BridgeBio Announces Durable Month 12 and 18 Phase 2 Cohort 5 Results of Oral Infigratinib in Achondroplasia, and First Participant Consented in ACCEL for Hypochondroplasia

Core Insights - BridgeBio Pharma announced sustained positive results from the PROPEL 2 Phase 2 trial of infigratinib in children with achondroplasia, indicating potential best-in-class efficacy and a favorable safety profile [1][2] Group 1: Clinical Trial Results - In Cohort 5 of PROPEL 2, infigratinib treatment resulted in a statistically significant increase in annualized height velocity (AHV) of +2.51 cm/year at Month 12 and +2.50 cm/year at Month 18 (p=0.0015) [1][2] - There was a statistically significant improvement in body proportionality, with the mean upper to lower body segment ratio changing from 2.02 at baseline to 1.88 at Month 18 (p=0.001) [1][2] - Infigratinib was well-tolerated, with no treatment-related adverse events reported in Cohort 5 [1][2] Group 2: Ongoing Studies and Future Plans - PROPEL 3, the global Phase 3 registrational study of infigratinib in achondroplasia, is on schedule for enrollment completion by the end of 2024 [1][3] - BridgeBio is expanding the development of infigratinib for hypochondroplasia, with the first child consented in the ACCEL observational study [1][4] - The ACCEL program will include a Phase 2/3 multicenter study to evaluate the efficacy and safety of infigratinib in children with hypochondroplasia [4] Group 3: Company Commitment and Community Impact - BridgeBio is committed to exploring the potential of infigratinib for broader medical and functional impacts on skeletal dysplasias, addressing significant unmet needs for families [7][11] - The company has received positive feedback from the U.S. FDA and EMA regarding the development of infigratinib for hypochondroplasia [4][5] - The Chandler Project, a patient advocacy organization, emphasizes the importance of addressing the needs of the skeletal dysplasia community through collaborative research efforts [6]