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1. WO2020004594 - PROPHYLACTIC OR THERAPEUTIC AGENT FOR SPINAL MUSCULAR ATROPHY

Publication Number WO/2020/004594
Publication Date 02.01.2020
International Application No. PCT/JP2019/025732
International Filing Date 27.06.2019
IPC
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
401
Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
02
containing two hetero rings
04
directly linked by a ring-member-to-ring- member bond
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
33
Heterocyclic compounds
395
having nitrogen as a ring hetero atom, e.g. guanethidine, rifamycins
495
having six-membered rings with two nitrogen atoms as the only ring hetero atoms, e.g. piperazine
505
Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
517
ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
21
Drugs for disorders of the muscular or neuromuscular system
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43
Drugs for specific purposes, not provided for in groups A61P1/-A61P41/108
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
401
Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
14
containing three or more hetero rings
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
403
Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/167
02
containing two hetero rings
04
directly linked by a ring-member-to-ring- member bond
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
413
Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
14
containing three or more hetero rings
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
417
Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/196
14
containing three or more hetero rings
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
471
Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/-C07D463/251
02
in which the condensed system contains two hetero rings
04
Ortho-condensed systems
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
487
Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/-C07D477/183
02
in which the condensed system contains two hetero rings
04
Ortho-condensed systems
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
491
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/-C07D459/290
02
in which the condensed system contains two hetero rings
04
Ortho-condensed systems
044
with only one oxygen atom as ring hetero atom in the oxygen-containing ring
048
the oxygen-containing ring being five-membered
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
498
Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
02
in which the condensed system contains two hetero rings
04
Ortho-condensed systems
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
498
Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
02
in which the condensed system contains two hetero rings
10
Spiro-condensed systems
[IPC code unknown for C12Q 1/686]
G PHYSICS
01
MEASURING; TESTING
N
INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
33
Investigating or analysing materials by specific methods not covered by groups G01N1/-G01N31/131
48
Biological material, e.g. blood, urine; Haemocytometers
50
Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
G PHYSICS
01
MEASURING; TESTING
N
INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
33
Investigating or analysing materials by specific methods not covered by groups G01N1/-G01N31/131
48
Biological material, e.g. blood, urine; Haemocytometers
50
Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
53
Immunoassay; Biospecific binding assay; Materials therefor
G PHYSICS
01
MEASURING; TESTING
N
INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
33
Investigating or analysing materials by specific methods not covered by groups G01N1/-G01N31/131
48
Biological material, e.g. blood, urine; Haemocytometers
50
Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
68
involving proteins, peptides or amino acids
C07D 401/04 (2006.01)
A61K 31/517 (2006.01)
A61P 21/00 (2006.01)
A61P 43/00 (2006.01)
C07D 401/14 (2006.01)
C07D 403/04 (2006.01)
CPC
A61K 31/517
A61P 21/00
A61P 43/00
C07D 401/04
C07D 401/14
C07D 403/04
Applicants
  • 株式会社リボルナバイオサイエンス REBORNA BIOSCIENCES, INC. [JP/JP]; 神奈川県藤沢市村岡東二丁目26番地の1 26-1, Muraoka-Higashi 2-chome, Fujisawa-shi, Kanagawa 2510012, JP
Inventors
  • 富士 晃嗣 FUJI, Koji; JP
  • 山崎 健 YAMASAKI, Takeshi; JP
  • 鈴木 俊也 SUZUKI, Shunya; JP
  • 小野 宏司 ONO, Koji; JP
  • 高萩 洋希 TAKAHAGI, Hiroki; JP
Agents
  • 一色国際特許業務法人 ISSHIKI & CO.; 東京都港区三田三丁目11番36号三田日東ダイビル Mita-Nitto Daibiru Bldg., 11-36, Mita 3-chome, Minato-ku, Tokyo 1080073, JP
Priority Data
2018-12255127.06.2018JP
Publication Language Japanese (JA)
Filing Language Japanese (JA)
Designated States
Title
(EN) PROPHYLACTIC OR THERAPEUTIC AGENT FOR SPINAL MUSCULAR ATROPHY
(FR) AGENT PROPHYLACTIQUE OU THÉRAPEUTIQUE POUR AMYOTROPHIE SPINALE
(JA) 脊髄性筋萎縮症の予防または治療剤
Abstract
(EN)
This prophylactic or therapeutic agent for spinal muscular atrophy includes a compound represented by formula (I) or a salt thereof (in the formula: W1, W2, and W3 are each independently selected from the group consisting of C-R2, C-R3, C-Rc, and C-Rd, and are defined as in any of (i) to (iv); (i) when W3 is C-R2, W1 is C-R3, W2 is C-Rc or N, and R1 is a hydrogen atom; (ii) when W3 is C-R3, W1 is C-R2, W2 is C-Rc or N, and R1 is a hydrogen atom, a C1–8 alkyl, or a C1–8 alkoxy; (iii) when W1 is C-R2, W2 is C-Rc or N, W3 is C-Rd, and R1 is an aliphatic heterocycle that contains at least one nitrogen atom and is optionally substituted with a non-aromatic substituent; (iv) when W2 is C-R2, W1 is C-Rc, W3 is C-Rd, and R1 is an aliphatic heterocycle that contains at least one nitrogen atom and is optionally substituted with a non-aromatic substituent; R2 is an aromatic ring of at least six members that is optionally substituted with a non-aromatic substituent; R3 is an aliphatic heterocycle that contains at least one nitrogen atom and is optionally substituted with a non-aromatic substituent; Q1 is selected from C-Ra and N; Q2 is selected from C-Rb and N; and Ra, Rb, Rc, and Rd are each independently selected from the group consisting of a hydrogen atom, halogen, a C1–8 alkyl, a C1–8 alkoxy, and a cyano group).
(FR)
La présente invention concerne un agent prophylactique ou thérapeutique pour amyotrophie spinale qui comprend un composé représenté par la formule (I) ou un sel de celui-ci (dans la formule : W1, W2 et W3 sont chacun indépendamment choisis dans le groupe constitué par C-R2, C-R3, C-Rc et C-Rd, et sont définis comme dans l'un quelconque de (i) à (iv) ; (i) lorsque W3 est C-R2, W1 est C-R3, W2 est C-Rc ou N, et R1 est un atome d'hydrogène ; (ii) lorsque W3 est C-R3, W1 est C-R2, W2 est C-Rc ou N, et R1 est un atome d'hydrogène, un alkyle en C1–8, ou un alcoxy en C1–8 ; (iii) lorsque W1 est C-R2, W2 est C-Rc ou N, W3 est C-Rd, et R1 est un hétérocycle aliphatique qui contient au moins un atome d'azote et est éventuellement substitué par un substituant non aromatique ; (iv) lorsque W2 est C-R2, W1 est C-Rc, W3 est C-Rd, et R1 est un hétérocycle aliphatique qui contient au moins un atome d'azote et est éventuellement substitué par un substituant non aromatique ; R2 est un cycle aromatique d'au moins six éléments qui est éventuellement substitué par un substituant non aromatique ; R3 représente un hétérocycle aliphatique qui contient au moins un atome d'azote et est éventuellement substitué par un substituant non aromatique ; Q1 est choisi parmi C-Ra et N ; Q2 est choisi parmi C-Rb et N ; et Ra, Rb, Rc et Rd sont chacun indépendamment choisis dans le groupe constitué par un atome d'hydrogène, un halogène, un groupe alkyle en C1–8, un groupe alcoxy en C1–8, et un groupe cyano).
(JA)
本発明の脊髄性筋萎縮症の予防または治療剤は、下記式(I):(式中、W、W、Wはそれぞれ独立してC-R、C-R、C-R、及びC-Rからなる群から選択され、下記(i)~(iv)のいずれかであり; (i)WがC-Rのとき、WがC-R、WがC-RまたはN、Rが水素原子; (ii)WがC-Rのとき、WがC-R、WがC-RまたはN、Rが水素原子、C1-8アルキル、またはC1-8アルコキシ; (iii)WがC-Rのとき、WがC-RまたはN、WがC-R、Rが非芳香性置換基で置換されてもよい、1つ以上の窒素原子を含む脂肪族複素環; (iv)WがC-Rのとき、WがC-R、WがC-R、Rが非芳香性置換基で置換されてもよい、1つ以上の窒素原子を含む脂肪族複素環; Rは、非芳香性置換基で置換されてもよい、6員以上の芳香環であり; Rは、非芳香性置換基で置換されてもよい、1つ以上の窒素原子を含む脂肪族複素環であり; QはC-R、Nから選択され; QはC-R、Nから選択され; R、R、R、Rはそれぞれ独立して水素原子、ハロゲン、C1-8アルキル、C1-8アルコキシ、及びシアノ基からなる群から選択される。) で表される化合物またはその塩を含む。
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