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EspeRare initiates the development of a new therapeutic approach for Duchenne


EspeRare is pleased to announce a collaboration between Duchenne UK, a Duchenne patient organisation, and Professor Dominic Wells at the Royal Veterinary College, to test a potential new anti-inflammatory and anti-fibrotic therapy on the mdx mouse model for Duchenne Muscular Dystrophy (DMD).

Duchenne UK is providing £67,980 to fund an eight-month program that will assess the potential benefits of EspeRare’s ER03 compound in DMD. This preclinical studies will look at the scientific rationale to “re-position” the compound as a treatment for DMD.

This compound has already been tested in several animal models of inflammation and fibrosis. It has also been tested for safety in healthy volunteers, in several Phase I studies as well as in a Phase II study.

Those living with Duchenne lack the muscle protein dystrophin which acts as a ‘shock absorber’ for muscle. Without a shock absorber, everyday use of muscles cause them to become damaged. This damage promotes inflammatory processes which in turn cause hardening and scar formation. Muscles damaged in this way are termed fibrotic. It is hoped that this potential new therapy will be effective in treating both inflammation and fibrosis in the damaged muscles of those living with Duchenne. This would be a treatment for all those living with Duchenne, regardless of their age or DMD gene mutation.

If this research program shows promise, the compound will be able to go straight to a Phase II Proof of Concept study in DMD patients. In line with our commitment to repositioning existing drug and bring medicines to market in an accelerated way, Duchenne UK and EspeRare are committed to further develop this treatment in Phase II, if the compound shows efficacy.

Emily Crossley and Alex Johnson, Co-Founders of Duchenne UK, said; “We are delighted to be able to join forces with Esperare and the Royal Veterinary College, to confirm the efficacy in DMD and advance our knowledge about this compound. By leveraging on the available data package and capitalizing on its safe use in humans in previous studies, we hope that if it shows promise, we could go into patients with DMD as soon as early 2018.

Florence Porte, CSO of EspeRare said: “Thanks to the generosity of Duchenne UK, it has been possible to transform this shelved asset into a potential novel therapeutic option for patients with DMD. This innovative repositioning approach may offer to patients with DMD a new and safe disease modifier addressing inflammation and fibrosis and which may be combined with dystrophin replacing therapies.

For more about Duchenne UK -> link

Duchenne UK news release -> link

For more about Royal Veterinary College -> link