Alexion, AstraZeneca Rare Disease has announced positive outcomes from its Japan Phase III clinical trial of acoramidis, an investigational treatment for adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).

The open-label, multicentre, prospective trial is designed to assess the safety and efficacy of acoramidis in Japanese adults diagnosed with ATTR-CM.

The trial involved 25 patients, with 22 completing the 30-month study period.

Participants were selected based on a confirmed diagnosis of ATTR-CM, which could be either wild-type TTR or a variant TTR genotype, along with clinical evidence of heart failure.

They were also required to exhibit Class I-III symptoms, according to the New York Heart Association Functional Classification.

Patients in the trial were administered acoramidis hydrochloride, at a dosage of 800mg twice daily.

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The initial treatment phase lasted 12 months, followed by another 18 months, culminating in a full 30-month treatment duration.

The trial’s primary endpoints included the variation from baseline in six-minute walk distance at month 12, as well as the rate of all-cause mortality and frequency of cardiovascular-associated hospital admission over 30 months.

Statistical assessments were performed post-completion of the 30-month active treatment by all participants.

Those who concluded the treatment phase were given the option to continue in a long-term extension period, which is currently in progress.

Data from the trial demonstrated consistency with the global Phase III ATTRibute-CM trial conducted by BridgeBio Pharma, particularly in survival rates, cardiac-associated hospitalisations, and improvements in functional and quality of life measures.

The Japanese trial was specifically designed to support regional registration efforts.

The success criterion was established as an increased estimated survival chance at 30 months versus placebo patients observed in the ATTRibute-CM trial.

Furthermore, acoramidis was reported to be well-tolerated among trial subjects without any significant safety concerns detected.

Alexion plans to submit the latest data to Japan’s health authority for regulatory consideration.

The company holds an exclusive licence agreement with Eidos Therapeutics, an affiliate of BridgeBio, to develop and commercialise acoramidis 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.”