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Drug Evaluation

Ivosidenib: an investigational drug for the treatment of biliary tract cancers

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Pages 301-307 | Received 11 Jan 2021, Accepted 03 Mar 2021, Published online: 26 Apr 2021

References

  • Valle JW, Borbath I, Khan SA, et al. Biliary cancer: ESMO clinical practice guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2016;27:v28–v37.
  • Shaib Y, El-Serag HB. The epidemiology of cholangiocarcinoma. Semin Liver Dis. 2004;24:115–125.
  • Nakeeb A, Pitt HA, Sohn TA, et al. Cholangiocarcinoma. A spectrum of intrahepatic, perihilar, and distal tumors. Ann Surg. 1996;224(4):463–475. discussion 73–5.
  • Bragazzi MC, Cardinale V, Carpino G, et al. Cholangiocarcinoma: epidemiology and risk factors. Translational Gastrointestinal Cancer. >2012;1:21-32.
  • Patel T. Worldwide trends in mortality from biliary tract malignancies. BMC Cancer. 2002;2(1):10.
  • Shin HR, Oh J-K, Masuyer E, et al. Comparison of incidence of intrahepatic and extrahepatic cholangiocarcinoma–focus on East and South-Eastern Asia. Asian Pac J Cancer Prev. 2010;11:1159–1166.
  • Rawla P, Sunkara T, Thandra KC, et al. Epidemiology of gallbladder cancer. Clinical and Experimental Hepatology. 2019;5(2):93–102.
  • Bray F, Ferlay J, Soerjomataram I, et al. Global cancer statistics 2018: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2018;68:394–424.
  • Primrose JN, Fox RP, Palmer DH, et al. Capecitabine compared with observation in resected biliary tract cancer (BILCAP): a randomised, controlled, multicentre, phase 3 study. Lancet Oncol. 2019;20:663–673.
  • Ben-Josef E, Guthrie KA, El-Khoueiry AB, et al. SWOG S0809: a phase ii intergroup trial of adjuvant capecitabine and gemcitabine followed by radiotherapy and concurrent capecitabine in extrahepatic cholangiocarcinoma and gallbladder carcinoma. J Clin Oncol. 2015;33:2617–2622.
  • Sharma A, Dwary AD, Mohanti BK, et al. Best supportive care compared with chemotherapy for unresectable gall bladder cancer: a randomized controlled study. J Clin Oncol. 2010;28:4581–4586.
  • Valle J, Wasan H, Palmer DH, et al. Cisplatin plus gemcitabine versus gemcitabine for biliary tract cancer. N Engl J Med. 2010;362:1273–1281.
  • Okusaka T, Nakachi K, Fukutomi A, et al. Gemcitabine alone or in combination with cisplatin in patients with biliary tract cancer: a comparative multicentre study in Japan. Br J Cancer. 2010;103:469–474.
  • Valle JW, Furuse J, Jitlal M, et al. Cisplatin and gemcitabine for advanced biliary tract cancer: a meta-analysis of two randomised trials. Ann Oncol. 2014;25:391–398.
  • Lamarca A, Jiménez-Fonseca P, Lamarca Á, et al. ABC-06 | a randomised phase III, multi-centre, open-label study of active symptom control (ASC) alone or ASC with oxaliplatin/5-FU chemotherapy (ASC+mFOLFOX) for patients (pts) with locally advanced/metastatic biliary tract cancers (ABC) previously-treated with cisplatin/gemcitabine (CisGem) chemotherapy. J clin oncol. 2019;37:4003.
  • Fostea RM, Fontana E, Torga G, et al. Recent progress in the systemic treatment of advanced/metastatic cholangiocarcinoma. Cancers (Basel) 2020 12 10.3390/cancers12092599
  • Marabelle A, Le DT, Ascierto PA, et al. Efficacy of pembrolizumab in patients with noncolorectal high microsatellite instability/mismatch repair-deficient cancer: results from the phase II KEYNOTE-158 Study. J Clin Oncol. 2020;38:1–10.
  • Boscoe AN, Rolland C, Kelley RK, et al. Frequency and prognostic significance of isocitrate dehydrogenase 1 mutations in cholangiocarcinoma: a systematic literature review. J Gastrointest Oncol. 2019;10:751–765.
  • Saha SK, Parachoniak CA, Ghanta KS, et al. Mutant IDH inhibits HNF-4alpha to block hepatocyte differentiation and promote biliary cancer. Nature. 2014;513:110–114.
  • DiNardo CD, Stein EM, De Botton S, et al. Durable remissions with ivosidenib in IDH1 -mutated relapsed or refractory AML. N Engl J Med. 2018;378(25):2386–2398.
  • Popovici-Muller J, Lemieux RM, Artin E, et al. Discovery of AG-120 (Ivosidenib): a first-in-class mutant idh1 inhibitor for the treatment of IDH1 mutant cancers. ACS Med Chem Lett. 2018;9:300–305.
  • Waitkus MS, Diplas BH, Yan H, et al. Biological role and therapeutic potential of IDH mutations in cancer. Cancer Cell. 2018;34(2):186–195.
  • De Botton S, Mondesir J, Willekens C, et al. IDH1 and IDH2 mutations as novel therapeutic targets: current perspectives. J Blood Med. 2016;7:171–180.
  • Dang L, Su S-SM. Isocitrate dehydrogenase mutation and (R)-2-hydroxyglutarate: from basic discovery to therapeutics development. Annu Rev Biochem. 2017;86(1):305–331.
  • Flavahan WA, Drier Y, Liau BB, et al. Insulator dysfunction and oncogene activation in IDH mutant gliomas. Nature. 2016;529(7584):110–114.
  • Figueroa ME, Abdel-Wahab O, Lu C, et al. Leukemic IDH1 and IDH2 mutations result in a hypermethylation phenotype, disrupt TET2 function, and impair hematopoietic differentiation. Cancer Cell. 2010;18(6):553–567.
  • Wishart DS, Feunang YD, Guo AC, et al., DrugBank 5.0: a major update to the DrugBank database for 2018. Nucleic Acids Res. 46(D1): D1074–D82. 2018.
  • Fan B, Mellinghoff IK, Wen PY, et al. Clinical pharmacokinetics and pharmacodynamics of ivosidenib, an oral, targeted inhibitor of mutant IDH1, in patients with advanced solid tumors. Invest New Drugs. 2020;38(2):433–444.
  • Lowery MA, Burris HA, Janku F, et al. Safety and activity of ivosidenib in patients with IDH1-mutant advanced cholangiocarcinoma: a phase 1 study. Lancet Gastroenterol Hepatol. 2019;4:711–720.
  • Abou-Alfa GK, Macarulla T, Javle MM, et al., Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study. Lancet Oncol. 21(6): 796–807. 2020.
  • Zhu AX, Macarulla T, Javle MM, et al. Final results from ClarIDHy, a global, phase III, randomized, double-blind study of ivosidenib (IVO) versus placebo (PBO) in patients (pts) with previously treated cholangiocarcinoma (CCA) and an isocitrate dehydrogenase 1 (IDH1) mutation. J clin oncol. 2021;39(3_suppl):266.
  • Lamarca A, Barriuso J, McNamara MG, et al. Molecular targeted therapies: ready for “prime time” in biliary tract cancer. J Hepatol. 2020;73:170–185.
  • Jusakul A, Cutcutache I, Yong CH, et al. Whole-genome and epigenomic landscapes of etiologically distinct subtypes of cholangiocarcinoma. Cancer Discov. 2017;7(10):1116–1135.
  • Ueno M, Morizane C, Kawamoto Y, et al. The nationwide cancer genome screening project in Japan, SCRUM-Japan GI-screen: efficient identification of cancer genome alterations in advanced biliary tract cancer. Ann Oncol. abstr 716P 2017;28: 244.
  • Prueksapanich P, Piyachaturawat P, Aumpansub P, et al. Liver fluke-associated biliary tract cancer. Gut Liver. 2018;12(3):236–245.
  • Chan-On W, Nairismägi M-L, Ong CK, et al. Exome sequencing identifies distinct mutational patterns in liver fluke–related and non-infection-related bile duct cancers. Nat Genet. 2013;45(12):1474–1478.
  • Lamarca A, Kapacee Z, Breeze M, et al. Molecular profiling in daily clinical practice: practicalities in advanced cholangiocarcinoma and other biliary tract cancers. J Clin Med 2020 Sep 3;9(9):2854
  • Zill OA, Greene C, Sebisanovic D, et al. Cell-Free DNA next-generation sequencing in pancreatobiliary carcinomas. Cancer Discov. 2015;5:1040–1048.
  • Andersen RF, Jakobsen A. Screening for circulating RAS/RAF mutations by multiplex digital PCR. Clin Chim Acta. 2016;458:138–143.
  • Mody K, Kasi PM, Yang J, et al. Circulating tumor DNA profiling of advanced biliary tract cancers. JCO Precis Oncol. 2019; (3):1–9. 10.1200/PO.18.00324.
  • Ettrich TJ, Schwerdel D, Dolnik A, et al. Genotyping of circulating tumor DNA in cholangiocarcinoma reveals diagnostic and prognostic information. Sci Rep. 2019;9(1):13261.
  • Kumari S, Tewari S, Husain N, et al. Quantification of circulating free DNA as a diagnostic marker in gall bladder cancer. Pathol Oncol Res. 2017;23(1):91–97.
  • Goyal L, Saha SK, Liu LY, et al. Polyclonal secondary FGFR2 mutations drive acquired resistance to fgfr inhibition in patients with FGFR2 fusion–positive cholangiocarcinoma. Cancer Discov. 2017;7(3):252–263.
  • Aguado E, Abou-Alfa GK, Zhu AX, et al. IDH1 mutation detection in plasma circulating tumor DNA (ctDNA) and association with clinical response in patients with advanced intrahepatic cholangiocarcinoma (IHC) from the phase III ClarIDHy study. J clin oncol. 2020;38:4576.
  • Borger DR, Goyal L, Yau T, et al. Circulating oncometabolite 2-hydroxyglutarate is a potential surrogate biomarker in patients with isocitrate dehydrogenase-mutant intrahepatic cholangiocarcinoma. Clin Cancer Res. 2014;20:1884–1890.
  • Heredia V, Mendiola M, Ortiz E, et al. AG-120, a novel IDH1 targeted molecule, inhibits invasion and migration of chondrosarcoma cells in vitro. Ann Oncol. 2017;28:v538.
  • Nicolay B, Narayanaswamy R, Aguado E, et al. EXTH-59. The IDH1 mutant inhibitor AG-120 shows strong inhibition of 2-HG production in an orthotopic IDH1 mutant glioma model in vivo. Neuro Oncol. 2017;19:vi86–vi.
  • Yen K, Chopra VS, Tobin E, et al. Abstract 4956: functional characterization of the ivosidenib (AG-120) and azacitidine combination in a mutant IDH1 AML cell model. Cancer Res. 2018;78:4956.
  • Ivosidenib (AG-120) with nivolumab in IDH1 mutant tumors. editor^, editors”. City,
  • Gemcitabine and cisplatin with ivosidenib or pemigatinib for the treatment of unresectable or metastatic cholangiocarcinoma. editor^, editors”. City
  • Roboz GJ, DiNardo CD, Stein EM, et al. Ivosidenib induces deep durable remissions in patients with newly diagnosed IDH1-mutant acute myeloid leukemia. Blood. 2020;135:463–471.
  • Golub D, Iyengar N, Dogra S, et al. Mutant isocitrate dehydrogenase inhibitors as targeted cancer therapeutics. Front Oncol. 2019;9:417.
  • Intlekofer AM, Shih AH, Wang B, et al. Acquired resistance to IDH inhibition through trans or cis dimer-interface mutations. Nature. 2018;559:125–129.
  • Harding JJ, Lowery MA, Shih AH, et al. Isoform switching as a mechanism of acquired resistance to mutant isocitrate dehydrogenase inhibition. Cancer Discov. 2018;8:1540–1547.

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