BioXcel Therapeutics, Inc. (BTAI), a biopharmaceutical company focusing on the development of innovative medicines using artificial intelligence, has revealed promising findings from its Phase 3 trial, TRANQUILITY II. The trial evaluated the efficacy and safety of BXCL501, a proprietary orally dissolving film formulation of dexmedetomidine, for the acute treatment of Alzheimer's disease-related agitation. The results demonstrate significant potential in addressing a critical need for episodic treatment options for patients with mild to moderate Alzheimer's disease.

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Positive Efficacy Results for BXCL501

TRANQUILITY II met its primary efficacy endpoint with the 60 mcg dose of BXCL501. The trial exhibited a statistically significant and clinically meaningful 7.5 point reduction in the Positive and Negative Syndrome Scale-Excitatory Component (PEC) total score after 2 hours, compared to a 5.4 point reduction with the placebo (p=0.0112). The 60 mcg dose also achieved the first key secondary endpoint by reducing agitation symptoms at 1 hour during the initial episode of agitation (p=0.0185).

Additionally, the efficacy of the 60 mcg dose was supported by various secondary measures, including the Clinical Global Impression of Improvement (CGI-I) and Agitation-Calmness Evaluation Scale (ACES). Impressively, 76% of patients responded favorably to the first 60 mcg dose, reporting being "Very Much" or "Much Improved" (CGI-I of 1 or 2), compared to 50% with the placebo.

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Safety and Tolerability of BXCL501

BXCL501 exhibited a favorable safety profile throughout the trial. The majority of safety events occurring within 24 hours of dosing were mild or moderate in severity and consistent with the current label for IGALMI™. Notably, dosing for subsequent episodes did not lead to a significant increase in adverse events, and no treatment-related serious adverse events were observed over the 12-week study period.

The trial also reported no syncope or falls related to the trial drug across all 443 episodes. Falls were primarily observed outside the 24-hour treatment window, with only one fall reported in the placebo group. These findings further validate the safety and tolerability of BXCL501.

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Expert Perspectives on BXCL501

Dr. George Grossberg, Professor and Director of the Division of Geriatric Psychiatry at St. Louis University School of Medicine, expressed excitement about the potential of BXCL501 in addressing Alzheimer's disease-related agitation. He highlighted the desirable onset of action and meaningful reduction in agitation at the 60 mcg dose, along with the favorable tolerability profile, emphasizing the potential positive impact on patients, caregivers, and families affected by this condition.