Ionis Launches Phase 1 Trial of IONIS-DMPK Rx to Treat DM1

Ionis Pharmaceuticals, Inc. (formerly Isis Pharmaceuticals, Inc.) announced today that it has launched a Phase 1 clinical trial for IONIS-DMPKRX. Ionis earned a $14 million milestone payment from Biogen Idec associated with this achievement. IONIS-DMPKRX is designed to reduce the production of toxic dystrophia myotonic-protein kinase (DMPK) RNA in cells, including muscle cells, for the treatment of Myotonic Dystrophy Type 1 (DM1).

"[IONIS]-DMPKRX is an example of the broad applicability of our antisense technology to develop novel drugs to treat patients with severe and rare disease. IONIS-DMPKRX is the first drug to enter our pipeline that is designed to target a toxic RNA, the first systemically administered drug to enter development from our Biogen Idec partnership and the second generation 2.5 drug to enter clinical development," said C. Frank Bennett, Ph.D., senior vice president of research at Isis.  "Myotonic dystrophy represents an ideal opportunity for antisense as the disease-causing gene produces a toxic RNA, which is not accessible by traditional therapeutic approaches but is uniquely accessible with our antisense technology. We look forward to rapidly advancing the development of IONIS-DMPKRX."

"Our collaboration with Biogen Idec has been very productive. [IONIS]-DMPKRX has rapidly advanced to the clinic, and we continue to make progress across the board in our drug discovery programs with Biogen Idec. All of these successes advance our neuromuscular disease franchise and translate into the potential for significant revenue as our drugs and programs progress," said B. Lynne Parshall, chief operating officer at Isis.

DM1 is a rare genetic neuromuscular disease characterized by progressive muscle atrophy, weakness and muscle spasms. DM1, the most common form of muscular dystrophy in adults, affects approximately 150,000 patients in the US, Europe and Japan. Patients with DM1 have a genetic defect in their DMPK gene in which a sequence of three nucleotides repeats extensively, creating an abnormally long toxic RNA, which accumulates in the nucleus of cells and prevents the production of proteins needed for normal cellular function. The number of triplet repeats increases from one generation to the next, resulting in the possibility of more severe disease in each subsequent generation. There are currently no disease-modifying therapies that address more than one symptom of the disease. IONIS-DMPKRX is designed to improve the underlying genetic defect that causes DM1.

"Myotonic dystrophy is a progressive and debilitating disease that affects thousands of patients for whom there are no direct therapeutic options. The innovative science behind IONIS-DMPKRX is compelling and targets the underlying genetic defect that causes myotonic dystrophy," said Molly White, executive director of MDF. "IONIS-DMPKRX has a chance to fill the therapeutic void for DM1 patients and transform the hopes and futures of thousands of patients and families."


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