We’re sharing news that ReveraGen BioPharma has licensed an option to develop Vamorolone with Actelion Ltd.

Vamorolone is the first in-human steroid like compound that has shown in animal models to have many of the benefits of corticosteroids, without the many side effects.

Duchenne Research Fund co-funded the successful Phase 1 study of Vamorolone, along with the Duchenne UK and Muscular Dystrophy Association (USA). Actelion’s agreement with ReveraGen means that it will support ReveraGen’s research and development over the next three years, and has the option to obtain the worldwide license for Vamorolone on receipt of the Phase 2b study results.

Media release 3 November 2016:

Vamorolone, a novel compound for the treatment of Duchenne Muscular Dystrophy, holds potential to better preserve muscle function and prolong ambulation for the patient, without some of the side effects associated with glucocorticoid therapy.

Actelion Ltd and ReveraGen BioPharma Inc., a privately held company engaged in the discovery and development of proprietary therapeutic products for neuromuscular and inflammatory diseases, today announced that they have entered into an agreement. By this agreement, Actelion has obtained an exclusive option to in-license ReveraGen’s lead compound vamorolone for the treatment of Duchenne Muscular Dystrophy at two different stages in its development.

Vamorolone is a novel compound that has the potential to preserve muscle function and prolong ambulation, without some of the known side effects associated with corticosteroids currently in use. This is important especially for very young patients with Duchenne, where glucocorticoid therapy is not appropriate due to these side effects, which include growth stunting and immune suppression.

Jean-Paul Clozel, MD and Chief Executive Officer of Actelion, commented: “We have been very impressed by the pioneering work performed by Dr. Hoffman and his team at ReveraGen, and by the encouragement the development of vamorolone receives from the patient community. With our proven scientific, regulatory and marketing expertise in the area of orphan drugs, Actelion is ideally positioned to support the development of this new therapeutic approach, to benefit the boys affected by Duchenne Muscular Dystrophy and help those who care for them.”

Eric Hoffman, Chief Executive Officer of ReveraGen, commented: “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.”

ReveraGen is pursuing parallel clinical development for vamorolone in both the US and Europe. Phase I clinical trials were completed in late 2015, funded through venture philanthropy contracts by organizations including the Duchenne Research Fund, Muscular Dystrophy Association (USA), Joining Jack (UK), and Duchenne Children’s Trust (UK).

A Phase IIa program is currently underway and investigates the safety and efficacy of vamorolone in 4-7 year old steroid-naïve boys with Duchenne (patients that have not taken prednisone or deflazacort). The DMD clinical program is being developed and run by a collaboration between the CINRG group and Newcastle University (Kate Bushby and Michela Guglieri). A Phase IIb program is in the planning stage.

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