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Isis Pharmaceuticals, Inc. (ISIS - Analyst Report) recently presented encouraging data on its candidate, ISIS-SMN, from a phase I study (n = 28). The data was presented at the 65th annual meeting of the American Academy of Neurology (AAN).

The open-label, single-dose, dose-escalation (1, 3, 6 and 9 mg) study evaluated the safety, tolerability and pharmacokinetic profile of ISIS-SMN in children with spinal muscular atrophy (SMA). It was found that ISIS-SMN was well tolerated at all dosages. Additionally, encouraging improvement in muscle function was also observed.

Isis Pharma is also evaluating ISIS-SMN in a multiple-dose phase Ib/2a study. The company expects to report data from this study by late 2013 or early 2014. Isis Pharma intends to initiate phase II/III studies on ISIS-SMN in infants in 2013 and children in the first half of 2014.

Last month, Isis Pharma received a $7.5 million milestone payment from GlaxoSmithKline (GSK - Analyst Report). The payment primarily relates to the initiation of a phase II/III clinical study of ISIS-TTRRx. The study will span 15 months enrolling 200 patients. Isis is developing ISIS-TTRRx in collaboration with Glaxo for the potential treatment of transthyretin (TTR) amyloidosis.

We note that Isis Pharma’s lead product, Kynamro (mipomersen sodium), received FDA approval in Jan 2013 for the treatment of familial hypercholesterolemia (FH). Isis Pharma’s partner Sanofi (SNY - Analyst Report) is marketing the product in the US. Sanofi is also looking to get the drug approved in the rest of the world including Europe.

Isis Pharma currently carries a Zacks Rank #3 (Hold). Cytokinetics, Inc. (CYTK - Snapshot Report) looks more attractive in the pharma sector with a Zacks Rank #1 (Strong Buy).

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