Benzinga | Feb 19, 2021 07:31AM EST

BridgeBio Pharma And Affiliate ML Bio Solutions Announce Dosing Of First Patient In Phase 2 Trial Of BBP-418 In Limb Girdle Muscular Dystrophy Type 2i

SAN FRANCISCO, Feb. 19, 2021 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (NASDAQ:BBIO),a clinical-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers,and affiliate ML Bio Solutions today announced that the first patient has been dosed in a Phase 2 trial of BBP-418 in patients with LGMD2i. BridgeBio and ML Bio's BBP-418 is the first-ever oral disease-modifying investigational treatment for LGMD2i. BBP-418 was granted Orphan Drug Designation for LGMD2i by the US Food and Drug Administration (FDA) in 2019, and for LGMD by the European Medicines Agency (EMA) in 2020.



LGMD2i is an inherited recessive muscular dystrophy caused by mutation of fukutin-related protein (FKRP). FKRP is an enzyme that adds a sugar molecule to a critical muscle cell structural protein called alpha-dystroglycan (?DG). Due to defective FKRP enzyme function, muscle cells of patients affected by LGMD2i lack the structural integrity that is provided by fully glycosylated ?DG protein. This leads to chronic muscle injury and loss, muscle weakness and disability. Many affected patients ultimately lose their ability to walk, and some develop a need for ventilatory support or die from heart failure. Currently, there are no disease-modifying therapies for people with LGMD2i.

BBP-418 is designed to bypass the metabolic defect in LGMD2i by providing the FKRP enzyme with precursor sugar molecules that supplement the body's natural sugars used by FKRP to glycosylate the ?DG protein on muscle cells. BBP-418 represents a novel substrate rescue approach with the potential to improve muscle strength and function.

The Phase 2 trial is expected to enroll up to 16 patients with a genetically-confirmed diagnosis of LGMD2i. In addition to safety, key efficacy measurements include changes in muscle ?DG glycosylation levels, and changes in functional measures including 10 meter walk and North Star for Dysferlinopathy (NSAD).