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.
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.
Behind the Buzz: Why Clinical Research Leaders Flock to SCOPE Summit
February 7th 2025In this episode, we meet with Micah Lieberman, Executive Conference Director for SCOPE Summit (Summit for Clinical Ops Executives) at Cambridge Innovation Institute. We will dive deep into the critical role of collaboration within the clinical research ecosystem. How do we bring together diverse stakeholders—sponsors, CROs, clinical trial tech innovators, suppliers, patients, sites, advocacy organizations, investors, and non-profits—to share best practices in trial design, program planning, innovation, and clinical operations? We’ll explore why it’s vital for thought leaders to step beyond their own organizations and learn from others, exchanging ideas that drive advancements in clinical research. Additionally, we’ll discuss the pivotal role of scientific conferences like SCOPE Summit in fostering these essential connections and collaborations, helping shape the future of clinical trials. Join us as we uncover how collective wisdom and cross-industry partnerships are transforming the landscape of clinical research.
Reaching Diverse Patient Populations With Personalized Treatment Methods
January 20th 2025Daejin Abidoye, head of solid tumors, oncology development, AbbVie, discusses a number of topics around diversity in clinical research including industry’s greatest challenges in reaching diverse patient populations, personalized treatment methods, recruitment strategies, and more.
POETYK PsA-2 Trial Shows Efficacy of Sotyktu as an Oral Therapy for Psoriatic Arthritis
March 11th 2025Sotyktu (deucravacitinib) demonstrated significant efficacy in improving psoriatic arthritis symptoms compared to placebo in the Phase III POETYK PsA-2 trial, with a well-tolerated safety profile.