SUMMIT PLC OUTLINES CLINICAL TRIAL PLANS FOR ITS DUCHENNE MUSCULAR DYSTROPHY PROGRAMME
Summit, a UK drug discovery company, today outlines its clinical trial plans for SMT C1100, a potential first-in-class disease modifying drug for the treatment of the fatal rare disease Duchenne Muscular Dystrophy.
DMD is a neuromuscular disease and is caused by the absence of dystrophin, a protein which is essential in maintaining the healthy function of muscles in the body. SMT C1100 is a small molecule that works by producing a naturally occurring protein called utrophin to substitute for the missing dystrophin. This is the only approach in development that continually makes new utrophin and has the potential to treat all DMD patients, regardless of their specific genetic mutation. A drug to treat DMD has the potential to generate annual sales in excess of $1 billion.
SMT C1100 has been extensively evaluated in non-clinical efficacy and safety studies and has demonstrated its ability to restore and maintain the function of muscles. A Phase I clinical trial in healthy volunteers will now be conducted by Summit. The trial will evaluate if the new formulation of SMT C1100 can provide consistent levels of the drug in the blood that non-clinical efficacy studies predicted would be required to confer therapeutic benefit in DMD patients, while also further assessing its safety. The new formulation will be appropriate for use by all DMD patients.
The manufacture and formulation of SMT C1100 is currently on-track and Summit expects to submit a clinical trial application (‘CTA’) to the Medicines and Healthcare products Regulatory Agency (‘MHRA’) in Q1 2012. If CTA approval is granted, the Phase I trial would commence with headline results from this study anticipated in Q3 2012. A successful outcome from the Phase I trial could lead to a Phase II study in DMD patients starting in H1 2013.
The Phase I clinical trial is completely funded by the $1.5 million agreement signed in December 2011 between Summit and the Muscular Dystrophy Association, Parent Project Muscular Dystrophy, Charley’s Fund, Cure Duchenne, the Foundation to Eradicate Duchenne and the Nash Avery Foundation.
Barry Price, PhD, Executive Chairman of Summit commented: “Summit is pleased to report that our plans to commence a new Phase I clinical trial for SMT C1100 are progressing well following the recent agreement signed with the DMD organisations. This clinical trial represents a key development milestone for the programme with a successful outcome having the potential to add considerable value to this asset, and bring an urgently needed treatment for this terrible disease a step closer.”
Valerie Cwik, M.D., MDA Interim President and Medical Director added: “We at MDA are excited about the continuing progress in the development of SMT C1100 as a therapy for DMD. MDA has supported utrophin upregulation as a therapeutic strategy for DMD for several years and is pleased to partner with Summit in the development of this promising drug.”