Benzinga | Nov 11, 2020 07:09AM EST

BioXcel Therapeutics Announces Completion Of Pre-NDA Meeting With FDA For BXCL501 For Acute Treatment Of Agitation In Patients With Schizophrenia And Bipolar Disorders

Initiated rolling submission of New Drug Application ("NDA") with U.S. Food and Drug Administration ("FDA")

On track to submit complete NDA in Q1 2021

NEW HAVEN, Conn., Nov. 11, 2020 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. ("BTI" or the "Company") (NASDAQ:BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence to identify improved therapies in neuroscience and immuno-oncology, today announced that it has completed its pre-NDA meeting with the FDA for BXCL501 for the acute treatment of agitation in patients with schizophrenia and bipolar disorders. BXCL501, which received Fast Track designation from the FDA, is the Company's novel, proprietary, orally dissolving, sublingual thin film formulation of dexmedetomidine ("Dex"). The purpose of the meeting was to discuss the FDA's expectations regarding the content and formatting of the NDA submission to ensure that all requirements for a complete application are met.

Prior to the meeting, BTI submitted a pre-NDA briefing document to the FDA that outlined the Company's preliminary data package, including clinical safety and efficacy, non-clinical results, Chemistry, Manufacturing and Controls ("CMC"), and other regulatory elements. After receiving the FDA's feedback, the Company believes its regulatory data package will be sufficient for submission of its NDA. The FDA also agreed to a rolling review of the NDA, allowing the Company to submit completed sections of the application early. BTI has already submitted part of the NDA to the FDA, which included non-clinical and product brand name content, with plans to submit the complete application in the first quarter of 2021.

"Based on the outcome of the pre-NDA meeting with the FDA, we are on track to submit a complete application to the FDA in the first quarter of 2021," commented Vimal Mehta, Chief Executive Officer of BTI. "As an oral, non-invasive product candidate, we believe BXCL501 is highly differentiated from the current standard of care and, if approved, will provide health professionals with the potential to target an underlying cause of agitation without the side effects seen with other medications. This NDA submission, once completed, will move BTI one step closer to bringing BXCL501 to the millions of patients suffering from agitation."

BTI previously announced positive topline results from its Phase 3 randomized, double-blinded, placebo-controlled, parallel group, adaptive studies (SERENITY I & SERENITY II) investigating the efficacy and safety of BXCL501 as an acute treatment for agitation in patients with schizophrenia and bipolar disorders. In both studies, BXCL501 consistently showed statistical superiority over placebo for the primary, secondary, and all exploratory endpoints. Both patient populations responded quickly to BXCL501, demonstrating statistically significant and clinically meaningful improvement in agitation beginning as early as 20 minutes in patients with bipolar disorders at both dose levels (120 mcg and 180 mcg), and as early as 20 minutes in patients with schizophrenia for the 180 mcg dose level. In addition, the duration of response lasted for at least four hours after treatment.

BXCL501 was well tolerated in both SERENITY trials. Overall, the most commonly reported adverse events from both trials were somnolence, dry mouth, and dizziness. All adverse events were characterized as mild or moderate in severity and none required further intervention or monitoring.