Isis Pharmaceuticals ISIS starts DMPK-Rx Phase 1 study for the treatment of myotonic dystrophy type 1

Isis Pharmaceuticals ISIS starts DMPK-Rx Phase 1 study for the treatment of myotonic dystrophy type 1 -

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced aujourd 'hui that it has launched a Phase 1 study for ISIS-DMPK _Rx . Isis received a payment of $ 14 million milestone from Biogen Idec associated with this achievement. ISIS-DMPK _Rx is designed to reduce the production of toxic substances myotonic protein kinase dystrophia (DMPK) RNA in cells, including muscle cells, for the treatment of myotonic dystrophy type 1 (DM1 ).

"ISIS-DMPK _Rx is an example of the broad applicability of our antisense technology to develop new drugs to treat patients with serious diseases and rare. ISIS-DMPK _Rx is the first drug to enter our pipeline that is designed to target a toxic RNA, the first drug administered systemically to get into developing our partnerships Biogen Idec and second generation 2.5 drug to enter development clinic, "said C. Frank Bennett, Ph .D., Vice President of research at Isis." myotonic dystrophy represents an ideal opportunity for antisense gene that causes the disease produces a toxic RNA, which are not accessible through traditional therapeutic approaches, but is only accessible with our antisense technology. We look forward to rapidly advancing the development of ISIS-DMPK _Rx . "

" Our collaboration with Biogen Idec was very productive. ISIS-DMPK _Rx quickly advanced to the clinic, and we continue to make progress on the board in our discovery programs drugs with Biogen Idec. these successes greatly advance our franchise neuromuscular disease and result in the potential for significant revenue that our medicines and progress of programs, "said B. Lynne Parshall, COO at Isis.

DM1 is a rare genetic neuromuscular disease characterized by muscle atrophy, weakness and progressive muscle spasms. DM1, the most common form of muscular dystrophy in adults, affects approximately 150,000 patients in the US , Europe and Japan. the DM1 patients have a genetic deficiency in their DMPK gene in which a sequence of three nucleotides repeat abundantly, creating an inordinately long time to toxic RNA, which accumulates in the nucleus of cells and prevents the production of proteins necessary for normal cell function. The number of triplet repeats increases from one generation to another, resulting in the possibility of a more serious disease in each subsequent generation. There are currently no disease-modifying treatment that treat more than one symptom of the disease. ISIS-DMPK _Rx is designed to enhance the underlying genetic defect that causes DM1.

"Myotonic dystrophy is a progressive and debilitating disease that affects thousands of patients for whom there is no direct treatment options. Innovative science behind ISIS-DMPK _Rx is convincing and target the underlying genetic defect that causes myotonic dystrophy, "said Molly White, executive director of the Foundation of myotonic dystrophy. "ISIS DMPK _Rx has a chance to fill the vacuum therapy for DM1 patients and transform the hopes and the future of thousands of patients and families."