公开(公告)号：WO2014209841A2

公开(公告)日：2014-12-31

申请号：PCT/US2014043577

申请日：2014-06-23

Applicant: HOFFMANN LA ROCHE ,  HOFFMANN LA ROCHE ,  PTC THERAPEUTICS INC

Inventor： DAKKA AMAL ,  GREEN LUKE ,  KARP GARY ,  NARASIMHAN JANA ,  NARYSHKIN NIKOLAI ,  PINARD EMMANUEL ,  QI HONGYAN ,  RATNI HASANE ,  RISHER NICOLE ,  WEETALL MARLA ,  WOLL MATTHEW

IPC: A61K31/4985

CPC classification number: C07D519/00 ,  C07D263/56 ,  C07D413/12 ,  C07D471/04 ,  C07D487/04 ,  C07D487/10

Abstract: The present invention provides compounds of formula (I) wherein A, B, X, Y, R1 and R2 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.

Abstract translation: 本发明提供其中A，B，X，Y，R 1和R 2如本文所述的式（I）化合物及其药学上可接受的盐。 此外，本发明涉及制备式（I）化合物，包含它们的药物组合物及其作为药物的用途。

公开(公告)号：WO2013119916A3

公开(公告)日：2013-10-24

申请号：PCT/US2013025292

申请日：2013-02-08

Applicant: PTC THERAPEUTICS INC ,  HOFFMANN LA ROCHE

Inventor： QI HONGYAN ,  CHOI SOONGYU ,  DAKKA AMAL ,  KARP GARY MITCHELL ,  NARASIMHAN JANA ,  NARYSHKIN NIKOLAI ,  TURPOFF ANTHONY A ,  WEETALL MARIA L ,  WELCH ELLEN ,  WOLL MATTHEW G ,  YANG TIANLE ,  ZHANG NANJING ,  ZHANG XIAOYAN ,  ZHAO XIN ,  GREEN LUKE ,  PINARD EMMANUEL ,  RATNI HASANE

IPC: A61K31/53 ,  A01N43/90 ,  A61K31/519 ,  C07D239/70

CPC classification number: C07D471/04 ,  A61K31/519 ,  C07D239/70 ,  C07D487/04 ,  C07D519/00

Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.

Abstract translation: 本文提供了化合物，其组合物，并用于治疗脊髓性肌肉萎缩。

公开(公告)号：WO2006037480A8

公开(公告)日：2006-05-18

申请号：PCT/EP2005010237

申请日：2005-09-22

Applicant: HOFFMANN LA ROCHE ,  DEHMLOW HENRIETTA ,  KUHN BERND ,  PANDAY NARENDRA ,  RATNI HASANE ,  WRIGHT MATTHEW BLAKE

Inventor： DEHMLOW HENRIETTA ,  KUHN BERND ,  PANDAY NARENDRA ,  RATNI HASANE ,  WRIGHT MATTHEW BLAKE

IPC: C07C69/92 ,  A61K31/085 ,  A61K31/192 ,  A61K31/216 ,  A61K31/235 ,  A61K31/421 ,  A61K31/423 ,  A61K31/428 ,  C07C43/23 ,  C07C59/68 ,  C07C65/24 ,  C07C69/734 ,  C07D261/20 ,  C07D263/32 ,  C07D275/04

CPC classification number: C07D275/04 ,  C07C43/23 ,  C07C59/68 ,  C07C65/24 ,  C07C69/734 ,  C07C69/92 ,  C07D249/06 ,  C07D261/08 ,  C07D261/20 ,  C07D263/32

Abstract: The invention is concerned with hexafluoroisopropanol substituted ether derivatives of formula (I), wherein R l to R 3 are as defined in the description and in the claims, as well as physiologically acceptable salts and esters thereof. These compounds bind to LXR alpha and LXR beta and can be used as medicaments.

Abstract translation: 本发明涉及式（I）的六氟异丙醇取代的醚衍生物，其中R 1至R 3如在说明书和权利要求书中所定义，以及生理学上 可接受的盐和酯。 这些化合物与LXRα和LXRβ结合，可用作药物。

公开(公告)号：WO2007014851A3

公开(公告)日：2007-05-31

申请号：PCT/EP2006064395

申请日：2006-07-19

Applicant: HOFFMANN LA ROCHE ,  BISSANTZ CATERINA ,  GRUNDSCHOBER CHRISTOPHE ,  RATNI HASANE ,  ROGERS-EVANS MARK ,  SCHNIDER PATRICK

Inventor： BISSANTZ CATERINA ,  GRUNDSCHOBER CHRISTOPHE ,  RATNI HASANE ,  ROGERS-EVANS MARK ,  SCHNIDER PATRICK

IPC: C07D401/06 ,  A61K31/454 ,  A61P9/12 ,  C07D209/22 ,  C07D209/42 ,  C07D401/12 ,  C07D403/06 ,  C07D403/12 ,  C07D405/12 ,  C07D405/14 ,  C07D417/12 ,  C07D495/04

CPC classification number: C07D209/42 ,  C07D209/22 ,  C07D401/06 ,  C07D401/12 ,  C07D403/06 ,  C07D403/12 ,  C07D405/12 ,  C07D405/14 ,  C07D417/12 ,  C07D495/04

Abstract: The present invention relates to indol-3-yl-carbonyl-piperidin and piperazin derivatives which act as V1a receptor antagonists and which are represented by Formula (I) wherein the residues R 1 to R 3 are as defined herein. The invention further relates to pharmaceutical compositions containing such compounds, their use in medicaments against dysmenorrhea, hypertension, chronic heart failure, inappropriate secretion of vasopressin, liver cirrhosis, nephrotic syndrome, obsessive compulsive disorder, anxious and depressive disorders, and methods of preparation thereof.

Abstract translation: 本发明涉及作为V1a受体拮抗剂起作用的吲哚-3-基 - 羰基 - 哌啶和哌嗪衍生物，其由式（I）表示，其中残基R 1至R 3 如本文所定义。 本发明进一步涉及含有这些化合物的药物组合物，它们在抗痛经，高血压，慢性心力衰竭，血管加压素分泌不当，肝硬化，肾病综合征，强迫症，焦虑和抑郁症的药物中的用途，以及它们的制备方法。

公开(公告)号：WO2008080844A9

公开(公告)日：2009-10-08

申请号：PCT/EP2007064183

申请日：2007-12-19

Applicant: HOFFMANN LA ROCHE ,  BISSANTZ CATERINA ,  GRUNDSCHOBER CHRISTOPHE ,  MASCIADRI RAFFAELLO ,  RATNI HASANE ,  ROGERS-EVANS MARK ,  SCHNIDER PATRICK

Inventor： BISSANTZ CATERINA ,  GRUNDSCHOBER CHRISTOPHE ,  MASCIADRI RAFFAELLO ,  RATNI HASANE ,  ROGERS-EVANS MARK ,  SCHNIDER PATRICK

IPC: C07D491/20 ,  A61K31/438 ,  A61P9/12 ,  A61P25/24

CPC classification number: C07D491/20

Abstract: The present invention is concerned with novel indol-3-yl-carbonyl-azaspiropiperidine derivatives as V1a receptor antagonists, their manufacture, pharmaceutical compositions containing them and their use as medicaments. The active compounds of the present invention are useful in the prevention and/or treatment of anxiety and depressive disorders and other diseases. In particular, the present invention is concerned with compounds of the general formula (I) wherein R1 to R6, U, V, W, X, Y and Z are as defined in the specification.

Abstract translation: 本发明涉及作为V1a受体拮抗剂的新型吲哚-3-基 - 羰基 - 氮杂螺哌啶衍生物，其制备方法，含有它们的药物组合物及其作为药物的用途。 本发明的活性化合物可用于预防和/或治疗焦虑和抑郁症等疾病。 特别地，本发明涉及通式（I）的化合物，其中R 1至R 6，U，V，W，X，Y和Z如说明书中所定义。

公开(公告)号：WO2008077810A3

公开(公告)日：2008-09-25

申请号：PCT/EP2007063879

申请日：2007-12-13

Applicant: HOFFMANN LA ROCHE ,  BISSANTZ CATERINA ,  GRUNDSCHOBER CHRISTOPHE ,  MASCIADRI RAFFAELLO ,  RATNI HASANE ,  ROGERS-EVANS MARK ,  SCHNIDER PATRICK

Inventor： BISSANTZ CATERINA ,  GRUNDSCHOBER CHRISTOPHE ,  MASCIADRI RAFFAELLO ,  RATNI HASANE ,  ROGERS-EVANS MARK ,  SCHNIDER PATRICK

IPC: C07D211/16 ,  A61K31/4355 ,  A61K31/437 ,  A61K31/438 ,  A61P9/00 ,  C07D471/10 ,  C07D491/10

CPC classification number: C07D491/107 ,  C07D211/16 ,  C07D221/20 ,  C07D401/06 ,  C07D405/06 ,  C07D471/10

Abstract: The present invention is concerned with novel spiro-piperidine derivatives as V1a receptor antagonists, their manufacture, pharmaceutical compositions containing them and their use as medicaments. The active compounds of the present invention are useful in the prevention and/or treatment of anxiety and depressive disorders and other diseases. The compounds of present invention are described with the general formula (I), wherein R 1 to R 5 , R 5' , R 7 to R 9 , R 7' , R 8' , X and Y are as defined in the specification.

Abstract translation: 本发明涉及作为V1a受体拮抗剂的新型螺 - 哌啶衍生物，它们的制造方法，含有它们的药物组合物以及它们作为药物的用途。 本发明的活性化合物可用于预防和/或治疗焦虑症和抑郁症以及其他疾病。 用通式（I）描述本发明的化合物，其中R 1至R 5，R 5，R 5，R 5，R 5，R 5， R 7，R 8，R 8，X和Y如说明书中所定义。

公开(公告)号：WO2005092856A8

公开(公告)日：2006-12-21

申请号：PCT/EP2005002869

申请日：2005-03-17

Applicant: HOFFMANN LA ROCHE ,  DEHMLOW HENRIETTA ,  KUHN BERND ,  MASCIADRI RAFFAELLO ,  PANDAY NARENDRA ,  RATNI HASANE ,  WRIGHT MATTHEW BLAKE

Inventor： DEHMLOW HENRIETTA ,  KUHN BERND ,  MASCIADRI RAFFAELLO ,  PANDAY NARENDRA ,  RATNI HASANE ,  WRIGHT MATTHEW BLAKE

IPC: A61K31/40 ,  C07D209/70 ,  C07D209/86 ,  C07D209/88 ,  C07D401/12 ,  C07D403/06 ,  C07D403/12 ,  C07D411/06 ,  C07D411/14 ,  C07D413/06 ,  C07D413/14 ,  C07D417/06 ,  C07D471/04

CPC classification number: C07D413/06 ,  C07D209/70 ,  C07D209/88 ,  C07D403/12 ,  C07D413/14 ,  C07D417/06

Abstract: The present invention relates to compounds of the formula (I) wherein R 1 , R 2 , R 3 , R 4 , R 5 , X 1 , X 2 , X 3 , X 4 , n, and k are defined in the description and claims, and pharmaceutically acceptable salts and/or pharmaceutically acceptable esters thereof. The compounds are useful for in the treatment and prophylaxis of diseases which are modulated by LXRa and/or LXRß agonists, including increased lipid and cholesterol levels, particularly low HDL-cholesterol, high LDL-cholesterol, atherosclerotic diseases, diabetes, particularly non-insulin dependent diabetes mellitus, metabolic syndrome, dyslipidemia, Alzheimer's disease, sepsis, inflammatory diseases such as colitis, pancreatitis, cholestasis/fibrosis of the liver, and diseases that have an inflammatory component such as Alzheimer's disease or impaired/improvable cognitive function.

Abstract translation: 本发明涉及式（I）的化合物，其中R 1，R 2，R 3，R 4， 其中，R 5，X 5，X 1，X 2，X 3，X 4， SUP，n和k在说明书和权利要求书中被定义，以及其药学上可接受的盐和/或其药学上可接受的酯。 该化合物可用于治疗和预防由LXRα和/或LXRβ激动剂调节的疾病，包括增加脂质和胆固醇水平，特别是低HDL-胆固醇，高LDL-胆固醇，动脉粥样硬化疾病，糖尿病，特别是非胰​​岛素 依赖性糖尿病，代谢综合征，血脂异常，阿尔茨海默氏病，败血症，炎性疾病如结肠炎，胰腺炎，胆汁淤积/肝纤维化，以及具有炎性成分如阿尔茨海默病和受损/可改善的认知功能的疾病。

公开(公告)号：EP2812004A4

公开(公告)日：2015-11-11

申请号：EP13747100

申请日：2013-02-08

Applicant: PTC THERAPEUTICS INC ,  HOFFMANN LA ROCHE

Inventor： QI HONGYAN ,  CHOI SOONGYU ,  DAKKA AMAL ,  KARP GARY MITCHELL ,  NARASIMHAN JANA ,  NARYSHKIN NIKOLAI ,  TURPOFF ANTHONY A ,  WEETALL MARLA L ,  WELCH ELLEN ,  WOLL MATTHEW G ,  YANG TIANLE ,  ZHANG NANJING ,  ZHANG XIAOYAN ,  ZHAO XIN ,  GREEN LUKE ,  PINARD EMMANUEL ,  RATNI HASANE

IPC: A61K31/53 ,  A61K31/4375 ,  A61K31/519 ,  A61P25/00 ,  C07D471/04 ,  C07D487/04 ,  C07D519/00

CPC classification number: C07D471/04 ,  A61K31/519 ,  C07D239/70 ,  C07D487/04 ,  C07D519/00

Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.

Abstract translation: 本文提供了化合物，其组合物，并用于治疗脊髓性肌肉萎缩。

公开(公告)号：EP3013345A4

公开(公告)日：2016-11-30

申请号：EP14816651

申请日：2014-06-23

Applicant: HOFFMANN LA ROCHE ,  PTC THERAPEUTICS INC

Inventor： DAKKA AMAL ,  GREEN LUKE ,  KARP GARY ,  NARASIMHAN JANA ,  NARYSHKIN NIKOLAI ,  PINARD EMMANUEL ,  QI HONGYAN ,  RATNI HASANE ,  RISHER NICOLE ,  WEETALL MARLA ,  WOLL MATTHEW

IPC: A61K31/497 ,  A61K31/4965 ,  C07D401/00

CPC classification number: C07D519/00 ,  C07D263/56 ,  C07D413/12 ,  C07D471/04 ,  C07D487/04 ,  C07D487/10

Abstract: The present invention provides compounds of formula (I) wherein A, B, X, Y, R1 and R2 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.

Abstract translation: 本发明提供其中A，B，X，Y，R 1和R 2如本文所述的式（I）化合物及其药学上可接受的盐。 此外，本发明涉及制备式（I）化合物，包含它们的药物组合物及其作为药物的用途。

公开(公告)号：EP2797592A4

公开(公告)日：2016-01-13

申请号：EP12862080

申请日：2012-12-28

Applicant: PTC THERAPEUTICS INC ,  HOFFMANN LA ROCHE

Inventor： WOLL MATTHEW G ,  CHEN GUANGMING ,  CHOI SOONGYU ,  DAKKA AMAL ,  HUANG SONG ,  KARP GARY MITCHELL ,  LEE CHANG-SUN ,  LI CHUNSHI ,  NARASIMHAN JANA ,  NARYSHKIN NIKOLAI ,  PAUSHKIN SERGEY ,  QI HONGYAN ,  TURPOFF ANTHONY A ,  WEETALL MARLA L ,  WELCH ELLEN ,  YANG TIANLE ,  ZHANG NANJING ,  ZHANG XIAOYAN ,  ZHAO XIN ,  PINARD EMMANUEL ,  RATNI HASANE

IPC: A61K31/353 ,  A61P25/00 ,  C07D231/12 ,  C07D311/16 ,  C07D311/18 ,  C07D405/04 ,  C07D405/12 ,  C07D405/14 ,  C07D413/04 ,  C07D413/14 ,  C07D417/04 ,  C07D417/14 ,  C07D471/04 ,  C07D487/04 ,  C07D491/04 ,  C07D495/04 ,  C07D513/04 ,  C07D519/00

CPC classification number: C07D487/04 ,  C07D231/12 ,  C07D311/16 ,  C07D311/18 ,  C07D405/04 ,  C07D405/12 ,  C07D405/14 ,  C07D413/04 ,  C07D413/14 ,  C07D417/04 ,  C07D417/14 ,  C07D471/04 ,  C07D491/04 ,  C07D495/04 ,  C07D513/04 ,  C07D519/00

Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. In a specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene. In another specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 into mRNA that is transcribed from the SMN1 gene. In yet another embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 and SMN2 into mRNA that is transcribed from the SMN1 and SMN2 genes, respectively.