Isis Pharmaceuticals announced the start of a Phase I study for its neuromuscular disease drug ISIS-DMPKRx. The initiation of the study entitled the company to a $14 million milestone payment from Biogen Idec under the terms of their collaboration.
ISIS-DMPKRx is an investigational treatment developed using Isis’ antisense technology to reduce the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells, including muscle cells, to treat Myotonic Dystrophy Type 1 (DM1).
C. Frank Bennett, SVP of research at Isis, said, "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 ISIS-DMPKRx.”
Myotonic Dystrophy Type 1 is a rare genetic neuromuscular disease which is estimated to affect around 150,000 patients in the U.S., Japan, and Europe. The hallmarks of the disease include progressive muscular atrophy, muscle spasms, and weakness. The disease is caused by a genetic defect in the DMPK gene, which results in an abnormally long toxic RNA and prevents the production of proteins crucial for normal cellular function. The condition exacerbates from one generation to the next as the genetic error, which increases from one generation to the next, is passed down from the parent to the offspring. Existing disease-modifying therapies typically address only one symptom of DM1 while ISIS-DMPKRx seeks to improve the underlying genetic defect of the disease.
B. Lynne Parshall, COO at Isis, said, “ISIS-DMPKRx has rapidly advanced to the clinic, and we continue to make progress across the board in our drug discovery programs with Biogen Idec.” Parshall said that each milestone helps the company’s neuromuscular disease business to advance into development and commercialization.