BioXcel Therapeutics announced that BXCL501, a proprietary sublingual thin film of dexmedetomidine, met the primary and secondary endpoints in the phase 3 SERENITY I and SERENITY II trials. Based on these results the company plans to file a New Drug Application with the U.S. FDA in Q1 of 2021.
The SERENITY studies were randomized, double-blinded, placebo-controlled parallel group adaptive trials in a total of 759 patients, 18 to 75 years of age. SERENITY I (n=381) enrolled patients with agitation associated with schizophrenia or schizoaffective disorder, with arms randomized to receive BXCL501 at 120 micrograms, or 180 micrograms or matching placebo, respectively. SERENITY II (n=378) enrolled patients with agitation associated with bipolar disorders, in three treatment arms randomized to receive BXCL501 at 120 micrograms, 180 micrograms or placebo, respectively. The primary endpoint of the trials was the reduction in acute agitation measured by the Positive and Negative Syndrome Scale – Excitatory Component (“PEC”) change from baseline compared to placebo.
Highly statistically significant and clinically meaningful reductions in the 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. The most common adverse effects were somnolence, dry mouth and dizziness.
BXCL501 is an investigational proprietary sublingual thin film of dexmedetomidine, a selective alpha-2a receptor agonist for the treatment of acute agitation. BXCL501 has been granted Fast Track Designation by the U.S. Food and Drug Administration for the acute treatment of agitation. BXCL501 is also being evaluated in a Phase 1b/2 trial (TRANQUILITY) for the treatment of agitation associated with dementia, and in a Phase 1b/2 study (RELEASE) for the treatment of opioid withdrawal symptoms.
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 22 million people are at risk of agitation, and 13 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. 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.