Natural Product Splicing Inhibitors: A New Class of Antibody-Drug Conjugate (ADC) Payloads

Bioconjugate Chemistry

Volumn 27, Issue 8, 2016, Pages 1880-1888

  Puthenveetil, Sujiet ^a   Loganzo, Frank ^b   He, Haiyin ^a   Dirico, Ken ^a   Green, Michael ^a   Teske, Jesse ^a,c   Musto, Sylvia ^b   Clark, Tracey ^b   Rago, Brian ^b   Koehn, Frank ^a   Veneziale, Robert ^b,d   Falahaptisheh, Hadi ^b   Han, Xiaogang ^b   Barletta, Frank ^b   Lucas, Judy ^b   Subramanyam, Chakrapani ^a   O'Donnell, Christopher J ^a   Tumey, L Nathan ^a   Sapra, Puja ^b   Gerber, Hans Peter ^b   Ma, Dangshe ^b,e   Graziani, Edmund I ^a   more..

^a GS and T   (United States)

^b PFIZER INC   (United States)

^c ABBVIE INC   (United States)

^d Takeda San Diego Inc   (United States)

^e REGENERON PHARMACEUTICALS INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AMINO ACIDS; CELLS; CYTOLOGY; DISEASES; DRUG THERAPY;

ANTIBODY-DRUG CONJUGATES; CANCER THERAPEUTICS; CYTOTOXIC; DNA DAMAGES; DRUG-RESISTANCE; MICROTUBULES; MODE OF ACTION; NATURAL PRODUCTS; POSSIBLE MECHANISMS; SPLICEOSOMES;

ANTIBODIES;

ACETAMIDE; ANTIBODY CONJUGATE; CARBOXYLIC ACID; CYSTEINE; CYTOTOXIC AGENT; EPIDERMAL GROWTH FACTOR RECEPTOR 2; LYSINE; MULTIDRUG RESISTANCE PROTEIN 1; NATURAL PRODUCT; NATURAL PRODUCT SPLICING INHIBITOR; TRASTUZUMAB; TRASTUZUMAB A THAILANSTATIN CONJUGATE; UNCLASSIFIED DRUG; BIOLOGICAL PRODUCT; MALEIMIDE; MALEIMIDE DERIVATIVE; MULTIDRUG RESISTANCE PROTEIN; PYRAN DERIVATIVE; THAILANSTATIN A;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIGEN EXPRESSION; AREA UNDER THE CURVE; ARTICLE; CANCER CELL LINE; CONJUGATION; CONTROLLED STUDY; CYTOTOXICITY; DIELS ALDER REACTION; DRUG EFFICACY; DRUG METABOLISM; DRUG POTENCY; ELIMINATION HALF-LIFE; IN VITRO STUDY; MOUSE; MULTIDRUG RESISTANCE; NONHUMAN; PLASMA CONCENTRATION-TIME CURVE; STOMACH CANCER; TUMOR XENOGRAFT; ANIMAL; CELL TRANSFORMATION; CHEMISTRY; HUMAN; METABOLISM; TISSUE DISTRIBUTION; TUMOR CELL LINE;

ANIMALS; BIOLOGICAL PRODUCTS; CARBOXYLIC ACIDS; CELL LINE, TUMOR; CELL TRANSFORMATION, NEOPLASTIC; CYSTEINE; HUMANS; IMMUNOCONJUGATES; LYSINE; MALEIMIDES; MICE; P-GLYCOPROTEIN; PYRANS; TISSUE DISTRIBUTION;

EID: 84983486690     PISSN: 10431802     EISSN: 15204812     Source Type: Journal    
DOI: 10.1021/acs.bioconjchem.6b00291     Document Type: Article

References (27)

1. Graziani, E. I. and Tumey, L. N. (2013) Chapter 6. Recent Advances in Antibody-Drug Conjugates, In Biotherapeutics: Recent Developments using Chemical and Molecular Biology, pp 145-175, The Royal Society of Chemistry.
2. Doronina, S. O., Toki, B. E., Torgov, M. Y., Mendelsohn, B. A., Cerveny, C. G., Chace, D. F., DeBlanc, R. L., Gearing, R. P., Bovee, T. D., and Siegall, C. B. et al. 2003, Development of potent monoclonal antibody auristatin conjugates for cancer therapy Nat. Biotechnol. 21, 778 10.1038/nbt832
3. Lewis Phillips, G. D., Li, G., Dugger, D. L., Crocker, L. M., Parsons, K. L., Mai, E., Blattler, W. A., Lambert, J. M., Chari, R. V., and Lutz, R. J. et al. 2008, Targeting HER2-positive breast cancer with trastuzumab-DM1, an antibody-cytotoxic drug conjugate Cancer Res. 68, 9280-9290 10.1158/0008-5472.CAN-08-1776
4. Junttila, M. R., Mao, W., Wang, X., Wang, B.-E., Pham, T., Flygare, J., Yu, S.-F., Yee, S., Goldenberg, D., and Fields, C. et al. 2015, Targeting LGR5+ cells with an antibody-drug conjugate for the treatment of colon cancer Sci. Transl. Med. 7, 314ra186-314ra186 10.1126/scitranslmed.aac7433
5. Moldenhauer, G., Salnikov, A. V., Luettgau, S., Herr, I., Anderl, J., and Faulstich, H. (2012) Therapeutic Potential of Amanitin-Conjugated Anti-Epithelial Cell Adhesion Molecule Monoclonal Antibody Against Pancreatic Carcinoma Journal of the National Cancer Institute 104, 622-634 10.1093/jnci/djs140
6. Dokter, W., Ubink, R., van der Lee, M., van der Vleuten, M., van Achterberg, T., Jacobs, D., Loosveld, E., van den Dobbelsteen, D., Egging, D., and Mattaar, E. et al. 2014, Preclinical Profile of the HER2-Targeting ADC SYD983/SYD985: Introduction of a New Duocarmycin-Based Linker-Drug Platform Mol. Cancer Ther. 13, 2618-2629 10.1158/1535-7163.MCT-14-0040-T
7. Kung Sutherland, M. S., Walter, R. B., Jeffrey, S. C., Burke, P. J., Yu, C., Kostner, H., Stone, I., Ryan, M. C., Sussman, D., and Lyon, R. P. et al. 2013, SGN-CD33A: a novel CD33-targeting antibody-drug conjugate using a pyrrolobenzodiazepine dimer is active in models of drug-resistant AML Blood 122, 1455-1463 10.1182/blood-2013-03-491506
8. He, H., Ratnayake, A. S., Janso, J. E., He, M., Yang, H. Y., Loganzo, F., Shor, B., O'Donnell, C. J., and Koehn, F. E. (2014) Cytotoxic Spliceostatins from Burkholderia sp. and Their Semisynthetic Analogues J. Nat. Prod. 77, 1864 10.1021/np500342m
9. Liu, X., Biswas, S., Berg, M. G., Antapli, C. M., Xie, F., Wang, Q., Tang, M.-C., Tang, G.-L., Zhang, L., and Dreyfuss, G. et al. 2013, Genomics-Guided Discovery of Thailanstatins A, B, and C As Pre-mRNA Splicing Inhibitors and Antiproliferative Agents from Burkholderia thailandensis MSMB43 J. Nat. Prod. 76, 685-693 10.1021/np300913h
10. Kaida, D., Motoyoshi, H., Tashiro, E., Nojima, T., Hagiwara, M., Ishigami, K., Watanabe, H., Kitahara, T., Yoshida, T., and Nakajima, H. et al. 2007, Spliceostatin A targets SF3b and inhibits both splicing and nuclear retention of pre-mRNA Nat. Chem. Biol. 3, 576-583 10.1038/nchembio.2007.18
11. Roybal, G. A. and Jurica, M. S. (2010) Spliceostatin A inhibits spliceosome assembly subsequent to prespliceosome formation Nucleic Acids Res. 38, 6664-6672 10.1093/nar/gkq494
12. Eustaquio, A. S., Chang, L. P., Steele, G. L., O'Donnell, C. J., and Koehn, F. E. (2016) Biosynthetic engineering and fermentation media development leads to gram-scale production of spliceostatin natural products in Burkholderia sp Metab. Eng. 33, 67-75 10.1016/j.ymben.2015.11.003
13. Eustáquio, A. S., Janso, J. E., Ratnayake, A. S., O'Donnell, C. J., and Koehn, F. E. (2014) Spliceostatin hemiketal biosynthesis in Burkholderia spp. is catalyzed by an iron/α-ketoglutarate-dependent dioxygenase Proc. Natl. Acad. Sci. U. S. A. 111, E3376 10.1073/pnas.1408300111
14. Hamblett, K. J., Senter, P. D., Chace, D. F., Sun, M. M. C., Lenox, J., Cerveny, C. G., Kissler, K. M., Bernhardt, S. X., Kopcha, A. K., and Zabinski, R. F. et al. 2004, Effects of drug loading on the antitumor activity of a monoclonal antibody drug conjugate Clin. Cancer Res. 10, 7063-7070 10.1158/1078-0432.CCR-04-0789
15. McDonagh, C. F., Turcott, E., Westendorf, L., Webster, J. B., Alley, S. C., Kim, K., Andreyka, J., Stone, I., Hamblett, K. J., and Francisco, J. A. et al. 2006, Engineered antibody-drug conjugates with defined sites and stoichiometries of drug attachment Protein Eng., Des. Sel. 19, 299-307 10.1093/protein/gzl013
16. Hamann, P. R., Hinman, L. M., Beyer, C. F., Greenberger, L. M., Lin, C., Lindh, D., Menendez, A. T., Wallace, R., Durr, F. E., and Upeslacis, J. (2005) An Anti-MUC1 Antibody-Calicheamicin Conjugate for Treatment of Solid Tumors. Choice of Linker and Overcoming Drug Resistance Bioconjugate Chem. 16, 346-353 10.1021/bc049795f
17. Trail, P. A., Willner, D., and Hellström, K. E. (1995) Site-directed delivery of anthracyclines for treatment of cancer Drug Dev. Res. 34, 196-209 10.1002/ddr.430340209
18. Dokter, W., Ubink, R., van der Lee, M., van der Vleuten, M., van Achterberg, T., Jacobs, D., Loosveld, E., van den Dobbelsteen, D., Egging, D., and Mattaar, E. et al. 2014, Preclinical Profile of the HER2-Targeting ADC SYD983/SYD985: Introduction of a New Duocarmycin-Based Linker-Drug Platform Mol. Cancer Ther. 13, 2618-2629 10.1158/1535-7163.MCT-14-0040-T
19. Jeffrey, S. C., Burke, P. J., Lyon, R. P., Meyer, D. W., Sussman, D., Anderson, M., Hunter, J. H., Leiske, C. I., Miyamoto, J. B., and Nicholas, N. D. et al. 2013, A Potent Anti-CD70 Antibody-Drug Conjugate Combining a Dimeric Pyrrolobenzodiazepine Drug with Site-Specific Conjugation Technology Bioconjugate Chem. 24, 1256-1263 10.1021/bc400217g
20. Eskens, F. A. L. M., Ramos, F. J., Burger, H., O'Brien, J. P., Piera, A., de Jonge, M. J. A., Mizui, Y., Wiemer, E. A. C., Carreras, M. J., Baselga, J., and Tabernero, J. (2013) Phase I Pharmacokinetic and Pharmacodynamic Study of the First-in-Class Spliceosome Inhibitor E7107 in Patients with Advanced Solid Tumors Clin. Cancer Res. 19, 6296-6304 10.1158/1078-0432.CCR-13-0485
21. Alley, S. C., Benjamin, D. R., Jeffrey, S. C., Okeley, N. M., Meyer, D. L., Sanderson, R. J., and Senter, P. D. (2008) Contribution of Linker Stability to the Activities of Anticancer Immunoconjugates Bioconjugate Chem. 19, 759-765 10.1021/bc7004329
22. Zhao, R. Y., Wilhelm, S. D., Audette, C., Jones, G., Leece, B. A., Lazar, A. C., Goldmacher, V. S., Singh, R., Kovtun, Y., and Widdison, W. C. et al. 2011, Synthesis and Evaluation of Hydrophilic Linkers for Antibody-Maytansinoid Conjugates J. Med. Chem. 54, 3606-3623 10.1021/jm2002958
23. Burke, P. J., Senter, P. D., Meyer, D. W., Miyamoto, J. B., Anderson, M., Toki, B. E., Manikumar, G., Wani, M. C., Kroll, D. J., and Jeffrey, S. C. (2009) Design, Synthesis, and Biological Evaluation of Antibody-Drug Conjugates Comprised of Potent Camptothecin Analogues Bioconjugate Chem. 20, 1242-1250 10.1021/bc9001097
24. Eckford, P. D. W. and Sharom, F. J. (2009) ABC Efflux Pump-Based Resistance to Chemotherapy Drugs Chem. Rev. (Washington, DC, U. S.) 109, 2989-3011 10.1021/cr9000226
25. Kovtun, Y. V., Audette, C. A., Mayo, M. F., Jones, G. E., Doherty, H., Maloney, E. K., Erickson, H. K., Sun, X., Wilhelm, S., Ab, O., and J et al. 2010, Antibody-Maytansinoid Conjugates Designed to Bypass Multidrug Resistance Cancer Res. 70, 2528-2537 10.1158/0008-5472.CAN-09-3546
26. Lyon, R. P., Bovee, T. D., Doronina, S. O., Burke, P. J., Hunter, J. H., Neff-LaFord, H. D., Jonas, M., Anderson, M. E., Setter, J. R., and Senter, P. D. (2015) Reducing hydrophobicity of homogeneous antibody-drug conjugates improves pharmacokinetics and therapeutic index Nat. Biotechnol. 33, 733-735 10.1038/nbt.3212
27. Dokter, W., Ubink, R., van der Lee, M., van der Vleuten, M., van Achterberg, T., Jacobs, D., Loosveld, E., van den Dobbelsteen, D., Egging, D., and Mattaar, E. et al. 2014, Preclinical profile of the HER2-targeting ADC SYD983/SYD985: introduction of a new duocarmycin-based linker-drug platform Mol. Cancer Ther. 13, 2618-2629 10.1158/1535-7163.MCT-14-0040-T