Advancing BBP-418 in Phase 3 Settings as Potential Limb Girdle Muscular Dystrophy Treatment: Douglas Sproule, MD, MSc

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"Our goal is to execute the study as effectively as possible, to advance this therapy as quickly and as effectively as possible, [and] to make this product available for all patients affected by this disease."

BBP-418, an investigational oral substrate supplementation therapy in development for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), is designed to stabilize muscle cells during contraction to potentially stop any further muscle damage. The therapy is engineered to provide supraphysiological levels of an endogenous substrate upstream of the mutant FKRP enzyme to help the drive of residual activity of the enzyme to glycosylate αDG. Recently, BridgeBio Pharma announced the dosing of the first patient in its phase 3 FORTIFY clinical trial (NCT05775848) assessing BBP-418 in patients living with LGMD2I/R9.¹

FORTIFY, a randomized, double-blind, placebo-controlled study aimed to explore the safety and efficacy of BBP-418, has a planned interim analysis at 12 months to assess glycosylated αDG as a surrogate end point with the potential of the findings to support an accelerated approval. Participants will be assessed using the North Star Assessment for Dysferlinopathy and many other secondary end points, with findings expected as confirmatory clinical data in late 2024/early 2025.

Recently, Douglas Sproule, MD, MSc, chief medical officer at ML Bio Solutions, an affiliate of BridgeBio, sat down with NeurologyLive® in an interview to discuss the company’s progress of BBP-418 from its initial animal experiments to phase 3 trials. He also talked about the key goals and expectations for the global phase 3 study for LGMD as well as the impact the agent could have on patients as a treatment for 2I/R9.

REFERENCES
1. BridgeBio pharma announces opportunity for accelerated approval pathway in limb-girdle muscular dystrophy type 2i (lgmd2i/r9) based on glycosylated alpha-dystroglycan (⍺dg) levels and announces first patient dosed in fortify phase 3 study. News Release. BridgeBio Pharma. Published July 31, 2023. Accessed September 12, 2023. https://investor.bridgebio.com/news-releases/news-release-details/bridgebio-pharma-announces-opportunity-accelerated-approval