Results from Anavex Life Sciences Landmark Phase IIb/III Trial of Blarcamesine Presented at Alzheimer's Association Conference
Anavex Life Sciences presented results from their Phase IIb/III trial of blarcamesine (ANAVEX®2-73) for early Alzheimer's disease at the 2024 Alzheimer's Association Conference. The oral, once-daily treatment significantly slowed clinical decline by 38.5% (50 mg) and 34.6% (30 mg) compared to placebo over 48 weeks. Key findings include:
1. Significant improvement in the primary cognitive endpoint (ADAS-Cog13)
2. Positive trends in functional measures (ADCS-ADL)
3. Significant results in the key secondary composite endpoint (CDR-SB)
4. Reduced brain atrophy in key regions
5. Good safety profile with no neuroimaging adverse events
Anavex plans to submit for EMA approval in Q4 2024. The company believes blarcamesine's oral administration and safety profile could provide broader access to early Alzheimer's patients.
Anavex Life Sciences ha presentato i risultati del loro studio clinico di Fase IIb/III su blarcamesina (ANAVEX®2-73) per la malattia di Alzheimer in fase iniziale durante la Conferenza dell'Associazione Alzheimer 2024. Il trattamento orale, da assumere una volta al giorno, ha significativamente ridotto il declino clinico del 38,5% (50 mg) e del 34,6% (30 mg) rispetto al placebo nel corso di 48 settimane. I risultati principali includono:
1. Miglioramento significativo nel principale parametro cognitivo (ADAS-Cog13)
2. Tendenze positive nelle misure funzionali (ADCS-ADL)
3. Risultati significativi nel principale endpoint secondario composito (CDR-SB)
4. Riduzione dell'atrofia cerebrale in aree chiave
5. Buon profilo di sicurezza senza eventi avversi nella neuroimaging
Anavex prevede di richiedere l'. L'azienda crede che la somministrazione orale di blarcamesina e il suo profilo di sicurezza potrebbero fornire un accesso più ampio ai pazienti con Alzheimer in fase iniziale.
Anavex Life Sciences presentó los resultados de su ensayo clínico de Fase IIb/III sobre blarcamesina (ANAVEX®2-73) para la enfermedad de Alzheimer en etapa temprana en la Conferencia de la Asociación de Alzheimer 2024. El tratamiento oral, que se toma una vez al día, retrasó significativamente el deterioro clínico en un 38,5% (50 mg) y 34,6% (30 mg) en comparación con el placebo durante 48 semanas. Los hallazgos clave incluyen:
1. Mejora significativa en el principal punto de referencia cognitivo (ADAS-Cog13)
2. Tendencias positivas en medidas funcionales (ADCS-ADL)
3. Resultados significativos en el punto de referencia secundario compuesto clave (CDR-SB)
4. Reducción de la atrofia cerebral en regiones clave
5. Buen perfil de seguridad sin eventos adversos en neuroimágenes
Anavex planea solicitar la aprobación de la EMA en el cuarto trimestre de 2024. La empresa cree que la administración oral de blarcamesina y su perfil de seguridad podrían proporcionar un acceso más amplio a los pacientes con Alzheimer en etapa temprana.
아나벡스 생명과학(Anavex Life Sciences)는 2024년 알츠하이머 협회 회의에서 조기 알츠하이머병을 위한 블라르카메신(blarcamesine, ANAVEX®2-73)의 IIb/III상 시험 결과를 발표했습니다. 하루에 한 번 복용하는 경구 치료는 48주 동안 위약에 비해 임상적 악화를 38.5%(50mg) 및 34.6%(30mg) 만큼 유의미하게 지연시켰습니다. 주요 발견 사항은 다음과 같습니다:
1. 주요 인지 지표(ADAS-Cog13)의 유의미한 개선
2. 기능성 측정치(ADCS-ADL)에서의 긍정적인 경향
3. 주요 이차 복합 지표(CDR-SB)에서의 유의미한 결과
4. 주요 영역에서의 뇌 위축 감소
5. 신경영상 부작용이 없는 좋은 안전성 프로필
아나벡스는 2024년 4분기 EMA 승인을 신청할 계획입니다. 회사는 블라르카메신의 경구 투여와 안전성 프로필이 조기 알츠하이머 환자들에게 더 넓은 접근성을 제공할 수 있을 것으로 믿고 있습니다.
Anavex Life Sciences a présenté les résultats de son essai clinique de Phase IIb/III sur blarcamesine (ANAVEX®2-73) pour la maladie d'Alzheimer précoce lors de la Conférence de l'Association Alzheimer 2024. Le traitement oral, à prendre une fois par jour, a significativement ralenti le déclin clinique de 38,5 % (50 mg) et de 34,6 % (30 mg) par rapport au placebo sur 48 semaines. Les résultats clés comprennent :
1. Amélioration significative du principal critère cognitif (ADAS-Cog13)
2. Tendances positives dans les mesures fonctionnelles (ADCS-ADL)
3. Résultats significatifs dans le critère composite secondaire clé (CDR-SB)
4. Réduction de l'atrophie cérébrale dans des régions clés
5. Bon profil de sécurité sans événements indésirables en neuroimagerie
Anavex prévoit de demander l'. La société estime que l'administration orale de blarcamesine et son profil de sécurité pourraient offrir un accès plus large aux patients atteints d'Alzheimer précoce.
Anavex Life Sciences präsentierte die Ergebnisse ihrer Phase IIb/III-Studie zu blarcamesine (ANAVEX®2-73) bei frühzeitigem Alzheimer während der Alzheimer Association Conference 2024. Die orale Behandlung, die einmal täglich eingenommen wird, verzögerte den klinischen Rückgang signifikant um 38,5% (50 mg) und 34,6% (30 mg) im Vergleich zu Placebo über einen Zeitraum von 48 Wochen. Die wichtigsten Ergebnisse umfassen:
1. Signifikante Verbesserung des primären kognitiven Endpunkts (ADAS-Cog13)
2. Positive Trends bei funktionalen Messungen (ADCS-ADL)
3. Signifikante Ergebnisse im wichtigen sekundären zusammengesetzten Endpunkt (CDR-SB)
4. Verminderte Gehirnatrophie in wichtigen Regionen
5. Gutes Sicherheitsprofil ohne neuroimaging bedingte unerwünschte Ereignisse
Anavex plant, im 4. Quartal 2024 die EMA-Zulassung zu beantragen. Das Unternehmen ist der Ansicht, dass die orale Verabreichung von blarcamesine und das Sicherheitsprofil einen breiteren Zugang für frühzeitig Alzheimer-Patienten ermöglichen könnten.
- Blarcamesine significantly slowed clinical decline by 38.5% (50 mg) and 34.6% (30 mg) vs placebo at 48 weeks
- Primary cognitive endpoint ADAS-Cog13 showed significant improvement for both 50 mg and 30 mg doses
- Key secondary endpoint CDR-SB significantly improved vs placebo in both dose groups
- Blarcamesine significantly slowed brain atrophy in key regions
- Good comparative safety profile with no neuroimaging adverse events like ARIA
- EMA submission expected in Q4 2024
- Functional co-primary endpoint ADCS-ADL did not reach significance at Week 48
- Common treatment-emergent adverse event of dizziness occurred in 35.8% of participants during titration
The results from Anavex Life Sciences' Phase IIb/III trial of blarcamesine for early Alzheimer's disease are highly significant. The drug demonstrated a 38.5% and 34.6% slowing of clinical progression at 48 weeks for the 50 mg and 30 mg doses, respectively, on the primary cognitive endpoint ADAS-Cog13. This is a substantial improvement over existing treatments.
Importantly, blarcamesine showed benefits on both amyloid-beta levels and brain volume, addressing two key pathological hallmarks of Alzheimer's. The drug significantly slowed brain atrophy, with impressive reductions of 37.6% in whole brain atrophy, 63.5% in total grey matter atrophy and 25.1% in lateral ventricle expansion. These neuroimaging results provide strong evidence of the drug's neuroprotective effects.
The safety profile is particularly noteworthy. Unlike some other Alzheimer's treatments, blarcamesine did not cause Amyloid-related imaging abnormalities (ARIA) or neurological tissue damage. This favorable safety profile, combined with oral administration, could make blarcamesine more accessible and easier to manage for patients.
While the functional co-primary endpoint (ADCS-ADL) didn't reach significance, this may be due to the scale's reduced sensitivity in early Alzheimer's. The significant improvement in the CDR-SB secondary endpoint provides additional support for the drug's efficacy.
Anavex's blarcamesine represents a potentially game-changing development in the Alzheimer's treatment landscape. As an orally administered small molecule, it offers significant advantages over injectable antibody therapies in terms of convenience and potential cost-effectiveness.
The drug's differentiated mechanism of action, focusing on improving autophagy through SIGMAR1 activation, sets it apart from current amyloid-targeting therapies. This novel approach could position blarcamesine as a complementary treatment to existing options, potentially expanding the therapeutic arsenal against Alzheimer's.
The planned EMA submission in Q4 2024 suggests a relatively rapid path to potential commercialization. If approved, blarcamesine could capture a significant share of the Alzheimer's market, estimated to be worth
However, investors should note that the functional co-primary endpoint did not reach significance, which could potentially complicate the regulatory process. The company's ability to address this in their submission will be crucial.
Overall, these results position Anavex as a strong contender in the highly competitive Alzheimer's drug development space, with potential for significant market impact if blarcamesine gains approval.
The results of this Phase IIb/III trial for blarcamesine are indeed promising for the field of Alzheimer's disease treatment. The significant slowing of clinical decline, as measured by the ADAS-Cog13, is clinically meaningful and compares favorably to other treatments in development.
What's particularly intriguing is the drug's effect on brain atrophy. The 63.5% reduction in total grey matter atrophy is remarkable and suggests a potent neuroprotective effect. This could translate to meaningful preservation of cognitive function over time, which is the ultimate goal of Alzheimer's treatment.
The safety profile is another standout feature. The absence of ARIA is a significant advantage over amyloid-targeting antibodies, which have been plagued by this side effect. The main adverse event, dizziness, appears manageable through dose titration.
However, it's important to note the lack of significance in the ADCS-ADL functional measure. While this may indeed be due to the scale's reduced sensitivity in early Alzheimer's, it will be important to demonstrate functional benefits in future studies or real-world use.
If these results are replicated and lead to approval, blarcamesine could represent a major advance in Alzheimer's treatment, offering a convenient, safe and effective option for early-stage patients.
Oral, once daily blarcamesine significantly slowed clinical decline for early Alzheimer's disease patients with good comparative safety profile and no associated neuroimaging adverse events
Clinical benefit of blarcamesine consistently observed for both 30 mg and 50 mg treatment groups
Benefits of blarcamesine on both amyloid-beta and brain volume, two underlying pathological hallmarks of Alzheimer’s disease
EMA submission expected in Q4
NEW YORK, July 28, 2024 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative, neurodevelopmental, and neuropsychiatric disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome, schizophrenia, and other central nervous system (CNS) diseases, today presented comprehensive results from the Phase IIb/III study showing that blarcamesine (ANAVEX®2-73), once daily orally, significantly slowed clinical decline in people with early Alzheimer's disease (AD). The data were presented by Marwan Noel Sabbagh, MD, Professor of Neurology at Barrow Neurological Institute and Chairman of the Scientific Advisory Board at the 2024 Alzheimer's Association International Conference (AAIC).
Blarcamesine significantly slowed clinical progression by
“These data are very exciting, particularly in a study that can demonstrate objective slowing of markers of neurodegeneration,” said Dr. Sabbagh. “The advantage of blarcamesine is that it is a small oral molecule that exerts clinical benefits on cognition and neurodegeneration and could be appealing because of its route of administration and good comparative safety profile. The neuroimaging evaluation performed in the Phase IIb/III study demonstrated no neurological tissue damage such as hemorrhage or Amyloid-related imaging abnormalities (ARIA), as documented with other anti-amyloid targeted therapies. We believe the scalable and convenient features of blarcamesine could reduce crucial barriers within the currently complex healthcare ecosystem for Alzheimer's disease and provide broader access to a diverse population with early Alzheimer's disease.”
Blarcamesine, a small molecule administered orally once daily, demonstrated numerically superior clinical efficacy 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, could represent a novel treatment that could be complementary to the currently approved anti-beta amyloid monoclonal antibody drugs.
Juan Carlos Lopez-Talavera, MD, PhD, Head of Research and Development of Anavex commented: “Anavex's precision medicine approach, tailored to improving autophagy, a key clearance mechanism that removes protein aggregates and misfolded proteins across the Alzheimer's disease continuum and uniquely positions the Company to develop innovative solutions for patients and their families.” He continued: “People living with early Alzheimer's disease have the desire to maintain their sense of self for as long as possible. The study results provide the potential for people with more time to engage in meaningful activities. Full regulatory submission of blarcamesine in Europe (EMA) is expected in Q4 2024.”
For the primary endpoint ADAS-Cog13, blarcamesine is significantly better than placebo for both 50 mg (−2.149; P = 0.021) at 48 weeks and for 30 mg blarcamesine dosage groups (−1.934; P = 0.026) at 48 weeks. The key secondary endpoint CDR-SB was significantly improved vs. placebo in both 50 mg (−0.465; P = 0.045) and 30 mg (−0.502; P = 0.020) assigned dose groups. CGI-I was significantly improved in both 50 mg (−0.314; P = 0.008) and 30 mg (−0.248; P = 0.024) groups.
In the respective safety population, common treatment-emergent adverse events included dizziness, which was transient and mostly mild to moderate in severity, and occurred in 120 participants (
“Alzheimer’s disease is such a devastating disease that affects tens of millions worldwide. The findings from this and previous studies with blarcamesine in Alzheimer’s disease further strengthen our belief in the potential of addressing the complex pathology in Alzheimer’s disease through an upstream precision medicine compensatory process, autophagy through SIGMAR1 activation,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “We like to thank all the people involved in the study for their invaluable contributions and we look forward to continuing our journey to address the high unmet need for Alzheimer’s disease patients with a potential new convenient orally available treatment option for Alzheimer’s disease.”
The presentation is available on the Investors section of the Company’s website at www.anavex.com. Data from the blarcamesine Phase IIb/III ANAVEX®2-73-AD-004 trial to be published in an upcoming peer-reviewed journal.
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, Rett syndrome, schizophrenia 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 that restores 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 US Food and Drug Administration. Early Alzheimer’s Disease: Developing Drugs for Treatment Guidance for Industry. 2024.
2 Potashman M, Pang M, Tahir M, Shahraz S, Dichter S, Perneczky R, et al. Psychometric properties of the Alzheimer’s Disease Cooperative Study – Activities of Daily Living for Mild Cognitive Impairment (ADCS-MCI-ADL) scale: a post hoc analysis of the ADCS ADC-008 trial. BMC Geriatr. 2023; 23(1).37.
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