Varegacestat (γ-Secretase Inhibitor) Shrinks Desmoid Tumors in 46–78% of Patients Across All Subgroups

In the RINGSIDE Phase 2 open label trial (NCT04871282), 42 adults with progressing desmoid tumors received the oral γ-Secretase Inhibitor, varegacestat 1.2 mg QD, 2 mg or 4 mg. (2 days on/5 off); median follow-up 23 months. Pooled objective response rate was 43–78% regardless of age (≤40 y 59% vs >40 y 71%), tumor size (< 70 mm 72% vs ≥ 70 mm 64%), prior therapy (0/1/≥2 lines 55–78%), site (intra-abdominal 44% vs extra-abdominal 70%), or mutation (APC 43% vs CTNNB1 68%). At cutoff, 55% remained on treatment; no new safety signals reported.

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