CA3169667A1 - Pyridazine derivatives for modulating nucleic acid splicing - Google Patents

Pyridazine derivatives for modulating nucleic acid splicing Download PDF

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Publication number
CA3169667A1
CA3169667A1 CA3169667A CA3169667A CA3169667A1 CA 3169667 A1 CA3169667 A1 CA 3169667A1 CA 3169667 A CA3169667 A CA 3169667A CA 3169667 A CA3169667 A CA 3169667A CA 3169667 A1 CA3169667 A1 CA 3169667A1
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Prior art keywords
compound
alkyl
heterocyclyl
heteroaryl
aryl
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CA3169667A
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English (en)
French (fr)
Inventor
Dominic Reynolds
Michael Walker SEILER
Anant A. AGRAWAL
Frederic VAILLANCOURT
Peter Smith
Allen T. Hopper
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Remix Therapeutics Inc
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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D403/00Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/14Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing three or more hetero rings
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D401/00Heterocyclic 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
    • C07D401/14Heterocyclic 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D471/00Heterocyclic 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
    • C07D471/02Heterocyclic 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
    • C07D471/10Spiro-condensed systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/50Pyridazines; Hydrogenated pyridazines
    • A61K31/501Pyridazines; Hydrogenated pyridazines not condensed and containing further heterocyclic rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D409/00Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms
    • C07D409/14Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing three or more hetero rings
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D417/00Heterocyclic 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
    • C07D417/02Heterocyclic 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 two hetero rings
    • C07D417/04Heterocyclic 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 two hetero rings directly linked by a ring-member-to-ring-member bond
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D451/00Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof
    • C07D451/02Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof
    • C07D451/04Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof with hetero atoms directly attached in position 3 of the 8-azabicyclo [3.2.1] octane or in position 7 of the 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D451/00Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof
    • C07D451/02Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof
    • C07D451/04Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof with hetero atoms directly attached in position 3 of the 8-azabicyclo [3.2.1] octane or in position 7 of the 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring system
    • C07D451/06Oxygen atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D471/00Heterocyclic 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
    • C07D471/02Heterocyclic 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
    • C07D471/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D471/00Heterocyclic 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
    • C07D471/02Heterocyclic 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
    • C07D471/08Bridged systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02Heterocyclic 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
    • C07D487/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02Heterocyclic 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
    • C07D487/08Bridged systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07BGENERAL METHODS OF ORGANIC CHEMISTRY; APPARATUS THEREFOR
    • C07B2200/00Indexing scheme relating to specific properties of organic compounds
    • C07B2200/05Isotopically modified compounds, e.g. labelled

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Saccharide Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Cosmetics (AREA)
  • Replacement Of Web Rolls (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
CA3169667A 2020-02-28 2021-02-28 Pyridazine derivatives for modulating nucleic acid splicing Pending CA3169667A1 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202062983537P 2020-02-28 2020-02-28
US62/983,537 2020-02-28
US202063007134P 2020-04-08 2020-04-08
US63/007,134 2020-04-08
US202063040474P 2020-06-17 2020-06-17
US63/040,474 2020-06-17
US202063072781P 2020-08-31 2020-08-31
US63/072,781 2020-08-31
US202063126491P 2020-12-16 2020-12-16
US63/126,491 2020-12-16
PCT/US2021/020160 WO2021174170A1 (en) 2020-02-28 2021-02-28 Pyridazine dervatives for modulating nucleic acid splicing

Publications (1)

Publication Number Publication Date
CA3169667A1 true CA3169667A1 (en) 2021-09-02

Family

ID=75278334

Family Applications (2)

Application Number Title Priority Date Filing Date
CA3169667A Pending CA3169667A1 (en) 2020-02-28 2021-02-28 Pyridazine derivatives for modulating nucleic acid splicing
CA3169643A Pending CA3169643A1 (en) 2020-02-28 2021-02-28 Pyridazine derivatives for modulating nucleic acid splicing

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA3169643A Pending CA3169643A1 (en) 2020-02-28 2021-02-28 Pyridazine derivatives for modulating nucleic acid splicing

Country Status (11)

Country Link
US (2) US20230365526A1 (https=)
EP (2) EP4110774A1 (https=)
JP (2) JP2023515617A (https=)
KR (2) KR20220157407A (https=)
CN (2) CN115551847A (https=)
AU (3) AU2021228286A1 (https=)
BR (2) BR112022017089A2 (https=)
CA (2) CA3169667A1 (https=)
IL (2) IL295953A (https=)
MX (2) MX2022010634A (https=)
WO (2) WO2021174176A1 (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019028440A1 (en) 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICING
WO2020163248A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
CN114126613A (zh) 2019-02-05 2022-03-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
EP3920915A4 (en) * 2019-02-05 2022-10-05 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
CN113661162A (zh) 2019-02-06 2021-11-16 斯基霍克疗法公司 用于调节剪接的方法和组合物
WO2020163544A1 (en) 2019-02-06 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
BR112022023025A2 (pt) 2020-05-13 2023-03-28 Chdi Foundation Inc Moduladores de htt para tratar doença de huntington
US20240368163A1 (en) 2021-08-30 2024-11-07 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034836A1 (en) * 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
US20250127789A1 (en) * 2021-11-04 2025-04-24 Skyhawk Therapeutics, Inc. Compositions useful for modulating splicing
US12281116B2 (en) 2021-11-17 2025-04-22 Chdi Foundation, Inc. HTT modulators for treating Huntington's disease
US20250101006A1 (en) * 2022-01-31 2025-03-27 Novartis Ag Process for the synthesis of pyrazolyl derivatives useful as anti-cancer agents
CA3242153A1 (en) * 2022-04-01 2023-10-05 Tyk Medicines, Inc. COMPOUND SERVING AS A KINASE INHIBITOR AND ITS USE
EP4577543A1 (en) * 2022-08-22 2025-07-02 Redx Pharma Limited Pyrido-pyrazoles as inhibitors of ddr's for the treatment of fibrotic disorders and cancer
WO2024182747A2 (en) * 2023-03-01 2024-09-06 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2025211457A1 (ja) * 2024-04-05 2025-10-09 日本新薬株式会社 Cugリピートrnaとmbnlタンパクとの結合阻害化合物および医薬

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EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
IL294124B2 (en) * 2015-12-10 2024-02-01 Ptc Therapeutics Inc Methods for treating huntington's disease
EA201991309A1 (ru) 2016-11-28 2019-11-29 Способы модуляции сплайсинга рнк
WO2018140578A1 (en) * 2017-01-25 2018-08-02 President And Fellows Of Harvard College Non-fullerene solar panel acceptor compounds
KR20200017476A (ko) * 2017-06-14 2020-02-18 피티씨 테라퓨틱스, 인크. Rna 스플라이싱의 변경 방법
WO2019028440A1 (en) * 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICING
KR20200057071A (ko) 2017-09-25 2020-05-25 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱 조절제의 스크리닝 및 확인을 위한 방법 및 조성물
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Publication number Publication date
EP4110774A1 (en) 2023-01-04
CN115551847A (zh) 2022-12-30
JP2023515618A (ja) 2023-04-13
MX2022010637A (es) 2023-01-19
WO2021174176A9 (en) 2022-09-15
IL295954A (en) 2022-10-01
EP4110771A1 (en) 2023-01-04
CN115551843A (zh) 2022-12-30
US20230365526A1 (en) 2023-11-16
WO2021174176A1 (en) 2021-09-02
BR112022017107A2 (pt) 2022-11-16
CA3169643A1 (en) 2021-09-02
US20230140983A1 (en) 2023-05-11
MX2022010634A (es) 2023-01-19
IL295953A (en) 2022-10-01
WO2021174170A1 (en) 2021-09-02
AU2021228286A1 (en) 2022-10-06
KR20220157407A (ko) 2022-11-29
BR112022017089A2 (pt) 2022-11-16
KR20220158236A (ko) 2022-11-30
JP2023515617A (ja) 2023-04-13
WO2021174170A9 (en) 2022-09-15
AU2021228288A1 (en) 2022-09-22
AU2024201568A1 (en) 2024-03-28

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