Treatment For Advanced Cancer Patients With p53 Y220C Mutation is Fast-Tracked
A New Jersey-based precision oncology company announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its lead product candidate PC14586, for the treatment of cancer patients with locally advanced or metastatic solid tumors that have a p53 Y220C mutation.
PC14586 is a first-in-class small molecule designed to structurally correct the p53 Y220C mutant protein.
According to PMV Pharmaceuticals, Inc.’s press statement on October 13, 2020, ‘there are currently no FDA-approved medicines that target the p53 Y220C mutation.’
PMV Pharma plans to conduct a Phase 1/2 open-label, multicenter study to assess the safety, tolerability, pharmacokinetics, and anti-tumor activity of PC14586 in adult patients with a p53 Y220C mutation in locally advanced or metastatic solid tumors.
PC14586 is an orally available structural corrector of the p53 Y220C mutant protein, designed to selectively bind to the crevice created by the p53 Y220C mutation, hence, restoring the wild-type, or normal, p53 protein structure and tumor-suppressing function.
p53 plays a pivotal role in cellular function by preserving the integrity of DNA and preventing abnormal cells from entering or progressing through the cell cycle, reports previous studies.
Mutant p53 takes on oncogenic properties that endow cancer cells with a growth advantage and resistance to anti-cancer therapy. Mutant p53 proteins are very common and are found in approximately half of all human cancers. The p53 Y220C mutation is associated with many cancers including but not limited to breast, non-small cell lung cancer, colorectal, pancreatic, and ovarian cancers.
“Fast Track designation reflects the recognition by the FDA that PC14586 has the potential to address a significant unmet medical need for advanced cancer patients with a p53 Y220C mutation,” said David Mack, Ph.D., President and Chief Executive Officer of PMV Pharma, in a press release.
“While targeted therapies have improved outcomes for many patients with advanced cancers, those with tumors carrying a p53 mutation do not yet have precision treatment options for these mutations and often have poor outcomes. We look forward to working closely with the FDA as we advance PC14586 through the clinic as part of our mission to discover and develop novel, tumor-agnostic, precision oncology therapies.”
Phase 1 is a first-in-human, open-label, dose-escalation study designed to up to 30 patients with solid tumors that have a p53 Y220C mutation using next-generation sequencing. Phase 2 is an open-label study designed to assess anti-tumor efficacy and safety in patients with solid tumors that have a p53 Y220C mutation. Phase 2 is expected to enroll up to 100 patients.
Fast Track designation is intended to facilitate the development and expedite the review of products to treat serious conditions while fulfilling an unmet medical need, enabling products to reach patients more rapidly.
PMV Pharma is a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53 mutations.
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