CN104302181B - 用于治疗脊髓性肌萎缩的化合物 - Google Patents
用于治疗脊髓性肌萎缩的化合物 Download PDFInfo
- Publication number
- CN104302181B CN104302181B CN201380023259.1A CN201380023259A CN104302181B CN 104302181 B CN104302181 B CN 104302181B CN 201380023259 A CN201380023259 A CN 201380023259A CN 104302181 B CN104302181 B CN 104302181B
- Authority
- CN
- China
- Prior art keywords
- pyridin
- pyrano
- alkyl
- formula
- amino
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
- 0 C*=C(C(O1)=**)N=*C1=* Chemical compound C*=C(C(O1)=**)N=*C1=* 0.000 description 17
- GCGVIKJQIRAKHH-UHFFFAOYSA-N CC(C)(C)OC(N(CCC1)CCN1c(cc1)nc(O2)c1C=C(C(C)=O)C2=O)=O Chemical compound CC(C)(C)OC(N(CCC1)CCN1c(cc1)nc(O2)c1C=C(C(C)=O)C2=O)=O GCGVIKJQIRAKHH-UHFFFAOYSA-N 0.000 description 1
- AFVXBFOVAWNVRR-UHFFFAOYSA-N CC(C)(C)OC(N(CCC1)CCN1c(cc1)nc(O2)c1C=C(C(CBr)=O)C2=O)=O Chemical compound CC(C)(C)OC(N(CCC1)CCN1c(cc1)nc(O2)c1C=C(C(CBr)=O)C2=O)=O AFVXBFOVAWNVRR-UHFFFAOYSA-N 0.000 description 1
- DJEXPSNGMUCDQG-UHFFFAOYSA-N CC(C)(C)OC(N(CCC1)CCN1c(cc1)nc(O2)c1C=C(c1c[n](cc[s]3)c3n1)C2=O)=O Chemical compound CC(C)(C)OC(N(CCC1)CCN1c(cc1)nc(O2)c1C=C(c1c[n](cc[s]3)c3n1)C2=O)=O DJEXPSNGMUCDQG-UHFFFAOYSA-N 0.000 description 1
- BRPQKOMBXSQDBC-UHFFFAOYSA-N CC(C)(C)OC(N(CCC1)CCN1c1ccc(C=O)c(O)n1)=O Chemical compound CC(C)(C)OC(N(CCC1)CCN1c1ccc(C=O)c(O)n1)=O BRPQKOMBXSQDBC-UHFFFAOYSA-N 0.000 description 1
- CUDLQBVFNTTZCL-UHFFFAOYSA-N CN(CCC1)CCN1c(cc1)nc(O2)c1C=C(c1c[n](cc[s]3)c3n1)C2=O Chemical compound CN(CCC1)CCN1c(cc1)nc(O2)c1C=C(c1c[n](cc[s]3)c3n1)C2=O CUDLQBVFNTTZCL-UHFFFAOYSA-N 0.000 description 1
- GUGNZJPYKLZTEV-UHFFFAOYSA-N O=C1Oc(nc(cc2)N3CCNCCC3)c2C=C1c1c[n](cc[s]2)c2n1 Chemical compound O=C1Oc(nc(cc2)N3CCNCCC3)c2C=C1c1c[n](cc[s]2)c2n1 GUGNZJPYKLZTEV-UHFFFAOYSA-N 0.000 description 1
Classifications
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular 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
- 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
- C07D491/04—Ortho-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
- C07D491/04—Ortho-condensed systems
- C07D491/044—Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring
- C07D491/052—Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring the oxygen-containing ring being six-membered
Landscapes
- 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)
- Physical Education & Sports Medicine (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Nitrogen Condensed Heterocyclic Rings (AREA)
- Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Cosmetics (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261605487P | 2012-03-01 | 2012-03-01 | |
| US61/605,487 | 2012-03-01 | ||
| PCT/US2013/028131 WO2013130689A1 (en) | 2012-03-01 | 2013-02-28 | Compounds for treating spinal muscular atrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN104302181A CN104302181A (zh) | 2015-01-21 |
| CN104302181B true CN104302181B (zh) | 2017-09-15 |
Family
ID=49083256
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201380023259.1A Active CN104302181B (zh) | 2012-03-01 | 2013-02-28 | 用于治疗脊髓性肌萎缩的化合物 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US9371336B2 (https=) |
| EP (1) | EP2819519B1 (https=) |
| JP (1) | JP6092264B2 (https=) |
| KR (1) | KR102099997B1 (https=) |
| CN (1) | CN104302181B (https=) |
| BR (1) | BR112014021531B1 (https=) |
| CA (1) | CA2865957C (https=) |
| EA (1) | EA029155B1 (https=) |
| MX (1) | MX352962B (https=) |
| WO (1) | WO2013130689A1 (https=) |
Families Citing this family (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX352861B (es) | 2011-12-30 | 2017-12-13 | Ptc Therapeutics Inc | Compuestos para tratar la atrofia muscular espinal. |
| MX357834B (es) | 2012-01-26 | 2018-07-26 | Ptc Therapeutics Inc | Compuestos de tipo isocumarina sustituidos para tratar la atrofia muscular espinal. |
| EP2812004B1 (en) | 2012-02-10 | 2018-06-27 | PTC Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| CA2868026C (en) | 2012-03-23 | 2021-02-16 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| CA2894992A1 (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 |
| EP3035935B1 (en) | 2013-08-19 | 2020-03-11 | F. Hoffmann-La Roche AG | Compounds for use in the prophylaxis and treatment of cancer |
| US10195202B2 (en) | 2013-12-19 | 2019-02-05 | Ptc Therapeutics, Inc. | Methods for modulating the amount of RNA transcripts |
| US10668171B2 (en) | 2015-05-30 | 2020-06-02 | Ptc Therapeutics, Inc. | Methods for modulating RNA splicing |
| EP3133065A1 (en) | 2015-08-21 | 2017-02-22 | Merck Patent GmbH | Compounds for optically active devices |
| EA201800367A1 (ru) | 2015-12-10 | 2019-02-28 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Способы лечения болезни хантингтона |
| JP2019535789A (ja) | 2016-11-28 | 2019-12-12 | ピーティーシー セラピューティクス，インコーポレーテッド | Ｒｎａスプライシングを調節する方法 |
| EP3363786A1 (en) | 2017-02-15 | 2018-08-22 | Merck Patent GmbH | Compounds for optically active devices |
| EP3363793A1 (en) | 2017-02-15 | 2018-08-22 | Merck Patent GmbH | Hydrophobic compounds for optically active devices |
| EP3634953B1 (en) | 2017-06-05 | 2024-01-03 | PTC Therapeutics, Inc. | Compounds for treating huntington's disease |
| JP2020523365A (ja) | 2017-06-14 | 2020-08-06 | ピーティーシー セラピューティクス，インコーポレーテッド | Ｒｎａスプライシングを改変する方法 |
| CN111182898B (zh) | 2017-06-28 | 2024-04-16 | Ptc医疗公司 | 用于治疗亨廷顿氏病的方法 |
| MX2019015580A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
| JP7312749B2 (ja) | 2017-08-04 | 2023-07-21 | 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 | スプライシングをモジュレートする方法および組成物 |
| EA202092001A1 (ru) | 2018-03-27 | 2021-01-29 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Соединения для лечения болезни гентингтона |
| CN112469707B (zh) | 2018-06-27 | 2024-06-21 | 利伯纳生物科学株式会社 | 脊髓性肌萎缩症的预防剂或治疗剂 |
| EP3814360B8 (en) | 2018-06-27 | 2024-11-06 | PTC Therapeutics, Inc. | Heteroaryl compounds for treating huntington's disease |
| HRP20240935T1 (hr) | 2018-06-27 | 2024-11-22 | Ptc Therapeutics, Inc. | Heterociklični i heteroaril spojevi za liječenje huntingtonove bolesti |
| WO2020005877A1 (en) | 2018-06-27 | 2020-01-02 | Ptc Therapeutics, Inc. | Heteroaryl compounds for treating huntington's disease |
| WO2020163405A1 (en) | 2019-02-05 | 2020-08-13 | Skyhawk Therapeutics, Inc. | Methods and compositions for modulating splicing |
| JP7551629B2 (ja) | 2019-02-05 | 2024-09-17 | 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 |
| 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 |
| JP7603594B2 (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 |
| JP7649257B2 (ja) | 2019-05-13 | 2025-03-19 | ピーティーシー セラピューティクス， インコーポレイテッド | ハンチントン病を処置するための化合物 |
| US11129829B2 (en) | 2019-06-17 | 2021-09-28 | Skyhawk Therapeutics, Inc. | Methods for modulating splicing |
| CN111116460A (zh) * | 2019-12-31 | 2020-05-08 | 阿里生物新材料（常州）有限公司 | 一种2-氯-4-羟基吡啶-3-甲醛氢溴酸盐的合成方法 |
| JP7763397B2 (ja) | 2020-05-20 | 2025-11-04 | エイエムオー・アイルランド | 光学活性装置で使用するためのアザクマリン及びアザチオクマリン誘導体 |
| WO2023114539A1 (en) * | 2021-12-17 | 2023-06-22 | Pretzel Therapeutics, Inc. | Amino chromen-2-one modulators of polrmt |
| AU2024286618A1 (en) * | 2023-06-09 | 2025-10-30 | F. Hoffmann-La Roche Ag | Inducible gene expression system |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2005046698A1 (en) * | 2003-11-10 | 2005-05-26 | Synta Pharmaceuticals, Corp. | Fused heterocyclic compounds |
| CN1628114A (zh) * | 2002-03-06 | 2005-06-15 | 诺瓦提斯公司 | 用作生物标记物的香豆素类化合物 |
| WO2009042907A1 (en) * | 2007-09-27 | 2009-04-02 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isoindoline compounds for the treatment of spinal muscular atrophy and other uses |
| WO2009151546A2 (en) * | 2008-05-27 | 2009-12-17 | Ptc Therapeutics, Inc. | Methods for treating spinal muscular atrophy |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| US3558618A (en) | 1968-04-01 | 1971-01-26 | Dow Chemical Co | Novel 4h-pyrazino(1,2-a)pyrimidine-4-ones |
| US4122274A (en) | 1977-05-25 | 1978-10-24 | Bristol-Myers Company | 3-Tetrazolo-5,6,7,8-substituted-pyrido[1,2-a]pyrimidin-4-ones |
| US4342870A (en) | 1980-03-28 | 1982-08-03 | Janssen Pharmaceutica N.V. | Novel 3-(1-piperidinylalkyl)-4H-pyrido[1,2-a]pyrimidin-4-one derivatives |
| US5089633A (en) | 1987-04-28 | 1992-02-18 | Georgia Tech Research Corporation | Substituted isocoumarins |
| US5726182A (en) | 1990-05-02 | 1998-03-10 | Abbott Laboratories | Quinolizinone type compounds |
| AU4231293A (en) | 1992-05-13 | 1993-12-13 | E.I. Du Pont De Nemours And Company | Substituted pyrido(1,2-A)pyrimidinone derivatives as fungicides |
| US5597922A (en) * | 1994-07-29 | 1997-01-28 | State Of Oregon, Acting By And Through The Oregon State Board Of Higher Education, Acting For And On Behalf Of The Oregon Health Sciences University And The University Of Oregon | Glycine receptor antagonist pharmacophore |
| US6630488B1 (en) | 1998-09-21 | 2003-10-07 | Biochem Pharma, Inc. | Quinolizinones as integrin inhibitors |
| EP1857443B1 (en) | 2000-01-24 | 2012-03-28 | AstraZeneca AB | Therapeutic morpholino-substituted compounds |
| WO2003104216A1 (en) | 2002-06-10 | 2003-12-18 | Acadia Pharmaceuticals Inc. | Urotensin ii receptor modulators |
| WO2004047766A2 (en) | 2002-11-25 | 2004-06-10 | Kaloidis Antonia C | Treatment for sma disease |
| US9068234B2 (en) | 2003-01-21 | 2015-06-30 | Ptc Therapeutics, Inc. | Methods and agents for screening for compounds capable of modulating gene expression |
| WO2004113335A2 (en) | 2003-06-20 | 2004-12-29 | Chiron Corporation | Pyridino[1,2-a]pyrimidin-4-one compounds as anticancer agents |
| US20050046698A1 (en) * | 2003-09-02 | 2005-03-03 | Knight Andrew Frederick | System and method for producing a selectable view of an object space |
| ATE373659T1 (de) | 2004-05-04 | 2007-10-15 | Warner Lambert Co | Pyrrolylsubstituierte pyrido(2,3-d)pyrimidin-7- one und derivate davon als therapeutische mittel |
| WO2006045010A2 (en) | 2004-10-20 | 2006-04-27 | Resverlogix Corp. | Stilbenes and chalcones for the prevention and treatment of cardiovascular diseases |
| CA2595477A1 (en) | 2005-01-21 | 2006-07-27 | Janssen Pharmaceutica N.V. | Novel heterocyclic benzo[c]chromene derivatives useful as modulators of the estrogen receptors |
| AR059339A1 (es) | 2006-02-09 | 2008-03-26 | Chugai Pharmaceutical Co Ltd | Derivados de la cumarina para trastornos proliferativos de celulas, composicion farmaceutica y agente terapeutico que los contiene |
| WO2008013997A2 (en) | 2006-07-27 | 2008-01-31 | The Trustees Of Columbia University In The City Of New York | Fluorescent substrates for monoamine transporters as optical false neurotransmitters |
| US8932818B2 (en) | 2008-08-13 | 2015-01-13 | Ptc Therapeutics, Inc. | Screening assays for compounds that modulate programmed ribosomal frameshifting |
| EP2501231B1 (en) | 2009-11-20 | 2016-12-21 | Merck Sharp & Dohme Corp. | Quinolizidinone carboxamide m1 receptor positive allosteric modulators |
| CN102812023A (zh) | 2010-01-13 | 2012-12-05 | 韩国巴斯德研究所 | 抗感染吡啶并(1,2-a)嘧啶类 |
| MX352861B (es) | 2011-12-30 | 2017-12-13 | Ptc Therapeutics Inc | Compuestos para tratar la atrofia muscular espinal. |
| MX357834B (es) | 2012-01-26 | 2018-07-26 | Ptc Therapeutics Inc | Compuestos de tipo isocumarina sustituidos para tratar la atrofia muscular espinal. |
| EP2812004B1 (en) | 2012-02-10 | 2018-06-27 | PTC Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| CA2868026C (en) | 2012-03-23 | 2021-02-16 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
-
2013
- 2013-02-28 CA CA2865957A patent/CA2865957C/en active Active
- 2013-02-28 MX MX2014010406A patent/MX352962B/es active IP Right Grant
- 2013-02-28 JP JP2014560007A patent/JP6092264B2/ja active Active
- 2013-02-28 BR BR112014021531-6A patent/BR112014021531B1/pt active IP Right Grant
- 2013-02-28 EP EP13755032.3A patent/EP2819519B1/en active Active
- 2013-02-28 CN CN201380023259.1A patent/CN104302181B/zh active Active
- 2013-02-28 US US14/380,385 patent/US9371336B2/en active Active
- 2013-02-28 KR KR1020147027577A patent/KR102099997B1/ko active Active
- 2013-02-28 WO PCT/US2013/028131 patent/WO2013130689A1/en not_active Ceased
- 2013-02-28 EA EA201491617A patent/EA029155B1/ru not_active IP Right Cessation
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN1628114A (zh) * | 2002-03-06 | 2005-06-15 | 诺瓦提斯公司 | 用作生物标记物的香豆素类化合物 |
| WO2005046698A1 (en) * | 2003-11-10 | 2005-05-26 | Synta Pharmaceuticals, Corp. | Fused heterocyclic compounds |
| WO2009042907A1 (en) * | 2007-09-27 | 2009-04-02 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isoindoline compounds for the treatment of spinal muscular atrophy and other uses |
| WO2009151546A2 (en) * | 2008-05-27 | 2009-12-17 | Ptc Therapeutics, Inc. | Methods for treating spinal muscular atrophy |
Also Published As
| Publication number | Publication date |
|---|---|
| BR112014021531A8 (pt) | 2021-06-15 |
| BR112014021531A2 (pt) | 2017-06-20 |
| WO2013130689A1 (en) | 2013-09-06 |
| JP2015509956A (ja) | 2015-04-02 |
| EP2819519A4 (en) | 2015-09-02 |
| US20150166575A1 (en) | 2015-06-18 |
| HK1200056A1 (en) | 2015-07-31 |
| MX2014010406A (es) | 2015-03-10 |
| EP2819519B1 (en) | 2019-10-23 |
| CA2865957C (en) | 2020-02-11 |
| KR20140131384A (ko) | 2014-11-12 |
| EP2819519A1 (en) | 2015-01-07 |
| JP6092264B2 (ja) | 2017-03-08 |
| CN104302181A (zh) | 2015-01-21 |
| US9371336B2 (en) | 2016-06-21 |
| EA201491617A1 (ru) | 2015-01-30 |
| MX352962B (es) | 2017-12-15 |
| KR102099997B1 (ko) | 2020-04-13 |
| CA2865957A1 (en) | 2013-09-06 |
| BR112014021531B1 (pt) | 2022-10-04 |
| EA029155B1 (ru) | 2018-02-28 |
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| GR01 | Patent grant | ||
| GR01 | Patent grant |