PDUFA target action date set for April 28, 2027
Varegacestat could provide a meaningful new oral treatment option for adults with desmoid tumors if approved
BOTHELL, Wash., July 08, 2026--(BUSINESS WIRE)--Immunome, Inc. (Nasdaq: IMNM), a biotechnology company committed to developing first-in-class and best-in-class targeted cancer therapies, today announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted its New Drug Application (NDA) for varegacestat, an investigational, oral, once-daily gamma secretase inhibitor (GSI), for the treatment of adults with desmoid tumors. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 28, 2027.
"The FDA's acceptance of our NDA for varegacestat is an important milestone for Immunome and for patients living with desmoid tumors," said Clay Siegall, Ph.D., President and Chief Executive Officer of Immunome. "We believe varegacestat has the potential to provide an important oral treatment option, supported by robust clinical data across all key efficacy endpoints. We look forward to working closely with the FDA throughout the review."
RINGSIDE Phase 3 Trial Results
The NDA is based on results from the Phase 3 RINGSIDE trial evaluating varegacestat in patients with progressing desmoid tumors. Key findings include:
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 (hazard ratio = 0.16, p<0.0001).
The trial also met all key secondary endpoints, including achieving an objective response rate of 56% vs. 9% with placebo (p<0.0001), as assessed by blinded independent central review.
Varegacestat demonstrated statistically significant improvement in worst pain intensity at week 12, with a clinically significant difference observed as early as the first evaluation at week 4.
In an exploratory analysis, varegacestat showed a median best change in tumor volume of -83% vs. +11% with placebo, as assessed by blinded independent central review.
Varegacestat was generally well tolerated with a manageable safety profile, consistent with the gamma secretase inhibitor class. The most common adverse events for participants in the treatment arm were diarrhea (82%), fatigue (44%), rash (43%), nausea (35%) and cough (34%). Most (95%) events were grade 1 or 2.
The results were presented in an oral abstract session at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting.