Summit enters biomarker collaboration for rare disease, Duchenne Muscular Dystrophy

Hannah Blake

pharmaphorum

A UK drug discovery company has teamed up with

Dr Yetrib Hathout from the Washington DC Children’s National Medical Center to develop utrophin biomarkers for the rare disease, Duchenne Muscular Dystrophy (DMD).

The company, Summit, is currently advancing therapies for DMD and C. difficile infections. At the end of 2012, Summit reported that in a phase 1 clinical trial of healthy volunteers, its lead candidate SMT C1100 was safe and well-tolerated.

The collaboration is being financially supported by a grant from the Foundation to Eradicate Duchenne and is part of a comprehensive biomarker programme being undertaken by Summit to advance its utrophin modulator programme for DMD.

"We are delighted to be working with Dr Hathout on developing new biomarkers that will help advance our understanding of DMD while supporting the progress of Summit's utrophin modulator programme. Developing biomarker indicators capable of accurately measuring utrophin protein levels in muscle will be vital in helping to confirm the activity of our clinical candidate SMT C1100 in future patient trials. We thank the Foundation to Eradicate Duchenne for their continuing support in advancing this important medical research."

Glyn Edwards, Chief Executive Officer of Summit.

DMD is caused by genetic mutations that prevent patients from making the structural protein dystrophin, which leads to progressive muscle wasting and is ultimately fatal. Summit is pioneering utrophin modulation to stimulate production of utrophin, a functionally similar protein to dystrophin that is expressed in foetal and regenerating muscle, and which has the potential to restore and maintain healthy muscle function. This disease modifying approach would benefit all DMD patients, regardless of the underlying genetic fault causing their illness.

The development of new biomarkers that accurately quantify utrophin protein levels in DMD muscles will play an important role in providing evidence for the potential effectiveness of Summit’s utrophin modulator drugs in future patient clinical trials.

Reference links:

Summit press release