Immunome submits FDA application for varegacestat in desmoid tumors

Immunome said on April 29, 2026, that it has submitted a New Drug Application to the US FDA for varegacestat, an investigational, once-daily oral gamma secretase inhibitor for adults with desmoid tumors. The filing is backed by results from the phase 3 RINGSIDE trial, which enrolled 156 patients with progressing desmoid tumors and, according to Immunome, met its primary endpoint with an 84% reduction in the risk of disease progression or death versus placebo. The company has also said the study met key secondary endpoints, including a 56% objective response rate versus 9% for placebo, with additional improvements in tumor volume and pain measures. Immunome had previously guided to an NDA filing in the second quarter of 2026, so this submission marks the transition from late-stage development to FDA review. (biospace.com)

Why it matters: For veterinary professionals tracking comparative oncology and the broader soft-tissue tumor pipeline, the filing adds to momentum in a rare-disease space that only recently saw its first FDA-approved systemic therapy, nirogacestat, in November 2023. Varegacestat would enter an emerging gamma secretase inhibitor class, and Immunome is positioning it around once-daily dosing and phase 3 data showing activity on progression-free survival, response rate, tumor volume, and pain. While desmoid tumors are a human indication, the case is relevant to clinicians watching how targeted therapies, symptom endpoints, and quality-of-life measures are shaping regulatory decisions in locally aggressive, non-metastatic tumors. (sec.gov)

What to watch: Next comes FDA’s filing review and potential acceptance decision, typically within about 60 days of submission, while Immunome has also said it expects to present the full RINGSIDE dataset at a major medical meeting. (businesswire.com)