Q2 2020 Capricor Therapeutics Inc Earnings Call
[music] greetings and welcome to the Capricorn Therapeutics second quarter, 20th 20th earnings call.
During the presentation, all participants will be in listen only mode.
Afterwards, we will conduct a question and answer session.
At that time, if you have a question. Please press the one followed by the four on your telephone at anytime during the presentation.
How's. It reminder, this conference is being recorded Thursday August 6th 2020.
I would not like to turn the conference overdue H, a Burger men Chief Financial Officer. Please go ahead.
Thank you and good afternoon, everyone before we start I would like to state there will be making certain forward looking statements starting today's presentation.
These statements May include statements regarding among other things the efficacy safety unintended utilization of our product candidates our future R&D plan, putting her anticipated conduct in time, you know preclinical in clinical studies or plants or is that a report additional data our plans regarding regulatory filings potential regulatory developments involving a product, Canada and or possibly uses of existing cash.
Yeah, that's my resources.
He's forward looking statements are based on current information assumptions and expectations that are subject to change in them. All the number of wrestling uncertainties. They may cause actual results did affirmatively from those contained in port looking statements.
Another Reds go describing a periodic filings name, but the FCC Queena quarterly an annual reports throw caution not the place undue rely on Sunday sport looking statement. Let me just claim any obligation to update such statements with that starts to call and turn it over to Linda My Bad C E O.
Good afternoon, and thank you for joining us for a second quarter update call I have never been more excited by Capric horse password well the world has been suffering with this horrible pandemic. The Capricorn team has been working harder than ever to advance our product candidates and our pipeline.
I will begin my remarks with update on our Cove it related programs.
Neither include our Cat 10, Oh, two sell products for the treatment of patients with Kobe 19, and a rapidly progressing bioengineered X as platform technology [noise].
[noise] to remind you kept N O. Two is broadly I mean, no modular Tori and are working hypothesis is that it is able to tamp down the cytokines storm that as a transitional event differentiating mild cove at 19 symptoms from does that lead to pneumonia and multi.
Oregon failure.
Our initial open label experience, where set with where seven patients with Coke at 19 were treated with Cat 10, Oh two.
Supported the notion that cat 10, Oh, two may modulate a hyper immune response and Cove at 19 patient.
Well, we were unable to definitively ascertain whether a cap 10, Oh two improved patient outcomes.
By analyzing blood samples and other test it was determined that the cat 10, Oh, two demonstrated identifiable improvements in certain patients such as a decrease in white blood cell count.
A decrease and I L. Six.
A decrease in C reactive protein.
[noise] and or a reduced reliance on supplemental oxygen.
We have concluded that the appropriate timing for treatment using cap 10, Oh, two isn't advanced patients who are showing early signs of cytokines store.
I have demonstrated by oxygen desaturation.
And size of a stomach and Clamatorial response in early Oregon damage.
We do not believe that patients who have only mild symptoms or those patients who are already in the most advanced stages of disease, and <unk> respirator dependent with and the state Oregon failure are the best candidates.
We believe that cashing patients who are heading downhill early and treating them with cat 10, Oh two represents the optimize timing to further study <unk>.
At 10, Oh two therapy.
Now open label studies produce headlines, but rarely provide definitive data to satisfy regulators.
And a certain clinicians.
Based on early experience, we were sufficiently competent to undertake a randomized placebo controlled trials.
We are committed to provide the highest standard of clinical data to the medical community.
A multicenter randomized clinical trial is now being prepared and subject. Your final regulatory approval will be our next clinical fade with cap 10, Oh two for coven 19th.
We're excited about the prospects of getting this trial executed.
Now today I'm going to provide the first updates on a rapidly advancing bioengineered accidental platform <unk>.
This too may have major a potential application in a coven 19 space.
But development and our Cove. It research are presenting opportunities for establishing a broad new platform for an array of other therapeutic opportunities down the road.
At present, however, we are laser focused on October 19th and I have some remarkable breakthroughs than I am able to highlight.
Now, let me tell me about the accident.
Saddam's, our natural nanoparticles at least by after all types that are capable of delivering potent signals and even molecule to desire types of action.
[noise] you previously heard me discuss C D C accidents.
[noise] active agent of our Cat 10, Oh, two cell therapy.
[noise] today.
The first time I will discuss our novel engineered non CDC extra salt.
What is remarkable about extra them is that soon if they are nature's vehicle for intracellular communication.
[noise] can modify or bioengineer of them to carry therapeutic molecules such as messenger Rnas.
[noise] inhibitory small ornate proteins or even trucks.
Furthermore, accident can be engineered to carry a specific markers, including receptor ligand or viral protein larcher antigens on their surface.
As such extra dorms are very well suited to be the basis of the next generation of vaccines.
Specifically they may be a more natural carrier of one or more messenger rna's or they may be bioengineered to form viruslike particles, otherwise known as B L. PS.
That mimic infectious theory odds suppose no risk of infection [noise].
Ah camera car there are two axes vaccine project underway, which I'm, making rapid progress.
Well, we are focused on October 19th this platform technology could be potentially adopted other vaccines as well.
We are very optimistic that one or more of the vaccines being developed under a project warped speed will be demonstrated to be useful for the human population.
That's sad first generation products, often leave room for improvement.
Each of the warp speed programs has its own pros and cons.
Hey discussion of each program is beyond the scope of this call, but I would like the highlight our program and some of the differences between copper court and others strategies.
We are investing in two unique vaccine products that have nowaday asked to animal studies.
[noise], both use <unk> derived from human self as he active terrier for the payload.
The excess almost messenger RMA vaccine can be differentiated from other messenger RNA based vaccines and too important ways.
First we formulate the messenger RNA vaccines, using the accident, which are safe non-toxic and a biological Oregon.
The <unk>, it's differentiates our approach from those that are using chemically derived lipid nanoparticles liposomes or other synthetic nanoparticle horrible.
Many of which had been associated with adverse reactions immune responses that limits repeat dosing and limited ability to interesting himself.
Now second we load the extra them with multiple messenger RNA molecules designed to elicit potent cellular and she moral antibody responses to all for structural protein I've Sars Kobe too.
Spike, which we've all heard about but also nucleocapsid N membrane M and envelope E.
This is critically important at all for proteins are targets of the immune response to Sars Kobe two infection.
And what's more N. A specific vaccines had been proven effective at preventing other corona virus infection.
Furthermore, the messenger rna's presenter spike receptor finding domain.
And protein and the soluble portions of the N N E protein informs that are optimized.
<unk> actually almost display.
And H C or major histocompatibility complex presentation.
With the goal of stimulating a broad based in long lasting cellular immunity to the virus.
In contrast, the other vaccines are unable to elicit immune responses to N M. R E N instead or only capable of eliciting an immune response to the spike protein.
Well, we agree that spike they critical components of any rational vaccine strategy.
And is incorporated in our approach as well.
We believe that a broader based approach that elicits immune responses to N N N E will have better outcomes.
I'm happy to say that studies in life have confirmed that the extra double messenger RNA vaccine induces strong immune responses to both spike in to the viral and protein.
Establishing the basic principle of extra they'll mediated mrna vaccinations.
This isn't important advance an accident bioengineering and nowadays to use of the accident platform for messenger RNA delivery in general and vaccination in particular.
Now the second vaccine candidates as a structural mimic about virus particle often referred to as a true viruslike particle D. L. P. As I stated earlier or viral extra salt.
This vaccine is generated by inducing human cells to produce accidents containing all for structural proteins.
The V L P producing cell line assemble C. S N N N E proteins into extra them that had the same basic protein and membrane composition as the actual virus.
However, the big difference between these V L PS and a virus particle is that the V. O P. S post no risk of infection, because they have no viral genomic RNA.
This means they are completely safe to manufacture safe to store and safe to administer.
To the body the immune system. However, this V. L. P vaccine looks just like the virus and is therefore, the closest one can come to a killed virus vaccine a classic vaccine that has been proven to be effective against animal Corona viruses.
We are hopeful that are ongoing preclinical research will continue to your old exciting result.
And it is our goal to bring these products rapidly forward in their development.
Furthermore, the work, we are doing and bio engineered extra them.
[noise] Stablish thing a plot for them not only for vaccines, but for the development of other potential therapeutic candidates.
However, leave this discussion to another time, given our present intense focus on Kobe 19.
I'll have further updates on this program very soon.
Finally, I would like to provide you with an update on our <unk> 10, 024, Duchenne muscular dystrophy program.
As we have previously presented we had very positive data and our hope too clinical trial.
And while it was a small trial.
Positive results suggest a high likelihood the captain Oh to improve improve both skeletal and cardiac muscle function and duchenne muscular dystrophy.
While the F. D. A has previously stated that it phase three clinical trial is necessary. We have continued to present the case for an accelerated approval.
Our position is based on the strong dataset with respect to both the performance of the upper Lim and improve cardiac function relative to the placebo controlled group.
As well as the excellent safety record of Captain O two.
And the present coven environment. It is unlikely that we would be able to undertake a phase through trial, but would seek some form of partnership should be not prevail in our discussions with the F. D I N.
We believe that for the older boys treated and hope to all of whom are steroid dependent and mostly non Angela captain Oh, two would present, an excellent option to modify intractable downhill clinical trajectory.
[noise] to increase dependent on caretaker and ultimately to death.
We can only hope that the FTA working with us it helps us find an experience path forward.
We are appreciative that FTA had been willing to hear our argument.
Now currently our main clinical focus is R Cove at 19 therapeutic and on the development of vaccine programs with the further development of the extra they'll plot for them to follow.
That's a J will update you want a moment, we are well capitalised to continue to deliver on or near term milestones in our building a rural cloth scientific product development team, but it's focus on the development of cells and engineered <unk> based therapeutic.
[noise] now I'd like to thank you for your time and attention today as you can see we have many milestones coming up I look forward to keeping updated on these very important programs.
I would like to know I'll turn the call over to a J Berkman are CFO Ajay.
Thanks went up so afternoons press release provided a summary, or second quarter ending June 30th 2020 financial Gonna got basic.
You May also referred or quarterly report on form tend to you, which we expect to become available very shortly will be available on the F. C C website.
As well as the financial sector Tomorrow, one SEC.
June 30th 2020th of companies cash cash equivalents marketable securities totaled approximately $36.3 million compared to approximately 9.9 million December 31st 2019.
During the first type of 2020, Capricorn raise approximately 29.4 million enough proceeds from the jail common stock and exercise a common warm.
<unk> comprised of approximately $19.5 million in that proceed.
And an average price for approximately 659 under our a T. M program that approximately 9.9 million. Additionally from the exercise a common one.
No turning quickly to the financial the first off of 2020 or gnatcatcher Houston operating activities was approximately $3.2 million for the second quarter of 2020 schooling stock-based compensation or research and development expense was approximately $1.8 million compared to approximately 1.6 million and cute too 2019.
Gunn, excluding stock-based comp our general an administrative expenses would you, possibly 1 million and cute too 20th 20th compared to approximately 800000 and cute to 2019.
Net lots of the first off the 20th 20th approximately five 6 million compared to a nut lots of possibly for 6 million for the first half of 2019 in summary, as we move forward. We continue to focus on the advancement of our core pipeline products Auckland articulated.
We will now open up the line for questions.
Q.
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One moment please for the first question.
And our first question is from the line, Jason Mccarthy with Maxim Group. Please go ahead.
Hi, Linda this is actually noreen on vacation. This afternoon. Congrats on all the progress I guess, we'll start with a more specific question and then that's my other brought her one you know you mentioned that seven patients were treated with cat Oh, two that had responses. It seemed with you know when you did.
Blood sample analyses, how many patients did you see these responses with.
We saw Ah changes in blood levels and every patient that we treated they all Ah showed reduction in white blood cell count ferritin and some of the other markers of information that I talked about a little bit earlier.
Okay, Great and then in terms of the.
Extra some program can you kind of help US understand you know when you read the literature you know they seem to be two sides of it you know there's some that believe it's a snapshot in time, which is really what extra phones are and the belief that it's not as effective and then there's the opposite you know that.
They do believe that it is so why would your approach be ideal for the indications that you have planned if he can remind us.
Yeah, I think snoring. So thank you forgive me the opportunity to talk for a moment that there's.
Extra zones that are being used clinically as a diagnostic tool and I think that's what you're referring to you take a sample the patient's blood and you can kind of see what's going on in their body because remember the most important thing about innoxious almost that they were in intercellular communicator. They are the words themselves. So of course was she a snapshot of what's going on within the human body, we have chose.
And for years to stay away from the diagnostic I used to that Saddam's, we're focusing both on the therapeutic you said extra jobs and then of course now on the development of vaccine. So we're taking advantage of the fact that the <unk> speak the language of the south and our nontoxic to the human body, but we're not sort of being passive bystanders and looking at what.
They maybe telling the story up.
Great and in terms of you know playing extra sums on the vaccine and I was just curious you know there's a reason jama article that discusses viral load and symptomatic children and adults and it sounds like children that are H five to 17 had similar viral loads two adults, but those at a younger had even higher one L.
And you know obviously, there's a risk that these children to transmit the virus. So when contemplating a strategy with your ex some vaccine ultimately are you considering uhm one didn't say teeth population given that unlike other many other companies you do actually have the experience in that population with your D. N. D studies would you imagine that to be.
Safe enough to apply to this population as well.
Yeah. So just to remind you we have two extra them based formulations for a vaccine one is the mrna loaded extra them. So we're taking a a human extra them and we're putting the four viral protein inside and the messenger RNA, so that will likely lead to us you.
Moral as well as a <unk>.
[noise] cellular immunity and the other approaches to make a virus like particle which is a standard method a vaccine production that's been around for a long time, but we've never been able to as a field take full advantage of it because we haven't had the appropriate delivery vehicles. So the extra them is the perfect viruslike particle.
And.
The the answer to your question is both of these types of vaccines could be wonderful for children their non-toxic, they're unlikely to be immunogenetics, we're not gonna be a clothing or for instance to a virus that they couldn't see in the future they're great per repeat dosing and you asked me to have a lot of experience is accompanying with a pediatric population.
So we're very comfortable what's your idea of a building towards children are current plan is to start the clinical work in adults, we'd like to make sure that you know we have treated dots population first before we go to the to the very.
Fragile child population a pediatric population.
Right of course, thank you that's it for me.
Our next question is from the line of island.
I don't watch news. Please go ahead.
[noise], Hi, Linda Hi, Jay Congratulations on the corner.
And when does you've mentioned help Tucson immunity is important not to 90 budrow sponsored dealing with coronavirus the popular Emilio to focus on anybody.
<unk> cellular immunity is important and then on top of about.
[noise] anything about the the T shirt immunity and the animal models are still on duck.
Yeah. So of course, we're looking at it keeps telling me in an animal model and what we're going to be <unk> name is presenting a full picture of the responses to the to vaccine candidate very soon so please stay tuned for that we're really really excited.
And she told immunity is probably the unheralded hero of the immune response right because it's where you harbor the opportunity to fight off future infection. So you have antibiotics antibodies come up antibodies come down and you have a memory response, but in T cells, but you really have the opportunity to stay at a these guys who are not coming in here again and uhm. So we are.
[noise] focusing hard on both the mediation of the cellular immunity as you said T. South and then also they she moral which is the antibody response and place they tune Island too you know the publication of of the state is it becomes available.
You have an <unk> experienced been going on in the with the.
Recent ashamed trial, how how long were would be Ah extension revokes occur.
If you're talking about the hope to open label expansion trial, which is the access to cop 10, Oh two for all the patients that we're in the hope to trial that is currently ongoing I will provide updated that becomes available I think I'd everybody knows you know getting kids into hospitals right now to do something like it's.
Nickel trial is risk that where I'm very aware of and in fact part of our conversations with FTAA regarding a request for accelerated approval.
And the fact that doing clinical trials and bring people at the hospital multiple times a year or just the test you know, they're they're limb stripes may not be the best way to to manage that population, let's keep him home and safe.
HM.
And then they're excited about your.
Phone platform, but you're building out and <unk>, you're right a number you're talking about the C. D. C to arrive extra films are are you're building a new platform off a variety of different sources are you pretty much.
I hate to use the term because it's not cool.
Pretty much building their own.
So we are being opportunistic and the selection of the salt hyper building. The exercise we became so excited a few years ago over the concept of building a bioengineered episome and I I've had to my colleagues and almost anybody that will listen to me actually sounds are going to do to biotechnology, what antibiotic therapy once did antibodies <unk>.
[noise] it off what twenty-five or 30 years ago and now the 12th most drunk revenue generating pharmaceuticals or antibody base and we think that extra dorms are are going to be that category of therapeutic opportunity. Because you can take them from different sell sources and you can tell them where to go and how to deliver the contents under markers on the outside.
[noise] allow them to bond to us and deliver contents to the nucleus. So you know Bob Langer Sad about 10 years ago, you know the problem with all of these Jean therapies is delivery delivery delivery well Wow.
Dr. Lager, we have a delivery vehicle for you.
Thank you very much.
And as a reminder to register a question. Please press the one four by by the four on your telephone.
And I will tell them to call back to Capricorn management at this time.
[noise] [noise]. Thank you very much for your questions. We look forward to providing you updates in the coming month since we build on our recent progress we expect 20th Tony to continue to be a productive in transformational year for copper Corp. We're committed to becoming a leading company in the development of Siler, an extra someone based therapeutics for diseases and as.
You know we were always focused on achieving our next milestones I Wanna. Thank are clinical investigators our scientific Advisory Board Dr. Steven goal to I've made our bioengineered program success to this point and I'm moving forward and my colleagues a capricorn [noise] for their steadfast dedication an outstanding efforts.
[noise] that does conclude the conference call for today.
Thank you for your participation and ask that you. Please disconnect your lines.
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