deramiocel (CAP-1002) / Capricor, Lonza, Nippon Shinyaku - LARVOL DELTA

A Novel In Vitro Potency Assay Demonstrating the Anti-Fibrotic Mechanism of Action of CDCs in Deramiocel. (PubMed, Biomedicines) - "Importantly, CDCs in Deramiocel lots classified as potent by this assay were shown to exert a clinically meaningful benefit in DMD patients in the HOPE-2 and HOPE-2 OLE studies. This assay represents a mechanistically informative, therapeutically relevant, reproducible, scalable, and regulatory-compliant approach for assessing Deramiocel potency, enabling consistent manufacturing, and facilitating the continued development of Deramiocel as a disease-modifying therapy for DMD."

Journal • Preclinical • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Fibrosis • Genetic Disorders • Immunology • Inflammation • Muscular Dystrophy

Capricor Therapeutics Responds to FDA Posting of Complete Response Letter (CRL) for Deramiocel (Capricor Press Release) - "The Company was not notified in advance that the CRL would be posted, but acknowledges the FDA’s decision to publish the letter, originally received in July 2025. However, the FDA did not release the comprehensive preliminary response that Capricor submitted shortly after receipt of the CRL. This written response provided clarifications to the Agency’s feedback and outlined the Company’s proposed plan to address the outstanding issues...'Looking ahead, we expect topline HOPE-3 data in the fourth quarter of 2025, and our discussions with the FDA have centered on how these data will inform and support the timing of our BLA resubmission.'"

CRL • P3 data: top line • Duchenne Muscular Dystrophy

Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy (Capricor Press Release) - "Capricor Therapeutics...announced that it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for Deramiocel, the Company’s lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy...the FDA stated that it had completed its review of the application but is unable to approve the BLA in its current form, specifically citing that the BLA does not meet the statutory requirement for substantial evidence of effectiveness and the need for additional clinical data...Capricor plans to submit data from the Phase 3 HOPE-3 clinical trial to provide additional evidence of effectiveness from an adequate and well-controlled study...with topline results expected in the third quarter of 2025."

CRL • P3 data: top line • Cardiomyopathy • Duchenne Muscular Dystrophy

Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy (GlobeNewswire) - "FDA has indicated that an Advisory Committee meeting is not required at this time; In-person late-cycle review meeting scheduled for mid-July; Biologics License Application (BLA) remains under Priority Review with PDUFA target action date of August 31, 2025; Four-year data presented at PPMD 2025 Conference demonstrate sustained cardiac function"

FDA event • PDUFA • Duchenne Muscular Dystrophy

Capricor Therapeutics Announces Orphan Drug Designation for Becker Muscular Dystrophy and Regulatory Progress for Duchenne Muscular Dystrophy Program (GlobeNewswire) - "Capricor Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Deramiocel, the company’s lead cell therapy candidate, for the potential treatment of Becker Muscular Dystrophy (BMD). This designation strengthens Capricor’s strategic position as it advances a fully integrated platform targeting the cardiac and skeletal complications of muscular dystrophy and expands the commercial potential of its lead asset."

Orphan drug • Duchenne Muscular Dystrophy

Capricor Therapeutics Announces Completion of Mid-Cycle Review Meeting with FDA on Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy (GlobeNewswire) - "Capricor Therapeutics...announced the completion of a mid-cycle review meeting with the U.S. Food and Drug Administration (FDA) for the Company’s Biologics License Application (BLA) seeking full approval for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. During the meeting, FDA stated that no significant deficiencies have been identified by the Review Committee and that the package is on track for a Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025. The FDA has also confirmed its intent to hold an advisory committee meeting, although an official date has not yet been set....The BLA submission is supported by Capricor’s cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to patient level data from an FDA-funded and published dataset on the natural history of DMD-cardiomyopathy and potential biomarkers of disease progression."

FDA event • PDUFA • Duchenne Muscular Dystrophy

Capricor Therapeutics Announces Positive Data Demonstrating Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular Dystrophy (GlobeNewswire) - P2 | N=13 | HOPE-2-OLE (NCT04428476) | Sponsor: Capricor Inc. | "Capricor Therapeutic...announced positive long-term data from its ongoing HOPE-2 open label extension ('OLE') clinical trial...The data is presented as a late breaking poster at this year’s Muscular Dystrophy Association Clinical and Scientific Conference, which began on March 16 and runs through March 19 in Dallas, Texas....Treatment effect increases year over year – Patients on deramiocel showed a reduction in disease progression, with a mean annual PUL 2.0 decline of 1.8 points in Year 1, 1.2 points in Year 2 and 1.1 points in Year 3; Potential disease-modifying effects – During a 1-year gap of treatment, those originally randomized to deramiocel showed a slower rate of decline (2.8 points per year) compared to untreated patients (3.7 points per year); Favorable safety profile – Deramiocel was well tolerated with no new safety signals identified..."

P2 data • Duchenne Muscular Dystrophy

Capricor Therapeutics Announces FDA Acceptance and Priority Review of its Biologics License Application for Deramiocel to Treat Duchenne Muscular Dystrophy (GlobeNewswire) - "Capricor Therapeutics...today announced the U.S. Food and Drug Administration ('FDA') has accepted for review its Biologics License Application ('BLA') seeking full approval for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy ('DMD') cardiomyopathy. Additionally, the FDA granted the BLA Priority Review with a Prescription Drug User Fee Act ('PDUFA') target action date of August 31, 2025 and at this time, the FDA has not identified any potential review issues....The BLA submission is supported by Capricor’s existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension ('OLE') trials..."

FDA filing • PDUFA • Priority review • Duchenne Muscular Dystrophy

HOPE-3: A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P3 | N=104 | Active, not recruiting | Sponsor: Capricor Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2026 ➔ Dec 2027 | Trial primary completion date: Dec 2024 ➔ Dec 2025

Enrollment closed • Trial completion date • Trial primary completion date • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Atrophy • Muscular Dystrophy

HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) (clinicaltrials.gov) - P2 | N=25 | Completed | Sponsor: Capricor Inc. | Phase classification: P1/2 ➔ P2

Phase classification • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Capricor Therapeutics Completes Submission of Biologics License Application to the U.S. FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy (GlobeNewswire) - "Capricor Therapeutics...today announced the completion of the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking full approval for deramiocel, an investigational cell therapy, to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy...The full submission of the rolling BLA was completed as the Company had previously guided in late December 2024 and is supported by Capricor’s existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to natural history data from an FDA funded and published dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression."

FDA filing • Duchenne Muscular Dystrophy

Capricor Therapeutics Granted Orphan Drug and ATMP Status for Deramiocel by European Medicines Agency (GlobeNewswire) - "Capricor Therapeutics...announced that the European Medicines Agency (EMA) has granted both Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations to its lead asset, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD)....Capricor expects to complete its full BLA submission by the end of 2024."

EMA filing • Orphan drug • Duchenne Muscular Dystrophy • Muscular Dystrophy

HOPE-2-OLE: Open-label Extension of the HOPE-2 Trial (clinicaltrials.gov) - P2 | N=13 | Active, not recruiting | Sponsor: Capricor Inc. | Trial completion date: Mar 2025 ➔ Mar 2026

Trial completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Capricor Therapeutics Announces Initiation of Rolling Submission of Biologics License Application (BLA) with U.S. FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy (GlobeNewswire) - "Capricor Therapeutics...announced today that it has initiated its rolling submission process with the U.S. Food and Drug Administration (FDA) for a Biologics License Application (BLA), seeking full approval for deramiocel to treat all patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy....Capricor plans to complete its rolling BLA submission by the end of 2024."

FDA filing • Cardiovascular • Duchenne Muscular Dystrophy

Regress-HFpEF: Regression of Fibrosis & Reversal of Diastolic Dysfunction in HFpEF Patients Treated With Allogeneic CDCs (clinicaltrials.gov) - P2 | N=27 | Completed | Sponsor: Cedars-Sinai Medical Center | N=40 ➔ 27

Enrollment change • Cardiovascular • Fibrosis • Heart Failure • Immunology

Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE) (clinicaltrials.gov) - P2 | N=8 | Completed | Sponsor: Capricor Inc.

New P2 trial • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Comparative Efficacy and Safety of Stem Cell Therapy with Standard Care in Patients with Muscular Dystrophy: A Systematic Review of Randomized Controlled Trials (ISPOR 2024) - "Two trials had reported the higher numbers of TEAEs with CAP-1002 versus control (RR: 1.14; 95% CI: 0.85-1.52; I 2 : 0%) in DMD. CONCLUSIONS : Considering limited RCTs and low certainty of evidence, suggest little to no difference in clinical improvement and QoL in persons with stem cell therapy among MD and highlights the necessity of larger trials, extended follow-ups, and meaningful outcomes, and meaningful outcomes to establish the potential role of stem cell therapy in MD."

Clinical • Review • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Transplantation • CD133

Long Term Safety and Efficacy of CAP-1002 in late-stage patients with DMD: A New Treatment Approach to Target Skeletal and Cardiac Muscle Pathogenesis (MDA 2024) - "Measurement of cardiac function (left ventricular ejection fraction; EF%) by MRI in the HOPE2-OLE at the 24-month timepoint, showed benefit in EF% in 67% of patients when compared to end of HOPE-2 (~3 years). CAP-1002 maintains a favorable safety profile in HOPE-2-OLE and continues to demonstrate cumulative, clinically meaningful benefit in preserving skeletal and cardiac muscle function by potentially modifying the underlying disease."

Clinical • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Fibrosis

Long Term Safety and Efficacy of CAP-1002 in late-stage patients with DMD: A New Treatment Approach to Target Skeletal and Cardiac Muscle Pathogenesis (MDA 2024) - No abstract available

Clinical • Duchenne Muscular Dystrophy

HOPE-2-OLE: Open-label Extension of the HOPE-2 Trial (clinicaltrials.gov) - P2 | N=13 | Active, not recruiting | Sponsor: Capricor Inc. | Trial completion date: Mar 2024 ➔ Mar 2025

Trial completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

HOPE-3: A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P3 | N=102 | Recruiting | Sponsor: Capricor Inc. | N=68 ➔ 102 | Trial completion date: Dec 2025 ➔ Dec 2026

Enrollment change • Trial completion date • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Atrophy • Muscular Dystrophy

Regress-HFpEF: Regression of Fibrosis & Reversal of Diastolic Dysfunction in HFpEF Patients Treated With Allogeneic CDCs (clinicaltrials.gov) - P2 | N=40 | Completed | Sponsor: Cedars-Sinai Medical Center | Active, not recruiting ➔ Completed | Trial completion date: Feb 2023 ➔ May 2023

Trial completion • Trial completion date • Cardiovascular • Fibrosis • Heart Failure • Immunology

Capricor Therapeutics Reports Second Quarter 2023 Financial Results and Provides Corporate Update (GlobeNewswire) - "Anticipated Milestones and Events: Continue discussions with FDA regarding our pathway towards BLA for CAP-1002 in DMD...Plan to share FDA feedback on the HOPE-3 clinical trial once final minutes are available; Plan to submit data from the ongoing HOPE-2-OLE study at a medical meeting in the fourth quarter of 2023; "

FDA event • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Capricor Therapeutics Reports Second Quarter 2023 Financial Results and Provides Corporate Update (GlobeNewswire) - "Anticipated Milestones and Events: Plan to complete enrollment of the currently designed HOPE-3 trial in the fourth quarter of 2023; Plan to report outcome from interim analysis of HOPE-3 in fourth quarter of 2023; Continue to explore opportunities for additional partnerships outside of the U.S. and Japan to support the potential commercialization of CAP-1002 in DMD."

Enrollment status • Licensing / partnership • P3 data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Capricor Therapeutics to Present 24-Month Results from Ongoing HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy (GlobeNewswire) - "Capricor Therapeutics...today announced that the Company will present 24-month safety and efficacy results from its ongoing HOPE-2 open-label extension (OLE) study with its lead asset, CAP-1002, for the treatment of Duchenne Muscular Dystrophy (DMD) at this year’s Parent Project Muscular Dystrophy (PPMD) Annual Conference."

P2 data • Duchenne Muscular Dystrophy