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Automatic cruiser to $50
Billion dollars mktcap coming upon BLA news very soon
Institutional investors keep accumulating cheap shares now
Big profit in a few months
Pps won’t retrace until BLA news soon
So much undervalued now
Pps will keep going up until BLA news next month
Looks like institutional investors buying huge here
i believe they have hidden value they barely scratched the surface of. we shall se it relatively soon with patience
CAPR will be trading around $50 before Thanksgiving day
1000% runner in the making
So much undervalued at $120 mil mktcap now but on the way to multi billion company in a few months
We will be millionaires soon
I like this part of CC
This is the strong case for accelerated approval definitely
As I said in my talk and I've been saying now for a few weeks' time is muscle what became very clear in the open label extension data is when they were off CAP-1002, they decline and as soon as they go back on CAP-1002, they get better. The placebo patients were on the steady course of decline similar to natural history for two straight years, the year on placebo and a year on gap and then as soon as they went on CAP-1002, the delay -- we saw the delay of the decline of the disease process by about 70%.
Would love to articulate the boggest question. Your booster therapeutics was thought to encapsulate and rebuild the walls on cells which I was most captivated by. As it has rejuvination charachteristics that can be an enhancement to move than your products. And can enable other treatments to effectuate and rebuild the affected areas. Are you trying to utilize this part of research toward that end?
nice. still holding for the big win. it's volcano time.
Hi Linda and A.J. Good afternoon. Thanks for taking the question. So, a couple questions actually. So, first, wanted to get a little more color or detail. Now, obviously, the answers will be predicated on what the FDA says, but how would you describe your wish-list or goal for what you hope to get out of your upcoming FDA meeting, where you said the primary goal is to present the OLE data? Are you looking to change the potential launch dynamic, have it potentially come to market earlier? I mean, what is the ultimate goal here?
Linda Marbán
Yes, I mean, our ultimate goal, Joe -- and first of all, it's great talking to you. I'm looking forward to seeing you in September. Our ultimate goal is to get CAP-1002 to market and to these DMD markets as quickly as possible.
As I said in my talk and I've been saying now for a few weeks' time is muscle what became very clear in the open label extension data is when they were off CAP-1002, they decline and as soon as they go back on CAP-1002, they get better. The placebo patients were on the steady course of decline similar to natural history for two straight years, the year on placebo and a year on gap and then as soon as they went on CAP-1002, the delay -- we saw the delay of the decline of the disease process by about 70%.
We think this data is extraordinary. We think it is representative of disease modifying activity. We're going strong to the FDA to show this data and we are excited to work with them to bring this to approval as quickly as we possibly can.
Joe Pantginis
And the muscle data certainly speak for itself in my belief, so I guess, I'll ask it in another way, as I'm thinking about it. So, with regard to the commercialization dynamic, so is there a potential I mean, let's just call it swing for the fences at this point with regard to does CAP-1002 have the potential to be on the market prior to the readout of HOPE-3? And when I say on the market, I mean, something like maybe a managed access program on a patient-by-patient basis based on the data you'd look to present?
Linda Marbán
Yes, so our goals right now are very focused, the building blocks are in place to get CAP-1002 ready for commercialization. That means meeting with the FDA regarding the CMC, making sure that we are BLA-ready, presenting the open label extension data to the FDA, getting their feedback, and then we'll decide our path forward there -- what's the best path forward.
The exciting thing with our regulatory designation of RMAT, we really got preferred access to FDA. So, they're going to work with us on this. And certainly everybody that we've shown the data to, is excited by it. And anecdotally, the patients are saying they're definitely feeling and functioning better. And so they're going to stand up and say -- and sometimes even shout the same thing.
So, certainly, we've put the team in place. We've got the manager there. And if anybody knows, I know I'm a big baseball fan, so swinging for the fences is not something I'm afraid to do.
Joe Pantginis
There you go. Okay. And then, I guess, with regard to either concomitant timing of the meeting or a separate meeting with regard to CMC, I'm glad you're, you're planning ahead with regard to your manufacturing, and supplementing out of San Diego lab, I'm glad you made those comments. How would you potentially project your needs beyond that from a manufacturing standpoint?
Linda Marbán
In terms of the manufacturing, we're working on that plan right now. We have the facility, as I just announced that we're building as part of our San Diego labs here, it is a relatively low cost option, which affords us a tremendous opportunity for not only getting this product ready for BLA, but also for commercial launch. We'll evaluate along with our partner NS, the need as it becomes more apparent. And we'll keep everybody updated on our plans for that as we move forward.
Joe Pantginis
Got it. And then just lastly, because obviously, the future has a lot of exosomes being discussed. So, you mentioned a couple things here and in your press release too, that you're going to be looking for some preclinical data, maybe get a little more visibility about what kind of things we might see without necessarily tipping your hand. And you said you also have a potential partner in candidate in the form of vaccine candidate, is that something apart from your COVID candidate that you've discussed previously?
Linda Marbán
Yes, it's sort of a twist on the COVID candidate. So, I can't really talk too much about it right now because we're really working very hard with our IP and legal teams to make sure we're protected on every level.
What I can tell you about this is it's going to be very exciting because it takes the exosomes, which you know we've been working on for a long time, we've standardized the manufacturing of such, so that they can be scaled up and manufactured in a way that will make them competitive with any lipid delivery system out there.
But we can put a targeting molecule on the outside, and we can put custom payloads on the inside. The current vaccine candidate is similar to a recombinant protein vaccine, so -- but has the advantage of being able to be made into other types of vaccine candidates very quickly. So, we use COVID, sort of, as our model system, rapidly available and something that we've been working on for a while, but in reality, could be translated to any infectious disease or other types of utility, where a protein-based vaccine might be necessary or desired.
Joe Pantginis
Got it. And my last question, if you don't mind and it's actually kind of important, I think just based on the current biotech environment, which looks like it's starting to bounce back, but cash is king right now. So, being able to have a balance into the second quarter of 2024 speaks volumes. So, I wanted to address one of your comments in the press release and A.J.'s comments about excluding potential strategic uses of this capital, is this something that would look to expand your pipeline externally -- from an external source or further expand an exosome program, or I guess what kind of things could we look at if that would be one of the options you consider?
Anthony Bergmann
Yes, Joe, happy to take a little bit of that question. I mean, obviously, we're being judicious with our cash spend. I think we have a nice runway into the second quarter, because we just articulated of 2024.
And really what we're prioritizing what our current mission is focused on CAP-1002 for DMD, and the exposome pipeline right now in a preclinical status. So, it's obviously out there, and we're considering all options, but we're going to do our best to effectively manage our burn rate moving forward.
Turning now to our exosomes technology. The last year has been focused on developing exosomes as a versatile platform for drug delivery and also on identifying potential applications. We have made significant progress in the manufacturing of exosomes as a competitive alternative to other lipid delivery systems with the additional benefit of having the potential to be targeted to a specific biomarker or cell type.
Our targeted delivery platform can carry therapeutic payloads that are produced via an exogenous process for loading certain types of payloads, which is similar to what most in our space are doing, or via an endogenous loading process for other types of payloads, including proteins.
This last approach relies on our proprietary technology, which allows for better consistency and preservation of the integrity of the cargo. We believe this positions Capricor to be able to attract potential partnerships and drive new therapeutic modalities.
The emerging exosomes platform will have potential applications in multiple domains, including vaccines, delivery of RNAs, including small interfering RNAs, and antisense molecules, as well as other payloads.
We have used our proprietary technology to develop an exosome-based vaccine with robust preclinical data. We plan on positioning this opportunity for partnering discussion.
At present, our focus is on the potential commercialization of CAP-1002 for DMD, while we continue to develop our exosomes platform technology for future pipeline opportunities. By prioritizing our core programs, we have the ability to efficiently utilize our current cash position, which carries us into the second quarter of 2024 to deliver on important political and related milestones.
Now, let me walk you through some of the key highlights and recent updates. First, turning to HOPE-3, our ongoing Phase III pivotal study, which was initiated in the second quarter, which included site selection and activation of certified Duchenne centers. HOPE-3 is a randomized double-blind placebo-controlled study with the goal to enroll 70 patients at approximately 15 to 20 investigative sites in the United States.
In July, we reported the initiation of enrollments and I am very pleased to inform you that as of today, we have enrolled seven patients. We have a growing list of interested candidates and we are optimistic that we will now gain momentum in the recruitment of the trial.
HOPE-2, our Phase II study, which was published in The Lancet last year, together with the recent late breaking open label extension data presented at this year's Parent Project for Muscular Dystrophy, or PPMD Annual Conference in June, are amplifying the interest in our HOPE-3 trial. Our current projections for enrollments are to be complete by the third quarter of 2023 or sooner.
The promise of HOPE-3 builds on the recently reported HOPE-2 open label extension data, which continue to underscore the therapeutic potential of CAP-1002 and highlight it's sustained safety and efficacy.
Let me recap that data for you to highlight its relevance to our regulatory strategy and the clinical development of CAP-1002. HOPE-2 open label extension was a very unique clinical study, which allows each patient to be used as their own control.
First, we conducted Hope-2 where one group received placebo and one group received CAP-1002. Those results showed statistical and clinically significant improvement in upper limb function in non-ambulant patients with DMD, as earlier mentioned and published.
Then, all patients went off treatment into what we call a gap phase, which was approximately one year. All patients no matter what group they were originally in declined in upper limb function during the gap days.
Then all patients went on CAP-1002 in the open label extension part of the trial and disease progression was attenuated up to 70%, most notable and those that were originally on placebo.
What is also interesting is that the original CAP-1002 group declined off therapy at the same rate that the placebo group did, but entered the open label portion of the trial with better upper limb function due to the fact that they had the benefit of one year of CAP-1002 in HOPE-2.
They started with better upper limb function and therefore, finished with better upper limb function. This is exemplary of potential disease modifying behavior of a therapeutic.
We believe this data must be shown to FDA, both because of its statistical power and clinical benefit, but also because time is muscle, based on this dataset, every year that patients are off CAP-1002, they lose function that cannot be recovered, based on our regulatory designations of RMAT, or Regenerative Medicine Advanced Therapy and orphan disease designation, and also the importance of this data to people with DMD.
We are requesting a meeting with FDA, which should occur this year to present this open label extension data, which we believe will further support our path forward towards potential regulatory approval.
However, we remain focused on executing on our HOPE-3 clinical trial, which is slated to be our pivotal trial. To remind you HOPE-3's primary endpoint is the performance of the Upper Limb 2.0 a validated tool to assess skeletal muscle function in non-ambulant patients, and also the measure in which we saw statistically significant results and HOPE-2 and in HOPE-2 open label extension. This meeting will complement a CMC meeting, which is required prior to BLA submission, which we are planning to hold later this year as well.
Another key priority for our CAP-1002 program involves preparing for the future potential commercial launch, including the scale up of manufacturing. While our current manufacturing site in Los Angeles is fully focused on supplying our HOPE-3 clinical trial, we are supplementing our manufacturing efforts by converting a portion of our San Diego labs to support potential early commercial launch.
We see this facility as a versatile and cost-effective measure. Additionally, our manufacturing plan encompasses the work we have done at Lonza as they may be an important part of our future scaled up commercial plan for CAP-1002.
As you know, we entered into a distribution and commercial agreement with Nippon Shinyaku and its U.S. subsidiary NS Pharma, an experienced and well-resourced commercial partner in the United States. NS Pharma has been a trusted DMD partner for the community and has already established a respected infrastructure to support patients and their caregivers.
If approved, we believe Nippon Shinyaku's leadership in this space will serve CAP-1002 well, enriching more eligible patients who could benefit from our therapy. As a reminder, Capricor's agreement with Nippon Shinyaku came with a $30 million upfront payment to Capricor and has a potential to reach up to $705 million in milestone payments, some of which, if achieved, will be paid during the course of HOPE-3.
To maximize the potential benefit of CAP-1002 and reach patients globally, we will continue to explore ex-U.S. partnership opportunities. Our goal is to continue to execute on our regulatory, clinical, CMC, and business development goals as I just outlined above, as we are committed to bring in CAP-1002 to patients as quickly as possible.
This is the beginning of 1000% run here
BLA news is humongous
Nothing can stop this beast now
Patience always pay off
Good luck all
I expect huge BLA news next month
$50 coming here
Compare with SRPT trading at $9 billion mktcap for DMD FDA approval
CAPR will be worth more than $1 billion after FDA approval
Pps will continue to go up leading to BLA news and Exosome cancer vaccine news very soon
This has only $120 mil mktcap now
This could pop over $1 billion mktcap upon FDA AA in a couple of months
1000% runner here
Pps will keep going up from now on in anticipation of BLA submission and Exosome vaccine partnership with big pharma
According to CC
BLA will be filed very soon
Exosome vaccine program to make partnership with big pharm later this year
We could see huge pps increase in the anticipation of BLA submission and Exosome partnership deal with big pharm
BLA will be submitted very soon
This is gonna explode huge
Any one listen to Dr. Marban today?
We went down to 4.10 after the loss was released - we closed after hours at 4.70. We have $51 million in cash - enough for almost 2 years. She will meet with FDA by the end of 2022 to try to convince them of an accelerated approval. As an MD, I think the results of the study are amazing and CAP 1002 should be approved asap.
https://viavid.webcasts.com/viewer/event.jsp?ei=1559162&tp_key=a70269ae2f
I am betting BLA submission before the end of 3rd Q
AA coming before the end of this year
This is $2 billion company upon AA soon
Still waiting.
PHROPHETS.
“news coming before the end of this quarter
$50 price target in a few months here “
You still have 24+k here?
I think at somepoint a bla will get filed maybe 4 months into the p3 study.
So patience then. Buylow sell at good price and work core till nees
I am expecting within 12 months we will get our AA.
why is this stagnent? does the company need to reassure the public as far as product development or is it just a lull before the storm?
This is 1000% runner potential now
Some group in accumulation mode
Super volcano in the making
Good news is on the way
BLA news coming
$20 coming this summer
give it to me.
Pps keep going up everyday
Looks like AA news coming
CAPR is severely undervalued around $100 mil mktcap with P3 DMD program for billion dollars revenue potential
This could be $50-$100 stock by the end of this year
Pps keep moving up everyday from now on until Accelerated Approval
The FDA has granted Capricor's CAP-1002 RMAT and Orphan Drug Designation, and the Company plans to present this data to the FDA and seek additional guidance on the best path forward for DMD patients.
New FDA commissioner Robert is advocate of Cap 1002
Looks like Capricor plan to meet FDA to persuade AA and confirmation P3 trial this time
I expect some news coining in 2 months
They gonna get Accelerated approval with P2 result or in the middle of P3
Good enough results with p2 extension trial Now
so much undervalued anyway
Let’s see what happen here this summer
I love this company but their big study for DMD is still recruiting and won’t have results until December 2024. How can we get approval before that?
https://clinicaltrials.gov/ct2/show/NCT05126758?term=Capricor&draw=2&rank=5
This is still a speculative play. Even though we like the OLE data, Capricor is still a one-trick pony until the exosome platform advances further into clinical trials. It is also dealing with cutting-edge technologies.
However, Capricor just showed standout data with statistical significance on a tiny sample. This is only possible because the clinical impacts are robust. From our vantage point, we think this is approvable data. However, we are not the FDA. The main concern going forward is safety: is there something that will show up in a larger sample?
Capricor sits on a valuation just under $110M. It was ridiculously undervalued when its recent valuation sunk under $70M. The readers took us to task during the Covid Shutdown for not being more optimistic. We felt that the main event was always the DMD program but also asserted that HOPE-2 would not results in an accelerated approval.
Now the good data has arrived. The case for CAP-1002 is stronger. We now match the enthusiasm of our former critics, but many are gone. This inefficiency should give you pause. If you were enthusiastic before, you should be delirious today.
We also said CAPR remains a speculative play…but today we are amused. As my former business partner and CEO used to say: The data is the data.
CAPR is presenting this data to FDA for Accelerated approval
This is $100 stock upon FDA approval this summer
The FDA has granted Capricor's CAP-1002 RMAT and Orphan Drug Designation, and the Company plans to present this data to the FDA and seek additional guidance on the best path forward for DMD patients. Capricor is currently conducting a pivotal Phase 3 trial, HOPE-3, designed as a randomized, double-blind, placebo-controlled study with approximately 70 patients and enrollment criteria similar to HOPE-2. The Phase 3 study is currently open for enrollment (NCT05126758).
Dr. Linda Marbán, CEO, will present the one-year open label extension results via a late-breaking session on June 25, 2022, at 5:05 p.m. MT.
Capricor will also deliver a clinical update as part of PPMD’s “Novel Approaches for Combatting Duchenne” session on June 25, 2022, at 11:00 a.m. MT.
The conference will take place in Scottsdale, Arizona from June 22-26, 2022, and will also feature an online component for attendees to join virtually.
$$$$$$$$$CARP @ $4.60 NOW$$$$$$$
Big news coming this week!
Fasten your seat belt
9:00a ET 6/17/2022 - Globe Newswire
Capricor Therapeutics to Present One-Year Efficacy Results from its Ongoing HOPE-2 Open Label Extension Study at 2022 Parent Project Muscular Dystrophy Annual Conference
Mentioned: CAPR
EQNX::TICKER_START (NASDAQ:CAPR), EQNX::TICKER_END Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases, today announced that the Company will present one-year, safety and efficacy results from its HOPE-2 open-label extension study with lead asset CAP-1002 for treating Duchenne muscular dystrophy (DMD) at this year's Parent Project Muscular Dystrophy (PPMD) Annual Conference.
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