BridgeBio announces publication of PROPEL 2 data in NEJM

BridgeBio (BBIO) announced that 18-month results from PROPEL 2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, were published as an original research article in the New England Journal of Medicine, or NEJM. Infigratinib is an investigational oral small molecule designed to inhibit FGFR3 signaling and target achondroplasia at its source. These data, which were also presented at the 62nd Annual European Society for Paediatric Endocrinology, or ESPE, meeting in Liverpool, demonstrate continued efficacy and a safety profile as an oral treatment option. Key results from the PROPEL 2 dataset were presented at ESPE in a talk titled “Oral infigratinib for children with achondroplasia: Month 18 results from the PROPEL 2 study demonstrate safety and durability of treatment effect on linear growth with improved body proportionality” by Melita Irving. The key data that were shared included: Statistically significant increase in annualized height velocity, or AHV, was observed in children in Cohort 5 who received a daily dose of 0.25 mg/kg/ day of infigratinib, with a mean change from baseline in AHV of +2.50 cm/year at Month 18. Mean change from baseline in height Z-score was +0.54 relative to an untreated achondroplasia population at Month 18. Statistically significant improvement in body proportionality was -0.12 at Month 18. Oral infigratinib was well tolerated at Month 18, with no serious adverse events or treatment-emergent adverse events leading to treatment discontinuation. Additionally, there was no accelerated progression of bone age, negative changes in bone mineral density, or other bone-related adverse events observed.