Parent Project Muscular Dystrophy (PPMD) is excited to join forces with Foundation to Eradicate Duchenne (FED) to award ReveraGen BioPharma (ReveraGen) a $1 million grant to help fund the chronic toxicology program for VBP15, a combined action drug that addresses multiple levels of Duchenne.
Currently in Phase 1 clinical trials in adult volunteers, VBP15 retains the anti-inflammatory activities of conventional glucocorticoids such as prednisone, but has been chemically modified to gain new membrane stabilization properties, and gain mineralocorticoid receptor antagonist properties relative to traditional steroids. In pre-clinical models of muscular dystrophy, VBP15 did not show side effect profiles such as growth stunting and immune suppression typically seen with commonly used steroidal anti-inflammatories.
VBP15 has moved through the regulatory path and is currently in studies of short-term treatment of adult volunteers (Phase 1 clinical trials). To enable long-term treatment of Duchenne patients, chronic toxicology studies in animals are required. “Chronic toxicology studies were our last hurdle to clear before entering long-term clinical trials in Duchenne”, said Eric Hoffman, CEO ReveraGen. “Chronic toxicology studies require large amounts of drug to carry out, and the cost is a high hurdle for small start-ups, such as ReveraGen,” continued Dr. Hoffman.
Pat Furlong, Founding President of PPMD, congratulated Dr. Hoffman and his team on VBP15. “Dr. Hoffman has been focused on Duchenne for many years now. The work he has led at ReveraGen has the potential to rewrite the progression of Duchenne for those living with the disease now, and those yet to be diagnosed. We are excited to support this project at this critical moment.”
Last year, PPMD initiated an innovative Xcelerate grant competition in recognition of the large number of exciting late stage preclinical and early stage clinical therapeutics in the pipeline for Duchenne. PPMD recognizes that the high costs of some required tests by FDA can slow bringing new drugs to Duchenne patients. To provide a bridge over this gap, PPMD developed the Xcelerate program to provide significant funding at these critical later stages of drug development.
“The Xcelerate program has filled a key niche in the drug development pipeline,” said John Porter, PhD, CEO of PPMD. “As the costs of these later stage projects can escalate, and the investment of PPMD is high, we leverage the international research community to carry out extensive due diligence and vet each project carefully,” continued Dr. Porter. A requirement of consideration of funding by the Xcelerate program is formal review by the TREAT-NMD Advisory Committee for Therapeutics (TACT) (www.treat-nmd.eu/tact).
PPMD will invest $750,000 in the VBP15 chronic toxicology program. An additional $250,000 has been provided by FED. “FED has been an enthusiastic supporter of the VBP15 program since its inception six years ago,” said Joel Wood, President of FED. “The Duchenne community needs a better steroid.”
With the completion of the chronic toxicology studies, Duchenne trials are expected to begin later in 2015.
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