The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Edward Tenthoff - Piper Sandler & Co. - Analyst
: So let's get right into it. Deramiocel composed of cardios deive cells or CDC S. Linda, I know you've been working a, a big chunk of your career on
these cells. What exactly are CDC S and how do they work?
Question: Edward Tenthoff - Piper Sandler & Co. - Analyst
: And we know quite a bit about D MD, but tell us about the cardiomyopathy that these boys experience and sadly, ultimately die from.
Question: Edward Tenthoff - Piper Sandler & Co. - Analyst
: So, walk us through the phase to hope to data that you've reported, maybe start with the trial design and, and tell us about the data that you've
reported.
Question: Edward Tenthoff - Piper Sandler & Co. - Analyst
: That's great. And tell us about the ole follow up and sort of the durability. So hope to a lot of the boys rolled over into the open label extension
study. Tell us about that data that you reported.
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DECEMBER 03, 2024 / 4:30PM, CAPR.OQ - Capricor Therapeutics Inc at Piper Sandler Healthcare Conference
Question: Edward Tenthoff - Piper Sandler & Co. - Analyst
: And these data were maintained out to three years if I'm not mistaken. So that's really durable. So what change have the FDA for them to sort of
reconsider how they're reviewing them myself? And what are the current regulatory plans?
Question: Edward Tenthoff - Piper Sandler & Co. - Analyst
: Excellent. And when do you think you could complete the filing and you know, how long would the review be? And I should mention, I think you
would be awarded a priority review voucher on approval, which could be very lucrative too. Right.
Question: Edward Tenthoff - Piper Sandler & Co. - Analyst
: Excellent. Now you are also conducting the phase three hope three trial which is a little bit less important now, because of this breakthrough with
the FDA in terms of reviewing for cardiomyopathy. But nonetheless, tell us about the trial design and what role could this play for DS L approval if
it's even needed?
Question: Edward Tenthoff - Piper Sandler & Co. - Analyst
: And you mentioned the manufacturing, I think I'm going to come visit you next week while I'm in San Diego and get to see the facility myself. But
maybe you can explain these potency assays and what is your plan to meet the significant demand for us launch.
Question: Edward Tenthoff - Piper Sandler & Co. - Analyst
: And Del has partnered with Nan Shou globally remind us of the economics and what does Nan Shinyaku bring to the commercial launch?
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DECEMBER 03, 2024 / 4:30PM, CAPR.OQ - Capricor Therapeutics Inc at Piper Sandler Healthcare Conference
Question: Edward Tenthoff - Piper Sandler & Co. - Analyst
: Excellent. So I'm just going to pause there and see if there's any questions about the cell or D&D or, and we mentioned earlier that one of the
mechanisms for the primary mechanism for the CDC S is through exosomes.
And you guys are working on developing engineered exosome therapies for I think in a COVID vaccine D MD. But there's really opportunity even
beyond it, tell us a little bit what about some of these efforts.
Question: Edward Tenthoff - Piper Sandler & Co. - Analyst
: Yes, excited to hear more about where you're going to intend to take that and maybe there's partnering opportunities around that too. Now,
following the recent offering and including the 20 million upfront from the for Europe, we estimate you guys have pro performer cash around 186
million, something like that. How long does this fund the company and what's it enable you to accomplish?
Question: Edward Tenthoff - Piper Sandler & Co. - Analyst
: Great, very exciting, Linda. Thank you so much. Good luck with.
Thank you. Looking forward to a lot of good news.
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