BioXcel Therapeutics (BTAI) investor relations material

Study background and clinical need

• Acute agitation in bipolar disorder and schizophrenia is a major burden, often leading to ED visits and hospitalization if not managed early.

• No FDA-approved therapies currently exist for at-home agitation management in these populations.

• IGALMI (dexmedetomidine sublingual film/BXCL501) is FDA-approved for institutional use; the SERENITY At-Home Phase III trial evaluated its safety and efficacy for self-administration at home, aiming for label expansion.

Study design and patient population

• SERENITY At-Home was a double-blind, placebo-controlled, 12-week trial with 246 randomized patients and over 2,600 treated agitation episodes.

• Included adults aged 18–75 with schizophrenia, schizoaffective, schizophreniform, or bipolar I/II disorder, stable on psychotropic regimens, and with recent agitation history.

• Excluded those with serious or unstable medical illness, certain personality disorders, or recent substance use disorder.

• Patients self-administered the film as needed, with or without a co-resident informant, and could use alternative coping strategies.

• Patients or caregivers completed a modified CGI-S assessment two hours after dosing.

Safety and tolerability results

• BXCL501 was well tolerated with no drug-related serious adverse events, falls, or syncopes reported.

• No patients discontinued due to treatment-emergent adverse events in the BXCL501 arm.

• Adverse event profile was consistent with prior institutional studies and the approved IGALMI label; most events were mild.

• Most common side effects: somnolence (22% first dose, 14% all doses vs. 17%/8% placebo), oral paresthesia/hypoesthesia, dizziness, dry mouth; incidence did not increase with repeat dosing.

• No new or unexpected safety signals emerged with repeated use.