Phase III trial of acoramidis in adults


London – Positive high-level results from the Japan Phase III trial of acoramidis in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) showed consistency to those in the global BridgeBio Pharma, Inc. (BridgeBio) ATTRibute-CM Phase III trial (NCT03860935), including survival, cardiac-related hospitalisations and other measures of improved functions (measured by 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.2

Alexion, AstraZeneca Rare Disease, maintains an exclusive licence with BridgeBio’s affiliate, Eidos Therapeutics, Inc. to develop and commercialise acoramidis in Japan. The success criterion in the open-label Phase III trial in Japan was defined as 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 stabilisation 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 stabiliser of transthyretin, designed to achieve maximal stabilisation and preserve native TTR.3 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.