Moderna, Inc. (NASDAQ:MRNA) Goldman Sachs 45th Annual Global Healthcare Conference June 10, 2024 1:20 PM ET
Company Participants
Stephen Hoge – President
Conference Call Participants
Salveen Richter – Goldman Sachs
Salveen Richter
Great. Good afternoon, everyone. Thank you so much for joining us. Really pleased to have Stephen Hoge, President of Moderna with us this morning.
Stephen Hoge
Thanks for having us.
Question-and-Answer Session
Q – Salveen Richter
After having just announced some news today on the COVID-flu combo, perhaps before we get into that, some big-picture questions, Moderna has over 40 programs in development. And in the context of R&D spend management, what is your strategy for pipeline prioritization?
Stephen Hoge
Great question. So, well, thank you, first of all. It’s really exciting to be here, the first time in Miami with you too. So, first on the question of prioritization, as you know well, because you’ve followed us for years, we are a platform company.
And so we actually start with the question of what can our platform technology, which in our case is messenger RNA, what can it do? And we don’t sort of say what we’d like it to do, we say, what are the pharmacology that it can do now. And as we’ve articulated, including our last R&D Day, there are really four pillars where we think our technology is mature and we’re scaling, we call them modalities.
The first is respiratory vaccines. The second is vaccines against other viruses, so latent viruses, and other infections. The third is our work in oncology, principally around our INT program with Merck. And then the fourth is rare diseases, so expressing proteins in the liver. And in those four modalities, we’re advancing anywhere between five and 10 medicines. Because we’re a platform company, we really think it’s important that it’s multiple medicines in those modalities because the probability of the success of the first one might be not that different than other technologies, but the second, third, fourth, and fifth can be quite high.
And so what we try to do is advance a broad portfolio across those four modalities and over time add more. Now when it gets down to the question of how do we pick within those modalities, a lot of that boils down to unmet need and where we see the opportunity, generally where there are things that we think our technology can uniquely do, like in rare diseases or cancer or in respiratory combinations. We will prioritize those and advance them forward. And that’s one of the most difficult parts of my job often as the Head of R&D is working with the team to say we can’t quite prioritize that program now and we hope to get it in the future.
Salveen Richter
And you had a collaboration recently with Blackstone Life Sciences and a funding agreement to kind of move the flu program forward. Just walk us through the strategies here for project financing and partnerships on the forward.
Stephen Hoge
Yes. Well, that becomes one of those examples of where we have the — fortunate to have a very strong balance sheet, $13 billion of cash as a result of the work through COVID. And we are translating that into projects, but investing about $4.5 billion a year in R&D, we do need to also manage that overall investment rate and scale it.
And where we think we’re sort of uniquely challenged is we have many more investment opportunities, many more programs. You mentioned the 40, but we’re trying to launch upwards of 15 products in the next five years commercially.
We have many more opportunities to invest than even we have capital. And so we’re always looking to create ways to expand the amount of capital we have. Blackstone Life Sciences was one of those opportunities that came up. They were really excited by our flu program, the potential for that in flu-COVID combo, which is something we’ve now got Phase-3 data in. And the idea was they would offer a P&L-sparing investment in that program, which would both give a short-term financial P&L relief, but also with $750 million commit to fund the development, which would provide the opportunity for us to invest in a different program.
And so while we really feel strongly about all of our programs, we have a very large respiratory portfolio and the opportunity to finance project finance a piece of that respiratory portfolio and reinvest that money elsewhere in the business made a lot of sense to us.
Salveen Richter
One of the recent opportunities that has come up is the avian flu and how Moderna might step into kind of think about this. First, help us understand how much of an issue this currently is because I think that what was being watched was human-to-human transmission and once signs of that would play out, then potentially orders could come in or more concrete contracts. So, help us understand where this stands.
Stephen Hoge
Yes. Well, if you look at the history of pandemics as we understand them, one every 15, 20 years does seem to happen. More often than not respiratory — well, almost always respiratory viruses, more often than not influenza, a couple of different coronavirus pandemics and we’ve just come out of one. So, these viruses are always out there. They’re always evolving and H5 like H7 and H9 have been strains of Avian or of influenza that we’ve been following — in the case of H5 avian influenza that we’ve been following because we’ve always had a concern that they could escape into human populations.
If they did, if they made that leap and became respiratory influenza viruses in humans, you would expect, unfortunately, a pandemic, not dissimilar to what we just went with COVID because none of us would have been exposed to those H5 strains and therefore, we would need to be vaccinated or become infected.
The other thing that’s quite concerning about H5 is in the rare cases, humans have contracted that mortality has been quite high. Currently, what we’re seeing circulating has really shown up in only a few instances, usually human to — animal to human transmission, a lot of more topical symptoms in the eye.
And that’s good news. I mean, I think that should be cause for some current reassurance, but the big concern was always that those viruses would make some evolutionary leap, that they would break through and be able to become aerosolized and then we’ve all seen when humans can breathe the virus, what can happen. And certainly, it’s happened countless times with influence in the past and it’s just a matter of exposure to humans before it could happen again.
And for that reason, I think we’re more concerned than not right now, given the way that the H5 strains have worked their way through animal populations in the wild; first with birds, and we all saw that last year. And now they’re in livestock with cattle. And obviously, the challenge with livestock, both cattle and chickens is there’s a lot of human contact and, obviously, it’s also moving closer and closer to us as a species. It’s just a matter of perhaps time until that last evolutionary step can happen.
So fortunately, we haven’t seen that yet and there’s reason to hope that maybe it never happens, but with the level of transmission in and around every Western economy in this country in cattle, it increasingly is becoming a concern.
Salveen Richter
And where does Moderna stand in terms of the discussions with the U.S. Government?
Stephen Hoge
So the U.S. Government, BARDA – HHS have confirmed ahead of us that we are in discussions as is another competitor for both development and potential supply of H5 vaccines. I think we’ve all seen how mRNA platforms can respond in pandemics and can provide a uniquely fast tool. And we have confirmed those conversations are ongoing. We have not said more at the moment, and so I’ll leave it at that.
Salveen Richter
Perfect. Let’s talk about the Phase 3 data that was released this morning on the combination seasonal flu-COVID vaccine, whereby the immune response from a single dose was found to be non-inferior versus the co-administered licensed comparators to start. Maybe you could just walk us through this data, put it in context, and help us think about that commercial opportunity.
Stephen Hoge
Yes. So this is really exciting from our perspective. We’ve been working, as you know, since the pandemic, actually before the pandemic on flu, on respiratory virus combination vaccines. And we, in the last couple of years, have made some breakthroughs in the combination of COVID vaccines and flu vaccines.
What we wanted to do through a couple of iterations is make sure we brought forward a program that we thought could be really best-in-class. And best-in-class to us meant we had to be better than the best flu vaccines and better than the best COVID vaccines.
And the data we announced today, we really feel like in that Phase 3 trial, we’ve hit that home run that we were looking for. First on the flu component, particularly in the enhanced flu component that’s — those age — over the age of 65 the comparator there was Fluzone HD, which is a market leader as an enhanced product. And we were able to demonstrate higher titers, actually statistically significantly higher titers for — against the three strains that are currently in influenza vaccine, those are H1, H3, and a B/Victoria strain.
That’s a tremendous accomplishment by itself, but the vaccine also included a COVID component and we were actually able to beat what we think is the best COVID vaccine, that won’t surprise you, it’s ours, Spikevax monovalent in that same clinical trial where we again demonstrated statistically significant neutralizing titers against that virus.
The fact that we could get those two into a combination vaccine and at a lower dose, it’s 40 micrograms and see a reactogenicity and safety profile that’s the same as what you would get if you got the component vaccines is really in some ways a dream come true from a development perspective, from an R&D perspective.
Now, from a public health perspective, we think this also represents a drug on board. Not only could it have the potential to be the best flu vaccine and can do that in combination. One of the biggest challenges we have right now is vaccinations. As we all know, the coverage for vaccines is about half of what it is for flu even in high-risk populations and that’s despite the fact that if you look at recent VA publications, you’re three times as likely to get hospitalized — three times likely to die if you’re hospitalized, but only half as likely if you get the vaccine.
When you look at research around this, a lot of people just prefer not to get two shots. Not many people want two shots actually if you could get one. But a lot of folks who do come in will preference getting the vaccine and then not follow-up — mean to follow-up, but not follow-up for their second shot off from the COVID vaccine or they’ll prefer they only get a shot in one arm and they don’t want one in both arms. And so we — the research we have done and that others have published shows that there could be a dramatic increase in compliance with public health recommendations or broader vaccination rates.
That has a huge impact on healthcare costs, decreased hospitalization, decreased illness. It also has a huge impact on the administration costs, right? A combination vaccine will take half as much time for a pharmacist than two separate vaccines and that allows them to spend that time on other things, taking care of other healthcare problems, twice as many doctors’ appointments and all the things that we know we have an issue with right now.
So, we’re pretty excited about the potential to create economic benefit for the delivery system, obviously, personal benefit for the individual, better compliance and to have all that wrapped up in a package where both products look like they’re as good or better than a market-leader is something we’re pretty pleased with right now.
Salveen Richter
And what are next steps here in terms of regulators?
Stephen Hoge
So, those conversations, now that we have the data are starting in earnest and talking to regulators about what it would take to submit this data, what other questions they would have and more follow-up they would want to see. But our goal will be to try and file for approval at least in some markets starting this year. And our goal would hopefully be that the product might be available as early as next year. But again, that all depends upon the regulators and they ultimately get to make those decisions about it?
Salveen Richter
One comment that comes up is, yes, individuals don’t want to take two doses, but there’s also this viewpoint of an mRNA-based vaccine versus a protein-based vaccine. So, how do you think about that in the context of a flu-based vaccine and the education that needs to be done about mRNA still just given the long-term safety differences?
Stephen Hoge
Yes. So, you want to if you look at the actual safety of mRNA the unprecedentedly large amount of safety data with the billions of doses that have been administered in the last couple of years. And in general, that is incredibly reassuring data. And so there’s an aspect of this, which is a perception or experience of having gone through the pandemic and maybe to some extent, a politicization of vaccination that happened there that was not related to the science of mRNA vaccine, not even related to the amount of data we have, but really related to the last number of years.
And as we go forward, we think that becomes less and less top-of-mind for people and more and more about what are the recommended vaccines we can get and what are the benefits of those. I think that our flu COVID combination vaccine or our flu vaccine will fall more into that category. There’s a strong association with the COVID vaccines with the two companies, particularly in this country that did ourselves and Pfizer that advanced them. But as you go into other categories and other medicines, we think that will become less top of mind for folks and they’ll really be focused on what their healthcare provider recommends.
As evidence for that, if you look at, where people are strongly recommended to get these vaccines, they actually do. And there’s not a specific resistance to it as long as it’s being brought forward by a trusted healthcare advisor.
Salveen Richter
Okay. On COVID, I think a source of upside to your ’24 guidance is the EU tender for up to 36 million doses. Can you remind us on how this tendering process works and when we might know the outcome of this process?
Stephen Hoge
Yes. So, the process is ongoing. The European Union opened up a tender, as you know, up until now, up until that process, the EU had procured — the ECA procured vaccines exclusively from Pfizer. But as a result of the tender, we’re going to be able to hopefully make our product available, perhaps as soon as this year. As you highlighted, it is up to 36 million doses a year, but that’s the upper bound. That certainly wouldn’t be the expectation of the number of doses.
And those processes are ongoing. We are in the rounds of negotiation. There are obviously things we have to do from a qualification perspective to go through that process. And we have not guided on when we expect that will close because we don’t actually control that. It’s really up to the European Commission to decide whether, first of all, we need all the requirements and then ultimately when the conclusion would be, but it is still our hope that we would be able to provide vaccines in Europe under such a procurement arrangement even this year.
Now, the amounts will really depend upon what individual countries submit for this year and the amounts this year because we’re sort of midyear might may or may not be the same as what they would do next year when they would have more time to plan overall supply.
Salveen Richter
Okay. You also have an RSV launch that’s coming up. So, congrats, on getting the drug approved.
Stephen Hoge
Thank you.
Salveen Richter
Can you walk us through the efficacy data that was included in the label and why there was a difference in the number of cases and the analysis presented there versus previously in the New England Journal of Medicine?
Stephen Hoge
Yes. So thank you for that. We’re pretty excited about it. The label data from the FDA approval process was 78.7% efficacy. The primary analysis, the per protocol primary analysis had been 83.7%, substantially the same numbers with the same overlap. So, what was different — during the process of the review with the FDA, they asked us to include a set of cases, about 20 cases that were not confirmed at the time of the primary analysis.
So these are people that might have developed a symptom, but at the time of the data cut-off by the DSMB, they had not had a PCR confirmation that it was RSV it could have been COVID, it could have been flu, lots of other things were diagnosed during that time. When you’re running a clinical trial and you’re following the protocol, when you hit that data cut-off, we’re blinded. We provide the data to DSMB and that blinded data was 83.7%.
But the FDA and regulators have the privilege of being able to look retrospectively and decide if they want to include those numbers. And because it added about 20 more cases to that primary analysis, we ultimately agreed with the agency to include those. Because they broke 15% and 5%, they slightly lowered the number by a couple of percent, but otherwise, it’s substantially the same result.
Salveen Richter
So if you step back, how competitive are you with the two other RSV vaccines that we’re watching with Glaxo and Pfizer and help us understand post-ACIP, the thoughts about contracting here.
Stephen Hoge
Yes. So contracting is — now that we’re approved, we can begin those negotiations and we’re actually able [technical difficulty] to finally get into the market. We believe we are very competitive. This year is a bit of a launch year, so it’s a transition year. Both of those products have been vaccinated in the market throughout the year.
But we intend to get out there and extol the benefits of mRESVIA, our product. First and foremost, we think we have really strong efficacy, a really compelling safety profile. So the strong efficacy that we’ve mentioned and the fact that we have not seen neuroinflammatory events or others in our clinical trial, we think bodes well for the product.
But the second piece that we talk a lot about is that it’s a prefilled syringe. And the advantages of that are again relieving stress for healthcare providers, whether it’s a pharmacist in a pharmacy or a doctor or a nurse in an office. And the reconstitution of the other vaccines, both of which require many steps and combination of diluents and shaking and all sorts of stuff, it is a lot more complicated than what people are used to with their COVID vaccines, their flu vaccines, which you just you pull it out-of-the refrigerator and you inject it in a prefilled syringe.
We do think that adds a lot of benefit to the healthcare system in terms of labor benefits as well. Obviously, it’s preferable for most providers or all. So, from our perspective, that’s a pretty competitive profile. Now we are entering, as you just said, into a contracting season that’s been underway for a couple of months without us. And so we’re actively engaging with retailers, with healthcare providers, integrated delivery networks across the country to try and make them aware of those features. Now I would say most have been aware that that’s coming.
And so this being our launch year, we’ll try and be pretty modest about how far we tell you our expectations are that we’ll get, but we’ll be aggressive as we can be. Our primary goal is to get the product into as many hands and pharmacies as we can because we actually think that combination of safety profile, efficacy profile, and real ease-of-use will win out in the market. And we do hope over one, two, three years of this launch, we will actually establish our fair share and maybe more with this product.
Salveen Richter
And the ACIP meeting that’s coming up, how important is that meeting and understanding these profiles? And I believe you’re going to share durability through a second season and some immunogenicity data across various populations.
Stephen Hoge
Yes. So we’ll do both of those. So ACIP recommendations are important for launch of vaccines in this country. Generally, they follow the — specifically they follow the approval process, the FDA and generally work-off of that label and we’ll do a health economic assessment on the vaccines. And so that’s important. It is our expectation that we incorporate it alongside the other two products in their recommendation. Now there is a question about the frequency of recommendation in the populations that’s independent of our product is going on around RSV right now.
It’s important to note that both of the competitor products had seen pretty steady waning over time through their — into their second season, still some efficacy in their second season. And most people are saying that somewhere between that second season and third season, there is expected to be a decline to background levels of protection, at which point people would probably need a booster to get back to that benefit that they were seeing at health economic value.
That was built into the ACIP analysis last year. We are coming forward with our 18-month data. So if you think of it as a six-month season, you wait a year, you go six more months, you’ve got two seasons worth of data, very analogous to what others had seen. We had already reported out some of the interim data. And like the other manufacturers, we have seen a decline in efficacy into that second season or heading into that second season. We will have that data and share it with ACIP, but we expect that our profile overall will be similar to other manufacturers.
And in general, what we’re waiting to see is how ACIP and other public health officials take the approach of revaccination. So the booster, is it a every two years, every three years, does it eventually become every year? Those are decisions for public health officials to make, but we do think that revaccination is going to be necessary for all of the RCx.
Salveen Richter
And the timeline for approval in other age groups like the 50 to 59 years?
Stephen Hoge
Yes. So, we’re — we will share some data on 18 to 59 years as soon as we have it. There actually is a Phase 3 study we expect to read out shortly as well. And our goal will be to submit that for approval if everything goes well this year, second-half of the year, so it’s available for 18-plus populations next year.
I think that one of our competitors is try to do something similar or another is still maybe also trying to get to the 18-plus high-risk populations. But we really do think that it’s those high-risk folks or people with immune compromise or other reasons for a concern about respiratory virus infections, 18 plus that will be the ultimate label for the product.
Salveen Richter
Any thoughts on the peak market share in the RSV market recognizing that I think on your guidance this year, the Street is sitting at well below $1 billion in sales?
Stephen Hoge
Well, we are the third entrant and we are coming in halfway into the year. And so there’s a little bit of a head-start that everybody else has. So we are trying to be focused about where we’re putting our effort in the second half of the year. On the question of where we expect to go over time, I feel much more confident saying we believe our efficacy and safety profile is as good or better and we believe our ease-of-use profile is better. And so we would hope to at least get our fair share, which I guess would be a third and hopefully beat that. But this year is a transition year. Next year is going to be the first full year of commercialization.
Salveen Richter
Perfect. Pivoting over to oncology. So you just came off ASCO where you had data from your INT program. Help us understand the key takeaways from this program and the read-through to other tumor types as well.
Stephen Hoge
Yes, that is an exciting area for us as well. So we’re partnered with Merck in the development of INT. And so with Merck, we were able to share the detailed three-year durability data. And what is remarkable is that across almost every measure now, the curves are really flattening out and we’re seeing really durable responses. So, first in terms of relapse-free survival, we’re still seeing essentially a 50% reduction in the rate of relapse out to a median follow-up of three years. That’s pretty remarkable.
That is actually, if you put it in absolute terms, it’s essentially one in five people with a Stage 3 or adjuvant melanoma — treated melanoma, not having a relapse in three years. It’s a 20-point difference, one in five. That’s pretty remarkable. Distant metastasis-free survival, which really predicts overall survival better than anything because it’s metastasis, we’re seeing still 60 plus percent reduction and the hazard ratio is I think 0.38%, you just don’t often get to see things like that oncology and lots of zeros in the p-value, which is exciting. And then we started to see the earliest signs of an overall survival trend. It’s early days, it’s preliminary, but at 2.5 years, the hazard ratio there is trending like the DMFS.
And so it’s a 0.45%, so about a 50% reduction in the rate of death, that’s overall survival. That fits scientifically, if you’re not having distant metastases and you’re not having relapse, you would expect to see that. But it’s extremely rare that you get to see those things as early as three years. And I think it all lines up really strongly in the case of adjuvant melanoma.
Now to your question on how we see that translating, adjuvant melanoma was the beachhead for IO therapy. And we all know that KEYTRUDA and other immune therapies have really translated that well across adjuvant settings, particularly in tumors with high mutational versions, lung cancer and non-small cell lung cancer and bladder cancer, renal cell carcinoma, so many others. And as we have announced with Merck, we’ve moved into clinical trials against all those because our view is there’s going to be an extremely high probability of translation across, just like there was for KEYTRUDA.
Salveen Richter
And you’ve also spoken about the three factors needed to take an accelerated approval pathway or to file for an accelerated approval pathway. Help us understand the alignment between you and Merck on these factors and what needs to be done to get that sign off from the FDA?
Stephen Hoge
Well, first and foremost our partner, Merck and us, we see these benefits and we kind of — we aligned on these criteria to some extent. We’ve talked about what is it going to take for us to move forward. And we do expect if we can achieve all three and agree that we’ll go-forward and ask the FDA and other regulators about the potential for an accelerated approval in this setting.
The three criteria that you mentioned, just to quickly cover them for those who aren’t as familiar, first, we wanted to see durability. We wanted to see at least three years of durability. I guess the answer on that one is check. We’ve got that with the most recent presentation at ASCO announcement from six months ago. We’ll happily follow that for another year or two, but once you get to four or five years, you really don’t expect those recurrences to happen and that’s good news.
We’ll, also follow that overall survival curve because that starts to become quite substantial. Again, that’s a 6% difference in overall survival, you know, small, not significant yet, but that’s one in five people not having a relapse and 1 in 20 people not dying and that, of course, would be pretty important. Second criteria, we have got to enroll the Phase 3. And so in that one, given where we are in terms of our obligation to regulators to demonstrate our diligence in confirmatory studies, we want to make sure that we had substantially enrolled, maybe completely enrolled in that Phase 3 study.
We have not, with our partner Merck, guided to our specific expectations, although there were a lot of questions after an investigator said they thought it could happen by the fall. What I can say is that we’re just really pleased with the brisk pace of enrollment. We think it speaks to the potential of that medicine. And the next thing that we would expect to do is with our partner, Merck, notify people that we’ve completed that enrollment.
And then that third criteria is we have to have a facility we make this. This is not like our other vaccines. In order to make an individualized medicine, it requires a completely different operation. We can do it in our R&D facility in Norwood, but actually to commercialize it at scale required building something purposeful. We’re building it in Massachusetts, and we’ve been on that for over a year. Our hope is that we can complete that work this year, but it’s not in our control alone. We actually have to complete the construction and the qualification and have confidence that facility is ready to support.
So all three of those stars seem to be aligning. And when we’re — when we and our partner are confident that we’ve satisfied our own criteria, then the next step would be to go forward with the FDA and others and ask them if they agree that the benefits of the product really far outweigh the risks and that it’s appropriate to move forward with accelerated approval. Our hope is that answer is yes, but it’s again not our choice.
Salveen Richter
Great. You also have Phase-3 interim data for the CMB program this year and that really was the lead program actually after you went public. So walk through the level of confidence you have in this analysis as well as what the bar is that you’re looking for.
Stephen Hoge
Yes. So our minimum bar statistically is 50% efficacy. We’d love to see something closer to 60%. There’s never been a vaccine against CMV. It’s, as you know, a leading cause of birth defects. And if we could prevent infection at that level, we would feel like we’d have a really big impact.
Obviously, we want to do better than that, but those are the statistical criteria. The first interim analysis is looking at the rates of seroconversions of infections. I think we’re pretty optimistic. I’m an optimist by nature. So, maybe on the bullish side of this. And the reason is that there have been prior attempts with CMV vaccines, including one recently published by Merck, not our partner on that program, but our partner on others.
And they have seen tantalizing evidence of efficacy. I think they’d seen 45% efficacy with a vaccine that produced about 10-fold lower levels of antibody titers, neutralizing antibodies against the virus. And so with our vaccine, we’re 10-fold higher than they are and again using seropositives as a reference, it wasn’t a head-to-head study.
We would expect to see some benefit 45% isn’t 60%, but maybe we can do better than that. The other thing is that the vaccines themselves also the way you measure infection actually can be quite complicated. And so in that same publication, Merck had shared that they did a seroconversion analysis or a surrogate for a seroconversion analysis looking at whether people had developed antibodies.
They couldn’t do it without that particular vaccine because it was a replication-deficient virus and so they kind of developed antibodies, but they looked at something they thought was more representative of that and they saw close to 60%, actually a little bit higher than 60% potential efficacy in that Phase 2 study. That actually is the endpoint — more analogous standpoint we have in our study.
So, I think the endpoint is likely more like their higher point estimate and we have higher titers. And so the combination of those things have me optimistic. I don’t know yet, but we could expect any moment now. And certainly, through the balance of this year, we would fully expect to get the interim analysis and we’ll be happy to share it with the world.
Salveen Richter
Great. And maybe just in the last few minutes that we have left, you do have a portfolio behind this, including a partnership with Vertex for a CF asset where we’re going to see data in the second half. Can you speak to the ability to have used combined with them and used MRA in an inhaled version to be able to treat this disease and how to think about that likelihood of success?
Stephen Hoge
Yes. We are fortunate to have two truly exceptional partners in Merck in — obviously, in oncology, and Vertex in cystic fibrosis. You couldn’t imagine a better partner. Their deep understanding about that disease, and our hopefully equally deep understanding about messenger RNA and lipid nanoparticles, was born as a partnership in 2016 and it took us a solid five, six years to get into the clinic. It took a lot of hard work scientifically to convince ourselves that we could get mRNA in lipid nanoparticles, aerosolized and then across the mucus barrier in the lungs of these patients with CF.
Vertex had an incredible system for evaluating that based on all of their work with the correctors and we were able to jump in there and over many years of hard engineering breakthrough, and so we’re — we and them are pretty excited. They announced earlier this year that we completed the single-ascending dose sort of safety portion of the study and had moved into multiple dose. And so this is daily, in some cases nebulized replacement. These are for patients who have no other hope because they don’t even have the gene, a functioning copy of the gene that a corrector could work on, they really need replacement.
And you know, I think we like our partner, Vertex, are pretty excited to see what that data looks like. I believe they’ve said publicly and it is a partner program, so I’ll defer to them. But I believe they’ve said publicly they expect to see that multiple-ascending dose by year-end. And so we’re quite keen to see whether or not we can improve respiratory outcomes measures.
So, all of the things they normally look like and look at for cystic fibrosis by the end of this year. And if that’s the case, get ready for us in the pulmonary space. It’s not the only rare disease we’re working on. As you know, we’re working in liver rare disease as well, not with a part, but obviously, we’re deeply committed to try and do as much as we can with this technology to help as many people as we can.
Salveen Richter
Great. Well, with that, Stephen, thank you so much. Take care.
Stephen Hoge
Thank you very much. Good to see you.