Mirum and Incyte Announce Phase 2 Results for Zilurgisertib
Mirum Pharmaceuticals (MIRM) and Incyte (INCY) announced Phase 2 results from Cohort 1 of the Progress study evaluating zilurgisertib, an investigational oral activin receptor-like kinase 2 inhibitor, in adolescents and adults with fibrodysplasia ossificans progressiva. Results were shared in a late-breaking rapid-fire presentation at ENDO 2026, the Endocrine Society's annual meeting. Key efficacy findings included: Fewer patients receiving zilurgisertib developed new HO lesions at Week 24, with an 81% reduction versus placebo. 99.9% reduction in total volume of new HO lesions in patients receiving zilurgisertib versus placebo at Week 24. Reduction in total existing HO lesion volume compared with an increase observed in placebo-treated patients at Week 24. Among patients receiving zilurgisertib, no new HO lesions were observed and total HO lesion volume continued to decrease from Week 24 to Week 48. Among patients who crossed over from placebo to zilurgisertib, no new HO lesions were observed and total HO lesion volume decreased from Week 24 to Week 48.
