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March 3, 2014

Isis Earns $1M in Another Milestone Payment from GSK

  • Isis Pharmaceuticals received a $1 million milestone payment from GlaxoSmithKline (GSK) as part of an open-label extension study of ISIS-TTRRx. The product is being offered to patients with familial amyloid polyneuropathy (FAP) who have completed dosing in a Phase II/III study.

    ISIS-TTRRx is an antisense drug in development with GSK for the treatment of transthyretin amyloidosis, a rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues.

    This milestone payment is the third of the $50 million Isis is eligible to receive as the study progresses. Including this milestone payment, Isis has earned $25 million in upfront and milestone payments for advancing ISIS-TTRRx.

    In July 2013, the company won a $2 million milestone payment from GSK for the antisense drug. In the deal, Isis received $20 million in up-front and milestone payments before the dosing of the first patient in the study.

    If GSK elects to exercise its option to exclusively license the ISIS-TTRRx program, Isis also is eligible to receive a license fee, regulatory and sales milestone payments and double-digit royalties on sales of ISIS-TTRRx. The Phase II/III trial 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.

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