FDA lists Sunovion’s, PsychoGenics’s SEP-363856 as breakthrough therapy for the treatment of schizophrenia

— SEP-363856 offers an innovative approach to the treatment of
schizophrenia including the potential to be the first agent for the
treatment of schizophrenia that does not bind to dopamine 2 (D2)
receptors —

— Schizophrenia affects more than 2 million people in the United
States —

MARLBOROUGH, Mass. & PARAMUS, N.J.–(BUSINESS WIRE)–Sunovion
Pharmaceuticals Inc. (Sunovion) and PsychoGenics
Inc. (PsychoGenics), today announced that the U.S. Food and Drug
Administration (FDA) has granted Breakthrough Therapy Designation for
SEP-363856, a novel agent for the treatment of people with schizophrenia.

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.¹

“Schizophrenia is a major public health challenge associated with
persistent abnormalities in thinking, perception and behavior, as well
as impairments in quality of life and functional skills, that affects
approximately 2.4 million people in the U.S.,”² said Antony
Loebel, M.D., President and Chief Executive Officer at Sunovion.
“Breakthrough Therapy Designation underscores the potential of
SEP-363856 as a novel treatment for patients with schizophrenia, for
whom few major advances in treatment have occurred since the advent of
antipsychotic pharmacotherapy in the 1950s. Investigational studies to
further evaluate the clinical benefit of SEP-363856 are in progress, and
we look forward to working closely with the FDA on this important
potential new therapy.”

SEP-363856 does not bind to dopamine 2 (D2) 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 57^th Annual Meeting of the American College
of Neuropsychopharmacology (ACNP) 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.¹

About SEP 361-201

SEP 361-201, a randomized, placebo-controlled, double-blind,
registration study, met its primary endpoint, demonstrating that
hospitalized 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<0.001). In addition, improvement was found in all major PANSS
(positive, negative and general psychopathology) subscales (p<0.02).

SEP-363856 was found to be generally well tolerated with notable
similarities to placebo treatment in discontinuation rates; proportion
of patients experiencing extrapyramidal symptoms or akathisia
(restlessness); and change in metabolic parameters such as weight,
lipids, glucose and prolactin.

About SEP-363856

SEP-363856 is a psychotropic agent with a novel, non-D2 mechanism of
action, distinct from currently marketed antipsychotics. Sunovion
discovered SEP-363856 in collaboration with PsychoGenics based in part
on a mechanism-independent approach using the in vivo phenotypic
SmartCube^® platform and associated artificial intelligence
algorithms. SEP-363856 was optimized for antipsychotic activity by
Sunovion medicinal chemists based on quantitative structure-activity
relationship analysis, in collaboration with PsychoGenics. SEP-363856 is
jointly owned by Sunovion and PsychoGenics. Sunovion has exclusive
rights to develop and commercialize SEP-363856 globally.

SEP-363856 is being studied in a global development program for
schizophrenia as well as for Parkinson’s disease psychosis, with
additional indications under consideration. Clinical trial results to
date demonstrate a predictable pharmacokinetic (PK) profile suitable for
once daily use.