Isis Pharmaceuticals Inc. (ISIS) announced Tuesday that it has earned a $7.5 million milestone payment from GlaxoSmithKline (GSK,GSK.L) related to the initiation of a Phase 2/3 clinical study for ISIS-TTRRx.
ISIS-TTRRx is an antisense drug in development with GlaxoSmithKline for the treatment of transthyretin or TTR amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues.
Recently, Isis and GSK amended the clinical development plan and financial terms relating to ISIS-TTRRx to support this registration-directed Phase 2/3 clinical study on ISIS-TTRRx.
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.
ISIS-TTRRx is part of the Isis-GSK strategic alliance to develop RNA therapeutics for rare and infectious diseases. For the initiation of the Phase 2/3 study, Isis will receive a $7.5 million milestone payment and is eligible to earn an additional $50 million in pre-licensing milestone payments to support the Phase 2/3 study of ISIS-TTRRx.
In addition, Isis is eligible to receive regulatory and sales milestone payments and double-digit royalties on sales of ISIS-TTRRx.
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