JP2024539655A - グルコセレブロシダーゼ活性の小分子モジュレーターおよびその使用 - Google Patents

グルコセレブロシダーゼ活性の小分子モジュレーターおよびその使用 Download PDF

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JP2024539655A
JP2024539655A JP2024522548A JP2024522548A JP2024539655A JP 2024539655 A JP2024539655 A JP 2024539655A JP 2024522548 A JP2024522548 A JP 2024522548A JP 2024522548 A JP2024522548 A JP 2024522548A JP 2024539655 A JP2024539655 A JP 2024539655A
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acceptable salt
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JP2024539655A5 (enExample
Inventor
ハント，ケビン
ジェン，ジェンビン
シェン，シダ
ブリッティン，ジェレマイア
レイ，ツィカン
シェイカ，グレッグ
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ヴァンキア バイオ，インコーポレイテッド
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Publication of JP2024539655A5 publication Critical patent/JP2024539655A5/ja
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00
    • 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
    • A61K31/438—The ring being spiro-condensed with carbocyclic or heterocyclic ring systems
    • 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
    • A61K31/4427—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems
    • A61K31/444—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring heteroatom, e.g. amrinone
    • 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
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/4965—Non-condensed pyrazines
    • A61K31/497—Non-condensed pyrazines containing further heterocyclic rings
    • 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
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/4985—Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
    • 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
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/506—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
    • 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
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • 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
    • A61K31/535—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
    • 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
    • A61K31/535—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
    • A61K31/5386—1,4-Oxazines, e.g. morpholine spiro-condensed or forming part of bridged ring systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • 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
    • C07D401/14—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 three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D417/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
    • C07D417/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/10—Spiro-condensed systems
    • 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
    • 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
    • C07D487/10—Spiro-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
    • C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
    • C07D491/10—Spiro-condensed systems
    • C07D491/107—Spiro-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D498/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D498/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D498/10—Spiro-condensed systems

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Hydrogenated Pyridines (AREA)
JP2024522548A 2021-10-13 2022-10-12 グルコセレブロシダーゼ活性の小分子モジュレーターおよびその使用 Pending JP2024539655A (ja)

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Application Number Priority Date Filing Date Title
US202163255272P 2021-10-13 2021-10-13
US63/255,272 2021-10-13
PCT/US2022/046386 WO2023064343A1 (en) 2021-10-13 2022-10-12 Small molecule modulators of glucocerebrosidase activity and uses thereof

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JP2024539655A true JP2024539655A (ja) 2024-10-29
JP2024539655A5 JP2024539655A5 (enExample) 2025-10-20

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US (1) US20250034170A1 (enExample)
EP (1) EP4415706A4 (enExample)
JP (1) JP2024539655A (enExample)
KR (1) KR20240102969A (enExample)
CN (1) CN118382436A (enExample)
AR (1) AR127355A1 (enExample)
AU (1) AU2022364718A1 (enExample)
CA (1) CA3235270A1 (enExample)
CL (1) CL2024001109A1 (enExample)
IL (1) IL312086A (enExample)
JO (1) JOP20240081A1 (enExample)
MX (1) MX2024004581A (enExample)
PE (1) PE20242105A1 (enExample)
TW (1) TW202327586A (enExample)
WO (1) WO2023064343A1 (enExample)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024355840A1 (en) * 2023-10-04 2026-03-19 The Board Of Trustees Of The University Of Illinois Fused heterocycles for treating neurodegenerative diseases
CN117659014A (zh) * 2023-12-05 2024-03-08 上海药坦药物研究开发有限公司 5,5-二氟-2,7-二氮杂螺[3.5]壬烷类化合物及其制备方法
WO2025237303A1 (zh) * 2024-05-14 2025-11-20 先声药业有限公司 β-葡萄糖脑苷脂酶变构调节剂、其药物组合物及用途

Citations (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008546820A (ja) * 2005-06-27 2008-12-25 グリュネンタール・ゲゼルシャフト・ミト・ベシュレンクテル・ハフツング 置換された１−オキサ−３，８−ジアザスピロ［４．５］−デカン−２−オン化合物及び医薬の製造へのその使用
JP2009528265A (ja) * 2006-01-24 2009-08-06 シンジェンタ パーティシペーションズ アクチェンゲゼルシャフト 殺虫剤として有用なジアザ−スピロ［４．５］デカン
JP2010503676A (ja) * 2006-09-15 2010-02-04 シェーリング コーポレイション 疼痛、糖尿病および脂質代謝の障害の治療
JP2012511546A (ja) * 2008-12-12 2012-05-24 アボツト・ヘルスケア・プロダクツ・ベー・ブイ Ｋｖ１．３カリウムチャンネル遮断薬としてのスピロアゼパン−オキサゾリジノン
JP2013531037A (ja) * 2010-07-13 2013-08-01 メルク・シャープ・エンド・ドーム・コーポレイション スピロ環式化合物
JP2013544893A (ja) * 2010-12-08 2013-12-19 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ グルコセレブロシダーゼ活性剤としての置換ピラゾロピリミジン類
JP2014508169A (ja) * 2011-03-14 2014-04-03 バーテックス ファーマシューティカルズ インコーポレイテッド イオンチャネルのモジュレーターとしてのモルホリン−スピロ環式ピペリジンアミド
JP2018525345A (ja) * 2015-07-01 2018-09-06 ノースウェスタン ユニバーシティ 置換キナゾリン化合物及びグルコセレブロシダーゼ活性の調節のためのその使用
US20200071334A1 (en) * 2018-08-31 2020-03-05 Northwestern University Pyrrolopyrimidine compounds and uses thereof for modulating glucocerebrosidase activity
JP2020530000A (ja) * 2017-08-02 2020-10-15 ノースウエスタン ユニバーシティ 置換縮合ピリミジン化合物およびその使用
JP2021501782A (ja) * 2017-11-03 2021-01-21 ブリストル−マイヤーズ スクイブ カンパニーＢｒｉｓｔｏｌ−Ｍｙｅｒｓ Ｓｑｕｉｂｂ Ｃｏｍｐａｎｙ ジアザスピロｒｏｃｋ阻害剤
WO2022232383A1 (en) * 2021-04-30 2022-11-03 Vanqua Bio, Inc. Small molecule modulators of glucocerebrosidase activity and uses thereof

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SE0302811D0 (sv) * 2003-10-23 2003-10-23 Astrazeneca Ab Novel compounds
JP2017502961A (ja) * 2013-12-23 2017-01-26 アレクトス セラピューティックス インコーポレイテッド グルコセレブロシダーゼモジュレーターおよびその使用

Patent Citations (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008546820A (ja) * 2005-06-27 2008-12-25 グリュネンタール・ゲゼルシャフト・ミト・ベシュレンクテル・ハフツング 置換された１−オキサ−３，８−ジアザスピロ［４．５］−デカン−２−オン化合物及び医薬の製造へのその使用
JP2009528265A (ja) * 2006-01-24 2009-08-06 シンジェンタ パーティシペーションズ アクチェンゲゼルシャフト 殺虫剤として有用なジアザ−スピロ［４．５］デカン
JP2010503676A (ja) * 2006-09-15 2010-02-04 シェーリング コーポレイション 疼痛、糖尿病および脂質代謝の障害の治療
JP2012511546A (ja) * 2008-12-12 2012-05-24 アボツト・ヘルスケア・プロダクツ・ベー・ブイ Ｋｖ１．３カリウムチャンネル遮断薬としてのスピロアゼパン−オキサゾリジノン
JP2013531037A (ja) * 2010-07-13 2013-08-01 メルク・シャープ・エンド・ドーム・コーポレイション スピロ環式化合物
JP2013544893A (ja) * 2010-12-08 2013-12-19 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ グルコセレブロシダーゼ活性剤としての置換ピラゾロピリミジン類
JP2014508169A (ja) * 2011-03-14 2014-04-03 バーテックス ファーマシューティカルズ インコーポレイテッド イオンチャネルのモジュレーターとしてのモルホリン−スピロ環式ピペリジンアミド
JP2018525345A (ja) * 2015-07-01 2018-09-06 ノースウェスタン ユニバーシティ 置換キナゾリン化合物及びグルコセレブロシダーゼ活性の調節のためのその使用
JP2020530000A (ja) * 2017-08-02 2020-10-15 ノースウエスタン ユニバーシティ 置換縮合ピリミジン化合物およびその使用
JP2021501782A (ja) * 2017-11-03 2021-01-21 ブリストル−マイヤーズ スクイブ カンパニーＢｒｉｓｔｏｌ−Ｍｙｅｒｓ Ｓｑｕｉｂｂ Ｃｏｍｐａｎｙ ジアザスピロｒｏｃｋ阻害剤
US20200071334A1 (en) * 2018-08-31 2020-03-05 Northwestern University Pyrrolopyrimidine compounds and uses thereof for modulating glucocerebrosidase activity
WO2022232383A1 (en) * 2021-04-30 2022-11-03 Vanqua Bio, Inc. Small molecule modulators of glucocerebrosidase activity and uses thereof

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CL2024001109A1 (es) 2024-09-13
US20250034170A1 (en) 2025-01-30
AR127355A1 (es) 2024-01-17
WO2023064343A1 (en) 2023-04-20
PE20242105A1 (es) 2024-10-28
TW202327586A (zh) 2023-07-16
CA3235270A1 (en) 2023-04-20
EP4415706A1 (en) 2024-08-21
KR20240102969A (ko) 2024-07-03
JOP20240081A1 (ar) 2024-04-14
EP4415706A4 (en) 2025-08-27
IL312086A (en) 2024-06-01
CN118382436A (zh) 2024-07-23
AU2022364718A1 (en) 2024-05-16
MX2024004581A (es) 2024-07-10

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