VBP15 for the treatment of DMD
ReveraGen BioPharma, Inc. USA Non-confidential report
VBP15 is a novel glucocorticoid derivative with NF-kB inhibition and membrane stabilizing effects but without GRE-transcriptional effects thought to yield the majority of side effects currently experienced by DMD boys currently treated with corticosteroids. ReveraGen presented extensive, high quality preclinical data suggesting good efficacy and low toxicity in the mdx mouse model of muscular dystrophy. Additional understanding of the PK profile of the compound as related to toxicity will be important. A comprehensive plan for clinical development was also considered to be of high quality. TACT recommended ReveraGen consider simplifying the clinical development program to facilitate recruitment and to shorten the timeline to regulatory approval.
Name of applicant: Erica Reeves Reviewed: October, 2012 in Prague, Czech Republic
VBP15 is a novel glucocorticoid derivative with NF-kB inhibition and membrane stabilizing effects but without GRE-transcriptional effects thought to yield the majority of side effects currently experienced by DMD boys currently treated with corticosteroids. ReveraGen presented extensive, high quality preclinical data suggesting good efficacy and low toxicity in the mdx mouse model of muscular dystrophy. Additional understanding of the PK profile of the compound as related to toxicity will be important. A comprehensive plan for clinical development was also considered to be of high quality. TACT recommended ReveraGen consider simplifying the clinical development program to facilitate recruitment and to shorten the timeline to regulatory approval.
