Isis Pharmaceuticals, Inc. has earned a $1 million milestone payment from GlaxoSmithKline for the advancement of the Phase II/III study of ISIS-TTRRx in familial amyloid polyneuropathy (FAP), a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues.
To date, Isis has earned $27 million in upfront and milestone payments. Should GSK exercise its option to license the ISIS-TTRRx program, Isis is eligible to receive a license fee, regulatory and sales milestones, and royalties on sales.
The Phase II/III study is designed to support an application for marketing approval of ISIS-TTRRx in FAP. The study will measure the effects of ISIS-TTRRx on neurological dysfunction and quality-of-life. ISIS-TTRRx is designed to inhibit the production of all forms of TTR, and to treat all types of transthyretin-related amyloidosis.