Good afternoon. Thanks for joining us for another session at the 43rd J.P. Morgan Healthcare Conference. I'm Brian Cheng. I'm a senior biotech analyst here at the firm. On stage, we have the team from Roivant. We have the CEO from Roivant. I'll now pass the mic to Matt for a short presentation, followed by a live audience Q&A. Matt, the stage is yours.
Great. Thank you. Thanks for having me, Brian. Thanks for having us, as always. Thank you all for listening. It's a pleasure to be here again. I think it's the same room as last year, so it's nice to be back in a familiar place. So my presentation is provocatively named this year. We were talking about what to call it. But we are just really, really excited about 2025 for Roivant. I feel like last year we came at sort of a transitional moment. We were focused on building up a pipeline. This year is really about harvesting. So sorry, I'm going to make some forward-looking statements. You can refer to these disclaimers on our website. So look, 2025 has truly transformational potential for us. And there's a whole bunch of things going on, and we spent a lot of time discussing internally.
I'm trying to focus everyone on these three. The first, which is also the most proximate, relates to Immunovant. We just put some more money on their balance sheet this morning, as you may have seen. But there's an opportunity this year to really prove, in our view, that our FcRn franchise has best-in-class potential, that the deeper IgG suppression that we can deliver with 1402 is going to yield better clinical benefit. And so we'll talk a little bit more about that in a second. The second major pillar is, last year when I spoke here, we were a commercial company. We had Vtama, our psoriasis drug. We sold that over the past year to Organon in a transaction that we think is good for all.
But we have the potential to get back on the board as a commercial company this year with our registrational DM data set in brepocitinib. That'll come in the middle of this year, second half. So we're really excited about that. I want to talk more about that and what that means for brepocitinib more broadly. And then finally this year, we have a jury trial in our litigation with Moderna over LNP patent infringement. And we are excited, after a long period of talking about that, to actually get in front of a jury and make our case and see how it progresses. So looking forward to that as well. So I'll start. I'm going to go through each of these in turn, and that'll be the focus of my remarks today. I'm going to start with Immunovant, which, again, and Pete's, I think, talking here tomorrow.
Just super excited about where we are and what we have an opportunity to do. So I think everyone knows this, but our lead candidate there, IMVT-1402, has some attributes that we think set us up to be best in class that are truly not seen in other anti-FcRns. We will be in 10 indications with that program by a little over a year from now. We've cleared five INDs already. So as a reminder of our properties there, first of all, we have, based on our phase I data, the opportunity to suppress IgG. We think deeper in practice than any of our FcRn competitors will go. We get to close to 80% IgG suppression in the studies that we've run there. And we're really excited to see that translate into what we think will be best-in-class clinical efficacy. We've also got a clean safety profile, convenient administration.
So we'll talk a little bit more about that. But I think relative to where our peer set is, we're going to be able to administer that with a relatively straightforward take-home auto-injector. So looking forward to talking more about that. And then really long patent life, up to 2043. So this is a really exciting program. Where we are focusing our attention on the future there is on indications where we think we can take the profile of that drug, the 1402, and turn it into a real benefit for patients. And probably what I am most excited about is where we're headed in Graves' disease. So in the past sort of six months, we've put out data in Graves' disease. Graves, for those who aren't familiar with it, is a disease of the thyroid.
You get autoantibodies that attack your thyroid, and you wind up with dysregulated thyroid hormone levels. And what we've now been able to generate in a phase II study of our first-generation anti-FcRn, batoclimab, is that truly one of a kind that's never been seen. We are able to deliver transformative benefit for Graves' patients. So almost 80%. So first of all, in our study, we saw very significant reductions in IgG. So we saw about 80% mean IgG reduction at week 12. So the way this study worked, for the first 12 weeks, patients were on high-dose batoclimab. For the second 12 weeks, they were on low-dose batoclimab. And what we saw, first of all, is almost 80% of patients who were in treatment at the end of that 12-week period got to normal thyroid hormone levels without an increase in their ATD, in their anti-thyroid drugs.
Then actually 60%, or almost 60% of patients were able to not only get to normal thyroid hormone levels, but get completely off anti-thyroid drugs. Graves is a widely prevalent disease. There are millions of patients. There are hundreds of thousands of patients who walk around every day uncontrolled. We were able to take close to 60% of those patients, get them clear of dysregulated thyroid hormone levels, and get them to ATD-free status. This trial also supported the point that I was making earlier. This trial also showed that deeper IgG suppression yields better clinical benefit. Because if you look at the second period of our study where we reduced to a lower dose of drug, we saw a reduction in responder rate.
We think this also sort of shores up for us our view that when we actually get to a commercial drug in Graves' disease, that we will have a potential best-in-class profile. Graves, and I talked about this a little bit, is just an unfathomably large unmet need. There has not been new development of a Graves' disease drug in literally decades. The current standard of care, methimazole and other anti-thyroid drugs, date back to like the 1950s. There are about 880,000 sort of prevalent Graves' disease patients, about 330,000 of which have either relapsed or otherwise are uncontrolled on ATDs and are choosing not to pursue the other alternatives, which are either surgery or radioiodine therapy. There are about 65,000 new patients every year, including about 20,000 new patients every year who are uncontrolled or intolerant.
About 25% to 30% of patients wind up relapsed, uncontrolled, or unable to take ATDs for some reason. I think there is a general view, and I think Graves is one of these indications. It's going to take time for people to fully understand, but a general view that in some corners of the world, this disease is relatively well controlled. What we have found is that is not true, and that we think our franchise offers a pretty unique opportunity to get back to control for patients who really need it. Meanwhile, the data we're generating this year is really going to underscore the hypothesis around deeper IgG suppression mattering. We actually, we for the most part, are already sold on this idea.
There are now 10 clinical trials across four FcRns and seven different indications across almost 700 patients demonstrating that deeper IgG suppression yields better clinical benefit. But the trials that we have reading out this year, mostly this quarter, a phase III study in MG, a phase II study in CIDP, and then later this year in TED will give us 500 more patients of evidence designed to show us how much better clinical benefit we think deeper IgG suppression can deliver for which patients and on what measures. And we think that will be really helpful in showing the whole world exactly what we have from the perspective of the strength of our franchise. So the second major pillar for us for 2025 is brepocitinib. So this is a dual inhibitor of TYK2 and JAK1. It is a drug that we got from Pfizer a number of years ago.
It is, simply put, one of the best anti-inflammatory drugs ever developed. It's a drug we are incredibly privileged to work on. You may be familiar with the general history of JAK inhibitors, but sort of going back from sort of nonspecific and pan-JAK inhibitors like tofacitinib or baricitinib to sort of single-JAK isoform-specific drugs like Rinvoq and Sotyktu, incredible widely used drugs right now broadly in inflammatory disease. Now we've got brepocitinib, which is a selective dual inhibitor of both TYK2 and JAK1, which we think has the potential to deliver unique benefit to patients who have diseases marked by pathogenic cytokines or marked by cytokines signaled by either of those kinases. Brepcitinib is a well-studied drug.
This is not news to anybody, but it's been studied in seven now positive phase II studies, including large studies in alopecia, psoriatic arthritis, UC, psoriasis, HS, Crohn's disease, where we put out some of the best modified clinical remission data ever seen, and our own studies that we ran since acquiring the program in non-infectious uveitis, which showed some of the lowest treatment failure rates ever seen in an NIU study. So really great data across a range of indications. We are studying it specifically in two places right now. One is non-infectious uveitis, which I'll talk a little bit more about in a second. And the second is dermatomyositis. So DM is a classic inflammatory disease. It's marked by rashes in the skin and muscle wasting.
It has tens of thousands of patients in the U.S. with a high morbidity and poor or no modern treatment options and an orphan price point and a concentrated prescriber base. We have pivotal data coming in dermatomyositis in the second half of 2025, which would be the first next approval for us of any drug. And it's the kind of drug that biotech companies like us are doing a really good job launching these days. So we're excited to get this data, set ourselves up for commercial launch, and get back on the board with a commercial franchise we feel really excited about. This is an indication we spent a fair amount of time talking about in the past couple of years as we've gotten it up and running. Recently, one of our competitors, Argenx, in a different modality progressed a myositis trial into phase III.
Now everyone believes it's a big market, so that's great. But we are excited to prove that out with the first new data in a new modality coming later this year. Then the other indication that I mentioned, we generated our own phase II data last year, is non-infectious uveitis, where we've shown best in indication potential. This is a disease. It's, again, a pretty prevalent disease of the eye. It's an inflammation of the eye. In particular, the patients we're most interested in are patients who have back of eye inflammation that is only treatable really with systemic high-dose steroids. Humira is approved in this indication. You can see the data from their study on the right-hand side of this chart. The endpoint in phase III for this indication is called time to treatment failure, which is what it sounds like.
It's how many months from the time that patients present to when they fail therapy and ultimately need to progress to either higher-dose steroids or, in some cases, to blindness and other bad disease progression. In our study at our high dose, whereas placebo in the Humira study was about three months treatment failure and about six months on Humira. I n our high dose at 12 months, which was the end of the study, half of our patients had not yet failed therapy, and so our time to treatment failure, all we could say about it was more than 12 months. This is a transformative, qualitatively different data set from anything that's been generated at NIU before, and we think this is a market with tens of thousands of patients currently receiving advanced therapy who would be eligible for something like brepcitinib.
So we are in a phase III study right now, actively enrolling at NIU. And we would be, I think, the first approved novel therapy at NIU at this time. And then finally, and I can't talk as much about this for obvious reasons, but this is a big year for us on something we've spent years discussing now, which is our active LNP litigation with Moderna and Pfizer. In particular, we have an actual jury trial in the Moderna case in September. I've been joking because I'm not sure people fully internalize what that means. That is a trial in front of a jury. This is like My Cousin Vinny. There will be a judge and lawyers, and we will make our case to the world. And it'll be an opportunity for everyone to hear what we think we've got.
Ultimately, there will be a decision in that case. The jurors will reach a verdict and give us both an infringement and a damages verdict if that case goes the way that we'd like it to. That will happen in September with some important summary judgment motions playing out in the middle of this year, sort of June, July, August, or May, June, July timeline before that. Then there's also ongoing progress expected in our Pfizer-BioNTech case. A big year for that, a year where we really get a sense for what we have and an opportunity to create a lot of value based on science done by a scientific team that's been working on this for literally decades based in Vancouver and who continue to advance the state of the art in LNP even to this day.
Look, we're really proud of what we've built, candidly. We think we have one of the best late-stage pipelines with some of the most important data coming of really any biotech company, certainly of our size and scale this year. We have a ton of data coming at Immunovant that we've talked about. We have data in brepcitinib. I haven't talked at all in this presentation. Maybe we'll talk a little bit about in the Q&A about mosliciguat, our inhaled sGC activator for pulmonary hypertension with interstitial lung disease. That's a program that we unveiled in the last year. And then the last thing I'll talk about is we aren't done, and we continue to do a fair amount of business development. And the environment for us is as good as it gets.
So the operating environment for us in terms of what we do best, in terms of bringing in new programs, has really never been better. We talked a little bit about this last year as well. But between strategic shifts in large pharma companies, there's really been a change, and we'll talk more about this, I'm sure, in terms of opportunities coming out of China and other regions. It looks like antibody manufacturing has just gotten a little bit and discovery has gotten a little bit easier around the world. And that means there's just more and more new opportunities coming around. There's been a temporary reduction in large pharma and M&A. Maybe today is a little bit of a change there with some of the announcements. But that has also led to people looking for different options, depressed biotech valuations, a challenging capital environment.
And so people are looking for creative options. And we feel like we present, both for biotech companies and for big pharma companies, a really unique, experienced, strategic, creative partner who can do the development work and generate data of the kind that we talked about earlier. Last but not least, look, this isn't just about 2025, although that's where I've been focused so far in these remarks. We feel like we have a long-term story that goes sort of beyond the near-term data generation. We think we have 10+ indications in development at Immunovant with multi-blockbuster launches potentially coming. We think brepcitinib will again be a large opportunity, not just anchored by DM and NIU, but with more indications.
We expect to unveil additional indications for Brepo, perhaps as soon as the first half of this year, and then mosliciguat generating data in the second half of 2026 in PH-ILD. So look, we are also in an incredibly privileged position. We have over $5 billion in cash. As you may remember from when we spoke last year, a lot of that was generated by the sale of our TL1A program to Roche. But just a super strong position in terms of our overall cash balance. We continue to repurchase shares. We've now repurchased about $1 billion worth of stock, about 100 million shares retired in 2024, leaving us in a strong capital position and continuing to strengthen the share count ahead of all of these data catalysts that I've mentioned for 2025. We closed on the sale of Dermavant. I alluded to it briefly before.
It's a program we're really proud to have brought to the market, a program that we think matters for dermatology patients and a program that we think Organon is going to do a heck of a good job continuing to market in the years to come, and we'll share in those benefits. And then we continue to do ongoing business development and are in ongoing negotiations for things that I am really excited to present in this forum and others before it next year. So really looking forward to that as well. So with that, I'm going to wrap up the prepared portion of this with a little bit of whirlwind and sit down, and Brian and I can chat a little bit about the business. So thank you all again.
Thank you, Matt. Let's start the Q&A process. For those who are in the audience, if you have any questions, feel free to raise your hand. We have a runner on the floor. For those joining us virtually, you can also submit questions on the portal. So Matt, I think there's that question always. I think we've done fireside chat. We've done dinners in the last 12 months or so. I always start with what's going on with your search for a new asset, New Vant. I guess you brought up a couple new points. And one of them is more acquisitions coming out of China, and there's reduced M&A activity, especially from the U.S. biotech side. Just how do you think about your urgency to execute? I'm sure that you have heard from investors asking you the same questions in different ways.
But as interest rate falls, do you feel a little bit more urgency to find something to fill that gap that investors are asking you about almost every time when we speak?
Yeah, look, thanks. It's a great question. I guess the first thing I'd say is, and this is a little bit what I was trying to hit in the presentation, I think 2025 is a pretty different year for us than 2024 was in the sense that I think 2024 was irretrievably a bit of a building year. We were generating phase II data in NIU. We were generating phase II data in Graves. 2025 is about the harvest for us.
It's about the seeds that we've planted over the years that have the potential to show what 1402 can be, that have the potential to generate a commercial product coming out of that DM data that will matter a lot to DM patients, that have the potential to really reveal after the years of work that we've put in both as a scientific matter in developing LNP and then as a practical matter on protecting our interests there.
I think there in some weird way, and I'll get to why I'm not resting on this, but there's never been less pressure, actually, on the BD side in the sense that there's so much happening in 2025 around generating clinical data and advancing patient interests that in some ways necessarily the BD takes a backseat to the rest of the story in terms of the way that we're going to talk about the business. Having said that, we are a deal making company at heart. We are asset hunters at heart. We have tens of people at this conference meeting with big pharma companies, biotech companies, academic institutions, other investors trying to figure out the ways to expand our pipeline that are most interesting.
We are in active discussion around a variety of different opportunities that we think will be head-turning and important if and when we close on them. But in terms of the time pressure of it, our first mantra on this stuff has always been the same. It's that the point is not to do a deal. The point is to do great deals and to bring in programs that matter for patients. And if that takes us another year, we'll go another year. I don't think it will. I'm a little bit surprised it's taken us quite as long as it has to close on some of the opportunities in front of us. But we're not out to be pressured. We're out to do the right deals and the deals that are going to ultimately produce value and help us advance the interests of patients with these programs.
And maybe just I think the Chinese piece is also interesting, right? I think are you geographically restricted to certain regions? And how are you thinking about the certain type of assets and also where are you looking specifically?
Yeah, it's a great question. So first of all, we, and this is as a matter of general strategy, we like to say that we exist sort of outside the, in the dough that is in between other people's cookie cutters, right? We're not the snowman. We're not the candy cane. We're the weird-shaped dough that exists between the two. And that means that we sort of can't set a precedent, oh, we only do X, Y, or Z region. We only do X, Y, or Z therapeutic area because that's necessarily limiting versus the opportunity set that we have in front of us. We are not sort of specific to one region. We're not specific to one TA. We sort of go where the opportunities are. I do think you mentioned it a couple of times in your questions here.
I do think what has happened in the last 12 months to the BD environment, not just for us, but more generally is interesting. I do think the proliferation of deals by big pharma companies buying assets from smaller biotech companies, and in particular from China, has been a little bit of a shift relative to where we were before. And one thing that I just want to highlight about that is I think there's versions of that shift that are potentially quite good for us. Namely, look, I think part of what we are seeing is not just, okay, there's incredibly impressive productivity from Chinese R&D, but part of that's just like the world has gotten better at discovering novel antibodies against at least some known targets. And so you see things like T-cell engagers with CD19 or CD3 or some of these other components.
There's just a lot of them. What I think that probably means is, first of all, the value of certain kinds of novel agents is lower because you can get one of many. Second of all, the value of good, thoughtful U.S. clinical development is higher, right? The companies that are going to win are the ones who can take an agent, hopefully a best-in-class agent, and can develop it creatively, can develop it thoughtfully, can focus it on new indications. I think that's exactly what we've done with brepocitinib. I think it's exactly what we're doing with 1402. I think it's exactly our sweet spot, taking good drugs and developing them thoughtfully.
And so I think this change in the environment where there's a sort of proliferation of comparatively less expensive programs coming from all over the place actually sets us up really well to compete in the marketplace. And I'm pretty excited for what that will mean for the kinds of deals that we do.
Let's talk about what's upcoming. I mean, I think you framed it nicely, the year of harvest just a minute ago. Which data matters the most to you as the head of Roivant and then why specifically?
Choosing between my children. Look, we have a lot of great data coming this year that I'm excited about. I guess if I were to highlight one, both because I think it is sort of necessarily long-term really important for our story and also because I think it's a little bit underappreciated. I think that brepocitinib as a drug and the dermatomyositis trial as a trial do tend to get lost a little bit in the shuffle of conversation around Roivant. And I think it's, first of all, I'm just really proud of what we've done there. It is a great, great drug. There are days where I pinch myself for the fact that brepocitinib is a Roivant molecule. It's one of the best JAK inhibitors in the world, a class of drugs that is enormously influential.
Rinvoq is going to be, I don't know how many tens of billions of dollars at peak. And we were able to get it because we were thoughtful at a moment where the world was maybe moving away about strategies that we could use to develop it for patients who need it, where some of the liabilities that people were sort of figuring out might not be as big a deal. And so anyway, this is really the beginning called the harvest. This is really the beginning of the next phase of life for brepocitinib. When we first brought it in, we said, okay, we're going to find some orphan indications where we think we can make a difference. And we chose dermatomyositis in NIU. We've got the NIU data, but DM is really the first of the pivotal trials, right?
This is the first time that we're reading out data from brepocitinib that if successful, this will lead to a commercial launch of the product, but obviously, the trial has to work. We have to get approved by FDA, but this is a trial designed to achieve that, and so that puts us in a position to get back out in front of patients and make a difference, and obviously, those are, we've been through it once before in our history. Those are transformational moments for companies. They put us in a different position, and I think we are starting to see in the biotech market, look, we talked about some of the M&A dynamics. You're seeing companies like Verona, like Madrigal, obviously like Argenx do a really great job with powerful agents well developed, and I think we have an opportunity to show something like that in brepocitinib.
And I think in some ways that opportunity begins the day after we read that study out this summer if it goes the way that we want or later this year. So I'm really excited about that data set.
Let's touch on something more immediate, which is the Immunovant data coming up. I cover Immunovant as well. I think I'm also getting a very similar line of questions. Like you said, this catalyst is interesting in a way that is I think that we already know that the deeper, the better. The deeper reduction, the better, right? But this one, a lot of investors view it as sort of the switch because I think MG has been very is very familiarized with a lot of investors. People want to see that you basically validate that notion, right? In your view, just based on how the trial is designed and what we could expect at the top line, how would you frame what a win scenario looks like coming later this quarter?
Yeah, we could talk a long time about parts of this question because it's a pretty interesting and textured question. One thing I'd say is I think we, at least at Roivant and Immunovant, potentially including you, have accepted the idea that deeper IgG suppression yields better clinical benefit. I'm not sure that like they where they is like the people in this room, beyond this room, like in the world have necessarily accepted that yet. And I think this data is an opportunity to really underscore and highlight the meaning of that claim, especially against a backdrop of just how well Argenx has done in launching Vyvgart in MG. What are we hoping to see? Look, I think there's a lot of dynamics at play here. Ultimately, the goal, the biology here is not very complicated, right? MG is an autoantibody-driven disease.
Downregulating FcRn impacts the re-uptake of IgG autoantibodies. So you downregulate all IgG antibodies, including the pathogenic ones that lead to MG. I think the premise, therefore, that further suppression of those antibodies yields better disease benefit isn't that controversial as a scientific matter. But it has been shown in our Graves data that I put up earlier. It's been shown in our TED data. It's been shown in J&J Sjögren's data. It's been shown in UCB's ITP data. But it hasn't really. It's been shown at the individual patient level in many MG studies, but it hasn't been shown at the cohort level. And it hasn't been conclusively shown to extend up to call it 80% IgG suppression where we can get to with batoclimab and IMVT-1402. So I think you'd really like to see that, right? You'd like to see a dose response.
You'd like to see a meaningful delta between placebo, obviously, and then low-dose FcRn therapy and then high-dose FcRn therapy. In particular, and we can talk a little bit about why, I think I'll be watching, for example, the absolute magnitude of the MG-ADL improvements from baseline in those various cohorts because I think if we can show a meaningful delta on that basis, we'll have a pretty clear picture in our own head of the fact that deeper IgG suppression can matter. Look, I think you'd like to see better placebo-adjusted separation as well. I think the only caveat there is placebo rates the trend on placebo over time in MG has been higher. Every doctor and every investigator and patient in an MG FcRn study knows that FcRns work in MG. MG-ADL is a relatively subjective endpoint in some ways.
So I think that's a higher bar to clear. But it'd be nice. So I think in order to see a clear dose response, ideally strong numerical improvements in MG-ADL. And in a perfect world, we'd see a relatively low placebo and be able to show that on a placebo-adjusted basis as well. But that's a harder thing to do. We will obviously look elsewhere as well. We'll look for deep responders. We'll look for responder rates. We'll look at QMG and other scales. There sort of is a totality of evidence that'll matter. I just think in this industry, the worst phrase you can utter with a data set is totality of evidence. And so I think the first place we'll be looking is overall MG-ADL improvement.
Okay. And how much of what would be a good improvement? Because I think when we look at what Vyvgart and Nipocalimab had reported in terms of correlation of IgG reduction versus MG-ADL, there's some precedent to kind of understand what that delta looks like. But like you said, placebo rates have been creeping up a little bit higher in some of the later, some of the more recent studies. So how should we think about what the delta could look like? And then is the ultimate goal to try to prove a win in the percentage of deep responders for the IMVT-1402 program?
Look, I think all of this is ultimately designed to underscore in the world's mind that deeper IgG suppression can yield clinical benefit that matters. So deep responders is obviously one way of showing that. Responder rate overall greater than 2. MG-ADL improvement is a way of showing that. There's lots of different sort of angles to show that. I don't know that it's about sort of any specific metric here so much as just about sort of underscoring the principle, especially because this is with batoclimab and most likely the commercial drug is 1402. I think Pete has given some numerical bars in other settings. I'm sure you're talking to him later. He may have some comments on that.
For me, it is less about setting a specific numeric bar for these deltas and more about, look, I think what I hope is that this data set is conclusive, that as I hope any reasonable person looking at it sees a clear dose response. I hope any reasonable person looking at it sees a drug that if you believe that our low dose is comparable to what, say, Vyvgart can deliver with a mid-60s percentage IgG suppression, and you believe in our data set that our high dose is outperforming, then I think you can look at that data set and you can walk away and think, yeah, there's an opportunity. This is really a best-in-class agent. So that, I think, is what we're really trying to show. And I think it's less about, is it 5% different or 10% different or whatever.
It's more about looking at the data and having a clear signal that dose matters.
Any questions from the audience? Okay. So switching gear to maybe just one more on I think we're hearing a little bit more on the route of administration, the differentiation there. How do you think about what we see across from the different competitors in the space? How differentiated do you think Immunovant's SubQ self-administer ROA is compared to others?
Yeah, I don't want to steal Pete's thunder when he comes to present. But I gave Pete a piece of advice that I doubt very much he'll take, which is I gave him the advice, we've got this timer here. And I said he should count out five to 10 seconds, which is how long a standard auto injector takes to administer. And so we can do that. And that's five seconds. And then I told him to count out 90 seconds, which is the upper end of the range given for the administration of 5 cc Halozyme product. And I'm not going to do that now because I think 90 seconds in this room will feel like a literal eternity. But it's just very different. Look, our competitor products are good. Vyvgart's an amazing product.
Argenyx has done an incredible job marketing that product, getting doc mindshare, and really getting that product out to patients is admirable. I think in practice, patients would prefer convenient at-home administration with short injection time, which I think there is no question. I think if we don't have a better product from an efficacy perspective, we can still win in lots of settings. We can still win in new indications. We can even still win share in MG. But ultimately, what I think these patients want is to be treated the best possible. And then I think route of administration comes in second as something people care about. That said, I think we have a significant advantage relative to the other SubQs that are currently out there.
Okay. And maybe just switching gears to the upcoming LNP cases. I know you can't speak much to it, but maybe just kind of help us to frame what are the potential outcomes that could happen after the hearing. And what could potentially will you be looking for in the best case scenario?
Yeah, see, this is where I go back to my comment about jury trials and My Cousin Vinny. Look, what do I think we're looking for? We believe that our scientists invented this technology and that it's been used in the COVID-19 vaccines. And we hope we can convince a jury of that. And we hope they find in favor of infringement. And we hope they find damages commensurate with the importance of the product. And so I think ultimately, what we're hoping for is that when we're sitting at this conference a year from now, we've got a clear answer from the jury that our product mattered or that our inventions matter for these products or at least the product that's the subject of the trial and a sense for just how important that is in terms of damages in terms of the future.
So look, we'll see how that plays out. Again, it's a trial. And so we're going to make our case. And they're going to make theirs. But I'm excited that we finally get our day in front of a jury to do that.
Just lastly, just touching on Priovant and I think this program has great data on NIU, but it has never really gotten traction with investors, but now we have DM data coming up in the back half of this year. How should we think about the bar that you need to hit for DM? And just to confirm, is this phase III alone sufficient for you to file? Or are there more data points that you need to collect before filing?
Yes, we think the answer to that question is yes. We think this data set is sufficient to file, and we hope it's approvable on the basis of this data set. Obviously, the data has to be successful for that to work. Look, DM is a very underserved patient population. The patient population with DM is very underserved. The only approved novel mechanism is IVIG, which has its own liabilities. It's a disease with high morbidity and high mortality. We said it has our estimate was about 40,000 patients in the U.S. I think Argenyx gave a 70,000 patient estimate in a different setting. It's a relatively large orphan market, and the truth is, I think the bar is a successful study. I think the bar is hitting statistical significance. I think the bar is delivering clinically meaningful benefit, which the study is powered to do.
And I think if we do that, this drug will be widely used. The truth is that JAK inhibition is already a relatively well-understood method of treatment among DM treating physicians. They're used off-label. There's an open-label study of tofacitinib that was done by an investigator. There are case reports of JAK inhibitors being used. And DM is an interferon, among other cytokine-driven, inflammatory disease. JAK inhibition should work. So the onus is on us to run a good study, to control placebo well, to demonstrate clinical benefit. And I think if we can succeed at that, there will be a lot of patient demand for a novel treatment option.
How should we think about placebo rate there?
Yeah, look, that's the rub with all of these studies, right? These patients are on background steroids. We are, I believe, the first large placebo-controlled DM study that has a mandatory steroid taper in it. And so we are set up for maximizing focus on the things that matter there in terms of controlling placebo response rate. I don't think there is a great answer to what should the placebo rate be for tests in a DM study. Excuse me. So it's hard to know exactly what we're solving for. But I think the parameters of the study are designed to focus maximally on achieving a good outcome.
Maybe just lastly, just want to touch on Mosliciguat. What are some of the upcoming catalysts that we could expect? Will there be additional data that you will sprinkle along throughout 2025, 2026 before we see data in PH-ILD? Just how should we think about the data flow for that program?
Yeah, no promises. The big data set there is second half of 2026 when the phase II study reads out. And that'll be a really important data set. We will not bluntly have translational data from Group 1 to Group 3 pulmonary hypertension until we have that data set. That's really the first time we get there. That having been said, Bayer conducted a very robust package of phase I studies for this molecule. There's about 170 subjects exposed, some combination of patients and healthy volunteers. It was the totality of that data that got us excited about the program. And that includes not just the single dose study that we shared, the ATMOS study that we shared this fall, but it includes multiple dose studies that drove durable cGMP production. It includes a whole bunch of PK and PD work that's been done.
And so I do think as we work to make sure that investigators are excited about the program, that people have a sense for what our thesis is, I do think there's an opportunity for us to share more of that data. But the next real sort of new data generated in that program is going to be the phase II study in the second half of 2026.
Okay. And then how translatable is the PVR reduction that you've seen towards PH-ILD? And just how much confidence can you have around the upcoming phase II?
On the one hand, the end of overall clinical trials run in PH-ILD is pretty small, right? Tyvaso was approved on the back of a clinical package that United Therapeutics ran. There was a study by Gossamer . A while ago, there were studies in some systemic sGC stimulators that were not successful for reasons that we think we understand, but very small numbers of studies. In those studies in general, other than the one issue where it's understood around V/Q mismatch, the predictive power of PVR has been good. That is, agents with better PVRs have resulted in better treatment. And agents with worse PVRs have resulted in worse treatment. And so I think it should be translatable both from PAH to PH-ILD and from PVR to treatment benefit in the PH-ILD patient population.
United Therapeutics with Tyvaso has done a beautiful job of paving an approval pathway and showing us how it's done. They are masters in this area. We're excited to follow in their footsteps. We think we have a shot at a best-in-indication kind of a program given the PVR data that we have in pulmonary hypertension group one.
Okay. Any questions from the audience? All right. That concludes the end of our Q&A. Thanks for.