Good morning, everyone, and thank you for joining us for HG Wainwright's 27th Annual Global Investment Conference. My name is Max Mahr, I'm an Analyst here on our Corporate Access team at H.C. Wainwright . I'd like now to turn our attention to John Climaco, who is CEO of CNS Pharmaceuticals. John, the floor is yours.
Thanks, Max, and thanks to H.C. Wainwright for having us. Yes, I'm the CEO of CNS Pharmaceuticals, and we are a drug developer that specializes in neuro-oncology treatments for primary and metastatic brain cancers. We take a little bit different approach. We are specializing in drugs that have a tried and true record that we know work against cancer cells, and we get them across the blood-brain barrier where the tumors are located in a way that no one else can. We've got a very strong financial position right now, over $12 million at close of Q2. We have a really unique proven clinical development engine, a global system of investigators and trial sites around the world that allows us to do this work extremely efficiently. We are advancing very rapidly back into the clinic with our lead compound, TPI 287, for the treatment of glioblastoma multiforme.
This is a late-stage novel blood-brain barrier penetrant abeotaxane. It's a member of the taxane class. Again, as I said earlier, a tried and true class of drugs that works, but taxanes do not historically get across the blood-brain barrier, so they're useless for brain cancer. Our taxane is different. It does, and it has an incredible track record that we'll talk about a little bit later. Studied in over 350 patients in multiple trials, both as monotherapy and combination. We reported our primary analysis of our second compound, Berubicin, a novel anthracycline also blood-brain barrier penetrant for second-line GBM, and we are doing ongoing analysis now and looking at strategic and regulatory next steps for that program, about which we are actually very optimistic. One of our main strengths really as a company is our focus. We are entirely dedicated to the neuro-oncology space and GBM in particular.
It's been that way since the day we founded this company, and seven years later, that is exactly what we're still doing. This is a very difficult problem to crack. It is one of the two, you might say, holy grails in oncology, the other being pancreatic cancer where the diagnosis is really a death sentence. Glioblastoma strikes as a lightning bolt for patients. We'll talk a little bit more about it in a second, but a very, very challenging disease to treat. We have an outstanding compound, TPI 287. It is ready to go into a phase II study, which we'll be getting the first half of next year, and this is a very exciting compound.
Again, you'll hear me say this numerous times, you know, what we believe is needed to move the needle in GBM and neuro-oncology is not necessarily the targeted therapies that grab the headlines and sound so sexy. We're still missing that foundational one-size-even-fits-most chemotherapy solution that is present all across oncology but is really absent in neuro-oncology because of the blood-brain barrier. We have two drugs that solve that problem, and they are outstanding candidates that can really make a difference for patients. The story for us is much bigger, and you should keep in mind beyond our focus, the next level once we see traction with these drugs is metastatic disease. Now, the primary brain cancer market is very healthy. This is a multi-billion dollar market around the world, even though we see these are orphan diseases. Only about 15,000 cases of glioblastoma each year in the United States.
However, take a look at that third box at the bottom, brain metastases. That's primarily triple negative breast cancer, very, you know, common cancer. 45,000 patients each year have metastases to the brain. Now, those patients, when their primary cancer in their breast metastasizes to the brain, they have exactly the same problem as the GBM patients have, sadly, which is they now have a tumor growing inside the blood-brain barrier, and their treatment options diminish almost to zero. This is a grave problem. We have an outstanding primary market, but we know that once these drugs come to the market for the primary cancer market, they're going to have tremendous utility for the metastatic market as well. I mentioned an engine that we have built over many years. We conducted a global study of Berubicin.
We enrolled 247 patients in 27 months, which is almost unheard of in an orphan disease like that globally. We had 46 centers in five countries around the world. These were hand-picked centers and investigators for their patient throughput, their expertise, their ability to follow protocol and produce clean data. The beauty of this engine, this network that we created for Berubicin, is that it can be entirely repurposed for our TPI 287 program. We are no longer going into the clinic from a standing start. We know our investigators. We know how they work. We know their institutions. We know who produces lots of data and who produces less. All of these things give us a huge time and money advantage in conducting these expensive and difficult to manage trials. We did this intentionally. We spent a lot. We did things that a lot of developers don't do.
We have patient concierge services. We made the participation in our study for investigators and for these very sick patients and their family as easy as possible because you have to understand, at the end of the day, this is a very difficult decision that patients and their families make along with their treating physician. There are hundreds of GBM trials going on globally at any one time. To choose what may be your last chance at a cure, your last chance for extension of progression-free survival or overall survival is literally a life and death decision for these folks. We made a great effort. We spent a great deal of money to make that decision as easy as possible.
I believe at the heart of the decision for us and why we've had such success recruiting when others have not is that our drugs, again, come from tried and true classes. There's nothing mysterious about a taxane. There's nothing mysterious about an anthracycline. These drugs have been on the market for decades treating other cancers in other formulations. What we have is something completely unique, but mechanistically, it is very well- understood by clinicians who can explain it to their patients and also by regulators. Just briefly, glioblastoma, I'm sure many of you are familiar with this disease. This is one of the most aggressive and deadly forms of cancer. The current standard of care, sadly, is totally ineffective in 60% of patients. Imagine that. We don't even have a one-size-fits-most chemotherapy solution for these patients. This can affect cognition. It can affect mood and behavior.
It essentially destroys who you are as a person before it ultimately kills you in 12- 18 months. There's about 50,000 new cases of this disease in the eight major markets each year, and it represents almost half of all primary brain malignant tumors. This is a real crisis. There is no cure. This is essentially a death sentence. As I said earlier, and I've mentioned this a couple of times, we believe that the nature of the problem here is not so much the tumor itself. Why do we believe that? Because we know that when we take glioblastoma tumors in a test tube and we introduce legions of common anti-cancer drugs to them, they all destroy the tumor cells beautifully at the bench. The problem is trying to destroy them in the human being.
The reason we believe that is such a difficult challenge is the blood-brain barrier. It is this specialized network of endothelial cells that surrounds your brain to protect it from potentially harmful substances. Unfortunately, almost all chemotherapy drugs are substrates of the blood-brain barrier's pumping mechanisms that are so powerful they can actively push these drugs out away from the brain faster than the heart can pump them in. Drug delivery to the site of the tumor, we believe, is the crux of the problem, and our drugs address it directly. Let's start with TPI 287. That's our lead compound. This is a late-stage novel blood-brain barrier-penetrant, abeotaxane , specifically designed to bypass the blood-brain barrier and treat primary and metastatic brain malignancies. Now, again, this is a classic taxane molecule at the bottom, paclitaxel. That molecule does not pass the blood-brain barrier.
There were deliberate and very careful serial chemistry changes to the central rings in that molecule that changed the lipophilicity of the molecule and changed the size of the molecule and its molecular weight in such a way that it is no longer in the form of TPI 287, our drug, a substrate for the blood-brain barrier, particularly the PGP pumping mechanism, which is upregulated in cells that become taxane resistant, and that is the crux of this problem. TPI 287 solves that beautifully. What's really significant, again, about this is we know this class of drugs works against these types of solid tumors. How do we know that? Because there's not an oncologist in the world today that didn't train in medical school on taxanes. That's how long they've been around. They are very effective. They are very safe. They are very well- tolerated.
Their Achilles heel when it comes to neuro-oncology is that all of them are substrates for the PGP protein pump. TPI 287 is not. We can see that the concentrations in animal models in the brain blood are very high for TPI 287, outstanding penetration. It proves out our hypothesis that this modified molecule will do what it was intended to do, but that's really a nice first step. The rubber meets the road, of course, when we see what this thing does in human beings. It's been evaluated already in extensive phase I and phase II clinical trials in over 350 patients in mono and combination therapy. It has fast-track designation. It has orphan designation. We are actively engaging with regulators on our trial design right now with the intention, again, of bringing this program back into the clinic in the first half of next year.
Specifically, what are we going to do with it? We're going to combine it with another drug called bevacizumab. Bevacizumab is part of the current armamentarium for glioblastoma. It's a very interesting and important drug. However, it has an Achilles heel of its own. Bevacizumab is very good at extending what is called progression-free survival, which is essentially a state of stable disease for the patient. The disease isn't getting any worse, but it's not getting any better. Unfortunately, with bevacizumab, eventually when you take it, you will progress, and there really isn't much you can do beyond that. What we're looking for, what FDA is looking for, is an extension of overall survival. Overall survival, in contrast to progression-free survival, is exactly what it sounds like. How long does the patient live overall?
We know that bevacizumab doesn't affect overall survival, but let's take a look at what we see in combination with TPI 287. We can see that on the right-hand graph, our median overall survival is 13.4 months, a significant extension of survival for these second-line GBM patients whose general overall survival is only six to eight months. 12-month overall survival is 64%, which is absolutely remarkable. In this particular study, there were multiple durable complete responses, which is effectively a cure. Those are people whose cancer has disappeared, the tumors are gone, and there is no evidence of the cancer at all. There were also multiple partial responses with tumor shrinkage up to 80%. Very, very significant and unique data.
We can see here again multiple studies overlaid on top of one another, but most importantly, take a look at the bevacizumab data alone, and that's 9.2 months in the lower overall survival graph. Bevacizumab plus TPI 287, 13.4 months, a six-month additional extension in overall survival. Let me tell you, for people who are projected statistically to only live six months, that's a doubling of their life expectancy. That is very significant for these patients, particularly when we have the opportunity to affect a durable complete response or a partial response, which in GBM is almost unheard of for current therapeutic options. This is a very powerful drug. This is a very safe drug. We know that from hundreds of patients who have been tested with it. It is very well- tolerated with a very well-managed safety profile.
We can build our safety and our tolerability on top of decades of clinical use in millions of patients with taxanes around the world. Clinicians know how to manage this type of drug very, very effectively. That is important because the last thing that you want to do is have a situation where the cure is bad or worse than the disease. Let's talk now about our second drug briefly, Berubicin. We are currently evaluating options for this drug. We completed earlier this year a global designed to be pivotal study of Berubicin. This is a novel blood-brain barrier-penetrant anthracycline, again, very similar in sort of philosophy to the taxane group. This is now an anthracycline. Anthracyclines have been around for 70 years in clinical use. They are effective. They are safe. They are very well- tolerated.
We tested this drug in 252 patients, randomized two to one against a drug called Lomustine, and we came out looking like this. Obviously, we were hoping for a much more significant separation, but what we see here is important for the following reason. Clearly, we see Lomustine and Berubicin behaving on overall survival almost identical. That is not statistically significant. It did not reach its primary endpoint. However, this is clinically extremely relevant, and the reason for that is as follows. Right now, we only have two drugs that we throw at glioblastoma. We have a drug called temozolomide, which I mentioned earlier as the primary standard of care today. That drug is only effective in 40% of patients. Imagine that. From a first-line perspective, you only have about 40%, four out of 10 patients who have a drug that's effective at all.
60% of those patients have no therapy on a primary first-line basis that's effective whatsoever. The second drug that we often throw at GBM is called Lomustine. It's another alkylating agent, mechanistically similar to temozolomide, effective but not incredibly effective. In combination, they can be used to extend overall survival. What our clinician investigators are telling us is that with only two drugs in the current armamentarium, a third drug, even if it's as good as Lomustine and no better, would be tremendously helpful. Not everyone responds to Lomustine. Not everyone can tolerate Lomustine, and everyone will eventually fail Lomustine, and when they do, there's very little to do for these patients. If Berubicin were available, they could bring in a mechanistically distinct drug for a third line to try to give patients an additional overall survival extension.
With recent changes at FDA focusing on rare diseases and focusing on patient access to novel therapies, we believe that there may be a pathway opening for this drug. We are very optimistic about this data, particularly because it is a very safe and well-tolerated drug. We have multiple patients that have been on this drug for over two years. That means they are obviously surviving, which is much longer than the six months they were expected to survive as recurrent GBM patients. What's also interesting about that is that anthracyclines, as a class, are known cardiotoxins. That is the Achilles heel of the anthracycline group. There is a black box label on all anthracyclines. There is a lifetime maximum dosing.
We have far exceeded that in two years with these patients on Berubicin, and we have, based on very sophisticated measures of troponins and right ventricular ejection fractions, no cardiotoxicity whatsoever. If that is proven out, this will be the first anthracycline demonstrated to not have cardiotoxicity. That is a massive breakthrough in and of itself, which could have utility far beyond the neuro-oncology space. While we didn't meet our primary endpoint on this study, we are very bullish on Berubicin and its possibilities going forward. Just a moment here on our finances. Again, $12.1 million in cash as of June 30, 2025. That takes us well out into 2026, into the second half of 2026. Our volume average is almost 25,000 shares. That's over three months, and we certainly have days when we are many, many times that. Our market cap, you can see here, 75% discount to cash.
Draw your own conclusion there. Right now, we're feeling very stable on the cash in a very difficult biotech market. We have two fantastic drugs. I'll mention here briefly, we have an outstanding team of executives here, two superstars in particular, Dr. Sandra Silberman, who was the Head of Global Clinical Development at Novartis and the clinician responsible for bringing the multi-billion dollar drug Gleevec to the market. Similarly, our CSO, Dr. Picker, he is really responsible for much of the very challenging chemistry behind these molecules. Carboplatin was one of his drugs, another multi-billion dollar global blockbuster. We are a small team, I think, that punches far above its weight given the decades of experience and history that we have behind us. CNS Pharmaceuticals, we trade on the NASDAQ. Our symbol is CNSP. We are a neuro-oncology drug development company.
We have two drugs very tightly focused on glioblastoma. We have plenty of cash out well into the second half of next year, and thank you for listening.