Akcea Therapeutics and Ionis Pharmaceuticals 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).

"The initiation of our CARDIO-TTRansform Phase 3 trial represents another important milestone further demonstrating Akcea and Ionis’ commitment to bring new treatment options to patients around the world living with TTR amyloidosis. Both the wild type and hereditary forms of ATTR cardiomyopathy are underdiagnosed and potentially fatal with limited treatment options available," said Damien McDevitt, Ph.D., interim chief executive officer at Akcea. "We remain very encouraged by the progress of our clinical studies with AKCEA-TTR-LRx thus far and look forward to working with our team of outstanding global investigators to continue to advance this clinical program in the months ahead."

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 percent at the highest dose.

"TTR amyloidosis is an under-recognized cause of heart failure in many adults. While there have been some advances in standard of care for this disease over the last few years, it remains a significant area of unmet need for patients around the world. One important element of the CARDIO-TTRansform trial is that patients with ATTR cardiomyopathy will be able to continue with approved treatments as we assess AKCEA-TTR-LRx. We have the potential to bring a new range of treatment options to patients living with both the hereditary and wild type forms of this fatal disease," said Stephen Heitner, M.D., associate professor of medicine at Oregon Health and Science University.

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.

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