California, USA-based biotech BridgeBio Pharma (Nasdaq: BBIO) yesterday announced positive results from PROPEL2, a Phase II trial of the investigational therapy infigratinib in children with achondroplasia, a genetic condition that slows bone growth, demonstrating potential best-in-class efficacy and a clean safety profile.
Infigratinib is an oral small molecule designed to inhibit FGFR3 and target achondroplasia at its source, noted BridgeBio, whose shares shot up 52% to $16.52 by close of trading on Monday. BridgeBio has a commercialization agreement for infigratinib with Swiss firm Helsinn.
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 10 children that have had six-month visits was +3.03 cm/yr (p = 0.0022).
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