AR122072A1 - Moduladores htt para tratar la enfermedad de huntington - Google Patents

Moduladores htt para tratar la enfermedad de huntington

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Publication number
AR122072A1
AR122072A1 ARP210101306A ARP210101306A AR122072A1 AR 122072 A1 AR122072 A1 AR 122072A1 AR P210101306 A ARP210101306 A AR P210101306A AR P210101306 A ARP210101306 A AR P210101306A AR 122072 A1 AR122072 A1 AR 122072A1
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Argentina
Prior art keywords
alkyl
optionally substituted
heterocyclyl
halo
haloalkyl
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ARP210101306A
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English (en)
Inventor
Celia Dominguez
Elizabeth M Doherty
Longbin Liu
Matthew Lee
Mark Stuart Chambers
Karine Fabienne Malagu
Perla Breccia
Alan F Haughan
Huw D Vater
Andrew J Stott
William R K Esmieu
Stephen John Webster
De Pol Amanda J Van
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Chdi Foundation Inc
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Publication of AR122072A1 publication Critical patent/AR122072A1/es

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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • 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
    • A61K31/4965—Non-condensed pyrazines
    • A61K31/497—Non-condensed pyrazines containing further heterocyclic rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—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
    • 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
    • C07D471/04—Ortho-condensed systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • 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
    • A61K31/4985—Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • 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
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/501—Pyridazines; Hydrogenated pyridazines not condensed and containing further heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • 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
    • A61K31/506—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/53—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with three nitrogens as the only ring hetero atoms, e.g. chlorazanil, melamine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings
    • C07D403/04—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D413/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D413/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings
    • C07D413/04—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D413/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D413/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
    • 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
    • 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
    • C07D487/10—Spiro-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D498/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D498/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D498/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Magnetic Treatment Devices (AREA)
  • Hydrogenated Pyridines (AREA)
  • Electrotherapy Devices (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

En el presente documento se proporcionan ciertos compuestos útiles como moduladores de HTT. Tales compuestos son útiles en el tratamiento de la enfermedad de Huntington. Reivindicación 1: Un compuesto de fórmula (1) o un análogo enriquecido isotópicamente, una sal, profármaco, tautómero, estereoisómero o una mezcla de estereoisómeros farmacéuticamente aceptables del mismo, en donde: X¹, X², X³, y X⁴ son CR⁴ o N, en donde al menos dos pero no más de tres de X¹, X², X³, y X⁴ son N; cada R⁴ es independientemente hidrógeno, halo, hidroxi, alquiloC₁₋₆, haloalquiloC₁₋₆, o alcoxiC₁₋₆; Y¹ es CR⁵ o N; R⁵ es hidrógeno, ciano, halo, hidroxi, alquiloC₁₋₆, haloalquiloC₁₋₆, alquiloC₁₋₆tio, alcoxiC₁₋₆, haloalcoxiC₁₋₆, heterociclilo, -NH₂, -NHR¹⁷, o -N(R¹⁷)₂, y opcionalmente sustituido en un átomo de nitrógeno disponible con alquiloC₁₋₆ o haloalquiloC₁₋₆; Y² está ausente, CR⁶ o N; R⁶ es hidrógeno, ciano, halo, hidroxi, alquiloC₁₋₆, haloalquiloC₁₋₆, alquiloC₁₋₆tio, alcoxiC₁₋₆, haloalcoxiC₁₋₆, heterociclilo, -NH₂, -NHR¹⁷, o -N(R¹⁷)₂, y opcionalmente sustituido en un átomo de nitrógeno disponible con alquiloC₁₋₆ o haloalquiloC₁₋₆; y Y³ es CR³ o N; R³ es hidrógeno, ciano, halo, hidroxi, alquiloC₁₋₆, haloalquiloC₁₋₆, alquiloC₁₋₆tio, alcoxiC₁₋₆, haloalcoxiC₁₋₆, heterociclilo, -NH₂, -NHR¹⁷, o -N(R¹⁷)₂, y opcionalmente sustituido en un átomo de nitrógeno disponible con alquiloC₁₋₆ o haloalquiloC₁₋₆; cada R¹⁷ es independientemente alquiloC₁₋₄, o dos R¹⁷ se unen, con cualquier átomo que intervenga, para formar un heterociclilo de 3 a 6 miembros; cada uno de Z¹ y Z² es C o N; anillo A y anillo B juntos forman un heteroarilo bicíclico de 9 ó 10 miembros que contiene 1 a 3 átomos de nitrógeno del anillo; Anillo B contiene 1 a 3 heteroátomos independientemente seleccionados de N, O, y S, y está opcionalmente sustituido en los átomos de carbono disponibles con 1 a 3 sustituyentes seleccionados independientemente de halo, hidroxi, alquiloC₁₋₆, haloalquiloC₁₋₆, alcoxiC₁₋₆, y haloalcoxiC₁₋₆, y opcionalmente sustituido en un átomo de nitrógeno disponible con alquiloC₁₋₆ o haloalquiloC₁₋₆; R¹ es -L¹-R¹¹, en donde L¹ es -O-, -S-, -S(O)-, -S(O)₂-, -N(R¹²)-, -alquilenoC₁₋₃-, -O-alquilenoC₁₋₃-, -N(R¹²)-alquilenoC₁₋₃-, o ausente, y R¹¹ es alquiniloC₂₋₆, cicloalquiloC₃₋₁₀, ariloC₆₋₁₀, heteroarilo, o heterociclilo, en donde R¹¹ está opcionalmente sustituido con 1 a 4 grupos R¹³; R¹² es hidrógeno o alquiloC₁₋₆; cada R¹³ se selecciona independientemente de halo, ciano, hidroxi, alquiloC₁₋₆ opcionalmente sustituido con R¹⁶, haloalquiloC₁₋₆, hidroxialquiloC₁₋₆, cicloalquiloC₃₋₁₀ opcionalmente sustituido con R¹⁶, cicloalquiloC₃₋₁₀-alquiloC₁₋₆ opcionalmente sustituido con R¹⁶, ariloC₆₋₁₀ opcionalmente sustituido con R¹⁶, ariloC₆₋₁₀-alquiloC₁₋₆ opcionalmente sustituido con R¹⁶, heteroarilo opcionalmente sustituido con R¹⁶, heteroaril-alquiloC₁₋₆ opcionalmente sustituido con R¹⁶, heterociclilo opcionalmente sustituido con R¹⁶, heterociclilo-alquiloC₁₋₆ opcionalmente sustituido con R¹⁶, OR¹⁴, -NH₂, -NHR¹⁴, -N(R¹⁴)₂, -alquilenoC₁₋₆-NH₂, -alquilenoC₁₋₆-NHR¹⁴, -alquilenoC₁₋₆-N(R¹⁴)₂, -C(O)R¹⁵, -C(O)OR¹⁵, -C(O)NHR¹⁵, -C(O)N(alquiloC₁₋₄)R¹⁵, -S(O)₂R¹⁵, -S(O)R¹⁵, -NHC(O)R¹⁵, -N(alquiloC₁₋₄)C(O)R¹⁵, -NHS(O)R¹⁵, -N(alquiloC₁₋₄)S(O)R¹⁵, -NHS(O)₂R¹⁵, y -N(alquiloC₁₋₄)S(O)₂R¹⁵; cada R¹⁴ se selecciona independientemente de alquiloC₁₋₆, cicloalquiloC₃₋₁₀, ariloC₆₋₁₀, heteroarilo, y heterociclilo; y cada R¹⁴ está opcionalmente sustituido con uno a seis halo, alquiloC₁₋₃, alcoxiC₁₋₃, cicloalquiloC₃₋₁₀, o -NHSO₂-aril-N(CH₃)₂; cada R¹⁵ es independientemente hidrógeno, -OH, alquiloC₁₋₆, haloalquiloC₁₋₆, cicloalquiloC₃₋₁₀, ariloC₆₋₁₀, heteroarilo, o heterociclilo; cada R¹⁶ es independientemente halo, ciano, hidroxi, -NH₂, -NHR²¹, -N(R²¹)₂, alquiloC₁₋₆, haloalquiloC₁₋₆, OR²¹, o cicloalquiloC₃₋₁₀; cada R²¹ se selecciona independientemente de alquiloC₁₋₆, cicloalquiloC₃₋₁₀, ariloC₆₋₁₀, heteroarilo, y heterociclilo, y cada R²¹ está opcionalmente sustituido con uno a seis halo o alcoxiC₁₋₃; y R² es hidrógeno o alquiloC₁₋₆.
ARP210101306A 2020-05-13 2021-05-12 Moduladores htt para tratar la enfermedad de huntington AR122072A1 (es)

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US (1) US11806346B2 (es)
EP (1) EP4149937A1 (es)
JP (1) JP2023532623A (es)
KR (1) KR20230028269A (es)
CN (1) CN116997548A (es)
AR (1) AR122072A1 (es)
AU (1) AU2021272972A1 (es)
BR (1) BR112022023025A2 (es)
CA (1) CA3182912A1 (es)
IL (1) IL298109A (es)
MX (1) MX2022013856A (es)
TW (1) TW202208358A (es)
WO (1) WO2021231571A1 (es)

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CN116997548A (zh) 2023-11-03
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MX2022013856A (es) 2023-04-05
US11806346B2 (en) 2023-11-07
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