Santhera Pharmaceuticals Holding AG and ReveraGen BioPharma, announced that the first patient has been dosed in a Phase 2 pilot study to assess vamorolone in Becker muscular dystrophy (BMD), funded by the U.S. Food and Drug Administration (FDA). This Phase 2 pilot study is a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability and exploratory clinical efficacy on motor function outcomes of vamorolone compared to placebo over a treatment period of 24 weeks in 39 males (aged =18 and <65 years) with BMD (ClinicalTrials.gov id: NCT05166109). Participants will be randomized 2:1 to vamorolone 500 mg (250 mg for body weight <50 kg) daily or placebo.

The clinical trial plans to enroll at sites in Pittsburgh (USA) and Padova (Italy). The study is funded by a USD 1.2 million grant from the FDA under their “Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)” grants program. The grant adds to existing grants from the National Institutes of Health (NIH) - National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), and the Foundation to Eradicate Duchenne to carry out a clinical trial of vamorolone in adults with BMD.