Phase III CARDIO-TTR study of IONIS TTRRx in TTR amyloid cardiomyopathy no longer proceeding- GSK/Ionis

GlaxoSmithKline (GSK) which has an option to exclusively license antisense drug IONIS-TTRRx, from Ionis, has decided not to initiate a Phase III outcome study, CARDIO-TTR, which was planned to evaluate IONIS-TTRRx in patients with TTR amyloid cardiomyopathy. As announced in April 2016, the FDA had placed this study on clinical hold as a result of safety findings in the ongoing NEURO-TTR study. GSK will consider options for TTR amyloid cardiomyopathy once additional clinical data are available from the ongoing studies.

Both the NEURO-TTR and an investigator-initiated Phase II open-label study in patients with TTR-related amyloid cardiomyopathy, should provide important data on patients with TTR amyloid cardiomyopathy, which could contribute to the design of an optimal Phase III study. In addition to the investigator-initiated study in patients with TTR-related amyloid cardiomyopathy, the NEURO-TTR study is also evaluating patients with TTR-related cardiomyopathy using a cardiac substudy to evaluate patients who have cardiac involvement in addition to their polyneuropathy. Approximately half of the patients in the NEURO-TTR study also have TTR-related amyloid cardiomyopathy.

Comment: the NEURO-TTR study and phase II study are both proceeding on track, with the data from the NEURO-TTR study planned in the first half of 2017. Updated data from the phase II study will be presented at the International Symposium on Amyloidosis in July 2016. Together, Ionis and GSK, are actively preparing for an NDA filing.