Isis, Xenon enter collaboration to discover and develop antisense drugs for AI

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.

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