EMA Accepts Anavex's Blarcamesine Application for Alzheimer’s Treatment

Anavex Life Sciences Corporation, 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 disorders, announced that the European Medicines Agency (EMA) has accepted for review the marketing authorization application (MAA) for blarcamesine (ANAVEX2-73), an investigational drug for the treatment of Alzheimer’s disease.

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, 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.”?

Marketing Authorization Application is supported by data from the randomized, double-blind, placebo-controlled Phase IIb/III, ANAVEX2-73-AD-004 trial and it’s up to 144 week open-label-extension (OLE) ATTENTION-AD ANAVEX2-73-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 weeks 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, President and CEO 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. 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. 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.

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.  The World Health Organization (WHO) estimated the cost in Europe of caring for people with dementia, including Alzheimer's disease, at $439 billion, or $31,144 per person in 2019. That includes hospital care, medicines, diagnostics, informal caregiver time, community services and long-term care facility costs.

Anavex's lead drug candidate, ANAVEX2-73, 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. ANAVEX2-73 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. ANAVEX2-73 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 ANAVEX2-73 for the treatment of Parkinson's disease. We believe that ANAVEX3-71, 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, ANAVEX3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation.

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