TRANQUILITY In-Care pivotal phase III trial plan with BXCL 501 for agitation associated with Alzheimer’s dementia

The Company’s plan to conduct this trial is based on feedback received from the FDA, following the recent receipt of minutes from the Type B/Breakthrough Therapy designation meeting held with the agency on February 20, 2024.

“The design of our upcoming TRANQUILITY In-Care trial largely mirrors TRANQUILITY II, which demonstrated positive efficacy and safety results with a 60 mcg dose of BXCL 501,” said Vincent O’Neill, M.D., Chief of Product Development and Medical Officer of BioXcel Therapeutics. “We have completed two recent meetings with the FDA on our TRANQUILITY program, and are pleased to have obtained clarity on the next steps for our AAD development path. This represents a major milestone since there is no U.S. regulatory precedent for episodic treatment of AAD. We are now intensely focused on advancing this important program.”

TRANQUILITY In-Care Pivotal Phase III Trial Design Summary The TRANQUILITY In-Care trial is designed as a double blind, placebo-controlled study to evaluate the efficacy and safety of a 60 mcg dose of BXCL 501 over a 12-week period. The trial is expected to enroll a total of approximately 150 patients 55 years and older across the spectrum of Alzheimer’s disease severity with mild, moderate, and severe dementia with mini-mental state examination (MMSE) scores of 0 to 25 who reside in skilled nursing facilities, memory care units, or assisted living facilities. The trial is expected to enroll patients with episodic agitation, with patients self-administering 60 mcg of BXCL 501 or placebo when agitation episodes occur over the trial period.

The primary endpoint is expected to be a change from baseline in the Positive and Negative Syndrome Scale-Excitatory Component (PEC) total score at two hours post-first dose. This is the same endpoint used in previous TRANQUILITY trials and in studies that supported the FDA approval of Igalmi (dexmedetomidine) sublingual film. Continued efficacy evaluations are expected to be conducted through performing additional PEC and complementary efficacy measures, including the global impression of change in agitation.

As part of the TRANQUILITY In-Care trial, the Company plans to include a feasibility cohort of 20 patients that would be evaluated in the home setting. The Company expects to generate additional Phase III efficacy and safety data in the TRANQUILITY In-Care trial to expand the database beyond the 70 patients who have already been treated with 60 mcg of BXCL 501 in TRANQUILITY I and II to date. The Company also plans to discuss the details of the requirement for long-term safety data at a future meeting with the FDA.