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Isis and Partners Present New Research at the American Association for Cancer Research 2009 Annual Meeting

DENVER and CARLSBAD, Calif., April 20 /PRNewswire-FirstCall/ -- Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) announced today that Isis and its collaborators presented results on antisense inhibition of novel targets that could offer new mechanisms to treat cancer during the American Association for Cancer Research (AACR) meeting. Research conducted by Isis and its collaborators demonstrated the feasibility of targeting a large non-coding RNA, sigma RNA also known as Malat-1, that could offer a new therapeutic mechanism to treat disease. Isis also presented new preclinical data showing that antisense inhibition of STAT5 resulted in potent anti-tumor activity. The oral presentations were presented today, April 20, at the AACR 100th Annual Meeting 2009 in Denver, Colorado by Isis and its collaborators.

The presentation by R. Lin from The Scripps Research Institute on work done in collaboration with scientists from Isis and titled "Molecular Control of RNA Alternative Splicing by sigma RNA, a Marker for Carcinomas" showed that antisense inhibition of Malat-1 altered the splicing of multiple mRNAs. Altering splicing results in the production of different variations of a protein, including variations that could be associated with cancer. Malat-1 is up-regulated in many human cancers including liver, breast, lung, pancreas, colon, and prostate.

Isis scientist Y. Kim presented research titled "Selective Inhibition of STAT5 Expression in Acute Myeloid Leukemia Cells Results in Potent Antitumor Activity" showing that antisense inhibition of STAT5 increased survival in preclinical models of acute myeloid leukemia (AML) suggesting that antisense drugs targeting STAT5 may offer a new avenue to treat AML and related blood disorders.

"The work presented at this year's AACR demonstrates the broad applicability of antisense technology and adds to our previously published work using antisense to target different classes of RNAs, including microRNAs and other non-coding RNAs. We and our collaborators have shown that antisense inhibition of non-coding RNAs can impact splicing patterns in cells to modulate the expression of multiple proteins that may be involved in disease," said C. Frank Bennett, Ph.D., Senior Vice President of Research at Isis Pharmaceuticals. "Furthermore, the work of our team on STAT5 provides yet another oncology target with the potential for anti-tumor activity. We believe that antisense drugs have pharmaceutical properties that make them attractive therapeutics for cancer, and we are expanding our efforts in cancer to include new targets and new approaches."


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 cancer. 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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