BioXcel Therapeutics Announces Positive Results from Correlation Study Supporting SERENITY At-Home Exploratory Efficacy Outcomes
BioXcel Therapeutics (Nasdaq: BTAI) reported positive correlation results supporting exploratory efficacy outcomes from the SERENITY At-Home program and plans to include the data in a supplemental New Drug Application (sNDA) targeted for Q1 2026.
In a prospective, open‑label in‑clinic study of 33 patients, the modified CGI‑S (mCGI‑S) scored by patients and caregivers strongly correlated with clinician PEC scores: ρ=0.89, p<0.0001 for patients and ρ=0.88, p<0.0001 for informants. No serious adverse events were reported and safety remained consistent with the IGALMI label. Company plans to include these findings in the sNDA and present additional data at upcoming medical meetings.
BioXcel Therapeutics (Nasdaq: BTAI) ha riportato risultati di correlazione positivi che supportano gli esiti di efficacia esplorativi dal programma SERENITY At-Home e piani di includere i dati in una domanda di New Drug (sNDA) supplementare mirata a Q1 2026.
In uno studio prospettico, in aperto in clinica di 33 pazienti, la CGI-S modificata (mCGI-S) valutata da pazienti e caregiver ha mostrato un'alta correlazione con i punteggi PEC del clinico: ρ=0.89, p<0.0001 per i pazienti e ρ=0.88, p<0.0001 per gli informatori. Nessun evento avverso grave è stato riportato e la sicurezza è rimasta coerente con l'etichetta IGALMI. L'azienda prevede di includere questi risultati nell'sNDA e presentare ulteriori dati nelle prossime riunioni mediche.
BioXcel Therapeutics (Nasdaq: BTAI) informó resultados de correlación positivos que respaldan los resultados de eficacia exploratoria del programa SERENITY At-Home y planes para incluir los datos en una solicitud de fármaco nuevo suplementaria (sNDA) dirigida a Q1 2026.
En un estudio prospectivo, abierto y en clínica de 33 pacientes, la versión modificada de CGI-S (mCGI-S) puntuó por pacientes y cuidadores, mostrando una fuerte correlación con los puntajes PEC de los clínicos: ρ=0.89, p<0.0001 para los pacientes y ρ=0.88, p<0.0001 para los informantes. No se reportaron eventos adversos graves y la seguridad se mantuvo consistente con la etiqueta IGALMI. La empresa planea incluir estos hallazgos en la sNDA y presentar datos adicionales en próximas reuniones médicas.
BioXcel Therapeutics (나스닥: BTAI)는 SERENITY At-Home 프로그램의 탐색적 효능 결과를 뒷받침하는 긍정적 상관 관계를 보고했으며 데이터가 보조 신약 신청(sNDA)에 포함될 계획이며 대상은 2026년 1분기입니다.
임상 현장에서 33명 환자를 대상으로 한 전향적, 공개적 오픈 라벨 연구에서, 환자와 간병인이 평가한 수정된 CGI-S(mCGI-S)가 임상의 PEC 점수와 강하게 상관되었으며: ρ=0.89, p<0.0001 환자들, ρ=0.88, p<0.0001 정보제공자들. 중대한 이상반응은 보고되지 않았고 안전성은 IGALMI 라벨과 일치하는 것으로 남아 있었다. 회사는 이 발견들을 sNDA에 포함시키고 향후 의학 학회에서 추가 데이터를 발표할 계획이다.
BioXcel Therapeutics (Nasdaq: BTAI) a rapporté des résultats de corrélation positifs soutenant les résultats d'efficacité exploratoire du programme SERENITY At-Home et prévoit d'inclure les données dans une demande de médicament nouveau supplémentaire (sNDA) ciblée pour Q1 2026.
Dans une étude prospective, ouverte en clinique et de 33 patients, le CGI-S modifié (mCGI-S) évalué par les patients et les soignants a montré une forte corrélation avec les scores PEC des cliniciens : ρ=0,89, p<0,0001 chez les patients et ρ=0,88, p<0,0001 chez les informants. Aucun effet indésirable grave n'a été signalé et la sécurité est restée conforme à l'étiquetage IGALMI. L'entreprise prévoit d'inclure ces résultats dans le sNDA et de présenter des données supplémentaires lors de prochaines réunions médicales.
BioXcel Therapeutics (Nasdaq: BTAI) berichtete positive Korrelationsergebnisse, die die explorativen Wirksamkeitsresultate des SERENITY At-Home-Programms unterstützen, und plant, die Daten in eine ergänzende Neuantrag (sNDA) aufzunehmen, die auf Q1 2026 abzielt.
In einer prospektiven, offenen In-Clinic-Studie mit 33 Patienten zeigte die modifizierte CGI-S (mCGI-S), bewertet von Patienten und Betreuern, eine starke Korrelation mit den PEC-Scores der Kliniker: ρ=0,89, p<0,0001 für Patienten und ρ=0,88, p<0,0001 für Informanten. Es wurden keine schweren unerwünschten Ereignisse gemeldet, und die Sicherheit blieb mit dem IGALMI-Label konsistent. Das Unternehmen plant, diese Ergebnisse in die sNDA aufzunehmen und weitere Daten bei kommenden medizinischen Meetings vorzustellen.
BioXcel Therapeutics (ناسداك: BTAI) أبلغت عن نتائج ارتباط إيجابية تدعم نتائج الفعالية الاستكشافية من برنامج SERENITY At-Home وتخطط لإدراج البيانات في طلب دواء جديد إضافي (sNDA) مستهدف لـ الربع الأول 2026.
في دراسة مستقبلية مفتوحة في العيادة شملت 33 مريضًا، تم تقييم CGI-S المعدلة (mCGI-S) من قبل المرضى ومقدمي الرعاية ووجد ارتباطاً قوياً مع نتائج PEC لدى الأطباء: ρ=0.89، p<0.0001 للمرضى وρ=0.88، p<0.0001 للمبلغين. لم تُبلغ عن أحداث سلبية خطيرة وظلت السلامة متوافقة مع تسمية IGALMI. تخطط الشركة لإدراج هذه النتائج في sNDA وتقديم بيانات إضافية في الاجتماعات الطبية القادمة.
BioXcel Therapeutics (纳斯达克: BTAI) 报告了积极相关性结果，支持 SERENITY At-Home 计划的探索性疗效结果，并计划将数据纳入针对性于 2026 年第一季度 的补充新药申请（sNDA）。
在一项前瞻性、开放标签的诊所内研究中，对 33 例患者，由患者和照护者评分的修订 CGI-S（mCGI-S）与临床医生的 PEC 评分呈强相关：ρ=0.89, p<0.0001 对于患者，ρ=0.88, p<0.0001 对于信息提供者。未报告严重不良事件，安全性与 IGALMI 标签一致。公司计划将这些发现纳入 sNDA，并在即将召开的学术会议上展示更多数据。
- Planned sNDA submission in Q1 2026
- Strong correlation: PEC vs mCGI-S ρ=0.89 (patients), ρ=0.88 (informants)
- No serious adverse events reported; safety consistent with IGALMI label
- Small study size of 33 patients
- mCGI‑S is an exploratory measurement rather than the standard PEC
Insights
Strong correlation supports using the mCGI-S for at‑home efficacy and bolsters an sNDA filing planned in
BioXcel completed the correlation study showing a high concordance between the clinician PEC and the patient/informant mCGI-S (ρ=0.89 and ρ=0.88, both p<0.0001), which directly supports using the mCGI-S as an exploratory at‑home efficacy measure. The finding addresses a measurement gap for outpatient, repeat‑dosing assessments and aligns with the plan to include these data in the supplemental NDA submission.
Risks and dependencies include regulatory acceptance of the mCGI-S as supportive evidence and whether the broader sNDA package (including completed trials) convinces reviewers; safety was unchanged with no serious adverse events reported and the profile remained consistent with the IGALMI® label. Watch for the formal sNDA filing and any FDA feedback after
Completes clinical trials required by FDA for sNDA submission planned for the first quarter of 2026
NEW HAVEN, Conn., Oct. 14, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced positive results from the correlation study related to exploratory efficacy outcomes from the SERENITY At-Home trial. The results, along with the data from the SERENITY At-Home trial, will be included in the supplemental New Drug Application (sNDA) submission that is planned for the first quarter of 2026.
The standard method for measuring acute agitation associated with schizophrenia and bipolar disorder is the Positive and Negative Syndrome Scale – Excited Component (PEC) administered by a trained clinician, which was used in the Serenity I & II Pivotal Trials. In order to evaluate BXCL501 for continued clinical effect with repeat dosing in the at-home setting using an exploratory efficacy measurement, the Company, in consultation with FDA, developed the modified CGI-S (mCGI-S) scale, which can be scored by patients and/or caregivers. The study assessed the correlation between PEC and mCGI-S in this prospective, open label, in-clinic trial in 33 patients.
The results demonstrated a strong correlation between the clinician assessments and the patient or caregiver (informant) rated outcomes, providing support for using mCGI-S to assess efficacy in the outpatient setting. A statistically significant and strong correlation between the PEC and mCGI-S with a correlation of ρ=0.89; p<0.0001 for patients and ρ=0.88; p<0.0001 for informants was observed.
“We are pleased with the strong and significant correlation observed with clinician assessment for both patients and informants, providing support for our exploratory efficacy outcomes in the SERENITY At-Home study.” said Dusan Kostic, Ph. D. Senior Vice President Medical Affairs and Clinical Development. “We plan to include these findings as a part of our upcoming sNDA package.”
There were no serious adverse events reported and the safety profile remains consistent with the IGALMI® label.
Additional data and results will be presented at upcoming medical meetings and conferences.
About Modified Clinical Global Impression – Severity Scale (mCGI-S)
The original clinician rated CGI-S scale describes an 8-point rating of agitation symptoms where zero means there was no assessment performed and scores of 1-7 rate increasing severities of the symptoms being assessed. For ease of implementation by the patient and informant in the home setting this scale was modified in consultation with FDA. The modified CGI-S is a 4-point scale where 0 is no agitation, and scores of 1-3 describe increasing severities of agitation (mild, moderate, or severe).
About the Positive and Negative Syndrome Scale - Excited Component (PEC)
The PEC is a 5-item assessment for rating acute psychomotor agitation within the following domains: excitement, hostility, tension, uncooperativeness, and poor impulse control (Montoya et al. 2011). Each item utilizes a 7-point scale, with domain-specific descriptions of the signs the rater may observe for each severity rating: 1 = Absent, 2 = Minimal, 3 = Mild, 4 = Moderate, 5 = Moderate Severe, 6 = Severe, and 7 = Extreme. The total PEC score is the sum of the five domain scores, ranging from 5 to 35. A PEC score of 14 is the threshold needed to treat an acute agitation episode.
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 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; potential market opportunity for BXCL501; release of data from the SERENITY At-Home 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’s completed, ongoing and proposed clinical trials to support regulatory approvals for its product candidates and change the treatment paradigm for agitation. 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
Russo Partners
Nic Johnson
nic.johnson@russopartnersllc.com
1.303.482.6405
Media
Russo Partners
David Schull
david.schull@russopartnersllc.com
1.858.717.2310
Source: BioXcel Therapeutics, Inc.
IGALMI® is a registered trademark of BioXcel Therapeutics, Inc.
FAQ
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