Good morning, everyone, and thank you for joining us for H.C. Wainwright's twenty-sixth Annual Global Investment Conference. My name is Eduardo Martinez. I'm a biotech equity research associate at H.C. Wainwright, and I have the pleasure of introducing Dr. Christopher Missling, President and CEO of Anavex Life Sciences. Thank you for joining us this morning.
Thank you very much for the kind introduction, and let me get started to introduce Anavex Life Sciences. Appreciate the kind introduction again and invitation. We are a public company, so I'd like you to read the forward-looking statement. Thank you very much. We're dedicated to pushing forward with new boundaries in scientific discovery, and especially with addressing unmet need and bringing relief to patients with novel oral small molecules. One of the biggest unmet need these days is still Alzheimer's disease, which is growing almost exponentially in most all regions of the world. This is also our lead indication, and you will see the pipeline today overall as well. Again, I want to remind, it's $20 trillion of cumulative cost of Alzheimer's disease dementia care from 2015 to 2050 expected.
One in three Medicare dollars will be spent on Alzheimer's disease and other dementia in two thousand and fifty, and 11 million are the number of Americans providing unpaid care for people with Alzheimer's and other dementia. Addressing this unmet need is a major endeavor for Anavex. We're focusing on small molecules, and we develop small molecules, especially for Alzheimer's, which is an orally once daily, convenient, and scalable treatment. We are now in the regulatory submission stage. We plan submitting for EMA approval in Europe by the end of this year. We've been invited to do that. As a reminder, we also have, of course, other regions we can cover.
And as a reminder, we've been really privileged by the fact that just recently, a comparison to the antibody drugs, Leqembi and donanemab, that blarcamesine demonstrated both safety as well as efficacy, superiority in their effect in Alzheimer's patients, in early Alzheimer's patients. And as a reminder, from a financial point of view, we have no debt and cash which allows us to operate for over four years, around about four years, which is, of course, also important for going forward projects. So again, I want to give you an overview of the pipeline. The focus is on solid oral formulations for Alzheimer's, but also we have intriguing data in phase two in Parkinson's dementia.
We have also an oral treatment opportunity there, as well as in Parkinson's itself, which we're planning to do a larger study with blarcamesine. But we also have a formulation which is convenient for children, patients who have problems with swallowing, and this is a liquid formulation for the drug, and we have already demonstrated very intriguing data in Rett syndrome, a rare disease, and we're planning to also expand this indication franchise of rare diseases into Fragile X and other following rare disease trajectories. Last but not least, I also want to remind that we have another project by itself, which is Anavex 3-71, an oral treatment for schizophrenia right now in phase 2, ongoing, and in the U.S., and very excited to have data coming out.
And we also have for frontotemporal dementia an orphan designation for 3-71 . We also have preclinical data for this compound in Alzheimer's disease. Again, reminder that oral administration upstream, and I'll come to it in a minute to the mechanism, might have additional advantages over antibodies, which are challenging biologics because of their administration route, but also by their safety challenges. How does the drug work or how the drug works? We're targeting an upstream mechanism, which is sigma-one, which is shown to be extremely intriguing from a pathological point of view, to reduce cellular stress overall, but also think about immune stimulation and oncology, translating that into CNS. This is really the best way to describe our drug profiles of sigma-one.
It's an upstream protein, which reduces cellular stress, which really tries to encapsulate and counter the pathology. And what's very intriguing is we demonstrated by looking at the expression level of the sigma-1 receptor, which was consistent with the response of patients in the previous trial, but also we identified genetic confirmation of this. When you look at the genes of the sigma-1, which is impaired, those patients who have an impaired genes didn't respond as strongly than those who had the wild type. And luckily, most patients have not an impaired or mutated sigma-1, so the majority, 80%, have a wild type gene of sigma-1, which is basically showing a very strong response if you activate that. And how does the activation works?
It's basically nicely shown in this schematic, where you see the aging and neurotoxicity, like pathology is challenging the survival of brain cells. And by showing how the antibodies work in Alzheimer's, specifically, they're trying to reduce A-beta, which they do by a downstream, I call it, effect, to remove this with an infusion. But the oral blarcamesine drug is able to do this more elegantly, actively through an upstream reduction of the removal of A-beta and tau, but also not only removing it, but also repairing the system and regenerate the biology in the brain. And also, we have demonstrated that shows resilience or building resilience in the brain.
So all this is a bit more elegant in addition to the oral administration, but think about the body helps himself by helping the body to help himself activation. We also know that sigma-1 is underexpressed in patients, and I want to now, however, show you that we increased that level of expression. But more importantly, now, I want to share with you what our upcoming catalysts are, which is very important. We have expressed great excitement that a paper will come out showing the full data of the ANAVEX 2-73 phase 2/3 study in Alzheimer's disease. We also are updating on the planned EMA submission this year. And also, we also have additional data from the Alzheimer's open-label study, which is coming out to be communicated.
One is the open-label study over 96 weeks, in totality 3 years, plus the 48 weeks, in addition to the data from the whole-genome somatic analysis to show data on the mechanism and the fidelity of the drug in its effect, but also, last but not least, I mentioned it before, we're expecting phase 2, placebo-controlled data readout of our schizophrenia trial with 3-71, so very excited about that one, and then in the pipeline, we're expanding and planning later-stage trials. One of them is a Parkinson's study, which is, in duration, probably one year long, and definitely longer than six months, and also, we're planning to reach another unmet need, which is the franchise of autism spectrum disorder.
In this case, is Fragile X, which we have very nice, solid preclinical data, and as recently as two months ago, presented at a Fragile X conference, additional biomarker data, showing that the drug really demonstrates a biological effect in the pathology of Fragile X, which is consistent also with the pathology in patients in animal models. And then last but not least, we also have another rare disease we're planning to show data about, start clinical data in. And last but not least, we have also additional papers expected throughout the remainder of the year. So maybe now let me share again the entire portfolio, which probably can describe really nicely as a platform with multiple drugs, not only one drug, in multiple indications, and we're very excited to move things forward.
As you can see, also, we are moving almost towards commercialization, especially in Alzheimer's disease direction, which is very exciting, and I will share with you more. The Alzheimer's disease franchise has been very substantiated with multiple studies, phase 1 study, phase 2 studies, and late-stage studies, and we're now in an open-label study. Now, when I mentioned that all regions, almost all regions of where the study took place, North America, Europe, and Southeast Asia, Australia specifically, we have reached patients which finished the phase 3 open-label study, and they almost requested all to stay on steady drug. We almost, in all regions of the world, are able to give the drug on a compassionate use to these patients who started with us. Parkinson's disease and Parkinson's dementia, I mentioned we're planning a study in Parkinson's very soon.
In Rett syndrome, we accomplished to have three studies completed, and we're planning to do another study to be able to accomplish what we want to do is to complete the task of helping patients in these indications. We had very strong support from investigators recently in a meeting that in patients at a conference, where gave us feedback that there's definitely still a lot of need for a drug like blarcamesine for Rett syndrome. That's also demonstrated in the real-world evidence, where patients really have told us that they have really seen improvement in their children, their girls, that they've got an improved mobility unexpectedly, and they also were able to reduce seizures, and there are more stories like that.
I also want to mention that all patients who finished the open-label studies were able and are able to get now the drug in our compassionate use programs in all regions and places where the study was tested. Some patients are now over four years on the study drug, which is extremely encouraging, and that certainly they would not do that if the drug wouldn't be safe or if they wouldn't see some benefit. Now, let me go to the Alzheimer's indication, which is really the key indication right now. There's a focus on that. Again, we're expecting a publication of the full data. It's three-arm study, over forty-eight weeks in over five hundred patients, and it's different doses, a medium dose and a high dose, target doses.
What's important is that we were able to demonstrate in the primary endpoint a positive outcome of ADAS-Cog13. What happened when we started the trial, we didn't know that in early Alzheimer, the ADCS-ADL is an additional measure, which was used constantly in all trials before, is not needed anymore for early Alzheimer. Because in early Alzheimer, the function, the ADL, is not impaired enough to show a separation, and then recently, as recently as March 2024, this year, the FDA acknowledged that it was published before that, acknowledged that fact, so for primary endpoint outcome, you don't need for regulatory approval, you don't seem to need that ADL outcome because of in early Alzheimer, you don't have the ability to show an improvement.
And so we were able to demonstrate a significant improvement also in CDR Sum of Boxes , is a key secondary endpoint, which is used very often as a primary endpoint in Alzheimer's disease. And we have also very strong data in another clinical outcome, CGI, as well as biomarker data in MRI, as well as biomarker of A-beta 42/40 ratio, which is really intriguing because it's part of the pathology. So when you show, for example, a picture of a brain, when it's healthy and when it's impaired, you see very clearly astonishing difference that the brain, which is impaired, builds holes and shrinks.
And what we've demonstrated in almost all regions of the brain with blarcamesine, a slowing down of this atrophy, of this shrinking of the brain, which is very intriguing because it goes to the core of the neuropathology of neurodegeneration itself. So in summary, we have oral administration of the drug, and you see a picture here of the drug, how it looks like, and with a novel target, and which impacts neurodegeneration at the core upstream. And we have very promising clinical data, with good safety profile, as well as, as I mentioned, a numerically superior efficacy compared to the antibodies.
It is demonstrated by giving you an example. Donanemab had an ADAS-Cog13 level value over 72 weeks of -1.35, so minus is an improvement, while our drug, the blarcamesine, demonstrated a -2.1 improvement over only 48 weeks, much earlier, numerically superior by 70%, around about 70%. That's why we're moving forward now in exploring commercial activities. That means we are actively now engaging with KOLs, with physicians, with Alzheimer's communities, to educate them about our drug, and when the paper comes out, this will be accelerating even further to explain the once daily oral administration potential in Alzheimer's disease. As a summary, you can see there's really large markets in almost all our indications.
Alzheimer's, of course, being the largest, with Europe being 7.8 million patients, which is larger even than the U.S., and Asia becoming the largest market these days with 23 million. So globally, 35 million patients in Alzheimer's, followed by 10 million in Parkinson's disease, schizophrenia, 24 million, and frontotemporal dementia is a rare disease as well as the other indications. We have very good strong patent protection. We aim for patent protection for all our drugs, all our indications, consistently. That's why we are up to 2039, protected globally. And last but not least important for the financial point of view, given the strong support also from advocacy groups like Michael J. Fox Foundation or Australian Government and Rett Syndrome Foundation, we're able to keep our burn rate a little bit modest, relatively modest.
We have, as of last quarter, $138.8 million in cash with no debt. Last year, the last fiscal year, we utilized $27.8 million. If you basically use that as a guidance for this year, which is probably about the range, most likely, then you can calculate easily that we have estimated four years of cash of runway, which is sustainable due to our disciplined operations and non-dilutive cash sources. I also like to mention that we enhanced and grew our team. We now have additional strong support with veterans from the biotech, but as well as big pharma. I want to mention that also that we have a strong support.
Regulatory side, we have a veteran from the FDA, from the neuro division, which joined us recently as well, which is important, of course, for our regulatory target groups. I also want to point out, we have a very strong support by scientific advisory board, with key opinion leaders in the relevant strong field. And what I mentioned, point out, Marwan Sabbagh, which is the Chairman of that scientific advisory board, and we're expanding as we see fit in that regard.
In summary, I'd like to point out that one of the key advantages, really, of our pipeline, of our platform, is really the precision medicine, that we know exactly what we're doing by activating sigma-1, which is down-regulated in pathology, it needs to be up-regulated, and we can do that with our small molecule, which shows that we're increasing the sigma-1 expression levels. What sigma-1 does 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-one to jump in and help to restore functionality, restore homeostasis, and bring proteins, which are misfolded quicker to washing them out, and enhance autophagy through Sigma-one activation, which is really nicely demonstrated in a paper with blarcamesine specifically, and it seems to be the key mechanism of Alzheimer's disease and probably other pathologies as well. So autophagy is impaired in many pathologies. Autophagy is the recycling mechanism of the neurons, which it cannot, like, let, you know, the trash out. They have to recycle it, and if that is impaired, the cells will eventually disintegrate or basically not function and lose brain mass.
And the fact that we demonstrated brain mass, delayed brain mass, delay of atrophy indicates that consistency. And last but not least, I also want to mention that our oral drug, since it's orally, once daily, can be shipped with Amazon or every means.
It is equitable, can be shipped to every regions in the land without any challenges at room temperature, is accessible to a diverse population, and is able, for that reason, to maintain overall more likely sustainable global healthcare system, given the challenges of expenses ramping up in the healthcare system, 'cause we don't need the margin of an antibody since we have a small molecule production, which is, of course, more convenient to from a financial point of view to develop. With that, if you have any more additional questions, please go to visit us on our website, www.anavex.com. Thank you very much for your attention. I appreciate it.