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@ARTICLE{Ammara:304121,
author = {A. Ammara and A. Carone and L. Lucarini and S. Sgambellone
and S. Marri and S. Villano and R. Matucci and G. Luga and
C. Fittipaldi and R. Pecori$^*$ and G. Pieraccini and C. Di
Serio and A. García-Llorca and T. Eysteinsson and S.
Kalinin and J. A. O. Viita and A. Urtti and F. Carta and S.
Selleri and C. T. Supuran},
title = {{R}epurposing {D}rug {M}etabolites into {D}ual
β-{A}drenergic {R}eceptor-{C}arbonic {A}nhydrase
{M}odulators as {P}otential {T}ools for {O}cular
{D}isorders.},
journal = {Journal of medicinal chemistry},
volume = {nn},
issn = {0095-9065},
address = {Washington, DC},
publisher = {ACS},
reportid = {DKFZ-2025-01784},
pages = {nn},
year = {2025},
note = {epub},
abstract = {We report the regioselective chemical derivatization of
(R)-2-((4-aminophenethyl)amino)-1-phenylethan-1-ol, the
primary metabolite of the β3-Adrenergic Receptor (β3-AR)
agonist mirabegron, with prototypical Carbonic Anhydrase
Inhibitors (CAIs) to afford the carbamates 10-14 and the
ureido derivatives 15-18. Such compounds were endowed in
vitro with distinct inhibition profiles for the human (h)
Carbonic Anhydrases (CAs) and showed preferential agonisms
for the β3-AR subtype. Among them, 14 induced remarkable
intraocular pressure (IOP) lowering in an in vivo transient
model of ocular hypertension, with the maximal effect at 120
min post-administration at $1\%$ w/v concentration.
Furthermore, the high stability of the compounds in rabbit
plasma and their ability to induce full vasodilation in
isolated porcine retinal arteries suggested that the
observed in vivo effects likely result from a combination of
conventional aqueous humor reduction and modulation of
ocular vascular tone, both of which are mediated by CAs and
β-ARs. The pronounced melanosomal accumulation of
representative compounds 14 and 16 highlights their
potential as ideal candidates for evaluating pharmacokinetic
profiles in ophthalmic applications. The results of this
study provide strong evidence for the biomedical repurposing
of a neglected metabolite through a novel class of
dual-targeting ligands, also offering a promising strategy
to help counteract the ongoing decline in drug discovery.},
cin = {D150},
ddc = {610},
cid = {I:(DE-He78)D150-20160331},
pnm = {314 - Immunologie und Krebs (POF4-314)},
pid = {G:(DE-HGF)POF4-314},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40857102},
doi = {10.1021/acs.jmedchem.5c01459},
url = {https://inrepo02.dkfz.de/record/304121},
}