PPMD is thrilled to see the continued progress of VBP-15, now known as Vamorolone. Progress to date includes a first in-human study of healthy volunteers and animal data which suggests it has none of the side effects of steroids, does not impact linear growth, and does not promote insulin resistance. NIH has awarded $3 million for Vamorolone to continue clinical studies in boys 4 -7 years of age. PPMD is proud to be an early funder of Vamorolone and are hopeful that this compound will one day prove to be an alternative option for steroids.
Read the Press Release:
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. firstname.lastname@example.org
Learn more by about about this project by watching the recording of Dr. Hoffman's presentation from our 2015 Connect Conference: