BioXcel Therapeutics Receives Second Positive Recommendation from Data Safety Monitoring Board (DSMB) to Continue SERENITY At-Home Pivotal Phase 3 Safety Trial for Acute Treatment of Agitation Associated with Bipolar Disorders or Schizophrenia
BioXcel Therapeutics (Nasdaq: BTAI) received a second positive recommendation from the Data Safety Monitoring Board (DSMB) to continue its SERENITY At-Home pivotal Phase 3 safety trial of BXCL501 without modifications. The trial evaluates the treatment of agitation associated with bipolar disorders or schizophrenia in at-home settings.
The DSMB's recommendation followed a review of unblinded safety data from 178 patients as of May 28, 2025. The trial, which is now fully enrolled with 200 patients, is testing a 120 mcg dose of BXCL501 in a double-blind, placebo-controlled study design. The company expects to release topline data in Q3 2025.
This trial aims to support a potential supplemental New Drug Application (sNDA) to expand the IGALMI® label for at-home use, addressing an unmet medical need as there are currently no FDA-approved therapies for bipolar and schizophrenia-related agitation in the at-home setting.
BioXcel Therapeutics (Nasdaq: BTAI) ha ricevuto una seconda raccomandazione positiva dal Data Safety Monitoring Board (DSMB) per proseguire senza modifiche la sua sperimentazione di sicurezza di fase 3 SERENITY At-Home con BXCL501. Lo studio valuta il trattamento dell'agitazione associata a disturbi bipolari o schizofrenia in contesti domiciliari.
La raccomandazione del DSMB è arrivata dopo la revisione dei dati di sicurezza non in cieco di 178 pazienti al 28 maggio 2025. Lo studio, ora completamente arruolato con 200 pazienti, sta testando una dose di 120 mcg di BXCL501 in uno schema di studio in doppio cieco e controllato con placebo. L'azienda prevede di pubblicare i dati principali nel terzo trimestre 2025.
Questo studio mira a supportare una potenziale domanda supplementare di nuovo farmaco (sNDA) per ampliare l'indicazione di IGALMI® per l'uso domiciliare, rispondendo a un bisogno medico insoddisfatto, dato che attualmente non esistono terapie approvate dalla FDA per l'agitazione correlata a bipolarismo e schizofrenia in ambito domiciliare.
BioXcel Therapeutics (Nasdaq: BTAI) recibió una segunda recomendación positiva de la Junta de Monitoreo de Seguridad de Datos (DSMB) para continuar su ensayo pivotal de seguridad en fase 3 SERENITY At-Home con BXCL501 sin modificaciones. El estudio evalúa el tratamiento de la agitación asociada con trastornos bipolares o esquizofrenia en entornos domiciliarios.
La recomendación del DSMB siguió a la revisión de datos de seguridad no cegados de 178 pacientes al 28 de mayo de 2025. El ensayo, que ya está completamente inscrito con 200 pacientes, está probando una dosis de 120 mcg de BXCL501 en un diseño de estudio doble ciego y controlado con placebo. La compañía espera publicar datos preliminares en el tercer trimestre de 2025.
Este ensayo tiene como objetivo apoyar una posible solicitud suplementaria de nuevo fármaco (sNDA) para ampliar la indicación de IGALMI® para uso en el hogar, abordando una necesidad médica no cubierta ya que actualmente no existen terapias aprobadas por la FDA para la agitación relacionada con el trastorno bipolar y la esquizofrenia en el entorno domiciliario.
BioXcel Therapeutics (나스닥: BTAI)가 데이터 안전성 모니터링 위원회(DSMB)로부터 BXCL501의 SERENITY At-Home 3상 주요 안전성 시험을 수정 없이 계속 진행하라는 두 번째 긍정적 권고를 받았습니다. 이 시험은 양극성 장애 또는 조현병과 관련된 초조함을 가정 환경에서 치료하는 것을 평가합니다.
DSMB의 권고는 2025년 5월 28일 기준 178명 환자의 비맹검 안전성 데이터를 검토한 후 나왔습니다. 현재 200명 환자가 완전히 등록된 이 시험은 이중맹검 위약 대조 연구 설계로 120 mcg 용량의 BXCL501을 테스트하고 있습니다. 회사는 2025년 3분기에 주요 결과 데이터를 발표할 예정입니다.
이 시험은 IGALMI®의 가정 내 사용 확대를 위한 보충 신약 신청(sNDA)을 지원하는 것을 목표로 하며, 현재 가정 환경에서 양극성 및 조현병 관련 초조함에 대해 FDA 승인 치료제가 없어 충족되지 않은 의료 수요를 해결합니다.
BioXcel Therapeutics (Nasdaq : BTAI) a reçu une deuxième recommandation positive du Data Safety Monitoring Board (DSMB) pour poursuivre son essai pivot de phase 3 SERENITY At-Home sur la sécurité de BXCL501 sans modifications. L'essai évalue le traitement de l'agitation associée aux troubles bipolaires ou à la schizophrénie en milieu domiciliaire.
La recommandation du DSMB fait suite à l'examen des données de sécurité non aveuglées de 178 patients au 28 mai 2025. L'essai, désormais entièrement recruté avec 200 patients, teste une dose de 120 mcg de BXCL501 dans un protocole en double aveugle contrôlé par placebo. La société prévoit de publier les données principales au 3e trimestre 2025.
Cette étude vise à soutenir une éventuelle demande d'autorisation de mise sur le marché supplémentaire (sNDA) pour étendre l'indication d'IGALMI® à l'usage à domicile, répondant à un besoin médical non satisfait car il n'existe actuellement aucun traitement approuvé par la FDA pour l'agitation liée au trouble bipolaire et à la schizophrénie en milieu domiciliaire.
BioXcel Therapeutics (Nasdaq: BTAI) erhielt eine zweite positive Empfehlung vom Data Safety Monitoring Board (DSMB), die SERENITY At-Home Phase-3-Sicherheitsstudie mit BXCL501 ohne Änderungen fortzusetzen. Die Studie bewertet die Behandlung von Unruhe im Zusammenhang mit bipolaren Störungen oder Schizophrenie in häuslichen Umgebungen.
Die Empfehlung des DSMB folgte auf die Überprüfung unblinder Sicherheitsdaten von 178 Patienten zum 28. Mai 2025. Die Studie ist nun vollständig mit 200 Patienten eingeschrieben und testet eine 120 mcg Dosis von BXCL501 in einem doppelblinden, placebokontrollierten Studiendesign. Das Unternehmen erwartet die Veröffentlichung der wichtigsten Daten im 3. Quartal 2025.
Diese Studie zielt darauf ab, eine potenzielle ergänzende Zulassungsanmeldung (sNDA) zu unterstützen, um die IGALMI®-Indikation für die Anwendung zu Hause zu erweitern und damit einen ungedeckten medizinischen Bedarf zu adressieren, da derzeit keine von der FDA zugelassenen Therapien für unruhige Zustände bei bipolarer Störung und Schizophrenie im häuslichen Umfeld existieren.
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Insights
Positive DSMB safety review strengthens BXCL501's potential for at-home agitation treatment, with pivotal data coming Q3 2025.
This second positive DSMB recommendation represents a significant de-risking milestone for BioXcel's SERENITY At-Home Phase 3 trial. With safety data from 178 patients passing independent review without modifications needed, the trial appears to be proceeding smoothly toward its Q3 2025 readout. The fully enrolled study evaluates BXCL501 (120 mcg) against placebo in 200 patients experiencing agitation from bipolar disorder or schizophrenia.
The at-home setting represents a crucial expansion opportunity for BXCL501 (marketed as IGALMI). Currently, IGALMI is only approved for use in healthcare facilities, but the vast majority of agitation episodes occur at home. A successful at-home label expansion would dramatically increase the addressable market, potentially reaching millions of patients who currently lack FDA-approved options.
This safety review is particularly important for an at-home indication, as regulators apply heightened scrutiny to self-administered acute psychiatric medications. The clean DSMB review suggests the drug's
If positive, this data would support a supplemental New Drug Application (sNDA) to expand IGALMI's label - a regulatory pathway that's typically more straightforward than the original approval process. The unmet need highlighted by the company is genuine, as psychiatric agitation episodes often escalate to emergency interventions when effective at-home options are unavailable.
DSMB recommended the continuation of trial as planned
Topline data expected in Q3 2025
NEW HAVEN, Conn., July 01, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced the second positive recommendation by an independent Data Safety Monitoring Board (DSMB) to continue, without modification, the SERENITY At-Home pivotal Phase 3 safety trial of BXCL501 for acute treatment of agitation associated with bipolar disorders or schizophrenia.
The DSMB recommendation followed a review of unblinded safety data from 178 patients dosed as of the May 28, 2025 cutoff date. The trial is fully enrolled and collection of data over the 12-week period is continuing.
“We are pleased with this additional favorable recommendation from the DSMB regarding our first at-home trial of BXCL501 and look forward to the upcoming data readout,” said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. “Bipolar and schizophrenia-related agitation in the at-home setting is a large, unmet medical need, with no FDA-approved therapies. We are highly motivated to address this need for millions of patients, with trial data intended to support a potential sNDA submission to expand the IGALMI® label in the at-home setting.”
The SERENITY At-Home Phase 3 trial is 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.
- Trial enrollment was completed in May, 2025
- Topline data expected in Q3 2025.
Additional information on the SERENITY At-Home trial is included in a corporate presentation in the Investors section of the Company’s website: bioxceltherapeutics.com.
About BXCL501
Outside 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 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 at-home setting. The trial is designed to evaluate 200 patients with a history of agitation episodes residing at home either alone or with caregivers/informants. Patients are self-administering 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. In addition, 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 exploratory endpoints 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 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 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 trial; potential market opportunity for BXCL501; completing enrollment and release of topline data from the ongoing SERENITY trial; submission of an sNDA; expanding the IGALMI® label in the at-home setting; the potential for the results from the Company’s completed, ongoing and proposed clinical trials to support regulatory approvals for its product candidates. 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 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’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
Investors
Russo Partners
Nic Johnson
1.303.482.6405
Media
Russo Partners
David Schull
1.858.717.2310
Source: BioXcel Therapeutics, Inc.
IGALMI® is a registered trademark of BioXcel Therapeutics, Inc.
FAQ
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