November 10, 2016
Cambridge, MA

Solid Adds Simvastatin Research Program To Therapeutic Pipeline

Grant from Duchenne UK to fund research to better understand the potential benefits of statins in patients with Duchenne muscular dystrophy (DMD).

Solid continues to grow its therapeutic pipeline with multiple candidates that have the potential to address the different facets of DMD. To this end, the company is excited to enter into a new research collaboration with Stanley Froehner, Ph.D., and Nick Whitehead, Ph.D., of the University of Washington to study the possible benefits of Simvastatin and other statins in patients with DMD. The research will be funded in part by a new $90,000 grant from Duchenne UK, a leading charity in the United Kingdom.

In Duchenne muscular dystrophy, muscle cells come under significant stress, which leads to cell death, chronic inflammation and fibrosis. Statins are a class of molecules approved in many countries for their known benefits in heart disease. Recent preclinical studies have shown that statins may also have anti-oxidative and anti-inflammatory effects, which have the potential to address the hallmarks of DMD disease progression.

The Solid statin program is spearheaded by preclinical studies by Drs. Froehner and Whitehead on Simvastatin, a common, readily-available statin. In early preclinical models of DMD, short-term administration of Simvastatin resulted in reduced skeletal muscle damage and enhanced muscle function.  Long-term administration in the same models showed an improvement of overall muscle health.

“The Simvastatin program is another example of our commitment to leaving no stone unturned in our search to find effective therapies for DMD,” said Gilad Hayeem, president of Solid Biosciences. “The program could not have been possible without the generous grant from Duchenne UK, one of our fearless advocacy partners in the fight to beat this disease.”

Solid and its collaborators at the University of Washington are currently performing several preclinical studies to better characterize the potential risks and benefits of statins and inform the optimal molecule to move into clinical studies for DMD.




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