The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to BXCL501 (dexmedetomidine; BioXcel Therapeutics) for the acute treatment of agitation associated with dementia.

Dexmedetomidine is currently available as a solution for intravenous infusion for use in clinical anesthesia and sedation in an intensive care setting. BXCL501 is an investigational oral thin film formulation of dexmedetomidine, a selective alpha-2a receptor agonist.

The designation is supported by data from the randomized, double-blind, placebo-controlled phase 1b/2 TRANQUILITY study ( NCT04251910), which evaluated the efficacy, safety and pharmacokinetics of BXCL501 in 54 patients aged 65 years and older with acute agitation associated with dementia; 87% of study participants had Alzheimer disease. Patients were randomly assigned to receive BXCL501 30mcg (n=16), 60mcg (n=20), 90mcg (n=4) or placebo (n=14).

Results showed statistically significant reductions in agitation measures (Positive and Negative Syndrome Scale-Excitatory Component; Pittsburgh Agitation Scale; and Modified Cohen-Mansfield Agitation Inventory) at 2 hours post-dose with the 30mcg and 60mcg doses compared with placebo. The most common adverse event reported was somnolence which was mild or moderate in severity.

“Managing dementia related agitation, specifically in elderly patients, represents a significant challenge for physicians and caregivers, as there are currently no FDA-approved therapies and off-label drugs come with black box warnings,” said Vimal Mehta, Chief Executive Officer of BioXcel. “The FDA’s decision to grant Breakthrough Therapy designation further underscores the significant unmet need for a new treatment for this underserved patient population, as well as highlights BXCL501’s potential in becoming the first therapeutic option, if approved, to address this debilitating medical condition.”

The Company also recently submitted a New Drug Application for BXCL501 to treat agitation associated with schizophrenia and bipolar disorders I and II.


  1. BioXcel Therapeutics receives FDA Breakthrough Therapy designation for BXCL501 for the acute treatment of agitation associated with dementia. [press release]. New Haven, CT: BioXcel Therapeutics, Inc.; March 15, 2021. 
  2. BioXcel Therapeutics announces BXCL501 met the primary and all secondary endpoints in the TRANQUILITY phase 1b/2 study for the acute treatment of agitation in dementia, including Alzheimer’s disease. [press release]. New Haven, CT: BioXcel Therapeutics, Inc.; January 5, 2021.