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Isis Pharmaceuticals Earns $2 Million from GSK for the Advancement of ISIS-TTR Rx
Date:7/30/2013

CARLSBAD, Calif., July 30, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today 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.

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