ReveraGen BioPharma is moving ahead with a phase 1 trial of an experimental drug in development, VBP15, to treat Duchenne muscular dystrophy (DMD). The drug, will be evaluated for safety in healthy people in this phase 1 study. VBP15 is an experimental compound intended to replicate the benefits of corticosteroid drugs like prednisone and deflazacort, which are widely used to treat DMD, but to eliminate their unwanted side effects. VBP15 is an oral drug that has shown efficacy in animal models of Duchenne muscular dystrophy through both anti-inflammatory and membrane stabilization properties. VBP15 shares some beneficial aspects of the glucocorticoid drug prednisone, but with fewer significant side effects and improved efficacy in pre-clinical trials. In studies, VBP15 reduced inflammation and stabilize cell membranes, both of which are important to treating DMD. There are no approved drugs for DMD, although daily prednisone or deflazacort are often used off label. The significant side effect profiles associated with these drugs, including stunting of growth, mood changes, bone fragility, and weight gain detract significantly from patient quality of life. Finding alternatives to glucocorticoids in DMD is an important priority and pending regulatory review and approval, clinical trials of these drugs will be an important milestone. If VBP15 is safe and well tolerated in healthy volunteers, the next step is likely to be a trial of the drug in people with DMD.
