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BridgeBio Pharma Presents 12-month Results from Phase 2 Study of BBP-418 in Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i)
Novel assay developed to assess the extent of alpha-dystroglycan (⍺DG) glycosylation, the core pathology of LDMD2i, from muscle biopsy samplesIncrease in the
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"Novel assay developed to assess the extent of alpha-dystroglycan (⍺DG) glycosylation, the core pathology of LDMD2i, from muscle biopsy samplesIncrease in the ratio of glycosylated αDG to total αDG from baseline towards normal levels, suggesting that the investigational oral therapy, BBP-418, has the potential to address the root cause of LGMD2iLarge, sustained reduction in creatine kinase (CK) (>75%), a key marker of muscle breakdown, after 12 months of treatmentImprovements in key functional measures, the north star assessment for dysferlinopathy (NSAD) and 10-meter walk test (10MWT), observed after 12 months of treatment with BBP-418If successful, BridgeBio believes BBP-418 could be the first approved therapy for the treatment of patients with LGMD2i PALO ALTO, Calif., Oct. 14, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, and its affiliate company ML Bio Solutions, today reported 12-month data from the Phase 2 study of BBP-418 in patients with limb-girdle muscular dystrophy type 2i (LGMD2i). These updated results were presented in an oral presentation at the 27th International Hybrid Annual Congress of the World Muscle Society (WMS), taking place in Halifax, Canada on October 11 – 15, 2022. These additional Phase 2 results continue to support the potential for BBP-418 to restore glycosylation of alpha-dystroglycan (αDG), reduce muscle damage, and preserve motor function for patients over time, as demonstrated by the sustained increase in the ratio of glycosylated-αDG over total αDG, reduction in creatine kinase, improvements in NSAD scores, and improvements in 10MWT velocity observed over a 12-month period. “LGMD2i dramatically impacts individuals living with the disease, fundamentally robbing people of their independence. As the disease progresses, individuals with LGMD2i lose the ability to function unaided, eventually becoming wheelchair-dependent and requiring ventilation assistance, the timing of which is dependent on the severity of their disease. Our Phase 2 data show our investigational therapy continues to be well-tolerated and may have the potential to improve or slow clinical decline associated with the disease,” said Douglas Sproule, M.D., M.Sc., chief medical officer of ML Bio Sol...