( RICHMOND Va.--(BUSINESS WIRE)--May 3 ...)Insmed Announces Promising Results From IPLEX Phase II Myotonic,Muscular Dystrophy Clinical Study,Health

Preliminary results of the clinical study, being conducted at the University of Rochester School of Medicine and Dentistry, showed that six months of treatment with doses of IPLEX up to 1 mg/kg/day in six patients met the primary study endpoints of being safe and well tolerated. In addition, IPLEX treatment was associated with improvements in muscle mass, cholesterol and triglycerides. During the six months of treatment, 5 out of 6 patients experienced an improvement in lean muscle mass. Patients also reported improvement in gastrointestinal function, endurance and cognitive function during treatment with IPLEX.

"We are very encouraged by the results from this preliminary study of IPLEX in myotonic dystrophy. The results not only indicate IPLEX was safe, well tolerated and had a positive effect on muscle and lipid metabolism, they also suggest IPLEX may have a positive effect on aspects of the disease that affect patients' daily living," said Dr. Geoffrey Allan, president and CEO of Insmed.

Study Description

The primary objectives of this ongoing open-label, Phase II dose escalation study are to examine the safety and tolerability of once-daily, subcutaneous injection of IPLEX in up to 15 patients with DM1 and to identify the maximum tolerated dose. The next cohort of patients will be treated for six months with a dose of IPLEX which will be titrated up to 2 mg/kg/day.

The study is funded by the National Institutes of Health and the Muscular Dystrophy Association, with supply of IPLEX drug provided by Insmed. A Phase II, placebo-controlled study to further investigate the safety and efficacy of IPLEX in a larger number of patients with DM1 is being des
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