Ryan’s Quest Clinical Trial Grant for Steroid Alternative
Hamilton, New Jersey, August 3, 2015—The Ryan’s Quest Foundation, a non-profit organization specific to Duchenne muscular dystrophy, announced today the award of their most recent contribution toward the fight to end Duchenne muscular dystrophy. The Foundation has awarded ReveraGen BioPharma $50,000 towards a clinical trial for a potential steroid alternative. The grant was facilitated through a newly formed partnership of Duchenne Foundations, the Duchenne Alliance.
VBP15 (Verolone) is a first-in-human clinical stage drug being developed for Duchenne muscular dystrophy. Verolone is structurally related to steroids (specifically glucocorticoids), but has been chemically altered to change a series of properties that may show optimization for Duchenne muscular dystrophy and other chronic inflammatory disorders. Specifically, in a large series of pre-clinical studies (in vitro and animal), Verolone:
Retains anti-inflammatory (transrepression) activity.
Decreases side effects due to transactivation activity.
Due to apparent loss of many side effects, the dose may be increased, potentially improving efficacy.
Changes the mineralocorticoid binding activity from agonist (prednisone) to antagonist (Verolone), possibly improving heart function in DMD (like epleronone)
Changes the effect on muscle cell membranes, from a destabilizing activity (prednisone) to a stabilizing activity (Verolone), possibly counteracting dystrophin deficiency
Verolone is currently midway through Phase 1 clinical trials in adult volunteers, and these trials will be completed in October 2015. Clinical trials in DMD boys are anticipated to begin in early 2016.
Verolone has been extensively peer reviewed and key aspects of the program independently validated. The pre-clinical and clinical development of Verolone has been supported by government agencies and foundations working with ReveraGen BioPharma, Children’s National Medical Center in Washington DC, Cooperative International Neuromuscular Research Group, The John Walton Neuromuscular Center at Newcastle University, and the co-inventors (Drs. John McCall, Kanneboyina Nagaraju, and Eric Hoffman).
The Duchenne Alliance Research Fund (DARF) is actively raising funds to support the clinical development program of Verolone. The following foundations are contributing to the DARF fundraising effort: Save Our Sons, Michael’s Cause, Pietro’s Fight, Alex’s Wish,
Ryan’s Quest, Duchenne Now, Hope for a Future, and Hope for Gus. DARF invites other foundations to join these efforts.
Ryan’s Quest co-founder, David Schultz explains, ”We are cautiously optimistic that trials such as Verolone will provide a promising treatment and better quality of life in Duchenne muscular dystrophy in the near future”.
The Ryan’s Quest Foundation was founded by Hamilton, New Jersey residents David and Maria Schultz on behalf of their son Ryan and thousands of other young men diagnosed with Duchenne muscular dystrophy, a life limiting disorder for which there is no cure. Ryan’s Quest is dedicated to finding a treatment that will ensure the survival and quality of life for this generation of affected young men and boys.
Duchenne muscular dystrophy occurs in approximately 1 out of every 3,600 male births. It is considered one of the most common and severe forms of muscular dystrophy. Considered a genetic disorder, approximately 35% of all cases appear to be by spontaneous mutation. Duchenne is typically diagnosed between 3 and 7 years of age, with the mean year of diagnosis around the age of 5. Duchenne follows a predictable progressive clinical course of muscle weakness. Loss of ambulation occurs by the age of 12 and death generally in early adulthood secondary to respiratory or cardiac failure.
Contact: David Schultz
One hundred percent of funds raised through The Ryan’s Quest Foundation are directed towards research for a treatment and cure of Duchenne muscular dystrophy. To support the efforts of The Ryan’s Quest Foundation, please visit www.ryansquest.org.