Immunome’s Varegacestat Delivers Landmark Efficacy in Phase 3 RINGSIDE Trial for Desmoid Tumors

RINGSIDE Study Reveals 84% Reduction in Progression Risk with Varegacestat

Immunome’s latest topline data from its global Phase 3 RINGSIDE trial has put varegacestat in the spotlight, with the oral therapy showing an 84% reduction in the risk of disease progression or death in patients with desmoid tumors compared to placebo. The trial, the largest and most comprehensive to date for this patient population, not only met its primary endpoint for progression-free survival but also outperformed in every key secondary measure, positioning varegacestat as a potential new standard of care.

Highest Response Rate Recorded: Varegacestat Achieves 56% ORR

The objective response rate (ORR)—the percentage of patients with meaningful tumor reduction—reached 56% for varegacestat, vastly outstripping the 9% rate observed in the placebo arm. On top of this, patients on varegacestat experienced a median best reduction in tumor volume of 83%, compared to an 11% increase with placebo, suggesting the drug is highly effective at shrinking these aggressive tumors.

Manageable Safety Profile Enhances Drug’s Clinical Promise

Despite the remarkable efficacy, varegacestat demonstrated a safety profile that aligns with other drugs in the gamma secretase inhibitor class. The most frequent side effects included diarrhea (82%), fatigue (44%), rash (43%), nausea (35%), and cough (34%). Importantly, most adverse events were grade 1 or 2, suggesting that side effects, while common, were generally mild to moderate and manageable for patients.

Major Regulatory Milestone on Horizon: NDA Filing Planned for 2026

Based on the strength of these results, Immunome is moving forward with plans to submit a New Drug Application (NDA) for varegacestat to the U.S. FDA in the second quarter of 2026. This timeline could see varegacestat becoming a key treatment option for a patient population currently underserved by existing therapies.

What This Means for Patients with Desmoid Tumors

Desmoid tumors, while not metastatic, often result in recurring, debilitating growths that impact physical function and quality of life. With only 1,000–1,650 new cases annually in the U.S. and around 10,000–11,000 patients being actively managed, effective treatments are critical. The improvements seen in progression-free survival, tumor volume reduction, and pain intensity underscore the potential impact varegacestat could have if approved.

Key Takeaways: Transformative Potential with Measurable Outcomes

The RINGSIDE trial results mark a pivotal achievement for Immunome and offer new hope for patients with desmoid tumors. Investors and stakeholders should monitor the company’s planned presentations and NDA progress through 2026. As the therapy’s clinical and regulatory trajectory advances, varegacestat’s strong data profile sets it apart in the targeted oncology field—potentially changing the standard of care for a challenging disease.

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