Approximately $2 million award will go toward Phase III development of Iplex as a therapy for myotonic MD.

Insmed received a grant of $2,087,325 from the Muscular Dystrophy Association (MDA). The support will help Insmed advance into Phase III development of its compound to treat myotonic muscular dystrophy (MMD).

Insmed decided to initiate the enabling trial based on results from an ongoing open-label, dose-escalation trial of Iplex™ in MMD. To date, up to 70% of patients have reported improvements in one or more symptoms associated with the disorder, the company points out. Furthermore, patients undergoing a standardized six-minute walk test reportedly improved their walking distance. The funding is expected to cover a substantial portion of the external costs associated with the 24-week late-stage study.

“We are delighted to have a well-respected organization such as the MDA take an active role in the development of Iplex for this important indication,” says Geoffrey Allan, Ph.D., president and CEO. “We believe third-party commitments such as this clearly demonstrate the significance of our results to date and the validity of this approach to treat patients with this severely debilitating disease.”

Iplex was approved in the U.S. in December 2005 for the treatment of children with growth failure due to severe primary IGF-I deficiency. The drug is a complex of recombinant human insulin-like growth factor-I and its predominant binding protein IGFBP-3.

Iplex is also in Phase II investigations as a therapy for ALS and HIV-associated adipose redistribution syndrome as well as a Phase I study in patients with retinopathy of prematurity.

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