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.