Key insights
- Immunome Inc. announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application for varegacestat, an oral treatment for desmoid tumors. This validation initiates the EMA's review process. The application is supported by positive Phase 3 RINGSIDE trial data, which showed significant improvements in progression-free survival, objective response rate, and pain reduction compared to placebo. The U.S. FDA has also accepted Immunome's New Drug Application for varegacestat.
BOTHELL, Wash. - Immunome Inc. (NASDAQ:IMNM) announced today that the European Medicines Agency has validated its Marketing Authorization Application for varegacestat, an oral gamma secretase inhibitor for treating adults with desmoid tumors.
The validation confirms the application is complete and the EMA will begin its review under the centralized marketing authorization procedure, according to a press release statement.
The application is supported by results from the Phase 3 RINGSIDE trial, a randomized, double-blind, placebo-controlled study involving 156 patients with progressing desmoid tumors. Data presented at the 2026 American Society of Clinical Oncology Annual Meeting showed the trial met its primary endpoint of progression-free survival with an 84% reduction in the risk of disease progression or death compared to placebo.
The trial also met all key secondary endpoints. The objective response rate was 56% versus 9% with placebo. Varegacestat showed statistically significant improvement in worst pain intensity at week 12, with differences observed at week 4. An exploratory analysis showed a median best change in tumor volume of -83% versus +11% with placebo.
The most common adverse events in the treatment arm were diarrhea (82%), fatigue (44%), rash (43%), nausea (35%) and cough (34%). Most events (95%) were grade 1 or 2.
The U.S. Food and Drug Administration accepted Immunome’s New Drug Application for varegacestat in July, with a target action date of April 28, 2027.
Desmoid tumors are aggressive non-metastatic soft tissue tumors prone to recurrence. Approximately 1,000 to 1,650 people are diagnosed with desmoid tumors annually in the United States, with about 10,000 to 11,000 actively managed patients.
Varegacestat is administered orally once daily. The RINGSIDE trial includes an ongoing open-label extension phase.
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