Biogen Idec, Isis Forge 2nd Alliance This Year for Rare Disease

Xconomy Boston — 

Biogen Idec and Isis Pharmaceuticals are apparently warming up to each other as partners, as they are doubling down on their efforts to treat rare muscular disorders.

The Weston, MA-based biotech giant (NASDAQ: BIIB) is announcing today that it has formed a new partnership with Carlsbad, CA-based Isis (NASDAQ: ISIS) to develop a treatment for myotonic dystrophy type 1 (DM1), sometimes called Steinert disease. Under the deal, Isis gets a $12 million upfront fee, and will be eligible for another $259 million in milestone payments, plus a double-digit percentage royalty if the companies are able to develop a marketable product for the disease. Isis will be responsible for development costs through mid-stage clinical trials, and Biogen will pick up the responsibility if it exercises its option to license the drug.

Biogen and Isis showed their mutual interest in genetic neuromuscular disorders in January, when they formed a collaboration around a drug for spinal muscular atrophy. Now the companies are building on that relationship, and expanding into myotonic dystrophy type 1. The condition, caused by a genetic defect, leads to muscle atrophy, weakness and spasms. An estimated 150,000 people in the U.S., Europe and Japan suffer from the condition, the companies said today in a statement. There are no treatments today that can slow down the progression of the disease.

Isis scientists are wagering they can fight myotonic dystrophy type 1 now that scientists have identified a gene that contains three individual DNA bases that repeat extensively, which leads to the production of long, toxic RNA molecules that accumulate in cells and cause the muscle problems. By creating an antisense molecule, Isis and Biogen are hoping to target and get rid of the toxic RNA.

“Myotonic dystrophy has an identifiable genetic cause, the program fits with our mission to bring innovative therapies to patients with serious neurologic diseases, and Isis’ antisense compound has the potential to make a real difference,” said Steve Holtzman, Biogen’s executive vice president of corporate development, in a statement. “This collaboration, which is our second with Isis, reflects the tremendous respect we have for their scientific leadership and expertise in antisense technology.”