Good afternoon. Welcome to Day two of the Jefferies Healthcare Conference. My name is Dennis Ding, Biotech Analyst here at Jefferies. I have the great pleasure of having CEO Matt Gline of Roivant Sciences here with me. Welcome.
Thanks for having me. It's great to be here.
I usually like to kick things off with just a very broad sort of topic, just like an intro of where the company is today, some of the progress that you guys have made over the last couple of years, and maybe mention TL1A. Then just your outlook going through the rest of the year in 2026.
Yeah, great. Thank you. Look, that is a super broad question. We are, for those who do not know us, and I think most of you do, we are a clinical stage biopharma company focused on, we ultimately focus on the same thing everybody else is, developing medicines that matter in ways that we hope add a bunch of value. We have built at this point a portfolio of late-stage programs that we are really excited about. There are probably three that we will at least touch on in the conversation today. One is our anti-FcRn franchise, which is at our subsidiary Immunovant. Another is a JAK1/TYK2 inhibitor that we are developing for dermatomyositis, non-infectious uveitis, and cutaneous sarcoidosis. And then the third is a PH-ILD program called mosliciguat.
We have a handful of other things we've worked on over time, as well as a couple of other aspects of the story, like we're in patent litigation with Pfizer and Moderna over their use of IP and their COVID-19 vaccine. We've had a busy couple of years building this portfolio. I think part of what Dennis was alluding to was we have just under $5 billion in cash. The main reason for that is because a couple of years ago, we bought and then sold an anti-TL1A antibody from Pfizer to Roche in a way that resulted in quite a lot of proceeds to us. We have been sort of deploying that cash cautiously and thoughtfully towards our existing pipeline, towards some new things that we're looking at, and then towards buying back stock, which we've been doing over the past couple of years as well.
You asked about the outlook from here. Look, I'm super excited about what our next couple of years look like. The first major event here, at least on the portfolio side, is we have a phase three readout for what we think will be registrational data in dermatomyositis at brepocitinib, which, if successful, will put us back into the sort of commercial hot seat, launching a program that we think could be very important to patients in what we think is a big and interesting market. That data will come in the second half of this year pretty soon. On top of that, portfolio development-wise, after that, we then have a bunch of data coming both for additional programs at brepocitinib. We have NIU data coming about a year and a half after that.
We have data coming in Immunovant and a whole bunch of indications and data coming the second half of next year in the PH-ILD program. A lot of clinical data in programs that advance significantly or become commercial really in the next couple of years. On top of that, we have a Moderna trial that we believe should be in the second half of this year, knock wood, assuming everything progresses according to plan. That will be meaningful as well.
Sure. That's a great overview. Before we kind of go into some of the pipeline, we'd love to ask you more broadly just around BD and some of your strategy. You've been talking about how the environment has been very ripe, very conducive to dealmaking for the last 12 to 18 months. I feel like now with so many companies trading under cash, et cetera, how do you view the BD landscape right now?
What are the really good companies trading under cash? Look, we're pretty excited about the stuff we see in the world. So we've always been drug hunters. It's a big part of the history of our business. Most of our partnerships, but by no means all, have been with big pharma companies that are in a moment of transition. And we have a lot of interesting things on the racket in conversations with various big pharma companies that we like a lot. And some of them, I hope we'll get done and will be awesome to talk about. The biotech market has had a rough four or five months. That's tough for everybody, but an opportunity for us as valuations get cheaper, expectations change. Stuff that was competitive last year is not competitive this year. So I think that's all been constructive as well.
We are long-term owners of the things we buy. I think we're pretty choosy and have taken our time. I think we're going to continue to be pretty choosy and take our time. There are definitely multiple things I'm excited about that could get done within probably months is the right time frame to articulate there, maybe a little sooner, depending on what comes together. Some really good stuff on the BD side.
Okay. Maybe also talk about, given just some of the uncertainties around tariffs.
One more comment on BD, which is I also think we're increasingly just excited about both the FcRn and JAK1/ TYK2 sort of opportunities. One kind of BD that we have done a fair amount of is just indication expansion for those businesses. I think you will also see more of that from a capital deployment perspective.
What do you mean by that? Like organically or you mean?
Yeah, organically. Like we've added cutaneous sarcoidosis. Yeah, exactly.
Okay.
Which we view in a very similar context to BD. It's spending more money on things we care about.
Okay. On tariffs, right? That has been definitely a point of uncertainty and pharma is going through. I'm sure there's scenario planning, worrying about margins and EPS, et cetera. How has the tone in these discussions with pharma changed or maybe not changed over the last couple of months during this period of transition?
Yeah, it's been a really interesting, I mean, honestly, it's been an interesting 18 months, but also especially an interesting few months on big pharma conversations, especially around M&A, which is the rhetoric is busy, right? The rhetoric is like teams working on things, looking at things, spending a lot of time, engaging actively. The pace isn't matching that. I think there's a bunch of reasons for that. Look, I think, first of all, we've seen more China deals out of big pharma. We've seen more value buying out of big pharma. I think that's had an impact on how they think about deploying their time and capital. Also, I was meeting with a big pharma CEO a few weeks ago. His comment was, this was before the MFN noise.
His comment was, I've spent 85% or 90% of my time the last month on tariffs. That means everything else involved in running a, whatever, multi-hundred billion dollar pharma company has been squeezed into 10% or 15% of this big pharma CEO's time. I think you can imagine then why big deals have been sometimes hard to get done. You can imagine why some of the stuff in general has been slower. That's probably been the main impact for us in terms of sentiment in these conversations. It's just been a real time suck trying to figure out which way is up. I'm sure the MFN stuff has made that even more complicated. I'm sure there's just like a lot of, I hope everybody's okay, a lot of trying to figure things out.
Do you feel like pharma needs to get clarity and visibility on both tariffs and MFNs before doing BD deals, or do you think they're okay with kind of running with it?
I think what we've learned this week is that the answer to that question is evidently not, which is great.
Okay. And then therapeutic areas in terms of BD and new assets?
Yeah. What we've always said is that we are opportunistic, that we'll go anywhere, that there are certain things that lend themselves better to our portfolio construction than others, that onc has always been a little bit harder for us because of the competitive dynamics, that gene therapy and cell therapy has always been a little bit harder for us because we don't have the concentrated exosome manufacturing, but that everything else is on the table. There's stuff that we like right now in immunology, respiratory, pulmonary, cardiometabolic, mostly outside of obesity, rare disease, and a little bit of what I would call like spec pharma.
Okay. Spec pharma, what do you mean by that exactly? Just.
Yeah. I mean, the stuff we've done before in this area has been like women's health, urology.
Like PAMA?
Sorry?
Like Patama?
Yeah, but also like relugolix and vibegron or like, yeah.
Okay.
That sort of stuff.
All right. All right. That's helpful. Now, kind of going to the pipeline and the most near term, I think, data card flip is the phase three dermatomyositis data in the second half of this year. Maybe you can frame that opportunity, the indication, the commercial opportunity, et cetera, for people.
Yeah. Dermatomyositis is a very severe, rare inflammatory condition that presents with both muscle wasting and very severe skin symptoms, like a very severe rash. It is often fatal and very difficult to live with. There are basically no good therapeutic options. These patients are on sort of the standard sort of cadre of steroids and methotrexate and stuff like that. IVIG, one IVIG is approved for use in DM and works okay, but has all the liabilities associated with IVIG. Other than that, there's really nothing else. We will be, if successful, the first oral sort of novel medication in DM and sort of the first next generation therapy. There's a variety of other things in development, including Argenx's FcRn and so on. We think the patient population in DM is, the number we've given is around 40,000 patients. Competitors give numbers up to 70,000.
The truth is like a little bit hard to know, but my guess is this is a market that is similar in overall size to something like myasthenia gravis and similar in support of overall price point to something like myasthenia gravis. The main difference between them is that we're just like earlier as an industry in terms of our understanding of what the commercial landscape looks like here. That makes me really excited about the position that I think we get to play as kind of the first novel therapy other than IVIG to market.
Okay. So then when the data reads out, what do you consider to be good data?
The great news for us about this space, first of all, is that the mechanism, our mechanism, JAK1/ TYK2 inhibition, and especially JAK inhibition generally, DM patients are mostly treated by rheumatologists. They have a tremendous amount of familiarity with JAK inhibition and a strong belief based on case studies and investigator-initiated studies and so on that JAK inhibitors will work to treat dermatomyositis patients. If they have, there are no unlabeled JAK inhibitors. If they have an unlabeled JAK inhibitor, we think they will reach for it. What that means to us is the bar for success here is mostly just a statistically significant trial. We need to hit a P-value on the primary, which in our case is mean tests. That's it. That's what we're looking for.
Okay. When you talk to doctors and you mentioned that many of them or some of them use off-label JAK inhibitors, can you kind of quantify that? Is that very common? Is that kind of a niche type of?
It's not like commercially common because JAK inhibitors are really expensive. And so there's just like no good way to do it. There are hundreds at this point. I think we're aware of about 600 published case studies of JAK use in dermatomyositis. So you can imagine that's certainly not every patient treated is getting a case study published on them. You can imagine it's some multiple of that in terms of treated patients, but still like a very small percentage of the overall treatment landscape.
Okay. When you think about the phase III, placebo is kind of very difficult to predict and handicap here. Can you just help investors frame what placebo would do? Because the whole goal is to show a delta over test. What are some of the mitigation strategies around placebo?
Yeah, first of all, I'll say, and look, I am the first to agree that placebo is, if you were going to be nervous about something in this study, the thing you'd be nervous about is placebo. Some of that's just a function of tests. Tests is like not a great endpoint. From a placebo perspective, it starts at zero and can only go up. And so that's just like mathematically not great. That having been said, I guess I'd like slightly object to the characterization that it is unpredictable in the sense that at this point we have a handful of studies. We have a 52-week corpus study that failed. We have the Argenx data that was just put out. We have all the IVIG studies, some of which have been successful and some haven't.
I'd say like at six months, if you had to try and dimension it out, like placebo tests ranges from, I don't know, 25-40, which is like a real range. It's not like in that sense, there's some real unpredictability there, but it is what it is. I think, look, we are doing the things that you would want in terms of managing placebo. We are the first, certainly first phase III study to have a mandatory steroid taper in the protocol, which tapers down to at a maximum of 5 mg of oral prednisone by 36 weeks. I think that should be helpful. That said, I was also just encouraged, Argenx is, and this is a different patient population, was pan-myositis data, but I felt like relatively high placebo. I think they had like a close to 40-point tests, different patient population.
Yeah, around five, yeah.
Nice separation from the drug effect and very low P-value in a way that, like, made tests feel reasonably, in their study, tests was reasonably well-behaved as an endpoint. I would hope for the same for us.
Okay. Knock on wood. Yes. And then.
This isn't real wood, which is making me a little nervous.
Going beyond 24 weeks because your primary endpoint is 52 weeks, how does placebo typically behave beyond the 24-week period?
I think there's only one study that went beyond 24 weeks. I think it was the corpus study. I don't really think there is a typical. This is definitely a question that no one knows the answer to. I think you would hope, first of all, that it would level off to some degree. Second of all, that like some patients, especially with the taper, will worsen or have to discontinue and that that will be net helpful from a mean test perspective.
Is there any data that you can point to for investors that could quantify, I guess, the placebo mitigation on tests?
From the steroid taper?
Yeah. Just anything that you can point people to because I feel like, like you said, to your point, the placebo is kind of the biggest point of variability here. If people feel a little bit more comfortable around how that could behave and if there is any kind of incremental data they could point people to, I feel like people could be a little bit more confident and feel better going to this data set.
I'm not 100% sure that my job is to convince people not to worry about placebo before the data set reads out. It's a real risk. People can worry about it. The thing that I'm most focused on is if and when the study reads out positively and we're happy with the outcome, people should understand that it's a big commercial market, that it's an exciting opportunity, that we've delivered a clinically meaningful benefit. I just, like, look, I think we are appropriately focused on placebo mitigation because it's one of the important things about running a study well in immunology. To people who came to me and said, I'm worried about placebo for tests, I'd probably say, me too. I'm also worried about placebo for tests.
I think the thing that is encouraging is at this point, several other drugs have been able to produce statistically significant deltas versus tests in a way that suggests it's a viable endpoint that works in clinical trials. There's a lot of evidence to suggest that JAK inhibitors and brepocitinib specifically should work in this indication. I feel cautiously optimistic that we're going to be able to work through that.
Okay. Can you comment on the small phase two study that's on clinicaltrials.gov around DM and flipped to completed just a couple of months ago? Just comment on what the purpose of that study was and.
Yeah, the purpose of that study was to achieve orphan drug designation with FDA, which requires certain specific clinical, there's like you need some clinical data in mechanism to do. And so we ran a small skin-focused study in DM. We haven't said what the results were. They were good. Whether we put that data out or not is an open question still. Anyway, the purpose was to get orphan drug designation, which has some real benefits for us. Gets us better user fee situation, better engagement with FDA, et cetera. It's been a good trade.
Okay. You guys will be hosting an investor event with Priovant around DM. Talk a little bit about that and just what kind of things will you be disclosing and sharing?
Yeah, I want to set the stage correctly for that. I don't think this is like a, I don't think the investor event ought to be like a stock catalyst. This is not like we're trying to put out some surprise data and get people to move. I think this is about making sure that people understand the trial design, some of the placebo stuff that we're doing and why and what it looks like, a little bit about baseline characteristics. Then just like disease state education, making sure people understand what dermatomyositis is, why we care about it, why we're focused on it, why we think the opportunity is big. I also think part of that is that test is a pretty coarse-grained measure.
We want people to understand what test is, all the different components of it, and then also what some of the other endpoints we're measuring are, which I think is the lesson. Look, obviously if we miss on test, it's done. Some of these other endpoints will actually be the commercially relevant endpoints, things like CDASI, the talk around skin presentation, DMOMS, which is a dermatomyositis, but test is not even specific to dermatomyositis. It is the same for all flavors of myositis. There are other subsets of test and other endpoints that the DM community is more focused on. We want to help the investor community understand better a little bit how we think docs will view the data set before we put it out and a little bit more of just like what does good look like.
Okay. That's very helpful. In terms of the regulatory path forward, if the phase III were positive and like you say, you just need to hit stat six on tests, you only need one trial for approval.
We believe that one trial with good data is sufficient.
Okay. How should, assuming the data is positive, how should we think about the launch? Do you think there would be a bulge of demand? Do you think it'd be kind of a more steady, slow and steady type of trajectory? Like, how should, how are you guys thinking about it?
These are also tough questions to answer because I feel like the right thing to do is to say it's going to be slow and crappy and then to beat every quarter. Look, I think DM is a big patient population. I think the nice thing about launching in DM is the prescriber base in the U.S. is concentrated. Many of these patients are treated at specialty myositis centers. Those centers have hundreds of DM patients. You can address like lots of patients in a relatively concentrated way, including in many sites that are already trial sites who are familiar with the program who are excited about it. I think it has a lot of ingredients that make it feel tractable as a launch that make it feel like we can get it done.
As a further benefit, look, this is not like VTAMA. This is a drug that will have a rare and orphan price point. And you do not need that many patients to have a successful early launch. I think we have a good bead on where to find those patients now. I feel like it is a launch that we are excited to take on.
Okay. Historically on pricing, you guys have always kind of pointed to what Pfizer said three or four years ago around being $150,000 net. Has that changed at all or do you still feel?
That price has gotten a little more expensive over time. Look, I think the bookends are clear at this point. On the bottom end of the range is IVIG, which is pushing $200,000 these days. At the top end of the range, you have Argenx coming in, albeit after us, but they are going to come in at an FcRn price point, which will be probably $600,000-$700,000 when all is said and done for a full year of therapy if it is similar to CIDP in terms of usage. I think that sets a pretty wide bookend. I expect us to come in within those bookends.
Okay. Okay. That's very helpful. And that's list price. Is that right? Or is that?
Yeah, although it's way too early to be talking about rebating and gross tenets and all that stuff. I think modern launches have a wide variety of approaches to that question.
Okay. Perfect. How are you guys thinking about product positioning once approved relative to IVIG?
I guess I think the answer to that question is ahead of or after. I do not think it's like I expect there to be a clear treatment paradigm that every patient who gets off of steroids immediately goes on to brepo and then IVIG or vice versa. I think it will be a viable alternative option. I guess my view is assuming our data supports this, many patients would prefer a once-daily oral to going on to IVIG. I think we have a really attractive form factor. The answer is I do not expect there to be a clear algorithm of one then the other. I think we are happy to exist ahead of IVIG in many patients' treatment journeys and happy to exist after IVIG in others.
Okay. You mentioned earlier around when you talk about BD is not necessarily inorganic BD and indication expansion. That is something that you guys have been kind of doing around brepocitinib. I am assuming if the DM data were positive, that you would take another hard look and maybe be a little bit more enthusiastic about that and announce multiple indications.
In truth, irrespective of the DM data, there are other indications for brepocitinib that we are excited about now that we are doing active work to explore.
Okay. Perfect. So we just spent about 20 minutes on DM. That's great.
I love it.
Maybe we could talk about Immunovant. I mean, that's another big topic of discussion among investors. Can you just remind us, can you remind us of just the data updates that you got that you had this year?
Yeah.
How are you thinking about the opportunity moving forward?
Yeah. Our lead drug in Immunovant is IMVT-1402. It's a next-generation anti-FcRn that's currently in pivotal trials in a bunch of indications: Graves and MG and CIDP and Sjogren's and some exploratory studies and some earlier ones. We had previously a drug called batoclimab. We have a drug called batoclimab. It's an earlier generation FcRn that is also a very good drug. It suppresses IgG quite deeply. It's a nice sub-Q, but it has the unfortunate effect of increasing, as you know, LDL cholesterol. It's therefore been, it's certainly our second most exciting asset, as it were, of two.
However, we had a set of phase three programs for that drug reading out, including in MG and CIDP, which is phase II B, but a set of programs reading out earlier this year that had a few purposes for us, probably the most important of which was to help us show that in an indication like MG, we could continue to deliver a dose response for deeper IgG suppression. That is, that deeper IgG suppression would yield clinical benefit for those patients. I think everybody can agree across the entire investor community that we've just put that to bed, that everybody in the world is now totally convinced that deeper IgG suppression yields better clinical benefit. No, I don't think that's true. Look, we were really happy with the data we put out there. We showed a clear dose response.
We ourselves are convinced that we have an opportunity to be a best-in-class drug in MG, especially when looking at more stringent measures like effectively clinical remission. That data was important, therefore, for painting a path forward for us in terms of how to focus on those endpoints for 1402, for example. We thought the CIDP data also looked really promising, especially when cut on high versus low IgG suppression patients.
Okay. The idea there and the strategy there is to redefine what is good in MG. Is that kind of fair to say? Because people have been kind of preoccupied with MG-ADL and you guys are coming out with this new minimal disease expression.
MSE.
Right. Right. So.
Argenx has started talking about MSE and docs have started talking about MSE. Look, I think MG is actually still pretty young as a commercial market. I think what we expect to see happen and what we would like to see happen and what we would like to be a part of engineering for the field is the same progression that many other immunology markets have followed, right? From IgA to PASI 75, PASI 50 to PASI 75, PASI 90 to PASI 100, from ACR 20 to ACR 50 to ACR 70, from clinical response in UC to clinical remission in UC.
I think we see a similar trend in the future of MG going from not just like MG-ADL improvement from baseline, but like deep MG-ADL responders, not just like two-point MG-ADL responders, but five and seven-point MG-ADL responders, not just MG-ADL improvement, but these MSE sort of clinical remission kind of patients and also durable MSE, patients who obtain clinical remission and then hang on to it for a long period of time. I think that is where MG patients deserve to see the market go and is where a whole bunch of people involved in MG, not just us, but like people developing T-cell engagers and people developing all kinds of other therapies are going to want to see the market go towards these like truly like patient-desired outcomes.
I think we can help move the FcRn field there in a way that I think is like what the next generation of data is going to look like anyway, and in a way that I think really highlights what we can deliver with deeper IgG suppression than what some of our competitors will deliver with lesser IgG suppression. That is how I think we see the market evolving. I think we'd like to be a big part of carrying it there. Now, having said that, look, the truth about MG is these are patients who had very few options before. Efgartigimod is a good drug for these patients. It works well. They like it. Docs are happy prescribing it. The patients are happy being on it. We obviously need to offer a real value proposition in order to take meaningful share.
It is why I put some of these other indications like Graves ahead of the value prop of MG. It is because in Graves, we get to be in Graves what Argenx is in MG. We get to define that commercial market and be out in front of it. It is why I think that is like an easier battle versus the cage match that will wind up being in MG. That said, I think there is a lot of opportunity in MG, to be clear, along the lines of what we were just talking about.
Sure. And then Graves?
Yeah. Graves is one of these fun markets where anyone trying to build a commercial model is going to result in a stupid number because there are 330,000 Graves patients who are refractory to all pharmacological treatment options. They've failed every drug available. The only remaining options for them are to feel shitty, sorry, feel crappy, or to have their thyroid surgically removed and go on lifelong Synthroid and still feel crappy. I think that is a pretty special market in terms of what we can offer to patients, especially given the data that we showed in phase two. It's a market where it's really exciting to be first. There will be a lot of other people showing up in Graves disease, of that I am confident. I will get questions about all of them.
The fun thing I get to say to those questions that I could never say in MG is, well, we're going to be ahead of them. You can just watch us first and then they can sort of figure out how to play from behind. That feels great.
How do you think about the future of your relationship with Immunovant? There have been a few changes, at least communication-wise over the last few months. What is the best case scenario for you? Where would you like that relationship going?
We'd like a good relationship with Immunovant. Look, we own 60% of the company, 58% of the company. That's a little bit higher than it was a year ago. We like the program. We think it's a really exciting place to be. We think, look, 1402 is a drug that's going to work in a variety of indications. It's a well-understood mechanism at this point. There's broad biology. There are many indications that people have not even begun to study and many others that are being actively studied now. It's the kind of mechanism that will stack commercially where there'll be a bunch of different indications that can get approved. There are error bars around all of them, but like many of those indications will certainly be nine-figure indications and could be blockbuster indications. Being able to stack those together is a pretty unique opportunity.
I love being involved in stories like that. I think it will stack nicely on top of brepocitinib and sort of be a part of a real and interesting commercial story. In that sense, I guess like what I hope is that we can continue to be close to Immunovant, develop that drug, commercialize it successfully to the extent that opportunities arise that we can own more and that we can be in that position. The only thing I'll say, which I think is clear and everyone knows that we feel this way, is we are not the only people in the world who have noticed that the commercial opportunity in FcRn is exciting. We get lots of inbound conversations and lots of ongoing dialogue with big pharma companies and others about the mechanism. We take all those conversations seriously.
We are, as we showed with the TL1A, ruthlessly economic in how we think about our programs.
Okay. Perfect. And then in the last 30 seconds, the LNP litigation?
In 30 seconds. I believe that Moderna and Pfizer are infringing on our IP. I believe we will have our day in court in the relatively near future. My hope is that that day in court is this fall. I think going to trial will be a good experience for us in terms of what it opens up.
There's going to be a sort of a pretrial hearing summary judgment/Delbare motion next week. Can you comment on that or just what expectations into that?
The thing that I am most interested in in that meeting is I hope that that meeting serves to put some additional timelines around the case, including potentially a timeline. I think it almost certainly will come with a timeline for filing summary judgment motions. I think it very well could, and I hope it does come with a timeline for the trial itself. If we do not like that timeline, we will talk to the judge about it. I think having a timeline is important at this stage.
Yes. Okay. Perfect. Thank you so much, Matt, for hanging out with us. Really excited about the floor here. It's great to see you.
Thank you very much for having me. Appreciate it. Thank you, everybody.