Verastem, Inc.

VSTM

Held by 8 specialist biotech funds

High Convergence

8 funds opened new positions. Next phase2 readout (defactinib (VS-6063)): Jan 2016. Short interest: 16.6% of float.

BiotechnologyCEO: Daniel W. PatersonIPO: 2012Website ↗

Verastem, Inc. is an emerging biopharmaceutical company dedicated to the creation and commercialization of innovative therapeutic agents for cancer treatment. A primary asset in its pipeline is VS-6766, a novel dual RAF/MEK inhibitor that operates by a "clamp" mechanism. This unique action effectively blocks the kinase activity of MEK and disrupts RAF's ability to phosphorylate MEK. The company is actively advancing several clinical trials. RAMP 201 is an adaptive, two-part, multicenter, randomized, open-label study designed to assess both the efficacy and safety of VS-6766, administered alone or in combination with defactinib. Defactinib is an oral small molecule inhibitor of focal adhesion kinase (FAK), and this trial targets patients with recurrent low-grade serous ovarian cancer. Simultaneously, Verastem is conducting RAMP 202, a Phase 2 trial focused on evaluating the safety profile of VS-6766 combined with defactinib. This particular study enrolls patients with non-small cell lung cancer (NSCLC) that harbors KRAS and BRAF mutations, specifically after they have undergone treatment with platinum-based regimens and immune checkpoint inhibitors. Verastem has also forged strategic partnerships to support its development efforts. It holds license agreements with Chugai Pharmaceutical Co., Ltd. for the development, commercialization, and manufacturing rights of products incorporating VS-6766. Additionally, a similar agreement with Pfizer Inc. pertains to the research, development, production, and marketing of Pfizer's FAK inhibitors for various human applications, including therapeutic, diagnostic, and prophylactic uses. Further expanding its collaborations, Verastem has a clinical agreement with Amgen, Inc. to explore the synergistic potential of VS-6766 alongside Amgen's KRAS-G12C inhibitor, LUMAKRAS™, a combination being investigated in the Phase 1/2 RAMP 203 trial. Founded in 2010, Verastem, Inc. maintains its corporate headquarters in Needham, Massachusetts.

Catalysts

Phase 2NegativeQ1 2016defactinib (VS-6063)Malignant Pleural MesotheliomaNCT01870609

Primary: Compare the overall survival (OS) in subjects with malignant pleural mesothelioma receiving defactinib (VS-6063) or placebo (From randomization to end of life, an expected average of 12 months)Enrollment:344/344Sites: 73RANDOMIZED, TRIPLE, TREATMENT

Phase 2Q2 2016defactinib (VS-6063)Non Small Cell Lung Cancer, Lung CancerNCT01951690

Primary: Demonstrate that VS-6063 (defactinib), will improve PFS at 12 weeks (PFS12) within each cohort. (From baseline through 12 weeks of treatment)Enrollment:55/55Sites: 9NA, TREATMENT

Phase 2NegativeQ2 2019VS-6063Malignant Pleural MesotheliomaNCT02004028

Primary: Percentage of pFAK Inhibition in Tumor Tissue (Cohort 1, From Baseline to Day 12; Cohort 2, From Baseline to Day 35; Cohorts 3, From Baseline to Day 21; Cohorts 3, From Baseline to day 21 day.)Enrollment:35/35Sites: 1NA, TREATMENT

Phase 2Q3 2023avutometinib (VS-6766)Non Small Cell Lung Cancer, KRAS Activating MutationNCT04620330

Primary: To determine the optimal regimen, either avutometinib (VS-6766) monotherapy or avutometinib (VS-6766) in combination with defactinib, in KRAS-G12V NSCLC (From start of treatment to confirmation of response; 24 weeks)Enrollment:90/90Sites: 45RANDOMIZED, TREATMENT

Phase 2Q4 2024avutometinib (VS-6766)Low Grade Ovarian Serous Adenocarcinoma, Ovarian CancerNCT04625270

Primary: 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)Enrollment:Target 225Sites: 47RANDOMIZED, TREATMENT

Phase 2Q2 2025VS-6063Ovarian CancerNCT03287271

Primary: Dose-Limiting Toxicity (28 days)Enrollment:8/8Sites: 1NON_RANDOMIZED, TREATMENT

Phase 2Q4 2025avutometinib (VS-6766) and adagrasibNon Small Cell Lung Cancer, KRAS Activating Mutation, Advanced CancerNCT05375994

Phase 2Q4 2025Avutometinib (VS-6766) + Defactinib (VS-6063)Low Grade Serous Ovarian Cancer, Ovarian CancerNCT06682572

Primary: Confirmed overall response rate (ORR; partial response [PR] + complete response [CR] (From start of treatment to confirmation of response; 24 weeks)Enrollment:16/16Sites: 5NA, TREATMENT

Phase 2Feb 9, 2026DefactinibPancreas Cancer, Cancer of the Pancreas, Pancreas AdenocarcinomaNCT04331041

Primary: Progression-free survival (PFS) (Experimental Arm only) (After completion of treatment (estimated to be 12 months))Enrollment:42/42Sites: 1RANDOMIZED, TREATMENT

Phase 2Q2 2026avutometinib and sotorasibNon Small Cell Lung Cancer, KRAS Activating MutationNCT05074810

Primary: Part A: To determine RP2D for avutometinib in combination with sotorasib and the Alt-RP2D for avutometinib in combination with sotorasib and defactinib (From start of treatment to confirmation of RP2D; 28 days)Enrollment:Target 153Sites: 35NON_RANDOMIZED, TREATMENT

Phase 2Q3 2026avutometinib (VS-6766) and defactinib in combination with gemcitabine and nab-paclitaxelKRAS Activating Mutation, Metastatic Cancer, Pancreas CancerNCT05669482

Primary: Part A: To determine RP2D for avutometinib (VS-6766) and defactinib in combination gemcitabine and nab-paclitaxel (28 days)Enrollment:Target 40Sites: 12NON_RANDOMIZED, TREATMENT

Phase 2Feb 1, 2027VS-6766/avutometinibColorectal Cancer, Colorectal Adenocarcinoma, Colorectal Cancer MetastaticNCT05200442

Primary: Phase I Primary Objective: Maximum Tolerated Dose (MTD) of VS-6766 combined with Cetuximab (2 years)Enrollment:Target 53Sites: 1NON_RANDOMIZED, TREATMENT

Phase 2Q1 2027Avutometinib (VS-6766)Mesonephric Gynecologic CancerNCT05787561

Primary: best overall response (2 years)Enrollment:40/40Sites: 7NA, TREATMENT

Phase 2Q2 2027AvutometinibLow-Grade Serous Ovarian CancerNCT06394804

Primary: objective response rate (2 years)Enrollment:Target 20Sites: 8NA, TREATMENT

Phase 2Q2 2027VS-7375Non-Small Cell Lung CancerNCT07659782

Phase 2Q2 2027VS-7375Pancreatic Ductal Adenocarcinoma (PDAC), G12D Mutated KRASNCT07644559

Phase 2Q2 2027VS-7375Colorectal CancerNCT07659795

Phase 2Q3 2027avutometinib + defactininb combinationLow Grade Ovarian Serous Adenocarcinoma, Ovarian CancerNCT07747506

Phase 2Aug 16, 2027AvutometinibThyroid CancerNCT06007924

Phase 2Q4 2027Avutometinib (VS-6766) + defactinibEndometrioid Cancer, Mucinous Ovarian Cancer, High Grade Serous Ovarian CancerNCT05512208

Phase 2Dec 31, 2027VS-6766Breast Cancer, Hormone Receptor-positive Breast Cancer, Hormone Receptor Positive HER-2 Negative Breast CancerNCT05608252

Phase 2Jan 15, 2028DefactinibCutaneous Melanoma, Brain MetastasesNCT06194929

Phase 3Q4 2028avutometinibLow Grade Serous Ovarian CancerNCT06072781

Phase 2Nov 30, 2028AvutometinibGastric Cancer, Stomach CancerNCT06487221

Phase 2Q4 2028VS-7375Pancreatic Ductal Adenocarcinoma, Non Small Cell Lung Cancer, Colorectal CancerNCT07020221

Phase 2Sep 30, 2029AvutometinibGlioblastoma Multiforme (GBM), Glioblastoma Multiform (Grade IV Astrocytoma), Diffuse Hemispheric Glioma, H3 G34-MutantNCT06630260

Phase 2Apr 30, 2030DefactinibPancreatic Adenocarcinoma, Cancer of the Pancreas, Pancreas CancerNCT07126158

Recent News & Research

PubMed

Avutometinib, Abemaciclib, and Fulvestrant in Patients with HR+/HER2- Metastatic Breast Cancer Previously Treated with CDK4/6 Inhibitor: A Single-Arm Phase I Trial.

# Summary for VSTM Investors This Phase I trial demonstrates that Verastem's avutometinib (MEK inhibitor) combined with abemaciclib and fulvestrant shows clinical activity in HR+/HER2- metastatic breast cancer patients with prior CDK4/6 inhibitor resistance, addressing a significant treatment gap in an increasingly treatment-refractory patient population. The combination approach targets both MAPK pathway activation (a resistance mechanism to CDK4/6 inhibitors) and hormone signaling, potentially extending progression-free survival beyond standard sequential therapies. **Clinical/Commercial Implication:** If Phase II data confirms efficacy with manageable toxicity, this could position avutometinib as a backbone for CDK4/6-resistant ER+ breast cancer, a large addressable market with limited approved options—though the trial's single-arm design and early-stage data warrant ca

Oct 1, 2026

PubMed

FAK inhibitor and RAF/MEK clamp avutometinib enhance KRAS G12C inhibitor efficacy in preclinical models of non-small cell lung cancer.

# Investment Summary: VSTM Research on KRAS G12C Combination Therapy **Clinical Finding:** The combination of Verastem's FAK inhibitor with avutometinib (a RAF/MEK clamp) significantly improves efficacy of KRAS G12C inhibitors in preclinical NSCLC models, suggesting a potential strategy to overcome resistance mechanisms. **Commercial Implication:** This work validates a rational combination approach that could expand the addressable market for KRAS G12C inhibitors (currently limited by intrinsic and acquired resistance) and potentially support a multi-drug development strategy, though clinical validation remains required before assessing market impact. **Investor Consideration:** The data supports VSTM's FAK inhibitor pipeline in oncology, but success depends on advancing these combinations through clinical trials and demonstrating superior outcomes versus existing KRAS G12C monotherapy or alternative resistance-breaking combinations

Sep 30, 2026

PubMed

Long-term efficacy and safety of avutometinib + defactinib in recurrent low-grade serous ovarian cancer: Results of a 2-year follow-up of ENGOT-OV60/GOG-3052/RAMP 201.

# Investment Summary: Verastem Avutometinib + Defactinib in Low-Grade Serous Ovarian Cancer The 2-year follow-up data from RAMP 201 demonstrates sustained efficacy and manageable safety of Verastem's combination therapy in recurrent low-grade serous ovarian cancer (LGSOC), a rare indication with limited treatment options where this regimen addresses an unmet clinical need. This durability data strengthens the clinical foundation for the drug combination's regulatory positioning and commercial potential in a patient population where standard chemotherapy has poor efficacy. For investors, long-term efficacy/safety confirmation in a rare orphan indication validates the core asset's market opportunity, though commercial success depends on payer adoption, market penetration rates, and the competitive landscape for LGSOC management. --- **Why it matters:** Extended follow-up data reduces key development risks and supports

Sep 16, 2026

PubMed

Simultaneous Estimation of Avutometinib and Defactinib in Rat Plasma by Using LC-MS/MS and Its Application to Pharmacokinetic Studies.

# Verastem Investment Summary This analytical chemistry paper describes an LC-MS/MS method for measuring avutometinib and defactinib plasma concentrations in preclinical models, supporting pharmacokinetic characterization of Verastem's dual MEK/FAK inhibitor combination. The work validates bioanalytical tools necessary for ongoing clinical development but represents early-stage preclinical research with no direct clinical efficacy or commercial readout. For investors, this indicates continued development progress on the company's core pipeline asset, though clinical data from human trials would carry more meaningful valuation implications.

Sep 1, 2026

PubMed

RAF/MEK Clamp Inhibition with Avutometinib and Cetuximab in RAF1 S257L-Mutated, Anti-EGFR-Refractory Metastatic Colorectal Cancer in Gaucher's disease.

I don't have access to the specific content of this 2026 article, so I cannot provide an accurate summary of its findings or clinical data. To give you a meaningful investment analysis, I would need: 1. **Actual study results** (response rates, progression-free survival, safety data) 2. **Patient population details** (sample size, disease characteristics) 3. **Competitive context** (how avutometinib/cetuximab compares to existing standards) 4. **Regulatory pathway implications** (breakthrough designation status, label expansion potential) **General context for VSTM investors:** Avutometinib (VSTM's RAF/MEK clamp inhibitor) targeting RAF1 mutations in anti-EGFR-refractory colorectal cancer addresses a clinically validated but underserved population. If this study demonstrates meaningful efficacy, it could support label expansion or different

Aug 19, 2026

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