BridgeBio's Dwarfism Candidate Shows Favorable Height Increase, Safety Profile

BridgeBio Pharma Inc BBIO announced results from PROPEL2 Phase 2 trial of infigratinib in children with achondroplasia, a genetic condition affecting a protein in the body called the fibroblast growth factor receptor.
In achondroplasia, this protein begins to function abnormally, slowing down the growth of bone in the cartilage of the growth plate.
Infigratinib is an oral small molecule designed to inhibit FGFR3 and target achondroplasia at its source.
At the highest dose level evaluated to date (Cohort 5, 0.25 mg/kg once daily), the mean increase from baseline in annualized height velocity (AHV) for the ten children with six-month visits was +3.03 cm/yr.
80% of the ten children with six-month visits were responders, with a change from baseline AHV of at least 25%. Among the responders, the average change from baseline in AHV was +3.81 cm/yr.
Related: BridgeBio Pharma Shares Preliminary Data From Gene Therapy Candidate In Ultra Rare Disorder.
Combined with the previously reported Cohort 4 change from baseline in AHV value of +1.52 cm/yr, the Cohort 5 data demonstrate a strong dose-response for infigratinib
Median follow-up across all cohorts is 71.1 weeks. The study has shown a well-tolerated safety profile, with no drug-related treatment-emergent adverse events in Cohort 5.
No serious adverse events or discontinuations due to AEs were reported in any cohort.
BridgeBio has started enrolling children in the run-in for a Phase 3 trial.
Additionally, BridgeBio expects to initiate development for infigratinib in hypochondroplasia, a skeletal dysplasia closely related to achondroplasia and similarly driven by FGFR3 gain-of-function variants.
Infigratinib has IP protection out to at least 2041.
Price Action: BBIO shares 60.30% at $17.50 on the last check Monday.

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