All right. Welcome, everybody, to Atea this morning. We've got JP, we've got the whole team, but we've got JP starting us off with a brief presentation on some recent announcements.
Okay, great. Thank you, John. Really appreciate the opportunity today. Just to remind you that we're forward-looking statements, and you can get more information with our more recent regulatory filing with the SEC. We have really a great year so far with great progress, and I am pleased to announce today that we have achieved our patient recruitment target. So the screening is closed, and on our phase III, C-BEYOND in North America, and we will announce in a few days that the enrollment will be completed as well with 880 patients in the study. So flawless execution, ahead of time, actually, and with results anticipated till mid-2026. The C-FORWARD is recruiting very well outside North America. We have about six months of differences with North America, really due to regulatory authorities' duration to get the green light.
We have 16 countries, 120 clinical sites, and as I said, we anticipate to get full enrollment also around mid-2026 with results at the end of next year. You can see next year being a pivotal year for the company. We're also very pleased and very excited. I'm a drug discoverer, so when we discover new drugs, I'm really excited. Here, this AT-587 and AT-2490 from our nucleotide platform as the latest asset where we have demonstrated very important in vitro antiviral activity. Why is it important? Because today, with the increased number of solid organ transplant or bone marrow transplant, hematologic malignancies, those immunocompromised patients actually were infected with hepatitis E, have no treatment, and lead to potential, actually, organ rejection, and unfortunately, very rapid evolution to cirrhosis within three to five years.
Last but not least, we have a solid balance sheet of almost $330 million at the end of September, which will allow us to go all the way through the end of 2027, beginning of 2028, with full delivery on our hepatitis C program. We anticipate to file the NDA first quarter of 2027, and we will have data also with the hepatitis C proof of concept and engaging in phase II, phase III, probably sometime in 2027. So may I have the next slide? Just to remind you that our phase III is a head-to-head against standard of care against Epclusa. Our regimen is a combination of bemnifosbuvir, which is the most potent nucleotide, and ruzasvir , which we have licensed from Merck. We believe that we have a potential best-in-class here with what the KOL number one is: short eight-week treatment duration.
We have no food effect, and really importantly for the new model that we are very excited to be able to, with our regimen, to optimize this test and t reat, which allow that to basically diagnose the patient and treat immediately, essentially the same day. And this is allowed by the low risk of drug-drug interaction. I think the best example is the lack of drug-drug interaction with the PPIs, where you have about 35% of HCV patients taking actually those drugs. We believe that our phase III will allow to confirm the robust efficacy: 98% you can see in the phase II, actually even 99% in non-cirrhotic. And as I said, we are looking forward for the data in mid-2026. Next slide. So for our hepatitis E, just this is a new field for a lot of buy-side and sell-side. You have about 20 million individuals infected worldwide.
You have the genotype 1 and genotype 2, mostly due to waterborne transmission. They are basically not very acute hepatitis, mostly an immunocompetent individual. The key issues are hepatitis genotype 3 and genotype 4. These are un developed countries due to foodborne transmission through uncooked meat. And next slide, please. And you can see that this is really this HCV infection with the new progress and really the amazing results in solid organ transplant recipients and hematopoietic stem cells, as I said, hematologic malignancy. We're talking about 450,000 individuals between the U.S. and Europe. And you can see that a significant number can develop rapid cirrhosis, and there is no approved treatment. The first line, actually, is to reduce the immunosuppression, but that's not ideal for organ rejection possibilities. And the second line is ribavirin. We all know the toxicity and the poor activity of ribavirin.
But still, they get about a cure in about 50% of the patients as off-label. Next slide, please. So as we anticipate, it's a very robust potential market opportunity in the range of $500 million-$750 million. About 3% are at risk of those 450,000. We are talking about U.S. and Europe only. And so about 15,000 patients annually. And obviously, that would be an orphan drug with all the positive effect in terms of drug development and pricing. We have a solid IP, both composition of matter and use patent for these new entities. Next slide, please. And here, what you have is the data, very potent, about 200-fold more potent than ribavirin. We're so far very clean profile in preclinical IND- enabling studies.
And I think that by JP Morgan, we'll be able to announce which one, the AT-587 or AT-2490, will go all the way to clinical development. Thank you, John.
Excellent. A lot to talk about. Thanks so much, JP. Let's go back for a second and start a little bit on hepatitis C, and then I definitely want to make sure we get back to HCV by the end of the presentation, but let's talk about the commercial landscape here. So you spent a lot of time talking about the unmet need here, the demands of the KOLs, but this is a market that most folks think is declining, so what's the opportunity here by the time you launch? In a few years, what is driving your confidence that the market isn't declining, and what's holding back the?
Thank you for the question, John. John, will address the question, our Chief Commercial Officer.
Sure. So with all respect to those individuals thinking it's declining, it's growing a lot faster. If you looked at 10 years ago when we first had the DAA appear in the United States, there was roughly around 2.5 million people with HCV. Today, it's closer to four or exceeding four. But each year in the United States, there's 150,000 new cases of HCV, and we're only treating about 100,000 of them. So we're not even making a dent into what is out there. It's why the CDC and others are calling this a public healthcare crisis when you start talking about that for the numbers. When we do look at our research amongst the highest prescribers, as well as some of our KOL events, like the one we recently had, what they really want is they want a product that is more suited for today's patients and for prescribers.
Specifically, what is the product profile? JP mentioned it. They want a short course of direct-acting therapy. They want something that's going to have minimal, if any, DDIs, and they want to make sure it's convenient with or without food. The reason that's important to them, JP did mention this whole idea behind test and t reat, and if you were to ask the KOLs today, "Why haven't we done what you were asking initially? Why aren't we treating more patients? Why aren't we curing more patients?", and what they believe is that we have to move to a test and t reat model. Put simply, a patient comes in, diagnosed, and they're treated, but in order to do that, they have to have confidence in the drug that they're prescribing.
The reason that our research has showed that they like our profile so much is because it's not only short-acting, but for the low potential for DDIs. Arantxa, do you want to comment on test and t reat model so people understand?
Yes. It's been increasingly applied in the United States. It's also done in Europe. And basically, the patient comes in, and the goal is that you will have the ability to test at the site, even with a PCR swab, PCR machines, to test the viral load, and then you give the bottle to the patient. You have already the bottle there, and you give it to the patient, and they go home. That's the ideal setting, and then there are some variations, and that's what now the investigators are implementing more and more. Because what happens is that when the patient comes, you're going to lose that patient if you have to send him to another place to get tested, and then come back to the clinic to get the result, and then come back to get the medication.
So you start losing them, and they go back to the community, and they start spreading it again, and each individual can spread it to another four or five people. And so that's why test and t reat is really kind of the new thing. That's why you're seeing a lot of talks about this at the CDC in our clinic.
Actually, this year at the liver disease meeting in Washington, it's the first time that you had so many presentations on test and t reat.
On test and t reat.
Never before.
But the reason this hasn't been adopted with existing regimens is the docs can't confidently prescribe to everybody who walks in the door?
It's a combination of logistical challenges. Now there is more emphasis because people have realized that this is the way you have to do it because the epidemic is exploding, and so they have to do something about it, and so now they're starting to implement logistically at the sites these methods. They also even now have vans that go to the sites and pick up these patients to identify them and bring them to the clinic.
The goal is a larger awareness of hepatitis C. You can see that both from the current administration, the Congress, with Senator Cassidy, with the new bill, with the White House, the CDC. I think what has been realized is that there's going to be 50% more liver cancer in the next five years in the United States. The only way to address that is we are going to have to decrease hepatitis C in this country tremendously. If we want to eradicate it, it's going to cost us $60 billion. As Arantxa mentioned, it's just the test and t reat. To make sure that, depending on the states, you know this, whether you can really treat immediately or not on the reimbursement.
So that's something else that will have to be changed also to be more friendly to the patient and prescribers.
Okay. So now you've also said that the sales cost of a commercial launch would be modest. You've spoken about the concentration of prescribers that are responsible for most prescriptions. But it does seem like there's sort of two things going on here. On the one hand, maybe the commercial launch for you as an individual company might be modest. But JP, you're just talking about a lot of regulatory changes, massive changes in logistics and the way that this is done countrywide. Doesn't that take lobbying effort, time, and spend somehow to affect those changes in addition to the sales force?
John, you want to take over that?
I think right now, the way the United States is, we have to accept the way it is right now. It's highly efficient with limited competition. So you have 6,000 physicians, right? 80% of DAA in the United States. So from that perspective, it's highly concentrated and very efficient. But at the same time, with lobbying efforts, such as JP is saying, with the bipartisan efforts to try to address this crisis and their intent to implement a test and t reat, the reason we're excited about this is because if you were to ask physicians in a test and t reat model which product profile is the best, it's our profile.
In our model, basically, I can tell you that we will have a significant number of treatment, very robust treatment by the time of launch 2027, 2028. We will launch with both bottle and blister pack, and that is covered in our budget as well, so we are talking about 20,000 - 30,000 treatment immediately available. We are wrapping up in terms of manufacturing with low digit cost of goods, which allows us to do that. The reason we are there is because we have a very robust process chemistry. We are at metric ton with bemnifosbuvir, and 100 kilos on ruzasvir. We are basically achieving, from a manufacturing standpoint, maximization already with the costs.
So you'll be ready to go at launch with all the form factors you need and all the material you need. Can we talk about maybe pricing? You're talking about a premium profile here. What should we expect for a price point relative to established players?
Well, during our managed care research, which represented a considerable amount of lives in the United States, when they saw the profile, that's one thing they were concerned about, is how would you price it because you're obviously offering a better profile. But our position is we would be competitive in the market with the current ones that are out there and in the various segments, whether it's commercial, government, or other.
Excellent.
And I think what's interesting is from a hepatitis C treatment, there was not much difference between the U.S. and Europe in terms of pricing. And actually, hey, the U.K. has announced that they will increase by 25% the pricing. So basically, we're right on target.
Excellent. And while we're on the subject of ex-U.S., you've talked about wanting a partnership there to pursue the global opportunities. Can you talk a little bit about your ideal partners, what you do?
Look, the ideal partner definitely has to have some space either in the antivirals or in liver disease and the adequate sales force, especially Europe and Japan. We can either go to a global partner or regional markets. We have quite an interaction with potential parties. We know what they want. It's clear that delivering positive data on our phase III, including both trials, will allow us to do a pretty large partnership. Look, it's always like that. The money is in the royalties. It's not in the upfront. So we believe that we would command definitely robust double-digit royalties for a product like that, which we anticipate will be definitely around $1 billion, especially ex-U.S.
And launch ready. Let's move in the last couple of minutes to HEV, since you just went over some of the market opportunity and the preclinical data there. Can you clarify the program that you're going to take forward? Is that going to be a pan-genotypic program?
Yeah. The target is the polymerase. So we inhibit the polymerase, and the polymerase is very well conserved across genotype. Just be aware that we are going to be developing this drug only for immunocompromised patients.
In the developed world.
Genotype 3 and for mostly genotype 3, U.S. and Europe. As I said, we will file for orphan designation both in the U.S. and Europe. And yeah, so we can move very rapidly. We anticipate to do next year a proof of concept. We don't know yet, and that's why we are going to do a preclinical chronic toxicity up to nine months upfront, whether it will be a three-month or six-month treatment. The goal is a cure. So it's clear that the clinical endpoint is very well known. And after a proof of concept, we'll go straight into a phase II, phase III.
And you'll be able to treat the ideal would be in the immunocompromised patients without any loss of the.
The beauty is that we have reported chemical structure. So it's no surprise. It's very close to bemnifosbuvir, with only a 4'-F atom in the sugar moiety. So what we have seen in drug-drug interaction with bemnifosbuvir, you can expect that you will have the same lack of drug-drug interaction with AT-587. So that's why we are very excited, especially with organ transplant recipients. You cannot afford drug-drug interaction.
Absolutely. And can you talk a little bit more about the development path there? So you just mentioned not knowing exactly how long the regimen will be going to be. You're going to have to explore.
We can move very rapidly here. As I said, the proof of concept will be 10 patients-12 patients across the board. It can be any of those immunocompromised, either hematologic malignancies or solid organ transplant infected with hepatitis E. What we need to finalize is the dose and the treatment duration. With the phase I, we'll have already an idea with the dose. We can predict as we have predicted before with hepatitis C, with bemnifosbuvir. And so after, we'll go straight into a phase II, phase III because the major unmet medical needs and going rapidly with phase II/III. And we'll see with the regulatory authorities, both the FDA and the EMA, the number of patients that they requested. I doubt that there will be a placebo control. It will not be practical to have a placebo control. So we are very excited about it.
Fair to assume if you're getting started with that proof of concept study in mid-2026, we could potentially see some data in.
No, thank you, John. We have phase I in mid-2026. So it will take about three months. So we will send the proof of concept end of 2026. We'll do first 12 weeks to see if it will do it.
In 2027.
And then 2027.
The results.
And then we'll start phase II/III in 2027. The beauty here again is that regardless you have a three-month or six-month treatment, it's not like hepatitis B that you're going to have a three-year clinical trial. But here you have a three to six months. And then we know it will be again 12 weeks for SVR to demonstrate a cure.
Rapidly.
For those patients, cure.
Much smaller trials than I assume.
Yeah, much smaller.
Yeah, yeah, yeah. I think we're probably going to talk about probably 100 to 200 patients to 50.
Like a non-hands-on situation kind of.
They are going to tell us, but we are not going to have to go to 1,000 patients.
Yeah.
Excellent. Awesome. Well, thank you so much, JP.
Thank you so much, John. Thank you again.