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Title: Design of gem-Difluoro-bis-Tetrahydrofuran as P2 Ligand for HIV-1 Protease Inhibitors to Improve Brain Penetration: Synthesis, X-ray Studies, and Biological Evaluation

Journal Article · · ChemMedChem
 [1];  [1];  [1];  [1];  [2];  [2];  [3];  [3];  [3];  [2];  [4]
  1. Purdue Univ., West Lafayette, IN (United States)
  2. Georgia State Univ., Atlanta, GA (United States)
  3. Kumamoto Univ. School of Medicine (Japan)
  4. Kumamoto Univ. School of Medicine (Japan); National Cancer Inst., Bethesda, MD (United States); National Center for Global Health and Medicine, Tokyo (Japan)

The structure–based design, synthesis, biological evaluation, and X–ray structural studies of fluorine–containing HIV–1 protease inhibitors are described. The synthesis of both enantiomers of the gem–difluoro–bis–THF ligands was carried out in a stereoselective manner using a Reformatskii–Claisen reaction as the key step. Optically active ligands were converted into protease inhibitors. Two of these inhibitors, (3R,3aS,6aS)–4,4–difluorohexahydrofuro[2,3–b]furan–3–yl(2S,3R)–3–hydroxy–4–((N–isobutyl–4–methoxyphenyl)sulfonamido)–1–phenylbutan–2–yl) carbamate (3) and (3R,3aS,6aS)–4,4–difluorohexahydrofuro[2,3–b]furan–3–yl(2S,3R)–3–hydroxy–4–((N–isobutyl–4–aminophenyl)sulfonamido)phenylbutan–2–yl) carbamate (4), exhibited HIV–1 protease inhibitory Ki values in the picomolar range. Both 3 and 4 showed very potent antiviral activity, with respective EC50 values of 0.8 and 3.1 nM against the laboratory strain HIV–1LAI. The two inhibitors exhibited better lipophilicity profiles than darunavir, and also showed much improved blood–brain barrier permeability in an in vitro model. A high–resolution X–ray structure of inhibitor 4 in complex with HIV–1 protease was determined, revealing that the fluorinated ligand makes extensive interactions with the S2 subsite of HIV–1 protease, including hydrogen bonding interactions with the protease backbone atoms. Moreover, both fluorine atoms on the bis–THF ligand formed strong interactions with the flap Gly 48 carbonyl oxygen atom.

Research Organization:
Argonne National Laboratory (ANL), Argonne, IL (United States)
Sponsoring Organization:
USDOE Office of Science (SC); US National Inst. of Health; US National Cancer Inst.; Ministry of Education, Culture, Sports, Science, and Technology of Japan; Ministry of Health, Welfare, and Labor of Japan; Cooperative Research Project on Clinical and Epidemiological Studies of Emerging and Reemerging Infectious Diseases (Kumamoto Univ.) of Monbu‐Kagakusho
Grant/Contract Number:
GM53386; GM62920; H15‐AIDS‐001
OSTI ID:
1245842
Journal Information:
ChemMedChem, Vol. 10, Issue 1; ISSN 1860-7179
Publisher:
ChemPubSoc EuropeCopyright Statement
Country of Publication:
United States
Language:
ENGLISH
Citation Metrics:
Cited by: 19 works
Citation information provided by
Web of Science

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Cited By (3)

Nature Inspired Molecular Design: Stereoselective Synthesis of Bicyclic and Polycyclic Ethers for Potent HIV-1 Protease Inhibitors journal June 2018
Design of Highly Potent, Dual-Acting and Central-Nervous-System-Penetrating HIV-1 Protease Inhibitors with Excellent Potency against Multidrug-Resistant HIV-1 Variants journal March 2018
Novel Central Nervous System (CNS)-Targeting Protease Inhibitors for Drug-Resistant HIV Infection and HIV-Associated CNS Complications journal May 2019