Good morning and welcome to the CNS Pharmaceuticals Corporate Update webinar. This morning, the company announced the expansion of its pipeline with the in-license of a late-stage novel potential blood-brain barrier permeable abeotaxane for the treatment of brain malignancies. My name is Janine Thomas. I am CEO of JTCIR, and I will be the moderator for today's event. As a brief reminder, all participants are currently in listen-only mode. Following the presentation, there will be a question-and-answer session. If you have a question, click on the Q&A button at the bottom of your screen and type in your answer. Note that this webinar is being recorded at the company's request, and a replay will be made available on the company's website following the end of the event.
At this time, I'd like to remind our listeners that remarks made during this webcast may state management's intentions, beliefs, expectations, or future projections. These are forward-looking statements and involve risks and uncertainties. Forward-looking statements on this call are made pursuant to the safe harbor provisions of the federal securities laws and are based on CNS Pharmaceuticals' current expectations, and actual results can differ materially. As a result, you should not place undue reliance on any forward-looking statements. Some of the factors that could cause actual results to differ materially from these contemplated by such forward-looking statements are discussed in the periodic report CNS Pharmaceuticals files with the Securities and Exchange Commission. These documents are available in the investor section of the company's website and on the Securities and Exchange Commission's website. We encourage you to review these documents carefully.
Additionally, certain information contained in this webcast relates to or is based on studies, publications, surveys, and other data obtained from third-party sources and the company's own estimates and research. While the company believes these third-party sources to be reliable as of the date of this presentation, it is not independently verified and makes no representation as to the adequacy, fairness, accuracy, or completeness of or that any independent source has verified any information obtained by the third-party sources. So here we are ready to get started. Joining us on today's webinar from CNS Pharmaceuticals' leadership team are John Climaco, Chief Executive Officer; Dr. Sandra Silberman, Chief Medical Officer; and we are also joined by Dr. Samuel Goldlust, a leading neuro-oncologist and key opinion leader who serves as the Medical Director of Neurology and Neuro-oncology at Saint Luke's Health System. I will now turn it over to you, John.
All right. Thanks, Janine. Thanks, everyone, for joining. Well, as Janine said, we made a major announcement today, and we are really thrilled. This has been a long time coming. I want to introduce this just by kind of reminding everyone what our mission is. You know, we are a drug development company, but we are a very specialized drug development company. We are working specifically on one of the worst of the worst cancers. That's glioblastoma, this indiscriminate killer currently considered incurable. We have an incredible drug in a late-stage trial, Berubicin, a novel anthracycline that crosses the blood-brain barrier. We are thrilled about that program. We are pushing it to its conclusion with the release of data, top-line data for that potentially pivotal study planned for the first half of next year.
Now, we have been so laser-focused, as we often say, on Berubicin that you might think that that's all we are doing. But in fact, for years, we have been looking for another compound to build off the success that we've had with Berubicin. And for this disease, that has been an incredibly challenging process. We have spent over four years evaluating 200+ compounds and projects in this space, looking for something that we felt was as clinically impressive as Berubicin. And we have found just such a drug in TPI 287. Next slide, please. This drug is the equal in terms of our excitement to Berubicin. This is, as Janine said, a late-stage novel abeotaxane that crosses the blood-brain barrier and has some really outstanding clinical data behind it.
So we think that this, because of its mechanism of action, which is completely distinct from Berubicin, how it could be used and the clinical data behind it, we think it is just an incredible complement to what we have and an expansion and furthering of our mission. We have always said, as excited as we are about Berubicin, there is unfortunately not going to be a silver bullet for this very difficult-to-treat disease and metastatic brain cancers like it that grow behind the blood-brain barrier, which make it very difficult to get potentially helpful drugs to the site of the tumor. So what we are announcing today is an exclusive license for TPI 287 from Cortice Biosciences in exchange for a little over 600,000 shares of the company's common stock. We will be developing this program starting immediately. And next slide, please.
Let me tell you just a little bit without stealing the thunder from my colleagues, Doctors Silberman and Goldlust. But TPI 287, again, builds on the success that we have with Berubicin. We have developed an incredible clinical network, and I like to call an engine for developing drugs like this. You know, it's one thing to have the molecule. It's an entirely different thing to plan, organize, and execute the type of trial that we're doing for Berubicin that we've often talked about, which is a drug like this deserves a conclusive answer. The patients, the FDA, our shareholders deserve a conclusive answer. And that's what we've designed with our CNS 201 study of Berubicin.
We are moving in the same direction with a drug that is equally deserving of a pivotal study, and that is this late-stage blood-brain permeable abeotaxane that was designed specifically for the treatment of cancers like this. Let me just tell you a couple of really exciting things about this. First of all, this drug has been studied in a number of phase one trials in over 350 patients to date. So this drug has seen a lot of action in human beings, demonstrating its safety and efficacy profile, which originally is what really excited us about this drug.
Specific to GBM, there's a few statistics I think that should jump off the page for anybody that's been following our story: a 60% overall response rate, 40% of patients with progression-free survival at 6 months, and most significantly, and I know Doctors Silberman and Goldlust will talk about this a lot, 13.4-month median overall survival for GBM. And these are recurrent patients, again. These are patients that failed standard of care treatment and are back with their cancer growing aggressively. That's an incredible number. We didn't see in our evaluation of, as I said, over 200 different compounds and projects, anything that looked that exciting to us. This is an orphan-designated disease. So again, that's excuse me, drug. So that's 7 years of marketing exclusivity.
So you can see we're lining up a program here that sounds a lot like what we found in Berubicin, something that could really change the game for these very, very sick patients. So we are just incredibly excited about this. It has taken a long, long time. We have always said that this was our mission and this was what we wanted to do. But we weren't just going to bolt on another project simply to have something else in our pipeline. We knew and believed very strongly that we have an incredible program in Berubicin. We wanted an equally incredible program to follow up, and I think that we've found that. So without any more from me, let me turn it over to Sandra and to Dr. Goldlust to talk specifically about this really incredible drug and program. I think you're on mute, Sandra.
As John said, we already have a program that is a potentially pivotal trial with Berubicin. But TPI 287 has been, as he said, also in a number of patients with glioblastoma, and we find ourselves with that compound going into phase 2 and discussions with regulatory authorities. Next slide, please. We know about glioblastoma because of its deadliness. It's the most aggressive and currently incurable brain cancer. The problem with it is that the blood-brain barrier limits the ability of cancer therapeutics to get access to them. There are tight junctions limiting the flow of materials into the brain, and there's efflux transporters, and those are lining the blood-brain barrier and transporting out chemicals that are not considered, you know, that are considered toxic to the brain, which are most chemotherapeutics. The next slide.
So TPI 287 fits into our armamentarium of drugs that are permeable through the blood-brain barrier. It is mainly based on the fact that it's not a substrate for the MDR, P-gp-like protein transporter. And it was designed so that it can get across the blood-brain barrier, but it has the same properties as a taxane. Next slide, please. So what you see here is it's compared to both paclitaxel and docetaxel, two of the most well-known members of the taxane family, to look at the permeability of the brain to these compounds. And as seen with wild-type PGP-positive animals, we see that paclitaxel and docetaxel have almost no penetration of the brain, whereas TPI 287 does. And when we knock out the PGP, we see that probably the greatest reason for lack of permeability is that transporter.
And when we look at the brain concentrations compared to the plasma concentrations, we see that there are sustained and very high levels in the brain of animals. Next slide. So what is this compound? It's, as we said, an abeotaxane. And the reason that it's an abeotaxane and not a taxane is because it has a five-membered ring rather than a six-membered ring at the core. It also has side chains that give it the properties of not binding to the P-gp receptor. The other thing to know about it is the mechanism of action is exactly that of the taxanes that bind to the microtubules in the spindle and prevent the mitotic mechanism from moving forward. Also, as John said, it's been studied in over 350 patients. So we know a lot about its activity as well as its safety.
In the phase 2 studies where we've looked at glioblastoma, there was considerable success. There was a 60% overall response rate. But even more important, as Dr. Goldlust will talk about, there was a 13.4-month median survival and a 64% one-year overall survival. These were for heavily pretreated patients with glioblastoma. Again, this is an orphan drug that's been in this indication and for which CNS Pharmaceuticals will be talking with regulators and our team of investigators that we've already established to develop a phase 3 program to look at the registration of this molecule. I'll turn it over to Dr. Goldlust.
Thank you, Sandra. So just as a little bit of background to build on what Sandra talked about, we've known as neuro-oncologists for decades that taxanes in a Petri dish are particularly effective at treating glioblastoma cells. It's the, in clinical practice, it's getting that mechanism of action, that type of drug or class of drug into the brain that's been the problem. And I think if we look at this data here, it really speaks to both support the idea that we know taxanes are effective in GBM, but also that it's getting into the brain and having a meaningful effect when it's formulated as TPI 287. So as the investigator, lead investigator in the phase 1 study, this chart shows us a compilation of all the patients that were treated.
I think the real takeaway, in addition to the fact that there were many durable survivors, keep in mind that the average survival of recurrence is around 6 months. There's no drugs that have been shown to prolong that 6-month interval. We can see the median overall survival here is 13.4 months. In addition, as indicated by the purple triangles on some of these patients, this 25, 24-patient group was heavily enriched with what we call MGMT unmethylated patients. Those are patients that historically don't respond to traditional chemotherapy with temozolomide, the first-line agent, but also historically don't live as long as patients that have the opposite marker or an MGMT methylated. So we have a 24-patient subset that had a long median overall survival, 64% alive at 1 year.
Again, that's at recurrence, coupled with the idea that these patients are some of the poorer prognostication patients from diagnosis. So really sort of puts this table, rather this chart into perspective. We can take a look at the next slide. Although the combination of TPI 287 and Avastin was in dose escalation and wasn't compared in a trial to other combinations, we've just superimposed historical data in terms of progression-free survival and overall survival, historical data versus the outcome of our phase 1 study. You can see, I think I'd really encourage you to hone in on the overall survival. If we look at that brown curve of the TPI 287 and Avastin or cefazolim, you can see it compares very favorably to other clinical trials of Avastin alone or Avastin plus X.
If you look at that Avastin alone arm, it hovers in the 6-9 range depending on the study. And again, the 13.4 months of OS or overall survival is quite impressive. Our plan, of course, is to verify this in a later stage study. Also worth noting that the toxicity of the drug was in line with what we expect of taxanes. Some peripheral neuropathy, some change in the white blood cell counts, both.
I think we lost Dr. Goldlust for now, but as he was saying, the safety is comparable to those of the other taxanes systemically. Again, what we saw was some myelosuppression with white counts lowering and some peripheral neuropathy, but there were no central cognitive changes in the patients and no obvious changes in their neurological function. So I think we have characterized the drug quite well at this time and are primed to go into a later stage design study and have discussions with regulatory authorities on what they think the design of those studies could be.
Great.
I'll send this back to John.
Yeah. So as you can see from that data, our excitement about this program, I think, is well-founded. It fits with our lead program. It's a very similar type of drug and situation in the sense that we have amazing data in terms of efficacy and safety from many, many patients in this disease and this drug. We know that it does things to this disease. We see signal altering the course of this drug. As Dr. Goldlust said, we don't see these kinds of overall survival numbers at anything like this from this disease, particularly in these unmethylated patients, which have such a poor prognosis. I want to point out one thing about this program that I'm sure is on investors' minds, and that is what sort of does this do to the company overall and our focus. So to begin with, this is our focus.
This fits squarely within our mission. It fits squarely within our focus. It's completely complementary to Berubicin. But most importantly, I think from the perspective of where we go from here and how we execute, you have to understand that in order to, as I mentioned earlier, in order to develop a drug like this, it's one thing to have the molecule. It's another thing to have a program and a network of clinicians and an ability to execute a potentially pivotal study like the one that is warranted for TPI 287 based on the existing data that we have and that we've just gone over. The advantage that we have as a company, and I think the thing that makes us the best folks in the world to investigate a drug like this and advance the standard of care is we have already built that network.
We have spent years developing our contacts, our relationships with key opinion-leading investigators in the United States and in Europe that allowed us to push the Berubicin program as fast as we did. I'll remind everyone that with Berubicin, we went from a standing start enrolling our first patient to complete enrollment of over 250 patients in a little more than 2.5 years, which is really remarkable given that a program of that size in this disease, a lot of folks told us it might take 5-6 years to enroll. I think that speaks to the quality of our investigators, the quality of the study that Sandra designed, and the quality of our drug, Berubicin. We can now repurpose that network. There is an equal amount of excitement in the neuro-oncology community based on Dr.
Goldlust's paper, which was recently published about the phase one study of TPI 287. You can find a link to that paper in our press release today. But basically, the community knows this is a very, very exciting cytotoxic chemotherapy possibility for this very desperate situation. We have that clinical network. We understand how to use it. We have built this program in conjunction with the Fast Track designation at FDA in terms of our Berubicin program. So we have a very, very good rapport with the FDA, and we have a very good understanding of what it is that they are looking for in a drug like this. All of that will help us to reduce both time and, importantly, cost to developing this program. Time really is money in the drug development business. The less time you can spend developing, the less money you will spend.
And so we want to make certain, I think, that investors understand that one of the things we're most excited about is really being able to, to borrow a tech phrase, to push more content through the platform and the engine that we have already built. We have spent years and invested millions of dollars in building this system. We can now push another very exciting, equally exciting program through it with our TPI 287 in license. So we really, as I said, we have spent years looking for a program like this, negotiating with our friends at Cortice Biosciences to pull this drug in, and our work on this drug begins today. So with that, I think we can open it up to questions, Janine.
Great. Okay. So to our participants, if you have a question for the team, click the Q&A button at the bottom of your screen, type in your question, and we will get to as many as time allows. So we already have people that know the drill here, so we do have some questions in queue. So the first question is, this looks like an excellent complementary asset. Can you talk about the differences in patient populations between the ongoing potentially pivotal program with Berubicin and the proposed phase 3 study with TPI 287 for GBM? Are there patient characteristics that would make one asset more ideal than the other? And can you give us a sense of the additional GBM patients that this asset targets beyond your focus with Berubicin?
Okay. That's a great question. Sandra, why don't we start with you on that?
Okay. Yeah. Our program with Berubicin took all patients with recurrent GBM, and they were both MGMT methylated and unmethylated. I think what we've seen with TPI 287 in the poor prognosis patient population, which is the unmethylated, that maybe we would focus on patients with recurrent disease that are unmethylated, for which current standard of care may not have as good of an outcome as it does with the methylated MGMT. The other thing is that we are probably going to move Berubicin because of its activity into an earlier stage disease. And so we may not be - we won't be overlapping as we go into primary recurrence after treatment with standard of care for TPI 287. As far as the combination, we've thought about that quite a bit. And anthracyclines and taxanes have been used for systemic disease throughout our treatment of major tumors.
The anthracyclines are actually limited by the fact that they're cardiotoxic. What we found with the Berubicin program is that there is no cardiotoxicity as we've been seeing it. The use of this compound for other diseases that are metastatic to the brain that would be sensitive to anthracyclines would be very interesting. The use of TPI 287 in combination with relatively non-overlapping toxicities would be something that we would be exploring in the future.
Okay. Our next question. You talked about Berubicin being a pipeline drug. Do you feel like TPI 287 has a similar kind of potential?
I'm going to again turn that over to Dr. Goldlust and Dr. Silberman, but my own personal opinion is absolutely. This is a very exciting drug with an incredible range of utility. As I mentioned, it's been tested in over 350 patients in a variety of diseases. It comes from a well-known, trusted family. You can hear a lot of similarities from what we've talked about with Berubicin, and that is because this is exactly the type of drug that we're looking for. So please, doctors.
I want to, I mean, I want to make sure I understand the question. Could you repeat it for me, please?
Okay. Apologies. So.
It's okay.
So you guys talked about Berubicin being a pipeline in a drug. Do you feel like TPI 287 has a similar kind of potential?
Well, I think in addition to what was mentioned with the last question, because TPI 287 is slightly earlier in development than Berubicin, we still have a dose expansion to do with TPI 287. Keep in mind that if you read the phase 1, we found an ideal dose, which was on the higher end of our escalation. We saw all of this benefit, including with some patients that may have received what we now know is a relatively subtherapeutic dose of the drug. My expectation and my hope is that if we can treat all patients with dose expansion at what we now know is the best dose, we may even get more activity out of the drug than we have seen to date.
I think we have a little bit more to learn in terms of how much mileage, so to speak, we can get out of it.
Yeah. So let me add to that in terms of pipeline. Will it treat other diseases? And as I have alluded to, we think that taxanes are used in quite a number. They're ubiquitously used for systemic disease. To have one that actually gets into the brain and has activity in the brain opens up a host of different opportunities for us, including those that are sensitive to taxanes as well as anthracyclines. And we know that breast cancer, for example, is something—is it a disease that will metastasize to the brain because it's a sanctuary and it doesn't get treated by systemic disease? Well, this no longer makes—this drug is now no longer allowing the brain to be a sanctuary. It's an area where the drug permeates. And as we've noted, it has success in a very difficult-to-treat disease, glioblastoma.
The safety is totally manageable because we've known taxanes for years, over 50 years of working with them. The same thing with anthracyclines. I do think that getting these two drugs with access to the brain speaks to a lot of the attempts that are being made to try to get both anthracyclines like doxorubicin and the taxanes like paclitaxel, liposomal, or albumin-associated drugs to try to get them into the brain. I think that these two drugs exemplify what we've been looking for for years.
Okay. Our next question, Dr. Goldlust, you were an investigator in the earlier trials. What is your personal take on 287? And what about the patients you tested?
So I've been an investigator on a lot of studies for recurrent GBM, including several immunotherapy studies. What I think we see, what I've seen in trials is that there are a small subset of patients that respond to the drug of interest, but without sort of an overall benefit for the group. But I've never seen or participated in a study that had such a profound improvement in OS at recurrence. So it really stands out among the 20 or so studies I've taken part of for recurrent disease.
Okay. Our next question, how did you find this asset? How did it compare to others you evaluated? What was your selection criteria?
Great question. So we found this drug. We've been aware of this drug for a number of years. Sandra and Dr. Don Picker, our Chief Scientific Officer, were actually involved in the development of this drug at a previous sponsor. So we've been aware of this drug for some time. We worked with Dr. Goldlust on our Berubicin program. So we were aware of the work that he was doing. And as exciting as it was, we knew as a company that we didn't want to suffer from any confirmation bias. So we set all the good things that we knew about this program aside and started canvassing the market and the scientific literature for other potential programs years ago. As I said, and I just sort of went back this morning to check this because it's a huge number.
We did look at over 200 different molecules and programs of various stages in this space. We had a number of consultants working with us for over a year kicking the tires in diligence on this particular molecule, on the clinical data, on the CMC work behind it, which is actually extraordinary. Just as an aside, a huge amount of time was spent by Cortice Biosciences developing a commercial pathway to manufacture this drug. So very advanced work there. But when we put all of those things together with the, you might say, the amazing kind of cherry on top that really lights it up, which is that OS number at recurrence, it became clear that the drug that we knew from our colleagues' work prior to the founding of CNS on this program really was the standout molecule in the space.
And if I think about it from my somewhat biased perspective, I'm not surprised. Sandra and Don are two of the best drug developers in this space anywhere in the world. It's not surprising that they worked on this very exciting molecule early on. But we completed, I would say, a full circle process. We started with something that we thought was interesting. We spent years evaluating it. We spent years evaluating other potential programs, all the while, of course, focusing on our lead program in Berubicin. But as we fully enrolled that study and saw its conclusion coming on the horizon, we felt that it was time, as I said, to take advantage of all the amazing work that we have done building this drug development engine and springboarding into another program.
It was clear based on our years of evaluation that this was the appropriate program for that.
Okay. Our next question. So how much additional cash do you need to get to results? Why do this now if you're confident in Berubicin since funding is limited?
Yeah, that's a great question. I mean, the basic answer to the second part of the question is the time is right. We have the bandwidth to do it. We have the clinical network to do it. The sooner we get started on this program, the better. We are incredibly confident in our Berubicin program that's going to deliver results next year. And we want to get started now. This drug was available, and we wanted to pull it in now and get to work on it because time is of the essence for these patients. We think that this is going to be an amazing value creator for the company going forward. And to the first part of the question, let me say this.
It's very important to understand that before you get to the point where you're dosing a first patient in a study of the kind that we've been executing with Berubicin and that we will execute for TPI 287, there are years of work and effort and planning that go into developing a program like that, getting that green light from the FDA to proceed, getting all of your investigators on board. All of that takes time, and all of that takes money. The important thing, I think, and we emphasize this in our press release today, is that because we can repurpose so much of the work that we did for Berubicin, we expect to be able to shortcut a lot of that time and a lot of that effort and resource spend on this program to get it started.
Now, that said, there's still 1 year to 18 months of work prior, let's say, from today to the point where we expect a dose of first patient. But that's probably half the time that it might have taken otherwise. Were we starting again from scratch as we did back in 2017 with this company and our Berubicin program? Overall, I would expect going forward that this program, in terms of overall spend over the years that will be occupied, will probably cost something similar to the Berubicin program. However, with the material savings upfront because of our ability, again, to repurpose that clinical program and all of the years spent building it and planning. All of those things really compress the timeframe significantly, which compresses the spend.
If we can save a year or a year and a half, we're probably looking at savings in the $10+-$15 million range on the overall ticket for this program. So we think that, again, we are the best people in the world to do this work because we have already done so much of this work. We have the machine ready. We're now going to push more content through it.
Okay. Our next question. Do you see this cannibalizing Berubicin commercially, but particularly with patient enrollment in clinical trials?
Sure. That's a good question. I'll also just say one thing and then let my colleagues answer it. But I think the answer on both fronts is no. First of all, we've already fully enrolled the Berubicin study, so there's no possibility that we'll be cannibalizing trial enrollment because one is already enrolled and the other hasn't started. In terms of treatment, I'm going to let Dr. Goldlust and Dr. Silberman talk about that.
I can comment on that, John. I think something to keep in mind as background is that most cancers, aside from glioblastoma, have a backbone of cytotoxic therapy that has been standard treatment for decades. GBM doesn't have that. We don't have anything that's biomarker agnostic to treat this disease aside from surgery and radiation. So there's a lot of progress to be made in GBM in terms of survival just to sort of get it up to the level of other cancers. So I think having two potentially active cytotoxic drugs, if you think of something like lung cancer, I mean, there's an order of magnitude more options, and they certainly don't interfere with the progress of efficacious agents.
I think the short answer is no, and the reason being that we have a lot of catching up to do on this disease, and there's certainly space for two efficacious molecules.
Okay. Next question. What is your timing for entering a pivotal trial with TPI 287? And do you have an idea on design and size, and will it be in combo with bevacizumab?
Great question. Mostly for my colleagues, I will say kind of the top-line answer is as soon as possible. We will be using all the work, as I've said many times now, that we've done for Berubicin in building this great clinical network and drug development engine to accelerate that process. Our hope is that we can be back in the clinic in 2025 with this drug. But Sandra, why don't you talk? This will be your program again.
Yeah. And so one of the great things is that Cortice, prior to us acquiring TPI 287, had already sat down with the FDA and designed what they thought would be the next study to do. And we know that there have been a myriad of compounds and cytotoxics that have been combined with Avastin and bevacizumab previously that have not shown an overall survival benefit. But they're looking at us having a program comparing bevacizumab plus TPI 287 to bevacizumab alone. And that's what we're looking at right now. And we will go back to the FDA and ensure that that's the program that they think is most reasonable. Sam, do you want to talk about the use of bevacizumab in combination with TPI?
Sure. I saw there was a question earlier about progression-free survival, and is that sort of a meaningful outcome in a study that used Avastin? And I think the short answer is probably not. And that's why we've really honed in on the much more meaningful endpoint of survival. But the world of neuro-oncology is always evolving in terms of what people are using clinically as their standard of care. So I think in going back to the FDA, we can sort of look at what experts are doing in design of clinical trials over the past five years since this concept was initiated and really make a thoughtful determination as to whether we're going to include bevacizumab in future studies, knowing that we're going to be focusing on survival, and we really want to make sure we can highlight the activity of the drug in that way.
I think we have a little bit of discovery to do over the coming months to really refine that study. Stay tuned on that.
Okay. Our next question. How much cash do you need to get to Berubicin results?
Yeah. So we have said that we will put results out in the first half of next year. So that's kind of a six-month window from starting roughly six months from now and going to roughly a year from now. We burn about $1 million a month on the trial, although that's descending now as patients are rolling off the study and enrollment is fixed. And we burn about $1 million a quarter on G&A. So the range there remaining to spend, not what we have to raise, is probably anywhere from roughly $8 million-$12 million. So somewhere in that range. Actually, maybe a little bit more on the high end, but that's kind of a rough estimate. It's inexact because we don't know exactly when our last patients will come off our CNS 201 study of Berubicin.
That will dictate, of course, when we take top-line data and announce it. So again, but we are completely on track for that in terms of our timing and our monitoring of the study. Reiterate again to everyone, first half of next year, data is expected, and our confidence in that remains very, very high, frankly, as it does in the performance of Berubicin based on everything that we've seen and talked about.
Okay. We have time for a couple more questions. You showed superior PFS with bevacizumab. How does that compare with beva or 287 alone?
I think we tackled that one with the last question.
Okay. Next. Can you talk about the goals for the upcoming FDA meeting and what a phase 3 program would look like for TPI 287?
Well, first of all, we've just acquired the drug, so we need to sit down with the FDA and plan the meeting. But as I said, Cortice had already discussed plans moving forward with them. And I think we will just use that as a template for us to use the Fast Track designation that it does have to be able to design the next study. And as I said, I think the most useful study would be a repeat or an expansion of the bevacizumab and TPI 287 at the recommended phase 2 dose, and then a randomized study against Avastin and bevacizumab alone. Using overall survival as the endpoint. Sorry. Very important.
Okay. What do you think would provide more investor confidence?
Yeah, that's a great question. So I think what would provide more investor confidence is an understanding that past is not necessarily prologue for this disease. GBM and pancreatic cancer, to my understanding, have developed a sort of mythic quality in oncology in that these diseases appear to be not just difficult to manage and incurable today, but because so many have failed, the bias, I think, is that everyone will fail. And I think if you listen carefully to what Doctors Goldlust and Silberman have said here, and you think about our approach to Berubicin, it's a very, very logical approach which extends to what we're doing in TPI 287 and why we chose this drug. And that is something that I have said over and over again. And I think if investors pay attention to what we've done and investors look at Dr.
Goldlust's paper on TPI 287 and understand just how extraordinary this OS data is for a drug like this, you will see that the approach that we have taken, I think, is what's going to carry the day and create that foundational one-size-fits-all cytotoxic chemotherapy foundation for this disease that is missing. While everyone has been fixated in recent years about these rifle shot programs to treat a handful of patients in precision medicine, and believe me, I'm a huge believer in precision medicine. I spent a decade in that space. But when you see the gap that exists here in the foundation of a treatment algorithm for these patients, you have to understand that the best way to approach this is to go back to the drawing board and say, "What type of molecule actually destroys these cells?
What do we know, in essence, colloquially speaking, works at the bench, and how can we translate it to patient care?" So take a look at TPI 287, and you know that this family that it comes from, the taxane family, can destroy these types of cells, these cancer cells in the Petri dish all day long. Again, very similar to anthracycline and how we've worked with Berubicin. So what makes this so special? Well, it's a very logical eight-step semisynthetic process that turns this taxane molecule originally derived from a natural source in the bark of the yew tree to a highly lipophilic, very specific molecule that bypasses both the active and passive mechanisms of the blood-brain barrier.
And the way I see that, the way our team sees that, is a very logical stacking of the deck in our favor because we're taking, once again, that hammer, this taxane family in the case of TPI 287, the anthracycline family in the case of Berubicin, and we're saying, "We know this hammer works. We know this hammer can hit the nail as long as we can bring it to the nail." In this case, that's exactly what we can do with TPI 287, the modification to that sort of existing starting point of a taxane to allow this drug to be highly lipophilic, bypass the P-glycoprotein MDR efflux pumps, and get into the brain. Now puts us in a situation where, as we have seen in the phase one study, this drug has efficacy. It's well tolerated.
To me, the only question is, can we demonstrate this conclusively at scale, properly powered and designed for the FDA to approve? That's what we're really good at. That's what we've done with Berubicin in a very short period of time. That's exactly what we're going to do here. I think for investors that understand the disease, that understand what these molecules can do, and understand that this disease is not something that will never be defeated. It has to be defeated in a stepwise fashion with an armamentarium of drugs that make sense, that were designed for this purpose, that can actually get on their own to the site of the tumor.
When you understand all of that, you realize, I think, as an investor, that yes, maybe you're taking a contrarian position to those who invest in this space in general because the common sense evaluation of the space would be, "Well, it's GBM. Therefore, everything is going to fail." That's an easy bet. But you get outsized returns by taking a contrarian position often. And I think that's really what investors have to understand here. We are 100% committed to this mission. We have demonstrated that commitment for years in our laser-focused execution of our Berubicin study. You should expect the exact same commitment to TPI 287 and to managing this disease in a way that others have been unsuccessful because they haven't taken this same approach.
I think that our approach is founded not just in optimism, not just in confirmation bias, but in very hard science that's now been published. This drug, if I can speak this way, this drug works. This disease doesn't spontaneously resolve. Now our mission, just as it is with Berubicin, is to prove that conclusively. I think we've demonstrated our expertise and our ability to do that. Hopefully, over time, investors will see that. My firm belief is that investors in this company, of which I am one, will be handsomely rewarded for their fortitude here going forward.
Just to build on John's excellent points there, I think if you take some kind of bird's eye view of the way the drug works and some of the surrounding data behind it, as John mentioned, we don't have a traditional chemotherapy backbone that works for all patients. We've run a lot of clinical trials of sort of similar targeted agents and immunotherapy agents as other cancers without having the benefit of that backbone at our disposal to help patients with GBM. But if you look at the phase one data in terms of survival, if you look at the drugs that we know have some benefit in this space, alkylated chemotherapies help a subset of patients. So we know cytotoxic drugs can be effective.
But if you look at what else is approved for this disease, I'm sure savvy investors kind of know the options that are out there. You look at a device called Optune, which is also FDA approved for the treatment of GBM. The mechanism of action proposed of Optune is to interrupt what's called metaphase array. That's where DNA is in one tumor cell is splitting from one tumor cell to two tumor cells, and that's how tumor cells grow. That's the almost identical mechanism of action of taxane drugs. Now, one is based on electrical activity with Optune, and one is based on chemical activity with taxane. But we know that that paradigm, interrupting metaphase array, has been a successful pursuit in GBM. So if we're just looking at what's out there and what's worked in the past, we don't have too many different options to draw from.
But this sort of borrows from both directions. It's a cytotoxic drug, which we know can be helpful, and also mechanistically, we've seen it work in other ways in this disease. So just to sort of couch it in terms of all of the things that tell us that this has promised.
Great. Okay. That does conclude the Q&A, John, or Sandra, Dr. Goldlust, any closing remarks?
I'll just say, first of all, thank you. This was a great program. Thank you, Dr. Goldlust, Dr. Silberman. I hope investors have a full picture of what we're up to now, why we've done this, how we found it, why we're so excited. But I can just tell you from my personal perspective, this has been a years-long pursuit. As I saw the company develop and our expertise be proven out, my hope and wish all along was that we could push more opportunities for patients through this engine that we have built. There's not a ton of companies that are as committed to this space as we are. No one else is really taking this approach. We think that this approach is going to carry the day for these patients. It's consistent with everything that we have said about our Berubicin program and our vision.
The armamentarium for these patients is sorely lacking. We really need that one-size-fits-all foundational cytotoxic chemotherapy treatment. And that's what we are developing in both programs now. They are totally complementary to one another. One, we believe very strongly is going to deliver game-changing results in the first half of next year. And just as we're doing that, we will be springboarding into the clinic with TPI 287 to give those patients hope again that we can continue to expand their overall survival and their quality of life with well-tolerated chemotherapy drugs because that's really the name of the game for these folks. And that's what we're all about as a company. So we could not be more excited. This is the embodiment of kind of everything that we have wanted to do since we founded the company. And now finally, here we are. So thanks, Janine.
We're just thrilled to get to work on this.
Wonderful. Well, this does conclude the CNS Pharmaceuticals Investor Corporate Update webcast. Everyone can disconnect their lines. Thank you.