BridgeBio Pharma Reports Results of BBP-418 in P-II Study for the Treatment of Limb-girdle Muscular Dystrophy Type 2i
Shots:
- The P-II study evaluates BBP-418 in 14 patients including ambulatory & non-ambulatory with LGMD2i. The results showed a 43% increase in the ratio of αDG to total αDG from baseline across all 3 dosing cohorts, 70% reduction in CK @90 day in all cohorts & 77% @180 day for cohorts 1 & 2
- Additionally, 11 patients received a 50% reduction in CK with 75% reaching 2x the normal range suggested a reduction in muscle breakdown, increase in 10MWT velocity @90 & 180 days, was well-tolerated with no treatment-related serious AEs, dose-limiting toxicities or discontinuations were identified
- The company plans to meet with regulatory authorities in 2022 to discuss the paths for approval and plans to initiate a P-III trial in H2’22
Ref: Globe Newswire | Image: Bridgebio
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