NINDS small business funding leads to new therapy for Duchenne muscular dystrophy

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Scientist holding drug.

ReveraGen BioPharma, a company supported by a small business grant from the National Institute of Neurological Disorders and Stroke (NINDS), has developed a new drug for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease. Agamree (vamorolone) received U.S. Food and Drug Administration approval for the treatment of DMD in children and adults aged 2 years and older. The novel drug offers an alternative treatment option for people living with DMD.

DMD is a rare form of muscular dystrophy primarily affecting young boys. The disease is caused by the loss of dystrophin protein in muscle tissues, leading to progressive muscle weakness and difficulties with walking, balance, and daily activities. DMD patients’ lives are drastically shortened by cardiomyopathy and respiratory complications. 

Corticosteroids such as prednisone have been widely used to increase muscle strength by modest amounts and slow disease progression by a couple of years. But steroids often come with negative side effects that can be especially troublesome in children, including weight gain, short stature, and decreased bone density, especially after long-term use.

Vamorolone is a novel steroidal, anti-inflammatory drug that provides the benefits of corticosteroids, but without some of the detrimental side effects, such as stunting of growth.

After years of promising preclinical work, with funding from NINDS, ReveraGen moved the drug into clinical trials to test its safety, efficacy, and potential to reduce unwanted side effects. Results from the pivotal phase 2b VISION-DMD study ultimately led to vamorolone’s approval.

In the trials, daily oral vamorolone was administered for up to 48 months. The treatment improved muscle strength and stature compared to placebo, with an efficacy comparable to prednisone. There was also a reduction in side effects related to bone health and growth. The VISION-DMD study involved 121 boys with DMD, aged 4 to 7 years.

ReveraGen is now testing vamorolone as a treatment for Becker muscular dystrophy in an ongoing trial (NCT05166109).

The clinical trials and other drug development efforts were funded by the National Institutes of Health’s NINDS Small Business Program (R44NS095423), Eunice Kennedy Shriver National Institute of Child Health and Human Development (U54HD090254), National Institute of Arthritis and Musculoskeletal and Skin Diseases (U34AR068616), as well as the Muscular Dystrophy Association and other nonprofit organizations. Vamorolone was developed through a partnership with the National Center for Advancing Translational Sciences Therapeutics for Rare and Neglected Diseases (TRND) program.