Defactinib

Phase 2Small 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 9 indications[3], with its most advanced program in Phase 2.[1]

Defactinib is one of 254 small molecule programs targeting Cutaneous Melanoma. It is currently in Phase 2.

Indications

Total Trials

Active Trials

253

Competitors

Pipeline

Status by Indication[3][1]

Indication

Cutaneous MelanomaRecruiting

AdenocarcinomaActive, not recruiting

Pancreatic AdenocarcinomaNot yet recruiting

Non-Small Cell Lung CancerCompleted

Gastric CancerRecruiting

GlioblastomaActive, not recruiting

MDSRecruiting

TrialActive, not recruiting

AvutometinibRecruiting

Cutaneous MelanomaPhase 2

Recruiting

AdenocarcinomaPhase 2

Active, not recruiting

Pancreatic AdenocarcinomaPhase 2

Not yet recruiting

Non-Small Cell Lung CancerPhase 2

Completed

Gastric CancerPhase 2

Recruiting

GlioblastomaPhase 1

Active, not recruiting

MDSPhase 1

Recruiting

TrialPhase 1

Active, not recruiting

AvutometinibPhase 2

Recruiting

Competition

Competitive Landscape

414 competitors in Non-Small Cell Lung Cancer

#DrugCompanyPhaseTrials

SKB264

SSichuan Kelun-Biotech Biopharmaceutical Co., Ltd.

Phase 3

5 2

Defactinib

VVerastem, Inc.

Phase 2

2 3

BPI-7711

BBeta Pharma Shanghai

Phase 3

3 4

Remeron

OOrganon and Co

Preclinical

1 5

Aqupla

Jiangsu Simcere Pharmaceutical Co., Ltd.

Preclinical

—6

Ftorafur

TThe First Affiliated Hospital with Nanjing Medical University

Preclinical

—7

Vibramycin

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

Treatment

1 9

PD-1 Inhibitors

BBioNTech US Inc.

Preclinical

2 10

Valemetostat Tosylate

Daiichi Sankyo

Preclinical

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

Timeline

Expected Milestones

Q1 2027

Phase 2Active, not recruiting

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

Q4 2027

Phase 1Active, not recruiting

Glioblastoma

NCT05798507

Phase 1Recruiting

MDS

NCT05636514

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

Pancreatic Ductal Adenocarcinoma2 trials

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

A Phase 1/2, Open-label Study of VS-7375, a KRAS G12D (ON/OFF) Inhibitor, as Monotherapy and in Combination, in Patients With Advanced KRAS G12D-Mutated Solid Tumors

Recruiting·VVerastem, Inc.·Preclinical·NCT07020221·616 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

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

17 more indications

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

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

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

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

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

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

Malignant Pleural Mesothelioma1 trial

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

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

Advanced Lymphoma1 trial

Molecular Analysis for Therapy Choice (MATCH)

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

Gynecologic Cancer1 trial

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

Active, not recruiting·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT05787561·40 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

Sources

References

[1]
ClinicalTrials.gov — 26 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 2[1]
Company: VVerastem, Inc.

Defactinib

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2027

Q2 2027

Q3 2027

Q4 2027

Trials by Indication[1]

Non-Small Cell Lung Cancer2 trials

Pancreatic Ductal Adenocarcinoma2 trials

Avutometinib2 trials

Low-grade Serous Ovarian Cancer2 trials

Pancreatic Adenocarcinoma1 trial

Advanced Solid Malignancies1 trial

Cutaneous Melanoma1 trial

Adenocarcinoma1 trial

Glioblastoma1 trial

Advanced Cancer1 trial

Gastric Cancer1 trial

Trial1 trial

MDS1 trial

Pleural Mesothelioma1 trial

Ovarian Cancer1 trial

Absorption1 trial

Malignant Pleural Mesothelioma1 trial

Hematologic Malignancies1 trial

Advanced Lymphoma1 trial

Thyroid Cancer1 trial

Gynecologic Cancer1 trial

Advanced Colorectal Cancer1 trial

References

Drug Info

Quick Facts

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