PE20211585A1 - Agente profilactico o terapeutico para atrofia muscular espinal - Google Patents

Agente profilactico o terapeutico para atrofia muscular espinal

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Publication number
PE20211585A1
PE20211585A1 PE2020002186A PE2020002186A PE20211585A1 PE 20211585 A1 PE20211585 A1 PE 20211585A1 PE 2020002186 A PE2020002186 A PE 2020002186A PE 2020002186 A PE2020002186 A PE 2020002186A PE 20211585 A1 PE20211585 A1 PE 20211585A1
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PE
Peru
Prior art keywords
aliphatic heterocycle
optionally substituted
muscular atrophy
spinal muscular
hydrogen atom
Prior art date
Application number
PE2020002186A
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English (en)
Inventor
Koji Fuji
Takeshi Yamasaki
Shunya Suzuki
Koji Ono
Hiroki TAKAHAGI
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Reborna Biosciences Inc
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Publication date
Application filed by Reborna Biosciences Inc filed Critical Reborna Biosciences Inc
Publication of PE20211585A1 publication Critical patent/PE20211585A1/es

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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/517—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
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    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/506—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
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    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
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    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • C07D401/04—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 containing two hetero rings directly linked by a ring-member-to-ring-member bond
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    • C07D471/02—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 C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
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    • 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
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    • A—HUMAN NECESSITIES
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Abstract

La presente invencion se refiere a compuestos representados por la formula 1 o una sal de estos, en donde: W1, W2 y W3 se seleccionan cada uno independientemente del grupo que consiste en C-R2, C-R3, C-Rc y C-Rd, y se definen mediante uno de los siguientes (i) a (iv): (i) cuando W3 es C-R2, entonces W1 es C-R3, W2 es C-Rc o N, y R1 es un atomo de hidrogeno; (ii) cuando W3 es C-R3, entonces W1 es C-R2, W2 es C-Rc o N, y R1 es un atomo de hidrogeno, alquilo C1-8 o alcoxi C1-8; (iii) cuando W1 es C-R2, entonces W2 es C-Rc o N, W3 es C-Rd, y R1 es un heterociclo alifatico que contiene uno o mas atomos de nitrogeno, donde el heterociclo alifatico se sustituye opcionalmente con un sustituyente no aromatico; y (iv) cuando W2 es C-R2, W1 es C-Rc, W3 es C-Rd, y R1 es un heterociclo alifatico que contiene uno o mas atomos de nitrogeno, donde el heterociclo alifatico se sustituye opcionalmente con un sustituyente no aromatico; R2 es un anillo aromatico de 6 o mas miembros opcionalmente sustituido con un sustituyente no aromatico; R3 es un heterociclo alifatico que contiene uno o mas atomos de nitrogeno, donde el heterociclo alifatico se sustituye opcionalmente con un sustituyente no aromatico; Q1 se selecciona de C-Ra y N; Q2 se selecciona de C-Rb y N; y Ra, Rb, Rc y Rd se seleccionan cada uno independientemente del grupo que consiste en un atomo de hidrogeno, halogeno, alquilo C1-8, alcoxi C1-8 y un grupo ciano. Son compuestos preferidos: (S)-6-(2,4-dimetilpiperazin-1-il)-2-(4,6-dimetilpirazolo[1,5-a]pirazin-2-il)quinazolin-4(3H)-ona; 2-(4,6-dimetilpirazolo[1,5-a]pirazin-2-il)-6-(4-metilpiperazin-1-il)quinazolin-4(3H)-ona, entre otros. Tambien se refiere a un medicamento, un regulador de empalme, un potenciador de expresion del ARNm de SMN con funcion normal y/o proteina SMN de funcion normal, un agente profilactico de atrofia muscular espinal (SMA) asociados al compuesto de la invencion. Los compuestos de la invencion tienen utilidad como agentes profilacticos o terapeuticos para la atrofia muscular espinal, potenciando la expresion del ARNm normal del gen de supervivencia de motoneuronas 1 (SMN1) o su proteina funcional.
PE2020002186A 2018-06-27 2019-06-27 Agente profilactico o terapeutico para atrofia muscular espinal PE20211585A1 (es)

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JP2018122551 2018-06-27
PCT/JP2019/025732 WO2020004594A1 (ja) 2018-06-27 2019-06-27 脊髄性筋萎縮症の予防または治療剤

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IL280213B1 (en) * 2018-07-26 2024-02-01 Domain Therapeutics Altered quinazolinone derivatives and their use as positive allosteric modulators of MGLUR4
CR20220484A (es) * 2020-02-28 2022-12-15 Remix Therapeutics Inc Amidas heterocíclicas y su uso para modular el empalme
CA3174353A1 (en) 2020-04-08 2021-10-14 Dominic Reynolds Compounds and methods for modulating splicing
CA3175193A1 (en) 2020-04-08 2021-10-14 Dominic Reynolds Compounds and methods for modulating splicing
US20240002375A1 (en) 2020-07-02 2024-01-04 Remix Therapeutics Inc. 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as modulators for splicing nucleic acids and for the tre
AU2021300260A1 (en) 2020-07-02 2023-02-09 Remix Therapeutics Inc. 5-[5-(piperidin-4-yl)thieno[3,2-c]pyrazol-2-yl]indazole derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
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