Isis and GlaxoSmithKline eye Phase III with a rare disease drug

GlaxoSmithKline ($GSK) and partner Isis Pharmaceuticals ($ISIS) are plotting a pivotal trial in the rare TTR amyloid cardiomyopathy, an inherited disease that impairs the heart muscle. GSK is planning to enroll about 500 patients with the disease in a multicenter study that will determine how well ISIS-TTR can improve outcomes including mortality, cardiac transplant and hospitalization, the companies said. ISIS-TTR is an antisense drug designed to combat the protein transthyretin, whose misfolding is associated with a few rare diseases. In an open-label Phase II study, the drug has successfully reduced TTR protein levels and charted a disease-stabilizing effect in amyloid cardiomyopathy, Isis said. Release

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