智通财经APP获悉，2025年3月30日，礼来(LLY.US)宣布了lepodisiran在2期临床研究中取得积极结果。Lepodisiran是一种在研的小干扰RNA（siRNA）疗法，旨在降低脂蛋白(a) [Lp(a)]的产生。Lp(a)是遗传性心脏病的危险因素。在2期ALPACA研究中，在最高测试剂量（400mg）治疗后的第60天至第180天期间内，lepodisiran使Lp(a)水平平均降低93.9%，达到了研究主要终点。接受lepodisiran 16mg和96mg剂量的参与者在同一时间内，其Lp(a)水平分别降低了40.8%和75.2%。
Lepodisiran还达成多项次要终点。在为期近18个月的研究中，所有评估时间点均显示，在接受lepodisiran三个测试剂量（16mg、96mg或400mg）的单次或两次给药后，均显示Lp(a)水平的降低。ii该研究设计为在基线和第180天时各给药一次lepodisiran（16mg、96mg或400mg），另设一组在基线时给药lepodisiran 400mg，在第180天给予安慰剂。额外剂量的lepodisiran效果尚未确定。
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