ISIS-TTR Rx is an antisense drug being developed by GSK to treat TTR amyloidosis, a genetic disease characterized by progressive dysfunction of peripheral nerves and/or heart tissue.
The accelerated development plan is expected to initiate a phase 2/3, registration-directed, clinical study later the year.
Isis antisense drug discovery senior vice president Brett Monia said "We anticipate initiating the next clinical study this year, which will evaluate the effects of ISIS-TTRRx on neurological dysfunction and on quality-of-life in patients with familial amyloid polyneuropathy."
As part of the altered agreement, Isis will receive a $2.5m upfront payment and will earn a $7.5m milestone payment upon the ISIS-TTRRx Phase 2/3 study initiation.
In addition, Isis will receive an additional $50m in pre-licensing milestone payments to support the ISIS-TTR phase 2/3 study as well as increased regulatory and sales milestones payments from GSK upon achieving product registration and certain sales thresholds.
Isis chief operating officer and chief financial officer Lynne Parshall said, "Under the updated plan, Isis continues to manage the clinical development of ISIS-TTRRx while benefiting from GSK’s late-stage development and commercial expertise."