AstraZeneca shared encouraging outcomes from the Japan phase 3 trial of BridgeBio-partnered acoramidis, a potential treatment for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).
ATTR-CM is a rare and fatal condition leading to heart failure, with a high fatality rate within four years of diagnosis. Acoramidis, an investigational oral small molecule stabilizer of transthyretin, demonstrated positive results in the ATTRibute-CM trial, meeting the defined success criterion of a greater estimated survival probability at 30 months compared to the placebo.
The trial enrolled 25 patients, with 22 completing the 30-month trial. Acoramidis was administered twice daily, showing positive outcomes in functional measures and quality of life.
The results mirror those of the global ATTRibute-CM phase 3 trial, showcasing improved survival rates, reduced cardiac-related hospitalizations, and enhanced functional measures at 30 months. The trial, designed to support local registration for the rare and fatal condition leading to heart failure, marks a significant step forward in addressing this challenging medical concern.
The drug is being developed and commercialized in Japan through an exclusive license held by Alexion, AstraZeneca Rare Disease, from BridgeBio's affiliate, Eidos Therapeutics. The data will be presented at a forthcoming medical meeting and submitted to Japan’s health authority for regulatory review. BridgeBio submitted its NDA for acoramidis to the U.S. FDA for the treatment of ATTR-CM in December.
If approved, acoramidis will go up against Pfizer’s similarly acting blockbuster, Vyndaqel, which became the first FDA-approved treatments for ATTR-CM in May 2019.