BioXcel Therapeutics Reports Financial Results for the Fourth Quarter and Full Year 2023
BioXcel Therapeutics, Inc. provides updates on late-stage clinical programs for BXCL501, focusing on treating agitation associated with Alzheimer’s dementia, bipolar disorders, and schizophrenia. The company plans to expand data for TRANQUILITY and SERENITY programs, emphasizing safety and efficacy. Financially, the company reported increased net revenue from IGALMI, reduced R&D and SG&A expenses, and a net loss for the fourth quarter and full year 2023. The company's cash and cash equivalents are estimated to fund operations through mid-2024.
Positive
BioXcel Therapeutics updates late-stage clinical programs for BXCL501 for treating agitation associated with various disorders.
The company plans to expand data for TRANQUILITY and SERENITY programs, focusing on safety and efficacy.
Financially, the company reported increased net revenue from IGALMI, reduced R&D and SG&A expenses, and a net loss for the fourth quarter and full year 2023.
BioXcel Therapeutics estimates its current cash and cash equivalents will fund operations through mid-2024.
Negative
The company reported a net loss for the fourth quarter and full year 2023, with reduced cash and cash equivalents compared to the previous year.
R&D and SG&A expenses were reduced, potentially impacting future research and development activities.
The company's estimated cash runway does not include potential additional capital, raising concerns about future financing activities.
The update on BioXcel Therapeutics' TRANQUILITY and SERENITY clinical programs is significant for stakeholders, as it indicates progress in the development of BXCL501 for treating acute agitation in Alzheimer's dementia and bipolar disorders/schizophrenia. The engagement with the FDA and the strategic planning to generate additional Phase 3 data are critical steps that could potentially lead to approval and commercialization. Such developments are likely to influence the company's stock value as they are closely watched by investors for signs of future revenue potential.
Moreover, the announcement of a permanent J-Code for IGALMI™ suggests improved reimbursement pathways, which may enhance product uptake and revenue. The patent portfolio updates provide insights into the company's efforts to secure long-term exclusivity for its products, which is a key factor in maintaining competitive advantage in the pharmaceutical industry.
From a financial perspective, the reported increase in net revenue from IGALMI, despite being modest, indicates initial market penetration and potential growth trajectory. However, the reported net losses and increased SG&A expenses highlight the financial burden of commercialization and development efforts. The current cash and cash equivalents suggest a limited runway, necessitating careful financial management or additional capital infusion to sustain operations beyond mid-2024. Investors should monitor the company's capital-raising activities and cost management strategies closely.
The clinical relevance of BXCL501 for acute agitation in Alzheimer’s dementia and related disorders is underscored by the lack of FDA-approved treatments for such indications. The company's focus on expanding the efficacy data in care facilities using the Positive and Negative Syndrome Scale-Excitatory Component (PEC) as a primary measure is a methodologically sound approach to demonstrate the drug's impact on patients' symptoms. The decision to re-evaluate the timing for the TRANQUILITY At Home study in light of prioritizing efficacy data collection in care facilities reflects strategic clinical development planning. The implications for patient care are substantial if BXCL501 proves to be effective and safe, as it would address a significant unmet medical need.
03/12/2024 - 07:00 AM
Provides update on two late-stage clinical programs for BXCL501 for potential treatment of agitation
Recently completed meetings with U.S. Food and Drug Administration for TRANQUILITY and SERENITY programs
C onference call and webcast set for 8:00 a.m. ET today
NEW HAVEN, Conn., March 12, 2024 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience and immuno-oncology, today provided an update on its late-stage TRANQUILITY and SERENITY clinical programs. In addition, the Company reported its financial results for the fourth quarter and full year 2023.
“We believe our late-stage programs evaluating BXCL501 for acute agitation associated with Alzheimer’s dementia, bipolar disorders, and schizophrenia represent groundbreaking opportunities to bring much-needed treatment options to large numbers of patients and caregivers,” said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. “We are pleased to be advancing the clinical development paths for TRANQUILITY and SERENITY, and plan to focus our resources on these high-priority programs.”
TRANQUILITY Program: Evaluating 60 mcg dose of BXCL501 for agitation associated with Alzheimer’s dementia (AAD)
On February 20, 2024, the Company held a Type B/Breakthrough Therapy designation meeting with FDA. Based on the FDA’s feedback, the Company plans to generate additional Phase 3 efficacy and safety data in care facilities to expand the database beyond the 70 patients who have already been treated with 60 mcg of BXCL501 in TRANQUILITY I and II to date. The Company plans to generate these data in a variety of relevant care settings and across severity of dementia using the Positive and Negative Syndrome Scale-Excitatory Component (PEC) as the primary efficacy measure, as used in the prior TRANQUILITY II study. The Company announced in November 2023 that it was planning to conduct a Phase 3 trial in the at-home setting, with safety as the primary objective (TRANQUILITY At Home). Given the priority to expand the database to generate additional efficacy data in care facilities, the Company is re-evaluating the timing for initiating TRANQUILITY At Home. Given no prior regulatory precedent for episodic treatment of agitation in AD patients, the Company plans to engage with the FDA regarding the requirement for collection of long-term safety data. The Company expects to provide further guidance regarding program plans following receipt of final meeting minutes from FDA. SERENITY Program: Evaluating potential at-home use of 120 mcg of BXCL501 for agitation associated with bipolar disorders or schizophrenia
On March 6, 2024, the Company held a Type C meeting with FDA. Based on FDA feedback, Company plans to conduct a Phase 3 trial by amending the SERENITY III protocol to evaluate the 120 mcg dose of BXCL501 (an approved dose of IGALMI™) in the at-home setting, with safety as the primary objective and efficacy measures as exploratory endpoints. The Company expects to provide further guidance regarding program plans following receipt of final meeting minutes from FDA. Corporate Updates
IGALMI™ Commercialization
Permanent J-Code for IGALMI™ (dexmedetomidine) sublingual film became effective Jan. 1, 2024; code helps to standardize the reimbursement process, simplify claims submission, and in turn streamline billing and reimbursement for customers. Dedicated Corporate Account Director (CAD) team has secured and continues to secure volume contracts, driving broader access to IGALMI™. Patent Portfolio
The Company continues to develop a broad global intellectual property portfolio, with over 100 patent applications in prosecution and multiple patents issued as of February 29, 2024.
Neuroscience franchise (BXCL501 and pipeline): Company’s patent portfolio includes 10 issued U.S. patents, with eight listed in the FDA's Approved Drug Products with Therapeutic Equivalence Evaluations (commonly known as the Orange Book), as well as three patents from Japan and 11 patents from other countries. Additionally, the portfolio has 19 utility patent applications and seven provisional applications in the U.S. and 116 utility patent applications in other countries, as well as two Patent Cooperation Treaty (PCT) applications not yet in the national phase. The expiry dates for the Orange Book listed patents covering Igalmi in the U.S. run from December 29, 2037 to January 12, 2043. Immuno-oncology franchise (BXCL701 and pipeline): Company’s patent portfolio includes two issued patents in the U.S., one in Japan, and 12 in other countries, as well as one provisional application and six utility patent applications in the U.S., and 31 utility patent applications in other countries. OnkosXcel Therapeutics
FDA designated as a Fast Track development program the investigation of BXCL701 in combination with a checkpoint inhibitor for treatment of patients with metastatic small cell neuroendocrine prostate cancer (SCNC) with progression on chemotherapy and no evidence of microsatellite instability. Reported completion of patient enrollment in safety portion of Phase 2 relapsed pancreatic cancer trial of BXCL701 in combination with KEYTRUDA® sponsored by Georgetown Lombardi Comprehensive Cancer Center. Initiated formal process to assess strategic options. Fourth Quarter and Full Year 2023 Financial Results
Net Revenue: Net revenue from IGALMI was $376,000 for the fourth quarter of 2023, compared to $238,000 for the same period in 2022.
Net revenue from IGALMI was $1.4 million for the full year of 2023, compared to $375,000 for 2022.
Research and Development (R&D) Expenses: R&D expenses were $9.9 million for the fourth quarter of 2023, compared to $32.5 million for the same period in 2022.
R&D expenses were $84.3 million for the full year of 2023, compared to $91.2 million for the full year of 2022. The decreased expenses for both the fourth quarter and the full year were primarily attributable to a decrease in clinical trial activity associated with the wind down of the SERENITY III and TRANQUILITY II studies, a decrease in chemical, manufacturing, and control (CMC) costs, and a decrease in personnel related to the Reprioritization.
Selling, General and Administrative (SG&A) Expenses : SG&A expenses were $9.6 million for the fourth quarter of 2023, compared to $20.7 million for the same period in 2022.
SG&A expenses were $83.4 million for the full year 2023, compared to $68.8 million for 2022. The increased costs for the full year were primarily attributable to an increase in legal and professional fees, costs associated with increased personnel, and related costs to support the commercialization of IGALMI in the U.S. prior to the Reprioritization.
Net Loss: BioXcel Therapeutics had a net loss of $22.3 million for the fourth quarter of 2023, compared to a net loss of $54.8 million for the same period in 2022. For the full year of 2023, BioXcel Therapeutics reported a net loss of $179.0 million , compared to a net loss of $165.8 million for the full year of 2022. The loss for the 2023 year includes approximately $18.6 million in non-cash stock-based compensation. Total cash expenditures for 2023 totaled approximately $155.0 million .
Cash and cash equivalents totaled $65.2 million on December 31, 2023, compared to $193.7 million on December 31, 2022. The Company estimates that its current cash and cash equivalents will fund its operations through mid-2024. This estimated cash runway does not include potential additional capital that may become available under the amendments to the strategic financing agreements or resulting from any potential financing activities that may be undertaken by the Company.
Conference Call and Webcast bioxceltherapeutics.com, and a replay will be available through June 12, 2024.
BioXcel Therapeutics may use its website as a distribution channel of material information about the Company. Financial and other important information regarding the Company is routinely posted on and accessible through the Investors sections of its website at www.bioxceltherapeutics.com . In addition, you may sign up to automatically receive email alerts and other information about the Company by visiting the “Email Alerts” option under the News/Events section of the Investors & Media website section and submitting your email address.
About IGALMI™ (dexmedetomidine) sublingual film
INDICATION
IGALMI™ (dexmedetomidine) sublingual film is a prescription medicine, administered under the supervision of a health care provider, that is placed under the tongue or behind the lower lip and is used for the acute treatment of agitation associated with schizophrenia and bipolar disorder I or II in adults. The safety and effectiveness of IGALMI has not been studied beyond 24 hours from the first dose. It is not known if IGALMI is safe and effective in children.
IMPORTANT SAFETY INFORMATION
IGALMI can cause serious side effects, including:
Decreased blood pressure, low blood pressure upon standing, and slower than normal heart rate, which may be more likely in patients with low blood volume, diabetes, chronic high blood pressure, and older patients. IGALMI is taken under the supervision of a healthcare provider who will monitor vital signs (like blood pressure and heart rate) and alertness after IGALMI is administered to help prevent falling or fainting. Patients should be adequately hydrated and sit or lie down after taking IGALMI and instructed to tell their healthcare provider if they feel dizzy, lightheaded, or faint.Heart rhythm changes (QT interval prolongation) . IGALMI should not be given to patients with an abnormal heart rhythm, a history of an irregular heartbeat, slow heart rate, low potassium, low magnesium, or taking other drugs that could affect heart rhythm. Taking IGALMI with a history of abnormal heart rhythm can increase the risk of torsades de pointes and sudden death. Patients should be instructed to tell their healthcare provider immediately if they feel faint or have heart palpitations.Sleepiness/drowsiness . Patients should not perform activities requiring mental alertness, such as driving or operating hazardous machinery, for at least 8 hours after taking IGALMI.Withdrawal reactions, tolerance, and decreased response/efficacy . IGALMI was not studied for longer than 24 hours after the first dose. Physical dependence, withdrawal symptoms (e.g., nausea, vomiting, agitation), and decreased response to IGALMI may occur if IGALMI is used longer than 24 hours.The most common side effects of IGALMI in clinical studies were sleepiness or drowsiness, a prickling or tingling sensation or numbness of the mouth, dizziness, dry mouth, low blood pressure, and low blood pressure upon standing.
These are not all the possible side effects of IGALMI. Patients should speak with their healthcare provider for medical advice about side effects.
Patients should tell their healthcare provider about their medical history , including if they suffer from any known heart problems, low potassium, low magnesium, low blood pressure, low heart rate, diabetes, high blood pressure, history of fainting, or liver impairment. They should also tell their healthcare provider if they are pregnant or breastfeeding or take any medicines, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Patients should especially tell their healthcare provider if they take any drugs that lower blood pressure, change heart rate, or take anesthetics, sedatives, hypnotics, and opioids.
Everyone is encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You can also contact BioXcel Therapeutics, Inc. at 1-833-201- 1088 or medinfo@bioxceltherapeutics.com.
Please see full Prescribing Information at igalmi.com .
About BXCL501
About the Positive and Negative Syndrome Scale-Excitatory Component Score (PEC or PANSS-EC) The PEC total score is a validated endpoint for use in clinical research to quantify the severity of a patient’s acute agitation. The PEC rating evaluates 5 elements associated with agitation: poor impulse control, tension, hostility, uncooperativeness, and excitement; each scored 1 (minimum) to 7 (maximum). The PEC total score is the sum of these 5 elements and thus ranges from 5 to 35.
About BioXcel Therapeutics, Inc. bioxceltherapeutics.com .
Forward-Looking Statements
Contact Information
Corporate ekopp@bioxceltherapeutics.com
Investor Relations bdoyle@bioxceltherapeutics.com
Media David.schull@russopartnersllc.com Scott.stachowiak@russopartnersllc.com
Source: BioXcel Therapeutics, Inc.
IGALMI™ is a trademark of BioXcel Therapeutics, Inc.
BioXcel Therapeutics, Inc. Statements of Operations (Unaudited, in thousands, except per share amounts) Three months ended December 31, Year ended December 31, 2023 2022 2023 2022 Revenues Product revenues $ 376 $ 238 $ 1,380 $ 375 Operating expenses Cost of goods sold 714 9 1,260 20 Research and development 9,934 32,459 84,326 91,239 Selling, general and administrative 9,603 20,664 83,413 68,761 Restructuring costs - - 4,163 - Total operating expenses 20,251 53,132 173,162 160,020 Loss from operations (19,875 ) (52,894 ) (171,782 ) (159,645 ) Other (income) expense Interest expense, net 3,435 2,921 13,314 8,213 Interest income (946 ) (1,487 ) (5,649 ) (2,528 ) Other (income) expense, net (108 ) 480 (394 ) 427 Net loss and comprehensive loss $ (22,256 ) $ (54,808 ) $ (179,053 ) $ (165,757 ) Net loss per share - basic and diluted $ (0.76 ) $ (1.95 ) $ (6.15 ) $ (5.92 ) Weighted average shares outstanding - basic and diluted 29,449 28,068 29,129 28,015 Condensed Balance Sheets (Unaudited, in thousands) December 31, December 31, 2023 2022 Cash and cash equivalents $ 65,221 $ 193,725 Total assets $ 73,702 $ 205,853 Total liabilities $ 130,210 $ 129,078 Total stockholders' equity (deficit) $ (56,508 ) $ 76,775
What are the late-stage clinical programs that BioXcel Therapeutics provided updates on?
BioXcel Therapeutics provided updates on the late-stage TRANQUILITY and SERENITY clinical programs for BXCL501.
What is the focus of the TRANQUILITY program?
The TRANQUILITY program focuses on evaluating the 60 mcg dose of BXCL501 for agitation associated with Alzheimer’s dementia.
What dose is being evaluated in the SERENITY program?
The SERENITY program is evaluating the potential at-home use of 120 mcg of BXCL501 for agitation associated with bipolar disorders or schizophrenia.
What financial results were reported by BioXcel Therapeutics for the fourth quarter and full year 2023?
BioXcel Therapeutics reported increased net revenue from IGALMI, reduced R&D and SG&A expenses, and a net loss for the fourth quarter and full year 2023.
How long does BioXcel Therapeutics estimate its current cash and cash equivalents will fund its operations?
BioXcel Therapeutics estimates its current cash and cash equivalents will fund its operations through mid-2024.
