Results from the phase 3 RINGSIDE trial showed that varegacestat (AL102) significantly improved outcomes for patients with progressing desmoid tumors. The findings have supported a New Drug Application submitted to the FDA in April 2026. The global study enrolled 156 adults with desmoid tumors that had progressed within the previous year and randomly assigned them to receive either oral varegacestat or placebo.
The trial met its primary endpoint, with varegacestat reducing the risk of disease progression or death by 84% compared with placebo. Median progression-free survival was not reached in the varegacestat arm, while it was 24.87 months with placebo. The objective response rate was 55.7% versus 9.1%, and patients experienced a median tumor volume reduction of 83%. Significant pain relief was also reported as early as four weeks after treatment began.
Common side effects included diarrhea, fatigue, rash, and nausea. No treatment-related deaths were reported, and ovarian toxicity observed in some premenopausal women was often reversible.