Drugs / Avutometinib
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Avutometinib

Small molecule targets MAP2K1, MAP2K2 via inhibition

Developed for
non-small cell lung carcinoma · ovarian serous tumor · endometrioid adenocarcinoma · glioblastoma · malignant ovarian serous tumor · malignant pancreatic neoplasm · metastatic malignant neoplasm · ovarian cancer
+28 more · carcinoma · cervical cancer · colorectal adenocarcinoma · colorectal cancer · cutaneous melanoma · diffuse gastric cancer · diffuse glioma, H3 G34 mutant · endometrial cancer · hormone receptor-positive breast cancer · low grade glioma · lung adenocarcinoma · lung cancer · mesonephric neoplasm · metastatic malignant neoplasm in the brain · mucinous ovarian cancer · neoplasm · neuroblastoma · neurofibromatosis type 1 · optic pathway glioma · ovarian serous adenocarcinoma · pancreatic adenocarcinoma · pancreatic ductal adenocarcinoma · plasma cell myeloma · plexiform neurofibroma · primary brain neoplasm · refractory malignant neoplasm · thyroid cancer · uveal melanoma
Investigated by
Verastem, Inc. · Memorial Sloan Kettering Cancer Center · Institute of Cancer Research, United Kingdom · Emory University · European Network of Gynaecological Oncological Trial Groups (ENGOT) · GOG Foundation
+26 more · M.D. Anderson Cancer Center · National Cancer Institute (NCI) · Royal Marsden NHS Foundation Trust · Adrienne G. Waks · Amgen · Australia New Zealand Gynaecological Oncology Group · Cambridge University Hospitals NHS Foundation Trust · Cancer Research UK · Chugai Pharma USA · Chugai Pharmaceutical · Dana-Farber Cancer Institute · Eli Lilly and Company · Hoffmann-La Roche · Japanese Gynecologic Oncology Group · Korean Gynecologic Oncology Group · Minderoo Foundation · Mirati Therapeutics Inc. · Ryan H. Moy, MD, PhD · The Foundation for Barnes-Jewish Hospital · Thomas Jefferson University · University of Cambridge · University of Chicago · University of Oklahoma · University of Utah · University of Virginia · Washington University School of Medicine

Regulatory milestones approvals, filings & regulatory actions · 2 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Filed borderline ovarian serous tumor 1 milestone
Filed US (FDA) — borderline ovarian serous tumor 2024-10-31

“Announced the initiation of a rolling New Drug Application (NDA) submission seeking accelerated approval of the combination of avutometinib and defactinib for adult patients...” verastem.com ↗

“Completed rolling NDA submission for avutometinib and defactinib combination in recurrent KRAS mutant low-grade serous ovarian cancer in October 2024” verastem.com ↗

“today announced that the Company has completed its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA)” verastem.com ↗

“Verastem has initiated a rolling NDA submission for avutometinib and defactinib combination in adults with recurrent LGSOC” verastem.com ↗

Approved Indication not stated 1 milestone
Approved US (FDA) AVMAPKI FAKZYNJA CO-PACK (COPACKAGED) — 2025-05-08 fda.gov ↗

Trials 28

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 01 trial
Phase 0 NCT05798507 Jul 2023 → Dec 2026 expected glioblastoma Emory University Active not recruiting No outcome recorded
Phase 16 trials · 1 met primary
Phase 1 NCT07318324 May 2026 → Aug 2028 expected endometrial cancer M.D. Anderson Cancer Center Recruiting No outcome recorded
Phase 1 NCT06104488 Oct 2023 → Oct 2029 expected carcinoma, low grade glioma, neuroblastoma, neurofibromatosis type 1 +4 Memorial Sloan Kettering Cancer Center Active not recruiting No outcome recorded
Phase 1 NCT03681483 Oct 2018 → Jul 2024 non-small cell lung carcinoma Memorial Sloan Kettering Cancer Center Completed No outcome recorded
Phase 1 NCT03875820 Dec 2017 → Apr 2023 overdue endometrioid adenocarcinoma, malignant pancreatic neoplasm, non-small cell lung carcinoma, ovarian serous tumor Institute of Cancer Research, United Kingdom Active not recruiting Met primary
Phase 1 NCT02407509 Jun 2013 → Oct 2025 lung cancer, ovarian cancer, plasma cell myeloma Royal Marsden NHS Foundation Trust Completed No outcome recorded
Phase 1 NCT00773526 Nov 2008 → Sep 2011 neoplasm Hoffmann-La Roche Completed No outcome recorded
Phase 1/28 trials
Phase 1/2 NCT07824960 Oct 2026 → Apr 2029 expected pancreatic ductal adenocarcinoma University of Virginia Not yet recruiting No outcome recorded
Phase 1/2 NCT06630260 Nov 2024 → Sep 2029 expected diffuse glioma, H3 G34 mutant, glioblastoma Institute of Cancer Research, United Kingdom Recruiting No outcome recorded
Phase 1/2 NCT06194929 Mar 2024 → Jan 2028 expected cutaneous melanoma, metastatic malignant neoplasm in the brain University of Utah Recruiting No outcome recorded
Phase 1/2 NCT05669482 Mar 2023 → Aug 2026 overdue malignant pancreatic neoplasm, metastatic malignant neoplasm Verastem, Inc. Active not recruiting No outcome recorded
Phase 1/2 NCT05608252 Feb 2023 → Dec 2027 expected hormone receptor-positive breast cancer Adrienne G. Waks Recruiting No outcome recorded
Phase 1/2 NCT05200442 Aug 2022 → May 2027 colorectal adenocarcinoma University of Chicago Suspended No outcome recorded Stop: Enrollment
Phase 1/2 NCT05375994 Aug 2022 → Jun 2026 metastatic malignant neoplasm, non-small cell lung carcinoma Verastem, Inc. Completed No outcome recorded
Phase 1/2 NCT05074810 Apr 2022 → Jun 2026 overdue non-small cell lung carcinoma Verastem, Inc. Active not recruiting No outcome recorded
Phase 212 trials · 1 met primary
Phase 2 NCT07126158 Apr 2026 → Apr 2030 expected pancreatic adenocarcinoma Washington University School of Medicine Recruiting No outcome recorded
Phase 2 NCT06369259 Feb 2025 → Feb 2029 expected colorectal cancer M.D. Anderson Cancer Center Recruiting No outcome recorded
Phase 2 NCT06682572 Oct 2024 → Dec 2025 overdue ovarian cancer, ovarian serous tumor Verastem, Inc. Active not recruiting No outcome recorded
Phase 2 NCT06487221 Oct 2024 → Nov 2028 expected diffuse gastric cancer Ryan H. Moy, MD, PhD Recruiting No outcome recorded
Phase 2 NCT06495125 Jul 2024 → Mar 2029 expected lung adenocarcinoma Emory University Recruiting No outcome recorded
Phase 2 NCT06394804 Apr 2024 → Apr 2027 expected malignant ovarian serous tumor Memorial Sloan Kettering Cancer Center Recruiting No outcome recorded
Phase 2 NCT06007924 Aug 2023 → Aug 2027 expected thyroid cancer Memorial Sloan Kettering Cancer Center Recruiting No outcome recorded
Phase 2 NCT05787561 Mar 2023 → Mar 2027 expected mesonephric neoplasm Memorial Sloan Kettering Cancer Center Active not recruiting No outcome recorded
Phase 2 NCT05512208 Feb 2023 → Dec 2027 expected cervical cancer, endometrioid adenocarcinoma, malignant ovarian serous tumor, mucinous ovarian cancer University of Oklahoma Recruiting No outcome recorded
Phase 2 NCT04720417 Jan 2021 → Apr 2024 uveal melanoma Thomas Jefferson University Terminated No outcome recorded Stop: Enrollment
Phase 31 trial
Phase 3 NCT06072781 Mar 2024 → Oct 2028 expected ovarian serous tumor Verastem, Inc. Recruiting No outcome recorded
Comparator or background therapy1 trial
Phase 1 NCT05187169 Comparator Dec 2021 → Apr 2022 — Verastem, Inc. Completed No outcome recorded

News releases announcing trial results or a regulatory action · 43

DateIssuerRelease
2026-10-02 Verastem, Inc. Results Verastem Oncology Announces New RAMP 201 Molecular Profiling and External Control Arm Analyses Describing Outcomes for Avutometinib Plus Defactinib in Recurrent Low-Grade Serous Ovarian Cancer to be Presented at IGCS 2026 verastem.com ↗
RAMP 201 molecular profiling showed clinically meaningful activity in KRAS wild-type recurrent LGSOC, including ORRs of 22% in patients with NRAS- or BRAF-mutated tumors and 18% in patients with KRAS/NRAS/BRAF triple wild-type tumors
2026-09-28 Verastem, Inc. Results Verastem Oncology Announces Publication in Gynecologic Oncology of Two-Year Median Follow-Up Data from RAMP 201 Study of AVMAPKI® FAKZYNJA® CO-PACK (avutometinib capsules; defactinib tablets) in Recurrent Low-Grade Serous Ovarian Cancer verastem.com ↗
Across patients with KRAS wild-type and KRAS-mutated recurrent LGSOC, the combination had a median duration of response of 31.1 months, median progression free survival of 12.9 months, and a safety profile consistent with the primary analysis
2026-06-17 Verastem, Inc. Results Verastem Oncology Announces Positive Updated Results from RAMP 205 Evaluating Avutometinib Plus Defactinib in Combination with Standard-of-Care Chemotherapy in First-Line Metastatic Pancreatic Cancer verastem.com ↗
The updated data from the RAMP 205 trial provide important clinical insights into the potential impact of combined RAF/MEK and FAK inhibition as a therapeutic option for pancreatic cancer.
2026-04-10 Verastem, Inc. Results Verastem Oncology Announces Two-Year Median Follow-Up Data on AVMAPKI® FAKZYNJA® Combination Therapy (avutometinib capsules; defactinib tablets) in Recurrent Low-Grade Serous Ovarian Cancer at the SGO 2026 Annual Meeting on Women’s Cancers verastem.com ↗
Patients in the Phase 2 RAMP 201 clinical trial demonstrated sustained clinical benefits across multiple efficacy measures highlighting durability of response with a median follow-up of two years
2026-03-17 Verastem, Inc. Results Verastem Oncology Announces Late Breaking and Regular Abstracts Accepted for Presentation at the AACR Annual Meeting 2026 verastem.com ↗
In mutant BRAF-driven models, the combination of the FAK inhibitor VS-4718 and the RAF/MEK clamp avutometinib, with or without the mutant BRAF inhibitor encorafenib, significantly delayed tumor onset, induced regression of established tumors and brain metastases, and prolonged overall survival.
2025-12-29 Verastem, Inc. Results Verastem Oncology Provides Update on RAMP 203 Phase 1/2 Clinical Trial for Advanced KRAS G12C Mutant Non-Small Cell Lung Cancer verastem.com ↗
While avutometinib plus defactinib combined well with a G12C inhibitor to drive early and sustained anti-tumor responses, next generation G12C inhibitors are establishing a new benchmark with higher response rates.
2025-07-11 Verastem, Inc. Results 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 Journal of Clinical Oncology verastem.com ↗
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).
2025-06-30 Verastem, Inc. Results 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 Serous Ovarian Cancer verastem.com ↗
FRAME study demonstrated a 42.3% ORR and 20.1 months median PFS for all patients with low-grade serous ovarian cancer (LGSOC), regardless of KRAS mutation; for patients with KRAS-mutated LGSOC, ORR and mPFS were 58.3% and 30.8 months, respectively
2025-05-23 Chugai Pharmaceutical Results Verastem Oncology’s Announcement Regarding AVMAPKI (Positive Updated Results from RAMP 205 Evaluating Avutometinib Plus Defactinib in Combination with Standard-of-Care Chemotherapy in Frontline Metastatic Pancreatic Ductal Adenocarcinoma) chugai-pharm.co.jp ↗
Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) announced that Verastem Oncology issued a press release on AVMAPKI TM (generic name: avutometinib) about positive updated results from RAMP 205 evaluating avutometinib plus defactinib in combination with standard-of-care chemotherapy in frontline metastatic pancreatic ductal adenocarcinoma on May 22, 2025.
2025-05-22 Verastem, Inc. Results Verastem Oncology Announces Positive Updated Results from RAMP 205 Evaluating Avutometinib Plus Defactinib in Combination with Standard-of-Care Chemotherapy in Frontline Metastatic Pancreatic Ductal Adenocarcinoma verastem.com ↗
today 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).
2025-05-09 Chugai Pharmaceutical Regulatory Verastem Oncology’s Announcement Regarding AVMAPKI (FDA Approval for AVMAPKI FAKZYNJA Combination Therapy as the First-Ever Treatment for Adult Patients with KRAS-Mutated Recurrent Low-Grade Serous Ovarian Cancer) chugai-pharm.co.jp ↗
the U.S. Food and Drug Administration (FDA) has approved for AVMAPKI TM FAKZYNJA TM CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC), who received prior systemic therapy.
2025-05-08 Verastem, Inc. Regulatory FDA Approves the AVMAPKI™ FAKZYNJA™ Combination Therapy as the First-Ever Treatment for Adult Patients with KRAS-mutated Recurrent Low-Grade Serous Ovarian Cancer verastem.com ↗
AVMAPKI FAKZYNJA CO-PACK is the first and only FDA-approved medicine for this disease.

All press releases naming this drug 59 releases

DateIssuerRelease

Evidence & citations 18 cited values

Every value below carries the sentence it was read from. 30 sources stand behind the page.

FieldValueCited text
Known as avutometinib “VS-6766/avutometinib” NCT05200442 ↗
21

NCT07824960 ↗

NCT06487221 ↗

NCT05787561 ↗

NCT05375994 ↗

NCT06682572 ↗

NCT06394804 ↗

NCT06104488 ↗

NCT05798507 ↗

NCT05074810 ↗

NCT06007924 ↗

Show 11 more sources

Known as avutometinib (VS-6766) “avutometinib (VS-6766)” NCT06072781 ↗
Known as AVUTOMETINIB POTASSIUM ChEMBL registry synonym — accepted as the source's own label CHEMBL3264002 ↗
Known as CH5126766 “CH5126766” NCT03875820 ↗
Known as CH5126766-002 ChEMBL registry synonym — accepted as the source's own label CHEMBL3264002 ↗
Known as CKI-27 ChEMBL registry synonym — accepted as the source's own label CHEMBL3264002 ↗
Known as R-7304 ChEMBL registry synonym — accepted as the source's own label CHEMBL3264002 ↗
Known as Rg-7304 ChEMBL registry synonym — accepted as the source's own label CHEMBL3264002 ↗
Known as RO-5126766 POTASSIUM ChEMBL registry synonym — accepted as the source's own label CHEMBL3264002 ↗
Known as RO5126766 ClinicalTrials.gov intervention name — accepted as the source's own label NCT03681483 ↗
1

NCT00773526 ↗

Known as RO5126766-002 ChEMBL registry synonym — accepted as the source's own label CHEMBL3264002 ↗
Known as VS-6766 “This study will assess the safety and efficacy of avutometinib (VS-6766) in combination with adagrasib in patients with G12C Non-Small Cell Lung Cancer (NSCLC) who have been...” NCT05375994 ↗
15

“This study will assess the safety and efficacy of avutometinib (VS-6766) and defactinib in combination with gemcitabine and nab-paclitaxel in patients with Pancreatic Ductal...” NCT05669482 ↗

“Avutometinib (also known as VS-6766) is an oral drug that blocks an important signaling pathway in cancer cells known as the MAP kinase pathway.” NCT06487221 ↗

“A Study of Avutometinib (VS-6766) and Defactinib in People With Mesonephric Gynecologic Cancer” NCT05787561 ↗

“Avutometinib (VS-6766), a RAF/MEK Clamp in Combination With Defactinib, a FAK Inhibitor” NCT06104488 ↗

“Avutometinib (VS-6766): will be administered at 3.2 mg orally twice a week” NCT05512208 ↗

“Avutometinib (VS-6766) (Dual RAF/MEK Inhibitor)” NCT04620330 ↗

“Avutometinib (VS-6766) + Defactinib (VS-6063)” NCT07824960 ↗

“Avutometinib (VS-6766) + Defactinib (VS-6063)” NCT06682572 ↗

“avutometinib (VS-6766)” NCT04625270 ↗

NCT03875820 ↗

Show 5 more sources

Known as VS-6766 POTASSIUM ChEMBL registry synonym — accepted as the source's own label CHEMBL3264002 ↗
Action Inhibit “Dual specificity mitogen-activated protein kinase kinase 2 inhibitor” CHEMBL3264002 ↗
2

“Dual specificity mitogen-activated protein kinase kinase 1 inhibitor” CHEMBL3264002 ↗

“a Dual Raf and MEK Inhibitor” NCT00773526 ↗

Modality Small molecule “VS-6766 is a unique small molecule drug” NCT05608252 ↗
1

CHEMBL3264002 ↗

Route Oral “Administered orally daily for 28 days” NCT00773526 ↗
Target MAP2K1 “Dual specificity mitogen-activated protein kinase kinase 1 inhibitor” CHEMBL3264002 ↗
Target MAP2K2 “Dual specificity mitogen-activated protein kinase kinase 2 inhibitor” CHEMBL3264002 ↗