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 (enExample) |
| EP (1) | EP2819519B1 (enExample) |
| JP (1) | JP6092264B2 (enExample) |
| KR (1) | KR102099997B1 (enExample) |
| CN (1) | CN104302181B (enExample) |
| BR (1) | BR112014021531B1 (enExample) |
| CA (1) | CA2865957C (enExample) |
| EA (1) | EA029155B1 (enExample) |
| MX (1) | MX352962B (enExample) |
| WO (1) | WO2013130689A1 (enExample) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2861609C (en) | 2011-12-30 | 2021-02-16 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| CA2862084C (en) | 2012-01-26 | 2021-05-11 | Ptc Therapeutics, Inc. | 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy |
| PL2812004T3 (pl) | 2012-02-10 | 2019-01-31 | Ptc Therapeutics, Inc. | Związki do leczenia rdzeniowego zaniku mięśni |
| MX358514B (es) | 2012-03-23 | 2018-08-24 | Ptc Therapeutics Inc | Compuestos para tratar la atrofia muscular espinal. |
| EA201500699A1 (ru) * | 2012-12-24 | 2015-12-30 | Рамот Эт Тель-Авив Юниверсити Лтд. | Агенты для лечения генетических заболеваний, возникающих в результате нонсенс-мутаций, и способы идентификации этих агентов |
| RU2016109324A (ru) | 2013-08-19 | 2017-09-26 | Ф. Хоффманн-Ля Рош Аг | Способ скрининга |
| 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 |
| KR102488323B1 (ko) | 2015-12-10 | 2023-01-12 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료 또는 개선을 위한 조성물 |
| 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 |
| EP3363793A1 (en) | 2017-02-15 | 2018-08-22 | Merck Patent GmbH | Hydrophobic compounds for optically active devices |
| EP3363786A1 (en) | 2017-02-15 | 2018-08-22 | Merck Patent GmbH | Compounds for optically active devices |
| KR20200033249A (ko) | 2017-06-05 | 2020-03-27 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료 화합물 |
| 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 |
| MX2019015578A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
| MX2019015580A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
| CN111194215B (zh) | 2017-08-04 | 2024-03-01 | 斯基霍克疗法公司 | 用于调节剪接的方法和组合物 |
| US12103926B2 (en) | 2018-03-27 | 2024-10-01 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
| AU2019294478B2 (en) | 2018-06-27 | 2023-03-23 | Ptc Therapeutics, Inc. | Heterocyclic and heteroaryl compounds for treating Huntington's disease |
| US11685746B2 (en) | 2018-06-27 | 2023-06-27 | Ptc Therapeutics, Inc. | Heteroaryl compounds for treating Huntington's disease |
| WO2020005877A1 (en) | 2018-06-27 | 2020-01-02 | Ptc Therapeutics, Inc. | Heteroaryl compounds for treating huntington's disease |
| MX2020014116A (es) | 2018-06-27 | 2021-06-15 | Reborna Biosciences Inc | Agente profilactico o terapeutico para atrofia muscular espinal. |
| WO2020163405A1 (en) | 2019-02-05 | 2020-08-13 | Skyhawk Therapeutics, Inc. | Methods and compositions for modulating splicing |
| 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 |
| 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 |
| WO2021233800A1 (en) | 2020-05-20 | 2021-11-25 | Merck Patent Gmbh | Azacoumarin and azathiocoumarin derivatives for use in optically active devices |
| WO2023114539A1 (en) * | 2021-12-17 | 2023-06-22 | Pretzel Therapeutics, Inc. | Amino chromen-2-one modulators of polrmt |
| WO2024251925A1 (en) * | 2023-06-09 | 2024-12-12 | 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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| 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 |
| HK1040076A1 (zh) | 1998-09-21 | 2002-05-24 | 希雷生物化学有限公司 | 作为整联蛋白抑制剂的喹嗪酮 |
| CN1263743C (zh) | 2000-01-24 | 2006-07-12 | 基纳西亚股份有限公司 | 用于治疗的吗啉代基取代的化合物 |
| US20040077529A1 (en) | 2002-06-10 | 2004-04-22 | Per-Fredrik Lehmann | Urotensin II receptor agents |
| US20040105849A1 (en) | 2002-11-25 | 2004-06-03 | 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 |
| AU2004249730A1 (en) | 2003-06-20 | 2004-12-29 | Novartis Vaccines And Diagnostics, Inc. | 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 |
| ES2292130T3 (es) | 2004-05-04 | 2008-03-01 | Warner-Lambert Company Llc | Pirido(2,3-d)pirimidin-7-onas pirrolil-sustituidas y derivados de las mismas como agentes terapeuticos. |
| EP2332527A3 (en) | 2004-10-20 | 2011-11-16 | Resverlogix Corp. | Flavanoids and Isoflavanoids for the prevention and treatment of cardiovascular diseases |
| EP1846397A1 (en) | 2005-01-21 | 2007-10-24 | Janssen Pharmaceutica N.V. | Novel heterocyclic benzoy[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 |
| US8337941B2 (en) | 2006-07-27 | 2012-12-25 | The Trustees Of Columbia University In The City Of New York | Fluorescent substrates for monoamine transporters as optical false neurotransmitters |
| WO2010019236A1 (en) * | 2008-08-13 | 2010-02-18 | Ptc Therapeutics, Inc | Methods for treating spinal muscular atrophy |
| US8754220B2 (en) | 2009-11-20 | 2014-06-17 | Merck Sharp & Dohme Corp. | Quinolizidinone carboxamide M1 receptor positive allosteric modulators |
| BR112012017473A2 (pt) | 2010-01-13 | 2019-09-24 | Institut National De La Sante Et De La Rech Medicale Inserm Epst | pirido(1,2-a) pirimidinas, seu uso, bem como composição farmacêutica compreendendo tais compostos |
| CA2861609C (en) | 2011-12-30 | 2021-02-16 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| CA2862084C (en) | 2012-01-26 | 2021-05-11 | Ptc Therapeutics, Inc. | 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy |
| PL2812004T3 (pl) | 2012-02-10 | 2019-01-31 | Ptc Therapeutics, Inc. | Związki do leczenia rdzeniowego zaniku mięśni |
| MX358514B (es) | 2012-03-23 | 2018-08-24 | Ptc Therapeutics Inc | Compuestos para tratar la atrofia muscular espinal. |
-
2013
- 2013-02-28 EA EA201491617A patent/EA029155B1/ru not_active IP Right Cessation
- 2013-02-28 EP EP13755032.3A patent/EP2819519B1/en active Active
- 2013-02-28 US US14/380,385 patent/US9371336B2/en active Active
- 2013-02-28 CN CN201380023259.1A patent/CN104302181B/zh 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 KR KR1020147027577A patent/KR102099997B1/ko active Active
- 2013-02-28 CA CA2865957A patent/CA2865957C/en active Active
- 2013-02-28 WO PCT/US2013/028131 patent/WO2013130689A1/en not_active Ceased
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 |
|---|---|
| JP2015509956A (ja) | 2015-04-02 |
| CA2865957C (en) | 2020-02-11 |
| EP2819519B1 (en) | 2019-10-23 |
| US20150166575A1 (en) | 2015-06-18 |
| US9371336B2 (en) | 2016-06-21 |
| CA2865957A1 (en) | 2013-09-06 |
| BR112014021531B1 (pt) | 2022-10-04 |
| CN104302181A (zh) | 2015-01-21 |
| EP2819519A1 (en) | 2015-01-07 |
| HK1200056A1 (en) | 2015-07-31 |
| WO2013130689A1 (en) | 2013-09-06 |
| MX2014010406A (es) | 2015-03-10 |
| JP6092264B2 (ja) | 2017-03-08 |
| EP2819519A4 (en) | 2015-09-02 |
| BR112014021531A8 (pt) | 2021-06-15 |
| MX352962B (es) | 2017-12-15 |
| EA029155B1 (ru) | 2018-02-28 |
| KR102099997B1 (ko) | 2020-04-13 |
| KR20140131384A (ko) | 2014-11-12 |
| BR112014021531A2 (pt) | 2017-06-20 |
| EA201491617A1 (ru) | 2015-01-30 |
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| GR01 | Patent grant | ||
| GR01 | Patent grant |