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Avutometinib

Phase 2

Gastric Cancer | Small molecule | Oncology |Verastem, Inc.|Last Updated: Aug 5, 2026

Target and mechanism

Molecular targetMAP2K1, MAP2K2
Target classInhibitor
ModalitySmall molecule

Also known as avutometinib and sotorasib, avutometinib (VS-6766)

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment27

FDA Designations

BREAKTHROUGH_THERAPYORPHAN_DRUG

Clinical trial landscape

Avutometinib · 5 trials · 10 indications

Phase 2 3Phase 1 1Early Phase 1 1
NCT07747506Continued Access to Avutometinib in Combination With Defactinib in Patients With Recurrent Low-Grade Serous Ovarian Cancer (LGSOC)Low Grade Ovarian Serous Adenocarcinoma
NOT YET_RECRUITING2 Analytics
NCT06487221Avutometinib and Defactinib in Diffuse Gastric CancerGastric Cancer
RECRUITING27 Analytics
NCT04625270A Study of Avutometinib (VS-6766) v. Avutometinib (VS-6766) + Defactinib in Recurrent Low-Grade Serous Ovarian Cancer With and Without a KRAS MutationLow Grade Ovarian Serous Adenocarcinoma
ACTIVE NOT_RECRUITING225 Analytics
PHASE2NOT YET_RECRUITING
Continued Access to Avutometinib in Combination With Defactinib in Patients With Recurrent Low-Grade Serous Ovarian Cancer (LGSOC)
Low Grade Ovarian Serous AdenocarcinomaUnlock trial analytics
PHASE2RECRUITING
Avutometinib and Defactinib in Diffuse Gastric Cancer
Gastric CancerUnlock trial analytics
PHASE2ACTIVE NOT_RECRUITING
A Study of Avutometinib (VS-6766) v. Avutometinib (VS-6766) + Defactinib in Recurrent Low-Grade Serous Ovarian Cancer With and Without a KRAS Mutation
Low Grade Ovarian Serous AdenocarcinomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Duration of Treatment Exposure to Avutometinib in Combination With Defactinib
Until treatment discontinuation (approximately 20 months)

Treatment exposure will be summarized descriptively for participants receiving continued treatment with avutometinib in combination with defactinib after completion of Study VS-6766-201 (RAMP-201).

Progression-free survival (PFS) Rate
6 months

To determine the efficacy of combination defactinib and avutometinib in patients with metastatic diffuse gastric cancer (DGC) as measured by 6-month progression-free survival (PFS) rate. PFS is defined as the duration of time from start of treatment to time of progression or death, whichever occurs first.

Part A: Determine optimal regimen of avutometinib (VS-6766) monotherapy or in combination with defactinib
From start of treatment to confirmation of response; 24 weeks

Confirmed overall response rate per RECIST 1.1

Part B: To determine the efficacy of the optimal regimen identified from Part A
From start of treatment to confirmation of response; 24 weeks

Confirmed overall response rate per RECIST 1.1

Part C: To evaluate additional efficacy parameters for the optimal regimen identified in Part A
From start of treatment to confirmation of response; 24 weeks

Confirmed overall response rate per RECIST 1.1

Part D:To evaluate additional efficacy parameters for a lower dose of avutometinib in combination with defactinib
From start of treatment to confirmation of response; 24 weeks

Confirmed ORR defined according to RECIST 1.1

Phase 1b - To evaluate the safety and tolerability of investigational agent in patients with malignant brain tumours
12 months

To identify the incidence, nature and severity of adverse events and laboratory abnormalities, with severity determined according to NCI CTCAE v5.0

Phase 1b - To determine the preliminary antitumour activity of the investigational agent administered at the RP2D in patients with molecularly defined malignant brain tumours
12 months

Antitumour activity will be defined on the basis of the following outcomes. If any of the following occur, patients will be considered to have clinically benefitted: For relapsed GBM: Achievement of overall response of CR or PR per Response Assessment in Neuro-Oncology (RANO) within 6 months or Free of disease progression or death at 6 months For front line unmethylated GBM (MRD): • Progression-free survival (PFS)

Phase 2 - To determine the antitumour activity of investigational agent administered at the RP2D in patients with molecularly defined malignant brain tumours
9 months

Antitumour activity will be defined on the basis of the following outcomes: Progression-free survival (PFS), defined as the time from enrolment to the first occurrence of disease progression or death from any cause (whichever occurs first), as determined by the investigator according to RANO Overall survival (OS), defined as the time from enrolment to death from any cause

Concentration of defactinib that accumulates in the glioblastoma (GBM) and brain around tumor
At time of surgery

Defactinib concentration will be measured in a sample of the glioblastoma, brain around the glioblastoma, and in a serum sample from each subject receiving Defactinib. Descriptive statistics, such as mean and standard deviation, will be generated with these results. Concentration will be compared between dose levels within study drug using two-sample t-tests or non-parametric equivalents such as Mann Whitney U tests.

Concentration of avutometinib (VS-6766) that accumulates in the GBM and brain around tumor
At time of surgery

VS-6766 concentration will be measured in a sample of the glioblastoma, brain around the glioblastoma, and in a serum sample from each subject receiving VS-6766. Descriptive statistics, such as mean and standard deviation, will be generated with these results. Concentration will be compared between dose levels within study drug using two-sample t-tests or non-parametric equivalents such as Mann Whitney U tests.

Incidence of adverse events associated with defactinib
Up to 2 weeks post surgery

Will be assessed by quantification of the recognized side effects of this agent including fatigue, nausea, diarrhea, vomiting, hyperbilirubinemia, decreased appetite, peripheral edema, dizziness, and headache and by monitoring for new or undescribed adverse events. Summary statistics will include frequencies and percentages of adverse events.

Incidence of adverse events associated with VS-6766
Up to 2 weeks post surgery

Will be assessed by quantification of the recognized side effects of this agent including rash, creatine phosphokinase elevation, visual disturbances, hypoalbuminemia, and fatigue and by monitoring for new or undescribed adverse events. Summary statistics will include frequencies and percentages of adverse events.

Secondary Endpoints

Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
From informed consent until 30 days after the last dose of study treatment (approximately 20 months plus 30 days).
Overall Response Rate (ORR)
6 months
Median Progression-Free Survival
24 months
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Continued Treatment of Avutometinib + DefactinibEXPERIMENTALTo provide continued treatment with avutometinib in combination with defactinib for participants with recurrent LGSOC who are deriving continued clinical benefit after completion of the VS-6766-201 (RAMP-201) study
Avutometinib & DefactinibEXPERIMENTALAvutometinib: Study participants will receive Avutometinib 3.2mg orally two times per week for three weeks in a row followed by one week of rest Defactinib: Study participants will receive Defactinib 200mg twice daily for three weeks in a row followed by one week of rest
Part AEXPERIMENTALTo determine the optimal regimen, either avutometinib(VS-6766) monotherapy or avutometinib (VS-6766) in combination with defactinib, for subsequent evaluation for efficacy in the Expansion Phase (Part B)
Part BEXPERIMENTALTo determine the efficacy of the optimal regimen identified from Part A
Part C:EXPERIMENTALTo evaluate additional efficacy parameters for the optimal regimen identified in Part A.
Part DEXPERIMENTALTo evaluate additional efficacy parameters for a lower dose of avutometinib in combination with defactinib
Phase 1bEXPERIMENTALThe Phase 1b will evaluate the safety and tolerability of combination of avutometinib and defactinib and determine its preliminary antitumour activity when administered at the recommended Phase 2 dose (RP2D) in patients with molecularly defined malignant brain tumours.
Phase 2EXPERIMENTALThe Phase 2 part of the study will determine the antitumour activity of investigational agents administered at the RP2D in patients with molecularly defined malignant brain tumours. Avutometinib and defactinib may be administered in combination with temozolomide (TMZ).
Arm I (Defactinib)EXPERIMENTALPatients receive 1 dose of defactinib PO while on study, prior to planned tumor resection. Patients undergo blood collection and donate resected tumor tissue while on study.
Arm II (Avutometinib)EXPERIMENTALPatients receive 1 dose of avutometinib PO while on study, prior to planned tumor resection. Patients undergo blood collection and donate resected tumor tissue while on study.

Interventions

NameTypeDescription
avutometinib + defactininb combinationDRUGAvutometinib: administered orally twice a week Defactinib: administered orally twice daily
AvutometinibDRUG3.2mg orally
DefactinibDRUG200 mg orally
avutometinib (VS-6766)DRUGavutometinib (VS-6766) monotherapy
avutometinib (VS-6766) and defactinibDRUGavutometinib (VS-6766) and defactinib combination
TemozolomideDRUGTemozolomide will be supplied as 5, 20, 100, 140, 180 or 250 mg hard capsules.
Biospecimen CollectionPROCEDUREUndergo blood and tissue sample collection
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Eligibility Criteria

Age Range18 Years to N/A
SexFEMALE
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: 1. Currently active on VS-6766-201(RAMP-201) study while receiving avutometinib in combination with defactinib, have not required prior permanent discontinuation of avutometinib plus defactinib. Any active dose hold from the RAMP-201 study should be ≤ 28 days at the time of Scre...

Countries:FranceUnited StatesBelgiumCanadaItalySpainUnited Kingdom
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Competitive Landscape -Gastric Cancer 112 trials

Top 20 of 45 competitors

CompanyTickerTrialsLead PhaseDrugs
AstraZeneca PLCAZN16PHASE3AZD0901, Ramucirumab, paclitaxel, Paclitaxel, Docetaxel
BeOne Medicines Ltd. Sponsored ADRONC4PHASE3Tislelizumab, Cisplatin, Leucovorin, 5-fluorouracil, Oxaliplatin
Jazz Pharmaceuticals Public Limited CompanyJAZZ3PHASE3Zanidatamab, Tislelizumab, Trastuzumab, Capecitabine, Oxaliplatin
Arcus Biosciences, Inc.RCUS2PHASE3Domvanalimab, Zimberelimab, Capecitabine, Fluorouracil, Leucovorin
Bristol-Myers Squibb CompanyBMY6PHASE2Pumitamig, Folfox, Capox, Nivolumab
Pfizer Inc.PFE7PHASE2disitamab vedotin, tucatinib
Agenus Inc.AGEN2PHASE3Balstilimab, Botensilimab, Folfox Protocol, XELOX, Nivolumab
AbbVie, Inc.ABBV2PHASE2Telisotuzumab Adizutecan, Budigalimab, Fluorouracil, Leucovorin, Oxaliplatin
Eli Lilly and CompanyLLY2PHASE2Ramucirumab, Paclitaxel
Compass Therapeutics, Inc.CMPX1PHASE2CTX-009, Paclitaxel
ALX Oncology Holdings, Inc.ALXO1PHASE2Evorpacept, Trastuzumab, Ramucirumab, Paclitaxel
GE Healthcare Technologies Inc.GEHC1PHASE2GEH300079 Positron-Emission Tomography/Computed Tomography
ImmunityBio IncIBRX1PHASE2N-803, Pembrolizumab
Apollomics Inc. Class AAPLM1PHASE2APL-101
Exelixis, Inc.EXEL2PHASE1cabozantinib, atezolizumab
Tango Therapeutics, Inc.TNGX2PHASE2Trifluridine/Tipiracil, Oxaliplatin, FOLFOX regimen, Nivolumab
Inhibrx Biosciences, Inc.INBX1PHASE1INBRX-106- Hexavalent OX40 agonist antibody, pembrolizumab, Carboplatin AUC-5, Pemetrexed/m2, Cisplatin/m2
Incyte CorporationINCY1PHASE2Capecitabine, Oxaliplatin, Retifanlimab
I-Mab Biopharma US LimitedIMAB2PHASE2Givastomig, Nivolumab, 5Fluorouracil, Leucovorin, Oxaliplatin
Enliven Therapeutics, Inc.ELVN1PHASE1ELVN-002, Trastuzumab, 5-Fluorouracil, Oxaliplatin, Capecitabine
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Recent Changes (Last 90 Days)

LOWAug 5, 2026NCT07747506NEW_TRIAL: changed

Frequently asked questions about Avutometinib

What is Avutometinib used for?

Avutometinib is an investigational small molecule being studied for the treatment of several cancers, including low grade serous ovarian cancer, glioblastoma multiforme (GBM), endometrioid cancer, and non small cell lung cancer. It is currently in Phase 2 clinical development for these indications.

What does Avutometinib target?

Avutometinib is a kinase inhibitor, as indicated by its '-tinib' suffix. It is being studied both as a monotherapy and in combination with other agents, such as defactinib, for the treatment of various solid tumors.

Who is developing Avutometinib?

Avutometinib is being developed by Verastem, Inc., a biopharmaceutical company. Verastem is listed on the stock exchange under the ticker symbol VSTM.

What phase is Avutometinib in?

Avutometinib is currently in Phase 2 clinical trials. It has received Breakthrough Therapy designation and Orphan Drug designation from the FDA for certain indications, reflecting its potential in treating serious conditions.

What clinical trials is Avutometinib in?

Avutometinib is being evaluated in several clinical trials, including NCT04625270, a Phase 2 study in recurrent low-grade serous ovarian cancer; NCT05798507, an early Phase 1 study in glioblastoma; NCT06630260, a Phase 1 study in malignant brain tumors; and NCT07747506, a continued access study in low-grade serous ovarian cancer.

Is Avutometinib the same as VS-6766?

Yes, Avutometinib is also known as VS-6766. In clinical trials, it is often referred to as avutometinib (VS-6766), particularly when studied in combination with defactinib for conditions like low-grade serous ovarian cancer and glioblastoma.