BridgeBio announces Phase 2 cohort 5 results of infigratinib

BridgeBio Pharma announced positive results from PROPEL2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, 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. To date, key results from the clinical trial include: At the highest dose level evaluated to date, the mean increase from baseline in annualized height velocity for the 10 children that have had six-month visits was +3.03 cm/yr. 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. The two remaining children who have not yet had six months of follow-up have a mean change from baseline in AHV of +8.8 cm/yr at three months. 80% of the 10 children with six-month visits were responders, with a change from baseline AHV of at least 25%. Preliminary analysis of Collagen X levels also saw a statistically significant increase from baseline in Cohort 5. 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. No serious adverse events or discontinuations due to AEs were reported in any cohort. "I am encouraged by these efficacy and safety results and thankful for our partnership with the physicians, community advocates, children, and families in this study. These results reach a new tier of efficacy, and coupled with our differentiated safety and convenience profile, provide us the opportunity to serve children with achondroplasia and other skeletal dysplasias. We look forward to exploring the potential of infigratinib on the wider medical and functional impacts of achondroplasia, hypochondroplasia and other skeletal dysplasias, which hold significant unmet needs for families," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. Based on the positive results to date, BridgeBio has started enrolling children in the run-in for a Phase 3 trial.