BridgeBio Reports Positive Phase 2 Results; Begins Enrolling Patients in Phase 3 Study

Commercial-stage biopharmaceutical company, BridgeBio (Nasdaq: BBIO), has announced positive results from its PROPEL2 Phase 2 clinical trial demonstrating potential best-in-class efficacy and a clean safety profile for its investigational therapy, infigratinib.

The company is assessing infigratinib as a treatment in children with achondroplasia, a genetic bone growth disorder that results in dwarfism.


  •  80% of children at six months were responders to the treatment.
  • Infigratinib demonstrated clear dose-responsiveness as a single daily oral therapy and was well-tolerated with no adverse events.
  • At the highest dose level, 0.25 mg/kg once daily, the mean increase from baseline in annualized height velocity (AHV) for the 10 children that have had six-month visits was +3.03 cm/yr (p = 0.0022).
  • The baseline AHV for the 10 children with six-month visits was in the expected range for children with achondroplasia at 3.73 cm/yr, rising to 6.77 cm/yr after treatment.
  • As a result of treatment, the median absolute AHV reached 7.6 cm/yr, which is beyond the 99th percentile of growth for children living with achondroplasia.
  • Based on the trial’s positive results, BridgeBio has started enrolling children in the run-in for a Phase 3 trial.

Infigratinib is an oral small molecule designed to inhibit FGFR3, a protein that becomes overactive in certain bone disorders. BridgeBio expects to initiate clinical development for infigratinib in hypochondroplasia, a skeletal dysplasia closely related to achondroplasia and similarly driven by FGFR3.

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BridgeBio Reports Positive Phase 2 Results; Begins Enrolling Patients in Phase 3 Study

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