10.09.14
Isis Pharmaceuticals, Inc. has earned an $18 million milestone payment from GlaxoSmithKline (GSK) related to the advancement of the Phase II/III study of ISIS-TTRRx in patients with familial amyloid polyneuropathy (FAP). The Phase II/III study aims 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 on quality-of-life.
ISIS-TTRRx is an antisense drug for the treatment of transthyretin amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues. To date, Isis has earned $45 million of the $70 million in upfront and milestone payments for advancing the development of ISIS-TTRRx. If GSK exercises its option to exclusively license the ISIS-TTRRx program, Isis is eligible to receive a license fee, regulatory and sales milestones, and royalties.
"ISIS-TTRRx is the most advanced drug in our collaboration with GSK, and aside from KYNAMRO, it is also the furthest advanced in our late-stage clinical pipeline. We have patients who have completed fifteen months of therapy and are now receiving ISIS-TTRRx in our open-label extension study," said B. Lynne Parshall, chief operating officer at Isis. "The rapid development of ISIS-TTRRx from a research-stage program to a drug in a late-stage clinical trial is a testament to the efficiency and productivity of our drug discovery technology platform. As this program advances, we will continue to benefit from GSK's resources and expertise as well as earn additional milestone payments."
ISIS-TTRRx is an antisense drug for the treatment of transthyretin amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues. To date, Isis has earned $45 million of the $70 million in upfront and milestone payments for advancing the development of ISIS-TTRRx. If GSK exercises its option to exclusively license the ISIS-TTRRx program, Isis is eligible to receive a license fee, regulatory and sales milestones, and royalties.
"ISIS-TTRRx is the most advanced drug in our collaboration with GSK, and aside from KYNAMRO, it is also the furthest advanced in our late-stage clinical pipeline. We have patients who have completed fifteen months of therapy and are now receiving ISIS-TTRRx in our open-label extension study," said B. Lynne Parshall, chief operating officer at Isis. "The rapid development of ISIS-TTRRx from a research-stage program to a drug in a late-stage clinical trial is a testament to the efficiency and productivity of our drug discovery technology platform. As this program advances, we will continue to benefit from GSK's resources and expertise as well as earn additional milestone payments."