Phase III Trial of Acoramidis for Transthyretin Amyloid Cardiomyopathy Provides Positive, High-level Results


Phase III trial of acoramidis in adults with transthyretin-mediated amyloid cardiomyopathy showed positive findings for survival and hospitalizations consistent with those of global BridgeBio Phase III trial.

Image Credit: © Egor -

Image Credit: © Egor -

AstraZeneca has announced positive results from its Japan Phase III trial of acoramidis in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), according to a company press release.1 The results were consistent with those of the BridgeBio ATTRibute-CM global Phase III trial. Among the positive findings were survival, cardiac-related hospitalizations, and other measures of improved functions.

ATTR-CM is a potentially fatal disease of the heart muscle, which is caused by the protein transthyretin building up in the heart, nerves, and other organs. The process can lead to cardiomyopathy and ultimately heart failure. According to the American Heart Association, the most common variant of the disease in the United States occurs in 1 in 25 of all African Americans and in older patients who may be misdiagnosed with high blood pressure-related heart disease.2

“As people living with ATTR-CM are at risk of significant morbidity and mortality, including heart failure, halting disease progression is essential to improving outcomes,” said professor Yukio Ando, MD, PhD, department of amyloidosis research, Nagasaki International University, Nagasaki, Japan, in the release. “These results offer further evidence that TTR stabilization with acoramidis may improve survival and reduce disease severity for patients by preventing further breakdown of these proteins.”

Acoramidis is a novel, next-generation, orally-administered, highly potent, small molecule transthyretin (TTR) stabilizer. The drug was developed to produce maximal stabilization and preserve native TTR, according to AstraZeneca.

The Phase III open-label trial in Japan evaluated the safety and efficacy of acoramidis in Japanese adults with ATTR-CM. The trial enrolled 25 patients, of whom 22 had completed the trial at 30 months. Enrolled patients received does of acoramidis hydrochloride twice per day for an initial treatment period of 12 months followed by an additional 18 months.

Alexion, AstraZeneca Rare Disease, maintains an exclusive license with BridgeBio’s affiliate, Eidos Therapeutics, Inc. to develop and commercialize acoramidis in Japan. Results from the Japan Phase III trial showed greater estimated survival probability at 30 months than that observed among placebo patients in the ATTRibute-CM trial.

“With one of the industry’s largest amyloidosis pipelines exploring multiple therapeutic modalities, we are working to redefine treatment and care as well as offer new hope for this underserved community. These positive results support our ambition to bring acoramidis to people living with ATTR-CM in Japan as soon as possible,” said Marc Dunoyer, CEO, Alexion, AstraZeneca Rare Disease in the press release.


  1. Positive high-level results from Japan Phase III trial of acoramidis in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) showed consistency with global ATTRibute-CM Phase III trial. News release. February 2, 2024. Accessed February 7, 2024.
  2. Transthyretin Amyloid Cardiomyopathy (ATTR-CM). American Heart Association. Accessed February 7, 2024.
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