BioXcel Therapeutics Announced The Achievement Of Clinical And Regulatory Progress For Its Pivotal Phase 3 Trials Of BXCLl501 For The Acute Treatment Of Agitation Associated With Bipolar Disorders Or Schizophrenia (SERENITY At-Home) And Agitation Associated With Alzheimer's Dementia (TRANQUILITY In-Care)

SERENITY At-Home Pivotal Phase 3 trial

• Designed to evaluate the safety of a 120 mcg dose of BXCL501 in the at-home setting for agitation associated with bipolar disorders or schizophrenia
  • The first patient has been randomized.
  • The Company announced trial initiation on September 5, 2024, with an expected trial duration of 9 to 12 months.
  • The double-blind, placebo-controlled, 12-week outpatient trial is expected to enroll approximately 200 patients residing at home.

TRANQUILITY In-Care Pivotal Phase 3 trial

• Designed to evaluate the efficacy and safety of a 60 mcg dose of BXCL501 for AAD over a 12-week period
  • Received feedback from the U.S. Food and Drug Administration on proposed protocol
  • The double-blind, placebo-controlled study trial is expected to enroll a total of approximately 150 patients aged 55 years and older across the spectrum of Alzheimer's disease severity with mild, moderate, and severe dementia with mini-mental state examination (MMSE) scores of 0 to 25 who reside in skilled nursing facilities, memory care units, or assisted living facilities.
  • The trial is expected to enroll patients with episodic agitation, with patients self-administering 60 mcg of BXCL501 or placebo when agitation episodes occur over the trial period.
  • The planned primary endpoint is a change from baseline in the Positive and Negative Syndrome Scale-Excitatory Component (PEC) total score at two hours post-first dose. This is the same endpoint used in previous TRANQUILITY trials and in studies that supported the FDA approval of IGALMI (dexmedetomidine) sublingual film.
  • Continued efficacy evaluations are expected to be conducted through performing additional PEC and complementary efficacy measures, including the global impression of change in agitation.