AR096684A1 - Compuestos para tratar atrofia muscular espinal - Google Patents

Compuestos para tratar atrofia muscular espinal

Info

Publication number
AR096684A1
AR096684A1 ARP140102349A ARP140102349A AR096684A1 AR 096684 A1 AR096684 A1 AR 096684A1 AR P140102349 A ARP140102349 A AR P140102349A AR P140102349 A ARP140102349 A AR P140102349A AR 096684 A1 AR096684 A1 AR 096684A1
Authority
AR
Argentina
Prior art keywords
alkyl
haloalkyl
halo
haloalkoxy
alkoxy
Prior art date
Application number
ARP140102349A
Other languages
English (en)
Inventor
Narasimhan Jana
Green Luke
Qi Hongyan
Mitchell Karp Gary
G Woll Matthew
K Risher Nicole
L Weetall Maria
A Naryshkin Nikolai
Dakka Amal
Ratni Hasame
Pinard Emmanuel
Original Assignee
Ptc Therapeutics Inc
Hoffmann La Roche
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Ptc Therapeutics Inc, Hoffmann La Roche filed Critical Ptc Therapeutics Inc
Publication of AR096684A1 publication Critical patent/AR096684A1/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D519/00Heterocyclic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/437Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/496Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/4985Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D263/00Heterocyclic compounds containing 1,3-oxazole or hydrogenated 1,3-oxazole rings
    • C07D263/52Heterocyclic compounds containing 1,3-oxazole or hydrogenated 1,3-oxazole rings condensed with carbocyclic rings or ring systems
    • C07D263/54Benzoxazoles; Hydrogenated benzoxazoles
    • C07D263/56Benzoxazoles; Hydrogenated benzoxazoles with only hydrogen atoms, hydrocarbon or substituted hydrocarbon radicals, directly attached in position 2
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D413/00Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D413/02Heterocyclic 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
    • C07D413/12Heterocyclic 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 linked by a chain containing hetero atoms as chain links
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D413/00Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D413/14Heterocyclic 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D471/00Heterocyclic 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
    • C07D471/02Heterocyclic 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
    • C07D471/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02Heterocyclic 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
    • C07D487/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02Heterocyclic 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
    • C07D487/10Spiro-condensed systems

Abstract

Reivindicación 1: Compuestos de fórmula (1) en la que X es N o CR³; Y es N o CR⁴; con la condición de que X e Y no son ambos N; A es un heteroarilo bicíclico de 9 miembros que comprende dos o tres heteroátomos seleccionados independientemente entre N u O, en la que A puede estar opcionalmente sustituido con uno, dos o tres R⁵; B es un heterocicloalquilo de 4 a 9 miembros mono o bicíclico saturado o parcialmente insaturado que comprende uno o dos átomos de nitrógeno en el anillo, siendo carbono los demás átomos en el anillo, en la que B puede estar opcionalmente sustituido con uno, dos o tres R⁶; R¹ es hidrógeno, halo, alquilo C₁₋₇, haloalquilo C₁₋₇, alcoxi C₁₋₇ o haloalcoxi C₁₋₇; R² es hidrógeno, halo, alquilo C₁₋₇ haloalquilo C₁₋₇, alcoxi C₁₋₇ o haloalcoxi C₁₋₇; R³ es hidrógeno, halo, alquilo C₁₋₇ haloalquilo C₁₋₇, alcoxi C₁₋₇ o haloalcoxi C₁₋₇; R⁴ es hidrógeno, halo, alquilo C₁₋₇, haloalquilo C₁₋₇ alcoxi C₁₋₇ o haloalcoxi C₁₋₇; cada R⁵ se selecciona independientemente entre halo, ciano, alquilo C₁₋₇, haloalquilo C₁₋₇ o cicloalquilo C₃₋₇; cada R⁶ se selecciona independientemente entre alquilo C₁₋₇ o dos R⁶ juntos forman un alquileno C₂₋₇; y sales farmacéuticamente aceptables de los mismos.
ARP140102349A 2013-06-25 2014-06-23 Compuestos para tratar atrofia muscular espinal AR096684A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP13173487 2013-06-25

Publications (1)

Publication Number Publication Date
AR096684A1 true AR096684A1 (es) 2016-01-27

Family

ID=48669815

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP140102349A AR096684A1 (es) 2013-06-25 2014-06-23 Compuestos para tratar atrofia muscular espinal

Country Status (13)

Country Link
US (2) US20160145270A1 (es)
EP (1) EP3013345B1 (es)
JP (1) JP6492071B2 (es)
KR (1) KR102314290B1 (es)
CN (1) CN105358151B (es)
AR (1) AR096684A1 (es)
BR (1) BR112015030288B1 (es)
CA (1) CA2913785C (es)
HK (1) HK1215399A1 (es)
MX (1) MX363456B (es)
RU (1) RU2673542C2 (es)
TW (1) TW201542543A (es)
WO (1) WO2014209841A2 (es)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105392790B (zh) 2013-08-19 2019-04-19 豪夫迈·罗氏有限公司 4H-吡啶并[1,2-a]嘧啶-4-酮化合物制备用于预防或治疗癌症的药物的用途
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
RU2725979C2 (ru) 2014-06-25 2020-07-08 Ф. Хоффманн-Ля Рош Аг ИМИДАЗО[1,2-а]ПИРАЗИН-1-ИЛ-БЕНЗАМИДЫ ДЛЯ ЛЕЧЕНИЯ СПИНАЛЬНОЙ МЫШЕЧНОЙ АТРОФИИ
KR102620328B1 (ko) 2014-10-03 2024-01-02 콜드스프링하버러보러토리 핵 유전자 산출량의 표적화 증강
WO2016161572A1 (en) 2015-04-08 2016-10-13 Merck Sharp & Dohme Corp. TrkA KINASE INHIBITORS, COMPOSITIONS AND METHODS THEREOF
SG11201802870RA (en) 2015-10-09 2018-05-30 Univ Southampton Modulation of gene expression and screening for deregulated protein expression
WO2017080967A1 (en) 2015-11-12 2017-05-18 F. Hoffmann-La Roche Ag Compositions for treating spinal muscular atrophy
JP6872550B2 (ja) 2015-12-10 2021-05-19 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft 架橋されたピペリジン誘導体
CN113750101A (zh) 2015-12-10 2021-12-07 Ptc医疗公司 用于治疗亨廷顿病的方法
CN109312343B (zh) 2015-12-14 2022-09-27 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
SG11201911615WA (en) 2017-06-05 2020-01-30 Ptc Therapeutics Inc Compounds for treating huntington's disease
MX2019015578A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
CA3067592A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
SG10202002990XA (en) 2017-08-04 2020-05-28 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
SG10202108375XA (en) 2017-08-25 2021-09-29 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
MX2020009957A (es) 2018-03-27 2021-01-15 Ptc Therapeutics Inc Compuestos para el tratamiento de enfermedad de hungtinton.
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
PE20211378A1 (es) 2018-06-27 2021-07-27 Ptc Therapeutics Inc Compuestos heterociclicos y de heteroarilo para tratar la enfermedad de huntington
JP2022517410A (ja) * 2019-01-18 2022-03-08 バイオジェン・エムエイ・インコーポレイテッド イミダゾ[1,2-a]ピリジニル誘導体及び疾患の処置におけるその使用
JP2022521467A (ja) 2019-02-05 2022-04-08 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
WO2021007378A1 (en) 2019-07-11 2021-01-14 Ptc Therapeutics, Inc. Compounds for use in treating huntington's disease
IL298063A (en) 2020-05-11 2023-01-01 Stoke Therapeutics Inc opa1 antisense oligomers for the treatment of conditions and diseases
TW202208358A (zh) 2020-05-13 2022-03-01 美商Chdi基金會股份有限公司 用於治療亨汀頓舞蹈症之htt調節劑
WO2022169230A1 (ko) * 2021-02-03 2022-08-11 단국대학교 천안캠퍼스 산학협력단 6-메톡시니코틴아미드를 유효성분으로 포함하는 근감소증의 예방 또는 치료용 조성물
WO2023102231A1 (en) * 2021-12-03 2023-06-08 Rgenta Therapeutics, Inc. 5-pyrimidinecarboxamide derivatives and methods of using the same

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SE9801526D0 (sv) * 1998-04-29 1998-04-29 Astra Ab New compounds
CN101128199B (zh) * 2004-04-23 2013-07-24 布里斯托尔-迈尔斯斯奎布公司 作为激酶抑制剂的单环杂环
AR056187A1 (es) * 2005-03-21 2007-09-26 S Bio Pte Ltd Derivados de imidazo[1,2-a)piridina: preparacion, composiciones farmaceuticas y uso para preparar medicamentos
US7666880B2 (en) * 2005-03-21 2010-02-23 S*Bio Pte Ltd. Imidazo[1,2-A]pyridine derivatives: preparation and pharmaceutical applications
US20090105264A1 (en) * 2005-11-03 2009-04-23 Christopher Hamblett Substituted Nicotinamide Compounds
EP2215074B1 (en) * 2007-09-27 2014-02-19 The United States of America, as Represented by the Secretary, Department of Health and Human Services Isoindoline compounds for the treatment of spinal muscular atrophy and other uses
WO2009151546A2 (en) * 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
EP2280959B1 (en) * 2008-06-05 2012-04-04 Glaxo Group Limited 4-amino-indazoles
WO2010019243A1 (en) * 2008-08-13 2010-02-18 Ptc Therapeutics, Inc. Methods for treating viral infections
UY32138A (es) * 2008-09-25 2010-04-30 Boehringer Ingelheim Int Amidas sustituidas del ácido 2-(2,6-dicloro-fenilamino)-6-fluoro-1-metil-1h-bencimidazol-5-carboxílico y sus sales farmacéuticamente aceptables
WO2012142459A1 (en) * 2011-04-14 2012-10-18 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isoindoline compounds for the treatment of spinal muscular atrophy and other uses

Also Published As

Publication number Publication date
WO2014209841A2 (en) 2014-12-31
TW201542543A (zh) 2015-11-16
RU2673542C2 (ru) 2018-11-28
EP3013345B1 (en) 2017-11-22
BR112015030288A2 (es) 2017-08-22
CN105358151A (zh) 2016-02-24
RU2015154676A3 (es) 2018-03-15
MX2015017345A (es) 2016-08-03
US20160145270A1 (en) 2016-05-26
BR112015030288B1 (pt) 2022-10-18
MX363456B (es) 2019-03-25
EP3013345A4 (en) 2016-11-30
CN105358151B (zh) 2019-04-12
EP3013345A2 (en) 2016-05-04
US10501482B2 (en) 2019-12-10
KR20160022312A (ko) 2016-02-29
WO2014209841A3 (en) 2015-10-29
CA2913785A1 (en) 2014-12-31
RU2015154676A (ru) 2017-07-28
CA2913785C (en) 2021-08-31
KR102314290B1 (ko) 2021-10-21
HK1215399A1 (zh) 2016-08-26
JP2016530231A (ja) 2016-09-29
US20180222925A1 (en) 2018-08-09
JP6492071B2 (ja) 2019-03-27

Similar Documents

Publication Publication Date Title
AR096684A1 (es) Compuestos para tratar atrofia muscular espinal
AR100809A1 (es) Inhibidores de fosfatidilinositol 3-quinasa
AR099379A1 (es) Compuestos tricíclicos como agentes antineoplásicos
AR100808A1 (es) Inhibidores de fosfatidilinositol 3-quinasa
AR100442A1 (es) Derivados de pirido[1,2-a]pirimidin-4-ona para tratar atrofia muscular espinal
AR104326A1 (es) Compuestos nucleósidos 5-sustituidos
AR099363A1 (es) Pirrolo[2,3-d]pirimidinil, pirrolo[2,3-b]pirazinil y pirrolo[2,3-d]piridinil acrilamidas
AR094668A1 (es) Amidas como moduladores de canales de sodio
GT201200346A (es) Derivados de imidazopiridina, su procedimiento de preparaciòn y su aplicaciòn en terapèutica
AR089671A1 (es) 1,4-dihidropirimidinas 4,4-disustituidas y su uso como medicamentos para el tratamiento de la hepatitis b
AR099415A1 (es) Benzoilfenil-formamidinas con actividad fungicida, sus composiciones agronómicas y su uso para el control de hongos fitopatogénicos de cultivos agrarios
AR097866A1 (es) Derivados de 4-azaindol
AR106053A1 (es) Compuestos imidazo[4,5-c]quinolin-2-ona y su uso para el tratamiento del cáncer
NI201200191A (es) Derivados de indolizina, su procedimiento de preparación y su aplicación en terapéutica
AR106100A1 (es) Compuestos bicíclicos como inhibidores duales atx / ca
MA38982A1 (fr) Nouvel octahydro-cyclobuta [1,2-c; 3,4-c'] dipyrrol-2-yl
AR094702A1 (es) Compuestos de azabencimidazol
EA201390163A1 (ru) Гетеробициклические производные в качетстве ингибиторов hcv
CL2019001694A1 (es) Compuestos de pirazolopirimidina y métodos de uso de los mismos.
AR101051A1 (es) Compuestos no esteroideos inhibidores de cyp17 / antiandrógenos
CR20160070A (es) Compuestos de triazolopiridina, composiciones y métodos de uso de los mismos
AR100776A1 (es) Compuestos herbicidas
AR090557A1 (es) DERIVADOS IMIDAZOLICOS AGONISTAS ADRENERGICOS a2
PE20151669A1 (es) Compuestos de azetidiniloxifenilpirrolidina
AR102258A1 (es) Compuestos de quinolina y quinazolina

Legal Events

Date Code Title Description
FB Suspension of granting procedure