Isis testing safety of a new myotonic muscular dystrophy drug that targets toxic RNA

US Isis Pharmaceuticals Inc (NASDAQ:ISIS) said yesterday it had started a Phase I trial of ISIS-DMPKRx, designed to lower the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells, including muscle cells, as a treatment for myotonic dystrophy type 1 (DM1).

The company will get a milestone payment of USD 14 million (EUR 10.3m) from Biogen Idec for reaching this milestone.

C. Frank Bennett, Isis senior vice president of research, said that ISIS-DMPK Rx is the first compound in the company's pipeline designed to target a toxic RNA, the first systemically administered drug to go into development from the partnerships with Biogen Idec and the second generation 2.5 drug to start clinical development.

DM1, affecting some 150,000 people in the United States, Europe and Japan, is a rare genetic neuromuscular disease distinguished by progressive muscle atrophy, weakness and muscle spasms. DM1 is the most common form of muscular dystrophy in adults.

DM1 patients have a genetic defect in their DMPK gene in which a sequence of three nucleotides repeats extensively, resulting in an abnormally long toxic RNA, which accumulates in the nucleus of cells and prevents the production of proteins necessary for normal cellular function. ISIS-DMPKRx is designed to alleviate the underlying genetic defect causing DM1.

(USD 1.0 = EUR 0.736)

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Isis testing safety of a new myotonic muscular dystrophy drug that targets toxic RNA