Capricor Therapeutics (CAPR) Soars on Positive Phase 3 HOPE‑3 Results: Latest Stock News, Analyst Targets and 2025 Outlook

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is back in the spotlight on December 3, 2025, after announcing positive topline results from its pivotal Phase 3 HOPE‑3 trial of deramiocel (CAP‑1002) in Duchenne muscular dystrophy (DMD). The news has reignited hopes for eventual U.S. approval of the therapy and sent CAPR shares sharply higher in premarket trading. ^[1]

With Wall Street price targets implying several‑hundred‑percent upside from current levels, and a high‑stakes regulatory saga still unfolding after the FDA’s rejection earlier this year, CAPR has become one of the most closely watched small‑cap biotech stocks of late 2025. ^[2]

Below is a detailed look at today’s news, the company’s evolving regulatory path, analyst forecasts, financial position, and the key risks investors should keep in mind.

What Happened Today: HOPE‑3 Hits Its Primary and Cardiac Endpoints

On December 3, 2025, Capricor reported that its pivotal Phase 3 HOPE‑3 trial of deramiocel in DMD met both its primary and key secondary endpoints, delivering statistically significant benefits in skeletal and cardiac function compared with placebo. ^[3]

According to Capricor and subsequent Reuters coverage:

Primary endpoint (skeletal muscle function)
HOPE‑3 measured upper limb function using the Performance of the Upper Limb 2.0 (PUL v2.0) scale. Patients treated with deramiocel showed a 54% slower decline in upper limb function versus placebo, with the result reaching statistical significance. ^[4]
Key secondary endpoint (cardiac function)
The trial also evaluated left ventricular ejection fraction (LVEF) via cardiac MRI. Deramiocel recipients experienced a 91% slower worsening of heart function compared with placebo, again with statistically significant results. ^[5]
Trial design and population
HOPE‑3 is a randomized, double‑blind, placebo‑controlled Phase 3 study enrolling 105–106 boys and young men with DMD. Participants received deramiocel or placebo every three months for one year. More than three‑quarters already had cardiomyopathy at baseline, underscoring the advanced disease stage in this population. ^[6]
Safety profile
Capricor reported that deramiocel was generally well tolerated, with no new major safety signals. ^[7]

The data directly target the heart and skeletal muscle decline that drive morbidity and mortality in DMD. Heart complications are the leading cause of death in these patients, often in their twenties or thirties. ^[8]

Market Reaction

Reuters reported that shares of Capricor jumped about 18% in premarket trading on the HOPE‑3 news. ^[9]

As of intraday trading on December 3, CAPR is changing hands in the mid‑$6 range, after closing at $6.36 on December 2 and about $6.65 on December 1. ^[10]

That puts the company’s market cap around $290–300 million, according to multiple data providers. ^[11]

From FDA Rejection to a Renewed Path Forward

Today’s positive data come just months after a major setback.

The July 2025 Complete Response Letter (CRL)

On July 11, 2025, the U.S. Food and Drug Administration issued a Complete Response Letter (CRL) declining to approve deramiocel for DMD‑associated cardiomyopathy. Reuters reported that the agency concluded the submitted evidence did not meet efficacy requirements and requested additional data. CAPR shares fell roughly 30% in early trading after the news. ^[12]

Patient advocacy group Parent Project Muscular Dystrophy later summarized the CRL, highlighting the need for more robust clinical data to support durable benefit in both cardiac and skeletal muscle function. ^[13]

Type A Meeting: HOPE‑3 Becomes the “Additional Study”

In September 2025, Capricor announced the outcome of a Type A meeting with the FDA to clarify the path forward. Key points from that regulatory update: ^[14]

The FDA agreed that HOPE‑3 can serve as the “additional study” requested in the CRL.
Capricor can submit HOPE‑3 data within the existing BLA, maintaining PUL v2.0 as the primary endpoint and using LVEF as a key secondary endpoint that the company hopes to include on the label.
Meeting minutes noted that the FDA “remains committed to collaborating” with Capricor and is willing to exercise “further regulatory flexibility” in reviewing HOPE‑3 data. ^[15]

Capricor has said that it plans to resubmit its response to the CRL using HOPE‑3 results, aiming for a label that reflects both cardiac and skeletal muscle benefits in DMD. The company expects this resubmission to be reviewed as a Type 2application, which typically carries a review period of up to six months once accepted by the FDA. ^[16]

Today’s positive HOPE‑3 topline readout is therefore more than just a clinical win—it’s potentially the missing piece in an amended approval package.

What Is Deramiocel and Why Does It Matter?

Deramiocel (CAP‑1002) is an allogeneic cell therapy made from cardiosphere‑derived cells (CDCs), a specialized population of cardiac cells. These cells are thought to exert anti‑fibrotic and immunomodulatory effects, largely through exosomes and soluble factors that can shift macrophages toward a more reparative, less inflammatory phenotype. ^[17]

Key points from recent scientific updates:

A peer‑reviewed paper in Biomedicines, highlighted by Capricor in November 2025, described a validated in‑vitro potency assay for deramiocel. The study showed that CDCs can suppress collagen I and III gene expression in human fibroblasts—supporting their anti‑fibrotic mechanism of action. ^[18]
Results were consistent across more than 100 manufacturing lots, strengthening the case that deramiocel’s biological activity is reproducible at commercial scale. ^[19]

Deramiocel carries multiple regulatory designations, including Orphan Drug, Regenerative Medicine Advanced Therapy (RMAT) in the U.S., Advanced Therapy Medicinal Product (ATMP) in Europe, and Rare Pediatric Diseasedesignation, which could qualify Capricor for a priority review voucher if the therapy is approved. ^[20]

Capricor has also signed an exclusive commercialization and distribution agreement with Nippon Shinyaku (NS Pharma) for deramiocel in the United States and Japan, positioning the company with an established commercial partner if approval is secured. ^[21]

Analyst Forecasts: Wall Street Sees Multi‑Bagger Potential

Despite the regulatory roller coaster, analyst sentiment on CAPR remains notably bullish.

Consensus Ratings and Price Targets

Different platforms report broadly similar conclusions:

StockAnalysis.com
- 6 covering analysts
- Consensus rating: “Strong Buy”
- Average 12‑month price target: $23.17
- Target range: $13 (low) to $30 (high), implying roughly +260% upside from recent levels in the mid‑$6 range. ^[22]
MarketBeat
- 9 analysts
- Average price target around $22.38, with the same $13–$30 range, implying ~250% potential upside versus recent prices. ^[23]
TipRanks
- Over the last three months, CAPR has an average analyst price target of about $21–22, with no sell ratingsreported. A recent snapshot showed 15 Buy and 7 Hold ratings for the month and an average target of $21.25. ^[24]
Zacks Investment Research
- Zacks lists a consensus target in the low‑$20s, with individual estimates generally spanning $13 to the high‑$20s, again implying substantial upside versus current trading levels. ^[25]

Several research notes—including those from Roth Capital and HC Wainwright—have reiterated buy‑equivalent ratingsfollowing regulatory updates, with targets typically in the low‑ to mid‑$20s and, in at least one recent case, adjusted from $12 to $13 while maintaining a Strong Buy call. ^[26]

Independent and Retail‑Focused Commentary

A MarketBeat feature described Capricor as a stock that had previously surged more than 100% on optimism around deramiocel and highlighted the current analyst target cluster in the low‑$20s. ^[27]
A Seeking Alpha article published after the FDA’s CRL framed the sell‑off as a potential “buying opportunity,” arguing that the HOPE‑3 readout and path to a 2026 approval could unlock significant value if the trial succeeded. ^[28]
Short‑term trading sentiment has been volatile. A recent StockTwits/Investing.com piece described CAPR as sparking a “retail frenzy” ahead of today’s data release, while options data showed heavy put activity earlier in November. ^[29]

Analyst enthusiasm is clearly anchored on deramiocel ultimately reaching the market; if approval does not materialize, these targets would almost certainly be revised lower.

The Martin Shkreli Short Call and Elevated Volatility

CAPR’s volatility spiked even before today’s good news.

In late November 2025, controversial former pharmaceutical executive Martin Shkreli publicly announced a short position in Capricor, arguing that deramiocel would fail the HOPE‑3 study and that the company’s prospects were overstated. ^[30]

Investing.com and Benzinga both reported that CAPR shares plunged roughly 15–17% on the day of Shkreli’s comments as traders reacted to the high‑profile short thesis. ^[31]
Around the same time, Defenseworld and other outlets noted a surge in put‑option volume, underscoring heightened bearish speculation. ^[32]

However, not all commentary sided with the short call. A recent piece in Cantech Letter highlighted a Roth Capital Markets note by analyst Boobalan Pachaiyappan, arguing that “shorters won’t take down Capricor” and suggesting the stock may be undervalued relative to its DMD opportunity. ^[33]

Today’s HOPE‑3 success directly contradicts the central premise of Shkreli’s thesis—that the trial would fail—though the market will now refocus on the FDA’s review and commercial execution.

Financial Position: Cash Runway Into Late 2026

Capricor remains a pre‑revenue biotech, so its ability to fund operations through the next phase of regulatory review is critical.

From the company’s Q3 2025 results: ^[34]

Cash, cash equivalents and marketable securities:
Approximately $98.6 million as of September 30, 2025 (down from about $151.5 million at the end of 2024).
Revenue:
$0 in Q3 2025 and year‑to‑date, as prior revenues were associated with recognition of milestone payments from its distribution agreement with Nippon Shinyaku and were fully recognized by the end of 2024.
Operating expenses:
Roughly $26.3 million in Q3 2025 and $79.0 million for the first nine months of 2025, reflecting higher clinical and manufacturing spend as deramiocel advances.
Net loss:
About $24.6 million in Q3 and $74.9 million for the first nine months of 2025.
Runway guidance:
Management expects its current cash balance to fund planned operations into the fourth quarter of 2026, excluding any additional milestones or strategic uses of capital.

Third‑party analyses, including pieces on Yahoo Finance and Simply Wall St, have generally concluded that although Capricor is burning cash, its runway appears adequate for the next couple of years, assuming no major delays or unexpected cost spikes. ^[35]

Given the likely need for commercial build‑out, post‑approval commitments, and potential label expansion studies, many investors still expect some form of future financing or partnership activity, especially if the FDA response takes longer than anticipated.

Beyond Deramiocel: The StealthX™ Exosome Platform

While deramiocel is the near‑term value driver, Capricor is also investing in a broader exosome‑based therapeutics platform.

Project NextGen StealthX Vaccine Trial

In August 2025, Capricor announced that the first subjects had been dosed in a Phase 1 clinical trial of its StealthX exosome‑based vaccine, funded by the U.S. National Institute of Allergy and Infectious Diseases (NIAID) under the federal Project NextGen program. ^[36]

Key details:

The vaccine uses engineered exosomes displaying SARS‑CoV‑2 proteins on their surface, aiming to offer an alternative to traditional mRNA platforms.
The Phase 1 study includes multiple dosing arms and may later incorporate a multivalent construct that also targets the nucleocapsid protein, pending additional FDA clearance. ^[37]
Initial topline data from this trial are currently expected in Q1 2026, subject to NIAID timelines. ^[38]

Scalable Loading of Therapeutic Oligonucleotides

In November 2025, Capricor presented new data at the American Association for Extracellular Vesicles (AAEV) 2025meeting describing a scalable framework for loading therapeutic siRNA and phosphorodiamidate morpholino oligomers (PMOs) into exosomes. ^[39]

The poster showed that:

Exosomes derived from 293F cells could be efficiently loaded with siRNA and PMO cargo using optimized electroporation conditions.
Combining scale‑up and scale‑out manufacturing strategies enabled production of significantly larger batches of exosome therapeutics with comparable loading efficiency to small‑volume methods. ^[40]

Together with the deramiocel potency‑assay publication, these data support Capricor’s long‑term goal of using exosomes not just as by‑products of cell therapy, but as a standalone, modular delivery platform for vaccines and nucleic‑acid‑based drugs. ^[41]

Legal and Shareholder Overhangs

The CRL and subsequent stock drop have attracted attention from securities law firms, several of which have announced investigations or potential class actions on behalf of shareholders. ^[42]

Notable recent items include:

Investigations by firms such as Bragar Eagel & Squire, Johnson Fistel, Wolf Popper and others into whether Capricor misled investors about the probability of FDA approval prior to the July 2025 rejection. ^[43]
Additional “investor alert” press releases following the CRL and the sharp decline in CAPR’s share price. ^[44]

These matters are still at an early stage, and no adverse findings have been announced. However, they add an extra layer of uncertainty that investors should factor into their risk assessment.

Key Risks for CAPR Investors

Even with today’s positive news, Capricor remains a high‑risk, high‑reward biotech name. Major risks include:

Regulatory risk
- The FDA has already issued one CRL, citing efficacy concerns. The agency will now scrutinize HOPE‑3 data to determine whether deramiocel truly delivers clinically meaningful benefit that outweighs risks. Approval is not guaranteed, even with statistically significant endpoints. ^[45]
Single‑asset concentration
- In the near term, Capricor is heavily reliant on deramiocel. If the FDA ultimately rejects the therapy or requires additional large trials, the company’s valuation could be severely impacted.
Financing and dilution
- While Capricor believes its current cash can fund operations into late 2026, a negative regulatory outcome or slower‑than‑expected ramp could prompt equity raises or other dilutive financings. ^[46]
Competition
- DMD is an active therapeutic area, with players such as Sarepta Therapeutics and others already marketing or developing gene and exon‑skipping therapies. Deramiocel aims to treat cardiomyopathy across mutation types, but it will likely compete in a crowded landscape. ^[47]
Litigation and reputational risk
- Ongoing shareholder investigations and the very public short thesis from Martin Shkreli underscore reputational and legal overhangs that could affect sentiment regardless of fundamentals. ^[48]
Execution risk in cell‑therapy manufacturing
- Although Capricor has completed a pre‑license inspection and validated potency assays, scaling and maintaining consistent cell‑therapy manufacturing at commercial volumes remains complex and resource‑intensive. ^[49]

Bottom Line: A Pivotal Day for CAPR Stock

For Capricor Therapeutics, December 3, 2025 marks a crucial inflection point:

The HOPE‑3 trial met its primary and key secondary endpoints, showing clinically relevant benefit in both skeletal and cardiac function in boys and young men with DMD. ^[50]
The results appear well aligned with the regulatory roadmap negotiated in the Type A meeting, setting the stage for a BLA resubmission that could be reviewed over roughly six months once accepted. ^[51]
Analysts remain broadly bullish, with consensus targets more than triple the current share price, though those valuations implicitly assume eventual approval and meaningful uptake. ^[52]
Capricor has cash runway into late 2026, a deepening body of mechanistic and potency data for deramiocel, and an emerging exosome platform that could provide long‑term diversification beyond DMD. ^[53]

At the same time, CAPR remains a speculative small‑cap biotech with substantial regulatory, financing, and litigation risk, and its stock has shown that it can swing double digits in a single session on news, opinion pieces, or social‑media‑driven sentiment. ^[54]

For investors following the story, the next major catalysts will be:

Formal BLA resubmission including HOPE‑3 data;
FDA acceptance and scheduling of a new PDUFA date;
Any additional detailed HOPE‑3 data presentations at scientific or investor meetings;
Early StealthX vaccine data expected in 2026. ^[55]

Disclaimer: This article is for informational and news purposes only and does not constitute investment advice, a recommendation to buy or sell any security, or a substitute for professional financial guidance. Biotech stocks can be highly volatile and speculative; always do your own research and consider consulting a licensed financial advisor before making investment decisions.