CL2016002836A1 - Compuestos derivados de pirido pirimidin con actividad moduladora del empalme genico de smn2; composicion farmaceutica y uso en el tratamiento de la atrofia muscular espinal (ame). - Google Patents

Compuestos derivados de pirido pirimidin con actividad moduladora del empalme genico de smn2; composicion farmaceutica y uso en el tratamiento de la atrofia muscular espinal (ame).

Info

Publication number
CL2016002836A1
CL2016002836A1 CL2016002836A CL2016002836A CL2016002836A1 CL 2016002836 A1 CL2016002836 A1 CL 2016002836A1 CL 2016002836 A CL2016002836 A CL 2016002836A CL 2016002836 A CL2016002836 A CL 2016002836A CL 2016002836 A1 CL2016002836 A1 CL 2016002836A1
Authority
CL
Chile
Prior art keywords
ame
pyrido
pyrimidin
treatment
muscular atrophy
Prior art date
Application number
CL2016002836A
Other languages
English (en)
Inventor
Hasane Ratni
Luke Green
Nikolai A Naryshkin
Marla L Weetall
Original Assignee
Hoffmann La Roche
Ptc Therapeutics Inc
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 Hoffmann La Roche, Ptc Therapeutics Inc filed Critical Hoffmann La Roche
Publication of CL2016002836A1 publication Critical patent/CL2016002836A1/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

Compuestos dericvados de 2-(imidazo[1,2-b]piridazin-6-il)-4h-pirido[1,2-a]pirimidin-4-ona; proceso de preparacion; composicion farmceutica; y su uso para el tratamiento o la prevencion de la atrofia muscular espinal (ame)
CL2016002836A 2014-05-15 2016-11-08 Compuestos derivados de pirido pirimidin con actividad moduladora del empalme genico de smn2; composicion farmaceutica y uso en el tratamiento de la atrofia muscular espinal (ame). CL2016002836A1 (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
CL2016002836A1 true CL2016002836A1 (es) 2017-04-21

Family

ID=53175048

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2016002836A CL2016002836A1 (es) 2014-05-15 2016-11-08 Compuestos derivados de pirido pirimidin con actividad moduladora del empalme genico de smn2; composicion farmaceutica y uso en el tratamiento de la atrofia muscular espinal (ame).

Country Status (35)

Country Link
US (1) US9969754B2 (es)
EP (3) EP3663296B1 (es)
JP (1) JP6236173B2 (es)
KR (2) KR102213740B1 (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) EA035068B1 (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) MA51988A (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) PL3143025T3 (es)
PT (1) PT3143025T (es)
RS (1) RS59718B1 (es)
SG (1) SG11201609497TA (es)
SI (2) SI3663296T1 (es)
TW (1) TWI667239B (es)
UA (1) UA119670C2 (es)
WO (1) WO2015173181A1 (es)
ZA (1) ZA201607026B (es)

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TR201813877T4 (tr) 2012-02-10 2018-11-21 Hoffmann La Roche Spi̇nal müsküler atrofi̇ni̇n tedavi̇ edi̇lmesi̇ne yöneli̇k bi̇leşi̇kler.
WO2015024876A2 (en) 2013-08-19 2015-02-26 F. Hoffmann-La Roche Ag Screening method
US10882868B2 (en) * 2014-05-15 2021-01-05 Hoffmann-La Roche Inc. Compounds for treating spinal muscular atrophy
WO2016184832A1 (en) 2015-05-20 2016-11-24 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy
WO2016196386A1 (en) 2015-05-30 2016-12-08 Ptc Therapeutics, Inc. Methods for modulating rna splicing
JP2018533594A (ja) * 2015-11-12 2018-11-15 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 筋萎縮性側索硬化症を処置するための化合物
WO2017080967A1 (en) * 2015-11-12 2017-05-18 F. Hoffmann-La Roche Ag Compositions for treating spinal muscular atrophy
LT3386511T (lt) 2015-12-10 2021-08-25 Ptc Therapeutics, Inc. Hantingtono ligos gydymo būdai
EP3386978B1 (en) 2015-12-10 2021-01-27 H. Hoffnabb-La Roche Ag Bridged piperidine derivatives
EA201991309A1 (ru) * 2016-11-28 2019-11-29 Способы модуляции сплайсинга рнк
EP3634953B1 (en) 2017-06-05 2024-01-03 PTC Therapeutics, Inc. Compounds for treating huntington's disease
KR20200017476A (ko) 2017-06-14 2020-02-18 피티씨 테라퓨틱스, 인크. Rna 스플라이싱의 변경 방법
EP3645121A4 (en) 2017-06-28 2021-03-17 PTC Therapeutics, Inc. HUNTINGTON'S DISEASE TREATMENT METHODS
CN111182898B (zh) 2017-06-28 2024-04-16 Ptc医疗公司 用于治疗亨廷顿氏病的方法
SG11202002610TA (en) * 2017-09-22 2020-04-29 Hoffmann La Roche 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
AU2018344402A1 (en) * 2017-10-03 2020-04-09 F. Hoffmann-La Roche Ag New treatment of SMA
US20210009590A1 (en) 2018-03-27 2021-01-14 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
EP3814357B1 (en) 2018-06-27 2024-05-01 PTC Therapeutics, Inc. Heterocyclic and heteroaryl compounds for treating huntington's disease
EP3814360A1 (en) 2018-06-27 2021-05-05 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
WO2020079203A1 (en) 2018-10-19 2020-04-23 F. Hoffmann-La Roche Ag New forms of pyrido[1,2-a]pyrimidin-4-one derivatives, its formulation and its process of making
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
CA3210678A1 (en) 2021-03-18 2022-09-22 F. Hoffmann-La Roche Ag Novel process
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)

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Publication number Priority date Publication date Assignee Title
JP3345021B2 (ja) * 1996-08-06 2002-11-18 ファイザー インク． 置換ピリド―またはピリミド―含有６，６―または６，７―二環式誘導体
CA2700841A1 (en) 2007-09-27 2009-04-02 The United States Of America, As Represented By The Secretary, Departmen T Of Health And Human Services Isoindoline compounds for the treatment of spinal muscular atrophy and other uses
US8633019B2 (en) 2008-05-27 2014-01-21 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
KR20100012134A (ko) 2008-07-28 2010-02-08 신호열 마우스
WO2010019236A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc Methods for treating spinal muscular atrophy
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
TR201813877T4 (tr) * 2012-02-10 2018-11-21 Hoffmann La Roche Spi̇nal müsküler atrofi̇ni̇n tedavi̇ edi̇lmesi̇ne yöneli̇k bi̇leşi̇kler.
AU2013289938A1 (en) 2012-07-13 2015-01-29 Indiana University Research & Technology Corporation Compounds for treatment of spinal muscular atrophy
WO2017080967A1 (en) 2015-11-12 2017-05-18 F. Hoffmann-La Roche Ag Compositions for treating spinal muscular atrophy

Also Published As

Publication number Publication date
EP3663296A1 (en) 2020-06-10
HRP20230637T1 (hr) 2023-09-29
WO2015173181A1 (en) 2015-11-19
SG11201609497TA (en) 2016-12-29
PH12016502081A1 (en) 2017-01-09
EA202090486A3 (ru) 2020-08-31
EA035068B1 (ru) 2020-04-23
IL270027B (en) 2020-08-31
IL248653B (en) 2019-11-28
PL3143025T3 (pl) 2020-03-31
HRP20192159T1 (hr) 2020-02-21
KR20210014219A (ko) 2021-02-08
UA119670C2 (uk) 2019-07-25
ES2949660T3 (es) 2023-10-02
US9969754B2 (en) 2018-05-15
CA2948561C (en) 2019-10-22
AU2015261046A1 (en) 2016-10-27
BR112016026205A8 (pt) 2021-07-20
MX2016014547A (es) 2017-02-23
HUS2100037I1 (hu) 2021-10-28
AU2015261046B2 (en) 2019-04-18
CR20160518A (es) 2017-02-21
SI3663296T1 (sl) 2023-08-31
EP3143025A1 (en) 2017-03-22
EA201692280A1 (ru) 2018-07-31
CA2948561A1 (en) 2015-11-19
TWI667239B (zh) 2019-08-01
FR21C1039I2 (fr) 2022-09-02
EP4241772A3 (en) 2023-11-15
MA39995B1 (fr) 2019-11-29
PE20170128A1 (es) 2017-03-16
MX371050B (es) 2020-01-14
SI3143025T1 (sl) 2020-01-31
DK3143025T3 (da) 2019-12-09
FR21C1039I1 (es) 2021-10-15
ES2761423T3 (es) 2020-05-19
LTPA2021010I1 (es) 2021-10-11
KR102213740B1 (ko) 2021-02-09
RS59718B1 (sr) 2020-01-31
NZ725008A (en) 2019-11-29
KR20170003687A (ko) 2017-01-09
JP6236173B2 (ja) 2017-11-22
EA202090486A2 (ru) 2020-06-30
IL248653A0 (en) 2017-01-31
AR100442A1 (es) 2016-10-05
NO2021035I1 (no) 2021-09-10
ZA201607026B (en) 2022-05-25
BR112016026205A2 (pt) 2017-08-15
NL301128I1 (es) 2021-09-22
HUE046491T2 (hu) 2020-03-30
AU2015261046C1 (en) 2019-07-25
PT3143025T (pt) 2019-12-03
PL3663296T3 (pl) 2023-08-07
TW201609738A (zh) 2016-03-16
JP2017515863A (ja) 2017-06-15
NL301128I2 (nl) 2021-10-25
MA51988A (fr) 2021-01-20
CN106459092A (zh) 2017-02-22
EP4241772A2 (en) 2023-09-13
CN106459092B (zh) 2019-10-15
MY174284A (en) 2020-04-01
EP3143025B1 (en) 2019-10-09
EP3663296B1 (en) 2023-05-17
US20170197990A1 (en) 2017-07-13
KR102256013B1 (ko) 2021-05-26

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