ReveraGen and Santhera Announce FDA Orphan Grant Funding for Clinical Trial with Vamorolone in Becker Muscular Dystrophy

Rockville, MD, USA, and Pratteln, Switzerland, September 27, 2021 – ReveraGen Biopharma and Santhera Pharmaceuticals (SIX: SANN) announce that ReveraGen has received a USD 1.2 million grant from the FDA under their “Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)” grants program. The grant adds to existing grants from the National Institutes of Health, NIAMS, and the Foundation to Eradicate Duchenne to initiate a clinical trial of vamorolone in adults and children with Becker muscular dystrophy, a progressive muscle wasting disease similar to Duchenne muscular dystrophy, but usually milder.

Vamorolone, a dissociative steroid drug, that has shown retention of efficacy and reduction of safety concerns typically associated with corticosteroids in Duchenne muscular dystrophy (DMD) will now be tested in a 24-week clinical exploratory trial in Becker muscular dystrophy (BMD). BMD is caused by mutations of the DMD gene but shows residual dystrophin protein in muscle, and variable onset and progression of muscle weakness. The double-blind trial will test efficacy and safety of daily vamorolone on motor outcomes and established biomarker outcomes, with participants randomized 2:1 vamorolone or placebo. The clinical trial plans to enroll at sites in Padova (Italy) and Pittsburgh (USA).

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

The mechanisms of actions, providing basis for vamorolone efficacy as demonstrated in the pivotal VISION-DMD study in the more severe DMD, are felt to be highly relevant to BMD too. In addition, vamorolone is hypothesized to increase dystrophin protein levels in BMD via inhibition of microRNAs that deleteriously target dystrophin, and this may further complement the mechanism of action specifically in BMD [1-3].

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