BridgeBio Pharma, Inc. (NASDAQ:BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases, today announced sustained positive results from PROPEL 2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, demonstrating continued potential best-in-class efficacy and an encouraging safety profile. Infigratinib is an oral small molecule designed to inhibit FGFR3 signaling and target achondroplasia and hypochondroplasia at their source. BridgeBio will also host an investor call on June 4, 2024, at 8:00 am ET with Ravi Savarirayan, M.D., Ph.D., of Murdoch Children’s Research Institute in Melbourne, Australia, and the global lead investigator for PROPEL 2, to discuss the results from the Phase 2 study.
To date, key results from the Cohort 5 dose escalation cohort in PROPEL 2 trial include:
- Sustained and statistically significant mean increase in AHV of +2.51cm/year from baseline at 12 months, and +2.50 cm/yr at 18 months (p=0.0015)
- Statistically significant improvement in body proportionality (mean upper to lower body segment ratio), from 2.02 at baseline to 1.88 at Month 18 (mean change from baseline, p=0.001)
- A continued well-tolerated safety profile, with no treatment-related adverse events assessed as related to infigratinib
