Q4 2020 Capricor Therapeutics Inc Earnings Call

Critics and welcome to the Capricorn Therapeutics fourth quarter full year, 'twenty, and 'twenty earnings and corporate update call.

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A question and answer session will follow the formal presentation.

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As a reminder, this conference is being recorded.

I'd now like to turn the conference over to your host AJ Bergmann Chief Financial Officer.

Mr. Brookman are you muted.

Yes, thank you very much the.

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 the anticipated conduct and timing of preclinical and clinical studies our plans for presenting.

The reported additional data our plans regarding regulatory filings.

Potential regulatory developments involving our product candidates and our possible uses of existing cash and investment of resources. These forward looking statements are based on current information assumptions and expectations that are subject to change and evolve the 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 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.

And I'll turn the call over to Linda Moore ban 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 access on the platform technology, which was highlighted in the paper published earlier this week and bio archive and will then provide a brief overview of our.

Other program.

We are extremely excited about the progress that we've made in 2020 and looking ahead, we are very enthusiastic about 2021.

Yeah.

Joining me today is Dr. Steven Kwok Professor of biological chemistry of Johns Hopkins University Doctor Gold has worked and the extra dome field for nearly two decades and works of cap of core both us and executive consultant and scientific collaborator Doctor Gold has helped accelerate cap of course excess of progress, bringing new ideas.

<unk> innovative technology and valuable Knowhow, while also helping us assembled the team of scientists and our Los Angeles labs to work on product development and manufacturing.

We have also entered into a sponsored research agreement with Johns Hopkins University to support the extra from program.

I will begin my remarks with a brief overview of the goals of our ex US on platform and then I will turn it over to Dr. Gould to walk through the data and its implications for Exosomes therapeutic development pipeline.

I hope you'll be as excited as I am by our findings and we will see the power of that this platform can potentially provide and the development of therapeutics for a broad spectrum of diseases.

Mrna therapeutics, our new class of medicines pioneered by companies such as Madonna and other 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.

Ed.

RNA medicines are sets of instructions and these instructions direct sales and the body to make all of the proteins required for life and.

Well as to prevent or fight disease the field.

And have 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 for you.

And we're excited to be part of this new wave of opportunities and biotechnology.

And the field and the whole looks beyond mrna vaccine development.

One of the most critical aspects to the repeat of delivery of nucleic acids to treat diseases are effective and safe delivery systems.

While we think about platform delivery systems for mrna and other nucleic acids and there are many systems that had been developed most notably the lipid nanoparticle, but it's our belief the nature of our communications system. The Exosomes offer great.

Potential due to their unique properties.

Now lets remind you extra films are produced by all cells abundant and all of bio fluids and have been demonstrated to be safe by decades of transfusion and transplantation medicine and.

Additionally, they can be directed or in other words targeted to the cell type and we'd like to treat and are readily able to deliver of payload to the self directing protein expression of.

Capricorn 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 on the answers and we believe that exosomes can provide a platform and drug development with that possibility.

The data that has been generated by Johns Hopkins and Capricorn is focused specifically on expression of functional proteins and using our <unk> platform delivery system as well as on the toxicity and safety effects when compared to traditional lipid nanoparticle.

And these aspects support the goals of Capricorn ex yourself 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 the skills.

Capricorn and 2021 is focused on becoming a leading extra zone RNA company and all of our work against Sars Cov, two is simply a prelude to the future development of exit non based vaccines and therapeutics with the ultimate goal of generating formulations.

Engineered exosomes and synthetic mrna to prevention and prevent and treat human diseases I.

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

Yes.

Thank you Dr <unk>.

Yeah, there's been a very clear presentation of the general approach that we've been pursuing here and Hopkins and.

And that we started a very deep and broad collaboration with cap of core over the past year.

We are very excited to be working with cap of core and allows us to expand our horizons. Both in terms of the basic research, but also on the applying the the rules of ex yourself by Genesis and production and targeting.

<unk> actual real world clinical problems.

Our approach is essentially.

Outlined and this wide here a bit.

A great need to make sure that we develop the <unk> platform and <unk>.

High throughput way, so that we can optimize the production index. So the loading of the.

And the formulation the manufacturing and the targeting and so what I'm going to start with is talking a little bit about.

And a small piece of the data that we've been collecting about the general utility of Exosomes platforms for RNA delivery.

One of the questions that you might be thinking of it right away.

How the excess compare other RNA delivery platforms, specifically lipid nanoparticles or L. N piece and so we would undertake 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'd like to show us.

You're here that reflects wage change need and response to the injection of equal.

Numbers of ask yourselves or equal numbers of lipid nanoparticles and black dots are the weight of the animals on day zero the.

Red squares of the weights of animals three days after the injection and what we have seen is that.

At <unk>.

<unk> injections not engender any growth adverse effects that can be detected upon visual examination of the animals weighing the animals.

And this is not quite true for L. M P's.

And then equal numbers of vehicles being rejected what we see is that of over a short period of time.

LNP injections can lead to a reduction in weight of the animals and reduction of weight and.

It's just a very useful marker for any kind of treatment that causes the animals to seal of bit off it.

It doesn't necessarily either.

Horrible toxicity of the brewing here, but it is indicative of the adverse effects of the treatment. So this is just inject the animals with ex yourselves that went back and Jackie and animals with an equal number of M. P's.

Causes of some distress to the animals now.

And that's just measuring weight of animals, the very easy thing for us to all understand.

And and we all know when we seal the deal well, we tend not to eat as much.

But the real key is looking and these animals by detail just the logical analysis, but that shown and this why now there's a lot of panels here.

To make it a little simpler let me go through it.

And the top row are histological samples from control mice and.

Page and these were done the multiple animals and each group.

Troll animals and examine for morphology of the art and test and salivary gland spleen, kidney liver heart, <unk> lung and brain and based on and independent histological analysis. These animals are totally normal.

And the same was true for all animals that were injected with ex yourself. So we're injecting animals, either with the saline or injected and with an equal number of extra some sort of L. N peds.

And the act of self inject the animals again, the histological report came back normal and all tissues and all animals and the same cannot be said for those animals that were injected with the same number of LNP and these LNP as are very similar.

The same base of composition and as the LNP that are being administered and some of the leading vaccines. So while there was no abnormalities and most tissues and there was an abnormality and most of the animals and their split and.

So what was specifically see what was specifically seeing and the L. M. P. Injected animals was an increase of a high increase and the amount of red pulp and the split and the bed pulp is is the indication of sort of non specific inflammation and.

And this goes hand in hand, with the weight loss data that we saw and these paired animals. So just some of these two pieces of data and what.

And we're showing you is that injections of Exosomes and large numbers of Exosomes have 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 ex yourselves.

And the people there is no.

Not associated with any adverse effects and so we're basically just reproducing what kind of what the Madison already knows.

In contrast injection of L N piece.

A mildly inflammatory by this assay.

And so that's just that's just the delivery vehicles, comparing lnp's of the vehicle ex items of the OCA one of the effects of those alone.

Well. The next question is well how did these deals.

Livery vehicles compare in terms of the efficiency with which they can function of we deliver and M. Rnas.

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 and I'm just going to show you one little snapshot of the data so well what youre looking at here is the expression.

And a luciferase a light emitting enzyme.

Net assay on sales that are grown and culture. So the yourselves all of these when they don't normally and any light of what we can do though is we can synthesize in vitro and.

Messenger RNA that encodes an enzyme elusive for ace that can be used to.

As a marker for how efficiently, we're delivering mrna into the living cells. So again the.

The the particles of the ex some of them.

Sales do not have this enzyme activity the LNP and do not have this activity. The activity is not strictly president and the army. The army has to get into the cytoplasm of other cell and has to be translated into of protein and that has to be done with a high degree of fidelity. So that that protein is.

Enzymatically active so he can emit light so once that's made.

And it's capable of the bidding when given the substrate. So what we've done here is we have control of cells. That's the bar on the far left you can see those control sales don't.

In response to the substrate and Theres a subsidiary called <unk>.

And at the enzymes present light is and if you add the substrates of the control sales, there's no activity and that's why all of those black circles of control are right at the bottom of zero light admission no. When we load a standard amount of constant amount of RNA into tens of the allowed.

And particles per mill.

The lipid nanoparticles, we do get good expression of this light admitting enzyme.

But when we do the exact same parallel experiment the same amount of RNA of the same numbers of Abaxis homes as of on teams, we get more light admission, which means we're more efficiently delivering that RNA into the cytoplasm of human cells, and it's being turned into the functional enzymes.

Exosomes are described neither of those two bars and the middle of showed that now.

Now when you go to a high dose administration and high dose administration is particularly important for therapeutics mrna delivery when youre going to have to be delivering a lot of RNA. Many many times in order to cure and I say genetic diseases and what we see is that the.

The difference between extra cell mediated mrna.

And LNP mediated RNA delivery is really dramatic.

Now more than a tenfold difference.

And as you can see what happens is that when you dose with large amounts of lipid nanoparticles and you get an adverse effect on the expression of the RNA of the big carry so there seems to be an upper limit on LNP and media. The mrna delivery that you don't have with Texas mediated RNA delivery and.

This is a really important point I know that this is just the tissue culture experiment, but what you see and human cells and tissue culture is very often of very good sort of.

Indication of what Youll see in vivo.

And I'm going to show you some in vivo data and the next slide and which we take the same mrna loaded ask yourselves, where the mrna and codes a light emitting protein.

Yeah and codes of light and many proteins and we're going to actually show you a picture of light coming out of the animals that needs treatment.

So what you see on the left are a couple of the animals that received no extra self injections and so they get don't receive.

Any of the mrna rotary axis films. These animals have been injected with the substrate for the slight of many enzyme it's called <unk> and.

And you see only background and just the normal pictures of these mice.

And these mice or all of the lives on the right are a pillar of the animals that were injected with our and I'm already loaded ex yourselves or the mrna and code the slight embedded inside the.

And the day before.

And then after a day to allow the extra so memory and a to.

And to deliver that the RNA into the cells of the App.

Animals and.

And that RNA being turned into a protein and.

And that protein folding properly so that it is enzymatically active.

If you then inject the substrate for that enzymes into the animals, the substrates and gets to where the enzyme is and now like comes out of these animals. So youre looking there and read those of the brightest.

Regions of why the mission and the scales on the right and.

You have an idea of how many millions per seconds of the Eaton and mitigate 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 ex some formulations and although this is not a therapeutic all of our day and it's not a vaccine and coding RNA and is showing all of the same biochemical molecular and cell biological.

Processes that would occur in any kind of vaccine development program or therapeutic development program.

Youre getting a real time live animal version of what gene expression and looks like from our formulations. So this shows very directly.

And our platform can deliver functional and designs not just protein but.

And you cannot proteins that are not profitable.

But functional enzymes and of course this is directly relevant to any kind of gene therapy of enzyme replacement therapy type of paradigms that are out there for viral vectors of enzyme injections. We can do the same thing with the exercise based on the names.

No.

I wanted to take just a little bit about the very earliest work we get.

And the Sars Covid, two vaccine and experiments that actually we performed a late last June early summer on of Sars Covid, two vaccine and just to reiterate our point of view when we started all of this we wanted to make sure that we could generate.

And of research level, but also and the manufacturing level safe non toxic acrosome mrna formulations, which we have achieved.

We wanted to not just drive antibody and and cellular immunity and fleet to the spike protein, but we wanted the broader based vaccine.

Vaccines that will draw.

Dry immunity to the other major structural protein of the virus, which is called nucleocapsid. So we have two mrna is that we synthesize we formulated them into the exosomes at the same time and.

And we use those formulations.

To elicit immune responses and mice and the data confirms that the multiplex mrna approach works, we get immune responses to both proteins now.

And I'm going to show you. This data from our very first study and I'll just preface this by saying that our.

Additional C of loading RNA has been optimized well beyond this our antigen.

Vaccine antigen design has been improved significantly and we're getting much better expression even there.

Early studies that we perform but even in that very early first study, where we had not yet had time to optimize our platform and our formulations and we got very strong immune responses to both of the nucleocapsid protein and the spike proteins and the.

The overall regimen of <unk>.

Vaccination and the testing dates as shown and.

Panel a at the top of that money diagram.

But low that the D shows that we elicited a strong anti nuclear capsid immune responses.

And in C that we generated strong anti spike immune and antibody responses as well and these antibody responses basically came up very early after vaccination and remain constant out with.

And with very little decline out through the end of the study and I'll also say that our dosing here is is quite low relative to where we are now in terms of the amount of RNA be delivered we were only delivering four micrograms at that time, we're now up to 30 micrograms 50 micrograms and our formulations.

Not only did we get the antibody responses, but the panel and the right demonstrates that we had CD eight positive and CD for positive cellular immune responses, both to the spike and nuclear capsid.

And so this is just a snapshot of our approach our approach and not limited just to the Sars cov two vaccines a bunch of other vaccines and more importantly, what we're really excited is our ongoing studies and 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, two with our vaccine development platform.

And we are also in the process of generating.

Very interesting research studies and other candidate excess of RNA vaccines.

We're very interested and the vaccines, but I want to reiterate that the vaccines are fantastic developmental program with which we are troubleshooting and optimizing the very issues that.

Also he has to be.

Addressed and optimized for therapeutics, RNA delivery and the very strong focus in the area.

And we have a number of the.

Mental projects that were in the middle of at present and.

I just wanted to bring you back to those mice that we looked at earlier.

Because the.

They really do demonstrate the potential of <unk>.

To sum mrna delivery platform that we've been working on here in Baltimore and also in collaboration with our colleagues of Capricorn.

We are delivering functional Rnas and not just two of net life from the animals, but to repair of genetic defects and animal models as a prelude towards generating and clinically useful therapies.

And I think that pretty much sums up what I have to say about the the research collaboration and the results that we posted online.

The archives earlier this week and I'll turn it back now to a doctor and barbell.

Thank you Dr. Trickled that was enlightening even to me who taught.

And to multiple times per day.

And now.

That you have seen this exciting data, let me tell you, where we are and our path to the clinic with our multi antigen and exit them 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 one clinical trial and Q3 of 2021 and if approved by the FDA the free.

Preclinical data presented late last year is compelling and we are looking for it for advancing this product and to the clinic of course as I mentioned previously we also view of this data is proof of the concept the mrna loaded exosomes make excellent vaccine candidates, which kind of potentially be applied.

The other infectious diseases.

Now, let me turn your attention to cash tenor to our cell therapy product, which I think of clinical development for Duchenne muscular dystrophy and the novel Coronavirus.

I would like to update you on the status of our G. M D program first.

We remain engaged and focused on the goal of advancing cap kind of O two towards approval for DMT as quickly as possible now.

Now allow me to recap, where we stand we have conducted two clinical trials using cap 10 O two and DMT.

Each trial showed both skeletal muscle and cardiac muscle improvement.

Hope to our phase II clinical trial showed significant improvement and the pool or the performance of the upper limb and measure of upper limb skeletal muscle function.

And also and ejection fraction the.

Gold standard of cardiac function.

It is important to emphasize that hope to was the first randomized placebo controlled clinical trial and Duchenne muscular dystrophy true.

The impact both cardiac and skeletal muscle function.

As a reminder.

Cardiomyopathy is typically the leading cause of death and those with DMT, So any improvement and cardiac function would be considered very relevant for both life and quality of life.

Now to tour, our Mac designation and the importance of this program over the past year, we have been and continuous conversations with FDA regarding the approval pathway for cap kind of true and DMT.

This process. The typically nausea work has been understandably delayed due to COVID-19.

We with the help of a cadre of consultants rare disease specialist advocates of the statisticians are working on additional analyses to support further discussion with FDA on our approval pathway.

We expect to have additional clarity and the final decision by the end of the third quarter of this year.

And while we have rigorously continued to make our case for an accelerated approval pathway.

And we had previously stated SCA has encouraged us to conduct a phase III clinical trial.

The design of the phase III trial is that it will be a small and focused study measuring those items that were positive and hope to the performance of the upper limb the pole and.

And the cardiac measures of ejection fraction and volume.

The size of the proposed phase III trial is estimated to be approximately 60 patients.

F D. A has indicated that the acceptance of this study design.

Further we are also and 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.

And we look forward to bringing you updates on the 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 Q2 and three of this year.

This trial is important because it may potentially down the state the immuno module of Torrey effect of top 10 of two which has been demonstrated not only and previous clinical trials, but also like for.

And of course 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'm 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 non dilutive sources of capital to fund future clinical work on our program.

I continue to be excited by the progress we are making both and the extra zone platform the latest of which you've heard about today, but also with regard to pursuing a pathway for captain of two towards potential approval and commercialization all of them.

And now I'll turn the call over to AJ 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 and the next few days and it'll 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 and.

December 31, 2019 day.

And our current plans and projections Capricorn expects that its cash and cash equivalents will fund our research and development programs and other operations for at least for the second quarter of 2023.

Turning quickly to the financials and the fourth quarter of 2020 of our net cash used in operating activities was approximately $3 8 million.

For the fourth quarter of 2020, excluding stock based compensation of research and development expense was approximately $2 7 million compared to approximately 800000 and Q4 2019.

Again, excluding stock based comp our general and administrative expense was approximately $1 1 million and Q4 2020.

And approximately 800000 and Q4 2019.

Net loss for the fourth quarter 2020 was approximately $4 2 million compared to a net loss of approximately $1 5 million for the fourth quarter of 2019 and.

The 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 of 2019.

We continue to remain financially focused on the development of our cell and <unk> Zone program.

We will now open the lineup for questions. Thank you a J.

Thank you we will now begin the question and answer session.

If you would like to ask a question press star one on your telephone keypad.

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One moment, please while we poll for questions.

Our first question today comes from Jason Mccarthy of Maxim Group. Please proceed with your question.

And as Michael of Qunar, which on the line for Jason Thanks for taking the question.

So for the first question I kind of want to.

Look of how we should look at cap record going forward and it seems like much of your future development is focused on the engineered of Exosomes.

So would it make sense to view cap of core more and the same space with companies like Kodiak or E box than the more traditional signaling based cell therapy companies going forward.

Thank you Michael and please send my regards to Jason Yes.

You have the pegged it exactly as we see it so we see ourselves and it's actually sitting somewhat between what I would call them of daughter and of Kodiak or any of Ark, and we are taking RNA medicines for putting them as act and Exosomes and delivery vehicles, and we are planning on deploying them for everything from the vaccine work you saw today all the way through.

Of the therapeutics for monogenic diseases, and other types of vaccines and other types of disease.

Diseases as well so we are definitely moving away from the cell therapy, where we remain bullish about top 10 of two it works, we believe very strongly and it.

We plan on taking it all the way through commercialization of one way or another but right now we are focusing most of our development efforts on the extra non platform.

Thank you.

And actually we're guarding 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 cap of core as a potential delivery partner or external collaborative programs.

Yeah. So our plan is similar to what you'll see sort of enrolling with Kodiak and any box and and some of those others is to have some programs that we maintain internally. We certainly have a good focus and have been building and monogenic diseases over the past few years as well as obviously some of the.

Vaccine technology that we're working on right now.

But you know we also plan on making the so translatable sort of plug and play that will attract the large number of partners and also be able to develop this in conjunction with them.

Okay.

Awesome and then one more if you don't mind on the actual mrna vaccine candidate.

And just like the ask if and ex of Exosomes based MRI and the RNA vaccine you would expect the tab of the same ultra low temp cold chain requirement.

And as the current mrna vaccines and then also given the improved delivery of the genes is there a possibility for having it is the single dose vaccine.

Yeah. So at this point, we're still working and 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 two doses as is again with the current mrna vaccines, but it's certainly possible.

That we could ultimately move towards the single dose.

Alright, and again, thank you very much for taking my questions and congrats on the progress.

Thanks, Thanks, a lot.

The next question is from Joseph Thank goodness of H C. Wainwright. Please proceed with your question.

Good afternoon, guys and one way of life.

And for Joe.

Thank you for taking my questions. So my first two questions are related to the development of the extra from based vaccine and for.

First of all on either I guess more sharp there and.

And you obviously have done a lot of work already and you're waiting for FDA feedback about the can you give us a sense of the work that may still be required for the entering the clinic and win.

And the vaccine.

Yeah. So you know we remain poised and ready to go into the clinic based on the feedback from the FDA, we cant anticipate what theyre going to ask for but yeah, and one of the advantages of cap of core has and the development of this new platform pipeline and that we've been in the clinic before we've been working with FDA for a lot of years, we know what it takes to get that.

The thing through the regulatory process and so I will tell you that we are ready to go as soon as soon as we complete the requirements are the FDA asks for and.

Got it thank you for that and like more on the long term and my guess what do you think the regulatory pathway could look like and given that there is no precedent yet for the approval of an extra the AUM based therapeutics I guess, what is the impression of the general sentiment of direct data and what do you think is the aspect of it would require more and more.

Mark.

Yeah, and we actually have and <unk> and active I N T.

And ex us on product that we have not completed but we got a lot of feedback and working with FDA on that program and gave US a lot of clarity on what they would look for of course, you know things can change and and the field is moving for a rapidly but I really do think that the regulatory process will be easier.

Neither of them of self certainly and probably similar to something that would be like and antibody. You know you have to go through all of the biologic and but I don't think there'll be anything very specific the ex is one of the great values of working with the engineered access to all of US and why we switched basically from using primarily of C. D. C based acts of them too.

And engineered accident and the functions like a drug and so you can load it.

With you know whatever you want to put inside there were using RNA now, but it could be approach and it could be another nucleic acid and then you tend to develop potency assays as well as other types of metrics of the mechanism of action and a very clear and concise way. So it should be a relatively straightforward regulatory pathway.

Hi, Thank you for that.

And switching to captain of two you mentioned you may pursue of partnerships for the program can you give us an idea of what would be the ideal popping out of for a couple of times too much of your government.

The idea of partner for cap of corporate Cat 10 of two development, obviously it would be of a corporate partner that has activity and of our commercial products and the duchenne muscular dystrophy space and it's.

Definitely you know the type of program.

And the exposure that we would like however, our conversations with multiple partners at the time and I think the intriguing clinical data the <unk>.

Strong and implications of the mechanism of action and the unmet medical need of the non ambulant boys and young men and makes us of really desirable target. So we're very excited about the pathway for captain of two right now.

Thank you very much.

The next question comes from Alan Liang of Bio Watch News. Please proceed with your question.

Hi, yes. Thank you for taking taking the time of answering my questions and congratulations on the positive preclinical data.

I was noticing that the there's a quite amount of the additional data points that you guys are for dealing with the inspire trial and I was wondering if you plan to go for a label expansion on the post COVID-19 patients.

Yeah. So you know we're looking at a lot of things and the inspire trial as you can imagine and as you know the story of Covid has unfolded. It it's very difficult to see exactly how the disease progresses, and then also you know what.

The improvement looks like so we built the trial with endpoint and so that we can best plan and the path forward and we're not ruling out label expansion and we're not ruling out that if the data were good enough, we wouldn't try and take at the FDA and see for some times of the accelerated program. We're also not ruling out that it might need further clinical development, which we would evaluate I'm doing at.

At the time based on the pandemic itself the docs.

The vaccine.

Applicability.

In terms of the general population and and that kind of thing so.

To summarize and we are really excited about and fire because it hits really closely to what we've demonstrated is the immuno module Tory impact of the sales over many years, including and D M D and <unk>.

We remain I'm curious and open to for the impact of Cop 10 of two and COVID-19.

Wonderful.

Regarding the 10 O two for for D. M D.

You mentioned that you were working on two additional studies with the outside collaborators is there any color that you can provide on that.

And what you might be looking for.

And so these are not clinical studies of what we are doing is we're working with them.

Insult and advocates as of.

Well the statisticians to fully understand the impact of cap kind of two and TNT and we feel that it's very important because this is even though it's a monogenic diseases, obviously very heterogeneous.

<unk> genius and its presentation and so what we're doing is we are doing analysis to figure out and 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 Yeah look I really look forward to it.

The it needs to be realized the the last question and I have for you is really just regarding delivery system of the ex as one platform and comparison of the lipid nanoparticles. If there was anything there.

You'd like to expand on that I view it as of a big problem and the space. So you know where do you what are you seeing in terms of market potential or how.

How much of interest has really been.

And then brought to your attention regarding that the step.

The platform.

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

Sure.

In the general area of RNA delivery.

No.

Challenge for therapeutics, whether your delivery of mrna is to treat a monogenic disease or small interfering RNA as of Microrna means to fight cancers.

You have to have a platform that is safe.

That is reproducible.

Repeatedly dose of <unk> and I think this is an area where extra sounds really shine through in terms of the potential.

And we.

Many many animals now we never see adverse effects of excess of injections, even when they are loaded with our day, even when they're dosed at very high levels, even when we dose very frequently.

And 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 and efficacy as well as tolerability. So those areas are clear cut opportunities for <unk> based.

<unk> therapeutics and vaccines.

Another issue that.

You know where ex or some type of a lot of potential lives and the area of targeting specific tissues.

Just on molecular tropism that you engineer into the ex yourselves, so the ex yourselves or delivery vehicle, but theyre, not completely and nurse and and while we do harvest stem from cell cultures, we have numerous avenues open to us for.

The engineering and molecular.

The targeting modalities small molecules buyer.

Biologics.

And what have you buy the various the protected and so we're very excited.

And by.

And by our.

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.

Monitor in real time gene expression from our platform. We can very quickly run through many many variability the variables and are in our design and our manufacturing that effect.

Targeting efficiencies I mean the.

This is an extremely important thing would be to do we know this from the extensive industry expertise of biologics, where it's not just having the right antibody, but knowing how to optimize it for proper stability and the blood stream et cetera, and so we're very interested.

And exploring the safety.

Our profile differences and and exploring the trophy and some differences and the repeat dosing differences and those are kind of the three areas, where I see extra sounds as having really high ceilings.

Yeah.

Awesome, Thank you and I'm excited for the future.

Thank you very much thank you both.

Are there any more questions.

As a reminder, if you would like to ask a question. Please press star one on your telephone keypad.

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

Thank you and thank you for joining us today and you can see and 2020, we wonder want a complete pivot.

Away from cell and gene therapy and towards the new access platform, which we think will bring great opportunities. Both in terms of the vaccine, but also in terms of therapeutic development. They look for it to providing you with updates on all of the programs that we have and please stay well during these very difficult times.

Yeah.

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

Yeah.

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