Isis Pharmaceuticals, Inc. (ISIS) and Xenon Pharmaceuticals Inc. Collaborate to Develop Antisense Drugs Against Hemojuvelin and Hepcidin  
11/16/2010 7:02:02 AM

VANCOUVER, Canada, Nov. 16, 2010 /PRNewswire-FirstCall/ -- Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) and Xenon Pharmaceuticals Inc. announced today a new collaboration to discover and develop antisense drugs as novel treatments for the common disease anemia of inflammation (AI).

Under the terms of the agreement, Isis will receive an undisclosed upfront payment in the form of a convertible promissory note from Xenon to discover and develop antisense drugs to the targets hemojuvelin and hepcidin. Upon the identification of a development candidate, Xenon has the option to exclusively license the development and worldwide commercialization rights for these antisense drugs from Isis. In addition to license and option fees, Isis will be eligible to receive development and commercial milestones and royalties on sales of drugs licensed to Xenon under the collaboration as well as a portion of sublicense revenue.

"We are excited to be working with Isis, a world leader in the discovery and development of RNA-targeted therapeutics," said Simon Pimstone, President & Chief Executive Officer of Xenon. "Using our human genetics platform, we validated hemojuvelin and hepcidin as novel targets for the treatment of anemia of inflammation where inhibition of these targets in the liver is predicted to treat AI by promoting red blood cell production. As such they are ideally suited to antisense therapeutics."

"Hemojuvelin and hepcidin are promising targets for the treatment of anemia of inflammation, which have been largely inaccessible using traditional drug discovery methods. We are pleased to be working with Xenon and excited to apply our technology to such interesting and clinically meaningful targets," said B. Lynne Parshall, COO and CFO of Isis. "A key component of our business strategy is to exploit the broad applicability of our antisense technology platform in order to target a wide range of diseases. By working with partners like Xenon, who are highly innovative and dedicated to a therapeutic area, we are able to expand the breadth of our antisense drug pipeline while staying focused on our internal drug discovery and development activities."

AI is the second most common form of anemia worldwide and is associated with a wide variety of conditions including infection, cancer and chronic inflammation. AI is caused by the disruption of normal levels of iron in the body leading to poor iron availability for red blood cell production. This restricted iron availability also hampers the current treatment option recombinant erythropoietin (epo) to promote red blood cell production. Antisense therapeutics targeting hemojuvelin and hepcidin should reverse iron disturbances observed in AI and facilitate new red blood cell production.

"A key aspect of erythropoietin's adverse effects, particularly in the cancer setting, has been the use of higher doses in some patients," said Paul Goldberg, Xenon's VP of Clinical Development. "This resistance to erythropoietin action is highly related to the restricted iron availability observed in AI. Inhibition of hemojuvelin or hepcidin may alleviate the erythropoietin resistance and offer alternative treatment options," he added.


Xenon is a privately owned, clinical genetics-based drug discovery and development company engaged in developing small molecule therapies focusing in the areas of pain, cardiometabolic disease, and diseases of iron metabolism. Xenon has multiple products in phase 1 and 2 clinical development. For more information, visit the Company's website at


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 22 drugs in development. Isis' drug development programs are focused on treating cardiovascular, metabolic, and severe neurodegenerative diseases and cancer. 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 has designed and executed a patent strategy that has provided the Company with strong and extensive protection for Isis' drugs as well as all aspects of antisense drug discovery, development and manufacturing. Additional information about Isis is available at


This press release includes forward-looking statements regarding Isis' collaboration with Xenon Pharmaceuticals and the discovery and development of Isis' antisense drugs for the treatment of anemia of inflammation. 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 drugs. 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, 2009 and its most recent quarterly report on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from the Company.

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


This release contains forward-looking statements that are not based on historical fact. These forward-looking statements involve risks, uncertainties and other factors that may cause the actual results, events or developments to be materially different from those expressed or implied by such forward-looking statements. Readers are cautioned not to place undue reliance on such forward-looking statements.

SOURCE Isis Pharmaceuticals, Inc.