Since we last spoke a year ago, you have raised $50 million in venture capital. Could you explain the significance of that and the progress of your Alzheimer’s medicine, ALZ-801/valiltramiprosate?
This funding marks another milestone for Alzheon, reflecting a decade of dedicated research in understanding and combating Alzheimer’s disease and of development of our treatment. Our scientific breakthroughs include discovery of the toxin causing the disease, effective diagnostic methods, and the use of plasma biomarkers for monitoring disease progression and therapeutic efficacy. With this funding, we have completed enrollment for the pivotal APOLLOE4 Phase 3 clinical trial of our novel oral medication, ALZ-801/valiltramiprosate, which as our data have shown, avoids certain adverse effects, such as brain edema and microhemorrhage, that have been seen with other treatments. This drug shows promise for not only improved accessibility but also potentially greater efficacy in treating Alzheimer’s.
What makes ALZ-801 different from other Alzheimer’s treatments?
ALZ-801 stands in a class of its own due to its oral administration, favorable safety profile, and potentially superior efficacy. In our Phase 2 study, spanning two years, we observed the drug’s ability to stabilize Alzheimer’s disease on cognitive measures, a potential improvement over currently approved treatments. ALZ-801 also has the potential to simplify delivery and access for patients, while its safety profile avoids the serious side effects seen in other Alzheimer’s drugs. Our goal is to offer a more accessible, safer, and more effective treatment to Alzheimer’s patients.
When do you anticipate ALZ-801 to be available on the market?
We expect to complete the pivotal Phase 3 trial this year and aim for regulatory approval shortly thereafter. If all goes according to plan, ALZ-801 could be available in the U.S. by 2025. In preparation for this launch, we are already scaling up production to meet the anticipated high demand, planning for the manufacture of substantial quantities of the drug in order to address the widespread need for an effective and accessible Alzheimer’s treatment.
Could you elaborate on the effects of ALZ-801? Does it cure Alzheimer’s or improve the disease outcome?
ALZ-801 is being studied in patients with Early Alzheimer’s disease, with our latest clinical data demonstrating the potential for this drug to substantially alleviate disease progression. ALZ-801 intervenes in the early stages of Alzheimer’s disease, focusing on the early symptomatic phase, when brain damage begins to present as clinical symptoms. By targeting the disease before it reaches an advanced stage, our drug holds the potential to prevent progression to severe symptoms. The treatment’s effectiveness is based on our understanding of Alzheimer’s as a decades-long process, where early intervention is crucial. Although ALZ-801 is currently being evaluated in the early clinical disease, our ultimate goal is to prevent patients from reaching the symptomatic stage of Alzheimer’s. This approach is based on groundbreaking research, including the identification of key biomarkers like tau protein, which enables the detection of Alzheimer’s disease years before the clinical symptoms appear.
How do you identify and target patients in the pre-symptomatic stage of Alzheimer’s?
Identification of pre-symptomatic AD patients is still evolving and is a combination of biomarker screening and genetic risk assessment.
We apply advancements in biomarker research, particularly the measurement of tau protein in plasma, to detect brain damage caused by Alzheimer’s years and decades before clinical symptoms manifest.
This method allows us to identify individuals who have active disease but have not yet shown symptoms. Additionally, we focus on high-risk groups, such as those with the apolipoprotein E4 gene, which increases the likelihood of developing Alzheimer’s at an earlier age. Our end goal is to target these high-risk individuals for early intervention, ideally preventing progression to the clinical stage of the disease.
Do you see a global resurgence in investment in neurological diseases, and are there plans to expand your research to other diseases beyond Alzheimer’s?
There has been a notable shift in investment focus towards neurological disorders in recent years. This sector, encompassing both psychiatry and neurology, has seen significant value creation, even surpassing traditional high-value areas such as oncology and orphan diseases. The change is largely due to a deeper understanding of the science behind these conditions. Previously, the vast investments of hundreds of billions in Alzheimer’s research yielded little progress, leading to investor hesitancy. However, recent breakthroughs have changed this narrative, transforming AD into one of the most promising and attractive investment areas.
At Alzheon, our technology is designed to prevent the formation of toxins that cause neurodegeneration with a portfolio of molecules that could potentially address a number of brain disorders. Our understanding of these disorders, particularly the process of protein clearance from the brain and its impairment with age, positions us to advance treatments for conditions such as Parkinson’s and ALS alongside Alzheimer’s.
What is your perspective on the Inflation Reduction Act and its impact on your work?
The Inflation Reduction Act brings both opportunities and challenges for us, particularly in the context of Medicare. The act’s provision to cap out-of-pocket expenses at $2,000 for Medicare Part D beneficiaries aligns well with our approach, as it reduces the financial burden on patients using our oral treatment. This change could be impactful considering the growing costs associated with Alzheimer’s care, projected to reach $1.1 trillion by 2050 in the U.S. alone. Our treatment, being oral and easy to administer, could offer a novel solution by potentially preventing patients from reaching the terminal, more costly stages of Alzheimer’s. This further aligns with our commitment to making a widely accessible and cost-effective treatment, addressing the needs of millions of patients globally.
Why did you choose to dedicate your life to Alzheimer’s research?
Alzheimer’s is considered one of the largest threats to humanity and my journey into Alzheimer’s research is rooted in a deep commitment to address the devastating impacts of neurodegenerative disorders on patients and their families. My background in computer science, medicine, and neurology, coupled with my experiences at Pfizer and leading several biotechnology companies, has led me to realize the urgency to address the massive need for effective treatments in this field. In addition to Alzheon and other companies, I also founded a global institute, International Neurodegenerative Disorders Research Center, INDRC, which applies data driven solutions, in particular big data, and artificial intelligence approaches, to accelerate identification of treatments for neurodegenerative disorders. Witnessing the profound effects of diseases like Alzheimer’s firsthand fueled my dedication to this cause.
The challenge in identification and development of new treatments has been immense, especially considering the long-standing reluctance of investors to fund research in this area. However, our team’s relentless focus on understanding of the disease process has led to significant breakthroughs, making Alzheon a pioneer in the field. This journey, driven by a mission to help millions suffering from neurodegenerative disorders, is a testament to our focused innovation and dedication, tackling some of the most challenging medical conditions of our time.