NEW HAVEN, Conn. - BioXcel Therapeutics, Inc. (NASDAQ: BTAI) announced plans to initiate a Phase 3 clinical trial, named TRANQUILITY In-Care, to examine the efficacy and safety of BXCL501, an investigational treatment for agitation in Alzheimer's dementia (AAD) patients.
This decision follows recent discussions with the U.S. Food and Drug Administration (FDA), including a meeting on February 20, 2024, where the company received guidance on its development path for AAD.
BXCL501 is an orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. The upcoming double-blind, placebo-controlled study aims to enroll approximately 150 patients aged 55 and older, residing in various care settings, to determine the treatment's impact on acute agitation episodes associated with Alzheimer's.
The trial's primary endpoint will measure changes in the Positive and Negative Syndrome Scale-Excitatory Component (PEC) total score two hours after the first dose, consistent with previous TRANQUILITY trials and studies supporting FDA approval of IGALMI™ (dexmedetomidine) sublingual film for schizophrenia and bipolar disorder I or II.
Dr. Vincent O’Neill, Chief of Product Development and Medical Officer at BioXcel Therapeutics, emphasized the significance of advancing the AAD program, noting the lack of FDA-approved therapies for the acute treatment of AAD. The company also plans to explore the feasibility of administering BXCL501 in a home setting with a cohort of 20 patients.
This initiative builds upon the data from TRANQUILITY I and II trials, where 70 patients have already been treated with a 60 mcg dose of BXCL501. Further discussions with the FDA will address long-term safety data requirements.
The company's approach to drug development involves leveraging artificial intelligence to identify new therapeutic indications for existing drugs. BXCL501 has received Breakthrough Therapy designation from the FDA for the acute treatment of agitation associated with dementia, among other designations for various neuropsychiatric disorders.
BioXcel Therapeutics' announcement is based on a press release statement, and the trial's commencement is a step toward addressing the unmet medical needs of Alzheimer's dementia patients.
Please note that the safety and efficacy of BXCL501 for the investigational uses have not been established. The information provided in this article is based on a press release statement from BioXcel Therapeutics.
InvestingPro Insights
As BioXcel Therapeutics, Inc. (NASDAQ: BTAI) gears up for a critical Phase 3 clinical trial for BXCL501, investors are closely monitoring the company's financial health and market performance. According to InvestingPro data, BioXcel Therapeutics currently has a market capitalization of approximately $78.58 million. The company's revenue has seen a substantial increase over the last twelve months as of Q4 2023, with a growth rate of 268.0%, signaling a potential upswing in its business activities.
Despite these positive signs, the company faces challenges as reflected in some of the InvestingPro Tips. Analysts are wary of the company's cash burn and weak gross profit margins, which stand at 8.7% as of the last twelve months ending Q4 2023. Additionally, the stock has experienced a notable decline over the past year, with a one-year price total return of -84.81% as of early 2024. These financial metrics are important considerations for investors as they weigh the prospects of BioXcel's investigational treatment and its impact on the company's future.
For those looking to delve deeper into BioXcel's financials and market performance, InvestingPro offers additional insights. With more InvestingPro Tips available, investors can gain an enhanced understanding of the company's valuation, which suggests a poor free cash flow yield and high revenue valuation multiple. To access these comprehensive analytics and tips, consider using the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription at https://www.investing.com/pro/BTAI.
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