This section highlights FDA-related milestones and regulatory updates for drugs developed by Verastem (VSTM).
Over the past two years, Verastem has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Avutometinib and RAMP. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
Avutometinib FDA Regulatory Timeline and Events
Avutometinib is a drug developed by Verastem for the following indication: For the treatment of low-grade serous ovarian cancer (LGSOC).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Avutometinib
- Announced Date:
- October 2, 2026
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology announced data from a new molecular profiling and tumor biomarker analysis of the Phase 2 RAMP 201 clinical trial of avutometinib plus defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC), as well as a new external control arm analysis comparing outcomes from RAMP 201 with conventional care.
Read Announcement- Drug:
- Avutometinib
- Announced Date:
- March 17, 2026
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology announced it will present new long-term AVMAPKI™ FAKZYNJA™ combination therapy (avutometinib capsules; defactinib tablets) data from the Phase 2 RAMP 201 clinical trial at the Society of Gynecologic Oncology (SGO) 2026 Annual Meeting on Women's Cancers.
AI Summary
Verastem Oncology announced it will present new long-term data from the Phase 2 RAMP 201 trial at the Society of Gynecologic Oncology (SGO) 2026 Annual Meeting on Women’s Cancers. The data will be shared as a plenary oral presentation and focus on extended follow-up in patients with recurrent low-grade serous ovarian cancer (LGSOC).
RAMP 201 evaluated the combination of AVMAPKI™ (avutometinib capsules) and FAKZYNJA™ (defactinib tablets). The long-term results aim to clarify how durable responses are, how the combination performs over time, and which patients—including those with KRAS mutations—may benefit most from this targeted approach.
These follow-up findings are expected to inform clinicians and researchers about the combination’s longer-term activity and safety, and to help guide future clinical research and treatment decisions for recurrent LGSOC.Read Announcement
- Drug:
- Avutometinib
- Announced Date:
- March 17, 2026
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology announced multiple abstracts have been accepted for presentation at the American Association for Cancer Research (AACR) Annual Meeting to be held April 17-22, 2026, in San Diego, CA.
AI Summary
Verastem Oncology announced that multiple abstracts have been accepted for presentation at the American Association for Cancer Research (AACR) Annual Meeting, taking place April 17–22, 2026, in San Diego, CA. Late‑breaking abstracts will be posted on the AACR meeting website on April 17 at 12:00 p.m. PT, and regular abstracts also will be presented during the conference.
Key preclinical data to be shared include results for VS-7375, an oral KRAS G12D (ON/OFF) inhibitor. In combination with PRMT5 inhibitors, VS-7375 produced strong, durable tumor regressions in KRAS G12D pancreatic cancer models. Across pancreatic, lung, and colorectal models, VS-7375 drove deeper and more sustained tumor shrinkage compared with ON-only G12D inhibitors or pan‑RAS inhibitors.
Other presentations will describe combinations such as the FAK inhibitor VS-4718 with the RAF/MEK clamp avutometinib, with or without encorafenib, which delayed tumor onset, caused regressions including brain metastases, and extended survival in mutant BRAF models. These data aim to advance treatments for RAS/MAPK pathway‑driven cancers.
Read Announcement- Drug:
- Avutometinib
- Announced Date:
- May 22, 2025
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology announced positive updated safety and efficacy results from the RAMP 205 Phase 1/2 trial evaluating avutometinib plus defactinib in combination with gemcitabine and Nab-paclitaxel in the front-line for patients with metastatic pancreatic ductal adenocarcinoma (PDAC).
AI Summary
Verastem Oncology announced positive updated safety and efficacy results from the RAMP 205 Phase 1/2 trial focused on front-line treatment of metastatic pancreatic ductal adenocarcinoma (PDAC). In the trial’s selected recommended Phase 2 dose, dose level 1, 83% of patients (10 out of 12) achieved a partial response when treated with a combination of avutometinib, defactinib, gemcitabine, and Nab-paclitaxel. These promising outcomes demonstrate strong tumor reduction and a manageable safety profile with no new safety signals.
Based on the encouraging data, plans are underway to expand the trial and begin a registrational Phase 3 study in 2026. These results support the potential of this combination therapy as a novel treatment option for front-line metastatic PDAC, offering hope for improved outcomes in this challenging patient population.
Read Announcement- Drug:
- Avutometinib
- Announced Date:
- March 20, 2025
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology highlighted recent progress.
AI Summary
Verastem Oncology recently highlighted significant progress in their clinical pipeline. Their novel combination of avutometinib plus defactinib for KRAS mutant recurrent low‐grade serous ovarian cancer received high praise from the FDA. The agency accepted the New Drug Application under the accelerated approval pathway and granted Priority Review, with a PDUFA action date set for June 30, 2025. CEO Dan Paterson stated that 2025 is expected to be transformational, as the company aims to launch the first FDA-approved treatment for this specific patient group and transition into a fully integrated commercial-stage organization.
Additionally, Verastem is advancing further studies, including initiating a Phase 1/2a trial for VS-7375—an oral KRAS G12D inhibitor—and progressing studies in pancreatic and non-small cell lung cancers. These efforts underline the company’s commitment to expanding treatment options in oncology.
Read Announcement- Drug:
- Avutometinib
- Announced Date:
- December 30, 2024
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) under the accelerated approval pathway for avutometinib, an oral RAF/MEK clamp, in combination with defactinib, an oral FAK inhibitor, for the treatment of adult patients with recurrent low-grade serous ovarian cancer (LGSOC), who received at least one prior systemic therapy and have a KRAS mutation.
AI Summary
Verastem Oncology announced that the FDA has accepted its New Drug Application (NDA) for review under the accelerated approval pathway. The application is for a new treatment that combines avutometinib, an oral RAF/MEK clamp, with defactinib, an oral FAK inhibitor. This treatment is aimed at adult patients with recurrent low-grade serous ovarian cancer (LGSOC) who have received at least one prior systemic therapy and have a KRAS mutation.
If approved, this would be the first FDA-approved treatment specifically for this patient group. The NDA, submitted in October 2024, has received Priority Review, with a target action date set for June 30, 2025. Verastem is hopeful that this new therapy will address a significant unmet need and offer a promising new option for patients facing this rare and challenging form of ovarian cancer.
Read Announcement- Drug:
- Avutometinib
- Announced Date:
- December 18, 2024
- Indication:
- For the treatment of low-grade serous ovarian cancer (LGSOC)
Announcement
Verastem Oncology announced preliminary clinical data for the triplet combination of avutometinib and sotorasib plus defactinib in the RAMP 203 Phase 1/2 study in KRAS G12C mutant advanced non-small cell lung cancer (NSCLC).
AI Summary
Verastem Oncology announced early clinical data from the RAMP 203 Phase 1/2 study, which is testing a new triplet combination of avutometinib, sotorasib, and defactinib in patients with advanced non-small cell lung cancer bearing the KRAS G12C mutation. In this trial, no dose-limiting toxicities were observed among patients who had already progressed on earlier treatments. Notably, two of the three patients treated in this cohort experienced initial tumor reductions of at least 20%, and all remain on the study treatment. The addition of defactinib, a focal adhesion kinase (FAK) inhibitor, is designed to boost anti-tumor activity by addressing resistance mechanisms. Verastem plans to enroll more patients and will present further data at a medical meeting in the second half of 2025, highlighting the promising nature of this combination approach in addressing difficult-to-treat lung cancer.
Read Announcement
RAMP 201 FDA Regulatory Timeline and Events
RAMP 201 is a drug developed by Verastem for the following indication: Vutometinib (VS-6766) alone and in combination with defactinib among patients with recurrent low-grade serous ovarian cancer (LGSOC).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- RAMP 201
- Announced Date:
- April 10, 2026
- Indication:
- Vutometinib (VS-6766) alone and in combination with defactinib among patients with recurrent low-grade serous ovarian cancer (LGSOC).
Announcement
Verastem Oncology announced two-year median follow-up data from the Phase 2 RAMP 201 clinical trial that evaluated AVMAPKI® FAKZYNJA® combination therapy (avutometinib capsules; defactinib tablets) in patients with recurrent low-grade serous ovarian cancer (LGSOC) will be presented today during an oral plenary session at the Society of Gynecologic Oncology (SGO) 2026 Annual Meeting on Women's Cancers taking place in San Juan, Puerto Rico, April 10-13, 2026.
AI Summary
Verastem Oncology announced two‑year median follow‑up data from the Phase 2 RAMP 201 trial of AVMAPKI FAKZYNJA (avutometinib capsules; defactinib tablets) in recurrent low‑grade serous ovarian cancer. The results were presented April 10, 2026, at the SGO Annual Meeting.
With a median follow‑up of 24.9 months, patients showed sustained clinical benefit across multiple efficacy measures. Fifty‑two percent of KRAS‑mutated and 30% of KRAS wild‑type patients remained on therapy for more than one year, and median duration of response and progression‑free survival were consistent with earlier findings.
The combination remained well tolerated after two years, with no new safety signals and a low 12% discontinuation rate due to adverse events. Safety analyses included 303 patients across FRAME, RAMP 201, and RAMP 202.
A new exposure‑response analysis showed the best effect when using the approved dose and schedule. AVMAPKI FAKZYNJA is approved under accelerated approval for adult patients with KRAS‑mutated recurrent LGSOC; continued approval may depend on confirmatory trials.
Read Announcement- Drug:
- RAMP 201
- Announced Date:
- July 11, 2025
- Indication:
- Vutometinib (VS-6766) alone and in combination with defactinib among patients with recurrent low-grade serous ovarian cancer (LGSOC).
Announcement
Verastem Oncology announced that the primary analysis of the Phase 2 RAMP 201 clinical trial was published online in the Journal of Clinical Oncology (JCO).
AI Summary
Verastem Oncology announced that the primary analysis of its Phase 2 RAMP 201 clinical trial was published online in the Journal of Clinical Oncology (JCO). The study examined the use of the drug combination avutometinib plus defactinib in patients with recurrent low‐grade serous ovarian cancer who had received multiple previous treatments.
The analysis revealed an overall confirmed response rate of 31%, with a higher response in patients with KRAS mutations (44%) compared to those with KRAS wild-type (17%). In addition, 82% of patients had a reduction in target lesions. The median progression-free survival was 12.9 months overall, with improved outcomes in the KRAS-mutant group. These promising results support the potential of this combination therapy to offer a durable and meaningful benefit for patients facing this challenging cancer, while ongoing trials continue to evaluate its effectiveness further.
Read Announcement- Drug:
- RAMP 201
- Announced Date:
- May 20, 2025
- Indication:
- Vutometinib (VS-6766) alone and in combination with defactinib among patients with recurrent low-grade serous ovarian cancer (LGSOC).
Announcement
Tempus Announces a Collaboration with Verastem to Develop CDx for First-Ever FDA-Approved KRAS-Mutant Recurrent Low-Grade Serous Ovarian Cancer Combination Treatment
AI Summary
Tempus AI and Verastem Oncology have joined forces to develop a companion diagnostic (CDx) test for a new targeted treatment. The collaboration aims to support the first-ever FDA-approved combination treatment with avutometinib and defactinib for KRAS-mutant recurrent low-grade serous ovarian cancer (LGSOC), a rare cancer that often affects younger women and has few treatment options. Tempus has already provided confirmatory testing in Verastem’s Phase 2 RAMP-201 trial using its FDA-approved xT CDx assay. This assay is now being used in the global Phase 3 RAMP-301 trial to help identify patients with KRAS mutations, ensuring they receive the most suitable treatment. The new diagnostic test is a key step to better group patients based on their cancer’s genetic profile, potentially leading to more effective and personalized therapies for LGSOC patients.
Read Announcement- Drug:
- RAMP 201
- Announced Date:
- February 20, 2025
- Indication:
- Vutometinib (VS-6766) alone and in combination with defactinib among patients with recurrent low-grade serous ovarian cancer (LGSOC).
Announcement
Verastem Oncology announced multiple oral and poster presentations, including an oral presentation of additional analyses from the ongoing Phase 2 RAMP 201 (ENGOT-ov60/GOG-3052) trial evaluating the investigational combination of avutometinib plus defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC), at the Society of Gynecologic Oncology (SGO) 2025 Annual Meeting on Women's Cancer, to be held on March 14-17 in Seattle, Washington.
AI Summary
Verastem Oncology announced that it will present new results from the ongoing Phase 2 RAMP 201 (ENGOT-ov60/GOG-3052) trial at the Society of Gynecologic Oncology (SGO) 2025 Annual Meeting on Women’s Cancer in Seattle, Washington, from March 14-17. The oral presentation will reveal additional analyses, including subgroup data by KRAS mutational status, from the study evaluating the combination of avutometinib and defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC). This data builds on the primary analysis that supported their New Drug Application currently under Priority Review by the FDA. The presentation is part of Verastem’s ongoing efforts to expand treatment options for patients with RAS/MAPK pathway-driven cancers, and it offers an opportunity for oncologists to gain further insights into the potential benefits of this targeted therapy combination.
Read Announcement- Drug:
- RAMP 201
- Announced Date:
- October 9, 2024
- Indication:
- Vutometinib (VS-6766) alone and in combination with defactinib among patients with recurrent low-grade serous ovarian cancer (LGSOC).
Announcement
Verastem Oncology announced further details for its late-breaking presentation of mature data from the ongoing Phase 2 RAMP 201 (ENGOT-ov60/GOG-3052) clinical trial to be presented as an oral presentation at a plenary session at the International Gynecologic Cancer Society (IGCS) Annual Global Meeting taking place October 16-18, 2024 in Dublin, Ireland.
AI Summary
Verastem Oncology announced details of its late-breaking presentation at the upcoming International Gynecologic Cancer Society (IGCS) Annual Global Meeting, which will be held in Dublin, Ireland from October 16-18, 2024. During the meeting’s plenary session on October 17, 2024 at 11:42 AM ET, the company will showcase mature data from the ongoing Phase 2 RAMP 201 trial (ENGOT-ov60/GOG-3052).
The oral presentation will share updated safety and efficacy findings for the combination of avutometinib, a novel RAF/MEK clamp, and defactinib, an oral selective FAK inhibitor, in patients with recurrent low-grade serous ovarian cancer (LGSOC). Professor Susana N. Banerjee, the global lead investigator, is set to present the data, highlighting metrics such as overall response rate, progression-free survival, and duration of response.
Read Announcement
