ReveraGen BioPharma has announced that it has received a $3 million grant from the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health to conduct phase 2 clinical trials of vamorolone (previously VBP15) in boys with Duchenne muscular dystrophy (DMD).

Vamorolone, a “dissociative steroid,” is an anti-inflammatory compound. Researchers hope it will provide the same benefits of traditional glucocorticoids, such as prednisone and deflazacort, without the unwanted side effects — including stunted growth, insulin resistance and weight gain — of those drugs. In clinical trials, glucocorticoid steroids have been shown to benefit DMD patients, but many find the side effects to be burdensome or intolerable.

The news follows encouraging results from a phase 1 trial conducted in healthy adult volunteers, and is an example of the success of MDA’s Venture Philanthropy program (MVP), which provided $2.9 million in support for early-stage and preclinical development of the drug, along with phase 1 clinical testing. Funds through MVP, the drug development arm of MDA’s translational research program, are leveraged to move promising drugs like vamorolone through the critical early stages of development to ensure that they attract follow-on funding from other sources and progress through the drug development pipeline towards market approval.

Additionally, through its basic research program, MDA is supporting development of biomarkers for glucocorticoids including vamorolone with a grant to Yetrib Hathout for $300,000, and research by Eric Hoffman to understand the effects of glucocorticoids including vamorolone on muscle repair with a grant for $321,659.

“Thanks in part to early funding through MDA’s basic and MVP grant programs, ReveraGen has received significant follow-on funding from the National Institutes of Health, which will allow it to move this promising experimental drug forward into phase 2 trials,” said MDA Scientific Program Officer Laura Hagerty, Ph.D.

The upcoming phase 2a multiple ascending dose studies to test safety, tolerability and pharmacokinetics (the drug levels in the body) will be conducted in boys with DMD ages 4 to 7 years, with enrollment expected to begin in the second quarter of 2016. A six-month extension study will follow, with the goal to test clinical efficacy, safety and pharmacodynamics (effects the drug has on the body).

“We are proud to have supported the early development and clinical testing of vamorolone, and we are gratified to see the drug progress through the development pipeline toward becoming a potential therapy for DMD,” Hagerty added. “We look forward to the results of the upcoming phase 2 trials. If vamorolone is proven safe and effective, it could someday be a valuable treatment option for our DMD families.”