ReveraGen Biopharma and Santhera Pharmaceuticals have secured a $1.2 million grant for funding their clinical trial of vamorolone in Becker muscular dystrophy (BMD) from the US Food and Drug Administration (FDA).

The orphan grant funding has been issued under Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01) grants program.

It adds to the other grants provided by the US National Institutes of Health, US National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), and the Foundation to Eradicate Duchenne.

The grants will be used by ReveraGen Biopharma and Santhera Pharmaceuticals for launching a 24-week clinical exploratory trial featuring both adults and children.

Becker muscular dystrophy is a progressive muscle wasting disease, which is identical to Duchenne muscular dystrophy (DMD), but is milder usually compared to the latter.

Paula Clemens — Professor of Neurology at the University of Pittsburgh School of Medicine, and co-principal investigator on the FDA, NIH, and Foundation to Eradicate Duchenne awards said: “There are currently no approved drugs for BMD in any country, and there is a high unmet need.”

Vamorolone, which is a dissociative steroid drug, has demonstrated efficacy retention and reduction of safety concerns that are associated usually with corticosteroids in Duchenne muscular dystrophy.

The dissociative steroid drug was discovered by US-based ReveraGen BioPharma. It is being developed in collaboration with Switzerland-based Santhera Pharmaceuticals which owns the global rights to the drug candidate for all indications.

According to Santhera Pharmaceuticals, the double-blind trial will evaluate the efficacy and safety of daily treatment with vamorolone on motor outcomes and established biomarker outcomes.

Participants in the clinical exploratory trial will be randomly grouped in a 2:1 ratio to receive vamorolone or placebo. The clinical trial is planned to enroll patients at sites in Padova in Italy and Pittsburgh in the US.

Elena Pegoraro — Professor of Neurology at the University of Padova in Italy said: “While the drug development pipeline has greatly expanded for DMD in recent years, there are very few clinical investigational efforts underway for BMD.

“Corticosteroids are often not tolerated by patients with BMD due to their side effects. Therefore, the lessened side effect burden of vamorolone seen in DMD trials may prove important to the underserved BMD patient community.”

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