Sage Therapeutics, Inc.

Praxis Precision Medicines, Inc.

Vertex Pharmaceuticals, Inc.

Sage Therpeutics, Inc.

Gabriel Martinez Botella

Francesco Gerald Salituro

Albert Jean Robichaud

Boyd Lynn Harrison

Andrew Mark Griffin

Brian Edward Marron

Richard Thomas Beresis

Kiran Reddy

Daniel La

Francois Maltais

Michael Robin Hale

Qing Tang

Judith Straub

Magnus Ronn

Paul A Laskar

Nelson B Olivier

Jon H Come

David Messersmith

Robert Jason Herr

Maria Jesus Blanco-Pillado

Valerie Marone

Robert Borbo Kargbo

Carlos Loya

Albert Charles Pierce

Mark Willem Ledeboer

Kevin Michael Cottrell

Huai Gao

John P Duffy

Arnaud Le Tiran

Alex Aronov

John Gregory Reid

Ronald Lee Grey, Jr

Michelle L Stewart

Emilie Porter Huck

James J Doherty

Marion Wittmann

Michael C Quirk

The invention relates to salts of Compound 1, crystalline forms thereof, methods of their preparation, pharmaceutical compositions thereof and methods of their use.

Salts and crystal forms of GABA-positive allosteric modulator

Provided, in part, are compounds of Formula I. pharmaceutically acceptable salts thereof, and pharmaceutical compositions thereof, which are useful in the treatment of conditions associated with the activity of sodium channels. Methods of treating a disease or condition relating to aberrant function of a sodium ion channel including neurological disorders (e.g., Dravet syndrome, epilepsy), pain, and neuromuscular disorders are also provided herein.

Substituted pyridines as ion channel modulators

Compounds are provided according to Formula (A): and pharmaceutically acceptable salts thereof, and pharmaceutical compositions thereof; wherein R, R, n, R, R, R, and Rare as defined herein. Compounds of the present invention are contemplated useful for the prevention and treatment of a variety of conditions.

C7 substituted oxysterols and methods of use thereof

The present invention is directed to, in part, a compound having the structure: or a pharmaceutically acceptable salt thereof, useful for preventing and/or treating a disease or condition relating to aberrant function of a voltage-gated, sodium ion channel, for example, abnormal late/persistent sodium current. Methods of treating a disease or condition relating to aberrant function of a sodium ion channel including neurological disorders (e.g., Dravet syndrome, epilepsy), pain, and neuromuscular disorders are also provided herein.

3-(ethoxydifluoromethyl)-6-(5-fluoro-6-(2,2,2-trifluoroethoxy)pyridin-3-yl)-[1,2,4]triazolo[4,3-α]pyrazine as an ion channel modulator

Compounds are provided according to Formula (I): Formula (I) and pharmaceutically acceptable salts thereof, and pharmaceutical compositions thereof; wherein R, R, R, R, R, R, and n are as defined herein. Compounds of the present invention are contemplated useful for the prevention and treatment of a variety of conditions.

Oxysterols and methods of use thereof

Provided herein are 19-nor C3,3-disubstituted C21-pyrazolyl steroids of Formula (I): and pharmaceutically acceptable salts thereof; wherein, R, R, R, R, R, R, R, R, and Rare as defined herein. Such compounds are contemplated useful for the prevention and treatment of a variety of CNS-related conditions, for example, treatment of sleep disorders, mood disorders, schizophrenia spectrum disorders, convulsive disorders, disorders of memory and/or cognition, movement disorders, personality disorders, autism spectrum disorders, pain, traumatic brain injury, vascular diseases, substance abuse disorders and/or withdrawal syndromes, and tinnitus.

19-nor C3,3-disubstituted C21-n-pyrazolyl steroids and methods of use thereof

Described herein are neuroactive steroids of the Formula (I): or a pharmaceutically acceptable salt thereof; wherein, R, R, R, Rand A are as defined herein. Such compounds are envisioned, in certain embodiments, to behave as GABA modulators. The present invention also provides pharmaceutical compositions comprising a compound of the present invention and methods of use and treatment, e.g., such for inducing sedation and/or anesthesia.

Compositions and methods for treating CNS disorders

Compounds are provided according to Formula (A): and pharmaceutically acceptable salts thereof, and pharmaceutical compositions thereof; wherein R, R, R, R, R, R, and Rare as defined herein. Compounds of the present invention are contemplated useful for the prevention and treatment of a variety of conditions.

The present invention is directed to, in part, fused heteroaryl compounds of Formula (III-II):

Substituted [1,2,4]triazolo[4,3-a]pyrazines as ion channel modulators

Compounds are provided according to Formula (I) and pharmaceutically acceptable salts thereof, and pharmaceutical compositions thereof, wherein X, Y, R, R, R, R, R, R, R, R, R, R, and Rare as defined herein. Compounds of the present invention are contemplated useful for the prevention and treatment of a variety of conditions.

Described herein are neuroactive steroids of the Formula (I): or a pharmaceutically acceptable salt thereof; wherein -------, R, R, R, A and L are as defined herein. Such compounds are envisioned, in certain embodiments, to behave as GABA modulators. The present invention also provides pharmaceutical compositions comprising a compound of the present invention and methods of use and treatment, e.g., such for inducing sedation and/or anesthesia.

Neuroactive steroids, compositions, and uses thereof

Described herein are neuroactive steroids of the Formula (I): or a pharmaceutically acceptable salt thereof, wherein, A, R, R, R, R, R, R, R, and Rare as defined herein. Such compounds are envisioned, in certain embodiments, to behave as GABA modulators. The present invention also provides pharmaceutical compositions comprising a compound of the present invention and methods of use and treatment, e.g., such for inducing sedation and/or anesthesia.

Provided, in part, are compounds of Formula I: pharmaceutically acceptable salts thereof, and pharmaceutical compositions thereof, which are useful in the treatment of conditions associated with the activity of sodium channels. Methods of treating a disease or condition relating to aberrant function of a sodium ion channel including neurological disorders (e.g., Dravet syndrome, epilepsy), pain, and neuromuscular disorders are also provided herein.

Ion channel modulators

Described herein are neuroactive steroids of Formula (I), Formula (V), or Formula (IX) or a pharmaceutically acceptable salt thereof; wherein each instance of R, R, R, R, R, R, R, R, R, R, R, R, andare as defined herein. Such compounds are envisioned, in certain embodiments, to behave as GABA modulators. Also provided are pharmaceutical compositions comprising a compound described herein and methods of use and treatment, e.g., such as for inducing sedation and/or anesthesia.

C7, C12, and C16 substituted neuroactive steroids and their methods of use

Described herein are neuroactive steroids of the Formula (II): or a pharmaceutically acceptable salt thereof; wherein A, R, R, R, R, R, R, R, R, Randare as defined herein. Such compounds are envisioned, in certain embodiments, to behave as GABA modulators. The present invention also provides pharmaceutical compositions comprising a compound of the present invention and methods of use and treatment, e.g., such for inducing sedation and/or anesthesia.

Growing community of inventors

Wayland, MA, United States of America

Gabriel Martinez Botella