AU2016219231B2 - Benzenesulfonamide upregulators of NPC1 for neimann-pick disease and other lysosomal storage disorders - Google Patents

Benzenesulfonamide upregulators of NPC1 for neimann-pick disease and other lysosomal storage disorders Download PDF

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Publication number
AU2016219231B2
AU2016219231B2 AU2016219231A AU2016219231A AU2016219231B2 AU 2016219231 B2 AU2016219231 B2 AU 2016219231B2 AU 2016219231 A AU2016219231 A AU 2016219231A AU 2016219231 A AU2016219231 A AU 2016219231A AU 2016219231 B2 AU2016219231 B2 AU 2016219231B2
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alkyl
alkoxy
halo
phenyl
substituted
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AU2016219231A1 (en
AU2016219231A2 (en
Inventor
Raul Rolando Calvo
Fannie CHEN
Seameen DEHDASTHI
Patricia DRANCHAK
Marc FERRER-ALEGRE
Yiannis Ioannou
Juan Jose Marugan
Samarjit Patnaik
Noel Southall
Mercedes TAYLOR
Wei Zheng
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Icahn School of Medicine at Mount Sinai
National Institutes of Health
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Icahn School of Medicine at Mount Sinai
National Institutes of Health
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C311/00—Amides of sulfonic acids, i.e. compounds having singly-bound oxygen atoms of sulfo groups replaced by nitrogen atoms, not being part of nitro or nitroso groups
    • C07C311/15—Sulfonamides having sulfur atoms of sulfonamide groups bound to carbon atoms of six-membered aromatic rings
    • C07C311/21—Sulfonamides having sulfur atoms of sulfonamide groups bound to carbon atoms of six-membered aromatic rings having the nitrogen atom of at least one of the sulfonamide groups bound to a carbon atom of a six-membered aromatic ring
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/16—Amides, e.g. hydroxamic acids
    • A61K31/165—Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/4164—1,3-Diazoles
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D207/00—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom
    • C07D207/02—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D207/30—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having two double bonds between ring members or between ring members and non-ring members
    • C07D207/32—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having two double bonds between ring members or between ring members and non-ring members with only hydrogen atoms, hydrocarbon or substituted hydrocarbon radicals, directly attached to ring carbon atoms
    • C07D207/33—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having two double bonds between ring members or between ring members and non-ring members with only hydrogen atoms, hydrocarbon or substituted hydrocarbon radicals, directly attached to ring carbon atoms with substituted hydrocarbon radicals, directly attached to ring carbon atoms
    • C07D207/335—Radicals substituted by nitrogen atoms not forming part of a nitro radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D213/00—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members
    • C07D213/02—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members
    • C07D213/04—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D213/24—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom with substituted hydrocarbon radicals attached to ring carbon atoms
    • C07D213/44—Radicals substituted by doubly-bound oxygen, sulfur, or nitrogen atoms, or by two such atoms singly-bound to the same carbon atom
    • C07D213/46—Oxygen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D233/00—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings
    • C07D233/54—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members
    • C07D233/64—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members with substituted hydrocarbon radicals attached to ring carbon atoms, e.g. histidine

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
AU2016219231A 2015-02-11 2016-02-11 Benzenesulfonamide upregulators of NPC1 for neimann-pick disease and other lysosomal storage disorders Active AU2016219231B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562114840P 2015-02-11 2015-02-11
US62/114,840 2015-02-11
PCT/US2016/017504 WO2016130774A1 (en) 2015-02-11 2016-02-11 Benzenesulfonamide upregulators of npc1 for neimann-pick disease and other lysosomal storage disorders

Publications (3)

Publication Number Publication Date
AU2016219231A1 AU2016219231A1 (en) 2017-09-28
AU2016219231A2 AU2016219231A2 (en) 2017-09-28
AU2016219231B2 true AU2016219231B2 (en) 2020-08-20

Family

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AU2016219231A Active AU2016219231B2 (en) 2015-02-11 2016-02-11 Benzenesulfonamide upregulators of NPC1 for neimann-pick disease and other lysosomal storage disorders

Country Status (8)

Country Link
US (1) US10239830B2 (cg-RX-API-DMAC10.html)
EP (1) EP3256116B1 (cg-RX-API-DMAC10.html)
JP (1) JP6887389B2 (cg-RX-API-DMAC10.html)
CN (1) CN107949380B (cg-RX-API-DMAC10.html)
AU (1) AU2016219231B2 (cg-RX-API-DMAC10.html)
CA (1) CA2976449C (cg-RX-API-DMAC10.html)
IL (2) IL253896B (cg-RX-API-DMAC10.html)
WO (1) WO2016130774A1 (cg-RX-API-DMAC10.html)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111289749B (zh) * 2018-12-10 2023-04-14 北京蛋白质组研究中心 C型1类尼曼-匹克蛋白检测物在制备筛查肝细胞癌产品中的应用
WO2021150695A1 (en) * 2020-01-22 2021-07-29 Icahn School Of Medicine At Mount Sinai Constrained n-substituted tetrahydrobenzoazepine sulfonamides as anticancer and neuroprotective agents
WO2022159566A1 (en) 2021-01-21 2022-07-28 Icahn School Of Medicine At Mount Sinai Benzenesulfonamide agonists of trap1 for treating organ fibrosis

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000027823A1 (en) * 1998-11-09 2000-05-18 James Black Foundation Limited Gastrin and cholecystokinin receptor ligands
WO2004035545A2 (en) * 2002-10-18 2004-04-29 E.I. Du Pont De Nemours And Company Azolecarboxamide herbicides

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6177447B1 (en) * 1996-07-15 2001-01-23 Universiteit Van Amsterdam Deoxynojirimycin derivatives and their uses as glucosylceramidase inhibitors
CA2881078C (en) 2004-06-24 2018-10-02 Vertex Pharmaceuticals Incorporated Modulators of atp-binding cassette transporters
US8354427B2 (en) 2004-06-24 2013-01-15 Vertex Pharmaceutical Incorporated Modulators of ATP-binding cassette transporters
MX2008013194A (es) 2006-04-11 2008-12-01 Vertex Pharma Composiciones utiles como inhibidores de canales de sodio regulados por voltaje.
US9732067B2 (en) * 2012-06-20 2017-08-15 University Of Kansas Compounds and methods for activating the apoptotic arm of the unfolded protein response
CA2880880C (en) * 2012-08-03 2021-01-26 John Mckew Cyclodextrin for the treatment of lysosomal storage diseases
US20140128352A1 (en) 2012-09-20 2014-05-08 Buck Institute For Research On Aging Compounds and methods for modulating mitochondrial metabolism and reactive oxygen species production
EP2823816A1 (en) 2013-07-09 2015-01-14 Tragex Pharma Inhibitors of Neuropilin and use thereof for the treatment of Neuropilin-related diseases

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000027823A1 (en) * 1998-11-09 2000-05-18 James Black Foundation Limited Gastrin and cholecystokinin receptor ligands
WO2004035545A2 (en) * 2002-10-18 2004-04-29 E.I. Du Pont De Nemours And Company Azolecarboxamide herbicides

Also Published As

Publication number Publication date
AU2016219231A1 (en) 2017-09-28
US20180044286A1 (en) 2018-02-15
CA2976449A1 (en) 2016-08-18
IL268811A (en) 2019-10-31
AU2016219231A2 (en) 2017-09-28
IL253896B (en) 2019-09-26
CN107949380A (zh) 2018-04-20
JP6887389B2 (ja) 2021-06-16
EP3256116B1 (en) 2022-08-17
EP3256116A1 (en) 2017-12-20
CN107949380B (zh) 2021-07-06
EP3256116A4 (en) 2018-07-11
WO2016130774A1 (en) 2016-08-18
JP2018510904A (ja) 2018-04-19
CA2976449C (en) 2022-08-09
US10239830B2 (en) 2019-03-26
IL253896A0 (en) 2017-10-31

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