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BridgeBio Pharma Announces Positive Phase 2 Data for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i)
- 43% increase in the ratio of glycosylated alpha-dystroglycan (αDG) to total αDG from baseline were measured across all three dosing cohorts, signifying the
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"- 43% increase in the ratio of glycosylated alpha-dystroglycan (αDG) to total αDG from baseline were measured across all three dosing cohorts, signifying the oral therapy has the potential to address both the root cause of LGMD2i and drive functional improvements for patients- Average of 70% reduction in creatine kinase (CK), a key marker of muscle breakdown, after 90 days of treatment and average 77% reduction after 180 days- Increase in velocity in the 10-meter walk test (10MWT) at day 90 and 180, which is an improvement over the decline in velocity seen in the natural history data- If the development program is successful, BridgeBio believes BBP-418 could be the first approved therapy for the treatment of patients with LGMD2i- BridgeBio to host investor call on March 14, 2022 at 8:00 AM ET PALO ALTO, Calif., March 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 announced positive data from the Phase 2 study of BBP-418 in patients with limb-girdle muscular dystrophy type 2i (LGMD2i). The results are featured in a poster at the Muscular Dystrophy Association (MDA) 2022 Annual Meeting, taking place in Nashville, Tennessee on March 13 – 16, 2022. BridgeBio believes these initial results indicate the potential for BBP-418 to increase glycosylation of αDG and drive functional improvements for patients, as well as reduce creatine kinase, a key marker of muscle breakdown. Furthermore, the 90- and 180-day data show improvements on walk tests from baseline, which the Company believes suggests a potential impact on clinical function and on the rate of disease progression. “To date, people with LGMD2i have no approved disease-modifying treatment options. Many of these patients see their quality of life deteriorate rapidly and lose their functional independence, including their ability to walk. Our preliminary trial data holds promise for this unmet patient need as our investigational therapy is shown to be generally well-tolerated and to improve several key markers associated with a patient’s decline,” said Douglas Sproule, M.D., M.Sc., chief medical officer of ML Bio Solutions. ML Bio Solutions, based in Charlotte, North Carolina, is the affiliate compa...