Phase 3 Study of BXCL501 for Agitation in Alzheimer Disease Initiated


Two parallel studies will evaluate the BioXcel Therapeutics’ drug’s effectiveness as an acute therapy in treating a full spectrum of agitation in 300 patients with AD.

Vimal Mehta, PhD, CEO, BioXcel Therapeutics

Vimal Mehta, PhD

A phase 3 program studying BXCL501, an orally dissolving thin film formulation of dexmedetomidine, has been initiated to study the safety and efficacy of the drug in the acute treatment of agitation in patients with Alzheimer disease (AD). The program consists of 2 studies, TRANQUILITY 2 and TRANQULITY 3, BioXcel Therapeutics announced.1

In March 2021, BXCL501 received breakthrough designation from the FDA for the acute treatment of agitation associated with dementia.2 The designation was supported by positive topline data from the phase 1b/2 TRANQULITY study (NCT04251910), with the treatment demonstrating significant reductions in agitation measures at both dose levels, 30 mcg and 60 mcg, at 2 hours post-dose. 

“Following multiple meetings with the FDA, we are pleased to announce the initiation of our phase 3 program,” Vimal Mehta, PhD, CEO, BioXcel Therapeutics, said in a statement.1 “This marks an important advancement in potentially bringing this novel treatment to the more than 4 million patients, who experience agitation as one of AD’s most devastating symptoms. We are leading the development path for this innovative therapy and are confident in BXCL501’s potential to treat acute, as well as intermittent, forms of agitation."

READ MORE: LIBRA Index Synopsizes Brain Health, Higher Scores Indicate Dementia Risk

The parallel phase 3 pivotal trials will be randomized, placebo-controlled, and adaptive, enrolling approximately 150 patients with dementia older than the age of 65 years in each study. When agitation occurs, patients will self-administer a 40-mcg or 60-mcg dose of BXCL501, or placebo over a 3-month period. 

The trials differ in terms of activities of daily living (ADL) criteria. TRANQUILITY 2 will enroll patients in assisted living or residential facilities who require minimal assistance with ADL, while TRANQUILITY 3 will enroll patients in nursing homes who have moderate to severe dementia and require moderate or greater assistance with ADL. 

To assess agitation, the trials will measure change from baseline in Positive and Negative Syndrome Scale-Excitatory Component (PEC) and Pittsburgh Agitation Scale scores. Change from baseline in PEC scores at 2 hours after initial dose and after subsequent doses will serve as the primary efficacy end point. Following completion of the phase 3 trials, participants will be eligible to enroll in a 52-week, open-label safety study to evaluate the efficacy of continued use of BXCL501. 

BXCL501 has previously had success in achieving anti-agitation results in clinical trials of several neuropsychiatric disorders, including schizophrenia-related agitation, bipolar disorder-related agitation, and dementia-related agitation. It has been granted fast track designation from the FDA for the acute treatment of agitation in each disorder.

1. BioXcel Therapeutics initiates pivotal phase 3 program of BXCL501 for acute treatment of agitation in patients with Alzheimer disease. News relase. BioXcel Therapeutics. December 15, 2021. Accessed December 15, 2021.
2. BioXcel Therapeutics receives breakthrough therapy designation for BXCL501 for the acute treatment of agitation associated with dementia. News release. BioXcel Therapeutics. March 15, 2021. Accessed December 15, 2021.
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