小中大靶点 适应症 在研药物
PI3K抑制剂 实体瘤、淋巴瘤、白血病 Idelalisib (filed), BKM120 (phase III), BYL719 (phase II), pictilisib (phase II), XL765 (phase II), PF-05212384 (phase II), PF-04691502 (phase II), GS-9820 (phase II), dactolisib (phase II), copanlisib (phase II), GDC-0941 (phase II), GSK2141795 (phase II), RG7422 (phase II), BGT226 (phase I/II), XL147 (phase I), SAR260301 (phase I), GSK2636771 (phase I), GSK2269557 (phase I), GSK2126458 (phase I), GSK1059615 (phase I, failed?), RG7604 (phase I), RG7666 (phase I), AMG 319 (phase I), MLN1117 (phase I), AZD5363 (phase I), AZD8186 (phase I), AZD6482 (phase I)
mTOR抑制剂 实体瘤 MK-8669 (phase II), XL765 (phase II), dactolisib (phase II), PF-05212384 (phase II), PF-04691502 (phase II), BGT226 (phase I/II), GSK1059615 (phase I, failed?), AZD8055 (phase I)
Akt抑制剂 实体瘤 MK-2206 (phase II), ipatasertib (phase II), GSK2110183 (phase I), GSK2141795 (phase I), GSK690693 (phase I, failed?), RG7440 (phase I)
Raf抑制剂 黑素瘤 Vemurafenib (approved), Dabrafenib (approved), LGX818 (phase II), RG7304 (phase I), MLN2480 (phase I)
MEK1/2抑制剂 黑素瘤、卵巢癌 Trametinib (approved), MEK162 (phase III), pimasertib (phase II), RG7167 (phase I), TAK-733 (phase I)
EGFR/HER2抑制剂 非小细胞肺癌、乳腺癌 Gefitinib (approved), erlotinib (approve*******tinib (approved), lapatinib (approved), dacomitinib (phase III, failed), AZD8931 (phase II), AEE788 (phase I/II, failed?), AZD9291 (phase I/II)
FGFR抑制剂 实体瘤 BGJ398 (phase II), LY2874455 (phase II), AZD4547 (phase II)
多靶点RTK抑制剂 实体瘤 TKI258 (phase III), nintedanib (phase III), motesanib (phase III)
Syk抑制剂 类风湿性关节炎、肿瘤 Fostamatinib (phase III, failed), GS-9973 (phase II), TAK-659 (phase I)
Aurora激酶抑制剂 白血病、淋巴瘤 MLN8237 (phase III), AZD1152 (phase II/III), tozasertib (phase II, failed?), AMG 900 (phase I), MK-5108 (phase I, failed?), TAK-901 (phase I, failed?), GSK1070916 (phase I, failed?), PF-03814735 (phase I, failed?)
ALK抑制剂 非小细胞肺癌 Crizotinib (approved), LDK378 (phase III), RG7853 (phase II)
Bcl-2抑制剂 白血病、淋巴瘤 RG7601 (phase II), ABT-199 (phase II), ABT-737 (phase II), ABT-263 (phase II)
HDM2/p53抑制剂 实体瘤 SAR405838 (phase I), RG7388 (phase I), JNJ-26854165 (phase I)
JAK1/2/3抑制剂 类风湿性关节炎、银屑病、骨髓增殖性肿瘤 Tofacitinib (approved), ruxolitinib (approved), momelotinib (phase III), baricitinib (phase III), LY2784544 (phase II), GSK2586184 (phase II), GLPG0634 (phase II), AZD1480 (phase I, failed)
c-Met抑制剂 实体瘤 Crizotinib (approved), foretinib (phase II), LY2801653 (phase II), SAR125844 (phase I), AMG 208 (phase I), AMG 337 (phase I)
HDAC抑制剂 多发性骨髓瘤、淋巴瘤 Vorinostat (approved), romidepsin (approved), LBH589 (phase III), JNJ-26481585 (phase II)
Hedgehog通路抑制剂 基底细胞癌、急性粒细胞白血病 Vismodegib (approved), erismodegib (phase II), BMS-833923 (phase II), LY2940680 (phase II), PF-04449913 (phase I)
ChK1抑制剂 实体瘤、淋巴瘤 MK-8776 (phase II), LY2603618 (phase II, failed), RG7741 (phase I), AZD7762 (phase I, failed)
PARP抑制剂 乳腺癌、卵巢癌 Veliparib (phase III), olaparib (phase III), rucaparib (phase III), niraparib (phase III), AZD2461 (phase I, failed), iniparib (phase III, failed)
CDK抑制剂 乳腺癌 Palbociclib (phase III), dinaciclib (phase III), LEE011 (phase II), LY2835219 (phase II), BAY 1000394 (phase I/II), AZD5438 (phase I, failed?)
p38 MAPK抑制剂 急性冠脉综合征、慢性阻塞性肺病、急性肺损伤、肿瘤 losmapimod (phase II), dilmapimod (phase II), LY2228820 (phase II)
Notch通路抑制剂 实体瘤 MK-0752 (phase I/II, failed?), PF-03084014 (phase I), LY3039478 (phase I)
BACE抑制剂 阿尔兹海默病 LY450139 (phase III, failed), MK-8931 (phase II/III), BMS-708163 (phase II, failed?), LY2811376 (phase I, failed?), AZD3839 (phase I, failed?)
SGLT1/2抑制剂 II型糖尿病 Canagliflozin (approved), dapagliflozin (approved), empagliflozin (filed), ertugliflozin (phase II), LIK066 (phase II), GSK 1614235 (phase I)