Immunome, Inc. submits FDA NDA for varegacestat in desmoid tumors, supported by Phase 3 RINGSIDE data showing significant PFS improvement and response rates
Written by: Farha Farheen, Pharm D
Reviewed By: Pharmacally Editorial Team
Immunome, Inc. announced that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration for varegacestat, an investigational oral, once-daily gamma secretase inhibitor, for the treatment of adults with progressing desmoid tumors, supported by findings from its Phase 3 RINGSIDE trial (NCT04871282).
The company stated that the global, randomized, double-blind, placebo-controlled study enrolled 156 patients and met its primary endpoint, with varegacestat demonstrating a statistically significant improvement in progression-free survival and reducing the risk of disease progression or death by 84% compared with placebo (hazard ratio 0.16; 95% CI: 0.071–0.375; p<0.0001).
Immunome, Inc. reported that secondary efficacy outcomes also favored varegacestat, including a confirmed objective response rate of 56% versus 9% with placebo (p<0.0001), along with a median best tumor volume reduction of −83% compared with a +11% increase in the placebo group.
The company added that the study met key secondary endpoints, including statistically significant improvements in tumor volume reduction and worst pain intensity.
Immunome, Inc. stated that varegacestat demonstrated a manageable safety profile consistent with the gamma secretase inhibitor class, with the most commonly reported adverse events being diarrhea (82%), fatigue (44%), rash (43%), nausea (35%), and cough (34%), most of which were grade 1 or 2 in severity.
The company, citing its Chief Executive Officer Clay B. Siegall, described the NDA submission as a key milestone reflecting the strength of the clinical data and development efforts, and indicated that varegacestat may provide a meaningful new oral treatment option for adults with progressing desmoid tumors, while acknowledging the contributions of patients, families, and investigators.
Immunome, Inc. noted that desmoid tumors are rare, non-metastatic but locally aggressive soft tissue tumors associated with high recurrence, chronic pain, and functional impairment, with approximately 1,000 to 1,650 new cases diagnosed annually in the United States.
The company also indicated that detailed data from the RINGSIDE trial have been selected for oral presentation at the American Society of Clinical Oncology Annual Meeting scheduled for May–June 2026.
Reference