Wednesday, January 18 at 1:00PM EST
Please join Parent Project Muscular Dystrophy and ReveraGen BioPharma as we host a webinar Wednesday, January 18 at 1:00PM EST to discuss vamorolone (VBP15) clinical trials in Duchenne.
Dr. Eric Hoffman will present a brief history of the vamorolone program, explain current and anticipated clinical trials, and leave time for discussion and fielding of questions.
Please submit questions in advance to firstname.lastname@example.org, with “ReveraGen Webinar” in the subject line by noon on Monday, 1/16.
Vamorolone is a new drug that has five different activities that may prove beneficial to Duchenne boys. It is a potent anti-inflammatory agent (similar to prednisone and deflazacort); It is a mineralocorticoid receptor antagonist, similar to epleronone, and may benefit Duchenne cardiac function; It is a membrane stabilizer, possibly counter-acting dystrophin deficiency at myofiber membranes; It is able to re-synchronize muscle regeneration, likely improving it.
These multiple aspects of efficacy must also be balanced with safety. With regards to side effects, vamorolone does not show many of the side effects seen with prednisone and deflazacort. Through blood biomarkers in adult volunteers, vamorolone does not show evidence of bone side effect, or metabolic side effects. In mouse studies, vamorolone did not show the stunting of growth seen in prednisolone-treated mice.