PMV Pharmaceuticals (NASDAQ:PMVP) reported significant clinical momentum for its lead candidate, rezatapopt, bolstered by a fresh FDA Orphan Drug Designation and clinical results showing high response rates in late-stage ovarian cancer patients.
The Princeton-based precision oncology company announced today that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation on March 2, 2026, for rezatapopt, its first-in-class p53 reactivator.
The designation, aimed at the treatment of TP53 Y220C-mutated ovarian cancer, follows the prestigious publication of Phase 1 data in the New England Journal of Medicine (NEJM) earlier this year.
Clinical updates from the pivotal Phase 2 PYNNACLE trial revealed an overall response rate (ORR) of up to 50% in the ovarian cancer cohort for patients whose tumors were TP53 Y220C mutated and KRAS wild-type.
The data, updated since the last cutoff, reinforces the drug's potential as a tumor-agnostic therapy.
PMV Pharma confirmed it remains on track to submit a New Drug Application (NDA) for rezatapopt in the first quarter of 2027.
On the financial front, PMV Pharmaceuticals ended the 2025 fiscal year with $112.9 million in cash, cash equivalents, and marketable securities.
The company reported a net loss of $77.7 million for the year, with net cash used in operations totaling $73.6 million.
Despite the clinical burn, management expects current capital to provide a functional runway into the end of the second quarter of 2027, covering the planned NDA submission period.