Latest news with #VerastemOncology
Yahoo
14-07-2025
- Health
- Yahoo
Verastem's avutometinib combination shows 31% ORR in ovarian cancer trial
Verastem Oncology has reported primary analysis data from the two-part multicentre Phase II RAMP 201 trial, demonstrating that avutometinib plus defactinib showed a confirmed overall response rate (ORR) of 31% in treating recurrent low-grade serous ovarian cancer (LGSOC) patients. The analysis was published in the Journal of Clinical Oncology (JCO). The parallel cohort, adaptive, open-label, randomised study assessed avutometinib alone and in conjunction with defactinib in those with recurrent LGSOC. These patients had undergone a median of three previous therapy lines, including hormonal therapy, chemotherapy, bevacizumab, and MEK inhibitors. Part A of the trial determined the choice of the go-forward regimen, which was the combination therapy against avutometinib alone, based on ORRs. Parts B and C, the trial's expansion phases, assessed the safety and efficacy of the go-forward regimen of 3.2mg of avutometinib bi-weekly, and 200mg of defactinib twice a day. Part D of the trial assessed a low dose of the combination to inform the reduction of the individualised dose. The data published stated that the combination resulted in an ORR of 31% in 109 evaluable subjects in the trial. The response rate was also higher in those with a KRAS mutation, at 44%, compared to 17% in KRAS wild-type patients. The median duration of response (DOR) was reported to be 31.1 months across all subjects, with similar results seen in the KRAS mutant population. Despite this, the KRAS wild-type population had a shorter median DOR of 9.2 months. Median progression-free survival (PFS) was 12.9 months for all subjects, extending to 22 months in the KRAS mutant population and 12.8 months in the KRAS wild-type population. The disease control rate (DCR) at six months or more was 61% in the total population, with a higher rate of 70% in the KRAS-mutated group and 50% in the KRAS wild-type group. Most patients, regardless of KRAS mutation status, experienced some minimisations in target lesions. Last year, Verastem announced the dosing of the first subject with GFH375/VS-7375 as part of a Phase I/II trial in China. "Verastem's avutometinib combination shows 31% ORR in ovarian cancer trial" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
11-07-2025
- Business
- Business Wire
Verastem Oncology Announces Publication of the Primary Results from the Phase 2 RAMP 201 Trial of Avutometinib in Combination with Defactinib in Patients with Recurrent Low-Grade Serous Ovarian Cancer in the
BOSTON--(BUSINESS WIRE)--Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced that the primary analysis of the Phase 2 RAMP 201 clinical trial was published online in the Journal of Clinical Oncology (JCO). The data reported in the publication showed that avutometinib plus defactinib demonstrated a confirmed overall response rate (ORR) of 31% in all patients with recurrent low-grade serous ovarian cancer (LGSOC). The full manuscript, titled 'Efficacy and Safety of Avutometinib ± Defactinib in Recurrent Low-Grade Serous Ovarian Cancer: Primary Analysis of ENGOT-OV60/GOG-3052/RAMP 201,' will also appear in the print publication of JCO. 'The publication of the primary analysis of the RAMP 201 study in recurrent low-grade serous ovarian cancer in the Journal of Clinical Oncology reflects the meaningful clinical advancement demonstrated by the combination of avutometinib plus defactinib for patients with recurrent low-grade serous ovarian cancer,' said John Hayslip, M.D., chief medical officer at Verastem Oncology. 'The findings supported the recent FDA approval of the combination in KRAS-mutated recurrent low-grade serous ovarian cancer and our ongoing global Phase 3 RAMP 301 trial of the combination in recurrent low-grade serous ovarian cancer with and without a KRAS mutation.' The Company will submit the RAMP 201 publication and the recent publication of the FRAME study to the National Comprehensive Cancer Network® (NCCN®) in support of its consideration of inclusion of the KRAS wild-type population evaluated in these trials in the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®). Currently, the combination is listed as an NCCN Category 2A recommendation for the treatment of KRAS-mutated recurrent LGSOC, which is aligned with the FDA-approved indication. JCO Publication of RAMP 201 The Phase 2 RAMP 201 trial evaluated the safety, tolerability, and efficacy of avutometinib with and without defactinib in patients with recurrent LGSOC who had received a median of three (range one to nine) prior lines of therapy, including chemotherapy, hormonal therapy, bevacizumab, and MEK inhibitors. The publication included the primary analysis of the Phase 2 RAMP 201 trial that showed a confirmed ORR of 31% (34/109) in all 109 evaluable patients, 44% (25/57) in patients with a KRAS mutation, and 17% (9/52) in patients with KRAS wild-type. The median duration of response (DOR) was 31.1 months for all patients, 31.1 months in the KRAS mutant population, and 9.2 months in the KRAS wild-type population. Median progression-free survival (PFS) was 12.9 months for all patients, 22.0 months in the KRAS mutant population, and 12.8 months in the KRAS wild-type population. The majority of patients (82%) had some reduction in target lesions, regardless of KRAS mutation status. The disease control rate (DCR) at 6 or more months was 61% in the total population, 70% in the KRAS-mutated population, and 50% in the KRAS wild-type population. 'Our paper showed that the combination of avutometinib plus defactinib demonstrated clinically meaningful and durable responses in patients with recurrent low-grade serous ovarian cancer. The recent FDA accelerated approval of the combination in KRAS-mutated recurrent low-grade serous ovarian cancer, which is based on these results, is fantastic news. The fact that a majority of patients had some reduction in target lesions, regardless of KRAS mutation status, underscores the advancement the combination represents in this rare ovarian cancer and its potential to be the new standard of care in recurrent low-grade serous ovarian cancer,' said Professor Susana Banerjee, M.B.B.S., M.A., Ph.D., F.R.C.P, Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust and Team Leader in Women's Cancers at The Institute of Cancer Research, London and Global Lead Principal Investigator of ENGOTov60/GOG3052/NCRI/RAMP201. The results also demonstrated that the combination is well-tolerated, with a 10% discontinuation rate due to adverse events (AEs). The most common AEs (all grades, grade ≥3) were nausea (67.0%, 2.6%), diarrhea (58.3%, 7.8%), and increased creatine phosphokinase levels (60.0%, 24.3%). 'The publication of the Phase 2 RAMP 201 trial supports our understanding of the role that the combination of avutometinib plus defactinib plays in treating patients with recurrent low-grade serous ovarian cancer. We are excited to continue to build on the findings from the trial and advance the research in this disease with the ongoing international Phase 3 RAMP 301 trial, which is evaluating the combination in patients with and without a KRAS mutation,' said Rachel Grisham, M.D., Section Head, Ovarian Cancer at Memorial Sloan Kettering Cancer Center in New York, NY and Global Lead Principal Investigator of GOG-3097/ENGOT-ov81/GTG-UK/RAMP 301. The RAMP 201 study results were initially presented in an oral plenary session at the International Gynecologic Cancer Society (IGCS) Annual Meeting in October 2024. About RAMP 201 RAMP 201 (ENGOTov60/GOG3052/NCRI) (NCT04625270) was an adaptive, two-part multicenter, parallel cohort, randomized, open-label Phase 2 registration-directed trial evaluating the efficacy and safety of avutometinib alone and in combination with defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC). The first part of the trial (Part A) determined the selection of the go-forward regimen, which was the combination of avutometinib and defactinib versus avutometinib alone, based on overall response rates. The expansion phases of the trial (Parts B and C) evaluated the safety and efficacy of the go-forward regimen of avutometinib 3.2 mg twice weekly and defactinib 200 mg twice daily. The Part D portion of the trial evaluated a low dose of the combination to inform individualized dose reduction. About Low-Grade Serous Ovarian Cancer (LGSOC) LGSOC is a rare ovarian cancer that is insidious and persistent. LGSOC is distinct and different from high-grade serous ovarian cancer (HGSOC) and requires different treatment. LGSOC is highly recurrent and less sensitive to chemotherapy compared to HGSOC. Approximately 6,000-8,000 women in the U.S. and 80,000 worldwide are living with this disease. LGSOC affects younger women with bimodal peaks of diagnosis at ages between 20-30 and 50-60 and has a median survival of approximately ten years. Approximately 70 percent of LGSOC shows RAS pathway-associated mutations, and 30 percent of people with LGSOC have a KRAS mutation. The majority of patients report a negative impact of LGSOC on their mental and physical health, fertility, and long-term quality of life. About AVMAPKI and FAKZYNJA Combination Therapy AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors. The U.S. Food and Drug Administration (FDA) approved AVMAPKI™ FAKZYNJA™ CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. Verastem is also evaluating avutometinib in combination with defactinib and other agents as a potential treatment for patients with advanced pancreatic cancer (RAMP 205; NCT05669482) and advanced KRAS G12C mutant non-small cell lung cancer (RAMP 203; NCT05074810). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use. AVMAPKI FAKZYNJA CO-PACK U.S. Indication Indication AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS -mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Important Safety Information Warnings and Precautions Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity. Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration. Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality. Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction. Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception. Adverse Reactions The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count. Drug Interactions Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK. Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK. Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin. Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid. Use in Specific Populations Lactation: Advise not to breastfeed. Fertility: May impair fertility in males and females. Click here for full Prescribing Information. About Verastem Oncology Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI™ FAKZYNJA™ CO-PACK in the U.S. 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, FAK inhibition, and KRAS G12D inhibition. For more information, please visit and follow us on LinkedIn. Forward-Looking Statements Notice This press release includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Verastem's expectations, beliefs, goals, plans or prospects including, without limitation, statements about AVMAPKI FAKZYNJA CO-PACK's potential to benefit adult patients living with KRAS-mutated recurrent low-grade serous ovarian cancer in the United States, AVMAPKI FAKZYNJA CO-PACK's potential to be a transformational medicine, and AVMAPKI FAKZYNJA CO-PACK's potential to be an important treatment option for patients with KRAS-mutated recurrent low-grade serous ovarian cancer should be considered forward-looking statements. These forward-looking statements generally can be identified by the use of words such as 'anticipate,' 'expect,' 'plan,' 'could,' 'may,' 'believe,' 'estimate,' 'forecast,' 'goal,' 'project,' and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology's programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of commercialization of product candidates, the timing of commencing and completing trials and compiling data, the expected timing of the presentation of data by the Company and the potential clinical value of various of the Company's clinical trials. Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others: the uncertainties inherent in research and development, such as the possibility of negative or unexpected results of clinical trials; that the development and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; that data may not be available when expected; the risk that our preliminary and interim data may not be representative of more mature data; uncertainties related to the recent change in the U.S. presidential administration, including regulatory and policy changes that may adversely affect our business; that our product candidates may not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients; that the launch of AVMAPKI FAKZYNJA CO-PACK in the United States may not be successful; that the product may not be adopted by health care professionals; that pricing for the product may not be viewed favorably and we may not be successful in securing reimbursement coverage from third-party payors, including government agencies, for the product; that we may not be able to establish the product as the standard of care for KRAS-mutant recurrent LGSOC; that the actions or advice of regulatory agencies may impact our ability to obtain and maintain regulatory approval for our product; that the market opportunities for the product, that are based on internal and third-party estimates, may prove to be incorrect; that there may be competitive developments affecting our product candidates; that our product may cause adverse safety events or unexpected concerns may arise from additional data or analysis, or result in unmanageable safety profiles as compared to its level of efficacy; that we may be unable to successfully validate, develop and obtain regulatory approval for companion diagnostic tests for our product that require or would commercially benefit from such tests, or experience significant delays in doing so; that our product may experience manufacturing or supply interruptions or failures; that we may face substantial competition, which may result in others developing or commercializing products before or more successfully than we do which could result in reduced market share or market potential for our product candidates; that we may not have sufficient cash to fund our contemplated operations, including certain of our product commercialization and development programs; that we may not attract and retain high quality personnel; that we may not be able to expand the approved indication for AVMAPKI FAKZYNJA CO-PACK; that we may not be able to successfully obtain, maintain and protect intellectual property on our development and marketed products; that we may not be able to manage growth and operating expenses through disciplined investments in operations and achieve a self-sustainable financial profile in the future and avoid or successfully manage unexpected expenditures; that we may be unable to maintain strategic business collaborations; that our dependence on third parties for the development and commercialization of certain products may prove to be unsuccessful; that we may face government investigations and substantial changes in governmental policies, regulations, funding and enforcement; and the risks identified under the heading "Risk Factors" as detailed in the Company's Annual Report on Form 10-K for the year ended December 31, 2024, as filed with the Securities and Exchange Commission (SEC) on March 20, 2025, as well as the other information we file with the SEC, are possibly realized. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties. NCCN makes no warranties of any kind whatsoever regarding their content, use, or application and disclaims any responsibility for their application or use in any way. Dr. Grisham has financial interests related to Verastem Oncology.
Business Wire
30-06-2025
- Business
- Business Wire
Verastem Oncology Announces Nature Medicine Publication of the Results from the First-in-Human Phase 1 FRAME Study of Avutometinib in Combination with Defactinib in Solid Tumors, including Low-Grade Serious Ovarian Cancer
BOSTON--(BUSINESS WIRE)--Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced that updated results from the Phase 1/2 FRAME study conducted by The Institute of Cancer Research, London, and The Royal Marsden NHS Foundation Trust were published online in Nature Medicine. The full manuscript, titled 'Defactinib with avutometinib in patients with solid tumors: the phase 1 FRAME trial,' was the first-in-human study to evaluate the safety, tolerability, and efficacy of avutometinib in combination with defactinib in patients with low-grade serous ovarian cancer (LGSOC), non-small cell lung cancer (NSCLC), and other solid tumor types. 'The FRAME study was the early foundation for the recent FDA approval of avutometinib plus defactinib in KRAS-mutated recurrent low-grade serous ovarian cancer and we are pleased to see that the mature data set continues to show the safety and tolerability of this combination therapy,' said Dan Paterson, president and chief executive officer of Verastem Oncology. 'This supports our ongoing commitment to advancing our research into the combination for use in other solid tumors, including RAMP 205 in first-line metastatic pancreatic cancer.' The FRAME study enrolled patients, across dose escalation and dose expansion cohorts, with RAS-MAPK-driven solid tumors including LGSOC, NSCLC, colorectal, pancreatic and endometrial cancers. The updated data include an extended follow-up period and more detailed analyses from the FRAME study, featuring efficacy and safety data that demonstrated the novel combination of avutometinib and defactinib continues to be well-tolerated and shows encouraging responses in patients with LGSOC, consistent with previous findings. 'This was the first clinical study to show significant clinical activity of the combination of avutometinib, a RAF/MEK clamp, with defactinib, a FAK inhibitor. The FRAME study demonstrated an important potential advancement in the treatment of solid tumor diseases like low-grade serous ovarian cancer where seventy percent of these tumors are driven by the RAS/MAPK pathway and with thirty percent of these patients carrying a KRAS mutation,' said Professor Udai Banerji, co-Director of the Drug Development Unit, The Institute of Cancer Research and The Royal Marsden Hospital NHS Foundation Trust, London. 'We are pleased to have contributed to the development of avutometinib and defactinib. With FRAME's encouraging efficacy signal, the recommended Phase 2 dose was selected and the intermittent dosing schedule evaluated, which is the dose and schedule recently FDA approved in the U.S. and being used in the global confirmatory Phase 3 RAMP 301 trial.' In 26 patients with LGSOC who were evaluable for efficacy, the overall response rate (ORR) was 42.3% (11/26) and median progression-free survival (mPFS) was 20.1 months. In the 24 patients whose samples could be sequenced for KRAS mutations, ORR and mPFS were 58.3% (7/12) and 30.8 months in the 12 patients with KRAS mutations, and 33.3% (4/12) and 8.9 months in the 12 patients without KRAS mutations. In 11 patients who had previously received a MEK inhibitor, the ORR was 27.3% (3/11). Additionally, in two patients with LGSOC who had brain metastases prior to enrolling, MRI imaging at 30 months post-treatment with the combination showed the metastases had shrunk in both patients. In 27 patients with LGSOC who were evaluable for safety, only one (4%) discontinued treatment due to Grade 3 skin toxicity. The five most common adverse events (AEs) (all grades, grade ≥3) were: rash (90%, 8%), elevated blood levels of creatine phosphokinase (56%, 9%), AST elevation (43%, 1%), hyperbilirubinemia (38%, 2%), and diarrhea (38%, 1%). The tolerability profile of avutometinib plus defactinib was comparable to the tolerability of each agent as monotherapy. 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 low-grade serous ovarian cancer (LGSOC), non-small cell lung cancer (NSCLC) and colorectal cancer (CRC). The FRAME study is being led by Professor Udai Banerji, MBBS, MD, DNB, PhD, FRCP, Deputy Director of the Drug Development Unit at The Institute of Cancer Research, London, and The Royal Marsden NHS Foundation Trust, 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 (avutometinib 3.2mg, defactinib 200mg); cohort 2a (avutometinib 4mg, defactinib 200mg); and cohort 2b (avutometinib 3.2mg, defactinib 400mg). The recommended Phase 2 dose was determined to be cohort 1 (avutometinib 3.2mg, defactinib 200mg). About Low-Grade Serous Ovarian Cancer (LGSOC) LGSOC is a rare ovarian cancer that is insidious and persistent. LGSOC is distinct and different from high-grade serous ovarian cancer (HGSOC) and requires different treatment. LGSOC is highly recurrent and less sensitive to chemotherapy compared to HGSOC. Approximately 6,000-8,000 women in the U.S. and 80,000 worldwide are living with this disease. LGSOC affects younger women with bimodal peaks of diagnosis at ages between 20-30 and 50-60 and has a median survival of approximately ten years. Approximately 70 percent of LGSOC shows RAS pathway-associated mutations, and 30 percent of people with LGSOC have a KRAS mutation. The majority of patients report a negative impact of LGSOC on their mental and physical health, fertility, and long-term quality of life. About AVMAPKI and FAKZYNJA Combination Therapy AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors. The U.S. Food and Drug Administration (FDA) approved AVMAPKI™ FAKZYNJA™ CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. Verastem is also evaluating avutometinib in combination with defactinib and other agents as a potential treatment for patients with advanced pancreatic cancer (RAMP 205; NCT05669482) and advanced KRAS G12C mutant non-small cell lung cancer (RAMP 203; NCT05074810). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use. AVMAPKI FAKZYNJA CO-PACK U.S. Indication Indication AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS -mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Important Safety Information Warnings and Precautions Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity. Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration. Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality. Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction. Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception. Adverse Reactions The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count. Drug Interactions Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK. Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK. Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin. Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid. Use in Specific Populations Lactation: Advise not to breastfeed. Fertility: May impair fertility in males and females. Click here for full Prescribing Information. About Verastem Oncology Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI™ FAKZYNJA™ CO-PACK in the U.S. 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, FAK inhibition, and KRAS G12D inhibition. For more information, please visit and follow us on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Verastem's expectations, beliefs, goals, plans or prospects including, without limitation, statements about AVMAPKI FAKZYNJA CO-PACK's potential to benefit adult patients living with KRAS-mutated recurrent low-grade serous ovarian cancer in the United States, AVMAPKI FAKZYNJA CO-PACK's potential to be a transformational medicine, and AVMAPKI FAKZYNJA CO-PACK's potential to be an important treatment option for patients with KRAS-mutated recurrent low-grade serous ovarian cancer should be considered forward-looking statements. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "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. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: the launch AVMAPKI FAKZYNJA CO-PACK in the United States including having the product available in the market following launch and thereafter; the adoption of the product by health care professionals; establishing favorable pricing for the product and securing reimbursement coverage from third-party payors, including government agencies, for the product; establishing the product as the standard of care for KRAS-mutant recurrent LGSOC; actions or advice of regulatory agencies and our ability to obtain and maintain regulatory approval for our product; the market opportunities for AVMAPKI FAKZYNJA CO-PACK are based on internal and third-party estimates that may prove to be incorrect; that there may be competitive developments affecting our product candidates; that data may not be available when expected; that our product may cause adverse safety events or unexpected concerns may arise from additional data or analysis, or result in unmanageable safety profiles as compared to its level of efficacy; that we may be unable to successfully validate, develop and obtain regulatory approval for companion diagnostic tests for our product that require or would commercially benefit from such tests, or experience significant delays in doing so; that our product may experience manufacturing or supply interruptions or failures; that we face substantial competition, which may result in others developing or commercializing products before or more successfully than we do which could result in reduced market share or market potential for our product candidates; the discovery and development of novel drug candidates and delivery approaches and successful demonstration of the efficacy and safety of our product candidates; the pre-clinical and clinical results for our product candidates; that we may not have sufficient cash to fund our contemplated operations, including certain of our product commercialization and development programs; that we may not attract and retain high quality personnel; that we may not be able to expand the approved indication for AVMAPKI FAKZYNJA CO-PACK; that we may not be able to successfully launch, market and sell AVMAPKI FAKZYNJA CO-PACK in the United States and realize all or a substantial portion of the potential market opportunity; that we may not be able to successfully launch, market and sell approved products globally; that there may be delays, interruptions or failures in the manufacture and supply of our product candidates or marketed products; that we successfully obtain, maintain and protect intellectual property on our development and marketed products; that we are able to manage growth and operating expenses through disciplined investment in operations and able to achieve a self-sustainable financial profile in the future and avoid or successfully manage unexpected expenditures; that we are able to maintain strategic business collaborations; the dependence on third parties for the development and commercialization of certain products; the risks and uncertainties related to the recent change in the U.S. presidential administration, including regulatory and policy changes that may adversely affect our business; the risk of future government investigations and substantial changes in governmental polices, regulations, funding and enforcement; as well as those risks and uncertainties more fully discussed in the 'Risk Factors' filed with Verastem's 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 20 th, 2025, as may be updated from time to time in Verastem's subsequent Quarterly Reports on Form 10-Q, and in other filings that Verastem makes with the SEC.
Yahoo
14-06-2025
- Business
- Yahoo
Tempus AI (NasdaqGS:TEM) Soars 51% In Last Quarter
In an exciting development for Tempus AI, the company experienced a significant 51% increase in its share price over the last quarter. This surge coincides with major events including their collaboration with The Abrams Research Center to advance Alzheimer's research and a partnership with Verastem Oncology for ovarian cancer diagnostics. Additionally, the introduction of innovative products like the liquid biopsy assay for treatment response monitoring reflects Tempus AI's active expansion in AI and precision medicine. With a broad market showing an 11% rise over the past year, Tempus AI's performance is notably robust amidst industry-wide growth projections. We've discovered 3 possible red flags for Tempus AI that you should be aware of before investing here. Rare earth metals are an input to most high-tech devices, military and defence systems and electric vehicles. The global race is on to secure supply of these critical minerals. Beat the pack to uncover the 24 best rare earth metal stocks of the very few that mine this essential strategic resource. Tempus AI's shares demonstrated impressive resilience, achieving a substantial 77.09% total return over the past year. This outpaced both the broader U.S. market, which returned 10.6%, and the life sciences industry, which saw a decline of 23.1%. The robust share performance aligns with several strategic alliances and innovative product launches over the last year, potentially enhancing Tempus AI's revenue streams in precision medicine and AI-driven diagnostics. Despite strong revenue forecasts with expected annual growth of 21.71%, Tempus AI faces challenges with its profitability, recording a loss of US$68.04 million in the first quarter of 2025. The significant share price increase, however, positions the company above the consensus analyst price target of US$65.50. While the developments may bolster revenue, the market's valuation of Tempus AI highlights a discrepancy given its current unprofitability and Price-To-Sales Ratio of 15.4x compared to the industry average. The next quarters will be crucial in determining whether the company's strategic initiatives translate into a sustainable financial turnaround. Examine Tempus AI's past performance report to understand how it has performed in prior years. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Companies discussed in this article include NasdaqGS:TEM. This article was originally published by Simply Wall St. Have feedback on this article? Concerned about the content? with us directly. Alternatively, email editorial-team@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
06-06-2025
- Business
- Yahoo
VSTM Stock Soars 22% in a Month on Ovarian Cancer Combo Drug Approval
Verastem Oncology VSTM shares rallied 22.2% in a month, primarily driven by the FDA approval of the company's novel combination regimen of avutometinib plus defactinib in early May for treating KRAS mutant recurrent low-grade serous ovarian cancer (LGSOC), a rare and distinct type of ovarian cancer. The FDA's decision came well in advance of the originally expected date of June 30, 2025. The approval was granted under the FDA's accelerated approval pathway and is commercialized in the U.S. market as an oral combination co-pack with the two prescription products, known as 'Avmapki Fakzynja Co-Pack.' Following the FDA approval, Avmapki Fakzynja Co-Pack is the first and only FDA-approved treatment for the LGSOC indication. Per Verastem Oncology, the combo offers a much-needed treatment option for patients and sets a new standard of care for women with recurrent LGSOC with a KRAS mutation. Full approval of the product for this use depends on the detailed evidence of clinical benefit in the follow-up phase III RAMP 301 confirmatory study, which will evaluate the combination in women with and without a KRAS mutation. Year to date, Verastem Oncology stock has surged 64.8% against the industry's 5.8% decline. Image Source: Zacks Investment Research Please note that the FDA's decision was based on the results from VSTM's phase II RAMP 201 study, which evaluated the combination of Avmapki and Fakzynja in adult patients with measurable KRAS-mutated recurrent LGSOC. Per the data readout, a 44% overall response rate was observed in patients treated with the combo therapy, with an acceptable safety profile. Additionally, the median duration of response ranged from 3.3 to 31.1 months in the KRAS mutant population. The Avmapki/Fakzynja combo enjoys the FDA's Breakthrough Therapy designation for the treatment of patients with recurrent LGSOC after one or more prior lines of therapy, including platinum-based chemotherapy, in the United States. Avutometinib alone or in combination with defactinib also enjoys the FDA's Orphan Drug designation for the same indication. Verastem Oncology is also looking to expand the label of its approved avutometinib/defactinib combo therapy beyond LGSOC. Last week, VSTM reported positive updated safety and efficacy results from the phase I/II RAMP 205 study evaluating avutometinib plus defactinib in combination with chemotherapy (gemcitabine and Nab-paclitaxel) for the first-line treatment of metastatic pancreatic ductal adenocarcinoma (PDAC) patients. This has also likely contributed to the stock price rally in the past month. The RAMP 205 study is evaluating 60 patients in five cohorts (12 per cohort) who are receiving one of five dose regimens of avutometinib/defactinib combo with chemotherapy for frontline metastatic PDAC. As of April 25, 2025, patients in the dose level 1 cohort achieved an overall response rate of 83% (eight confirmed and two unconfirmed who remain on treatment). Based on this encouraging outcome, Verastem Oncology selected dose level 1 as the recommended phase II dose. Additionally, it was found that the dose level 1 showed the best response, and overall, 92% of patients across all dose levels achieved tumor shrinkage. The safety profile of the avutometinib/defactinib combo was acceptable across all the treatment cohorts and no new safety signals were identified. Verastem Oncology is currently gearing up to initiate a registrational phase III front-line metastatic PDAC study in 2026. Apart from these indications, the company is simultaneously evaluating avutometinib and defactinib in combination with a KRAS G12C inhibitor, sotorasib, for treating non-small cell lung cancer in a separate mid-stage study. Verastem, Inc. price-consensus-chart | Verastem, Inc. Quote Verastem Oncology currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector are Bayer BAYRY, Lexicon Pharmaceuticals LXRX and Amarin AMRN, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. In the past 60 days, estimates for Bayer's earnings per share have increased from $1.19 to $1.25 for 2025. During the same time, earnings per share have increased from $1.28 to $1.31 for 2026. Year to date, shares of Bayer have gained 45.3%. BAYRY's earnings beat estimates in one of the trailing four quarters, matched twice and missed on the remaining occasion, the average negative surprise being 13.91%. In the past 60 days, estimates for Lexicon's loss per share have narrowed from 37 cents to 32 cents for 2025. During the same time, loss per share estimates for 2026 have narrowed from 35 cents to 31 cents. Year to date, shares of LXRX have lost 6.6%. LXRX's earnings beat estimates in three of the trailing four quarters and missed the same on the remaining occasion, delivering an average surprise of 11.97%. In the past 60 days, estimates for Amarin's loss per share have narrowed from $5.33 to $3.48 for 2025. During the same time, loss per share estimates for 2026 have narrowed from $4.13 to $2.67. Year to date, shares of AMRN have gained 17.1%. AMRN's earnings beat estimates in two of the trailing four quarters, matched once and missed the same on the remaining occasion, delivering an average surprise of 29.11%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Bayer Aktiengesellschaft (BAYRY) : Free Stock Analysis Report Lexicon Pharmaceuticals, Inc. (LXRX) : Free Stock Analysis Report Amarin Corporation PLC (AMRN) : Free Stock Analysis Report Verastem, Inc. (VSTM) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research Sign in to access your portfolio
