BridgeBio Reports Sustained Phase 2 Results of Infigratinib for Achondroplasia and ACCEL Consent for Hypochondroplasia

BridgeBio Pharma, Inc., a biopharmaceutical company specializing in genetic disorders, recently reported encouraging results from its Phase 2 clinical trial, PROPEL 2, focused on the investigational drug infigratinib for children with achondroplasia. The trial demonstrated promising efficacy and a strong safety profile, solidifying infigratinib's potential as a leading treatment option.

Achondroplasia is characterized by disproportionate short stature and numerous health complications. It is caused by a mutation in the FGFR3 gene. Infigratinib works as an oral small molecule inhibitor targeting FGFR3 signaling, aiming to address the root cause of achondroplasia and a related condition, hypochondroplasia.

Key findings from Cohort 5 of the PROPEL 2 trial, which involved a dose of 0.25 mg/kg/day, were particularly notable:

1. Annualized Height Velocity (AHV): There was a significant and sustained increase in AHV, with a mean rise from baseline of +2.51 cm/year at Month 12 and +2.50 cm/year at Month 18 (p=0.0015).
2. Body Proportionality: At Month 18, there was a marked improvement in body proportionality (p=0.001), with the upper to lower body segment ratio improving from 2.02 at baseline to 1.88.
3. Safety: Infigratinib was well-tolerated with no treatment-related adverse events reported in any participant in Cohort 5.

Dr. Ravi Savarirayan, the global lead investigator for PROPEL 2, highlighted the significance of these findings, noting the improvement in growth velocity and body proportionality. These results suggest infigratinib not only boosts growth but may also enhance functionality for individuals with achondroplasia. The ongoing PROPEL 3 study, a global Phase 3 trial, aims to confirm these benefits, with completion expected by the end of 2024.

BridgeBio is also advancing the development of infigratinib for hypochondroplasia, another FGFR3-related skeletal dysplasia. This condition shares many characteristics with achondroplasia, including short stature and potential complications. The ACCEL study, an observational run-in phase for hypochondroplasia, has begun with the first participant enrolled in May 2024. The subsequent ACCEL 2/3 will be a global Phase 2/3 study to assess the efficacy and safety of 0.25 mg/kg/day of infigratinib in children with this condition.

Dr. Daniela Rogoff, Chief Medical Officer for Skeletal Dysplasias at BridgeBio, expressed optimism about the potential of infigratinib to improve not only growth but also body proportions, which could significantly impact the lives of children with skeletal dysplasias. She emphasized the importance of continued evaluation in other related genetic conditions.

Chandler Crews, founder of The Chandler Project, a patient advocacy organization, praised the collaborative efforts with BridgeBio and QED to prioritize the community's needs in the development process. She noted the potential of infigratinib to address functional complications, which is crucial for those living with skeletal dysplasias.

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