Sponsor: Memorial Sloan Kettering Cancer Center (other)
Phase: 2
Start date: April 29, 2024
Planned enrollment: 20
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: Assess the efficacy and safety of upfront avutometinib, defactinib, and letrozole for low-grade serous ovarian cancer (LGSOC).
Patients: Adults with histologically confirmed ovarian or peritoneal LGSOC, RECIST-measurable disease, and no prior systemic treatment for LGSOC or serous borderline disease. Eligible patients are not candidates for primary debulking or have measurable residual disease after attempted debulking. Most must have an ECOG performance status of 0–1; patients with a status of 2 may qualify if the impairment is cancer-related.
Design: Single-arm, nonrandomized phase 2 treatment study in the neoadjuvant setting or after suboptimal debulking. There is no control group.
Treatments: Avutometinib 3.2 mg orally twice weekly and defactinib 200 mg orally twice daily are given for three weeks followed by one week off in each 28-day cycle. Letrozole 2.5 mg is taken orally daily; premenopausal or perimenopausal patients with an ovary in situ also receive leuprolide for ovarian suppression. Avutometinib inhibits RAF–MEK signaling by stabilizing an inactive RAF–MEK complex, while defactinib inhibits FAK signaling that may enable adaptive resistance. In a prior phase 2 study of the two-drug combination in previously treated recurrent LGSOC, the objective response rate was 31% overall and 44% in KRAS-mutated disease. That evidence does not establish efficacy for this three-drug regimen in the upfront setting; letrozole adds endocrine therapy.
Outcomes: The primary outcome is RECIST v1.1 objective response rate, defined as complete or partial response, assessed over two years. The secondary outcome is adverse-event occurrence, graded using CTCAE v5.0 over two years.
Burden on patient: Moderate. Treatment involves multiple oral dosing schedules and, for some patients, recurring leuprolide injections. Response imaging, safety laboratory testing, and monitoring for cardiac and ocular risks are likely to require clinic visits. The protocol information does not specify visit or scan frequency, and no serial research biopsies or intensive pharmacokinetic blood draws are described.
Last updated: Sep 2026
Inclusion Criteria:
* Female patients ≥ 18 years of age
* Histologically-confirmed LGSOC (ovarian, peritoneal) by tissue biopsy read by pathology at study institution. NOTE: Patients with a prior history of serous borderline tumors without prior systemic (cytotoxic or hormonal) treatment but a new diagnosis of low-grade serous ovarian cancer are eligible.
* Determination that the patient is not a primary surgical candidate by a gynecologic oncologist surgeon; or has undergone an attempted primary debulking with residual RECIST measurable disease.
* Measurable disease according to RECIST 1.1. Measurable disease is defined as at least one lesion that can be accurately measured in at least one dimension. Each lesion must be ≥10mm when measured by CT or MRI. Lymph nodes must be ≥15mm by short axis when measured by CT or MRI.
* An Eastern Cooperative Group (ECOG) performance status of ≤1. Patients with an ECOG performance status of 2 are permitted on trial if this is deemed to be secondary to cancer but not to other comorbidities.
* Patients with treated brain metastases are eligible if follow-up brain imaging after CNS-directed therapy shows no evidence of progression. Patients with asymptomatic brain metastases that do not require intervention are also eligible.
* HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial.
* For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated.
* Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on anti-HCV treatment, they are eligible if they have an undetectable HCV viral load.
* Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial.
* Female patients with reproductive potential agree to use highly effective method of contraceptive during the trial and for 1 month following the last dose of study intervention, unless surgical menopause is conferred. Women of child-bearing potential must have a negative pregnancy test within 14 days prior to commencement of study treatment. Non-hormonal methods of highly effective contraception include:
* intrauterine device (IUD)
* bilateral tubal occlusion
* vasectomized partner
* sexual abstinence
* Patients must have adequate cardiac function with left ventricular ejection fraction ≥ 50% by echocardiography (ECHO) or multiple-gated acquisition (MUGA) scan.
* Baseline QTc interval \< 460 ms (Common Terminology Criteria for Adverse Events \[CTCAE\] Grade 1) using Fredericia's QT correction formula. NOTE: This criterion does not apply to patients with a right or left bundle branch block.
* Adequate organ function, defined by the following parameters:
°Adequate hematologic function
* Hemoglobin \[Hb\] ≥ 9.0 g/dL. If a red blood cell transfusion has been administered the Hb must remain stable and ≥ 9.0 g/dL for at least 1 week prior to first dose of study intervention.
* Platelets ≥ 100,000/mm\^3
* Absolute neutrophil count \[ANC\] ≥ 1000/mm\^3
°Adequate hepatic function:
* Total bilirubin ≤1.5 × upper limit of normal \[ULN\] for the institution; patients with Gilbert syndrome may enroll if total bilirubin is \<3.0mg/dL (51 μmole/L) upon discussion with MSK PI.
* Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 × ULN (or \<5 xULN in patients with liver metastases).
* Adequate renal function with creatinine clearance rate of ≥50 mL/min as calculated by the Cockcroft-Gault formula or serum creatinine of ≤ 1.5 x ULN.
* Creatine phosphokinase (CPK) ≤2.5 x ULN.
Exclusion Criteria:
* Patients who are deemed in the opinion of their treating physician to be appropriate candidates for a primary debulking surgery are not eligible for this trial, unless measurable disease remains after a primary cytoreductive surgery.
* Prior systemic anti-cancer therapy for LGSOC or serous borderline disease.
* Major surgery within 4 weeks, minor surgery within 2 weeks, or palliative radiotherapy within 1 week of the first dose of study intervention. Open and close laparotomy and/or laparoscopy will be considered minor surgery.
* Treatment with warfarin. Patients on warfarin for deep vein thrombosis/pulmonary embolism can be converted to low-molecular-weight heparin or direct oral anticoagulants (DOACs).
* Patients with the inability to swallow oral medications or impaired gastrointestinal absorption due to gastrectomy or drainage PEG tube. Patients with diagnosis of bowel obstruction \<3 months from study enrollment will be excluded unless surgically cured or approved by study PI.
* Symptomatic brain metastases requiring steroids or other interventions. Patients with new asymptomatic CNS metastases detected during the screening period must receive radiation therapy and/or surgery for CNS metastases. Following treatment, these patients may then be eligible if all other criteria are met.
* 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.
* Patients with a history of corneal erosion (instability of corneal epithelium), corneal degeneration, active or recurrent keratitis, and other forms of serious ocular surface inflammatory conditions.
* History of rhabdomyolysis.
* Patients with a history of hypersensitivity to any of the active (Avutometinib, defactinib) or inactive (hydroxypropylmethylcellulose, mannitol, magnesium stearate) ingredients of the investigational product.
* Female patients who are breastfeeding.
* Any other medical condition (e.g., cardiac, gastrointestinal, pulmonary, psychiatric, neurological, genetic, etc.) that in the opinion of the Investigator would places the patient at unacceptably high risk for toxicity.
* Exposure to medications (with or without prescriptions), supplements, herbal remedies, or foods with potential for drug-drug interactions with study interventions within 14 days prior to the first dose of study intervention and during the course of therapy (Appendix 1), including:
* strong CYP3A4 inhibitors or inducers, due to potential drug-drug interactions with both Avutometinib and defactinib.
* strong CYP2C9 inhibitors or inducers, due to potential drug-drug interactions with defactinib.
* P-glycoprotein (P-gp) inhibitors or inducers, due to potential drug-drug interactions with defactinib.
Atlanta, Georgia, 30322, United States
No email / 404-778-3401
Status: Recruiting
Basking Ridge, New Jersey, 07920, United States
No email / 646-888-4653
Status: Recruiting
Montvale, New Jersey, 07645, United States
No email / 646-888-4653
Status: Recruiting
Middletown, New Jersey, 07748, United States
No email / 646-888-4653
Status: Recruiting
New York, New York, 10065, United States
No email / 646-888-4653
Status: Recruiting
Uniondale, New York, 11553, United States
No email / 646-888-4653
Status: Recruiting
Commack, New York, 11725, United States
No email / 646-888-4653
Status: Recruiting
Harrison, New York, 10604, United States
No email / 646-888-4653
Status: Recruiting