Capricor Therapeutics Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy

Rhea-AI Summary

Capricor Therapeutics (NASDAQ: CAPR) reported positive topline results from the pivotal Phase 3 HOPE-3 trial of Deramiocel in Duchenne muscular dystrophy on Dec 3, 2025. The randomized, double-blind, placebo-controlled study (n=106) met its primary endpoint, PUL v2.0 (54% slowing vs placebo; p=0.029), and its key secondary cardiac endpoint, LVEF (91% preservation vs placebo; p=0.041). Results were statistically significant across type‑1‑error controlled secondary endpoints and safety was consistent with prior studies. The company plans to submit a response to its prior Complete Response Letter incorporating HOPE-3 data.

Positive

• PUL v2.0 slowing of progression: 54% (p=0.029)
• LVEF preservation vs placebo: 91% (p=0.041)
• Phase 3 randomized double-blind placebo-controlled trial with n=106
• Favorable safety and tolerability consistent with prior experience

Negative

• Key cardiac endpoint evaluated in a smaller subset: n=83
• Primary and key secondary p-values are near significance threshold (p=0.029 and 0.041)
• Company previously received a Complete Response Letter requiring a regulatory response

News Market Reaction

+371.07% 24.6x vol

73 alerts

+371.07% News Effect

+439.0% Peak in 5 hr 22 min

+$1.45B Valuation Impact

$1.85B Market Cap

24.6x Rel. Volume

On the day this news was published, CAPR gained 371.07%, reflecting a significant positive market reaction. Argus tracked a peak move of +439.0% during that session. Our momentum scanner triggered 73 alerts that day, indicating high trading interest and price volatility. This price movement added approximately $1.45B to the company's valuation, bringing the market cap to $1.85B at that time. Trading volume was exceptionally heavy at 24.6x the daily average, suggesting very strong buying interest.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

HOPE-3 sample size: 106 participants PUL v2.0 effect: 54% slowing PUL v2.0 p-value: p=0.029 +5 more

8 metrics

HOPE-3 sample size 106 participants Pivotal Phase 3 HOPE-3 DMD trial

PUL v2.0 effect 54% slowing Primary endpoint vs placebo; p=0.029

PUL v2.0 p-value p=0.029 Primary skeletal muscle function endpoint

LVEF effect 91% preservation Key secondary cardiac endpoint vs placebo; p=0.041

LVEF p-value p=0.041 Key secondary cardiac MRI endpoint

Dose level 150 million cells Deramiocel per infusion every three months

OLE duration 48 months HOPE-2 open-label extension follow-up

DMD prevalence US 15,000 individuals Estimated affected population in the United States

Market Reality Check

Price: $21.57 Vol: Volume 1,820,521 is below...

low vol

$21.57 Last Close

Volume Volume 1,820,521 is below the 20-day average of 5,796,033, suggesting limited pre-news positioning. low

Technical Trading above 200-day MA of 9.41, indicating a pre-existing longer-term uptrend into this catalyst.

Peers on Argus

Pre-news, CAPR was up 4.71% while peers were mixed: CADL and ALEC showed double-...

Pre-news, CAPR was up 4.71% while peers were mixed: CADL and ALEC showed double-digit gains, OMER and LCTX declined, and FHTX was modestly positive. This pattern points to stock-specific dynamics rather than a coordinated biotech move.

Historical Context

5 past events · Latest: Dec 05 (Negative)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Dec 05	Equity offering pricing	Negative	+5.4%	Pricing of $150M common stock offering at $25.00 per share.
Dec 04	Equity offering launch	Negative	-14.9%	Announcement of proposed underwritten public offering of common stock.
Dec 03	Pivotal trial results	Positive	+371.1%	HOPE-3 Phase 3 met primary and key secondary endpoints in DMD.
Nov 24	Platform data update	Positive	-19.3%	New exosome manufacturing data presented at AAEV 2025.
Nov 10	Earnings and pipeline	Negative	-6.2%	Q3 2025 loss and update on HOPE-3 timing and regulatory plans.

Pattern Detected

Recent news often triggers large, directional moves, with dilutive offerings sometimes met with unexpectedly positive price action and core Deramiocel milestones drawing outsized reactions.

Recent Company History

Over the last six months, Capricor’s trajectory has centered on Deramiocel and funding its development. Earlier clinical and regulatory updates for Deramiocel, including orphan and other designations, set expectations for pivotal HOPE-3 data. The current Phase 3 topline success on Dec 3, 2025 follows that roadmap and precedes capital-raising offerings on Dec 4 and Dec 5. Exosome-platform data and quarterly results also shaped sentiment but drew less sustained positive reaction than today’s pivotal readout.

Regulatory & Risk Context

Active S-3 Shelf · $300,000,000 of securities plus up to $150,000,000 of common stock via equity distribution

Shelf Active

Active S-3 Shelf Registration 2025-09-10

$300,000,000 of securities plus up to $150,000,000 of common stock via equity distribution registered capacity

An effective S-3 shelf filed on Sep 10, 2025 authorizes Capricor to issue up to $300,000,000 of securities and separately sell up to $150,000,000 of common stock under an equity distribution agreement, highlighting substantial capacity to raise additional capital that could be dilutive to existing shareholders if utilized.

Market Pulse Summary

The stock surged +371.1% in the session following this news. A strong positive reaction aligns with ...

Analysis

The stock surged +371.1% in the session following this news. A strong positive reaction aligns with the pivotal nature of HOPE-3, which delivered statistically significant benefits on both skeletal (PUL v2.0) and cardiac (LVEF) endpoints and showed a 371.07% move on the day of the topline release. Historically, clinical updates moved CAPR only modestly, so such a spike could also reflect prior high short interest and future capital-raising capacity under the $300,000,000 shelf and $150,000,000 equity distribution.

Key Terms

pul v2.0, lvef, cardiomyopathy, exosomes, +4 more

8 terms

pul v2.0 medical

"met the primary endpoint (PUL v2.0) and the key secondary cardiac endpoint"

PUL v2.0 is an updated version of the PUL system or protocol that changes how the product works, usually to make it faster, safer, or cheaper to use. For investors, it matters because the update can change potential returns and risks — it might lower costs and boost performance, or introduce new features that change how money is managed.

lvef medical

"key secondary cardiac endpoint (LVEF), both achieving statistical significance"

Left ventricular ejection fraction (LVEF) is a percentage that measures how much blood the heart’s main pumping chamber pushes out with each beat, like the share of water a pump empties from a bucket each cycle. Investors watch LVEF because it’s a key medical yardstick used to diagnose and track heart function, shaping demand for drugs, devices, clinical trials, insurance costs and the financial outlook of healthcare-related businesses.

cardiomyopathy medical

"designed to treat Duchenne cardiomyopathy, the leading cause of mortality"

A condition that weakens or stiffens the heart muscle, reducing its ability to pump blood effectively; think of the heart as an engine that becomes less powerful or less flexible. For investors, cardiomyopathy matters because it can drive demand for medical treatments, affect healthcare costs, influence the value of companies developing drugs or devices, and trigger regulatory or insurance impacts that change revenues and risks across the healthcare sector.

exosomes medical

"preclinical development focused on vaccinology and the targeted delivery of oligonucleotides, proteins, and small-molecule therapeutics"

Exosomes are tiny particles released by cells that carry proteins, genetic material, and other molecules from one cell to another. They act like biological messengers, helping cells communicate and coordinate their activities. For investors, understanding exosomes is important because they are being explored as potential tools for disease diagnosis and treatment, which could influence healthcare markets and innovation.

orphan drug designation regulatory

"Deramiocel has received Orphan Drug Designation for the treatment of Duchenne"

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

regenerative medicine advanced therapy regulatory

"granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S."

Regenerative Medicine Advanced Therapy (RMAT) is a U.S. regulatory designation for cell, gene, and tissue‑based therapies intended to treat serious or life‑threatening conditions; it gives developers a “fast lane” with more frequent agency interaction and eligibility for accelerated review pathways. For investors, an RMAT label signals that a therapy may reach market faster and face less regulatory uncertainty than a standard program, which can raise the potential value and reduce timeline risk—though it is not a guarantee of approval.

advanced therapy medicinal product regulatory

"Advanced Therapy Medicinal Product (ATMP) designation in Europe"

Medicines made from living cells, genes, or engineered tissues that aim to treat or cure disease by changing biological processes rather than using traditional chemical drugs. They matter to investors because they can command high prices and rapid growth if approved, but also carry large development costs, complex manufacturing and regulatory hurdles, and binary outcomes (success or failure) that can dramatically affect a company’s value—think of them as high-risk, high-reward bespoke therapies.

rare pediatric disease designation regulatory

"granted Regenerative Medicine Advanced Therapy (RMAT) designation... and Rare Pediatric Disease Designation"

A rare pediatric disease designation is an official regulatory status given to a drug or therapy that targets a serious or life‑threatening condition primarily affecting children and is uncommon in the population. It matters to investors because the status often brings financial and development perks — such as tax credits, reduced fees, faster review and periods of market protection — which can lower costs, speed approval and improve the commercial outlook; think of it as a VIP pass that makes bringing a scarce, child‑focused treatment to market easier and potentially more profitable.

AI-generated analysis. Not financial advice.

• Pivotal Phase 3 randomized, double-blind, placebo-controlled study (n=106) met the primary endpoint (PUL v2.0) and the key secondary cardiac endpoint (LVEF), both achieving statistical significance (p=0.03 and p=0.04, respectively)
• Statistical significance was achieved in all type 1 error controlled secondary endpoints
• Results demonstrate clinically meaningful and statistically significant skeletal and cardiac benefits, supporting Deramiocel as a potential first-in-class therapy designed to treat Duchenne cardiomyopathy, the leading cause of mortality in Duchenne
• Deramiocel maintained a favorable safety and tolerability profile consistent with prior clinical experience
• Company plans to submit its response to the Complete Response Letter incorporating HOPE-3 data, following prior alignment with FDA
• Conference call and webcast today at 8:00 a.m. ET
  
  

SAN DIEGO, Dec. 03, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced positive topline results from its pivotal Phase 3 HOPE-3 trial evaluating Deramiocel, the Company’s investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD).

“HOPE-3 delivered strong and definitive evidence that Deramiocel can meaningfully improve the course of Duchenne muscular dystrophy, demonstrating statistically significant improvements in both skeletal and cardiac function,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “These results reinforce the durable benefits seen in HOPE-2 and its open-label extension, which has continued for over 48 months, and highlight the strength, consistency, and reproducibility of Deramiocel’s clinical profile after more than a decade of rigorous clinical development. We believe these pivotal study results, in addition to the evidence from the HOPE-2 and HOPE-2 OLE studies, position us to address the clinical issues in the Complete Response Letter received earlier this year, consistent with prior FDA guidance that HOPE-3 results should be sufficient to support regulatory approval.”

HOPE-3 is a randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating Deramiocel in boys and young men with Duchenne muscular dystrophy. The study randomized 106 participants across 20 leading U.S. clinical sites. Participants received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for a 12-month period. The average age of participants was approximately 15 years, and all were on a stable corticosteroid regimen throughout the study. Baseline demographics were well balanced between treatment arms, approximately 90 percent were receiving cardiac medications at baseline, and over 75 percent had a clinical diagnosis of cardiomyopathy. Deramiocel maintained a favorable safety and tolerability profile consistent with prior clinical experience.

Topline Efficacy Results

Endpoint	% Slowing of Progression3 (Deramiocel vs. Placebo)	p-value
Performance of Upper Limb (PUL v2.0) Total Score¹ (Primary, n=105)	54%	p=0.029
Left Ventricular Ejection Fraction (LVEF %)² (Key Secondary, n=83)	91%	p=0.041

¹ n reflects the number of patients in the ITT population with evaluable PUL v2.0 assessments at 12 months.
² n reflects the number of patients in the ITT population with centrally reviewed and evaluable cardiac MRI LVEF assessments at 12 months.
³ Percent slowing is calculated as the treatment difference divided by the placebo change from baseline.

“We believe the HOPE-3 PUL results show statistically and clinically meaningful and significant treatment effects on both upper limb function and cardiomyopathy,” said Craig McDonald, M.D., Distinguished Professor of Physical Medicine & Rehabilitation and Pediatrics at UC Davis Health, and National PI of the HOPE-3 trial. “A nearly 54 percent slowing of skeletal muscle disease progression is extraordinary in Duchenne and directly linked to maintaining independence and quality of life in the most severely affected patients with greatest unmet need. The preservation of function reflected in PUL v2.0 translates into real, practical benefits for boys and young men living with this disease, and the effect of Deramiocel on cardiomyopathy will potentially translate to improved long-term survival. The HOPE-3 study is the first-ever Phase 3 trial in a largely non-ambulatory population with DMD to successfully meet its primary endpoint and to support the development of an innovative therapy over many years with this level of impact has been a profound privilege.”

“The cardiac findings from HOPE-3 represent a significant advance in the management of Duchenne muscular dystrophy cardiomyopathy,” said Jonathan Soslow, M.D., MSCI, Professor of Pediatrics (Cardiology) at Vanderbilt University Medical Center. “Cardiomyopathy is the leading cause of mortality in Duchenne, and stabilizing cardiac function has remained a major unmet need. The statistically and clinically significant preservation of left ventricular ejection fraction in patients treated with Deramiocel observed in HOPE-3 underscores the potential of Deramiocel to address one of the most critical aspects of the disease.”

Dr. Marbán continued, “For families living with Duchenne who are looking for therapies that preserve functional ability, protect the heart and maintain independence, today’s results provide real momentum and meaningful progress, offering renewed confidence as we work to advance Deramiocel toward potential regulatory approval.”

We expect that detailed HOPE-3 results will be submitted for presentation at a future scientific meeting and for publication in a peer-reviewed journal.

Conference Call and Webcast

To participate in the conference call, please dial 1-800-717-1738 (Domestic) or 1-646-307-1865 (International) and reference the conference ID: 52151. Participants can use guest dial-in numbers above and be answered by an operator or click the Call me™ link for instant telephone access to the event. To participate via a webcast, please click here. A replay of the webcast will be available following the conclusion of the live broadcast and will be accessible on the Company’s website.

About Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited.

About Deramiocel

Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials.

Deramiocel has received Orphan Drug Designation for the treatment of Duchenne Muscular Dystrophy (DMD) from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.

About the HOPE-3 Phase 3 Trial

HOPE-3 is a Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial consisting of two cohorts evaluating the safety and efficacy of Deramiocel in participants with DMD. Non-ambulatory and ambulatory boys who meet eligibility criteria are randomly assigned to receive either Deramiocel or placebo every 3 months for a total of four doses during the first 12 months of the trial. A total of 106 eligible subjects were randomized in the dual-cohort trial. For more information, please visit ClinicalTrials.gov (NCT05126758).

About Capricor Therapeutics

Capricor Therapeutics (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, Deramiocel, an allogeneic cardiac-derived cell therapy that is currently in late-stage clinical development for the treatment of Duchenne muscular dystrophy (DMD). Extensive preclinical and clinical data have demonstrated Deramiocel’s potent immunomodulatory and anti-fibrotic effects in helping to preserve cardiac and skeletal muscle function in DMD. Capricor is also leveraging the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on vaccinology and the targeted delivery of oligonucleotides, proteins, and small-molecule therapeutics, with the potential to treat and prevent a wide range of diseases. At Capricor, we are committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and X.

Cautionary Note Regarding Forward-Looking Statements

Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including future interactions with regulatory authorities and the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; the potential that required regulatory inspections may be delayed or not be successful which would delay or prevent product approval; the ability to achieve product milestones and to receive milestone payments from commercial partners; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2024, as filed with the Securities and Exchange Commission on March 26, 2025, and in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2025, as filed with the Securities and Exchange Commission on November 10, 2025. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.

Capricor has entered into an agreement for the exclusive commercialization and distribution of Deramiocel for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel and the StealthX™ vaccine are investigational candidates and have not been approved for commercial use in any indication.

For more information, please contact:

Capricor Media Contact:
Raquel Cona
KCSA Strategic Communications
rcona@kcsa.com
212.896.1204

Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755

FAQ

What did Capricor (CAPR) announce on Dec 3, 2025 about Deramiocel?

Capricor announced positive topline HOPE-3 Phase 3 results: PUL v2.0 54% slowing (p=0.029) and LVEF 91% preservation (p=0.041).

How large was the HOPE-3 trial for CAPR Deramiocel and who was enrolled?

HOPE-3 randomized 106 boys and young men with DMD across 20 U.S. sites; average age ≈15 years.

Did HOPE-3 show cardiac benefit for Deramiocel (CAPR)?

Yes—HOPE-3 met the key secondary cardiac endpoint with 91% preservation of LVEF versus placebo (p=0.041) in evaluable patients.

What is Capricor’s next regulatory step after the HOPE-3 topline results?

The company plans to submit a response to its prior Complete Response Letter incorporating HOPE-3 data.

Were there any safety concerns reported in the HOPE-3 topline results for CAPR?

Deramiocel maintained a favorable safety and tolerability profile consistent with prior clinical experience.

Will detailed HOPE-3 results be published for CAPR Deramiocel?

Capricor expects to submit detailed HOPE-3 results for presentation at a future scientific meeting and for peer-reviewed publication.