BioXcel Therapeutics Announces First Patient Dosed in TRANQUILITY III Phase 3 Trial for Acute Treatment of Agitation in Patients With Alzheimer’s Disease

BioXcel Therapeutics, Inc. recently announced the first patient has been dosed in the pivotal Phase 3 TRANQUILITY III trial of BXCL501 (dexmedetomidine) sublingual film, the company’s proprietary, orally dissolving film, under investigation for the acute treatment of agitation in patients with Alzheimer’s disease (AD). AD is the most prevalent type of dementia in the US. The TRANQUILITY program includes two investigational studies, TRANQUILITY II and TRANQUILITY III, which are designed to evaluate the safety and efficacy of BXCL501 for the acute treatment of Alzheimer’s-associated agitation in adults 65 years and older in assisted living or residential care facilities and nursing homes.

“The prevalence of Alzheimer’s disease is unfortunately increasing and there remains no FDA-approved product indicated for patients experiencing agitation associated with this condition,” said Robert Risinger, MD, Chief Medical Officer, Neuroscience of BioXcel Therapeutics. “With two pivotal trials underway in our TRANQUILITY program, we are aiming to expand BXCL501’s potential to treat the full spectrum of episodic and intermittent chronic agitation market, and address the costly health-care burden related to Alzheimer’s agitation.”

There are approximately 100 million reported agitation episodes that occur in the US each year related to Alzheimer’s disease. The number of adults over the age of 65 with AD is expected to double from 5.8 million in 2020 to 11.8 million in 2040, representing a significant and growing market opportunity for BXCL501. This potential opportunity is in addition to the current acute treatment of agitation associated with schizophrenia or bipolar I or II disorder market opportunity. Approximately 16 million episodes of schizophrenia and bipolar disorder-associated agitation occur in institutional settings and, when combined with at-home episodes, 23 million annually in the US.

TRANQUILITY II and III will evaluate the safety and efficacy of BXCL501 in patients who experience agitation across diverse settings and across the range of dementia severity. Each trial will enroll approximately 150 patients with dementia ages 65 years and older who will self-administer 40mcg or 60mcg of BXCL501 or placebo under the supervision of a trained research staff member whenever agitation episodes occur over a three-month period. TRANQUILITY II will assess patients in assisted living or residential care facilities requiring minimal assistance with activities of daily living. TRANQUILITY III will assess patients residing predominantly in nursing homes with moderate to severe dementia who require moderate or greater assistance with activities of daily living. The primary efficacy endpoint for both studies is change in Positive and Negative Syndrome Scale-Excitatory Component (PEC) total score from baseline measured at two hours after the initial dose and subsequent doses.

BXCL501 is an investigational proprietary, orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. BioXcel Therapeutics believes that BXCL501 potentially targets an important mediator of agitation, and the Company has observed anti-agitation results in multiple clinical studies across several neuropsychiatric disorders. BXCL501 is under investigation for the acute treatment of agitation associated with bipolar I or II disorder or schizophrenia in the at-home setting, for the acute treatment of Alzheimer’s-related agitation, and as an adjunctive treatment for major depressive disorder. The safety and efficacy of BXCL501 for these investigational uses have not been established. BXCL501 has been granted Breakthrough Therapy designation for the acute treatment of agitation associated with dementia and Fast Track designation for the acute treatment of agitation associated with schizophrenia, bipolar disorders, and dementia.

BioXcel Therapeutics, Inc. is a biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology. The company’s drug re-innovation approach leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indications. The company’s commercial product, IGALMI (developed as BXCL501), is a proprietary, sublingual film formulation of dexmedetomidine approved for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults. The safety and effectiveness of IGALMI has not been established beyond 24 hours from the first dose. For more information, please visit IGALMIhcp.com and also see the IGALMI full Prescribing Information. BXCL501 is under evaluation for at-home use for the acute treatment of agitation in bipolar and schizophrenia patients, for acute treatment of Alzheimer’s-related agitation, and as an adjunctive treatment for major depressive disorder. The safety and efficacy of BXCL501 for these uses have not been established. The company is also developing BXCL502 as a potential therapy for chronic agitation in dementia. Under its subsidiary, OnkosXcel Therapeutics, the Company is developing BXCL701, an investigational, orally administered, systemic innate immune activator for the treatment of aggressive forms of prostate cancer. The safety and efficacy of BXCL502 and BXCL701 have not been established. For more information, visit bioxceltherapeutics.com.