Anavex Life Sciences Announces Oral Blarcamesine Cognitive Resilience Results Approximating Normal Aging in New Precision Medicine Clinical Data from Phase IIb/III Alzheimer’s Disease Trial
Anavex Life Sciences (NASDAQ:AVXL) has announced groundbreaking results from its Phase IIb/III Alzheimer's disease trial for oral blarcamesine. The drug demonstrated remarkable cognitive stabilization in early Alzheimer's patients, with outcomes approaching normal aging profiles. Key findings include an 84.7% reduction in cognitive decline compared to placebo on the ADAS-Cog13 endpoint at 48 weeks.
The study's Precision Medicine population showed minimal decline, with a 48-week change from baseline of 0.853 on ADAS-Cog13 and 0.465 on CDR-SB, comparable to prodromal aging adults. The treatment could benefit up to 70% of early Alzheimer's patients through its novel mechanism of autophagy restoration via SIGMAR1 activation, representing a significant advancement in individualized Alzheimer's care.
Anavex Life Sciences (NASDAQ:AVXL) ha reso noti risultati rivoluzionari dal suo trial di fase IIb/III sull'Alzheimer per l'antidoto orale blarcamesine. Il farmaco ha mostrato una notevole stabilizzazione cognitiva nei pazienti nelle fasi iniziali della malattia, con performance vicine a quelle dell'invecchiamento normale. Tra i punti chiave, un riduzione dell'84,7% del declino cognitivo rispetto al placebo sull'endpoint ADAS-Cog13 a 48 settimane.
La popolazione di Precision Medicine dello studio ha evidenziato un declino minimo, con una variazione a 48 settimane dal basale di 0,853 sull'ADAS-Cog13 e di 0,465 sul CDR-SB, valori paragonabili agli adulti in età prodromica. Il trattamento potrebbe avvantaggiare fino al 70% dei pazienti con Alzheimer precoce grazie al suo nuovo meccanismo di ripristino dell'autofagia tramite attivazione del SIGMAR1, rappresentando un importante passo avanti nella cura personalizzata dell'Alzheimer.
Anavex Life Sciences (NASDAQ:AVXL) ha anunciado resultados pioneros de su ensayo de fase IIb/III en Alzheimer con la administración oral de blarcamesina. El fármaco demostró una notable estabilización cognitiva en pacientes con Alzheimer temprano, con resultados cercanos a los del envejecimiento normal. Entre los hallazgos clave, una reducción del 84,7% del deterioro cognitivo frente a placebo en el endpoint ADAS-Cog13 a las 48 semanas.
La población de Medicina de Precisión del estudio mostró un deterioro mínimo, con un cambio a las 48 semanas desde la línea base de 0,853 en ADAS-Cog13 y de 0,465 en CDR-SB, valores comparables a los de adultos en etapa prodrómica. El tratamiento podría beneficiar hasta al 70% de los pacientes con Alzheimer temprano mediante su novedoso mecanismo de restauración de la autofagia por activación de SIGMAR1, representando un avance significativo en la atención individualizada del Alzheimer.
Anavex Life Sciences (NASDAQ:AVXL)는 경구용 블라르카메신에 대한 알츠하이머병 2상/3상 임상시험의 획기적인 결과를 발표했습니다. 이 약물은 초기 알츠하이머 환자에서 인지 기능의 현저한 안정화를 보였으며, 결과는 정상 노화 수준에 근접했습니다. 주요 결과로는 48주 시점 ADAS-Cog13 평가에서 위약 대비 인지 저하 84.7% 감소가 포함됩니다.
정밀의학 집단에서는 감소 폭이 거의 없었으며, 기준선 대비 48주 변화가 ADAS-Cog13에서 0.853, CDR-SB에서 0.465로 전구기 성인의 값과 유사했습니다. 이 치료법은 SIGMAR1 활성화를 통한 오토파지 회복이라는 새로운 기전을 통해 초기 알츠하이머 환자의 최대 70%까지 혜택을 줄 수 있어 맞춤형 알츠하이머 치료에 중요한 진전을 의미합니다.
Anavex Life Sciences (NASDAQ:AVXL) a annoncé des résultats révolutionnaires de son essai de phase IIb/III sur la blarcamesine orale pour la maladie d'Alzheimer. Le médicament a montré une stabilisation cognitive remarquable chez des patients en phase précoce, avec des résultats proches du profil du vieillissement normal. Les points clés incluent une réduction de 84,7% du déclin cognitif par rapport au placebo sur l'endpoint ADAS-Cog13 à 48 semaines.
La population de médecine de précision de l'étude a présenté un déclin minimal, avec un changement à 48 semaines depuis la valeur de référence de 0,853 sur l'ADAS-Cog13 et de 0,465 sur le CDR-SB, comparable aux adultes en phase prodromique. Le traitement pourrait bénéficier jusqu'à 70% des patients atteints d'Alzheimer précoce grâce à son mécanisme novateur de restauration de l'autophagie via l'activation de SIGMAR1, représentant une avancée significative dans les soins individualisés contre l'Alzheimer.
Anavex Life Sciences (NASDAQ:AVXL) hat bahnbrechende Ergebnisse aus seiner Phase-IIb/III-Studie zu oral verabreichtem Blarcamesin bei Alzheimer bekanntgegeben. Das Präparat zeigte eine bemerkenswerte kognitive Stabilisierung bei Patienten im Frühstadium von Alzheimer, mit Ergebnissen, die dem normalen Alterungsprofil nahekommen. Zu den wichtigsten Befunden zählt eine Reduktion des kognitiven Abbaus um 84,7% gegenüber Placebo beim ADAS-Cog13-Endpunkt nach 48 Wochen.
Die Precision-Medicine-Population der Studie verzeichnete nur einen minimalen Rückgang, mit einer Veränderung nach 48 Wochen gegenüber dem Ausgangswert von 0,853 beim ADAS-Cog13 und 0,465 beim CDR-SB, vergleichbar mit prodomalen Erwachsenen. Durch seinen neuen Wirkmechanismus der Wiederherstellung der Autophagie über SIGMAR1-Aktivierung könnte die Behandlung bis zu 70% der Patienten im frühen Alzheimerstadium zugutekommen und stellt einen bedeutenden Fortschritt in der individualisierten Alzheimer-Therapie dar.
- None.
- Results limited to early-stage Alzheimer's disease patients
- Treatment effectiveness depends on specific genetic profiles
- Drug still in investigational phase without regulatory approval
Insights
Anavex's oral Alzheimer's drug shows remarkable 84.7% reduction in cognitive decline vs placebo, potentially benefiting ~70% of patients through precision medicine.
Anavex's Phase IIb/III trial results for oral blarcamesine in early Alzheimer's disease (AD) represent a significant breakthrough in the AD treatment landscape. The data demonstrates the 30mg dose produced an 84.7% reduction in cognitive decline compared to placebo on the ADAS-Cog13 primary endpoint at 48 weeks. Most remarkably, patients in the precision medicine cohort showed cognitive decline patterns closer to normal aging rather than typical AD progression.
The cognitive stabilization is particularly compelling: the blarcamesine treatment group showed only a 0.853 point change on ADAS-Cog13 compared to 5.592 points decline in the placebo group. Similarly, on the CDR-SB scale, patients showed minimal progression (0.465 points) comparable to prodromal aging patterns. These results are unprecedented for an oral AD therapy.
The mechanism of action differentiates blarcamesine from other AD treatments. Rather than targeting amyloid directly, it activates SIGMAR1 to restore autophagy - an upstream approach that addresses the disease's fundamental cellular dysfunction. This approach appears particularly effective in patients with specific genetic profiles (ABCLEAR3 population), potentially benefiting up to ~70% of the global AD population.
What's particularly promising is the combination of efficacy with convenience - as an oral, once-daily medication, blarcamesine could significantly improve treatment accessibility compared to infusion-based therapies. This could transform the early AD treatment paradigm, especially given the favorable safety profile mentioned, though specific safety data wasn't detailed in this release.
While these results are impressive, it's important to note they come from a defined precision medicine population rather than the total intent-to-treat group, indicating genetic testing would be necessary to identify optimal candidates for treatment.
New clinical Precision Medicine population 48-week data demonstrates unprecedented cognitive stabilization in early Alzheimer’s disease
Cognitive outcomes observed in the oral blarcamesine 30 mg Precision Medicine cohort move toward normal aging profiles across validated clinical scales, supporting its relevance in early-stage Alzheimer’s care
Blarcamesine could represent a novel treatment option for up to ~
NEW YORK, Sept. 09, 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, announced today the latest findings for blarcamesine, an oral small molecule for the potential treatment of early Alzheimer’s disease (AD).
On all standard scales for measuring Alzheimer’s disease and cognitive decline, after 48 weeks, the defined Precision Medicine population ABCLEAR31 consisting of early AD patients with confirmed and progressed pathology taking 30 mg once-daily oral blarcamesine demonstrated barely detectable decline. This was comparable to minimally perceptible decline in prodromal (pre-dementia) aging adults.
- For ADAS-Cog13 (the standardized neuropsychological test used in Alzheimer's disease research to measure cognitive function and track disease progression), blarcamesine showed a 48-week change from baseline of 0.853 compared to ~1 point typical annual decline in prodromal (pre-dementia) aging adults.
- For CDR-SB (Clinical Dementia Rating, standard test that evaluates the severity of cognitive impairment and functional decline in individuals with AD), blarcamesine demonstrated a change from baseline of 0.465, aligning with the 0-0.5 point annual range seen in prodromal aging.
These data are similar to referenced barely detectable prodromal AD decline, in spite of the more advanced stage of AD impairment at baseline of the blarcamesine population.2,3
| Baseline | |||||
| ADAS-Cog13, mean [SD] | CDR-SB, mean [SD] | ||||
| Blarcamesine ABCLEAR3 population* | 28.4 [9.10] | 4.02 [1.853] | |||
| Prodromal population3 | 23.22 [6.79] | 2.11 [0.97] | |||
| Change from Baseline | |||||
| ADAS-Cog13 | CDR-SB | ||||
| Blarcamesine ABCLEAR3 population*, 48 weeks | 0.853 | 0.465 | |||
| Prodromal population, 52 weeks3 | 1.26 | 0.56 | |||
| * ABCLEAR3 = Alzheimer’s Blarcamesine Cognition Efficacy and Resilience Genes, 30 mg Group | |||||
Oral blarcamesine treatment over 48 weeks in a Precision Medicine population, including up to ~
In comparison, the respective placebo group in the ANAVEX2-73-AD-004 Phase IIb/III ABCLEAR34 population, ADAS-Cog13 LS mean declined by 5.592 points resulting in an ADAS-Cog13 LS mean difference of -4.739 [
"Given the strong interest in living a longer life without Alzheimer’s dementia, novel therapeutic directions are required. Blarcamesine’s mechanism of autophagy restoration via SIGMAR1 activation addresses a non-amyloid, upstream target, representing such a highly transformative clinical innovation,” said Marwan Noel Sabbagh, MD, Professor of Neurology, and Chairman of the Anavex Scientific Advisory Board. “We are thrilled that today's findings show the superior effect of blarcamesine Precision Medicine population shifting the previous cognitive decline of Alzheimer’s disease to barely detectable decline resembling prodromal older cognitive decline.”
“Today’s data exemplify how precision medicine is transforming Alzheimer’s care – oral blarcamesine enables a shift from fit-for-all approaches to individualized, tailored therapy. We are finally moving toward measurable individualized benefit with a high confidence of the meaningful clinical effectiveness of blarcamesine at 30 mg dose level and a 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, Audrey Gabelle, MD, PhD and Member of the Anavex Scientific Advisory Board. “Ensuring equitable access to effective Alzheimer’s treatments is not just a scientific goal, but a societal responsibility. Blarcamesine combines scientific precision with a bold commitment to equity by offering easy-to-implement, safe, scalable and equitable access of care for people living with Alzheimer’s.”
The mechanistic confirmation that blarcamesine restores impaired autophagy through SIGMAR1 activation by acting upstream of amyloid and tau pathologies at the molecular level was previously established both in vitro and in vivo. Specifically, studies demonstrated enhanced autophagic flux in human cells and in C. elegans as well as increased proteostasis capacity, ultimately ameliorating paralysis caused by protein aggregation in C. elegans.5,6
A clinical Precision Medicine approach confirmed that the prespecified SIGMAR1 non-mutated population, termed ABCLEAR17, which represents up to ~
“Our company’s goal is to provide potential high-quality options for patients with Alzheimer’s disease. Today’s new clinical efficacy data update from our Phase IIb/III trial is critical, as it adds contemporary context to Precision Medicine data showing strong protection from Alzheimer’s disease with an oral once daily potential solution with the aim to alleviate significant medical and economic burden,” commented Juan Carlos Lopez-Talavera, MD, PhD, Head of Research and Development of Anavex. “We’re excited by the meaningful improvements seen with blarcamesine, confirmed by results of patients identified and confirmed through Precision Medicine.”
“We believe these data reinforce the opportunity to potentially transform the treatment paradigm for individuals living with Alzheimer’s disease. In this Precision Medicine population blarcamesine’s once-daily oral administration may support broader implementation across diverse care settings, offering a patient-friendly and scalable option for early-stage Alzheimer’s disease,” said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. “Blarcamesine’s combination of efficacy and safety may streamline treatment pathways in the complex Alzheimer’s ecosystem, paving the way for broader adoption and equitable access among patients.”
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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1 ABCLEAR3 = Alzheimer’s Blarcamesine Cognition Efficacy and Resilience gene variants non-carrier population (SIGMAR1 wild type [WT]/COL24A1 wild type [WT]).
2 MMSE, mean [SD] was 23.5 [3.21] for blarcamesine ABCLEAR3 population and 25.68 [2.2] for Prodromal AD population.
3 McDougall, F et al. “Psychometric Properties of the Clinical Dementia Rating - Sum of Boxes and Other Cognitive and Functional Outcomes in a Prodromal Alzheimer's Disease Population.” JPAD. vol. 8,2 (2021): 151-160.
4 ABCLEAR3 = Alzheimer’s Blarcamesine Cognition Efficacy and Resilience gene variants non-carrier population (SIGMAR1 wild type [WT]/COL24A1 wild type [WT]).
5 Christ, M G et al. “Sigma-1 Receptor Activation Induces Autophagy and Increases Proteostasis Capacity In Vitro and In Vivo.” Cells vol. 8,3 211. 2 Mar. 2019.
6 Baeken, M W et al. “Conserved LIR-specific interaction of Sigma-1 receptor and GABARAP.” iScience Volume 28, Issue 9, 2025, 113287.
7 ABCLEAR1 = Alzheimer’s Blarcamesine Cognition Efficacy and Resilience gene variant non-carrier population (SIGMAR1 wild type [WT])
FAQ
What are the key results of Anavex's Phase IIb/III Alzheimer's trial for blarcamesine?
How does AVXL's blarcamesine work differently from other Alzheimer's treatments?
What percentage of Alzheimer's patients could potentially benefit from AVXL's blarcamesine?
What are the administration benefits of Anavex's blarcamesine for Alzheimer's patients?
What cognitive scales were used to measure blarcamesine's effectiveness in the AVXL trial?
