Mirum Pharmaceuticals, Inc. 与因塞特医疗联合宣布，其候选药物Zilurgisertib在治疗进行性骨化性纤维发育不良（FOP）的关键性2期PROGRESS研究中，取得了积极的顶线结果。
该研究达到了其主要终点，显示出统计学上显著且具有临床意义的疗效。与安慰剂相比，接受Zilurgisertib治疗的患者在关键疾病活动指标上表现出显著改善。
安全性方面，Zilurgisertib的耐受性普遍良好，观察到的不良事件与先前研究数据一致。详细的研究结果计划在即将召开的医学会议上公布，并提交同行评议期刊发表。
此次积极数据标志着在治疗这种罕见、致残性遗传疾病方面的一个重要进展。两家公司正与监管机构讨论下一步开发计划。
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