Capricor Therapeutics, Inc. engages in the discovery, development and commercialization of biological therapies for the treatment of cardiac and other serious medical conditions. Its product candidate consists of CAP-1002, Cenderitide, Exosomes, CAP-1001, CU-NP and CSps. The company was founded on June 17, 1996 and is headquartered in San Diego, CA.
相关临床试验
11
2 进行中
药物批准
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监管机构
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成立时间
2005
进行中（未招募）
2
18.2%
已完成
6
54.5%
No Longer Available
1
9.1%
终止
1
9.1%
Unknown
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9.1%
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- Capricor Therapeutics will release its second quarter 2026 financial results after market close on Thursday, August 13, 2026. - Management will host a conference call and webcast at 4:30 p.m. ET the same day to discuss results and provide a recent corporate update. - The company's lead candidate, Deramiocel, is an allogeneic cardiac-derived cell therapy in late-stage development for Duchenne muscular dystrophy (DMD). - Capricor is also advancing its StealthX exosome platform for targeted delivery of oligonucleotides, proteins, and small-molecule therapeutics.
- FDA Advisory Committee meetings remain pivotal regulatory catalysts that can trigger sharp biotech stock swings, as illustrated by Moderna's 9.6% intraday gain following a unanimous positive vote for its influenza vaccine mRNA-1010. - The FDA is not bound by AdCom votes; it has approved drugs despite negative panel recommendations (e.g., Zynquista in 2024) and rejected drugs after positive votes (e.g., Teplizumab in 2021). - The recent Capricor Therapeutics AdCom for deramiocel in Duchenne muscular dystrophy highlighted the need for temporary voting members with specialized disease expertise on advisory panels. - Experts argue FDA should reform AdComs by aggressively recruiting disease-specific specialists, refining conflict-of-interest policies, and asking committees to vote on specific scientific questions rather than a single yes-or-no on approvability.
- FDA briefing documents released ahead of an advisory committee meeting concluded that Capricor's deramiocel data did not provide "substantial evidence of effectiveness" for Duchenne muscular dystrophy. - Agency staff noted that approval generally requires at least two adequate and well-controlled studies, while deramiocel's BLA rests on a single pivotal trial. - The FDA raised concerns that Capricor made a "litany of changes" to its statistical analysis plan after the study was completed, potentially to generate a positive outcome. - Capricor shares fell more than 60% following the news, and Block & Leviton has launched a securities fraud investigation into the company's prior representations about deramiocel's regulatory path.
- A Complete Response Letter (CRL) is the FDA's formal notice that a drug application cannot be approved in its current form, citing deficiencies in safety, efficacy, manufacturing, or labeling. - CRLs can trigger severe market reactions, with single-day stock declines ranging from 2% (AbbVie) to 75% (Aldeyra Therapeutics), depending on the nature and severity of the deficiencies. - Companies can pursue Class 1 resubmissions for minor fixes (approximately two-month review) or Class 2 resubmissions for substantial changes (approximately six-month review), with successful resubmissions enabling significant stock recoveries. - Fortress Biotech's CUTX-101 and Capricor Therapeutics' deramiocel demonstrate that CRLs are not terminal, as both programs advanced toward approval following resubmission.
- The Duchenne Muscular Dystrophy market reached approximately $2.15 billion in 2023 and is expected to grow significantly due to increased drug uptake and anticipated gene therapy launches. - Over 75 companies are actively developing pipeline therapies for DMD, with recent FDA designations including Atossa Therapeutics' (Z)-Endoxifen receiving Rare Pediatric Disease designation. - Capricor Therapeutics announced positive Phase 3 HOPE-3 trial results for Deramiocel, while the FDA accepted their BLA for review with Priority Review designation. - Current approved treatments include EMFLAZA, VYONDYS 53, EXONDYS 51, AMONDYS 45, VILTEPSO, and gene therapy ELEVIDYS in the US, with AGAMREE launched in Germany in 2024.
- Capricor Therapeutics presented new data at AAEV 2025 demonstrating a scalable framework for loading therapeutic siRNAs and PMOs into exosomes using optimized electroporation conditions. - The company successfully demonstrated that both scale-up and scale-out strategies produce comparable loading efficiencies to standard small-volume electroporation methods. - The integrated manufacturing approach enables production of significantly larger batches of therapeutic exosomes, providing a feasible pathway for clinically relevant quantities. - This advancement supports the versatility of Capricor's exosome technology platform and its potential application across a broad range of diseases.
- Multiple biotechnology companies including UniQure, Biohaven, and Capricor have faced unexpected FDA reversals on previously agreed-upon evidence requirements for drug approvals since July 2024. - UniQure's Huntington's disease gene therapy AMT-130, which showed 75% disease slowing in pivotal trials, had its biologics license application timeline thrown into uncertainty after FDA changed its stance on acceptable evidence. - The regulatory instability coincides with significant leadership changes at FDA, with the Center for Drug Evaluation and Research changing hands four times since early 2025 and philosophical differences emerging between current and previous FDA leadership. - Industry analysts suggest these reversals may signal a shift toward stricter regulatory standards, requiring more compelling risk-benefit profiles despite companies having aligned with FDA on trial designs and statistical analyses.
- Capricor Therapeutics published a peer-reviewed study in Biomedicines describing a novel in-vitro potency assay that characterizes the anti-fibrotic mechanism of action of Deramiocel for Duchenne muscular dystrophy treatment. - The study demonstrated that cardiosphere-derived cells suppress collagen I and III gene expression in primary human fibroblasts through secreted exosomes and soluble factors, with consistent results across over 100 manufacturing lots. - The validated assay enhances quality control and product consistency for Deramiocel as it advances through late-stage development, with Phase 3 HOPE-3 trial topline data expected in mid-fourth quarter 2025. - Deramiocel has received multiple regulatory designations including Orphan Drug status from FDA and EMA, and Regenerative Medicine Advanced Therapy designation, supporting its potential for treating this rare neuromuscular disorder.
- The FDA initially encouraged Capricor Therapeutics to file for approval of their cardiac-derived cell therapy for Duchenne muscular dystrophy after being impressed with clinical data. - Despite statistically significant heart data, the FDA rejected the application this summer due to changes in leadership and data review processes. - A 22-year-old patient who has shown remarkable improvement on the quarterly treatment is now advocating directly to Congress and the FDA for continued access. - The family warns that boys with DMD are experiencing irreparable heart damage while waiting for regulatory approval of this potentially life-changing therapy.
- The FDA under President Trump is abandoning its decades-old policy of convening outside expert panels to review individual drug applications, with officials claiming these meetings are redundant and time-consuming. - George Tidmarsh, head of the FDA's Center for Drug Evaluation and Research, stated the agency "would like to get away" from assembling expert panels because "I don't think they're needed" for specific drug evaluations. - The FDA has already reduced advisory committee meetings from 22 in the same period last year to only seven since Trump's return, while beginning to publish complete response letters as an alternative transparency measure. - Former FDA officials and academics strongly oppose this shift, arguing that expert panels provide crucial independent oversight and public transparency that cannot be replaced by published rejection letters alone.
