ES2687854T3 - Pirazolopiridazinas y métodos para tratar las enfermedades degenerativas de la retina y la pérdida de audición asociada con el síndrome de Usher - Google Patents
Pirazolopiridazinas y métodos para tratar las enfermedades degenerativas de la retina y la pérdida de audición asociada con el síndrome de Usher Download PDFInfo
- Publication number
- ES2687854T3 ES2687854T3 ES13848995.0T ES13848995T ES2687854T3 ES 2687854 T3 ES2687854 T3 ES 2687854T3 ES 13848995 T ES13848995 T ES 13848995T ES 2687854 T3 ES2687854 T3 ES 2687854T3
- Authority
- ES
- Spain
- Prior art keywords
- compound
- pharmaceutically acceptable
- acceptable salt
- usher
- syndrome
- 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
- 206010011878 Deafness Diseases 0.000 title claims description 13
- 208000014769 Usher Syndromes Diseases 0.000 title claims description 13
- 208000016354 hearing loss disease Diseases 0.000 title claims description 13
- 231100000888 hearing loss Toxicity 0.000 title claims description 9
- 230000010370 hearing loss Effects 0.000 title claims description 9
- 238000000034 method Methods 0.000 title claims description 7
- 210000001525 retina Anatomy 0.000 title claims description 5
- 208000015122 neurodegenerative disease Diseases 0.000 title claims description 4
- NMEPLWZDUIIAAC-UHFFFAOYSA-N 1h-pyrazolo[4,3-c]pyridazine Chemical class C1=NN=C2C=NNC2=C1 NMEPLWZDUIIAAC-UHFFFAOYSA-N 0.000 title description 3
- 150000001875 compounds Chemical class 0.000 claims abstract description 37
- 150000003839 salts Chemical class 0.000 claims abstract description 26
- 208000011580 syndromic disease Diseases 0.000 claims description 8
- FWBHETKCLVMNFS-UHFFFAOYSA-N 4',6-Diamino-2-phenylindol Chemical compound C1=CC(C(=N)N)=CC=C1C1=CC2=CC=C(C(N)=N)C=C2N1 FWBHETKCLVMNFS-UHFFFAOYSA-N 0.000 claims description 4
- 208000017442 Retinal disease Diseases 0.000 claims description 4
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims description 4
- 230000003412 degenerative effect Effects 0.000 claims description 4
- 239000000203 mixture Substances 0.000 claims description 4
- 239000003937 drug carrier Substances 0.000 claims description 2
- 102200123931 rs111033258 Human genes 0.000 claims description 2
- 239000003981 vehicle Substances 0.000 claims description 2
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims 1
- 201000007737 Retinal degeneration Diseases 0.000 claims 1
- 206010064930 age-related macular degeneration Diseases 0.000 claims 1
- 238000004458 analytical method Methods 0.000 claims 1
- 238000003709 image segmentation Methods 0.000 claims 1
- 208000002780 macular degeneration Diseases 0.000 claims 1
- 230000004258 retinal degeneration Effects 0.000 claims 1
- 230000011218 segmentation Effects 0.000 claims 1
- LMBFAGIMSUYTBN-MPZNNTNKSA-N teixobactin Chemical compound C([C@H](C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](CO)C(=O)N[C@H](CCC(N)=O)C(=O)N[C@H]([C@@H](C)CC)C(=O)N[C@@H]([C@@H](C)CC)C(=O)N[C@@H](CO)C(=O)N[C@H]1C(N[C@@H](C)C(=O)N[C@@H](C[C@@H]2NC(=N)NC2)C(=O)N[C@H](C(=O)O[C@H]1C)[C@@H](C)CC)=O)NC)C1=CC=CC=C1 LMBFAGIMSUYTBN-MPZNNTNKSA-N 0.000 claims 1
- -1 Pyrazolopyridazine compound Chemical class 0.000 description 10
- 230000004438 eyesight Effects 0.000 description 7
- 201000004569 Blindness Diseases 0.000 description 6
- IAZDPXIOMUYVGZ-UHFFFAOYSA-N Dimethylsulphoxide Chemical compound CS(C)=O IAZDPXIOMUYVGZ-UHFFFAOYSA-N 0.000 description 5
- ZMXDDKWLCZADIW-UHFFFAOYSA-N N,N-Dimethylformamide Chemical compound CN(C)C=O ZMXDDKWLCZADIW-UHFFFAOYSA-N 0.000 description 5
- JGFZNNIVVJXRND-UHFFFAOYSA-N N,N-Diisopropylethylamine (DIPEA) Chemical compound CCN(C(C)C)C(C)C JGFZNNIVVJXRND-UHFFFAOYSA-N 0.000 description 4
- 208000018769 loss of vision Diseases 0.000 description 3
- 230000004393 visual impairment Effects 0.000 description 3
- LMDZBCPBFSXMTL-UHFFFAOYSA-N 1-ethyl-3-(3-dimethylaminopropyl)carbodiimide Chemical compound CCN=C=NCCCN(C)C LMDZBCPBFSXMTL-UHFFFAOYSA-N 0.000 description 2
- DBMJMQXJHONAFJ-UHFFFAOYSA-M Sodium laurylsulphate Chemical compound [Na+].CCCCCCCCCCCCOS([O-])(=O)=O DBMJMQXJHONAFJ-UHFFFAOYSA-M 0.000 description 2
- 231100000895 deafness Toxicity 0.000 description 2
- 230000007850 degeneration Effects 0.000 description 2
- 238000000132 electrospray ionisation Methods 0.000 description 2
- 238000004128 high performance liquid chromatography Methods 0.000 description 2
- 238000004895 liquid chromatography mass spectrometry Methods 0.000 description 2
- 230000005043 peripheral vision Effects 0.000 description 2
- 230000002207 retinal effect Effects 0.000 description 2
- FPQQSJJWHUJYPU-UHFFFAOYSA-N 3-(dimethylamino)propyliminomethylidene-ethylazanium;chloride Chemical compound Cl.CCN=C=NCCCN(C)C FPQQSJJWHUJYPU-UHFFFAOYSA-N 0.000 description 1
- 208000012639 Balance disease Diseases 0.000 description 1
- 239000006144 Dulbecco’s modified Eagle's medium Substances 0.000 description 1
- 208000026350 Inborn Genetic disease Diseases 0.000 description 1
- 206010025421 Macule Diseases 0.000 description 1
- 208000001140 Night Blindness Diseases 0.000 description 1
- 229910019142 PO4 Inorganic materials 0.000 description 1
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 1
- 206010047571 Visual impairment Diseases 0.000 description 1
- 238000000451 chemical ionisation Methods 0.000 description 1
- 230000007547 defect Effects 0.000 description 1
- 238000003795 desorption Methods 0.000 description 1
- 235000012489 doughnuts Nutrition 0.000 description 1
- 210000003027 ear inner Anatomy 0.000 description 1
- 208000016361 genetic disease Diseases 0.000 description 1
- GQZXNSPRSGFJLY-UHFFFAOYSA-N hydroxyphosphanone Chemical compound OP=O GQZXNSPRSGFJLY-UHFFFAOYSA-N 0.000 description 1
- 229910052744 lithium Inorganic materials 0.000 description 1
- 231100000864 loss of vision Toxicity 0.000 description 1
- 230000014759 maintenance of location Effects 0.000 description 1
- 238000004949 mass spectrometry Methods 0.000 description 1
- 239000011159 matrix material Substances 0.000 description 1
- 238000001840 matrix-assisted laser desorption--ionisation time-of-flight mass spectrometry Methods 0.000 description 1
- 230000035772 mutation Effects 0.000 description 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 description 1
- 239000010452 phosphate Substances 0.000 description 1
- 108090000623 proteins and genes Proteins 0.000 description 1
- 230000001105 regulatory effect Effects 0.000 description 1
- WHRNULOCNSKMGB-UHFFFAOYSA-N tetrahydrofuran thf Chemical compound C1CCOC1.C1CCOC1 WHRNULOCNSKMGB-UHFFFAOYSA-N 0.000 description 1
- 208000027491 vestibular disease Diseases 0.000 description 1
- 208000029257 vision disease Diseases 0.000 description 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
- C07D487/02—Heterocyclic 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
- C07D487/04—Ortho-condensed systems
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Life Sciences & Earth Sciences (AREA)
- Neurosurgery (AREA)
- Biomedical Technology (AREA)
- Neurology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Ophthalmology & Optometry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Nitrogen Condensed Heterocyclic Rings (AREA)
- Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261718611P | 2012-10-25 | 2012-10-25 | |
| US201261718611P | 2012-10-25 | ||
| US201361775402P | 2013-03-08 | 2013-03-08 | |
| US201361775402P | 2013-03-08 | ||
| PCT/US2013/066939 WO2014066836A1 (en) | 2012-10-25 | 2013-10-25 | Pyrazolopyridazines and methods for treating retinal-degenerative diseases and hearing loss associated with usher syndrome |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2687854T3 true ES2687854T3 (es) | 2018-10-29 |
Family
ID=50545356
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES13848995.0T Active ES2687854T3 (es) | 2012-10-25 | 2013-10-25 | Pirazolopiridazinas y métodos para tratar las enfermedades degenerativas de la retina y la pérdida de audición asociada con el síndrome de Usher |
Country Status (10)
| Country | Link |
|---|---|
| EP (1) | EP2911671B1 (enExample) |
| JP (1) | JP6291502B2 (enExample) |
| AU (1) | AU2013334139B2 (enExample) |
| CA (1) | CA2889494C (enExample) |
| ES (1) | ES2687854T3 (enExample) |
| HU (1) | HUE039970T2 (enExample) |
| IL (1) | IL238288B (enExample) |
| PL (1) | PL2911671T3 (enExample) |
| WO (1) | WO2014066836A1 (enExample) |
| ZA (1) | ZA201502655B (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2834049C (en) | 2011-04-25 | 2018-05-22 | Usher Iii Initiative, Inc. | Pyrazolopyridazines and methods for treating retinal-degenerative diseases and hearing loss associated with usher syndrome |
| JP6290910B2 (ja) * | 2012-10-25 | 2018-03-07 | アッシャー・サード・イニシアティブ・インコーポレイテッド | ピラゾロピリダジンならびに網膜変性疾患およびアッシャー症候群に伴う聴力損失を処置するための方法 |
| US9227976B2 (en) | 2012-10-25 | 2016-01-05 | Usher Iii Initiative, Inc. | Pyrazolopyridazines and methods for treating retinal-degenerative diseases and hearing loss associated with usher syndrome |
| WO2017045955A1 (en) * | 2015-09-14 | 2017-03-23 | Basf Se | Heterobicyclic compounds |
| AU2018282154B2 (en) | 2017-06-05 | 2022-04-07 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
| AU2019243048A1 (en) | 2018-03-27 | 2020-10-15 | Ptc Therapeutics, Inc. | Compounds for treating Huntington's disease |
| IL279688B2 (en) | 2018-06-27 | 2025-01-01 | Ptc Therapeutics Inc | Heterocyclic and heteroaryl compounds for the treatment of Huntington's disease |
| BR112020026534A2 (pt) | 2018-06-27 | 2021-03-23 | Ptc Therapeutics, Inc. | Compostos de heteroarila para o tratamento da doença de huntington |
| WO2020161209A1 (en) | 2019-02-06 | 2020-08-13 | Syngenta Crop Protection Ag | Herbicidal fused pyridazine compounds |
| WO2020161208A1 (en) | 2019-02-06 | 2020-08-13 | Syngenta Crop Protection Ag | Herbicidal fused pyridazine compounds |
| CN114245794B (zh) | 2019-05-13 | 2024-09-13 | Ptc医疗公司 | 用于治疗亨廷顿氏病的化合物 |
| WO2022011091A1 (en) | 2020-07-09 | 2022-01-13 | Usher Iii Initiative, Inc. | Treatment of cancer, inflammatory diseases and autoimmune diseases |
| WO2023139091A1 (en) * | 2022-01-19 | 2023-07-27 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Geralexin and uses thereof for the treatment of retinal degenerative diseases |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB0206723D0 (en) * | 2002-03-21 | 2002-05-01 | Glaxo Group Ltd | Novel compounds |
| US20090306045A1 (en) * | 2005-12-22 | 2009-12-10 | Ira Mellman | Inhibition of Glycogen Synthase Kinase and Methods of Treating Autoimmune or Immune Inflammatory Disease |
| CN101917999A (zh) * | 2007-11-07 | 2010-12-15 | 弗尔德里克斯制药股份有限公司 | 蛋白质运输的调节 |
| CA2834049C (en) * | 2011-04-25 | 2018-05-22 | Usher Iii Initiative, Inc. | Pyrazolopyridazines and methods for treating retinal-degenerative diseases and hearing loss associated with usher syndrome |
| JP6290910B2 (ja) * | 2012-10-25 | 2018-03-07 | アッシャー・サード・イニシアティブ・インコーポレイテッド | ピラゾロピリダジンならびに網膜変性疾患およびアッシャー症候群に伴う聴力損失を処置するための方法 |
-
2013
- 2013-10-25 EP EP13848995.0A patent/EP2911671B1/en active Active
- 2013-10-25 ES ES13848995.0T patent/ES2687854T3/es active Active
- 2013-10-25 JP JP2015539868A patent/JP6291502B2/ja active Active
- 2013-10-25 WO PCT/US2013/066939 patent/WO2014066836A1/en not_active Ceased
- 2013-10-25 HU HUE13848995A patent/HUE039970T2/hu unknown
- 2013-10-25 CA CA2889494A patent/CA2889494C/en active Active
- 2013-10-25 PL PL13848995T patent/PL2911671T3/pl unknown
- 2013-10-25 AU AU2013334139A patent/AU2013334139B2/en active Active
-
2015
- 2015-04-14 IL IL238288A patent/IL238288B/en active IP Right Grant
- 2015-04-20 ZA ZA2015/02655A patent/ZA201502655B/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP2911671B1 (en) | 2018-06-27 |
| HUE039970T2 (hu) | 2019-02-28 |
| IL238288A0 (en) | 2015-06-30 |
| HK1214180A1 (en) | 2016-07-22 |
| PL2911671T3 (pl) | 2018-11-30 |
| ZA201502655B (en) | 2017-11-29 |
| WO2014066836A1 (en) | 2014-05-01 |
| EP2911671A4 (en) | 2016-09-14 |
| JP6291502B2 (ja) | 2018-03-14 |
| CA2889494C (en) | 2018-12-18 |
| AU2013334139A1 (en) | 2015-04-30 |
| AU2013334139B2 (en) | 2017-08-03 |
| EP2911671A1 (en) | 2015-09-02 |
| JP2015535257A (ja) | 2015-12-10 |
| IL238288B (en) | 2018-04-30 |
| CA2889494A1 (en) | 2014-05-01 |
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