BioXcel Therapeutics, Inc. Q3 FY2023 earnings call

• Development updates: Significant progress with BXCL501's TRANQUILITY and SERENITY III late-stage clinical programs, aligned with FDA recommendations for Phase 3 trials in home settings. - Financing: Entered binding term sheet with Oaktree Capital Management and Qatar Investment Authority to amend financing agreements, enhancing financial flexibility. - IGALMI: Focus on commercial efforts, J-Code issuance by CMS to streamline reimbursement, and patent protection extended to 2043. - Other programs: Progress on NIDA-funded trial for opioid use disorder, emerging neuroscience programs, and positive survival data for BXCL501 in prostate cancer trials; focus on strategic options for OnkosXcel Therapeutics.

Net revenue of IGALMI was approximately $341,000 for the third quarter of 2023. Research and development expenses were $19.6 million for Q3 2023, down from $22.1 million in the same period of 2022. Selling, general and administrative expenses were $24.3 million in Q3 2023, up from $17.1 million in Q3 2022. BioXcel Therapeutics had a net loss of $50.5 million for Q3 2023, compared to a net loss of $41.8 million in Q3 2022. Cash and cash equivalents totaled $90 million as of September 30, 2023, with an estimated cash runway lasting through mid-2024.

• Expect to finalize study protocols for TRANQUILITY and SERENITY III trials and initiate them soon. - Confidence in using TRANQUILITY II data for an sNDA submission based on independent audit. - Amended financing terms provide financial flexibility to prioritize development programs.

• Uncertainties in clinical trial outcomes and regulatory reviews for TRANQUILITY and SERENITY III. - Risks related to cash runway and prioritization of development efforts among programs. - Market adoption risks for IGALMI and BXCL501 in respective indications.

Q: With respect to the 100 patient trial for TRANQUILITY, can you give more color on the development strategy of going for a broad label initially and criteria for success?

A: Vimal Mehta stated they have alignment with FDA for a home-setting trial, aiming for broad access to patients, and Rob Risinger mentioned it's a double-blind, placebo-controlled study focusing on safety and caregiver-assessed clinical benefit.

Q: Can you clarify if you're able to use all data from TRANQUILITY II for the sNDA package?

A: Vimal Mehta said the company believes, based on independent audit and available info, they can use the TRANQUILITY II data announced in June.

Q: What's the ultimate path forward for 501 in chronic agitation and timeline?

A: Vimal Mehta said they'll work with the agency to explore a path, but current focus is on acute treatment in home setting for Alzheimer's-related agitation.

Q: On the SERENITY III program, what led to going with 120-microgram dose and discussions with FDA?

A: Vimal Mehta explained 120-microgram dose has significant safety data, efficacy established, and allows evaluation in home setting to capture more episodes than hospital setting.

Q: When might you finalize protocols and initiate the at-home study for TRANQUILITY?

A: Vimal Mehta said protocol is under development, will be submitted to FDA, and they'll provide guidance on trial initiation and patient dosing timeline once protocol is finalized.