Moderna, Inc.'s (MRNA) Management Presents at Chardan 5th Annual Genetic Medicines Conference (Transcript)

Moderna, Inc. (NASDAQ:MRNA) Chardan 5th Annual Genetic Medicines Conference October 5, 2021 1:00 PM ET

Company Participants

Stephen Hoge - President

Conference Call Participants

Geulah Livshits - Chardan Capital Markets

Geulah Livshits

Hello. And it's now my pleasure to introduce our next fireside chat guest, Dr. Stephen Hoge, President of Moderna. Stephen, thank you again for joining us today. And maybe let's start off by a big picture item. The last year, when you participated in our conference, Moderna was a Phase 3 company and now millions of people have received the Moderna COVID-19 vaccine. We've seen the pipeline advance and you've announced a number of research and manufacturing initiatives.

Question-and-Answer Session

Q - Geulah Livshits

So before we talk about these topics in a bit more detail, can you comment at a high level on Moderna's trajectory and priorities going forward?

Stephen Hoge

Well, thank you, and thanks again for having me back. I enjoyed it so much last year and the year before. So this will be an exciting chance to reprice where we've been. It has been a very busy year, as you described. I think I talk least about our progress in COVID, which is a place that's been well covered and maybe focus a lot on all of the other progress that's been happening in our clinical development pipeline.

As we've announced throughout the year, we've had a number of key transitions, a few that I'd highlight in the respiratory vaccine space to start out, which is, we've been moving forward with both our flu and RSV vaccines. These are respiratory vaccines that we think we will eventually use in combinations, both for older adults and pediatric populations. We've been advancing them very quickly in both of those populations and actually have completely enrolled those studies and announced at a recent -- at our recent R&D Day that we're moving forward towards pivotal studies in the near-term, including a Phase 2/3 program for RSV, based on some very exciting clinical data that we shared a month ago, showing what we think are some of the best titers possible in older adults with RSV vaccine.

That's obviously incredibly important and something that we think in the future could be combined with a second program that's moving very quickly, which is our seasonal influenza vaccine. We have a quadrivalent, so four different mRNAs against flu program as well, as I said, a couple of further generations, so 1010, 1020 and 1030 on our mRNA pipeline that are moving forward quite quickly. And we do hope that seasonal flu program, which is fully enrolled, will be able to read out quickly and allow us to transition rapidly towards pivotal development there as well.

And of course, we've always intended, as you know, to do combinations in and around that space. In fact, if you go back a couple of years and you look at our vaccines days and our R&D days, we've always talked about doing respiratory combinations. Now we know those combinations will almost certainly include COVID visitors. And so that's a foundational piece of it. But we do expect that, particularly for older adults, who have waning immunity in the influenza and against RSV, that there will be eventually combinations of those as well. And we've actually announced the beginning of our work to look at some of those combinations, a combination of flu and COVID as well as a combination of RSV in hMPV and pediatric populations. So both of those recently announced and again, moving forward quickly.

That's just in the respiratory vaccine space. I won't go as fast, but just a quick -- sorry, I won't go as slow. I'll go really fast on just a few other key milestones. We are excited to start the CMV pivotal study this year. That is still on track. That's going to be a very big deal. As you know, who followed us for a while, we've been passionate about that cytomegalovirus vaccine. We think it is a major unmet need and a huge opportunity for us. And we're on track to start that Phase 3 study imminently, which is exciting.

And then we've continued to progress in therapeutics. And again, for those who’ve followed us for a while, that has been a longstanding story. Our most advanced programs are in Phase 2. And so we have a VEGF program with AstraZeneca, which is looking to do cardiac regeneration in patients undergoing CABG surgery. And that has actually -- we hope to have that data in the near-term. So we have our fingers crossed that when we see that data, that will be encouraging for that program.

I'll remind you that could be a substantial opportunity to change the standard of care in people who've got cardiac ischemia disease. And that's in partnership with AstraZeneca. And we also have Phase 2 -- another Phase 2 program, a mid- to late-stage program in PCV, in cancer vaccines, where we're fully enrolled now in that cancer vaccine study. That's 150 participants randomized against KEYTRUDA to show whether or not we can extend the relapse-free survival in melanoma patients, a very exciting program, too. And so again, we're sort of on pins and needles waiting for that data, both respectively in cancer and cardiovascular disease.

And then the last thing I'd highlight are the progress in the rare disease pipeline. We have now enrolled the first cohort of the propionic acidemia program, PA, which is one of our first rare -- as our first rate metabolic disease to be dosed in humans. And we've actually enrolled the first subjects in our MMA program over the course of the summer and announced that, which is another important milestone.

And so we'll be looking closely in the near-term for data from those early-stage rare disease programs, which because the biomarkers are synonymous with the name of the disease, we think if we're able to see an impact on that biomarker relatively quickly, we'll know that we have a great path forward and those programs can move very quickly.

I'm not going to get to autoimmune and other things and pulmonary and others, that’s going well, but we are incredibly pleased with the progress in the pipeline across. And now over 30 medicines in development, a couple of dozen in different -- in the early stages of the clinical development, later stages. And as you said, also now a registered product, an authorized product in the United States and a conditional license product outside the United States is exciting.

Geulah Livshits

Great. So I'd like to touch on some of those different programs that you mentioned. And we won't spend too much time on COVID, but we do have to talk about it a little bit.

Stephen Hoge

Of course.

Geulah Livshits

So, obviously, since we've now seen a real-world elevation for the COVID vaccines and the conversation, as you said, has shifted more to booster than the long-term strategy there. So can you comment on how -- and obviously, the conversation shift every day with each new data readout and real-world study regarding immunity duration, et cetera. So can you comment on how you're currently thinking about the boosters in light of, for example, current regulatory stances on that front?

Stephen Hoge

Sure. So we have already been achieved authorizations or licenses around use of the booster. For instance, in the UK, where it's recommended regardless of the primary vaccination series. And as everybody probably saw, the FDA is holding a VRBPAC on the 14th. We will be presenting around our data in the United States. We think the data is incredibly supportive of the use of the booster. But ultimately, I want to defer that judgment to both those advisory committees, the FDAs and eventually, CDC and ACIPs.

But the data that we see that I think is compelling from my side is there -- we are very pleased to see the durability of 1273 of Spikevax. It has held up, we think, best among vaccines. And if you look at the Phase 3 data, that's true. And if you look at the real-wold evidence, that's been true. And generally, we've been encouraged to see that, that efficacy is very high even against the Delta variant. But it is not going to be permanent.

And just like other vaccines, we do see waning immunity measured by neutralizing antibody titers. And our sense is that sometime, maybe it's nine months, maybe it's 12 months, I don't think we ever want to wait and be too late to find out. But at some point, you will start to see substantial breakthrough infections. We've seen some for sure already, and those have been reported, but we expect them to go up. And that's why for now, we think a booster dose sometime after six months, to make sure that we maintain high immunity in the populations, frankly, we think broadly is of importance. And that's why we've been seeking that authorization, including in the United States for those populations.

Now again, public health officials have to decide if they agree and then if they recommend that. But I think personally, selfishly, that there's so much going on right now in the pandemic in terms of circulation of the virus still. Delta is still a big threat. And as we head into the winter season, the last thing we want to see is declining protection, waning immunity leading to a spike in breakthrough cases on top of perhaps a seasonal epidemic on flu and other things that will add an incredible burden to our healthcare systems.

And so I think that if we know we have a tool in a vaccine that can suppress that, we do know that fresh vaccination is able to substantially lower the number of those breakthrough cases. And I think it's prudent for us to plan to use that broadly as a third dose booster. Now looking forward, we actually do think that there's good reason to keep doing that seasonally every year, particularly for high-risk populations. And that's not just immunocompromised, I would say, those who are older adults over the age of 50 or at least over the age of 65.

And then there's the other healthcare and economic arguments that maybe those under the age of 50 might make as well. We have seen increases in long COVID as a result, even associated with breakthrough infections. There's a concern that this virus can have all sorts of long-term sequela. And of course, you can recover from those things, but who wants to be out for either a week with mild COVID and dealing with all the testing and disruptions associated with that and our families or maybe a month or two recovering from more chronic syndromes.

And so our goal -- our view is that there's a strong rationale to try and prevent all of that morbidity that can happen associated with those diseases. And we think that seasonal boosters will actually be a big part of that because it's been able to show a benefit so far.

Geulah Livshits

Great. So you just talked about the seasonal boosters. And previously, you mentioned the overall strategy that you've talked about before regarding combinations for the respiratory franchise. And so putting those together a little bit, can you talk a little bit about how you envision the path for putting the combination vaccines in the respiratory program, including potentially COVID together with respect to regulatory agencies and your discussions on that front?

Stephen Hoge

Sure. So it's important to say that, in some cases, the fastest path towards combination may be proceeding with monovalents and then combining them, except after approval. We do -- we started with the COVID vaccine, where we have -- we already have clear efficacy. Now we do not yet have a BLA. We've found for that, has been talked about. And some countries actually have achieved full approval already. And so that is an anchor point. And I think what we would want to show is as we bring in, let's say, a flu vaccine for which there are very clear surrogates and approvable end points, that we would be able to show that there's not interference in those combinations.

Now whether you go all the way to sort of approving the flu by itself or whether you combine it with the COVID in the later stages of development, just to show non-interference with COVID and then ultimately, your pivotal studies, your pivotal immunity and safety or efficacy and safety studies are with that come up, that's something that's subject to conversations with regulators.

I mean at the end of the day, we're all discovering together the best way to use mRNA technology to do combination vaccines. And it may evolve. It may be that we choose to proceed in the first booster combinations with monovalents and then post approval, do a small study bridging immunogenicity and safety, so that you can combine them and then advance that combination vaccine. But that in subsequent stories, we might actually be doing things where we're folding-in in pivotal studies combination sooner because we just want to get there a little bit faster perhaps be a little efficient.

So it's not that we're dogmatic about one approach is better than the other, and how we're doing it. We're really going to follow the science and work with regulators to make sure that we're giving them the data they need so they can make the choices around this.

What I would say, though, is that as a final comment is that booster vaccines aren't that foreign. They're foreign in adults. But if you really look at it in pediatrics, there's a long history of bringing forward vaccines, combining them and actually, therefore, efficiently delivering in a single injection, many potential forms of protection. We think we can learn a lot from that experience as we approach the respiratory vaccine approach in older adults.

Geulah Livshits

Just one last quick question on the respiratory and the combo vaccine programs. Would you envision -- is there still value to developing and potentially commercializing a standalone vaccines or like as a kind of an a la carte vaccine strategy? Or do you envision primarily focusing on these combinations once you identify the optimal combos for your target population?

Stephen Hoge

Yes. Well, you asked the question in two ways. And I'd say, is there still value? Of course. There's never -- value is never zero, right? And so there’s a - if there's a role to play for a monovalent vaccine in a market, then of course, we want to make sure that we provide it. However, you asked the question, the other, what's our primary approach? I think our primary approach is we really think that there's -- it's a failure of imagination that people aren't doing these respiratory combinations already, right? Or it's a failure, it's just an effective history as opposed to this following the science.

The truth is that these respiratory viruses, they're top five global killers. They're -- it's horrible scourge. And in fact, they impact the same at-risk populations with roughly the same seasonality in sort of the six months going through the fall, into the early spring. And so there's -- it's really, if you look at it, why do we need to make people a human thing cushion and give them five needles when we could all get that into one needle and do a single combination injection? And if along the way, that meant that somebody didn't see all four or five of those viruses that year, yes. But they were prophylaxed, protected against the full set. Is that really that different than covering four strains of flu? And is that really that sort of crazy as an idea?

So we do think primarily the advantage is, I want one booster. I want one shot in my arm. I want to know that I'm protected against respiratory viruses for the better part of the season of the year. I don't want two or three. And I think it creates a big a la carte problem, both for healthcare systems and for us to try and get too cute with influenza virus. So if they're needed, will we provide them? Of course. But we actually think there's a better way, and that's what we're trying to get to.

Geulah Livshits

Got it. And so let's talk a little bit more regarding the other pipeline and how perhaps the COVID program has enabled some of the advances there. The COVID program has obviously developed on a pretty accelerated timeline. Can you speak a little bit to the development timelines for your other programs, either within the infectious disease franchise or outside of it? For example, in the rare disease setting, where, again, you also mentioned your clinical trial momentum there.

Stephen Hoge

Yes. Well, again, so we are -- we think COVID has shown us what's possible, particularly in the vaccine space. And I don't know that I -- I would certainly not promise that we are going to continue to average 11 months from Phase 1 to authorization, right, or even less than that every year. But there's no reason we need to fall back to a much more laconic timeline. And if you look what we're doing even in the RSV space, as we announced that we've completed the Phase 1 dosing of RSV, and we're moving into a Phase 2/3 study that we hope to be pivotal. And we're trying to do that in the near-term, even this year if we're able to.

And that approach, if you look at the timing, maybe it's 12, 18 months from first in-human to approaching that pivotal study. And the pivotal study will run what it will run. It will be event-driven as all respiratory studies will be in efficacy. And so we'll have to follow that out. But it's actually pretty fast if you think about it.

You start talking about rapidly from IND filing into the pivotal phase. Our flu program, we hope to move similarly on very fast time horizons. Now there are going to be some programs outside of respiratory disease in particular, where I think you're going to -- you see slightly longer because you want to learn more about the vaccine in the early stages of clinical development. Maybe it's not as clear what the dosing regimen be. CMV is an example.

But even in CMV, I'd point to, it's only been a couple, maybe 3, 4 years, right, from first in-human to starting a pivotal study, and that's actually quite fast in the grand scheme of things. And we hope to do faster even in the future. But I think we imagine these as low single-digit year time horizons. Again, subject to the science being there, really has to work, the data has to be right and regulators feeling comfortable with that approach. But we do think we can learn a lot, not just from Moderna's experience, but from all of the manufacturers’ experience in the COVID world and realize that actually probably faster is possible without actually sacrificing anything in terms of the quality of the data or the safety of the participants.

Now rare diseases, and in cancer and you alluded to that, the burden of disease is so high. You've always been able to go fast. In fact, the unmet need is so high that the benefit risk is absolutely clear if you can provide it. You need to go fast. And that has always been there as a pathway. We've seen it in cancer, and I think we've seen it in rare diseases. And our goal is to bring that kind of transformative benefit so that actually people clear the path, and you're working collaboratively with regulators and communities to get those drugs available as fast as possible.

Geulah Livshits

Got it. And so you mentioned the route of this program. Is there any additional color that you want to provide on the timelines and/or what you'd want to see, for example, to promote intracellular to a core modality?

Stephen Hoge

Core modality is, as you know, I love that language. Thank you for that. For us, that will be -- we believe we have enough scientific data and we'd share with the world that we can predict the pharmacology. We really know how to do this. And so the next one, we can go fast and not skip steps, but move at risk with a bunch of steps in parallel in the technical side and into the studies.

It is where we think we have been, as you know, for a couple of years in vaccines. And we showed what that meant in COVID when we went fast just 1 company at -- in Cambridge, Massachusetts. And so we believe that same approach is going to play out in how we approach rare diseases. I haven't seen the data yet. And so the thing I'm looking for is what is that data going to say. But when do we know we see enough of that pharmacology that we can say across a range of different populations, we can predict the dose response for seeing the biomarker move in the way we want. It could happen in PA. It could happen in MMA. It could happen in both. We continue to conduct those studies. And because there are different biologies and different patient populations, it may not be the one is 100% clear, but the other is.

But when we see enough data, enough patients and enough predictability in that response, we will be sure to share it and say, "Look, we're there." And we would then call it a core modality and say, "We're going to go faster because we believe this is now reproducible, and we can call these shots going forward." We're not there yet. And we have a little work to do.

And look, if it doesn't show up in the first one, we've got GSD1a coming right behind. And we've had really positive signals around that. And so we're hoping to -- preclinically and with the FDA designation. So we're looking to raise that one forward as well, and we'll keep going. Our mantra is, this is how science progresses. This is how technology progresses. We'll run these studies and hopefully show very quickly that one of them is successful. And then we will do a lot of that thing to address the other diseases.

Geulah Livshits

Got it. So speaking of science progressing and technology progressing, last month, you announced the Moderna Genomics and your plans to get into more genomic editing using the existing platform as well as novel nucleic acid technology. So can you elaborate a little bit on the approach and what you can at least and how it fits in Moderna’s broader strategy going forward?

Stephen Hoge

Yes. So we -- look, we have been following the genomic medicine space for years. We existed before it in some place. I -- and so we've been very much aware. Our first experience is expressing nucleases 8, 9 years ago. You see that back in the patent [years ago]. But we've been looking at this space for a very long time. We have felt that there was a need for some further evolution definition in the best places to use that technology. And so that's how we sort of followed it over time and focus most of our energy in our existing therapeutic areas.

But in the last couple of years, it feels like we're not there yet, but the time is right to start thinking about what are the payloads that we would want to express in mRNAs and lipid nanoparticles and our core technologies, our core competencies to more permanently correct diseases, a range of different things. And the answer is a question of, well, are you thinking about -- and you list them off. Are you thinking about the knockoff and base editing and gene editing and rewriting all of this stuff? The answer is yes. Anything that you can do with an mRNA and the lipid nanoparticle because that's our core competency. That's what -- that's frankly a science that we've invented, and we're still the people that do that in humans, including across a range of therapeutic programs.

And so that competency, anywhere we believe it can be valuable with a range of partners or just using our own technology, we’re able to move, try and bring it forward. There has been massive convergence not yet on what's the enzyme or the nuclease, but there has been massive convergence on the idea we're proud of that mRNA and LNP might be the way to go forward with this. And we couldn't agree more. That's why we created it. And we built the infrastructure to do many of these things at a different level of quality and experience that we think anybody else will be in. We hope to add value that way very quickly.

So we're looking broadly. We are agnostic on the enzyme. We are agnostic on that approach. Right now, we're going to follow the science and perhaps try many different approaches. But the one thing that you can count on is that it will be an mRNA and an LNP.

Geulah Livshits

Great. And so in the last couple of minutes, again, just moving back to big picture, looks like you had robust performance year-to-date. So looking ahead from your perspective, what do you see as the hidden value growth drivers for the company that might not still be appreciated by investors? We've obviously appreciated the stock to this date.

Stephen Hoge

Yes. Well, I think a lot of our story has been still about COVID. And I still like, therefore, everything non-COVID is the answer to that question, because we spend a lot of energy on it. I understand why, it's an incredibly large and commercial product. I get it. But at the end of the day, if I paint the story forward 5 or 10 years from now, even the COVID vaccine in my mind is a part of a broader story in preventing respiratory disease. And in that sense, I would imagine it's subsumed by those respiratory combos. And of course, it may stand alone as a separate product in the future, but I actually really don't think that's going to be the right approach for healthcare systems.

And so what I would follow is what is the progress on flu? What's the progress on our RSV? How is our data looking? Are we executing on those studies well? How are we doing combination? Because that could move very quickly. These are older adult, respiratory adults. And the kinds of things that you might say, oh, these are barriers to that product moving quickly in the past, like can you make enough doses? Nobody asked that question anymore, right? I mean we're making a lot of doses. And they're coming here, [even worse]. The capacity will be there.

As the question about, have you given it enough people? Is there enough safety and comfort with the platform? This platform has been giving more to people than any -- than the entire world of flu vaccines last year and the years before. In the history, I think there's this is one of those things that's been transformational. So many of those fundamental questions are kind of gone, and now it can we execute this combination strategy. I think that's something to watch.

And then the other -- the wildcard in that is the rare diseases and cancer work. And I think I would also add to that where we come out with VEGF, which is if we show progress in the therapeutic spaces, any of them, but certainly, many of them, if we show that, I think it is not currently appreciated that mRNA and the technology we've built is not principally about preventing disease, but it can also be used to treat it. And I think that, that will be perhaps an eye opener for some who don't follow the story as closely.

Geulah Livshits

Fantastic. So I think that brings us to the end of the session. So I'd like to thank you again, Stephen, for your participation, fielding our questions and the great discussion.

Stephen Hoge

Perfect. Thank you so much. Pleasure to be here again.