The Phase 1 study of ISIS-TTRRx was a blinded, randomized, placebo-controlled, dose-escalation study designed to assess the safety and pharmacokinetic profile of ISIS-TTRRx in healthy volunteers. ISIS-TTRRx was evaluated in single and multiple doses ranging from 50 mg per week up to 400 mg per week. After only four weeks of dosing, subjects in the 200 mg and 400 mg multiple-dose cohorts displayed a mean reduction of 44 percent and 81 percent in TTR levels, respectively. ISIS-TTRRx was generally well tolerated in all subjects.
"In this study, we observed substantial dose-dependent reductions in TTR protein of greater than 80 percent. Based on the mechanism of action and the early data we have presented, we believe that ISIS-TTRRx could provide benefit to patients with FAP," said Brett Monia, Ph.D., Senior Vice President, Drug Discovery and Corporate Development of Isis. "Together with our partner GSK, we are finalizing the development plan for ISIS-TTRRx, which is intended to achieve an efficient route to registration. Our next clinical study for ISIS-TTRRx will begin in 2012 and evaluate the effects of the drug on disease progression and other measures of disease burden/improvements on quality of life in patients with FAP."
Transthyretin amyloidosis is a genetic disease in which the patient inherits a mutant gene that produces a misfolded form of TTR, which progressively accumulates in tissues, impairing their function. In patients with transthyretin amyloid
|SOURCE Isis Pharmaceuticals, Inc.|
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