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Mirum reports ATEBRIOZ reduced HO lesion volume by 14.0 cm³ vs placebo in pediatric FOP study
MIRMMirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals announced positive Week 24 results from Cohort 2 of the pivotal PROGRESS study for ATEBRIOZ (zilurgisertib) in pediatric patients aged 6 to <12 years with fibrodysplasia ossificans progressiva. Treatment reduced total heterotopic ossification lesion volume by a mean of -8.09 cm³ compared to an increase of 9.14 cm³ for placebo, yielding a treatment difference of -14.0 cm³ (p=0.003). No treated patients developed new lesions, and joint mobility scores improved significantly versus placebo. The company plans to submit a supplemental New Drug Application in the first half of 2027 to expand the indication to this younger age group. Mirum Pharmaceuticals operates as a biopharmaceutical company focused on developing treatments for rare diseases.