公开(公告)号：US20240132509A1

公开(公告)日：2024-04-25

申请号：US18507660

申请日：2023-11-13

Applicant: PTC THERAPEUTICS, INC.

Inventor： Nadiya Sydorenko ,  Md Rauful Alam ,  Michael A. Arnold ,  Suresh Babu ,  Anuradha Bhattacharyya ,  Guangming Chen ,  Aleksey I. Gerasyuto ,  Gary Mitchell Karp ,  Andrew J. Kassick ,  Anthony R. Mazzotti ,  Young-Choon Moon ,  Jana Narasimhan ,  Jigar Patel ,  Anthony Turpoff ,  Matthew G. Woll ,  Wuming Yan ,  Nanjing Zhang

IPC: C07D487/04 ,  A61P25/14 ,  A61P25/28 ,  C07D491/048 ,  C07D495/04 ,  C07D519/00

CPC classification number: C07D487/04 ,  A61P25/14 ,  A61P25/28 ,  C07D491/048 ,  C07D495/04 ,  C07D519/00

Abstract: Disclosed are quinoline and quinazoline compounds which modulate the activity of the gated ion channels. Compounds that modulate these gated ion channels are useful in the treatment of diseases and disorders related to pain, inflammation, the neurological system, the gastrointestinal system and genitourinary system. Preferred compounds include quinoline or quinazoline derivatives substituted at the 4-position via N(H), C(O) or O moieties.

公开(公告)号：US11180483B2

公开(公告)日：2021-11-23

申请号：US16583442

申请日：2019-09-26

Applicant: PTC Therapeutics, Inc.

Inventor： Chang-Sun Lee ,  Ramil Baiazitov ,  Liangxian Cao ,  Thomas W. Davis ,  Wu Du ,  Ronggang Liu ,  Young-Choon Moon ,  Steven D. Paget ,  Hongyu Ren ,  Nadiya Sydorenko ,  Richard Gerald Wilde

IPC: A61K31/506 ,  A61K45/06 ,  A61N5/10 ,  C07D403/04 ,  C07D471/04 ,  C07D487/04 ,  C07D403/12

Abstract: Amine substituted reverse pyrimidine compounds and forms thereof that inhibit the function and reduce the level of B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.

公开(公告)号：US20210330647A1

公开(公告)日：2021-10-28

申请号：US17183342

申请日：2021-02-24

Applicant: PTC Therapeutics, Inc.

Inventor： Neil G. Almstead ,  Peter Seongwoo Hwang ,  Young-Choon Moon

IPC: A61K31/4245 ,  C07D271/06

Abstract: The present invention relates to crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid, pharmaceutical compositions and dosage forms comprising the crystalline forms, methods of making the crystalline forms and methods for their use for the treatment, prevention or management of diseases ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay.

公开(公告)号：US20210009590A1

公开(公告)日：2021-01-14

申请号：US17040751

申请日：2019-03-26

Applicant: PTC THERAPEUTICS, INC.

Inventor： Nadiya Sydorenko ,  Suresh Babu ,  Anuradha Bhattacharyya ,  Young-Choon Moon ,  Jana Narasimhan ,  Jigar S. Patel

IPC: C07D471/08 ,  C07D401/14 ,  C07D401/12 ,  A61P25/28

Abstract: The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted monocyclic heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.

公开(公告)号：US10584115B2

公开(公告)日：2020-03-10

申请号：US15037514

申请日：2013-11-21

Applicant: PTC Therapeutics, Inc.

Inventor： Ramil Baiazitov ,  Soongyu Choi ,  Wu Du ,  Seongwoo Hwang ,  Chang-Sun Lee ,  Ronggang Liu ,  Young-Choon Moon ,  Steven D. Paget ,  Hongyu Ren ,  Nadiya Sydorenko ,  Richard Gerald Wilde

IPC: C07D403/04 ,  C07D401/14 ,  C07F5/04 ,  A61K31/69 ,  C07D471/04 ,  A61K31/519 ,  C07D405/14 ,  C07D417/14 ,  C07D401/04 ,  A61K31/4985 ,  C07D413/04 ,  C07D403/14 ,  A61K45/06 ,  C07D487/04 ,  A61K31/497 ,  C07D513/04

Abstract: Amine substituted pyridine and pyrazine compounds and forms thereof that inhibit the function and reduce the level of B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.

公开(公告)号：US09975878B2

公开(公告)日：2018-05-22

申请号：US15038039

申请日：2013-11-21

Applicant: PTC Therapeutics, Inc.

Inventor： Hiroo Koyama ,  Ramil Baiazitov ,  Wu Du ,  Chang-sun Lee ,  Young-Choon Moon ,  Steven D. Paget ,  Hongyu Ren ,  Nadiya Sydorenko

IPC: C07D403/04 ,  A61K31/53 ,  A61K31/5377 ,  C07D413/14 ,  C07D401/14 ,  C07D405/14 ,  A61K45/06 ,  C07D471/04 ,  C07D403/14

CPC classification number: C07D403/04 ,  A61K31/53 ,  A61K31/5377 ,  A61K45/06 ,  C07D401/14 ,  C07D403/14 ,  C07D405/14 ,  C07D413/14 ,  C07D471/04

Abstract: Amine substituted triazine compounds and forms thereof that inhibit the function and reduce the level of B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.

公开(公告)号：US09861617B2

公开(公告)日：2018-01-09

申请号：US14940345

申请日：2015-11-13

Applicant: PTC Therapeutics Inc.

Inventor： Gary Mitchell Karp ,  Seongwoo Hwang ,  Guangming Chen ,  Neil Gregory Almstead ,  Young-Choon Moon

IPC: A61K31/4245 ,  A61K31/4439 ,  A61K31/454 ,  A61K31/4545 ,  A61K31/5377 ,  A61K31/655 ,  C07D271/06 ,  C07D271/12 ,  C07D285/14 ,  C07D413/02 ,  C07D413/04 ,  C07D413/10 ,  A61K45/06 ,  A61K9/00 ,  A61K9/12 ,  A61K9/48

CPC classification number: A61K31/4245 ,  A61K9/0053 ,  A61K9/12 ,  A61K9/48 ,  A61K31/4439 ,  A61K31/454 ,  A61K31/4545 ,  A61K31/5377 ,  A61K31/655 ,  A61K45/06 ,  A61P27/02 ,  C07D271/06 ,  C07D271/12 ,  C07D285/14 ,  C07D413/02 ,  C07D413/04 ,  C07D413/10

Abstract: Novel 1,2,4-oxadiazole benzoic acid compounds, methods of using and pharmaceutical compositions comprising an 1,2,4-oxadiazole benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith.

公开(公告)号：US20160297798A1

公开(公告)日：2016-10-13

申请号：US15038039

申请日：2013-11-21

Applicant: PTC THERAPEUTICS, INC.

Inventor： Hiroo Koyama ,  Ramil Baiazitov ,  Wu Du ,  Chang-sun Lee ,  Young-Choon Moon ,  Steven D. Paget ,  Hongyu Ren ,  Nadiya Sydorenko

IPC: C07D403/04 ,  A61K31/53 ,  A61K31/5377 ,  C07D413/14 ,  C07D401/14 ,  C07D405/14 ,  A61K45/06 ,  C07D471/04

CPC classification number: C07D403/04 ,  A61K31/53 ,  A61K31/5377 ,  A61K45/06 ,  C07D401/14 ,  C07D403/14 ,  C07D405/14 ,  C07D413/14 ,  C07D471/04

Abstract: Amine substituted triazine compounds and forms thereof that inhibit the function and reduce the level of B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.

Abstract translation: 抑制B细胞特异性莫洛尼鼠白血病病毒整合位点1（Bmi-1）蛋白的功能和降低水平的胺取代三嗪化合物及其形式及其用于抑制Bmi-1功能并降低Bmi水平的方法 -1治疗由Bmi-1介导的癌症。

公开(公告)号：US20160280685A1

公开(公告)日：2016-09-29

申请号：US15037514

申请日：2013-11-21

Applicant: PTC THERAPEUTICS, INC

Inventor： Ramil Baiazitov ,  Soongyu Choi ,  Wu Du ,  Seongwoo Hwang ,  Chang-Sun Lee ,  Ronggang Liu ,  Young-Choon Moon ,  Steven D. Paget ,  Hongyu Ren ,  Nadiya Sydorenko ,  Richard Gerald Wilde

IPC: C07D403/04 ,  C07D401/14 ,  C07F5/04 ,  A61K31/69 ,  C07D471/04 ,  A61K31/519 ,  C07D405/14 ,  C07D417/14 ,  C07D401/04 ,  A61K31/4985 ,  C07D413/04 ,  C07D403/14 ,  A61K45/06 ,  C07D487/04 ,  A61K31/497

Abstract: Amine substituted pyridine and pyrazine compounds and forms thereof that inhibit the function and reduce the level of B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.

Abstract translation: 胺取代的吡啶和吡嗪化合物及其形式，其抑制B细胞特异性莫洛尼鼠白血病病毒整合位点1（Bmi-1）蛋白的功能和水平降低，以及它们用于抑制Bmi-1功能和降低水平的方法 的Bmi-1治疗由Bmi-1介导的癌症。

公开(公告)号：US20160081988A1

公开(公告)日：2016-03-24

申请号：US14940345

申请日：2015-11-13

Applicant: PTC Therapeutics Inc.

Inventor： Gary Mitchell Karp ,  Seongwoo Hwang ,  Guangming Chen ,  Neil Gregory Almstead ,  Young-Choon Moon

IPC: A61K31/4245 ,  A61K31/454 ,  A61K31/5377 ,  A61K9/12 ,  A61K31/655 ,  A61K9/48 ,  A61K9/00 ,  A61K31/4439 ,  A61K31/4545

CPC classification number: A61K31/4245 ,  A61K9/0053 ,  A61K9/12 ,  A61K9/48 ,  A61K31/4439 ,  A61K31/454 ,  A61K31/4545 ,  A61K31/5377 ,  A61K31/655 ,  A61K45/06 ,  A61P27/02 ,  C07D271/06 ,  C07D271/12 ,  C07D285/14 ,  C07D413/02 ,  C07D413/04 ,  C07D413/10

Abstract: Novel 1,2,4-oxadiazole benzoic acid compounds, methods of using and pharmaceutical compositions comprising an 1,2,4-oxadiazole benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith.