BridgeBio Pharma, Inc. (NASDAQ: BBIO) has announced positive and sustained results from its Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia. The trial, known as Propel 2, demonstrated continued potential best-in-class efficacy and an encouraging safety profile for infigratinib. The treatment resulted in a statistically significant and sustained increase in annualized height velocity (AHV), with a mean change from baseline of +2.51cm/year at month 12 and +2.50cm/year at month 18. Additionally, at month 18, there was a statistically significant improvement in body proportionality, with the mean upper to lower body segment ratio improving from 2.02 at baseline to 1.88 (p=0.001).
Furthermore, the results indicate that infigratinib was well-tolerated as a single daily oral therapy, with no adverse events assessed as treatment-related in any participant in cohort 5. The global Phase 3 registrational study of infigratinib in achondroplasia, known as Propel 3, continues to enroll on schedule, with completion estimated by the end of 2024.
In addition to the positive results in achondroplasia, BridgeBio has announced the first child consented in Accel, an observational run-in study for infigratinib in children living with hypochondroplasia, a skeletal dysplasia closely related to achondroplasia and similarly driven by FGFR3 gain-of-function variants. The company is committed to expanding the FGFR3-related skeletal dysplasias franchise for infigratinib by accelerating development in hypochondroplasia.
The initiation of the observational study in hypochondroplasia and the obtainment of FDA and EMA alignment on the interventional study underline the potential of infigratinib as a treatment option for children with hypochondroplasia. The company aims to evaluate infigratinib in other FGFR-related skeletal dysplasias and genetic conditions, with the initiation of the observational study in hypochondroplasia representing a significant step forward.
A link to the webcast discussing the Phase 2 data at months 12 and 18 of infigratinib in children with achondroplasia will be available on BridgeBio's website. The results from the Phase 2 trial indicate the potential for infigratinib to provide benefits that could impact the lives of children with achondroplasia, and the company is committed to exploring the potential of infigratinib on wider medical and functional impacts of achondroplasia, hypochondroplasia, and other skeletal dysplasias, which hold significant unmet needs for families.
Achondroplasia is the most common cause of disproportionate short stature, affecting approximately 55,000 people in the United States and the European Union, including up to 10,000 children and adolescents with open growth plates. Hypochondroplasia, another FGFR3-associated skeletal dysplasia, is a rare condition with a similar prevalence to achondroplasia. Currently, no treatments for hypochondroplasia are approved in the United States.
BridgeBio Pharma is a commercial-stage biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases. The company's pipeline of development programs ranges from early science to advanced clinical trials, with a commitment to applying advances in genetic medicine to help patients as quickly as possible. Following these announcements, the company's shares moved -3.1%, and are now trading at a price of $27.12. Check out the company's full 8-K submission here.
