Alzheon is a Phase 3 clinical-stage biopharmaceutical company developing oral disease- modifying therapies for Alzheimer’s disease.
What does the new White House administration’s focus on prevention imply for the approach to Alzheimer’s? And where do you see Alzheon fitting in?
The annual cost per Alzheimer’s patient to Medicare is approximately $70,000 a year, for an annual total of $175 billion, largely due to the enormous burden of nursing care. Even the U.S. cannot sustain this indefinitely, so there is growing pressure to address this burgeoning problem. This is why we have been in discussions with policymakers about prevention and early intervention.
Two years ago, and in parallel to Alzheon, I launched a second company, Tolion Health AI, which uses AI for prevention of dementia. Families with strong genetic risk factors have been approaching us asking how to avoid the disease. For example, in patients with two copies of the APOE4 risk gene, developing Alzheimer’s pathology is almost certain by age 65 with many starting to show decline in memory and other cognitive abilities. However, lifestyle interventions can dramatically reduce the risk of having these symptoms—by nearly half, but early intervention is critical. Because Alzheon’s drug is oral, with favorable safety profile and targets one of the earliest amyloid pathologies, it can be used preventively before people start showing any cognitive decline. What we call Alzheimer’s dementia is the terminal stage of disease where patients have lost so much of their cognitive skills that they become unable to reason or function independently.
The key finding from our last Phase 3 study with valiltramiprosate is that treatment must start at the earliest signs of memory loss, before patients have lost their functional abilities, this stage is called mild cognitive impairment or MCI. That is where our treatment makes the biggest difference.
Could you expand on the key takeaways from your Phase 3 results from this spring?
The main takeaway from our Phase 3 trial is that early intervention, at the earliest symptomatic stage (MCI) is crucial. Alzheimer’s is driven by toxic amyloid buildup as the brain’s ability to clear it declines with age. Our drug, valiltramiprosate, prevents this buildup, which is why it is effective in the early stages of Alzheimer’s. In contrast, the approved antibody therapies that target amyloid clear existing amyloid plaques and show greater effect later in the disease.
Our data demonstrates that when the drug is given to patients at the MCI stage, it slows the loss of memory and cognitive abilities by half compared to a placebo, and maintains functional performance for the whole study duration of 1.5 years. On cognition, treated patients stayed above baseline for a full year and on daily function they remained above baseline for 18 months, while placebo patients declined progressively over this period. The drug effect is strongest early in the disease, when there’s still enough healthy brain tissue to protect—particularly in areas like the hippocampus, which is responsible for memory formation. Indeed, the MCI patients treated for 1.5 years showed significant tissue preservation in all the brain regions, including the hippocampus.
Would you make the more general statement that prevention should now be the main focus in our approach to Alzheimer’s?
Yes, but regulatory systems are built around treating patients who already show symptoms. Approval pathways are still tied to these later stages, even though the disease begins decades earlier and can now be detected through brain imaging or blood-based biomarkers. No drug has yet received approval for pre-symptomatic treatment.
Our data suggests that treatment with valiltramiprosate at earliest symptomatic stage (MCI) can stabilize patients for years, allowing them to remain functional and independent much longer. We have seen this in multiple clinical studies showing consistent clinical benefits on both cognition and function. Translating this into practice could mean delaying the disability and loss of independence that leads to nursing home placement by almost a decade, which would have a profound impact on patients, their families, and healthcare systems alike.
You mentioned Tolion Health AI. Could you explain Tolion’s added value in simple terms?
Tolion aims to combine and analyze all existing medical knowledge concerning brain health and performance with AI to guide personalized prevention strategies and lifestyle changes to improve brain function and protect it from damage. Alzheimer’s affects 55 million people globally, and roughly half of those cases could be prevented through modifiable factors such as exercise, social engagement, diet, hearing and vision care, and overall lifestyle.
Our goal is threefold: to consolidate knowledge through AI tools, identify what works for each individual, and help change behavior accordingly. By addressing modifiable risks factors early—decades before symptoms appear—we can dramatically reduce the disease burden and improve quality of life.
We are making prevention accessible across all income groups with the goal to reduce the enormous human as well as monetary costs of dementia care — currently reaching USD 384 billion per year in the U.S. alone. By focusing on prevention, we strive to help healthcare systems save billions of dollars that would otherwise be spent on managing this disease.
What are your main milestones and regulatory priorities for Alzheon for the next year?
Our next focus is to seek patient access to the drug as soon as possible while running additional studies. Patients carrying the APOE4 risk gene are also the ones at the highest risk of neurovascular complications called ARIA (brain edema with or without bleeds) with the approved amyloid antibodies, which carry boxed warnings from the FDA. We will conduct trials in patients with one and two copies of the risk gene, who together make up approximately two-thirds of the Alzheimer’s population. These studies will allow us to extend the benefits of treatment across the majority of patients.
We will also be engaging closely with regulators to advance our approval process, building on the strong clinical data we already have. The ultimate goal is to make our therapy widely available as soon as possible to a group of AD patients that need safe drugs that can target amyloid but avoid the risk of serious ARIA seen with the antibodies.
How have investors reacted to your Phase 3 results?
Positively. These studies cost hundreds of millions of dollars, but investor response has been encouraging because the potential value is enormous—both in magnitude of patient-lives affected and in potential market value. The key question is confidence in approval and reproducibility, and we have demonstrated consistent positive effects across four studies and datasets.
Our drug’s safety and efficacy profile, especially its protective effect on brain atrophy and neurodegeneration, is very compelling. In contrast, antibody therapies on the market have high risks of brain swelling and bleeds and are extremely expensive—potentially costing patients over $100,000 a year for the combined cost of drug infusions and the ancillary follow-up MRIs, safety monitoring, and PET scans. With our simple treatment paradigm with an oral pill, the potential safety, cost, and accessibility advantages are clear.
Do you expect any special support from the new administration?
Yes, there is a strong political will to act. The economic and social burden of Alzheimer’s is simply too profound to ignore, especially with an aging population. The cost of AD care for the US government is staggering. Policymakers recognize that prevention and early treatment are both cost-effective and essential.
Lifestyle interventions in mid-life as recommended by Tolion Brain Coach mobile application can halve the disease burden, while treatments like ours can keep early symptomatic patients functional and out of nursing homes for longer. Starting our drug at the pre-symptomatic stage may also be effective at allaying Alzheimer’s symptoms for years, akin to what early intervention with lipid lowering drugs have done to cardiovascular disease. Infusions are not a sustainable long-term solution, so an oral, safe, effective drug is exactly what the system needs. Alzheimer’s has become a major economic burden to the US government, and it is encouraging to see it taken seriously at the highest levels.
Where would you like to see Alzheon one year from now?
Our priorities are clear: work with regulators to bring our lead drug candidate valiltramiprosate to patients as soon as possible, complete financing for our upcoming studies, and advance our next-generation compounds.
Beyond that, we are exploring other indications where our molecules could be effective, including Down syndrome patients who overproduce amyloid and develop Alzheimer’s decades earlier. The long-term vision is to expand this platform to protect as many patients as possible.
