Immunome Submits NDA for Varegacestat to FDA

Immunome announced the submission of a New Drug Application to the U.S. Food and Drug Administration for varegacestat, an investigational, oral, once-daily gamma secretase inhibitor, for the treatment of adults with desmoid tumors. The NDA is supported by results from the global, randomized, double-blind, placebo-controlled Phase 3 RINGSIDE trial of varegacestat in patients with progressing desmoid tumors. The trial met its primary endpoint of improving progression-free survival, demonstrating a statistically significant and clinically meaningful improvement vs. placebo, with an 84% reduction in the risk of disease progression or death. The confirmed objective response rate based on RECIST v1.1 was 56% with varegacestat vs. 9% with placebo, as assessed by blinded independent central review. In an exploratory analysis, varegacestat demonstrated a median best change in tumor volume of -83% vs. +11% with placebo, as assessed by blinded independent central review. In addition, the trial met all key secondary endpoints, with varegacestat achieving statistically significant improvements vs. placebo in landmark tumor volume reduction and worst pain intensity. Varegacestat was generally well tolerated, with a manageable safety profile consistent with the GSI class. The most common adverse events for participants in the treatment arm were diarrhea, fatigue, rash, nausea and cough. Most events were grade 1 or 2.