Benzinga | Jul 20, 2020 07:07AM EDT

BioXcel Therapeutics Announces Primary And Secondary Endpoints Met In Two Pivotal Phase 3 Trials Of BXCL501 For Acute Treatment Of Agitation In Patients With Schizophrenia And Bipolar Disorder

Highly statistically significant improvements in PEC score observed vs. placebo (p<0.0001) at two hours in the SERENITY trials for both doses tested



Statistically significant improvements in PEC score observed as early as 20 minutes after treatment

All exploratory endpoints demonstrated statistically significant and clinically meaningful reductions in agitation measures that were durable

BXCL501 was well tolerated with no serious adverse events

NDA submission to U.S. Food and Drug Administration planned for Q1 2021

Company to host conference call today at 8:30 a.m. ET

NEW HAVEN, Conn., July 20, 2020 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. ("BTI" or "Company") (NASDAQ:BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence to identify improved therapies in neuroscience and immuno-oncology, today announced that BXCL501, the Company's proprietary sublingual thin film of dexmedetomidine, met the primary and secondary endpoints of SERENITY I and SERENITY II, demonstrating a robust treatment effect in the trials. Results demonstrated that BXCL501 was well tolerated, with rapid and durable reductions in agitation.

In patients with schizophrenia (SERENITY I) and a second study of bipolar disorder (SERENITY II), highly statistically significant and clinically meaningful reductions in the Positive and Negative Syndrome Scale, Excitatory Component ("PEC") score at two hours, the primary endpoint, were reported for both high and low dose cohorts of BXCL501 compared to placebo (p<0.0001). Both studies also met the key secondary endpoint, demonstrating improvement in PEC scores beginning as early as 20 minutes in patients with bipolar disorder, at both dose levels, and as early as 20 minutes in patients with schizophrenia for the 180 mcg dose level. Exploratory efficacy endpoints confirmed the primary endpoint, with duration of response lasting at least four hours after treatment.

"These compelling Phase 3 results show that BXCL501, if approved, has the potential to become an important new treatment option for patients suffering from acute agitation," commented Vimal Mehta, Ph.D., Chief Executive Officer of BTI. "We are extremely pleased that rapid and robust reductions in agitation were demonstrated in both patient populations despite differing neuropsychiatric diagnoses. We believe these results suggest that BXCL501 may have potential to treat agitation across a wide range of conditions. As we initiate steps toward regulatory submissions in these first two indications, we are also rapidly advancing the investigation of BXCL501 in additional disorders with significant unmet medical need including dementia, hyperactive delirium and opioid withdrawal symptoms."