Defactinib

Phase 3Small molecule

Defactinib is being investigated as a cancer treatment that works by blocking a protein called FAK, which helps cancer cells survive and spread. It's being tested in clinical trials for various solid tumors.[1] Defactinib is currently being investigated across 14 indications[3], with its most advanced program in Phase 3.[1]

Defactinib is one of 330 small molecule programs targeting Non-Small Cell Lung Cancer. It is currently in Phase 3.

Indications

Total Trials

Active Trials

329

Competitors

Pipeline

Status by Indication[3][1]

Indication

Non-Small Cell Lung CancerCompleted

Advanced LymphomaActive, not recruiting

Gynecologic CancerRecruiting

Low-Grade Serous Ovarian CancerRecruiting

Advanced Colorectal CancerRecruiting

TrialActive, not recruiting

AdenocarcinomaActive, not recruiting

Pancreatic AdenocarcinomaNot yet recruiting

Gastric CancerRecruiting

GlioblastomaActive, not recruiting

Non-Small Cell Lung CancerPhase 2

Completed

Advanced LymphomaPhase 2

Active, not recruiting

Gynecologic CancerPhase 2

Recruiting

Low-Grade Serous Ovarian CancerPhase 3

Recruiting

Advanced Colorectal CancerPhase 2

Recruiting

TrialPhase 1

Active, not recruiting

AdenocarcinomaPhase 2

Active, not recruiting

Pancreatic AdenocarcinomaPhase 2

Not yet recruiting

Gastric CancerPhase 2

Recruiting

GlioblastomaPhase 1

Active, not recruiting

Showing 10 of 14 indications

Competition

Competitive Landscape

449 competitors in Non-Small Cell Lung Cancer

#DrugCompanyPhaseTrials

Leqembi

Eisai

Phase 3

1 2

Krazati

KKaruna Therapeutics, Inc., a Bristol Myers Squibb company

Phase 3

6 3

Defactinib

VVerastem, Inc.

Phase 3

2 4

Gotistobart

OOncoC4, Inc.

Phase 3

1 5

Remeron

OOrganon and Co

Preclinical

1 6

Aqupla

Jiangsu Simcere Pharmaceutical Co., Ltd.

Preclinical

—7

Ftorafur

TThe First Affiliated Hospital with Nanjing Medical University

Preclinical

—8

Vibramycin

PPfizer's Upjohn has merged with Mylan to form Viatris Inc.

Treatment

1 10

Valemetostat Tosylate

Daiichi Sankyo

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 1Active, not recruiting

Trial

NCT03875820

Phase 2Active, not recruiting

Advanced Lymphoma

NCT02465060

Q1 2027

Phase 2Recruiting

Gynecologic Cancer

NCT05787561

Q2 2027

Phase 2Recruiting

Low-Grade Serous Ovarian Cancer

NCT06394804

Phase 2Active, not recruiting

Adenocarcinoma

NCT04331041

Q3 2027

Phase 2Recruiting

Thyroid Cancer

NCT06007924

Clinical

Trials by Indication[1]

Non-Small Cell Lung Cancer2 trials

Defactinib, Avutometinib and Nivolumab for the Treatment of Anti-PD1 Refractory LKB1-Mutant Advanced Non-Small Cell Lung Cancer

Recruiting·EEmory University·Phase 2·NCT06495125·50 enrolled

Phase II Study of VS-6063 in Patients With KRAS Mutant Non-Small Cell Lung Cancer

Completed·VVerastem, Inc.·Phase 2·NCT01951690·55 enrolled

Avutometinib2 trials

5G-RUBY: Avutometinib and Defactinib in Malignant Brain Tumours

Recruiting·IInstitute of Cancer Research, United Kingdom·Phase 1/2·NCT06630260·182 enrolled

Phase Ib Study of Avutometinib, Defactinib, and Everolimus in RAS Pathway Mutant Endometrial Cancer

Recruiting·MM.D. Anderson Cancer Center·Phase 1·NCT07318324·31 enrolled

Low-grade Serous Ovarian Cancer2 trials

A Phase 3, Randomized, Open-Label Study of Combination Therapy With Avutometinib Plus Defactinib Versus Investigator's Choice of Treatment in Patients With Recurrent Low-Grade Serous Ovarian Cancer (LGSOC) (RAMP 301)

Recruiting·VVerastem, Inc.·Phase 3·NCT06072781·270 enrolled

Combination Targeted and Hormonal treAtMEnt of Low-gradE Serous Ovarian Cancer in the upfroNt Setting

Recruiting·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT06394804·20 enrolled

Ovarian Cancer1 trial

Phase I/Ib Study of Paclitaxel in Combination With VS-6063 in Patients With Advanced Ovarian Cancer

Completed·VVerastem, Inc.·Phase 1·NCT01778803·22 enrolled

Absorption1 trial

A Phase I, Open-label Study of Absorption, Metabolism, and Excretion of Defactinib (VS-6063) in Healthy Male Subjects

Completed·VVerastem, Inc.·Phase 1·NCT02913716·5 enrolled

17 more indications

MDS1 trial

Combined Evaluation of Epigenetic and Sensitising Therapy in AML and MDS

Recruiting·CClinical Hub for Interventional Research (CHOIR)·Phase 1·NCT05636514·12 enrolled

Pleural Mesothelioma1 trial

Pembrolizumab + Defactinib In Pleural Mesothelioma

Withdrawn·—·Phase 1·NCT04201145

Malignant Pleural Mesothelioma1 trial

Placebo Controlled Study of VS-6063 in Subjects With Malignant Pleural Mesothelioma

Terminated·VVerastem, Inc.·Phase 2·NCT01870609·344 enrolled

Gastric Cancer1 trial

Avutometinib and Defactinib in Diffuse Gastric Cancer

Recruiting·—·Phase 2·NCT06487221·27 enrolled

Trial1 trial

Phase I Trial of Defactinib and VS-6766.

Active, not recruiting·IInstitute of Cancer Research, United Kingdom·Phase 1·NCT03875820·87 enrolled

Pancreatic Ductal Adenocarcinoma1 trial

Study of Pembrolizumab With or Without Defactinib Following Chemotherapy as a Neoadjuvant and Adjuvant Treatment for Resectable Pancreatic Ductal Adenocarcinoma

Completed·LLei Zheng·Phase 2·NCT03727880·28 enrolled

Advanced Solid Malignancies1 trial

Study of FAK (Defactinib) and PD-1 (Pembrolizumab) Inhibition in Advanced Solid Malignancies (FAK-PD1)

Unknown status·NNHS Greater Glasgow and Clyde·Phase 1/2·NCT02758587·59 enrolled

Cutaneous Melanoma1 trial

Defactinib and Avutometinib, With or Without Encorafenib, for the Treatment of Patients With Brain Metastases From Cutaneous Melanoma

Recruiting·UUniversity of Utah·Phase 1/2·NCT06194929·33 enrolled

Glioblastoma1 trial

Identification of Treatment Concentrations of Defactinib or VS-6766 for the Treatment of Patients With Glioblastoma

Active, not recruiting·EEmory University·Phase 1·NCT05798507·14 enrolled

Advanced Cancer1 trial

Defactinib Combined With Pembrolizumab and Gemcitabine in Patients With Advanced Cancer

Completed·WWashington University School of Medicine·Phase 1·NCT02546531·43 enrolled

Adenocarcinoma1 trial

Stereotactic Body Radiotherapy and Focal Adhesion Kinase Inhibitor in Advanced Pancreas Adenocarcinoma

Active, not recruiting·WWashington University School of Medicine·Phase 2·NCT04331041·42 enrolled

Hematologic Malignancies1 trial

Phase I Dose Escalation Study of VS-6063 in Japanese Subjects With Non-Hematologic Malignancies

Completed·VVerastem, Inc.·Phase 1·NCT01943292·9 enrolled

Pancreatic Adenocarcinoma1 trial

Stereotactic Body Radiotherapy Plus FAK and RAF/MEK Inhibition in Advanced Pancreatic Adenocarcinoma

Not yet recruiting·WWashington University School of Medicine·Phase 2·NCT07126158·36 enrolled

Advanced Colorectal Cancer1 trial

Open-label Phase 2 Study of Avutometinib (RAF/MEK Clamp) in Combination With Defactinib (FAK Inhibitor) and Cetuximab in Patients With Unresectable, Anti-EGFR-Refractory Advanced Colorectal Cancer

Recruiting·MM.D. Anderson Cancer Center·Phase 2·NCT06369259·33 enrolled

Advanced Lymphoma1 trial

Molecular Analysis for Therapy Choice (MATCH)

Active, not recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT02465060·6,452 enrolled

Gynecologic Cancer1 trial

Single Arm Phase II Study of Avutometinib (VS-6766) and Defactinib in Advanced or Recurrent Mesonephric Gynecologic Cancer

Recruiting·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT05787561·40 enrolled

Thyroid Cancer1 trial

Phase II of Avutometinib (VS-6766) and Defactinib In RAF Dimer-Driven RAI-Refractory Differentiated and Anaplastic Thyroid Cancer Patients

Recruiting·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT06007924·30 enrolled

Sources

References

[1]
ClinicalTrials.gov — 25 trial records for Defactinib. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Defactinib compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Defactinib. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Verastem, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Focal Adhesion Kinase 1 Inhibitor; Protein Tyrosine Kinase 2 Beta Inhibitor[2]
Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: VVerastem, Inc.

Defactinib

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q1 2027

Q2 2027

Q3 2027

Trials by Indication[1]

Non-Small Cell Lung Cancer2 trials

Avutometinib2 trials

Low-grade Serous Ovarian Cancer2 trials

Ovarian Cancer1 trial

Absorption1 trial

MDS1 trial

Pleural Mesothelioma1 trial

Malignant Pleural Mesothelioma1 trial

Gastric Cancer1 trial

Trial1 trial

Pancreatic Ductal Adenocarcinoma1 trial

Advanced Solid Malignancies1 trial

Cutaneous Melanoma1 trial

Glioblastoma1 trial

Advanced Cancer1 trial

Adenocarcinoma1 trial

Hematologic Malignancies1 trial

Pancreatic Adenocarcinoma1 trial

Advanced Colorectal Cancer1 trial

Advanced Lymphoma1 trial

Gynecologic Cancer1 trial

Thyroid Cancer1 trial

References

Drug Info

Quick Facts

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