Angewandte
Chemie
DOI: 10.1002/anie.201309682
Enzyme Inhibitors
Structure-Based Design of Inhibitors of the Aspartic Protease
Endothiapepsin by Exploiting Dynamic Combinatorial Chemistry**
Milon Mondal, Nedyalka Radeva, Helene Kçster, Ahyoung Park, Constantinos Potamitis,
Maria Zervou, Gerhard Klebe,* and Anna K. H. Hirsch*
Abstract: Structure-based design (SBD) can be used for the
design and/or optimization of new inhibitors for a biological
target. Whereas de novo SBD is rarely used, most reports on
SBD are dealing with the optimization of an initial hit.
Dynamic combinatorial chemistry (DCC) has emerged as
a powerful strategy to identify bioactive ligands given that it
enables the target to direct the synthesis of its strongest binder.
We have designed a library of potential inhibitors (acylhydra-
zones) generated from five aldehydes and five hydrazides and
used DCC to identify the best binder(s). After addition of the
aspartic protease endothiapepsin, we characterized the protein-
bound library member(s) by saturation-transfer difference
NMR spectroscopy. Cocrystallization experiments validated
the predicted binding mode of the two most potent inhibitors,
thus demonstrating that the combination of de novo SBD and
DCC constitutes an efficient starting point for hit identification
and optimization.
from the DCL. Saturation-transfer difference (STD) NMR
spectroscopy, which enables the direct characterization of
target—ligand interactions in solution,[3] has been applied to
identify DCL members bound to the target.[4] In addition to
DCC, structure-based design (SBD) is also a powerful
strategy to design and/or optimize bioactive compounds.[5]
Whereas de novo SBD is rarely used,[6] most reports on SBD
deal with the optimization of an initial hit discovered by other
means. Therefore, the combination of SBD and DCC would
represent a highly efficient hit-identification strategy in
a range of medicinal-chemistry projects. In the present
study, we have used de novo SBD in combination with
1
acylhydrazone-based DCC and H-STD-NMR spectroscopy
to identify a new family of potent hits for endothiapepsin,
which belongs to the notoriously challenging class of aspartic
proteases.[7] Finally, we have validated the proposed binding
mode of the inhibitors by X-ray crystallography.
Endothiapepsin belongs to the family of pepsin-like
aspartic proteases, which are involved in a wide range of
diseases such as hypertension and malaria.[7] Endothiapepsin
has been used as a model enzyme for mechanistic studies[8] as
well as for the development of renin[9] and b-secretase[10]
inhibitors. Eukaryotic aspartic proteases comprise two struc-
turally similar subunits, each contributing an aspartic acid
residue to the catalytic dyad (Asp35 and Asp219 in endo-
thiapepsin) that cleaves the peptide bond of the substrate
using a bound water molecule.
O
ver the past decade, dynamic combinatorial chemistry
(DCC)[1] has emerged as a powerful strategy to identify
ligands for biological targets.[2] DCC allows the formation of
a dynamic combinatorial library (DCL), in which the bonds
between the building blocks are reversible. Upon addition of
a target, one or more library members are bound, thereby
leading to selection and amplification of the strongest binders
[*] M. Mondal, Dr. A. K. H. Hirsch
The formation of an imine-type bond has been applied to
medicinal-chemistry-based DCC projects.[11] In aqueous so-
lution, which is required for any biological application, imines
themselves are inherently too unstable. Acylhydrazones, on
the other hand, offer the right kinetic and thermodynamic
balance.[12] Despite its pH dependence, this type of reversible
linkage has started to attract attention.
This linkage requires the target protein to be stable at
room temperature for one week (pH 7.2),[2c] the use of aniline
as a nucleophilic catalyst (pH 6.2),[13] or an acidic buffer
system (pH < 6). Acylhydrazones are attractive for biological
DCC given that the building blocks are readily available,
afford an amide-type linkage that offers both hydrogen-bond
donor and -acceptor sites for molecular recognition by the
target and are sufficiently stable both at acidic and physio-
logical pH values (see Figure S1 in the Supporting Informa-
tion) to enable direct analysis of the DCL. The optimum
pH value of endothiapepsin is 4.5, and we have shown that the
enzyme is stable under these conditions at room temperature
for more than 20 days (see Figure S2 in the Supporting
Information), thus making it an ideal target enzyme for an
SBD project that exploits acylhydrazone-based DCC.
Stratingh Institute for Chemistry, University of Groningen
Nijenborgh 7, 9747 AG Groningen (The Netherlands)
E-mail: A.K.H.Hirsch@rug.nl
hirsch/
N. Radeva, Dr. H. Kçster, Dr. A. Park, Prof. Dr. G. Klebe
Institute of Pharmaceutical Chemistry
Marbacher Weg 6, 35032 Marburg (Germany)
E-mail: Klebe@Staff.Uni-Marburg.de
Dr. C. Potamitis, Dr. M. Zervou
Institute of Biology, Medicinal Chemistry & Biotechnology
National Hellenic Research Foundation
48,Vas. Constantinou Ave, Athens 11635 (Greece)
[**] We thank Pieter van der Meulen for useful suggestions and
discussions about STD NMR spectroscopy. Funding was granted by
the Netherlands Organisation for Scientific Research (NWO-CW,
VENI grant to A.K.H.H.), by the Dutch Ministry of Education,
Culture, Science (Gravity program 024.001.035) and European
Union’s Seventh Framework Programme (FP7-REGPOT-2009-1)
under grant agreement no. 245866 (to C.P. and M.Z.), and by the
ERC advanced grant no. 268145 DrugProfilBind kindly provided by
the EU.
Supporting information for this article is available on the WWW
Angew. Chem. Int. Ed. 2014, 53, 3259 –3263
ꢀ 2014 Wiley-VCH Verlag GmbH & Co. KGaA, Weinheim
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