AstraZeneca's Phase III trial in Japan for acoramidis, a treatment for rare, fatal heart condition ATTR-CM, shows positive results in survival, hospitalizations, and quality of life.

BridgeBio Pharma's acoramidis demonstrated positive results in a single-arm Phase 3 study in Japanese patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). The trial showed improvement in functions and quality of life measures after 30 months with no safety issues identified. The data will be presented at a medical meeting and submitted for regulatory review by Japan's health authority. Acoramidis is an investigational, oral, highly potent small molecule stabilizer of transthyretin, designed to achieve maximal stabilization and preserve native TTR.

February 02, 2024
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