAS mutant tumors are present in 30% of all human cancers and have historically presented a difficult treatment challenge and are often associated with significantly worse prognosis. Challenges associated with identifying new treatment options for these types of cancers include resistance to single agents, identifying tolerable combination regimens with MEK inhibitors and new RAS inhibitors in development addressing only a minority of all RAS mutated cancers.

    The combination of VS-6766 and defactinib has been found to be clinically active in KRAS mutant tumors (KRASmt). In an ongoing investigator-initiated Phase 1/2 FRAME study, the combination of VS-6766 and defactinib is being evaluated in patients with LGSOC, KRASmt NSCLC and colorectal cancer (CRC). Based on an observation of higher response rates seen in patients with KRAS-G12V mutations in the study, Verastem will also be further exploring the role of VS-6766 and defactinib in KRAS-G12V NSCLC. The FRAME study was expanded in August 2020 to include new cohorts in pancreatic cancer, KRASmt endometrial cancer and KRAS-G12V NSCLC.

    About Verastem Oncology

    Verastem Oncology (NASDAQ:VSTM) is a development-stage biopharmaceutical company committed to the advancement of new medicines to improve the lives of patients diagnosed with cancer. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition and focal adhesion kinase (FAK) inhibition. For more information, please visit www.verastem.com.

    Forward-Looking Statements Notice

    This press release includes forward-looking statements about Verastem Oncology's strategy, future plans and prospects, including statements related to the potential clinical value of the RAF/MEK/FAK combination and the timing of commencing a registration-directed trial for the RAF/MEK/FAK combination. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," "can," "promising" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement.

    Applicable risks and uncertainties include the risks and uncertainties, among other things, regarding: the success in the development and potential commercialization of our product candidates, including defactinib in combination with VS-6766 (; the occurrence of adverse safety events and/or unexpected concerns that may arise from additional data or analysis or result in unmanageable safety profiles as compared to their levels of efficacy; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the scope, timing, and outcome of any legal proceedings; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of our product candidates; whether preclinical testing of our product candidates and preliminary or interim data from clinical trials will be predictive of the results or success of ongoing or later clinical trials; that the timing, scope and rate of reimbursement for our product candidates is uncertain; that third-party payors (including government agencies) may not reimburse; that there may be competitive developments affecting our product candidates; that data may not be available when expected; that enrollment of clinical trials may take longer than expected; that our product candidates will experience manufacturing or supply interruptions or failures; that we will be unable to successfully initiate or complete the clinical development and eventual commercialization of our product candidates; that the development and commercialization of our product candidates will take longer or cost more than planned; that we or Chugai Pharmaceutical Co., Ltd. will fail to fully perform under the VS-6766 (CH5126766) license agreement; that we may not have sufficient cash to fund our contemplated operations; that we may be unable to make additional draws under our debt facility or obtain adequate financing in the future through product licensing, co-promotional arrangements, public or private equity, debt financing or otherwise; that we will be unable to execute on our partnering strategies for defactinib in combination with VS-6766; that we will not pursue or submit regulatory filings for our product candidates, and that our product candidates will not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients.

    Other risks and uncertainties include those identified under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2019 as filed with the Securities and Exchange Commission (SEC) on March 11, 2020 and in any subsequent filings with the SEC. The forward-looking statements contained in this press release reflect Verastem Oncology's views as of the date hereof, and the Company does not assume and specifically disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law.

    References

    1 Chénard-Poirier, M. et al. Results from the biomarker-driven basket trial of RO5126766 (CH5127566), a potent RAF/MEK inhibitor, in RAS- or RAF-mutated malignancies including multiple myeloma. Journal of Clinical Oncology 2017: 35. 10.1200/JCO.2017.35.15_suppl.2506.

    2 https://clinicaltrials.gov, NCT03875820

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  3. Verastem, Inc. (NASDAQ:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to developing and commercializing new medicines for patients battling cancer, today announced that management will present virtually at the following upcoming investor conferences:

    • H.C. Wainwright 22nd Annual Global Investment Conference on Monday, September 14, 2020 at 1:30 p.m. Eastern Time
    • Cantor Virtual Global Healthcare Conference on Thursday, September 17, 2020 at 3:20 p.m. Eastern Time

    A live webcast of the presentations will be available on the investors section of the Company's website at www.verastem.com. An archived presentation will be available for 90 days.

    About Verastem Oncology

    Verastem Oncology (NASDAQ:VSTM) is a…

    Verastem, Inc. (NASDAQ:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to developing and commercializing new medicines for patients battling cancer, today announced that management will present virtually at the following upcoming investor conferences:

    • H.C. Wainwright 22nd Annual Global Investment Conference on Monday, September 14, 2020 at 1:30 p.m. Eastern Time
    • Cantor Virtual Global Healthcare Conference on Thursday, September 17, 2020 at 3:20 p.m. Eastern Time

    A live webcast of the presentations will be available on the investors section of the Company's website at www.verastem.com. An archived presentation will be available for 90 days.

    About Verastem Oncology

    Verastem Oncology (NASDAQ:VSTM) is a development-stage biopharmaceutical company committed to the development and commercialization of new medicines to improve the lives of patients diagnosed with cancer. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition and focal adhesion kinase (FAK) inhibition. For more information, please visit www.verastem.com.

    View Full Article Hide Full Article
  4. Announced Path Forward for VS-6766 and Defactinib Combination Following Meeting with FDA

    Phase 2 Registration-Directed Trials Expected to Commence by Year End 2020 in Both Low-Grade Serous Ovarian Cancer and KRAS Mutant Non-Small Cell Lung Cancer

    Company Monetizes COPIKTRA® (duvelisib) Providing Cash Runway Until at Least 2024

    Verastem, Inc. (NASDAQ:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to developing and commercializing new medicines for patients battling cancer, today reported financial results for the three months ending June 30, 2020, and provided an overview of recent corporate highlights.

    "The first half of 2020 has been a time of transformational change at Verastem Oncology. We recently…

    Announced Path Forward for VS-6766 and Defactinib Combination Following Meeting with FDA

    Phase 2 Registration-Directed Trials Expected to Commence by Year End 2020 in Both Low-Grade Serous Ovarian Cancer and KRAS Mutant Non-Small Cell Lung Cancer

    Company Monetizes COPIKTRA® (duvelisib) Providing Cash Runway Until at Least 2024

    Verastem, Inc. (NASDAQ:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to developing and commercializing new medicines for patients battling cancer, today reported financial results for the three months ending June 30, 2020, and provided an overview of recent corporate highlights.

    "The first half of 2020 has been a time of transformational change at Verastem Oncology. We recently announced our newest strategic transaction, the sale of COPIKTRA to Secura Bio, which allows us to monetize this asset while focusing our resources and efforts on advancing the VS-6766 and defactinib combination program in KRAS mutant solid tumors," commented Brian Stuglik, Chief Executive Officer of Verastem Oncology. "We are now looking forward to a catalyst-driven second half of 2020, including reporting updated data from the LGSOC arm of the investigator-initiated Phase 1/2 FRAME study in September and commencing registration-directed clinical trials in low-grade serous ovarian cancer (LGSOC) and KRAS mutant non-small cell lung cancer (NSCLC) by the end of this year."

    Second Quarter 2020 and Recent Highlights

    • Announced Path Forward for VS-6766/Defactinib Combination in LGSOC Following Meeting with U.S. FDA. Verastem announced today that the company met with the FDA in July 2020 to discuss the registration-directed study design for the VS-6766/defactinib combination in patients with LGSOC. The FDA was supportive of the Company's development strategy and adaptive design for LGSOC. Verastem's NSCLC study will also be an adaptive design with a focus on patients with KRAS-G12V mutant tumors. Verastem intends to seek input from the FDA after completing the initial cohort of the lung cancer study. Verastem expects to commence registration-directed clinical trials for potential accelerated approval in LGSOC and KRAS mutant NSCLC by the end of 2020.



    • Selling COPIKTRA Franchise to Secura Bio in a Deal Totaling $311 Million, Plus Royalties. Verastem recently announced its entry into a definitive agreement to sell its global commercial and development rights to COPIKTRA in all oncology indications to Secura Bio, Inc. The transaction, which carries a total deal value of up to $311 million, plus royalties, will provide Verastem's current programs with a cash runway until at least 2024 and will allow the Company to focus its resources and efforts on the clinical development of VS-6766, its RAF/MEK inhibitor, and defactinib, its FAK inhibitor, in KRAS mutant solid tumors. Verastem is pursuing development of this combination in LGSOC and KRAS mutant NSCLC.



    • Presented Preliminary Results from Investigator-initiated Phase 1 FRAME Study Evaluating the Combination of VS-6766 and Defactinib in KRAS Mutant Solid Tumors at AACR 2020 Virtual Meeting I. In a virtual poster presentation, Udai Banerji, MBBS, MD, DNB, PhD, FRCP, NIHR, Professor of Molecular Cancer Pharmacology at The Institute of Cancer Research and Honorary Consultant in Medical Oncology at The Royal Marsden NHS Foundation Trust, highlighted data from this ongoing, open-label, dose-escalation and expansion study in patients with KRAS mutant advanced solid tumors, including LGSOC and NSCLC. Preliminary data demonstrated a 67% overall response rate and long duration of therapy among patients with LGSOC. Based on higher response rates seen in NSCLC patients with KRAS-G12V mutations, Verastem will also be further exploring the role of the VS-6766/defactinib combination in KRAS-G12V NSCLC. Expansion cohorts remain ongoing in LGSOC and NSCLC and the study will be expanding to include new cohorts in pancreatic, KRAS mutant endometrial and KRAS-G12V NSCLC.



    • Presented New Preclinical VS-6766/Defactinib Combination Data in Uveal Melanoma at AACR 2020 Virtual Meeting II. In this study, researchers identified and reinforced that FAK inhibition is a viable pathway to inhibit downstream from the GNAQ pathway, which is constitutively active in uveal melanoma. It was observed that co-targeting FAK and RAF/MEK signaling led to tumor collapse in uveal melanoma xenograft and liver metastasis models in vivo. Based on these encouraging results, Verastem plans to support an investigator-sponsored, Phase 2 clinical testing of the VS-6766/defactinib combination in uveal melanoma, which is expected to commence by the end of 2020.



    • Appointed John H. Johnson to the Board of Directors. In April, Verastem Oncology announced the appointment of John H. Johnson to its Board of Directors. Mr. Johnson's career spans multiple executive management roles at leading global corporations where he was responsible for overseeing oncology and immunology drug development initiatives and commercialization. Mr. Johnson will serve on the Compensation and Nominating and Governance Committees.

    Upcoming Milestones

    • Close transaction with Secura Bio during the third quarter of 2020.



    • Present updated data from the LGSOC cohort of the investigator-initiated Phase 1/2 FRAME study evaluating VS-6766 and defactinib in KRAS mutant solid tumors in September, including at the 2nd Annual RAS-Targeted Drug Development Conference on September 16, 2020.



    • Present new preclinical data from studies investigating VS-6766 and defactinib in combination with KRAS-G12C inhibitors in September, including at the 2nd Annual RAS-Targeted Drug Development Conference on September 16, 2020.



    • Commence registration-directed clinical trials in LGSOC and KRAS mutant NSCLC by the end of 2020.



    • Submit updated data from the NSCLC cohort of the investigator-initiated Phase 1/2 FRAME study to the International Association for the Study of Lung Cancer (IASLC) World Lung Cancer Conference, taking place in January 2021.

    Second Quarter 2020 Financial Results

    Net product revenue for the three months ending June 30, 2020 (2020 Quarter) was $4.2 million, compared to $3.0 million for the three months ending June 30, 2019 (2019 Quarter). License and collaboration revenue for both the 2020 Quarter and 2019 Quarter was $0.1 million.

    Total operating expenses for the 2020 Quarter were $25.6 million, compared to $41.4 million for the 2019 Quarter.

    Research and development (R&D) expense for the 2020 Quarter was $9.3 million, compared to $11.3 million for the 2019 Quarter. The decrease of $2.0 million, or 18%, was primarily related to a decrease in contract research organization (CRO) costs and lower employee related expense.

    Selling, general and administrative (SG&A) expense for the 2020 Quarter was $15.4 million, compared to $29.3 million for the 2019 Quarter. The decrease of $13.9 million, or 47%, primarily resulted from the company's shift in strategic direction which led to lower commercial program and employee related expense.

    Net loss for the 2020 Quarter was $23.0 million, or $0.14 per share (basic and diluted), compared to $42.2 million, or $0.57 per share (basic and diluted), for the 2019 Quarter.

    For the 2020 Quarter, non-GAAP adjusted net loss was $20.5 million, or $0.12 per share (diluted), compared to non-GAAP adjusted net loss of $35.6 million, or $0.48 per share (diluted), for the 2019 Quarter. Please refer to the GAAP to Non-GAAP Reconciliation attached to this press release.

    Verastem Oncology ended the second quarter of 2020 with cash, cash equivalents and short-term investments of $160.8 million.

    Financial Guidance and Outlook

    With the proceeds from the sale of COPIKTRA, Verastem has a cash runway until at least 2024 to deliver on the current programs for VS-6766 and defactinib, including clinical and regulatory milestones and development in LGSOC and KRASmt NSCLC. Verastem expects its 2020 operating expenses to be approximately 40% lower than its 2019 operating expenses. As a result of its new strategic direction and operating plans, along with the expected sale of the COPIKTRA franchise during the third quarter and associated transition activities, the Company expects total operating expenses for the full year 2020 to be in the range of $80 million to $90 million. Beginning in 2021 Verastem expects its annual operating expenses to be approximately $50 million.

    Use of Non-GAAP Financial Measures

    To supplement Verastem Oncology's condensed consolidated financial statements, which are prepared and presented in accordance with generally accepted accounting principles in the United States (GAAP), the Company uses the following non-GAAP financial measures in this press release: non-GAAP adjusted net loss and non-GAAP net loss per share. These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP. Management believes this non-GAAP information is useful for investors, taken in conjunction with the Company's GAAP financial statements, because it provides greater transparency and period-over-period comparability with respect to the Company's operating performance and can enhance investors' ability to identify operating trends in the Company's business. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of the Company's operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three months ended March 31, 2020 and 2019 are included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.

    About VS-6766

    VS-6766 (formerly known as CH5126766, CKI27 and RO5126766) is a unique inhibitor of the RAF/MEK signaling pathway. In contrast to other MEK inhibitors in development, VS-6766 blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows VS-6766 to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors.

    About Defactinib

    Defactinib (VS-6063) is an oral small molecule inhibitor of FAK and PYK2 that is currently being evaluated as a potential combination therapy for various solid tumors. The Company has received Orphan Drug designation for defactinib in ovarian cancer and mesothelioma in the US, EU and Australia. Preclinical research by Verastem Oncology scientists and collaborators at world-renowned research institutions has described the effect of FAK inhibition to enhance immune response by decreasing immuno-suppressive cells, increasing cytotoxic T cells, and reducing stromal density, which allows tumor-killing immune cells to enter the tumor.i,ii

    About the VS-6766/Defactinib Combination

    RAS mutant tumors are present in 30% of all human cancers and have historically presented a difficult treatment challenge and are often associated with significantly worse prognosis. Challenges associated with identifying new treatment options for these types of cancers include resistance to single agents, identifying tolerable combination regimens with MEK inhibitors and new RAS inhibitors in development addressing only a minority of all RAS mutated cancers.

    The combination of VS-6766 and defactinib has been found to be clinically active in KRAS mutant tumors. In an ongoing investigator-initiated Phase I/2 FRAME study, the combination of VS-6766 and defactinib is being evaluated in patients with LGSOC, KRAS mutant NSCLC and colorectal cancer (CRC). Preliminary data from this study presented at the American Association for Cancer Research (AACR) 2020 Virtual Annual Meeting I demonstrated a 67% overall response rate and long duration of therapy among patients with KRASmt LGSOC. Based on an observation of higher response rates seen in patients with KRAS-G12V mutations in the study, Verastem will also be further exploring the role of VS-6766 and defactinib in KRAS-G12V NSCLC. The FRAME study is expanding in August 2020 to include new cohorts in pancreatic, KRAS mutant endometrial and KRAS-G12V NSCLC.

    About COPIKTRA® (duvelisib)

    COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first approved dual inhibitor of PI3K-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells. PI3K signaling may lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.iii,iv,v COPIKTRA is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies and relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. COPIKTRA is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), for which it has received Fast Track status and Orphan Drug Designation, and is being investigated in combination with other agents through investigator-sponsored studies.vi For more information on COPIKTRA, please visit www.COPIKTRA.com. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

    About Verastem Oncology

    Verastem Oncology (NASDAQ:VSTM) is a commercial biopharmaceutical company committed to the development and commercialization of new medicines to improve the lives of patients diagnosed with cancer. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including phosphoinositide 3-kinase (PI3K), focal adhesion kinase (FAK) and RAF/MEK inhibition.

    Our first FDA approved product is available for the treatment of patients with certain types of indolent non-Hodgkin's lymphoma (iNHL).

    For more information, please visit www.verastem.com.

    Forward-Looking Statements Notice

    This press release includes forward-looking statements about Verastem Oncology's strategy, future plans and prospects, including statements related to the expected sale of COPIKTRA, the Company's future funding requirements and the potential clinical value of the RAF/MEK/FAK combination. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," "can," "promising" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement.

    Applicable risks and uncertainties include the risks and uncertainties, among other things, regarding: the satisfaction of closing conditions with respect to the sale of the COPIKTRA assets to Secura Bio; the ability of Secura Bio to achieve the clinical and sales milestones necessary to result in additional consideration payable to Verastem; the inherent uncertainty in forecasting expected funding needs of the Company in advancing its product candidates; the success in the development and potential commercialization of our product candidates, including defactinib in combination with VS-6766; the occurrence of adverse safety events and/or unexpected concerns that may arise from additional data or analysis or result in unmanageable safety profiles as compared to their levels of efficacy; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the scope, timing, and outcome of any legal proceedings; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of our product candidates; whether preclinical testing of our product candidates and preliminary or interim data from clinical trials will be predictive of the results or success of ongoing or later clinical trials; that the timing, scope and rate of reimbursement for our product candidates is uncertain; that third-party payors (including government agencies) may not reimburse; that there may be competitive developments affecting our product candidates; that data may not be available when expected; that enrollment of clinical trials may take longer than expected; that our product candidates will experience manufacturing or supply interruptions or failures; that we will be unable to successfully initiate or complete the clinical development and eventual commercialization of our product candidates; that the development and commercialization of our product candidates will take longer or cost more than planned; that we may not have sufficient cash to fund our contemplated operations; that we may be unable to make additional draws under our debt facility or obtain adequate financing in the future through product licensing, co-promotional arrangements, public or private equity, debt financing or otherwise; that we will be unable to execute on our partnering strategies for defactinib in combination with VS-6766; that we will not pursue or submit regulatory filings for our product candidates, and that our product candidates will not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients.

    Other risks and uncertainties include those identified under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2019 as filed with the Securities and Exchange Commission (SEC) on March 11, 2020 and in any subsequent filings with the SEC. The forward-looking statements contained in this press release reflect Verastem Oncology's views as of the date hereof, and the Company does not assume and specifically disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law.

    Verastem, Inc.

    Condensed Consolidated Balance Sheets

    (in thousands)

    (unaudited)

     

     

     

     

     

     

     

     

     

     

    June 30,

     

    December 31,

     

     

     

    2020

     

    2019

     

     

     

     

     

     

     

     

    Cash, cash equivalents, & investments

     

    $

    125,328

     

    $

    75,506

     

    Accounts receivable, net

     

     

    1,500

     

     

    2,524

     

    Inventory

     

     

    6,316

     

     

    3,096

     

    Restricted cash, Prepaid expenses and other current assets

     

     

    11,448

     

     

    3,835

     

    Property and equipment, net

     

     

    791

     

     

    947

     

    Intangible assets, net

     

     

    19,223

     

     

    20,008

     

    Right-of-use asset, net

     

     

    2,909

     

     

    3,077

     

    Restricted cash and other assets

     

     

    31,017

     

     

    36,053

     

    Total assets

     

    $

    198,532

     

    $

    145,046

     

     

     

     

     

     

     

     

     

    Current Liabilities

     

    $

    28,784

     

    $

    29,890

     

    Long-term debt

     

     

    30,899

     

     

    35,067

     

    Convertible senior notes

     

     

    20,381

     

     

    68,556

     

    Lease Liability, long-term

     

     

    3,225

     

     

    3,489

     

    Other liabilities

     

     

    870

     

     

    870

     

    Stockholders' equity

     

     

    114,373

     

     

    7,174

     

    Total liabilities and stockholders' equity

     

    $

    198,532

     

    $

    145,046

     

     

    Verastem, Inc.

    Condensed Consolidated Statements of Operations

    (in thousands, except per share amounts)

    (unaudited)

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    Three months ended June 30,

     

    Six months ended June 30,

     

     

     

    2020

     

    2019

     

    2020

     

    2019

     

    Revenue:

     

     

     

     

     

     

     

     

     

     

     

     

     

    Product revenue, net

     

    $

    4,235

     

    $

    3,019

     

    $

    9,269

     

    $

    4,690

     

    License and collaboration revenue

     

     

    72

     

     

    117

     

     

    94

     

     

    117

     

    Total revenue

     

     

    4,307

     

     

    3,136

     

     

    9,363

     

     

    4,807

     

    Operating expenses:

     

     

     

     

     

     

     

     

     

     

     

     

     

    Cost of sales - product

     

     

    392

     

     

    377

     

     

    887

     

     

    534

     

    Cost of sales - intangible amortization

     

     

    393

     

     

    392

     

     

    785

     

     

    785

     

    Research and development

     

     

    9,344

     

     

    11,346

     

     

    20,268

     

     

    21,103

     

    Selling, general and administrative

     

     

    15,442

     

     

    29,298

     

     

    35,046

     

     

    55,331

     

    Total operating expenses

     

     

    25,571

     

     

    41,413

     

     

    56,986

     

     

    77,753

     

    Loss from operations

     

     

    (21,264)

     

     

    (38,277)

     

     

    (47,623)

     

     

    (72,946)

     

    Other expense

     

     

     

     

     

     

    (1,313)

     

     

     

    Interest income

     

     

    122

     

     

    1,268

     

     

    478

     

     

    2,765

     

    Interest expense

     

     

    (1,868)

     

     

    (5,185)

     

     

    (12,542)

     

     

    (10,115)

     

    Net Loss

     

    $

    (23,010)

     

    $

    (42,194)

     

    $

    (61,000)

     

    $

    (80,296)

     

    Net loss per share—basic and diluted

     

    $

    (0.14)

     

    $

    (0.57)

     

    $

    (0.45)

     

    $

    (1.09)

     

    Weighted average common shares outstanding used in computing net loss per share—basic and diluted

     

     

    165,395

     

     

    73,877

     

     

    136,775

     

     

    73,865

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    Verastem, Inc.

    Reconciliation of GAAP to Non-GAAP Financial Information

    (in thousands, except per share amounts)

    (unaudited)

     

     

     

    Three months ended June 30,

     

    Six months ended June 30,

     

     

    2020

     

    2019

     

    2020

     

    2019

    Net Loss Reconciliation

     

     

     

     

     

     

     

     

     

     

     

     

    Net Loss (GAAP basis)

     

    $

    (23,010)

     

    $

    (42,194)

     

    $

    (61,000)

     

    $

    (80,296)

    Adjust:

     

     

     

     

     

     

     

     

     

     

     

     

    Amortization of acquired intangible asset

     

     

    393

     

     

    392

     

     

    785

     

     

    785

    Stock-based compensation expense

     

     

    1,659

     

     

    3,065

     

     

    3,029

     

     

    5,313

    Non-cash interest, net

     

     

    480

     

     

    1,207

     

     

    9,259

     

     

    2,815

    Severance and Other

     

     

    11

     

     

    1,957

     

     

    1,798

     

     

    1,994

    Change in fair value of derivative

     

     

     

     

     

     

    1,313

     

     

    Chugai license payment

     

     

     

     

     

     

    3,000

     

     

    Adjusted Net Loss (non-GAAP basis)

     

    $

    (20,467)

     

    $

    (35,573)

     

    $

    (41,816)

     

    $

    (69,389)

     

     

     

     

     

     

     

     

     

     

     

     

     

    Reconciliation of Net Loss Per Share

     

     

     

     

     

     

     

     

     

     

     

     

    Net Loss per share – diluted

    (GAAP Basis)

     

     

    (0.14)

     

     

    (0.57)

     

     

    (0.45)

     

     

    (1.09)

    Adjust per diluted share

     

     

     

     

     

     

     

     

     

     

     

     

    Amortization of acquired intangible asset

     

     

     

     

     

     

    0.01

     

     

    0.01

    Stock-based compensation expense

     

     

    0.01

     

     

    0.04

     

     

    0.02

     

     

    0.07

    Non-cash interest, net

     

     

    0.01

     

     

    0.02

     

     

    0.07

     

     

    0.04

    Severance and Other

     

     

     

     

    0.03

     

     

    0.01

     

     

    0.03

    Change in fair value of derivative

     

     

     

     

     

     

    0.01

     

     

    Chugai license payment

     

     

     

     

     

     

    0.02

     

     

    Adjusted Net Loss per share – diluted

    (non-GAAP Basis)

     

    $

    (0.12)

     

    $

    (0.48)

     

    $

    (0.31)

     

    $

    (0.94)

    Weighted average common shares outstanding used in computing net loss per share—diluted

     

     

    165,395

     

     

    73,877

     

     

    136,775

     

     

    73,865

     
     

    i Gerber D. et al. Phase 2 study of the focal adhesion kinase inhibitor defactinib (VS-6063) in previously treated advanced KRAS mutant non-small cell lung cancer. Lung Cancer 2020: 139:60-67.

    ii Chénard-Poirier, M. et al. Results from the biomarker-driven basket trial of RO5126766 (CH5127566), a potent RAF/MEK inhibitor, in RAS- or RAF-mutated malignancies including multiple myeloma. Journal of Clinical Oncology 2017: 35. 10.1200/JCO.2017.35.15_suppl.2506.

    iii Winkler D.G., Faia K.L., DiNitto J.P. et al. PI3K-delta and PI3K-gamma inhibition by IPI-145 abrogates immune responses and suppresses activity in autoimmune and inflammatory disease models. Chem Biol 2013; 20:1-11.

    iv Reif K et al. Cutting Edge: Differential Roles for Phosphoinositide 3 kinases, p110-gamma and p110-delta, in lymphocyte chemotaxis and homing. J Immunol 2004:173:2236-2240.

    v Schmid M et al. Receptor Tyrosine Kinases and TLR/IL1Rs Unexpectedly activate myeloid cell PI3K, a single convergent point promoting tumor inflammation and progression. Cancer Cell 2011;19:715-727.

    vi www.clinicaltrials.gov, NCT03372057.

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  5. Verastem Will Receive $70 Million Up-Front with Total Deal Value Up to $311 Million, Plus Double-Digit Sales Royalties

    Upon Closing, Verastem's Current Programs Will Be Funded Until At Least 2024 to Develop VS-6766 and Defactinib in Low-Grade Serous Ovarian Cancer and KRAS Mutant Non-Small Cell Lung Cancer

    Phase 2 Registration-Directed Trials Expected to Commence by Year End 2020 in Both Low-Grade Serous Ovarian Cancer and KRAS Mutant Non-Small Cell Lung Cancer

    Enrollment in Ongoing Investigator-Initiated Phase 1/2 FRAME Study of VS-6766 and Defactinib Now Expanding to Include Pancreatic, KRAS Mutant Endometrial and KRAS-G12V Non-Small Cell Lung Cancer Cohorts

    Verastem, Inc. (NASDAQ:VSTM) (also known as Verastem Oncology), a biopharmaceutical…

    Verastem Will Receive $70 Million Up-Front with Total Deal Value Up to $311 Million, Plus Double-Digit Sales Royalties

    Upon Closing, Verastem's Current Programs Will Be Funded Until At Least 2024 to Develop VS-6766 and Defactinib in Low-Grade Serous Ovarian Cancer and KRAS Mutant Non-Small Cell Lung Cancer

    Phase 2 Registration-Directed Trials Expected to Commence by Year End 2020 in Both Low-Grade Serous Ovarian Cancer and KRAS Mutant Non-Small Cell Lung Cancer

    Enrollment in Ongoing Investigator-Initiated Phase 1/2 FRAME Study of VS-6766 and Defactinib Now Expanding to Include Pancreatic, KRAS Mutant Endometrial and KRAS-G12V Non-Small Cell Lung Cancer Cohorts

    Verastem, Inc. (NASDAQ:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to advancing new medicines for patients battling cancer, today announced that it has entered into a definitive agreement to sell its global commercial and development rights to COPIKTRA (duvelisib), its marketed oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first FDA-approved dual inhibitor of PI3K-delta and PI3K-gamma, to Secura Bio, Inc., an integrated biopharmaceutical company dedicated to the worldwide commercialization of significant oncology therapies.

    Verastem's sale of COPIKTRA follows the Company's previously announced strategic direction to focus on maximizing the broad potential of its RAF/MEK inhibitor (VS-6766) and FAK inhibitor (defactinib) program in KRAS mutant (KRASmt) solid tumors. Upon closing of the transaction with Secura Bio, Verastem will be dedicated to the development of this program and to deliver on clinical and regulatory milestones for the first potential indications in low-grade serous ovarian cancer (LGSOC) and KRASmt non-small cell lung cancer (NSCLC). Both LGSOC and KRASmt NSCLC are areas of high unmet patient need as there are no approved treatments and existing therapies have low response rates.

    "By focusing our expertise and efforts on rapidly advancing the RAF/MEK/FAK development program, we believe we will be providing the best path forward for patients, customers, our shareholders and our company. These strategic decisions will enable us to best deliver on our mission to advance new medicines on behalf of cancer patients," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "The agreement with Secura Bio will ensure COPIKTRA continues to help more patients, leveraging the established commercial structure, support of ongoing clinical study and potential expansion into new indications."

    Terms of the Definitive Sale Agreement

    Verastem will receive an up-front payment of $70 million upon the closing of the transaction and is eligible to receive up to a total deal value of $311 million if certain regulatory and sales-based milestones are successfully met by Secura Bio and COPIKTRA's other rest-of-world partners, including:

    • A total of $45 million from two separate milestone payments for U.S. Food and Drug Administration (FDA) and European Medicines Agency approvals of COPIKTRA with label indicated for peripheral T-cell lymphoma
    • A total of $50 million for cumulative worldwide net sales of COPIKTRA beginning at $100 million of cumulative net sales
    • Verastem will receive low double-digit royalties on net sales over $100 million in U.S., Europe and the United Kingdom
    • Verastem will also receive 50% of licensing milestones (up to $146 million) and royalties outside of U.S., Europe and the United Kingdom

    In exchange, Secura Bio will receive an exclusive worldwide license for the research, development, commercialization and manufacture of COPIKTRA in all oncology indications. Secura Bio will assume all operational and financial responsibility for activities that were previously part of Verastem's duvelisib program, including commercialization efforts in the United States and Europe, ongoing clinical trials, Verastem's partnerships with Yakult, CSPC and Sanofi and existing royalty obligations. Secura Bio and Verastem are also in discussions related to the transfer of Verastem's field sales and medical professionals.

    The transaction with Secura Bio is subject to customary closing conditions and is expected to close in the third quarter of 2020.*

    VS-6766 and Defactinib Program Progress and Registration-Directed Trials

    Verastem announced today that the company met with the FDA in July 2020 to discuss the registration-directed study design for the VS-6766/defactinib combination in patients with LGSOC. The FDA was supportive of the Company's development strategy and adaptive design for LGSOC.

    Verastem's NSCLC study will also be an adaptive design with a focus on patients with KRAS-G12V mutant tumors. Verastem intends to seek input from the FDA after completing the initial cohort of the lung cancer study. Verastem expects to commence registration-directed clinical trials for potential accelerated approval in LGSOC and KRASmt NSCLC by the end of 2020.

    Verastem is continuing its clinical collaboration with the Drug Development Unit at ICR/Royal Marsden Hospital. The ongoing investigator-initiated Phase 1/2 FRAME study evaluating the combination of VS-6766 with defactinib in LGSOC, KRASmt NSCLC and colorectal cancer (CRC) has resumed normal accrual and reporting rates following the global lockdown resulting from the COVID-19 pandemic. The FRAME study is now expanding to include new cohorts in pancreatic cancer, KRASmt endometrial cancer and KRAS-G12V NSCLC. Verastem expects that additional data from the LGSOC cohort of the FRAME study will be made available in September, including presentation at the 2nd Annual RAS-Targeted Drug Development Conference. The Company also expects that additional data from the NSCLC cohort of the FRAME study will be submitted to the International Association for the Study of Lung Cancer (IASLC) World Lung Cancer Conference, taking place in January 2021.

    The Company has also begun preclinical combination studies investigating VS-6766 and defactinib in combination with KRAS-G12C inhibitors and initial data will be presented at the 2nd Annual RAS-Targeted Drug Development Conference. Based on the positive preclinical data presented at the AACR 2020 Virtual Annual Meeting II, Verastem plans to support a Phase 2 investigator-initiated study evaluating the combination of VS-6766 and defactinib in uveal melanoma, which is expected to begin in late 2020.

    Corporate and Financial Overview

    With the sale of COPIKTRA, Verastem will become a focused development company with reduced annual expenses of approximately $50 million. The company is in a position of financial strength with a cash runway expected to fund the clinical and regulatory milestones and development of VS-6766 and defactinib in LGSOC and KRASmt NSCLC until at least 2024.

    About VS-6766

    VS-6766 (formerly known as CH5126766, CKI27 and RO5126766) is a unique inhibitor of the RAF/MEK signaling pathway. In contrast to other MEK inhibitors in development, VS-6766 blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows VS-6766 to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors.

    About Defactinib

    Defactinib (VS-6063) is an oral small molecule inhibitor of FAK and PYK2 that is currently being evaluated as a potential combination therapy for various solid tumors. The Company has received Orphan Drug designation for defactinib in ovarian cancer and mesothelioma in the US, EU and Australia. Preclinical research by Verastem Oncology scientists and collaborators at world-renowned research institutions has described the effect of FAK inhibition to enhance immune response by decreasing immuno-suppressive cells, increasing cytotoxic T cells, and reducing stromal density, which allows tumor-killing immune cells to enter the tumor.1,2

    About the VS-6766/Defactinib Combination

    RAS mutant tumors are present in 30% of all human cancers and have historically presented a difficult treatment challenge and are often associated with significantly worse prognosis. Challenges associated with identifying new treatment options for these types of cancers include resistance to single agents, identifying tolerable combination regimens with MEK inhibitors and new RAS inhibitors in development addressing only a minority of all RAS mutated cancers.

    The combination of VS-6766 and defactinib has been found to be clinically active in KRASmt. In an ongoing investigator-initiated Phase I/2 FRAME study, the combination of VS-6766 and defactinib is being evaluated in patients with LGSOC, KRASmt NSCLC and colorectal cancer (CRC). Preliminary data from this study presented at the American Association for Cancer Research (AACR) 2020 Virtual Annual Meeting I demonstrated a 67% overall response rate and long duration of therapy among patients with KRASmt LGSOC. Based on an observation of higher response rates seen in patients with KRAS-G12V mutations in the study, Verastem will also be further exploring the role of VS-6766 and defactinib in KRAS-G12V NSCLC. The FRAME study is expanding in August 2020 to include new cohorts in pancreatic, KRASmt endometrial and KRAS-G12V NSCLC.

    About COPIKTRA® (duvelisib)

    COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first approved dual inhibitor of PI3K-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells. PI3K signaling may lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.3,4,5 COPIKTRA is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies and relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. COPIKTRA is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), for which it has received Fast Track status and Orphan Drug Designation, and is being investigated in combination with other agents through investigator-sponsored studies.6 For more information on COPIKTRA, please visit www.COPIKTRA.com. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

    *MTS Health Partners, L.P and Ropes & Gray acted as advisors to Verastem Oncology on this transaction.

    About Verastem Oncology

    Verastem Oncology (NASDAQ:VSTM) is a commercial biopharmaceutical company committed to the development and commercialization of new medicines to improve the lives of patients diagnosed with cancer. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including phosphoinositide 3-kinase (PI3K), focal adhesion kinase (FAK) and RAF/MEK inhibition.

    Our first FDA approved product is available for the treatment of patients with certain types of indolent non-Hodgkin's lymphoma (iNHL).

    For more information, please visit www.verastem.com.

    Forward-Looking Statements Notice

    This press release includes forward-looking statements about Verastem Oncology's strategy, future plans and prospects, including statements related to the expected sale of COPIKTRA, the Company's future funding requirements and the potential clinical value of the RAF/MEK/FAK combination. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," "can," "promising" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement.

    Applicable risks and uncertainties include the risks and uncertainties, among other things, regarding: the satisfaction of closing conditions with respect to the sale of the COPIKTRA assets to Secura Bio; the ability of Secura Bio to achieve the clinical and sales milestones necessary to result in additional consideration payable to Verastem; the inherent uncertainty in forecasting expected funding needs of the Company in advancing its product candidates; the success in the development and potential commercialization of our product candidates, including defactinib in combination with VS-6766; the occurrence of adverse safety events and/or unexpected concerns that may arise from additional data or analysis or result in unmanageable safety profiles as compared to their levels of efficacy; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the scope, timing, and outcome of any legal proceedings; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of our product candidates; whether preclinical testing of our product candidates and preliminary or interim data from clinical trials will be predictive of the results or success of ongoing or later clinical trials; that the timing, scope and rate of reimbursement for our product candidates is uncertain; that third-party payors (including government agencies) may not reimburse; that there may be competitive developments affecting our product candidates; that data may not be available when expected; that enrollment of clinical trials may take longer than expected; that our product candidates will experience manufacturing or supply interruptions or failures; that we will be unable to successfully initiate or complete the clinical development and eventual commercialization of our product candidates; that the development and commercialization of our product candidates will take longer or cost more than planned; that we may not have sufficient cash to fund our contemplated operations; that we may be unable to make additional draws under our debt facility or obtain adequate financing in the future through product licensing, co-promotional arrangements, public or private equity, debt financing or otherwise; that we will be unable to execute on our partnering strategies for defactinib in combination with VS-6766; that we will not pursue or submit regulatory filings for our product candidates, and that our product candidates will not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients.

    Other risks and uncertainties include those identified under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2019 as filed with the Securities and Exchange Commission (SEC) on March 11, 2020 and in any subsequent filings with the SEC. The forward-looking statements contained in this press release reflect Verastem Oncology's views as of the date hereof, and the Company does not assume and specifically disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law.

    1 Gerber D. et al. Phase 2 study of the focal adhesion kinase inhibitor defactinib (VS-6063) in previously treated advanced KRAS mutant non-small cell lung cancer. Lung Cancer 2020: 139:60-67.

    2 Chénard-Poirier, M. et al. Results from the biomarker-driven basket trial of RO5126766 (CH5127566), a potent RAF/MEK inhibitor, in RAS- or RAF-mutated malignancies including multiple myeloma. Journal of Clinical Oncology 2017: 35. 10.1200/JCO.2017.35.15_suppl.2506.

    3 Winkler D.G., Faia K.L., DiNitto J.P. et al. PI3K-delta and PI3K-gamma inhibition by IPI-145 abrogates immune responses and suppresses activity in autoimmune and inflammatory disease models. Chem Biol 2013; 20:1-11.

    4 Reif K et al. Cutting Edge: Differential Roles for Phosphoinositide 3 kinases, p110-gamma and p110-delta, in lymphocyte chemotaxis and homing. J Immunol 2004:173:2236-2240.

    5 Schmid M et al. Receptor Tyrosine Kinases and TLR/IL1Rs Unexpectedly activate myeloid cell PI3K, a single convergent point promoting tumor inflammation and progression. Cancer Cell 2011;19:715-727.

    6 www.clinicaltrials.gov, NCT03372057.

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