BioXcel Therapeutics provides updates on clinical programs for BXCL501

TRANQUILITY Program: Evaluating 60 mcg dose of BXCL501 for agitation associated with Alzheimer’s dementia: On February 20, 2024, the Company held a Type B/Breakthrough Therapy designation meeting with FDA. Based on the FDA’s feedback, the Company plans to generate additional Phase 3 efficacy and safety data in care facilities to expand the database beyond the 70 patients who have already been treated with 60 mcg of BXCL501 in TRANQUILITY I and II to date. The Company plans to generate these data in a variety of relevant care settings and across severity of dementia using the Positive and Negative Syndrome Scale-Excitatory Componentas the primary efficacy measure, as used in the prior TRANQUILITY II study. The Company announced in November 2023 that it was planning to conduct a Phase 3 trial in the at-home setting, with safety as the primary objective. Given the priority to expand the database to generate additional efficacy data in care facilities, the Company is re-evaluating the timing for initiating TRANQUILITY At Home. Given no prior regulatory precedent for episodic treatment of agitation in AD patients, the Company plans to engage with the FDA regarding the requirement for collection of long-term safety data. The Company expects to provide further guidance regarding program plans following receipt of final meeting minutes from FDA. SERENITY Program: Evaluating potential at-home use of 120 mcg of BXCL501 for agitation associated with bipolar disorders or schizophrenia: On March 6, 2024, the Company held a Type C meeting with FDA. Based on FDA feedback, Company plans to conduct a Phase 3 trial by amending the SERENITY III protocol to evaluate the 120 mcg dose of BXCL501 in the at-home setting, with safety as the primary objective and efficacy measures as exploratory endpoints. The Company expects to provide further guidance regarding program plans following receipt of final meeting minutes from FDA.