Capricor Therapeutics’ stock exploded higher on December 3, 2025 after the biotech announced that its pivotal Phase 3 HOPE‑3 trial of Deramiocel (CAP‑1002) in Duchenne muscular dystrophy (DMD) met both its primary and key secondary endpoints. [1]
Below is a deep dive into today’s news, the regulatory backdrop, the company’s finances and pipeline, and how analysts have been thinking about CAPR stock heading into this inflection point.
Key Takeaways
- HOPE‑3 hit its goals: The 106‑patient Phase 3 trial showed a 54% slowing of upper limb decline and 91% preservation of heart function versus placebo, both statistically significant. [2]
- Stock goes vertical: CAPR ripped from roughly $6.36 at Tuesday’s close to about $40 intraday, a gain of more than 500% on the HOPE‑3 topline data. [3]
- Path to approval re‑opened: The new data are intended to answer the FDA’s July 2025 Complete Response Letter (CRL) and support a resubmission of the Deramiocel BLA, with the company and FDA already aligned on HOPE‑3 as the “additional study” the agency asked for. [4]
- Cash runway into late 2026: Capricor ended Q3 2025 with about $98.6 million in cash and securities and guides runway into Q4 2026 under its current plan. [5]
- Analysts were already bullish pre‑data: Prior to today’s surge, Wall Street consensus targets sat in the low‑$20s with “Strong Buy” ratings; Roth Capital, for example, had a $13 target and modeled a 2026 U.S. launch. [6]
Capricor Stock Today: A Biotech Super‑Spike
As of early afternoon on December 3, 2025, Capricor Therapeutics (NASDAQ: CAPR) was trading around $39–40 per share, up from approximately $6.36 at the previous close. That’s a single‑day move of just over 500% based on real‑time quote data. [7]
Pre‑market, Benzinga reported CAPR around $6.93, already up sharply as traders reacted to the early press release detailing HOPE‑3 success. [8] By regular trading hours, the stock had transitioned from “nice pop” to “nosebleed roller coaster,” with intraday prints nearing $40 and multi‑million share volume.
This move is happening against a backdrop of intense retail interest and short‑seller drama:
- A December 1 Stocktwits piece highlighted that CAPR had become a retail favorite ahead of the readout, with message volume at “high” levels and sentiment still bullish despite volatility. [9]
- In late November, controversial ex‑pharma executive Martin Shkreli publicly announced a short position in Capricor, arguing that Deramiocel would fail HOPE‑3. [10]
Today’s data do the opposite of “fail,” which goes a long way to explaining why the stock just went orbital.
What HOPE‑3 Actually Showed
The market isn’t just reacting to a headline; the magnitude and quality of the HOPE‑3 signal are genuinely striking.
Trial design
- Study: HOPE‑3 Phase 3, randomized, double‑blind, placebo‑controlled
- Population: Boys and young men with DMD (largely non‑ambulatory), average age ~15 years
- Size: 106 participants across 20 U.S. clinical sites
- Dosing: 150 million Deramiocel cells vs placebo, IV infusion every 3 months for 12 months (4 doses)
- Background therapy: Nearly all patients on standard cardiac medications; all on stable corticosteroids; >75% had cardiomyopathy at baseline. [11]
Primary and key secondary endpoints
According to Capricor’s topline release: [12]
- Primary endpoint – Performance of Upper Limb (PUL v2.0) total score
- 54% slowing of disease progression vs placebo
- p = 0.029
- Key secondary endpoint – Left Ventricular Ejection Fraction (LVEF %)
- 91% preservation of cardiac function vs placebo
- p = 0.041
Both endpoints were part of a type‑1‑error‑controlled hierarchy, and Capricor reports that all controlled secondary endpoints also achieved statistical significance. [13]
Investigators did not mince words. Craig McDonald, M.D., the national principal investigator, described a ~54% slowing of skeletal muscle decline as “extraordinary in Duchenne,” especially in severely affected, mostly non‑ambulatory patients, linking it directly to preservation of independence and quality of life. [14] Cardiologist Jonathan Soslow, M.D., called the cardiac data a “significant advance” in DMD cardiomyopathy, where heart failure is the leading cause of death. [15]
Safety
The company says Deramiocel maintained a favorable safety and tolerability profile, consistent with prior HOPE‑2 and HOPE‑2 OLE experience, with no new safety signals emerging in HOPE‑3. [16]
In plain language:
- The trial was well‑controlled and reasonably sized for a rare disease.
- It hit both the muscle function and heart function targets the FDA asked for.
- The effect size is big enough that clinicians are excited, not just mildly pleased.
For a DMD program that just took a regulatory punch in July, this is exactly the kind of data package investors were hoping for.
From FDA Rejection to a Credible Path Back
This morning’s good news only makes sense in the context of a tough 2025.
The July 2025 Complete Response Letter
In July, the U.S. FDA issued a Complete Response Letter (CRL) rejecting Capricor’s original Biologics License Application (BLA) for Deramiocel in DMD cardiomyopathy. The agency said the submission did not provide “substantial evidence of effectiveness” and explicitly requested additional clinical data from an adequate and well‑controlled study. [17]
Translation: “We’re not saying ‘never,’ but we’re not convinced yet; bring more robust data.”
Type A meeting and regulatory reset
Capricor met with the FDA for a Type A meeting, a formal post‑CRL discussion, and published a detailed regulatory update on September 25, 2025. Key points from that update: [18]
- The FDA agreed that HOPE‑3 will serve as the “additional study” requested in the CRL.
- The current BLA can be salvaged: HOPE‑3 data will be submitted within the existing application rather than starting from scratch.
- PUL v2.0 remains the primary endpoint; LVEF is a key secondary endpoint that Capricor will ask to include in the label.
- The FDA emphasized in its meeting minutes that it would “exercise further regulatory flexibility” in reviewing HOPE‑3 data.
The company reiterated today that it plans to submit its CRL response incorporating HOPE‑3 results, completing the data package needed for the FDA to take another formal look at Deramiocel. [19]
Designations and incentives
Deramiocel comes with a full backpack of regulatory designations: [20]
- Orphan Drug (US and EU)
- RMAT (Regenerative Medicine Advanced Therapy) in the U.S.
- ATMP (Advanced Therapy Medicinal Product) in Europe
- Rare Pediatric Disease Designation, potentially qualifying Capricor for a Priority Review Voucher if approved.
Those designations don’t guarantee approval, but they do grease the regulatory rails and improve the economics if the therapy makes it to market.
Financial Position: Cash Into Late 2026, Still Pre‑Revenue
Capricor is pre‑revenue in 2025 and relies on partnership funds and its balance sheet to power R&D.
From the Q3 2025 report: [21]
- Cash, cash equivalents and marketable securities:
- ~$98.6 million as of September 30, 2025 (down from ~$151.5M at year‑end 2024).
- Q3 2025 revenue:
- $0 (prior revenue was recognition of milestone payments from Nippon Shinyaku, fully recognized by end of 2024).
- Operating expenses (Q3 2025):
- ~$26.3 million vs ~$15.3 million in Q3 2024.
- Net loss (Q3 2025):
- ~$24.6 million, or $0.54 per share.
- Runway guidance:
- Management believes current cash should fund operations into the fourth quarter of 2026, excluding any new milestones or strategic spending beyond its base plan.
These numbers fit the profile of a small‑cap, late‑stage biotech: high burn, no product revenue yet, but enough runway to get through the next major regulatory milestones if things stay on schedule. Ts2 Tech
Commercial partner: Nippon Shinyaku / NS Pharma
Capricor has already signed an exclusive commercialization and distribution agreement with Nippon Shinyaku (NS Pharma) covering Deramiocel for DMD in the United States and Japan, subject to regulatory approval. [22]
This matters because:
- It offloads some commercial execution risk.
- It creates the possibility of additional milestone and royalty payments if Deramiocel is approved and launched.
Beyond Deramiocel: The StealthX™ Exosome Platform
Deramiocel is the near‑term value driver, but Capricor is also quietly building an exosome‑based therapeutics platform that could matter a lot if the company survives the current DMD gauntlet.
Project NextGen StealthX vaccine trial
In August 2025, Capricor announced that the first subjects were dosed in a Phase 1 trial of its StealthX exosome‑based vaccine, funded by the U.S. National Institute of Allergy and Infectious Diseases (NIAID) under Project NextGen. [23]
- The vaccine uses engineered exosomes displaying SARS‑CoV‑2 proteins, as an alternative to mRNA approaches.
- The Phase 1 study explores multiple dose levels and could expand to multivalent designs pending FDA feedback.
- Topline data are expected in Q1 2026, subject to NIAID timelines. [24]
Scalable exosome loading data
On November 24, 2025, Capricor presented data at the AAEV 2025 meeting showing a scalable framework for loading therapeutic siRNA and PMO oligonucleotides into exosomes using optimized electroporation and integrated scale‑up/scale‑out strategies. [25]
Key takeaways:
- Exosomes from 293F cells were efficiently loaded with siRNA and PMO cargo.
- Large‑scale manufacturing with comparable loading efficiency looks feasible.
Management explicitly positions this as part of a strategy to evolve exosomes from a “nice side effect” of cell therapy into a standalone delivery platform for vaccines and nucleic‑acid therapeutics. [26]
For equity holders, this is optionality: the stock still lives or dies on Deramiocel in the near term, but StealthX could become a second act.
How Wall Street Has Been Valuing CAPR
Before today’s moonshot, analysts were already fairly enthusiastic about Capricor — but their models were built around a single‑digit share price, not a $40 rocket ship.
Roth Capital: “Shorters won’t take down Capricor”
In a November 26, 2025 note, Roth Capital Markets analyst Boobalan Pachaiyappan reiterated a “Buy” rating and $13 price target on CAPR after a sharp drop triggered by a short‑seller report. [27]
Highlights from that analysis:
- The sell‑off was seen as disconnected from Deramiocel’s existing data and company disclosures.
- Phase 2b data previously showed a 107% slowing of cardiac disease progression over 12 months, which the analyst thought could justify regulatory flexibility even if skeletal muscle endpoints proved tougher. [28]
- Roth’s DCF model assumed:
- ~55% probability of approval,
- 12% discount rate,
- 0% terminal growth,
- U.S.‑only revenues (no ex‑U.S. modeled).
- The model implied an equity value of about $581 million, supporting the $13 target, and assumed a U.S. launch in 4Q 2026 with market exclusivity to 2039. [29]
Today’s move obviously blows past that target; if Deramiocel’s risk profile has just changed for the better, street models will need a full re‑write.
Consensus targets before HOPE‑3
A December 3 feature from TechStock² aggregated several data providers and painted the pre‑readout consensus picture like this: Ts2 Tech
- StockAnalysis.com:
- ~6 covering analysts
- Rating: “Strong Buy”
- Average 12‑month target: about $23.17
- Range: $13–30 per share
- MarketBeat:
- ~9 analysts
- Average target: roughly $22.38, with the same $13–30 range
- TipRanks & Zacks:
- Consensus targets in the low‑$20s, with no sell ratings reported.
Those targets implied ~250–260% upside from late‑November prices in the mid‑$6 range. Now that the stock itself is hovering around $40, those “upside” numbers are obsolete; we should expect a round of target hikes, downgrades, or both as analysts digest HOPE‑3.
Independent analysis
A September 29 Seeking Alpha deep dive framed Capricor as a high‑risk, high‑reward DMD cardiomyopathy play with: [30]
- A credible unmet‑need niche (non‑ambulatory DMD cardiomyopathy).
- Strong regulatory designations and a supportive partner (Nippon Shinyaku).
- A reasonable cash position — but meaningful burn and single‑asset concentration risk.
That article — like many others — treated HOPE‑3 as the make‑or‑break event for the stock. Today, that coin landed on “make,” at least on the clinical front.
Volatility, Shorts and Retail Mania
Heading into today:
- Stocktwits reported that retail sentiment had cooled slightly from “extremely bullish” to “bullish”, but message volume remained high as traders waited for the HOPE‑3 readout. [31]
- Shkreli’s short thesis — arguing cell therapy limitations, weak HOPE‑2 data, and a likely HOPE‑3 failure — had helped drive the stock down double digits in late November. [32]
TechStock² noted a “retail frenzy” and elevated put‑option activity around CAPR, as bulls and bears essentially gambled on a binary outcome. Ts2 Tech
Now that HOPE‑3 has clearly succeeded on its prespecified endpoints, the immediate focus shifts from “Will the trial work?” to:
- How will the FDA interpret clinical meaningfulness?
- How aggressively will payers reimburse a cell therapy in DMD?
- Can Capricor and Nippon Shinyaku execute on commercialization and manufacturing at scale?
The stock’s intraday behavior — and the squeeze potential for short positions — suggests volatility is not going away anytime soon.
Key Risks for CAPR Investors
Even after a day like this, the Capricor story is not suddenly low‑risk. Major risk buckets include: Ts2 Tech+1
- Regulatory risk
- The FDA has already issued one CRL; approval is not automatic just because endpoints were met. The agency will scrutinize whether the magnitude and durability of benefit truly justify approval and potential label claims across both skeletal and cardiac function.
- Single‑asset dependence
- In the near term, Capricor is heavily reliant on Deramiocel. A negative FDA decision, or a demand for yet another large trial, would be brutal for the stock.
- Financing and dilution
- Management says cash lasts into late 2026, but commercial build‑out, post‑approval studies, or delays could force equity raises.
- Competition in DMD
- DMD is a crowded field, with players like Sarepta and others pushing gene therapies and exon‑skipping drugs. Deramiocel’s differentiation — mutation‑agnostic and cardiomyopathy‑focused — will have to be clearly communicated and clinically compelling. [33]
- Litigation and sentiment overhangs
- Several securities law firms have launched investigations around the July CRL and prior disclosures, adding headline risk even if nothing ultimately comes of them. Ts2 Tech
- Manufacturing and logistics for cell therapy
- Scaling an allogeneic cell product with consistent potency, quality and supply is non‑trivial, even with prior inspections completed. [34]
This is still a classic “high‑beta biotech” situation — just one with a much stronger hand than it had 24 hours ago.
Bottom Line
December 3, 2025 is a genuine pivot point for Capricor Therapeutics stock:
- Clinically, HOPE‑3 did what it needed to do, delivering statistically and clinically meaningful improvements in both upper limb and cardiac function in a tough‑to‑treat DMD population. [35]
- Regulatorily, the data appear well aligned with the Type A meeting roadmap, giving Capricor a realistic shot at turning a July CRL into a 2026 approval attempt. [36]
- Financially and strategically, the company has runway into late 2026, a commercial partner in Nippon Shinyaku, and an emerging exosome platform that could eventually diversify the story beyond DMD. [37]
At the same time, CAPR is now trading in a very different price neighborhood than the one used in recent analyst models. Whether today’s 500%+ move proves to be the start of a re‑rating or just the latest chapter in biotech’s volatility anthology will depend on the FDA’s next steps, the strength of detailed HOPE‑3 data, and Capricor’s ability to execute.
References
1. www.globenewswire.com, 2. www.globenewswire.com, 3. www.investing.com, 4. www.capricor.com, 5. www.capricor.com, 6. www.cantechletter.com, 7. www.investing.com, 8. www.benzinga.com, 9. stocktwits.com, 10. stocktwits.com, 11. www.globenewswire.com, 12. www.globenewswire.com, 13. www.globenewswire.com, 14. www.globenewswire.com, 15. www.globenewswire.com, 16. www.globenewswire.com, 17. stocktwits.com, 18. www.capricor.com, 19. www.globenewswire.com, 20. www.globenewswire.com, 21. www.capricor.com, 22. www.capricor.com, 23. www.capricor.com, 24. www.capricor.com, 25. www.nasdaq.com, 26. www.nasdaq.com, 27. www.cantechletter.com, 28. www.cantechletter.com, 29. www.cantechletter.com, 30. seekingalpha.com, 31. stocktwits.com, 32. stocktwits.com, 33. www.parentprojectmd.org, 34. www.nasdaq.com, 35. www.globenewswire.com, 36. www.capricor.com, 37. www.capricor.com
