The California-based biotech announced positive results from their latest limb-girdle muscular dystrophy type 2i (LGMD2i) program trials, linking their drug, BBP-418, to positive biomarker changes and functional endpoints in the right direction.
LGMD2i is a rare form of muscular dystrophy caused by mutations in the FKRP gene, for which there are currently no approved treatment options. Mutations in the gene impair the addition of a specific sugar molecule to a protein that acts as a molecular ‘shock absorber’ in cells and normally helps prevent damage from everyday muscle use. BBP-418, also called ribitol, is said to work by catalyzing the enzyme to function again, increasing sugar-protein interactions, and theoretically enabling muscle cells to repair themselves.
The phase 2 study included 14 patients with LGMD2i, and BridgeBio tested ascending doses of the drug candidate in 3 different cohorts, assessing participants after 90 and 180 days.
Among the promising results, BridgeBio reported a 43% increase in the ratio of these sugar-protein interactions; an average of 70% reduction in the levels of creatine kinase, a marker of muscle breakdown; and an increase in walking velocity.
“The positive results from the Phase 2 study exceed expectations and are incredibly exciting as they demonstrate consistent improvements in key markers of muscle function and support further study,” concluded Amy Harper, primary investigator of the trial.