Pratteln - Santhera Pharmaceuticals (SIX: SANN) announces that partner ReveraGen Biopharma Inc. and their academic collaborators have published new open-label, long-term clinical data on the safety, tolerability and efficacy of vamorolone in patients with Duchenne muscular dystrophy (DMD).
These 18-month treatment data extend previously published 24-week treatment data, and show a reduction of corticosteroid-specific side effects and sustained efficacy with vamorolone including clinical improvement through the 18-month follow-up period.
This publication in the journal PLOS Medicine  provides peer-reviewed and detailed open-label data in patients with DMD treated for 18 months with vamorolone. A multi-center, open-label, 24-week trial (VBP15-003; [2, 3]) with a total 24-month long-term extension (VBP15-LTE; ) was conducted by the Cooperative International Neuromuscular Research Group (CINRG) and evaluated drug-related effects of vamorolone on motor outcomes and corticosteroid-associated safety concerns. This publication covers the 24-week Phase 2a trial (VBP15-003) and the first 12 months of the open-label extension trial (VBP15-LTE) adding up to a total treatment period of 18 months.
'This long-term study showed significant continued clinical improvement of all outcomes measured over an 18-month follow-up period,' said Edward C. Smith, MD, Associate Professor of Pediatrics, Duke University, Durham (North Carolina, USA), clinical investigator and lead-author of the publication. 'Treatment-related efficacy responses with vamorolone were similar to those seen in an external control group with corticosteroid-treated patients. Both 4-stair climb and 10-meter run/walk tests were significantly improved when compared to steroid-naive natural history control subjects.'
'Importantly, we also found that vamorolone did not show stunting of growth seen with deflazacort and prednisone, and vamorolone also showed fewer physician-reported adverse events such as mood disturbance, excessive hair growth, and Cushingoid appearance,' noted Eric Hoffman, PhD, Vice President of Research at ReveraGen BioPharma, Inc. and co-author of the study.
DMD trial participants (4 to
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