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Isis Pharmaceuticals, Inc. recently published encouraging new data in the journal Circulation Research related to antisense technology. It was found that antisense targeting of apolipoprotein C-III (apoC-III) showed significant decline in apoC-III and triglycerides. Both apoC-III and triglycerides are risk factors associated with cardiovascular diseases.

Isis Pharma currently has an antisense apoC-III inhibitor, ISIS-APOCIII, in its pipeline. ISIS-APOCIII is being evaluated in a phase II study for the treatment of severe hypertriglyceridemia. Top-line data from the study are expected to be released in mid-2013. On completion of the phase II study the company plans to initiate phase III study on the candidate.

We are impressed by the development of antisense technology and believe that the number of potential therapeutic applications is enormous. Antisense drugs may have significant potential to treat a number of diseases where small molecules and biologic compounds have failed. Although still in its early stage, antisense technology with mechanisms such as small interfering RNA (siRNA), RNA interference (RNAi), alternate splicing, and microRNA (mRNA) have the potential to change how diseases are treated.

Earlier this year, Isis Pharma received a $7.5 million milestone payment from GlaxoSmithKline . 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 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. looks more attractive in the pharma sector with a Zacks Rank #1 (Strong Buy).

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