Reported Sunday, BridgeBio Pharma Presented Results From The Exploratory Cardiac Magnetic Resonance Imaging Substudy Of ATTRibute-CM, Its Phase 3 Trial Of Acoramidis In ATTR-CM
Reported Sunday, BridgeBio Pharma Presented Results From The Exploratory Cardiac Magnetic Resonance Imaging Substudy Of ATTRibute-CM, Its Phase 3 Trial Of Acoramidis In ATTR-CM
周日报道，BridgeBio Pharma公布了Attribute-CM的探索性心脏磁共振成像子研究的结果，这是其在ATTR-CM中对阿科拉米德的3期试验
- - In this exploratory substudy, treatment with acoramidis was associated with possible cardiac structural and functional improvement compared with placebo, with potential cardiac amyloid regression
- - The data demonstrate that targeting near-complete transthyretin (TTR) stabilization with acoramidis may enable cardiac remodeling and functional recovery in patients with ATTR-CM
- - These results are the first prospective, longitudinal evaluation of cardiac structure and function by CMR imaging in a double-blind, placebo-controlled, interventional study in ATTR-CM
- - The findings from this study build upon positive results from BridgeBio's global ATTRibute-CM Phase 3 trial, wherein the primary endpoint was met (Win Ratio of 1.8) with a high statistical significance (p<0.0001); the substudy data are consistent with the cardiovascular clinical benefits observed with acoramidis
- - Acoramidis was well-tolerated, with no safety signals of potential clinical concern observed
- - BridgeBio's New Drug Application (NDA) has been accepted by the U.S. Food and Drug Administration (FDA) with a PDUFA action date of November 29, 2024; a Marketing Authorization Application (MAA) for acoramidis has been accepted by the European Medicines Agency (EMA), with an expected decision in 2025
- -在这项探索性子研究中，与安慰剂相比，使用阿科拉米德治疗可能改善心脏结构和功能，并可能出现心脏淀粉样蛋白回归
- -数据表明，使用阿科拉米德靶向近乎完全的甲状腺素（TTR）稳定剂可以实现 ATTR-CM 患者的心脏重塑和功能恢复
- -这些结果是ATTR-CM的一项双盲、安慰剂对照的介入性研究中首次通过CMR成像对心脏结构和功能进行前瞻性的纵向评估
- -这项研究的发现建立在BridgeBio全球Atribie-CM 3期试验的积极结果基础上，该试验达到了主要终点（胜率为1.8），具有很高的统计学意义（p
- -Acoramidis 耐受性良好，未观察到潜在临床问题的安全信号
- -BridgeBio的新药申请（NDA）已获得美国食品药品监督管理局（FDA）的批准，PDUFA的生效日期为2024年11月29日；阿科拉米的上市许可申请（MAA）已被欧洲药品管理局（EMA）接受，预计将在2025年做出决定