Sponsor: Ryan H. Moy, MD, PhD (other)
Phase: 2
Start date: Oct. 28, 2024
Planned enrollment: 27
Defactinib (PF-04554878; VS-6063) is an oral small-molecule kinase inhibitor developed primarily for cancer treatment. Although still investigational in many tumor types, it is no longer wholly investigational in the United States: on May 8, 2025, the FDA granted accelerated approval to defactinib combined with avutometinib for adults with previously treated, KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC). Defactinib monotherapy is not FDA-approved. (fda.gov)
Defactinib inhibits focal adhesion kinase (FAK/PTK2) and the related kinase PYK2/PTK2B. These non-receptor tyrosine kinases transmit signals from integrins and growth-factor receptors that can promote tumor-cell survival, proliferation, migration and invasion. FAK inhibition may also affect cancer stem-like cells and the tumor microenvironment. In combination with avutometinib, defactinib is intended to suppress adaptive FAK signaling that may limit responses to RAF–MEK pathway inhibition. (pubmed.ncbi.nlm.nih.gov)
Overall, meaningful clinical efficacy has been demonstrated principally when defactinib is combined with avutometinib in KRAS-mutated LGSOC; single-agent activity has generally been limited.
In the initial monotherapy phase I trial, common adverse events included nausea (37%), fatigue (33%), vomiting (28%), diarrhea (22%) and headache (22%). Reversible dose-limiting toxicities included headache, fatigue and unconjugated hyperbilirubinemia. (pubmed.ncbi.nlm.nih.gov)
For avutometinib plus defactinib, frequent adverse reactions and laboratory abnormalities included increased creatine phosphokinase, gastrointestinal symptoms, fatigue, rash, edema, musculoskeletal pain, elevated liver enzymes or bilirubin, cytopenias and ocular effects including vitreoretinal disorders and visual impairment. Because these data concern combination treatment, individual events cannot necessarily be attributed to defactinib alone. (fda.gov)
Last updated: Sep 2026
Avutometinib (RO5126766/CH5126766/VS-6766) is an oral small-molecule RAF/MEK inhibitor developed for RAS–RAF–MEK–ERK pathway-driven cancers. Although still investigational in most settings, it is no longer wholly investigational in the United States: on May 8, 2025, the FDA granted accelerated approval to avutometinib plus the FAK inhibitor defactinib for adults with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) previously treated with systemic therapy. (fda.gov)
Avutometinib is described as a RAF/MEK clamp. It binds allosterically to MEK1/2, inhibits MEK activity, and stabilizes an inactive RAF–MEK complex in which MEK cannot be phosphorylated and released by RAF. This suppresses both MEK activity and the compensatory RAF reactivation that can limit conventional MEK inhibitors, thereby reducing downstream ERK signaling. (pmc.ncbi.nlm.nih.gov)
Defactinib inhibits focal adhesion kinase (FAK), an adaptive signaling pathway that may become activated after RAF/MEK inhibition. This provides the rationale for combining the two drugs. (pubmed.ncbi.nlm.nih.gov)
The principal clinical evidence comes from the phase II RAMP-201 study of avutometinib plus defactinib in 115 heavily pretreated patients with recurrent LGSOC:
At approximately two years of follow-up, median duration of response remained 31.1 months and median PFS was 12.9 months overall. Median PFS was 19.6 months in KRAS-mutated and 12.7 months in KRAS-wild-type disease. (pubmed.ncbi.nlm.nih.gov)
The FDA approval population comprised 57 patients with KRAS-mutated LGSOC; blinded independent review found a 44% ORR (95% CI, 31%–58%), with responses lasting 3.3 to 31.1 months at the analysis cutoff. (fda.gov)
Earlier phase I monotherapy testing in 52 patients with advanced solid tumors produced three partial responses—two in BRAF-mutated melanoma and one in NRAS-mutated melanoma—and tumor shrinkage in approximately 40% of patients, although this was not a tumor-specific response rate. (pubmed.ncbi.nlm.nih.gov)
In the 115-patient RAMP-201 combination cohort, the most frequent grade 3 or higher treatment-related adverse events were:
Approximately 10%–12% discontinued treatment because of adverse events, and no treatment-related deaths were reported in the two-year analysis. (pubmed.ncbi.nlm.nih.gov)
Other commonly reported toxicities include rash or acneiform dermatitis, nausea, fatigue, liver-enzyme elevations, edema, musculoskeletal symptoms, gastrointestinal effects and ocular or vitreoretinal disorders. Early monotherapy studies also identified creatine phosphokinase elevation and blurred vision as dose-limiting toxicities. (fda.gov)
Last updated: Sep 2026
Goal: Evaluate whether avutometinib plus defactinib controls previously treated metastatic or unresectable diffuse-type gastric cancer, with a primary focus on the proportion of patients alive without progression at 6 months. Safety and other efficacy measures will also be assessed.
Patients: Adults with unresectable or metastatic gastric or gastroesophageal junction carcinoma that is diffuse-type, poorly cohesive, signet ring cell, or mixed type; patients with pathogenic CDH1 or RHOA mutations are eligible regardless of histology. Patients must have received at least one platinum- and fluoropyrimidine-containing regimen for advanced disease, have an ECOG performance status of 0–1, adequate organ function, radiographically evaluable disease, and tumor accessible for a fresh biopsy or malignant ascites sampling.
Design: Single-arm, nonrandomized phase II treatment study; all participants receive the same combination. There is no comparator arm.
Treatments: Avutometinib 3.2 mg orally twice weekly plus defactinib 200 mg orally twice daily, both given for three weeks followed by one week off. Avutometinib inhibits RAF–MEK signaling, while defactinib inhibits focal adhesion kinase (FAK), a potential adaptive survival pathway. In previously treated low-grade serous ovarian cancer, the combination produced a 31% response rate overall in a phase II study, including 44% in KRAS-mutated disease; those results do not establish efficacy in gastric cancer.
Outcomes: The primary endpoint is the 6-month progression-free survival rate. Secondary endpoints include RECIST 1.1 objective response rate, median progression-free survival, overall survival, disease control rate, and duration of response. The listed disease-control definition includes progressive disease, which appears inconsistent with the conventional definition and warrants clarification.
Burden on patient: Moderate anticipated burden. Treatment is oral but requires dosing on multiple days each week in repeating four-week cycles, follow-up visits and imaging for response assessment, and safety monitoring. Eligibility requires fresh tumor sampling or paracentesis of malignant ascites, adding a procedure; cardiac, laboratory, and ECG screening may also be needed. Visit and imaging frequency are not specified, and intensive pharmacokinetic blood sampling is not described.
Last updated: Sep 2026
Inclusion Criteria:
1. Histologic or cytologic evidence of gastric/gastroesophageal junction carcinoma, classified as diffuse type, poorly cohesive, signet ring cell, or mixed type. Patients with known pathogenic CDH1 and/or RHOA mutations will be allowed regardless of histology.
2. Prior therapy with at least one line of therapy for unresectable/metastatic disease, which must include platinum and fluoropyrimidine.
3. ECOG performance status of 0 or 1
4. Age ≥ 18 years
5. Adequate organ function, defined by the following laboratory parameters:
a. Adequate hematologic function, including hemoglobin \[Hb\] ≥ 9.0 g/dL; platelets ≥ 100,000/mm3; and absolute neutrophil count \[ANC\] ≥ 1500/mm3. If a red blood cell transfusion or erythropoiesis-stimulating agent has been administered the Hb must remain stable and ≥ 9 g/dL for at least 1 week prior to first dose of study intervention.
(i) Subjects with Hgb ≥ 8.5 g/dL and \<9.0 g/dL are eligible if there is no history of significant cardiovascular risk features as per the investigator (i.e., prior myocardial infarction) b. Adequate hepatic function: (i) total bilirubin ≤ 1.5 × upper limit of normal \[ULN\] for the institution; patients with Gilbert syndrome may enroll if total bilirubin is \< 3.0 mg/dL (51 μmol/L); (ii) alanine aminotransferase (ALT) and aspartate aminotransferase (AST)
≤ 2.5 × ULN (or \< 5 x ULN in patients with liver metastases). c. Adequate renal function with creatinine clearance rate of ≥ 50 mL/min, as calculated by the Cockcroft-Gault formula d. International normalized ratio (INR) ≤ 1.5 and partial thromboplastin time (PTT) ≤ 1.5 x ULN in the absence of anticoagulation or therapeutic levels in the presence of anticoagulation.
e. Albumin ≥ 3.0 g/dL (451 μmol/L). f. Creatine phosphokinase (CPK) ≤ 2.5 x ULN. g. Adequate cardiac function with left ventricular ejection fraction ≥ 55% by echocardiography (ECHO) or multiple-gated acquisition (MUGA) scan.
6. Disease that can be evaluated radiographically, which can be measurable disease or non-measurable disease per RECIST 1.1. All radiology studies must be performed within 28 days prior to start of study-directed therapy.
7. Tumor that is amenable to fresh biopsy, which may include malignant ascites that is amenable for paracentesis.
8. Baseline QTc interval \< 460 ms for females and ≤ 450 ms for males (average of triplicate readings) using Fredericia's QT correction formula. NOTE: This criterion does not apply to subjects with a right or left bundle branch block.
9. Adequate recovery from toxicities related to prior treatments to at least Grade 1 by CTCAE v 5.0. Exceptions include alopecia and peripheral neuropathy grade ≤ 2. Subjects with other toxicities that are stable on supportive therapy may be allowed to participate with prior approval from the principal investigator.
10. The effects of the study drugs on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (see Section 7.6 for Contraception Guidance) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 3 months after the last dose of the study drug.
11. Ability to understand and the willingness to sign a written informed consent document.
Exclusion Criteria:
1. Systemic anti-cancer therapy within 3 weeks of the first dose of study therapy, or within 5 half-lives of the previous drug, whichever is shorter.
2. Patients currently receiving any other investigational agent.
3. Subjects may not have had a history of malignancy other that esophagogastric cancer within two years prior to screening, with the exception of those with a negligible risk of metastasis or death (e.g., 5-year overall survival \>90%) such as adequately treated carcinoma in situ of the cervix, non-melanoma skin carcinoma, localized prostate cancer, ductal carcinoma in situ, or stage I uterine cancer.
4. Major surgery within 4 weeks or palliative radiotherapy within 1 week of the first dose of study therapy.
5. Treatment with warfarin. Subjects on warfarin for DVT/PE can be converted to low- molecular-weight heparin (LMWH).
6. History of treatment with a direct and specific inhibitor of FAK, MEK, or KRAS.
7. Patients who had exposure to medications (with or without prescriptions), supplements, herbal remedies, or foods with potential for drug-drug interactions with avutometinib and/or defactinib within 14 days prior to the first dose of study intervention and during the course of therapy, including:
1. Strong CYP3A4 inhibitors or inducers.
2. Strong CYP2C9 inhibitors or inducers.
3. Strong P-glycoprotein (P-gp) inhibitors or inducers
4. Strong breast cancer resistance protein (BCRP) inhibitors or inducers
8. Symptomatic brain metastases requiring steroids or other local interventions. Subjects with previously diagnosed brain metastases are eligible if they have completed their treatment and have recovered from the acute effects of radiation therapy or surgery prior to study entry, have discontinued corticosteroid treatment for these metastases for at least 4 weeks prior to first dose of study therapy, and are neurologically stable.
9. Known hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) infection that is active and/or requires therapy. HIV patients on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with the investigational agents.
10. Active skin disorder that has required systemic therapy within the past 1 year.
11. History of rhabdomyolysis.
12. Concurrent ocular disorders including the following:
1. Patients with history of glaucoma, history of retinal vein occlusion (RVO), predisposing factors for RVO, including uncontrolled hypertension, uncontrolled diabetes.
2. Patients with history of retinal pathology or evidence of visible retinal pathology that is considered a risk factor for RVO, intraocular pressure \> 21 mm Hg as measured by tonometry, or other significant ocular pathology, such as anatomical abnormalities that increase the risk for RVO.
3. Patients with active or chronic, visually significant corneal disorders, other active ocular conditions requiring ongoing therapy or clinically significant corneal disease that prevents adequate monitoring of drug-induced keratopathy. Examples of visually significant corneal disorders include corneal degeneration, active or recurrent keratitis, and other forms of serious ocular surface inflammatory conditions. Visually significant corneal disorders do NOT include dry eyes, blepharitis, and uncomplicated corneal erosions.
13. Concurrent congestive heart failure, prior history of class III/IV cardiac disease (New York Heart Association \[NYHA\]), myocardial infarction within the last 6 months, unstable arrhythmias, unstable angina or severe obstructive pulmonary disease.
14. Inability to swallow oral medications.
15. History of hypersensitivity to any of the inactive ingredients (hydroxylpropylmethylcellulose, mannitol, magnesium stearate) of the investigational product.
16. Female subjects who are pregnant or breastfeeding. Pregnant women are excluded from this study because the risk for teratogenic or abortifacient effects with the investigational agents is unknown. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother, breastfeeding should be discontinued if the mother is treated with the investigational agents.
17. Any other medical condition (e.g., cardiac, gastrointestinal, pulmonary, psychiatric, neurological, genetic, etc.) that in the opinion of the investigator would place the subject at unacceptably high risk for toxicity.
New York, New York, 10032, United States
[email protected] / 212-342-5162
Status: Recruiting