BridgeBio shares long-term data from Phase 2 trial in LGMD2I/R9

BridgeBio Pharma shared new long-term data from its Phase 2 trial in patients with limb-girdle muscular dystrophy type 2I/R9 at the Annual Congress of World Muscle Societyin Charleston, South Carolina. The new long-term data remains consistent with earlier data from the Phase 2 study showing a well-tolerated safety profile and encouraging preliminary efficacy. Additionally, early changes in glycosylated DG levels at 3 months appear to be associated with ambulatory improvements at 9 months, providing support for the possible use of glycosylated DG levels as a surrogate endpoint in the ongoing Phase 3 study for accelerated approval. BridgeBio presented 21-month results from its ongoing Phase 2 trial, including: Large sustained reduction in creatine kinase observed over an extended treatment period; Stabilization in NSAD scores and ambulatory measures observed over 21-months of BBP-418 treatment; BBP-418 continues to be well-tolerated with longer-term treatment; No treatment-related SAEs or dose limiting toxicities observed with 21-months of BBP-418 dosing. “There is a serious unmet need for those with LGMD2I/R9. The consistent improvement observed in biomarkers as well as stabilization in ambulatory measures are encouraging and help predict the clinical benefit for patients. The data for BBP-418 give me hope that similar results will be carried through in the currently enrolling Phase 3 trial, FORTIFY, for patients with LGMD2I/R9,” said Amy Harper, M.D., professor in the department of neurology at Virginia Commonwealth University and primary investigator of the Phase 2 clinical trial in LGMD2I/R9. BridgeBio recently announced the first patient dosed in FORTIFY, its Phase 3 registrational study in patients with LGMD2I/R9 and is continuing to enroll throughout the U.S. with clinical trial sites planned for Europe and Australia. The Phase 3 FORTIFY registrational study is a randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of BBP-418. FORTIFY has a planned interim analysis at 12 months focused on assessing glycosylated alphaDG as a surrogate endpoint to potentially support an accelerated approval. The NSAD and secondary endpoints will be evaluated at 36 months, and results are expected to provide confirmatory clinical data.