Today, and maybe we can start with a quick round of intro. Jean-Paul, do you want to start first?
Yeah, thank you. Thanks for having us. I can see that not everyone is back to school. It's a start. I am Jean-Paul Kress, CEO of VOR Bio. I joined when we e-licensed telitacicept, our asset we have to speak about today.
Sandy do you want to go next?
Yes, Sandy Mahatme, the CFO and the CBO of the company, joined a few weeks after[unintelligible].
And Dallan.
Dallan Murray I'm the Chief Commercial Officer, and I joined at the beginning of August.
Yeah, Qim Zuraw. I'm Chief Development Officer. I joined in mid-July.
Carl.
Thank you so much. Thanks a lot for being with us here today. Maybe we start off. I know one of the key trends for the last year really is a lot of the licensing deals of China assets. Here, our assets are centered around China licensing. Maybe start from there, Jean-Paul. With all these assets out there, how did this particular asset catch your eyes and eventually come with this deal with RemeGen?
Yeah, and that's a very exciting time for VOR Bio. We call it VOR 2.0 because we really repurposed the company throughout this e-licensing for the global rights of telitacicept. We'll say teli. It's a bit easier to say. Here we have the perfect storm for value creation. The asset is a BAFF/APRIL inhibitor. We'll come back to it. It has a dual inhibition of those two very important factors for B-cell lineage activation and survival. It works on the upstream and the downstream, which is a big opportunity to fill a met need in many autoimmune diseases. It's an advanced late-stage product. It's commercial in China. 70,000 patients have been treated with the product. It's a huge advantage. There is a huge database of real-life experience with teli. It's also very well studied throughout phase II and phase III in several key autoimmune indications.
We have 3,000 patients who have been investigated through these late-stage trials in China. That's a very important point because sometimes when you partner with a China company, you deal with less mature or less, I would say, business-ready companies. You have a bit more risk. Here is the risk because the asset itself has a lot of experience and the profile you will see is very compelling. At the same time, our partner, RemeGen, is very strong. It's a large company which is in charge of the whole value chain. They have research capabilities, state-of-the-art development skills. They also have cutting-edge manufacturing plants which can produce for the drug substance. We are in good hands in terms of partnership. That was a key thing in the deal. Now we could talk about the asset, but I'm sure you have questions about that.
I would also say that this is, you've seen the flurry of deals coming from China. We can comment on that as well. We like to say that we are probably the first big autoimmune deal coming from China with this late-stage asset and the risk asset. A superb opportunity.
Got it. Just to your point, there is, as you mentioned, a fairly robust set of data on various autoimmune diseases for this particular asset in China already. Historically, people had some concerns about the transferability of the China data to a much more global-oriented population. I think we have now moved past the point where people question the data quality, but now, there was a more homogeneous population that was studied in China versus now a much more global population in the U.S. and in European or other markets. Do you have some concerns like that, like as you were looking through the asset?
On the contrary, I think it's good to step back a little bit and contemplate what China has achieved over the last five years and expansion exponentially over the last two, three years. The biotech industry in China and the innovation from biopharma in general in China has caught up with the level of the U.S. If I quote a couple of numbers, you have as many assets in the China pipeline, all stages from IND to BLA, NDA, as in the U.S. pipeline. Because it's growing, it will probably overcome the U.S. by the China number of assets. If you look at the global pharma pipeline worldwide, same across the spectrum, China has one third of it. I'm not talking about generics or copycats. I'm talking about cutting-edge modalities, biologics, bispecifics, fusion proteins like teli and others, even in cell therapies and gene therapies. This is incredibly impressive.
Probably even more related to your question, the number of assets is important, but also the fact that China has, the authorities have put in place a plan called China Health 2030, which has for imperative to facilitate and ease up the approvals and the regulatory process and also help the execution of key clinical trials. The maturity of execution by the China ecosystem is incredibly strong and has caught up with the West. The integrity is very important. Last but not least, the talents there are best in class. You have China nationals who have gone to the U.S., studied here, learned in great organizations here or academics or private, go back there, set up their startups or work in the ecosystem. That's been incredibly powerful for them. Last but not least, the capital is flowing. They lack a little bit.
That's why they need us in the West and in the U.S. for that. That's a perfect match for value creation. That's a general statement. Now for us at VOR with teli, very important to mention again the quality of our partner, RemeGen, public company, commercial company, several thousands of employees, decades of experience from R to commercial with manufacturing capabilities. Sandy was reminding us that they have 150,000L- tank facilities for production of biologics. This is incredibly powerful for our value creation opportunity. I'm actually happy to be part of this China innovation, which I think we can be proud to have harvested and a set of this quality and of this potential for a company like us, a biotech, which you know over the last couple of weeks have been reforming itself with a new management team of great quality.
I was able to pick on some people I knew from previous experiences. I mean, just, I mean, you didn't, I didn't spend time on me, but just for telling you a bit of who we are. I'm an MD and immunologist by training. I spent more than 30 years in biopharma working with organizations like AbbVie, Gilead, Sanofi across the world and mostly in the U.S. Most recently, I was the CEO of MorphoSys, where we launched an anti-CD19 in lymphoma. We acquired Constellation Pharmaceuticals to replenish the late-stage pipeline, and we got acquired by Novartis for $3 billion. Before that, I was the CEO of Syntimmune, an FCRN company, which was acquired by Alexion in 2018. I've been a bit around and that helped me to quickly form a great new team to basically take advantage of this opportunity.
I totally agree with you, especially on the China assets part and some of the clinical data transferability. I think another kind of unique aspect about teli is also this is an approved product in China, right? You can start seeing some of the commercial launch and how the uptake has been in some of these incredibly important indications like SLE, MG, and RA. Just to basically kind of double click on that question itself, the market in China, the fact that it's approved, it's obviously a huge advantage. We also recognize the market in China, the commercial market around autoimmune is very different, say like the market in the U.S., right? The competition in the U.S. for some of these indications are comparably a little bit more fierce than it is in China.
How do you feel like, you know, in some of these indications you are going after where the competitive dynamics in the U.S. might be a little bit different? How this would potentially differentiate?
I think it's important to come back to the strengths of the asset. I'll describe a little bit the modality, which makes it a huge proposal for many autoimmune diseases and there are actually even in the West unmet medical needs. teli is a BAFF, dual BAFF/ APRIL inhibitor. It acts on two different pathways at different places of the B-cell lineage, on the upstream by normalizing the B cells and on the downstream by normalizing the long-lived plasma cells in the bone marrow. This spectrum of activity ultimately decreases or cuts off the pathogenic autoantibodies, which are responsible for many autoimmune diseases, not restricted to IgGs because we actually control or normalize the pathogenic IgGs, IgAs, IgMs. In many diseases, those are involved, including myasthenia gravis. This very elegant and comprehensive mode of action addresses not only the symptoms, which most therapies actually commercialize like the FCRN do.
They just basically improve symptoms, but they don't help with durability. It's pretty short-lived. Here we have the opportunity to have a durable effect with what we will call disease modification. That's new. When you talk to physicians in several autoimmune diseases, take MG, which is a flagship slash beachhead indication for us, the physicians, they want durable products with the potential for disease modifying. There has been the 1.0 version of the biologics in autoimmune with the FCRNs , obviously, but it was in 2019 or 2020. It's exactly like in other diseases. It's a great wave of innovation, but now we're ready for the version 2.0 of biologic therapies like products like telitacicept.
Maybe just to actually double click on one of the comments you made, MG is going to be your beachhead indication. Why do you think, like I know that in China, teli is actually approved in several indications. Why do you ultimately pick MG as the beachhead, like the launchpad for potentially other indications as well?
In China, teli is approved in RA, lupus, systemic lupus, and MG most recently. We have positive and best in disease in many regards. Phase III results in China in those diseases, plus IgAN and Sjögren’s disease. We'll come back to that. There are other indications investigated. That gives us, as we mentioned earlier, an incredible advantage. Which biotech comes in the U.S. with this wealth of data coming out of the country where the validation is proven now. It's a great advantage to choose our indications. Why did we choose MG? MG is a bit like the iconic indication of autoimmune diseases. It's what actually Argenx started with, MG and ITP. There is still a lot of unmet medical needs with MG. I mentioned the need for durability and disease modification. That's what every neuromuscular physician treating MG will want.
You hear about even sometimes cell therapy or in CD19, which is very long-term vision. Some people are working on that, but we are plugging a huge space here. There is unmet medical need. The other reason is that this market is growing. If you look at the business part of things, it's a $4 billion market in the U.S. It's projected to be $10 billion in 2030, thanks to the arrival of new agents and somehow with the growth of some current agents. There are significant shortfalls with current agents. The FCRN s, they are a bit long-lived. There is on and off things. If you stop, they just flush the IgGs. They are very narrow. They very effectively flush the IgGs, but as soon as you stop the treatment, it comes back.
There has been work recently showing that the MG disease is actually multifactorial and much more complex. We bridge a gap here and we fill an unmet medical need with our upstream and downstream approach. We think there is still a lot of possibility to make business impact and obviously help patients in many ways.
Maybe just a follow-up on that. You know, as we have seen some of the recent papers that actually showed where some of these, especially MG, is actually driven by a particular subtype within the IgG class. How do you think, you know, to your comment on the differentiation from the FCRN class, how do you feel like the modulation itself could potentially be different, especially, you know, based on this kind of a new study on, as we understand the disease biology a little bit more?
You're probably referring to the O'Connor work out of Yale. This is very exciting data. He and his team basically confirmed that the MG physiopathology is more complex than what we thought. It's just not one type or subtype of Ig. It's actually more than 20% of the patients also have IgAs and IgMs, pathogenic ones, autoantibodies. You have a not unsignificant proportion of patients who don't have complement disorders. If you combine all that, if you factor those together, because it's in addition for the complexity, it fluctuates with time. Patients starting with IgG, then they have IgMs coming up and IgA. You need to actually encapsulate all that in your therapeutic proposal. It's certainly not the FCRNs who do that because they just target the IgG. By definition, you will miss these new or other things which are physiopathologic or physiopathogenic.
A product like telitacicept, with this upstream-downstream effect on the lineage, is actually very adapted to address the complexity of the disease in a durable way. This is where we come. This is where we act. I think this is a key advantage. The science is actually claiming for better modalities like ours. That's what the clinicians and the patients also need and want.
Just maybe follow up on that comment as well, right? For the BAFF and APRIL, we have also seen other assets within the same class, although at different indications. How do you feel like thinking about the competitive dynamics as you think about the other drugs in the same class? A lot of them are not going to gMG at this point, but I think ultimately that, you know, some of the other indications we're thinking of, like Sjögren’s, they start becoming a focus. How do you feel like our positioning against some of the other assets within the same class?
Number one, we are the most advanced BAFF/ APRIL in the world by far. 70,000 treated patients, commercial in China, 3,000 patients in mid-late stage clinical trials. The other two assets you're mentioning don't have that. Number two, we have the opportunity to play on a bigger landscape. It doesn't stop within the BAFF/ APRIL limiter. It's good that there are actually others because that shows that the modality makes sense and it works. I mean, the results from our competitors in nephrology, especially, we have results in IgAN as well. We have great phase III results coming from China. It also validates what they find in the West or in the U.S. globally, validates what we found in China. It's very aligned in IgAN, for instance. That's a great thing. It's beyond that.
The point is that the playground or the landscape of universe of opportunities is way beyond the BAFF/ APRIL inhibitors. We have the opportunity to compensate for the shortfalls of the FCRN , which are dominating a lot of the autoimmune diseases, MG, for instance. Here we can play a big role. There is a lot of shares to take from these other modalities. This is big, actually. This is actually in terms of number of indications because it starts with MG for us, but it doesn't stop with MG. Also in terms of potential market increase or expansion within each of these indications because as we know, not all the MG patients are being addressed right now with the current therapies because of either side effects or shortfalls. I think that's the most important point.
You could argue about small, you know, chemical or preclinical differences between the assets, but I don't think that's the point. We have an asset which works. We have best-in-class and disease data in several of our indications, definitely in MG. In MG, we're advanced by years compared to these products because they have not even started.
Maybe just follow up on your comment on the safety side, right? We, again, like I think the first class of these FCRN drugs, etc., was a huge transformative change for the MG space and several other autoimmune indications. These themselves are not perfect. We have seen like some of the RAMS programs, even black- box, etc. How do you feel like based on the existing safety data you have seen in China, how do you feel that could potentially translate into some differentiation?
It's another great advantage of having this wealth of data and this number of patients having been treated by our product in China. Now we also have patients globally because we started our MG trial. We'll start to collect ex-China data. There is no difference between the China patients and the Western patients, at least physiological. It's not like in Japan, for instance. The safety database is huge. The safety profile is extremely favorable and very consistent across the indication spectrum. In some indications or trials, we're actually very close to the placebo. That's super important because these diseases are chronic. Patients have to be treated the long term and sometimes for life. We don't want, we're not talking about, you know, oncology indications where the life expectancy is a couple of weeks, where you can tolerate more, like with CD19s. I was the CEO of MorphoSys.
We launched a CD19, Monjuvi. It was in lymphoma. You don't treat lymphoma patients. They die in a couple of weeks, especially RDLBCL. Here it's different. These patients, they have symptoms which can become very bad, but they have more time. You don't want to hamper them with a lot of side effects. Our product is very, the profile is extremely favorable for this kind of indications.
We spent a fair amount of time talking about the indication that's had a lot of validations, but I also wanted to congratulate the team for the recent Sjögren’s data, the RemeGen team that your partner has put out. I personally always thought Sjögren’s is such a huge market with very little, with no current real targeted treatment and with a lot of potential. I wanted to first congratulate the team here, but also get your sense, is this another indication you're thinking of beyond MG? How do you feel the drug's potential in this new, a very exciting wide space of indication compared to others?
You're right. Sjögren’s is an incredible opportunity for a couple of reasons. It's a large indication. Unfortunately, many patients, mostly women, suffer from this disease. It's a disease where the pathogenic autoantibodies attack the exocrine glands in a various way. It's very polymorphic. The typical symptoms at the beginning of the disease are the lack of tears and the lack of saliva. It sounds trivial, but try to imagine even when you have a contact lens and you don't have enough tears, it's not great. These people don't even have any at some point. It's really, really affecting the quality of life. It's actually affecting other organs and parts of the body in a bad way with time. It's a graveyard of molecules in development. There have been a lot of failures in the past for various reasons. It's difficult to address. Currently, it's an incredibly underserved indication.
There is nothing beyond symptom control. Recently, two events happened. Number one, we, RemeGen, and us announced that our phase III with more than 300 patients, I believe, in China has hit its primary endpoint. Just remembering the phase II trial, which was communicated on a couple of months ago or years ago, I don't remember, we showed that we have great efficacy in this indication. The primary endpoint is called SDI, it's a composite score of various complex things. We showed that we have an improvement of four points placebo-adjusted. If you compare that with other modalities, like FCRN s or BAFF receptor inhibitor, we almost double compared to them. It's inter-trial comparison, but it gives you an idea. That's very, very exciting. We will communicate on the phase III results at an upcoming conference in the fall. Bear with us.
We can't talk about that because we are under embargo per conference policy, but the results are very, very good. We're super excited. I will also mention a large pharma who has communicated recently on hitting the park on their primary endpoint in Sjögren’s in two phase IIIs. That's very good because it shows that we can now track the code. We're confident that the play, I mean, the landscape is very big, 300,000 patients in the U.S. for several players. Our profile seems to be best in disease. We're very excited. As you mentioned, we're planning to start a phase III trial, a global phase III trial in the next couple of months, which, by the way, has already been approved by the FDA. We're cleared by the FDA to start this trial. It's extremely exciting. It will be our second indication, bigger than MG. MG is the beachhead.
We're going to start momentarily a phase III in Sjögren’s.
I look forward to seeing more of the Sjögren’s data. I'm personally very excited. I wanted to know more details at the medical conference coming up. Maybe just on the kind of commercial part of Sjögren itself, you know, historically, I always feel like there is a lot of generics and there is a general pushback from payers saying some of these symptoms are not very severe. You have dry eyes, dry mouth can be managed with some generics on the market. Do you feel like you would potentially get some of the pushbacks in terms of the commercial landscape? That's one. Two, do you think as you think about your development strategy, would you actually mean skew more towards the, you know, moderate to severe rather than an all-comers study?
That's something we'll figure out in the next weeks or months. We really have time for that. What we hear consistently is that there is not enough to help the patients. Yes, the market will need to be shaped, but the unmet medical need and the demand from the treaters and the patients is very high. That's also a reason why a competitor has launched two phase III in this indication a few years ago. We're not concerned by that. There will be a way to address the patient while doing good business in this indication. You can somehow compare it to systemic lupus, but lupus is a bit more difficult to develop, more busy. This one is more, I would say, a white space. You mentioned that. With great results, we'll have a strong value proposition and we'll be able to extract good value creation out of this indication.
It's very large.
Yeah, it is a large, high indication, I'll say, with nothing approved. From the headline numbers I think the RemeGen team put out, it really does seem like BAFF in disease compared to the, even the data we've seen from Novartis on their BAFF inhibitors as well. Congratulations to the team here again. I know we talked some about MG and the Sjögren’s. Could you just share your general vision beyond these two indications? How are you thinking about the future of telitacicept?
I think we'll play it by ear. We have the incredible advantage to have so many data already from China in other indications. The question will be how we deploy capital and how we stay disciplined with indication choice and timing. You can easily imagine that we would turn into like some FCRN companies who have compounded. Remember that it took time with the first two indications of the leader there. It started to go with another one, and it opened up. There was a mix of proof of concept, discipline. We have an advantage because we already have data, very robust, high-quality data coming from China. That gives us a decision power, which is a bit unparalleled.
That's one of the great things with China innovation because you get this wealth of data, almost like a gift, which you could not do if you would be de novo in our own geography because you just need the time. We would be in phase II right now in MG. We just start ourselves and we would not know what it would be on a big sample. Here we have, I mean, the question on China is actually how do we deal with all this great data, right? How do we keep a little bit of cool and say, hey, we still need to do the work here and comply with the regulatory authorities' demand?
Maybe just follow up on that. I agree with the future of this particular asset. A question I think a lot of investors wanted to understand better is what are the key milestones that are going to come up in the next 6 to 12 months? A little bit of shorter term, but do you mind just to kind of outline that for the investors?
Definitely a very important question. We have a very clear roadmap for the next 6 to 12 months. Number one, we will continue to execute on our MG global phase III trial, meaning recruiting. It's going very well. Qin is in charge of that. She's doing a wonderful job. Qin was actually the Chief Development Officer of RemeGen. She's been transitioning with her team to us from the U.S. She was running the global trial before we did the deal, and we have a continuity advantage here as well. We continue to execute on that, and we will prepare for the filing. We're already thinking about the way we'll do that, but it should be pretty straightforward. The number two opportunity we just discussed at length is preparing for Sjögren’s. It's even a bigger opportunity. We're opening a very big chapter here, a bit more uncharted territories.
The great thing is that we have another company, a large pharma, who is doing it. They will kind of clean the air a little bit for market access, all the potential challenges that Dallan, our Chief Commercial , will need to work on. That's great, actually. We will not be alone in front of well, establishing a huge indication like that. It's like if Sanofi Regeneron would be just like a small biotech trying to establish Dupixent. You need a bit of muscle. We're not from the last train, but we also have the, I would say, the environment from a big pharma doing that as well.
It's a goldilock of a scenario, right? Like you have someone to kind of plot in the white space for you a little bit. I do think Novartis has left a lot of efficacy on the table for additional agents too.
Music to my ear. That's absolutely true if you look at the phase II. I can tell you, bear with us for the phase III. It's very good. Very strong, best in disease. We have an advantage. We'll reproduce it with our own study. It's very strong. We have the possibility to be the leader in this indication. Starting this study with the protocol, which is already approved by the FDA, is a very strong milestone in the next 12 months, I would say. I would mention the flurry of data, which is coming up in the next couple of weeks and months. We have, number one, the Sjögren phase III data from China. Very expected. I don't think you'll be disappointed. It's coming at a conference in a couple of weeks, a major one.
We have very important, we talked about the need to ensure sustainability and disease modification in myasthenia gravis. We have long-term 48 weeks data from our China phase III in MG. It's coming also at the conference. I think we can name the conference. It's the AANEM, yes, in a couple of weeks in San Francisco. You'll see the durability of our asset and the great controlling of the primary endpoint and secondary endpoint over time, almost a year. Super important and differentiating. Very also important, we didn't talk about it, but we will have IgAN data from a phase III in China. A 24 weeks data, which will be presented at a nephrology conference in the fall. It's another proof of concept of our spectrum of possibilities.
We'll see what we do with it, but expect that as a catalyst for proof of concept and showing the consistency of data across the spectrum, both for efficacy and safety. I'm not sure if I'm forgetting anything, but it's already five catalysts, which are pretty important in the next few months.
Got it. These are all, in a sense, a larger commercial opportunity, right? Maybe I think another question a lot of investors could most likely be interested in is, what is the kind of the financing strategy around it? What are we thinking about some of the financing strategy to support our ambitions within the clinical development and eventually commercial?
Yeah, as of now, at the end of the quarter, we had about $199 million in cash. I think we're fairly well set to carry out the phase III for both myasthenia gravis as well as Sjögren's. We'll be opportunistic in terms of how we are thinking about adding funds. At this point, I think we're in a fairly good position for what we're projecting.
Thank you so much. In the cognitive time, I just wanted to open up to the audience here and see if there are questions from the audience to the award team here. Go ahead.
I just have a question. How are you working on that?
We don't work with Ousia simply because RemeGen is our manufacturer for our drug substance. They have significant capacity, as you know, almost 150,000 L . Most of the drug substance comes from there, but it's very stable. It's almost stability for four years for the drug substance. We're in a place where we think that we can stockpile a lot of it in the United States, which is where most of our finishing occurs for the drug product. Thermo Fisher does that currently. The supply chain we think is very secure. We will have to stockpile in case there's any bumpiness to ensure supply assurance. The cost of goods is fairly manageable from our perspective. The ability to manage it with our partner is something we feel very comfortable about. We don't think any bumpiness should occur from our situation on supply.
Maybe actually just double click on that. The geopolitical is one of the key themes of some of the recent China-U.S. partnership discussions, et cetera. Do you feel, do you like in a way, this is a successful partnership? Do you think that could potentially affect how you think about your future collaborations with them? Assuming, you know, how you potentially build additional pipeline on top of teli as well?
Great, great question. No, I mean, I don't have a crystal ball on what could happen in terms of geopolitics, although I have a bit of an observation tower with my board seat in this big pharma. No, and it's back to one of the things I said earlier. RemeGen is a large company, public company, so there is a lot of transparency. A lot of things are being disclosed, like here. It's very mature. I wouldn't say it's a geopolitical topic, but it's also a way to be a bit in the dark. We don't have that. Sandy described the supply chain, which is very effective and clean for us and optimized. I think we have a good view of the things.
When we did the due diligence with our investors, world-class investors, by the way, I didn't mention that as a reason for me to join in and join in the deal in the company. We really looked at these things and we felt confident also with all the legal advice and everything that we were in good standing here together with them. We'll see. I think there is so much happening between the two countries. You've seen the big announcements recently with massive, mostly oncology deals, with competition between financial institutions and Big Pharma to get the assets. I think it's almost too big that there is no solution that can be figured out on a global way because it's beneficial to both countries, actually. The innovation there, the capital will flow back to China. Here we can also generate huge value creation.
This deal helps us to bypass a lot of steps because we leverage a pretty low cost, actually, something which already has been matured very much in China. We can complement things. Definitely one thing that the larger pharma companies in China have not cracked is how to launch a drug globally. This is where we come to the equation here in the U.S. This is how it's virtuous.
I know we're coming up on time, but just a last question for my own curiosity, maybe a little bit of, you know, to your point you made earlier. Historically, most of the China assets deal are centered around oncology. Do you actually see more and more, you know, deals coming out of China assets around autoimmune as well?
There is a lot of pipeline, you know, to immune in China. I've seen some charts recently where it's as many as oncology. Immunology in general, not only autoimmune, but immunology is a big chunk of the pipeline in China with great innovative modalities. I mean, OX40s and all these things. I mean, future biospecifics or trispecifics. Yes, definitely there will be a wave of immunology deals. Big Pharma is looking at these immunology deals. We saw a lot of oncology deals because there has been the super fashion of the VEGF1, PD-L1 deals recently because of the size of the opportunity and the fear of missing out of several organizations. Although it's too busy. You mentioned busyness in MG, but I mean, that's really busy, right? That's a really busy factor there. I'm glad we're not in that. It's big numbers, but it's easy to be dwarfed.
I think we have a superb opportunity because we think we are, I think we are leading the wave in immunology and certainly in autoimmune diseases with China innovation and leveraging the U.S. wonderful ecosystem.
Thank you so much for the board team to be here with us today. Really appreciate you guys joining us.
Thank you very much for having me.