Japan ATTR-CM Trial Shows Promise for Acoramidis, Mirroring Global Results

Encouraging Outcomes in Japan Phase III Trial of Acoramidis for Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) Consistent with Global ATTRibute-CM Phase III Trial

Positive high-level results from the Japan Phase III trial of acoramidis in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) showed consistency with those in the global BridgeBio Pharma, Inc. (BridgeBio) ATTRibute-CM Phase III trial (NCT03860935), including survival, cardiac-related hospitalizations, and other measures of improved functions (measured by the six-minute walk test) and quality of life (measured by the Kansas City Cardiomyopathy Questionnaire Overall Summary Score) at 30 months. This trial in Japan was conducted to support local registration.

ATTR-CM is a rare, systemic, progressive, and fatal condition that leads to heart failure and high rates of fatality within four years from diagnosis.

Alexion, AstraZeneca Rare Disease, maintains an exclusive license with BridgeBio’s affiliate, Eidos Therapeutics, Inc. to develop and commercialize acoramidis in Japan. The success criterion in the open-label Phase III trial in Japan was defined as a greater estimated survival probability at 30 months than that observed among placebo patients in ATTRibute-CM.

Professor Yukio Ando, MD, PhD, Department of Amyloidosis Research, Nagasaki International University, Nagasaki, Japan, said: “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. 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.”

Marc Dunoyer, Chief Executive Officer, Alexion, AstraZeneca Rare Disease, said: “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.”

Acoramidis is an investigational, next-generation, oral, highly potent small molecule stabilizer of transthyretin, designed to achieve maximal stabilization and preserve native TTR.

Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified. The data will be presented at a forthcoming medical meeting and submitted to Japan’s health authority for regulatory review.

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