JP2021502403A5 - - Google Patents

Info

Publication number

JP2021502403A5

JP2021502403A5 JP2020526304A JP2020526304A JP2021502403A5 JP 2021502403 A5 JP2021502403 A5 JP 2021502403A5 JP 2020526304 A JP2020526304 A JP 2020526304A JP 2020526304 A JP2020526304 A JP 2020526304A JP 2021502403 A5 JP2021502403 A5 JP 2021502403A5

Authority

JP

Japan

Prior art keywords

alkyl

optionally substituted

syndrome

substituted

hydrogen

Prior art date

2018-11-09

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

• Espacenet
• Global Dossier
• Discuss

• 229910052739 hydrogen Inorganic materials 0.000 claims 12
• 239000001257 hydrogen Substances 0.000 claims 12
• 125000004435 hydrogen atom Chemical class [H]* 0.000 claims 12
• 208000011580 syndromic disease Diseases 0.000 claims 12
• 206010015037 epilepsy Diseases 0.000 claims 11
• 125000002887 hydroxy group Chemical group [H]O* 0.000 claims 10
• 241000124008 Mammalia Species 0.000 claims 9
• 239000008194 pharmaceutical composition Substances 0.000 claims 9
• 125000004404 heteroalkyl group Chemical group 0.000 claims 8
• AURFZBICLPNKBZ-YZRLXODZSA-N 3alpha-hydroxy-5beta-pregnan-20-one Chemical compound C([C@H]1CC2)[C@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H](C(=O)C)[C@@]2(C)CC1 AURFZBICLPNKBZ-YZRLXODZSA-N 0.000 claims 7
• PGTVWKLGGCQMBR-FLBATMFCSA-N Ganaxolone Chemical compound C([C@@H]1CC2)[C@](C)(O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H](C(=O)C)[C@@]2(C)CC1 PGTVWKLGGCQMBR-FLBATMFCSA-N 0.000 claims 7
• AURFZBICLPNKBZ-UHFFFAOYSA-N Pregnanolone Natural products C1CC2CC(O)CCC2(C)C2C1C1CCC(C(=O)C)C1(C)CC2 AURFZBICLPNKBZ-UHFFFAOYSA-N 0.000 claims 7
• ORNBQBCIOKFOEO-YQUGOWONSA-N Pregnenolone Natural products O=C(C)[C@@H]1[C@@]2(C)[C@H]([C@H]3[C@@H]([C@]4(C)C(=CC3)C[C@@H](O)CC4)CC2)CC1 ORNBQBCIOKFOEO-YQUGOWONSA-N 0.000 claims 7
• 229950006567 ganaxolone Drugs 0.000 claims 7
• ORNBQBCIOKFOEO-QGVNFLHTSA-N pregnenolone Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H](C(=O)C)[C@@]1(C)CC2 ORNBQBCIOKFOEO-QGVNFLHTSA-N 0.000 claims 7
• 229960000249 pregnenolone Drugs 0.000 claims 7
• 208000014644 Brain disease Diseases 0.000 claims 6
• 208000032274 Encephalopathy Diseases 0.000 claims 6
• 125000000217 alkyl group Chemical group 0.000 claims 6
• 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 6
• 208000035475 disorder Diseases 0.000 claims 6
• 125000000547 substituted alkyl group Chemical group 0.000 claims 6
• 230000001037 epileptic effect Effects 0.000 claims 5
• 239000002858 neurotransmitter agent Substances 0.000 claims 5
• 125000003118 aryl group Chemical group 0.000 claims 4
• 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 4
• GRVDJDISBSALJP-UHFFFAOYSA-N methyloxidanyl Chemical group [O]C GRVDJDISBSALJP-UHFFFAOYSA-N 0.000 claims 4
• 125000004043 oxo group Chemical group O=* 0.000 claims 4
• 229910052760 oxygen Inorganic materials 0.000 claims 4
• 229910052717 sulfur Inorganic materials 0.000 claims 4
• 239000003814 drug Substances 0.000 claims 3
• 150000003839 salts Chemical class 0.000 claims 3
• 125000000008 (C1-C10) alkyl group Chemical group 0.000 claims 2
• 208000009575 Angelman syndrome Diseases 0.000 claims 2
• 102100034746 Cyclin-dependent kinase-like 5 Human genes 0.000 claims 2
• 101000945692 Homo sapiens Cyclin-dependent kinase-like 5 Proteins 0.000 claims 2
• 101001072243 Homo sapiens Protocadherin-19 Proteins 0.000 claims 2
• 102100036389 Protocadherin-19 Human genes 0.000 claims 2
• 208000006289 Rett Syndrome Diseases 0.000 claims 2
• 208000005392 Spasm Diseases 0.000 claims 2
• QAOWNCQODCNURD-UHFFFAOYSA-L Sulfate Chemical compound [O-]S([O-])(=O)=O QAOWNCQODCNURD-UHFFFAOYSA-L 0.000 claims 2
• JLRGJRBPOGGCBT-UHFFFAOYSA-N Tolbutamide Chemical compound CCCCNC(=O)NS(=O)(=O)C1=CC=C(C)C=C1 JLRGJRBPOGGCBT-UHFFFAOYSA-N 0.000 claims 2
• MENQCIVHHONJLU-FZCSVUEKSA-N [(3s,5s,8r,9s,10s,13s,14s,17s)-17-acetyl-10,13-dimethyl-2,3,4,5,6,7,8,9,11,12,14,15,16,17-tetradecahydro-1h-cyclopenta[a]phenanthren-3-yl] hydrogen sulfate Chemical compound C([C@@H]1CC2)[C@@H](OS(O)(=O)=O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H](C(=O)C)[C@@]2(C)CC1 MENQCIVHHONJLU-FZCSVUEKSA-N 0.000 claims 2
• 125000003710 aryl alkyl group Chemical group 0.000 claims 2
• 238000003556 assay Methods 0.000 claims 2
• 125000002618 bicyclic heterocycle group Chemical group 0.000 claims 2
• 239000012472 biological sample Substances 0.000 claims 2
• 150000001875 compounds Chemical class 0.000 claims 2
• 208000013500 continuous spikes and waves during sleep Diseases 0.000 claims 2
• 230000007812 deficiency Effects 0.000 claims 2
• 229940079593 drug Drugs 0.000 claims 2
• 229950007402 eltanolone Drugs 0.000 claims 2
• 229910052736 halogen Inorganic materials 0.000 claims 2
• 150000002367 halogens Chemical class 0.000 claims 2
• 230000003340 mental effect Effects 0.000 claims 2
• 125000001570 methylene group Chemical group [H]C([H])([*:1])[*:2] 0.000 claims 2
• 125000003107 substituted aryl group Chemical group 0.000 claims 2
• 125000002023 trifluoromethyl group Chemical group FC(F)(F)* 0.000 claims 2
• XMRPGKVKISIQBV-UHFFFAOYSA-N (+-)-5- Pregnane-3,20-dione Natural products C1CC2CC(=O)CCC2(C)C2C1C1CCC(C(=O)C)C1(C)CC2 XMRPGKVKISIQBV-UHFFFAOYSA-N 0.000 claims 1
• XMRPGKVKISIQBV-BJMCWZGWSA-N 5alpha-pregnane-3,20-dione Chemical compound C([C@@H]1CC2)C(=O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H](C(=O)C)[C@@]2(C)CC1 XMRPGKVKISIQBV-BJMCWZGWSA-N 0.000 claims 1
• 206010010904 Convulsion Diseases 0.000 claims 1
• FMGSKLZLMKYGDP-USOAJAOKSA-N dehydroepiandrosterone Chemical compound C1[C@@H](O)CC[C@]2(C)[C@H]3CC[C@](C)(C(CC4)=O)[C@@H]4[C@@H]3CC=C21 FMGSKLZLMKYGDP-USOAJAOKSA-N 0.000 claims 1
• 238000002483 medication Methods 0.000 claims 1