Deramiocel (Cap-1002)

As the PDUFA date nears (Aug 22nd) will keep patients, families, investors, science communities updated with news, articles, and AI insights

[Deramiocel (Cap-1002) @1002Cap]

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

[Capricor Therapeutics @Capricor]

Capricor’s quarterly conference call and webcast is starting at 4:30 p.m. ET today. <<<Click the link to access the webcast https://t.co/ddU5SDEbSx>>>
$CAPR #RareDisease

[DC_Draino @DC_Draino]

This is Kai and he’s a teenager with Duchenne Muscular Dystrophy, a genetic disease that kills most boys before 25. 

His life was improved when he joined a clinical trial for a promising drug called “Deramiocel” from Capricor Therapeutics.

Then that drug’s review process was put on pause by the FDA until the company refiled with the best data ever in treating DMD. Kids had 54% better function at the end of a clinical trial called HOPE-3. Thank goodness, it's finally under review again. 

Since 2025, FDA reviewers have rejected 23 rare disease drugs and some believe this was driven by Vinay, but others suggest it's the near-sighted FDA reviewers.  Trump finally pushed Vinay out for his inability to do the job the American people demand.  So there is hope that treatments for rare diseases may see some light.

Moms of dying kids don't care about the deep state’s "process." They care if their child sees Christmas.

Acting U.S. FDA Commissioner Kyle Diamantas said he wants to reopen approvals for these rare disease drugs. 

Here’s hoping he’s serious.

And moves fast.

#CapricorPartner

[Deramiocel (Cap-1002) @1002Cap]

Science will prevail🙏🏻 $capr #TimeIsMuscle #Deramiocel #DMD

[DC_Draino @DC_Draino]

This is Kai and he’s a teenager with Duchenne Muscular Dystrophy, a genetic disease that kills most boys before 25. 

His life was improved when he joined a clinical trial for a promising drug called “Deramiocel” from Capricor Therapeutics.

Then that drug’s review process was put on pause by the FDA until the company refiled with the best data ever in treating DMD. Kids had 54% better function at the end of a clinical trial called HOPE-3. Thank goodness, it's finally under review again. 

Since 2025, FDA reviewers have rejected 23 rare disease drugs and some believe this was driven by Vinay, but others suggest it's the near-sighted FDA reviewers.  Trump finally pushed Vinay out for his inability to do the job the American people demand.  So there is hope that treatments for rare diseases may see some light.

Moms of dying kids don't care about the deep state’s "process." They care if their child sees Christmas.

Acting U.S. FDA Commissioner Kyle Diamantas said he wants to reopen approvals for these rare disease drugs. 

Here’s hoping he’s serious.

And moves fast.

#CapricorPartner

[Elijah Stacy @Elijahjstacy]

Let the phrase “time is muscle” actually mean something and get this approved immediately!

[Deramiocel (Cap-1002) @1002Cap]

We look forward to a transformational summer 🙏🏻 #Deramiocel #DMD $CAPR

[CureDuchenne @CureDuchenne]

Happy Mother’s Day 💙

The love, strength, and fierce dedication of Duchenne moms and mom figures is unmatched—and today, we’re celebrating all of you.

Thank you for being the heart of this community and for everything you do, seen and unseen.

#MothersDay #Mom

[Deramiocel (Cap-1002) @1002Cap]

@Capricor didn’t just check the box they set a new standard for what DMD evidence looks like. $CAPR #deramiocel #timeismuscle #DMD

[Deramiocel (Cap-1002) @1002Cap]

HOPE-3 is the first Phase 3 randomized controlled trial in advanced Duchenne muscular dystrophy to hit every pre-specified primary and secondary endpoint. No therapy in the history of this disease has done that in this patient population.

[Capricor Therapeutics @Capricor]

At the American Academy of Neurology (AAN) 2026 Annual Meeting in Chicago, Capricor will be presenting Phase 3 HOPE-3 results for Deramiocel in a late-breaking oral presentation later today. The presentation highlights previously reported topline data demonstrating significant functional benefits in patients with Duchenne muscular dystrophy (DMD). For more information, please click https://t.co/icJhFL0gA3
$CAPR #Deramiocel #DMD #AANAM #Duchenne

[Deramiocel (Cap-1002) @1002Cap]

@CoalitionDMD @DuchenneNow @DMDfoundation @DuchenneUK @DuchenneDF @ParentProjectMD @mdnewstoday_ #timeismuscle

[Deramiocel (Cap-1002) @1002Cap]

@US_FDA @DrMakaryFDA No excuses to keep boys with Duchenne waiting for a now proven therapy. With all the public talk about speed and flexibility, you owe it to a community that has neither. Capricor has met the standard every step of the way. The time is now

[Deramiocel (Cap-1002) @1002Cap]

It has already earned Orphan Drug, RMAT, Rare Pediatric Disease, and ATMP designations across the US and EU, all recognizing serious unmet need and promising evidence. Families are still waiting for access to the very therapy those designations were meant to speed.
$CAPR

[Exosome RNA @ExosomeRNA]

Exosomes deliver key reproductive signals between neurons, revealing new biology. - @uOttawa https://t.co/7Z6FCyqEnz

[CureDuchenne @CureDuchenne]

Today, we celebrate the incredible siblings in our CureDuchenne community 💙

You are advocates, teammates and best friends. Your role is powerful, and your impact is immeasurable.

We see you and we appreciate you.

#NationalSiblingsDay #DMD #Siblings #DuchenneCommunity

[Deramiocel (Cap-1002) @1002Cap]

At this point a second CRL from the FDA wouldn’t just be a regulatory decision, it would be an institutional choice to be adversarial without scientific justification against dying children. The data speaks for itself $CAPR #timeismuscle 🙏🏻 #ApproveIt #DMD

[Deramiocel (Cap-1002) @1002Cap]

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

[Deramiocel (Cap-1002) @1002Cap]

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

[Deramiocel (Cap-1002) @1002Cap]

@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

[Markland55 @markland_55]

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

[Deramiocel (Cap-1002) @1002Cap]

@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

[Deramiocel (Cap-1002) @1002Cap]

$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