The results from the Japanese trial were on par with the ATTRibute-CM trial in terms of survival rates, cardiac-associated hospitalisations, and improvements in functional and quality of life measures
AstraZeneca announces positive results from Phase 3 ATTR-CM trial of acoramidis in Japan. (Credit: Nappy on Unsplash)
AstraZeneca said that its rare disease arm Alexion’s Phase 3 trial of acoramidis in Japan in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) demonstrated consistency with the ATTRibute-CM Phase 3 trial conducted by BridgeBio Pharma.
Acoramidis is an investigational, next-generation, oral, small molecule stabiliser of transthyretin (TTR). It is designed to achieve maximal stabilisation and preserve native TTR.
Alexion has an exclusive licence with BridgeBio Pharma’s affiliate Eidos Therapeutics to develop and commercialise the drug candidate in Japan.
The open-label, multicentre, prospective trial in Japan involved 25 patients, out of which 22 finished the trial at 30 months. It enrolled patients who had a confirmed diagnosis of ATTR-CM with either wild-type TTR or a variant TTR genotype along with clinical evidence of heart failure.
The participants were administered with acoramidis hydrochloride twice daily at a dose of 800mg. This was continued for an initial treatment phase of 12 months followed by a further 18 months.
The late-stage study’s primary endpoint included the change from baseline to month 12 in a six-minute walk distance. It also consists of the rate of all-cause mortality and frequency of cardiovascular-associated hospitalisation over 30 months.
Following the completion of 30 months of intensive therapy for each participant, statistical analyses were carried out.
The patients who completed this treatment period were eligible to enter a continuous long-term extension period, which is currently in progress.
The results showed consistency with the ATTRibute-CM trial in terms of survival rates, cardiac-associated hospitalisations, and improvements in functional and quality of life measures.
AstraZeneca said that the Japanese trial was specifically designed to support local regulatory submission in Japan.
Alexion, AstraZeneca Rare Disease CEO Marc Dunoyer 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.”