US FDA Grants Expanded Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

About Us

Our mission is to empower people living with neuromuscular diseases to live longer, more independent lives.

ReveraGen BioPharma Announces Start of Phase 1 Clinical Trial of VBP15 Dissociative Steroid Drug

Product holds potential for replacing glucocorticoids in Duchenne muscular dystrophy and other chronic inflammatory states

SILVER SPRING, M.D./ CHICAGO – February 18, 2015 – ReveraGen BioPharma today announced the start of a Phase 1, first-in-human, clinical trial of a novel dissociative steroidal drug, VBP15, under development for Duchenne muscular dystrophy.  Recruitment in the trial is underway, with the initial doses in the first volunteers having been received. 

“Glucocorticoids are a mainstay of therapeutics for Duchenne muscular dystrophy, but the side effect profiles detract from patient quality of life, and VBP15 promises to retain or increase efficacy while reducing side effect profiles,” said Eric Hoffman, CEO of ReveraGen BioPharma. 

“MDA is funding research projects worldwide to help move discoveries from the science lab to families. We look for research that not only works to find treatments and cures, but that also improves the quality of life for those fighting muscle diseases,” said MDA Executive Vice President and Chief Medical and Scientific Officer Valerie A. Cwik. “The goal of funding development of VBP15 is to find a drug that provides the benefits of currently available glucocorticoids, but that doesn't cause the negative side effects associated with these medications.”

Joining Jack CEO Alex Johnson  said, “We are thrilled to see the VBP15 program progress into the clinical stage and look forward to the start of clinical trials in DMD boys later this year.”  Ronald Cohn, head of the The Duchenne Research Fund scientific advisory board added, “We are delighted to be supporting the development of VBP15 and would like to thank the team behind it. The possibility of developing a drug that has the potential to be used instead of glucocorticoids with at least the same benefits, but less side effects is incredibly exciting and we hope that efforts for developing this drug will move forward as swiftly as possible.”

Financial support for the Phase 1 trials was provided by venture philanthropy funding from four non-profit foundations, Muscular Dystrophy Association (USA), Joining Jack (UK), DRF (UK) and Duchenne Children’s Trust (UK).  Foundation to Eradicate Duchenne, Parent Project Muscular Dystrophy and Save Our Sons (Australia) have also provided key financial support for the VBP15 drug development program.

Preclinical development of VBP15 was accomplished through a public/private partnership with researchers from the National Center for Advancing Translational Sciences’ Therapeutics for Rare and Neglected Diseases (TRND) program.

The Phase 1, first-in-human study will evaluate the safety, tolerability and pharmacokinetics of VBP15 in healthy adult volunteers. 

About MDA

The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to saving and improving the lives of people with muscle disease, including muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research to find treatments and cures; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement.

Visit and follow us at and @MDAnews. Learn more about MDA's mission by watching this video.

About Joining Jack

Jack has Duchenne muscular dystrophy, DMD. There is no cure at the moment. We want to find a cure. We're raising money to fund research to find an effective set of treatments to combat this condition. We want to make a difference to this generation of people living with DMD.

About Duchenne Children’s Trust

The Duchenne Children’s Trust was set up by Emily and Nick Crossley, after their son, Eli was diagnosed with Duchenne muscular dystrophy. They are currently co-funding 3 clinical trials, and are funding projects they hope will make a difference to this generation of children and adults living with DMD.

About The Duchenne Research Fund

The Duchenne Research Fund, formerly known as the GM Trust, was established in May 2007 to identify and fund research into finding a cure for this devastating disease.  The Duchenne Research Fund exists to find and fund research that will bring a cure for DMD closer, improving the conditions of those living with DMD along the way. We are committed to looking at any possible route for such a cure and will not stop until a cure is found. We will facilitate and fund new research into minority strains of DMD and focus on the exceptions of the condition with the belief that this will hold the key to a cure.