BR112023022524A2 - Moduladores de molécula pequena de atividade de glicocerebrosidase e usos dos mesmos - Google Patents
Moduladores de molécula pequena de atividade de glicocerebrosidase e usos dos mesmosInfo
- Publication number
- BR112023022524A2 BR112023022524A2 BR112023022524A BR112023022524A BR112023022524A2 BR 112023022524 A2 BR112023022524 A2 BR 112023022524A2 BR 112023022524 A BR112023022524 A BR 112023022524A BR 112023022524 A BR112023022524 A BR 112023022524A BR 112023022524 A2 BR112023022524 A2 BR 112023022524A2
- Authority
- BR
- Brazil
- Prior art keywords
- activity
- compounds
- small molecule
- glicocerebrosidase
- molecule modulators
- Prior art date
Links
- 230000000694 effects Effects 0.000 title abstract 3
- 150000003384 small molecules Chemical class 0.000 title abstract 2
- 150000001875 compounds Chemical class 0.000 abstract 4
- 208000015872 Gaucher disease Diseases 0.000 abstract 2
- 102000004547 Glucosylceramidase Human genes 0.000 abstract 2
- 108010017544 Glucosylceramidase Proteins 0.000 abstract 2
- 208000012902 Nervous system disease Diseases 0.000 abstract 2
- 208000025966 Neurological disease Diseases 0.000 abstract 2
- 208000018737 Parkinson disease Diseases 0.000 abstract 2
- 102000004190 Enzymes Human genes 0.000 abstract 1
- 108090000790 Enzymes Proteins 0.000 abstract 1
- 208000037765 diseases and disorders Diseases 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
- C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
- C07D487/04—Ortho-condensed systems
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- 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
- A61K31/4427—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems
- A61K31/4439—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
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- A—HUMAN NECESSITIES
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- 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
- A61K31/445—Non condensed piperidines, e.g. piperocaine
- A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
- A61K31/454—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
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- A—HUMAN NECESSITIES
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- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
- A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
- A61K31/4545—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. pipamperone, anabasine
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- A61K31/46—8-Azabicyclo [3.2.1] octane; Derivatives thereof, e.g. atropine, cocaine
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- A—HUMAN NECESSITIES
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- A61K31/4965—Non-condensed pyrazines
- A61K31/497—Non-condensed pyrazines containing further heterocyclic rings
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- A—HUMAN NECESSITIES
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- A61K31/4985—Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
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- A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
- A61K31/506—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
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- A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
- A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
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- A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
- A61K31/5375—1,4-Oxazines, e.g. morpholine
- A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
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- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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- C—CHEMISTRY; METALLURGY
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- C07D—HETEROCYCLIC COMPOUNDS
- C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
- C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
- C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
- C07D211/08—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms
- C07D211/18—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms
- C07D211/20—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms with hydrocarbon radicals, substituted by singly bound oxygen or sulphur atoms
- C07D211/22—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms with hydrocarbon radicals, substituted by singly bound oxygen or sulphur atoms by oxygen atoms
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- C—CHEMISTRY; METALLURGY
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- C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
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- C07D211/18—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms
- C07D211/26—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms with hydrocarbon radicals, substituted by nitrogen atoms
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- C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
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- C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
- C07D211/40—Oxygen atoms
- C07D211/44—Oxygen atoms attached in position 4
- C07D211/46—Oxygen atoms attached in position 4 having a hydrogen atom as the second substituent in position 4
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- C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
- C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
- C07D211/40—Oxygen atoms
- C07D211/44—Oxygen atoms attached in position 4
- C07D211/52—Oxygen atoms attached in position 4 having an aryl radical as the second substituent in position 4
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- C07D401/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
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- C07D401/04—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings directly linked by a ring-member-to-ring-member bond
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Abstract
moduladores de molécula pequena de atividade de glicocerebrosidase e usos dos mesmos. a presente invenção refere-se a compostos que modulam a glicocerebrosidase (gcase), uma enzima cuja atividade está associada a doenças e distúrbios neurológicos (por exemplo, doença de gaucher, doença de parkinson). também são fornecidas composições farmacêuticas e kits compreendendo os compostos e métodos de tratamento de doenças e distúrbios relacionados à gcase (por exemplo, doença de gaucher, doença de parkinson) com os compostos em um indivíduo, administrando os compostos e/ou composições descritos neste documento.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
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US202163182728P | 2021-04-30 | 2021-04-30 | |
PCT/US2022/026715 WO2022232383A1 (en) | 2021-04-30 | 2022-04-28 | Small molecule modulators of glucocerebrosidase activity and uses thereof |
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BR112023022524A2 true BR112023022524A2 (pt) | 2024-01-02 |
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BR112023022524A BR112023022524A2 (pt) | 2021-04-30 | 2022-04-28 | Moduladores de molécula pequena de atividade de glicocerebrosidase e usos dos mesmos |
Country Status (10)
Country | Link |
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US (1) | US20240262832A1 (pt) |
EP (1) | EP4329881A1 (pt) |
JP (1) | JP2024518000A (pt) |
KR (1) | KR20240005805A (pt) |
CN (1) | CN117642404A (pt) |
AU (1) | AU2022265691A1 (pt) |
BR (1) | BR112023022524A2 (pt) |
CA (1) | CA3218259A1 (pt) |
MX (1) | MX2023012882A (pt) |
WO (1) | WO2022232383A1 (pt) |
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WO2024123967A1 (en) * | 2022-12-08 | 2024-06-13 | Senya Pharmaceuticals, Inc. | Sarm1 inhibitors, pharmaceutical compositions, and therapeutic applications |
WO2024158931A1 (en) * | 2023-01-25 | 2024-08-02 | Epirium Bio Inc. | Pgdh inhibitors and methods of making and using |
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CA2507348C (en) * | 2004-05-13 | 2013-07-16 | The Hospital For Sick Children | Real time methylumbelliferone-based assay |
AU2015342883B2 (en) * | 2014-11-06 | 2020-07-02 | Bial - R&D Investments, S.A. | Substituted pyrrolo(1,2-a)pyrimidines and their use in the treatment of medical disorders |
US10882865B2 (en) * | 2018-08-31 | 2021-01-05 | Northwestern University | Pyrrolopyrimidine compounds and uses thereof for modulating glucocerebrosidase activity |
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2022
- 2022-04-28 WO PCT/US2022/026715 patent/WO2022232383A1/en active Application Filing
- 2022-04-28 CA CA3218259A patent/CA3218259A1/en active Pending
- 2022-04-28 AU AU2022265691A patent/AU2022265691A1/en active Pending
- 2022-04-28 BR BR112023022524A patent/BR112023022524A2/pt unknown
- 2022-04-28 MX MX2023012882A patent/MX2023012882A/es unknown
- 2022-04-28 US US18/288,928 patent/US20240262832A1/en active Pending
- 2022-04-28 JP JP2024513163A patent/JP2024518000A/ja active Pending
- 2022-04-28 EP EP22796720.5A patent/EP4329881A1/en active Pending
- 2022-04-28 CN CN202280046263.9A patent/CN117642404A/zh active Pending
- 2022-04-28 KR KR1020237041098A patent/KR20240005805A/ko unknown
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Publication number | Publication date |
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WO2022232383A1 (en) | 2022-11-03 |
AU2022265691A1 (en) | 2023-11-09 |
US20240262832A1 (en) | 2024-08-08 |
CN117642404A (zh) | 2024-03-01 |
CA3218259A1 (en) | 2022-11-03 |
MX2023012882A (es) | 2024-01-17 |
EP4329881A1 (en) | 2024-03-06 |
KR20240005805A (ko) | 2024-01-12 |
JP2024518000A (ja) | 2024-04-23 |
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