PPMD is pleased to share that ReveraGen Biopharma and Santhera Pharmaceuticals announced that ReveraGen has received a $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 is a dissociative steroid drug that has shown retention of efficacy and reduction of safety concerns typically associated with corticosteroids in Duchenne and will now be tested in a 24-week clinical exploratory trial in Becker. 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).

The mechanisms of actions, providing the basis for vamorolone efficacy as demonstrated in the pivotal VISION-DMD study in Duchenne, are felt to be highly relevant to Becker as well. In addition, vamorolone is hypothesized to increase dystrophin protein levels in Becker via the inhibition of microRNAs that deleteriously target dystrophin, and may further complement the mechanism of action specifically in Becker.

For the full release, click here.