Media Release
June 1, 2021
Santhera and ReveraGen Announce Positive and Statistically Highly Significant Topline Results with Vamorolone in Pivotal VISION-DMD Study
Vamorolone demonstrates strong efficacy across primary and secondary endpoints in both doses of 6 mg/kg/day and 2 mg/kg/day
Primary endpoint Time to Stand [TTSTAND] velocity is met for vamorolone 6 mg/kg/day versus placebo [p=0.002]
Secondary endpoints for Six-Minute Walk [6MWT] and Time to Run/Walk 10 meters [TTRW] tests achieve statistical significance versus placebo
Study confirmed good safety and tolerability profile of vamorolone for both doses
Santhera plans to file a New Drug Application [NDA] with the US FDA in Q1-2022, requesting priority review
Pratteln, Switzerland, and Rockville, MD, USA, June 1, 2021 – Santhera Pharmaceuticals [SIX: SANN] and ReveraGen BioPharma, Inc [US: private] announce positive key results from the VISION-DMD study, demonstrating robust efficacy across multiple efficacy endpoints and favorable safety and tolerability of vamorolone in the treatment of patients with DMD.
VISION-DMD is a pivotal Phase 2b study designed to demonstrate efficacy and safety of vamorolone compared to placebo and prednisone [active control] in the treatment of DMD. In the first 24-week double-blind period, of which the topline data readout is presented here, 121 ambulant boys aged 4 to