Excellent. Thank you, Alex. Good morning, everybody, and thanks very much for your time. It's a pleasure to be here, to catch up again. It's been a couple of months since we last met. Actually quite a bit has happened since we last met in December. I wanna talk about that. I'm gonna jump straight into a couple of updates, and focus on those for the course of this presentation. As you'll note, I think, first of all, there's been a lot of real and meaningful activity in recent months, and I hope you'll see that as we talk about what's been going on.
I also think it's an incredibly exciting time to be at Alpha Tau and to be looking at the story just given what has set up to be a really, really transformative year for us in 2026. I'm just gonna start with a quick reminder. I think most of you are already familiar with the story, just to remember who we are. Alpha Tau has developed the Alpha DaRT, which is a local intratumoral injection directly into tumors. It is the only way that we know how to get alpha particle radiation directly into tumors. That's a much more efficient and much more potent form of radiation, such that we're able to use a fraction of the dose that other radiation therapies are using. Every other radiotherapy in cancer you'll look at is dosed in millicuries.
What we're doing is dosed in microcuries, obviously orders of magnitude less. So much so that we can ship it in a cardboard box on DHL or FedEx. So much so that we can treat in a regular room without the need for a nuclear bunker in the hospital. We don't need the lead vest and the lead in the walls and the lead in the floors. It is incredibly potent, incredibly focused dose of radiation that we inject directly into the tumor, and it stays where we put it. We have seen it to be broadly applicable across a number of different cancer types. Having done this in animals in about 20 different tumor types and never seeing a tumor type show resistance, we're now trying it in multiple different human cancers and have seen fantastic data so far.
I'm gonna leave it there just to remind you guys, as you know, we're gonna inject this treatment directly into the tumor. The Alpha DaRT injection is coated with radium-224. The radium will decay multiple times. As it decays, it recoils into the tumor and releases alpha particles deeper and deeper into the tumor. Rather than the 40-micron range we normally see from alpha particles, which makes them useless, now we see closer to 4-5 millimeters, which gives us a nice and very conformal dose of potent radiation. Again, I think most of you have seen this before, and so I'll leave it there. I wanna focus on what's happened even in just three short months since we met at the Sidoti conference in December 2025.
I think when we had met last time, we had just started treating glioblastoma patients. Obviously, a very important cancer for us, a deadly cancer, one in which a brain cancer in which we have not only Breakthrough Device Designation but also are accepted into the FDA's TAP program. That's the Total Product Lifecycle Advisory Program. It's effectively an incubator that the FDA maintains internally for some very select group of therapies that they want to help accelerate them commercially. They give us access to payers and to commercial considerations even during the trials that we're doing in the clinical pathway. GBM is a very big focus area for us, and I'm gonna talk about how that's going in a second, but that was just starting when we had last spoken.
Since then, there have been a couple of important announcements, and I want to hit on a few of those. First of all, as we had initially discussed would be happening, we had data released at the ASCO GI conference in January. ASCO, of course, being the premier cancer conference, the conference that all of the biggest cancer players want to be at presenting their data. Our first study in pancreatic cancer, which was led by a number of clinicians in Montreal, completed the study and then reported data which they presented at ASCO's Gastrointestinal Conference earlier this year. They actually received two separate presentations, which is quite notable. The first of which was focused on the final data coming out of those patients.
Again, very similar data to what we had shown last year in our interim, where we saw fantastic safety as well as disease control, some good response rates as well. Again, similar data to what we saw before, but with a higher n, with a larger patient population now that study has finished recruiting. The other point that was noted there was they actually got a separate presentation just to discuss some of the immune effects they had been studying. In particular, radiation therapy is known to be incredibly immunosuppressive. It effectively destroys the immune system because of the very broad approach, the long range of the gamma radiation and the X-rays, et cetera.
It was very unique when they were able to test patients, the immune systems before versus after treatment of the pancreas with the Alpha DaRT, and they saw no meaningful difference whatsoever. Actually, that immune protection was quite meaningful, quite differentiated versus what is usually seen with radiation therapies. Again, that also got them a second presentation at the ASCO GI conference. Again, we're very excited to be presenting at some of these premier cancer conferences as more and more data comes out. The second thing we announced since we last met was that we've actually started a submission of our application for approval to the FDA. The FDA has given us what's called a rolling submission.
In this case, it's a Modular PMA, which is a unique approach where they allow us to start submitting the package of the information for our approval in recurrent cutaneous SCC, the skin cancer pivotal study that we're running right now. We've already started submitting the application even before the trial is finished. The idea being that normally one would take a 10,000-page application and dump it on the FDA's desk and say, "See you in six or nine months." In this case, what they're trying to do to accelerate us is that they're letting us submit it in portions. Even before the clinical trial has finished, we've already submitted the preclinical, nonclinical work there, and are getting feedback from the FDA. We're working through that. We'll be submitting the module related to the quality and manufacturing, et cetera.
Also get some feedback on that. Hopefully, the goal being that by the time we finish the clinical study, which we'll talk about in just a bit and submit that data, the clinical package, the FDA will have already seen and reviewed two of the three parts of the application. Again, setting us up for a swifter application process in light of having done some of those points in advance. Finally, one of the most important things, I think, a very meaningful milestone and one that we had communicated would be coming at the beginning of this year, was that we did receive approval in Japan for the Alpha DaRT in the locally advanced unresectable head and neck cancer as well as the locally recurrent head and neck cancer as well. A couple of important things.
First of all, again, this is our first approval outside of Israel. Japan is a very difficult regulatory regime to get approved, a very challenging one, and so we're very excited, and we feel very validated to be able to show that we got that done. I would note that the indication was actually broader than we expected. Remember, the study that we ran in Japan was focused on the most difficult recurrent patients, and so we expected to get a label that matched that study. The pivotal study really focused on recurrent patients. Actually, if you look at the indication that we got, they gave us a broader indication than we expected. Not only are we able to treat and to commercialize this for locally recurrent head and neck cancer, we can also treat first-line locally advanced unresectable head and neck cancer as well.
Very excited to see that vote of confidence there. Very appreciative of the medical societies in Japan. There were six different medical societies who went to bat for us, who spoke strongly about the need for this product in the country. Japan is obviously one of the most important markets for us. It is a large, unified healthcare market with a penchant for advanced technologies, especially in the radiation therapy setting, for cancer. Very excited to have that under our belts. For now, we're focused on satisfying our post-marketing surveillance requirements. The PMDA, the Japanese FDA, which gave us the approval, as is fairly standard there, they condition the approval on what we call PMS, post-marketing surveillance, or often called Phase IV, right?
That once the treatment is approved, they wanna see you treat patients in the real world and demonstrate that, in fact, the data that you're seeing in the commercial setting looks similar to what you are seeing, what you saw in the trial, that there's no major difference. We are gonna focus on satisfying those requirements. We need to treat 66 patients in five sites, and that will be our focus now is to get that done together with our partner, HekaBio, in Japan. We also are gonna look to discuss with them additional studies. Now that we have this trial done, we're gonna look at other indications that we wanna run. I'd say one that is very much in focus has been pancreatic cancer. Obviously, we've seen some great momentum and some great data in pancreatic cancer elsewhere in the world.
They have a particularly large pancreatic cancer problem in Japan. They have nearly as many cases a year as we have in the US, and yet they have maybe a third of the population, so there's a very big incidence of pancreatic cancer in Japan. We've seen a lot of interest from KOLs in the country, very keen to see us bring this trial into Japan as well. We were waiting first to finish the first application for approval in head and neck cancer. Now that that's done, we're going to discuss with the Japanese regulator, with the PMDA, whether we can run a study now in the pancreas as well. Finally, of course, now that we have approval, we can also begin to discuss with the Ministry of Health, Labor, and Welfare the potential reimbursement for the product.
Again, for now, we're not focused on a large commercial launch, but nonetheless, until now, we weren't able to discuss with the Ministry of Health whether they were gonna reimburse the product and at what level. You have to wait for the approval to do that. Now that the approval is at hand, we will also discuss with them potential reimbursement, and we may even get some reimbursement during the course of this PMS discussion of the post-marketing surveillance trials. In any event, our focus is really on completing those requirements and launching additional studies rather than doing a large launch. Again, just a couple of highlights. You can see here how in three months since we last met at the Sidoti Conference, tremendous activity at the company, very, very busy.
I think more importantly, I would say, as we stand towards the beginning of 2026, I think this is a really momentous year for us, and it's important for me that people understand this will be a transformative year for Alpha Tau. If I think about the upcoming milestones, we did have one milestone we were looking at in the Japanese approval, which of course I've removed from the slide now that we just got that approval. We were able to meet that target as expected. We're now focused on a couple of US studies, and what I would say is that these are probably our most important studies that are all coming to a head over the next few months.
First of all, recall in the U.S., we're running our pivotal study in skin cancer, the recurrent cutaneous SCC, where we're. This is our last trial for approval, hopefully. As I mentioned, we've already started submitting that package to the FDA even while this trial is running. We expect to finish treating patients sometime in the next few weeks. Around the end of this quarter, or, you know, early April, we'll finish treating these patients. You know, then we'll be in that lovely twilight that biotech companies love to be in, where you're sitting around waiting for the data. Investors are getting excited and coming in ahead of the data and things like that.
Again, looking forward to having that trial done, and waiting for the data to come out, expecting the data in the second half of this year. Again, looking to get a lot of the package in before that, and then as that data comes in, remember, we're looking at response rate as well as six-month durability. That means we would probably need 7-8 months after the last patient is treated to get that final data, then submit that to the FDA. Since we have breakthrough designation here, which gives us fast-track review, as well as the fact that the FDA will already have seen some of the modules in advance, we are hoping for a relatively swift review by the FDA. The second one is our pancreatic cancer pilot study, our phase two-like study.
Remember, this is a trial that we launched in second half of last year just after Labor Day. The trial has been going very, very nicely in terms of recruitment. Sadly, there is not much to offer these patients newly diagnosed with pancreatic cancer, and so there really has not been difficulty in recruiting patients for this study, and so we expect to finish treating patients probably in the next quarter. Again, that's a two or three-quarter trial. It's very rapid for us, and looking for data in the second half of the year. Finally, the GBM study, as I mentioned earlier, just started, excuse me, in December. Glioblastoma being a deadly disease, we're very excited by how that study has been going. The first patient that came in, everybody was terrified.
For the first time, we're doing this in humans, and we're just ecstatic at how well it went. The first patient got up and went home the next day. We got 95% coverage in that tumor. Since then, we treated more, but the idea is we will treat three patients, one per month, and then stop and look at the safety. Those of you who've been following us for a while will know this is very reminiscent of what we had done the first time we treated in the pancreas as well, right? We treated patients in the pancreas with a very similar cautious approach, concerned that we shouldn't do damage to the patients or have major safety issues, and looking to monitor safety very carefully as we treat the first few patients and increase the radioactivity.
Just as there, we treated the first five patients, one per month, and then stopped and checked the safety and reported that it was clean, and then moved ahead more quickly. Same approach here. We're gonna treat the first three patients. We're gonna stop and check the safety. We expect to report that next quarter, and then if that looks good, we will continue treating patients and look to finish the recruitment in the second half of the year.
As I stand back and look at what this year looks like for us, how it's set up, our most transformative studies, our phase 3, our pivotal study in the skin in the U.S., our pilot study in the pancreas in the U.S., and our pilot study in recurrent GBM in the U.S. are all set up for very meaningful milestones over the course of this year. We're also, of course, active on a number of other fronts, a number of other indications that are being started. As you know, we just got approval just before we met last time. We had gotten approval from the FDA for a locally recurrent prostate cancer study, and so we are looking to start treating patients there in the coming months as we get those sites online.
We are very active in the strategic front, as we continue to see increased interest from potential partners who want to find ways to work together. That level of dialogue is increasing meaningfully, and there may be something to announce in 2026 as well. This has been a very busy couple of months, and we are expecting that to continue to be very busy over the course of the year. I think by next year we will look like a very different company versus where we are today. I'll say very quickly as our financials came out recently, we have been public, as you know, for just about four years. We had $77 million in cash and deposits at the end of the year.
We continue to be burning about $5 million-$6 million a quarter for our run rate operations. Again, putting aside the one-off CapEx that we've had over the course of the past year for the first phase of construction of the facility in New Hampshire, the actual trials and operations themselves remain at about $5-$6 million a quarter. Again, feeling very good about our financing position, our ability to execute on the studies that we have running right now. With that, I will pause. I'll take any questions, and I thank you all very much for the time.
Raphi, thank you very much for the presentation and overview. Maybe I can kick things off. You know, you've mentioned radiation, surgery where possible, you know, as current standards of care. Could you talk a little bit about not just the coverage, but maybe some of the outcomes that the DaRTs are producing in skin, head and neck, you know, pancreatic cancer? What are you seeing in terms of results?
Sure. Obviously the pivotal study we can't discuss in the skin. We'll have to wait and see if that reads out. There are all sorts of penalties for reading out early, so we haven't done that. I'd say pretty consistently across the skin, we've seen incredibly high response rates, near 100%, where tumors that were not responding to surgery or to other forms of radiation are responding to our treatment, and we tend not to see any of the systemic side effects that one normally sees with radiation oncology. We don't see any of the nausea, the fatigue, the vomiting, et cetera, because the radiation is really confined to the tumor. There's no reason you should see those systemic side effects, and in fact, we really haven't been seeing those.
What we've been seeing is a very mild treatment that avoids the need for a very drastic treatment like a major resection or some sort of other more, you know, drastic treatment like a checkpoint inhibitor, because of the fact that the treatment that we're using is so focused, so powerful, it overcomes the other form of resistance. As you know, the last trial that we read out in this was our US trial in recurrent skin cancer, where we saw 100% complete response rate. Every tumor disappeared, of course, and we saw no serious side effects from the product, which was an A plus. The metrics that we use in other indications are a bit different.
For example, in the pancreas, where number one, unfortunately everybody tends to die eventually, and number two, the treatment paradigm tends to be more of combinations rather than monotherapy use, the question we're being asked is, can we show that when we add our treatment into standard of care chemotherapy, that the patients are living longer? While we haven't done head-to-head studies there yet, we have looked at some of the interim data that we released last year, where we saw that, in fact, each group of patients we examined seemed to live meaningfully longer than what they were expected to live based on the literature. Again, there the data has been very promising, albeit early, but very promising in terms of survival. You know, a little bit of a different metric depending on where we're looking.
What I would say is across the different tumor types that we've done, everything seems to reinforce our view, which has been there for 10 years, which is that if it works in one tumor, it should work in others as well. It's a very broad mechanism. It's not specific to antigens or to mutations or things like that. We've always been of the view that if it works here, it should work there, and so far everything we've seen continues to support that.
Great. We have a couple of questions, you know, on the commercial path. Maybe just to start with on skin cancer, could you talk a little bit about how many patients, you know, have been treated in that trial, how many are left to treat, and then the path to what you see as the ultimate size of the skin cancer market addressable by DaRT?
Sure. We don't read out, you know, any, you know, patient by patient in terms of the recruitment of the study. It's an 86-patient study, and we have single digits left, right? We are really approaching the end there. I'll leave it at that, right? That's why, as we say, we expect to finish over the next few weeks. We're really at the very end of that trial. In terms of the market opportunity, there are 1.8 million new cutaneous SCC cases a year in the U.S. alone. That's the kind of cancer we're going after. We don't anticipate going after all of those patients, of course.
Those we can get to treat the tumor lopped off by a dermatologist should do so. We're not gonna compete with the price of a scalpel. We will go after the most difficult patients. According to the literature, about 3.5% of SCC of the skin will have a local recurrence or a nodal spread. Those are the more stubborn cancer cases we're going after. So 3.5% of 1.8 million is about 64,000 cases a year. I would call that our TAM. Of course, one has to take views as to what will be the penetration and the rate of penetration, which we haven't given guidance. Anybody can take a view on that, of course. These are patients who in some cases do have other options. They could do surgeries.
Those surgeries get more and more grotesque as they try to do it again and again. Not everyone can get surgery, and so again, we'll have to figure out exactly what portion of the market that we take. I think 64,000 is generally the number we look at as the overall market that we're looking to target and then take a slice of it where those patients will be best served by the Alpha DaRT.
Great. Thanks, Raphi. You know, similar sort of questions about commercial path to Japan. Could you talk a little bit more about, you know, what qualifies as like the advanced type of indications, you know, that would be eligible for treatment by DaRT? Same thing you just did, like how many sort of cases you estimate there to be. Then ultimately, you know, in Japan, like what are your distribution plans in terms of treatment and pricing and things like that?
Yeah, absolutely. A lot of stuff in there. In terms of, you know, the requirements or the labeling of the approval, locally advanced, of course, is generally stage three. It's a specific definition of the type of cancer, one which is not easily resectable, right? It's already started spreading to the areas around it. Again, those first-line patients should qualify for Alpha DaRT. I mean, the Alpha DaRT is approved for first-line locally advanced patients. Those who are being treated in a recurrent setting, it's anybody who's locally recurrent. Again, that can be after a dose of radiation, for example, or previous treatment, surgical treatment, they could qualify. They, you know, the second line criteria are broader than the first line criteria, let's say.
In terms of the plans for Japan, I would say, you know, we do see there's a market there for the approval that we have, which is the locally advanced unresectable and the locally recurrent head and neck cancer. It's probably a few hundred to low thousands of patients a year. I don't think that, again, we don't intend on doing a large commercial launch ourselves there, because we think that this will be best done with a partner. For now, we're working with HekaBio, which is the partner in Japan who's also worked with us on the clinical development. They are plugged into those five hospitals where we're gonna start the PMS, and so we expect them to do the distribution for us.
Longer term, as we bring new indications in, I think we'll likely look to launch in a different indication in Japan after launching in the U.S. Again, you guys have heard me say this before, we think that the U.S. is our most important market, the one where we want to spend and prioritize our marketing dollars for a commercial launch there first. As you've heard me say before, it's not obvious to me that we want to launch in the skin or the head and neck when we think the pricing power and the uptake and the commercial ramp will be much faster in something like the pancreas or GBM.
For now, the goal is to focus on the PMS requirements to make sure that any qualifications for the approval are satisfied, to begin other indications like the pancreas and eventually others as well, like the brain in Japan. Then to first stage a U.S. launch of large commercial scale, and then bring that to Japan later on as well.
Perfect. Very good. Thanks, Raphi. I know you mentioned briefly partnerships.
Yep
In terms of, you know, being something that's gotten a little bit more interest. Could you talk a little bit about, you know, what types of partnerships you're considering and, you know, is it treatment specific or region specific? How are you thinking about that?
Yeah, it's a good question. We've gotten a lot of different inbounds in a few different ways, and I'll explain what I mean. We are looking at a couple of different ways to do this. One of them is geographic, right? For example, we are exploring partnerships in Japan, as I mentioned. We are working with HekaBio right now for satisfying the PMS. Longer term, as new indications come online, we would expect to bring in potentially additional partners.
I would say I've been pleasantly surprised by the number of blue-chip, well-known, you know, multinational or Japanese, and either way, either Japanese or multinational companies that you know very well, you know, household names in the biopharma space who have come to us and said, "We are very strong in oncology in Japan, and we'd like to market the Alpha DaRT for you." There, for example, we could consider giving somebody all of Japan, all the indications. Of course, since we're not under pressure to do a massive launch there right now, we could. You know, we don't need to rush and sign a transaction like that right now.
As we continue to de-risk the various indications that we're working on and sweeten the pot for what is in Japan, then the offers available to us are ever larger. That's something we're considering, but not pushing forward very hard because the more work we do in Japan, the more time is on our side for getting a better deal there. We have gotten a couple of inbounds on companies looking to take distribution and commercialization rights for specific tumor types. That is something we're exploring very selectively. Remember, this product is fundamentally the same platform going into different tumors. We don't want different companies calling the same doctor to talk about different indications with the same product. We have to be very careful about that.
If there is something that we think is sectionable where we can say, "This particular point of call, this doctor will be with that company," and then that won't interfere with what we're doing elsewhere, then we will examine that, and it is some of the stuff that we're looking at for this year. Finally, I'd say another, you know, two other points of interest. One of them has been, the data that we've shown on the combination with checkpoint inhibitors has been incredibly interesting for strategics, just given how active and how belligerent I'd say the market is in competition between the various large pharma players competing over who has a 20 versus a 25% response rate in a given tumor.
If our treatment can really triple, quadruple, quintuple those response rates to the checkpoint inhibitor, as we've seen from some of the early interim data last year, then I think that's very meaningful. We have seen players come forward and say, "We'd like to try this with our checkpoint inhibitor. We'd like to work with you on the combination therapy and how it gets marketed," and things like that. Now, last point I'd mention is there's also been interest, more at the nuts and bolts level on ways to work together on some of the physical products we use, right? R&D co-development, supply, right? Remember, we are using existing delivery mechanisms like endoscopes and bronchoscopes and biopsy needles.
Companies that are making those products are coming and saying, "Can we work on our next gen product to make sure that it works with the Alpha DaRT? Can we talk about supplying some of these needles to you?" and things like that. There are some areas of discussion there too.
Great. Very helpful. Raphi, are there any disruptions to the company's operations, you know, with current sort of military actions in the Middle East?
Not meaningfully, no. Obviously, it's you know, it's challenging times, and we go to work, and we have some people are bringing their kids to the office 'cause there's no school. Every once in a while we'll have to stop and go for shelter, and that's uncomfortable and unfortunate, but you know, we're strong, and we hope that ultimately we'll come out better from this. There has not been material disruptions to the operations. We continue to manufacture treatments and ship them out.
Great. Glad to hear. I guess just, you know, zooming out a little bit, you know, for folks who are sort of newer to the Alpha Tau story, maybe they're coming from, you know, skin cancer or they're coming from other cancer treatments, what would you sort of, you know, say to them as the value proposition, you know, if they're looking at you guys now?
I'd say in general, not specific to Neil in particular, in general, I'd say we are offering an incredibly focused, incredibly potent approach, which is orthogonal in many ways to what others are doing, for better or for worse, how you can see it as you wish. Right today, cancer treatment is getting increasingly more focused and niche-y. I wanna target a specific mutation, right, in a specific tumor, and that is great for the patients who have those tumors. They have all sorts of systemic off-target effects to worry about. But in general, that MO is wonderful for patients. Our treatment is approaching it differently. We are saying, "I will approach this with a local approach.
I will target the tumors geographically rather than via some sort of antibody or antigen, or peptide or things like that." In many ways it is different but also complementary, right? We find ourselves working very well together with other therapies because we don't have overlapping side effect profiles which tend to prevent the ability to combine treatments. A very broadly applicable treatment, which we think is very powerful, has a very strong safety profile, and we're doing that together with other therapies where needed. I'd say in particular, the value proposition for today is the fact that we are standing at the beginning of an incredibly transformative year for Alpha Tau.
I expect to be back here a year from today looking at a very different company than where we are today, just given what we expect to happen this year in our pivotal study, in our brain cancer study, in our pancreatic cancer study, all really coming to a head over the course of the next 12 months.
That's perfect, and I think that's a great place to end. I'd like to thank you, Raphi, for sharing the Alpha Tau story with us, and also thank everybody listening for spending time with us today.
Same here. Thank you, Alex, and thanks everybody for joining us. Have a great day.