Ptc Therapeutics, Inc.

Ramil Y Baiazitov

Young-Choon Moon

Nadiya Sydorenko

Steven David Paget

Hongyu Ren

Chang-Sun Lee

Wu Du

Richard Gerald Wilde

Ronggang Liu

Liangxian Cao

Thomas W Davis

Gary Mitchell Karp

Zhenrong Xu

Seongwoo Hwang

Suresh D Babu

Soongyu Choi

Nanjing Zhang

Jana Narasimhan

Anthony A Turpoff

Anuradha Bhattacharyya

Matthew G Woll

Jigar S Patel

Anthony R Mazzotti

Scott J Barraza

Lukiana Amedzo

Hiroo Koyama

Md Rauful Alam

Nathaniel T Kenton

Sudipta Pal

Nicholas W Mszar

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) or Formula (II), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.

Compounds for treating Huntington's disease

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.

Substituted reverse pyrimidine Bmi-1 inhibitors

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.

Substituted pyridine and pyrazine BMI-1 inhibitors

Methods of using and pharmaceutical compositions comprising (2R,3S,4R,5R,6S)-5-amino-6-(((1R,2S,3S,4R,6S)-4,6-diamino-3-(((2R,3R,4R,5R)-3,5-dihydroxy-5-methyl-4-(methylamino)tetrahydro-2H-pyran-2- yl)oxy)-2-hydroxycyclohexyl)oxy)-2-(hydroxymethyl)tetrahydro-2H-pyran-3,4-diol are disclosed, including methods of treating or preventing a nonsense mutation mediated disease associated with premature translation termination or a premature stop codon resulting from a germline or somatic nonsense mutation.

Use of an aminoglycoside for nonsense mutation suppression and the treatment of disease

Amine substituted 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.

Substituted pyrimidine Bmi-1 inhibitors

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.

Substituted triazine BMI-1 inhibitors

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East Brunswick, NJ, United States of America

Ramil Y Baiazitov