Mirum Pharmaceuticals Announces Positive Results from Cohort 2 of the Pivotal PROGRESS Study of ATEBRIOZ™ (zilurgisertib) in Pediatric Patients Aged 6 to <12 Years with Fibrodysplasia Ossificans Progressiva
Key Highlights
- ➤HO lesion volume fell 8.09 cm³ with zilurgisertib versus rose 9.14 cm³ with placebo
- ➤Treatment difference favored zilurgisertib by 14.0 cm³; nominal p=0.003
- ➤No zilurgisertib-treated patients developed new HO lesions versus 1 placebo patient
- ➤Mirum plans H1 2027 sNDA seeking ATEBRIOZ expansion to ages 6 to <12
- ➤Cohort 2 safety was consistent with Cohort 1, with no new safety signals
Expert Statements
Rob Myers, Chief Medical Officer of Mirum Pharmaceuticals
“These results represent an important advance for younger children living with FOP”
Rob Myers, Chief Medical Officer of Mirum Pharmaceuticals
“The reduction in total HO volume and absence of new lesions seen with zilurgisertib build on the positive results previously observed in adolescents and adults in Cohort 1 of PROGRESS.”
Rob Myers, Chief Medical Officer of Mirum Pharmaceuticals
“We look forward to discussing these findings with the FDA to expand access to ATEBRIOZ for younger children living with this debilitating disease.”
Frederick S. Kaplan, M.D., Isaac and Rose Nassau Professor of Orthopaedic Molecular Medicine at the University of Pennsylvania Perelman School of Medicine
“FOP is a devastating genetic disease that leads to progressive loss of mobility, independence, quality of life, and shortened lifespan”
Frederick S. Kaplan, M.D., Isaac and Rose Nassau Professor of Orthopaedic Molecular Medicine at the University of Pennsylvania Perelman School of Medicine
“Childhood is a critical window in FOP, so a therapy targeting the underlying cause of the disease is especially important for this young age group.”
Frederick S. Kaplan, M.D., Isaac and Rose Nassau Professor of Orthopaedic Molecular Medicine at the University of Pennsylvania Perelman School of Medicine
“Results such as these offer hope that we may be able to slow disease progression in children before its most serious consequences arise.”
Mirum Pharmaceuticals Announces Positive Results from Cohort 2 of the Pivotal PROGRESS Study of ATEBRIOZ™ (zilurgisertib) in Pediatric Patients Aged 6 to <12 Years with Fibrodysplasia Ossificans Progressiva
- Zilurgisertib reduced total HO lesion volume and improved clinician-assessed joint mobility; no zilurgisertib-treated patients developed new HO lesions
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