Pediatric FOP Data Show ATEBRIOZ Halts New Bone Lesions, sNDA Set for H1 2027
MIRM sits 27% above its 52-week low of $63.232.
Summary
Cohort 2 of the pivotal PROGRESS study hit its efficacy measures in children aged 6 to <12 with FOP: total HO lesion volume fell 8.09 cm3 on zilurgisertib versus a 9.14 cm3 increase on placebo (nominal p=0.003), and no treated patient developed new lesions. Joint mobility improved 0.80 CAJIS points versus 0.09 on placebo, against roughly 0.5 points of annual worsening in untreated patients. Safety tracked Cohort 1 with no new signals and no growth plate closure. This extends the label beyond the September 2026 FDA approval, which covered ages 12 and older, and Mirum plans an sNDA in H1 2027 for the younger group; Cohort 3 in ages 2 to <12 is enrolling. The data add a label-expansion path to an already-approved franchise, though the addressable population is small — roughly 300 FOP patients in the U.S. and 900 worldwide. Full Cohort 2 results are slated for an upcoming medical meeting.
At the time of this announcement, MIRM was trading at $80.15 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $5.2B. The 52-week trading range was $63.23 to $130.00. This news item was assessed with positive market sentiment and an importance score of 7 out of 10. Source: BusinessWire.