Ptc Therapeutics, Inc.

Novartis Ag

Other

Samit Hirawat

Langdon Miller

Liangxian Cao

Marla L Weetall

Thomas W Davis

Cristian Massacesi

Harry H Miao

Gary Mitchell Karp

Young-Choon Moon

Neil Gregory Almstead

Guangming Chen

Ellen Welch

Seongwoo Hwang

James Jan Takasugi

Richard Gerald Wilde

Peter Seongwoo Hwang

Hongyu Ren

Charles Romfo

Emmanuelle Di Tomaso

Paul D Kennedy

Paul Kennedy

Jemes J Takasugi

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.

Compositions for an orally active 1,2,4-oxadiazole for the treatment of disease

Methods for dosing an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy

The present invention relates to a dosage regimen for a phosphatidylinositol 3-kinase (PI3K) inhibitor compound of formula (I) or a pharmaceutically acceptable salt thereof. More specifically, the present invention relates to a dosage regimen for the treatment of patients suffering from a proliferative disease, such as, for example, cancer, with a phosphatidylinositol 3-kinase (PI3K) inhibitor compound of formula (I) or a pharmaceutically acceptable salt thereof.

Dosage regimen for a PI-3 kinase inhibitor

Methods for dosing an orally active 1,2,4-oxadiazole for nonsense mutation supression therapy

A pharmaceutical combination comprising: (a) a phosphatidylinositol-3-kinase inhibitor selected from 5-(2,6-di-morpholin-4-yl-pyrimidin-4-yl)-4-trifluoromethyl-pyridin-2-ylamine, (S)-Pyrrolidine-1,2-dicarboxylic acid 2-amide 1-({4-methyl-5-[2-(2,2,2-trifluoro-1,1-dimethyl-ethyl)-pyridin-4-yl]-thiazol-2-yl}-amide) or any pharmaceutically acceptable salt thereof and (b) a gonadorelin agonist and, optionally, (c) an antiestrogen agent, particularly for use in the treatment or prevention of a cancer; uses of such a combination in the preparation of a medicament for the treatment or prevention of a cancer; pharmaceutical compositions of the combination of said therapeutic agents and methods of treating a cancer in a subject comprising administering to said subject a therapeutically effective amount of such a combination.

Pharmaceutical combinations

A pharmaceutical combination comprising (a) a phosphatidylinositol-3-kinase (PI3K) inhibitor compound of formula (I), as defined herein, or a pharmaceutically acceptable salt thereof and (b) paclitaxel or a pharmaceutically acceptable salt thereof for simultaneous, separate or sequential use, for the treatment of a cancer of the head and neck; a pharmaceutical composition comprising said combination; the use of said combination for the preparation of a medicament for the treatment of a cancer of the head and neck; a method of treating or preventing a cancer of the head and neck comprising administering a jointly therapeutically effective amount of such a combination to a subject in need thereof; and a commercial package thereof.

Combination of a PI3 kinase inhibitor with pacitaxel for use in the treatment or prevention of a cancer of the head and neck

Methods for dosing an orally active 1,2,4-Oxadiazole for nonsense mutation suppression therapy

The present invention relates to functional proteins encoded by nucleic acid sequences comprising a nonsense mutation. The present invention also relates to methods for the production of functional proteins encoded by nucleic acid sequences comprising a nonsense mutation and the use of such proteins for prevention, management and/or treatment of diseases associated with a nonsense mutation(s) in a gene.

Methods for the production of functional protein from DNA having a nonsense mutation and the treatment of disorders associated therewith

Methods for dosing an orally active 1,2,4-oxadiazole

Growing community of inventors

Chatham, NJ, United States of America

Samit Hirawat