Isis Pharmaceuticals, Inc. ISIS announced that its partner Biogen Inc. BIIB has started a phase I/II study on ISIS-SOD1Rx (previously known as ISIS-BIIB3Rx) in patients suffering from amyotrophic lateral sclerosis (ALS).
The randomized, placebo-controlled, dose-escalation study is being conducted to evaluate the safety and activity of ISIS-SOD1Rx in patients with ALS, including patients with superoxide dismutase 1-ALS. Patients will receive ISIS-SOD1Rx through an injection directly into the cerebral spinal fluid.
Isis and Biogen are developing ISIS-SOD1Rx under a collaboration agreement that is focused on the discovery and development of antisense drugs for the treatment of neurological and neuromuscular disorders. As the study advances, Isis has the opportunity to earn a $2.5 million milestone payment from Biogen. Apart from ISIS-SOD1Rx, candidates that are being developed under this collaboration include nusinersen (spinal muscular atrophy), ISIS-DMPK-2.5Rx (myotonic dystrophy type 1), and an undisclosed neurodegenerative disease candidate, ISIS-BIIB4Rx.
We are encouraged by the companies’ progress on ISIS-SOD1Rx. Given the lack of approved treatments for ALS, there is strong unmet need for drugs addressing this disease.
We note that Isis has partnership agreements with leading health care companies like GlaxoSmithKline plc GSK and others. The company’s agreements with these companies not only validate its antisense technology but also provide it with funds in the form of upfront, milestone and other payments.
Meanwhile, Isis has several candidates in mid-stage clinical development that could trigger future licensing opportunities. We expect investor focus to remain on pipeline updates from the company.
Isis is a Zacks Rank #3 (Hold) stock. A better-ranked stock in the health care sector is Achillion Pharmaceuticals, Inc. ACHN, carrying a Zacks Rank #1 (Strong Buy).
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