The company reported positive results from its PROPEL 3 study of its experimental oral therapy, infigratinib, in children with achondroplasia, a genetic form of dwarfism.
BridgeBio Pharma Inc. (BBIO) on Thursday reported encouraging phase-3 study data for its experimental oral therapy infigratinib, saying the drug improved growth outcomes in children with achondroplasia, a genetic form of dwarfism.
The topline findings stem from PROPEL 3, a global, yearlong Phase 3 trial that enrolled children ages 3 to younger than 18 with open growth plates. Participants were randomly assigned at a 2:1 ratio to receive infigratinib or placebo, with statistically significant improvements across key growth measures through Week 52.
The study met its main goal by demonstrating a meaningful increase in annualized height velocity compared with placebo. Children receiving the therapy grew faster on average, with a treatment benefit of more than 2 centimeters per year compared with placebo.
Following the update, BridgeBio Pharma's stock traded over 17% higher in Thursday’s premarket.
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