- Varegacestat treatment resulted in statistically significant improvements in primary and all key secondary efficacy endpoints
- 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
- Progression-free survival benefit was consistent across prespecified patient subgroups, including tumor location, baseline tumor size, patient age and prior systemic desmoid tumor therapy
- New Drug Application (NDA) for varegacestat submitted to the U.S. Food and Drug Administration (FDA) in April 2026, with Marketing Authorization Application (MAA) submission to the European Medicines Agency (EMA) planned by the end of 2026
BOTHELL, Wash.--(BUSINESS WIRE)-- Immunome, Inc. (Nasdaq: IMNM), a biotechnology company committed to developing first-in-class and best-in-class targeted cancer therapies, today announced detailed efficacy and safety results from RINGSIDE, the global, randomized, double-blind, placebo-controlled Phase 3 trial of varegacestat in patients with progressing desmoid tumors. The data are being presented today in an oral abstract session at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago. Immunome submitted an NDA for varegacestat to the FDA in April 2026.
“Desmoid tumors can be locally aggressive, painful and unpredictable, creating a high disease burden for patients and a continued need for new treatment options,” said Mrinal M. Gounder, M.D., sarcoma medical oncologist and drug development specialist at Memorial Sloan Kettering Cancer Center, and the RINGSIDE primary investigator who is presenting the data. “The RINGSIDE data show a compelling progression-free survival benefit with varegacestat that is consistent across relevant patient subgroups, complemented by a high response rate and reduction in tumor volume. The data also show a rapid, clinically meaningful reduction in worst pain intensity, which is an important element for patients. These findings confirm varegacestat could become standard of care in the treatment of desmoid tumors.”
“The detailed RINGSIDE data presented at ASCO reinforce varegacestat’s differentiated efficacy and manageable safety profile,” said Clay Siegall, Ph.D., President and Chief Executive Officer of Immunome. “The depth and consistency of benefit observed across RINGSIDE point to varegacestat’s potential to deliver a meaningful advance for patients with desmoid tumors. These results form the basis for the NDA we submitted in April 2026 and the planned MAA submission for Europe.”
RINGSIDE Key Clinical Data Presented at 2026 ASCO Meeting
As previously reported, the RINGSIDE trial met its primary endpoint and all key secondary endpoints. Varegacestat demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) vs. placebo, with an 84% reduction in the risk of disease progression or death (hazard ratio [HR] = 0.16, 95% confidence interval [CI]: 0.071, 0.375; p