Release Details

Antisense Technology Provides Insights Into Role of STAT-3 in the Growth and Survival of Blood Cell Cancers

CARLSBAD, Calif., May 18 /PRNewswire-FirstCall/ -- Scientists from Isis Pharmaceuticals, Inc. (Nasdaq: ISIS), working in collaboration with scientists at the Department of Pathology and NYU Cancer Institute, New York University School of Medicine and the Department of Pathology and Center for experimental Research and Medical Studies (CERMS), University of Torino, have reported in Nature Medicine that the STAT-3 gene is required for the generation of B-lymphomas in mice carrying a mutation implicated in some human lymphomas and that reducing the expression of STAT-3 with a second-generation antisense drug targeted to STAT-3 resulted in impaired growth of human and mouse lymphoid tumors in animals. These results suggest that the second-generation antisense inhibitor of STAT-3 might be an effective treatment for human lymphomas, multiple myeloma, and other cancers. The drug, ISIS 345794, is in preclinical development at Isis.

"It has been known for sometime that spontaneously occurring rearrangements on genes in human chromosomes can cause some cancers. For example, in some forms of human lymphomas, a cancer-causing gene is aberrantly relocated in the chromosome and inappropriately activated. We used genetic techniques to show that in a mouse model of human lymphoma and myeloma that the loss of STAT-3 prevents progression of disease and, in some cases, reverses the growth of existing lymphomas," said Dr. Giorgio Inghirami, senior author of the publication. "Although modern genetic methods can lead to important insights into diseases, one of the central challenges is to rapidly convert the information to new drugs that will benefit patients. Antisense technology gives researchers the ability to target a specific protein, such as STAT-3, to clearly determine its pathogenetic role, and to efficiently treat the cause of the disease."

"This study was one of the studies that supported our understanding of the biological function of STAT-3 and reinforced our enthusiasm for this very exciting drug. Through the use of antisense technology, we confirmed with our collaborators that inhibition of STAT-3 prevented the progression of cancer," said Brett P. Monia, Ph.D., Isis' Vice President of Antisense Drug Discovery. "Once we have a functional understanding of a particular molecular target, we can progress the specific antisense inhibitor used to validate the target rapidly into drug development. ISIS 345794 is an excellent example of the value and efficiency of the technology for the treatment of human disease."

ABOUT ISIS PHARMACEUTICALS, INC.

Isis Pharmaceuticals, Inc. is exploiting its expertise in RNA to discover and develop novel human therapeutic drugs for its pipeline and for its partners. The Company has successfully commercialized the world's first antisense drug and has 11 antisense products in development to treat metabolic, cardiovascular and inflammatory diseases, and cancer. Through its Ibis division, Isis is developing a system to identify infectious organisms. As an innovator in RNA-based drug discovery and development, Isis is the owner or exclusive licensee of more than 1,500 issued patents worldwide. Additional information about Isis is available at http://www.isispharm.com.

This press release includes forward-looking statements regarding antisense technology, the development and therapeutic potential of antisense drugs to specific inflammation targets, and the success of Isis and its partners' anti-cancer drug discovery programs. Any statement describing our goals, expectations, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement, including those statements that are described as Isis' clinical goals. 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. Our forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause our results to differ materially from those expressed or implied by such forward-looking statements. Although our forward-looking statements reflect the good faith judgment of our management, these statements are based only on facts and factors currently known by us. As a result, you are cautioned not to rely on these forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those discussed in Isis' Annual Report on Form 10-K for the year ended December 31, 2004, and quarterly report on Form 10-Q for the quarter ended March 31, 2005, which are on file with the U.S. Securities and Exchange Commission (SEC), and available from the Company.

SOURCE Isis Pharmaceuticals, Inc.
05/18/2005
CONTACT: Claudine Prowse, Ph.D. of Isis Pharmaceuticals, Inc., +1-760-603-2331

Web site: http://www.isispharm.com
(ISIS)
05/18/2005 07:55 EDT http://www.prnewswire.com