Leland Gershell - CFO Linda Marban - CEO Anthony Bergmann - VP, Finance.

Joe Pantginis - Rodman Ted Tenthoff - Piper Jaffray.

Good day ladies and gentlemen and welcome to Capricor Therapeutics Fourth Quarter and Full Year 2016 Financial Results and Business Highlights Conference Call. At this time, all participants are in a listen-only-mode. Later, we will conduct a question-and-answer session and instructions will follow at that time.

As a reminder, today's conference call is being recorded. I'd now like to turn the conference over to your host Dr. Leland Gershell, Capricor's Chief Financial Officer. Sir, the floor is yours..

Thank you, Cathy [ph]. Good afternoon and thank you all for joining us in today's call. Our press release providing details of Capricor's financial results for the quarter and year ended December 31, 2016 as well as an update on our business was issued a while ago. This press release is available on our website at capricor.com.

Joining me on the call today are Dr. Linda Marban, Capricor's President and Chief Executive Officer; and Anthony Bergmann, our Vice President of Finance. As a reminder, during today's call we will be making certain forward-looking statements.

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 contact and timing of pre-clinical and clinical studies, our plans to present or report additional data, our plans to regarding regulatory filings and our possibly usage 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 are other risks are described in our periodic filings made with Securities and Exchange Commission including our quarterly and annual report. You are cautioned not to place undue reliance on these forward-looking statements and we disclaim any obligations to update such statements. With that, I'd now like to turn the call over to Dr.

Linda Marban, Capricor's President and Chief Executive Officer..

Thank you, Leland, and thanks all of you for joining us. On today's call, I will share some updates on our drug development programs as well as on some other areas of our business and then Leland will summarize the financials that we reported today.

As we had announced on our last quarterly update in November, we were expanding the scope of our development program in the Duchenne muscular dystrophy for the conduct of a planned clinical trial and also aim to evaluate CAP-1002 with potential ability to improve respiratory and skeletal muscle pathology.

We've recently gathered key opinion leaders in Duchenne to collectively discuss approaches to the design of this clinical trial, which we expect to initiate in the second half of this year subject to regulatory approvals.

We've reached beyond our core group of highly respectively experts who are focused on the heart disease as DMD and assembled a larger panel of thought leader both from the U.S. and abroad to share their insights.

The meeting was tremendously informative for our team and start continuing dialog and collaboration, which we expect will be very useful to us as we design and execute our clinical development plans to Duchenne muscular dystrophy.

An important feature of the next stage in this program is that we planned to administer CAP-1002 by systemic infusion in contract with the intracoronary delivery method that we have used in the past. We look forward to sharing more details on this evolving program as we move through the year.

In the meantime, preclinical data showing positive effects of cardiosphere-derived cells or again CAP-1002, on non-cardiac striated muscle in DMD mouse model continues to accumulate both from around work and data parts collaborator.

As a result, we are also gaining greater clarity on the mechanism of actions of [Indiscernible] and their importance on its solitary effect. We expect some of these data to be published by our academic collaborators and still reviewed scientific journal article on the next coming months.

So now with respect to our ongoing Phase I/II HOPE-Duchenne trial in boys and young men with DMD associated cardiomyopathy, we look forward to reporting initial six month results this April. As a remainder, the HOPE trial randomized 13 patients to receive CAP-1002 by intracoronary infusion and 12 patients to receive usual care with no infusion.

Just to remind, these 70% of the patients were enrolled to the HOPE trial are non-ambulant, our group for room therapeutic options as well as clinical trial opportunities are few and far between. We will follow all trial participants for at least 12 months.

Those who do not receive the study medications maybe eligible to receive open label CAP-1002 after all participants have completed the control portion of the study and the data safety monitoring work has given its recommendation to proceed with the open label expansion.

In addition to the primary safety end point, we are exploring potential efficacy in HOPE by a variety of analysis including cardiac magnetic resonance imaging and performance on several functional measures including the performance of the Upper Limb test, the six minute walk test for those who are able to perform it and test of respiratory function.

We will also evaluate patients on quality of life measures.

Given the broadening scope of our therapeutic development program for DMD and our growing involvement with the Duchenne community, in the coming months, we look forward to hosting an R&D event in New York at which we and several of our key opinion leaders will discuss Capricor's unique approach to therapeutic development for Duchenne muscular dystrophy.

This event will be webcast for the benefit of those who will be unable to attend in person. Let's quickly touch on our development of CAP-1002 in adult heart disease. Patients follows up continues on our randomized, double-blind, placebo controlled Phase II ALLSTAR clinical trial.

ALLSTAR is powered to detect a statistical difference between the active and controlled arms with respect to the potential ability of CAP-1002 to reduce myocardial scar size at 12 months. The trial has been conducted at 30 centers in the U.S.

and in Canada and all of the cardiac MRIs will be read by a single core emerging lab at Johns Hopkins in Baltimore. This facility which is the same one that for the interpretation of the MRI from the positive [Indiscernible] produce study on which the design of ALLSTAR base is headed by Dr.

Jalal [Indiscernible] an internationally recognized leader in the use of MRI to evaluate the heart. Next quarter, we will deliver Janssen Biotech the results of this six month administrative analysis of the ALLSTAR data.

Let me remind you the Janssen will have 60 days following the delivery of the six months data to exercise its options to in license CAP-1002 for certain cardiovascular indications.

As many of you know, we have been developing our manufacturing process for CAP-1002 in close collaboration with Janssen for approximately three years and we are delighted to have made so much progress in this area.

Our exosome program continues to generate a great joy of enthusiasm among us internally and perhaps more importantly among some of the key opinion leaders we have engaged. This is driven by a growing body of preclinical data that supports the activity and potency of cardiosphere-derived cell exosome or CAP-2003 in model of inflammatory disease.

We planned to submit an investigational new drug application for IND that is FDA for CAP-2003 in the second half of 2017 to enable the clinical development of CAP-2003 in an inflammatory disease indication.

Our development efforts of this exosome candidate remains centered on graft versus host disease otherwise known as OGVHD, a relatively common complication of bone marrow transplantation that is totally addressed by current treatment options.

As we continue to gather preclinical data on our exosome, we are evaluating several indications including ocular as well systemic OGVHD for an initial IND. As we continue to build Capricor with the objective of becoming a commercial state biotechnology company, I'd like to mention a few personnel appointments that we've made in recent months.

In the fall, we appointed Karen Edward to oversee our GMP operations at Capricor. Karen has over 20 years of involvements with biotech product developments and manufacturing including quality and regulatory compliance management. Karen come to us from Mesoblast whereas Vice President of Quality Assurance.

She is reporting commercial readiness efforts by establishing manufacturing capabilities as well as creating and implementing quality control and supply chain management systems.

Karen has become a key asset in the context of our -- those interactions of the CMC at Janssen and are in evaluation of potential contract manufacturing organizations, as we elevate our manufacturing process to commercial scale. And earlier this year, we welcome Dr. Maris McIntyre to our team.

Maris also brings to Capricor nearly 20 years of strategic regulatory and product development experience. Her multi-disciplinary work has involved the variety of therapeutic programs within the industry including several which presented complex regulatory challenges.

Prior to her role with corporate sponsors, Maris served to the Center for Biological Evaluation and Research otherwise known as CBER at the FDA.

Maris' most recent position was as Executive Vice President of Regulatory Affairs and Product Developments at [Indiscernible] Therapeutics, which had been developing drug candidates for orphan neurological and neuromuscular disorders including Duchenne muscular dystrophy until its acquisition by Pfizer last year.

We believe Maris will be a great value to Capricor as we work with the FDA as well as other regulatory agencies to advance our product candidates for a hopeful commercialization. At this time, I would like to now turn the call over to Leland, so he can provide a brief review of our financials.

Leldand?.

Thank you, Linda. Please refer to the today's press release for a presentation of our fourth quarter and full year 2016 financials. We may also refer to our annual report on Form 10-K for 2016, which we expect to become available in the next few days and can be accessed in the financial section of our website at capricor.com.

During the quarter excluding the effect of stock-based compensation, we spent approximately $2.5 million in research and development expenses and approximately $900,000 in general and administrative expenses.

For the full year 2016 again excluding stock-based compensation, we spent approximately $15.5 million in R&D and approximately $3.5 million in G&A. We ended 2016 with cash, cash equivalents and marketable securities totaling approximately $16.3 million, as compared to approximately $13.6 million at December 31, 2015.

We expect these resources to fund our operations into the fourth quarter of this year. And with that, I’ll now turn the call back over to Linda for closing comments..

Thank you, Leland. So, the first few months of the year have been particularly busy one for all of us at Capricor as we prepare to enter into a very dynamic period for the Company.

With regards to our product development programs next month, we will report top line six months data from HOPE and in the fourth quarter we expect to report 12 months data from HOPE as well as from ALLSTAR.

We remain on track to submit an IND for CAP-2003 in the latter half of this year and on a strategic front, we expect to learn Janssen decision to its license options by the third quarter. And finally, we look forward to holding an R&D Day focus on our Duchenne program in New York City in the late spring or early summer.

Okay, I’ll now turn the call over to the operator to open up the call for questions. Thank you for your time today. It's been a pleasure..

Thank you. The floor is now open for questions. [Operator Instruction] And our first question comes from Joe Pantginis. Joe, go ahead..

Hi good afternoon, it's Joe Pantginis from Rodman. Hi Linda and Leland, couple of questions if you don't mind. First obviously, you have a major catalyst coming up with the HOPE Duchenne study next month and I guess I want to relate it to your current agreement with the Janssen.

You talked about certain cardiovascular indications when you initially signed -- I am sorry signed the partnership, the HOPE or the Duchenne program really wasn’t getting, wasn’t ongoing yet.

So since cardiomyopathy is such a problem with a lot of Duchenne patient, I was curious to see if the DMD program is part of the potential offices of the collaboration with Janssen? And then I have a follow-up. Thanks..

At this point, we can say that we are in active negotiations with Janssen and because of that we are not going to be discussing that topic at all at this point..

No that’s fair, thanks I understand, it's certainly an ongoing process. The second question, I wanted to follow up which is on the exosome program.

So based on pending IND and the potential broad applicability of CAP-2003 as well as the other exosome you might be developing, you had originally said you are going to be looking at the ocular GVHD program, but you are potentially looking now as I heard it correctly to look at potential systemic GVHD as well.

So, I wanted to see how you are looking to potentially adjust the program here from the original plans?.

When we started our clinical development program for exosome, we were really looking for a compartment that we can put the exosomes and where we felt they could have direct sale to sell communication ability to know that's how they worked biologically.

And we also have developed a manufacturing paradigm to develop large numbers of the exosomes needed phonetically for systemic delivery.

So, we were very interested in graft versus host disease and we thought that we can focus on the ocular portion of that for both of those reasons that we can put the exosomes into a compartment and we can make total numbers of them.

However, a lot of it happened to have great team that's working on this and keeping that it happened that are changed my direction somewhat at this point. One, as we've had really start when we positive data entering systemic graft versus host disease in a mouse model.

Just preliminary, we're going to follow up with some follow on studies, but really the kind of data that may twisted and case noticed and can't ignore it, and the changes were above and beyond making the eyes better.

And the other is that the manufacturing team has worked with the product development team and we now feel that we could be able to manufacture large enough doses to treat patients systemically.

So, while we still have [Indiscernible] graft, ocular graft versus host disease, we feel that it would be important to continue this exploration into the systemic manifestation to see if this will be the appropriate clinical development pathway for the exosomes..

And our next question comes from Ted Tenthoff. Ted, go ahead. .

I guess when it comes to the systemic delivery with DMD, how are we going to be working at endpoints beyond just MRI, you actually look at the and evaluate the skeletal muscle improvement?.

Yes, Ted, so the catalog meeting that we had in New York at the beginning of February was in my mind disruptive to anything that we ever had around the MD. I just had the most extraordinarily brilliant people sitting around the table, talking about the pathophysiology of Duchenne muscular dystrophy.

And it was the first time that we had addressed directly the idea of treating patients with the earlier stage manifestations of the skeletal muscle myopathy that was focused on the cardiomyopathy. So, what we really have that we have two unique opportunities with our product to treat the patients with Duchenne.

We have the opportunity to treat the cardiomyopathy. And we probably can do this systemically as well as through intracoronary methods.

And then we also have the opportunity to treat the skeletal muscle myopathy where we don't believe at this point that the endpoint we would look at would be MRI based, but perhaps partial base which still sort of trying to discern that.

What we've learned is that we need to study the skeletal muscle myopathy potentially in the younger patients, so that we can track changes overtime while they still have a fair bit of skeletal muscle performance ability. This is all being worked out now, clinical trials, our plans are being development.

And I promise that to keep you frontline informed as we move this program forward. .

And there appeared to be no questions at this time. .

Thank you very much for joining us today. And we look forward to talking to you as our results become available at our R&D event that's coming up and through the other milestones that we have planned for 2017. Have a nice afternoon..