BREAKING NEWS: New drug approved by the FDA for Duchenne muscular dystrophy. Learn more about this new treatment option.

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Vamorolone Will Advance to Phase 2 Testing for Treatment of DMD

Background: 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 that researchers hope will convey 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.

These first-in-patient studies of vamorolone follow the successful completion of a phase 1 trial to test the drug’s safety and tolerability in healthy adult volunteers. A phase 2a multiple ascending dose study 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).

Two MDA Venture Philanthropy (MVP) grants totaling $1.9 million to ReveraGen supported preclinical development and validation of the drug, and another MVP grant for $1,015,200 supported the phase 1 clinical trial to evaluate safety and tolerability in healthy adult volunteers. MVP is the drug development arm that operates within MDA’s Translational Research Program.

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.

Statement from MDA Scientific Program Officer Laura Hagerty, Ph.D.: 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. 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.

MDA is committed to bringing safe and effective treatments and cures to kids and adults with life-threatening neuromuscular diseases as quickly as possible, and 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.  

About MDA
MDA is leading the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America. Learn how you can fund cures, find care and champion the cause at

Claire Orphan
Media Relations Manager


See below to read ReveraGen BioPharma’s Press Release:

ReveraGen BioPharma receives $3M for Phase 2 studies for first-in-patient studies of vamorolone in Duchenne muscular dystrophy

Rockville, MD

The National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health has awarded a $3M grant to ReveraGen BioPharma for the conduct of clinical trials of vamorolone (previously VBP15) in boys with Duchenne muscular dystrophy (DMD).  DMD is caused by loss of the dystrophin protein, leading to progressive muscle damage, weakness, and disability.  Vamorolone slows this muscle damage in mouse models of DMD.

These first-in-patient studies of vamorolone follow the successful completion of Phase I trials in adult volunteers, which showed safety and tolerability of vamorolone.  Phase IIa multiple ascending dose (MAD) studies will be carried out in 4 to <7 year old DMD boys, followed by a six month extension study.  Safety, tolerability, and pharmacokinetics will be studied in the initial two week MAD phase.  Clinical efficacy, safety, and pharmacodynamic biomarkers will be followed in the 6 month extension study, with the goal of dose selection for future clinical trials.

Vamorolone is a first-in-human dissociative steroidal agent that aims to separate safety concerns of traditional glucocorticoid drugs, such as prednisone and deflazacort, from key aspects of efficacy.   In pre-clinical studies in mouse models of muscular dystrophy and inflammatory disease, vamorolone has retained efficacy while losing side effects such as stunting of growth. “Phase I data supported our previous findings in pre-clinical models, including absence of findings to suggest insulin resistance, and reduced suppression of the adrenal axis. The Phase IIa trial will begin to help validate the improved safety profile in DMD boys, and provide insights into an appropriate dose for efficacy studies,” said Eric Hoffman, CEO of ReveraGen, and principal investigator of the NINDS grant. 

“We are grateful for the assistance of the NIH for moving the clinical program forward,” said John McCall, lead chemist and co-founder of the vamorolone program.  “This new NINDS funding follows key partnerships with the NIH NCATS TRND program, and critical financial support by non-profit foundations in the US, UK and Australia”. 

The new funding includes extensive studies of pharmacodynamic safety and efficacy biomarkers. “Our inclusion of biomarkers in the vamorolone program promises to provide acute and objective read-outs of drug activity”, said Kanneboyina Nagaraju, co-founder of ReveraGen.  Phase I clinical trials were supported by the Muscular Dystrophy Association USA, and three United Kingdom foundations (Joining Jack, Duchenne Children’s Trust, and Duchenne Research Fund).  Dr. Paula Clemens from the University of Pittsburgh will lead the Phase IIa studies that will enroll DMD boys at 8 US sites in the Cooperative International Neuromuscular Research Group (CINRG).  “The upcoming Phase II studies have been carefully planned over the last year, with the NIH National Institute of Arthritis and Musculoskeletal (NIAMS) providing a critical funding through a clinical trial planning grant to support the project”, noted Dr. Clemens.  Dr. Clemens is also principal investigator on the NINDS clinical trial grant.  Initial enrollment of 4 to <7 year old steroid-naïve DMD boys is expected in 2Q 2016. 

About ReveraGen BioPharma
ReveraGen is a privately held clinical-stage drug development company focused on neuromuscular disease.  ReveraGen’s lead compound, vamorolone, is a novel anti-inflammatory in development for Duchenne muscular dystrophy (DMD).  

Contact: Eric P Hoffman, CEO.