Acoramidis Shows Positive Phase 3 Results in Japanese ATTR-CM Trial, Supporting Regulatory Submission

Feb 05, 2024 09:42 CST Updated 09:42

BridgeBio

Pharmaceutical R&D Developer

AstraZeneca

Biopharmaceutical Manufacturer

▎Edited by the WuXi AppTec content team

Recently, AstraZeneca and BridgeBio Pharma jointly announced positive results from a Phase 3 clinical trial conducted in Japan for the investigational therapy acoramidis. This trial aims to treat patients with transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM).The trial results will support the regulatory application for acoramidis in Japan.

This clinical trial is an open-label Phase 3 clinical trial that enrolled 25 patients, of whom 22 completed the 30-month follow-up. The evaluation criterion for the trial was that the estimated probability of survival at 30 months would be higher than that of the placebo group in the global Phase 3 clinical trial ATTRibute-CM.

The test results showed,No patient deaths were observed at 30 months, and the results for acoramidis in terms of patient survival, heart-related hospitalizations, functional improvement metrics, and quality of life indicators in this Phase 3 clinical trial were consistent with the global Phase 3 clinical trial ATTRibute-CM results.Detailed results will be announced at a future medical conference and submitted to Japanese regulatory authorities.

BridgeBio announced positive results from the ATTRibute-CM trial in July last year.Top-line Results. The clinical results at the 30th month of the study include:

The primary endpoint achieved a highly statistically significant improvement (stratified analysis priority order: all-cause mortality, then frequency of cardiovascular disease-related hospitalizations, then change in N-terminal pro-brain natriuretic peptide from baseline, then change in 6-minute walking distance from baseline), with a Win Ratio of 1.8 (p<0.0001). Unlike the results observed in previous transthyretin (TTR) stabilizer trials,At 30 months, a consistent reduction in the risk of cardiovascular-related hospitalizations was observed across all pre-specified patient subgroups.。
The curves for the time to the first occurrence of all-cause mortality and cardiovascular-related hospitalization began to separate at the 3rd month between the treatment group and the placebo group, continuing until the 30th month, with a hazard ratio of 0.645 (p=0.0008).

▲Mechanism of Action of Acoramidis (Image Source: Reference [3])

Acoramidis is a new-generation, orally administered, highly potent TTR small molecule stabilizer. It is designed to mimic the function of the protective TTR T119M mutation, maintaining the normal tetrameric conformation of TTR protein and preventing the formation of toxic amyloid proteins.

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References:

[1] BridgeBio Pharma Shares Positive Results of Single-Arm Phase 3 Study of Acoramidis in Japanese Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Including No Mortality Reported in the Trial at 30 Months. Retrieved February 2, 2024, from https://www.globenewswire.com/news-release/2024/02/02/2822760/0/en/BridgeBio-Pharma-Shares-Positive-Results-of-Single-Arm-Phase-3-Study-of-Acoramidis-in-Japanese-Patients-with-Transthyretin-Amyloid-Cardiomyopathy-ATTR-CM-Including-No-Mortality-Rep.html

[2] Positive high-level results from Japan Phase III trial of acoramidis in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) showed consistency with global ATTRibute-CM Phase III trial. Retrieved February 2, 2024, from https://www.astrazeneca.com/media-centre/press-releases/2024/positive-results-japan-phase-iii-trial-acoramidis-transthyretin-mediated-amyloid-cardiomyopathy-attr-cm-consistency-global-attribute-cm-phase-iii.html

[3] BBIO - ATTRibute-CM Detailed Results From ESC Congress 2023. Retrieved August 28, 2023 from https://investor.bridgebio.com/static-files/f3645003-5c8f-4ec2-b943-03bbad0c156e

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