Welcome everyone to the 40th annual J.P. Morgan Healthcare Conference. My name is Tess Romero. I'm one of the senior biotech analysts here at J.P. Morgan. I'm joined by Taylor Hanley from the team. Our next presenting company is Roivant. 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, Tess. Appreciate it, and good afternoon, everybody. Thank you for having me at the conference. I'm gonna 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. 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 two, I'd like to remind everybody that I'm gonna be making forward-looking statements as a part of today's presentation, including about our financial conditions and product candidates, and you can see more information about that on slide two.
I'm gonna 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. Then I'll get into some of our current projects and some recent updates as well. 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. We've really planted two stakes in the ground in particular in how we plan to do that differentially.
The first of them is something that people who are 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. 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 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 vants 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.
This ranges from technology that I'd call on the more boring 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 computational molecular dynamics and machine learning that help us design new molecules and advanced programs using creative development strategies. A wide variety of tools that add up to, we think, a differential benefit to each of our vants. 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. On page four, I'll just give a brief overview of some of the things that excite me about the business today.
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 or expected decision from FDA in the Q2 of this year and a launch shortly thereafter, and with an ongoing trial on atopic dermatitis. I will talk more about that program shortly. We also have a broad differentiated pipeline of clinical-stage programs, including some such as Betoclimab, our anti-FcRn antibody at Immunovant, which is a public company, that people may be familiar with, as well as one 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, we've talked about this in other presentations, that preclinical data, in part, is generated from our... what we believe it 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.
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 stakes, including 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 wanna highlight, as sort of the last high level point here, a little bit about our track record. If you wanna 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 nine 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 four 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. 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 psoriasis drug that I'll talk about soon. Tapinarof is now under review by FDA for approval. You know, 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 had built. We think about our technologies, the computational tools, also as commercial opportunities, and we had built a tech company called Datavant that helps us better understand patient populations. It allows for the linking of de-identified data across siloed healthcare 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.
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. The first of those things, which we've talked about a little bit publicly before, but just as a refresher with an update starting on page seven, I wanna 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 agent that we are aware of in clinical development. You know, tapinarof, in short, as I've mentioned, we expect to be a potential multi-blockbuster.
Really, that to us comes down to the five 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 continued 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 zero, completely cleared their psoriasis.
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 four months, which is an extraordinary remittive benefit, again, something that hadn't been seen with a topical. This is combined with a safety and 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 limit and in our study, was studied across the entire body surface with no limitation. Unlike a corticosteroid, and I'm a psoriasis patient, 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 corticosteroid 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 has neither of those limitations, and so it is both a more efficacious and more tolerable therapy than the topical corticosteroid.
It has the ability, in our view, to be an important first-line therapy, really the mainstay 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. You know, I'll talk a little bit on page eight and nine again about our data.
Starting on page 8 with 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.
On page nine, as a reminder, I mentioned this on the prior slide as well, we see a remittive benefit for those patients where they spend 115 days median or 130 days 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. A truly differentiated treatment option. On page ten, 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 tapinarof and psoriasis is completely on track.
We have no expectation of an advisory committee, and we expect the PDUFA date in the Q2 of this year. You know, we feel completely ready from a manufacturing commercial production capability standpoint with the team at tapinarof, 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 have begun to make the relevant hires already, and the Dermavant team expects them to be fully set up.
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 at Dermavant working on developing and launching this product, including a management team that has deep expertise in topical therapy and dermatology, really a deep understanding of the field. That's something that the Vant model allows for that makes us confident and excited going into this launch. You know, we also had a milestone recently, which is that data from our main phase III study, PSOARING 1 and 2, was published in the New England Journal of Medicine.
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 tapinarof, with top-line data expected in the first half of 2023. 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. We have phase II data in atopic dermatitis supporting that effect. Tapinarof is a drug that has been in our portfolio for a while, and so we've talked about it publicly before. You know, I wanna spend a little bit of time today now talking about a new program for us.
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. We have antibodies, we have lentiviral gene therapy, we have topical agents, and we have small molecules. One such small molecule, RVT-2001, I want to spend a few minutes on today, is a new therapy that will stand as the basis of a new Vant called Hemavant. I want to just take a minute on page 13 and remind people of a feature of how Roivant thinks about programs. I've got a subset of the programs that we've acquired over time on this list.
You know, we've always built our pipeline in part through in-licensing. We have the basic research facility. We're working on discovery, especially with targeted protein degraders. 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. One of the things that we pride ourselves on, we are in an era of extraordinarily expensive deal-making in biotech. Sometimes we have to pay up for therapy, and we've done so, but we pride ourselves on being highly efficient in making deals for in-licensing. You can see on this list a number of the deals, including some of the medicines that we'll talk about today.
You can see down at the bottom two 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. 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 Roivant equity. In the case of the Priovant therapies, $10 million upfront, plus 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. Certainly, I feel like with both of these new therapies, we've achieved that. 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. On page 15, I'd like to introduce RVT-2001. 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. You know, I think if you're not familiar with that indication, MDS is a form of blood cancer that is severe and difficult.
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. With about two-thirds of patients, lower risk patients, who wind up with chronic disease that needs treatment. In particular, these patients wind up dependent, in many cases, on red blood cell transfusions to treat very severe anemia. It's a very difficult disease for those patients. There are not good therapies or good options apart from those transfusions, which winds up being difficult and painful and challenging. Recently in 2020, a BMS drug called luspatercept was approved for the treatment of lower risk MDS for transfusion independence, to achieve transfusion independence. As you may know, with that drug, it's been quite successful.
It's annualizing at greater than $500 million five quarters after launch. BMS at launch, BMS has signaled a potential peak sales of greater than $4 billion. 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 multi-pronged approach to optimize our development strategy. You know, on page 16, just to situate RVT-2001 in our in the treatment landscape for lower-risk MDS. You know, for these patients with transfusion-dependent anemia and lower-risk MDS, there are some stratifications based on various markers.
You know, lenalidomide is approved for the treatment of a subset, about 10%-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. Otherwise, patients first go 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. It's a big potential addition to the treatment landscape and something that we think should matter, especially because luspatercept only has responder rates in the 40%.
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. 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 populations of interest to us. Our ability to evaluate that data is in part drawn from cross-trial comparisons with other medicines. We've seen something really exciting here, which is that 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 pre-treated patients.
They had been pre-treated with lenalidomide and/or HMAs. That's important because we saw a 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 pre-treatment population, in that heavily pre-treated population, other therapies have stumbled significantly more. 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 HI-E is not actually a transfusion independence endpoint. It's something more mild than that. Only 12% of patients in the lenalidomide trial with prior HMA exposure in an investigator-sponsored study achieved that sort of reduction in transfusions associated with the HI-E endpoint.
Those are both significantly lower rates in those patient populations than our admittedly somewhat small N, but significantly higher responder rate of greater than 30% in our study in those pretreated patient populations. That's against a backdrop across the 84 patient treatments of a relatively well-tolerated therapy, with the majority of events classified as Grade 1. There's a significant need here. In addition to being excited about our potential to deliver better response rates in heavily pretreated populations, which if this study continues to show the same data we would be able to achieve there's good precedent for significant improvement when you move these therapies into earlier line patients.
For example, that 13% for luspatercept among patients who had been pretreated with lenalidomide, when you go to patients without prior lenalidomide exposure to earlier line patients, it increased to 44% in their phase II trial. Luspatercept phase III trial actually excluded prior lenalidomide exposure because they had seen significantly reduced transfusion independence rates in lenalidomide-exposed patients in their phase II. In that same lenalidomide investigator-sponsored trial that I mentioned before, the 12% post HMA response rate increased to 38% in the HI-E with prior to HMA therapy. In both cases, you saw significant improvements in response rate once you moved to an earlier line patient.
We expect to enroll earlier line patients in our RVT-2001 phase I and II trial, who have been more responsive. If we see improvements of the same kind, we have a potential for an overall best-in-category therapy for these patients. We think it's pretty exciting data, and we're excited to run that phase I, II study. We expect to enroll 50-60 additional patients, and we expect data in early 2023. You know, 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 MDS patients with SF3B1 mutations, which is about 30% of the MDS patient population.
Obviously, there's good biological rationale there, given that we are an SF3B1 modulator. Then also, here's some additional exciting data. You know, in the subset of patients who have this TMEM14C, aberrant TMEM14C transcripts, which is a biomarker associated with SF3B1 mutations, we have 7 patients, of which 5 were responders. A 71% responder rate for transfusion independence. You know, we're also expanding the dataset specifically in that biomarker population, with the hopes that that's an additional potential path for us to validate demonstrating a higher rate even in that subset of the population. We're also optimizing dosage. We think there's a possibility to extend the pharmacodynamic effect by optimizing dosage of ARBG 2001 in this phase I/II study.
Finally, the only other point that I would make here is, while I've characterized this study as higher risk, 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 decay between phase II and phase III in other MDS therapies. 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. Thank you. That's what I've got on this new ARBG 2001 therapy, and we're excited to continue to provide updates as that trial gets up and running.
I'll next very briefly review some updates from a few other events, noting that I'm not gonna spend as much time on this. Starting on page 20 with an update that Immunovant provided last week. Again, Immunovant is a public company, speaking later in the conference. 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 in myasthenia gravis that is expected to start in the first half of calendar year 2022, as well as additional pivotal studies in two other indications, including possibly, TED, WAIHA, or two new indications that we expect to announce by August 2022.
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 the subcutaneous delivery that batoclimab offers. I won't spend a lot of time on this, but maybe if you just look at page 22 briefly. You know, I think one of the things that differentiates our approach with batoclimab from the other anti-FcRn 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.
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. This mirrors the way that autoimmune disease therapy works in other indications, and it's something we feel is uniquely enabled by batoclimab's product attributes. On page 23, I just lay this out versus a couple of the other anti-FcRn agents that are really the others in late-stage clinical development, including efgartigimod and nipocalimab.
You know, both of which in various ways are not able to administer in sort of the same framework. You know, efgartigimod, which is currently an IV therapy that was approved last month, is really a sort of symptomatic exacerbation approach where it's treated with sort of cyclical therapy, four weeks on, followed by four weeks off. Not with this sort of induction and 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. Only we have this sort of full induction maintenance and rescue paradigm, including with fully subcutaneous administrations. 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. 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.
We believe the same attributes that allow us to take a differentiated approach to use of batoclimab in MG will allow us to take a differentiated approach in some of these other indications. I'm not gonna talk about other events today, but I'll just briefly pull up on slide 28 a few slides at a time. I'm gonna skip our event 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 three pivotal initiations of batoclimab, including a phase II study in namilumab for sarcoidosis at Kinevant, where the IND has been accepted as of last month that we're on track to initiate a multiple ascending dose trial of our lysine-based anti-infective at Lysovant, with an IND submitted last month.
The robust open label expansion of the ongoing phase I/II trial in RVT-2001 that I mentioned before, among other trials. 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 development 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. Genevant is a scientific leader in the field of lipid nanoparticles with scientists who have been working on this technology for a very long time.
We are some of the earliest people to have worked on lipid nanoparticles, and we have innovative scientific collaborations, including with folks like Sarepta and Takeda on novel uses for LNP. 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 lipid nanoparticles 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.
You know, 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. Recently, as of last month, the Federal Circuit affirmed the validity of these patents, rejecting Moderna's appeal. We feel that IP estate has been affirmed. 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 and of this IP estate and the continued growth into novel LNP. It's an area to keep an eye on. There has been some confusion, and I'll just sort of on page 31 highlight.
You know, I think the easiest way to think about this is via our ownership stakes in Vants. We retain a 76% exposure on a basic basis or a 62% on a fully diluted basis on an economic interest in any royalties or otherwise derived from that patent estate. That's the sort of Roivant exposure, considering all the different pieces. I'm not gonna spend time today talking about the Roivant discovery 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.
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 in 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 Aruvant, as well as continued information from patients progressing in our phase I/II trial at Aruvant and sickle cell disease this year.
Initiation of our programs in sarcoidosis and at Lysovant, expansion and continued progress in MDS, as I discussed, and phase I initiation for our first degrader candidate, as well as multiple preclinical programs across Roivant Discovery and Proteovant, producing data that we should be able to share from a preclinical perspective and entering IND-enabling studies. 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 wanna thank you for taking the time to listen to it. I wanna call back to Tessa, who I think is gonna take some Q&A.
Yep. Thanks so much for that. 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?
Yeah. That's a great question. Pricing is one that we get from time to time. You know, 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.
First of all, there's a very wide range and an innovation gap right now between highly priced systemic therapies like SKYRIZI or whatever, which are tens of thousands of dollars a year or more, or Otezla, which is $3,500 a month. Then on the lower end of the spectrum, the corticosteroids that are generic and often inexpensive. There's a very wide band of possible pricing outcomes, and we haven't given guidance on price. You know, 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.
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. It's not difficult to get through step edits. We haven't said whether we expect step edits. 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. To be candid, systemic therapies haven't struggled with step edits before, so I'm not sure that's exactly the way that we think about the category.
From a pricing perspective, we haven't said, and I think we have a wide range of possibilities given the profiles of the molecule. We're gonna watch closely what's going on with some of the other topical therapies, and especially Opzelura in atopic dermatitis, which Incyte 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. 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 on-
Yeah.
Timing on when we could hear next steps?
Yeah. Thank you. I can't comment publicly on any action of any kind in any kind of infringement. You know, I'll say we were pleased by the outcome, obviously, in the appeals court, which affirmed the validity of the patents. I think we're sort of watching for that process to work its way through. You know, even after the appeal, even after the appeal court decision at the beginning of December, there's sort of a couple month tail to how that process plays out. 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. You touched a little bit on the myasthenia gravis study. What are the key endpoints and bar for success for this study? I think-
Yeah.
Yep, go ahead.
Go ahead, please.
I guess the second part of the question was just around safety and monitoring LDL elevations in that study.
Yeah. Yeah. 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. You know, in terms of endpoints and sort of the bar for success in mg obviously the only approved FcRn, anti-FcRn antibody at this point is efgartigimod from argenx, which got approved last month. That obviously sets an important bar in terms of how people think about it. You know, the main endpoint that people look at is MG ADL, and how these therapies perform against that bar. You know, the argenx study, which led to the approval of efgartigimod, studied this intermittent dosing paradigm.
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. 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. We think that's an important measure, and it's something that we're focused on. You know, in terms of cholesterol monitoring, I think Immunovant's comments on this are the most important, but in general, we feel very comfortable with the cholesterol position. We have added exclusion criteria.
Immunovant has added exclusion criteria in the study, especially for patients with LDL levels above 190, which should exclude somewhere between 5%-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. 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.
No problem.
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. We can.
Anything you'd highlight on the potential for differentiation.
Sure. We continue to enroll patients in our ongoing phase I/II study there. As has been our practice, we will continue to report data on those patients, sort of as it comes in. 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. By the way, there's data on this on slides 26 and 27 in the presentation materials. You know, I'd say on differentiation, and this is a point that's incredibly important. You know, one of the interesting things about the sickle cell disease sort of treatment landscape right now is that genetic medicines in sickle cell disease, the bar really is curative clinical efficacy.
It's something that Bluebird has shown, it's something that CRISPR's shown, and it's something that we, after our process development work, have shown in ARU-1801. 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 busulfan-based regimens. These patients, they spend literally months, in some cases, in the hospital. You know, these are young patients and women, for example, on busulfan. Now, this is not toxicity associated with the drug, it's toxicity associated with the preconditioning. Women on the preconditioning regimens have 70%-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 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 modified form of the gene for fetal hemoglobin, which has better rates of anti-sickling and therefore and better oxygen-binding 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. What that means is we use a melphalan-based regimen right now with far shorter neutropenia recovery times.
You know, a hospital stay of five days or less on median, and sometimes 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 preconditioning. We think this is gonna make a massive difference to patients. I 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-based preconditioning. We think this preconditioning is gonna 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 busulfan-based preconditioning.
Well, thank you so much, Matthew, for joining us today at the Healthcare Conference. It was a privilege to host you guys. Thanks to all of our listeners for joining. We hope everyone has a great rest of the conference.
Thank you so much for hosting. It was my pleasure to be here.