BioXcel Therapeutics, Inc., a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, announced that BXCL501, the Company's investigational, proprietary, orally dissolving thin film formulation of dexmedetomidine ("Dex"), has been granted Breakthrough Therapy designation from the U.S. Food and Drug Administration ("FDA") for the acute treatment of agitation associated with dementia.

The Breakthrough Therapy designation is intended to expedite the development and review of certain product candidates designed to treat serious or life-threatening diseases or conditions, and the designation includes increased interaction and guidance from the FDA.

“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," stated 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. We look forward to working closely with the FDA to advance BXCL501 into a pivotal dementia program, in hopes of quickly bringing this therapy to the millions of patients across treatment settings that lack alternative options.”

The Breakthrough Therapy designation for BXCL501 was supported by the positive topline data from the Phase 1b/2 TRANQUILITY study for the acute treatment of agitation associated with dementia, including Alzheimer's disease. BXCL501 demonstrated statistically significant reductions in agitation measures at 2 hours post-dose with both the 30 and 60 mcg doses as measured by multiple scales. The dose-dependent response observed has the potential to support the Company’s plans to evaluate BXCL501 for use across the full range of dementia care settings.