Phase 2 trial evaluates safety, tolerability, and efficacy of oral BBP-418 in limb-girdle muscular dystrophy type 2I/R9, suggesting therapeutic potential for restoring alpha-dystroglycan.
Limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9 or LGMDR9 FKRP-related) is caused by biallelic partial loss-of-function of the fukutin-related protein (FKRP) gene, which results in hypoglycosylation of alpha-dystroglycan (αDG). BBP-418 is an orally administered glycosylation substrate intended to saturate the partially functional FKRP enzyme, restoring effective αDG glycosylation and potentially ameliorating the root cause of LGMD2I/R9. The MLB-01-003 phase 2 study evaluates the safety, tolerability, clinical efficacy, and pharmacodynamic (PD) assessments in participants with LGMD2I/R9 receiving ascending doses of BBP-418.
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Harper et al. (2026) studied this question.
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