Earnings Labs

Capricor Therapeutics, Inc. (CAPR)

Q2 2018 Earnings Call· Tue, Nov 13, 2018

$34.07

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Transcript

Operator

Operator

Welcome to Capricor Therapeutics' Second Quarter 2018 Conference Call. My name is Catherine and I will be your operator for today's call. [Operator Instructions]. I will now like to turn the conference call over to Capricor's CFO, AJ Bergmann.

AJ Bergmann

Analyst

Thank you. Good afternoon everyone. Before we start, I would like to state that we will be making certain forward-looking statements during today's presentation. These statements may include statements regarding, among other things, the efficacy, safety and intended utilization of our product candidates, our future research and development plans including our anticipated conduct and timing of preclinical and clinical studies, our plans to present or report additional data, our plans regarding regulatory filings, potential regulatory developments involving our product candidates and our possible uses of existing cash and investment resources. These forward-looking statements are based on current information, assumptions and expectations that are subject to change and involve a number of risks and uncertainties that may cause actual results to differ materially from those contained in the forward-looking statements. These and other risks are described in our periodic filings made with the Securities and Exchange Commission, including our quarterly and annual reports. You are cautioned not to place undue reliance on these forward-looking statements and we disclaim any obligation to update such statements. With that, I would like to turn the call over to Linda Marbán, CEO. Linda Marbán: Thank you, AJ. Good afternoon and thank you for joining us for our second quarter update and conference call. These are busy days at Capricor as we continue to strive forward in our DMD as well as in the development of our exosome [ph] platform technology. We are making good progress in both so I'm delighted to provide you with some color on both programs today. As you may know we are actively enrolling HOPE-2 our potential registration and clinical trial in Duchenne muscular dystrophy, in that trial we will be dosing up to 84 later stage DMD patients with either CAP-1002 or placebo every three months with the primary efficacy…

AJ Bergmann

Analyst

Thank you, Linda. This afternoon's press release provided a summary of our second quarter 2018 financials on a GAAP basis. You may also refer to our quarterly report on Form 10-Q, which we expect to become available in the next few days and will be accessible on the SEC website, as well as in the financial section of the company website. As of June 30, 2018, the company's cash, cash equivalents and marketable securities totaled approximately $12.3 million, compared to approximately $14.1 million on December 31, 2017. Based on our current plans and projections, Capricor expects that this cash, cash equivalents and marketable securities will fund its research and development programs and other operations into the second quarter of 2019. In the second quarter of 2018, our net cash used in operating activities was approximately $3.7 million. Excluding stock-based compensation, our research and development expense was approximately $3.3 million in Q2 2018, compared to approximately $3 million in Q2 2017. Again, excluding stock-based compensation our general and administrative expense was approximately $900,000 in both Q2 2018 and Q2 2017 And with that I will now turn the call back over to Linda. Linda Marbán: Thank you, AJ. Thank you for joining us today and we will now open the line for questions.

Operator

Operator

[Operator Instructions]. Our first question comes from Jason McCarthy with Maxim Group. Your line is now open.

Jason McCarthy

Analyst

Couple of questions, first Linda could you discuss a little bit about the differences in patient populations and DMD? Capricor is targeting older patients I think word is gene therapy has not been able to target so far. Gene therapy seems to be more on the earlier age patients but the data in that space has been really positive but I think limited in terms of time and their abilities well safety as you know. Can you discuss a little bit where a cell therapy in addition to treating older patients and keeping them on their feet longer and may play a role in earlier age patients even potentially complementary with gene therapy or exon skipping approaches? Linda Marbán: So very good question. So CAP-1002 is uniquely positioned to be either adjunctive therapy used alongside gene therapy and the early stage or hopefully as we understand the gene therapy better in older patients or as a standalone and patients that either may not be able to get gene therapy or where gene therapy hasn't been effective for that. So basically what we've done and we were able to show this in HOPE-1 was that in these later stage patients that have an established cardiomyopathy and also a significant skeletal muscle deficit in that 80% of the patients in HOPE-2 were non-ambulant. We were able to show improvement in the performance of the upper limbs specifically the mid-level which our thought leaders tell us is the most important level because of the way in which it changes and over time but also because of the strength that it can potentially confer on the boys and young men and maintaining their independence. So we are developing CAP-1002 to use in DMD because of its powerful immunomodulatory activities, ability to help the immune system, make good decisions in terms of not breaking down the muscular dystrophy [ph], encouraging new muscle to be made and we know this because we've been able to see preclinical that new muscle cells are made and then also hopefully that will translate into the dated HOPE-2 where patients will have improvement in the performance upper limb. The older patients represent about half of additional Duchenne community at any point in time so we're looking at about a prevalence of about 10,000 U.S. that are at this point qualifying for HOPE-2 by not being either ambulant or later stage of ambulation.

Jason McCarthy

Analyst

And just in terms of HOPE-2 you know you will measure obviously the primary [indiscernible] but you've discussed and Craig has in the webinar the immunomodulatory effect, anti-fibrotic effect and changes in bio-energetics as well. Are those exploratory or even secondary endpoints that would be evaluated in HOPE-2? Linda Marbán: Yes, so Jason you know HOPE-1 gave us a really terrific opportunity where we saw a performance of the upper limb improvements in patients which gave us an ideal efficacy outcome measure that can lead to full approval so that's why we were able to power HOPE-2 as based on the performance of the upper limb mid-level and we are targeting it to be a registration trial which we will determine as we in the forward with the FDA of course. The other side benefit of CAP-1002 are absolutely not to be minimized. So the most important thing is we develop this therapy to be used to treat the cardiomyopathy associated with Duchenne which actually no other therapies have really shown evidence of improvement in any realistic way in these patients so we are absolutely laser focusing as a secondary endpoint on cardiac function and structure. We also have some pulmonary measures built-in because we saw pre-clinically improvements in diaphragmatic functions and that might have translated to exercise capacity and of course we are looking indirectly at energetics by using some of these skeletal muscle improvers. So to that end we are absolutely focusing not only on the primary efficacy endpoint which hopefully would lead us to quick registration and commercialization but also to the secondary endpoint that are as important to the function advocate.

Operator

Operator

Our next question comes from Bill Koski with BioWatch News. Your line is now open.

Bill Koski

Analyst · BioWatch News. Your line is now open.

Couple of questions for you, the first is I was hoping if you could shed some color on the collaboration with army, what obligation is Capricor going carry forward and what are you looking at in terms of licensing and royalties, we need to yield any future licensing and royalty payments? Linda Marbán: Right now the relationship is truly on a research basis and so those types of specifics will be determined later on as the program becomes more developed.

Bill Koski

Analyst · BioWatch News. Your line is now open.

Okay. Thank you. It also looks like in some of the previous presentations you started using lot of [ph] exosome products at room temperature, is this your preferred product that you're hoping you can use in future trials and if you can speak kind of in broad terms about how the cost of goods compares for exosome product versus the cellular product? Linda Marbán: We are exploring all kinds of formulation opportunities with the exosomes now and that's part of this relationship with the army. Ideally we will want something that a med can [ph] carry in his or her pack that will be easy to use and quick to deploy. So we'll look forward to providing you with more information as formulation becomes available. I don't think the exosomes are going to be one color in terms of what they're used for or how they're used, it will be indication specific and again that sort of will unfold over time. In terms of cost of goods we're anticipating that the cost of goods or the exosomes are going to be significantly less than for the cells. We haven't sharpened our pencils and done those numbers yet but as we do we'll be able to provide more clarification again it will be probably indication specific.

Operator

Operator

[Operator Instructions]. And I'm showing no further questions at this time. I'd like to turn the call back to Ms. Linda Marbán for any closing remarks. Linda Marbán: Thank you. We look forward to seeing you at various scientific and investor conferences through the course of the remainder of the year and with that we can disconnect the call. Thank you for your time.

Operator

Operator

Ladies and gentlemen thank you for participating in today's conference. This concludes today's program. You may all disconnect. Everyone have a great day.