BridgeBio Pharma Announces Publication in the New England Journal of Medicine of Phase 2 PROPEL 2 Study of Infigratinib for Children Living with Achondroplasia

Core Insights - BridgeBio Pharma announced positive results from the PROPEL 2 Phase 2 trial of infigratinib for children with achondroplasia, showing a significant increase in annualized height velocity and an encouraging safety profile [1][2][3] Group 1: Clinical Trial Results - In Cohort 5 of PROPEL 2, a daily oral treatment of infigratinib at 0.25 mg/kg resulted in a mean change in annualized height velocity of +2.50 cm/year at Month 18 (P=0.001) [1][3] - The mean change from baseline in height Z-score was +0.54 (P<0.001) compared to an untreated achondroplasia population at Month 18 [3] - Statistically significant improvement in body proportionality was observed with a mean upper to lower body segment ratio change of -0.12 (P=0.001) at Month 18 [3] Group 2: Safety and Tolerability - Oral infigratinib was well tolerated at Month 18, with no serious adverse events or treatment-emergent adverse events leading to treatment discontinuation [3] - No accelerated progression of bone age or negative changes in bone mineral density were observed, indicating a favorable safety profile [3] Group 3: Regulatory Designations - Infigratinib has received Breakthrough Therapy Designation, Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation from the U.S. FDA [1][4] - These designations highlight the potential of infigratinib to demonstrate substantial improvement in efficacy over existing therapies [2][4] Group 4: Future Developments - PROPEL 3, the global Phase 3 registrational study of infigratinib in achondroplasia, is on schedule for enrollment completion by the end of 2024 [1][5] - The company is committed to exploring the broader medical and functional impacts of infigratinib on skeletal dysplasias [5] Group 5: Company Overview - BridgeBio Pharma focuses on discovering and delivering transformative medicines for genetic diseases, with a pipeline that spans from early science to advanced clinical trials [7] - Founded in 2015, the company aims to apply advances in genetic medicine to benefit patients as quickly as possible [7]