阿斯利康在日本针对 acoramidis 进行的 III 期试验（一种治疗罕见致命性心脏病 ATTR-CM 的药物）在生存率、住院率和生活质量方面显示出积极的结果。 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 的 acoramidis 在日本转甲状腺素蛋白介导的淀粉样心肌病 (ATTR-CM) 患者的单组 3 期研究中显示出积极的结果。 BridgeBio Pharma's acoramidis demonstrated positive results in a single-arm Phase 3 study in Japanese patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). 该试验显示 30 个月后功能和生活质量指标有所改善，且未发现任何安全问题。 The trial showed improvement in functions and quality of life measures after 30 months with no safety issues identified. 这些数据将在医学会议上公布，并提交日本卫生当局进行监管审查。 The data will be presented at a medical meeting and submitted for regulatory review by Japan's health authority. Acoramidis 是一种在研、口服、高效小分子转甲状腺素蛋白稳定剂，旨在实现最大程度的稳定并保留天然 TTR。 Acoramidis is an investigational, oral, highly potent small molecule stabilizer of transthyretin, designed to achieve maximal stabilization and preserve native TTR.