Immunome, Inc., a leading biotechnology company dedicated to establishing and introducing excellent, first-in-class, and best-in-class focused cancer therapies, has announced robust, positive topline results from its pivotal Phase 3 RINGSIDE trial of varegacestat, an oral, investigational once-daily gamma secretase inhibitor (GSI), in patients with progressive desmoid tumors.
The trial has achieved its primary endpoint of enhancing progression-free survival, illustrating a clinically significant improvement versus placebo, with 84% mitigation in the challenge of death or disease progression. The qualified objective response rate (ORR) related to RECIST v1.1 was around 56% with varegacestat versus 9% with placebo.
This is examined by the trusted independent central review. Under the discovered analysis, varegacestat illustrated a median commendable change in the tumor percentage. Additionally, the trial has achieved each of the major secondary endpoints. Alongside, the varegacestat met the statistical noteworthy improvement need versus the placebo in familiar tumor volume diminishing and unbearable pain.
As observed, the varegacestat showed an impeccable tolerability with a balanced safety profile well committed to the GSI class. The basic negative events for the candidates during the treatment time were fatigue 44%, nausea 35%, cough 34%, rash 43% and diarrhoea 82%. Probably most of the events were either grade 1 or 2. As per this data, Immunome intends to file a New Drug Application in Q2 2026 to the U.S. Food and Drug Administration.
The CEO of Immunome, PhD, Clay Siegall, said, “RINGSIDE is the vast and most robust clinical trial organised to date in desmoid tumor patients, and the results making headlines have introduced the highest objective response rate seen in a shuffled clinical trial in this patient cohort. These findings illustrate the capability of the varegacestat to deliver remarkable results in a once-daily, oral drug that might give new lives to the patients.”
The M.D., sarcoma medical oncologists and drug development specialist at Memorial Sloan Kettering Cancer Center and RINGSIDE primary investigator, Mrinal M. Gounder, said, “Desmoid tumors can come with a destructive emotional and physical impact on the patient group, have a different, unpredictable nature and the boundaries of recent treatment alternatives. The progression-free survival advantage, mitigation in tumor volume and peak response rate with varegacestat in the RINGSIDE trial are striking.”
“Overall, these findings accelerate the duty of GSIs and qualify varegacestat that might become a standard of care for the desmoid tumor treatment.”
Mansi Kadam is a market research writer with over 3 years of experience analyzing trends in the healthcare industry. At Towards Healthcare, she covers innovations in medical sector, sustainability initiatives, and the evolving regulatory landscape.
