varegacestat (AL102) - (RINGSIDE)

July 8, 2026: Immunome, Inc. (NASDAQ: IMNM) announced the FDA accepted its New Drug Application (NDA) for varegacestat (formerly AL102), an investigational oral, once-daily gamma secretase inhibitor, for treatment of adults with desmoid tumors. The FDA set a PDUFA target action date of April 28, 2027.

The NDA is supported by the global, randomized, double-blind, placebo-controlled Phase 3 RINGSIDE trial (NCT04871282) in progressing desmoid tumors (n=156; varegacestat 1.2 mg daily vs placebo). The study met its primary endpoint of progression-free survival, showing an 84% reduction in risk of progression or death vs placebo (HR 0.16; p<0.0001). Key secondary endpoints were also met, including objective response rate (56% vs 9% placebo; p<0.0001) and improved worst pain intensity at week 12, with a difference observed as early as week 4. Exploratory tumor volume analysis showed median best change of -83% vs +11% placebo.

Safety was described as generally well tolerated; common adverse events included diarrhea (82%), fatigue (44%), rash (43%), nausea (35%), and cough (34%); 95% were grade 1–2. Results were presented at the 2026 ASCO Annual Meeting. Immunome plans to submit an EMA marketing authorization application by end of 2026.