Biogen begins ISIS-SOD1 Rx Phase 1/2 clinical study in patients with amyotrophic lateral sclerosis

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today announced that its partner, Biogen, has initiated a Phase 1/2 clinical study of ISIS-SOD1_Rx (BIIB067) in patients with amyotrophic lateral sclerosis (ALS). ISIS-SOD1_Rx is part of Isis' strategic collaboration with Biogen to discover and develop antisense drugs to treat neurological diseases. ISIS-SOD1_Rx, previously referred to as ISIS-BIIB3_Rx, is a Gen. 2.0+ antisense drug designed to reduce the production of superoxide dismutase 1 (SOD1). A mutation in the SOD-1 gene results in an inherited form of ALS, referred to as SOD1-ALS. SOD1-ALS is the second most common familial form of ALS, accounting for up to 20 percent of familial ALS. Familial ALS represents approximately 10 percent of all cases of ALS. Currently, treatment options for patients with ALS are extremely limited with no drugs that significantly slow disease progression.

"There is substantial evidence that mutations in the SOD1 gene are responsible for a toxic gain of function that can lead to progressive loss of motor neurons in patients with SOD1-ALS. As a result, patients with SOD1-ALS experience muscle weakness, loss of movement, difficulty in breathing and swallowing and eventually succumb to their disease. Our antisense technology provides us with the unique ability to act at the genetic level to reduce the production of the SOD1 protein, including the mutant SOD1 protein that is believed to be the cause of the disease," said C. Frank Bennett, Ph.D., senior vice president of research at Isis Pharmaceuticals. "We have previously demonstrated that we can safely administer an antisense drug in patients with familial ALS by intrathecal injection. In the study initiated today, we are evaluating a more potent antisense drug designed to directly reduce the production of the SOD1 protein. This approach has the potential to provide therapeutic benefit to patients with SOD1-ALS by slowing or even halting progression of this fatal disease."

"Together with Biogen, we have made significant progress developing antisense drugs to treat neurological and neuromuscular disorders. The advancement of ISIS-SOD1_Rx into clinical development is an important milestone for this collaboration and reflects Biogen's and our commitment to developing drugs for patients with ALS. The advancement of this program is also another example of the broad applicability of our technology to address diseases that are unapproachable by other therapeutic modalities," said B. Lynne Parshall, chief operating officer of Isis Pharmaceuticals.

ALS is a rare, fatal neurodegenerative disorder. Patients with ALS suffer progressive degeneration of the motor neurons, which results in a declining quality of life and ultimately death. ISIS-SOD1_Rx, which targets the best understood genetic cause of familial ALS, is the first antisense drug to treat ALS to enter clinical development under the strategic collaboration between Isis and Biogen. As the study progresses, Isis has the opportunity to earn a $2.5 million milestone payment from Biogen.

Biogen is conducting the randomized, placebo-controlled, dose escalation Phase 1/2 clinical study that will evaluate the safety and activity of ISIS-SOD1_Rx in patients with ALS, including patients with SOD1-ALS. In this study, ISIS-SOD1_Rx will be administered intrathecally as an injection directly into the cerebral spinal fluid. Intrathecal administration of other antisense drugs has been shown to be well tolerated in multiple clinical studies. For further study information, please visit www.clinicaltrials.gov and search for ISIS-SOD1_Rx.

SOURCE Isis Pharmaceuticals, Inc.