BioXcel Therapeutics Announces Database Lock in SERENITY At-Home Pivotal Phase 3 Safety Trial for Acute Treatment of Agitation Associated with Bipolar Disorders or Schizophrenia
BioXcel Therapeutics (Nasdaq: BTAI) has announced the completion of database lock for its SERENITY At-Home pivotal Phase 3 safety trial, evaluating BXCL501 for acute treatment of agitation in bipolar disorders or schizophrenia patients. The study, conducted across 22 sites nationwide, enrolled over 200 patients and collected data from more than 2,600 agitation episodes.
The trial evaluated a 120 mcg dose of BXCL501 in a double-blind, placebo-controlled study. The company implemented robust oversight controls, including patient eligibility reviews and DSMB reviews. BXCL501 has received Fast Track Designation, addressing an unmet need as there are currently no FDA-approved therapies for acute agitation treatment in at-home settings. Topline results are expected in August.
BioXcel Therapeutics (Nasdaq: BTAI) ha annunciato il completamento del lock del database per il suo trial pivotale di Fase 3 SERENITY At-Home, che valuta BXCL501 per il trattamento acuto dell'agitazione in pazienti con disturbi bipolari o schizofrenia. Lo studio, condotto in 22 centri su tutto il territorio nazionale, ha arruolato oltre 200 pazienti e raccolto dati su pi霉 di 2.600 episodi di agitazione.
La sperimentazione ha testato una dose di 120 mcg di BXCL501 in uno studio in doppio cieco controllato con placebo. L'azienda ha adottato rigorosi controlli di supervisione, inclusi esami di idoneit脿 dei pazienti e revisioni da parte del DSMB. BXCL501 ha ottenuto la Fast Track Designation, rispondendo a un bisogno clinico non soddisfatto, dato che attualmente non esistono terapie approvate dalla FDA per il trattamento acuto dell'agitazione in ambito domiciliare. I risultati preliminari sono attesi per agosto.
BioXcel Therapeutics (Nasdaq: BTAI) ha anunciado el cierre de la base de datos del ensayo pivotal SERENITY At-Home de Fase 3, que eval煤a BXCL501 para el tratamiento agudo de la agitaci贸n en pacientes con trastornos bipolares o esquizofrenia. El estudio, realizado en 22 centros a nivel nacional, reclut贸 a m谩s de 200 pacientes y recopil贸 datos de m谩s de 2.600 episodios de agitaci贸n.
El ensayo evalu贸 una dosis de 120 mcg de BXCL501 en un estudio doble ciego controlado con placebo. La compa帽铆a implement贸 controles de supervisi贸n s贸lidos, incluidos revisiones de elegibilidad de los pacientes y evaluaciones del DSMB. BXCL501 ha recibido la Fast Track Designation, cubriendo una necesidad no satisfecha ya que actualmente no existen terapias aprobadas por la FDA para el tratamiento agudo de la agitaci贸n en el entorno domiciliario. Se esperan los resultados provisionales en agosto.
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鞁滍棙鞚� 鞚挫毵龟瞼 鞙勳暯雽�臁� 氚╈嫕鞙茧 120 mcg 鞖╇焿鞚� BXCL501鞚� 韽夑皜頄堨姷雼堧嫟. 須岇偓電� 頇橃瀽 鞝侁博靹� 瓴�韱犾檧 DSMB 瓴�韱� 霌� 觳犾爛頃� 臧愲弲 韱奠牅 鞛レ箻毳� 霃勳瀰頄堨姷雼堧嫟. BXCL501鞚� 韺姢韸疙姼霝� 歆�鞝�(Fast Track Designation)鞚� 氚涭晿鞙茧┌, 順勳灛 臧�鞝曥棎靹滌潣 旮夓劚 齑堨“ 旃橂毳� 鞙勴暅 FDA 鞀轨澑 旃橂鞝滉皜 鞐嗢柎 氙胳订臁� 靾橃殧毳� 頃搓舶頃╇媹雼�. 欤检殧 瓴瓣臣電� 8鞗旍棎 氚滍憸霅� 鞓堨爼鞛呺媹雼�.
BioXcel Therapeutics (Nasdaq: BTAI) a annonc茅 la cl么ture de la base de donn茅es de son essai pivot de phase 3 SERENITY At-Home, 茅valuant BXCL501 pour le traitement aigu de l'agitation chez des patients atteints de troubles bipolaires ou de schizophr茅nie. L'茅tude, men茅e sur 22 sites 脿 l'茅chelle nationale, a recrut茅 plus de 200 patients et collect茅 des donn茅es sur plus de 2 600 茅pisodes d'agitation.
L'essai a 茅valu茅 une dose de 120 mcg de BXCL501 dans le cadre d'une 茅tude randomis茅e en double aveugle versus placebo. La soci茅t茅 a mis en place des contr么les de supervision rigoureux, incluant des v茅rifications d'茅ligibilit茅 des patients et des revues du DSMB. BXCL501 a re莽u la Fast Track Designation, r茅pondant 脿 un besoin non couvert car il n'existe actuellement aucune th茅rapie approuv茅e par la FDA pour le traitement aigu de l'agitation 脿 domicile. Les r茅sultats principaux sont attendus en ao没t.
BioXcel Therapeutics (Nasdaq: BTAI) hat den Abschluss des Database Locks f眉r die SERENITY At-Home pivotalen Phase鈥�3鈥慡icherheitsstudie bekanntgegeben, die BXCL501 zur akutiven Behandlung von Agitation bei Patienten mit bipolaren St枚rungen oder Schizophrenie bewertet. Die Studie wurde an 22 Standorten landesweit durchgef眉hrt, schloss mehr als 200 Patienten ein und sammelte Daten zu 眉ber 2.600 Agitations鈥慐pisoden.
Untersucht wurde eine 120 mcg-Dosis von BXCL501 in einer doppelblinden, placebokontrollierten Studie. Das Unternehmen setzte strenge Aufsichtsma脽nahmen um, darunter 脺berpr眉fungen der Patientenberechtigung und DSMB鈥態egutachtungen. BXCL501 erhielt die Fast Track Designation und adressiert damit einen ungedeckten Bedarf, da derzeit keine von der FDA zugelassenen Therapien f眉r die akute Behandlung von Agitation im h盲uslichen Umfeld existieren. Topline鈥慐rgebnisse werden im August erwartet.
- BXCL501 received Fast Track Designation from FDA, potentially expediting approval process
- High patient retention with vast majority completing the full 12-week study
- Successful collection of extensive data from over 2,600 agitation episodes
- First-mover advantage in addressing unmet need with no current FDA-approved therapies
- Requires FDA approval with no guarantee of success
- Limited patient population with only 200 participants in the trial
Insights
BioXcel's database lock for BXCL501 Phase 3 safety trial signals imminent results for a potential first-in-class at-home agitation treatment.
The database lock announcement for BioXcel's SERENITY At-Home pivotal Phase 3 safety trial represents a critical late-stage milestone in the development pathway for BXCL501. This procedural step essentially freezes the clinical trial database, preventing further modifications before statistical analysis begins 鈥� a necessary precursor to the topline data readout expected this month.
Several aspects of this trial deserve attention. The study collected data from over 2,600 agitation episodes across more than 200 patients, providing a substantial safety dataset. The trial design appears robust with careful site distribution (no site enrolled more than 11% of patients) and balanced recruitment between bipolar disorder and schizophrenia populations.
The company's implementation of multiple oversight mechanisms 鈥� including Data Safety Monitoring Board reviews, sponsor eligibility verification, and independent GCP compliance checks 鈥� suggests awareness of regulatory scrutiny that safety studies for psychiatric medications typically face. This methodical approach aligns with the expectations for a pivotal safety trial intended to support regulatory approval.
With BXCL501's Fast Track Designation and the stated absence of FDA-approved therapies for at-home treatment of acute agitation, BioXcel is positioning this as addressing a significant unmet need. If positive, these results could complete the safety package needed for FDA submission, potentially positioning BXCL501 as a first-in-class treatment for this indication in the outpatient setting.
The clinical significance of this trial extends beyond regulatory milestones. Acute agitation in bipolar disorder and schizophrenia represents a critical treatment gap in psychiatric care, particularly in non-institutional settings. When these episodes occur at home, patients and caregivers currently have limited options, often resulting in emergency department visits, involuntary hospitalizations, or suboptimal crisis management.
BXCL501's development for at-home use addresses a genuine clinical need. The 120 mcg dose being tested suggests the company is targeting an effective yet manageable dosage suitable for self or caregiver administration. The 12-week study duration with high completion rates ("vast majority of patients") is encouraging from a tolerability perspective, as dropout rates in psychiatric trials are typically substantial.
The balanced enrollment between bipolar disorder and schizophrenia populations is methodologically sound, as agitation presents differently across these conditions. While symptomatically similar, the underlying neurobiology can vary significantly, potentially affecting treatment response and safety profiles.
The collection of data from over 2,600 agitation episodes is particularly valuable, as it likely captures the heterogeneous nature of these events 鈥� varying in intensity, duration, and precipitating factors. This comprehensive dataset should provide insights into real-world effectiveness across different episode characteristics, though the press release doesn't specify the primary safety endpoints being measured.
Data from more than 2,600 agitation episodes collected
Topline data readout is on track for August听听听听听听听听
NEW HAVEN, Conn., Aug. 19, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical听company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced completion of the database lock for its SERENITY At-Home pivotal Phase 3 safety trial for acute treatment of agitation associated with bipolar disorders or schizophrenia. Topline results from the study are expected in August.
"The database lock is a significant step forward, and we are thrilled to have reached this critical milestone in an efficient manner as we look forward to reporting top-line results from the SERENITY At-Home pivotal Phase 3 safety trial soon," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "I want to extend our heartfelt gratitude to the clinical team, our patients, principal investigators and their study staff, and all our independent service providers for their dedication and collaborative efforts as we seek to make BXCL501 available to patients, as no FDA-approved therapies are available for the acute treatment of agitation in听the at-home听setting, which remains a significant need."
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 at-home setting:
- The trial enrolled more than 200 patients across 22 sites nationwide, with no single site enrolling more than
11% of the total patient population. - The distribution of enrolled patients was balanced between the two patient populations, bipolar disorders and schizophrenia.
- The vast majority of patients dosed completed the full 12-week study.
- Data from more than 2,600 agitation episodes was collected.
BioXcel partnered with Worldwide Clinical Trials (WCT), a recognized Clinical Research Organization (CRO), to conduct the Serenity At-Home trial. Robust sponsor oversight controls were implemented to听oversee the collaborators and patient safety, including patient eligibility reviews, two DSMB reviews during the peak recruitment period, and ensuring that no clinical site contributed an outsized percentage of the randomized patients. Independent industry experts were engaged by BioXcel for additional oversight of high enrolling sites for GCP compliance.
BXCL501 was granted Fast Track Designation for the acute treatment of agitation associated with bipolar disorders or schizophrenia.听There are no FDA-approved therapies for the acute treatment of agitation in听the at-home听setting.
About the SERENITY At-Home Phase 3 Trial
The trial was designed to study 200 patients with a history of agitation episodes despite being on stable treatment for their underlying bipolar or schizophrenia residing at home either alone or with caregivers/informants. Patients were required to self-administer 120 mcg of BXCL501 (the approved dose under medical supervision) or placebo when they experienced agitation episodes over the 12-week trial period, and their safety data (adverse events) was collected during the trial. In addition, patients or caregivers/informants completed 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 assess their experience in the outpatient setting.
About BXCL501
Outside of its approved indication by the U.S. Food and Drug Administration as IGALMI庐 (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鈥檚 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 IGALMI庐 (dexmedetomidine) sublingual film听听
听听
滨狈顿滨颁础罢滨翱狈听
IGALMI庐 (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.听
IMPORTANT SAFETY INFORMATION听
IGALMI can cause serious side effects, including:听
- 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.听
- 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.听
- 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.听
- 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.听
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.听
These are not all the possible side effects of IGALMI. Patients should speak with their healthcare provider for medical advice about side effects.听
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.听
Everyone is encouraged to report negative side effects of prescription drugs to the FDA. Visit鈥�痮r call 1-800-FDA-1088. You can also contact BioXcel Therapeutics, Inc. at 1-833-201- 1088 or鈥�.听
Please see full prescribing information at .
About听BioXcel听Therapeutics,听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鈥檚 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听.
Forward-Looking Statements
This press release includes 鈥渇orward-looking statements鈥� 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鈥檚 planned advancement of its TRANQUILITY and SERENITY trials and the trial designs thereof; potential market opportunity for BXCL501; release of topline data from the ongoing SERENITY trial; the submission of an sNDA to the FDA; the supply of IGALMI through existing distribution channels; the potential for the results from the Company鈥檚 completed, ongoing and proposed clinical trials to support regulatory approvals for its product candidates. When used herein, words including 鈥渁nticipate,鈥� 鈥渂elieve,鈥� 鈥渃an,鈥� 鈥渃ontinue,鈥� 鈥渃ould,鈥� 鈥渄esigned,鈥� 鈥渆stimate,鈥� 鈥渆xpect,鈥� 鈥渇orecast,鈥� 鈥済oal,鈥� 鈥渋ntend,鈥� 鈥渕ay,鈥� 鈥渕ight,鈥� 鈥減lan,鈥� 鈥減ossible,鈥� 鈥減otential,鈥� 鈥減redict,鈥� 鈥減roject,鈥� 鈥渟hould,鈥� 鈥渢arget,鈥� 鈥渨ill,鈥� 鈥渨ould鈥� 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鈥檚 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庐, BXCL501, BXCL502, BXCL701 and BXCL702 and other product candidates; the number of episodes of agitation and the size of the Company鈥檚 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鈥檚 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鈥檚 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 鈥渇raud and abuse鈥� laws; and its ability to commercialize its product candidates, as well as the important factors discussed under the caption 鈥淩isk Factors鈥� in its Annual Report on Form 10-K for the fiscal year ended December 31, 2024, as such factors may be updated from time to time in its other filings with the SEC, which are accessible on the SEC鈥檚 website at www.sec.gov and the Investors section of the Company鈥檚 website at . 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鈥檚 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鈥檚 views as of any date subsequent to the date of this press release.
Contact Information
Corporate/Investors
Russo Partners
Nic Johnson
听
1.303.482.6405
Media
Russo Partners
David Schull
听
1.858.717.2310
Source: BioXcel Therapeutics, Inc.
IGALMI庐听is a registered trademark of BioXcel Therapeutics, Inc.
FAQ
What are the key findings from BioXcel's (BTAI) SERENITY At-Home Phase 3 trial?
What is the potential market opportunity for BXCL501?
How many patients participated in BioXcel's SERENITY At-Home trial?
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What dosage of BXCL501 was tested in the SERENITY At-Home trial?
