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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