Clinical Trials Update from Ionis Pharmaceuticals

The Phase 3 outcome study of CARDIO-TTR, evaluating patients with TTR amyloid cardiomyopathy through the IONIS-TTRRx program will not be initiated.

The other IONIS-TTRRx studies are continuing and include the NEURO-TTR Phase 3 study and Dr. Merrill Benson’s investigator-initiated Phase 2 open-label study in patients with TTR-related amyloid cardiomyopathy.

The GSK/Ionis Pharmaceuticals NEURO-TTR Phase 3 study also evaluates patients who have cardiac involvement in addition to their polyneuropathy. They have noted that over half of the patients in the NEURO-TTR study have cardiac involvement. There is promising data noted from Dr. Benson’s study in patients with the cardiomyopathy form of the disease.  We look forward to seeing Dr. Benson’s data on the study that will be presented at the International Symposium on Amyloidosis in July 2016.

GSK exclusively licenses IONIS-TTRSRx  and Dr. Helen Merianos, Medicine Development Leader for GSK’s TTR amyloidosis program, said: “GSK remains committed to developing innovative medicines for the treatment of amyloidosis and to our ongoing collaboration with Ionis to develop IONIS – TTRRx for TTR amyloidosis. We will assess the results from the NEURO-TTR study to inform our next steps for TTR amyloid cardiomyopathy.”