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Isis Pharmaceuticals Earns $1 Million from GSK for Advancing ISIS-TTR Rx
Date:7/16/2014

CARLSBAD, Calif., July 16, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $1 million milestone payment from GlaxoSmithKline (GSK) related to the advancement of the 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.  Including this milestone payment, Isis has generated $27 million in upfront and milestone payments for advancing ISIS-TTRRx.  This $1 million milestone payment is the fifth of the nearly $58 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.  ISIS-TTRRx 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 www.clinicaltrials.gov and search for the identifier number NCT01737398.

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