Review
Copyright ©The Author(s) 2017.
World J Hepatol. Jul 28, 2017; 9(21): 907-920
Published online Jul 28, 2017. doi: 10.4254/wjh.v9.i21.907
Table 1 Targeted drugs under evaluation in advanced hepatocellular carcinoma
DrugMolecular targetStudy designDCRPFSOSTTPTolerabiltyPhase III study
Lenvatinib[46]VEGFR, FGFR, PDGFR,RET, KITPhase I/II (first line)NRNR18.7 mo12.8 moFavorable profileOngoing (E7080)
Cabozantinib[47]VEGFR-2, MET, RETPhase II (second line)68% at 12 wk4.2 moNRNRFavorable profileOngoing (NCT01908426 – CELESTIAL)
Tivantinib[48]c-METPhase II (vs placebo, second line)MET low NS MET high 50% vs 20%NRMET low NS MET high 7.2 mo vs 3.8 mo; P = 0.01MET low NS MET high 2.7 mo vs 1.4 mo; P = 0.04Severe neutropeniaOngoing (NCT01755767)
Apatinib[49]VEGFR2Phase II (first line)NRNR9.7 mo4.2 moFavorable profileOngoing (NCT02329860)
Refametinib[50]MEKPhase II (+ sorafenib)43%1NR290 d1122 d1High incidence of 3/4 grade adverse eventsNR
Foretinib[51]MET, RON, AXL, TIE-2, VEGFRPhase I/II (first line)79%NRNR4.2 moFavorable profileNR
Tepotinib[52]c-METPhase Ib/II (vs sorafenib, first line) - OngoingNRNRNRNRFavorable profileNR
Capmatinib[53]c-METPhase INRNRNRNRFavorable profileNR
Golvantinib[54]c-METPhase I/IIb (+ sorafenib) - OngoingNRNRNRNRFavorable profileNR
Emibetuzumab[55]c-METPhase I (monotp vs emibetuzumab + erlotinib)NRNRNRNRFavorable profileNR
LY2157299[56]TGF-βPhase II (second-line)NRNR36 wk212 wk2Favorable profileOngoing
Pazopanib[57]VEGFR1-3, PDGFRα-β, c-kitPhase INRNRNRNRFavorable profileNR
Axitinib[58]VEGFR1-3Phase II (vs placebo, second line)NR3.6 mo vs 1.9 mo; P = 0.00412.7 mo vs 9.7 mo; P = 0.2873.7 mo vs 1.9 mo; P = 0.006Acceptable profileNR