Welcome, everyone, to the 44th Annual JP Morgan Healthcare Conference. My name is Anupam Rama. I'm one of the Senior Biotech Analysts here at JP Morgan. I'm joined by my squad: Ratih Pinhay, Joyce Zhou, and Priyanka Grover. Our next presenting company is Vor and presenting on behalf of the company, we have CEO Jean-Paul Kress.
Thank you, Anupam. And good morning, everyone. Pleasure to be here and to update you on our progress at Vor. First, I'd like to say that I will be making forward-looking statements. This is our disclosure slide. I'll let you read it quickly. And at Vor, we have the ambition to redefine the way we treat autoimmune B-cell mediated diseases. And we want to improve the standard of care as we pivoted the company mid-2025 to an autoimmune powerhouse from a cell therapy company. So it's a brand new Vor. We call it Vor 2.0. And for doing so, we in-licensed what we think is one of the most promising autoimmune assets in the block called telitacicept. Telitacicept is coming from the China Biotech Innovation Hub. We got it from RemeGen, a company who made the news a couple of days ago.
I'd like to say that I'm very pleased to see our partner and my partner there in crime, Jianmin Fang, the CEO of RemeGen here. Telitacicept, and congrats for your deal with AbbVie, by the way. It was beautiful. Telitacicept is a BAFF/ APRIL inhibitor, which has the ability to remodulate the immune system without broad and deep B-cell depletion of immune suppression. This is really important because patients with autoimmune diseases need to be treated lifelong, and many treatments currently don't support that. Very importantly, thanks to the work of our partner in China, Telitacicept has been validated clinically in eight plus autoimmune diseases. I'll talk more about that in China, which gives us an incredible position to leverage the data there and also work on indication selection, which is a big deal in autoimmune diseases.
More than 10,000 patients have been treated there, and it has de-risked telitacicept, which has a very favorable safety profile. The pipeline and the product potential of telitacicept is broad, and we have selected our first two indications with MG, myasthenia gravis, and Sjögren's disease. We are currently conducting our global Phase 3 trial in MG and rolling patients, and soon, within the next few months, we will start our Sjögren's disease trial, and very importantly, we are well capitalized with $450 million on the balance sheet, so it's very healthy. We have the funds necessary for leveraging our opportunities and delivering on our key catalysts in the midterm, so we are very well positioned to become a powerhouse in autoimmune disease. Now, after this brief overview, I'd like to tell you about how it works, what's the mode of action of telitacicept. It's a very compelling mode of action.
BAFF and APRIL are two key cytokines necessary for the development, the maturation, and the survival of B-cells. By blocking BAFF, telitacicept inhibits the abnormal development and maturation of B-cells, and by blocking APRIL, it inhibits the pathogenic production of autoantibodies by plasma cells. This dual modality enables a remodulation of the immune system without deep or broad immune suppression, which again is a high unmet medical need, so we believe we have an extremely elegant and effective mechanism which supports a breakthrough in autoimmune disease treatment, so the modality is compelling, but what's even more interesting for an asset of this kind is the number of data that our partner has produced in China. As you can see here, the clinical profile is consistent and basically clinically meaningful in all instances. Three commercial approvals there in systemic lupus, RA, and myasthenia gravis.
Recently, two BLA submissions in Sjögren's and in IgA nephritis, and in the clinical development program delivered best-in-disease profile in three indications. So it's great to have this data, which gives us the status of actually the most advanced BAFF/ APRIL inhibitor in the world and gives us such a lead for efforts globally. It's true in clinical efficacy, but it's also true in safety, which is extremely important in this chronic disease. Tens of thousands of patients have been treated commercially in China, in addition to the almost 2,000 patients enrolled in the clinical development there, Phase 2, Phase 3s. And the product is basically de-risked safety-wise. No burdensome vaccination requirements, no signature B-cell depletion-associated serious adverse events, and in general, mild to moderate adverse events. So we're very encouraged and confident that this profile will support a best-in-class or best-in-disease approach in our space.
Here you can see the pipeline and the product overview of telitacicept. The wealth of data generated by our partner in China, combined with our own efforts globally now, will put us in a very favorable position. You can see the number of late-stage studies who have already delivered or are on the verge of doing so, and again, the strong cash position supports a runway into mid-2028, which should cover the key milestones midterm. I'd like to tell you more now about our beachhead indication, myasthenia gravis, so why did we choose myasthenia gravis, so why we think it's actually a great blockbuster opportunity? Number one, it's a sizable market. By the end of the decade, the forecast for the market in the US only is supposed to be around $10 billion, so it's growing. It's also expanding to more patients eligible to biologics.
Number two, there is an unmet medical need for durable and disease-modifying treatments. Currently, we address symptoms, but we don't go on the origin of the disease, and we're not very durable. We believe telitacicept has the potential for doing so. Here again, the clinical development program in China has delivered on a very impressive efficacy profile. MG-ADL is the primary endpoint in myasthenia gravis. It's a patient symptom score, very well-known in clinical practice. This disease, by the way, is a very formidable disease. It's well-known. It's well-studied, thanks to other modalities. Actually, it's also why we have chosen MG. It's well-characterized. Here you can see the comparison of the China Phase 3 trial results with other modalities.
On the MG-ADL improvement, the result is extremely impressive with a minus 4.8 improvement on this score, which gives us an incredible amount of room for delivering on our global study. That's impressive at week 24, which was the first part of the study, but the study also expanded in time with an open-label expansion program, which added 24 weeks of investigation in the study. You can see here, and it's extremely important in this disease, but also in all autoimmune diseases, it sustains. It's durable. There is continuing improvement beyond week 24. Here it was studied until week 48 through the OLE. You can see that the MG-ADL improvement, or actually absolute improvement, non-placebo adjusted, was 7.5, which is incredibly high.
So here you're talking about almost symptom remission, which gives us a strong product profile to compete in a market which is now dominated by a few classes which have shortfalls. We are enrolling our global Phase 3 trial in the U.S. and other Western countries. It's well underway. It's going well. There is excitement from the investigators on the product, which addressed the upstream once again and have the disease modification potential. We've made some changes. It's basically the same than the China trial, but we've made some improvements at the demand of our investigators, especially in the open-label extension part of it. We have extended it to 48 weeks, which we believe will give us even more data long-term for substantiating the differentiating durability aspect of telitacicept.
In a nutshell, MG, well-known disease, high unmet medical need despite busy space, and the opportunity to compete very effectively with the best-in-disease profile that we are generating now and with the help of our China colleagues. Now let's turn to the next big frontier, Sjögren's disease. Completely different opportunity for different reasons. It's also a very compelling and exciting opportunity. It's probably the most underserved autoimmune disease currently. It's a large disease, or at least a large population of patients affected by the disease. Almost 300,000 patients in the U.S. only. 100,000 patients currently addressable by biologics. These patients are suffering from a multitude of symptoms. Traditionally, it's dryness and fatigue disease, but there is much more than that. It's a systemic polysymptomatic polycausal disease, more complex than MG, involving B-cells in a big deal compared to antibodies or autoantibodies only.
And here you can see the severity scoring on the left, which is measured by the SDI endpoint, which is a physician score, which shows that patients evolve through stages of severity, which become very handicapping and taxing. They cannot really function. And even more concerning, they often transform into B-cell lymphoma in a significant proportion. And obviously, B-cell lymphoma has a pretty poor prognosis. So currently, these patients are treated by older therapies like corticosteroids or immunosuppressants, which don't support long-term treatment and disease modification. And again, we believe here that the BAFF/ APRIL inhibition remodulation of the immune system modality has an immense potential in this indication. So it's not only a belief because it's already proven. That's the beauty about this asset. Thanks to the effort of our China partner, who has delivered on best-in-disease efficacy profile on the Sjögren's Phase 3 trial there.
You can see here that this study actually in China had the particularity of having patients with no background therapies, no steroids, no DMARDs. So I call them pure patients. What you see is what you get. No background noise, a true signal. It's really the effect of the drug. So this drug works very well in Sjögren's. It was true at week 24. And again, here, you can see here on the two endpoints, SDI and ESSPRI, which are the regulatory endpoints in Sjögren's. It was true at week 24 with very compelling numbers. But it's also true in the expansion period here until week 48. So it shows that it also sustains and supports disease modification, which again is key in these autoimmune diseases, even more so for those complex diseases like Sjögren's disease. And I mentioned best-in-disease profile here with the caveat is inter-trial comparison.
We try to see how we compare with other modalities being explored. There are several modalities, actually several products, not so many modalities, but several products being explored, a couple of FcRns, a BAFF receptor inhibitor, and another agent. You can see the results here, placebo-adjusted, which give an incredible picture of the profile of the product and the differentiation we can hope. So this gives us a high level of confidence in our ability to deliver a best-in-disease profile in the West globally. We are working on our Phase 3 trial, which is ready. We have a protocol approved, and we will start in the next few months our enrollment. Very exciting opportunity, multi-billion dollars. And again, we think here that we will have the best-in-disease profile in the label, which should support a leadership position in this incredibly high unmet medical need indication.
Sjögren's is super exciting, and we have also the teams to deliver on that. I mentioned the potential. MG and Sjögren's, beachhead and follow-on, are on their own already blockbuster indications, as mentioned. Multi-billion-dollar opportunity. It doesn't stop here. In theory, we could address all B-cell-mediated autoimmune disease. We have data in IgAN, in China, etc., etc., and lupus, etc. We didn't decide yet, but we are already thinking about our next wave of indications. Here we will be good stewards of capital, and we'll pay attention on the absolute imperative to deliver on the current studies versus potential new opportunities. As a conclusion, I hope I was able to convey our excitement and enthusiasm for the potential of our company and its lead asset. In MG and in Sjögren's, these are blockbuster opportunities. We are very well placed.
We have the means, the teams, and the resources to deliver. We have assembled in record time in the last seven months a team of experts. We have people coming from the best autoimmune disease companies around who are joining us because they know the asset has so much potential. We can transform the standard of care here. And we'll not stop here because we'll try to expand in time to a true pipeline in the product approach. And last but not least, it's also about money. We have $450 million on the balance sheet, which gives us what we need for delivering on our next wave of catalysts. Thank you very much. And now I'd like to call my team to address the Q&A. Anupam, you will lead.
Thank you, Jean-Paul. Do you want to quickly introduce who's on stage with you? Yeah.
Thank you very much, Anupam.
So we have Sandy Mahatme, who is our CFO, tons of experience in raising money and all kinds of financing. And he's also our Chief Business Officer because we don't forget that we might have some BD opportunities and M&A opportunities in the future. We've got Dallan Murray, who is our Chief Commercial Officer. He has long experience in best-in-class leading specialty care companies, including Gilead, Vertex, and Sarepta. And at some point, I was asked, "Do you need a chief commercial officer that early?" I said, "Are you kidding me? We are pretty late.
We're in Phase 3, and we have to prepare for our launches." And last but not least, we have Jeremy Sokolove, who is our Chief Medical Officer, an expert in autoimmune disease, rheumatologist, immunologist, who has made a big difference already since he joined us in the program and in interacting with our KOLs and our stakeholders.
I'm going to ask the first couple of questions, but of course, I'm going to ask the audience if they have any questions. And if you do, just raise your hand, and we'll make sure you get your question in. I just wanted to ask a really broad question. And I think this is one of the key questions as it relates to transferability to the regional China data to a global population.
What are the factors that kind of give you confidence in extrapolating this regional telitacicept data to a global scale?
Thanks. It's probably a good question for Jeremy.
Great. Thanks, Anupam. So I think that the China population is clearly representative of a relatively homogeneous biology compared to a global population. That gives the opportunity to really evaluate the efficacy of the modality, specifically in the disease of interest and across six different diseases plus, they've demonstrated significant efficacy. So the consistency of the data is really important. Number two is the ability to translate the biology from one population to another with prior studies. So with Benlysta, belimumab, the data in China, very similar to the global data. With our IgAN data, the IgAN data in China, very similar to global IgAN data with other similar BAFF and APRIL mechanisms.
We think there's every reason to believe that the biology and the opportunity translates. There will be some potential dilution in the effect size due to increased variability as we go from a more homogenous to a more heterogeneous population. We think that will be relatively small. This is a mechanism with very significant efficacy, and that's been proven over and over again, both in the Chinese population and in the global population for IgAN. We're very confident in the translatability. And again, as Jean-Paul showed, even if there's a small decrement in the effect size by that heterogeneous population globally, that's something that still maintains a best-in-disease profile.
And then for myasthenia gravis, so where are you in terms of site initiation and enrollment curve, how we should think about that? And just this is a global study. How will it break down regionally?
Yeah.
We need to do the traditional equation in terms of number of patients, geography. The U.S. has to be around 20%. It's important. And then we can play around the globe with trying to see how we mitigate some effects which are well-known in immunology. But maybe Jeremy, you can elaborate on how we want to optimize our study.
Yeah, the study is going really well. RemeGen did a very nice job initiating the study globally. We've taken over a good part of that, and we've expanded further. We've increased our number of U.S. sites to make sure we have adequate U.S. representation, as well as some of our Western European sites. The study is going very well. We are on track to our catalyst, and we're really optimistic.
Question from the audience? Feel free to raise your hand. And then I guess you talked about the Sjögren's study.
Have you met with regulators and got sign-off on the study? And now we're just in a study initiation process? And where are we in the study initiation process?
Yeah. Well, RemeGen had started these efforts before our time. They did a good job to prepare the field there, and they met with regulators. You don't need to meet every month with regulators. There is sometimes a misconception that you need to meet with them every two weeks. It's not frequent, actually. So we've done some things to, well, you remember very importantly that in Sjögren's, Novartis published their results at the same time as us at ACR last fall. And that was pivotal, if I might say, for the space because tons of learnings on the first global study, Phase 3 study, emerged from their presentation.
We don't really have time to go there, but a lot of issues were surfaced in their trial in their results. They were the pioneer here. They had to deal with an unknown study with inexperienced physicians and investigators, complex study, etc., etc. They gave us a huge favor. They were bold. They did a good job. Yeah, they got a bit of casualty of that. The good news for us is that we have actually this learning that we can apply to our study. We did some modifications on draft protocol to try to include and reflect these learnings in our own endeavors. We believe that now we have a very robust protocol to maximize what could be disconnect with what we observed in China, which was a prior study.
And then you presented a lot of data in the second half of last year on both MG and Sjögren's. I'm just wondering, as you met with KOLs on a more global basis, what the feedback on the data were and any pushbacks?
Yeah. No pushbacks. Lots of enthusiasm, guys. Yeah. There's excitement, a lot of excitement and enthusiasm. In MG, they're excited about targeting the disease upstream and disease modification. In the rheumatology community, they're really deep in immunology. They understand the differentiation of this mechanism. And the feedback has been excitement. They want to see the data. They want to see global data, and they want to participate in our global trials and be part of this exciting mechanism.
Questions from the audience? And then I guess RemeGen has the China rights to the product. How are you thinking about OUS rights outside of China for the product?
So I'll stop with a generic answer. Right now, the U.S. is obviously prominent. It always was prominent. It's even more prominent now. The more recent MFN discussions have wounded Europe pretty badly in terms of prices. And there is a big disconnect now, which will actually increase. Currently, the view from pharma is that the U.S. will be even more the field for pharma. I put China aside. But Europe, traditionally, you always wanted to, okay, to U.S. and Europe, say, "Yes, you want to help the patients globally and make sure that you address them properly." But on the financial return on investment, U.S. will stay disproportionately larger than any other territory. So our absolute imperative is to secure the U.S. and be ready for that. We have the team for that.
We already have a medical affairs team with people coming from the best companies around, Argenx, etc., and we, of course, will address the other geographies in time, but we have a bit of time because in addition to not really rewarding innovation, some of these geographies are late to approve drugs and price the drugs. So we'll cover everything in time, but priority to the US for obvious reasons.
And then, Jean-Paul, I was wondering if you could expand on your comments on one of the last slides where you talked about we're thinking about the next wave of indications for telitacicept. The push-pull levers when you go into indication selection, almost walking us through the rationale of why you picked MG and Sjögren's.
Yeah, so MG was picked by RemeGen before us.
We are super pleased that they did so because one of the possibilities would have been IgAN. We work beautifully in IgAN. We've got great data, which actually are from China, which are actually validating the question mark on how we're going to reproduce globally because our data are very similar to others by putting in with your companies who have done global trials. But besides that, the choice of MG was very good because for all the reasons I mentioned, we think we can achieve blockbuster status in a high-end medical need. We have a lead versus any other BAFF inhibitor company if and when they will decide to go for MG. We have at least two and a half years of lead.
I didn't mention that we have a great advantage, which is we have a very rock-solid dose-ranging demonstration with our asset, which is not the case for the other ones. Now, other indications for any asset in immunology or autoimmune disease is a mix of unmet medical need, market potential, capability to differentiate in the thing. IgAN, for instance, IgAN, you very much an APRIL mechanism. So you have some APRIL inhibitor out there. So you might wonder, do you want to go with a BAFF APRIL? So these kind of things. And also, obviously, pricing. Pricing is very important because some indications support a higher pricing because of established drugs and number of patients and burden of disease, and some indications don't. So you have to mix all that to do the right choice. We have some proxy.
I mean, some other autoimmune disease companies have done some work around that, but we'll have to pioneer because our potential in terms of indications is very large. But we'll not go blind. We need to exert all these criteria to make choices.
Any final questions from the audience? I have one more. Yeah, go ahead.
What is the biggest improvement of Sjögren's? So the question is, what could be the biggest improvement of your Sjögren's trial compared to Novartis?
Yeah, that's a great question. And thank you. Jeremy?
Yeah. I think we have the advantage that Novartis has taught us a lot in the last several years about Sjögren's development. So I think one is Novartis has taught us that you have to go to experience sites. The SDI is not a simple tool. And Novartis brought on many sites to conduct such a large global study.
We have the advantage of now having those well-trained sites. We decrease the risk of having imprecision in the baseline measurement and over time, which can result in regression to the mean. We're very pleased about Novartis's education of the community and specifically investigators. That's one thing. The other is well-controlled background medications. We know that there is a syndrome of sudden compliance where patients who we think are on stable background medicines, maybe we're not. At the initiation of our clinical trial, they begin taking them. We've been able to put in place several mechanisms to assure that people who are not taking their medicines don't suddenly start, and those who are taking them take them in a consistent way. That even goes more for corticosteroids, which are very important that there's not a patient-derived variability in how they use the steroids.
We've incorporated that into our clinical protocol. These are all things that go to mitigate that placebo response that is inherent when those go wrong. And again, Novartis did an amazing job opening Sjögren's to the whole world. And we're going to be able to leverage their learnings to improve our study delivery.
Thank you. Maybe final one from me. You've had a lot of recent financing activity. What's the current cash runway, and then what key milestones are assumed in there?
Yeah, so we have about $450 million in cash. It gives us a very nice runway of two and a half years, right through the middle of 2028, supported by very long investors.
What it gets us through is the Phase 3 trial, completion of the MG Phase 3 trial, the commercial launch of our MG launch that Dallan will be handling, as well as the medical affairs efforts that are needed. Furthermore, the manufacturing ramp that's needed and stockpiling of the drug in the United States, all of that will be paid for. Then lastly, it'll also take us most of the way through our Sjögren's trial. It gives us a very nice runway, as I mentioned.
Thank you, Jean-Paul and team.
Thank you, Anupam. Thank you very much.