Isis Pharmaceuticals received a $1 million milestone payment from GlaxoSmithKline (GSK) as part of an open-label extension study of ISIS-TTRRx. The product is being offered to patients with familial amyloid polyneuropathy (FAP) who have completed dosing in a Phase II/III study.
ISIS-TTRRx is an antisense drug in development with GSK for the treatment of transthyretin amyloidosis, a rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues.
This milestone payment is the third of the $50 million Isis is eligible to receive as the study progresses. Including this milestone payment, Isis has earned $25 million in upfront and milestone payments for advancing ISIS-TTRRx.
In July 2013, the company won a $2 million milestone payment from GSK for the antisense drug. In the deal, Isis received $20 million in up-front and milestone payments before the dosing of the first patient in the study.
If GSK elects to exercise its option to exclusively license the ISIS-TTRRx program, Isis also is eligible to receive a license fee, regulatory and sales milestone payments and double-digit royalties on sales of ISIS-TTRRx. The Phase II/III trial 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.