Annual Global Investment Conference. My name is Li Chen, and I am an equity research associate. I'd like to welcome our next speaker, John Climaco, CEO of CNS Pharmaceuticals. It's a clinical stage biotech company focusing on development of therapies for treatment of brain tumors. John, please go ahead.
Thank you so much. Great to be here, and thanks, everyone, for watching. So yes, I'm John Climaco. I'm the CEO of CNS Pharmaceuticals. We trade on Nasdaq under the ticker CNSP. Obviously, we will be making forward-looking statements today, so please, review this. So we are a drug development company in neuro-oncology, and right now we are specializing in glioblastoma, the most aggressive and common primary form of brain cancer. We have two programs. Our lead program is a novel anthracycline called Berubicin. It's the first drug of its kind to cross the blood-brain barrier. We have a fully enrolled, designed-to-be-pivotal study that will release top-line data in the first half of 2025, so we're very excited about that. This drug is extremely exciting.
Again, it is the first anthracycline, first drug of its kind, shown to cross the blood-brain barrier, and there is importantly no evidence of cardiotoxicity with this molecule. That would also make it unique among the 60-plus-year-old in clinical use anthracycline family. It was developed at the MD Anderson Cancer Center, ranked the number one cancer center in the world. Our second drug, which we just announced as an in-license, is TPI-287. This is a novel late-stage abeotaxane, a blood-brain penetrable drug designed specifically for the treatment of brain malignancies. What's exciting about this molecule is, again, it is the first drug of its kind to penetrate the blood-brain barrier. It's been studied in over 350 patients to date, including both combination and monotherapies.
It has an orphan designation, as does our lead program, and we'll talk a lot more about that in a moment. So just taking a look at our pipeline, we are very, very sharply focused on neuro-oncology and glioblastoma in particular. So let's talk a little bit about glioblastoma. Again, the most aggressive, deadly, and currently incurable brain cancer, 12 months -18 months average life expectancy at diagnosis. We are studying Berubicin, and ultimately, we'll be studying TPI-287 in refractory or relapsed patients. And if you can believe it, these folks have an average life expectancy of 4 months -6 months, so very bleak situation. No drugs approved for secondary or relapse patients. Over 50,000 cases a year in the 8 major markets around the world. This is an incurable cancer.
There's basically one drug used right now up front in first-line therapy, that's called temozolomide, only works in about four out of 10 cases, and yet this drug delivers many billions in annual sales, despite being off patent for over 12 years. So what is it about this cancer that is so difficult to treat? Primarily, it is not just its aggressiveness and how it grows intertwined among the healthy brain tissue, it's that it is located as a primary tumor inside the blood-brain barrier, this very specialized network of cells that protects the brain from harmful substances. But unfortunately, it also limits the ability of many classes of potentially helpful chemotherapy drugs to attack and destroy these tumors.
So you have the passive tight junctions of the endothelial cells themselves, and then you have these very important active efflux pumps that are molecular-level pumps that really do the bulk of the work of the blood-brain barrier. They are so powerful they can defeat the heart itself in terms of its ability to pump potentially helpful substances up into the brain. So why are we so excited about our drugs? Well, primarily, we are excited about these drugs because they both come from known successful classes of drugs, anthracyclines and taxanes, and these drugs typically do not penetrate the blood-brain barrier, but Berubicin and TPI-287 do. What you can see here are very interesting results from the phase I study of Berubicin, where 44% of patients demonstrated stable disease or better.
We had two partial responders with up to 80% tumor shrinkage. This drug was extremely well-tolerated. We've seen no off-target toxicities, and we had one durable, complete response. This gentleman is still cancer-free, seventeen years after treatment with Berubicin. You can see his images on the right. These are basically unheard of, results for a cancer that, as I said earlier, tends to destroy people, when they relapse, and almost everyone will, in under a year. Now, I think from an investor perspective, what's exciting about what we are doing is that the Berubicin program is about as de-risked as you can get a drug development program, certainly in neuro-oncology. All of our investigators are contracted, all of our patients are dosed, and as you see on the right side of this image, that's 252 patients.
If this sounds more like a phase III study already than a phase II, it's because this study was designed to be pivotal. We said when we examined the phase I data, we know, colloquially speaking, this drug works. It is having activity in this cancer because you don't see people living years and months longer. You don't see their tumors shrinking or resolving completely spontaneously. It must be the impact of this molecule. So again, de-risked, all the investigators, all the patients are dosed, all the drug supply is in hand, and the pivotal endpoint of overall survival, that is how long these patients are living in this blinded multi-center international study being conducted in the United States and in Europe. We will reach that endpoint in six to 12 months.
It will be in the first half of next year, really under twelve months, frankly. Independent Data Safety Monitoring Board in December recommended the continuation of the trial after examining the data at midpoint. That was both efficacy and safety data. So as you can see, we have this very large study. This is one of the largest glioblastoma studies being conducted anywhere in the world. In fact, it may be the largest study. The focus is entirely on Berubicin. There is no confounding or noise-creating data. What we are really looking at is, does this very interesting blood-brain barrier-penetrant anthracycline help these patients to live longer than they would otherwise? And again, anthracyclines have been around in clinical use for nearly seventy years, so this is an incredibly trusted, well-studied, well-understood, well-regulated set of molecules. What makes this one special?
It was designed to penetrate the blood-brain barrier. Now, there are about fifteen thousand cases in the U.S. each year of this disease. That's about a $2.3 billion market. Fifty thousand cases globally. Again, no drug anywhere in the world is approved for relapsed high-grade gliomas. Those are glioblastomas. But what are the really exciting things about this molecule is, given that it comes from this well-understood and trusted in the clinic by millions of physicians over decades, treating, you know, millions of patients with a variety of tumors, is that many of the tumors that we know are sensitive to anthracycline therapy have a well-known metastatic pathway to the brain.
Unfortunately for those patients, whether they are breast cancer patients or other patients in different areas of the body, when their cancer metastasizes to the brain, they have the same problem that the GBM patients have. They have a tumor now growing inside that very difficult-to-penetrate blood-brain barrier. So even knowing that their cancers are anthracycline sensitive, when they grow inside the blood-brain barrier, currently available anthracyclines are not going to be able to help these patients. That's where Berubicin steps into the fray. We have chosen GBM, not just because it's a primary tumor, and that this drug can penetrate the blood-brain barrier. We chose it because the regulatory bar globally is as low as it gets. There is simply no competing molecule or therapeutic because there isn't anything approved.
But assuming we do achieve this very lofty goal, and our trial data and the appearance of, you know, the trial and the patients so far looks very good, we have what we often refer to as a pipeline in a drug. That is a drug that we could study in other cancers with well-known metastatic pathways to the brain for the rest of our career, to the benefit of these patients and our shareholders. And it, when you see the estimated population of these cancers in the U.S. alone, you can see that metastatic breast cancer at 45,000 cases a year absolutely dwarfs the number of primary brain tumors that you see. It dwarfs the pediatric. It's a huge population. It's a massive unmet need.
We have a molecule that shows every indication of a possibility of addressing these markets. So we're not just excited about the neuro-oncology opportunities, we're excited about what comes next. Intellectual property for Berubicin, it is an orphan drug. It is a new chemical entity. We are working on additional patent protections, particularly related to our manufacturing discoveries for this drug. And we will be making an orphan filing in Europe when we have the data from this study. So we feel we have a very robust fence around this molecule. So let's talk about the new molecule, our latest program, in-licensed TPI-287. This is a late-stage, novel, blood-brain barrier permeable abeotaxane designed for the treatment of brain malignancies. So what is exciting about this molecule?
Well, again, this drug has been very well-studied, over 350 patients to date. It has shown an outstanding efficacy and safety profile in prior trials for glioblastoma, and much like our Berubicin program, this molecule comes ultimately beginning at the taxane and to the abeotaxane class of drugs. This molecule has been designed specifically to do what no other molecule in its family does, and that is cross the blood-brain barrier. That's all great theory, but the rubber meets the road when you put these drugs into human beings and you see how they behave. And take a look at these very exciting data. 60% overall response rate, that's 39 partial responses and three complete responses. 96% disease control rate and a 13.4-month median and 64% one-year overall survival.
This is far in excess of the median survival for glioblastoma, so we are very excited that we have yet another molecule from a trusted, tried, and true class of drugs that has been in clinical use for decades, that can do something that none of those other molecules in that class can do, and that is cross the blood-brain barrier and actually attack these tumors in human beings, so take a look at some of this data. Readily penetrates the blood-brain barrier, much in excess of docetaxel and paclitaxel, from which this is ultimately derived. Incredible concentration inside the blood-brain barrier in animal models, but when we take a look again at that human data, where really the rubber meets the road, a 60% overall objective response rate, 96% disease control rate, and most importantly, 13.4-month overall median survival.
That is far in excess of the four to six months that these patients typically have when untreated, so this slide alone tells you why did we license this drug, and why are we starting a second program? And the reason is, there are simply vanishingly few drugs that will cross the blood-brain barrier, and even fewer that have shown anything like these kind of results. You can take a look at incredible improved glioblastoma survival in combination with bevacizumab, which is also known as Avastin, typically referred to as the last drug that you take, but when in combination with TPI-287, we see tremendous progression-free survival and overall survival improvement in these patients, so we've got two drugs. Again, they are both attacking the same problem. We have always said there is not going to be a magic bullet for glioblastoma.
Our job is to expand the armamentarium and give patients and their treating clinicians the tools they need to extend overall survival and quality of life while they fight this disease. We've got an outstanding team with great pedigrees. I'll just point out our two scientific leads, Dr. Sandra Silberman, our Chief Medical Officer. She was the former global head of clinical development at Novartis, where she developed Gleevec. So this is someone who not just has studied these cancers and these diseases, but really knows how to design trials that get blockbuster drugs approved. Similar to Dr. Picker, over thirty-five years of drug development on the CMC front, an incredible chemist. Carboplatin, another blockbuster, global blockbuster chemotherapy drug, was Don's design.
So we have got, for a small organization, the chops to pull this audacious goal off. We have done nothing if we haven't executed exactly on our plan, more or less on schedule, the way we laid it out years ago when we first went public. We started with that orphan drug designation in 2020. Our dosing of patients in this study commenced for Berubicin in September of 2021. A little more than two years later, while other studies around the world struggled to enroll patients, we enrolled over 250 of them. Why would you say that is? It's because people understand that this drug, as an anthracycline, is essentially the hammer in the oncologist's toolkit, is a tried and true way of destroying cancer cells.
It's just never been able to be deployed inside the blood-brain barrier for primary and metastatic brain cancers before Berubicin, because no one had created a molecule that could cross the blood-brain barrier, and we have. We completed enrollment in Berubicin in January 2024, and later this year, of course, we went ahead and in-licensed TPI-287. You can expect us to build on this executional success, because what you're really seeing here is a tremendous investment of time, resources, and manpower to build a global clinical network of the highest performing, highest throughput, best scientific clinicians out there in neuro-oncology, who can bring us the patients that we need to study these drugs on. We are going to do it again with TPI-287.
So again, as investment highlights, while we wrap this up, our lead program, Berubicin, a novel anthracycline, the hammer in the oncologist's toolbox. This class of drugs has been in use for almost seven decades. They are safe, they are effective, but historically, they do not cross the blood-brain barrier. We have solved that problem with Berubicin. It is the first drug of its kind to do that. We have a fully enrolled, designed-to-be-pivotal study that will read top-line data in the first half of two thousand and twenty-five. From a capital perspective, we have the money that we need to finish this study and deliver those results, so we are feeling that this program has been as de-risked for investors as one can have.
It is now a pure play in chemistry and biology, as this very exciting and potent molecule comes in direct contact with this very difficult cancer. Our pipeline has now been expanded. We have spent years studying hundreds of potential molecules that we might be interested in working with. We landed on TPI-287, a novel blood-brain penetrant abeotaxane, again, specifically designed to cross the blood-brain barrier, to take abeotaxanes where they have never gone before, and to treat primary and metastatic brain cancers, in a way that has never been available to clinicians and patients before. So this is a late-stage molecule. It has been studied in over 350 patients. It is very safe, it is well-tolerated, and what we intend to find out is just how effective it is against brain cancer.
We know, as I said, that we have seen very exciting results, both in terms of complete responses, partial responses, increases to overall survival and tolerability. It is an orphan-designated drug, and our goal is to put this drug back in the clinic, studying glioblastoma, in 2025. So thank you very much, H.C. Wainwright, for having us. Thank you for watching this, and that is CNS Pharmaceuticals. I'm John Climaco, the CEO. We trade on the Nasdaq under the ticker CNSP. Thank you very much.