Capricor Therapeutics Inc (CAPR) 2020 Q4 法說會逐字稿

Greetings, and welcome to Capricor Therapeutics Fourth Quarter, Full Year 2020 Earnings and Corporate Update Call. (Operator Instructions) As a reminder, this conference is being recorded.

I would now like to turn the conference over to your host, A.J. Bergmann, Chief Financial Officer. Mr. Bergmann, are you muted?

Yes. Thank you very much. Before we start, I would like to state that we will be making certain forward-looking statements during today's presentation. These statements may include statements regarding, among other things, the efficacy, safety and intended utilization of our product candidates; our future research and development plans, including our anticipated conduct and timing of preclinical and clinical studies; our plans to present or report additional data; our plans regarding regulatory filings, potential regulatory developments involving our product candidates; and our possible uses of existing cash and investment resources.

These forward-looking statements are based on current information, assumptions and expectations that are subject to change and involve a number of risks and uncertainties that may cause actual results to differ materially from those contained. These and other risks are described in our periodic filings as made with the SEC, including our quarterly and annual reports. You are cautioned not to place undue reliance on these forward-looking statements, and we disclaim any obligation to update such statements.

With that, I'll turn the call over to Linda Marbán, CEO.

Good afternoon, and thank you for joining us for our fourth quarter and full year 2020 financial results and corporate update call. We will begin today with an update on our exosome platform technology, which was highlighted in a paper published earlier this week on bioRxiv. I will then provide a brief overview of our other programs. We are extremely excited about the progress that we made in 2020. And looking ahead, we are very enthusiastic about 2021.

Joining me today is Dr. Stephen Gould, Professor of Biological Chemistry at Johns Hopkins University. Dr. Gould has worked in the exosome field for nearly 2 decades and works with Capricor both as an executive consultant and scientific collaborator. Dr. Gould has helped accelerate Capricor's exosome program, bringing new ideas, innovative technology and valuable know-how while also helping us assemble a team of scientists in our Los Angeles labs to work on product development and manufacturing. We have also entered into a sponsored research agreement with John Hopkins University to support the exosome program.

I will begin my remarks with a brief overview of the goals of our exosome platform, and then I will turn it over to Dr. Gould to walk through the data and its implications for exosome therapeutics development pipeline. I hope you will be as excited as I am by our findings and will see the power that this platform can potentially provide in the development of therapeutics for a broad spectrum of diseases.

mRNA therapeutics are a new class of medicines pioneered by companies such as Moderna and others. mRNA medicines are not small molecules like traditional pharmaceutical drugs, and they are not traditional biologics such as recombinant proteins and monoclonal antibodies, which were the origins of the biotech industry. Instead, mRNA medicines are sets of instructions, and these instructions direct cells in the body to make all the proteins required for life as well as to prevent or fight disease. The field has broad market opportunities for many diseases. And as we have seen with the success of the mRNA vaccines currently on the market, we believe that the concept is rapidly adaptable. We are excited to be part of this new wave of opportunities in biotechnology. As the field as a whole looks beyond mRNA vaccine development, one of the most critical aspects to the repeated delivery of nucleic acids to treat diseases are effective and safe delivery systems. When we think about platform delivery systems for mRNA and other nucleic acids, there are many systems that have been developed, most notably lipid nanoparticles. But it's our belief that nature's own communication systems, the exosomes, offer great potential due to their unique properties.

Now to remind you, exosomes are produced by all cells, abundant in all biofluids and have been demonstrated to be safe by decades of transfusion and transplantation medicine. Additionally, they can be directed or, in other words, targeted, to the cell types that we would like to treat and are readily able to deliver a payload to the cell, directing protein expression. Capricor has some exciting developments underway focused specifically on targeting technology, which we plan to share at a later date this year. These are the goals of drug delivery that remain unanswered, and we believe that exosomes can provide a platform in drug development with vast possibilities.

The data that has been generated by Johns Hopkins and Capricor is focused specifically on expression of functional proteins using our exosome platform delivery system as well as on the toxicity and safety effects when compared to traditional lipid nanoparticles. These aspects support the goals of Capricor's exosome platform, which we are developing. And I believe strongly that exosomes, again, the body's own drug delivery system, provide a unique opportunity for the expansion of this field.

Capricor in 2021 is focused on becoming a leading exosome RNA company. And all of our work against SARS-CoV-2 is simply a prelude to the future development of exosome-based vaccines and therapeutics, with the ultimate goal of generating formulations of engineered exosomes and synthetic mRNAs to potentially prevent and treat human diseases.

I would like to now turn the call over to Dr. Gould, who will walk us through the data and its implications for our platform. Steve?

Thank you, Dr. Marbán. Yes, there's been a very clear presentation of the general approach that we've been pursuing here at Hopkins and that we started a very deep, broad collaboration with Capricor over the past year. We are very excited to be working with Capricor. It allows us to expand our horizons both in terms of basic research but also in applying the rules of exosome biogenesis and production and targeting 2 actual real-world clinical problems.

Our approach is essentially outlined in this slide here. There's a great need to make sure that we develop the exosome platform in an efficient, high-throughput way so that we can optimize the production of exosomes, the loading of them, the formulation, the manufacturing and the targeting. And so what I'm going to start with is talking a little bit about a small piece of the data that we've been collecting about the general utility of exosome platforms for RNA delivery.

So one of the questions that you might be thinking of right away is, how do exosomes compare to other RNA delivery platforms, specifically with the nanoparticles or LNPs? And so we've undertaken a wide variety of experiments, and I'm going to show you a very small piece of our data on that issue. The first thing I would like to show is a figure here that reflects weight change data in response to the injection of equal numbers of exosomes or equal numbers of lipid nanoparticles. And black dots are the weight of the animals on day 0. The red squares are the weights of animals 3 days after the injection.

And what we have seen is that exosome injections did not engender any gross adverse effects that can be detected upon visual examination of the animals, weighing the animals. And this is not quite true for LNPs. In equal numbers of vesicles being injected, what we see is that over a short period of time, LNP injections can lead to a reduction in weight of the animals. And reduction in weight is just a very useful marker for any kind of treatment that causes animals to feel a bit off. It doesn't necessarily mean there's a horrible toxicity brewing here, but it is indicative of an adverse effect of the treatment. So this is just injecting animals with exosomes. There's no effect. Injecting animals with an equal number of LNPs causes some distress to the animals.

Now that's just measuring weight of animals, a very easy thing for us to all understand. And we all know when we feel -- don't feel well, we tend not to eat as much. But the real key is looking in these animals by a detailed histological analysis, and that's shown in this slide. Now there's a lot of panels here. But to make it a little simpler, let me go through it. The top row are histological samples from control mice, same age, and these were done on multiple animals in each group. The control animals have been examined for morphology of their intestines, salivary glands, spleen, kidney, liver, heart, diaphragm, lung and brain. And based on an independent histological analysis, these animals are totally normal, and the same was true for all animals that were injected with exosomes. So we're injecting animals either with saline or injecting them with an equal number of exosomes or LNPs.

And the exosome-injected animals, again, the histological report came back normal in all tissues in all animals. The same could not be said for those animals that were injected with the same number of LNPs. And these LNPs are very similar, the same basic composition as the LNPs that are being administered in some of the leading vaccines. So while there was no abnormalities in most tissues, there was an abnormality in most of the animals in their spleen. So what was specifically seen? What was specifically seen in the LNP-injected animals was an increase -- actually a high increase in the amount of red pulp in the spleen. And the red pulp is an indication of sort of nonspecific inflammation, and this goes hand in hand with the weight loss data that we saw in these paired animals.

So to sum up these 2 pieces of data, what we're showing you is that injections of exosomes and large numbers of exosomes has no adverse effects. And this is entirely expected because nearly a century of transfusion and transplantation medicine has demonstrated that the delivery of massive amounts of exosomes into people, that is not associated with any adverse effects. So we're basically just reproducing kind of what the medicine already knows. In contrast, injection of LNPs are mildly inflammatory by this assay. And so that's just the delivery vehicles, comparing LNPs as a vehicle and exosomes as a vehicle, what are the effects of those alone.

Well, the next question is, well, how do these delivery vehicles compare in terms of the efficiency with which they can functionally deliver mRNAs? And we have a little bit of data that we can show you today on that. Again, we've done many, many studies on this. I'm just going to show you one little snapshot of the data. So what you're looking at here is the expression of a luciferase, a light-emitting enzyme. That's assays on cells that are grown in culture. So these cells, obviously, they don't normally emit any light. What we can do, though, is we can synthesize in vitro a message RNA that encodes an enzyme, a luciferase, that can be used to -- as a marker for how efficiently we're delivering mRNA into these living cells. So again, the particles, the exosomes themselves, do not have this enzyme activity. The LNPs do not have this activity. The activity is not strictly present in the RNA. The RNA has to get into the cytoplasm of a cell. It has to be translated into a protein, and that has to be done with a high degree of fidelity so that, that protein is enzymatically active so it can emit light. So once it's made, it's capable of emitting light when given the substrate.

So what we've done here is we have control cells. That's the bar on the far left. You can see those control cells don't -- in response to the substrate. There's a substrate called DTZ you add. And if the enzyme is present, light is emitted. If you add the substrate to the control cells, there's no activity. And that's why all those black circles of control are right at the bottom at 0 light emission.

Now when we load a standard amount -- a constant amount of RNA into 10^11 particles per ml of lipid nanoparticles, we do get good expression of this light-emitting enzyme. But when we do the exact same parallel experiment, same amount of RNA with same numbers of exosomes as LNPs, we get more light emission, which means we're more efficiently delivering that RNA into the cytoplasm of human cells, and it's being turned into functional enzyme. So exosomes are, by this graph, better. Those 2 bars in the middle show that.

Now when you go to high-dose administration, and high-dose administration is particularly important for a therapeutic mRNA delivery, where we're going to have to be delivering a lot of RNA many, many times in order to cure, say, genetic diseases, what we see is that the difference between exosome-mediated mRNA and LNP-mediated RNA delivery is really dramatic. It's now more than a tenfold difference. And as you can see, what happens is that when you dose with large amounts of lipid nanoparticles, you get an adverse effect on the expression of the RNA that they carry. So there seems to be an upper limit on LNP-mediated mRNA delivery that you don't have with exosome-mediated RNA delivery. And this is a really important point. I know that this is just a tissue culture experiment, but what you see in human cells in tissue culture is very often a very good sort of indication of what you'll see in vivo.

And I'm going to show you some in vivo data in the next slide, in which we take the same mRNA-loaded exosomes, where the mRNA encodes a light-emitting protein -- I mean, yes, encodes a light-emitting protein, and we're going to actually show you a picture of light coming out of the animals that we've treated. So what you see on the left are a couple of animals that received no exosome injections. So they didn't receive any of the mRNA-loaded exosomes. These animals have been injected with the substrate for this light-emitting enzyme. It's called DTZ. And you see only background there with just the normal pictures of these mice, and these mice are all alive. On the right are a pair of animals that were injected with our mRNA-loaded exosomes, where the mRNA encodes this light-emitting enzyme, the day before and then after a day to allow the exosome mRNA to deliver that RNA into the cells of the animals and then that RNA being turned into a protein, and that protein folding properly so that it's enzymatically active.

If you then inject the substrate for that enzymes into the animals, the substrate gets to where the enzyme is, and now light comes out of these animals. So you're looking there in red, those are the brightest regions of light emission. And the scale is on the right, and it gives you an idea of how many lumens per second are being emitted in these different areas of the animal. This exposure is just -- I think it's just about a minute or so.

Anyway, so this is a real-time, in vivo live animal evidence of protein expression from our RNA-loaded exosome formulations. And although this is not a therapeutic RNA and it's not a vaccine encoding RNA, it is showing all the same biochemical, molecular and cell biological processes that would occur in any kind of vaccine development program or therapeutic development program. You're getting a real-time live animal version of what gene expression looks like from our formulations. So this shows very directly that our platform can deliver functional enzymes, not just protein but -- because you can have proteins that are properly folding, but functional enzymes. And of course, this is directly relevant to any kind of gene therapy or enzyme replacement therapy type of paradigms that are out there for viral vectors or enzyme injections. We can do the same thing with exosome-based RNAs.

Now I want to say just a little bit about the very earliest work we did on the SARS-CoV-2 vaccine and experiments that actually we performed late last June, early summer on a SARS-CoV-2 vaccine. And just to reiterate our point of view, when we started all this, we wanted to make sure that we could generate at a research level, but also in the manufacturing level, safe, nontoxic exosome mRNA formulations, which we have achieved. We wanted to not just drive antibody and cellular immunity to the spike protein, but we wanted a broader-based vaccine, a vaccine that would drive immunity to the other major structural protein of the virus, which is called nucleocapsid. So we have 2 mRNAs that we synthesized. We formulated them into exosomes at the same time, and we used those formulations to elicit immune responses in mice. And the data confirms that the multiplexed mRNA approach works. We get immune responses to both proteins.

Now I'm going to show you this data from our very first study, and I will just preface this by saying that our efficiency of loading RNA has been optimized well beyond this. Our antigen -- vaccine antigen design has been improved significantly, and we are getting a much better expression even in this very early study that we performed. But even in that very early first study where we had not yet had time to optimize our platform and our formulations, we got very strong immune responses to both the nucleocapsid protein and the spike protein. And the overall regimen of the vaccination and the testing dates is shown in panel A at the top, that line diagram. Below that, in B, shows that we elicited strong anti-nucleocapsid immune responses and in C, that we generated strong anti-spike immune antibody responses as well. And these antibody responses basically came up very early after vaccination and remained constant out with very little decline out through the end of the study.

I'll also say that our dosing here is quite low relative to where we are now in terms of the amount of RNA we delivered. We were only delivering 4 micrograms at that time. We're now up to 30 micrograms, 50 micrograms in our formulations. Not only did we get antibody responses, but the panel on the right demonstrates that we had CD8-positive and CD4-positive cellular immune responses both to spike and to nucleocapsid. And so this is just a snapshot of our approach. Our approach is not limited just to SARS-CoV-2 vaccines but to other vaccines. And more importantly, what we're really excited is our ongoing studies in therapeutics development.

And so I'm just going to say just a couple of words about that. Our view of the vaccines is that we hope to very quickly help in the fight against SARS-CoV-2 with our vaccine development platform. And we are also in the process of generating very interesting research studies on other candidate exosome RNA vaccines. We're very interested in the vaccines, but I want to reiterate that the vaccines are a fantastic developmental program with which we are troubleshooting and optimizing the very issues that also have to be addressed and optimized for therapeutics RNA delivery. We have a very strong focus in the area of therapeutics, and we have a number of developmental projects that we're in the middle of at present.

And I just want to bring you back to those mice that we looked at earlier because they really do demonstrate the potential of exosome mRNA delivery platform that we've been working on here in Baltimore and also in collaboration with our colleagues at Capricor, where we are delivering functional RNAs not just to emit light from animals but to repair genetic defects in animal models as a prelude towards generating clinically useful therapies.

And I think that pretty much sums up what I have to say about the research collaboration and the results that we posted online at bioRxiv earlier this week. And I'll turn it back now to Dr. Marbán.

Thank you, Dr. Gould. That was enlightening even to me who talks to you multiple times per day. Now that you have seen this exciting data, let me tell you where we are in our path to the clinic with our multi-antigen exosome mRNA vaccine.

Currently, we are waiting for feedback from FDA on our pre-IND package. We expect to receive that before the end of this quarter. Our current plan is to move into the clinic and aim to initiate a Phase I clinical trial in Q3 of 2021, if approved by the FDA. The preclinical data presented late last year is compelling, and we are looking forward to advancing this product into the clinic. Of course, as I mentioned previously, we also view this data as proof of the concept that mRNA-loaded exosomes make excellent vaccine candidates, which can potentially be applied to other infectious diseases.

Now let me turn your attention to CAP-1002, our cell therapy product, which is in clinical development for Duchenne muscular dystrophy and the novel coronavirus. I would like to update you on the status of our DMD program first. We remain engaged and focused on the goal of advancing CAP-1002 towards approval for DMD as quickly as possible.

Now allow me to recap where we stand. We have conducted 2 clinical trials using CAP-1002 in DMD. Each trial showed both skeletal muscle and cardiac muscle improvements. HOPE-2, our Phase II clinical trial, showed significant improvement in the PUL or the performance of the upper limb, a measure of upper limb skeletal muscle function, and also in ejection fraction, the gold standard of cardiac function. It is important to emphasize that HOPE-2 was the first randomized, placebo-controlled clinical trial in Duchenne muscular dystrophy to impact both cardiac and skeletal muscle function. As a reminder, cardiomyopathy is typically the leading cause of death in those with DMD. So any improvement in cardiac function would be considered very relevant for both length and quality of life.

Now due to our RMAT designation and the importance of this program, over the past year, we have been in continuous conversations with FDA regarding the approval pathway for CAP-1002 in DMD. This process, though typically not short, has been understandably delayed due to COVID-19. We, with the help of a cadre of consultants, disease specialists, advocates and statisticians, are working on additional analyses to support further discussion with FDA on our approval pathway. We expect to have additional clarity and a final decision by the end of the third quarter of this year.

Now while we have rigorously continued to make our case for an accelerated approval pathway, as we have previously stated, FDA has encouraged us to conduct a Phase III clinical trial. The design of this Phase III trial is that it will be a small and focused study measuring those items that were positive in HOPE-2: The performance of the upper limb, the PUL; and the cardiac measures of ejection fraction and volume. The size of the proposed Phase III trial is estimated to be approximately 60 patients. FDA has indicated its acceptance of this study design. Further, we are also in discussions with several parties regarding a potential partnership opportunity for this program. Regardless, management is committed to moving this asset forward, and we are assessing all strategic options available to us. Currently, we are not planning on conducting the trial until we have final clarity from FDA, and then we will determine the appropriate path forward. We look forward to bringing you updates on this very important program as we have them.

Lastly, we continue to enroll the INSPIRE clinical trial to treat severe but not critical COVID-19 patients. This trial is designed for up to 60 patients and has a 30-day readout. We anticipate data from this by the end of Q3 of this year. This trial is important because it may potentially validate the immunomodulatory effects of CAP-1002, which has been demonstrated not only in previous clinical trials but also in preclinical publications over the past few years. Again, we will provide updates as they become available.

In summary, as you can see, we continue to support this exciting platform. And I am happy to say that we have sufficient resources to execute on our near-term plans and have assembled a world-class team of scientists helping us move this technology forward. We continue to pursue nondilutive sources of capital to fund future clinical work on our programs. I continue to be excited by the progress we are making both in the exosome platform, the latest of which you heard about today, but also with regards to pursuing a pathway for CAP-1002 towards potential approval and commercialization.

I will now turn the call over to A.J. Bergmann, our CFO, for an update on the financials.

Thank you, Linda. This afternoon's press release provided a summary of our fourth quarter and full year 2020 financials on a GAAP basis. You may also refer to our annual report on Form 10-K, which we expect to become available in the next few days and will be accessible on the SEC website as well as the financial section of the company website.

As of December 31, 2020, the company's cash, cash equivalents and marketable securities totaled approximately $32.7 million compared to approximately $9.9 million on December 31, 2019. Based on our current plans and projections, Capricor expects that its cash and cash equivalents will fund our research and development programs and other operations through at least the second quarter of 2023.

Turning quickly to the financials. In the fourth quarter of 2020, our net cash used in operating activities was approximately $3.8 million for the fourth quarter of 2020. Excluding stock-based compensation, our research and development expense was approximately $2.7 million compared to approximately $800,000 in Q4 of 2019. Again, excluding stock-based comp, our general and administrative expense was approximately $1.1 million in Q4 2020 and approximately $800,000 in Q4 2019. Net loss for the fourth quarter of 2020 was approximately $4.2 million compared to a net loss of approximately $1.5 million for the fourth quarter of 2019, and net loss for the full year of 2020 was approximately $13.7 million compared to a net loss of approximately $7.6 million for the full year 2019. We continue to remain financially focused on the development of our cell and exosome programs.

We will now open the line up for questions.

Thank you, A.J.

(Operator Instructions) Our first question today comes from Jason McCarthy of Maxim Group.

This is Michael Okunewitch on the line for Jason. So for the first question, I kind of want to look at how we should look at Capricor going forward. It seems like much of your future development is focused on the engineered exosomes. So would it make sense to view Capricor more in the same space as companies like Codiak or Evox than the more traditional signaling-based cell therapy companies going forward?

Thank you, Michael. Yes, you have pegged it exactly as we see it. So we see ourselves as actually sitting somewhere between what I would call a Moderna and a Codiak or an Evox. We are taking RNA medicines. We're putting them in exosomes as delivery vehicles, and we are planning on deploying them for everything from the vaccine work you saw today all the way through to therapeutics for monogenic diseases, other types of vaccines and other types of diseases as well. So we are definitely moving away from cell therapy. We remain bullish about CAP-1002. It works. We believe very strongly in it. We plan on taking it all the way through commercialization one way or another. But right now, we are focusing most of our development efforts on the exosome platform.

And actually, regarding the mRNA strategy, is that going to be focused more so on developing your own internal mRNA pipeline? Or are you going to position Capricor as a potential delivery partner for external collaborative programs?

Yes. So our plan's similar to what you'll see sort of on rolling with Codiak and Evox and some of those others, is to have some programs that we maintain internally. We certainly have a good focus and have been building in monogenic disease over the past few years as well as obviously some of this vaccine technology that we're working on right now. But we also plan on making this so translatable, so plug-and-play that we'll attract a large number of partners and also be able to develop this in conjunction with them.

Awesome. And then one more, if you don't mind, on the actual mRNA vaccine candidates. I'd just like to ask if an exosome-based mRNA vaccine, you would expect it to have the same ultralow temp cold chain requirement as the current mRNA vaccines? And then also, given the improved delivery of the genes, is there a possibility for having it as a single dose vaccine?

Yes. So at this point, we're still working on storage. But we're anticipating the cold chain type of storage that's necessary for the current mRNA vaccines. And in terms of single dose, we remain open to that possibility. The current design that we have is 2 doses as is, again, with the current mRNA vaccines. But it's certainly possible that we could ultimately move towards a single dose.

The next question is from Joseph Pantginis of H.C. Wainwright.

This is Emanuela on for Joe. So my first 2 questions are related to the development of the exosome-based vaccine. The first one is, I guess, more short term. You obviously have done a lot of work already, and you're waiting for FDA feedback. But can you give us a sense of the work that may still be required for entering the clinic with the vaccine?

Yes. So we remain poised and ready to go into the clinic based on the feedback from the FDA. We can't anticipate what they're going to ask for, but one of the advantages that Capricor has in the development of this new platform pipeline is that we've been in the clinic before. We've been working with the FDA for a lot of years. We know what it takes to get something through the regulatory process. And so I will tell you that we are ready to go as soon as we complete the requirements that FDA asks for.

Got it. And like more on the long term, I guess, what do you think the regulatory pathway could look like, given that there is no precedent yet for approval of an exosome-based therapeutics? I guess like what is your impression of the general sentiment of the regulator? And what do you think is the aspect that would require more work, if any?

So we actually have an IND, an active IND with an exosome product that we have not completed. But we got a lot of feedback and working with the FDA on that program. It gave us a lot of clarity on what they would look for. Of course, things can change, and the field is moving forward rapidly. But I really do think that the regulatory process will be easier than with cells, certainly and probably similar to something that would be like an antibody. You have to go through all of the biologics and -- but I don't think there'll be anything very specific.

The exosomes -- one of the great values of working with the engineered exosomes and why we switched basically from using primarily a CDC-based exosome to an engineered exosome is it functions like a drug. So you can load it with whatever you want to put inside there. We're using RNA now, but it could be a protein. It could be another nucleic acid. And then you can develop potency assays as well as other types of metrics of mechanism of action in a very clear and concise way. So it should be a relatively straightforward regulatory pathway.

Got it. And switching to CAP-1002, you mentioned you may pursue a partnership for the program. Can you give us an idea of what would be the ideal partner for CAP-1002 development?

The ideal partner for Capricor for CAP-1002 development, obviously, would be a corporate partner that has activity and/or commercial product in the Duchenne muscular dystrophy space. It's definitely the type of program and exposure that we would like. However, we're in conversations with multiple partners at this time. And I think the intriguing clinical data, the strong implications of the mechanism of action and the unmet medical need of the non-ambulant boys and young men, make this a really desirable target. So we're very excited about the pathway for CAP-1002 right now.

The next question comes from Alan Leong of BioWatch News.

Congratulations on the positive preclinical data. I was noticing that there's quite amount of additional data points that you guys are doing with the INSPIRE trial. I was wondering if you plan to go for a label expansion on post-COVID patients?

Yes. So we're looking at a lot of things in the INSPIRE trial. As you can imagine and as the story of COVID has unfolded, it's very difficult to see exactly how the disease progresses and then also what improvement looks like. So we built the trial with end points so that we can best plan the path forward, and we're not ruling out label expansion. We're not ruling out that if the data were good enough, we wouldn't try and take it to FDA and see for some type of an accelerator program. We're also not ruling out that it might need further clinical development, which we would evaluate doing at the time based on the pandemic itself, the vaccine applicability in terms of the general population and that kind of thing.

So to sort of summarize, we are really excited about INSPIRE because it hits really closely to what we've demonstrated, is the immunomodulatory impact of the cells over many years, including in DMD. But we remain curious and open to the impact of CAP-1002 in COVID-19.

Wonderful. Yes. Regarding 1002 for DMD, you mentioned that you were working on some additional studies with outside collaborators. Is there any color that you can provide on that, on what you might be looking for?

Yes. So these are not clinical studies. What we are doing is we're working with consultants, advocates as well as statisticians to fully understand the impact of CAP-1002 in DMD. We feel that it's very important because this is -- even though it's a monogenic disease, it's obviously very heterogeneous in its presentation. And so what we're doing is we are doing analyses to best figure out how the patients are impacted. I can tell you that we are extremely excited about what we are finding, and we're talking to the FDA about it currently. And so stay tuned for more information there.

Wonderful. Yes, I really look forward to it. It needs to be realized. The last question I had for you is really just regarding delivery system of the exosome platform in comparison to lipid nanoparticles, if there was anything that you'd like to expand on that. I view it as a big problem in the space. So what do you see in terms of market potential? Or how much interest has really been brought to your attention regarding that platform?

Yes. So I'm going to actually ask Steve to elaborate a little bit on that one. Steve, if you're still on the line, would you be willing to answer that question for Alan?

Sure. Yes. I mean, in the general area of RNA delivery, a challenge for therapeutics, whether you're delivering mRNAs to treat a monogenic disease or small interfering RNAs or microRNAs to fight cancers, you have to have a platform that is safe, that is reproducibly -- repeatedly doseable. And I think this is an area where exosomes really shine through in terms of potential. We -- the many, many animals now, we never see adverse effects of exosome injections even when they're loaded with RNA, even when they're dosed at very high levels, even when we dose very frequently over and over and over again. And we have some -- a lot of really interesting data that we'll be presenting in the not-too-distant future, I hope, on repeat dosing efficacy as well as tolerability. So those areas are clear-cut opportunities for exosome-based therapeutics and vaccines.

Another issue that -- where exosomes have a lot of potential is in the area of targeting to specific tissues based on molecular tropism that you engineer into the exosomes. So the exosomes are delivery vehicle, but they're not completely inert. And while we do harvest them from cell cultures, we have numerous avenues open to us for engineering in molecular targeting modalities, small molecules, biologics, what have you, by various different techniques. And so we're very excited by our ongoing efforts in this area.

And I just want to point your attention back to those mice we showed. Because we can so easily a monitor in real-time gene expression from our platform, we can very quickly run through many, many variables in our design, in our manufacturing that affect targeting efficiencies. I mean, this is an extremely important thing to be able to do. We know this from the extensive industry expertise in biologics, where it's not just having the right antibody but knowing how to optimize it for proper stability in the bloodstream, et cetera. And so we're very interested both in exploring the safety profile differences and in exploring the tropism differences and the repeat dosing differences. Those are kind of the 3 areas where I see exosomes as having really high ceiling.

(Operator Instructions) There are no additional questions at this time. I would like to turn the call back to company management for closing remarks.

Thank you. And thank you for joining us today. As you can see, in 2020, we underwent a complete pivot away from cell and gene therapy and towards this new exosome platform, which we think will bring great opportunities both in terms of vaccine but also in terms of therapeutic development. We look forward to providing you with updates on all of the programs that we have, and please stay well during these very difficult times. Goodbye.

This concludes today's conference. You may disconnect your lines. Thank you for your participation.