AstraZeneca shares positive phase 3 results for acoramidis in ATTR cardiomyopathy
05 Feb 2024
Phase 3Clinical Result
Preview
Source: PMLiVE
AstraZeneca (AZ) has shared positive high-level results from a late-stage trial of acoramidis in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM)transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a rare and progressive condition that leads to heart failure and high rates of mortality within four years of diagnosis.
In ATTR-CM, the transthyretin (TTR) protein that normally circulates in the bloodstream becomes misshapen and builds up in various parts of the body, including the heart, resulting in cardiomyopathy.
Up to 500,000 people worldwide are affected by the condition, but many of those patients remain undiagnosed, AZ said.
Acoramidis is an investigational, next-generation oral small-molecule stabiliser of TTR, designed to achieve maximal stabilisation and preserve native TTR.
Results from the phase 3 trial of the candidate, conducted in Japan, showed consistency with those in the global BridgeBio Pharma ATTRibute-CM phase 3 study, including survival, cardiac-related hospitalisations and other measures of improved functions and quality of life at 30 months.
Acoramidis was well-tolerated, AZ said, adding that the new results will be submitted for regulatory review in Japan, where the company’s rare disease unit, Alexion, maintains an exclusive licence with BridgeBio’s affiliate, Eidos Therapeutics, to develop and commercialise the asset.
Marc Dunoyer, chief executive officer of Alexion, 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.”
Professor Yukio Ando, department of amyloidosis research, Nagasaki International University, added: “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.”
The results come just two weeks after AZ announced positive data from a late-stage study evaluating its immunotherapy Imfinzi (durvalumab) in a subset of patients with hepatocellular carcinoma (HCC), the most common type of liver cancer.
The phase 3 EMERALD-1 trial has been comparing Imfinzi plus transarterial chemoembolisation (TACE), followed by Imfinzi with or without Genentech’s Avastin (bevacizumab), until progression against TACE alone in more than 600 patients with unresectable HCC eligible for embolisation.
For more details,please visit the original website
The content of the article does not represent any opinions of Synapse and its affiliated companies. If there is any copyright infringement or error, please contact us, and we will deal with it within 24 hours.
Organizations
Targets
Drugs
Hot reports
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Leverages most recent intelligence information, enabling fullest potential.