BioXcel Therapeutics (BTAI) Jefferies Global Healthcare Conference summary

Market expansion and strategic vision

• Preparing for two pivotal Phase 3 trials to expand BXCL501 into home settings for schizophrenia, bipolar, and Alzheimer's agitation, targeting a potential 123 million additional episodes annually.

• IGALMI, the currently approved drug, covers 16 million episodes, with a focused strategy to maintain brand equity and expand physician and caregiver experience.

• Expansion into home settings addresses a significant unmet need, as no FDA-approved drugs exist for agitation in these environments.

• Volume contracting and targeted commercial strategy have led to revenue uptick and efficient brand maintenance.

SERENITY program updates

• SERENITY At-Home trial will evaluate the 120 microgram dose for safety, with protocols submitted and site selection underway.

• Market research indicates strong patient and provider support for home use, with 80% positive response.

• Institutional and home settings are complementary, with both 120 and 180 microgram doses used based on patient needs.

• The program is a near-term value driver, aiming for sNDA submission upon successful trial completion.

TRANQUILITY program and Alzheimer's opportunity

• TRANQUILITY targets agitation in Alzheimer's, a market with about 100 million episodes and no approved acute treatments.

• TRANQUILITY I led to breakthrough therapy designation; TRANQUILITY II showed positive Phase 3 results.

• TRANQUILITY In-Care trial will enroll 150 patients, simulating real-life settings across disease severity, with efficacy as the primary endpoint.

• Strategy allows for staggered or parallel trials to optimize sNDA submission for both in-care and home settings.