This Novel Therapy Could Reverse the Biological Causes of DMD

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Source: pixabay.com

Mitobridge, Inc., a pharmaceutical company that focuses on improving mitochondrial function, just announced the achievement of a vital milestone in the first-in-human clinical trial of PPAR-delta modulator, MA-0211.

This study will yield the foundation for an up and coming trial program for patients with Duchenne muscular dystrophy (DMD).

Duchenne muscular dystrophy, or DMD for short, is a rare genetic condition that is caused by dystrophin gene mutations.

These mutations cause dystrophin genes to lose their function, in turn causing a series of progressive skeletal, cardiac, and muscle weakness in patients. To learn more about DMD, click here.

Currently, there is no cure for DMD, and the standard-of-care is merely to manage its symptoms. However, MA-0211, which is actually the first clinical compound out of Mitobridge’s mitochondrial enhancement platform, aims to reverse the mitochondrial deficits in DMD. These mitochondrial deficits contribute to the dystrophic muscles in patients and thus, play the main role causing DMD and its progression.

Mitobridge has backed up this theory with a ton of nonclinical data from genetic animal models and patient samples, such as the mdx mouse model. These models had improved mitochondrial function, overall energy metabolism, and muscle regeneration after being administered MA-0211.

These data provided enough evidence to then advance MA-0211 into clinical development phases, in which the safety, tolerability, and pharmacokinetics of MA-0211 will be tested in healthy volunteers. After this study, MA-0211 will be administered to patients with DMD.

Even though this therapy is still only in the early stages of development, starting is always the hardest part. Hopefully, this is the start of MA-0211 picking up more speed and it won’t be long until DMD patients can begin registering for a later-phase clinical trial.

To learn more about Mitobridge’s novel treatment in Business Wire, click here!


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