AstraZeneca and its partner Ionis Pharmaceuticals have announced that the Phase III CARDIO-TTRansform trial evaluating Wainua (eplontersen) for adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not meet its primary efficacy endpoint, marking a significant setback for the investigational indication.
The global study assessed whether Wainua could reduce the composite outcome of cardiovascular death and recurrent cardiovascular clinical events over a treatment period of up to 140 weeks compared with placebo. While the drug demonstrated a safety profile consistent with previous studies and was generally well tolerated, it failed to achieve a statistically significant improvement in the primary endpoint.
Despite the disappointing outcome, AstraZeneca and Ionis stated that they will continue analyzing the full dataset, including secondary endpoints and predefined subgroup analyses, to better understand the trial results and determine potential next steps with regulatory authorities.
Wainua is already approved in several countries for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The unsuccessful ATTR-CM trial does not affect the medicine's existing approved indication but limits its expansion into the much larger cardiomyopathy patient population.
The CARDIO-TTRansform study enrolled more than 1,400 patients, making it one of the largest clinical trials conducted in ATTR-CM. The complete findings are expected to be presented at a future medical congress and shared with the scientific community.
