Biogen starts phase 1/2 clinical study of ISIS-SOD1 Rx in ALS patients

Patients with ALS fatal neurodegenerative disorder suffer progressive degeneration of the motor neurons, which leads to a declining quality of life and even could lead to death, the company said.

ISIS-SOD1Rx is a Gen. 2.0+ antisense drug designed to lower the production of the superoxide dismutase 1 (SOD1) enzyme.

A mutation in the SOD-1 gene causes an inherited form of ALS, which is the second most common familial form of ALS, accounting for about 20% of familial cases and 10% of all ALS cases.

Under the randomized, placebo-controlled, dose escalation phase 1/2 clinical study, Biogen will administer ISIS-SOD1Rx as an injection directly into the cerebral spinal fluid.

Other antisense drugs administered in the similar way have been well-tolerated in several clinical trials.

Isis Pharmaceuticals COO Lynne Parshall said: "Together with Biogen, we have made significant progress developing antisense drugs to treat neurological and neuromuscular disorders.

"The advancement of ISIS-SOD1Rx 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 partnership of Isis and Biogen is focused on using antisense technology to advance the treatment of neurological and neuromuscular disorders.

The alliance combines Isis’ experience in antisense technology for assessing potential neurological targets and discovering antisense drugs with Biogen’s capability to develop neurological disorders therapies.