CAPR Capricor Therapeutics Inc

$CAPR I finished solid 30-minute call with AJ Bergmann. He sounds more confident than he did the day after the adcom Most of his time is now on commercial activities. They’re occupying the new building (with the option to terminate the lease if needed) and the tone was clear: they’re preparing to launch. NS arbitration is going smoother than expected and they are extremely impressed with Skadden. They still have the door open to pivot back to the PI hearing if required, but arbitration remains the preferred path and it’s progressing well. He’ll be at more investor conferences this year, including Jefferies in the first week of December right after PDUFA. Overall, he came across as highly constructive on the path forward this review cycle. Feeling very good about where things stand. I will taken questions and answer as best as possible

$CAPR The FDA already concluded that they are using SAP 1.0 in their analysis the current review isn’t designed to convince them to use a different sap It’s about providing additional data to demonstrate that ANY sap is irrelevant because all versions of the analysis favor drug over placebo In rare disease setting .04 vs .09 is meaningless it’s about the totality of evidence and various data sets

$CAPR My “educated guess”: We get approval for skeletal muscle for males 9 and older non-ambulatory DMD patients and a 20% chance that the label includes accelerated approval for cardiomyopathy with a confirmatory trial. My prediction still stands regarding approval time: October - 85% November - 15% I think approval is coming next week. The FDA wants to say they approved deramiocel a month before PDUFA. It will show they are expediting rare terminal pediatric diseases. A win/win for all parties. I bought more this morning. 👍💰

$CAPR The spider(institutions) has spun its web(the shares short). Now it is a waiting game for its prey. The institutions don’t care about the price in the short term. They are loaning out their shares and collecting interest payments while they wait. Here is what is going to happen in the future: T1 halt pending an announcement. The stock opens north of $40 after the approval PR and the institutions start buying in the open market forcing some shorts to cover. And then the second wave happens, the institutions start calling back their shares and creates a squeeze!!! It will rocket the shares $60+ and with a potential gamma squeeze we are talking north of $80+. 👍💰💰💰 I bought more today. 👊👏👍

$CAPR Capricor’s VP of Biometrics liked the FDA’s post about transforming rare disease approvals. I’d like to think if the FDA was playing hardball with Capricor while still making posts like this, she would not be liking their message.

$CAPR FDA will wait until the week after the end of October meeting soliciting input for their new framework. Even though there will be no official framework for some time, FDA will incorporate concepts discussed in conjunction with this new framework into an approval in first week of November. That allows them to save face on using the outdated SAP at adcom while still moving forward with the approval that is absolutely necessary and beneficial and FDA knows it. Their lawyers just need them to introduce evolving thought to avoid a lawsuit on adcom. Approval Nov 6 at latest sets a target no more that 15 business days out. MM shorts etc. can do what they want between now and then but this drug is getting approved and shorts will suffer losses.

$CAPR it seems to me that it would be really odd for FDA to come out with a patient focused rare disease program to then turn around and deny a DMD rare disease drug begged for by patients

$CAPR I would like to come back to Markland55 phone call with AJ Bergmann, if I understood correctly. Please correct me if I got anything wrong!!! • Physical Expansion: The team is already actively moving into the new offices in San Diego. Although the special termination clause runs until Dec 31 (protection against FDA rejection), management is creating facts for the commercial launch ahead of the PDUFA date (Nov 22). • Skadden Power: In the NS arbitration, they are going all out. Capricor has hired Skadden, one of the most powerful elite law firms in the world. According to Bergmann, the process is going very positively!?? • Jefferies Conference: Bergmann is locked in for the first week of December—exactly after the FDA deadline. Conclusion: Nobody goes to the Jefferies conference after a failure, pays huge legal fees, and moves into exclusive new offices right now if they don't firmly expect approval... or did I misunderstand that?

$CAPR Forget the money. Yesterday I read about families fighting DMD, and I genuinely cried. It makes you realize we should never take anything for granted. Every single one of us needs to appreciate just being able to move our hands and legs. This isn't just about stocks; it’s about life. Praying for health and strength for these kids. May God bless them. 🙏

$CAPR Spotted in the wild tonight. n=1, no control arm, no pre-specified analysis plan ..but the effect size was striking. ;) Either the universe is front-running the PDUFA or I've spent too many nights with the Lancet supplementary appendix..😃

$CAPR will be buying another 20k shares next Tuesday when funds will have cleared. Actually hoping it stays around this price! All in!!

$CAPR I don't pay much attention to analyst target prices. Many analyst are wrong the majority of the time. And once Deramiocel is approved it is a new game! And when Capricor does its own US marketing its a new game! And when the new clean rooms get going its a new game! And with an European/ROW partnership its a new game! And with StealthX drugs in clinic its a new game! And with a nice short squeeze its a new game! This is going over $100. Sooner than later!

$CAPR FDA meeting about rare disease today. Mikhail Talking about how to design these treatments with insight for the people involved. What the people need and want and how to measure outcomes . Its not about any specific treatment but how the FDA can change their framework to include recipients voices and outcomes in rare diseases.

$CAPR I expect the FDA to approve Deramiocel. But I agree with the 90% of voters who told a FOX poll that corruption is their top concern. It even ran ahead of inflation! If Deramiocel is not approved it can only be because of corruption. Deramiocel data is not only solid ,it is ROBUST! Statistician Dr. Nathan Hogan... "To sum it all, we tested the HOPE-3 result more than 600 ways and the effect did not materially move, which is the opposite behavior of a fragile result. Furthermore, we increased the sample by pooling with HOPE-2 and the same treatment effect appeared with an even stronger p-value confirming the original uncertainty was not about the efficacy of the drug, but rather about sample size. This is a common concern in rare and heterogenous diseases. We ran every sensitivity analysis that was a topic in the committee, and all 8 agreed confirming the benefit of deramiocel. Cardiac data points the same way. We wanted to put all that in front of you transparently ..."

$CAPR You think Linda would have submitted an amended BLA so quickly without being certain that a CRL would be out of the question? No CRL is coming folks. No ad-com is coming either. It's going to be full approval and likely encompass both skeletal and cardio. Only 5 weeks left so buckle up!

$CAPR Interesting timing with the FDA’s proposed overhaul on rare disease framework. Less than a month before Capricor’s PDUFA. They know the ADCOM was a sham and here is their opportunity to save face and do the right thing. I’m hoping for full approval.

$CAPR FDA Clinical Studies Labeling Guidance requires the primary trial providing support for an approval to be described in Section 14 of the label. So what happens if the FDA wants to approve deramiocel but objects to the use of SAP 3.0 for HOPE-3? IMO, the likely resolution is to omit the variables (such a p value) over which there is disagreement. Section 14 of the label will be comprised of the design, baseline demographics, and 12m outcomes of HOPE-3 based on CBER-accepted sensitivity models and treatment differences—focusing on the mean treatment effect (~1.1 to 1.29 pt with a 95% CI). This would hopefully be complemented by a subsection detailing the Integrated Summary of Efficacy (ISE) mapped pooled analysis (p=0.0087) and 24m OLE functional trajectory curves. There is clear regulatory precedent for this approach in rare disease approvals where primary endpoint analyses faced internal debate, most notably in CBER's labeling of Sarepta Therapeutics' Elevidys and Exondys 51.

$CAPR Would be so awesome that 1. we get approval this month 2. $SLS announces surprise BO this month 3. Able to buy more $CADL before BLA and potential BO maybe next year? 😁

$CAPR FDA APPROVAL IS COMING ! .. VERY SOON ! Approval news could drop any day now moving forward

$CAPR it is good to see approvals happening left and right. Stay the course and don’t wake up without shares after news then halted. The FDA explicitly recognizes that traditional clinical benchmarks are often structurally unattainable for rare conditions. The agency is shifting toward a "fit for purpose" evidentiary model for rare diseases, moving away from rigid, clinical trial designs. This flexibility is a realistic adaptation to small patient population. Have a great weekend.