CA2862084C - 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy - Google Patents

1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy Download PDF

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Publication number
CA2862084C
CA2862084C CA2862084A CA2862084A CA2862084C CA 2862084 C CA2862084 C CA 2862084C CA 2862084 A CA2862084 A CA 2862084A CA 2862084 A CA2862084 A CA 2862084A CA 2862084 C CA2862084 C CA 2862084C
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Prior art keywords
isochromen
amino
pyrazin
compound
formula
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CA2862084A
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English (en)
French (fr)
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CA2862084A1 (en
Inventor
Guangming Chen
Amal DAKKA
Gary Mitchell Karp
Chunshi Li
Jana Narasimhan
Nikolai Naryshkin
Maria L. Weetall
Ellen Welch
Xin Zhao
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PTC Therapeutics Inc
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PTC Therapeutics Inc
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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D513/00Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00
    • C07D513/02Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00 in which the condensed system contains two hetero rings
    • C07D513/04Ortho-condensed systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D311/00Heterocyclic compounds containing six-membered rings having one oxygen atom as the only hetero atom, condensed with other rings
    • C07D311/02Heterocyclic compounds containing six-membered rings having one oxygen atom as the only hetero atom, condensed with other rings ortho- or peri-condensed with carbocyclic rings or ring systems
    • C07D311/76Benzo[c]pyrans
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D405/00Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
    • C07D405/02Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings
    • C07D405/04Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D405/00Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
    • C07D405/14Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D407/00Heterocyclic compounds containing two or more hetero rings, at least one ring having oxygen atoms as the only ring hetero atoms, not provided for by group C07D405/00
    • C07D407/02Heterocyclic compounds containing two or more hetero rings, at least one ring having oxygen atoms as the only ring hetero atoms, not provided for by group C07D405/00 containing two hetero rings
    • C07D407/04Heterocyclic compounds containing two or more hetero rings, at least one ring having oxygen atoms as the only ring hetero atoms, not provided for by group C07D405/00 containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • 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
    • C07D409/02Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing two hetero rings
    • C07D409/04Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • 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
    • 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
    • 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
    • C07D497/00Heterocyclic compounds containing in the condensed system at least one hetero ring having oxygen and sulfur atoms as the only ring hetero atoms
    • C07D497/02Heterocyclic compounds containing in the condensed system at least one hetero ring having oxygen and sulfur atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D497/04Ortho-condensed systems

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Pyrane Compounds (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Cosmetics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA2862084A 2012-01-26 2013-01-25 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy Active CA2862084C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261591102P 2012-01-26 2012-01-26
US61/591,102 2012-01-26
PCT/US2013/023067 WO2013112788A1 (en) 2012-01-26 2013-01-25 Compounds for treating spinal muscular atrophy

Publications (2)

Publication Number Publication Date
CA2862084A1 CA2862084A1 (en) 2013-08-01
CA2862084C true CA2862084C (en) 2021-05-11

Family

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Family Applications (1)

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CA2862084A Active CA2862084C (en) 2012-01-26 2013-01-25 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy

Country Status (12)

Country Link
US (1) US9399649B2 (OSRAM)
EP (1) EP2809322B9 (OSRAM)
JP (2) JP6193888B2 (OSRAM)
KR (1) KR102064624B1 (OSRAM)
CN (1) CN104203239B (OSRAM)
BR (1) BR112014018027B1 (OSRAM)
CA (1) CA2862084C (OSRAM)
EA (1) EA028344B1 (OSRAM)
ES (1) ES2733644T3 (OSRAM)
MX (1) MX357834B (OSRAM)
PL (1) PL2809322T3 (OSRAM)
WO (1) WO2013112788A1 (OSRAM)

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BR112014019750B1 (pt) 2012-02-10 2020-03-03 F. Hoffmann-La Roche Ag. Composto, composição farmacêutica e seus usos
MX352962B (es) 2012-03-01 2017-12-15 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal.
US9914722B2 (en) 2012-03-23 2018-03-13 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
WO2014102778A2 (en) * 2012-12-24 2014-07-03 Ramot At Tel-Aviv University Ltd. Agents for treating genetic diseases resulting from nonsense mutations, and methods for identifying the same
KR20160045063A (ko) 2013-08-19 2016-04-26 에프. 호프만-라 로슈 아게 스크리닝 방법
EP3082820B1 (en) 2013-12-19 2022-07-20 PTC Therapeutics, Inc. Methods for modulating the amount of rna transcripts
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US10882868B2 (en) 2014-05-15 2021-01-05 Hoffmann-La Roche Inc. Compounds for treating spinal muscular atrophy
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MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
WO2019005980A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
CN111499615B (zh) 2017-08-04 2024-02-02 斯基霍克疗法公司 用于调节剪接的方法和组合物
AU2019243048A1 (en) 2018-03-27 2020-10-15 Ptc Therapeutics, Inc. Compounds for treating Huntington's disease
BR112020026534A2 (pt) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. Compostos de heteroarila para o tratamento da doença de huntington
EP3814360B8 (en) 2018-06-27 2024-11-06 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
JOP20200338A1 (ar) * 2018-06-27 2020-12-24 Reborna Biosciences Inc عامل وقائي أو علاجي للضمور العضلي نخاعي المنشأ
MX2020014098A (es) 2018-06-27 2021-05-27 Ptc Therapeutics Inc Compuestos heterocíclicos y de heteroarilo para tratar la enfermedad de huntington.
EP3920915A4 (en) 2019-02-05 2022-10-05 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
JP7603595B2 (ja) 2019-02-06 2024-12-20 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
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Also Published As

Publication number Publication date
ES2733644T3 (es) 2019-12-02
JP2015511224A (ja) 2015-04-16
BR112014018027A8 (pt) 2017-07-11
EP2809322B9 (en) 2019-10-30
JP2017171667A (ja) 2017-09-28
BR112014018027B1 (pt) 2020-10-27
MX357834B (es) 2018-07-26
KR20140127271A (ko) 2014-11-03
HK1202069A1 (en) 2015-09-18
CN104203239A (zh) 2014-12-10
PL2809322T3 (pl) 2019-11-29
BR112014018027A2 (OSRAM) 2017-06-20
US9399649B2 (en) 2016-07-26
EP2809322A1 (en) 2014-12-10
EP2809322A4 (en) 2015-12-23
EA201491412A1 (ru) 2014-12-30
CA2862084A1 (en) 2013-08-01
JP6193888B2 (ja) 2017-09-06
EA028344B1 (ru) 2017-11-30
EP2809322B1 (en) 2019-05-15
KR102064624B1 (ko) 2020-01-09
CN104203239B (zh) 2017-09-08
US20150126515A1 (en) 2015-05-07
MX2014008875A (es) 2014-10-14
WO2013112788A1 (en) 2013-08-01

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