CAPRICOR THERAPEUTICS, INC.
CAPR evidence brief
What we can confirm
- BioSniper Score:
- 4.5 / 10
- Market Cap:
- $502.3M
- Lead Asset:
- Deramiocel (Phase 2)
What it means
What to watch
Prepared research question
What matters most for CAPR—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · CAPR
Capricor Therapeutics is a biotechnology company developing cell and exosome-based therapeutics, with its lead candidate Deramiocel for Duchenne muscular dystrophy under FDA BLA review and a PDUFA date of November 22, 2026.
Key risk: Binary PDUFA outcome
BioSniper Score
Based on 5 of 6 signals
Financial data as of Jun 30, 2026.
Tracking 1 pipeline candidate · 2 clinical trials · 1 catalyst for CAPRICOR THERAPEUTICS, INC..
Similar companies
Capricor Therapeutics is a biotechnology company developing cell and exosome-based therapeutics, with its lead candidate Deramiocel for Duchenne muscular dystrophy under FDA BLA review and a PDUFA date of November 22, 2026.
- Deramiocel BLA for Duchenne muscular dystrophy is under active FDA review with a PDUFA date of November 22, 2026 [catalyst].
- HOPE-3 Phase 3 trial for Deramiocel achieved its primary endpoint of upper limb function at p=0.029, with results published in The Lancet [news · published 2026-08-13].
- Cash, cash equivalents and marketable securities were approximately $238 million as of June 30, 2026 [news · published 2026-08-13]; latest reported financials (as of 2026-06-30) show cash & equivalents of $20.9M, net income -$40.7M, and shareholders' equity $246.2M [sec].
- Deramiocel has received Orphan Drug, RMAT, and Rare Pediatric Disease designations from the FDA [pipeline].
- Capricor is also developing an exosome-based platform, including a StealthX™ vaccine under Phase 1 study by NIAID as part of Project NextGen [sec_mda · as of 2026-05-13].
What works
- Deramiocel achieved a statistically significant primary endpoint in its Phase 3 HOPE-3 trial (p=0.029) [news · published 2026-08-13].
- The company has a substantial cash position of approximately $238 million as of June 30, 2026, providing runway through the PDUFA decision [news · published 2026-08-13].
- Deramiocel benefits from multiple FDA designations (Orphan Drug, RMAT, Rare Pediatric Disease) that may expedite development and review [pipeline].
- Capricor has a commercialization partner, Nippon Shinyaku Co., Ltd., as exclusive distributor for DMD in the U.S. and Japan [pipeline].
What to weigh
- The company has not yet generated any product revenue and reported a net loss of -$40.7M for the six months ended June 30, 2026 [sec].
- Deramiocel's approval is uncertain; the PDUFA date of November 22, 2026 is a binary catalyst that could significantly impact the company's future [news · published 2026-09-13].
- The exosome platform is in early-stage development, with only $1.3 million spent in the first half of 2026, and the StealthX™ vaccine showed limited neutralizing antibody responses in early analyses [sec_mda · as of 2026-08-14].
- The company faces significant competition from exon-skipping oligonucleotides and gene therapies in the DMD space [pipeline].
From the filings
Quoted directly from source documents.
Deramiocel BLA is under active FDA review with a PDUFA date of November 22, 2026.
“Deramiocel Biologics License Application (BLA) under active FDA review”
News · August 13, 2026
Phase 3 HOPE-3 trial achieved primary endpoint of upper limb function at p=0.029.
“HOPE-3 Phase 3 results published in The Lancet; primary endpoint of upper limb function achieved at p=0.029”
News · August 13, 2026
Cash position of approximately $238 million as of June 30, 2026.
“Cash, cash equivalents and marketable securities of approximately $238 million as of June 30, 2026”
News · August 13, 2026
Deramiocel has Orphan Drug, RMAT, and Rare Pediatric Disease designations.
“Regulatory Status: Orphan Drug, RMAT, Rare Pediatric Disease”
Catalyst timeline
- 2026-11-22 · PDUFA · Deramiocel — FDA decision on the Deramiocel BLA for Duchenne muscular dystrophy; approval would enable commercial launch, while rejection would require additional development.
Risks & what to watch (4)›
Key risks
- Binary PDUFA outcome — Deramiocel's approval decision on Nov 22, 2026 is a binary catalyst; rejection would severely impact the company [news · published 2026-09-13].
- No product revenue — Capricor has no approved products and reported a net loss of -$40.7M for H1 2026 [sec].
- Early-stage exosome platform — Exosome platform is preclinical/early clinical; StealthX vaccine showed limited neutralizing antibody responses [sec_mda · as of 2026-08-14].
- Competitive DMD landscape — Deramiocel faces competition from exon-skipping oligonucleotides and gene therapies [pipeline].
What to watch
- FDA decision on Deramiocel BLA by PDUFA date November 22, 2026 [catalyst].
- Final results from NIAID's Phase 1 study of the StealthX™ vaccine, expected later in 2026 [sec_mda · as of 2026-08-14].
- Company's financial outlook and capital allocation decisions following regulatory clarity on Deramiocel [sec_mda · as of 2026-08-14].
- Potential commercial launch preparations and partnership activities with Nippon Shinyaku if Deramiocel is approved [pipeline].
Sources: Catalysts · News · Pipeline · SEC MD&A · Generated October 4, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 5 of 6 signals · derived from public data
Cash runway data unavailable
D/E 0.50 · 0% dilution
Lead: Phase 2
1 candidate
1 upcoming, next in ~39d
0 buys · 8 sells (180d)
Catalysts
Pipeline
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
177
Total value held
$1.03B
| Holder | Shares | Value |
|---|---|---|
| suvretta capital management, llc | 3,818,600 | $116.1M |
| blackrock, inc. | 3,421,815 | $104.0M |
| tang capital management llc | 3,399,900 | $103.4M |
| state street corp | 2,181,901 | $66.3M |
| vanguard capital management llc | 2,027,732 | $61.6M |
| ra capital management, l.p. | 1,507,844 | $45.8M |
| geode capital management, llc | 1,118,326 | $34.0M |
| goldman sachs group inc | 911,976 | $27.7M |
| jones hill capital lp | 909,207 | $27.6M |
| point72 asset management, l.p. | 821,179 | $25.0M |
Showing top 10 of 177 institutional holders of CAPRICOR THERAPEUTICS, INC.. Open the full 13F history after sign-in.
Short Interest
as of May 20, 2026- % of float
- 20.1%
- Days to cover
- 10.7
- Shares short
- 10,515,168
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
CAPR short interestGovernance
Frequently asked questions
What is the AI investment thesis for CAPRICOR THERAPEUTICS, INC.?
Capricor Therapeutics is a biotechnology company developing cell and exosome-based therapeutics, with its lead candidate Deramiocel for Duchenne muscular dystrophy under FDA BLA review and a PDUFA date of November 22, 2026.
What is CAPRICOR THERAPEUTICS, INC.'s lead drug candidate?
CAPRICOR THERAPEUTICS, INC.'s most advanced tracked candidate is Deramiocel (Phase 2) for Muscular Dystrophies; Duchenne Muscular Dystrophy.
What is CAPRICOR THERAPEUTICS, INC.'s next catalyst?
CAPRICOR THERAPEUTICS, INC.'s next tracked catalyst is a PDUFA event — "PDUFA: 0001104659-26-100071" — expected November 22, 2026.
Who is the CEO of CAPRICOR THERAPEUTICS, INC.?
Dr. Linda Marbán Ph.D. is the chief executive officer of CAPRICOR THERAPEUTICS, INC. (CAPR).
Where is CAPRICOR THERAPEUTICS, INC. headquartered?
CAPRICOR THERAPEUTICS, INC. (CAPR) is headquartered in San Diego, CA, United States.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.