Mirum reports positive Cohort 2 results for ATEBRIOZ in pediatric FOP patients aged 6 to <12
Mirum Pharmaceuticals said its 25-patient Cohort 2 trial of ATEBRIOZ (zilurgisertib) in children aged 6 to under 12 with fibrodysplasia ossificans progressiva showed reduced heterotopic ossification lesion volume and improved joint mobility at Week 24 compared with placebo. The company plans to submit a supplemental New Drug Application in the first half of 2027 to expand the approved indication to this age group.
Key points
- Mean total HO lesion volume fell 8.09 cm³ with ATEBRIOZ versus a 9.14 cm³ increase with placebo (nominal p=0.003).
- No ATEBRIOZ-treated patients developed new HO lesions, compared with 1 patient (7.7%) on placebo.
- Mean CAJIS joint mobility score improved 0.80 points with ATEBRIOZ versus 0.09 points with placebo at Week 24.
- The company plans a supplemental NDA submission in the first half of 2027; full Cohort 2 results will be presented at an upcoming medical meeting.
The filing
Written by AI on Nov 9, 2026, 11:43 AM ET, from this filing only. Written later: this filing was briefed after the fact, when past filings were added to the site, so the brief may be read with hindsight; the filing itself is the record of what was known on October 26, 2026.
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