Roivant Sciences Ltd. (ROIV) Earnings Call Transcript & Summary

Welcome, everyone, to the 40th Annual JPMorgan Healthcare Conference. My name is Tessa Romero. I'm one of the senior biotech analysts here at JPMorgan. I'm joined by Taylor Hanley from the team. Our next presenting company is Roivant. And presenting on behalf of the company, we have CEO, Matthew Gline. Before I turn it over to Matthew, I just wanted to highlight to all of our listeners to please use the ask a question feature in the portal to ask any of your questions, and I'll get it in on your behalf. With that, I'd like to hand it over to you, Matthew.

Thank you. Appreciate it, and good afternoon, everybody. Thank you for having me at the conference. I'm going to start today with a presentation outlining a little bit about Roivant and some of the things we're working on, and then we'll end with some time for Q&A. So thank you very much. I think the slides are self advanced, so I'll call out slide numbers as I go. Starting with on Slide 2, I'd like to remind everybody that I'm going to be making forward-looking statements as part of today's presentation, including about our financial conditions and product candidates, and you can see more information about that on Slide 2. So I'm going to begin, and I'll start on Slide 3, with a sort of 30,000 foot view, just a little bit about Roivant and who we are for those that may be less familiar with the company. And then I'll get into some of our current projects and some recent updates as well. So as a reminder, for those that are new to the company, Roivant was born in 2014, not really with the goal of being a single product biotech company or a single platform, even biotech company. but rather to rethink the big pharma model from end to end, and we've really planted 2 stakes in the ground in particular, in how we plan to do that differentially. The first of them is something that people were familiar with the company will know from the outside is our Vant model, shown schematically at left here, which is that we believe that biotech R&D is best conducted, and we believe this has been shown over a long period at this point at small innovative entrepreneurial biotech companies. And so rather than building a single command and control R&D organization, we've built a family of small, nimble, independent entrepreneurial biotech companies that we call Vants, each with their own leadership team. Each with their leadership team incentivized for the programs at that Vant and each focusing on specific therapeutic candidates or technologies in a narrow area. We built that family together because we believe there are benefits to scale that come from combining these companies and that we can get the best of both worlds by providing the incentive alignment and the focus of the Vant, while providing the access to analytics and access to other benefits as a part of being a part of the family. The other stake that we've planted in the ground is that we've been focused on the development of novel computational tools, the use of software and data and analytics to improve our ability to identify opportunities, discover new drugs, develop drugs and ultimately, to commercialize them. And this ranges from technology that I'd call on a more boarding end, like moving data from point A to point B in ways that allow us to run clinical trials faster or better identify and understand patient populations, to sophisticated technology like computation molecular dynamics and machine learning that help us design new molecules and advance programs using creative development strategy. So a wide variety of tools that add up to, we think, a differential benefit to each of our Vants. And so Roivant at the center, in some ways, feels like a technology company with engineers building tools that we then make available to the Vants in the family. So -- On Page 4, I'll just give a brief overview of some of the things that excite me about the business today. So starting with -- at the top of this list, we have a near-term commercial launch of a product that we believe has the potential to be a multiple blockbuster. It's a topical therapy for psoriasis with an expected approval from FDA. We expect the decision from FDA in the second quarter of this year and a launch shortly thereafter and is an ongoing trial in atopic dermatitis. So we'll talk more about that program shortly. We also have a broad differentiated pipeline of clinical stage programs, including some such as betoclimab or anti-FcRn antibody at Immunovant, which is a public company, that people may be familiar with as well as 1 program that I'll talk about for the first time publicly today, RVT-2001, a potential first-in-class oral SF3B1 modulator, which we're developing for transfusion-dependent anemia in patients with lower-risk myelodysplastic syndrome. In addition to that, we have several sources of asymmetric potential upside even above and beyond our clinical portfolio. That includes an intellectual property estate at Genevant that has been the focus of recent attention that we'll talk a little bit about later in the presentation, as well as upside from preclinical data across a range of therapeutic areas. Notably, and we've talked about this in other presentations, that preclinical data in part is generated from our -- what we believe to be a leading computational drug discovery platform with proprietary tools, both for atom by atom, molecular dynamic simulations and machine learning, tightly integrated with unique wet lab capabilities and biophysical capabilities to give us the ability to discover new drugs against difficult targets particularly in the field of targeted protein degradation. And finally, we're privileged with a strong capital position with $2.5 billion in cash as of September 30, a significant portfolio of public equity securing in Immunovant and other public companies. and private holdings that include a 12% stake in a tech company called Datavant that we built, that I'll talk more about in a moment. On Page 5, I want to highlight as sort of the last high level point here, a little bit about our track record. And so if you want to build a big pharma company, it turns out there's no substitute for developing drugs. We've put over 40 medicines into development and have run 9 pivotal trials in our history, of which 8 of them have been positive, and we lay out those trials here. Those have resulted in now 4 FDA-approved products. All of them advanced that we sold to Sumitomo Dainippon. They're now owned by Sumitovant, a collaboration that we have with Sumitomo Dainippon where those products have been approved and in that collaboration from back in 2019, we took in $3 billion of upfront cash that we've now been able to put into research into our next stage of development candidates, including tapinarof, which is the psoriases drug that I'll talk about soon. Tapinarof is now under review by FDA for approval. We also have a strong financial track record. I mentioned that $3 billion transaction with Sumitomo Dainippon that has really put us in a strong capital position. I mentioned the $2.5 billion in cash. We also did a deal last year where we took a tech company that we have built. We think about our technology, the computational tools also as commercial opportunities, and we have built a company called Datavant that helps us better understand patient populations. It allows for the linking of de-Identified data across siloed health care data sources in the U.S. We merged that company into another health data company, a company called Ciox Health in a transaction that preserves our strategic access to the tool, but also gave us a $320 million cash upfront payment and a 12% equity stake in the new company still called Datavant. So we're excited about that track record excited about some of the things we've been able to do in the past. But I'd like to spend the remaining of our time today talking about some things that we're excited about for the future. So the first of those things, which we've talked about a little bit publicly before, but just as a as a refresher with an update, starting on Page 7. I want to talk a little bit about tapinarof, which is our furthest along program. It is, as I've mentioned, a topical agent for the treatment of psoriasis. It's an aryl hydrocarbon modulating agent, and it is the only such agents that we are aware of in clinical development. Tapinarof, in short, as I've mentioned, we expect to be a potential multi-blockbuster. And really that to us comes down to the 5 major attributes of the product, an extraordinary treatment effect of a kind, frankly, never before seen in a topical agent. With durability studied in our combination of Phase III studies that included a 52-week extension study that showed improved treatment effect and continue to use beyond 12 weeks. A remittive benefit that, again, is something we've never seen before in a topical. I'll highlight this data again in a moment. But over 40% of the patients in our trial achieved a PGA score of 0, completely cleared their psoriasis. And consistently in our long-term extension study, we saw that those patients when we took them off therapy remained clear or mostly clear for an average or median of 4 months, which is an extraordinary relative benefit again something that hadn't been seen with the topical. And this is combined with a safety tolerability profile that are also differentiated versus the standard of care. In fact, tapinarof is more tolerable than many of the existing corticosteroids with no duration of use to limit and with our -- in our study was studied across the entire body surface with no limitation. So unlike a corticosteroid, so I'm a psoriasis patients, so I live this reality, when you walk out of a dermatologist office,with a prescription for a corticosteroid, which are the current mainstay of therapy for psoriasis, you can only use it if it's a potent corticosteroids for a few weeks at a time because of skin thinning. You can only use the most potent ones on certain parts of your body because of tolerability issues with potent corticosteroids. Tapinarof as neither of those limitations. And so it is both a more efficacious and more tolerable therapy than the topical corticosteroid. So it has the ability in our view to be an important first-line therapy, really, the mainstay of future therapy for psoriasis patients as well as to offer a real chronic option for patients to keep them off systemic therapy and biologics, which otherwise have been the next path for these patients over the past years as there's been limited innovation in topical therapy. I'll talk a little bit on Page 8 and 9, again, about our data, starting on Page 8. Was just a reminder that we have this effect that a significant percentage of our patients achieved a PGA of 0, 40.9% overall in the study, including about 43% who entered the long-term extension study on tapinarof achieved a PGA score of 0, completely cleared their psoriasis, which is a level of disease clearance that we are not aware of ever having been shown in a topical therapy before. And then on Page 9, as a reminder, and I mentioned this on the prior slide as well, we see a remittive benefit for those patients where they spend 115 days on median or 130 days on mean in the larger group that achieved the PGA in the extension study of disease clearance before they have a recurrence which is significantly longer than with corticosteroids, where you see generally rapid flare ups shortly after patients go off therapy. So that's really differentiated treatment option. On Page 10, I'll just sort of quickly recap some recent updates on the program. First of all, as we get closer to the PDUFA date, we sometimes get questions. In short, we believe the NDA submission for inter-offin psoriasis is completely on track. We have no expectation of an advisory committee, and we expect the PDUFA date in the second quarter of this year. We feel completely ready from a manufacturing and commercial production standpoint with the team at tapinarof -- with the team at Dermavant on track to ensure supply available for the launch of the drug, which we expect to happen shortly after the PDUFA. We are not waiting to build our commercial organization until after the approval. So we are begun to make the relevant hires already, and the Dermavant team expects them to be fully set up. And by the way, I'll pause there and just say one of the things about the Vant model that is at play with tapinarof is we have a truly extraordinary and capable team Dermavant working on developing and launching this product, including a management team that has deep expertise in topical therapy really deep understanding of the field. And that's something that the Vant model allows for that makes us confident and excited going into this launch. And we also had a milestone recently which is the data from our main Phase III study PSOARING 1 and 2 was published in the New England Journal. And finally, although we're not talking about it today, we continue to enroll patients in ADORING 1 and 2, which are atopic dermatitis trials for the intro with top line data expected in the first half of 2023. And we think tapinarof has the potential to be an important medicine in atopic dermatitis as well where there has been a similar lack of innovation in topical therapy, including nothing that can be used chronically in the way that tapinarof currently has data in psoriasis. And we have Phase II data and atopic dermatitis supporting that effect. So tapinarof is a drug that has been in our portfolio for a while, and so we've talked about it publicly before. I want to spend a little bit of time today now talking about a new program for us. And if you look at Page 12, you can see our entire development stage pipeline here with tapinarof as I mentioned at the top. A number of other therapies, and a reminder that we typically develop therapies that span modalities, span therapeutic areas. So we have antibodies. We have lentiviral gene therapy. We have topical agents. And we have small molecules. And 1 such small molecule, RVT-2001, I want to spend minutes on today, is a new therapy that will stand as the basis of a new Vant called Kinevant. So I want to just take a minute on Page 13 and remind people of a feature of how Roivant thinks about programs. And I've got a subset of the programs that we've acquired over time on this list. We've always built our pipeline in part through when licensing. We have the basic research facility. We're working on discovery, especially target proacintegrators, but where we can acquire programs in indications and with mechanisms of action that we're excited about, we always like to in-license programs and acquire programs that are in the clinic, where we have data that we can wrap our heads around. And 1 of the things that we pride ourselves on, we are in an era of extraordinarily expensive dealmaking in biotech. And sometimes, we have to pay up for therapy, and we've done so. But we proud ourselves on being highly different in making deals for in-licensing. And you can see on this list, a number of the deals, including some of the medicines that we'll talk about today. And you can see down at the bottom, 2 new acquisitions listed here. Hemavant RVT-2001, which we'll talk about today, and Priovant, which is a to be announced therapy that we've acquired that we're developing for severe autoimmune disease. We will talk more about that in the coming months. But you can see both of these deals, again, very capital efficient, in the way that we've acquired the therapy. In the case of RVT-2001, a $15 million total upfront, almost half of which in woven equity in the case of the Priovant therapies. $10 million upfront is a minority equity stake in the new Vant. In all cases, we like to risk share with our partners. We like them to come in and benefit with us if we are successful and we like to put most of our capital towards clinical development. And certainly, I feel like with both of these new therapies, we've achieved that. So that's definitely something we're proud of something we expect to continue to do and something that should be thought of as the context for these programs. So on Page 15, I'd like to introduce RVT-2001. So as I said at the beginning of today's call, it's a potential first-in-class small molecule SF3B1 modulator, which we're developing for the treatment of transfusion-dependent anemia in patients with lower-risk myelodysplastic syndrome. I think if you're not familiar with that indication, so MDS is a form of blood cancer that is severe and difficult. And in general, patients with MDS are stratified into higher risk and lower risk patients, with higher-risk patients ultimately having progressive disease that needs different treatment options, but with about 2/3 of patients, lower-risk patients, who wind up with chronic disease that needs treatment. And in particular, these patients wind up dependent in many cases, on red blood cell transfusions to treat very severe anemia. And so it's a very difficult disease for those patients. And there are not good therapies or good options apart from those transfusions, which winds up being difficult and painful and challenging. So recently, in 2020, a BMS drug called luspatercept was approved for the treatment of lower-risk MDS for transfusion independence. As you may know, with that drug, it's been quite successful. It's annualizing at greater than $500 million, 5 quarters at launch. And BMS launch and BMS has signal of potential peak sales of greater than $4 billion. So it's a market that is validated and that we think is important. We have encouraging proof-of-concept data that I will talk about a little bit later in this presentation and a multipronged approach to optimize our development strategy. On Page 16, just to situate RVT-2001 in our treating the treatment landscape for lower-risk MDS. For these patients with transfusion-dependent anemia and lower-risk MDS, there are some stratifications based on various markers lenalidomide is approved for the treatment of a subset, about 10% to 15% of these patients. And it works okay in those patients, but lenalidomide has significant toxicity, so it's limited to that sort of 5q minus patient population. But otherwise, patients first grow through ESAs as first-line therapy. And then currently, the only available option really is luspatercept, that recently approved medicine. We believe RVT-2001 can provide a new option both as second-line therapy alongside luspatercept for the patient population in question as well as potentially for therapy for luspatercept refractory patients. And so it's a big potential addition to the treatment landscape and something that we think should matter especially because luspatercept is only has responder rates in the 40%. And so there's many patients who don't respond to luspatercept. And luspatercept is most effective in patients with an already low transfusion burden. I think on Page 17, we lay out our thesis for this program and why we're excited about it. And I'll start by saying, look, this is a relatively high risk program in the sense that our data to date is based on relatively small studies, especially in the patient population of interest to us. And our ability to evaluate that data is in part drawn from cross-trial comparisons with other medicines. But we've seen something really exciting here which is that -- so in the lower risk transfusion-dependent MDS patients, of which out of the 80 patients studied here, 19 of them were lower risk transfusion-dependent MDS patients. 15 of them were heavily pretreated patients. They have been pretreated with lenalidomide and/or HMAs. And that's important because we saw transfusion independence rate in that study in that patient population of greater than 30% in our Phase I/II study, which isn't so remarkable overall but actually in that refractory treatment population in a heavily pretreated population. Other therapies have tumbled significantly more. So for example, luspatercept has shown a rate of 13% transfusion independence among patients with prior lenalidomide exposure in its Phase II trial. And lenalidomide itself, HIE is not actually a transfusion independence endpoint. It's something more mild than that. But only 12% of patients in the lenalidomide trial with prior HMA exposure in an investigator sponsored study achieve that sort of reduction in transfusions associated with the HIE endpoint. So those are both significantly lower rates in those patient populations than our admittedly somewhat small end but significantly higher responder rate of greater than 30% in our study in those pretreated patient populations. And that's against a backdrop across the 84 patient treatments treated a relatively well-tolerated therapy with the majority of events classified as Grade 1 and there's a significant need here. Now in addition to being excited about our potential to deliver better response rates in heavily pretreated population, which if this study continues to show the same data we would be able to achieve, there's good there's good precedent for significant improvement when you move these therapies into earlier line patients. And so for example, that 13% for luspatercept among patients who have been pretreated with lenalidomide, when you go to patients without prior lenalidomide exposure, to earlier line patients, it increased to 44% in their Phase III trial -- in their Phase II trial. And luspatercept Phase III trial actually excluded prior lenalidomide exposure because they have seen significantly reduced transfusion independence rates in lenalidomide exposed patients in their Phase II. And then in that same lenalidomide investigative [indiscernible] trial that I mentioned before, the 12% post HMA response rate increased to 38% in the HIE endpoint prior to HMA therapy. So in both cases, you saw significant improvements in response rate once you move from an earlier line patients. We expect to enroll earlier-line patients in RVT-2001 Phase I and II trial who have been more responsive. And if we see improvements of the same kind, we have a potential for an overall best-in-category therapy for the patients. And so we think it's a pretty exciting data and we're set to run that Phase I/II study. We expect to enroll 50 to 60 additional patients, and we expect data in 2023 -- early 2023. Now there's a couple of other things on Page 18 that we're doing to improve our analysis of those patients. One of them is that we are specifically selectively enrolling lower-risk patients with SF3B1 mutations, which about 30% of the MDS patient population. Obviously, there's good biological rationale there, given that we are an SF3B1 modulator. And then also -- and here's some additional exciting data. In the subset of patients who have this TMEM14C -- TMEM14C transcripts, which is a biomarker associated with SF3B1 mutation, we have 7 patients of which 5 were responders. So a 71% responder rate for transfusion independence. We're also expanding the data set specifically in that biomarker population with the hope that, that's an additional potential path for us to value -- demonstrating a higher rate even in that subset of the population. But we're also optimizing dosage. We think there's a possibility to extend the pharmacodynamic effect by optimizing dosage of VITI-201 in this Phase I/II study. And finally, the only other point that I would make here is, well, I've characterized this study is higher risk and as we go from smaller end to larger end, it's important that these effects hold. What we have generally seen is that if we demonstrate these sorts of effects in our overall Phase I/II study, that there's generally minimal data decade between Phase II and Phase III and other MDS therapies. So we believe that if next year, we're able to replicate some of these effects, that should put us in a good position in terms of our ability to run a pivotal trial starting thereafter. So thank you. That's what I've got on this new RVT-2001 therapy. And we're excited to continue to provide updates as that trial gets up and running. So I'll next very briefly review some updates from a few other Vants, noting that I'm not going to spend as much time on this. Starting on Page 20 with an update that Immunovant provided last week. And again, Immunovant is a public company speaking later in the conference. But critically and for those that have been following the story, Immunovant has now announced that we've reached alignment with FDA to move forward with a pivotal program Myasthenia Gravis that is expected to start in the first half of calendar year 2022 as well as additional pivotal studies in 2 other indications, including possibly TED, WAIHA or 2 new indications that we expect to announce by August 2022. And our Phase III trial, which I'll talk a little bit more about, is specifically designed to address unmet patient need by leveraging the broad window and subcutaneous delivery that batoclimab offers. So I won't spend a lot of time on this, but maybe if you just look at Page 22 briefly. I think one of the things that differentiates our approach with batoclimab from the other antisera agents in development is our Phase III trial in MG is designed to treat patients the way that other autoimmune therapies are often administered with induction, maintenance and ongoing long-term therapy. And so we have an induction phase where we're dosing with a higher dose, 340 or 680 milligrams, on a weekly basis, followed by a maintenance phase where we put patients on either 340 or 340 every other week dosing and then a long-term extension phase during which patients have the ability to titrate up or down based on their need. So this is -- mirrors the way that autoimmune disease therapy works in other indications, and it's something we feel is uniquely enabled by the batoclimab product attributes. And on Page 23, I just laid this out versus a couple of the other anti-FcRn agents that are really the others in late-stage clinical development, including [indiscernible] and nipocalimab, both of which in various ways are not able to administer and for the same framework. So [indiscernible], which is currently an IV therapy that was approved last month. It is really a sort of symptomatic exacerbation approach where it's treated with sort of cyclical therapy, 4 weeks on, followed by 4 weeks off. so not with this sort of induction in an ongoing maintenance approach. Nipocalimab has done more in the direction of an ongoing maintenance approach with a single loading dose followed by an extended period at a relatively lower dose for 22 weeks. Again, currently studying IV administration. And only we have this sort of full induction maintenance in rescue paradigm, including with fully subcutaneous administrations. So we're excited about that. We believe we have a protocol, and Immunovant has talked more about this that properly manages for and addresses any concerns about the cholesterol elevations that we've seen with this drug. We have exclusion criteria that keep a small percentage of the very most at-risk cardiovascular patients out of the trial, and we're excited to get that program underway. And on Page 24, as a reminder, that's not relevant just for myasthenia gravis, but it's relevant across the portfolio of potential autoimmune diseases addressable via FcRn, including multiple pivotal studies and proof-of-concept indication and proof-of-concept studies across multiple indications, and we believe the same attributes that allow us to take a differentiated approach to use of Nipocalimab in MG will allow us to take a differentiator both in some of the other indications. So I'm not going to talk about other Vants today, but I'll just briefly pull up on Slide 28, a few slides at a time. I'm going to skip [indiscernible] in the interest of time. Just to highlight that 2022 was really a packed year for us in terms of growth and advancements in our pipeline with multiple studies expected, including 3 pivotal initiations of batoclimab, including a Phase III study in namilumab for sarcoidosis at Kinevant, where the IND has been accepted as of last month. By that -- we're on track to initiate a multiple ascending dose trial of our lysine based anti-effective at lysovant with an IND submitted last one, and then the robust open-label expansion of the ongoing Phase I/II trial in RB211 that I mentioned before, among other trials. So a really exciting year for the development of our clinical pipeline. The last thing that I'll spend time on today, and I'll only spend a brief moment on it, is on Pages 30 and 31, which is to highlight the other developments that many have been paying attention to in our company. which is that we have a portfolio of intellectual property related to the use of lipid nanoparticles at Genevant. So Genevant is a scientific leader in the field of liponanoparticles with scientists who have been working on this technology for a very long time. We are some of the earliest people who have worked on lipid nanoparticles and the innovative scientific collaborations, including with folks like Sarepta and Takeda and novel uses for LNP. And we're excited about those scientific collaborations. As a part of that, we also have a quite broad IP estate related to lipid nanoparticles. You can see some of the key patents on Slide 30 that cover a wide range of attributes of LNPs, including the molar ratios of liponanoparticles that are often useful in therapeutic applications and vaccine applications as well as the morphology of various lipid nanoparticle formulations and in particular, formulations using mRNA and LNP together. The reason this has been an area of focus is because earlier in 2018, Moderna filed IPRs to invalidate several of these patents prior to COVID. And recently, as of last month, the Federal Circuit affirmed the validity of these patents rejecting the earnest appeal. And so we feel that IP estate has been earned. We are excited about what that means for the patent portfolio, and we think there's some upside to us potentially in the continued development of this IP estate and the continued growth into novel LNP. So it's an area to keep an eye on. There has been some confusion, and so I'll just sort of, on Page 31, highlight. I think the easiest way to think about this is via our ownership stakes in Genevant -- We retain a 76% exposure on a basic basis or 62% on a fully diluted basis and an economic interest in any royalties or otherwise derived from that patent estate. So that's the sort of Roivant exposure considering all the different pieces. So I'm not going to spend time today talking about the recovery engine. We'll have more opportunity to do that in the future, and we've done so, including at our R&D Day last year. So I'll just end here on Page 35 with a reminder that we really do have a catalyst packed 2022 ahead with FDA approval decision for tapinarof coming up as well as top line data for tapinarof and our Phase III atopic dermatitis studies in the first half of next year, multiple trial initiations and some additional information coming at Immunovant. The initiation of our Phase III study at Alyvant as well as continued information from patients progressing in our Phase I/II trial in Alyvant and sickle cell disease this year, initiation of our program in sarcoid lysovant expansion and continued progress in MDS, as I discussed, and Phase I initiation for our first greater candidate as well as multiple preclinical programs across [indiscernible] and proteovant producing data that we should be able to share from a pre-clinical perspective and entering IND-enabling study. So a number of exciting catalysts for the business. It's an exciting moment and a fair amount of content to cover in a short call, but I want to thank you for taking the time to listen to it, and I want to call it back to Tessa, who I think is going to take some Q&A.

Yes. Thanks so much for that. So my first question is really around launch preparedness activities for tapinarof. What sort of prior authorization or step edits are you expecting to put in place? And what are you thinking in terms of a pricing strategy?

Yes. So that's a great question, and pricing is one that we get from time to time. The way that I think about access for tapinarof is a couple of things. First of all, as a therapy that has the potential to be both more efficacious and more tolerable than the standard of care, you would expect that patients and physicians would be excited to use the drug. And it's a category that is a little bit complicated and has been very challenging from a payer perspective because the systemic therapies like the biologics are very expensive, so they're priced quite high. So first of all, there's a very wide range in an innovation gap right now between highly priced systemic therapies like SKYRIZI or whatever, which are in tens of thousands of dollars a year or more, or OTEZLA, which is $3,500 a month. And then on the lower end of the spectrum, the corticosteroids that are generic and often inexpensive. So there's a very wide band of possible pricing outcomes, and we haven't given guidance on price. The interesting thing about step edits in this space is it's been incredibly difficult for payers to control access to systemic therapy because the corticosteroids can only be used for a short period. And so even when the payers have put in place multiple rounds of step edits to try and defer patients from systemic therapy, patients can fail all of the corticosteroids available over the course of a few months, and most patients have already failed all of them. And so it's not difficult it's not difficult to get through step edits. So we haven't said whether we expect step edits and I think we have a value proposition to payers that should be really attractive to them and should help make the class sort of easier from an access perspective. But to be candid, systemic therapies haven't struggled except at its before. So I'm not sure that's exactly the way that we think about the category. And then from a pricing perspective, we haven't said, and I think we have a wide range of possibilities given the given the profiles of the molecule, we're going to watch closely what's going on with some of the other topical therapies and especially [indiscernible] in atopic dermatitis with insight launched recently, where they set a higher price. And we're sort of watching what that access picture looks like, and I think we'll be able to provide guidance on pricing as we get closer to the launch.

Great. And Matthew, you talked a little bit about the LNP patent estate in your prepared comments. We did get a portal question that was around kind of recent wins that you've had on the patent appeal side, how you're thinking about potential next steps as it relates to asserting potential infringement on these patents by COVID-19 vaccine makers. Any timing on timing on when we could hear next steps?

Yes, thank you. So I can't comment publicly on any action of any kind of infringement. But I'll say we were pleased by the outcome, obviously, in the appeals court, which affirmed the validity of the patents. And I think we're sort of watching for that process to work its way through. And even after the appeal -- even after the [indiscernible] at the beginning of December, there's sort of a couple of month tail to how that process plays out. So I would say until that's done, I wouldn't expect that we'll provide an update. But afterwards, we may be able to say more about our feelings.

Okay. Great. That's helpful. And then you touched a little bit on the Myasthenia Gravis study. What are the key endpoints in bar for success for this study? And I think there was -- yes, go ahead. Go ahead.

Go ahead, please.

I guess the second part of the question was just around safety and monitoring LDL elevations in that study.

Yes. Yes, perfect. So Immunovant has commented on this in their presentation last week, and I'm sure we'll comment on it again in their presentation later at this conference. In terms of endpoints and sort of the bar for success in MG, obviously, the only approved FCR anti-FcRn antibody at this point is [indiscernible] from Argenx which got approved last month. And that obviously sets an important bar in terms of how people think about it. The main endpoint that people look at is MG-ADL and how these therapies perform against that bar. The Argenx study, which led to the approval of [indiscernible] studied this intermittent dosing paradigm. And frankly, one of the things that we think is exciting about IMVT-1401, about batoclimab is that we are able to deliver a very high level of IgG suppression, above 70% in some of our studies at higher doses. And so we think we have the ability for patients that need it to get very consistent IgG suppression, which we do think will translate into a high level of efficacy. So we think that's an important measure and it's something that we're focused on. In terms of cholesterol monitoring. So again, I think Immunovant's comments on this are the most important. But in general, we feel very comfortable with the cholesterol position. We had added exclusion criteria -- Immunovant's added exclusion criteria in the study, especially for patients with LDL levels above 190, which should exclude somewhere between 5% and 10% of the patient population that have quite high levels of LDL. If those patients are controlled on a statin and their LDL comes lower, then they could be entered into the trial. But patients with LDL levels above 190 or patients who have active cardiovascular disease and LDL above 160 are excluded from the trial. Other than that, we expect physicians to monitor and treat LDL increases, especially during the long-term extension as they might otherwise, patients are not allowed to initiate a new statin during the induction or maintenance phases of the trial, although they are allowed to be on a statin if they were on a statin at the time they were entered into the trial.

Okay. I guess I'm sort of hopping around the different Vants here. But you touched a little bit on the ARU-1801 program. Maybe you can just hit on for us when we could expect to see data from this program?

Yes. So...

And anything you'd highlight on the potential for differentiation?

Sure. So we continue to enroll patients in our ongoing Phase I/II study there. And as has been our practice, we will continue to report data on those patients sort of as it comes in. So I think there will be more data including time updates on our existing patients as well as information about new patients as we dose them throughout this year. We also -- and by the way, there's data on this, on Slides 26 and 27, in the presentation materials. I'd say on differentiation, and this is a point that's incredibly important, one of the interesting things about the sickle cell disease treatment landscape right now is that genetic medicines in sickle cell disease, the bar really is curative clinical efficacy. And it's something that Bluebird has shown, it's something that [ CRISPR ] shown and it's something that we, after our process development work have shown in ARU- 1801. And I think a number of genetic medicines, both gene therapies and CRISPR-based gene editing approaches will be able to achieve curative efficacy. The challenge, and it's laid out on Page 27 of the presentation is all of the existing clinical development candidates in this area, use very high intensive myeloablative preconditioning, mostly busulfin-based regimens. And these patients, they spend literally months in some cases in the hospital. These are young patients and women, for example, on busulfan. This is not toxicity associated with the drug. It's toxicity associated with the preconditioning. Women on the preconditioning regimens have 70% to 80% risk of infertility or ovarian failure. There's long neutropenia recovery times. These are very, very difficult preconditioning regimens. We are the only clinical stage genetic medicine for sickle cell disease and the only near-term therapy for sickle cell disease that has shown clinical benefit using a reduced intensity conditioning regimen. This relates to the fundamental attribute of the program. the program uses a genetically modified form of the gene for fetal hemoglobin, which has better rates of anti-sickling and therefore -- and better oxygen buying characteristics. And therefore, it's fundamentally a potency delta. We can get the same clinical effect with less engraftment, and therefore, with less preconditioning than the other therapies can. And what that means is we use a melphalan-based regimen right now with far shorter recovery times, a hospital stay of 5 days or less on median. And in some sense, we think this is likely to be an outpatient administered procedure by the time that we reach the sort of commercial end of the spectrum with much lower rates of ovarian failure, much lower sort of toxicity associated with the preconditioning. And we think this is going to make a massive difference to patients. You know I'd point out, stem cell transplants are a current potentially curative treatment option for sickle cell disease. And most patients don't undergo stem cell transplants, in part because they have to go through the same busulfan both preconditioning. So we think this preconditioning is going to matter a lot to patients. And we think we will be the first therapy, even though we'll be a couple of years behind some of the other genetic medicines in sickle cell disease, we will be the first therapy to offer a truly differentiated profile by avoiding this busulfin based preconditioning.

Well, thank you so much, Matthew, for joining us today at the Healthcare Conference. It was a privilege to host you guys. And thanks to all of our listeners for joining. We hope everyone has a great rest of the day, great rest of the conference.

Thank you so much for hosting. It was my pleasure to be here.