公开(公告)号：US20240336613A1

公开(公告)日：2024-10-10

申请号：US18293349

申请日：2022-07-29

Applicant: PTC THERAPEUTICS, INC.

Inventor： Suresh BABU ,  Rauful ALAM ,  Anuradha BHATTACHARYYA ,  Guangming CHEN ,  Matthew S. EASTWOOD ,  Seyedmorteza HOSSEYNI ,  Yao JIANG ,  Gary Mitchell KARP ,  Young-Choon MOON ,  Jana NARASIMHAN ,  Hongyu REN ,  Nadiya SYDORENKO ,  Matthew G. WOLL ,  Nanjing ZHANG

IPC: C07D487/04 ,  A61K31/5025

CPC classification number: C07D487/04 ,  A61K31/5025

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 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：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：US11938101B2

公开(公告)日：2024-03-26

申请号：US17692133

申请日：2022-03-10

Applicant: PTC Therapeutics, Inc.

Inventor： Paul Mollard ,  Peter Giannousis ,  Shazad Suchit ,  Mahmoud Mirmehrabi ,  Christopher R. Cornell ,  Kieron E. Wesson

IPC: C07C235/78 ,  A61K31/122 ,  A61K31/164 ,  C07B57/00 ,  C07C215/08 ,  C07C215/28 ,  C07C215/30 ,  C07C231/12 ,  C07D215/28

CPC classification number: A61K31/122 ,  A61K31/164 ,  C07B57/00 ,  C07C215/08 ,  C07C215/28 ,  C07C215/30 ,  C07C231/12 ,  C07C235/78 ,  C07B2200/13 ,  C07C2601/16 ,  C07C231/12 ,  C07C235/78

Abstract: Disclosed herein are polymorphic and amorphous forms of anhydrate, hydrate, and solvates of (R)-2-hydroxy-2-methyl-4-(2,4,5-trimethyl-3,6-dioxocyclohexa-1,4-dienyl)butanamide and methods of using such compositions for treating or suppressing oxidative stress disorders, including mitochondrial disorders, impaired energy processing disorders, neurodegenerative diseases and diseases of aging. Further disclosed are methods of making such polymorphic and amorphous forms.

公开(公告)号：US20230339835A1

公开(公告)日：2023-10-26

申请号：US18347068

申请日：2023-07-05

Applicant: PTC THERAPEUTICS, INC.

Inventor： Andrew W. Hinman ,  Charles R. Holst ,  Angela Minnella ,  Paul Mollard ,  Sean Pintchovski ,  Jeffrey K. Trimmer ,  Eric Torrey

IPC: C07C50/04 ,  A61P25/16 ,  C07C46/10

CPC classification number: C07C50/04 ,  A61P25/16 ,  C07C46/10 ,  C07B2200/13

Abstract: Disclosed herein are methods of treating or suppressing a disorder selected from the group consisting of α-synucleinopathies, tauopathies, ALS, traumatic brain injury, and ischemic-reperfusion related injuries.ury, comprising administering to a subject in need thereof a therapeutically effective amount of a compound of the formula: or the hydroquinone form thereof; or a solvate or hydrate thereof.

公开(公告)号：US20230218587A1

公开(公告)日：2023-07-13

申请号：US17963838

申请日：2022-10-11

Applicant: PTC Therapeutics, Inc.

Inventor： Samit Hirwat ,  Langdon Miller

IPC: A61K31/4245 ,  A61K9/14 ,  A61K9/00

CPC classification number: A61K31/4245 ,  A61K9/145 ,  A61K9/0053

Abstract: The present invention relates to specific doses of and dosing regimens for using a 1,2,4-oxadiazole benzoic acid compound in treating or preventing diseases associated with nonsense mutations. In particular, the invention relates to specific doses and dosing regimens for the use of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid in mammals having diseases associated with nonsense mutations.

公开(公告)号：US11685746B2

公开(公告)日：2023-06-27

申请号：US17254848

申请日：2019-06-25

Applicant: PTC THERAPEUTICS, INC.

Inventor： Guangming Chen ,  Anuradha Bhattacharyya ,  Yao Jiang ,  Gary Mitchell Karp ,  Jana Narasimhan ,  Anthony Turpoff ,  Nanjing Zhang

IPC: C07D513/04 ,  C07D519/00

CPC classification number: C07D513/04 ,  C07D519/00

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 bicyclic heteroaryl compounds of Formula (I), Formula (II), Formula (III), or Formula (IV), 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：US20230172915A1

公开(公告)日：2023-06-08

申请号：US17923825

申请日：2021-05-07

Applicant: PTC THERAPEUTICS, INC.

Inventor： Nikolai A. NARYSHKIN ,  Jason GRACI ,  John BAIRD ,  Joseph COLACINO ,  Marla L. WEETALL ,  Liangxian CAO

IPC: A61K31/437 ,  A61K31/675 ,  A61P31/14

CPC classification number: A61K31/437 ,  A61K31/675 ,  A61P31/14

Abstract: One aspect described herein includes a method for treating coronavirus 2019 disease (COVID-19) caused by severe respiratory syndrome coronavirus 2 (SARS-CoV-2) by administering an effective amount of a DHODH inhibitor. Another aspect described herein includes a method of treating COVD-19 by administering 4-chlorophenyl (S)-6-chloro-1-(4-methoxyphenyl)-1,3,4,9-tetrahydro-2H-pyrido[3,4-b]indole-2-carboxylate (PTC299), or a pharmaceutically acceptable salt thereof.

公开(公告)号：US11638706B2

公开(公告)日：2023-05-02

申请号：US17093194

申请日：2020-11-09

Applicant: PTC Therapeutics, Inc.

Inventor： Suresh Babu ,  Anuradha Bhattacharyya ,  Seongwoo Hwang ,  Minakshi Jani ,  Young-choon Moon ,  Nadiya Sydorenko

IPC: C07D417/14 ,  A61K31/4468 ,  A61K31/506 ,  A61K31/433 ,  C07D403/14 ,  C07D405/14 ,  C07D401/12 ,  C07D417/04 ,  C07D487/04 ,  C07D401/04 ,  C07D409/14 ,  C07D487/10 ,  C07D237/20 ,  C07D471/10 ,  C07D413/14 ,  C07D487/08 ,  C07D405/12 ,  C07D403/10 ,  C07D471/08 ,  C07D403/12 ,  C07D417/10 ,  C07D401/14 ,  C07D417/12 ,  A61K31/50 ,  A61K31/415 ,  A61P25/28 ,  A61K31/4439 ,  A61K31/4709 ,  A61K31/501 ,  A61K31/517 ,  A61K31/5377 ,  A61K31/5513 ,  G01N33/68

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：US20230057568A1

公开(公告)日：2023-02-23

申请号：US17783941

申请日：2020-12-07

Applicant: PTC THERAPEUTICS, INC.

Inventor： Nanjing ZHANG ,  Michael A. ARNOLD ,  Amal DAKKA ,  Gary Mitchell KARP ,  Tom Tuan LUONG ,  Christie MORRILL ,  Jana NARASIMHAN ,  Nikolai A. NARYSHKIN ,  Anthony TURPOFF ,  Jiashi WANG ,  Xiaoyan ZHANG

IPC: C07D495/04

Abstract: The present description relates to compounds useful for improving pre-mRNA splicing in a cell. In particular, another aspect of the present description relates to substituted thieno [3,2-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof and methods of use for treating or ameliorating familial dysautonomia.

公开(公告)号：US11560364B2

公开(公告)日：2023-01-24

申请号：US17511432

申请日：2021-10-26

Applicant: PTC Therapeutics, Inc.

Inventor： Peter Giannousis ,  Paul Mollard ,  Noah M. Benjamin ,  Jeffrey D. Butler ,  Olivier Dapremont ,  James B. Falabella

IPC: C07D311/72

Abstract: Provided herein are methods for making an alpha-tocotrienol enriched tocol mixture, such as from a plant or plant-derived material. Also provided herein are simulated moving bed purification methods for tocotrienol compounds such as alpha tocotrienol.