Isis Pharmaceuticals Earns $2 Million From GSK for the Advancement of ISIS-TTR Rx

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Friday, August 2, 2013

CARLSBAD, Calif., - Isis Pharmaceuticals, Inc., announced that it has earned a $2 million milestone payment from GlaxoSmithKline (NYSE: GSK) related to the advancement of the ongoing Phase 2/3 study of ISIS-TTRRx in patients with familial amyloid polyneuropathy (FAP).

ISIS-TTRRx is an antisense drug in development with GSK for the treatment of transthyretin amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues. Isis has earned $20 million in upfront and milestone payments prior to first patient dosed in the ongoing Phase 2/3 study. The $2 million milestone payment announced today is the first milestone of the $50 million in milestone payments Isis is eligible to earn as the Phase 2/3 study progresses. In addition, if GSK elects to exercise its option to exclusively license the ISIS-TTRRx program, Isis is eligible to receive a license fee, regulatory and sales milestone payments and double-digit royalties on sales of ISIS-TTRRx.

The Phase 2/3 study of ISIS-TTRRx is a randomized, double-blind, placebo-controlled, international study designed to support an application for marketing approval of ISIS-TTRRx in patients with FAP. The fifteen month study will measure the effects of ISIS-TTRRx on neurological dysfunction and on quality-of-life. For further study information, please visit www.clinicaltrials.gov and search for the identifier number NCT01737398.

ABOUT ISIS PHARMACEUTICALS, INC.

Isis is exploiting its leadership position in antisense technology to discover and develop novel drugs for its product pipeline and for its partners. Isis' broad pipeline consists of 28 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, and cancer. Isis' partner, Genzyme, is commercializing Isis' lead product, KYNAMRO™, in the United States for the treatment of patients with HoFH. Genzyme is also pursuing marketing approval of KYNAMRO in other markets. Isis' patents provide strong and extensive protection for its drugs and technology. Additional information about Isis is available at www.isispharm.com.

CONTACT:

D. Wade Walke, Ph.D.
Executive Director, Corporate Communications and Investor Relations,
760-603-2741;
 
Amy Blackley, Ph.D.,
Associate Director, Corporate Communications,
760-603-2772

Source: Isis Pharmaceuticals, Inc.

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