BridgeBio Pharma, Inc. (NASDAQ:BBIO) ("BridgeBio" or the "Company"), a new type of biopharmaceutical company focused on genetic diseases, announced that positive 18-month results from PROPEL 2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, were published as an original research article in the New England Journal of Medicine (NEJM) today. Infigratinib is an investigational oral small molecule designed to inhibit FGFR3 signaling and target achondroplasia at its source. These data, which were also presented today at the 62nd Annual European Society for Paediatric Endocrinology (ESPE) Meeting in Liverpool, demonstrate continued potential best-in-class efficacy and an encouraging safety profile as a first-in-class oral treatment option.

"Publication of results from PROPEL 2 in the New England Journal of Medicine and Breakthrough Therapy Designation of infigratinib by the FDA underscore the significance and importance of these data and the potential of this oral therapy to not only increase height, but more importantly, enhance functionality for children with achondroplasia," said Ravi Savarirayan, M.D., Ph.D., of Murdoch Children's Research Institute in Melbourne, Australia, the global lead investigator for PROPEL 2 and lead author of the NEJM publication.

Key results from the PROPEL 2 dataset were presented today at ESPE in a talk titled "Oral infigratinib for children with achondroplasia: Month 18 results from the PROPEL 2 study demonstrate safety and durability of treatment effect on linear growth with improved body proportionality" by Melita Irving, M.D., a clinical geneticist at Guy's and St Thomas' NHS Foundation Trust, London, UK and investigator for the infigratinib clinical program at the Evelina London Children's Hospital. The key data that were shared included:

• Statistically significant increase in annualized height velocity (AHV) was observed in children in Cohort 5 who received a daily dose of 0.25 mg/kg/ day of infigratinib), with a mean change from baseline in AHV of +2.50 cm/year (P=0.001) at Month 18
• Mean change from baseline in height Z-score was +0.54 (P<0.001) relative to an untreated achondroplasia population at Month 18
• Statistically significant improvement in body proportionality (mean upper to lower body segment ratio) was -0.12 (P=0.001) at Month 18
• Oral infigratinib was well tolerated at Month 18, with no serious adverse events (SAE) or treatment-emergent adverse events (TEAEs) leading to treatment discontinuation
  • Additionally, there was no accelerated progression of bone age, negative changes in bone mineral density, or other bone-related adverse events observed
    
    
    
     

"We are encouraged to see no safety signals and no adverse changes in bone age or bone mineral density," said Dr. Irving. "These results point to the potential of infigratinib for children living with skeletal dysplasia, and we look forward to further evaluation of infigratinib in PROPEL 3, the ongoing Phase 3 trial, as well as in the Phase 2 portion of the ACCEL program in children with hypochondroplasia."

Infigratinib has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) based on the shared results from the PROPEL 2 clinical trial, which meet the FDA's requirement of potentially demonstrating substantial improvement in efficacy over available therapies on clinically significant endpoints. In addition to receipt of Breakthrough Therapy Designation, infigratinib has also received Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation for achondroplasia from the FDA. If infigratinib is approved, BridgeBio may qualify for a Priority Review Voucher.