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news.Insmed has reported that its investigational drug Iplex has met primary endpoints in a phase II study in patients with myotonic muscular dystrophy.
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Preliminary results of the clinical study showed that six months of treatment with doses of Iplex up to 1mg/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.
“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.
Iplex was approved in the US in December 2005 for the treatment of children with growth failure due to severe primary IGF-I deficiency. The drug is also being investigated for various other indications with unmet medical needs, including retinopathy of prematurity and amyotrophic lateral sclerosis (also known as ALS or Lou Gehrig’s disease) and HIV-associated adipose redistribution syndrome.