Of Alpha Tau Medical, ticker DRTS. During the presentation, please feel welcome to submit your questions using the Zoom Q&A interface at the bottom of your screen. After the presentation, we will open to your questions. With that, Raphi, I will turn it over to you.
Great. Thank you, Alex, and thank you to everybody who's joined us. It's a pleasure to be here back at the Sidoti Conference. We always enjoy the chance to meet with a wide range of investors. I am happy to tell our story for those who are hearing it for the first time, and also really want to focus on a couple of important clinical updates since we were last at the conference. Thank you very much for the opportunity, and definitely feel free to submit any questions, and happy to get to those at the end. Just for those of you who don't know the story, or those of you who need a quick reminder, we are the only company who's figured out how to deliver alpha particles directly into a tumor.
I'll explain what this means, but it's a much more focused and potent form of radiation, one which has been known for years to be much more efficient and much more direct in destroying cells. No one's been able to use it for local delivery into the tumors. I'll explain why that is and how we've overcome that problem. In the end, we end up with a product which can deliver an incredibly tightly controlled, conformal, but very, very powerful dose of radiation into the tumor. We are focused on really testing and delivering this in a number of different tumor types. In the end, the treatment is physics-based. We've always been of the view that if you inject this treatment into the tumor, it should do its job.
We just need to really develop ways to do those injections and then test those across a number of tumors and generate the data needed for approvals. We are doing that now. We have done this in 20 or so different tumor types in animals, as well as already in a number of types in humans, and have yet to see a tumor type that does not respond. We are really building out and really building tremendous momentum as we work through different cancer types, generating data, including some data I am going to share with you now, data that will come out in the coming months and years, and looking for our first FDA approval as early as next year. Just to set the stage on the scientific side, traditionally, cancer patients are very commonly receiving local radiation therapy. More than half of cancer patients are getting it.
They're all doing it using the traditional beta or gamma-focused types of radiation, which are the kinds of radiation that penetrate tissues and bodies and walls, etc., and get stopped by metal or by concrete. Those are X-rays, gamma rays, etc. Now, those are useful because we're able to generate enough range to penetrate the body and cover the tumor. However, the shortcoming, of course, is that they are very inefficient. They require high doses. Because of that, we're going to put a large dose into a tumor. It's going to spread into the area around it and do meaningful damage. At the other end of the spectrum are the alpha particles that we're using. These are radioisotope emissions that are very heavy. They've got a lot of energy to them. They're very, very direct in the way they're destroying cancers. We need much lower doses.
The issue is that they are so heavy and unwieldy. They are very short in range. When we place an alpha emitter inside of a tumor, we find that we can get 40-90 microns of range, which is 40-90 millionths of a meter, which means we will kill three or four cells. The fundamental inability to get the alpha particles to move to any useful range in the tissue is the reason nobody does this. Even though they're more efficient and they're more potent, we can't harness them if we can't get them to move into the tumor. The way in which we overcome this is by using the natural decay chain of radium-224, which will break down naturally six times before it stabilizes.
It will release a bunch of alpha particles, each of which will smack into something and kill it and stop very quickly. Excuse me, what we've managed to do is we've taken a small piece of metal, a source that we've coated with radium-224, where the radium is right near the surface. The way we've done this traps the radium but allows its daughter atoms, the other steps in the chain, to escape and move into the tissue. We position the radium at the surface so that it breaks down on the source. Once its daughter atoms are released, they recoil off of the source into the tumor, kind of the way a gun recoils when you fire it and it flies in the opposite direction.
These isotopes will move deeper and deeper into the tissue and release these alpha particles deeper into the tissue. Hopefully, my video comes through. You can see here an example. We'll place one or more of these sources into the tissue, into the tumor. We'll leave it there. As it breaks down, while the radium is trapped, its daughter atoms move into the tumor, and they continue to diffuse over their total of 12 hours of half-life and release these particles deeper and deeper into the tissue. Excuse me. It's not that we're actually shooting the particles any harder. We just have them released deeper into the tumor. Now, instead of that 40 microns of range that we get from the alpha particles themselves, we get about 4-5ml of range, which is really being driven by this repeat diffusion of isotopes.
That is quite a bit more useful. We know what to do with 4-5ml of range here. We can use that to put a couple of these sources into the tumor and then have them drive that coverage that we need and ensure that we have really covered the tumor. That is what we are focused on doing, on delivering these sources into the tumor. Really, for us, the question is, where do we want to focus our time, given that we see the relevance here to a very broad range of tumors, ultimately to any solid tumor? We need to choose where to focus our efforts.
Historically, where we've spent most of our time, and those of you who joined us in the past have seen the data that we've generated, the really fantastic data, mostly where we've spent our time has been in localized and unresectable tumors. These are tumors that have other available options, like skin cancer, head and neck cancers, prostate cancers. The patients who try those therapies and fail them find that they've run out of options very quickly. We have found we can be a very compelling later-line solution. We've done this now, having started in skin and head and neck tumors, what we call superficial tumors. We started there because it was easy to access that area and deliver these sources, control where they're going, monitor for side effects. Once we had good preclinical data, we got started.
We treated hundreds of tumors to date. First of all, I'd say the safety data has been fantastic. We don't see the traditional serious systemic side effects that one sees, the nausea, the fatigue, the vomiting, etc. We don't see that because, again, the radiation is incredibly tightly controlled. The historical shortcoming of alpha particles, their short range, has now become their strength because, again, they're just not leaving a tumor and doing any damage. We've also seen fantastic efficacy data. I'll show you some examples of that. We've already been approved in Israel for treating SCC of the skin and oral cavity. We've submitted a request for approval from the PMDA in Japan, and we're hoping to hear back in the next quarter or two. Really looking forward to getting that approval. Really, our focus is on the US, where we expect to do our first launch.
We are currently in a pivotal study in the US in this recurrent skin cancer. This is going to be our first trial that will get us an approval, hopefully, in the FDA. We're running that right now. I'll talk about that in a second. Just to give you a taste for what these data looks like, as an example, this is data that was generated in the US in a small pilot skin cancer study that was led by Memorial Sloan Kettering Cancer Center in New York. Just to give you an example, this patient here has a recurring tumor. He already had surgery. The tumor has grown back on the nose. The surgical option is quite deformative now, really, to cut out a chunk of his nose. Instead, we just inject a couple of these sources.
You can see here, three months later, the tumor is gone. The nose looks great. This gentleman avoided a very drastic treatment with a very simple one without really any serious side effects. Again, we treated now across this study 10 patients. We saw no related serious adverse events from the product. Again, the mild side effect profile that we talked about earlier. Again, a 100% complete response rate, meaning every single tumor we treated disappeared completely. Really unbelievable data. It's the best we could have possibly hoped for. Again, very consistent with some of the other trials that we've done. Now we are running that study in the US to get our first approval.
This is being done under the breakthrough designation from the FDA, which is where the FDA says, "We recognize you have something that may help patients who don't have good options available." They have given us a tremendous amount of feedback and guidance as we built this study. We will also get fast-track review when we submit. We are running that study now across the US, treating 86 patients. We expect to finish recruitment in Q3 of this year and then look to get that data with the six-month durability early next year and submit it for approval again with the six-month review, looking to be approved as early as the end of next year. Just to give you a sense of the market opportunity here, which we think is tremendous, there are 1.8 million new CSCC cases a year in the US.
Obviously, most of those skin cancers will go for a simple resection in a dermatologist's office. We're not going to compete with the price of a scalpel. We see this as a premium product at tens and tens of thousands of dollars per treatment. We are going to focus on the most difficult patients, specifically the high-risk cases, and really focus on the patients who have already had insufficient response from other treatments. The literature seems to indicate that about 3.5% of patients will be in that target focus area for us, where they have local recurrences or spread into the lymph nodes around the area of treatment. Those are the patients we will go after, about 64,000 patients a year. We estimate new cases a year that will get to that stage just in the United States alone.
We're going to go out there and explain to those doctors, "Look, you can cut off this gentleman's nose or finger or whatever it might be, or put them on a systemic therapy, which is really a drastic outcome. We have a much better focused solution that doesn't have a lot of the side effects of the other treatments and has fantastic outcomes." This is the market we're going to go after. That's where we've historically shown a lot of our data. Those of you who participated before in the stability conferences have seen it. What we really want to focus on now is some of the newer areas that we're really exploring, the two other pillars of our strategy. One of them is the metastatic patients. Of course, there are plenty of ways to use a local therapy on a metastatic patient.
You could give it, even if they're receiving chemotherapy or immunotherapy, you may still want to add in our treatment to help destroy the bigger tumors. Think about it like a heavily soiled shirt that you're putting into the laundry, right? You may realize the laundry is not smart enough, as it were, to find those really big stains and break them up. You can say, "I, as an intelligent human being, see those large stains, and I'm going to pre-spot them directly onto that stain so that when I put it in the laundry, I'll be able to do its job." You could do that with a metastatic patient as well. We've done plenty of those. I'm referring to something else here. I'm referring to the fact that when we treat a tumor, we find that we're generating an immune response.
The immune system seems to be picking up on these tumors and helping fight them elsewhere in the body. We've seen that in all sorts of ways. In preclinical work, we will treat mice, and they will become immune to that cancer but susceptible to other cancers. We've shown we can activate immunotherapy drugs that mice who are not responding to checkpoint inhibitors, for example, will respond in the presence of our treatment. Most importantly, we've seen patients like this woman here, one of multiple, where this woman came in with multiple SCC tumors on the legs. We treated the first tumor. You can see it's a 3 cm tumor. A month later, the tumor has disappeared. This is a suture from a biopsy looking for cancer and not finding it. This woman had a complete response on the treated lesion.
We then said, "Let's treat the other lesions." We found that when we went to measure them, they had already disappeared. We never touched them, and this lady was on no other treatments. We've seen a couple of cases like this where we will treat a tumor, and the body will somehow pick up on it and treat the tumors elsewhere in the body. The question we're asking ourselves is, as we seem to be generating this immune response from the treatment, can we harness that somehow to help metastatic patients, not just by destroying the tumor that we're treating, but by really helping the body fight these tumors elsewhere? One of the things that we've just shared a couple of weeks ago at our R&D Day is some incredible data from our first combination study.
This is a unique study for us. We are modeling it off of the Keynote 48 study, which is the trial that Merck used to get approval for Keytruda, its blockbuster $30 billion drug, for approval in head and neck patients. We've modeled the trial off of this, looking at a very similar patient population, the same treatments with the pembrolizumab, the Keytruda they were getting in the trial itself, just adding in one Alpha DaRT treatment along the way. Now, what's unique here for us is that, number one, it's our first combination study. Number two, it's our first time where we treat a tumor and do not care about that tumor. We care about the other tumors in the body. Are we helping the body utilize and harness the Keytruda drug to do its job finding tumors elsewhere in the body?
We're comparing it to the trial that got them the approval, the KEYNOTE-48, which showed that for these patients in this specific group, the probability of any response was 19%. The probability of a cure, basically of a complete response of all the tumors, was a miserable 5%. Those were the chances of success. That was sufficient to get them approval for these patients, even though 81% of patients are not seeing a response because, unfortunately, there's really nothing to offer these patients. For that, that was enough to get them approved. We want to know if we can do better using the Alpha DaRT to kickstart the response to this drug. We released data in January. We had only treated eight. We'd only recruited eight patients so far.
Two of them unfortunately died even before they got Alpha DaRT, but that's how these trials work. These are very desperate patients. Those count against us. Even when you include those, if you look at the other six patients, three had a complete response where their tumors disappeared across the body, and three had a partial response where the tumors shrank with the Keytruda thanks to our help. Again, even with those two patients we never got to touch, in the denominator, we're looking at a whopping 37.5% who had a complete resolution of their tumors versus the 5% you would have expected to see on Keytruda alone, and again, a staggering 75% who had an objective response versus the 19% that normally would have been seen through Keytruda.
Again, it seems to be that the treatment is somehow helping the body fight these tumors and really driving the responses to Keytruda that we hope to see. The ability to really kickstart the body's response to the Keytruda seems to be driving this data here. Just to give you an example of one of these case studies and why we get so excited about it, here's an example of a 96-year-old woman who has tumors in the mouth. You can see here, really in the jaw as well as the lip. It's a really, really brutal tumor to try and resect in anybody, especially in a 96-year-old woman. She also had skin metastases in the neck, as we'll see in a second. We use those skin metastases as a platform. We inject our treatment, take it out a few weeks later.
You can see here those tumors have all disappeared. That's not surprising. We know the Alpha DaRT seems to do its job and destroy tumors. What's really cool, though, is the way this served as a platform because we never touched the tumor in the mouth. You can see this hideous tumor has now disappeared completely. You can see it on the PET scans as well. There's quite a bit of activity on the PET scan in the mouth, which has now completely resolved itself. In the end, this patient took the Keytruda for a year and stopped and is still alive last time we checked over two years after diagnosis.
Again, a 96-year-old woman who would have had really no real options available to her for this metastatic cancer in some of these really difficult areas to treat, like in the jaw, and somehow using the Alpha DaRT as a platform to awaken the immune system to the presence of these tumors has then driven the response of the Keytruda, where she's now cancer-free, which is, again, this is exactly what we want to see. This is one of multiple case studies like this, but a really, really fantastic outcome for these patients. Finally, again, we've made a tremendous amount of focus and progress on tumors of high unmet need.
The thought process here is, if we have a treatment that really is indifferent to the nature of the tumor, we should go after those tumors that do not have good options, like pancreatic cancer, like glioblastoma, multiforme of the brain. There are so many cancers that are desperate needs for these patients who do not have good options available for them. We want to see if we can be helpful there. We are running a number of important trials in internal organs, in the pancreas, in the liver, in the lung, in the prostate, very soon in the brain. I want to just spend a few minutes talking about some of the data that we showed a few weeks ago. We talked about some of these cancers in the liver and in the lung and in the rectum.
I want to focus on our pancreas studies, where we read some initial data from the three trials that were running in Canada and in Israel, taking patients with pancreatic cancer who do not have great options available to them. We had treated at that time 41 patients. It is now much more than that. At the time when we read this out, 41 patients had been treated. 33 had already reached the response time frame, and we saw great responses. Even with partial coverage of the tumors, we saw nearly 20% response rates where tumors were really already regressing, and nearly 100% where the disease was kept in check, where the tumors were not growing and the patients were keeping stable, which is a fantastic outcome for a very aggressive tumor, which tends to grow very quickly.
The question we asked ourselves was, and what we learned that really clinicians and patients and investors want to understand is, in the setting of pancreatic cancer, ultimately, people understand it's unfortunately a death sentence. The question is, can you extend their survival? No one cares about the specific tumor that we treated and the ability to keep it in check. The question is, are these patients living longer? They're going to get chemotherapy. If we add on Alpha DaRT, can we show a benefit in terms of helping them live longer? We did look at, even though these studies weren't designed to look at survival, it's a pretty easy question to ask, is the patient alive or dead? We were able to collect that data. We saw great survival data.
We saw patients were alive 11 months median survival since they were treated, 19 months since they started that current round of chemotherapy. The problem is that because these were heterogeneous studies, really the first time we're treating patients with the pancreas, we took whoever was available and interested in the treatment. It is a very heterogeneous population, and it's hard to know what to compare this to. How long should they have lived if we didn't give them the Alpha DaRT? What we did was we actually looked at different patient populations and found that even when we focus on those different groups, we can see benefit versus how long they should have been expected to survive.
One such group was, excuse me, patients who never received chemo, patients who ostensibly were too sick to get chemo or were refusing to get chemo because of how brutal it is. The data show that if you do not treat pancreatic cancer, the patient will die in three to three and a half months. We showed that by giving them a treatment they could and were willing to receive, they survived seven and a half months median survival. Again, already more than double what would have been expected, offering them a treatment where one was not really relevant and showing a meaningful survival benefit. The next group we looked at were patients who were metastatic, had already received first-line FOLFIRINOX chemotherapy, which is the gold standard, and had failed and come to us.
Now, the gold standard FOLFIRINOX, the expected survival is about 10-11 months according to literature. Since the initiation of the chemo, we found that actually we did not know what the median survival was because 15 months in, not enough patients had died. They were just refusing to die. We do not know what the median is yet. We know it is more than 15 months, so we know that they have already done much better than they would have done on chemo. We will have to wait and see if they survive 15 or 20 or 30 or however long it is. Until enough patients die, we can actually get a view for what the median survival is. Obviously, a fantastic outcome for these patients. Finally, we had patients who came in who had already failed second-line gemcitabine and Abraxane chemotherapy. Now, again, they have already failed second-line therapy.
They're expected to have survived seven and a half to 10 months on that chemotherapy. We saw a whopping 23 months of survival since the initiation of that chemo. Again, adding in the Alpha DaRT had a tremendous benefit. Now, obviously, there are a massive number of patients with desperate unmet need here. We are focused on the 87% of patients who are nonsurgical when they get diagnosed because those who can get a resection should do that. That at least has some chance of helping them. Again, 87% of patients are nonsurgical of diagnosis. Now, out of the 67,000 cases a year that are diagnosed just in the US, that means about 59,000 will be nonsurgical. Those are the ones that we're focused on where we think we can have the most value to add there.
One of the things we did announce as well, very exciting for us, is that we have FDA approval to start a study. Again, tremendous focus on increasing the momentum in the pancreas in particular. You think about that group that had failed first-line FOLFIRINOX and not enough patients have died. We do not even know how long they are going to survive, but obviously well more than they should have on the FOLFIRINOX alone. We are going to run a trial focused on that group, taking newly diagnosed locally advanced pancreatic cancer or metastatic pancreatic cancer, giving them FOLFIRINOX, but adding the Alpha DaRT on top of it and asking ourselves, when we get these patients earlier on, before they have gotten too sick, when they are newly diagnosed, can we have a real survival benefit for these patients?
Very much looking forward to getting that trial underway in the US Again, where historically those of you who've been on the Sidoti conferences before have seen us really focused on the first pillar of our strategy, the localized and unresectable patients, we're now starting to see great momentum and real data, I think very exciting data on our ability to generate that response to the checkpoint inhibitors and help the metastatic patients, as well as the ability to drive real meaningful difference for the patients with a high unmet need. Now, of course, looking forward as a company trading in a biotech company, obviously looking very much focused on milestones, clinical and regulatory milestones.
There are a number coming up now, given how many shots we have on goal, including first patient coming in in the US next quarter for the pancreatic cancer, looking to get an FDA approval as well for a brain cancer study and start treating patients in Israel on brain cancer. Afterwards, in Q3, finish the recruitment of the patients in the US for the pivotal trial for approval in skin, get that data to submit early next year. As well, just another important milestone, looking for that approval from the PMDA in Japan, hopefully in Q3 of this year. We've been public for three years. We had $63 million in cash and deposits at our annual report that we just published last week. We are still burning about $5 million a quarter on a run rate basis.
There's some one-off CapEx of a few million dollars that we're spending on a facility that we're building, a commercial scale manufacturing facility in New Hampshire. Other than that, again, I think we run incredibly leanly for a company with 130-ish employees and 15 or so clinical programs to spend $5 million a quarter is very lean. We are really here just telling our story. We think the momentum that's building is fantastic. The data is amazing. We've got some great plans coming up for the coming quarters. Obviously, looking to get new investors to join the ride. You can find our share at DRTS on NASDAQ. Again, we're going to continue to execute and hopefully generate additional results. With that, I'm going to pause. I thank you very much for the time, and we'd love to take any questions.
Great. Thank you very much for the presentation, Raphi. Maybe we could circle back on the R&D update day. I think you touched on one of the biggest things that you shared, which is this amazing systemic response rate. I think you touched on this as well. You said chemotherapy can be quite brutal. Some people do not want it. When you have your systemic effects, are there side effects that are unpalatable, or is it kind of a much smoother treatment process for patients?
Yeah, we have not seen really concerning serious adverse events coming out of the product in general. Specifically, when we look at combinations, we have not seen that be an issue. I talked about those first eight patients we treated in combination with Keytruda. There were no serious adverse events there either from the treatment.
Again, we haven't seen that be an issue in the combinations either. I'd say in general, when we think about combinations with chemotherapy, with immunotherapy, the biggest issue we have, of course, is that those other therapies have brutal side effects. If you're on chemotherapy and Alpha DaRT, you'll probably have the same side effects at least as chemotherapy, and that's brutal. Obviously, to the extent we can generate that immune response more systematically and push patients towards immunotherapy over chemotherapy, I think that has a lot of benefit there as well. No, we don't see our product driving really meaningful serious adverse events. That's not a concern, nor have we seen anything unique coming from the combination of the two.
I think it's really the background side effects from the other therapies or from the cancer itself that are really going to be the challenge going forward.
Makes sense. Rafi, you spoke about some of the CapEx investment in facilities, and I know you've made some investments in your supply chain to be able to distribute the DaRTs. We have a question from the audience about the overall patent protection, maybe both for the DaRTs and for the supply chain.
Yeah, absolutely. We've got a wealth of patents. We spent quite a bit of focus there. We've got over 150 patents that are granted around the world. That includes the product itself, the little piece of metal with an alpha-emitting isotope on it.
That includes the way in which we manufacture it, the delivery devices we've come up with for injecting this into different parts of the body safely, the combinations that we do, the way in which we plan the treatments, the way in which we really specify the right way to use Alpha DaRT in different cancers. We have patents on Alpha DaRT for prostate cancer, Alpha DaRT for glioblastoma, et cetera. These are granted already, as well as over 200 applications that are pending. We have a fantastic patent franchise. We also have a significant amount of know-how, and really that supply chain that you mentioned becomes very important. We have to get this messy thorium-228 that we buy from the US Department of Energy and elsewhere, which is an unwanted byproduct from other nuclear reactions.
We have to purify it and really get the thorium into a form where we can milk out the radium and trap it onto those sources. Again, there is a tremendous amount of know-how there as well.
Great. Thank you. Another question from the audience. Could you talk about distribution plans? I know we have spoken about Israel, Japan, the US. Could you talk a little bit about distribution plans once approval happens?
Of course. We are focused on launching first in the US. That is where we see our most attractive pricing and most attractive commercial adoption. We are exploring a couple of other countries around the world, as I mentioned.
We do already have distribution partnerships signed in Canada and in Israel, and we are currently in discussions on some other ones in Asia as well, just given that those are countries where we would be less likely to launch our own Salesforce. We are hoping to do our own. We are planning on and really planning our own launch for the US commercial opportunity. Keep in mind that we already expect to have our first FDA approval as early as late next year. We have to be getting ready for that, and we are doing that. We could launch ourselves in the US once we get FDA approval and are focused on doing that. We may choose to launch a little bit later if we see, for example, that pancreas is knocking you out of the park and it's a few months behind.
We may choose to launch in pancreas, for example, over the launch in skin, which will be our first approval. In general, our plan is to build our own Salesforce. We know exactly which doctors we need to go after and expect that we'll be able to do that very successfully.
That's great context. Thank you. Maybe the last question to end it, if we could zoom out a little bit and you could sum up the value proposition for investors, they might be looking across radiotherapy, novel cancer treatment, but yeah, we'd love to hear the DaRT case.
Sure. I appreciate that. I'd say, look, there are a lot of people working on tackling cancer. It's a very important disease, there's no doubt. No one is doing local alpha radiation like this.
No one's getting into the tumors, and that has a real role to play here. Systemic therapies are great. They're generally very niche-focused, but that doesn't mean they don't have important roles. For those cancers, they have a very important role. We can make a really transformational difference in these difficult cancers in our ability to deliver an incredibly focused and incredibly potent dose of radiation and spare the tissue around it. I think as we continue to generate our data from expansion into multiple tumors, going from the skin to the pancreas, now to the brain and the liver and the lungs and the prostate and the rectum, et cetera, we see this having tremendously broad market opportunities, really focused as well on building our manufacturing capacity to keep up with the opportunity, which continues to outstrip our expectations.
We expect to make a real transformation in the way people treat cancer and to be an important part of cancer treatment a couple of years from now. I think there is a lot of good here for patients, but I also think that given how unique the data are and how difficult these cancers are, we should see a very, very meaningful price for this product and really have tremendous sales opportunity with a very captive audience who do not have a lot to use unfortunately. I think this would be a great opportunity both in terms of value we are driving and really improving patients' lives, as well as an unbelievable business going forward.
That is great. Thank you, Raphi. With that, we are at time. I would like to thank you for the presentation and Q&A, and also thank everybody listening for spending time with us.
Yeah, it was great to be here and thank you, everybody, for the time.