Q1 2022 Capricor Therapeutics Inc Earnings Call
Good day, ladies and gentlemen, and welcome to the Capri called Therapeutics, Inc. First quarter 2022 financial earnings call.
As a reminder, today's call is being recorded at this time I would like to turn the call over to Mr. Backman Kupiec with Therapeutics Chief Financial Officer. Please go ahead Sir.
Thank you. Thank you for joining today's call.
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 are planned to present.
Additional data our plans regarding regulatory filings potentially potential regulatory developments involving our product candidates potential milestone payments 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 in the forward looking statements. 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 with that I'll turn the call over to Linda Moore Van CEO .
Good afternoon, and thank you for joining us for our first quarter 2022 conference call.
I will provide updates on our lead product cap 10 O two as well as our <unk> platform technology.
We have made significant progress this quarter highlighted first and foremost by securing a commercial partnership with Nippon <unk> for Duchenne muscular dystrophy program.
And $30 million in upfront cash to fund our phase III program.
Further we saw the publication of our phase III hope to resolve in the lancet one of the most prestigious medical journals in the world.
If you haven't read this publication I encourage you to please do so as it will strengthen your understanding of the positive results demonstrated in the phase II hope to trial of cap <unk>.
The paper speaks to both the scientifically.
And clinical rationale for our phase III hope three clinical trial that I'll discuss in greater detail in a few minutes.
We continue to prudently invest in the future of Capricorn.
<unk> of our near term value is the Caf two program for the treatment of older Boys with Duchenne muscular dystrophy.
We have a new pension yaacov and its U S subsidiary and as pharma and experience well resource commercial partner in the United States.
We remain active in seeking further partnerships for our key markets around the world.
Our goal is to partner with commercially talented companies.
Repair us for global expansion of the DMD program.
The second pillar of value is our bio engineered access our program. We will have more detailed updates on this program in the future.
<unk> to say that we are extremely pleased by the progress being made in this platform technology program.
We have assembled a world class team in research quality manufacturing and clinical operations. Yeah. We continue to maintain a lean organization in order to remain capital efficient.
We are fortunate to have a strong balance sheet in these very turbulent equity markets and are also fortunate to have no near term need to conduct an equity financing.
Our summary financials announced prior to this call report over $58 million in cash providing at least two years of runway to execute on our strategies.
Turning to our DMD phase III program.
I'd like to provide an update on the status of our hope three phase III clinical trial.
<unk> III is a randomized double blind placebo controlled clinical study designed to enroll approximately 70 patients in the United States.
We will be treating patients who are largely non ambulant and in the later stage of the disease process for whom very few therapeutic options success.
All of our patients are required to be on best medical therapy, which includes corticosteroids.
This patient population comprises over one half of the DMD market or about 10000 boys and young men per year in the USA.
We are actively onboarding sites for <unk> III and are now actively screening patients at our first site.
Our objected to bring on more than 20 sites in the next several months.
Based on the strength of the hope to data the publication in the lancet and anecdotal reports of the patients the enthusiasm for hope III run strong in the patient community.
As you know <unk> agreement with Nippon <unk> comes with potentially up to $705 million more in milestone payments some of which if achieved will be paid during the course of hope three.
We also upon FDA approval will qualify for a rare pediatric coupon voucher for which <unk> retains the rights.
In the past these coupons have in and of themselves become very valuable assets for these companies that have had their products approved.
If cap <unk> was approved for the older Boys, we believe that there will be a substantial opportunity to expand the indication to even younger patients with duchenne muscular dystrophy.
This population similarly has a material unmet needs.
Further the existing exon skipping drugs have well described limitations and the gene therapy space is moving much more slowly than many had hoped.
There is nothing inconsistent with the concept of combining cap 10 or two in any of the multiple therapeutic options currently available or in clinical development.
Lastly, I would like to reiterate that.
As additional data clinical data becomes available our goal is to continue to discuss our path to registration with the FDA.
Okay.
Finally, I would like to give you an update on our <unk> platform technology.
We continue to advance this platform on multiple fronts.
Let me remind you that extra dumped our nature's communication devices. They are capable of delivering cargo directly into the cytoplasm and we believe that they should have a safety and efficacy profile far better than a manufactured lipid nanoparticle.
Most important aspect of the <unk> is that they can be targeted.
Using extra particular factors too which are targeting molecule can be attached.
Our team is hard at work expanding the payload and targeting capabilities of our platform.
These include but are not limited to loading M. Rnas.
<unk> Rnas or small inhibitory RNA is.
Small molecule and proteins within or on the surface of the <unk>.
Along with developing high affinity targeting capability and moving towards a scalable and cost effective manufacturing paradigm.
The options for both vaccine platform and therapeutics based on our end bio engineered <unk> platform are extensive and I will be providing more color on the development of the <unk> platform at future meetings and in publications.
As always our partnering strategy is central to our business model with the objective of finding excellent commercial and development partners.
As well as additional non equity capital.
In conclusion stay tuned for further updates on our phase III DMD program and the continued advancement of our <unk> platform technology.
I want to reinforce that over the last year, we have delivered on our goals. These include a partnership for DMD. The publication of our phase two results in a highly respected medical journal.
Enhancement of our management team and manufacturing to build capabilities as well as the advancement of the <unk> platform technology.
We currently are focused externally on DMG and hope III, we also recognize the value and importance of the <unk> platform.
So with careful management of capital.
<unk> strong clinical data we approach the second quarter was tremendous energy and optimism.
Over the next few months, we plan to present at various banking scientific and advocacy conferences, including several high profile RNA and non viral delivery symposiums at Boston focus on our Exosomes program.
We'll also be providing an update on our DMD program at the annual parent project muscular dystrophy meeting in Scottsdale in June of this year.
I will now be turning the call over to AJ Bergmann, our CFO for more detailed update on the financials.
Hey.
Thank you Linda.
This afternoon's press release provided a summary of our first quarter of 2022 financials on a GAAP basis may also refer to our quarterly report on Form 10-Q, which we expect to become available shortly it will be accessible on the SEC website as well as the financial section of the company website.
As of March 31, 2022, the company's cash and cash equivalents totaled approximately $58 3 million compared to approximately $34 9 million on December 31 2021.
Turning quickly to the financials in the first quarter of 2022, our net cash provided by operating activities was approximately $24 million driven by the $30 million upfront payment from the bunch and yaacov.
For the first quarter of 2022, excluding stock based compensation, our research and development expense was approximately $4 9 million compared to approximately $3 2 million in Q1 2021.
And excluding stock based compensation, our general and administrative expense was approximately $1 9 million in Q1, 2022, and approximately $1 3 million in Q1 2021.
Net loss for the first quarter 2022 was approximately $7 8 million compared to a net loss of approximately $5 2 million for the first quarter of 2021 well.
We will now open the lineup for questions.
Thank you, ladies and gentlemen, if you would like to ask a question. Please signal by pressing star one on your telephone keypad if.
If youre using a speakerphone. Please make sure your mute function is turned off to allow youll see the to reach our equipment.
Press Star one to ask a question with take first question from Sean.
I'm, so sorry, Joe and genius with deficiency Wainwright. Your line is open. Please go ahead.
Hey, guys. Good afternoon, thanks for taking the question.
A few if you don't mind, so first I guess just going back to your prepared comments Linda.
<unk> three you talked about even potentially expanding to younger patients non advanced in the future. Just curious if this could be something that nippon might be interested in funding and are accelerating and how we could think about broadening the market there.
Yeah.
Yes, Joe so great to hear your voice. Thank you so much for joining us today.
We haven't discussed the younger patients specifically with <unk>, we are very busy launching <unk> and getting that program up and running.
Say this the relationship is very warm with them as well.
Very excited at all of the opportunities ahead of us.
For the non ambulant and the ambulant patients.
Got it got it so if we focus on hope three a little bit. Obviously, you said you are screening patients at the moment.
I'm going to make an assumption here. So let me know, if I'm sort of right or wrong or going in the right direction.
There might be.
A decent backlog of patients that are ready to be screened either.
Patients that were ready for hope to or just where that's gotten around from different advocacy groups or what have you. So it seems like this could enroll relatively quickly.
Yes that certainly is our thinking we certainly got a lot of feedback from families from the community from physicians that there's a lot of work sort of in the community that top $10. Two is delaying the progression of Duchenne muscular dystrophy. Both we hear this anecdotally and also of course.
The published data supports that so yes, we're expecting that the trial will enroll well.
Okay got it and then.
I know you sort of said, we'll get more detailed and exosomes in the future I don't know if you wanted to.
Tease us with any potential favorite indications, but maybe specific part of that question is.
Yes level of advancement, so far like do you have the relative models and hand, the requisite models in hand, I should say.
Yeah. So as you know I'm really excited about the <unk> platform.
<unk> has performed as.
We pretended that it would it's showing efficacy in the treatment of DMD and we believe that our <unk> will have.
As brighter or brighter future as drug delivery vehicles.
We've done has been laying the groundwork building the foundation of the home framing it in I was learning how to manufacture these things to scale getting the agreements in place to make large numbers of exosomes working on targeting technologies can we send an accident should deliver to the kidney to the muscle to the brain and then ultimately custom loading content.
On the inside or on the outside for therapeutic opportunities. What I can tell you is that we're very far ahead in the development of these sort of core technologies now and are identifying indications, which we're going to be coming forward with very soon very soon in the next couple of quarters.
Got you got you that's helpful. And then maybe just shifting gears a little bit maybe for a J.
If I read the press release correctly, you have got the $30 million. This was it in the first quarter or the second quarter that you received a $30 million and just curious about how youre looking to account for it.
Yeah. Thanks, Joe the money came right at the end of the first quarter. So it's booked on the balance sheet as you'll see with our cash position going up substantially.
Likely be right turning to the accounting concept of it we'll be ratably recognizing this income really over the duration of our clinical development program pretty standard recognition from an accounting concept, but the money is in the bank and it gives us a nice runway of well over two years at this point. So it's good to have.
Perfect I appreciate the color you too thanks a lot.
Thanks, Joe Thank you Joe.
Once again, ladies and gentlemen, please press star one to ask a question.
It appears there are no further question at this time I would like to turn the call back to Linda Marvin for any additional or closing remarks.
Thank you for joining us today, we look forward to seeing you at meetings as we are now finally able to be out and about in the community and stay healthy during this tumultuous time.
Good day.
Okay.
This concludes today's call. Thank you for your participation you may now disconnect.
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