BridgeBio Pharma Touts Encouraging Data From Dwarfism Candidate

  • BridgeBio Pharma Inc BBIO has announced interim results from PROPEL 2 Phase 2 trial of infigratinib in children with achondroplasia, the most common form of short-limbed dwarfism.
  • At the highest dose level (0.128 mg/kg once daily), the mean increase in annualized height velocity (AHV) was 1.52 cm/yr over baseline in children five years and older. 
  • Earlier cohorts in PROPEL 2 did not achieve the target efficacious exposure as suggested by preclinical data, and no dose-response was observed. 
  • An increase in AHV over a baseline of 0.22 cm/yr was observed across these earlier combined cohorts.
  • Related: BridgeBio Pharma Posts Promising Data From Gene Therapy Trial In Ultra-Rare Disease.
  • A single case of mild hyperphosphatemia (abnormally high serum phosphate levels) led to a dose reduction in a participant in Cohort 3 (dose: 0.064 mg/kg once daily).
  • No children have discontinued treatment as the result of an adverse event. No bone-related AEs were observed to date.
  • BridgeBio has begun enrolling Cohort 5 participants who will receive approximately twice the dose of Cohort 4 (0.25 mg/kg once vs. 0.128 mg/kg once daily). 
  • Price Action: BBIO shares are up 6.53% at $11.75 during the premarket session on the last check Tuesday.
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