On October 26, Mirum Pharmaceuticals announced preliminary results from Cohort 2 (ages 6 to < 12) of the PROGRESS clinical trial of zilurgisertib.
It's important to remember that this press release is Mirum's first announcement of trial results; it is NOT an announcement of regulatory review results by the U.S. Food & Drug Administration or any other regulatory agency. For children ages 6 to < 12, zilurgisertib is still only available to those enrolled in the trial.
The press release outlines three key points:
- Zilurgisertib reduced total HO lesion volume and improved clinician-assessed joint mobility; no zilurgisertib-treated patients developed new HO lesions.
- Mirum plans to submit a supplemental New Drug Application (sNDA) in the first half (H1) of 2027 to expand the ATEBRIOZ indication to pediatric patients aged 6 to < 12 years.
- Safety and efficacy findings consistent with PROGRESS Cohort 1; no new safety signals.
We are grateful to the 25 pediatric patients ages 6 to < 12 years with FOP from around the world who enrolled in Cohort 2 of the Phase 2 PROGRESS Trial.
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