Q4 2020 Earnings Call Transcript

Published: 11 Mar 2021

[Operator:]

Greetings, and welcome to Capricor Therapeutics Fourth Quarter Full Year 2020 Earnings and Corporate Update Call. At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. As a reminder, this conference is being recorded. I would now like to turn the conference over to your host, AJ Bergmann, Chief Financial Officer. Mr. Bergmann, are you muted?

[AJ Bergmann:]

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.

[Linda Marbán:]

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 Exosomes 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 two decades and works at 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 Johns 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 exosomes therapeutic development pipeline. I hope you will be as excited as I am by our findings. And we'll see the power that this platform can potentially provide and 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 steps 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.

[Stephen Gould:]

Thank you, Dr. Marbán. Yes, it has 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 and 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 to actual real world clinical problems.

[Linda 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 and our path to the clinic with our multi-antigen exosome-mRNA vaccine. Currently, we're 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 1 clinical trial and Q3 of 2021, if approved by the FDA. The preclinical data presented late last year was 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 two clinical trials using CAP-1002 and DMD, each trial showed both skeletal muscle and cardiac muscle improvements. HOPE-2 our Phase 2 clinical trial showed significant improvements in the pole or the performance of the upper limb, a measure of upper limb skeletal muscle function, and also an ejection fraction, the goal 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 lengths and quality of life. Now due to our RMAT designations 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 and DMD. This process fall typically not short has been understandably delayed due to COVID-19. We with the help of a cardiac consultants, rare disease specialists, advocates and statisticians are working on additional analysis to support further discussion with FDA on our approval pathways. 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 3 clinical trial. The design of this Phase 3 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 volumes. The size of the proposed Phase 3 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 or 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 trials 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 down-state, the immunomodulatory effect of CAP-1002, which has been demonstrated not only in previous clinical trials, but also in pre-clinical 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 programs. I continue to be excited by the progress we were 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 AJ Bergmann, our CFO for an update on the financials.

[AJ Bergmann:]

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, it 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 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 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 ourselves in exosome programs. We will now open the line up for questions.

[Linda Marbán:]

Thank you, AJ.

[Operator:]

Thank you. We will now begin the question-and-answer session. Our first question today comes from Jason McCarthy of Maxim Group. Please proceed with your question.

[Michael Okunewitch:]

Hi, this is Michael Okunewitch on the line for Jason. Thanks for taking the question. 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 makes 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?

[Linda Marbán:]

Thank you, Michael, and send my regards to Jason. Yes, you have pegged it exactly as we see it. So we see ourselves as actually sitting somewhat between what I would call a Madrona 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 therapeutics for monogenic diseases, other types of vaccines and other types of diseases as well. So we are definitely moving away from cell therapy, where we remain bullish about CAP-1002 at 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.

[Michael Okunewitch:]

Thank you. 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 or external collaborative programs?

[Linda Marbán:]

Yes. So our plan is similar to what you'll see sort of enrolling 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 diseases 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 will attract a large number of partners and also be able to develop this in conjunction with them.

[Michael Okunewitch:]

Awesome. And then one more, if you don't mind on the actual mRNA vaccine candidate. I'd just like to ask if an exosome based mRNA vaccine, you would expect it to have the same ultra-low temp cold chain requirements 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?

[Linda Marbán:]

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 two doses as is again with the current mRNA vaccines, but it's certainly possible that we could ultimately move towards a single dose.

[Michael Okunewitch:]

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

[Linda Marbán:]

Thanks, thanks a lot.

[Operator:]

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

[Emanuela Branchetti:]

Good afternoon, guys. This is Emanuela on for Joe. Thank you for taking my questions. So my first two 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?

[Linda Marbán:]

Yes. 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 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.

[Emanuela Branchetti:]

Got it. Thank you for that. 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 toolbar of an exosome based therapeutic? 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.

[Linda Marbán:]

So we 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 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 self, certainly, and probably similar to something that would be like an antibiotic, you have to go through all of the biologic hoops, but I don't think there'll be anything very specific. The exosome is 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, as 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 approaching, it could be another nucleic acid, and then you can develop potency assays as well as other types of metrics of mechanism of action and a very clear and concise way. So it should be a relatively straightforward regulatory pathway.

[Emanuela Branchetti:]

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

[Linda Marbán:]

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.

[Emanuela Branchetti:]

Thank you very much.

[Operator:]

The next question comes from Alan Leong of BioWatch News. Please proceed with your question.

[Alan Leong:]

Hi. Yes, thank you for taking the time to answer my questions and congratulations on the positive preclinical data. I was noticing that there's a quite amount of the additional data points that you guys are doing with the INSPIRE trial. And I was wondering if you plan to go for a label expansion on post-COVID patients?

[Linda Marbán:]

Yes. So we're looking at a lot of things in the INSPIRE trial. As you can imagine, 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 a trial with endpoints so that we can best plan the path forward. And we're not ruling out label expansion. We're not ruling out if the data were good enough, we wouldn't try and take it to FDA and see, for some type of 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 as 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.

[Alan Leong:]

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

[Linda Marbán:]

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 a 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 analysis to best figure out how the patients are impacted. I can tell you that we are extremely excited, but what we are finding and we're talking to the FDA about it currently. And so stay tuned for more information there.

[Alan Leong:]

Wonderful, yes, I really look forward to it. It needs to be realized. The last question I have 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 this 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?

[Linda Marbán:]

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?

[Stephen Gould:]

Sure. Yes. I mean, in the general area of RNA delivery, 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 dosable. And I think this is an area where exosomes really shine through in terms of potential. We've done many, many animals now. We never see adverse effects of exosomes 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 monitor in real-time gene expression from our platform. We can very quickly run through many, many variables in our design and 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 and 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 three areas where I see exosomes is having really high ceilings.

[Alan Leong:]

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

[Linda Marbán:]

Thank you very much. Thank you both. Are there any more questions?

[Operator:]

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

[Linda Marbán:]

Thank you. And thank you for joining us today. As you can see, in 2020, we wonder when 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 the 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. Good bye.

[Operator:]

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