Business
BridgeBio Pharma Shares Preliminary Findings on Novel Bioassay Measuring Glycosylated Alpha-dystroglycan (⍺DG) in Patients with Limb-girdle Muscular Dystrophy Type 2I (LGMD2I)
- BridgeBio has developed a validated bioassay that directly measures glycosylated ⍺DG, which is central to LGMD2I disease, and enables monitoring of
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"- BridgeBio has developed a validated bioassay that directly measures glycosylated ⍺DG, which is central to LGMD2I disease, and enables monitoring of responses to disease-modifying therapies in LGMD2I patients - BridgeBio also shared 15-month results from its ongoing Phase 2 study, which showed a doubling of glycosylated ⍺DG in LGMD2I patients treated with BBP-418 - A sustained decrease of ≥70% in creatine kinase (CK), a marker of muscle breakdown, was observed with BBP-418 treatment at 15 months - Improvements in ambulatory and clinical function measures were observed after 15 months of treatment with BBP-418 - Based on the Phase 2 results, BridgeBio is embarking on a registration-enabling Phase 3 study with an initiation in mid-2023 PALO ALTO, Calif., March 21, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced the Company’s novel validated muscle tissue-based bioassay, which measures the amount of glycosylated ⍺DG in patients with LGMD2I, at the MDA 2023 Annual Meeting. Additionally, BridgeBio shared updated results at Month 15 for its Phase 2 clinical trial and the design for its pivotal Phase 3 study for patients with LGMD2I. BridgeBio will host an investor call with Jeffrey Rosenfeld, M.D., Ph.D., a specialist in neuromuscular medicine and professor of neurology at Loma Linda University School of Medicine on Tuesday, March 21 at 8:30 am ET to discuss the results shared at the meeting. The novel bioassay was developed by BridgeBio to measure the amount of glycosylated ⍺DG, and to evaluate the impact of BBP-418 treatment. When deficient, glycosylated ⍺DG is the direct cause of muscle breakdown in LGMD2I. The bioassay allowed the Company to evaluate natural history and Phase 2 data to inform the Phase 3 study design. Highlights of the results include: Development of a novel, validated, quantitative method to measure glycosylated ⍺DG in muscleObservation of ~90% reduction in glycosylated ⍺DG in muscle biopsies from patients with LGMD2I compared to healthy individualsHigher glycosylated ⍺DG in L276I/L276I homozygous patients (~11%) who are characterized by a milder clinical phenotype than other fukutin-related protein (FKRP) genotypes (~5%)Stable glycosylated ⍺DG levels in untreated p...