Welcome, everybody, to our final company presentation of the day, one we're very excited about. We're joined today by the CEO of Longeveron, Wa'el Hashad. Thank you for joining us. Longeveron is based in Miami, Florida. It's a clinical stage biotech company developing cell therapies for aging-related diseases, sorry, conditions and other, sorry, life-threatening conditions as well. There is a breakout room afterwards, but feel free to enjoy the presentation. The floor is yours.
Thank you, Eric.
Good afternoon, everyone. I'm sure by now everybody's tired, so I'll try to make it very engaging, and we do have a very exciting story to tell here today. So this is. We are a public company, so this is our forward-looking statement. You can see it completely. It's available on our website, so what is Longeveron? You can tell from the name the company was founded 10 years ago with an objective to address a large unmet need area of aging-related illnesses. It started with two programs, one around aging-related frailty, and the other one is around Alzheimer's dementia, and then, as it expanded, we added another life-threatening condition, actually in an opposite age group, which is called hypoplastic left heart syndrome, and it's a rare disease that affects children, and it gets diagnosed intrauterine. We'll get into that detail. The company has one product, its lead product.
It's called Lomecel-B, and we had conducted five or completed five clinical trials, phase I and phase II-B in aging-related frailty, phase I and phase II-A in Alzheimer's dementia, and phase I in HLHS, and currently wrapping up phase II-B in that indication, so that's the sixth trial, so we literally have a clinical pipeline and one product that addresses these three conditions, and because we have conducted these five trials, of course, we share the data and the information with the agency, with the FDA, and we have been able, based on the results of these trials, to obtain some important designations, so for the HLHS indications, we have orphan drug designation, we have a rare pediatric disease designation, and we have fast-track designation, and for the Alzheimer's indication, we have fast-track designation and RMAT designation, which is the Regenerative Medicine Advanced Therapy designation.
We have cash and cash equivalents to fund the company through the end of this year, if not even a little bit beyond that. And we carry no debts whatsoever on our books. And we have built a very proven management team that we built and strengthened our C-suite and the rest of the organization as we continue to advance our work. So what is Lomecel-B? Lomecel-B is an allogeneic donor-derived medicinal signaling cells or mesenchymal stem cells. We obtain it from healthy adult volunteers aged 18 to 45. We take the cells in our labs in Florida, in Miami, Florida. That's where the company is based. And we harvest these cells. We isolate them and then harvest them, and we produce billions of cells out of each donation. And then we use these cells and formulate.
The formulation is different between the hypoplastic left heart syndrome and the other indication. So after we do the final formulations, we cryopreserve them and keep them until the clinical use of these products. How does this allogeneic MSCs work? We believe it works through multifaceted mode of action. So anti-inflammatory is one that we have a lot of data to support that mechanism. We believe also there is cell-to-cell interaction, exosome and microRNA release, and mitochondrial transfer as well. As I mentioned, we have conducted five trials. So we finished phase I and phase II in Alzheimer's disease phase II-A and phase I and phase II-B in aging-related frailty. And currently, we are over 80% enrolled in our hypoplastic left heart syndrome phase II-B for this one. These three indications represent a big market potential. Of course, Alzheimer's is very well known.
It's 6.9, almost 7 million patients in the U.S. that are affected by that disease. It is up to a $10 billion market opportunity just in the U.S. and even double that amount outside of the U.S. Aging-related frailty, it's a growing population as the age continues to grow, 8.1 million. Estimated opportunity from a market size of about $4 billion-$8 billion, and the hypoplastic left heart syndrome, it's about 1,000 babies in the U.S., about 10,000 worldwide, and up to a $1 billion market potential in the U.S. alone, so very large market opportunity from a financial standpoint. I'll start reviewing each one of the indications that we work with. Hypoplastic left heart syndrome, not a lot of people know it. It's, as I said, it's an ultra-rare disease, so what is this condition and how our drug can potentially help?
You can see from the diagram on the left, you can see the normal heart. If you go remember the anatomy, there are four chambers. One of them is a very important one, which is the left ventricle. That's where the chamber that pumps the blood into the entire body. On the right-hand side, you can see the Hypoplastic Left Heart Syndrome. Babies are born with this condition. Basically, as you can see, the left ventricle is not fully developed. You can diagnose this condition, as I said, intrauterine. So before a mother gives birth, we know that these children have that condition. Right now, the way that these patients are treated is they go through a three-stage procedure.
The first one happens in the first week of life, and then the second one is about three to four months after birth, and then the third one, three to four years after birth, and they call the Norwood procedures, Glenn procedure, and Fontan procedure. The basic premise of these procedures is to convert or reconfigure the heart to allow the right ventricle to perform the left ventricle function. One thing you can notice here from the picture is that the right ventricle wall thickness, I don't know if I have here, you can see that the wall thickness of the right ventricle is much thinner than the left ventricle. So when you do the reconfiguration of that to allow the right ventricle to perform the left ventricle function, over time, with the load of work that now the right ventricle has to perform, it sometimes fails.
And ultimately, that leads either to death or the need to transplant the patient with a new heart. So where is the value of our product here? I will come to that. One thing that I want to tell you, because of this overload on the right ventricle, we have seen that the survival of these children with this procedure goes up to 80% five years survival and 50% into adolescence. So there is still an opportunity for these kids to improve on survival benefits that these surgeries provide. So what do we do? We are in the second surgery, which is the Glenn procedure. We inject our cells directly during the procedure, during the surgery. We inject them into the right ventricle wall. Sorry. You can see here, these are the eight different spots that we inject our cells.
The goal here is to strengthen or provide more support to the right ventricle to perform its function. How do we know that this is going to translate into a clinical outcome? We look for several things. We look for the ejection fraction, the amount of blood being pumped from the right ventricle. We also look at survival benefits, which is the most important clinical endpoint, which is, can these kids survive? We have conducted, as I mentioned, a phase I trial. In the phase I trial, we had 10 patients that enrolled. All of them received the cells during the second-stage surgery. What is the outcome of this? We have seen that these kids, all of the 10, have had safety. There were no issues, but most importantly, all of them had survival 100% without the need for transplant over a five-year period.
That is compared to the standard of care, which is, or the historical data that showed 80% survival in the same or 20% mortality, which is the opposite of that in the same period of time, and by the way, the 80% is also coming from one of the largest data sets for this one. It's called the Single Ventricle, and I will show you the data in a minute. There were no major adverse events, especially MACE, major adverse cardiovascular events. There were no other safety issues. There were no other hypersensitivity or reaction from this type of cells that has been given to these kids. You can compare the historical data, which is in the red. This is, as I said, coming from the largest data set for the hypoplastic left heart syndrome patients, over 160 patients.
This data set was a study that was sponsored by the NIH and the NHLBI. And in that, you can see that the survival after five years is only at 80%. In our phase I, we have seen 100% survival without the need of transplant. This data was the basis for us to receive all the designations that I mentioned, which is the fast-track rare pediatric and orphan disease designation. That encourages us to move to the next stage, which is doing a phase II-B, in which we are conducting a head-to-head trial versus the standard of care. So this trial is 38 patients. The first group is 19 patients. They will receive only the standard of care, which is the surgeries. And the other group will receive the standard of care plus the cells being injected into the right ventricle during the second-stage surgery.
We continue to evaluate the patients after the surgery for 12 months. And we are evaluating the right ventricular ejection fraction, and we are also looking at the 12-month survival as well as the length of hospitalization for these patients as primary outcome endpoints. Some of them are clinical, and some of them are surrogate endpoints. We're conducting this study in the top academic institutions in the country. You can see here, we're doing it at Mass General, Lurie, which is Northwestern, Emory Children's Hospital of Atlanta, and University of Utah, University of Texas, Children's Hospital of Philadelphia. So all the top academic institutions are participating in this trial. And this trial is also co-sponsored by the NIH and the NHLBI. We are over 80% enrolled, and we expect to finish enrollment earlier this year.
We'll follow, as I mentioned, the patients for 12 months, and then we'll have the final data readout. We met with the FDA in August of last year. We had a Type C meeting, and we confirmed with the FDA that this trial will be accepted as a pivotal trial for full approval. They also agreed to all of the endpoints as well as all the other requirements for approval, including potency assay and CMC plan. We are right now full steam ahead preparing for our BLA, and we plan to file for a BLA sometime in 2026 once we have the data readout. Second indication that we're working on is Alzheimer's disease. As many of you know, Alzheimer's is a big issue and had its own fair challenges of getting drugs even approved to the market.
I think over the years, we have learned a lot about Alzheimer's disease and that it's actually much more complex than just that we initially thought, and one of the things that we believe that affects Alzheimer's or associated with Alzheimer's is that we believe that patients with Alzheimer's exhibit a high level of inflammation, and this is where our products bring, as I mentioned, one of the mechanisms of action for our cells is an anti-inflammatory plus it's a pro-vascular, so based on the mechanism of action, we believe that there could be a value for this product in this disease. We have conducted two trials. The first one was a 33 patients head-to-head with placebo, and we tested just a single dose of Lomecel-B, one low dose, one high dose.
And in the first trial, we have seen a positive signal on the efficacy, and there was absolutely met the endpoints when it comes to the safety of the product. We did not have any single death or infusion-related reaction or any other major adverse events. That encourages us to move to the second phase II-A, again, head-to-head trial. And in this one, we decided to test multiple dosing. So you can see we had one to one to one to one. One group was placebo, and the other three groups were a single low dose, multi-dose of multi-low dose and multi-high dose, 25 million and 100 million cells. And we followed up at the primary endpoint with safety, and the secondary endpoint was a composite Alzheimer's disease score of imaging, neurocognitive, and score. So cognition as well as activity and quality of life and activity of daily living.
The trial results were positive. We presented this at the Alzheimer's Association International Conference in last July, and as a featured presentation for the conference, we also submitted the results of the trial to the FDA, and we obtained an RMAT designation, as I mentioned, and we plan to meet with the FDA in the 1st quarter to discuss the development plan for the trial. The trial achieved its primary and secondary endpoints and showed the statistical significance in all the pre-specified endpoints that we had. The product has continued to show an established safety profile. We did not have any, as I mentioned, any infusion-related reaction. There was no single case of ARIA or amyloid-related imaging abnormalities. We also had no association of any negative cognitive function.
To the contrary, we have seen an improvement in cognitive functions in some of the scales compared to placebo, such as the Montreal Cognitive Assessment and MMSE. One of the most important findings as well, and we conducted this, is we have wanted to employ imaging because we believe that imaging is a very precise measure. We have done MRI. All of them were done at a central lab reading to avoid any differences. What we saw from the brain MRI was that we had 49% less decline in the brain atrophy in the treated group versus the placebo group. That was a very, and that was highly statistical significant for this trial. The results with the CLEAR MIND trial support the further development with the program, which we intend to do.
As I said, we're planning to meet with the agency in the 1st quarter to discuss the development plan. And we believe that the best path forward on Alzheimer's is that we, through partnership, as everybody knows, it's an expensive program. So we definitely like to get the support. And we're waiting for the FDA minutes for the meetings, and then we'll engage in that partnership discussions. Last program, which I will get to, the aging-related frailty. It's a disease that affects a large population as we continue to see the age or the life expectancy go higher. It affects about 15% of the population above the age of 65. As I mentioned, we conducted two trials, phase I and phase II-B. We used a test called Six-Minute Walk Test, which is basically how many steps a patient can do in six minutes. And it is a patient-reported outcome.
It's a validated test. And we have seen a significant improvement in our Phase II-B in nine months between the treated group and the placebo group. So that supports the indication. However, for just a prioritization, we decided to focus mostly on the HLHS and the Alzheimer's program as our top priority for the time being. As I mentioned, we have about $23 million, which can take us all the way to the end of 2025, if not into the earlier part of 2026. And we have 14.8 million shares of common stock being traded in the public. This is a management team that runs the company. And all of them, and by the way, more details are available on our website, but you can see a lot of them have been part of driving innovative medicine and getting drugs approved. And we continue to be committed.
So in summary, I want to tell you that we are a stem cell company. We have one product and multiple indications. Our pipeline in HLHS and aging-related frailty represents a very large opportunity in the U.S. and the international markets. We have multiple designations from the FDA, including two fast-tracks: RMAT, orphan drug designation, and rare pediatric disease. I forgot to mention also that the rare pediatric disease comes with a priority review voucher that is available to us at the time of approval. The FDA, we have cash and cash equivalent throughout the early part of 2026, and we have a proven management that's really dedicated and committed to advancing our work. With that, I thank you for your attendance, and I'm happy to take questions later on. Thank you. If you might have a question. All right. So every batch, yeah.
So we do not do the collection ourselves. We get GMP-grade bone marrow that is approved from large suppliers. So that is a normal part of the work that we do. And that's why the FDA required the employment of a potency assay that we have to perform to ensure that batch-to-batch variability is not going to be affected. So yeah. No. It's mesenchymal stem cell. It's not programmed. Yeah. So we isolate the mesenchymal stem cells, and we expand them in our lab. But we don't program them or anything like that. And then we infuse them. And I can have Dr. Hare here, who can probably, he's the founder of the company and the owner of the technology, can do this.
But basically, because of the high level of inflammation in the brain that is associated with Alzheimer's, that attracts the cells to the site, and that's how we go there and reach the site of impact. I don't believe they have any chemotaxis. I don't know about that. Josh, do you want to? Yes. Yeah. As I mentioned, it's a multidisciplinary mechanism of action. It's a pro-vascular anti-inflammatory. And it's also, we believe these cells excrete exosome and microRNA. And all of this is the reason why they do the effect. So it's not, I know it is with cell therapy, everybody expects a singular approach to mechanism of action. With cell therapy, it's actually a multidisciplinary approach to action. So the action doesn't happen just because one, multiple things lead to this outcome. Systemic action, yeah. So we had a meeting with the FDA, and they agreed to our proposed plan.
Not just the clinical plan and the primary endpoints and the study design and so on, but they also reviewed everything from potency assay, CMC plan, and everything. They agreed to all of our plans. The FDA likes to see clinical endpoints. That's the most important one. Survival or mortality plus hospitalization are very important to them. Then the surrogate endpoint that they agreed to is the ejection fraction, the right ventricular ejection fraction. It's a combined endpoint. Yeah.
Thank you so much to Wa'el Hashad, CEO of Longeveron.