BridgeBio Presents Additional Data From Interim Analysis Of FORTIFY, Phase 3 Clinical Trial Of Oral BBP-418, In Individuals With Limb-Girdle Muscular Dystrophy Type 2I/R9; To Submit NDA To FDA In H1 2026

- Phase 3 FORTIFY interim analysis results presented in a late-breaking oral presentation at MDA highlight the broad and consistent efficacy of BBP-418 across key clinical endpoints and prespecified subgroups

- Early separation from placebo observed in 100MTT, with improvements in ambulation evident as early as three months following initiation of BBP-418 treatment

- Based on the positive interim analysis results, BridgeBio intends to submit an NDA to the FDA for traditional approval in the first half of 2026 with a U.S. launch anticipated in late 2026/early 2027

- If successful, BBP-418 could be the first approved therapy for individuals living with LGMD2I/R9, potentially representing the first approval of a therapy for any form of LGMD

PALO ALTO, Calif., March 11, 2026 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (NASDAQ:BBIO) ("BridgeBio" or the "Company"), a biopharmaceutical company focused on developing medicines for genetic conditions, today presented additional positive data from the interim analysis of FORTIFY, the Phase 3 clinical trial of oral BBP-418, in individuals with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). These data were presented at the MDA Clinical and Scientific Conference in a late-breaking oral presentation by Katherine Mathews, M.D., Professor of Pediatrics and Neurology at the University of Iowa's Roy J. and Lucille A. Carver College of Medicine.

New efficacy findings presented from the interim analysis include the following:

• Demographics, baseline characteristics, and baseline values across key endpoints were generally well balanced between the BBP-418 and placebo arms
• Treatment with BBP-418 resulted in a rapid and sustained reduction in serum creatine kinase (CK), with decreases observed as early as three months and maintained through 12 months. Among individuals treated with BBP-418 for 12 months, 59.6% achieved CK levels within 2x the Upper Limit of Normal (ULN), and 38.3% achieved normalization of CK levels. The ULN is the highest value within the normal range for serum CK
• BBP-418 demonstrated a rapid time to separation on the 100-meter timed test (100MTT), with improvements in ambulation showing separation from placebo as early as three months post treatment. At 12 months, individuals treated with BBP-418 completed the 100MTT approximately 31 seconds faster than those receiving placebo 
• On another measure of ambulation, the 10-meter walk test (10MWT), BBP-418-treated participants improved from baseline by 0.13 m/s, compared to a decline of 0.10 m/s in the placebo arm
• Consistent treatment effect observed across all prospectively defined subgroups, including genotype, age, and baseline forced vital capacity (FVC), with point estimates favoring BBP-418 for alpha-dystroglycan (αDG), CK, 100MTT, and FVC
   

New safety findings presented from the interim analysis include the following:

• BBP-418 was generally well tolerated, with a safety profile comparable to placebo. Treatment-emergent adverse events (TEAEs) were reported in 93.2% of individuals receiving BBP-418 and 100% of individuals receiving placebo. Grade ≥3 TEAEs occurred at similar rates between groups (5.4% for BBP-418 vs. 5.3% for placebo)
• Serious TEAEs were reported in 5.4% of BBP-418-treated individuals compared to 7.9% of those receiving placebo. No treatment-related serious TEAEs were reported in either arm, and no deaths occurred during the study
• The most common treatment-emergent adverse events (≥10% in the BBP-418 arm) were diarrhea (39.2% vs. 52.6% for placebo), procedural pain (20.3% vs. 10.5%), fall (18.9% vs. 26.3%), nasopharyngitis (17.6% vs. 21.1%), nausea (16.2% vs. 7.9%), arthralgia (14.9% vs. 10.5%), headache and COVID-19 (10.8% vs. 13.2%), and influenza (10.8% vs. 7.9%)
• All events of diarrhea were mild to moderate (Grade 1–2) and no serious TEAEs related to diarrhea were reported
• No new or unexpected safety findings, relative to prior clinical experience, with BBP-418 were observed. There was no laboratory pattern suggestive of hepatic, renal, cardiac, hematologic, or metabolic toxicity, and no clinically meaningful changes were observed in ECG or echocardiographic parameters
   

In addition to the late-breaking oral presentation at the MDA and Clinical Scientific Conference, one additional oral presentation and four posters were also shared, which focused on advancing the understanding of BBP-418 and LGMD2I/R9. These findings included:

• A High-Throughput Assay for Measuring Ribitol Response Across FKRP Variants, presented by Yujiao Yang, Ph.D., Postdoctoral Associate in the Laboratory of Monkol Lek at Yale School of Medicine
  • This high-throughput, cell-based assay provides a scalable framework for systematically evaluating therapeutic responsiveness across the full spectrum of possible FKRP variants in LGMD2I/R9.
• Systematic Literature Review of Clinical Outcomes and Disease Burden in LGMD2I/R9, presented by Karen Bartley, Ph.D., MPH, Senior Director of Value and Health Economics at BridgeBio
  • Findings from this systematic literature review highlight the substantial impact on health due to LGMD2I/R9 and identify key evidence gaps, including limited data on economic and caregiver burden
• Real-World Insights into LGMD, Including Subtype 2I/R9: Treatment Patterns, Health Care Resource Utilization, and Costs, presented by Karen Bartley, Ph.D., MPH, Senior Director of Value and Health Economics at BridgeBio
  • Limited data exist on healthcare resource utilization (HCRU) in LGMD2I/R9. Results from this analysis demonstrate substantial clinical burden, elevated comorbidity, and significantly higher HCRU and medical costs among individuals with LGMD2I/R9. Introduction of the specific ICD-10-CM code for LGMD2I/R9, G71.036, should improve identification and enable more robust research in this population
• Long-Term Survival, Quality-Adjusted Life-Years, and Economic Burden in LGMD2I/R9: A Health Outcomes Model, presented by Karen Bartley, Ph.D., MPH, Senior Director of Value and Health Economics at BridgeBio
  • LGMD2I/R9 was associated with reduced life years and quality-adjusted life years, driven primarily by cardiomyopathy and loss of ambulation, with outcomes and lifetime burden varying by genotype. Findings from this health outcomes model are consistent with patterns observed in other muscular dystrophies and underscore the substantial burden of LGMD2I/R9
• The Journey of BBP-418: From LGMD2I/R9 Disease Pathophysiology to Registrational Clinical Trials, presented by Canan Bilgin, M.D., Senior Director of Clinical Development at BridgeBio
  • Over a decade of foundational research has elucidated the pathophysiology of LGMD2I/R9, identified a potential disease-modifying therapy, and enabled both functional and biomarker-driven clinical evaluation of BBP-418 in LGMD2I/R9
     

BridgeBio intends to submit an NDA to the FDA for traditional approval in the first half of 2026 with a U.S. launch anticipated in late 2026/early 2027. The Company is also engaging regulatory agencies to identify an expedited path to approval for BBP-418 in Europe. If successful, BBP-418 could be the first approved therapy for individuals living with LGMD2I/R9, potentially representing the first approval of a therapy for any form of LGMD. BridgeBio intends to initiate clinical studies of BBP-418 in LGMD2I/R9 for individuals less than 12 years of age and in LGMD2M/2U in the near future.