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Sarconeos announced as new potential treatment for Duchenne

Posted on: September 11th, 2017 | 0 comments

Biophytis clinical stage drug-candidate Sarconeos demonstrates efficacy in preclinical models of Duchenne muscular dystrophy

An abstract entitled “Sarconeos demonstrates sharp functional improvement and anti-fibrotic properties in an animal model of Duchenne muscular dystrophy” has been selected for presentation at the World Muscle Society international congress being held on 3-7 October 2017 in St Malo, France.

Stanislas Veillet, CEO of Biophytis said: “The abstract highlights exciting data demonstrating Sarconeos’ potential ability to improve muscle function not only in sarcopenia but also in other neuro-muscular diseases, such as Duchenne. Duchenne muscular dystrophy is a debilitating progressive disease with few treatment options for patients. Sarconeos’ oral delivery and safety profile could offer a promising treatment alone or combined with gene therapies when they become available. We are now designing the possible clinical roadmap for this new Orphan indication, which offers a clear regulatory pathand accelerated timelines to market.”

About the Sarconeos study

In the study, mdx mice were treated daily with either Sarconeos for 8 weeks. At completion of the study, mice treated with Sarconeos showed statistically significant improvements in running distances (2.4-fold) when compared to untreated mdx mice. Additionally, the treated mice showed improvements in maximal muscle force of 15% when treated with Sarconeos. Gene expression markers in the heart for fibrosis (CTGF) and hypertrophy (my7, BMP4) were reduced in treated mice, and histopathological analysis revealed a clear decrease of muscle lesion profile, as well as a decrease in fibrosis in the treated mdx animals.

These results demonstrate the efficacy of Sarconeos in the improvement of dystrophic muscle functionality and in the prevention of fibrosis appearance and potentially open a new therapeutic option for people living with Duchenne.

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