Benzinga | Jan 5, 2021 07:17AM EST

BioXcel Therapeutics Announces BXCL501 Met Primary And All Secondary Endpoints In TRANQUILITY Phase 1b/2 Study For Acute Treatment Of Agitation In Dementia, Including Alzheimer's Disease

Statistically significant, clinically meaningful, rapid and durable reductions in agitation achieved with the 60 mcg dose as measured by multiple agitation scales



BXCL501 was well tolerated with no severe or serious adverse events

Results provide a clear path toward initiating a pivotal program for BXCL501 in dementia

Agitation associated with dementia affects an estimated 4 million patients in the U.S.

Company to host conference call today at 8:30 a.m. ET

NEW HAVEN, Conn., Jan. 05, 2021 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. ("BioXcel" 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 BXCL501, the Company's proprietary, orally dissolving thin film formulation of dexmedetomidine, met the primary and secondary endpoints of the TRANQUILITY trial at the 60 mcg dose level. Topline results from the evaluated doses showed that BXCL501 was generally well tolerated, with rapid and durable reductions observed in measures of acute agitation with the 60 mcg dose.

The TRANQUILITY Phase 1b/2 randomized, placebo controlled, adaptive, ascending dose finding study enrolled 54 patients in assisted living facilities with agitation related to dementia, 87 percent of which had Alzheimer's disease. Patients received BXCL501 at either 30 mcg (n=16), 60 mcg (n=20), 90 mcg (n=4) or placebo (n=14). The study's primary safety and tolerability endpoints were met, with no severe or serious adverse events reported. Adverse events in the trial included hypotension (10%, 0% and 0%, for 60 mcg, 30 mcg and placebo, respectively), orthostatic hypotension (5%, 6.3% and 0%, respectively) and dizziness (5%, 6.3% and 0%, respectively). The most common adverse event was somnolence characterized as either mild (55% for 60 mcg, 50% for 30 mcg and 7.1% for placebo) or moderate (5%, 0% and 0%, respectively). Orthostasis and dizziness were observed in some patients receiving the highest 90 mcg dose. Higher exposure levels of BXCL501 were observed in this elderly patient population compared to earlier trials and, as a result, the Company focused on studying the 30 and 60 mcg doses. Notably, there were no reported cases of syncope or falls in any of the patients studied.

The trial met its secondary efficacy endpoints with the 60 mcg dose compared to placebo in all three primary agitation scales--the Positive and Negative Syndrome Scale-Excitatory Component ("PEC"), the Pittsburgh Agitation Scale ("PAS"), and the Modified Cohen-Mansfield Agitation Inventory ("Mod-CMAI")--demonstrating statistically significant and clinically meaningful reductions in total scores at two hours post-dosing. The reductions were both rapid and durable with numerical separation from placebo in PEC total score seen as early as 30 minutes, and with statistically significant separation from placebo in both PEC and PAS total scores observed at 60 minutes* and lasting through eight hours post-dosing. The 30 mcg dose cohort showed numerical improvements at all three measures.