BioXcel Therapeutics, Inc. (NASDAQ: BTAI) today announced full enrollment of its pivotal Phase 3 SERENITY trials evaluating BXCL501 for the acute treatment of agitation in patients with schizophrenia and bipolar disorder. The Company expects to report topline data from both trials in July of 2020.

“Completing patient enrollment in our two Phase 3 studies marks a significant milestone for BTI, and lays the foundation for future growth as we look to pursue clinical trials for several additional indications for BXCL501,” commented Vimal Mehta, Ph.D., Chief Executive Officer of BTAI. “The BTI team has remained focused, with our clinical programs on track despite the ongoing COVID-19 pandemic. We’re looking forward to reporting results from the two SERENITY registration trials, which we expect will be in July, to be followed by a potential New Drug Application submission in early 2021.”

The SERENITY studies are randomized, double-blinded, placebo-controlled, adaptive trials of up to 750 patients, 18 to 75 years of age. SERENITY I is evaluating patients with agitation associated with schizophrenia, with each of three arms receiving BXCL501 at 120 micrograms, 180 micrograms or placebo, respectively. SERENITY II is evaluating patients with agitation associated with bipolar disorder, also in three arms, receiving BXCL501 at 120 micrograms, 180 micrograms or placebo, respectively. The primary endpoint of the trials is reducing acute agitation measured by the Positive and Negative Syndrome Scale, examining the Excited Component (“PEC”) change from baseline compared to placebo. A key secondary endpoint includes determining the earliest time where an effect on agitation is apparent as measured by the change from baseline in PEC total score.

About Agitation in Neuropsychology

Agitation is a common and difficult to manage symptom associated with a number of psychiatric conditions, including schizophrenia and bipolar disorder. It is estimated that approximately 19 million people are at risk of agitation, and 8.3 million in the U.S. suffer from agitation each year, costing approximately $40 billion annually in treatment related expenses. Early identification and prompt intervention to relieve agitation are essential to avoid symptomatic escalation and emergence of aggression. Recent consensus guidelines emphasize the need for non-coercive management strategies to protect the therapeutic alliance between patients and their healthcare providers—an alliance that is critical for the effective management of chronic psychiatric conditions. A non-invasive therapy that causes rapid symptom relief and de-escalates agitation may be necessary to avoid the costly and traumatic use of coercive techniques, like physical restraint and seclusion, which require admission and prolonged hospitalization.

About BXCL501

BXCL501 is an investigational proprietary sublingual thin film of dexmedetomidine, a selective alpha-2a receptor agonist for the treatment of acute agitation. BTI believes that BXCL501 directly targets a causal agitation mechanism, and the Company has observed anti-agitation effects in multiple clinical studies across multiple neuropsychiatric indications. BXCL501 has been granted Fast Track Designation by the U.S. Food and Drug Administration for the acute treatment of agitation.

A Phase 1b safety and efficacy study of BXCL501 in patients with schizophrenia yielded positive dose-response data. BXCL501 is being evaluated in the SERENITY program, consisting of two Phase 3 studies for the acute treatment of agitation in patients with schizophrenia (SERENITY I) and bipolar disorder (SERENITY II). BXCL501 is also being evaluated in a Phase 1b/2 trial (TRANQUILITY) for the treatment of agitation associated with dementia, and the Company is preparing to initiate a Phase 1b/2 study (RELEASE) of BXCL501 for the treatment of opioid withdrawal symptoms.