Isis and Collaborators Present New Research at the ATVB Annual Conference

Isis Pharmaceuticals and collaborators presented data on numerous advanced programs from Isis' cardiovascular franchise

CARLSBAD, CA, USA I May 1, 2009 I Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) announced today that Isis and collaborators presented data on numerous advanced programs from Isis' cardiovascular franchise. The data were presented during the Arteriosclerosis, Thrombosis and Vascular Biology's (ATVB) Annual Conference. The presentations included data from a post-hoc analysis of a recently completed clinical study of mipomersen in which treatment with mipomersen resulted in a decrease in apoC-III. In addition, Isis presented preclinical data from two late-stage research programs, one on a direct antisense inhibitor of apoC-III, which may provide a promising new drug to lower triglycerides, and a second on an antisense inhibitor to Factor XI, which has the potential to create a drug to decrease clotting without increasing bleeding. Both of these programs are expected to produce near-term clinical development candidates that have the potential to provide unique treatments for cardiovascular disease. Isis and collaborators also presented data on antisense inhibitors against 11 beta-HSD1 and apoF, potential new cardiovascular targets. The research presented at this year's ATVB in Washington D.C. showcases Isis' prolific antisense drug discovery capabilities and supports the continued expansion of Isis' cardiovascular franchise.

At ATVB, Isis collaborator, Dr. Frank Sacks, Professor of Cardiovascular Disease Prevention at the Harvard School of Public Health, presented a post-hoc analysis on a Phase 2 study in patients with high cholesterol who were treated with mipomersen as a single agent. Dr. Sacks' analysis showed that treatment with mipomersen reduced circulating apoC-III. ApoC-III is an apolipoprotein that transports triglycerides in the blood. Patients with cardiovascular disease frequently have elevated levels of triglycerides and apoC-III.

Isis scientist, J. Crosby, presented new research titled "Antisense oligonucleotide-mediated depletion of Factor XI results in effective anticoagulation with a favorable risk/benefit profile in mice" demonstrating that antisense drugs that inhibit Factor XI can prevent thrombus formation in models of stroke without causing an increase in bleeding, the most common side effect observed with currently available antithrombotic agents. The Isis antisense drug inhibiting Factor XI is in advanced research and representative of significant advances in creating antisense drugs that can more safely reduce undesired clotting.

"Factor XI is the first representative drug candidate for development from our new thrombosis effort. It is another example of the power of our antisense technology to develop new drugs to nearly any genomic target," said Brett Monia, Ph.D., Vice President of Drug Discovery and Corporate Development at Isis Pharmaceuticals. "After rapidly and efficiently evaluating individual clotting factors produced in the liver, we selected Factor XI as the optimal target to inhibit thrombosis without causing bleeding. In addition to Factor XI, we will have drugs that inhibit other individual clotting factors that demonstrate potential therapeutic benefit and represent other novel approaches to treating cardiovascular disease."

Isis scientist, A. Mullick, also presented new research titled "Antisense inhibition of apolipoproteinC-III mitigated features of metabolic syndrome and reduced atherosclerosis in LDL receptor knockout mice" showing the ability of antisense drugs in a late-stage research program targeting apoC-III to selectively reduce triglycerides in both liver and blood. Antisense drugs that selectively reduced levels of apoC-III dramatically reduced triglycerides and atherosclerosis in animal models.

"The potential to add an apoC-III inhibitor in the near-term to our cardiovascular pipeline, which already includes mipomersen, a CRP inhibitor and a PCSK9 inhibitor is quite exciting," said Richard Geary, Ph.D., Senior Vice President of Development at Isis Pharmaceuticals. "Inhibiting apoC-III is a promising new way to lower triglycerides to treat a potential independent risk factor for cardiovascular disease. Futhermore, the anti-atherotic effect of lowering apoC-III, which we have seen in animals, suggests that lowering apoC-III could produce reductions in atherosclerosis in man as well."

Finally, Isis and collaborators reported on the effects of antisense drugs targeting 11 beta-HSD1 and apoF. In a presentation titled "Antisense-mediated inhibition of 11 beta-HSD1 significantly decreases hepatic lipogenesis, triglyceride content and triglyceride secretion" Isis collaborator, G. Li from Columbia University, showed that antisense inhibition of 11 beta-HSD1 reduced triglycerides in both liver and blood, while also reducing body fat in animal models. 11 beta-HSD1 is an enzyme that inactivates steroids and may contribute to metabolic disease, suggesting the potential therapeutic benefit of antisense drugs inhibiting 11 beta-HSD1 to treat high triglycerides and diabetes. In a presentation titled "In vivo knock down of apolipoprotein F decreases liver triglyceride levels" S. Khetarpal from University of Pennsylvania presented research demonstrating that antisense drugs that selectively inhibit apoF reduced triglycerides in blood and liver. ApoF is another protein that carries triglycerides in blood and could offer potential therapeutic benefit in patients with diseases associated with elevated triglycerides including cardiovascular disease.

"The efficiency of our drug discovery platform offers us virtually unlimited opportunities to expand the scope of diseases we can treat with antisense drugs. We have shown that antisense inhibition of a number of new disease targets could have potential therapeutic benefits across a wide range of cardiovascular diseases," said C. Frank Bennett, Ph.D., Senior Vice President of Research at Isis Pharmaceuticals. "The work we presented at this year's ATVB conference demonstrates the broad applicability of our technology and how we can take advantage of the growing number of genetically identified therapeutic targets, especially targets associated with cardiovascular disease. Of course, we see similar efficiency and equally exciting advances in therapeutic areas outside of cardiovascular disease, including severe neurodegenerative and metabolic diseases, and cancer."


Isis is exploiting its expertise in RNA to discover and develop novel drugs for its product pipeline and for its partners. The Company has successfully commercialized the world's first antisense drug and has 19 drugs in development. Isis' drug development programs are focused on treating cardiovascular and metabolic diseases. Isis' partners are developing antisense drugs invented by Isis to treat a wide variety of diseases. Isis and Alnylam Pharmaceuticals are joint owners of Regulus Therapeutics Inc., a company focused on the discovery, development and commercialization of microRNA therapeutics. Isis also has made significant innovations beyond human therapeutics resulting in products that other companies, including Abbott, are commercializing. As an innovator in RNA-based drug discovery and development, Isis is the owner or exclusive licensee of over 1,600 issued patents worldwide. Additional information about Isis is available at

This press release includes forward-looking statements regarding Isis' business, its drug discovery and development pipeline, and the therapeutic potential of antisense drugs for the treatment of cardiovascular disease. Any statement describing Isis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such products. Isis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Isis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Isis' programs are described in additional detail in Isis' annual report on Form 10-K for the year ended December 31, 2008, which is on file with the SEC. Copies of this and other documents are available from the Company.

In this press release, unless the context requires otherwise, "Isis," "Company," "we," "our," and "us" refers to Isis Pharmaceuticals and its subsidiaries, including Regulus Therapeutics Inc.

Isis Pharmaceuticals is a registered trademark of Isis Pharmaceuticals, Inc. Regulus Therapeutics is a trademark of Regulus Therapeutics Inc.

SOURCE: Isis Pharmaceuticals, Inc.

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