Ionis Pharmaceuticals Inc (IONS)

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7:08AM ISIS Pharm initates Phase 2/3 study Of ISIS-TTR Rx and earns $7.5 million milestone payment from GlaxoSmithKline (GSK) (ISIS) 14.43 : Co announced today that it has earned a $7.5 million milestone payment from GlaxoSmithKline (GSK) related to the initiation of a Phase 2/3 clinical study for ISIS-TTRRx. ISIS-TTRRx is an antisense drug in development with GlaxoSmithKline (GSK) for the treatment of transthyretin amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues. The Phase 2/3 study of ISIS-TTRRx is a randomized, double-blinded, placebo-controlled, international study designed to support an application for marketing approval of ISIS-TTRRx in patients with FAP. The fifteen month study will enroll approximately 200 patients randomized 2:1 to receive 300 mg/week of ISIS-TTRRx or placebo and will measure the effects of ISIS-TTRRx on neurological dysfunction and on quality-of-life. The United States Food and Drug administration granted ISIS-TTRRx fast track designation and orphan drug status for the treatment of FAP.