Hey, good afternoon, everyone. My name is Jess Fye. I'm the Large-Cap Biotech Analyst at JP Morgan, and we're delighted to be continuing the conference today with Moderna. Good news this year, we don't all have to switch rooms for Q&A. Stéphane's gonna give the presentation, and then we're gonna go straight into Q&A after that. A couple ways you can ask a question. There's mic runners in the room, so if you wanna raise your hand, we'll try to get a mic to you. Alternatively, you can submit a question electronically, and I'll read it off the iPad up here. With that, let me pass it over to Moderna's CEO, Stéphane Bancel.
Thank you, Jess. Good afternoon, everybody, and thank you so much for joining us today. It's quite an impressive room for this presentation. Before I start, let me remind you that we'll be making forward-looking statements that investing in Moderna entails some risk that you can find on the SEC website or on the website. As you all know by now, mRNA is like software. As you know, computers use a binary system to code any piece of software where life across species on the planet uses a quaternary system to code for any protein. That makes mRNA an information molecule. It's this belief and this understanding that led us 10 years ago to build this company. We believe that the key main advantages of mRNA versus small or large molecule are very profound in what they can mean for patients and for creating value.
The first thing that's very exciting to us is a very large product opportunity ahead of us, not only this year or next year, but in the next 10, 20 years. A very large wide canvas to paint on. We can do secreted protein like the biotech industry, but what excites us the most is we can do transmembrane protein, those protein on the membrane of a cell. We can do intracellular protein, including protein inside compartments of a cells. Like one of our rare disease program is inside the mitochondria that we're getting the protein in. A very large product opportunity. We can make very complex protein. In the transmembrane, you will see one of that is actually made of five protein, for which we make five mRNA. That is actually in phase III right now, this is not science fiction.
That's something we know how to do. We can combine mRNA. We have program when we have two, three, four, five, six mRNAs in protein, going up to 15 mRNAs in one dose. The flexibility we have to do the right biology to get a drug to work is very profound and very different from small or large molecules. The other piece that has excited us since the beginning, and that excites me a lot, having been in large pharma before, is the belief that we have that this technology will lead to higher probability of technical success of drugs getting to the clinic and drugs getting to market. Why is that? It's because we always use the same chemistry. What we inject in the human body is the same for every product.
We believe this is gonna have a massive impact in change of probability of technical success. This is the industry average that a lot of people are using to assess assets chance to get to market. The first piece that we believe that I think we have demonstrated a couple times is the ability to go at a very different speed in term of cycle time of development versus small or large molecule. Why is that? It's because we always make the same product using the same processes. In large pharmaceuticals, you have to invent how to make the product every time the team in the labs have a cool new molecules.
Well, in our case, when the teams in the lab have a molecule they want to take to a clinic, it's the same manufacturing process than the other molecule, allowing us to go really quickly, sometime in 30, 60 days, from a candidate to starting a clinical trial and filing an IND. Last but not least, because we use the same manufacturing process for everything, we use the same factory with the same people in the same room using the same CapEx. Literally, one week we can make a COVID-19 vaccine, and a week after, in the same room with the same machine with same people, you're gonna make a flu vaccine or a rare disease drug or a cardiology drug. The flexibility that gives us the efficiency of capital is really profound.
Based on those beliefs that we had 10 years ago, we set up a very different vision and strategy for how to build this mRNA company. We kind of threw the biotech playbook by the window because it was a different science. We believe it was not the right way to build an mRNA company than it was done for real technologies. The way we thought about it is that if we invest in technology, mRNA technology, delivery technology, and for an application that we call modality, we can make the first drug work safely in human. The next drugs and the next drug in that same application is like copy and paste. Use the same chemistry for the mRNA, use the same lipid, the same route of administration, the same manufacturing process.
You just change the order of the sequence, and here you go again. You could do that across many applications. That's how we set up to build Moderna. What is interesting is inventing a new application, a new modality is difficult, it's time-consuming, and is expensive. Why? 'Cause you have to figure out how to deliver the mRNA to the new cell type that you want to have a biological effect. That's hard. Then you need to deal with immunity. How do you make sure that the immune system doesn't react in the way that you don't want about your product? Then, of course, all the translation.
What is quite interesting in the last 10 years, but even more interesting now that we have $18 billion of cash and 4,000 people in the company. I believe that Moderna is uniquely positioned to continue to lead in the space of mRNA. First, it's all we do. Two is we have critical mass in science. We have digitalized the company to allow speed. We have the capital that we can invest in science. What we have done over the last 10 years, as you can imagine, we've been a bit distracted with the pandemic, we have developed seven modalities, seven applications that you see on this slide that all have drugs in humans. We have many more applications or modalities that our teams are working on in the lab that we hope to be able to introduce if the science is positive.
I won't have time today to walk you through all the programs, there's 48 of those, through all the modalities. I'm going to pick a few, and those I'm not going to cover are in the appendix of this deck that you can find on our website. Let me start by the first modality, infectious disease vaccine. We think there's an incredible opportunity to have a massive impact on human health through mRNA technology for vaccines. There are more than 200 viruses documented by scientists that hurts human. 200. There are vaccine against around 20 of those. That's a lot of white space. I want time to cover everything, but let me start by the big vaccines that we have a very active pipeline on. First, respiratory viruses. You are aware, of course, of SARS-CoV-2, of influenza.
Now RSV is becoming more popular because it's doing a lot of damage. You can see on this slide some of the leading respiratory viruses that circulate in the U.S., in Europe, around the world every year. You can see with the red those viruses that do not have a vaccine approved on the market. Our vision for respiratory viruses is quite simple. We want to develop vaccine against all of those viruses. We want to combine them, so we don't need three or four shots every fall. We want to adapt them to the variant circulating in the country where you live, not a single variant for the entire planet, which we believe some years doesn't make scientific sense. That's a bit the opportunity we're trying to address. Let me say a couple words on COVID.
We reported this morning $18.4 billion dollar of sales for the year. Those are, of course, at this stage unaudited. We confirmed that we have at least $5 billion dollar of contract already signed or deferrals from 2022 into 2023 that we qualify kind of as a floor in terms of sales for the year. In this $5 billion number, you have some countries like Canada, U.K., and so on, but this number assumes no sales in the U.S. Of course, we are working actively with the channels in the U.S. to provide the updated 2023 booster into U.S. pharmacy, hospitals, and doctors. No new contract in Europe is assuming the $5 billion. No new contract in Japan, Middle East, and many other parts of the world. RSV.
We're very excited about RSV because how much damage it does to the community every year. We've announced that our phase III study, 36,000 participants, is fully enrolled. This is a phase III study that's a registration study looking at safety and vaccine efficacy. We announced this morning that we crossed the number of cases, it's a case-driven study, 64 confirmed cases. The requirement was 42 for the first interim analysis. We should expect it very soon, and I'm talking weeks, not months, the vaccine efficacy coming out of this. On flu, we are running two phase III studies. In the Southern Hemisphere, fully enrolled, looking at immunogenicity because some countries told us that we use that for an approval.
In Northern Hemisphere, efficacy study, 22,000 participants, that could come as early as this winter given the number of cases that has been happening across the world, in the fall and this winter. Let me now pivot to another family of viruses, latent viruses. Those are viruses that once in our body, never leave our bodies. They create short-term, healthcare damage, but some of them create very important long-term damage. I won't go through the list, I don't have the time, but same thing on the right side, you can see all the viruses for which there's no vaccine on the market. CMV is currently in phase III. CMV, cytomegalovirus, is the number one cause of birth defects in this country and around the world.
1 in 200 kids in the U.S. is born every year with CMV disease. The phase III is 40% enrolled, and we are announcing that we are now also enrolling in Japan. We believe the CMV opportunity is $2 billion-$5 billion annual sales for this product, given there's nothing on the market. As you can see, we believe that we can build a very important portfolio for patients and on the way to create value for investors across the different vaccines we have in the pipeline: CMV, EBV, herpes, VZV, and also HIV. That's a quick run by vaccines. I wish I could spend more time on vaccine. You guys can join us on our vaccine day in April to learn more. Now Modality 2, cancer vaccines.
As many of you know, in December, we're very excited to share some positive data in a combination with KEYTRUDA, with a personalized cancer vaccine. This study was powered. The control arm was KEYTRUDA monotherapy. The other arm was Moderna personalized cancer vaccine plus KEYTRUDA. We're very delighted to show a hazard ratio of 0.56, meaning a 44% reduction in risk of recurrence or death versus getting KEYTRUDA monotherapy. You can see the p- value. It's the first time with mRNA that any company has shown you a randomized clinical study positive outcome. As you know, cancer vaccines have been, for the last couple of decades, a graveyard of candidates. We're very excited what this could mean for patients. Because the mechanism is teaching T-cell how to recognize epitopes from the cancer of every individualized patient.
We think it's applicable to a lot of other tumor types. with our colleagues at Merck, we're actively preparing a phase III study that could start as early as this year, and also planning several additional phase IIIs in indications where KEYTRUDA immunotherapy works with the same belief that we could improve the response versus KEYTRUDA alone, combining KEYTRUDA and monoclonal drugs. we're gonna be expanding and testing that technology in many, many tumor types, and potentially also going much earlier in disease progression, kind of watch this space. We're gonna be investing aggressively in that space. I'm skipping a couple modality for lack of time. Modality 5, we're very happy to announce this morning a very exciting program in cardiology for chronic heart failure. As you know, it's a massive medical problem around the world.
Just in the U.S., 1 million hospitalizations every year. The molecular vaccine is really interesting because it's a naturally occurring hormone in humans. It's used actually by pregnant women during pregnancy. We believe that we can code that molecule to help people with heart failure. We've announced this morning that we've initiated a phase I study in patients, we'll share with you data as we learn more about this program that could move pretty quickly if a phase I was positive. Another important Modality 6 is rare genetic disease in the liver. There are a couple of those in rare genetic disease in the liver that cannot be drugged using small molecule or using recombinant. Why? Because the kids are missing a protein inside the hepatocyte of their liver.
We developed technology, IV injection going into the liver, delivering it to what we believe is the hepatocyte, the mRNA molecule. We now have six patients years of experience on drug. Some kids have been more than a year with dosage every two weeks. It's generally well-tolerated to date, and we're already seeing at low dose a reduction in number of metabolic decompensation event, which the regulators have deemed is the endpoint for the pivotal study for this program. We're monitoring this. We'll update data as we have more, but we are very excited because if this works, it would unleash the entire modality. As we all know, in rare genetic disease, the biology risk is very, very low. Another one that we're very excited about, which took a few years to make work in the labs, is the ability to deliver inhaled mRNA to patients.
We partnered with our colleagues in Boston, Vertex, to work on developing a CFTR mRNA program, coding for the entire CFTR gene to help those kids that don't respond to the standard treatment, where Vertex has a massive impact on so many lives. There are still several thousand kids that don't respond to treatment. What we believe is that if we're able to give them a full length CFTR protein in their lungs, we could basically restore their lung functions. We developed that technology. We're very excited that the R&D was opened by the FDA, that this got a fast-track designation. This morning, our colleagues at Vertex announced that we've already started dosing in human, in patients, of course. This should be able to go very quickly because those kids have no hope. They have no option.
If you think about it, if it works in this application, then we can open a brand new space, a new modality in lung disease. Think about all the lung disease that we could go after inhaled mRNA. That is extremely exciting for us. This is a typical slide we use to just give you a quick summary of where we are as of January 2023. I won't read everything. Just to tell you, we have now 48 programs in development. 48. The company is close to 4,000 people, and our budget calls for adding 2,000 employees this year because we are scaling research, we're scaling development, we're scaling manufacturing, and of course, we're scaling commercial. As of the end of the year, we had around $18 billion of cash. How do we intend to deploy that capital?
Our number one priority, and we have been saying that for a couple of years, is to invest in the company. We spent 10 years to develop a platform that could scale. One of the biggest challenge of pharmaceutical companies is what is next in your pipeline. We have an information-based molecule. We build a platform around it. With the management team and the board of directors, we understand our science. We believe our ability to scale. What we announced this morning is the board-approved budget for 2023 is actually $4.4 billion of R&D investments. Many phase III, as I said, in cancer vaccine with our colleagues at Merck, which is a 50/50 cost share and profit share, we're going to be very aggressive. We've also announced we bought a priority review voucher, which now makes...
We have two of them, and we have two phase III that should read out, flu and RSV this quarter. Those two can be handy to be able to move very quickly towards approval if the results are positive. In term of partnership, we do not believe we are on everything being able to the best science in the world. We believe there's amazing science happening outside our walls of our labs. We want to tap that technology. What we have done last year, you can see the top line, Metagenomi OriCiro, and already this year, actually last week, announcing two new partnership, an acquisition in Japan, and also a licensing agreement. What are we trying to do with those partnerships? We're trying to expand the size of a mRNA operating system.
We want to provide our therapeutic area with more and more technologies to be able to do more and more drugs using mRNA. We believe there's so many things that we are still learning and exploring to keep expanding the mRNA operating system. OriCiro is a good example. Small private company in Japan with amazing new technology. They figure out how to make plasmid, which is the raw material to all the mRNA we make, without using E. coli. Synthetic, cell-free, enzymatic reaction. Huge impact in term of speed, scalability, and purity of products. That will help us across the company. Using that technology, we can go faster to a clinic for personalized cancer vaccine, which could have an impact on efficacy and more people responding because they are dying of a cancer.
It can help us in the labs to go faster from idea to data, to just expand the mRNA operating system faster, to get into the clinic faster. Think about when there's a new variant. The FDA told us on June twenty-eighth they wanted the BA.5 Omicron variant. It was in pharmacy on September second, we spent all of July waiting for the plasmids, GMP plasmids. This technology will allow us to shed that time by maybe half. That's gonna mean a lot of impact for patients. The excess cash, we will return to shareholder. We already announced two share buybacks. In 2022, we bought for $3.3 billion dollar of stock at an average price of $143.
There's still $2.8 billion of remaining capacity in the current plan. The board will continue to review as appropriate the use of cash between investing in the company, which is our number one priority, doing partnership to expand the OS, and the excess to return to our shareholders. What is exciting for me is how the company is accelerating. The biopharmaceutical world is an analog world, where companies have drugs that are all different. In our case, we have a platform. If you look at some of our historical data, at the end of 2018, no commercial product of course, 21 product in development, mostly early stage. The end of 2020, commercial product, 25 products moving to a later stage. 2022, because in 2020 we are kind of pretty busy with pandemic. 2022, 48 program in development.
Four program in phase III, nine program in phase II. You can see how the pipeline is expanding into new modalities and is accelerating because we have a capital to do so. If we invest $4.5 billion in R&D this year, what do you think it's gonna look like in two, three, four years from now? I want to be quick on my slide. Our R&D budget in 2019 was less than $500 million. If you think about it, this picture was generated with much lower R&D budget than where we are now. $4.5 billion is around 10x. The R&D investment we're gonna do in 2023 versus what we did in 2019. This is gonna drive a lot of change in our pipeline, and I think a big impact on patients.
Because of where the company is and the growth of the company is, we spent quite some time in the last few months to think about where are we heading. What you see on this slide is our original mission, the one we set up when we started the company. Our goal 10 years ago was to figure out how to make mRNA work because we were convinced that if we could make one drug work, because mRNA is information, we could do a lot of drugs. What we could do for society and patient over the next five, 10, 20, 30 years could be transformational. Potentially as big, if not larger, than what recombinant as an industry has done over the last 40 years. Where we are today, we know mRNA works.
We thought about what's our next 10 years journey, what's our mission, what should drive us in the morning? What you see here is what we think is really important to who we are and what we want to become, which is as a team, we want to deliver the greatest possible impact to people through mRNA medicine. What we're optimizing for is the maximum impact we can have in the next five, 10, 20 years on the world, thanks to this amazing science. I hope you will join us for some of the key event this year, and I would like to thank you for your attention. I'll be happy just to take any question. Thank you.
Great. Thanks for that presentation. Just maybe starting out with a bigger picture question. Can you talk about how you think about the future as we move toward treating COVID as an endemic disease and as the U.S. shifts towards a commercial model?
Sure. Like everybody, you know, I've never managed a transition from pandemic to endemic, so we are learning as we go. We're trying to use, you know, other viruses, other disease to kind of help us think about it, at least directionally. We think that flu is a pretty good model for where we think things will end up. The need to update the products regularly based on the biology, potentially different booster in different geographies, which we can do. We're trying to really help us by even building plants around the world, you know.
I don't have to talk about it, in this presentation, but we're building a plant in Canada, we're building a plant in Melbourne, we're building a plant in the U.K., where we've had 10 years agreement with those governments, 10-year supply agreement that have been signed to basically procure from Moderna respiratory vaccines that we can adapt with the local authorities to what they want for their people. I think for the 50 and above, 50 years old and above, you know, people that have, you know, immunocompromised, seriously sick, the need for an annual booster is gonna be important. I don't think that's the only population. Now, the example I use, you know, is I've done a flu shot over the last 20 years.
You know, we provided the flu shot at Moderna since the last 10 years, every year as a benefit, like many companies do. Have I taken a flu shot? Now I'm 50 now, so since I was 30, because I thought I was gonna be hospitalized or dying? No. They want to be sick, they want to give it to somebody else. I think a lot of people are gonna do that as well. Do I believe everybody's gonna do it? No. I think if you look at the 50+ and people at high risk and people who are just gonna want to be protected, I think it's gonna be an interesting market in term of size.
I think as we get more and more combination, you know, we're trying to work on a COVID plus flu, COVID plus flu plus RSV, because nobody really likes needles so much. When we talk to payers, they are very worried as we get into a COVID plus flu and soon a flu plus RSV, is how we ensure compliance. What they want is if those vaccine exists, they want people to use them, especially people at a high risk that might end up in hospitals. Through the discussions we're having with payers, with private payers, but also health ministers and so on, we believe that there's a very, very important demand out there for combinatory vaccines, and that's exactly where, as you know, we're going in term of pipeline and strategy.
I think combinations, and I think the customization of products by geography is gonna be driving a big competitive advantage.
Okay. You touched on this a little bit, but you've got some other viral vaccines in development where there is a bunch of competition and some other ones where there isn't a bunch of competition. How do you decide where to go? Like, what's your kind of overarching...
Yeah.
-infectious kind of vaccine strategy?
Sure. Let me start by the easy ones. If there is a good vaccine out there, we most probably are not gonna do anything. A good example is hepatitis. You know, there's a good vaccine for Hepatitis A and Hepatitis B. We've never even talked about should we do an Hepatitis A or Hepatitis B vaccine because we don't see the medical need and how much value we could bring, and given other things we could do, we'd rather do something else with our talent and our capital. Respiratory is different because first, we believe we can get a much higher efficacy on flu. We don't think we get that done on the mRNA-1010, the first flu program, because it was not designed for that. It was designed to be non-inferior to commercial flu products to go to the market quickly.
We believe a COVID plus flu, COVID with, you know, Moderna's product has shown the best protection against hospitalization. If we have a non-inferior flu vaccine, we think that we'll provide a lot of value to the healthcare system. That's what we want to do first. If you think about flu, there's a lot of things that can be done with flu with mRNA to increase the efficacy, because as we all know, the current vaccines don't have a great efficacy. We can do HA antigen and NA antigens to actually improve, we believe, the efficacy of a vaccine. H3 strain is the most important one. Why? It drives 90% of hospitalization. Why only pick one H3 strain, which other technology are constrained to do because they cannot do more? What about doing two or three H3 strain in a single dose?
You look at the different strains circulating around the world, because it's really hard to guess where the virus is gonna be in a few months when we have vaccines in pharmacies. We think we can use the technology in quite an interesting way to develop different products. You know, I even been talking to a country that I won't mention because it's a discussion with sovereign, whose public health experts and health ministers say, "Hey, should we put pandemic strain of flu in the flu product for our country?" Think about like an H5, and then the year after an H7, and the other an H10, because you could create some herd immunity so that if there is a new pandemic with flu, this time not the coronaviruses, you could have a bit of herd immunity and you could rotate that every year.
There's a lot of ideas that you could have using mRNA. If you design the product a bit like it's done in the tech world, which is start by the problem you're trying to solve, and design the product backwards. In biopharma, people have one molecule, and they're trying to do the best they can with that molecule and to find a way it could be used. The way we design drugs is very different. We start with the medical problem, the science, and we move backward to say, "What do we need to do in term of components so that the product has a high chance of working?" Like CMV. Most pharma company doing vaccine have tried the CMV vaccine because it has been the number one priority of the National Academy of Sciences for 20 years.
Some vaccine went into phase II, but they failed because efficacy was too low. Why? They only coded for the gB antigen. It is well known by scientists that the virus can also get into human cells via the pentamer. It was believed in the industry that if you don't have a pentamer, you have no chance. A lot of pharma company, because they only could do the gB, 'cause the pentamer, as the name says, it's five protein that have to come together. Good luck with our recombinant to do that. They still went into the clinic. These are type of things we will not do because we have so many other things we can do, but we think the science makes sense to increase the chance of a drug to get to market.
Okay. Yeah, we have a couple of questions from the audience here. Can you talk about how Moderna thinks about original antigenic sin or immune priming with respect to COVID?
Okay. I think the question is really around the different populations, because what I think is quite unique about this virus versus other virus that we are dealing with in the respiratory space is most of us, and I think I can say all of us in this room, have never seen the virus as young people. Which is why I think people who think that the need for boosting in the elderly won't be there, I just don't understand the science. We believe that because we have not seen, again, the older ones in the room, we've not seen this virus, you know, in our early years of life. Especially in our teenager years with a very strong immune system and memory, that the need for boosting the subjects are gonna be very important.
The next one here is, it's got a statement and a question. Moderna's technologies can change how we do gene editing. You established a great collaboration with Metagenomi. Can you tell us more about how that's going, and what your plans are to go to tissues beyond the liver with LNPs or other delivery systems?
Sure. I think there's two questions in there. First is about increasing the application of mRNA. As you saw, the seven modalities use different lipid or different route of administration, like the lung, the liver, and so on. We're investing, you know, large amounts of money to invent new modalities to go to new cell types. We can do that for either using mRNA to code for protein as a therapeutics or going to the part of the question around gene editing. We can use the mRNA to code for an enzyme that's gonna do gene editing. While we have all been excited about, you know, CRISPR-Cas9 system, because I'm not aware of any technology that human has touched, where the first version of a technology is the best one.
We think that's just the beginning of a very exciting time for science and biology to find different gene editing systems. That's where kind of Metagenomi comes into play, where we want to figure out for different cell types, potentially for different jobs to be done by the enzyme, where you might want to use different enzymes. We believe that's one tool to do everything might not how you build a house. We are very long in genomics. You know, we have a group that soon is gonna be up to 200 people. They are leveraging the entire Moderna infrastructure, so they design mRNA for new gene editing enzymes, and it goes to the same robot that makes everything else that we use for the portfolio.
We can move things to the clinical so very quickly because the idea here is the same, is using the same delivery system, using the same mRNA just to do gene editing that's used just making human protein. We think that if you look at the level of investments we are making in gene editing and the infrastructure we have at Moderna in research, development, and GLP manufacturing, that potentially, you know, in a couple of years from now, we're gonna be actually a very large player in gene editing.
Okay. maybe switching to PCV. Obviously, we got the encouraging top-line results. Can you talk a little bit about where you see this product going, whether it's what tumor types or what other settings?
Sure. Given the data, and how we think it's a very profound impact, and as you know, the study was powered and randomized and kind of where the p- value and the hazard ratios are and so on, we and our colleagues at Merck are really excited about the technology. We've shown and presented ASCO in 2019, I think, or 2018... 2019, I think. The fact that we were able to get T-cells that did not recognize epitopes coding in our products by taking the blood of cancer patients before dosing our product and taking their blood after several injection and showing that then the T-cell recognized the epitope we know we coded in our products. We think that we have mechanism of action, now we have clinical outcome.
We believe this should be applicable to a lot of tumor types across the board. We're gonna, of course, start where KEYTRUDA works, but we've had discussions with our colleagues at Merck to say what type of tumors will actually KEYTRUDA did not work, but it might make sense to go back because maybe by combining personalized cancer vaccine to KEYTRUDA, you might get the immune system to the right place where you can have a clinical impact that is beneficial to patients. We want to also go earlier in disease, we want to go later in disease. Could you think, and now I'm kind of brainstorming for a minute, it's not forward-looking statements.
Could you, for example, with the improvements in liquid biopsy, could you see a world where you try this combining with products like GRAIL and others, where you basically, through blood work, figure out some mutation that you got in the vaccine, that you basically give very early on? Because we really believe, given what is known about immunology, that getting younger patient and much earlier in disease should lead to better outcome because of what happens to the immune system through the disease progression. We're gonna be creative, we're gonna be bold in terms of how many things we do. The great thing with Merck, as they've shown the world with KEYTRUDA, they will not try a lot of different studies at the same time.
I think that experience, also what's happening with the product patent expiry that they have on their hand, and our belief in the science of mRNA and the balance sheet that we have, I think you have two companies. You know, I spoke to Rob Davis, the CEO of Merck, several times, in the last few months. We're both very eager to be really bold about what we think this can do for patients and, as a consequence, creating value for shareholders.
I think you mentioned you have enough events in the RSV trial to do your first interim?
First interim.
What are the kind of scenarios here if you hit or don't hit on the first interim? What happens next?
Sure. If we don't hit on the first interim, then we keep going. The study is ongoing, and UN will be for quite a while to monitor people for much more than the 12 months required for filing. Because of how the statistics works, it's not because we missed the first interim that necessarily we have a bad efficacy. If we hit it and we have vaccine efficacy, I think the thing we're gonna be looking at is first, what's the efficacy against, you know, disease? Because the vaccine that have published data so far, I think have a very good, you know, 80-ish% protection against hospitalization. I think, or I hope it...
One could do better on the efficacy against disease, because anybody who gets a vaccine will hope, of course, not to get hospitalized, but we hope not to get sick. I think a prevention of disease is also gonna be important. Then there's a tolerability profile. One of the vaccine that has already got data out there is using a pretty strong adjuvant. So those are the three things I think we're gonna be looking at: VE against severe disease, VE against mild disease, and tolerability.
That comes very soon?
That should come very soon. Like I said, days or weeks, not months.
Okay.
I mean, the team is analyzing all the data and so on, so when everything is good and QC'd, we will release it.
Okay. you've also got a number of efforts ongoing in rare diseases in addition to vaccines.
Yes.
in addition to oncology. Where do rare diseases fit in kinda Moderna's strategy?
I think it goes back to the same theme, which is we want to use this technology to help patients. If you think about those rare genetic disease in the liver, those kids and those parents have no hope. They have an enzyme that you and I have that they're missing, and every time they just get a runny nose or get a viral infection or something, the parents rush to the hospital, the kid's in the ICU, and they might lose their children. That's the standard of care today. They try to diet and other things to minimize the risk, but every decomposition event could lead to death for those childrens. Because of some of those diseases leads very high level of acid, you have a lot of brain damage every time you have an incident.
The way it fits the strategy is we can help patients because the biology risk on rare disease is very low. If you think about cancer or HIV is through the roof, but rare genetic disease that have single mutation are pretty low biology risk. If we can do it once to get mRNA into the hepatocyte of a liver, then the next time we just change the sequence and we go again. Could you get a couple dozen drugs? Some might be, you know, $1 billion, some might be $500 million, some might be $200 million or $400 million because of the size of the market.
If you don't need to build any plans for it, if the drugs are gonna be developed very quickly, if once you have them on the market, it's gonna be very hard for anybody else to come behind us in terms of doing a study 'cause it'd be unethical to take kids out of a drug working. Until genomics really works really well, you might have those kids, you know, forever, and then, and the newborn kids with those disease. As we work on genomics, we might be able to go back to offer our parents and doctors and the patients either a repeat dosing, like every two weeks, like enzyme replacement therapy, using mRNA to code the protein like we're doing today, and/or propose a gene editing solution because we know how to get into the same cell type.
Mm.
That's a bit how we see the strategy. A bit like you see Vertex, we talked about it, you know, going back with an mRNA solution to treat the patients that they could not treat using the current products. We're trying to really obsess about patients and the families and say, "How do we help them? How do we create the right product or the right products for each disease?" Then we think we'll create value on the way because, again, small clinical trials that are pretty quick, no plans to build. If you think about just the economics of rare disease for Moderna, it's actually extremely attractive.
Okay, great. Well, we're out of time, so we'll leave it there. Thank you.
Thank you.
Sure.