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ISIS Pharmaceuticals Earns $1 Million From GSK For Advancing ISIS-TTR Rx

CARLSBAD, Calif. , March 3, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $1 million milestone payment from GlaxoSmithKline (GSK) related to the initiation of an open-label extension study of ISIS-TTR Rx, which is being offered to those patients with familial amyloid polyneuropathy (FAP) who have completed dosing in the Phase 2/3 study of ISIS-TTR Rx.  


ISIS-TTR Rx 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.   Including this milestone payment, Isis has earned $25 million in upfront and milestone payments for advancing ISIS-TTR Rx.   This $1 million milestone payment is the third 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-TTR Rx program, Isis is eligible to receive a license fee, regulatory and sales milestone payments and double-digit royalties on sales of ISIS-TTR Rx.

"We are very pleased with the progress we are making with ISIS-TTR Rx.   We have patients who have been on therapy for almost a year and enrollment continues to proceed as planned," said B. Lynne Parshall, chief operating officer at Isis.     

The Phase 2/3 study of ISIS-TTR Rx is a randomized, double-blind, placebo-controlled, international study designed to support an application for marketing approval of ISIS-TTR Rx in patients with FAP.   The fifteen month study will measure the effects of ISIS-TTR Rx on neurological dysfunction and on quality-of-life.   ISIS-TTR Rx is an investigational drug that is designed to inhibit the production of all forms of TTR, and to treat all types of transthyretin-related amyloidosis.   For further study information, please visit 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 31 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, including neurological disorders, and cancer.   Isis' partner, Genzyme, is commercializing Isis' lead product, KYNAMRO ®, in the United States and other countries for the treatment of patients with homozygous FH.   Isis' patents provide strong and extensive protection for its drugs and technology.   Additional information about Isis is available at

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