PMV Pharmaceuticals' Rezatapopt Shows Promising Responses in 8 Tumor Types

PMV Pharmaceuticals released interim data from the Phase 2 pivotal portion of the PYNNACLE clinical trial, showing confirmed responses in 8 tumor types, including ovarian, lung, breast, and colorectal cancer. The overall response rate was 33%, with the median time to response at 1.4 months and the median duration of response at 6.2 months. The company plans to submit an NDA for platinum-resistant/refractory ovarian cancer by the end of Q1 2027.

PRINCETON, N.J., Sept. 10, 2025 (GLOBE NEWSWIRE) -- PMV Pharmaceuticals, Inc. (Nasdaq: PMVP), a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53, today announced interim data from the Phase 2 pivotal portion of the PYNNACLE clinical trial. The ongoing Phase 1/2 PYNNACLE clinical trial is evaluating rezatapopt in patients with advanced solid tumors harboring a TP53 Y220C mutation.

The Phase 2 clinical trial data, summarized as of an August 4, 2025 data cutoff date, showed a 33% overall response rate (ORR) across 97 evaluable patients, with confirmed responses in eight different tumor types. Key highlights include a 43% ORR in ovarian cancer patients with a median duration of response of 7.6 months. The trial also demonstrated a 60% response rate in endometrial cancer, albeit in a small sample (n=5). The median duration of response was 6.2 months across heavily pretreated patients.

The safety profile showed mostly Grade 1-2 treatment-related adverse events, with a low 3.7% discontinuation rate. The FDA provided feedback, leading PMV to plan to enroll an additional 20-25 platinum-resistant/refractory ovarian cancer patients and submit a New Drug Application (NDA) in Q1 2027.

Rezatapopt, a first-in-class p53 reactivator, demonstrated significant clinical activity against tumors harboring the specific TP53 Y220C mutation. The 33% overall response rate and 43% response rate in ovarian cancer represent substantial breakthroughs in targeting the historically "undruggable" p53 pathway. The tumor-agnostic efficacy across eight different cancer types underscores the potential for a broad addressable market beyond just ovarian cancer.

The median time to response was 1.4 months, and the median duration of response was 6.2 months, suggesting durable benefits. The safety profile appears manageable, with predominantly Grade 1-2 adverse events and low discontinuation rates.

PMV Pharma will host an investor webinar today at 8:00 AM ET to review the PYNNACLE Phase 2 interim data and provide a regulatory update. The event will feature presentations by PMV Pharma management and Ramez N. Eskander, M.D., Professor of Obstetrics, Gynecology, and Reproductive Sciences at the University of California, San Diego.

The PYNNACLE Phase 2 data positions rezatapopt as a potentially first-in-class p53-targeting therapy with promising clinical validation. The observed efficacy across eight tumor types suggests a substantial addressable market beyond just ovarian cancer. Several metrics stand out from an investment perspective: the 33% overall response rate provides proof-of-concept for PMV's precision oncology approach targeting the specific Y220C mutation of TP53; the 43% response rate in ovarian cancer, including one complete response, represents a compelling potential first approval pathway; the 60% response rate in endometrial cancer, albeit in a small sample (n=5), signals another promising indication; and the median duration of response of 6.2 months across heavily pretreated patients suggests meaningful clinical benefit. The clear regulatory guidance from the FDA provides a defined pathway to market.