Isis Pharmaceuticals, Inc. has earned a $2 million milestone payment from GlaxoSmithKline for the advancement of an ongoing Phase II/III study of ISIS-TTR in patients with familial amyloid polyneuropathy (FAP), a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues.
The Phase II/III study is a randomized, double-blind, placebo-controlled, study will support an application for marketing approval of ISIS-TTR in patients with FAP. The study will measure the effects of ISIS-TTR on neurological dysfunction and on quality-of-life.
To date, Isis has earned more than $20 million in upfront and milestone payments for advancing ISIS-TTR. Also, if GSK elects to exercise its option to exclusively license the ISIS-TTR program, Isis is eligible to receive a license fee, regulatory and sales milestones, and royalties on sales.