PE20170128A1 - Compuestos para tratar atrofia muscular espinal - Google Patents

Compuestos para tratar atrofia muscular espinal

Info

Publication number
PE20170128A1
PE20170128A1 PE2016002128A PE2016002128A PE20170128A1 PE 20170128 A1 PE20170128 A1 PE 20170128A1 PE 2016002128 A PE2016002128 A PE 2016002128A PE 2016002128 A PE2016002128 A PE 2016002128A PE 20170128 A1 PE20170128 A1 PE 20170128A1
Authority
PE
Peru
Prior art keywords
compounds
muscular atrophy
alkyl
spinal muscular
methylimidazo
Prior art date
Application number
PE2016002128A
Other languages
English (en)
Inventor
Luke Green
Marla L Weetall
Hasane Ratni
Nikolai A Naryshkin
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 PE20170128A1 publication Critical patent/PE20170128A1/es

Links

Classifications

    • 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
    • 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
    • A61K31/5025—Pyridazines; Hydrogenated pyridazines 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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • 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
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • 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
    • 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
    • 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

Abstract

Se refiere a compuestos de formula (I) y sales farmaceuticamente aceptables de los mismos, donde: A es N-heterocicloalquilo, NR12R13; R1 es H o alquilo C1-C7; R2 es H, CN, entre otros; R3 es H, alquilo C1-C7 o cicloalquilo C3-C8; R12 es heterocicloalquilo opcionalmente sustituido y que comprende un atomo de N; R13 es H, alquilo C1-C7 o cicloalquilo C3-C8. Son compuestos preferidos: 2-(2-metilimidazo[1,2-b]piridazin-6-il)-7-(4-metilpiperazin-1-il)pirido[1,2-a]pirimidin-4-ona; 7-[(8aR)-3,4,6,7,8,8a-hexahidro-1H-pirrolo[1,2-a]pirazin-2-il]-2-(2-metilimidazo[1,2-b]piridazin-6-il)pirido[1,2-a]pirimidin-4-ona; entre otros. Tambien se refiere a un proceso, composiciones farmaceuticas y un metodo. Dichos compuestos son moduladores de empalme genico de supervivencia de neuronas motoras (SMN2) siendo utiles en el tratamiento de la atrofia muscular espinal (AME)
PE2016002128A 2014-05-15 2015-05-11 Compuestos para tratar atrofia muscular espinal PE20170128A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201461993839P 2014-05-15 2014-05-15

Publications (1)

Publication Number Publication Date
PE20170128A1 true PE20170128A1 (es) 2017-03-16

Family

ID=53175048

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2016002128A PE20170128A1 (es) 2014-05-15 2015-05-11 Compuestos para tratar atrofia muscular espinal

Country Status (35)

Country Link
US (1) US9969754B2 (es)
EP (3) EP3143025B1 (es)
JP (1) JP6236173B2 (es)
KR (2) KR102256013B1 (es)
CN (1) CN106459092B (es)
AR (1) AR100442A1 (es)
AU (1) AU2015261046C1 (es)
CA (1) CA2948561C (es)
CL (1) CL2016002836A1 (es)
CR (1) CR20160518A (es)
DK (1) DK3143025T3 (es)
EA (2) EA202090486A3 (es)
ES (2) ES2949660T3 (es)
FR (1) FR21C1039I2 (es)
HR (2) HRP20230637T1 (es)
HU (2) HUE046491T2 (es)
IL (2) IL248653B (es)
LT (1) LTPA2021010I1 (es)
MA (2) MA39995B1 (es)
MX (1) MX371050B (es)
MY (1) MY174284A (es)
NL (1) NL301128I2 (es)
NO (1) NO2021035I1 (es)
NZ (1) NZ725008A (es)
PE (1) PE20170128A1 (es)
PH (1) PH12016502081A1 (es)
PL (2) PL3663296T3 (es)
PT (1) PT3143025T (es)
RS (1) RS59718B1 (es)
SG (1) SG11201609497TA (es)
SI (2) SI3143025T1 (es)
TW (1) TWI667239B (es)
UA (1) UA119670C2 (es)
WO (1) WO2015173181A1 (es)
ZA (1) ZA201607026B (es)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA037123B1 (ru) 2012-02-10 2021-02-09 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения спинальной мышечной атрофии
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
US10882868B2 (en) * 2014-05-15 2021-01-05 Hoffmann-La Roche Inc. Compounds for treating spinal muscular atrophy
JP6749343B2 (ja) 2015-05-20 2020-09-02 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 脊髄性筋萎縮症を処置するための化合物
US10668171B2 (en) 2015-05-30 2020-06-02 Ptc Therapeutics, Inc. Methods for modulating RNA splicing
CN108137601A (zh) * 2015-11-12 2018-06-08 豪夫迈·罗氏有限公司 用于治疗肌萎缩性侧索硬化症的化合物
JP6659841B2 (ja) * 2015-11-12 2020-03-04 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 脊髄性筋萎縮症を処置するための組成物
EP3386978B1 (en) 2015-12-10 2021-01-27 H. Hoffnabb-La Roche Ag Bridged piperidine derivatives
LT3386511T (lt) 2015-12-10 2021-08-25 Ptc Therapeutics, Inc. Hantingtono ligos gydymo būdai
AU2017363369A1 (en) * 2016-11-28 2019-05-30 Ptc Therapeutics, Inc Methods for modulating RNA splicing
IL300875A (en) 2017-06-05 2023-04-01 Ptc Therapeutics Inc Compounds for the treatment of Huntington's disease
CN111372611A (zh) 2017-06-14 2020-07-03 Ptc医疗公司 修饰rna剪接的方法
US11395822B2 (en) 2017-06-28 2022-07-26 Ptc Therapeutics, Inc. Methods for treating Huntington's disease
MX2019015578A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
EP3684766A1 (en) * 2017-09-22 2020-07-29 H. Hoffnabb-La Roche Ag Process for the prepration of 7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-one derivatives
JP7423515B2 (ja) * 2017-10-03 2024-01-29 エフ． ホフマン－ラ ロシュ アーゲー Ｓｍａの新たな処置
WO2019191092A1 (en) 2018-03-27 2019-10-03 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
SG11202012674PA (en) 2018-06-27 2021-01-28 Ptc Therapeutics Inc Heterocyclic and heteroaryl compounds for treating huntington's disease
KR20210079317A (ko) 2018-10-19 2021-06-29 에프. 호프만-라 로슈 아게 피리도[1,2-a]피리미딘-4-온 유도체의 형태, 그의 제형 및 그의 제조 방법
CN113966219A (zh) 2019-06-12 2022-01-21 豪夫迈·罗氏有限公司 Sma的新治疗方法
US20220315607A1 (en) 2019-07-31 2022-10-06 Teva Pharmaceuticals International Gmbh Solid state forms of risdiplam and process for preparation thereof
WO2022048675A1 (zh) * 2020-09-07 2022-03-10 苏州科睿思制药有限公司 Risdiplam晶型及其制备方法和用途
WO2022162107A1 (en) 2021-02-01 2022-08-04 Sandoz Ag Crystalline form of risdiplam
KR20230157367A (ko) 2021-03-17 2023-11-16 에프. 호프만-라 로슈 아게 신규 티아졸로피리미디논 유도체
CN116981673A (zh) * 2021-03-17 2023-10-31 豪夫迈·罗氏有限公司 新颖噻二唑并嘧啶酮衍生物
BR112023018593A2 (pt) 2021-03-18 2023-10-24 Hoffmann La Roche Processo
WO2023057404A1 (en) 2021-10-06 2023-04-13 F. Hoffmann-La Roche Ag Novel combined administration
WO2023202501A1 (zh) * 2022-04-18 2023-10-26 深圳市塔吉瑞生物医药有限公司 取代的吡啶并嘧啶酮类化合物及包含该化合物的组合物及其用途
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2024081932A1 (en) 2022-10-14 2024-04-18 Genentech, Inc. Methods for treating spinal muscular atrophy

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69719193T2 (de) * 1996-08-06 2003-09-25 Pfizer Substituierte pyrido- oder pyrimidogruppen enthaltende 6,6- oder 6,7-bicyclische derivate
WO2009042907A1 (en) 2007-09-27 2009-04-02 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
KR20100012134A (ko) * 2008-07-28 2010-02-08 신호열 마우스
WO2010019243A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc. Methods for treating viral infections
JP2011529722A (ja) * 2008-08-14 2011-12-15 カーディアック ペースメイカーズ， インコーポレイテッド 音響通信リンクの性能評価および適合
BR112014016287B1 (pt) * 2011-12-30 2022-08-09 F. Hoffmann - La Roche Ag Composto e composição farmacêutica
EA037123B1 (ru) * 2012-02-10 2021-02-09 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения спинальной мышечной атрофии
EP2872493B1 (en) 2012-07-13 2018-11-14 Indiana University Research and Technology Corporation 5,6,7-trimethoxy 4-phenyl quinolin-2-one derivatives for treatment of spinal muscular atrophy
JP6659841B2 (ja) 2015-11-12 2020-03-04 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 脊髄性筋萎縮症を処置するための組成物

Also Published As

Publication number Publication date
EA202090486A2 (ru) 2020-06-30
HRP20230637T1 (hr) 2023-09-29
KR20170003687A (ko) 2017-01-09
IL248653A0 (en) 2017-01-31
IL248653B (en) 2019-11-28
JP6236173B2 (ja) 2017-11-22
LTPA2021010I1 (es) 2021-10-11
EA202090486A3 (ru) 2020-08-31
FR21C1039I1 (es) 2021-10-15
CN106459092A (zh) 2017-02-22
KR102213740B1 (ko) 2021-02-09
AU2015261046C1 (en) 2019-07-25
PL3143025T3 (pl) 2020-03-31
SI3143025T1 (sl) 2020-01-31
US9969754B2 (en) 2018-05-15
BR112016026205A2 (pt) 2017-08-15
AU2015261046B2 (en) 2019-04-18
PL3663296T3 (pl) 2023-08-07
UA119670C2 (uk) 2019-07-25
NL301128I1 (es) 2021-09-22
EP3143025A1 (en) 2017-03-22
HRP20192159T1 (hr) 2020-02-21
CL2016002836A1 (es) 2017-04-21
IL270027B (en) 2020-08-31
EA201692280A1 (ru) 2018-07-31
MX371050B (es) 2020-01-14
TW201609738A (zh) 2016-03-16
MA51988A (fr) 2021-01-20
PT3143025T (pt) 2019-12-03
CN106459092B (zh) 2019-10-15
HUS2100037I1 (hu) 2021-10-28
SI3663296T1 (sl) 2023-08-31
EP4241772A3 (en) 2023-11-15
AR100442A1 (es) 2016-10-05
MY174284A (en) 2020-04-01
CA2948561C (en) 2019-10-22
SG11201609497TA (en) 2016-12-29
EP3663296A1 (en) 2020-06-10
BR112016026205A8 (pt) 2021-07-20
EA035068B1 (ru) 2020-04-23
NO2021035I1 (no) 2021-09-10
EP3143025B1 (en) 2019-10-09
ZA201607026B (en) 2022-05-25
US20170197990A1 (en) 2017-07-13
EP4241772A2 (en) 2023-09-13
PH12016502081A1 (en) 2017-01-09
CR20160518A (es) 2017-02-21
JP2017515863A (ja) 2017-06-15
CA2948561A1 (en) 2015-11-19
HUE046491T2 (hu) 2020-03-30
WO2015173181A1 (en) 2015-11-19
FR21C1039I2 (fr) 2022-09-02
KR20210014219A (ko) 2021-02-08
RS59718B1 (sr) 2020-01-31
DK3143025T3 (da) 2019-12-09
KR102256013B1 (ko) 2021-05-26
MA39995B1 (fr) 2019-11-29
ES2761423T3 (es) 2020-05-19
TWI667239B (zh) 2019-08-01
NZ725008A (en) 2019-11-29
NL301128I2 (nl) 2021-10-25
MX2016014547A (es) 2017-02-23
ES2949660T3 (es) 2023-10-02
AU2015261046A1 (en) 2016-10-27
EP3663296B1 (en) 2023-05-17

Similar Documents

Publication Publication Date Title
PE20170128A1 (es) Compuestos para tratar atrofia muscular espinal
NI201900061A (es) Imidazopirrolopiridina como inhibidores de la familia de quinasas jak
NI201900060A (es) Inhibidores de moléculas pequeñas de la familia de quinasa jak
PE20171307A1 (es) Compuestos de triazolopirimidina y usos de los mismos
EA202090659A1 (ru) СОСТАВЫ, СОДЕРЖАЩИЕ 6-(2-ГИДРОКСИ-2-МЕТИЛПРОПОКСИ)-4-(6-(6-((6-МЕТОКСИПИРИДИН-3-ИЛ)МЕТИЛ)-3,6-ДИАЗАБИЦИКЛО[3.1.1]ГЕПТАН-3-ИЛ)ПИРИДИН-3-ИЛ)ПИРАЗОЛО[1,5-a]ПИРИДИН-3-КАРБОНИТРИЛ
PE20230731A1 (es) Compuestos heterociclicos como inmunomoduladores
PE20190811A1 (es) Derivados de pirazolopirimidinas como inhibidor de quinasa
MX2018003215A (es) Derivados de imidazo[4,5-c]quinolina e imidazo[4,5-c][1,5]naftirid ina novedosos como inhibidores de lrrk2.
PE20160588A1 (es) Compuestos de triazolopiridinas y triazolopirazinas inhibidores de c-met y composiciones de los mismos
GT201300291A (es) Derivados de piridin-2(1h)-ona útiles como medicamentos para el tratamiento de trastornos mieloproliferativos, rechazo de trasplantes, enfermedades mediadas por el sistema inmune y enfermedades inflamatorias.
MX2023000782A (es) 7-bencil-4-(2-metilbencilo)-2,4,6,7,8,9-hexahidroimidazo [1,2-a]pirido[3,4-e]pirimidin-5(1h)-ona, analogos y sales del mismo y su uso en terapia.
EA201991109A1 (ru) ПРОИЗВОДНЫЕ [1,2,4]ТРИАЗОЛО[1,5-a]ПИРИМИДИНА В КАЧЕСТВЕ ИНГИБИТОРОВ PDE2
CL2008003578A1 (es) Compuestos derivados de imidazo[1,5-a]-quinoxalina, inhibidores pde10; composicion farmaceutica que los comprende y/o kit; procedimiento de preparacion de estos; y su uso en el tratamiento de enfermedades snc
CL2012002487A1 (es) Compuestos derivados de imidazo[1,2-a]pirazina, inhibidores de pde10; composicion farmacéutica que los comprende; proceso para preparar la composicion farmaceutica; utiles para el tratamiento de enfermedades neurológicas, psiquiátricas o metabólicas tales como esquizofrenia, parkinson y diabetes, entre otras.
EA201891067A1 (ru) [1,2,4]ТРИАЗОЛО[1,5-a]ПИРИМИДИН-7-ИЛЬНОЕ СОЕДИНЕНИЕ
PE20141023A1 (es) Derivados de (1,2,4) triazolo [4,3-a] quinoxalina como inhibidores de fosfodiesterasas
ECSP19043790A (es) Pirazolo[3,4-b]piridinas e imidazo[1,5-b]piridazinas como inhibidores de PDE1
PE20150754A1 (es) Derivados de azaindol que actuan como inhibidores de pi 3k
PE20170295A1 (es) Compuestos de imidazopiridazina
CL2017000223A1 (es) [1,2,4]triazolo[4,3-b]piridazinas para su uso en el tratamiento de enfermedades proliferativas
NI202000100A (es) Inhibidores de moléculas pequeñas de la familia de quinasa jak
EA201991094A1 (ru) СОЕДИНЕНИЯ [1,2,4]ТРИАЗОЛО[1,5-a]ПИРИМИДИНА В КАЧЕСТВЕ ИНГИБИТОРОВ PDE2
EA201990973A1 (ru) СОЕДИНЕНИЯ [1,2,4]ТРИАЗОЛО[1,5-a]ПИРИМИДИНА В КАЧЕСТВЕ ИНГИБИТОРОВ PDE2
BR112015000459A2 (pt) inibidores da enzima fosfodiesterase 10
PE20150965A1 (es) Azaindolinas