阿斯利康(AZN.US)和默沙东(MRK.US)联合公布KOMET临床3期试验的积极顶线结果

智通财经
13 Nov 2024

智通财经APP获悉,今日,阿斯利康(AZN.US)和默沙东(MRK.US)联合公布KOMET临床3期试验的积极顶线结果。分析显示,两家公司共同开发的Koselugo(selumetinib)用于治疗患有症状性、不可手术丛状神经纤维瘤(PN)的1型神经纤维瘤病(NF1)成人患者时,能够使患者的肿瘤体积显著缩小,达成试验主要终点。

NF1是一种罕见,不可治愈的遗传性疾病。NF1是因为合成神经纤维瘤蛋白(neurofibromin)的NF1基因发生突变引起的。该基因突变可扰乱RAS/MAPK信号通路(RAS-RAF-MEK-ERK),进而导致肿瘤的生长。MEK是RAS/MAPK信号通路中的关键蛋白激酶。Koselugo能够选择性地抑制MEK1和MEK2,从而让失调的信号通路恢复正常,进而缓解NF1患者的病情。

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