Immunome reported that its Phase 3 RINGSIDE trial of the oral gamma‑secretase inhibitor varegacestat met its primary endpoint, showing an 84 % reduction in the risk of disease progression or death versus placebo (hazard ratio 0.16, p < 0.0001). The study also achieved a 56 % objective response rate and a median best change in tumor volume of –83 % for varegacestat compared with +11 % for placebo, with most adverse events being grade 1 or 2 and a manageable safety profile.
The results represent the highest objective response rate observed in a randomized clinical trial for desmoid tumors and exceed the 41 % response rate reported for the only other approved drug in this indication, Ogsiveo. The magnitude of tumor shrinkage and the durability of responses suggest that varegacestat could become a best‑in‑class treatment for this rare, locally aggressive disease, potentially expanding the market for targeted therapies in a patient population with limited options.
Financially, Immunome posted a net loss of $57.5 million for the quarter ended September 30, 2025, with R&D expenses of $49.2 million. The company ended the quarter with $272.6 million in cash and cash equivalents, giving it a runway into 2027 and providing the liquidity needed to support the regulatory and commercialization path for varegacestat.
CEO Clay Siegall said, “RINGSIDE is the largest and most comprehensive clinical trial conducted to date in patients with desmoid tumors, and the topline results represent the highest objective response rate observed in a randomized clinical trial in this patient population.” He added that the company anticipates filing a New Drug Application in Q2 2026 and that the data “demonstrate the potential of varegacestat to offer best‑in‑class results in a convenient, once‑daily, oral medicine that may help patients reclaim their lives.”
The announcement was met with strong investor interest, reflecting confidence in the drug’s potential to fill a significant unmet need and to position Immunome as a leader in the rare‑disease oncology space. The company’s robust cash position and the favorable trial data together support a positive outlook for the upcoming NDA filing and future commercialization.
Immunome also highlighted its broader pipeline, noting progress on IM‑1021, a ROR1‑targeted antibody‑drug conjugate, and IM‑3050, a FAP‑targeted radiotherapy, which are in earlier development stages. These assets underscore the company’s strategy to diversify its portfolio and to build a sustainable growth engine beyond varegacestat.
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