Anavex Life Sciences, a clinical-stage biopharmaceutical company, has presented groundbreaking results from its Phase IIb/III trial of blarcamesine, or Anavex 2-73, for early Alzheimer’s disease at the 2024 Alzheimer’s Association International Conference. The study demonstrates significant potential in slowing clinical decline and addressing underlying pathological hallmarks of the disease.
Key Findings
The trial, involving oral, once-daily administration of blarcamesine, showed:
— Significant slowing of clinical progression by 38.5% and 34.6% at 48 weeks in 50 milligrams and 30 milligrams groups, respectively, compared to placebo.
— Improvement in the primary cognitive endpoint ADAS-Cog13.
— Positive trends in the functional co-primary endpoint ADCS-ADL, though not reaching statistical significance.
— Significant improvement in the key secondary composite endpoint CDR-SB for both dosage groups.
— Beneficial effects on biomarkers, including plasma Aβ42/40-ratio and reduction of brain atrophy.
Implications for Alzheimer’s Treatment
Dr. Marwan Noel Sabbagh, professor of neurology and chairman of Anavex’s scientific advisory board, emphasized the excitement surrounding these results, particularly the objective slowing of neurodegeneration markers. “These data are very exciting, particularly in a study that can demonstrate objective slowing of markers of neurodegeneration,” said Sabbagh.
“We believe the scalable and convenient features of blarcamesine could reduce crucial barriers within the currently complex health care ecosystem for Alzheimer’s disease and provide broader access to a diverse population with early Alzheimer’s disease.”
Anavex Life Sciences’ approach focuses on improving autophagy, a key clearance mechanism for protein aggregates and misfolded proteins in Alzheimer’s disease. This precision medicine strategy positions the company uniquely in developing innovative solutions for patients across the disease continuum.
Safety Profile and Next Steps
The study reported common treatment-emergent adverse events, primarily mild to moderate dizziness, which were manageable through adjusted titration schedules. Importantly, blarcamesine doesn’t require routine MRI monitoring, distinguishing it from some other Alzheimer’s treatments.
Anavex plans to submit a full regulatory application for blarcamesine to the European Medicines Agency in Q4 2024. The company believes that blarcamesine’s efficacy, combined with its oral administration and safety profile, could offer a complementary treatment option alongside currently approved therapies.
About Anavex Life Sciences
Anavex Life Sciences Corp. (Nasdaq: AVXL) is dedicated to developing novel therapeutics for neurodegenerative, neurodevelopmental, and neuropsychiatric disorders. Its lead drug candidate, blarcamesine, has shown promise in clinical trials for Alzheimer’s disease, Parkinson’s disease dementia, and Rett syndrome. The company’s approach focuses on restoring cellular homeostasis by targeting sigma-1 and muscarinic receptors.
As Alzheimer’s disease continues to affect millions worldwide, Anavex’s innovative approach and promising results offer hope for a new, orally available treatment option. The scientific community eagerly awaits further developments and the potential impact of blarcamesine on the lives of those affected by this devastating disease.
Note: This article discusses investigational uses of a product in development and does not intend to make conclusions about efficacy or safety. There is no guarantee that blarcamesine will successfully complete clinical development or gain health authority approval.