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BridgeBio Pharma FORTIFY Trial Shows BBP-418 Significantly Improves Cardiac Health

BridgeBio Pharma presented new exploratory cardiac data from the 12-month interim analysis of FORTIFY, the Phase 3 clinical trial of oral BBP-418 in individuals living with limb-girdle muscular dystrophy type 2I/R9. The data were presented in an oral presentation at the 31st annual Congress of the World Muscle Society in Hiroshima, Japan by Volker Straub of Newcastle University, UK. Cardiomyopathy affects approximately 30% of individuals with LGMD2I/R9 and approximately 60% of those with other FKRP genotypes, and is progressive, with an annual loss of approximately 0.4% of left ventricular ejection fraction. In FORTIFY, 22.9% of participants had elevated high-sensitivity troponin I at baseline and 17.1% had an LVEF below 50%. Exploratory cardiac findings at Month 12 showed: HS troponin I is a blood test used to detect injury to heart muscle. 100% of the participants treated with BBP-418 returned to the normal range for HS troponin l by Month 12 versus 40% in the placebo arm; HS troponin I declined more with BBP-418 than placebo. At Month 12, 54% of participants treated with BBP-418 had stable or improved LVEF, a measurement of how much blood the heart's main chamber pushes out with each beat, compared with 25% of those on placebo. Both HS troponin I and LVEF will continue to be monitored in the ongoing FORTIFY study. A consistent pattern across the FORTIFY interim analysis is the proportion of treated individuals reaching normal or near-normal levels on markers of the disease e.g., 38% of patients whose CK reached normal levels. In addition to the HS troponin I finding, in which 100% of evaluable BBP-418-treated participants with elevated levels at baseline returned to the normal range by Month 12, separate data presented at WMS 2026 by Anne Katrine Kjemso Jensen, a medical student in the laboratory of John Vissing, Copenhagen Neuromuscular Center of Rigshospitalet, DK, quantified aDG glycosylation in 34 asymptomatic heterozygous FKRP carriers. Results showed: asymptomatic carriers showed significantly reduced glycosylation versus healthy controls, thereby establishing a reference point for the level associated with normal muscle function. In FORTIFY, BBP-418 treatment increased mean glycosylated aDG above carrier levels by Month 12, suggesting that BBP-418 can restore glycosylated aDG to asymptomatic levels. While many therapies in progressive neuromuscular disease aim to slow decline, BBP-418 was developed to target LGMD2I/R9 at its source, and the pattern of biomarker normalization observed to date, alongside functional improvement from baseline rather than stabilization, is consistent with the potential for BBP-418 to be a disease modifying therapy. In the Phase 3 FORTIFY trial, BBP-418 met all primary and secondary endpoints at the pre-specified 12-month interim analysis, showing treated individuals improving while placebo recipients declined across every key measure. These results were presented as a late-breaking oral presentation at the 2026 MDA Clinical and Scientific Conference, which are available here. BridgeBio believes BBP-418 is positioned to become the first and only approved therapy for individuals living with LGMD2I/R9, addressing a significant unmet need in this disease and potentially representing the first approval of a therapy for any form of LGMD. Approximately 7,000 individuals currently live with LGMD2I/R9 and other addressable a-dystroglycanopathies in the U.S. and Europe. BBP-418 is under FDA Priority Review with a PDUFA target action date of November 27, 2026. The company is also engaging regulatory agencies to identify an expedited path to approval for BBP-418 in Europe. BBP-418 has previously received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the FDA and Orphan Drug Designation from the European Medicines Agency. BBP-418 received Priority Review from the FDA, highlighting the potential for BBP-418 to address unmet need in LGMD2I/R9. Consistent with Rare Pediatric Designation from the FDA, if BBP-418 is approved, BridgeBio may qualify for a Priority Review Voucher. The company intends to initiate clinical studies of BBP-418 in LGMD2I/R9 for individuals less than 12 years of age in the first half of 2027 and in LGMD2M/R13 and LGMD2U/R20 in the near future.

BridgeBio Pharma Presents New Data in Children with Achondroplasia Study

BridgeBio Pharma presented new exploratory analyses from PROPEL 3, the global Phase 3 study of oral infigratinib in children living with achondroplasia, showing directionally favorable trends beyond growth across medical complications associated with achondroplasia, including sleep apnea, otitis media, and body composition. These data were presented at the Annual European Society for Paediatric Endocrinology, or ESPE, Meeting 2026 in Marseille, France. The mean total apnea-hypopnea index. or AHI, at 52 weeks remained consistent with the baseline mean in the oral infigratinib group, with a 10.4% increase, versus a 49.2% increase in the placebo group. In children younger than eight years of age, the mean total AHI at 52 weeks was unchanged for the oral infigratinib group, versus a 63.2% increase for the placebo group. The estimated annualized rate of otitis media, a recurrent complication in children with achondroplasia that can affect hearing and speech development, was 38% lower in the oral infigratinib group compared to the placebo group, and in children younger than 8 years of age, 47% lower in the oral infigratinib group compared to the placebo group Impact on body composition: Mean change from baseline in body mass index was smaller in the oral infigratinib group compared to placebo, with a greater increase in lean body mass and smaller increases in body fat mass and visceral fat volume compared to placebo.