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Isis Pharmaceuticals: Antisense Value

 November 27, 2012 05:16 PM

By John McCamant, editor The Medical Technology Stock Letter

Isis Pharmaceuticals (ISIS) has had a lots of news. The first was the announcement that the company will receive $1.1 million from Alnylam as its portion of the upfront fees from a collaboration with Genzyme.  

The company also announced that they will receive a $1.25 million contingent payment from Pfizer triggered by Pfizer's decision to advance EXC 001 into a Phase II trial. Pfizer's commitment to advancing EXC 001 is a testament to ISIS' business strategy investment.

The market opportunity to treat hypertrophic scars is very large and Pfizer has the drug development expertise and global commercial infrastructure to support the continued advancement of EXC 001, which ISIS could not do on their own.  

The ability to develop a drug development candidate to proof of concept and then partner it on attractive terms is a winning formula for ISIS and their shareholders.

ISIS also had a few pieces of positive clinical news. The company announced the initiation of a Phase Ib/IIa trial evaluating ISIS-SMNRx in children with spinal muscular atrophy (SMA). SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality.  

Under the terms of the January 2012 agreement, ISIS is responsible for global development and Biogen has the option to license the compound until completion of the first successful Phase 2/3 trial.

The company also announced the initiation of a Phase II comparator-controlled trial evaluating ISIS-FXIRx in patients undergoing knee replacement surgery, also referred to as total knee arthroplasty (TKA).  

ISIS-FXIRx inhibits the production of Factor XI, a coagulation factor produced in the liver that is involved in the formation of clots.  

The combination of robust anti-thrombotic activity with an improved safety profile to currently available anticoagulants could be broadly useful in many therapeutic settings where thrombosis is a serious problem.  

ISIS has also announced that preliminary data from the Phase I trial of ISIS-STAT3Rx in patients with cancer were  presented at the Oligonucleotide Therapeutics Society in Boston.  

The early data on ISIS-STAT3Rx is intriguing as it suggests that this drug development candidate can produce responses in refractory patients with advanced cancer who have failed to respond to other therapies.

ISIS continues to create significant long-term value for their shareholders despite Wall Street's continued reservations regarding antisense technology. ISIS is a buy under $12.  

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