Isis Pharmaceuticals, Inc. has earned a $1 million milestone payment from GlaxoSmithKline (GSK) related to the advancement of the phase 2/3 study of ISIS-TTRRx in patients with familial amyloid polyneuropathy (FAP).

SIS-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 milestone payment, Isis has generated $27 million in upfront and milestone payments for advancing ISIS-TTRRx.  This $1 million milestone payment is the fifth of the nearly $58 million in milestone payments Isis is eligible to earn as the phase 2/3 study progresses.  In addition, if GSK elects to exercise its option to exclusively license the ISIS-TTRRx programme, 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.  

Isis is exploiting its leadership position in antisense technology to discover and develop novel drugs for its product pipeline and for its partners.  Isis' broad pipeline consists of 32 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, including neurological disorders, and cancer.