Defactinib Evidence: Trial Results and Peer-Reviewed Publications

Pancreatic Neoplasms (15)

• Phase II Study of Vismodegib in Patients With SMO- or PTCH1-Mutated Tumors: Results From the National Cancer Institute Molecular Analysis for Therapy Choice Eastern Cooperative Oncology Group-American College of Radiology Imaging Network Trial (EAY131) Subprotocol T.
  JCO Precis Oncol · 2025 · PMID 41066729 · NCT02465060
• PIK3CB Inhibitor GSK2636771 in Cancers With PTEN Mutation/Deletion or Loss of PTEN Protein Expression: Results From the NCI-MATCH ECOG-ACRIN Trial (EAY131) Subprotocols N and P.
  JCO Precis Oncol · 2025 · PMID 40865033 · NCT02465060
• Phase II Study of Copanlisib in Patients With PTEN Loss: Results From NCI-MATCH ECOG-ACRIN Trial (EAY131) Subprotocols Z1G and Z1H.
  JCO Precis Oncol · 2025 · PMID 39913886 · NCT02465060
• Defactinib with avutometinib in patients with solid tumors: the phase 1 FRAME trial.
  Nat Med · 2025 · PMID 40579546 · NCT03875820
• Revolutionizing cancer drug development: Harnessing the potential of basket trials.
  Cancer · 2024 · PMID 37934000 · NCT02465060

Carcinoma, Non-Small-Cell Lung (2)

• Single agent VS-6766 or VS-6766 plus defactinib in KRAS-mutant non-small-cell lung cancer: the RAMP-202 phase II trial.
  Future Oncol · 2022 · PMID 35285277 · NCT04620330
• A first-in-Asian phase 1 study to evaluate safety, pharmacokinetics and clinical activity of VS-6063, a focal adhesion kinase (FAK) inhibitor in Japanese patients with advanced solid tumors.
  Cancer Chemother Pharmacol · 2017 · PMID 27025608 · NCT01951690

Ovarian Neoplasms (2)

• Efficacy and Safety of Avutometinib ± Defactinib in Recurrent Low-Grade Serous Ovarian Cancer: Primary Analysis of ENGOT-OV60/GOG-3052/RAMP 201.
  J Clin Oncol · 2025 · PMID 40644648 · NCT04625270
• Preclinical efficacy of RAF/MEK clamp avutometinib in combination with FAK inhibition in low grade serous ovarian cancer.
  Gynecol Oncol · 2024 · PMID 38493021 · NCT04625270

Uveal Melanoma (1)

• Gαs-Protein Kinase A (PKA) Pathway Signalopathies: The Emerging Genetic Landscape and Therapeutic Potential of Human Diseases Driven by Aberrant Gαs-PKA Signaling.
  Pharmacol Rev · 2022 · PMID 34663687 · NCT04720417

Mesothelioma, Malignant (1)

• Maintenance Defactinib Versus Placebo After First-Line Chemotherapy in Patients With Merlin-Stratified Pleural Mesothelioma: COMMAND-A Double-Blind, Randomized, Phase II Study.
  J Clin Oncol · 2020 · PMID 30785827 · NCT01870609

Glioblastoma (1)

• Window of opportunity study measuring defactinib and avutometinib delivery in glioblastomas.
  Cancer Chemother Pharmacol · 2026 · PMID 41790275 · NCT05798507

2026 (2 papers)

1. Window of opportunity study measuring defactinib and avutometinib delivery in glioblastomas.
   Del Valle MM, Colon EM, Kettimuthu K, et al. · Cancer Chemother Pharmacol · 2026 · Derived
   PubMed: PMID 41790275 · NCT05798507 · Glioblastoma
2. The combination of avutometinib and defactinib in treating recurrent low-grade serous ovarian cancer: a plain language summary of the Phase II clinical trial ENGOT-OV60/GOG-3052/RAMP 201.
   Banerjee SN, Van Nieuwenhuysen E, Aghajanian C, et al. · Future Oncol · 2026 · Derived
   PubMed: PMID 41384753 · NCT06072781 (RAMP 301)

2025 (6 papers)

1. Phase II Study of Vismodegib in Patients With SMO- or PTCH1-Mutated Tumors: Results From the National Cancer Institute Molecular Analysis for Therapy Choice Eastern Cooperative Oncology Group-American College of Radiology Imaging Network Trial (EAY131) Subprotocol T.
   Tsao AS, Song Z, Ho AL, et al. · JCO Precis Oncol · 2025 · Derived
   PubMed: PMID 41066729 · NCT02465060 · Pancreatic Neoplasms
2. PIK3CB Inhibitor GSK2636771 in Cancers With PTEN Mutation/Deletion or Loss of PTEN Protein Expression: Results From the NCI-MATCH ECOG-ACRIN Trial (EAY131) Subprotocols N and P.
   Janku F, Jegede OA, Puhalla SL, et al. · JCO Precis Oncol · 2025 · Derived
   PubMed: PMID 40865033 · NCT02465060 · Pancreatic Neoplasms
3. GOG-3097/ENGOT-ov81/GTG-UK/RAMP 301: a phase 3, randomized trial evaluating avutometinib plus defactinib compared with investigator's choice of treatment in patients with recurrent low grade serous ovarian cancer.
   Grisham R, Monk BJ, Van Nieuwenhuysen E, et al. · Int J Gynecol Cancer · 2025 · Derived
   PubMed: PMID 39375168 · NCT06072781 (RAMP 301)
4. Phase II Study of Copanlisib in Patients With PTEN Loss: Results From NCI-MATCH ECOG-ACRIN Trial (EAY131) Subprotocols Z1G and Z1H.
   Gouda MA, Wei Z, Rodon J, et al. · JCO Precis Oncol · 2025 · Derived
   PubMed: PMID 39913886 · NCT02465060 · Pancreatic Neoplasms
5. Efficacy and Safety of Avutometinib ± Defactinib in Recurrent Low-Grade Serous Ovarian Cancer: Primary Analysis of ENGOT-OV60/GOG-3052/RAMP 201.
   Banerjee SN, Van Nieuwenhuysen E, Aghajanian C, et al. · J Clin Oncol · 2025 · Derived
   PubMed: PMID 40644648 · NCT04625270 (RAMP 201) · Ovarian Neoplasms
6. Defactinib with avutometinib in patients with solid tumors: the phase 1 FRAME trial.
   Banerjee S, Krebs MG, Greystoke A, et al. · Nat Med · 2025 · Derived
   PubMed: PMID 40579546 · NCT03875820 (FRAME) · Pancreatic Neoplasms

2024 (3 papers)

1. Revolutionizing cancer drug development: Harnessing the potential of basket trials.
   Subbiah V, Burris HA, Kurzrock R, et al. · Cancer · 2024 · Derived
   PubMed: PMID 37934000 · NCT02465060 · Pancreatic Neoplasms
2. Preclinical efficacy of RAF/MEK clamp avutometinib in combination with FAK inhibition in low grade serous ovarian cancer.
   McNamara B, Demirkiran C, Hartwich TMP, et al. · Gynecol Oncol · 2024 · Derived
   PubMed: PMID 38493021 · NCT04625270 (RAMP 201) · Ovarian Neoplasms
3. Phase II Study of Sunitinib in Tumors With c-KIT Mutations: Results From the NCI MATCH ECOG-ACRIN Trial (EAY131) Subprotocol V.
   Gien LT, Song Z, Poklepovic A, et al. · JCO Precis Oncol · 2024 · Derived
   PubMed: PMID 39666929 · NCT02465060 · Pancreatic Neoplasms

2023 (3 papers)

1. Trametinib in Patients With NF1-, GNAQ-, or GNA11-Mutant Tumors: Results From the NCI-MATCH ECOG-ACRIN Trial (EAY131) Subprotocols S1 and S2.
   Wisinski KB, Flamand Y, Wilson MA, et al. · JCO Precis Oncol · 2023 · Derived
   PubMed: PMID 37053535 · NCT02465060 · Pancreatic Neoplasms
2. The NCI-MATCH trial: lessons for precision oncology.
   O'Dwyer PJ, Gray RJ, Flaherty KT, et al. · Nat Med · 2023 · Derived
   PubMed: PMID 37322121 · NCT02465060 · Pancreatic Neoplasms
3. Mind the (guidance) signals! Translational relevance of semaphorins, plexins, and neuropilins in pancreatic cancer.
   Fard D, Giraudo E, Tamagnone L, et al. · Trends Mol Med · 2023 · Derived
   PubMed: PMID 37598000 · NCT04331041 · Pancreatic Neoplasms

2022 (5 papers)

1. Platelet p110β mediates platelet-leukocyte interaction and curtails bacterial dissemination in pneumococcal pneumonia.
   Schrottmaier WC, Kral-Pointner JB, Salzmann M, et al. · Cell Rep · 2022 · Derived
   PubMed: PMID 36351402 · NCT02465060 · Pancreatic Neoplasms
2. Gαs-Protein Kinase A (PKA) Pathway Signalopathies: The Emerging Genetic Landscape and Therapeutic Potential of Human Diseases Driven by Aberrant Gαs-PKA Signaling.
   Ramms DJ, Raimondi F, Arang N, et al. · Pharmacol Rev · 2022 · Derived
   PubMed: PMID 34663687 · NCT04720417 · Uveal Melanoma
3. Phase II Study of Taselisib in PIK3CA-Mutated Solid Tumors Other Than Breast and Squamous Lung Cancer: Results From the NCI-MATCH ECOG-ACRIN Trial (EAY131) Subprotocol I.
   Krop IE, Jegede OA, Grilley-Olson JE, et al. · JCO Precis Oncol · 2022 · Derived
   PubMed: PMID 35138919 · NCT02465060 · Pancreatic Neoplasms
4. Phase II Study of Copanlisib in Patients With Tumors With PIK3CA Mutations: Results From the NCI-MATCH ECOG-ACRIN Trial (EAY131) Subprotocol Z1F.
   Damodaran S, Zhao F, Deming DA, et al. · J Clin Oncol · 2022 · Derived
   PubMed: PMID 35133871 · NCT02465060 · Pancreatic Neoplasms
5. Single agent VS-6766 or VS-6766 plus defactinib in KRAS-mutant non-small-cell lung cancer: the RAMP-202 phase II trial.
   Capelletto E, Bironzo P, Denis L, et al. · Future Oncol · 2022 · Derived
   PubMed: PMID 35285277 · NCT04620330 (RAMP202) · Carcinoma, Non-Small-Cell Lung

2021 (2 papers)

1. Molecular Landscape and Actionable Alterations in a Genomically Guided Cancer Clinical Trial: National Cancer Institute Molecular Analysis for Therapy Choice (NCI-MATCH).
   Flaherty KT, Gray RJ, Chen AP, et al. · J Clin Oncol · 2021 · Derived
   PubMed: PMID 33048619 · NCT02465060 · Pancreatic Neoplasms
2. Phase II Study of AZD4547 in Patients With Tumors Harboring Aberrations in the FGFR Pathway: Results From the NCI-MATCH Trial (EAY131) Subprotocol W.
   Chae YK, Hong F, Vaklavas C, et al. · J Clin Oncol · 2021 · Derived
   PubMed: PMID 32463741 · NCT02465060 · Pancreatic Neoplasms

2020 (1 paper)

1. Maintenance Defactinib Versus Placebo After First-Line Chemotherapy in Patients With Merlin-Stratified Pleural Mesothelioma: COMMAND-A Double-Blind, Randomized, Phase II Study.
   Fennell DA, Baas P, Taylor P, et al. · J Clin Oncol · 2020 · Derived
   PubMed: PMID 30785827 · NCT01870609 (COMMAND) · Mesothelioma, Malignant

2017 (1 paper)

1. A first-in-Asian phase 1 study to evaluate safety, pharmacokinetics and clinical activity of VS-6063, a focal adhesion kinase (FAK) inhibitor in Japanese patients with advanced solid tumors.
   Shimizu T, Fukuoka K, Takeda M, et al. · Cancer Chemother Pharmacol · 2017 · Derived
   PubMed: PMID 27025608 · NCT01951690 · Carcinoma, Non-Small-Cell Lung

2016 (1 paper)

1. Is the NCI MATCH trial a match for gynecologic oncology?
   Moore KN, Mannel RS · Gynecol Oncol · 2016 · Derived
   PubMed: PMID 26586415 · NCT02465060 · Pancreatic Neoplasms