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SAGE Therapeutics: Rare Central Nervous System Disorders

May 14, 2015

SAGE Therapeutics, a Cambridge, Massachusetts-based biopharmaceutical company launched in 2010, is developing treatments for rare Central Nervous System (CNS) disorders – in particular for seizure medicines. SAGE Therapeutics’ proprietary chemistry platform targets GABAA and NMDA receptors, which are broadly accepted as impacting many psychiatric and neurological disorders. The Company’s lead candidate is SAGE-547, in clinical development for Super-Refractory Status Epilepticus (SRSE).

SAGE Therapeutics’ programs are aimed at treating different stages of the rare disease Status Epilepticus (SE). SE is a life-threatening form of epilepsy or seizures that occurs in approximately 150,000 US patients each year, with a mortality rate of 20%. Refractory SE, in which currently available treatment options are not effective, occurs in approximately 1/3 of SE patients. Per a recent Press Release:

“SRSE is a critical condition in which the brain is in a state of persistent seizure, where patients are placed in a medically induced coma in an attempt to stabilize them and where conventional and approved therapies fail to awaken the patients. Currently, there are no therapies specifically approved for SRSE”.

SAGE-547, an intravenous (IV) agent, is in clinical development as an adjunctive therapy for patients with SRSE. In April 2014, SAGE-547 receives a FDA Orphan Drug Designation (ODD) for SE and a Fast Track Designation for SE in July 2014.

On May 14th, SAGE Therapeutics announces 1st Q 2015 financial results and data from a completed Phase I/II SAGE-547 clinical trial for SRSE – an achievement of a 77% response rate. A Phase III clinical trial (STATUS) is expected to start by mid-2015. According to an online Yahoo Finance article,   SAGE Therapeutics’ Chief Executive Jeff Jonas says that if all goes well, the drug could be commercially available by 2018. Also per the article, the shares of the company have risen more than three times since the company went public in July 2014.

SAGE Therapeutics is also developing treatments for other rare diseases:

•   Dravet Syndrome

•   Rett Syndrome.

SAGE-547 FDA ODD Database Record

Generic Name: Allopregnanolone
Trade Name: n/a
Date Designated: 04-20-2014
Orphan Designation: Status Epilepticus
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: SAGE Therapeutics215 First StreetSuite 220 Cambridge, MA 02142



SAGE Therapeutics Pipeline.

Please Note: “HERO (Human Epilepsy Research Opportunities) Badge” courtesy of the HERO website.

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