Anavex Presents at H.C. Wainwright 26th Annual Global Investment Conference
Heather Arranie 
Highlighting the Importance of Blarcamesine
Anavex Life Sciences’ recent investor presentation has provided valuable insights into the importance of Blarcamesine as a treatment for central nervous system diseases.
Anavex, the clinical-stage biopharmaceutical developing innovative small molecule treatments for debilitating central nervous system (CNS) diseases – recently presented at the H.C. Wainwright 26th Annual Global Investment Conference to provide key insights into its global operations. Here are some highlights from this investor presentation that emphasize the continued importance of Anavex’s work developing treatments like Blarcamesine for diseases like Alzheimer’s, Parkinson’s, and Rett Syndrome.
Accounting for Global Demand:
With worldwide Alzheimer’s and dementia cases expected to exceed 130 million by 2050- an increase of over 80 million from today- Anavex Life Sciences is one company uniquely equipped to meet the increasing demands for treatments for CNS diseases.
As a small-molecule, orally-available Sigma-1 activator, the company’s investigational drug, Blarcamesine, offers a safe, convenient, and effective treatment for neurodegeneration in Early Alzheimer’s Disease and Parkinson’s-related dementia and has shown promise in improving symptoms for individuals with Rhett Syndrome.
“We’ve been really privileged by the fact that just recently [following] a comparison to the antibody drugs, Leqembi and Donanemab, Blarcamesine demonstrated superiority both in regards to safety as well as efficacy in their effect in Alzheimer’s patients [and] in early Alzheimer’s patients,” Anavex Life Sciences’ President and CEO, Dr. Christopher Missling said in his presentation.
As a brain with Alzheimer’s ages, it becomes susceptible to neurotoxicity, leading to neurodegeneration and brain plaque. While most monoclonal antibody treatments for Alzheimer’s focus on the downstream removal of these plaques after Alzheimer’s has progressed by activating Sigma-1 proteins key to cellular homeostasis, Blarcamesine focuses on upstream prevention of neurodegeneration by repairing and protecting damaged brain cells.
“Sigma-1 is a bit like the fire truck in your neighborhood; you never need the fire truck if you never have a fire, but if you have a severe fire, you really need it badly. And that’s the ability of Sigma-1 to jump in and help to restore functionality [and] restore homeostasis,” explained Dr. Missling in his presentation.
Given that around 84% of the best-selling pharmaceutical products are orally administered, yet only around 60% of small molecule drugs are commercially available orally, Anavex continues to meet the demands of underserved communities with its CNS treatments like Blarcamesine. As a once-daily, orally available treatment, Blarcamesine has the potential for widespread accessibility with large-scale, room-temperature shipping capabilities
“We have a formulation which is convenient for children, patients who have problems with swallowing, and a liquid formulation for the drug, and we have already demonstrated intriguing data in Rett syndrome, a rare disease. We are planning to also expand this indication franchise of rare disease into Fragile X and other following rare disease trajectories,” said Dr. Missling.
Transforming Lives:
As Anavex continues its clinical trials for treating Alzheimer’s, Parkinson’s, and Rhett’s Syndrome, the company continues to transform the lives of its patients.
Not only have 93% of Rhett’s Syndrome pediatric patients who completed the open-label extension study of Blarcamesine opted to continue treatment through the Compassionate Use Program, but some patients have been using the treatment for over four years, and caretakers of those with Rhett’s Syndrome around the world have shared the resounding benefits of treatment.
“I was hopeful that we would see some change and some kind of decrease in her symptoms,” said Donna after her daughter Annelise began the Blarcamesine trial. “Her seizures reduced quite dramatically. She would have multiple seizures a day, and now we’re lucky to get one every couple of weeks.”
With its promising clinical trial results and patient testimonies, Anavex has begun educating the medical community about the benefits of Blarcamesine in preparation for its eminent regulatory approval.
“We are now in the regulatory submission stage. We are planned to submit for potential EMA approval in Europe by the end of this year. And as a reminder, we also have, of course, other regions we are planning to cover,” stated Dr. Missling.
