BioXcel Therapeutics, Inc. ("BioXcel" or the "Company") (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, today announced that the U.S. Food and Drug Administration (“FDA”) has accepted for filing the New Drug Application (“NDA”) for BXCL501, the Company’s proprietary, investigational, orally dissolving thin film formulation of dexmedetomidine, for the acute treatment of agitation associated with schizophrenia and bipolar disorders I and II. The FDA has assigned a Prescription Drug User Fee Act (“PDUFA”) target action date of January 5, 2022. At this time, the FDA is not planning to hold an advisory committee meeting to discuss the application.

“The filing of our NDA marks an important milestone toward our goal to provide a new treatment option for the millions of patients with schizophrenia and bipolar disorders struggling with acute agitation,” said Vimal Mehta, Chief Executive Officer of BioXcel. “We believe that BXCL501, if approved, would represent a significant improvement in the care and management of agitation in these patients, potentially easing the burden for physicians and allied caregivers. While the FDA reviews our application, we will continue to execute on our comprehensive commercial strategy to ensure we are well positioned for potentially bringing BXCL501 – which is designed to address an important unmet need – to both patients and health care providers across the U.S.”

The application is supported by data from two randomized, double-blinded, placebo-controlled, parallel group Phase 3 studies (SERENITY I & SERENITY II) of BXCL501 for the acute treatment of agitation associated with schizophrenia and bipolar disorders I and II, respectively. In both trials, BXCL501 was well-tolerated and met the primary and secondary endpoints at the 120 mcg and 180 mcg doses, demonstrating statistically significant, rapid and durable improvements from baseline versus placebo across multiple agitation scales.

About Schizophrenia and Bipolar Disorder Related Agitation

Agitation is a common and difficult to manage symptom associated with multiple neuropsychiatric conditions, including schizophrenia and bipolar disorders I and II. These two disease states alone have an estimated U.S. prevalence of approximately 9 million adults with more than 3 million experiencing agitation each year. On average, patients with these conditions experience more than a dozen episodes per year, the majority requiring pharmacologic treatment. Early identification and prompt intervention to relieve agitation are essential to avoid symptomatic escalation and the emergence of aggression. Expert consensus best-practice guidelines have recommended that agitation should be treated by a combination of behavioral calming techniques, verbal de-escalation, and medications that are voluntarily accepted by patients without coercion, with the pharmacologic goal of “calming without excessive sedation.” A non-invasive therapy that causes rapid and sustained symptom relief may be helpful to avoid the costly and traumatic use of coercive techniques, like physical restraint and seclusion, which may result in admission and prolonged hospitalization.

About BXCL501

BXCL501 is an investigational, proprietary, orally dissolving thin film formulation of dexmedetomidine, a selective alpha-2a receptor agonist for the treatment of agitation and opioid withdrawal symptoms. BioXcel believes that BXCL501 potentially targets a causal agitation mechanism, and the Company has observed anti-agitation results in multiple clinical studies across several neuropsychiatric disorders, including schizophrenia related agitation (SERENITY I), bipolar disorder related agitation (SERENITY II) and dementia related agitation (TRANQUILITY). BXCL501 has been granted Breakthrough Therapy designation for the acute treatment of agitation associated with dementia and Fast Track designation for the acute treatment of agitation associated with schizophrenia, bipolar disorders and dementia. The Company recently received acceptance of its New Drug Application for BXCL501 for the acute treatment of agitation associated with schizophrenia and bipolar disorders. The safety and efficacy of BXCL501 has not been established.