Afternoon. Welcome to the Jefferies Healthcare Conference in London. My name is Dennis Ding, Biotech Analyst here at Jefferies. I have the wonderful pleasure of having the CEO of Roivant here with us, Matt. Welcome.
Thanks. Thanks for having me. I feel like I am everything that is standing between these people and a drink.
That's true. I guess, look, 2025 has been quite a transformational year for the company. A lot of pieces that you guys have set up over the last several years have played out this year positively for the company. Maybe just highlight some of the progress and some of the data that you guys have reported this year that have generated so much excitement.
Yeah, thank you. Yeah, look, it's always fun. Look, a stressful thing about Biotech is you plant seeds, and then it takes a long time to see what's going to come out of them. It's always fun to kind of see it actualize. Roivant is a transformed company relative to earlier this year. For those who don't know us very well, we're about a $14.5 billion market cap now, public biopharma company, mostly focused on developing late-stage drugs that we think should matter for underserved patient populations. That's probably what half the companies on the stage have said today. This year, in particular, our portfolio has moved forward in a really meaningful way, probably across two programs.
The 1st earlier this year is we generated some data in our FCRN franchise that we think helped underscore that we have a potential best-in-class drug with deep IgG suppressions leading to better clinical benefit in myasthenia gravis in CIDP. Then more recently in that franchise as well, showing that we can drive clinical remission in Graves' disease, which we think sets that program up for enormous success. Now, that's probably nonetheless not the main event of the year at this point, because after that, in September, we just put out some phase III data for brepocitinib, our dual inhibitor of TYK2 and JAK1 in Dermatomyositis, which is just one of these great, horrible orphan indications where there's enormous unmet need from these patients who are very sick. It's an inflammatory disease marked by a terrible skin rash and a bad muscle inflammation that leads to wasting.
These patients can't climb stairs, they can't lift things, they can't live their daily lives. The rash is itself debilitating. We demonstrated this is basically, other than IVIG, which has been approved and in use for a long time, this is really the first novel drug to succeed in a Dermatomyositis study. We generated data showing extraordinary benefit, hit every primary and every secondary endpoint across muscle and skin and different parts of the disease, and showed that all while on a background of reducing Steroid burden, which is something you don't actually hear about. Just a transformative moment for us in terms of being able to bring a drug like that to patients, which now we're on a path to registration for. We're really looking forward to that.
Sure. What has the feedback from Doctors been like around brepo?
Yeah, one of the great things about this program is there's so much unmet need. We had Docs literally in tears the day this study came out because these Docs have all been trialists across multiple failed programs in Dermatomyositis. For them, it's such a remarkable moment to see a study finally succeed, to see clinical benefit coming for these patients. Just unbelievable feedback. To be honest, it's one of these things where investors tell me they've done Doc calls, and I immediately know it's going to be a good meeting because the Docs are just like, the Doc Community is so far behind this drug.
Okay. So you guys will file the NDA in the 1st half?
Yeah, that's what we've said. Yeah, really drafting the clinical section is really what's involved at this point, and the team's hard at work at doing just that.
Okay. How should we think about the launch trajectory once you guys get approved? Are there any good analogs that are relevant to brepo that you should point us to?
I'm looking out across the room for my Head of IR and CFO, who I know are sitting in the room, because the answer I'm supposed to give is slow and steady. The launch is going to be slow and steady, gradual. Look, I think the nice thing about this launch is it pattern matches to a lot of the great recent launches in biotech. It's an orphan disease. It's got 40,000 currently treated patients. Some of our competitors say 70,000 from an Epidemiological perspective. It's a good size market. It's going to have a sort of orphan rare disease price point. We haven't obviously decided on price yet, but the other therapies in the category are sort of orphan price point.
We feel like we have an opportunity to learn from Roivant and Madrigal and Horizon and BridgeBio and Argenx and Insmed and so many others, and to try and learn from all of those examples. I think we're doing the best we can to understand what happens in new markets like MG, where companies bring drugs to market in an area of high unmet need without a lot of other drugs. I think that's sort of how we're thinking about trajectory. Obviously, for a new-to-market category, new-to-market indication, we're going to need to educate the Doc base. That part's been going well. We're going to need to sort out market access and payer dynamics. There's a lot of work to do. I think I don't have specific guidance to point to, but I think there's a huge opportunity here at peak penetration.
I think it'd work for us to do in front of us to build to that. The only other thing I'll say is in the best possible sense, and this analogy is not a guarantee of anything, but look, I think there's nothing you could have said about myasthenia gravis in 2019 before the FCRN showed up that you couldn't now say about DM in terms of size, in terms of opportunity, in terms of unmet need, in terms of severity. I feel like there's just some really good analogs in the world for situations where launches like this have gone well.
Okay. Where do you see the most amount of adoption happening once you get launched? Do you see yourself being like a Post-IVIG sort of option or Pre-IVIG? Just give some color there.
Yeah. IVIG, for context for those who aren't super familiar with Dermatomyositis, is the only recent trial that has been successful in DM has been an IVIG study. There are approved IVIGs for use in DM. Among the standard treatment paradigms for IVIG and DM, the probably most common is 40 hours a month of infusions, sometimes spread out over five consecutive days. These are patients who are giving a work week a month to going into Infusion Clinic. IVIG is a tough therapy for these patients. It is only used in about 13% of the DM market now, in part for the reasons I just said. It's just a tough drug to use for that patient population. There is another, call it 11%, who are using off-label stuff, including a lot of things that have failed clinical trials, Remicade, rituximab, et cetera.
I think the short answer is, if you ask Docs, they talk about writing brepo very broadly. They talk about using it in 30%, 40%, 50%, 50%+ of their treated patients, which obviously is a stretch to believe on day zero. I think there's a huge opportunity across the patient population is, I guess, the 1st answer to that question. There's obviously the low-hanging fruit, the severe patients, the refractory patients, the patients who are otherwise considering a course of IVIG or who are on IVIG and don't like it, the patients who are on very high-dose steroids or very high-dose Methotrexate. I think those are all kind of low-hanging fruit patients. The honest answer is, we're an oral therapy for disease where the majority of patients are on oral steroids and immunosuppressants. I think almost every patient is conceivably eligible.
The question is really just where are Docs going to start in terms of trying the drug out.
Right. I think it does help that there is some usage of JAK inhibitors currently in DM just off-label.
Yeah, I totally agree with that. I think the Docs are familiar with the mechanism. Look, DM is treated in the U.S., there's about half the patients, a little bit more than half are treated at specialty myositis referral centers. At those centers, the Docs are split about 50/50 between Dermatologists and Rheumatologists. Obviously, those are both Subspecialties, Rheumatologists especially, but both Subspecialties that are intensely familiar with JAK inhibition as a mechanism. Some of these Docs are pretty heavy users of off-label tocilizumab because, for example, some investigators have run studies at TOFO that have shown reasonably decent clinical benefit. I think we get to benefit from that tailwind. Even the Docs using off-label JAK inhibitors, first of all, our drug is not a pure JAK.
It's a combination of a JAK1 and TYK2, and there are good reasons to believe that TYK2 is contributing meaningfully to clinical benefit. These Docs are just desperate for an on-label option where they can get coverage, where they can get support from a sponsor, where they can do all of these things that allow them to use the drug more reliably and more effectively. I think that absolutely there will be a tailwind from Docs converting off-label TOFA patients to brepocitinib as well. I think that's driven a lot of the Doc familiarity with the mechanism and the Doc enthusiasm here.
Yeah. Okay. I feel like DM is just a start. There's also NIU. You guys also started phase II in cutaneous sarcoid. Should we be expecting additional indications over the next one or two years?
Yeah. I think the short answer to that question for brepocitinib is absolutely yes. Look, this is a great drug. I think take a tiny step back from a historical perspective. We unlicensed this drug from Pfizer in 2021. At the time, JAK inhibitors were on their back foot as a mechanism, right? The study had just come out that showed that they could cause elevated either Cardiometabolic risk or other things. It sort of was not clear where the market was headed. We were like, "Oh, okay, but everyone's throwing the baby out with the bathwater here. We know these are incredibly effective anti-inflammatory drugs." We looked at this sort of orphan disease swimlane as ours. Now, you fast forward to 2025, look, in 2021, Rinvoq was a $3 billion drug, and I think everyone kind of thought it was going to flatline.
In 2025, Rinvoq is an $8 billion drug that's probably on a path to being a $15 billion drug. The market has spoken, and physicians are obviously, in certain categories, very comfortable using these agents. Really, the orphan swimlane for JAK inhibitors is wide open. It's ours to inhabit. It just feels like a really big opportunity. Obviously, we have the program in DM that's now in registration effectively or will be filed soon. We have NIU, which reads out next year, or sorry, reads out in 2027, I should say. We have a phase II study in cutaneous sarcoid that reads out next year. Absolutely, I mean, at some level, any orphan inflammatory disease with 20,000-100,000 patients for which there's high unmet need, not a lot of other approved options, should be viewed as an eligible place for us to go.
You can imagine that we've pretty aggressively canvassed that landscape, especially for places where both JAK1 and TYK2 should play a role. It feels like a really big opportunity.
Yeah. Kind of sounds like Rinvoq for rare disease.
Yeah, I'll take that as an analog. Rinvoq's a good drug.
Okay. If I can ask on NIU, just remind us of some of the phase II data you guys have shown and just what standard of care there is and what's the incremental benefit that brepo has.
NIU, it's an eye inflammatory center for non-infectious uveitis. It's an eye inflammatory disease. It's what it sounds like. It's for non-infectious inflammation of the eye. There's about 400,000 NIU patients in the U.S., of which the majority are front of eye inflammatory patients who are treated with steroid eye drops. About 70,000 of those patients have inflammation in the back of the eye, and you just can't get there with topical therapy. Those patients are treated on Systemic Immunosuppressants and Systemic Anti-inflammatories. What you should be thinking is, Ophthalmologists have very low tolerance for eye inflammation and will do anything they can to get it under control. These patients are on very, very high-dose systemic prednisone, for example, like super high-dose bursts of Systemic Immunosuppressants. The idea there is to get the eye inflammation under control.
Even then, it doesn't always work. Also, for those who have been on a Pred Pack or whatever, imagine doing that, but harder and longer. It's like a miserable experience for these patients. If it doesn't work, these patients can go blind, which is one of the reasons why eye inflammation is so poorly tolerated by Physicians. Standard of care is high-dose immunosuppressants and steroids. HUMIRA is approved. It works okay, not great, and is not very widely used for that reason because there's just not a lot of risk-taking among these Docs. If something doesn't work, they don't want to use it. I think in general, the sort of field is open for a new entrant. You asked about our phase II data. Last year, we generated phase II data in a study.
It was a blinded study, but there was no placebo. It was two different doses of brepocitinib. As we calculated, HUMIRA's treatment failure rate, for example, is about two-thirds, a little bit less than two-thirds. Our treatment failure rate was sub-30%. It was about twice as many patients are succeeding on therapy on our drug as in the HUMIRA studies. That just feels like a remarkable sea change for these patients and something we're really excited to see through. That is now in two registrational studies that will read out, as I said, in 2027.
Perfect. As we think about 2026, can you just outline some of the catalysts that you see through the year?
Yeah, 2026 is a busy year. We have that cutaneous sarcoid study that I mentioned, that proof of concept study for brepocitinib. We have, in FCRN land, multiple studies reading out. We have a proof of concept study in CLE, and we have the 1st period of our rheumatoid arthritis study. We have a large phase II- B study in PH-ILD for mosliciguat, which is our inhaled vasodilator for PH-ILD, which has really promising phase I data in PH patients. That is another really large market where we get to be probably the 1st N on-treprostinil mechanism, which feels really exciting. That data will come next year as well. Those are all the sort of major clinical catalysts. On top of that, in the Moderna litigation, we have the actual jury trial with Moderna that should play out in March.
Just a lot going on in 2026.
Perfect. Which one of those do you think is the most underappreciated from investors?
I think investors are still only really valuing the most sort of front and center indications for each of our programs. I think for Immunovant, it's Graves. I think for brepo, it's DM. Even then, I think the DM opportunity is probably underappreciated today. I think NIU, which granted is not a 2026 event right now, is significantly underappreciated. I think very few people have done real work on it at this stage. I think cutaneous sarcoid is a flyer as far as the world's concerned. I look at some of our competitors in Pulmonary Hypertension. I'm sure Insmed, which is a company we very much aspire to copy in life, has gotten an enormous amount of credit and value out of their PH-ILD, their Treprostinil Program.
I hope a year from now we're sitting on phase II- B data that puts us in that league. I think that could be a huge opportunity. People are starting to ask questions about mosliciguat, but it's really just starting to scratch the surface. In many ways, both mosliciguat and NIU right now feel to me like DM felt, I don't know, a year or 18 months ago. Just a huge opportunity for people to come up on the curve on both of those.
Yeah. I guess maybe we can double-click on mosliciguat a little bit for PH-ILD. I feel like that is quite an important catalyst that is not really in the stock at all. It is a sizable market opportunity too, where you already have pretty promising PVR data in phase II. Just talk about that readout. What does good data look like? Maybe next steps after that.
One of the great things about Pulmonary Hypertension is, look, in Graves' disease, for example, I think there is very much a growing appreciation for how big Graves' disease is as an opportunity. It took education. It is a new indication for people. There is not like an established investor community of Graves' disease investors. Pulmonary Hypertension, if you have worked in the buy-side for more than 10 years, you have made money on Pulmonary Hypertension companies. You have been following this space. You know it well. PAH is a well-developed market with many different categories working successfully in tandem. Several companies have been built and grown. Merck today up meaningfully on data in a related field. This is just like a well-understood area. PH-ILD is a subset of that area.
Was difficult to invest in new frontier up until a couple of years ago when United Therapeutics, one of the real pioneers in PH, demonstrated efficacy with TYVASO, what is now TYVASO with treprostinil, in a way that has created the category. We owe a huge debt of gratitude to those companies. That's an enormous step forward. I think at this point, it's, I think, clear. PH-ILD is a market that looks a little bit like the PAH market, to be honest. It's a large patient population. Pulmonary Hypertension is what it sounds like. It's hypertension in the lung. It's devastating. I mean, these patients die ultimately. It's a really, really bad disease. These are patients who have Pulmonary Hypertension by dint of Interstitial Lung Disease. Things like Idiopathic Pulmonary Fibrosis. For the 1st time, we can treat that Pulmonary Hypertension with TYVASO.
Our view is it's time for that market to go the way of PAH, that is, to have other therapeutic categories, other opportunities to treat these patients, which will be used on top of TYVASO, earlier line, later line, like across the board. Just a huge opportunity for new kinds of therapies in PH-ILD. We think we have a great shot. Now, the way these studies are usually run in phase II is that you study PVRs, like right-hearted blood pressure, which is our primary endpoint, is measured. These patients, you can tell how sick they are because they're doing this for clinical trial under General Anesthesia through a Heart Catheter. You measure that and you show that you are improving blood pressure in the relevant vessels. Ultimately, the clinical endpoint in a registrational study will be either six-minute walk or time to clinical worsening.
is a measure of clinical benefit for the patient. In this study, the primary endpoint is PVR. Based on what we have seen in PAH in group one patients, I expect we will hopefully see meaningful reductions in PVR in this phase II- B study. What we are really looking for is how that translates into six-minute walk or time to clinical worsening in terms of seeing clinical benefit for these patients. I think statistic is obviously nice, but just seeing a real benefit for these patients will matter a lot.
Okay. In terms of PVR, I believe the phase II data in PAH had around 37-38%.
That's exactly right. Yep. Some of the deepest, I think the deepest PVR reductions ever observed in a clinical program.
Yeah. In terms of placebo, there's really sort of minimal improvement in placebo.
Yeah. You would not see a lot of PVR. Again, this is six-minute walk is a difficult endpoint. It is a measure of how far people can walk in six minutes. Obviously, there are a lot of things that drive that. When you are anesthetized on an Operating Room with a Catheter in, there is not that much that is going to drive a placebo response on blood pressure. You do not see that much placebo response on PVR.
Okay. 38% is pretty much 38%. That's a good number. I guess talk about the translatability.
That's really the question. The whole question is the 38% is in patients with Pulmonary Arterial Hypertension. Those are different Etiology of patients than the PH-ILD patient population. You just do not know for sure exactly how that will translate. The truth is only treprostinils have really been studied in both. What do we know? We know that treprostinils showed good PVR reductions in PAH and also good PVR reductions in PH-ILD, and that translated to clinical benefit when they were inhaled. We know that Systemic Vasodilators, both treprostinils and actually our drug, I do not think I even said this, is an SGC activator. It is a different mechanism. There in the past has been a similar drug to ours, an SGC stimulator studied systemically in PH-ILD. Like systemic treprostinil, it is not very good in PH-ILD.
It turns out in Pulmonary Hypertension patients with lung disease, Systemic Vasodilators have an issue, which is that while you treat the healthy lung tissue well, you wind up vasodilating the diseased lung tissue as well. You end up basically giving back all or sometimes even more than all of the benefit at the same time. What you really need is an inhaled targeted vasodilator that only gets into the healthy lung tissue and drives better like TGMP production and oxygenation in the healthy tissue. I think that translatability, we know that Inhaled Vasodilators work in PH-ILD in general, and we've seen that mostly with inhaled treprostinil. Not a huge N, but that's what gives us confidence to try.
Yeah. Okay. In PAH, I believe anything north of 20% PVR is considered pretty positive. Do you think that's also a similar, I guess, I don't want to call it a bar, but for PH-ILD?
As a Biotech CEO, I've come to hate the word bar. Look, I think the answer is if anything in PH-ILD, it's a little bit of a lower number because these patients have lower baseline PVRs generally. Whatever. In PAH studies, the baseline are like whatever, maybe nine or 10 Woods units, whereas in PH-ILD, it's maybe more like 7. I think even a somewhat lower number than 20 would be good. Again, 38 is much higher than 20. My hope is that we're not coming close here either.
Okay. Got it. That's super helpful. Another 2026 catalyst is the Immunovant RA data.
Yeah.
Talk a little bit about that. I believe it's an open label readout in 2026.
That's right.
Just help us understand the interpretability of that data and what do you consider to be positive.
Yeah, perfect. Look, RA was always a stretch indication for an FCRN, right? RA is clearly not a Vanilla Autoantibody-driven disease. It's multifactorial. There's a bunch of different reasons that people get RA. What we know is that in some RA patients, there are significant IgG Autoantibodies. We know that in early line patients that J&J with nipocalimab was able to show clinical benefit, modest, but meaningful clinical benefit, that that clinical benefit was higher and more interesting if they looked only at the Autoantibody-positive, ACPA-positive patient population. They then ran a different study. They ran a study combining an FCRN with a TNF, which was a tough study for them. It didn't pan out the way they wanted it to.
We've decided to go a different direction, which is to study ours in a late line RA patient population, sort of 4th line, difficult to treat RA, where the bar for clinical meaningfulness is lower. These patients don't have a lot of options. Where we're focused specifically on the ACPA-positive, the Autoantibody-positive patient population with the hope that we can make a difference. The study is a randomized withdrawal design. It's a phase II- B. We, one of two pivotal programs if successful. The readout next year is the sort of run-in period, if you will. All of the patients are on drug, and then responders will be randomly taken off, hence randomized withdrawal. Look, what we're going to get is an open label response rate, which means the bar for us being excited and starting that 2nd phase III is high.
If the, whatever, if the ACR20s are low, it's not going to be that exciting. If we see like a really meaningful response rate where Docs are excited and patients are excited, I think that's the kind of thing that gets us moving forward. I don't have like a numerical number, but I think the answer is because it's open label, because it's RA, the bar is high. This is really, although it is structured as one of two pivotals, it's really a signal-finding study.
Got it. Got it. I guess that kind of answers my next question, which was just the rationale behind designing a trial that way. I guess it's to get a signal before it's submitting.
It's to get a signal fast, exactly. I think the idea is that there's very patient-friendly design, right? Like you get everyone's on drug at the beginning. This has been among the easiest studies that we are running to enroll.
Perfect. I think we have two more minutes, but would love to pick your brain about BD. Just thoughts on the landscape and what you're seeing. I feel like it's been a little bit over a year or two just since your latest announcement. What are you seeing out there? What are you most interested in?
Yeah. Look, I think one of the most interesting things about the moment that we're in as a company is we don't, I don't mean that we don't need BD, right? I think like the thing that carries us from here to a $30 billion or $40 billion or even $50 billion market cap is our existing pipeline. It's launching well in DM. It's launching well in NIU. It's stacking these things together. I think brepo could be a large, large drug. I think FCRN could be a large, large drug, large, large category for us. Like these are big opportunities. The don't mess it up bar or imperative is high. I think therefore correspondingly the opportunity to be, look, we are historically a Transactional company. We have at this very conference people running around meeting with pharma companies, talking through ideas.
There's lots of things we'd like, but I think the bar for actually transacting is high because we don't want anything that's going to distract us from the sort of core job of making these existing programs successful. Bluntly, we want things that if we become a $30 billion or $40 billion or $50 billion company are going to be needle-moving, are going to be sufficiently important that they add a real leg to the stool. That said, we absolutely 100% see things that meet that description within the portfolios of Big Pharma companies. Some of those things we have been excited about at this point for two years.
We have been doing the slow, steady, important work of bringing our would-be partners along and helping them get excited about us as a partner, helping them get excited about the opportunity, seeing their own businesses evolve, seeing the need on their end evolve. I think, look, a big part of our business is sitting around outside Big Pharma companies like a puppy waiting for just the right moment to transact. I think we continue to focus on that kind of activity because we see a lot of opportunity to be a good, effective partner to big pharma companies.
Got it. I think that's all the time that we have. Thank you so much for hanging out with us. Excited for 2026 and hope you get that drink.
Thank you. Yeah, I hope you all get that drink.