BridgeBio Launches New Subsidiary to Tackle Limb-Girdle Muscular Dystrophy

BridgeBio launched Charlotte, N.C.-based ML Bio Solutions to develop the LGMD2i asset BBP-418, a substrate supplementation therapy.

Bay Area-based BridgeBio LLC is launching another subsidiary to tackle neuromuscular diseases such as Limb-Girdle Muscular Dystrophy Type 2 (LGMD2i).

This morning, BridgeBio launched Charlotte, N.C.-based ML Bio Solutions to develop the LGMD2i asset BBP-418, a substrate supplementation therapy. BridgeBio did not provide information regarding how much financing its spinout will have at its disposal or the number of employees. In its announcement, BridgeBio said it is “committing R&D expertise” to advance the development of BBP-418. The new company will begin a natural history study of the asset later this year and plans to initiate interventional trials shortly thereafter.

Limb-Girdle Muscular Dystrophy Type 2 is an autosomal recessive disease. It is caused by loss-of-function mutations in the FKRP gene, which lead to decreased glycosylation of alpha-dystroglycan (α-DG). Patients with LGMD2i cannot effectively bind proteins to stabilize the muscle cell, which ultimately causes damage to the muscle and leads to a loss of strength. LGMD2i is a progressive and debilitating disease that affects an estimated 7,000 patients across the United States and European Union. . It is associated with loss of mobility and ultimately respiratory and cardiac failure, and there is presently no approved treatment.

The BridgeBio subsidiary is not alone in working toward a treatment for Limb-Girdle Muscular Dystrophy. Sarepta Therapeutics is also focused on the disease. In May, the Cambridge, Mass.-based company acquired a new asset to bolster its other pipeline therapies aimed at the disease.

For ML Bio Solutions, its asset BBP-418 aims to bypass the core metabolic defect in LGMD2i by providing a pro-drug that can be metabolized into the missing substrate in the α-DG sugar backbone.

“BBP-418 is a novel approach that targets LGMD2i at the source of disease, and exemplifies the BridgeBio mission of creating life-altering medicines by pairing well-understood genetically- driven phenotypes with treatments whose mechanisms target the sources of disease,” said Uma Sinha, chief scientific officer of BridgeBio Pharma. “We are thrilled to partner with the foremost experts in the field to explore the translation of these important discoveries into clinical impact for patients who currently lack effective treatment options.”

As ML Bio Solutions moves forward with its program, it will be supported by BridgeBio, as well as the McColl Lockwood Laboratory for Muscular Dystrophy Research at Atrium Health, which is where the therapeutic approach for BBP-418 originated.

“By combining the scientific leadership of the McColl-Lockwood Laboratory with the rare disease development expertise at BridgeBio, we believe we can accelerate the path to making a safe and effective disease-modifying treatment available to LGMD2i patients as soon as possible,” Luther Lockwood, chairman of the board and president of ML Bio Solutions said in a statement.

The launch of ML Bio Solutions comes about a year after BridgeBio launched QED Therapeutics to develop infigratinib, a tyrosine kinase receptor FGFR that had been discontinued by Novartis in 2017. BridgeBio’s business model is to launch a subsidiary to develop each of its 15 pipeline assets. Two years ago, the company launched Eidos Therapeutics, to develop a novel small-molecule treatment for transthyretin (TTR) amyloidosis.