Actelion In-Licenses Vamorolone for DMD; Recruitment Open for Phase 2a Clinical Trial

Actelion and ReveraGen BioPharma have entered an agreement in which Actelion has obtained the exclusive option to in-license ReveraGen’s lead compound vamorolone (VBP15) for the treatment of Duchenne muscular dystrophy (DMD) at two different development stages.

Vamorolone is a novel compound with the potential to preserve muscle function and prolong ambulation without some of the side effects commonly associated with corticosteroids that are used now. This is particularly relevant for very young DMD patients, for whom glucocorticoid therapy is not appropriate because of side effects, which include growth stunting and immune suppression.

Vamorolone is the first-in-human steroid-like product that showed evidence of effectively separating a series of sub-properties seen in corticosteroids. Vamorolone has been shown, in animal models, to retain anti-inflammatory effects and new membrane stabilization. Treatment with vamorolone in the earlier stages of DMD potentially could increase preservation of muscle function, in comparison to currently used corticosteroids.

Vamorolone completed Phase 1 clinical trials in late 2015, funded through philanthropist organizations including the Muscular Dystrophy Association (U.S.), Joining Jack (U.K.), Duchenne Research Fund (U.K.) and Duchenne Children`s Trust (U.K.).

With both U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) orphan drug designations, vamorolone is now in a Phase 2a clinical trial (NCT02760277), led by a collaborative research team (CINRG group and Newcastle University).

The Phase 2a clinical trial is now recruiting 48 participants to investigate the safety and effectiveness of vamorolone in four- to seven-year-old steroid-naïve (who have not taken prednisone or deflazacort) DMD boys.

The study will evaluate if it is safe to use vamorolone for more than two weeks in children with DMD, if boys with DMD taking vamorolone improve muscle function in comparison to other boys with DMD in other studies who are not taking any type of steroid, and to see if boys with DMD who take vamorolone gain less weight in comparison to boys with DMD in a prior study who took another type of steroid called prednisone. Study participants will take vamorolone for 24 weeks.

“Duchenne has been the focus of our efforts for many years, and we now have the chance to slow the progression of this devastating disease. The project has already greatly benefited from broad philanthropic support, and we are delighted with the option agreement, which will bring Actelion’s scientific and commercial competencies to the table, enabling vamorolone to rapidly reach patients with Duchenne and their families,” Eric Hoffman, ReveraGen’s chief executive officer, said in a press release.