Date: 2013-02-06
Type of information: R&D agreement
Compound: utrophin biomarkers
Company: Summit (UK) The Foundation to Eradicate Duchenne Muscular Dystrophy - Children\'s National Medical Center (USA)
Therapeutic area: Genetic diseases - Neuromuscular diseases - Rare diseases
Type agreement: R&D
Action mechanism:
Disease: Duchenne Muscular Dystrophy (DMD)
Details: Summit has entered into a collaboration with Dr Yetrib Hathout from Children\'s National Medical Center in Washington DC, for the development of utrophin biomarkers for DMD. 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. 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. In late 2012, Summit reported that in a Phase 1 trial in healthy volunteers, its lead candidate SMT C1100 was safe and well-tolerated. 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.
This collaboration is part of Summit\'s comprehensive biomarker programme developing a range of assays that will measure biological endpoints to demonstrate muscle benefit after treatment with small molecule utrophin
modulators.
Financial terms:
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