礼来公司$(LLY)$周日公布了其小干扰RNA (siRNA)候选药物lepodisiran的2期积极结果。试验结果显示，服用最高剂量的候选药物后，该药物将心脏病遗传风险因素的水平降低94%。
Lepodisiran旨在降低一种靶向脂蛋白(a)(也称：Lp(a))的产生，这是一种导致心脏病的遗传风险因素。一些患者的Lp(a)水平持续较低近1.5年。二期试验达到主要终点：最高测试剂量(400毫克)治疗后60至180天内，lepodisiran使脂蛋白(a)水平平均显著降低93.9%，达到主要终点。接受16毫克和96毫克lepodisiran剂量组的受试者，同期脂蛋白(a)水平分别降低40.8%和75.2%。
礼来公司负责糖尿病、肥胖和心脏代谢研究的集团副总裁Ruth Gimeno说：“长期以来，降低高脂蛋白(a)患者的遗传性心血管风险一直是一个严重未满足的需求。这些结果为长期、持久的治疗选择提供了希望。我们将继续在正在进行的3期心血管效果试验中评估lepodisiran的潜在益处。”
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