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 patients with familial amyloid polyneuropathy (FAP). GSK is developing ISIS-TTRRx as a treatment for transthyretin amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues.
To date, Isis has earned $26 million in upfront and milestone payments for advancing 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 milestone payments and royalties.
"We are very pleased with the progress we are making with ISIS-TTRRx. In addition to being on schedule for enrollment, we have initiated an open-label extension study of ISIS-TTRRx, which is being offered to those patients with FAP who have completed dosing in the Phase II/III study of ISIS-TTRRx," said B. Lynne Parshall, chief operating officer at Isis.