BioXcel Therapeutics Announces First Patient Dosed in TRANQUILITY II Phase 3 Trial for Acute Treatment of Agitation in Patients with Alzheimer’s Disease

• Top-line data for TRANQUILITY II anticipated in Q4 2022/early Q1 2023
• Phase 3 TRANQUILITY III study underway with patient enrollment initiating in H2 2022

NEW HAVEN, Conn., May 03, 2022 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a commercial-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, today announced the first patient has been dosed in the Phase 3 TRANQUILITY II study of BXCL501, the Company’s proprietary, orally dissolving thin film formulation of dexmedetomidine, for the acute treatment of agitation in patients with Alzheimer’s disease (AD). The pivotal Phase 3 TRANQUILITY program includes two studies, TRANQUILITY II and TRANQUILITY III, which are designed to evaluate the safety and efficacy of BXCL501 in adults 65 years and older in assisted living or residential facilities and nursing homes.

“There are an estimated 100 million agitation episodes annually in the U.S. associated with Alzheimer’s disease¹, which have a devastating impact on patients and their caregivers,” said Robert Risinger, M.D., Chief Medical Officer of BioXcel Therapeutics. “We believe the recent FDA approval of BXCL501 for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults has laid a strong foundation for pursuing this Alzheimer’s-related agitation program to potentially address this debilitating symptom for patients. Importantly, we are also expanding TRANQUILITY II to more than 10 clinical trial sites in the U.S. and with no current FDA approved treatments for agitation associated with this disease, we are making strong and swift efforts to potentially bring BXCL501 and its proven ability to address agitation to this large market.”

The Company’s decision to continue the evaluation of both the 40 and 60 mcg dosing regimens in the TRANQUILITY II and III pivotal trials is further supported by results from a recent 46 patient, multicenter, placebo-controlled study evaluating the efficacy, safety and tolerability of BXCL501 40 mcg dose in patients with agitation associated with dementia. Previously, BXCL501 was granted Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for the acute treatment of agitation associated with dementia. BXCL501 demonstrated statistically significant reductions in agitation measures with both the 30 and 60 mcg doses as measured by multiple scales with no severe or serious adverse events.