BridgeBio Pharma has reported positive results from Phase 3 ATTRibute-CM study of acoramidis for patients with transthyretin amyloid cardiomyopathy (ATTR-CM).
Acoramidis is an investigational, next-generation, orally-administered small molecule stabiliser of transthyretin (TTR).
ATTRibute-CM study was designed to assess the efficacy and safety of acoramidis.
In the trial, the TTR stabiliser showed statistically significant improvement in the primary endpoint by a Win Ratio of 1.8.
The on-treatment survival rate was observed at 81% against 74% on placebo. The absolute risk reduction was 6.43% and the relative risk reduction was found at 25%.
Additionally, a highly statistically significant relative risk reduction of 50% was recorded on the frequency of cardiovascular-related hospitalisation.
Furthermore, BridgeBio found a statistically significant treatment effect at 30 months across additional measured markers of morbidity, quality of life, and function.
The investigational drug also showed highly statistically significant and clinically meaningful treatment benefits observed at 30 months on the secondary endpoints of N-terminal prohormone of brain natriuretic peptide (NT-proBNP), KCCQ, and a six-minute walk distance.
The comparative exploratory post hoc analyses, made possible by tafamidis drop-in, revealed that acoramidis increased blood TTR levels by 42% more and improved median NT-proBNP by 92% compared to placebo + tafamidis.
BridgeBio Cardiorenal president and chief medical officer Jonathan Fox said: “Our heartfelt thanks go out to the patients, their caregivers, investigators, and study staff who have actively participated in ATTRibute-CM and continue to contribute to this pivotal research.
“We are extremely encouraged by the robustly positive and consistent findings of the ATTRibute-CM study, which confirm our position that highly potent TTR stabilisation has the potential to profoundly impact patients’ lives. We look forward to presenting the data to health authorities to bring acoramidis to patients as expeditiously as possible.”
The biopharmaceutical company plans to submit its New Drug Application (NDA) to the US Food and Drug Administration (FDA) by the end of this year, and regulatory submissions in additional markets will follow in 2024.
