Mirum reports Phase 2 pediatric study shows ATEBRIOZ cuts bone lesion volume and improves mobility
+2 new sources since this summary was written
Mirum Pharmaceuticals announced that in the PROGRESS Cohort 2 trial, children aged 6 to under 12 with fibrodysplasia ossificans progressiva who received zilurgisertib experienced a reduction in heterotopic ossification volume and better joint function compared with placebo. No treated patients developed new bone lesions, while one placebo patient did. The company intends to file a supplemental NDA in the first half of 2027 to broaden the ATEBRIOZ label to this younger age group.
Why it matters
The data support expanding ATEBRIOZ to a younger patient population, which could increase Mirum's market opportunity and drive future revenue. The company’s plan to submit an sNDA in H1 2027 signals a near‑term regulatory milestone that may affect the stock price.
Key facts
- 1The double‑blind portion enrolled 25 children aged 6 to under 12, randomized 1:1 to zilurgisertib 75 mg daily or placebo for 24 weeks. rttnews.com
- 2Patients on zilurgisertib showed an average decrease of 8.09 cm³ in total heterotopic ossification lesion volume, whereas placebo patients showed an average increase of 9.14 cm³. rttnews.com
- 3The treatment difference in lesion volume was 14.0 cm³ favoring zilurgisertib, with a nominal p‑value of 0.003. rttnews.com
- 4Mean CAJIS joint‑mobility score improved by 0.80 points with zilurgisertib versus 0.09 points with placebo. rttnews.com
- 5No patient receiving zilurgisertib developed a new bone lesion; one placebo patient did. rttnews.com
- 6Mirum plans to submit a supplemental New Drug Application in the first half of 2027 to add the 6‑to‑<12‑year indication. stocktitan.net
Summary written by AlphAI AI Desk from 4 of 4 sources. Not investment advice. Figures are as stated by the linked sources.