Isis Pharmaceuticals partner Biogen has commenced a Phase I / II clinical trial of ISIS-SOD1Rx (BIIB067) for patients with amyotrophic lateral sclerosis (ALS).
ALS is a rare and fatal neurodegenerative disorder.
Formely known as ISIS-BIIB3Rx, the ISIS-SOD1Rx is a Gen. 2.0+ antisense drug, which is designed to reduce the production of superoxide dismutase 1 (SOD1).
ISIS-SOD1Rx is part of the firms' strategic collaboration, which was established to develop antisense drugs to treat neurological diseases.
Isis Pharmaceuticals research senior vice-president Dr Frank Bennett said: "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."
The company has designed the Phase I / II clinical trial to assess the safety and activity of ISIS-SOD1Rx in patients with ALS, including patients with SOD1-ALS.
In the trial, ISIS-SOD1Rx will be injected directly into the cerebral spinal fluid.
According to Isis, the intrathecal administration of other antisense drugs has been shown to be well tolerated in multiple clinical studies.
Isis Pharmaceuticals COO Lynne Parshall said: "Together with Biogen, we have made significant progress developing antisense drugs to treat neurological and neuromuscular disorders."