International Consortium Provides $2M Funding for Phase 1 Clinical Trial of ReveraGen DMD Drug July 17, 2014

Washington, DC - A novel anti-inflammatory in development for the chronic treatment of Duchenne muscular dystrophy by ReveraGen BioPharma,  a private spin-off company from Children’s National Health System, has received $2 million from four nonprofit organizations.

The Muscular Dystrophy Association,  based in Chicago, Ill, and three British organizations, the Joining Jack Foundation, The Duchenne Research Fund,  and the Duchenne Children’s Trust, provided Phase 1 clinical trial funding for the first-in-human studies of VBP15.

The funding is slated for the Phase 1 clinical trial funding for the lead compound known as VBP15, an oral drug that has shown efficacy in animal models of Duchenne muscular dystrophy (DMD) through anti-inflammatory and membrane stabilization.  ReveraGen, based in Silver Spring, MD, is a privately held drug development company engaged in the discovery and development of proprietary therapeutic products for neuromuscular and inflammatory diseases.

DMD, characterized by progressive muscle degeneration and weakness, affects about one in every 5,000 boys with over 50,000 patients worldwide.  There are currently no approved drugs for DMD, although daily prednisone is often used off label with significant side effects, such as stunting of growth, mood changes, bone fragility, and weight gain that detract from a patient’s quality of life.

While VBP15 shares some beneficial aspects of the glucocorticoid (corticosteroid) drug prednisone, it has fewer significant side effects and has shown improved efficacy in pre-clinical trials. In studies, VBP15 reduced inflammation and stabilized cell membranes, both of which are believed to be relevant to treating DMD, a disease in which muscle fiber membranes are unstable.

“We are delighted to continue to develop VBP15 with the critical assistance of nonprofit organizations,” said Eric Hoffman, PhD, a ReveraGen co-founder and geneticist at Children’s National.

“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,” said ReveraGen clinical consultant and former Chief Medical Officer Ed Connor.

“VBP15 is being developed for all DMD children, regardless of the type of gene mutation, and the drug shows efficacy in pre-clinical models of other types of neuromuscular disease and chronic inflammatory states,” said ReveraGen co-founder Kanneboyina Nagaraju.  

John McCall, a medicinal chemist and ReveraGen co-founder noted, “We have a good chemistry here, and I am thrilled we are preparing to move into clinical trials.”

MDA Executive Vice President and Chief Medical and Scientific Officer Valerie Cwik, MD, said, “The Phase 1 clinical trial funding is MDA’s third venture philanthropy investment in the VBP15 program, which shows the success of the drug in meeting all milestones, and our continued enthusiasm about the therapeutic potential of this compound.”  

Joining Jack CEO Alex Johnson noted, “As a new foundation, we are focused on potential therapies for DMD moving into clinical trials, and support of drug developers using innovative approaches to speed the drug approval process; VBP15 and ReveraGen was a good fit with our model.”

Emily Crossley from the Duchenne Children’s Trust said, “This project fits with our mission to fund translational research. We are delighted to be supporting VBP15, and look forward to the potential this compound has to treat all patients with DMD.”

Doron Rosenfeld from the Duchenne Research Fund added “We are very excited to be working with ReveraGen on this ground-breaking project, alongside Joining Jack, the Duchenne Children’s Trust and the MDA. We hope and pray that through our combined efforts the VBP15 program will readily present itself as an effective, viable and preferable alternative to existing DMD therapies.”

The $2 million  funding is under a coordinated venture philanthropy model, providing return on investment based upon future drug sales.

About ReveraGen BioPharma

ReveraGen is a privately held drug development company engaged in the discovery and development of proprietary therapeutic products for neuromuscular and inflammatory diseases. We are pursuing small molecule drug development for selected indications. ReveraGen’s lead compound, VBP15, is a novel anti-inflammatory currently in development for the chronic treatment of DMD. Additional indications and second-generation product candidates are in the pipeline.

About MDA

The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to saving and improving the lives of anyone 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 mda.org and follow us at facebook.com/MDAnational and @MDAnews. Learn more about MDA's mission by watching this video.

About Joining Jack Foundation

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 Crossleyafter their son, Eli, was diagnosed with Duchenne Muscular Dystrophy.  Their mission is to help the global fight to bring treatments to market as quickly as possible, by funding translational research that will bring potential drugs out of the laboratory and into the clinic.

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. www.duchenne.org.uk


About Children’s National Health System

Children’s National Health System, based in Washington, DC, has been serving the nation’s children since 1870. Children’s National’s hospital is Magnet® designated, and is consistently ranked among the top pediatric hospitals by U.S.News & World Report. Home to the Children’s Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children’s National is one of the nation’s top NIH-funded pediatric institutions. With a community-based pediatric network, eight regional outpatient centers, an ambulatory surgery center, two emergency rooms, an acute care hospital, and collaborations throughout the region, Children’s National is recognized for its expertise and innovation in pediatric care and as an advocate for all children. For more information, visit ChildrensNational.org, or follow us on Facebook and Twitter.

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