Anavex Life Sciences Corp. has achieved a significant milestone with the European Medicines Agency (EMA) accepting the Marketing Authorization Application (MAA) for blarcamesine (ANAVEX2-73) for the treatment of Alzheimer's disease. This acceptance marks a crucial step in potentially bringing a new oral treatment option to millions of patients in Europe affected by this debilitating condition.
The MAA is supported by data from the randomized, double-blind, placebo-controlled Phase IIb/III ANAVEX2-73-AD-004 trial, along with its 144-week open-label extension. These studies investigated the efficacy and safety of blarcamesine in patients with early Alzheimer's disease.
Clinical Efficacy and Safety
Blarcamesine, administered orally once daily, demonstrated clinically meaningful improvement over 48 weeks. The primary endpoint, ADAS-Cog13 score, showed an improvement of more than 2 points, suggesting a significant impact on cognitive function. This improvement indicates a superior numerical clinical efficacy compared to currently approved therapies, while also demonstrating a slowing of neurodegeneration in early Alzheimer's disease patients.
Importantly, blarcamesine's safety profile indicates that routine MRI monitoring is not required, offering a more convenient treatment option for patients and potentially reducing healthcare costs. The advantage of blarcamesine lies in its ease of administration and good comparative safety profile.
Mechanism of Action: SIGMAR1 Activation
Data from the Phase IIb/III trial revealed that blarcamesine exerts its clinical efficacy through upstream SIGMAR1 activation. SIGMAR1, an integral membrane protein, activates a compensatory process, inducing autophagy and restoring cellular homeostasis. This mechanism is particularly relevant as impaired autophagy precedes both amyloid beta and tau tangles, key pathological hallmarks of Alzheimer's disease. Therefore, the stabilization or restoration of autophagy can be seen as an early preventative measure against the progression of Alzheimer's disease.
Alzheimer's Disease Burden in Europe
There are an estimated 7 million people in Europe living with Alzheimer's disease, and this number is projected to double by 2030, according to the European Brain Council. The World Health Organization (WHO) estimated the cost of caring for people with dementia, including Alzheimer's disease, in Europe at $439 billion in 2019. This figure includes hospital care, medicines, diagnostics, caregiver time, community services, and long-term care facility costs.
Management Perspective
"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."
Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex, added, "We look forward to working with the EMA and thank the team at Anavex for their work on the submission. We are committed to improving patient access to novel and accessible oral medicines."
