Immunome Submits NDA for Varegacestat, FDA Target Date April 28, 2027

Immunome announced the FDA has accepted its New Drug Application, or NDA, for varegacestat, an investigational, oral, once-daily gamma secretase inhibitor, or GSI, for the treatment of adults with desmoid tumors. The FDA assigned a Prescription Drug User Fee Act target action date of April 28, 2027. Desmoid tumors - also known as aggressive fibromatosis or desmoid-type fibromatosis - are aggressive non-metastatic soft tissue tumors that are prone to recurrence. The NDA is based on results from the Phase 3 RINGSIDE trial evaluating varegacestat in patients with progressing desmoid tumors. The registrational trial met its primary endpoint of improving progression-free survival vs. placebo, with a statistically significant and clinically meaningful 84% reduction in the risk of disease progression or death. The trial also met all key secondary endpoints, including achieving an objective response rate of 56% vs. 9% with placebo. Varegacestat demonstrated statistically significant improvement in worst pain intensity at week 12. In an exploratory analysis, varegacestat showed a median best change in tumor volume of -83% vs. +11% with placebo. Varegacestat was generally well tolerated with a manageable safety profile.