Anavex Life Sciences Announces Publication of Oral Blarcamesine Describing a New Class of Clinical Precision Medicine from Phase IIb/III Alzheimer’s Disease Trial
Anavex Life Sciences (NASDAQ:AVXL) has published results from its Phase IIb/III trial of oral blarcamesine for early Alzheimer's disease treatment. The study demonstrates that the once-daily oral medication showed significant clinical efficacy and improved Quality of Life (QoL-AD) scores in patients.
Using a Precision Medicine approach, the treatment benefited up to 70% of Alzheimer's disease participants with unprecedented effect size. The drug demonstrated superior clinical efficacy versus approved therapies, with a strong safety profile and no requirement for routine MRI monitoring.
The results are currently available on medRxiv as a preprint and are being submitted to a peer-reviewed medical journal. The company will continue evaluating the Phase IIb/III early Alzheimer's disease and ATTENTION-AD trial data for future presentations at international conferences.
Anavex Life Sciences (NASDAQ:AVXL) ha pubblicato i risultati del suo trial di fase IIb/III sull'uso orale di blarcamesina per il trattamento precoce della malattia di Alzheimer. Lo studio ha dimostrato che il farmaco assunto una volta al giorno ha mostrato un'efficacia clinica significativa e un miglioramento dei punteggi QoL-AD nei pazienti.
Adottando un approccio di medicina di precisione, il trattamento ha beneficiato fino al 70% dei partecipanti con malattia di Alzheimer con un effetto di grande magnitudine senza precedenti. Il farmaco ha dimostrato un'efficacia clinica superiore rispetto alle terapie approvate, accompagnata da un profilo di sicurezza solido e senza necessità di monitoraggio MRI di routine.
I risultati sono attualmente disponibili su medRxiv come preprint e sono in fase di sottomissione a una rivista medica sottoposta a peer review. L'azienda continuerà a valutare i dati del trial Phase IIb/III in Alzheimer precoce e ATTENTION-AD per futuri interventi a conferenze internazionali.
Anavex Life Sciences (NASDAQ:AVXL) ha publicado los resultados de su ensayo de fase IIb/III de blarcamesina oral para el tratamiento de la enfermedad de Alzheimer en etapas tempranas. El estudio demuestra que el medicamento oral de una dosis diaria mostró eficacia clínica significativa y mejoras en las puntuaciones de Calidad de Vida QoL-AD en los pacientes.
Utilizando un enfoque de Medicina de Precisión, el tratamiento benefició a hasta el 70% de los participantes con Alzheimer con un tamaño del efecto sin precedentes. El fármaco demostró una eficacia clínica superior frente a las terapias aprobadas, con un perfil de seguridad sólido y sin necesidad de resonancia magnética de rutina.
Los resultados están disponibles actualmente en medRxiv como preprint y se están presentando para su publicación en una revista médica revisada por pares. La empresa continuará evaluando los datos del ensayo Phase IIb/III para Alzheimer temprano y del ensayo ATTENTION-AD para futuras presentaciones en conferencias internacionales.
Anavex Life Sciences (NASDAQ:AVXL)는 조기 알츠하이머병 치료를 위한 경구 블라르케미인(blarcamesine)의 2b/3상 시험 결과를 발표했다. 연구에 따르면 하루 한 번 복용하는 경구 약물이 환자에서 유의미한 임상 효능과 QoL-AD 점수 향상을 보여주었다.
정밀의료(Precision Medicine) 접근법을 사용하여 치료는 복수의 알츠하이머 병 참가자 중 최대 70%에게 전례 없는 효과 크기로 혜택을 주었다. 이 약은 승인된 치료제 대비 우수한 임상 효능을 보였고, 강한 안전성 프로파일과 정기 MRI 모니터링의 필요 없음이 특징이다.
이 결과는 현재 medRxiv의 프리프린트로 공개되었으며 동료심사를 거친 저널에 제출 중이다. 회사는 향후 국제 학회에서의 발표를 위해 초기 알츠하이머 및 ATTENTION-AD 시험 데이터의 추가 평가를 계속할 것이다.
Anavex Life Sciences (NASDAQ:AVXL) a publié les résultats de son essai de phase IIb/III sur l’utilisation orale de la blarcamesine pour le traitement de la maladie d’Alzheimer à un stade précoce. L’étude démontre que le médicament oral pris une fois par jour a démontré une efficacité clinique significative et une amélioration des scores QoL-AD chez les patients.
En adoptant une approche de médecine de précision, le traitement a bénéficié jusqu’à 70% des participants atteints de la maladie d’Alzheimer avec une taille d’effet sans précédent. Le médicament a démontré une efficacité clinique supérieure par rapport aux thérapies approuvées, avec un profil de sécurité solide et sans besoin de surveillance par IRM de routine.
Les résultats sont actuellement disponibles sur medRxiv en tant que préprint et font l’objet d’une soumission à une revue médicale évaluée par des pairs. L’entreprise continuera d’évaluer les données de l’essai Phase IIb/III pour Alzheimer précoce et l’essai ATTENTION-AD pour de futures présentations lors de conférences internationales.
Anavex Life Sciences (NASDAQ:AVXL) hat Ergebnisse aus der Phase-IIb/III-Studie zu oralem Blarcamesin zur Behandlung der frühesten Alzheimer-Krankheit veröffentlicht. Die Studie zeigt, dass das einmal täglich verabreichte orale Medikament signifikante klinische Wirksamkeit und Verbesserungen der QoL-AD-Werte bei den Patienten aufwies.
Unter Verwendung eines Präzisionsmedizin-Ansatzes hat die Behandlung bis zu 70% der Alzheimer-Teilnehmer mit beispielloser Effektgröße profitieren lassen. Das Medikament zeigte überlegene klinische Wirksamkeit gegenüber zugelassenen Therapien, mit sicherem Profil und ohne Notwendigkeit einer routinemäßigen MRI-Überwachung.
Die Ergebnisse stehen derzeit auf medRxiv als Preprint zur Verfügung und werden in eine begutachtete medizinische Fachzeitschrift eingereicht. Das Unternehmen wird die Phase-IIb/III-Daten zur frühen Alzheimer-Krankheit und zur ATTENTION-AD-Studie weiterhin evaluieren, um sie in zukünftigen internationalen Konferenzen vorzustellen.
Anavex Life Sciences (NASDAQ:AVXL) نشرت نتائج تجربتها من المرحلة IIb/III لدواء بلاركامسين الفموي لعلاج مرض الزهايمر في مراحله المبكرة. أظهرت الدراسة أن الدواء الفموي بعدة قرص يومياً قد أظهر فعالية سريرية كبيرة وتحسين درجات جودة الحياة QoL-AD لدى المرضى.
باستخدام نهج الطب الدقيق، استفاد العلاج حتى 70% من المشاركين المصابين بالزهايمر مع حجم تأثير غير مسبوق. أظهر الدواء فعالية سريرية متفوقة مقارنة بالعلاجات المعتمدة، مع ملف سلامة قوي وبدون حاجة لمراقبة MRI روتينية.
تتوفر النتائج حالياً على medRxiv كمسودة قبل النشر وتُقدَّم للنشر في مجلة طبية مقومة بمراجعة الأقران. ستواصل الشركة تقييم بيانات تجربة Phase IIb/III لمرض الزهايمر المبكر وتجربة ATTENTION-AD للمشاركات المستقبلية في المؤتمرات الدولية.
Anavex Life Sciences (NASDAQ:AVXL) 已公布其口服 blarcamesine 针对早期阿尔茨海默病治疗的 Phase IIb/III 试验结果。研究表明，该每天一次口服药物在患者中呈现 显著的临床疗效并改善 QoL-AD 生活质量评分。
采用 精准医疗（Precision Medicine） 方法，治疗使多达 70% 的阿尔茨海默病参与者获益，效应大小空前。该药显示 优于已获批治疗的临床疗效，安全性强且无需常规 MRI 监测。
结果目前以预印本形式在 medRxiv 公布，正在提交给同行评审的医学期刊。公司将继续评估早期阿尔茨海默病的 Phase IIb/III 以及 ATTENTION-AD 试验数据，以便在国际会议上进行未来展示。
- Treatment benefited up to 70% of Alzheimer's disease participants using Precision Medicine approach
- Demonstrated superior clinical efficacy compared to approved therapies
- Strong safety profile with no need for routine MRI monitoring
- Once-daily oral dosing format enhances practical implementation
- Significant improvement in Quality of Life scores for patients
- Results are currently only published as a preprint, pending peer review
- Investigational drug status with no guarantee of regulatory approval
Insights
Anavex's blarcamesine shows strong clinical efficacy in Alzheimer's trial with precision medicine approach benefiting up to 70% of early-stage patients.
The Phase IIb/III trial results for blarcamesine represent a significant development in Alzheimer's treatment. The drug demonstrated significant improvements in Quality of Life (QoL-AD) scores that actually reversed the typical negative trajectory seen in Alzheimer's progression—an exceedingly rare outcome in AD clinical research.
What sets this approach apart is the precision medicine strategy that identified genetic biomarkers allowing up to ~70% of Alzheimer's patients to benefit with what's described as an "unprecedented effect size." This targeted approach contrasts with most AD treatments that show modest benefits across broader populations.
Particularly noteworthy is the once-daily oral tablet formulation, which offers substantial practical advantages over infusion-based therapies like monoclonal antibodies. The release also emphasizes no requirement for routine MRI monitoring—a significant safety and cost advantage over some existing therapies that require extensive monitoring for side effects like ARIA (Amyloid-Related Imaging Abnormalities).
The mechanism of action—enhancing the brain's autophagy (cellular cleaning) mechanism—represents a different approach from both traditional cholinesterase inhibitors and newer amyloid-targeting therapies. If validated, this could establish an entirely new therapeutic class for neurodegenerative conditions.
However, we should note this data is currently published on medRxiv, a preprint server, and is still awaiting peer review. While promising, these results require further validation through the formal scientific review process and additional confirmation in larger studies before clinical adoption.
Publication title: “Oral Blarcamesine Phase IIb/III Trial Confirms Identified Precision Medicine Patient Population – Significant Broad Clinical and Quality of Life Improvements for Early Alzheimer’s Disease Patients”
Significant improvement in self-assessed Quality of Life (QoL-AD) scores indicating a reversal of negative trajectory for Alzheimer's disease clinical trial participants was observed
Using a novel Precision Medicine approach, up to ~
Oral blarcamesine tablet, by enhancing the brain's autophagy internal clearing mechanism could alleviate significant medical and economic burden
Publication available on medRxiv, in submission to a peer-reviewed medical journal
NEW YORK, Sept. 30, 2025 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company focused on developing innovative treatments for Alzheimer's disease, Parkinson's disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome, and other central nervous system (CNS) disorders, today announced the publication “Oral Blarcamesine Phase IIb/III Trial Confirms Identified Precision Medicine Patient Population – Significant Broad Clinical and Quality of Life Improvements for Early Alzheimer’s Disease Patients” to be available online as a preprint at medRxiv, and in submission to a peer-reviewed medical journal.
“This publication of blarcamesine, a once-daily oral small molecule, is very exciting, with demonstrated superior clinical efficacy versus approved therapies and slowed neurodegeneration in early Alzheimer’s patients,” said Marwan Noel Sabbagh, MD, Professor of Neurology, and Chairman of the Anavex Scientific Advisory Board. “Coupled with a strong safety profile and no need for routine MRI monitoring, its unique mechanism of action supports a Precision Medicine approach—potentially benefiting up to
“I am very thrilled about the strong cognitive and functional improvements observed in Alzheimer’s patients—alongside enhanced Quality of Life and the favorable safety profile,” commented Professor of Neurology and Doctor in Neurosciences at the Memory Resources Research Center, the European Neurodegenerative Excellence Center of Montpellier University, France, Audrey Gabelle, MD, PhD and Member of the Anavex Scientific Advisory Board. “Blarcamesine’s targeted approach has the potential to significantly reduce both the medical and economic burden of the disease.”
“These findings highlight the potential of blarcamesine to reshape Alzheimer’s care, particularly within genetically defined populations,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “Its once-daily oral dosing and favorable safety profile make it a practical and scalable option for early-stage treatment across diverse healthcare settings. We believe this approach could simplify care delivery and expand access for patients navigating the complexities of Alzheimer’s disease.”
The publication is available at https://www.medrxiv.org/content/10.1101/2025.09.27.25336656v1.full.pdf.
Anavex will continue to evaluate the Phase IIb/III early Alzheimer’s disease and ATTENTION-AD trial data, which will be published and presented at international AD conferences.
This release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that any investigational uses of such product will successfully complete clinical development or gain health authority approval.
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of novel therapeutics for the treatment of neurodegenerative, neurodevelopmental, and neuropsychiatric disorders, including Alzheimer's disease, Parkinson's disease, schizophrenia, Rett syndrome, and other central nervous system (CNS) diseases, pain, and various types of cancer. Anavex's lead drug candidate, ANAVEX®2-73 (blarcamesine), has successfully completed a Phase 2a and a Phase 2b/3 clinical trial for Alzheimer's disease, a Phase 2 proof-of-concept study in Parkinson's disease dementia, and both a Phase 2 and a Phase 3 study in adult patients and one Phase 2/3 study in pediatric patients with Rett syndrome. ANAVEX®2-73 is an orally available drug candidate designed to restore cellular homeostasis by targeting SIGMAR1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer's disease. ANAVEX®2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson's Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX®2-73 for the treatment of Parkinson's disease. We believe that ANAVEX®3-71, which targets SIGMAR1 and M1 muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer's disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid, and tau pathologies. In preclinical trials, ANAVEX®3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at www.anavex.com. You can also connect with the Company on Twitter, Facebook, Instagram, and LinkedIn.
Forward-Looking Statements
Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company’s most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.
For Further Information:
Anavex Life Sciences Corp.
Research & Business Development
Toll-free: 1-844-689-3939
Email: info@anavex.com
Investors:
Andrew J. Barwicki
Investor Relations
Tel: 516-662-9461
Email: andrew@barwicki.com
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FAQ
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