ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS) announced that it has earned a $2.8 million milestone payment from Biogen related to the advancement of the ongoing Phase 1/2a study of ISIS-DMPK-2.5Rx in patients with myotonic dystrophy type I (DM1).

“Our collaboration with Biogen has been very productive.  ISIS-DMPK-2.5Rx has rapidly advanced into patients with DM1, and we continue to make progress across the board in our drug discovery programs with Biogen.  Progress like this advances our severe and rare disease pipeline for neurological disorders, which may, as our programs advance, translate into the potential for significant revenue.  Together with Biogen, we have advanced four drugs into the pipeline and have other targets in late-stage research.  To date and including the $2.8 million earned today, we have generated nearly $27 millionfrom Biogen related to the advancement of ISIS-DMPK-2.5Rx,” said B. Lynne Parshall, chief operating officer at Isis Pharmaceuticals.

ISIS-DMPK-2.5Rx is designed to reduce the production of the toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells, including muscle cells, for the potential treatment of DM1.  ISIS-DMPK-2.5Rx is being evaluated in a randomized, placebo-controlled, dose-escalation Phase 1/2a study in patients with DM1.   For further study information, please visit and search for ISIS-DMPKRx or by the study number: NCT02312011.

DM1 is a debilitating rare genetic neuromuscular disease characterized by progressive muscle atrophy painful muscle spasms, cataracts, heart conduction defects, endocrine abnormalities and myotonia.  DM1, the most common form of muscular dystrophy in adults, affects approximately 150,000 patients in the United States, Europe and Japan. There are currently no disease-modifying therapies. ISIS-DMPK-2.5Rx is the only program in clinical testing that is designed to address the underlying genetic defect that causes DM1.  ISIS-DMPK-2.5Rx is a product of Isis’ antisense technology, a unique drug discovery and development platform that can directly address the genetic cause of diseases, like DM1, that have been largely untreatable with current therapeutic approaches.

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 that accumulates in the nucleus of cells and prevents the production of proteins needed for normal cellular function.  The number of triplet repeats can increase from one generation to the next, with the devastating consequences of more severe disease in each subsequent generation. (Original Source)

Shares of Isis Pharmaceuticals closed yesterday at $62.77. ISIS has a 1-year high of $77.80 and a 1-year low of $37.38. The stock’s 50-day moving average is $45.93 and its 200-day moving average is $54.56.

On the ratings front, Piper Jaffray analyst Joshua Schimmer upgraded ISIS to Buy, with a price target of $80, in a report issued on November 5. The current price target implies an upside of 27.4% from current levels. According to, Schimmer has a total average return of -2.6%, a 38.9% success rate, and is ranked #3457 out of 3832 analysts.

Isis Pharmaceuticals Inc is engaged in antisense drug discovery and development, exploiting a novel drug discovery platform it created to generate a broad pipeline of first-in-class drugs.