Blarcamesine Receives EMA Filing Acceptance for Treatment of Alzheimer’s Disease
Anavex Life Sciences (NASDAQ: AVXL) announced that the European Medicines Agency (EMA) has accepted their Marketing Authorization Application (MAA) for blarcamesine, an oral treatment for Alzheimer's disease. The application is supported by data from the Phase IIb/III ANAVEX®2-73-AD-004 trial and its 144-week open-label extension study.
The once-daily oral medication demonstrated meaningful clinical improvement over 48 weeks, with the primary endpoint ADAS-Cog13 score exceeding 2 points. Blarcamesine works through SIGMAR1 activation, inducing autophagy to restore cellular homeostasis. The drug showed a favorable safety profile without requiring routine MRI monitoring, potentially offering an alternative or complement to injectable anti-beta amyloid antibody treatments.
Anavex Life Sciences (NASDAQ: AVXL) ha annunciato che l'Agenzia Europea per i Medicinali (EMA) ha accettato la loro Domanda di Autorizzazione all'Immissione in Commercio (MAA) per blarcamesine, un trattamento orale per la malattia di Alzheimer. La domanda è supportata dai dati dello studio clinico di Fase IIb/III ANAVEX®2-73-AD-004 e del suo studio di estensione open-label di 144 settimane.
Il farmaco orale, somministrato una volta al giorno, ha dimostrato un miglioramento clinico significativo dopo 48 settimane, con il punteggio del parametro primario ADAS-Cog13 che ha superato le 2 punti. Blarcamesine agisce attraverso l'attivazione di SIGMAR1, inducendo l'autofagia per ripristinare l'omeostasi cellulare. Il farmaco ha mostrato un profilo di sicurezza favorevole, senza richiedere un monitoraggio routinario con risonanza magnetica, offrendo potenzialmente un'alternativa o un complemento ai trattamenti iniettabili con anticorpi anti-beta amiloide.
Anavex Life Sciences (NASDAQ: AVXL) anunció que la Agencia Europea de Medicamentos (EMA) ha aceptado su Solicitud de Autorización de Comercialización (MAA) para blarcamesine, un tratamiento oral para la enfermedad de Alzheimer. La solicitud está respaldada por datos del ensayo clínico de Fase IIb/III ANAVEX®2-73-AD-004 y su estudio de extensión abierto de 144 semanas.
El medicamento oral, administrado una vez al día, demostró una mejora clínica significativa durante 48 semanas, con la puntuación del criterio principal ADAS-Cog13 superando las 2 puntos. Blarcamesine actúa mediante la activación de SIGMAR1, induciendo la autofagia para restaurar la homeostasis celular. El fármaco mostró un perfil de seguridad favorable sin requerir un monitoreo rutinario por resonancia magnética, lo que podría ofrecer una alternativa o complemento a los tratamientos inyectables con anticuerpos anti-beta amiloide.
Anavex Life Sciences (NASDAQ: AVXL)는 유럽 의약품청(EMA)이 알츠하이머병에 대한 경구 치료제 blarcamesine의 마케팅 승인 요청(MAA)을 수락했다고 발표했습니다. 이 요청은 Phase IIb/III ANAVEX®2-73-AD-004 시험 및 그 144주 공개 연장 연구의 데이터를 기반으로 하고 있습니다.
하루 한 번 복용하는 이 경구 약물은 48주 동안 의미 있는 임상 개선을 보여주었으며, 주요 지표인 ADAS-Cog13 점수가 2점을 초과했습니다. Blarcamesine은 SIGMAR1 활성화를 통해 작용하여 세포 항상성을 회복하기 위해 자가포식을 유도합니다. 이 약물은 정기적인 MRI 모니터링을 요구하지 않는 우수한 안전성 프로필을 나타내어, 주사형 항베타 아밀로이드 항체 치료의 대안 또는 보완책을 제공할 가능성이 있습니다.
Anavex Life Sciences (NASDAQ: AVXL) a annoncé que l'Agence européenne des médicaments (EMA) a accepté sa Demande d'Autorisation de Mise sur le Marché (MAA) pour blarcamesine, un traitement oral pour la maladie d'Alzheimer. La demande est soutenue par des données de l'essai ANAVEX®2-73-AD-004 de Phase IIb/III et de son étude d'extension en ouvert de 144 semaines.
Le médicament oral, administré une fois par jour, a montré une amélioration clinique significative sur 48 semaines, le score du critère principal ADAS-Cog13 dépassant 2 points. Blarcamesine agit par l'activation de SIGMAR1, induisant l'autophagie pour restaurer l'homéostasie cellulaire. Le médicament a montré un profil de sécurité favorable sans nécessiter de surveillance IRM de routine, offrant ainsi potentiellement une alternative ou un complément aux traitements injectables par anticorps anti-bêta-amyloïde.
Anavex Life Sciences (NASDAQ: AVXL) gab bekannt, dass die Europäische Arzneimittelagentur (EMA) ihren Antrag auf Marktzulassung (MAA) für blarcamesine, eine orale Behandlung für Alzheimer, akzeptiert hat. Der Antrag wird durch Daten aus der Phase-IIb/III-Studie ANAVEX®2-73-AD-004 und deren 144-wöchiger offener Verlängerungsstudie unterstützt.
Das einmal täglich verabreichte orale Medikament zeigte über 48 Wochen hinweg erhebliche klinische Verbesserungen, wobei der primäre Endpunkt ADAS-Cog13-Score um mehr als 2 Punkte übertroffen wurde. Blarcamesine wirkt durch die Aktivierung von SIGMAR1, was zur Induktion der Autophagie führt, um die zelluläre Homöostase wiederherzustellen. Das Medikament wies ein günstiges Sicherheitsprofil auf, ohne dass routinemäßige MRT-Überwachungen erforderlich sind, was potenziell eine Alternative oder Ergänzung zu injizierbaren Anti-Beta-Amyloid-Antikörperbehandlungen bietet.
- EMA acceptance of Marketing Authorization Application for blarcamesine
- Clinical trials showed meaningful improvement over 48 weeks with ADAS-Cog13 score >2 points
- Favorable safety profile without need for routine MRI monitoring
- Once-daily oral administration offers advantage over injectable alternatives
- Product still in investigational phase with no guarantee of approval
- Efficacy and safety conclusions not yet finalized
Insights
The EMA's acceptance of blarcamesine's MAA represents a important milestone for Anavex Life Sciences. The drug's unique mechanism targeting SIGMAR1 activation and autophagy enhancement addresses upstream Alzheimer's pathology, potentially offering advantages over existing treatments.
The Phase IIb/III trial data shows an improvement in ADAS-Cog13 scores of more than 2 points over 48 weeks, suggesting meaningful clinical efficacy. Notably, the oral administration and favorable safety profile without requiring routine MRI monitoring could provide significant advantages over injectable amyloid antibodies, potentially improving patient compliance and reducing healthcare burden.
For investors, key differentiators include: 1) The drug's potential as both a standalone and complementary treatment option, 2) The extensive data package including a 5-year Phase 2a trial and 3) The broader market opportunity in early Alzheimer's disease patients who may prefer oral administration over injections.
The EMA filing acceptance significantly enhances Anavex's market position in the $12+ billion global Alzheimer's treatment market. With a market cap of
Three key market advantages stand out: First, the oral administration route addresses a major market gap, as current approved antibody treatments require injections. Second, the favorable safety profile without MRI monitoring requirements could reduce healthcare costs. Third, the drug's mechanism targeting early-stage disease opens a larger potential patient pool compared to late-stage treatments.
The timing aligns with growing market demand for more accessible Alzheimer's treatments, particularly as healthcare systems seek cost-effective alternatives to expensive antibody therapies.
Blarcamesine: Potential novel oral treatment to target upstream Alzheimer’s disease pathology through autophagy enhancement
Submission based on favorable ANAVEX®2-73-AD-004 trial results in patients with early Alzheimer’s disease
Once daily oral administration of blarcamesine meaningfully slowed clinical decline in early Alzheimer's disease patients, demonstrating a favorable safety profile with no associated neuroimaging adverse events
NEW YORK, Dec. 23, 2024 (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 European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for blarcamesine (ANAVEX®2-73), an investigational drug for the treatment of Alzheimer’s disease. The Company is looking forward to working with the EMA.
Blarcamesine is an orally once daily administered small molecule for the potential treatment of patients with Alzheimer’s disease.
“The EMA filing acceptance for blarcamesine to review the Marketing Authorization Application potentially brings us a step closer offering broader patient access to a new treatment option in Europe, and we look forward to continued engagement with the EMA,” said Juan Carlos Lopez-Talavera, MD, PhD, Head of Research and Development of Anavex. “Through our clinical development program, we have seen that blarcamesine has the potential to slow the progression of this relentless and ultimately fatal disease.”
The MAA is supported by data from the randomized, double-blind, placebo-controlled Phase IIb/III, ANAVEX®2-73-AD-004 trial and it’s up to 144 week open-label-extension (OLE) ATTENTION-AD ANAVEX®2-73-AD-EP-004 trial investigating blarcamesine in early Alzheimer’s disease. Former study was recently accepted in a peer-reviewed medical journal with focus on Alzheimer’s disease titled, “Blarcamesine for the treatment of Early Alzheimer’s Disease: Results from the ANAVEX2-73-AD-004 Phase IIB/III trial.”
Strengthening the submission is the 265 week (~5-year) Phase 2a trial investigating blarcamesine in mild-to-moderate Alzheimer’s disease, with interim preliminary data published in the peer-reviewed medical journal, Alzheimer’s & Dementia, titled: “A precision medicine framework using artificial intelligence for the identification and confirmation of genomic biomarkers of response to an Alzheimer's disease therapy: Analysis of the blarcamesine (ANAVEX2-73) Phase 2a clinical study.”
“We look forward to working with the EMA and thank the team at Anavex for their work on the submission,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “We are committed to improving patient access to novel and accessible oral medicines.”
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.
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.
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 AD 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.
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 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
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.
FAQ
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