Deramiocel (Cap-1002)

nothing in Duchenne cardiomyopathy has come close to 4 years of ejection fraction stability in non-ambulant patients. This level of sustained cardiac preservation is unprecedented and not commonly discussed because it hasn’t existed—until now. $CAPR #DMD #Deramiocel #RareDisease

@Elijahjstacy 🙏🏻 Hope you both continue to win this fight

I saw this and had to repost, as it reminds me of my mom so much. Even as she is battling stage IV pancreatic cancer (getting a lot better btw), she still puts her kids before herself. I have an even greater respect for her since being on this cancer journey. And I have to admit a new belief of mine: you will never hate cancer enough until someone close to you has it. I sometimes debate if I would hate cancer the same if I had it vs. watching my mother have it — I don’t think I would, to be honest. It’s for this very experience that I, in some interesting way, better relate or understand the pain of DMD parents around the world. I am a better man because of this hardship, and I take pleasure in carrying the burdens of others. I am created to serve. I still see the light at the end of the tunnel, and it shines brighter than ever before for all of us!

durability. The bar is “substantial evidence of effectiveness.” HOPE-3 delivers that. $capr @Capricor @DrMakaryFDA @RobertKennedyJr @CureDuchenne @ParentProjectMD @mdnewstoday_

@US_FDA you have a double-blind, placebo-controlled Phase 3 trial that hit pre-agreed endpoints, in a disease that kills children, with a therapy that has no approved cardiac comparator, backed by 10+ years of clinical development and 48-month open-label extension data showing

Arnold Ventures (founded by John Arnold) is laser-focused on FDA accountability. They demand rigorous Phase 3 RCTs, confirmed clinical benefits, and no more surrogate-endpoint shortcuts that fail patients (they’ve repeatedly called out eteplirsen-style approvals in DMD as exactly what’s wrong with the system). Deramiocel from Capricor checks every single box they want: 1. Not accelerated approval- full BLA seeking traditional approval. 2. Pivotal Phase 3 HOPE-3 trial (randomized, double-blind, placebo-controlled) just hit its primary endpoint. 3. Real, meaningful clinical outcomes: - 54% reduction in upper-limb function decline - 91% slowing of cardiac decline - Cardiac MRI data showing actual reduction in fibrotic segments (late gadolinium enhancement) This isn’t hype on a biomarker — it’s disease-modifying data on both skeletal muscle AND cardiomyopathy, the #1 cause of death in DMD. Exactly what Arnold Ventures has been begging the FDA to require: strong, confirmatory evidence BEFORE approval so patients and payers actually get value. PDUFA date: August 22, 2026 BLA already back under active review after the CRL was lifted this month If Arnold Ventures had to design the ideal next DMD therapy to restore trust in the approval process… this is it. $CAPR patients win. Evidence wins. The system wins. What do you think — will AV publicly praise this one? 👀 #DMD #Deramiocel #CAPR #RareDisease #FDA @DrMakaryFDA

@US_FDA a narrow label will functionally deny access to patients who could benefit based on the actual trial evidence hence why an approved broad label(skeletal & cardiomyopathy) will give these kids & families a fighting chance #hope @ParentProjectMD @CureDuchenne @CoalitionDMD

$capr DMD is progressive. Ideally, you want a therapy like #deramiocel working before significant cardiac damage accumulates but a narrow cardiac label essentially forces families to wait until their child’s heart is already deteriorating

At the heart of this decision is something simple: giving patients a chance at a better, longer life. @RobertKennedyJr @DrMakaryFDA @CureDuchenne @ParentProjectMD @MarbanLab @CoalitionDMD #timeismuscle 🙏🏻

Capricor answered with a standard trial and delivered: the therapy hit every endpoint, and most importantly, it has shown a strong safety profile. We may be looking at something truly groundbreaking not just for this disease, but for the future of exosome-based medicine.

$capr @Capricor is about more than making investors money. This is about advancing real medical innovation for young boys and some women, living with a devastating terminal disease. The @US_FDA asked for stronger evidence beyond the Phase 2 and OLE data, and that was fair.

Tissue (LGE), pump function (LVEF), arm scores (PUL), patient impression, and real‑world video tasks, we see consistent, statistically significant benefit. $capr #deramiocel 🕰️🙏🏻

CEO of @Capricor @linda_marban on what clinicians should know about the company's investigational #Duchenne #celltherapy Deramiocel. #MDAconference (@MDAorg)

Today we announced that the U.S. FDA has resumed review of our BLA for Deramiocel for the treatment of Duchenne muscular dystrophy and set a PDUFA target action date of August 22, 2026. Click here to read the announcement: bit.ly/4bz2qk2 #DMD #Deramiocel $CAPR

@Elijahjstacy 🙏🏻

I decided to go the hospital. Rather safe than sorry situation. Having lots of respiratory issues still. Big problem in DMD.

Mr. President, I’m calling on you to act on Capricor’s Duchenne therapy and save hearts and lives, including mine! Let’s unleash American innovation and continue to be the medical leader in the world. Mr. President you can this happen and make history in DMD by ensuring the swift approval of deramiocel! USA USA USA 🇺🇸🇺🇸🇺🇸 @realDonaldTrump @WhiteHouse @EricTrump @DonaldJTrumpJr @POTUS @JDVance redstate.com/redstate-guest…

$capr time is muscle 🕰️ #DMD #Deramiocel #almostime

I just wrote a letter to the FDA urging them to review @Capricor’s deramiocel quickly after positive Phase III data so patients can get access sooner. ONLY if you’re a DMD patient or a parent waiting for this treatment, please sign your name here: submit.jotform.com/253445274576061 Let’s HOPE the FDA moves fast!

$capr #timeismuscle

$Capr science will always and continue to prevail🙏🏻

Science will prevail🙏🏻

Time is too precious to waste 🙏🏻

Science will prevail. Although an unprecedented turn of events has delayed the inevitable, it has also tragically deferred these boys the right to try but soon, HOPE 3 will confirm to the FDA that time is the most critical factor this community can’t afford to lose $capr