16 May 2019 - - The US Food and Drug Administration has granted Breakthrough Therapy Designation for SEP-363856, a novel agent for the treatment of people with schizophrenia, US-based Sunovion Pharmaceuticals Inc. and PsychoGenics Inc. said.

Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or life-threatening conditions when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on one or more clinically significant endpoints.

SEP-363856 does not bind to dopamine 2 or serotonin 2A (5-HT2A) receptors, which are thought to mediate the effects of currently available antipsychotic medicines.

Although the exact mechanism of action is unknown, SEP-363856 is believed to activate TAAR1 (trace amine-associated receptor 1) in addition to 5-HT1A (serotonin 1A) receptors.

The FDA granted Breakthrough Therapy Designation for SEP-363856 based on pivotal, Phase 2 data from Study SEP361-201, which were presented by Sunovion at the 57th annual meeting of the American College of Neuropsychopharmacology in December 2018, as well as data from Study SEP361-202, a six-month, open-label, safety and tolerability extension study.

With this Breakthrough Therapy Designation SEP-363856 is eligible for intensive guidance from the FDA on the drug development program and priority review.

SEP 361-201, a randomised, placebo-controlled, double-blind, registration study, met its primary endpoint, demonstrating that hospitalised patients with acute exacerbation (worsening) of schizophrenia treated with SEP-363856 showed statistically significant and clinically meaningful improvement in the Positive and Negative Syndrome Scale (PANSS) total score compared to placebo after four weeks of treatment (-17.2 vs. -9.7, respectively; p=0.001).

Patients treated with SEP-363856 also showed improvement in the overall severity of illness as assessed by the Clinical Global Impression Scale - Severity (CGI-S) (p