Mar. 11 at 7:43 PM
$BBIO BridgeBio presents Phase 3 FORTIFY interim analysis results
BridgeBio Pharma 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. These data were presented at the MDA Clinical and Scientific Conference in a late-breaking oral presentation. "Treatment with BBP-418 resulted in a rapid and sustained reduction in serum creatine kinase, 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, 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," the company stated. 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.
"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," the company added.
