Pratteln, Switzerland, June 2, 2020 – Santhera Pharmaceuticals (SIX: SANN) announces that partner ReveraGen Biopharma Inc. has completed a long-term, open-label extension study of 24 months duration with vamorolone in patients with Duchenne muscular dystrophy (DMD). Including 6 months treatment in the preceding study, ReveraGen has now obtained safety and efficacy data with vamorolone over a period of 2.5 years in 41 boys with DMD.
Eligible for enrolment into the now completed 24-month long-term, open-label extension study (VBP15-LTE, clinicaltrials.gov ID: NCT03038399) were boys who had previously completed the 6-month dose escalation study VBP15-003 (clinicaltrials.gov ID: NCT02760277). Data from this VBP15-003 study in comparison to natural history study data demonstrated dose-dependent improvement in timed function tests. Vamorolone was reported to be safe and well tolerated up to the highest dose tested (6.0 mg/kg/day).
“With most participants continuing treatment with vamorolone long-term, we have assembled a solid safety database, with 106 patient-years of vamorolone exposure in DMD boys, with no serious adverse events attributable to vamorolone to date,” said Eric Hoffman, PhD, Vice President of Research at ReveraGen BioPharma. “We will now analyze the efficacy data and plan to report the results in upcoming scientific conferences and publications.”
All 46 patients who completed the VBP15-003 study requested to continue vamorolone treatment in the long-term extension, rather than transition to corticosteroids. This VBP15-LTE study enabled dose escalation and de-escalation at the preference of the physician and family (suggested range 2.0 to 6.0 mg/kg/day). Of the 41 participants completing end-of-study visit after 24 months, 27 ended at 6.0 mg/kg/day (66%), 11 at 2.0 mg/kg/day (27%), and 3 at 4.0 mg/kg/day (7%). Thus, the majority of physicians/families chose treatment at the highest tested dose of vamorolone by the end of the VBP15-LTE study. Upon their expressed wish, the large majority of the boys completing the 2-year VBP15-LTE study have transitioned to Expanded Access Programs (USA, Canada, Israel) or compassionate use programs (UK, Sweden, Australia) to receive continued vamorolone treatment.
The currently ongoing Phase 2b VISION-DMD study (VBP15-004; clinicaltrials.gov: NCT03439670) is designed as a pivotal trial to demonstrate efficacy and safety of vamorolone administered orally at doses of 2.0 mg/kg/day and 6.0 mg/kg/day versus prednisone 0.75 mg/kg/day and placebo over a treatment period of 24 weeks. The study is currently being conducted at 33 sites across North America, Europe, Israel and Australia.
Vamorolone has been granted Orphan Drug status in the US and in Europe, and has received Fast Track and Rare Pediatric Disease designations by the US FDA. In November 2018, Santhera acquired from Idorsia Pharmaceuticals Ltd (SIX: IDIA), who has an option to an exclusive, worldwide license to vamorolone, the option to an exclusive sub-license to vamorolone in all indications and all countries worldwide, except Japan and South Korea.