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On March 14, 2022, BridgeBio Pharma, Inc.
LGMD2i is a monogenic autosomal recessive disorder caused by partial loss-of-function mutations in the FKRP gene, resulting in hypoglycosylation of the αDG protein associated with stable muscle cells
BBP-418 is designed to properly glycosylate αDG in muscle cells, restore normal function, and potentially improve muscle strength and function in patients
The Phase 2 clinical trial enrolled 14 subjects, including ambulatory and ambulatory LGMD2i patients
Participants had an average 43% increase in the ratio of glycosylated αDG to total αDG from baseline, suggesting that oral therapy has the potential to address the underlying causes of LGMD2i and promote functional improvements in patients
BridgeBio believes that these preliminary results suggest that BBP-418 has the potential to increase glycosylation of αDG and drive functional improvements in patients, as well as reduce CK, a key marker of muscle breakdown
References:
[1] BridgeBio Pharma Announces Positive Phase 2 Data for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i).
(Original abridged)
