Phase 3 Program Will Continue to Assess BXCL501 to Treat Agitation
The new program consists of two clinical trials — TRANQUILITY II and TRANQUILITY III — and will assess the safety and efficacy of BXCL501 in adults ages 65 and older in assisted living or residential facilities and nursing homes.
“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 [Alzheimer’s disease] 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,” Vimal Mehta, PhD, BioXcel Therapeutics CEO, said in a press release.
Each study will enroll 150 patients. Patients in assisted living or residential facilities who require minimal assistance for daily living activities will participate in the TRANQUILITY II trial. Those living in nursing homes with moderate to severe dementia and in need of frequent or daily assistance with daily living activities will join TRANQUILITY III.
In each study, patients will self-administer an under-the-tongue (sublingual) dissolving thin film containing one of two doses — 40 micrograms (mcg) or 60 mcg — of BXCL501 or a matched placebo whenever agitation occurs. Treatment will be underway for three months.
The trial’s main efficacy goal, in both studies, is to measure changes relative to the start of the trial (baseline) in agitation, as measured by the Positive and Negative Syndrome Scale-Excitatory Component (PEC), within two hours of each dose.
Another measure of agitation used in the trials is the Pittsburgh Agitation Scale (PAS). In both scales, lower scores reflect less agitation.
Patients who complete the TRANQUILITY studies will be eligible to enroll in an open-label, 52-week study that will continue to assess the therapy’s safety and efficacy.
BXCL501 is a selective agonist of alpha-2a adrenergic receptors with potent anti-anxiety and sedative properties. It is normally used to sedate patients undergoing invasive procedures or in intensive care settings.
According to BioXcel, BXCL501 has the potential to cause fewer side effects than other antipsychotics used in clinical practice.
The new Phase 3 program follows the positive top-line results from the Phase 1b/2 TRANQUILITY clinical trial (NCT04251910), which showed BXCL501 was well-tolerated and able to rapidly and sustainably lower agitation in patients with different forms of dementia, including Alzheimer’s disease.
Based on those results, the U.S. Food and Drug Administration (FDA) granted BXCL501 breakthrough therapy designation for the acute treatment of agitation associated with dementia.
Breakthrough therapy designation is intended to accelerate the development and review of a potential therapy for a serious or life-threatening disease, with clinical evidence of substantial improvement over existing options.
“We received FDA breakthrough therapy designation for BXCL501 in March 2021 based on our Phase 1b/2 TRANQUILITY study. Following multiple meetings with the FDA, we are pleased to announce the initiation of our Phase 3 program,” Mehta said.