Akcea Therapeutics, Inc. and Ionis Pharmaceuticals, Inc. announced the initiation of the CARDIO-TTRansform Phase 3 cardiovascular outcomes study for AKCEA-TTR-LRx in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR cardiomyopathy). AKCEA-TTR-LRx is an antisense drug developed using Ionis' proprietary LIgand Conjugated Antisense (LICA) technology platform and is designed to inhibit production of TTR. It was discovered by Ionis and is being co-developed by Ionis and Akcea.

In a Phase 1 clinical trial, patients treated with AKCEA-TTR-LRx experienced reductions in TTR of up to 94% at the higher dose. CARDIO-TTRansform is a global, double-blind, randomized, placebo-controlled Phase 3 cardiovascular outcome study. It is designed to compare AKCEA-TTR-LRx to placebo in patients with both wild type and hereditary ATTR cardiomyopathy who are on the current available standard of care.

The primary composite endpoint is cardiovascular (CV) mortality and frequency of CV clinical events comparing the two study arms at Week 120. Secondary endpoints include the change from baseline in the 6-minute walk test and in the Kansas City Cardiomyopathy Questionnaire (KCCQ) scores, as well as the rates of CV mortality, CV clinical events, and all-cause mortality at Week 120. AKCEA-TTR-LRx is also being studied in patients with polyneuropathy caused by hereditary TTR amyloidosis, or hATTR amyloidosis.

For more information on the Phase 3 NEURO-TTRansform study.