PMV Pharmaceuticals (PMVP) Jefferies Global Healthcare Conference 2025 summary

Company overview and scientific approach

• Founded to harness the power of p53, a key tumor suppressor protein, with a focus on targeting the TP53 Y220C mutation in solid tumors.

• Lead candidate Rezatapopt progressed from phase I to ongoing phase II registrational study, targeting multiple tumor types with a basket design.

• The Y220C mutation is present in about 1% of all solid tumors and 3% in ovarian cancer, guiding the study's cohort structure.

• Rezatapopt stabilizes the mutant p53 protein, restoring its tumor-suppressing function.

Clinical development and study design

• Phase II study includes five cohorts, with ovarian cancer as the anchor indication due to higher mutation frequency and unmet need.

• Interim analysis in mid-2025 will share data from 50 patients (20 with ovarian cancer) with at least 18 weeks of follow-up.

• Study is powered to show an 85% chance of detecting a 30% response rate in ovarian cancer and 95% in the tumor-agnostic cohort.

• Median duration of response (DOR) expectation is six months; phase I showed a seven-month median DOR.

• Enrollment is on track to complete 114 patients by year-end 2025, with NDA submission planned for late 2026.

Key findings and regulatory alignment

• Phase Ib showed a 38% confirmed overall response rate at the recommended dose, with efficacy across six tumor types.

• Safety profile was favorable, with mostly grade 1/2 GI toxicities mitigated by food, which also increased drug exposure by 40%.

• FDA alignment achieved on phase II design and dose, navigating Project Optimus requirements.

• Accelerated approval will likely be sought for ovarian cancer, with post-approval commitments to transition to full approval.