Isis Pharmaceuticals secures $18 million from GSK to advance ISIS-TTR Rx Phase 2/3 study

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned an $18 million milestone payment from GlaxoSmithKline (GSK) related to the advancement of the Phase 2/3 study of ISIS-TTR_Rx in patients with familial amyloid polyneuropathy (FAP).

"ISIS-TTR_Rx is the most advanced drug in our collaboration with GSK, and aside from KYNAMRO, it is also the furthest advanced in our late-stage clinical pipeline. We have patients who have completed fifteen months of therapy and are now receiving ISIS-TTR_Rx in our open-label extension study," said B. Lynne Parshall, chief operating officer at Isis. "The rapid development of ISIS-TTR_Rx from a research-stage program to a drug in a late-stage clinical trial is a testament to the efficiency and productivity of our drug discovery technology platform. As this program advances, we will continue to benefit from GSK's resources and expertise as well as earn additional milestone payments."

ISIS-TTR_Rx 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-TTR_Rx in development. In addition, if GSK elects to exercise its option to exclusively license the ISIS-TTR_Rx program, Isis is eligible to receive a license fee, regulatory and sales milestone payments and double-digit royalties on sales of ISIS-TTR_Rx.

The Phase 2/3 study of ISIS-TTR_Rx is a randomized, double-blind, placebo-controlled, international study designed to support an application for marketing approval of ISIS-TTR_Rx in patients with FAP. The fifteen month study will measure the effects of ISIS-TTR_Rx on neurological dysfunction and on quality-of-life. ISIS-TTR_Rx 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.