Bridgebio Pharma Presents 12-Month P-II Study Results of BBP-418 for Limb-Girdle Muscular Dystrophy Type 2i at WMS 2022
Shots:
- The P-II study evaluating the safety, tolerability, feasibility, and usefulness of selected clinical efficacy & PD of BBP-418 in 14 patients (incl. ambulatory & non-ambulatory) with LGMD2i
- The results showed increased glycosylation of ⍺DG in all dose cohorts with an avg. increase in ⍺DG ratio of +0.21 @Day 90, ≥75% reduction in CK sustained @12mos., improvements from baseline in NSAD (0.95) & 10MWT velocity (0.09m/s) @12mos. with no treatment-related SAEs or dose-limiting toxicities, reduction in muscle damage & motor function
- BridgeBio & ML Bio Solutions closely work with regulatory agencies & will initiate a P-III trial in 2023. BridgeBio also highlighted the development of a novel bioassay to evaluate the extent of ⍺DG glycosylation from muscle biopsy samples
Ref: Bridgebio Pharma | Image: Bridgebio Pharma
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