BioXcel Therapeutics, Inc. (NASDAQ:BTAI) is a biopharmaceutical company leveraging artificial intelligence (AI) to develop transformative medicines in neuroscience and immuno-oncology. The company's strategic focus is on advancing its late-stage clinical programs, BXCL501 and BXCL701, while driving the commercialization of its approved product, IGALMI™.

Financials

In the fiscal year ended December 31, 2023, BioXcel Therapeutics reported annual revenue of $1,380,000 and a net loss of $179,053,000. The company's annual operating cash flow was -$155,006,000, and its annual free cash flow was -$155,026,000.

For the first quarter of 2024, the company reported net revenue from IGALMI™ of $582,000, a 182% year-over-year increase from $206,000 in the same period of 2023. The sequential quarterly revenue increased 55% in the first quarter of 2024 compared to the fourth quarter of 2023, primarily attributable to increasing demand from existing customers, the addition of new customer orders, and volume-based contracting.

Research and development expenses for the first quarter of 2024 were $11.4 million, down from $27.8 million in the same period of 2023. The decrease was primarily due to the wind-down of the SERENITY III and TRANQUILITY II and III trials, as well as decreased professional fees, personnel, and related costs. Selling, general, and administrative expenses were $13.3 million for the first quarter of 2024, compared to $23.6 million in the same period of 2023, with the reduction primarily attributable to a decrease in personnel and costs associated with the commercialization of IGALMI™.

BioXcel Therapeutics reported a net loss of $26.8 million for the first quarter of 2024, compared to a net loss of $52.8 million in the same period of 2023. The company used approximately $17.7 million in operating cash during the first quarter of 2024. As of March 31, 2024, the company had $74.1 million in cash and cash equivalents, which includes the $25 million from the registered direct offering announced on March 25, 2024.

Neuroscience Pipeline Advancement

BioXcel Therapeutics is focused on advancing its late-stage neuroscience programs, BXCL501 and BXCL502, to address significant unmet needs in the treatment of agitation associated with various neuropsychiatric and neurodegenerative disorders.

BXCL501 (Dexmedetomidine) for Agitation

BXCL501, the company's lead neuroscience candidate, is an investigational, proprietary, orally dissolving film formulation of dexmedetomidine (Dex) targeting symptoms from stress-related behaviors such as agitation. The company is evaluating BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home setting (SERENITY program) and for the acute treatment of agitation (non-daily) associated with dementia due to probable Alzheimer's disease in care facilities and at-home settings (TRANQUILITY program).

SERENITY Program: Agitation Associated with Bipolar Disorders and Schizophrenia (At-Home Setting)

The SERENITY At-Home trial is designed as a double-blind, placebo-controlled study to evaluate the safety and efficacy of a 120-microgram dose of BXCL501 over a 12-week period in the outpatient setting. The primary objective is safety, with efficacy measures as exploratory endpoints. The company believes this program may provide a near-term opportunity to seek a label expansion for the previously approved 120-microgram dose of IGALMI™.

TRANQUILITY Program: Acute Treatment of Agitation Associated with Dementia due to Probable Alzheimer's Disease (AAD)

The TRANQUILITY In-Care trial is designed as a double-blind, placebo-controlled study to evaluate the efficacy and safety of a 60-microgram dose of BXCL501 over a 12-week period in care facilities. The primary endpoint is the change in PEC (Positive and Negative Syndrome Scale-Excitatory Component) score at 2 hours post-first dose, which is the same endpoint used in previous positive TRANQUILITY trials and in studies that supported the FDA approval of IGALMI™.

The company plans to discuss the details of the requirement for long-term safety data with the FDA to support the submission of a supplemental new drug application (sNDA) for BXCL501 in the treatment of agitation associated with dementia due to probable Alzheimer's disease.

BXCL502 Development

BioXcell Therapeutics is also developing BXCL502, a second neuropsychiatric drug candidate identified through its AI-based platform. The company plans to evaluate BXCL502 initially as a monotherapy and possibly in combination with BXCL501 for the chronic treatment of agitation in patients with dementia and acute stress disorder.

Immuno-Oncology Pipeline Advancement

In the company's immuno-oncology segment, the lead candidate is BXCL701, an investigational oral innate immune activator being developed by the company's wholly owned subsidiary, OnkosXcel Therapeutics, as a potential therapy for the treatment of aggressive forms of prostate cancer, pancreatic cancer, and other solid and liquid tumors.

BXCL701 for Metastatic Pancreatic Cancer

BioXcel Therapeutics recently announced the completion of patient enrollment in the safety portion of the Phase 2 trial of BXCL701 in combination with KEYTRUDA® (pembrolizumab) in metastatic pancreatic ductal adenocarcinoma. The company stated that a late-breaking abstract on the trial results has been selected for presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting.

IGALMI™ Commercialization Progress

BioXcel Therapeutics obtained FDA approval for IGALMI™ (dexmedetomidine) sublingual film in April 2022 for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults. The company has implemented a revised commercial strategy, focusing on a hospital/Integrated Delivery Network (IDN) contracting approach with a Corporate Account Director (CAD) team. This strategy has yielded results, with a 55% sequential quarterly increase in net revenue from IGALMI™ in the first quarter of 2024 compared to the fourth quarter of 2023.

The company also announced that the Centers for Medicare & Medicaid Services (CMS) assigned a new J-Code for IGALMI™ (J1105), which became effective on January 1, 2024. The J-Code is expected to facilitate expanded access to IGALMI™ for patients with agitation associated with bipolar disorder or schizophrenia.

Intellectual Property Expansion

BioXcel Therapeutics has been actively expanding its intellectual property portfolio to protect its neuroscience and immuno-oncology assets. As of April 2024, the company has 30 granted or allowed patents and more than 140 additional patent applications in prosecution. The company also has 8 U.S. patents for IGALMI™ listed in the FDA's Orange Book, with 2 additional recently allowed patents eligible for listing.

Outlook

BioXcel Therapeutics had $74.1 million in cash and cash equivalents as of March 31, 2024, which includes the $25 million from the registered direct offering announced on March 25, 2024. The company is focused on further strengthening its balance sheet and exploring multiple financing options, including potential partnerships, to extend its cash runway.

The company's strategic reprioritization, announced in August 2023, has resulted in a reduction of approximately 60% of its workforce to preserve cash and prioritize investment in its core clinical programs. BioXcel Therapeutics continues to evaluate its operating expenses and explore ways to optimize its cost structure to support the advancement of its neuroscience and immuno-oncology pipelines.

Conclusion

BioXcel Therapeutics is making significant progress in advancing its neuroscience and immuno-oncology pipelines, while also driving the commercialization of its approved product, IGALMI™. The company's focus on its late-stage clinical programs, BXCL501 and BXCL701, and the strategic shift in IGALMI™ commercialization have yielded positive results. With a strengthened intellectual property portfolio and a focus on optimizing its financial position, BioXcel Therapeutics appears well-positioned to continue its transformation and create value for its shareholders.