Drugs / Defactinib
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Defactinib

Small molecule targets PTK2, PTK2B via inhibition

Developed for
non-small cell lung carcinoma · malignant pancreatic neoplasm · ovarian cancer · ovarian serous tumor · endometrioid adenocarcinoma · glioblastoma · malignant ovarian serous tumor · malignant pleural mesothelioma
+26 more · pancreatic adenocarcinoma · plasma cell myeloma · acute myeloid leukemia · adult lymphoma · cancer · cervical cancer · chronic myelomonocytic leukemia · colorectal cancer · cutaneous melanoma · diffuse gastric cancer · diffuse glioma, H3 G34 mutant · endometrial cancer · lung adenocarcinoma · malignant epithelial tumor of ovary · mesonephric neoplasm · mesothelioma · metastatic malignant neoplasm · metastatic malignant neoplasm in the brain · mucinous ovarian cancer · myelodysplastic syndrome · ovarian serous adenocarcinoma · pancreatic ductal adenocarcinoma · pancreatic neoplasm · refractory malignant neoplasm · thyroid cancer · uveal melanoma
Investigated by
Verastem, Inc. · National Cancer Institute (NCI) · Merck Sharp & Dohme LLC · Memorial Sloan Kettering Cancer Center · Washington University School of Medicine · Cancer Research UK
+31 more · Emory University · European Network of Gynaecological Oncological Trial Groups (ENGOT) · GOG Foundation · Institute of Cancer Research, United Kingdom · M.D. Anderson Cancer Center · The Foundation for Barnes-Jewish Hospital · Amgen · Australia New Zealand Gynaecological Oncology Group · Australian National University · Cambridge University Hospitals NHS Foundation Trust · Clinical Hub for Interventional Research (CHOIR) · Japanese Gynecologic Oncology Group · Korean Gynecologic Oncology Group · Lei Zheng · Michael McHale · Minderoo Foundation · NHS Greater Glasgow and Clyde · Nine Girls Ask · Queen's University, Belfast · Raphael Bueno, MD · Royal Marsden NHS Foundation Trust · Ryan H. Moy, MD, PhD · The University of New South Wales · Thomas Jefferson University · University of Cambridge · University of Edinburgh · University of Glasgow · University of Leicester · University of Oklahoma · University of Southampton · University of Utah

Regulatory milestones approvals, filings & regulatory actions · 1 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved US (FDA) AVMAPKI FAKZYNJA CO-PACK (COPACKAGED) 2025-05-08 fda.gov

Trials 36 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 01 trial
Phase 0 NCT05798507 Jul 2023 → Dec 2026 expected glioblastoma Emory University Active not recruiting No outcome recorded
Phase 110 trials
Phase 1 NCT07318324 May 2026 → Aug 2028 expected endometrial cancer M.D. Anderson Cancer Center Recruiting No outcome recorded
Phase 1 NCT05636514 Dec 2022 → Aug 2026 overdue acute myeloid leukemia, chronic myelomonocytic leukemia, myelodysplastic syndrome Clinical Hub for Interventional Research (CHOIR) Recruiting No outcome recorded
Phase 1 NCT04201145 Sep 2020 → Dec 2021 malignant pleural mesothelioma Raphael Bueno, MD Withdrawn No outcome recorded
Phase 1 NCT03875820 Dec 2017 → Apr 2023 overdue endometrioid adenocarcinoma, malignant pancreatic neoplasm, non-small cell lung carcinoma, ovarian serous tumor Institute of Cancer Research, United Kingdom Active not recruiting No outcome recorded
Phase 1 NCT02943317 Oct 2016 → Aug 2018 malignant epithelial tumor of ovary Verastem, Inc. Terminated No outcome recorded
Phase 1 NCT02546531 Feb 2016 → Nov 2017 malignant pancreatic neoplasm Washington University School of Medicine Completed No outcome recorded
Phase 1 NCT02913716 Aug → Sep 2015 Verastem, Inc. Completed No outcome recorded
Phase 1 NCT01943292 Sep 2013 → Jun 2014 Verastem, Inc. Completed No outcome recorded
Phase 1 NCT01778803 Feb 2013 → Feb 2015 ovarian cancer Verastem, Inc. Completed No outcome recorded
Phase 1 NCT00787033 Dec 2008 → Mar 2011 cancer Verastem, Inc. Completed No outcome recorded
Phase 1/26 trials
Phase 1/2 NCT06630260 Nov 2024 → Sep 2029 expected diffuse glioma, H3 G34 mutant, glioblastoma Institute of Cancer Research, United Kingdom Recruiting No outcome recorded
Phase 1/2 NCT06194929 Mar 2024 → Jan 2028 expected cutaneous melanoma, metastatic malignant neoplasm in the brain University of Utah Recruiting No outcome recorded
Phase 1/2 NCT05669482 Mar 2023 → Aug 2026 overdue malignant pancreatic neoplasm, metastatic malignant neoplasm Verastem, Inc. Active not recruiting No outcome recorded
Phase 1/2 NCT05074810 Apr 2022 → Jun 2026 overdue non-small cell lung carcinoma Verastem, Inc. Active not recruiting No outcome recorded
Phase 1/2 NCT03287271 Feb 2018 → Apr 2025 overdue ovarian cancer Michael McHale Recruiting No outcome recorded
Phase 1/2 NCT02758587 Jul 2017 → May 2019 mesothelioma, non-small cell lung carcinoma, pancreatic neoplasm NHS Greater Glasgow and Clyde Unknown No outcome recorded
Phase 218 trials
Phase 2 NCT07126158 Apr 2026 → Apr 2030 expected pancreatic adenocarcinoma Washington University School of Medicine Recruiting No outcome recorded
Phase 2 NCT06369259 Feb 2025 → Feb 2029 expected colorectal cancer M.D. Anderson Cancer Center Recruiting No outcome recorded
Phase 2 NCT06682572 Oct 2024 → Dec 2025 overdue ovarian cancer, ovarian serous tumor Verastem, Inc. Active not recruiting No outcome recorded
Phase 2 NCT06487221 Oct 2024 → Nov 2028 expected diffuse gastric cancer Ryan H. Moy, MD, PhD Recruiting No outcome recorded
Phase 2 NCT06495125 Jul 2024 → Mar 2029 expected lung adenocarcinoma Emory University Recruiting No outcome recorded
Phase 2 NCT06394804 Apr 2024 → Apr 2027 expected malignant ovarian serous tumor Memorial Sloan Kettering Cancer Center Recruiting No outcome recorded
Phase 2 NCT06007924 Aug 2023 → Aug 2027 expected thyroid cancer Memorial Sloan Kettering Cancer Center Recruiting No outcome recorded
Phase 2 NCT05787561 Mar 2023 → Mar 2027 expected mesonephric neoplasm Memorial Sloan Kettering Cancer Center Recruiting No outcome recorded
Phase 2 NCT05512208 Feb 2023 → Dec 2027 expected cervical cancer, endometrioid adenocarcinoma, malignant ovarian serous tumor, mucinous ovarian cancer University of Oklahoma Recruiting No outcome recorded
Phase 2 NCT04331041 Aug 2021 → Feb 2026 overdue pancreatic adenocarcinoma Washington University School of Medicine Active not recruiting No outcome recorded
Phase 31 trial
Phase 3 NCT06072781 Mar 2024 → Oct 2028 expected ovarian serous tumor Verastem, Inc. Recruiting No outcome recorded
Also used as a comparator or background therapy in 2 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial
Phase 1 NCT02372227 Background Jan → Oct 2015 malignant mesothelioma Verastem, Inc. Terminated No outcome recorded
Phase 21 trial
Phase 2 NCT01870609 Comparator Sep 2013 → Jan 2016 malignant pleural mesothelioma Verastem, Inc. Terminated No outcome recorded

Evidence & citations 12 cited values

Every value below carries the sentence it was read from. 41 sources stand behind the page.

FieldValueCited text
Known as defactinib “VS-6063 (also known as defactinib or PF-04554878) is a second-generation inhibitor of focal adhesion kinase (FAK) and proline-rich tyrosine kinase-2 (Pyk2).” PMID 26334219 Sep 2015
33

NCT06682572

NCT06394804

NCT06072781

NCT05669482

NCT04331041

NCT05636514

NCT03875820

NCT06630260

NCT02913716

NCT05798507

NCT04625270

NCT06194929

NCT04201145

NCT06495125

NCT02546531

NCT01943292

NCT06369259

NCT01778803

NCT01870609

NCT06487221

NCT01951690

NCT06007924

NCT03727880

NCT04620330

NCT07126158

NCT02465060

NCT05787561

NCT02758587

NCT05074810

NCT07318324

NCT04439331

NCT04720417

NCT05512208

Known as DEFACITINIB ChEMBL registry synonym — accepted as the source's own label CHEMBL3137331
Known as defactinib (VS-6063) defactinib (VS-6063)NCT06072781
Known as defactinib hydrochloride “Patients receive defactinib hydrochloride (defactinib) orally (PO) 400 mg twice daily (BID) on days 1-28.” NCT04439331
Known as PF-04554878 “PURPOSE: VS-6063 (also known as defactinib or PF-04554878) is a second-generation inhibitor of focal adhesion kinase and proline-rich tyrosine kinase-2.” PMID 27025608 Mar 2016
1

NCT00787033

Known as VS-6063 “This study is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study of defactinib (VS-6063) in subjects with malignant pleural mesothelioma (MPM)” NCT01870609
8

VS-6063 (also known as defactinib or PF-04554878) is a second-generation inhibitor of focal adhesion kinase (FAK) and proline-rich tyrosine kinase-2 (Pyk2).” PMID 26334219 Sep 2015

“PURPOSE: VS-6063 (also known as defactinib or PF-04554878) is a second-generation inhibitor of focal adhesion kinase and proline-rich tyrosine kinase-2.” PMID 27025608 Mar 2016

“Avutometinib (VS-6766) + Defactinib (VS-6063)” NCT06682572

VS-6063NCT03875820

NCT02372227

NCT03287271

NCT02943317

NCT02004028

Known as VS-6063 HYDROCHLORIDE ChEMBL registry synonym — accepted as the source's own label CHEMBL3137331
Action Inhibit “Protein tyrosine kinase 2 beta inhibitor” CHEMBL3137331
2

“a focal adhesion kinase inhibitor” NCT01943292

“Focal adhesion kinase 1 inhibitor” CHEMBL3137331

Modality Small molecule CHEMBL3137331
Route Oral Oral defactinib (VS-6063) administered twice daily” NCT01778803
Target PTK2 “Focal adhesion kinase 1 inhibitor” CHEMBL3137331
Target PTK2B “Protein tyrosine kinase 2 beta inhibitor” CHEMBL3137331