BridgeBio Announces First Child Dosed in PROPEL 3, its Phase 3 Clinical Trial for Infigratinib in Children with Achondroplasia

- BridgeBio has dosed the first child in PROPEL 3, a one-year, 2:1 randomized, placebo-controlled Phase 3 pivotal trial evaluating the efficacy and safety of infigratinib in children with achondroplasia

-  The U.S. Food and Drug Administration (FDA) and European Union (EU) European Medicines Agency (EMA) shared positive feedback that the PROPEL 3 trial design would be acceptable as a registrational study to support a marketing application

- Data from PROPEL 2, BridgeBio’s Phase 2 clinical trial of infigratinib in children with achondroplasia, supports infigratinib’s potential as an oral, well-tolerated treatment option; results shared at ENDO 2023 included a significant increase in annual height velocity (AHV) with a mean change of +3.38 cm/year from baseline at six months, and early but promising trends towards improvement in proportionality, as measured by the upper and lower body segment ratio

- BridgeBio expects to begin development of infigratinib in hypochondroplasia, with the initiation of an observational lead-in study in the first half of 2024

PALO ALTO, Calif., Dec. 13, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the first child has been dosed in PROPEL 3, a Phase 3 clinical trial studying the efficacy and safety of infigratinib in children with achondroplasia. Both the U.S. FDA and the EU EMA indicated the trial design for PROPEL 3 would be acceptable as a registrational study to support a marketing application for the treatment of children with achondroplasia.

“The Phase 2 data for infigratinib has been very promising and suggests potential to increase growth, improve functionality and reduce the associated medical complications in children with achondroplasia. With the recent dosing of the first child in the Phase 3 trial, I am hopeful that we are one step closer to providing a safe, effective oral therapy to the people in the achondroplasia community who are seeking treatment,” said Dr. Ravi Savarirayan, M.D., Ph.D., clinical geneticist and leader of the molecular therapies research group at the Murdoch Children’s Research Institute in Melbourne, Australia and the global lead investigator for PROPEL 3.

PROPEL 3 is a global, one-year, 2:1 randomized, double-blinded placebo-controlled clinical trial, which will evaluate the efficacy and safety of infigratinib in children with achondroplasia aged 3 to