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The Children's National Research Institute

Steroid Drug Development and Inflammatory Bowel Diseases

Dissociative Steroid Drug Development

Kanneboyina Nagaraju, D.V.M., Ph.D., and Eric Hoffman, Ph.D., (prior directors of the Center for Genetic Medicine Research) worked with medicinal chemist John McCall, Ph.D., to develop dissociative steroids, a new series of drugs that are able to improve the efficacy and decrease the side effects associated with glucocorticoid drugs. The team created a technology transfer company, ReveraGen BioPharma, Inc. (the first for-profit spin-off from Children’s National) and developed the lead compound VBP15 (Vamorolone). ReveraGen developed the drug for use in patients with Duchenne muscular dystrophy (DMD) in collaboration with NIH Therapeutics for Rare and Neglected Diseases (TRND) and with financial support from five nonprofit foundations: Muscular Dystrophy Association (USA), Joining Jack (UK), DRF (UK), Duchenne Children’s Trust (UK) and Parent Project Muscular Dystrophy (USA). Although VBP15’s origins are in the treatment of DMD, the center, working with ReveraGen, has received NIH (STTR) funding to assess efficacy of vamorolone in asthma, sickle cell disease, rheumatoid arthritis, multiple sclerosis and inflammatory bowel disease (IBD) models.

Phase 1 clinical trials have been successfully completed in about 80 adult volunteers (Hoffman et al. 2018.) Phase 2a studies in DMD children started in July 2016 and are ongoing. ReveraGen has worked with Newcastle University in the United Kingdom to obtain a prestigious European Union grant (Horizons 2020) to support DMD trials. Recently, two papers were published and a U54 Center Grant awarded ($4.3 M from NIH / NICHD- PI, Dr. van den Anker) to develop pharmacodynamic biomarkers for dissociative steroids, as well as other steroids and other anti-inflammatory drugs. These biomarkers will enable investigators to sensitively detect responses to treatment in patients with DMD or (IBD), simply by testing patient blood samples.

Inflammatory Bowel Disease

Inflammatory bowel disease (Crohn’s disease, ulcerative colitis) affects more than 1.4 million Americans, about one in four of whom are children. Glucocorticoids, such as prednisone, remain one of the most effective and commonly prescribed therapies to induce remission in inflammatory bowel disease. However, lasting side effects, such as growth stunting, hypertension and osteoporosis, limit long-term use. ReveraGen has identified a dissociative steroidal compound, vamorolone (formerly VBP15) which is effective in reducing inflammation without major side effects (Damsker et al. 2016). The team also identified pharmacodynamic biomarkers in serum of patients who respond to treatment with both steroid and biologic drugs (Heier et al. 2016). Further clinical studies, funded by a NIH U54 Center Grant, are under way to bridge these blood-based biomarkers to endoscopic and clinical outcomes. These studies are important steps toward the group’s ultimate goals of improving clinical care and developing Vamorolone as an improved alternative to conventional steroid therapy for patients with IBD.