JP2023537484A - 嚢胞性線維症膜コンダクタンス制御因子モジュレーター - Google Patents

嚢胞性線維症膜コンダクタンス制御因子モジュレーター Download PDF

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JP2023537484A
JP2023537484A JP2023507621A JP2023507621A JP2023537484A JP 2023537484 A JP2023537484 A JP 2023537484A JP 2023507621 A JP2023507621 A JP 2023507621A JP 2023507621 A JP2023507621 A JP 2023507621A JP 2023537484 A JP2023537484 A JP 2023537484A
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formula
compound
optionally substituted
pharmaceutically acceptable
halogen
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JP2023537484A5 (https=
JPWO2022032068A5 (https=
Inventor
ジェレミー ジェイ． クレメンス，
ブレット シー． ブックサー，
トーマス クリーブランド，
ティモシー アール． クーン，
ミシェル ガラント，
ペーター グルーテンユイス，
ルア， サラ サビナ ハディダ
ジュリー ラテルール，
ヴィート メリロ，
マーク トーマス ミラー，
プラスナ パラセッリ，
イーマン ケー． ラムトフル，
スムクンタ ジャガデースワル レディー，
クラウディオ ストリノ，
リノ バルデス，
ジンラン ジョウ，
ミンソン ベック，
ウィリアム シュルツ ベカラ，
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Vertex Pharmaceuticals Inc
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Vertex Pharmaceuticals Inc
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Publication of JP2023537484A publication Critical patent/JP2023537484A/ja
Publication of JP2023537484A5 publication Critical patent/JP2023537484A5/ja
Publication of JPWO2022032068A5 publication Critical patent/JPWO2022032068A5/ja
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D413/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D413/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings
    • C07D413/04—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • 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
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A61K31/403—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil condensed with carbocyclic rings, e.g. carbazole
    • A61K31/404—Indoles, e.g. pindolol
    • 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
    • A61K31/439—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 the ring forming part of a bridged ring system, e.g. quinuclidine
    • 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
    • A61K31/443—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom
    • 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
    • A61K31/4439—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
    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • 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
    • C07D498/12—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 three hetero rings
    • C07D498/18—Bridged systems

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pulmonology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Oscillators With Electromechanical Resonators (AREA)
  • Amplifiers (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023507621A 2020-08-07 2021-08-06 嚢胞性線維症膜コンダクタンス制御因子モジュレーター Pending JP2023537484A (ja)

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Application Number Priority Date Filing Date Title
US202063063194P 2020-08-07 2020-08-07
US63/063,194 2020-08-07
PCT/US2021/044895 WO2022032068A1 (en) 2020-08-07 2021-08-06 Modulators of cystic fibrosis transmembrane conductance regulator

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JP2023537484A true JP2023537484A (ja) 2023-09-01
JP2023537484A5 JP2023537484A5 (https=) 2024-08-13
JPWO2022032068A5 JPWO2022032068A5 (https=) 2024-08-13

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US (2) US12269831B2 (https=)
EP (1) EP4192829B1 (https=)
JP (1) JP2023537484A (https=)
KR (1) KR20230066348A (https=)
CN (1) CN116171154A (https=)
AR (1) AR123183A1 (https=)
AU (1) AU2021322301A1 (https=)
CA (1) CA3190583A1 (https=)
CL (1) CL2023000375A1 (https=)
CO (1) CO2023002072A2 (https=)
CR (1) CR20230120A (https=)
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DO (1) DOP2023000024A (https=)
EC (1) ECSP23015370A (https=)
ES (1) ES3032183T3 (https=)
FI (1) FI4192829T3 (https=)
GE (2) GEAP202516158A (https=)
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JO (1) JOP20230022A1 (https=)
LT (1) LT4192829T (https=)
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MD (1) MD4192829T2 (https=)
MX (1) MX2023001465A (https=)
PE (1) PE20231377A1 (https=)
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PL (1) PL4192829T3 (https=)
PT (1) PT4192829T (https=)
RS (1) RS66953B1 (https=)
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SM (1) SMT202500214T1 (https=)
TW (1) TWI908847B (https=)
UY (1) UY39374A (https=)
WO (1) WO2022032068A1 (https=)
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JP7214743B2 (ja) 2018-02-15 2023-01-30 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 嚢胞性線維症膜コンダクタンス制御因子のモジュレーターとしての大環状分子、それらの医薬組成物、嚢胞性線維症の治療におけるそれらの使用、及びそれらの製造方法
UY38630A (es) 2019-04-03 2020-10-30 Vertex Pharma Agentes moduladores del regulador de la conductancia transmembrana de la fibrosis quística
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TWI899097B (zh) 2019-08-14 2025-10-01 美商維泰克斯製藥公司 製備cftr調節劑之方法
EP4058434A1 (en) 2019-11-12 2022-09-21 Genzyme Corporation 6-membered heteroarylaminosulfonamides for treating diseases and conditions mediated by deficient cftr activity
CR20230120A (es) 2020-08-07 2023-09-01 Vertex Pharma Moduladores del regulador de la conductancia transmembrana de la fibrosis quística
IL300413A (en) 2020-08-13 2023-04-01 Vertex Pharma Crystal forms of CFTR modulators
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CA3249865A1 (en) 2022-02-03 2023-08-10 Vertex Pharmaceuticals Incorporated PROCESSES FOR PREPARATION AND CRYSTALLINE FORMS OF (6A,12A)-17-AMINO-12-METHYL-6,15-BIS(TRIFLUOROMETHYL)-13,19-DIOXA-3,4,18-TRIAZATRICYCLO[12,3,1,12,5]NONADECA-1(18),2,4,14,16-PENTAEN-6-OL
JP2025506382A (ja) 2022-02-03 2025-03-11 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 嚢胞性線維症の治療方法
JP2025505643A (ja) * 2022-02-08 2025-02-28 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 嚢胞性線維症膜コンダクタンス制御因子の調節因子
CA3258118A1 (en) 2022-04-06 2023-10-12 Vertex Pharmaceuticals Incorporated MODULATORS OF THE TRANSMEMBRANE CONDUCTANCE REGULATOR IN CYSTIC FIBROSIS
JP2025517323A (ja) * 2022-05-16 2025-06-05 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 Ｃｆｔｒモジュレーターとしての大環状化合物の固体形態及びその調製
WO2023224931A1 (en) * 2022-05-16 2023-11-23 Vertex Pharmaceuticals Incorporated Methods of treatment for cystic fibrosis
JP2025530153A (ja) * 2022-09-07 2025-09-11 シオナ セラピューティクス インコーポレイテッド 大環状化合物、組成物、及びそれらの使用方法
WO2024054851A1 (en) * 2022-09-07 2024-03-14 Sionna Therapeutics Macrocyclic compounds, compositions and methods of using thereof
WO2024054845A1 (en) * 2022-09-07 2024-03-14 Sionna Therapeutics Macrocycic compounds, compositions, and methods of using thereof
WO2024097355A1 (en) * 2022-11-02 2024-05-10 Vicinitas Therapeutics, Inc. Deubiquitinase-targeting chimeras and related methods
WO2025076240A1 (en) 2023-10-04 2025-04-10 Vertex Pharmaceuticals Incorporated Solid forms of modulators of cystic fibrosis transmembrane conductance regulator
WO2025076235A1 (en) 2023-10-04 2025-04-10 Vertex Pharmaceuticals Incorporated Modulators of cystic fibrosis transmembrane conductance regulator
WO2025129104A1 (en) * 2023-12-15 2025-06-19 Sionna Therapeutics Inc. Compounds, compositions, and methods of using thereof

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