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Isis Pharmaceuticals begins Phase 1/2 clinical study of ISIS-SOD1 Rx in ALS patients.

M2 EQUITYBITES-December 14, 2015-Isis Pharmaceuticals begins Phase 1/2 clinical study of ISIS-SOD1 Rx in ALS patients


Drug discovery company Isis Pharmaceuticals (NasdaqGS:ISIS) reported on Friday the launch of a Phase 1/2 clinical study of ISIS-SOD1Rx (BIIB067) in patients with amyotrophic lateral sclerosis (ALS) by its partner, Biogen.

The company added that ISIS-SOD1Rx is part of its strategic collaboration with Biogen to discover and develop antisense drugs to treat neurological diseases, including ALS, a rare, fatal neurodegenerative disorder.

According to the company, ISIS-SOD1Rx 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% of familial ALS.

ISIS-SOD1Rx, 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. Isis has the opportunity to earn a USD2.5m milestone payment from Biogen.

In conjunction, the company is conducting the randomised, placebo-controlled, dose escalation Phase 1/2 clinical study that will evaluate the safety and activity of ISIS-SOD1Rx in patients with ALS, including patients with SOD1-ALS. ISIS-SOD1Rx will be administered intrathecally as an injection directly into the cerebral spinal fluid.

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Publication:M2 EquityBites (EQB)
Date:Dec 14, 2015
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