Limb Girdle Muscular Dystrophy: First Patient Dosed in Phase 2 Trial

According to a story from One News Page, the biopharmaceutical company BridgeBio Pharma and its affiliate ML Bio Solutions have recently announced that dosing has begun in its phase 2 clinical trial. This clinical trial is investigating the company’s experimental product candidate BBP-418 as a potential treatment for limb girdle muscular dystrophy type 2i (LGMD2i). It is the first orally available medicine developed for this indication. BridgeBio is focused on developing impactful treatments for patients living with genetic diseases and cancers with a genetic basis.

About Limb Girdle Muscular Dystrophy (LGMD)

Limb girdle muscular dystrophy (LGMD) also known as Erb’s muscular dystrophy, is a group of muscular dystrophies that are most characterized by progressive muscle atrophy that most prominently affects the muscles of the shoulders and hips. The precise genetic cause of the disorder varies depending on the subtype, but the end result is the same: the muscles are unable to form certain proteins that are necessary for normal muscular function. Symptoms may include oversized muscle fibers, weak shoulder muscles, respiratory muscle weakness, facial muscle weakness, lower back pain/discomfort, and heart palpitations. The rate of disease progression varies between individual cases. Onset is usually between the ages of 10 to 30 years. There are no disease modifying therapies for limb girdle muscular dystrophy; management usually consists of physical, occupational, respiratory, and speech therapy. Though not always fatal, heart and respiratory muscle weakness can be life threatening. To learn more about limb girdle muscular dystrophy, click here.

About BBP-418 and The Clinical Trial

The type 2i variant of this disorder is linked to mutations impacting the FKRP gene. BBP-418 was developed to bypass the defect in this disease by supplying the FKRP enzyme precursor molecules that are intended to supplement the sugars used by the enzyme, which acts on the αDG protein in the muscle cells. The ultimate goal is to help improve the muscle function and strength in these patients. BBP-418 has earned Orphan Drug designation from the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

The trial is anticipated to include a total of 16 patients. Endpoints include improvements in measures of muscle function and changes to αDG glycosylation levels. With no disease modifying treatments, the results of this trial could have a major impact on the outcomes in this disease.

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