JP2018507886A5 - - Google Patents
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- Publication number
- JP2018507886A5 JP2018507886A5 JP2017547448A JP2017547448A JP2018507886A5 JP 2018507886 A5 JP2018507886 A5 JP 2018507886A5 JP 2017547448 A JP2017547448 A JP 2017547448A JP 2017547448 A JP2017547448 A JP 2017547448A JP 2018507886 A5 JP2018507886 A5 JP 2018507886A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- composition according
- group
- compound
- pharmaceutically acceptable
- 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.)
- Granted
Links
- 239000008194 pharmaceutical composition Substances 0.000 claims 23
- 150000001875 compounds Chemical class 0.000 claims 14
- 229910052736 halogen Inorganic materials 0.000 claims 10
- 150000002367 halogens Chemical class 0.000 claims 10
- 239000000651 prodrug Substances 0.000 claims 10
- 229940002612 prodrug Drugs 0.000 claims 10
- 150000003839 salts Chemical class 0.000 claims 10
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 7
- -1 nitro, hydroxy, thio Chemical group 0.000 claims 6
- 102000004169 proteins and genes Human genes 0.000 claims 6
- 108090000623 proteins and genes Proteins 0.000 claims 6
- 125000003545 alkoxy group Chemical group 0.000 claims 5
- 208000035475 disorder Diseases 0.000 claims 5
- 229910052739 hydrogen Inorganic materials 0.000 claims 5
- 239000001257 hydrogen Substances 0.000 claims 5
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims 4
- PXGOKWXKJXAPGV-UHFFFAOYSA-N Fluorine Chemical compound FF PXGOKWXKJXAPGV-UHFFFAOYSA-N 0.000 claims 3
- 201000011240 Frontotemporal dementia Diseases 0.000 claims 3
- 125000003277 amino group Chemical group 0.000 claims 3
- 125000004093 cyano group Chemical group *C#N 0.000 claims 3
- 229910052731 fluorine Inorganic materials 0.000 claims 3
- 239000011737 fluorine Substances 0.000 claims 3
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 3
- KXDHJXZQYSOELW-UHFFFAOYSA-M Carbamate Chemical compound NC([O-])=O KXDHJXZQYSOELW-UHFFFAOYSA-M 0.000 claims 2
- 208000009829 Lewy Body Disease Diseases 0.000 claims 2
- 201000002832 Lewy body dementia Diseases 0.000 claims 2
- 208000018737 Parkinson disease Diseases 0.000 claims 2
- 208000032859 Synucleinopathies Diseases 0.000 claims 2
- 208000034799 Tauopathies Diseases 0.000 claims 2
- 125000000217 alkyl group Chemical group 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 239000002552 dosage form Substances 0.000 claims 2
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 2
- 125000001449 isopropyl group Chemical group [H]C([H])([H])C([H])(*)C([H])([H])[H] 0.000 claims 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims 2
- 125000006273 (C1-C3) alkyl group Chemical group 0.000 claims 1
- OJCLMSVFVVKWJX-UHFFFAOYSA-N 1-azabicyclo[2.2.2]octan-3-yl n-[2-[3-(4-fluorophenyl)phenyl]propan-2-yl]carbamate Chemical compound C1N(CC2)CCC2C1OC(=O)NC(C)(C)C(C=1)=CC=CC=1C1=CC=C(F)C=C1 OJCLMSVFVVKWJX-UHFFFAOYSA-N 0.000 claims 1
- 125000006163 5-membered heteroaryl group Chemical group 0.000 claims 1
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 208000014644 Brain disease Diseases 0.000 claims 1
- 125000000882 C2-C6 alkenyl group Chemical group 0.000 claims 1
- 125000003320 C2-C6 alkenyloxy group Chemical group 0.000 claims 1
- 125000003601 C2-C6 alkynyl group Chemical group 0.000 claims 1
- 206010012289 Dementia Diseases 0.000 claims 1
- 208000032274 Encephalopathy Diseases 0.000 claims 1
- 208000002339 Frontotemporal Lobar Degeneration Diseases 0.000 claims 1
- 208000003098 Ganglion Cysts Diseases 0.000 claims 1
- 208000032612 Glial tumor Diseases 0.000 claims 1
- 206010018338 Glioma Diseases 0.000 claims 1
- 208000023105 Huntington disease Diseases 0.000 claims 1
- 208000001089 Multiple system atrophy Diseases 0.000 claims 1
- 206010033645 Pancreatitis Diseases 0.000 claims 1
- 208000027089 Parkinsonian disease Diseases 0.000 claims 1
- 206010034010 Parkinsonism Diseases 0.000 claims 1
- 208000000609 Pick Disease of the Brain Diseases 0.000 claims 1
- 241000031708 Saprospiraceae Species 0.000 claims 1
- BQCADISMDOOEFD-UHFFFAOYSA-N Silver Chemical compound [Ag] BQCADISMDOOEFD-UHFFFAOYSA-N 0.000 claims 1
- 208000005400 Synovial Cyst Diseases 0.000 claims 1
- 208000026911 Tuberous sclerosis complex Diseases 0.000 claims 1
- 238000009825 accumulation Methods 0.000 claims 1
- 125000005133 alkynyloxy group Chemical group 0.000 claims 1
- 125000003118 aryl group Chemical group 0.000 claims 1
- 210000004227 basal ganglia Anatomy 0.000 claims 1
- 125000001797 benzyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C([H])([H])* 0.000 claims 1
- 239000004305 biphenyl Substances 0.000 claims 1
- 210000004720 cerebrum Anatomy 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- 210000000349 chromosome Anatomy 0.000 claims 1
- 230000001054 cortical effect Effects 0.000 claims 1
- 125000001995 cyclobutyl group Chemical group [H]C1([H])C([H])([H])C([H])(*)C1([H])[H] 0.000 claims 1
- 125000001559 cyclopropyl group Chemical group [H]C1([H])C([H])([H])C1([H])* 0.000 claims 1
- 230000007850 degeneration Effects 0.000 claims 1
- 208000017004 dementia pugilistica Diseases 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 125000001153 fluoro group Chemical group F* 0.000 claims 1
- 201000008361 ganglioneuroma Diseases 0.000 claims 1
- 125000001072 heteroaryl group Chemical group 0.000 claims 1
- 125000005842 heteroatom Chemical group 0.000 claims 1
- 150000002431 hydrogen Chemical class 0.000 claims 1
- 125000004356 hydroxy functional group Chemical group O* 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 230000007138 neurofibrillary change Effects 0.000 claims 1
- 125000000449 nitro group Chemical group [O-][N+](*)=O 0.000 claims 1
- 125000004043 oxo group Chemical group O=* 0.000 claims 1
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 claims 1
- 201000002212 progressive supranuclear palsy Diseases 0.000 claims 1
- 229910052709 silver Inorganic materials 0.000 claims 1
- 239000004332 silver Substances 0.000 claims 1
- 208000011580 syndromic disease Diseases 0.000 claims 1
- 210000001519 tissue Anatomy 0.000 claims 1
- 208000009999 tuberous sclerosis Diseases 0.000 claims 1
- 0 CC(C)(c1cccc(-c2ccc(*)cc2)c1)NC(OC1C(CC2)CCN2C1)=O Chemical compound CC(C)(c1cccc(-c2ccc(*)cc2)c1)NC(OC1C(CC2)CCN2C1)=O 0.000 description 2
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021078459A JP7374149B2 (ja) | 2015-03-10 | 2021-05-06 | タンパク質症を処置するための方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562131071P | 2015-03-10 | 2015-03-10 | |
| US62/131,071 | 2015-03-10 | ||
| PCT/US2016/021512 WO2016145046A1 (en) | 2015-03-10 | 2016-03-09 | Methods for treating proteinopathies |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021078459A Division JP7374149B2 (ja) | 2015-03-10 | 2021-05-06 | タンパク質症を処置するための方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018507886A JP2018507886A (ja) | 2018-03-22 |
| JP2018507886A5 true JP2018507886A5 (enExample) | 2019-04-25 |
| JP6990110B2 JP6990110B2 (ja) | 2022-02-10 |
Family
ID=55587378
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017547448A Active JP6990110B2 (ja) | 2015-03-10 | 2016-03-09 | タンパク質症を処置するための方法 |
| JP2021078459A Active JP7374149B2 (ja) | 2015-03-10 | 2021-05-06 | タンパク質症を処置するための方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021078459A Active JP7374149B2 (ja) | 2015-03-10 | 2021-05-06 | タンパク質症を処置するための方法 |
Country Status (17)
| Country | Link |
|---|---|
| US (2) | US20180036295A1 (enExample) |
| EP (2) | EP3267983B1 (enExample) |
| JP (2) | JP6990110B2 (enExample) |
| KR (1) | KR20170123329A (enExample) |
| CN (1) | CN107872976A (enExample) |
| AU (1) | AU2016229826A1 (enExample) |
| CA (1) | CA2978883A1 (enExample) |
| EA (1) | EA201791993A1 (enExample) |
| ES (1) | ES2973101T3 (enExample) |
| HK (1) | HK1244217A1 (enExample) |
| HU (1) | HUE065628T2 (enExample) |
| IL (1) | IL254393A0 (enExample) |
| MX (1) | MX2017011598A (enExample) |
| PL (1) | PL3267983T3 (enExample) |
| PT (1) | PT3267983T (enExample) |
| TW (1) | TW201642855A (enExample) |
| WO (1) | WO2016145046A1 (enExample) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MA37975B2 (fr) | 2012-09-11 | 2021-03-31 | Genzyme Corp | Inhibiteurs de synthase de glucosylcéramide |
| BR112019027029A2 (pt) * | 2017-06-19 | 2020-07-14 | Kainos Medicine, Inc. | moduladores de alfa-sinucleina |
| TWI644673B (zh) * | 2017-07-27 | 2018-12-21 | 百朗克股份有限公司 | 頭孢曲松的用途 |
| WO2020163244A1 (en) * | 2019-02-04 | 2020-08-13 | Genzyme Corporation | Methods for treating symptoms and disorders associated with lysosomal storage diseases |
| US12527776B2 (en) | 2019-02-04 | 2026-01-20 | Genzyme Corporation | Treatment of ciliopathies using inhibitors of glucosylceramide synthase (GCS) |
| BR112022009409A2 (pt) * | 2019-11-15 | 2022-08-09 | Yuhan Corp | Novos derivados que têm grupamento 2,3-dihidro-1h-indeno ou 2,3-dihidrobenzofurano ou sal farmaceuticamente aceitável dos mesmos e composições farmacêuticas que os compreendem |
| EP4100009A1 (en) | 2020-02-03 | 2022-12-14 | Genzyme Corporation | Methods for treating neurological symptoms associated with lysosomal storage diseases |
| KR20230005813A (ko) | 2020-02-28 | 2023-01-10 | 프락시스 프리시젼 메디신즈, 인크. | Kcnt1 억제제 및 사용 방법 |
| BR112022017530A2 (pt) | 2020-03-17 | 2022-10-18 | Sumitomo Pharma Co Ltd | Derivado de oxadiazol |
| BR112022019041A2 (pt) * | 2020-03-23 | 2022-12-27 | Praxis Prec Medicines Inc | Inibidores de kcnt1 e métodos de uso |
| KR20230043024A (ko) * | 2020-07-24 | 2023-03-30 | 젠자임 코포레이션 | 벤글루스타트를 포함하는 제약 조성물 |
| MX2023001285A (es) | 2020-07-30 | 2023-02-22 | Genzyme Corp | Metodos para reducir la concentracion de glicoesfingolipidos en el tejido cerebral y metodos de tratamiento de enfermedades neurodegenerativas que implican los mismos. |
| US11773088B2 (en) | 2020-11-02 | 2023-10-03 | Praxis Precision Medicines, Inc. | KCNT1 inhibitors and methods of use |
| KR20250106321A (ko) | 2022-12-01 | 2025-07-09 | 젠자임 코포레이션 | 강력하거나 중간 수준인 cyp3a4 억제제와 조합한 벤글루스타트 |
| WO2025262570A1 (en) | 2024-06-17 | 2025-12-26 | Genzyme Corporation | Venglustat as an inhibitor of glucosylceramide synthase in subjects having hepatic impairment |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4683A (en) | 1846-08-08 | waring and richard e | ||
| US195A (en) | 1837-05-15 | Machine fob cutting and dressing stone | ||
| BE640616A (enExample) | 1962-12-19 | |||
| US3492397A (en) | 1967-04-07 | 1970-01-27 | Warner Lambert Pharmaceutical | Sustained release dosage in the pellet form and process thereof |
| US4060598A (en) | 1967-06-28 | 1977-11-29 | Boehringer Mannheim G.M.B.H. | Tablets coated with aqueous resin dispersions |
| US3538214A (en) | 1969-04-22 | 1970-11-03 | Merck & Co Inc | Controlled release medicinal tablets |
| US4173626A (en) | 1978-12-11 | 1979-11-06 | Merck & Co., Inc. | Sustained release indomethacin |
| US4683195A (en) | 1986-01-30 | 1987-07-28 | Cetus Corporation | Process for amplifying, detecting, and/or-cloning nucleic acid sequences |
| CN103917094B (zh) * | 2011-03-18 | 2016-11-09 | 建新公司 | 葡萄糖基神经酰胺合酶抑制剂 |
| US9845327B2 (en) * | 2011-06-22 | 2017-12-19 | The General Hospital Corporation | Treatment of proteinopathies |
| MA37975B2 (fr) * | 2012-09-11 | 2021-03-31 | Genzyme Corp | Inhibiteurs de synthase de glucosylcéramide |
-
2016
- 2016-03-08 TW TW105107080A patent/TW201642855A/zh unknown
- 2016-03-09 US US15/556,444 patent/US20180036295A1/en not_active Abandoned
- 2016-03-09 EA EA201791993A patent/EA201791993A1/ru unknown
- 2016-03-09 HU HUE16711088A patent/HUE065628T2/hu unknown
- 2016-03-09 CA CA2978883A patent/CA2978883A1/en not_active Abandoned
- 2016-03-09 PL PL16711088.1T patent/PL3267983T3/pl unknown
- 2016-03-09 ES ES16711088T patent/ES2973101T3/es active Active
- 2016-03-09 AU AU2016229826A patent/AU2016229826A1/en not_active Abandoned
- 2016-03-09 WO PCT/US2016/021512 patent/WO2016145046A1/en not_active Ceased
- 2016-03-09 PT PT167110881T patent/PT3267983T/pt unknown
- 2016-03-09 KR KR1020177027604A patent/KR20170123329A/ko not_active Withdrawn
- 2016-03-09 EP EP16711088.1A patent/EP3267983B1/en active Active
- 2016-03-09 CN CN201680026839.XA patent/CN107872976A/zh active Pending
- 2016-03-09 JP JP2017547448A patent/JP6990110B2/ja active Active
- 2016-03-09 MX MX2017011598A patent/MX2017011598A/es unknown
- 2016-03-09 HK HK18103748.9A patent/HK1244217A1/zh unknown
- 2016-03-09 EP EP23217745.1A patent/EP4349408A3/en active Pending
-
2017
- 2017-09-10 IL IL254393A patent/IL254393A0/en unknown
-
2020
- 2020-03-02 US US16/807,102 patent/US20200197374A1/en active Pending
-
2021
- 2021-05-06 JP JP2021078459A patent/JP7374149B2/ja active Active
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