Bipolar II disorder

Enrollment exceeds 60% of required number of patients in SERENITY At-Home pivotal Phase 3 safety trial evaluating 200 patients for acute treatment of agitation associated with bipolar disorders or schizophrenia Topline data expected in second half of 2025 intended to support potential sNDA submission for label expansion of IGALMI® in the at-home setting Strengthened cash position to advance BXCL501 program NEW HAVEN, Conn., March 27, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today reported its financial results for the fourth quarter and full year 2024. “We believe that our SERENITY program presents an exciting opportunity to address a substantial unmet medical need — the 23 million episodes of bipolar and schizophrenia-related agitation that occur annually in the United States at home1-3 — and expand the market potential for our lead neuroscience asset BXCL501,” said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. “We are pleased that patient enrollment in our SERENITY At-Home pivotal Phase 3 trial is progressing well and that we have recently strengthened our cash position to further advance this important study.” BXCL501 Late-Stage Clinical Programs SERENITY Program SERENITY At-Home Phase 3 Trial: designed as a double-blind, placebo-controlled study to evaluate the safety of a 120 mcg dose of BXCL501 in 200 patients for acute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home setting. 24 clinical trial sites have been opened.127 patients have been enrolled, representing 63% of the required enrollment.A Data Safety Monitoring Board (DSMB) is planned to assess safety.Topline data expected in the second half of 2025. TRANQUILITY Program TRANQUILITY In-Care Phase 3 Trial: designed as a double-blind, placebo-controlled study to evaluate the efficacy and safety of a 60 mcg dose of BXCL501 for acute treatment of agitation associated with Alzheimer’s dementia in the care setting. Developed plans following receipt of FDA feedback on trial protocol. TRANQUILITY II Phase 3 Trial Site Inspection Closed by FDA The Company believes the reliability of data from its TRANQUILITY II Phase 3 trial of BXCL501 is further supported by the FDA closure of its site inspection under 21 C.F.R.20.64(d)(3) and release of the Establishment Inspection Report designating “Voluntary Action Indicated” for the site. IGALMI® Market Presence The Company is continuing to supply IGALMI® (dexmedetomidine) sublingual film to current and future patients and providers through existing distribution channels, with minimal commercial support. Equity Financing Closed $14 million equity financing and strengthened cash position to approximately $35 million, as of March 4, 2025, to advance SERENITY program. Fourth Quarter and Full Year 2024 Financial Results Net revenue from IGALMI® was $366,000 for the fourth quarter of 2024, compared to $376,000 for the same period in 2023. Net revenue from IGALMI® was $2.3 million for the full year of 2024, compared to $1.4 million for 2023. Cost of Goods Sold for the three months ended December 31, 2024 and 2023, were $832,000 and $714,000, respectively. Cost of goods sold is related to the costs to produce, package, and deliver IGALMI to customers, as well as costs related to excess or obsolete inventory. The increase in Cost of goods sold for the three months ended December 31, 2024 is the result of higher charges for reserves for excess or obsolete inventory compared to the same period in 2023. Charges for reserves for excess or obsolete inventory were $778,000 and $696,000 in the three months ended December 31, 2024 and 2023, respectively. Cost of Goods Sold was $2.1 million for the full year of 2024, compared to $1.3 million for 2023. The increase in Cost of goods sold is the result of higher charges for reserves for excess or obsolete inventory in 2024 compared to 2023. Charges for reserves for excess or obsolete inventory were $2.0 million and $1.2 million for 2024 and 2023, respectively. Research and Development (R&D) expenses were $5.9 million for the fourth quarter of 2024, compared to $9.9 million for the same period in 2023. R&D expenses were $30.4 million for the full year of 2024, compared to $84.3 million for the full year of 2023. The decreased expenses for both the fourth quarter and the full year were primarily attributable to a decrease in clinical trial activity associated with previously completed Phase 3 studies, a decrease in chemical, manufacturing, and control (CMC) costs, and a decrease in personnel related to the company’s reprioritization. Selling, General and Administrative (SG&A) expenses were $4.1 million for the fourth quarter of 2024, compared to $9.6 million for the same period in 2023. SG&A expenses were $34.5 million for the full year 2024, compared to $83.4 million for 2023. The decreased costs for the fourth quarter and the full year 2024 were primarily attributable to a decrease in personnel and related costs, lower non-cash stock compensation costs, lower legal and professional fees, and lower commercial and marketing costs resulting from restructuring actions taken in 2024 and 2023. Net Loss: BioXcel Therapeutics had a net loss of $10.9 million for the fourth quarter of 2024, compared to a net loss of $22.3 million for the same period in 2023. For the full year of 2024, BioXcel Therapeutics reported a net loss of $59.6 million, compared to a net loss of $179.1 million for the full year of 2023. The loss for the 2024 year includes approximately $6.2 million in non-cash stock-based compensation. Total cash used in operating activities for 2024 totaled approximately $72.0 million, down $83.0 million from 2023 cash used in operating activities of approximately $155.0 million. Cash and cash equivalents totaled $29.9 million on December 31, 2024, compared to $65.2 million on December 31, 2023. About BXCL501Outside of its approved indication by the U.S. Food and Drug Administration as IGALMI® (dexmedetomidine) sublingual film, BXCL501 is an investigational proprietary, orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. BXCL501 is under investigation by BioXcel Therapeutics for the acute treatment of agitation associated with Alzheimer’s dementia and for the acute treatment of agitation associated with bipolar I or II disorder or schizophrenia in the at-home setting. The safety and efficacy of BXCL501 for these investigational uses have not been established. BXCL501 has been granted Breakthrough Therapy designation by the FDA for the acute treatment of agitation associated with dementia and Fast Track designation for the acute treatment of agitation associated with schizophrenia, bipolar disorders, and dementia. About the SERENITY At-Home Phase 3 TrialThe SERENITY At-Home Phase 3 trial is a double-blind, placebo-controlled study designed to evaluate the safety of a 120 mcg dose of BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home setting. The trial is evaluating 200 patients with a history of agitation episodes residing at home either alone or with caregivers/informants. Patients will self-administer 120 mcg of BXCL501 or placebo when agitation episodes occur over the 12-week trial period, with safety data (adverse events) collected during the trial. Patients or caregivers/informants will complete a modified global impression of severity (mCGIs) and a clinical global impression of change (mCGI-C) two hours after dosing as an exploratory endpoint to evaluate use in the outpatient setting. About the TRANQUILITY In-Care Phase 3 TrialThe TRANQUILITY In-Care trial is designed as a double-blind, placebo-controlled study to evaluate the efficacy and safety of a 60 mcg dose of BXCL501 over a 12-week period for agitation associated with Alzheimer’s dementia in the care setting. The trial is expected to enroll approximately 150 patients 55 years and older who have mild, moderate, or severe dementia with mini-mental state examination scores of 0 to 25 and who reside in skilled nursing facilities, memory care units, or assisted living facilities. Patients will self-administer 60 mcg of BXCL501 or placebo when episodic agitation episodes occur over the trial period. The primary endpoint is expected to be a change from baseline in the Positive and Negative Syndrome Scale-Excitatory Component (PEC) total score at two hours post-first dose. Additional PEC and Clinical Global Impressions – Improvement Scale measurements will also be obtained during the trial. 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 BioXcel Therapeutics, Inc.BioXcel Therapeutics, Inc. (Nasdaq: BTAI) is a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience. Its wholly owned subsidiary, OnkosXcel Therapeutics, is focused on the development of medicines in immuno-oncology. The Company’s drug re-innovation approach leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indications. For more information, please visit bioxceltherapeutics.com. Forward-Looking StatementsThis press release includes “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended and Section 21E of the Securities Exchange Act of 1934, as amended. All statements contained in this press release other than statements of historical fact should be considered forward-looking statements, including, without limitation, statements related to: the Company’s planned advancement of its TRANQUILITY and SERENITY trials and the trial designs thereof; potential market opportunity for BXCL501; the DSMB meeting for the ongoing SERENITY trial; the supply of IGALMI through existing distribution channels; the potential for the results from the Company’s completed, ongoing and proposed clinical trials to support regulatory approvals for its product candidates; the Company’s current patent applications and potential Orange Book listings. When used herein, words including “anticipate,” “believe,” “can,” “continue,” “could,” “designed,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, though not all forward-looking statements use these words or expressions. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon the Company’s current expectations and various assumptions. The Company believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. The Company may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation: its limited operating history; its incurrence of significant losses; its need for substantial additional funding and ability to raise capital when needed; the impact of the reprioritization; its significant indebtedness, ability to comply with covenant obligations and potential payment obligations related to such indebtedness and other contractual obligations; the Company has identified conditions and events that raise substantial doubt about its ability to continue as a going concern; its limited experience in drug discovery and drug development; risks related to the TRANQUILITY program; its dependence on the success and commercialization of IGALMI®, BXCL501, BXCL502, BXCL701 and BXCL702 and other product candidates; the number of episodes of agitation and the size of the Company’s total addressable market may be overestimated, and approval that the Company may obtain may be based on a narrower definition of the patient population; its lack of experience in marketing and selling drug products; the risk that IGALMI or the Company’s product candidates may not be accepted by physicians or the medical community in general; the Company still faces extensive and ongoing regulatory requirements and obligations for IGALMI; the failure of preliminary data from its clinical studies to predict final study results; failure of its early clinical studies or preclinical studies to predict future clinical studies; its ability to receive regulatory approval for its product candidates; its ability to enroll patients in its clinical trials; undesirable side effects caused by the Company’s product candidates; its novel approach to the discovery and development of product candidates based on EvolverAI; the significant influence of and dependence on BioXcel LLC; its exposure to patent infringement lawsuits; its reliance on third parties; its ability to comply with the extensive regulations applicable to it; impacts from data breaches or cyber-attacks, if any; risks associated with the increased scrutiny relating to environmental, social and governance (ESG) matters; risks associated with federal, state or foreign health care “fraud and abuse” laws; and its ability to commercialize its product candidates, as well as the important factors discussed under the caption “Risk Factors” in its Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2024, as such factors may be updated from time to time in its other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov and the Investors section of the Company’s website at www.bioxceltherapeutics.com. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While the Company may elect to update such forward-looking statements at some point in the future, except as required by law, it disclaims any obligation to do so, even if subsequent events cause our views to change. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this press release. Contact Information Corporate/InvestorsBioXcel TherapeuticsErik Kopp1.203.494.7062 MediaRusso PartnersDavid Schull 1.858.717.2310 Source: BioXcel Therapeutics, Inc.IGALMI® is a registered trademark of BioXcel Therapeutics, Inc. References Data on file relating to agitation episodes associated with schizophrenia or bipolar I or II disorder. BioXcel Therapeutics, Inc. New Haven, CT December 2020. Episode estimations may not reflect potential treatable episodes, and actual addressable market may be smaller.Data from Wu EQ, Shi L, Birnbaum H, et al. Annual prevalence of diagnosed schizophrenia in the USA: a claims data analysis approach. Psychol Med. 2006;36(11):1535-1540. Estimates based on whether indications are approved for at-home use for the intended patient population and such patients are treatable. Episode estimations may not reflect potential treatable episodes, and actual addressable market may be smaller.National Institute of Mental Health. Prevalence of bipolar disorder in adults. November 2017. Accessed December 16, 2022. https://www.nimh.nih.gov/health/statistics/bipolar-disorder. Episode estimations may not reflect potential treatable episodes, and actual addressable market may be smaller. BioXcel Therapeutics, Inc. Statements of Operations (Unaudited, in thousands, except per share amounts) Three months ended December 31, For the year ended December 31, 2024 2023 2024 2023 Revenues Product revenues $366 $376 $2,266 $1,380 Operating expenses Cost of goods sold 832 714 2,143 1,260 Research and development 5,901 9,934 30,435 84,326 Selling, general and administrative 4,094 9,603 34,492 83,413 Restructuring costs 32 - 2,441 4,163 Total operating expenses 10,859 20,251 69,511 173,162 Loss from operations (10,493) (19,875) (67,245) (171,782)Other (income) expense Interest expense, net 4,032 3,435 15,129 13,314 Interest income (368) (946) (2,602) (5,649)Other (income) expense, net (3,298) (108) (20,173) (394)Net loss and comprehensive loss $(10,859) $(22,256) $(59,599) $(179,053) Net loss per share - basic and diluted $(3.57) $(12.09) $(23.51) $(98.33)Weighted average shares outstanding - basic and diluted 3,039 1,841 2,535 1,821 Condensed Balance Sheets (Unaudited, in thousands) December 31, December 31, 2024 2023 Cash and cash equivalents $29,854 $65,221 Total assets $38,338 $73,702 Total liabilities $131,439 $130,210 Total stockholders' equity (deficit) $(93,101) $(56,508)

Topline data expected in second half of 2025 to support potential sNDA submission for label expansion of IGALMI® in the home setting Company has $35M of cash following closing of recent equity financing NEW HAVEN, Conn., March 11, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced that, following the successful raise of $14 million gross proceeds in an equity financing that closed on March 4, 2025, it has approximately $35 million in cash. The strengthened cash position will support continued advancement of the Company’s pivotal Phase 3 SERENITY At-Home trial. The trial is designed to evaluate the safety of BXCL501, BioXcel Therapeutics’ investigational, proprietary, orally dissolving film formulation of dexmedetomidine, in the at-home setting for the acute treatment of agitation associated with bipolar disorders or schizophrenia. “We are pleased the recent financing enables us to progress our SERENITY At-Home trial,” said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. “Patient enrollment is continuing and topline data results expected in the second half of 2025 are intended to support a potential sNDA submission to expand the label for IGALMI® in the at-home setting. We are highly motivated by the prospect of bringing — for the first time — an acute treatment option for agitation to millions of additional patients in this setting.” About the SERENITY At-Home Phase 3 Trial The SERENITY At-Home Phase 3 trial is a double-blind, placebo-controlled study designed to evaluate the safety of a 120 mcg dose of BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia in the home setting. The trial is enrolling 200 patients with a history of agitation episodes residing at home either alone or with caregivers/informants. Patients will self-administer 120 mcg of BXCL501 or placebo when agitation episodes occur over the 12-week trial period, with safety data (adverse events) collected during the trial. Patients or caregivers/informants will complete a modified global impression of severity (mCGI-S) and a clinical global impression of change (mCGI-C) two hours after dosing as an exploratory endpoint to evaluate use in the outpatient setting. 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. About BXCL501Outside of its approved indication by the U.S. Food and Drug Administration as IGALMI® (dexmedetomidine) sublingual film, BXCL501 is an investigational proprietary, orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. BXCL501 is under investigation by BioXcel Therapeutics for the acute treatment of agitation associated with Alzheimer’s dementia and for the acute treatment of agitation associated with bipolar I or II disorder or schizophrenia in the at-home setting. The safety and efficacy of BXCL501 for these investigational uses have not been established. BXCL501 has been granted Breakthrough Therapy designation by the FDA for the acute treatment of agitation associated with dementia and Fast Track designation for the acute treatment of agitation associated with schizophrenia, bipolar disorders, and dementia. About BioXcel Therapeutics, Inc.BioXcel Therapeutics, Inc. (Nasdaq: BTAI) is a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience. Its wholly owned subsidiary, OnkosXcel Therapeutics, is focused on the development of medicines in immuno-oncology. The Company’s drug re-innovation approach leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indications. For more information, please visit bioxceltherapeutics.com. Forward-Looking Statements This press release includes “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended and Section 21E of the Securities Exchange Act of 1934, as amended. All statements contained in this press release other than statements of historical fact should be considered forward-looking statements, including, without limitation, statements related to: the Company’s planned advancement of its SERENITY program; the expected submission of an sNDA to expand the label for IGALMI® in the home setting; the expansion of IGALMI® market potential; bringing IGALMI® as a treatment option to additional patients in the home setting; ; the potential to bring new treatment options to patients, and statements regarding the Company’s operational progress. When used herein, words including “anticipate,” “believe,” “can,” “continue,” “could,” “designed,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, though not all forward-looking statements use these words or expressions. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon the Company’s current expectations and various assumptions. The Company believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. The Company may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation: its limited operating history; its incurrence of significant losses; its need for substantial additional funding and ability to raise capital when needed; the impact of the reprioritization; its significant indebtedness, ability to comply with covenant obligations and potential payment obligations related to such indebtedness and other contractual obligations; the Company has identified conditions and events that raise substantial doubt about its ability to continue as a going concern; its limited experience in drug discovery and drug development; risks related to the TRANQUILITY and SERENITY programs; its dependence on the success and commercialization of IGALMI®, BXCL501, BXCL502, BXCL701 and BXCL702 and other product candidates; the number of episodes of agitation and the size of the Company’s total addressable market may be overestimated, and approval that the Company may obtain may be based on a narrower definition of the patient population; its lack of experience in marketing and selling drug products; the risk that IGALMI or the Company’s product candidates may not be accepted by physicians or the medical community in general; the Company still faces extensive and ongoing regulatory requirements and obligations for IGALMI; the failure of preliminary data from its clinical studies to predict final study results; failure of its early clinical studies or preclinical studies to predict future clinical studies; its ability to receive regulatory approval for its product candidates; its ability to enroll patients in its clinical trials; undesirable side effects caused by the Company’s product candidates; its novel approach to the discovery and development of product candidates based on EvolverAI; the significant influence of and dependence on BioXcel LLC; its exposure to patent infringement lawsuits; its reliance on third parties; its ability to comply with the extensive regulations applicable to it; impacts from data breaches or cyber-attacks, if any; risks associated with the increased scrutiny relating to environmental, social and governance (ESG) matters; risks associated with federal, state or foreign health care “fraud and abuse” laws; and its ability to commercialize its product candidates, as well as the important factors discussed under the caption “Risk Factors” in its Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2024, as such factors may be updated from time to time in its other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov and the Investors section of the Company’s website at www.bioxceltherapeutics.com. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While the Company may elect to update such forward-looking statements at some point in the future, except as required by law, it disclaims any obligation to do so, even if subsequent events cause our views to change. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this press release. Contact Information Corporate/InvestorsBioXcel TherapeuticsErik Kopp1.203.494.7062 MediaRusso PartnersDavid Schull 1.858.717.2310 Source: BioXcel Therapeutics, Inc. IGALMI® is a registered trademark of BioXcel Therapeutics, Inc.

Phase 2b clinical trial of GH001 in patients with treatment-resistant depression on track for completion of last patient visit in the open-label extension in Q1 2025 Phase 1 clinical trial to evaluate proprietary aerosol delivery device in healthy volunteers is ongoing in the UK Full response to the IND hold on track for submission in mid-2025 Cash, cash equivalents, other financial assets and marketable securities of $182.6 million as of December 31, 2024 Net cash proceeds of an additional $139.8 million from public offering received in February 2025 DUBLIN, Feb. 27, 2025 (GLOBE NEWSWIRE) -- GH Research PLC (Nasdaq: GHRS), a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients by developing a practice-changing treatment in depression, today reported financial results for the year ended December 31, 2024, and provided updates on its business. Business Updates GH001 in Patients with TRD GH001, our proprietary inhalable mebufotenin product candidate, is currently being investigated in a multi-center, randomized, double-blind, placebo-controlled Phase 2b trial in approximately 80 patients with treatment-resistant depression (TRD) (GH001-TRD-201). We recently announced that the trial met its primary endpoint with a significant placebo-adjusted Montgomery-Åsberg Depression Rating Scale (MADRS) reduction from baseline of -15.5 on Day 8 (p<0.0001). The majority of the patients treated with GH001 achieved remission (MADRS≤10) with a 57.5% remission rate on Day 8 compared with 0% in the placebo group (p<0.0001). All other secondary endpoints were met with clinically and statistically significant improvements on Day 8, compared with placebo. During the double-blind part, GH001 was well tolerated and no serious adverse events (SAE) were reported. There was no evidence of treatment-emergent suicidal ideation or behavior. Safety analysis has not yet been completed for the open label extension (OLE) as it remains ongoing, but as of January 22, 2025, no SAEs have been reported throughout the OLE. As of January 22, 2025, 77.8% of the OLE completers were in remission at the 6-month visit, with infrequent treatments. Patients who had remission on Day 8 after their first active treatment had a 91.7% remission rate at 6 months. The OLE is on track for completion of last patient visit in the open-label extension in Q1 2025. GH001 Administered with Proprietary Aerosol Delivery Device Our Phase 1 clinical pharmacology trial to evaluate our proprietary aerosol delivery device for administration of GH001 in healthy volunteers (GH001-HV-106) is ongoing in the United Kingdom. This trial is designed to support our global program for GH001, by bridging to the clinical data generated with the commercially available device that we have used in our clinical trials to date. Update on IND for GH001 As previously announced, our Investigational New Drug Application (IND) for GH001 administered using our proprietary aerosol delivery device has been placed on clinical hold by the U.S. Food and Drug Administration (FDA), with the FDA requesting that we provide (i) an inhalation toxicology study in a non-rodent species and an additional inhalation toxicology study in rats, (ii) additional device design verification information and (iii) updates to our investigator brochure, to resolve the hold. We have recently announced the completion of all FDA requests to address IND hold. The requested additional inhalation toxicology study in a non-rodent species has now been completed. The pathology report concludes that there are no histology findings in the respiratory tract of any dogs at any dose level evaluated in the study. The requested additional inhalation toxicology study in rats has now been completed which showed histology findings consistent with our previously completed study in rats. This supports our position that these findings are rat specific. Based on previously announced FDA interactions, the response to their request for additional device design verification information is being prepared and, together with the completion of the inhalation toxicology studies, provides the final piece of information requested by the agency. We are preparing to engage with the FDA in advance of providing a full response to the IND hold which we are on track to submit in mid-2025. Proof-of-Concept Trials with GH001 We have recently announced the completion of and results from two Phase 2a proof-of-concept (POC) trials with GH001 in postpartum depression (PPD) and bipolar II disorder with a current major depressive episode (BDII). The primary endpoint of the Phase 2a POC trial for GH001 in PPD was met with a significant reduction from baseline of –35.4 points (96.3%) in MADRS total score on Day 8 after administration of GH001 (p<0.0001, n=10). On Day 8, 100% of patients were in remission (MADRS ≤ 10). GH001 was well tolerated and no treatment-related serious adverse events were reported. All treatment-emergent adverse events (TEAEs) were mild or moderate. The primary endpoint of the Phase 2a POC trial for GH001 in BDII was met with a significant reduction from baseline of –16.8 points (51.9%) in MADRS total score on Day 8 after administration of GH001 (p=0.0099, n=6). On Day 8, 33.3% of patients were in remission (MADRS ≤ 10). GH001 was well tolerated and no treatment-related serious adverse events were reported. The majority of TEAEs were mild or moderate and there were no reported TEAEs of hypomania or mania. Full Year 2024 Financial Highlights Cash position Cash, cash equivalents, other financial assets and marketable securities were $182.6 million as of December 31, 2024, compared to cash, cash equivalents, other financial assets and marketable securities of $222.7 million as of December 31, 2023. Other financial assets are comprised of money market funds, and marketable securities are comprised of investment grade bonds. Research and development expenses R&D expenses were $35.0 million for the year ended December 31, 2024, compared to $29.8 million for the full year 2023. The increase is primarily due to increased expenses relating to clinical development activities including clinical trial and non-clinical activities. These increases have been partly offset by the recognition of a research and development tax credit and a decrease in technical development expenses. Employee expenses also increased primarily due to the hiring of personnel to support our research and development activities. General and administrative expenses G&A expenses were $15.3 million for the year ended December 31, 2024, compared to $11.4 million for the full year 2023. The increase was primarily due to higher professional fees, which has been partly offset by a decrease in insurance costs. Employee expenses increased due to an increase in headcount to support our growth initiatives. Net loss Net loss was $39.0 million, or $0.75 loss per share, for the year ended December 31, 2024, compared to $35.6 million, or $0.68 loss per share, for the full year 2023. About GH Research PLC GH Research PLC is a clinical-stage biopharmaceutical company dedicated to transforming the treatment of psychiatric and neurological disorders. GH Research PLC's initial focus is on developing its novel and proprietary mebufotenin therapies for the treatment of patients with treatment-resistant depression (TRD). About GH001 Our lead product candidate, GH001, is formulated for mebufotenin administration via a proprietary inhalation approach. Based on the observed clinical activity in our Phase 2b GH001-TRD-201 trial, where the primary endpoint was met with a MADRS reduction from baseline of -15.5 points compared with placebo on Day 8 (p<0.0001), we believe that GH001 has potential to change the way TRD is treated today. About GH002 GH002 is our mebufotenin product candidate formulated for administration via a proprietary intravenous approach. We have completed a Phase 1 trial of GH002 in healthy volunteers. Forward-Looking Statements This press release contains statements that are, or may be deemed to be, forward-looking statements. All statements other than statements of historical fact included in this press release, including statements regarding our future results of operations and financial position, business strategy, product candidates, medical devices required to deliver these product candidates, research pipeline, ongoing and currently planned preclinical studies and clinical trials, regulatory submissions and approvals and their effects on our business strategy, including our plans and expectations for discussions with the FDA and the outcomes and resolution of such discussions related to the clinical hold on the GH001 IND, research and development costs, cash runway, timing and likelihood of success, as well as plans and objectives of management for future operations, are forward-looking statements. Forward-looking statements appear in a number of places in this press release and include, but are not limited to, statements regarding our intent, belief or current expectations. Forward-looking statements are based on our management’s beliefs and assumptions and on information currently available to our management. Such statements are subject to risks and uncertainties, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various factors, including, but not limited to, those described in our filings with the U.S. Securities and Exchange Commission. No assurance can be given that such future results will be achieved. Such forward-looking statements contained in this press release speak only as of the date hereof. We expressly disclaim any obligation or undertaking to update these forward-looking statements contained in this press release to reflect any change in our expectations or any change in events, conditions, or circumstances on which such statements are based unless required to do so by applicable law. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Investor Relations: Julie Ryan GH Research PLC investors@ghres.com