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BridgeBio Pharma’s ML Bio Solutions Announces Dosing of First Subject in Phase 1 Clinical Trial of BBP-418 For Limb Girdle Muscular Dystrophy Type 2i (LGMD2i)
ML Bio Solutions has initiated a Phase 1 clinical trial of BBP-418 in healthy volunteersML Bio Solutions’ sponsored lead-in study is enrolling patients with
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"ML Bio Solutions has initiated a Phase 1 clinical trial of BBP-418 in healthy volunteersML Bio Solutions’ sponsored lead-in study is enrolling patients with LGMD2i across twelve sites in the U.S. and internationally, in collaboration with the LGMD-GRASP consortium SAN FRANCISCO, June 11, 2020 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) affiliate ML Bio Solutions today announced that the first subject has been dosed in the Phase 1 clinical trial of BBP-418, an orally-administered small molecule therapy being evaluated for the treatment of limb girdle muscular dystrophy type 2i (LGMD2i). The Phase 1 clinical trial is designed to assess safety, tolerability, pharmacokinetics and food effect of BBP-418 in healthy volunteers. In collaboration with the GRASP-LGMD consortium based at Virginia Commonwealth University, which is led by Nicholas Johnson, M.D., M.S.C.I., ML Bio Solutions is also enrolling a LGMD2i lead-in study across multiple centers in the U.S. and Europe. ML Bio Solutions also announced that the U.S. Food and Drug Administration (FDA) approved its Investigational New Drug (IND) application for BBP-418 in March 2020.\n “The FDA’s acceptance of the investigational new drug application for BBP-418 marks a turning point for patients with LGMD2i, who currently have no targeted treatment options. BBP-418 has demonstrated efficacy in both mild and severe animal models of LGMD2i, and the start of the Phase 1 trial brings us one step closer to translating its potential to change the course of this incurable and debilitating disease,” said Uma Sinha, Ph.D., chief scientific officer of ML Bio Solutions and BridgeBio. LGMD2i is an inherited muscular dystrophy that is associated with two mutant copies of a gene called fukutin-related protein (FKRP). The FKRP enzyme enables the proper functioning of a fully glycosylated alpha-dystroglycan (α-DG) protein on muscle cells. While fully glycosylated α-DG anchors the inside and the outside of cells to act as a “shock-absorber,” hypo-glycosylated α-DG – such as occurs in LGMD2i and fukutinopathies – leads to muscle cells that cannot withstand the stress of normal cellular activities. ML Bio Solutions’ oral therapy BBP-418 is designed to enhance the function of enzymes like FKRP in order to restore glycosylation levels of α-DG protein and thereby recover its shock absorber funct...