Marathon Pharmaceuticals has announced pivotal Phase 3 data evaluating the investigational drug deflazacort for the treatment of Duchenne muscular dystrophy in the journal Neurology.

The study results show that deflazacort demonstrated significant improvement in muscle strength compared with placebo in 12 weeks. In addition, results show that deflazacort was associated with less weight gain than prednisone at 12 and 52 weeks.

“Families living with Duchenne muscular dystrophy need safe and effective therapies as soon as possible, said Valerie A. Cwik, M.D., who is a co-author on the Neurology paper. “These data results show that deflazacort can be a positive alternative to prednisone for our families living with DMD, and we are encouraged by the FDA’s acceptance of two new drug applications for deflazacort.”

The Phase 3 study examined the efficacy of deflazacort vs. placebo. The study also compared the efficacy and safety of deflazacort to prednisone.

This pivotal study is one of two clinical trials exclusively licensed by Marathon, which also conducted a full clinical pharmacology program for deflazacort, including eight clinical pharmacology and safety studies and nine preclinical studies. Data from this Phase 3 trial helped form the basis for two New Drug Applications (NDAs) for deflazacort, one for an immediate-release tablet formulation and one for an oral suspension formulation. The NDAs are currently under Priority Review by the U.S. Food and Drug Administration (FDA).

Deflazacort, a glucocorticoid, works as an anti-inflammatory and immunosuppressant. In the United States, the drug is considered an investigational therapy, as it has not been approved by the FDA for any disorder. Versions of deflazacort are approved in other countries, but not for DMD.

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