Anavex Life Sciences Announces Peer-Reviewed Publication of Oral Blarcamesine Phase IIb/III Data in The Journal of Prevention of Alzheimer’s Disease
Anavex Life Sciences announced the peer-reviewed publication of Phase IIb/III trial results for oral blarcamesine (ANAVEX®2-73) in The Journal of Prevention of Alzheimer's Disease. The study demonstrated that once-daily oral blarcamesine significantly slowed clinical progression by 36.3% at 48 weeks in early Alzheimer's Disease patients, and by 49.8% in the SIGMAR1 wild-type gene group on the ADAS-Cog13 cognitive endpoint.
The drug showed a favorable safety profile with no associated neuroimaging adverse events. Blarcamesine works through SIGMAR1 activation, inducing autophagy and restoring cellular homeostasis. The treatment could represent a novel approach for early Alzheimer's disease, potentially serving as a complementary or alternative option to injectable anti-beta amyloid drugs. The data is currently under review by the EMA as part of an MAA filing.
Anavex Life Sciences ha annunciato la pubblicazione peer-reviewed dei risultati della fase IIb/III dello studio clinico per il blarcamesine orale (ANAVEX®2-73) nella rivista The Journal of Prevention of Alzheimer’s Disease. Lo studio ha dimostrato che il blarcamesine orale assunto una volta al giorno ha notevolmente rallentato la progressione clinica del 36,3% dopo 48 settimane nei pazienti con Alzheimer precoce e del 49,8% nel gruppo con gene wild-type SIGMAR1, secondo il parametro cognitivo ADAS-Cog13.
Il farmaco ha mostrato un profilo di sicurezza favorevole senza eventi avversi associati all’imaging neuroimaging. Il blarcamesine agisce attraverso l'attivazione del SIGMAR1, inducendo l'autofagia e ripristinando l'omeostasi cellulare. Questo trattamento potrebbe rappresentare un approccio innovativo per l'Alzheimer precoce, potenzialmente fungendo da opzione complementare o alternativa ai farmaci anti-beta amiloide iniettabili. I dati sono attualmente sotto revisione da parte dell'EMA nell'ambito di una richiesta di autorizzazione all'immissione in commercio (MAA).
Anavex Life Sciences anunció la publicación revisada por pares de los resultados del ensayo de fase IIb/III del blarcamesina oral (ANAVEX®2-73) en The Journal of Prevention of Alzheimer's Disease. El estudio demostró que el blarcamesina oral administrado una vez al día ralentizó significativamente la progresión clínica en un 36,3% a las 48 semanas en pacientes con enfermedad de Alzheimer en etapa temprana, y en un 49,8% en el grupo con el gen wild-type SIGMAR1 en el punto final cognitivo ADAS-Cog13.
El fármaco mostró un perfil de seguridad favorable sin eventos adversos asociados a neuroimágenes. La blarcamesina actúa a través de la activación de SIGMAR1, induciendo autofagia y restaurando la homeostasis celular. Este tratamiento podría representar un enfoque novedoso para la enfermedad de Alzheimer temprana, potencialmente sirviendo como una opción complementaria o alternativa a los medicamentos anti-beta amiloide inyectables. Los datos están actualmente bajo revisión por la EMA como parte de una solicitud de autorización de comercialización (MAA).
Anavex Life Sciences는 경구용 블라르카메신(ANAVEX®2-73)의 IIb/III상 임상 시험 결과에 대한 동료 검토 출판을 발표했습니다. 이 연구는 초기가 Alzheimer 환자에서 블라르카메신을 하루에 한 번 투여했을 때, 48주 후에 임상 진행이 36.3% 느려진 것으로 나타났으며, SIGMAR1 와일드타입 유전자 그룹에서는 ADAS-Cog13 인지 Endpoint에서 49.8% 느려짐을 보였습니다.
이 약물은 신경영상학적 부작용이 없는 유리한 안전성 프로필을 보여주었습니다. 블라르카메신은 SIGMAR1 활성화를 통해 작용하며, 자가 포식을 유도하고 세포 항상성을 회복합니다. 이 치료법은 초기 알츠하이머병에 대한 새로운 접근법을 나타낼 수 있으며, 주사형 항베타 아밀로이드 약물에 대한 보완 또는 대안 옵션으로 기능할 수 있습니다. 데이터는 현재 MAA 제출의 일환으로 EMA에 의해 검토되고 있습니다.
Anavex Life Sciences a annoncé la publication par des pairs des résultats de l'essai de Phase IIb/III pour le blarcamesine orale (ANAVEX®2-73) dans The Journal of Prevention of Alzheimer’s Disease. L'étude a démontré que le blarcamesine oral pris une fois par jour a considérablement ralenti la progression clinique de 36,3% à 48 semaines chez les patients atteints de la maladie d'Alzheimer précoce, et de 49,8% dans le groupe de gènes SIGMAR1 de type sauvage sur le critère cognitif ADAS-Cog13.
Le médicament a montré un profil de sécurité favorable sans événements indésirables neuroimaging associés. Le blarcamesine agit par l'activation du SIGMAR1, induisant l'autophagie et rétablissant l'homéostasie cellulaire. Ce traitement pourrait représenter une approche innovante pour la maladie d'Alzheimer précoce, servant potentiellement d'option complémentaire ou alternative aux médicaments anti-bêta amyloïdes injectables. Les données sont actuellement en cours d'examen par l'EMA dans le cadre d'une demande d'autorisation de mise sur le marché (MAA).
Anavex Life Sciences gab die peer-reviewed Veröffentlichung der Ergebnisse der Phase IIb/III-Studien für das orale Blarcamesin (ANAVEX®2-73) im The Journal of Prevention of Alzheimer’s Disease bekannt. Die Studie zeigte, dass die einmal täglich verabreichte orale Blarcamesin die klinische Progression bei frühen Alzheimer-Patienten in 48 Wochen um 36,3% signifikant verlangsamte, und um 49,8% in der SIGMAR1 Wildtyp-Gruppierung beim ADAS-Cog13 kognitiven Endpoint.
Das Medikament zeigte ein günstiges Sicherheitsprofil ohne damit verbundene neuroimaging Nebenwirkungen. Blarcamesin wirkt durch die Aktivierung von SIGMAR1, induziert Autophagie und stellt die zelluläre Homöostase wieder her. Diese Behandlung könnte einen neuartigen Ansatz zur frühen Alzheimer-Krankheit darstellen und potenziell als komplementäre oder alternative Option zu injizierbaren Anti-beta-Amyloid-Arzneimitteln dienen. Die Daten werden derzeit von der EMA im Rahmen eines MAA-Antrags überprüft.
- Significant clinical progression slowdown of 36.3% at 48 weeks in general population
- Enhanced efficacy of 49.8% at 48 weeks in SIGMAR1 wild-type gene group
- Favorable safety profile with no neuroimaging adverse events
- MAA filing under review by EMA
- Oral administration advantage over injectable alternatives
- Drug still in investigational phase without regulatory approval
- Results to early Alzheimer's Disease patients
Insights
The publication of Phase IIb/III data for blarcamesine in JPAD represents a significant milestone in Alzheimer's disease treatment development. The data shows a 36.3% slowdown in clinical progression at 48 weeks, increasing to 49.8% in patients with the SIGMAR1 wild-type gene. This efficacy level compares favorably with existing treatments.
The mechanism of action targeting autophagy through SIGMAR1 activation is particularly noteworthy, as it addresses the disease process upstream of both amyloid beta and tau tangles. This novel approach could potentially offer better outcomes than current treatments that target downstream pathology.
Simple explanation: Think of Alzheimer's as a clogged drain. While current treatments try to unclog it (removing amyloid), blarcamesine helps prevent the clog from forming in the first place by maintaining proper cellular "cleaning" processes.
The peer-reviewed publication validates blarcamesine's potential market position with several key competitive advantages:
- Oral administration versus injectable competitors
- No requirement for routine MRI monitoring, reducing healthcare costs
- Potential for use as either standalone or complementary treatment
For AVXL, with a market cap of
Simple explanation: Imagine a new pain medication that works as well as current options but comes as a pill instead of a shot and doesn't need constant medical monitoring. That's what makes this drug special from a business perspective.
Blarcamesine potential novel oral treatment to target upstream Alzheimer’s disease pathology through autophagy enhancement
Impairment of autophagy precedes both amyloid beta and tau tangles, and therefore anticipates the neurodegenerative process in Alzheimer’s disease
NEW YORK, Jan. 15, 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 that The Journal of Prevention of Alzheimer’s Disease (JPAD) has published peer-reviewed detailed results from the Phase IIb/III study evaluating oral blarcamesine (ANAVEX®2-73) for the treatment of early Alzheimer’s Disease (AD).
Once daily oral blarcamesine, demonstrating a safety profile with no associated neuroimaging adverse events, significantly slowed clinical progression by
Oral once daily blarcamesine could represent a novel treatment in early Alzheimer’s disease and be complementary or alternative to injectable anti-beta amyloid drugs.
“The Alzheimer’s disease community has been actively pursuing new medicines for decades. To have data like these published in JPAD gives us energy and hope. We are now seeing in the data what we suspected about blarcamesine for a long time – that it has the potential to make a clinical difference for people living with early Alzheimer’s disease,” said senior author behavioral neurologist Professor Dr. Marwan Noel Sabbagh MD, Chairman of the Scientific Advisory Board. “The advantage of blarcamesine lies in its small oral formulation, which offers clinical benefits for cognition and neurodegeneration. Its ease of administration and favorable safety profile make it an appealing option.”
Data from the Phase IIb/III trial demonstrated oral once daily blarcamesine pre-specified clinical efficacy through upstream SIGMAR1 activation. SIGMAR1 is an integral membrane protein which activates an upstream compensatory process: Blarcamesine induces autophagy through SIGMAR1 activation resulting in restoring cellular homeostasis.
“The results from the Phase IIb/III blarcamesine study show real promise for patients living with early Alzheimer’s disease,” said lead author Associate Professor Dr. Stephen Macfarlane, FRANZCP, Head of Clinical Services at the Dementia Centre, HammondCare and Principal Investigator. “Early Alzheimer’s disease can progress swiftly, and the potential for blarcamesine to target on a constitutional level of the pathology should bring hope and excitement to persons living with Alzheimer’s disease and those of us who care for them.”
Impaired autophagy precedes both amyloid beta and tau tangles, and therefore anticipates the neurodegenerative process in Alzheimer’s disease.1 Hence, stabilization or restoration of autophagy can be seen as an early preventative measure countering the Alzheimer’s disease pathology.
“Publication of the pivotal placebo-controlled Phase IIb/III blarcamesine early Alzheimer’s disease trial allows for a broader dissemination of these important data and highlights blarcamesine’s impressive and consistent clinical profile that has led to meaningful results for these patients,” said Juan Carlos Lopez-Talavera, MD, PhD, Head of Research and Development of Anavex. “These data serve as the foundation for the MAA filing that is currently being reviewed by the EMA. We are aiming to potentially advance an important medicine to patients in need.”
Blarcamesine, a small molecule administered orally once daily, demonstrated clinically meaningful improvement over 48 weeks with primary endpoint ADAS-Cog13 score being larger than 2 points.2 This suggests superior numerical clinical efficacy compared to approved therapies while also slowing neurodegeneration in early Alzheimer’s disease patients. Blarcamesine’s safety profile indicates not requiring routine MRI monitoring, and given its differentiated mechanism of action, oral blarcamesine could represent a novel treatment that could be complementary or an alternative to injectable anti-beta amyloid monoclonal antibody drugs.
“The peer-reviewed publication of these data underscores the significance of the findings for both the scientific community and those focused on Alzheimer's disease,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “Alzheimer's disease is a highly complex condition, and this dataset plays a crucial role in advancing our understanding of the Phase IIb/III results. We are grateful to the dedication from participants, their families, and the sites for taking part in this important study.”
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.
The publication may be accessed here.
About Alzheimer’s Disease
There are an estimated 7 million people in Europe with Alzheimer’s disease, a number expected to double by 2030, according to the European Brain Council.3 The World Health Organization (WHO) estimated the cost in Europe of caring for people with dementia, including Alzheimer's disease, at
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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1 Christ MG, Clement AM, Behl C. The Sigma-1 Receptor at the Crossroad of Proteostasis, Neurodegeneration, and Autophagy. Trends Neurosci. 2020 Feb;43(2):79-81; Chen, J., He, HJ., Ye, Q. et al. Defective Autophagy and Mitophagy in Alzheimer’s Disease: Mechanisms and Translational Implications. Mol Neurobiol 58, 5289–5302 (2021).
2 Muir RT, Hill MD, Black SE, Smith EE. Minimal clinically important difference in Alzheimer's disease: Rapid review. Alzheimers Dement. 2024;20(5):3352-3363. doi:10.1002/alz.13770
3 https://www.braincouncil.eu/projects/rethinking-alzheimers-disease/
4 Jönsson L. The personal economic burden of dementia in Europe. Lancet Reg Health Eur. 2022 Jul 25;20:100472. doi: 10.1016/j.lanepe.2022.100472. PMID: 35910037; PMCID: PMC9326307.
5 World Health Organization (WHO); 2021. Global status report on the public health response to dementia.
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