Santhera: Full trial to pave way for a US regulatory submission for Duchenne muscular dystrophy drug

The 48-week 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 in ambulant boys ages 4 to <7 years with DMD. Efficacy outcome measures after the first 24-week treatment period are muscle strength and motor function outcomes with Time to Stand test (TTSTAND) as the primary study endpoint. Additional analyses compare various safety and tolerability markers between the vamorolone dose groups, placebo and prednisone. The second 24-week treatment period where all patients receive one of two vamorolone doses will evaluate the persistence of effect in the longer term. In addition to efficacy, the study aims to confirm the favorable tolerability profile of vamorolone with the potential to offer an alternative to current standard of care. Although glucocorticoids are part of the current care recommendations for DMD, their adverse effect profile limits their use.

With the current number of 121 patients recruited, the VISION-DMD study has reached its pre-planned enrollment target. The last patient is expected to complete the first 24-week treatment period in the first quarter of 2021 with a topline data readout in the second quarter of 2021. Subject to positive results of this first 24-week treatment period, this would pave the way for a regulatory submission to the US FDA in the fourth quarter of 2021.

Vamorolone has been granted Orphan Drug status in the US and in Europe, and has received Fast Track and Rare Pediatric Disease designations from the US FDA and Promising Innovative Medicine (PIM) status from the UK MHRA. On September 2, Santhera signed agreements with ReveraGen and Idorsia that granted Santhera an exclusive license to vamorolone for all indications worldwide.

“We are very pleased having achieved full enrollment of our pivotal trial with vamorolone in patients with Duchenne muscular dystrophy,” said Eric Hoffman, PhD, Vice President of Research at ReveraGen BioPharma. “On behalf of the entire study team we would like to thank patients and their families for their enthusiastic engagement to help advance the clinical development of vamorolone. We also thank investigators and study site personnel for their interest and commitment in completing this study as planned.”

“We congratulate ReveraGen on this accomplishment of fully enrolling the VISION-DMD trial, in times where the conduct of any clinical trial is very challenging for patients and families, study sites and study personnel,” said Kristina Sjöblom Nygren, MD, Chief Medical Officer and Head of Development at Santhera. “It is already clear from existing data from previous studies that vamorolone has tremendous therapeutic potential for patients with Duchenne muscular dystrophy and we are looking forward to the outcome of the 6-month randomized, placebo- and prednisone-controlled period of the VISION-DMD trial.”