Pipeline

Avutometinib is a RAF/MEK Clamp that induces inactive complexes of MEK with ARAF, BRAF and CRAF, potentially creating a more complete and durable anti-tumor response through maximal RAS pathway inhibition.

Defactinib is a best-in-class selective FAK inhibitor that has been studied as a monotherapy and in combination in patients with solid tumors.

Verastem Oncology’s pipeline is focused on RAS/MAPK-driven cancers, specifically novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition and FAK inhibition.

Indication Study Name Discovery Preclinical Phase 1 Phase 2 Phase 3

R/R LGSOC

Regimen: avutometinib + defactinib vs investigator’s choice of treatments (ICT)

Study Name: RAMP 301

RAMP 301
Discovery
Preclinical
Phase 1
Phase 2
Phase 3

R/R LGSOC1,2

Regimen: avutometinib +/- defactinib

Study Name: RAMP 201

RAMP 201
Discovery
Preclinical
Phase 1
Phase 2
Phase 3

R/R NSCLC (KRAS G12C)

Regimen: avutometinib + sotorasib +/- defactinib

Study Name: RAMP 203

RAMP 203
Discovery
Preclinical
Phase 1
Phase 2
Phase 3

Pancreatic Ductal Adenocarcinoma

Regimen: avutometinib + gemcitabine/nab-paclitaxel + defactinib

Study Name: RAMP 205

RAMP 205
Discovery
Preclinical
Phase 1
Phase 2
Phase 3

Verastem Oncology entered into a collaboration agreement with GenFleet Therapeutics that includes three discovery programs and provides Verastem with exclusive options to license three compounds selected for collaboration after successful completion of pre-determined milestones in Phase 1 trials. The licenses would give Verastem Oncology development and commercialization rights outside of the GenFleet territories of mainland China, Hong Kong, Macau, and Taiwan.

Verastem has identified GFH375/VS-7375, a potential best-in-class, potent and selective oral KRAS G12D (ON/OFF) inhibitor, as the lead discovery program from the collaboration. GenFleet’s IND for GFH375/VS-7375 was approved in China in June 2024 and the Phase 1/2 trial in KRAS G12D-mutant solid tumors was subsequently initiated and the first patient was dosed in July 2024.

Indication Study Name Discovery Preclinical Phase 1 Phase 2 Phase 3

KRAS G12D-mutant Solid Tumors

Regimen: Oral KRAS G12D (ON/OFF) Inhibitor
GenFleet initiated Phase 1/2 trial in China.

Discovery
Preclinical
Phase 1
Phase 2
Phase 3

Undisclosed

Discovery
Preclinical
Phase 1
Phase 2
Phase 3

Undisclosed

Discovery
Preclinical
Phase 1
Phase 2
Phase 3
Indication Study Name Discovery Preclinical Phase 1 Phase 2 Phase 3

R/R LGSOC

Regimen: avutometinib + defactinib

Study Name: IST-FRAME

IST-FRAME
Discovery
Preclinical
Phase 1
Phase 2
Phase 3

Gynecological Cancers (RAS Pathway-driven)3

Regimen: avutometinib + defactinib

Study Name: IST

IST
Discovery
Preclinical
Phase 1
Phase 2
Phase 3

Mesonephric

Regimen: avutometinib + defactinib

Study Name: IST

IST
Discovery
Preclinical
Phase 1
Phase 2
Phase 3

R/R NSCLC (KRAS mt)

Regimen: avutometinib + everolimus (mTORi)

Study Name: IST

IST
Discovery
Preclinical
Phase 1
Phase 2
Phase 3

R/R Colorectal Cancer

Regimen: avutometinib + cetuximab (EGFRi)

Study Name: IST

IST
Discovery
Preclinical
Phase 1
Phase 2
Phase 3

ER+ Breast Cancer

Regimen: avutometinib + abemaciclib + fulvestrant

Study Name: IST

IST
Discovery
Preclinical
Phase 1
Phase 2
Phase 3

1FDA Breakthrough Therapy Designation
2Registration-directed trial

These studies are investigating treatments or outcomes that have not received approval from a health authority. We are working diligently to demonstrate the safety and efficacy of these products, but there is no guarantee that the outcome of these studies will result in approval. We invite you to review our expanded access policy.


We believe in the power of collaboration to help us push forward with new and promising cancer treatments. Our Verastem Oncology Investigator Sponsored Trial (IST) program strives to advance medical and scientific knowledge for our product candidates and disease states of interest. We welcome partnerships with researchers and sponsoring institutions that build on this mission.

For additional details regarding our IST program or to submit a research request, please download, complete and submit the form below via email to verastem-IST@verastem.com.

Verastem Oncology also accepts proposals for preclinical research collaborations. For more information, please reach out to us at research@verastem.com.