Isis Pharmaceuticals Inc. announced that it has earned a $1 million milestone payment from GlaxoSmithKline related to the advancement of the Phase 2/3 study of ISIS-TTRRx in patients with familial amyloid polyneuropathy (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 milestone payment, Isis has generated $26 million in upfront and milestone payments for advancing ISIS-TTRRx. This $1 million milestone payment is the fourth of the $50 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 program, Isis is eligible to receive a license fee, regulatory and sales milestone payments and double-digit royalties on sales of ISIS-TTRRx.
“We are very pleased with the progress we are making with ISIS-TTRRx. In addition to being on schedule for enrollment, we have initiated an open-label extension study of ISIS-TTRRx, which is being offered to those patients with FAP who have completed dosing in the Phase 2/3 study of ISIS-TTRRx,” said B. Lynne Parshall, chief operating officer at Isis.
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.
Date; June 24, 2014
Source: Isis Pharmaceuticals
