Isis Pharma Earns $18 Mln From GSK For Advancing ISIS-TTR Rx - Quick Facts

(RTTNews) - Isis Pharmaceuticals Inc. (ISIS) announced that it has earned an $18 million milestone payment from GlaxoSmithKline (GSK, GSK.L) related to the advancement of the Phase 2/3 study of ISIS-TTRRx in patients with familial amyloid polyneuropathy or 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 $18 million milestone payment, Isis has generated $45 million of the $70 million in upfront and milestone payments Isis is eligible to earn for advancing ISIS-TTRRx in development.

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.