Mirum Reports Positive PROGRESS Cohort 2 Topline for zilurgisertib in Pediatric FOP

PROGRESS Cohort 2 met its efficacy endpoints at Week 24, showing zilurgisertib reduced total HO lesion volume by 8.09 cm³ (vs +9.14 cm³ placebo), prevented new HO lesions in all treated patients, and improved CAJIS joint-mobility scores. Safety was consistent with Cohort 1 with no new signals. These data will support an sNDA filing in H1 2027 to expand the existing approval (ages ≥12) to children aged 6 to <12 years.

Thông tin chính

Nộp lúc: 20:47 26 thg 10, 2026
Sự kiện: Kết quả topline
Chiều hướng: Tích cực
Công ty: MIRMMirum
Tài sản: zilurgisertib
Nguồn: SEC 8-K
Độ tin cậy: Có nguồn
Thay đổi CAJIS: -0,80 so với -0,09 điểm
tổn thương HO mới: 0% so với 7,7%
Thay đổi thể tích tổn thương HO: -8,09 so với +9,14 cm³ (p=0,003)

Trích đoạn nguồn

Mirum Pharmaceuticals Announces Positive Results from Cohort 2 of the Pivotal PROGRESS Study of ATEBRIOZ(TM) (zilurgisertib) in Pediatric Patients Aged 6 to <12 Years with Fibrodysplasia Ossificans Progressiva Mirum Pharmaceuticals Announces Positive Results from Cohort 2 of the Pivotal PROGRESS Study of ATEBRIOZ(TM) (zilurgisertib) in Pediatric Patients Aged 6 to Mirum Pharmaceuticals Announces Positive Results from Cohort 2 of the Pivotal PROGRESS Study of ATEBRIOZ™ (zilurgisertib) in Pediatric Patients Aged 6 to - Zilurgisertib reduced total HO lesion volume and improved clinician-assessed joint mobility; no zilurgisertib-treated patients developed new HO lesions - Mirum plans to submit an sNDA in H1 2027 to expand th e ATEBRIOZ indication to pediatric patients aged 6 to <12 year

SEC 8-K