Poster Presentation The 47th Lorne Conference on Protein Structure and Function 2022

Priming Sunflower Trypsin Inhibitor as a potent inhibitor of Kallikrein 8 (#158)

Alex Duff 1 , Alex Jackson 1 , Cameron Grant 1 , Darren Leahy 1 , Jonathan Harris 1
  1. Queensland University of Technology, Kelvin Grove, QLD, Australia

The serine protease kallikrein 8 (KLK8) is emerging as an important regulator of the neural microenvironment, playing key roles in the regulation of memory1, 2, neural plasticity3, 4, and the development of Alzheimer’s Disease5-7. Despite its attractiveness as a druggable target, there are few KLK8 inhibitors available apart from inhibitory antibodies. Previously the 14 amino acid cyclic peptide sunflower trypsin inhibitor-1 (SFTI-1), has been used as a starting point to design variants to block the activity of a range of serine proteases8-11. Here, we have used SFTI-1 as a scaffold for the development of a series of KLK8 inhibitors, using a combination of in silico molecular dynamics simulations and in vitro kinetics assays to guide substitutions within SFTI-1. Residues at positions P2’ and P5’ were optimised for KLK8 inhibition and selectivity through iterative SFTI diversity libraries of 18 natural amino acids, identifying P2’ Ile – Leu and P5’ Ile – Arg as optimal residues respectively. Incorporation of P2’ Ile – Leu into a P4-TCLR-P1 SFTI optimised for KLK8 binding12 resulted in a 3-fold increase in inhibitory potency from 10.6 nM to 3.1 nM Ki. In contrast, the incorporation of P5’ Ile – Arg resulted in substrate cleavage of the inhibitor and a dramatic decrease of inhibitory potency to 28.0 nM Ki. Though P5’ Arg can result in inhibitor-substrate conversion, molecular dynamics simulations identified similar increases in ranked binding energy, derived from the formation of a stable enzyme-inhibitor hydrogen bond network between P5’ Arg and Glu 97 of KLK8. This approach has allowed for the development of a low nanomolar inhibitor of KLK8 and provides a structural basis for future inhibitor design.

  1. Lu, Z.-x., Huang, Q., and Su, B. (2009) Functional characterization of the human-specific (type II) form of kallikrein 8, a gene involved in learning and memory, Cell Research 19, 259-267.
  2. Konar, A., Kumar, A., Maloney, B., Lahiri, D. K., and Thakur, M. K. (2018) A serine protease KLK8 emerges as a regulator of regulators in memory: Microtubule protein dependent neuronal morphology and PKA-CREB signaling, Sci Rep 8, 9928-9928.
  3. Matsumoto-Miyai, K., Ninomiya, A., Yamasaki, H., Tamura, H., Nakamura, Y., and Shiosaka, S. (2003) NMDA-dependent proteolysis of presynaptic adhesion molecule L1 in the hippocampus by neuropsin, The Journal of neuroscience : the official journal of the Society for Neuroscience 23, 7727-7736.
  4. Tamura, H., Kawata, M., Hamaguchi, S., Ishikawa, Y., and Shiosaka, S. (2012) Processing of neuregulin-1 by neuropsin regulates GABAergic neuron to control neural plasticity of the mouse hippocampus, The Journal of neuroscience : the official journal of the Society for Neuroscience 32, 12657-12672.
  5. Shimizu-Okabe, C., Yousef, G. M., Diamandis, E. P., Yoshida, S., Shiosaka, S., and Fahnestock, M. (2001) Expression of the kallikrein gene family in normal and Alzheimer's disease brain, Neuroreport 12, 2747-2751.
  6. Herring, A., Münster, Y., Akkaya, T., Moghaddam, S., Deinsberger, K., Meyer, J., Zahel, J., Sanchez-Mendoza, E., Wang, Y., Hermann, D. M., Arzberger, T., Teuber-Hanselmann, S., and Keyvani, K. (2016) Kallikrein-8 inhibition attenuates Alzheimer's disease pathology in mice, Alzheimer's & Dementia 12, 1273-1287.
  7. Herring, A., Kurapati, N. K., Krebs, S., Grammon, N., Scholz, L. M., Voss, G., Miah, M. R., Budny, V., Mairinger, F., Haase, K., Teuber-Hanselmann, S., Dobersalske, C., Schramm, S., Jöckel, K. H., Münster, Y., and Keyvani, K. (2020) Genetic knockdown of Klk8 has sex-specific multi-targeted therapeutic effects on Alzheimer's pathology in mice, Neuropathol Appl Neurobiol.
  8. Swedberg, J. E., Nigon, L. V., Reid, J. C., de Veer, S. J., Walpole, C. M., Stephens, C. R., Walsh, T. P., Takayama, T. K., Hooper, J. D., Clements, J. A., Buckle, A. M., and Harris, J. M. (2009) Substrate-Guided Design of a Potent and Selective Kallikrein-Related Peptidase Inhibitor for Kallikrein 4, Chemistry & Biology 16, 633-643.
  9. Li, C. Y., de Veer, S. J., White, A. M., Chen, X., Harris, J. M., Swedberg, J. E., and Craik, D. J. (2019) Amino Acid Scanning at P5′ within the Bowman–Birk Inhibitory Loop Reveals Specificity Trends for Diverse Serine Proteases, Journal of Medicinal Chemistry 62, 3696-3706.
  10. Swedberg, J. E., de Veer, S. J., Sit, K. C., Reboul, C. F., Buckle, A. M., and Harris, J. M. (2011) Mastering the canonical loop of serine protease inhibitors: enhancing potency by optimising the internal hydrogen bond network, PLoS ONE 6, e19302-e19302.
  11. de Veer, S. J., Wang, C. K., Harris, J. M., Craik, D. J., and Swedberg, J. E. (2015) Improving the Selectivity of Engineered Protease Inhibitors: Optimizing the P2 Prime Residue Using a Versatile Cyclic Peptide Library, Journal of Medicinal Chemistry 58, 8257-8268.
  12. Debela, M., Magdolen, V., Skala, W., Elsässer, B., Schneider, E. L., Craik, C. S., Biniossek, M. L., Schilling, O., Bode, W., Brandstetter, H., and Goettig, P. (2018) Structural determinants of specificity and regulation of activity in the allosteric loop network of human KLK8/neuropsin, Sci Rep 8, 10705-10705.