BioXcel Therapeutics Receives 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 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 safety of a 120 mcg dose for acute treatment of agitation in bipolar disorders or schizophrenia patients in at-home settings.
The DSMB's recommendation followed a review of unblinded safety data from the first 115 patients. The trial is fully enrolled with over 205 patients dosed, and more than 150 patients have received multiple doses during the 12-week trial period. Topline data is expected in Q3 2025. The results aim to support a potential sNDA submission for IGALMI® label expansion in the at-home setting, addressing an unmet medical need as there are currently no FDA-approved therapies for bipolar or schizophrenia-related agitation in this environment.
BioXcel Therapeutics (NASDAQ: BTAI) ha ricevuto una raccomandazione positiva dal Data Safety Monitoring Board (DSMB) per proseguire senza modifiche lo studio pivotale di Fase 3 SERENITY At-Home sulla sicurezza di BXCL501. Lo studio valuta la sicurezza di una dose da 120 mcg per il trattamento acuto dell'agitazione in pazienti con disturbi bipolari o schizofrenia in ambiente domiciliare.
La raccomandazione del DSMB è arrivata dopo la revisione dei dati di sicurezza non in cieco dei primi 115 pazienti. Lo studio è completamente arruolato con oltre 205 pazienti trattati e più di 150 hanno ricevuto dosi multiple durante il periodo di 12 settimane. I dati principali sono attesi nel terzo trimestre 2025. I risultati mirano a supportare una possibile sNDA per l'espansione dell'indicazione di IGALMI® in ambiente domiciliare, rispondendo a un bisogno medico insoddisfatto, dato che attualmente non esistono terapie approvate dalla FDA per l'agitazione correlata a disturbi bipolari o schizofrenia in questo contesto.
BioXcel Therapeutics (NASDAQ: BTAI) recibió una recomendación positiva del Comité de Monitoreo de Seguridad de Datos (DSMB) para continuar sin modificaciones el ensayo pivotal de Fase 3 SERENITY At-Home sobre seguridad de BXCL501. El ensayo evalúa la seguridad de una dosis de 120 mcg para el tratamiento agudo de la agitación en pacientes con trastornos bipolares o esquizofrenia en entornos domiciliarios.
La recomendación del DSMB se basó en la revisión de datos de seguridad no enmascarados de los primeros 115 pacientes. El ensayo está completamente reclutado con más de 205 pacientes dosificados y más de 150 han recibido dosis múltiples durante el período de 12 semanas. Se esperan datos principales en el tercer trimestre de 2025. Los resultados buscan apoyar una posible presentación de sNDA para la expansión de la etiqueta de IGALMI® en el entorno domiciliario, atendiendo una necesidad médica no cubierta ya que actualmente no existen terapias aprobadas por la FDA para la agitación relacionada con trastornos bipolares o esquizofrenia en este contexto.
BioXcel Therapeutics (NASDAQ: BTAI)는 데이터 안전성 모니터링 위원회(DSMB)로부터 수정 없이 SERENITY At-Home 주요 3상 안전성 시험을 계속 진행하라는 긍정적인 권고를 받았습니다. 이 시험은 가정 환경에서 양극성 장애 또는 정신분열증 환자의 급성 초조 상태 치료를 위해 120mcg 용량의 BXCL501 안전성을 평가합니다.
DSMB의 권고는 처음 115명의 환자에 대한 비맹검 안전성 데이터를 검토한 후에 나왔습니다. 시험은 205명 이상의 환자가 투여되어 완전 모집되었으며, 150명 이상이 12주 시험 기간 동안 다회 투여를 받았습니다. 주요 데이터는 2025년 3분기에 발표될 예정입니다. 이 결과는 가정 환경에서 IGALMI® 적응증 확대를 위한 잠재적 sNDA 제출을 지원하는 것을 목표로 하며, 현재 FDA 승인된 양극성 장애 또는 정신분열증 관련 초조 치료제가 없는 미충족 의료 수요를 해결합니다.
BioXcel Therapeutics (NASDAQ : BTAI) a reçu une recommandation positive du Data Safety Monitoring Board (DSMB) pour poursuivre sans modification son essai pivot de phase 3 SERENITY At-Home évaluant la sécurité de BXCL501. L’essai évalue la sécurité d’une dose de 120 mcg pour le traitement aigu de l’agitation chez des patients atteints de troubles bipolaires ou de schizophrénie en milieu domiciliaire.
La recommandation du DSMB fait suite à l’examen des données de sécurité non en aveugle des 115 premiers patients. L’essai est entièrement recruté avec plus de 205 patients traités, et plus de 150 ont reçu plusieurs doses durant la période d’essai de 12 semaines. Les données principales sont attendues au troisième trimestre 2025. Les résultats visent à soutenir une éventuelle soumission de sNDA pour l’extension de l’indication d’IGALMI® en milieu domiciliaire, répondant à un besoin médical non satisfait, puisqu’aucun traitement approuvé par la FDA n’existe actuellement pour l’agitation liée aux troubles bipolaires ou à la schizophrénie dans ce contexte.
BioXcel Therapeutics (NASDAQ: BTAI) erhielt eine positive Empfehlung vom Data Safety Monitoring Board (DSMB), die SERENITY At-Home entscheidende Phase-3-Sicherheitsstudie zu BXCL501 ohne Änderungen fortzusetzen. Die Studie bewertet die Sicherheit einer 120 mcg-Dosis zur akuten Behandlung von Erregungszuständen bei Patienten mit bipolaren Störungen oder Schizophrenie in häuslicher Umgebung.
Die Empfehlung des DSMB erfolgte nach Überprüfung unblinder Sicherheitsdaten der ersten 115 Patienten. Die Studie ist vollständig eingeschrieben mit über 205 behandelten Patienten, von denen mehr als 150 während der 12-wöchigen Studienphase mehrfach dosiert wurden. Topline-Daten werden für das dritte Quartal 2025 erwartet. Die Ergebnisse sollen eine mögliche sNDA-Einreichung zur Erweiterung der IGALMI®-Indikation für die häusliche Anwendung unterstützen und einen ungedeckten medizinischen Bedarf adressieren, da derzeit keine von der FDA zugelassenen Therapien für Erregungszustände bei bipolarer Störung oder Schizophrenie in diesem Umfeld existieren.
- Positive DSMB safety review recommendation to continue trial without modifications
- Trial is fully enrolled with over 205 patients, exceeding the 200-patient target
- Potential to address unmet medical need as no FDA-approved at-home therapies exist for this indication
- Multiple dose administration successful in over 150 patients
- None.
Insights
BTAI's Phase 3 trial for at-home agitation treatment receives positive DSMB continuation; potential label expansion represents significant commercial opportunity.
The DSMB's recommendation to continue the SERENITY At-Home trial without modifications is a positive safety signal for BioXcel's BXCL501. This pivotal Phase 3 safety trial has successfully enrolled over 205 patients (exceeding the 200 target), with more than 150 patients receiving multiple doses over the 12-week period.
The trial's significance lies in its potential to expand IGALMI's label to include at-home treatment of agitation in bipolar disorder and schizophrenia patients. Currently, no FDA-approved therapies exist for this specific use case, representing a substantial unmet medical need and market opportunity.
The company has completed patient enrollment, suggesting efficient trial execution. With topline data expected in Q3 2025, a successful outcome could support a supplemental New Drug Application (sNDA) to expand IGALMI's approved uses beyond its current indications.
While this DSMB review focused primarily on safety (reviewing unblinded data from 115 patients), the continuation recommendation indicates no concerning safety signals have emerged that would warrant trial modification or termination. For biotechnology companies, especially those with limited commercial products, label expansions for existing approved therapies represent lower-risk opportunities to expand market reach without the full development costs of new compounds.
DSMB recommended the continuation of trial as planned
Topline data expected in Q3 2025
NEW HAVEN, Conn., May 27, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced that an independent Data Safety Monitoring Board (DSMB) recommended that the SERENITY At-Home pivotal Phase 3 safety trial of BXCL501 for acute treatment of agitation associated with bipolar disorders or schizophrenia continue without modification.
The DSMB recommendation followed a review of unblinded safety data from the first 115 patients dosed as of the May 2, 2025 cutoff date. The trial is fully enrolled and collection of data over the 12-week period is continuing.
“We are pleased with the favorable DSMB meeting outcome and are excited about the upcoming data readout for our first at-home trial with BXCL501,” said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. “Results are intended to help support a potential sNDA submission for label expansion of IGALMI® in the at-home setting — a sizeable unmet medical need given there are no FDA-approved therapies for bipolar or schizophrenia-related agitation in this environment.”
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 is complete:
- More than 205 patients have been dosed.
- More than 150 patients have received multiple doses for agitation over the 12-week trial period.
- 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 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 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; 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
Corporate/Investors
BioXcel Therapeutics
Erik Kopp
1.203.494.7062
Media
Russo Partners
David Schull
1.858.717.2310
Source: BioXcel Therapeutics, Inc.
IGALMI® is a registered trademark of BioXcel Therapeutics, Inc.
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