Alzheon is a clinical-stage biopharmaceutical company developing oral treatments for Alzheimer's disease and other neurological disorders. The company recently completed a Phase 3 trial in patients with early stages of Alzheimer’s disease with its lead candidate ALZ-801/valiltramiprosate that targets neurotoxic amyloid oligomers.
Much has happened in the U.S. life science sector in the past 12 months. What have been the headline achievements for Alzheon?
2024 has been a tremendous year of advancements for Alzheon. Most importantly, we completed the APOLLOE4 Phase 3 trial of our oral Alzheimer’s drug candidate ALZ-801/valiltramiprosate – a 78-week gold-standard study focusing on high-risk patients with two copies of the APOE4 gene (APOE4 homozygotes). This group represents about one million individuals in the U.S. alone, and APOLLOE4 is the only Phase 3 study conducted solely in this population. We are now on the cusp of presenting the breakthrough results of this study and meeting with regulatory authorities including FDA.
Additionally, we published four new scientific papers this year, bringing our total to 13, outlining our scientific path to effective treatment. Another significant milestone was the closing of our $100 million funding round.
What distinguishes ALZ-801/valiltramiprosate from existing disease modifying therapies, and what does that mean for patients?
One primary distinction is that ALZ-801 is an oral pill, offering a potentially more accessible and less expensive alternative to existing Alzheimer's treatments. Secondly, it addresses many troublesome side effects and patient access challenges that have limited adoption, including brain swelling and microhemorrhages and the burdensome patient journey surrounding infusion therapies. Some healthcare systems, like the Veterans Health Administration, exclude APOE4 homozygotes from current AD treatments entirely. This means that ALZ-801/valiltramiprosate could potentially fill an unmet need for a patient population with the greatest genetic risk for developing Alzheimer’s disease and very limited therapeutic options.
Our studies, including the APOLLOE4 Phase 3 trial, focus on this high-risk population. In addition to clinical effects, we have observed compelling results in slowing progressive brain shrinkage and neurodegeneration biomarkers caused by Alzheimer’s disease. Importantly, ALZ-801 remains the only oral anti-amyloid treatment in Phase 3 studies. We plan to announce this data at a major scientific conference and publish our pathbreaking results in a peer-reviewed journal next year. This is what the industry has been working toward for decades: a treatment that addresses the disease both in the earliest symptomatic stages, and that is well-suited for prevention.
One-third of people with Alzheimer’s are undiagnosed. How is science advancing for early identification using blood-based biomarkers?
Blood-based biomarkers are one exciting innovation that will greatly help earlier diagnosis, which is much needed especially for presymptomatic patients so we can intervene earlier to prevent as much damage as possible. Advances in genetic testing will also improve precise diagnosis for appropriate patients.
Alzheon has been a leader in many of these breakthroughs including understanding the toxin causing the disease, accurately diagnosing Alzheimer’s, using blood-based biomarkers to track progression and efficacy, and understanding genetic risk factors like APOE4.
The APOE4 gene is present in about two-thirds of patients with Alzheimer’s, making genetics the second greatest risk factor after age. These advancements in understanding are enabling earlier detection, which is critical, since Alzheimer’s begins in the 40s or 50s and progresses silently for 20 to 30 years. Currently, patients are usually diagnosed only after showing clinical symptoms of cognitive decline. By this stage, the brain has already sustained significant damage, limiting recovery potential.
You are also the founder of the AI-enabled brain health company Tolion Health. What potential does artificial intelligence hold for addressing neurological diseases?
Tolion Health represents the culmination of our efforts to provide actionable, AI-powered brain health guidance. Tolion addresses an urgent need to support families and patients at risk for neurodegenerative disorders and dementia but not yet symptomatic by helping to provide them with actionable science-backed lifestyle interventions.
I founded Tolion to apply AI in ways that empower people, especially healthy individuals, by providing access to medical knowledge that is not readily available. One of the challenges in healthcare is efficiently leveraging the sheer volume of information and knowledge, which is rapidly growing every day, so no physician can track it all. AI can help address this by focusing on precise personalized and preventive healthcare that assesses where a person stands and how they can avoid disease onset and progression. Ultimately, our goal is to transform the landscape of brain health, guided by a shared mission to deliver targeted, individualized care and a future free from the burden of dementia.
There has been a surge in investor confidence in Alzheimer’s research as studies yield more promising results. Do you see funding for this segment becoming more forthcoming in 2025?
Absolutely. When I founded Alzheon 11 years ago, funding for Alzheimer’s research was nearly impossible. Hundreds of programs failed and, for decades, Alzheimer’s investments at large pharmaceutical companies totaling hundreds of billions yielded no results.
Recent breakthroughs in understanding the disease’s biology, the role of toxic amyloid and the introduction of anti-amyloid antibody treatments validating this approach have completely changed the interest of investors. Alzheimer’s is now seen as the next major-value opportunity after the success of obesity treatments.
With that in mind, do you anticipate any regulatory or policy changes that may expedite commercialization of neurological disease treatments?
Governments and regulators are increasingly recognizing the staggering cost of Alzheimer’s care. For instance, a recent analysis from the University of Southern California Brain Observatory showed slowing disease progression by 30% could save $2.4 trillion in the U.S. over 20 years. Regulatory agencies worldwide are working with companies to expedite effective therapies that can reduce this economic burden.
We are optimistic about the FDA and other regulators’ willingness to utilize imaging and fluid biomarkers and surrogate measures to revolutionize Alzheimer’s drug approvals. Changing this paradigm is essential for reaching patients earlier and enabling the introduction of presymptomatic preventive treatments. As the population ages and the number of patients grows, the urgency to address this disease becomes more critical. Policy makers understand this, and we hope to see policies that further incentivize innovation and accessibility.
What is on the horizon for Alzheon in 2025?
Our primary goal is to make ALZ-801/valiltramiprosate accessible to appropriate patients as quickly as possible. We are meeting with regulatory agencies to discuss the potential path to a market introduction and planning to start another study to expand the patient population for our treatment. Additionally, we are building a platform that includes studies with optimized molecules and improved versions of our current drug to target multiple neurodegenerative disorders more effectively.
Beyond that, we anticipate a busy start to the year, particularly with the J. P. Morgan Healthcare Conference in January. It is an opportunity to share our progress, discuss upcoming plans, meet with investors and Pharma companies, and further solidify relationships with those interested in advancing Alzheimer’s treatments alongside us.
