BioXcel Therapeutics Announces Initiation of SERENITY At-Home Pivotal Phase 3 Safety Trial of BXCL501 for Acute Treatment of Agitation Associated with Bipolar Disorders or Schizophrenia
BioXcel Therapeutics (Nasdaq: BTAI) has initiated patient enrollment in its SERENITY At-Home pivotal Phase 3 trial. The study will evaluate the safety of BXCL501, an investigational orally dissolving film formulation of dexmedetomidine, for the acute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home setting. The trial is expected to last 9-12 months and will enroll approximately 200 patients. Participants will self-administer 120 mcg of BXCL501 or placebo during agitation episodes over a 12-week period. The primary objective is safety, with an exploratory endpoint using a modified clinical global impression of change (mCGI-C) to evaluate patient and caregiver impressions. This trial addresses a significant unmet need, as there are currently no FDA-approved therapies for at-home treatment of agitation in these conditions.
BioXcel Therapeutics (Nasdaq: BTAI) ha avviato l'arruolamento dei pazienti nel suo studio clinico di Fase 3 SERENITY At-Home. Lo studio valuterà la sicurezza di BXCL501, una formulazione sperimentale di film orale disgregabile di dexmedetomidina, per il trattamento acuto dell'agitazione associata a disturbi bipolari o schizofrenia in un contesto domestico. La sperimentazione dovrebbe durare tra i 9 e i 12 mesi e prevedere l'arruolamento di circa 200 pazienti. I partecipanti autogestiranno 120 mcg di BXCL501 o placebo durante gli episodi di agitazione per un periodo di 12 settimane. L'obiettivo primario è la sicurezza, con un endpoint esplorativo che utilizza un'impressione clinica globale modificata di cambiamento (mCGI-C) per valutare le impressioni dei pazienti e dei caregiver. Questo trial affronta un bisogno significativo non soddisfatto, poiché attualmente non ci sono terapie approvate dalla FDA per il trattamento dell'agitazione a casa in queste condizioni.
BioXcel Therapeutics (Nasdaq: BTAI) ha iniciado la inscripción de pacientes en su ensayo clínico pivotal de Fase 3 SERENITY At-Home. El estudio evaluará la seguridad de BXCL501, una formulación experimental de película disolvente oral de dexmedetomidina, para el tratamiento agudo de la agitación asociada a trastornos bipolares o esquizofrenia en el hogar. Se espera que el ensayo dure de 9 a 12 meses y se inscriban aproximadamente 200 pacientes. Los participantes se autoadministrarán 120 mcg de BXCL501 o placebo durante episodios de agitación durante un período de 12 semanas. El objetivo principal es la seguridad, con un punto final exploratorio que utiliza una impresión clínica global modificada de cambio (mCGI-C) para evaluar las impresiones de pacientes y cuidadores. Este ensayo aborda una necesidad significativa insatisfecha, ya que actualmente no hay terapias aprobadas por la FDA para el tratamiento en casa de la agitación en estas condiciones.
BioXcel Therapeutics (Nasdaq: BTAI)는 SERENITY At-Home 주요 3상 시험에 환자 등록을 시작했습니다. 이 연구는 집에서의 양극성 장애 또는 정신분열증과 관련된 급성 초조 치료를 위한 덱스메데토미딘의 경구 용해 필름 제형인 BXCL501의 안전성을 평가할 것입니다. 시험은 9-12개월 동안 진행될 예정이며 약 200명의 환자를 등록할 계획입니다. 참가자들은 초조 에피소드 동안 12주 동안 120 mcg의 BXCL501 또는 위약을 자가 투여합니다. 주요 목표는 안전성이며, 환자와 보호자의 인상을 평가하기 위해 수정된 임상 글로벌 인상 변화(mCGI-C)를 사용하는 탐색적 최종점을 설정합니다. 이 시험은 이러한 조건에서 집에서의 초조 치료를 위한 FDA 승인 치료제가 현재 존재하지 않기 때문에 상당한 unmet need을 해결합니다.
BioXcel Therapeutics (Nasdaq: BTAI) a lancé l'inscription des patients pour son essai clinique pivot de Phase 3 SERENITY At-Home. L'étude évaluera la sécurité de BXCL501, une formulation expérimentale sous forme de film oral dissous de dexmedetomidine, pour le traitement aigu de l'agitation associée à des troubles bipolaires ou à la schizophrénie à domicile. L'essai devrait durer de 9 à 12 mois et recruter environ 200 patients. Les participants s'autoadministreront 120 mcg de BXCL501 ou de placebo durant des épisodes d'agitation sur une période de 12 semaines. L'objectif principal est la sécurité, avec un critère d'évaluation exploratoire utilisant une impression globale clinique modifiée de changement (mCGI-C) pour évaluer les impressions des patients et des soignants. Cet essai répond à un besoin non satisfait important, car il n'existe actuellement aucune thérapie approuvée par la FDA pour le traitement à domicile de l'agitation dans ces conditions.
BioXcel Therapeutics (Nasdaq: BTAI) hat die Patientenanmeldung für seine SERENITY At-Home entscheidende Phase-3-Studie gestartet. Die Studie wird die Sicherheit von BXCL501, einer experimentellen oral auflösenden Filmformulierung von Dexmedetomidin, zur akuten Behandlung von Agitation, die mit bipolaren Störungen oder Schizophrenie in einer häuslichen Umgebung verbunden ist, bewerten. Die Studie wird voraussichtlich 9-12 Monate dauern und etwa 200 Patienten einschließen. Die Teilnehmer werden während Agitations-Episoden über einen Zeitraum von 12 Wochen 120 mcg BXCL501 oder ein Placebo selbst verabreichen. Das Hauptziel ist die Sicherheit, während ein explorativer Endpunkt eine modifizierte klinische globale Eindrucksänderung (mCGI-C) verwendet, um die Eindrücke von Patienten und Betreuern zu bewerten. Diese Studie adressiert ein signifikantes ungedecktes Bedürfnis, da es derzeit keine von der FDA zugelassenen Therapien für die häusliche Behandlung von Agitation in diesen Erkrankungen gibt.
- Initiation of SERENITY At-Home pivotal Phase 3 trial for BXCL501
- Addressing an unmet need with an estimated 23 million annual agitation episodes in the at-home setting
- Potential to reduce emergency room visits and treatment costs
- Building on previous trial experience and FDA engagement
- Exploring a new market with no current FDA-approved therapies for at-home treatment
- Trial results and potential FDA approval still uncertain
- 9-12 month duration for trial completion, delaying potential market entry
- to testing only one dose (120 mcg) of BXCL501
Insights
The initiation of the SERENITY At-Home Phase 3 trial for BXCL501 is a significant step in addressing the unmet need for at-home treatment of agitation in bipolar disorders and schizophrenia. With an estimated 23 million annual agitation episodes in the at-home setting and no FDA-approved therapies currently available, this trial could potentially fill a critical gap in patient care.
The 120 mcg dose being tested is already approved for IGALMI™, which suggests a potentially smoother regulatory pathway if safety in the home setting is established. The trial's focus on safety, with a 12-week duration and 200 patient enrollment, is appropriate for assessing real-world risks. The inclusion of caregiver/informant feedback in the safety data collection is important for a comprehensive safety profile in the outpatient setting.
This trial initiation is a positive development for BioXcel Therapeutics (NASDAQ: BTAI). The potential market for at-home agitation treatment is substantial, with 23 million annual episodes representing a significant untapped opportunity. If successful, BXCL501 could become a first-in-class therapy for this indication, potentially driving substantial revenue growth.
Investors should note that the trial's 9 to 12-month duration suggests results may be available in late 2025 or early 2026. While this timeline is relatively short for a Phase 3 trial, it still represents a medium-term catalyst for the stock. The company's use of AI in drug development could also be viewed favorably by the market, potentially supporting a premium valuation if this approach proves successful.
The SERENITY At-Home trial addresses a critical need in psychiatric care. Currently, agitation episodes often result in emergency room visits, which are costly and potentially traumatic for patients. An at-home treatment option could significantly improve patient outcomes and reduce healthcare system burden.
The use of the modified clinical global impression of change (mCGI-C) as an exploratory endpoint is noteworthy. This patient-reported outcome measure will provide valuable insights into the real-world effectiveness of BXCL501. However, it's important to note that the primary focus on safety suggests that efficacy data may be , potentially necessitating further trials to fully establish the drug's benefit-risk profile in the home setting.
Estimated 23 million annual agitation episodes in the at-home setting 1-3
No FDA-approved therapies in the at-home setting for acute treatment of agitation associated with bipolar disorders or schizophrenia
NEW HAVEN, Conn., Sept. 05, 2024 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience and immuno-oncology, today announced the initiation of patient enrollment in its SERENITY At-Home pivotal Phase 3 trial. The trial will evaluate the safety of BXCL501, the Company’s 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. The trial duration currently is expected to be 9 to 12 months.
“The management of agitation for patients at home is extremely important but it is a significant clinical challenge,” said Dr. John Krystal, M.D., the Robert L. McNeil, Jr. Professor of Translational Research and Chair of the Department of Psychiatry at Yale School of Medicine. “Treating agitation early at home could help reduce patient suffering and trips to the emergency room, promoting patient safety and reducing the cost of treatment. New therapeutic options could be important in addressing this treatment gap.”
“We are pleased to have initiated our SERENITY At-Home trial, which is based on our extensive engagement with the FDA and our considerable previous trial experience with BXCL501. We are also encouraged by the safety results observed for BXCL501 in our programs to date and by the experience of patients and prescribers with IGALMI™,” said Vincent J. O’Neill, M.D., Executive Vice President, Chief of Product Development and Medical Officer of BioXcel Therapeutics. “The primary objective of the study is the characterization of the safety profile in the home setting of the 120 mcg dose, an approved dose of IGALMI.”
SERENITY At-Home Pivotal Phase 3 Trial Design Summary
The SERENITY At-Home Phase 3 trial is a double-blind, placebo-controlled study to evaluate the safety of a 120 mcg dose of BXCL501 in the home setting.
- The outpatient trial will enroll approximately 200 patients residing at home either alone or with caregivers/informants with at least one treated episode of agitation.
- Patients will self-administer 120 mcg of BXCL501 or placebo when agitation episodes occur over the 12-week trial period.
- Safety data will be collected from individual subjects as well as caregivers/informants, as is typical for outpatient trials. The investigator or designee will assess and record adverse events at all in-clinic visits based on telephone interviews and agitation episode diaries from patients and (when applicable) caregivers/informants.
- The primary objective is safety. Patients or caregivers/informants will complete a modified clinical global impression of change (mCGI-C) two hours after dosing as an exploratory endpoint to evaluate their impression of use in the outpatient setting.
A corporate presentation, including information on the SERENITY At-Home trial, is available on the Events & Presentations page under the “News/Events” tab in the Investors & Media section of the Company’s website at 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 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 advancement of its SERENITY trial and the trial design and expected timing thereof; potential market opportunity for BXCL501; and 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, 2023, as such factors may be updated from time to time in its other filings with the SEC, including without limitation its Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2024, which are accessible on the SEC’s website at www.sec.gov. 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
BioXcel Therapeutics
Erik Kopp
1.203.494.7062
ekopp@bioxceltherapeutics.com
Investors
Russo Partners
Nic Johnson
1.303.482.6405
Nic.Johnson@russopartnersllc.com
Media
Russo Partners
David Schull
1.858.717.2310
David.schull@russopartnersllc.com
Source: BioXcel Therapeutics, Inc.
IGALMI™ is a trademark of BioXcel Therapeutics, Inc.
BT BIOXCEL THERAPEUTICS is a registered trademark of BioXcel Therapeutics, Inc.
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References
1. 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.
2. 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.
3. 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.
FAQ
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