Phase IIa study of VBP for Duchenne muscular dystrophy

DESCRIPTION provided by applicant ReveraGen BioPharma is a clinical stage drug development company that is developing VBP a novel dissociative steroidal class drug The initial indication for development is Duchenne muscular dystrophy where VBP holds promise for retaining or increasing efficacy of glucocorticoids while reducing side effects bone fragilit stunting of growth VBP is currently in Phase I clinical trials in adult volunteers This proposed Phase II SBIR research is to carry out a Phase IIa clinical trial in Duchenne muscular dystrophy boys steroid na ve ages yrs Aim is to test four dose levels of VBP in a multiple ascending dose MAD trial design with two weeks on drug Patients completing the Phase IIa clinical trial will be offered enrollment into a six month extension study The goal is t test pediatric pharmacokinetics tolerability and safety in the Phase IIa clinical trial and assessments of efficacy time to stand velocity and safety change in body mass index in the six month extension study to aid dose selection in the future Phase IIb registration trial Aim i to develop a pharmacodynamics biomarker panel that can assess safety and efficacy markers in acute time frames from peripheral blood We have completed a natural history of DMD serum biomarkers using both SomaScan and proteomics discovery methods and present preliminary data on glucocorticoid associated efficacy and safety biomarkers The Phase II SBIR research will compare VBP biomarkers to glucocorticoid biomarkers as a means of assessing comparative safety and potential efficacy PUBLIC HEALTH RELEVANCE The proposed Small Business Innovation Research application is to carry out a clinical trial of an innovative steroidal drug VBP in young boys with Duchenne muscular dystrophy VBP shows promise in reducing side effects of glucocorticoids while retaining or increasing efficacy A key innovation is the definition of pharmacodynamic biomarkers for both safety and efficacy with likely application to other drug development programs in the muscular dystrophies