ReveraGen BioPharma, based in Silver Spring, Md., is moving ahead with a phase 1 trial of an experimental compound in development to treat Duchenne muscular dystrophy (DMD), made possible by a $2 million grant from U.S.-based MDA and three United Kingdom-based DMD organizations.
The drug, dubbed VBP15, will be evaluated for safety in healthy people in this phase 1 study.
“This new trial of VB15 in healthy volunteers is something MDA and ReveraGen have worked hard to achieve and is an important step forward,” said neurologist Valerie Cwik, MDA’s chief medical and scientific officer. “If found to be safe in healthy volunteers, the next likely step would be to begin testing in those with Duchenne muscular dystrophy. And, since it does not target a specific DMD-causing mutation, this drug has the potential to help everyone with DMD.”
VBP15 designed to provide benefits, not side effects, of prednisone
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.
Benefits of corticosteroids (also known as glucocorticoids) in DMD are thought to be dampening of muscle inflammation and stabilization of muscle-fiber membranes. Among the unwanted side effects that accompany chronic use of corticosteroids are weight gain, cessation of growth, thinning of the bones and skin, high blood pressure, cataracts, suppression of the immune system, and mood changes.
VBP15 and similar compounds may also have applications in other muscle diseases in which inflammation plays a role, such as Becker muscular dystrophy (BMD), type 2B limb-girdle muscular dystrophy (LGMD), Miyoshi myopathy, dermatomyositis (DM) and polymyositis (PM).
Additional MDA support for compound’s development
In 2012, MDA awarded a $1.5 million grant to ReveraGen for development of this type of compound. And in 2013, an MDA-supported study in mice with a DMD-like disorder found that VBP15 improved muscle strength and decreased muscle inflammation, while preserving immune system function, growth and bone structure.
In August 2013, MDA awarded a three-year grant to Jyoti Jaiswal at Children’s National Health System and the George Washington University, both in Washington, D.C., to conduct experiments in mice and patient cells for clues to the possible value of VBP15 in treating type 2B LGMD and Miyoshi myopathy.
Next step likely to be trial in DMD patients
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. When more details are known, they will be posted on the MDA website.