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

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: May 2026 ➔ Mar 2027

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

Outstanding Research Award in Pediatric Cardiology: Cardiac Effects of Deramiocel in Duchenne Muscular Dystrophy: Randomized Placebo Controlled Phase-3 Trial Results and Natural History Comparison (AHA 2026) - "Abstract is embargoed at this time."

Clinical • P3 data • Cardiovascular • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pediatrics

HOPE-3: A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P3 | N=106 | Active, not recruiting | Sponsor: Capricor Inc. | Trial completion date: Dec 2026 ➔ Mar 2027

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

Capricor Therapeutics…announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) target action date for its Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), from August 22, 2026 to November 22, 2026 (GlobeNewswire) - "As part of its ongoing discussions with the FDA following the July 2026 Advisory Committee meeting, Capricor submitted an amendment to the BLA that includes 24-month open-label extension data from its pivotal Phase 3 HOPE-3 study and additional robustness analyses, with a request that the FDA review the existing and new data in support of a refined proposed indication focused on upper limb function, the primary endpoint of HOPE-3....The FDA has classified the submission as a major amendment and extended the PDUFA target action date by three months to allow additional time to review the information."

FDA filing • PDUFA • Duchenne Muscular Dystrophy

Commercial and Manufacturing Readiness (GlobeNewswire) - "The Company's GMP manufacturing facility in San Diego is operational and positioned to support an initial commercial launch, if Deramiocel is approved. The second-floor expansion is targeted for full validation and FDA inspection in 2027, as planned."

Commercial • Duchenne Muscular Dystrophy

FDA Advisory Committee Outcome (GlobeNewswire) - "On July 29, 2026, the Cellular, Tissue and Gene Therapies Advisory Committee voted 3 in favor and 9 against on whether available evidence provides substantial evidence of effectiveness of Deramiocel for the treatment of cardiomyopathy in patients with DMD. The Committee was not asked to vote on the HOPE-3 primary endpoint or on overall benefit-risk, and in a separate discussion of upper limb function its feedback was directionally supportive of the HOPE-3 clinical evidence. The recommendation is advisory and non-binding....Capricor is continuing to work with the FDA on the review of its BLA. The Company plans to provide a regulatory update on its conference call today and will provide further updates as they become available."

FDA event • Duchenne Muscular Dystrophy

Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial. (PubMed, Lancet) - P3 | "Deramiocel safely slows disease progression in advanced DMD, preserving skeletal muscle function. Administered quarterly in a simple outpatient regimen, deramiocel is a promising treatment for DMD, agnostic to the precise underlying genetic lesion."

Journal • P3 data • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Myositis

Capricor Therapeutics…shares dropped 40% on Monday after the U.S. Food and Drug Administration published briefing documents ahead of an upcoming advisory committee meeting to review the company's lead cell therapy candidate (Yahoo Finance) - "In its briefing materials, the FDA reiterated that substantial evidence of effectiveness generally requires at least two adequate and well-controlled clinical studies, with each trial independently demonstrating convincing results....Capricor's original BLA relied on data from the Phase 2 HOPE-2 trial and its open-label extension. According to the FDA, those studies did not demonstrate evidence of effectiveness in improving skeletal or cardiac function, including measurements based on left ventricular ejection fraction and other cardiac endpoints."

FDA event • Stock price • Duchenne Muscular Dystrophy

Capricor Therapeutics…reported positive five-year data for its Duchenne muscular dystrophy cell therapy, deramiocel, and confirmed that the U.S. Food and Drug Administration will hold an advisory committee meeting to review its biologics license application on July 29. (Asianet Newsable) - "The company added that the biologics license application for deramiocel remains on track for the Prescription Drug User Fee Act target action date of Aug. 22, 2026...Deramiocel continued to slow disease progression over five years, with patients showing less decline in upper limb function than expected...The therapy also maintained stable cardiac function throughout the study and continued to demonstrate a favorable safety profile, with no new safety concerns reported after more than 800 intravenous infusions."

P3 data • Duchenne Muscular Dystrophy

Capricor Therapeutics Announces Late-Breaking Presentation of HOPE-3 Phase 3 Results at the American Academy of Neurology 2026 Annual Meeting (Capricor Press Release) - "'No therapeutic has ever delivered a statistically and clinically meaningful difference in the upper limb functional primary endpoint of Performance of the Upper Limb (PUL v2.0),' said Dr. Aravindhan Veerapandiyan, Associate Professor and Director of the Comprehensive Neuromuscular Program at Arkansas Children's Hospital. 'These findings were further supported by an alternative measure of upper limb function, specifically video recordings of tasks performed at home (the Duchenne Video Assessment, or DVA), which showed meaningful slowing of disease progression in the ability to self-feed, a function central to patient independence. These data reinforce the potential of Deramiocel to make a meaningful difference in the lives of those living with Duchenne.'"

P3 data • Duchenne Muscular Dystrophy

Capricor Therapeutics…announced that the Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) of the U.S. Food and Drug Administration (FDA) is planning to convene an advisory committee meeting to discuss the Company’s Biologics License Application (BLA) seeking approval of Deramiocel, an investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD) (GlobeNewswire) - "The BLA is supported by the Company's Phase 2 HOPE-2 trial and long-term outcomes from the HOPE-2-OLE trial, as well as positive results from the Phase 3 HOPE-3 trial, which achieved statistical significance on its primary endpoint (PUL v2.0), the key secondary cardiac endpoint (LVEF), and all other Type I error-controlled secondary endpoints. The date for the Advisory Committee meeting is July 29, 2026..."

FDA event • Duchenne Muscular Dystrophy

Phase-3 efficacy in Duchenne muscular dystrophy in HOPE-3 confirms skeletal and cardiac muscle functional benefit and supports the clinical translation of anti-fibrotic activity for deramiocel, an allogeneic cardiac derived cell therapy (ASGCT 2026) - "Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet 399, 1049-1058 (2022)."

Clinical • P3 data • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Fibrosis • Genetic Disorders • Immunology • Muscular Dystrophy

Deramiocel Significantly Slows Upper Limb Functional Decline in Duchenne Muscular Dystrophy: Skeletal Muscle Outcomes from the Phase 3 HOPE-3 Trial (AAN 2026) - "Conclusions Treatment with deramiocel resulted in a clinically meaningful and statistically significant slowing of upper limb disease progression over 12 months. The magnitude of effect, concordance across standardized functional scales, and home-based real-world video assessments support deramiocel as a therapy with the potential to modify the course of skeletal muscle decline and preserve independent function in patients with DMD."

Late-breaking abstract • P3 data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Musculoskeletal Diseases

Capricor Therapeutics Announces Establishment of New PDUFA Date for Deramiocel BLA (GlobeNewswire) - "Capricor Therapeutics...announced that the U.S. Food and Drug Administration ('FDA') has lifted the previously issued Complete Response Letter and resumed review of its Biologics License Application ('BLA') seeking full approval of Deramiocel, an investigational cell therapy, for the treatment of Duchenne muscular dystrophy ('DMD') cardiomyopathy. The submission has been classified as a Class 2 resubmission, with a Prescription Drug User Fee Act ('PDUFA') target action date of August 22, 2026...Capricor also expects to be eligible to receive a Priority Review Voucher ('PRV') upon potential approval of Deramiocel."

FDA filing • PDUFA • Duchenne Muscular Dystrophy

Capricor Therapeutics…announced that the U.S. Food and Drug Administration ('FDA') has lifted the previously issued Complete Response Letter and resumed review of its Biologics License Application ('BLA') seeking full approval of Deramiocel, an investigational cell therapy, for the treatment of Duchenne muscular dystrophy ('DMD') cardiomyopathy (GlobeNewswire) - "The Company received a Complete Response Letter ('CRL') from the FDA in July 2025. Following submission of data and supporting documentation from the HOPE-3 clinical trial, the FDA resumed review of the application and assigned a PDUFA target action date of August 22, 2026....Capricor also expects to be eligible to receive a Priority Review Voucher ('PRV') upon potential approval of Deramiocel."

PDUFA • Cardiomyopathy • Duchenne Muscular Dystrophy

Capricor Therapeutics…provided a regulatory update regarding its Biologics License Application (BLA) for Deramiocel, the Company’s investigational first-in-class cell therapy for the treatment of Duchenne muscular dystrophy (DMD). (The Manila Times) - "As previously disclosed, the Company provided topline results from its Phase 3 HOPE-3 clinical study to the U.S. Food and Drug Administration (FDA) in late 2025. Following its review of these data, the FDA has formally requested the full HOPE-3 clinical study report (CSR) and supporting data to address the Complete Response Letter (CRL). The FDA did not request any additional clinical studies or new patient data as part of this request. Preparation of the HOPE-3 CSR is well underway, and the Company plans to submit the requested materials to the FDA in February 2026."

FDA event • 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=106 | Active, not recruiting | Sponsor: Capricor Inc. | Trial completion date: Dec 2027 ➔ Dec 2026 | Trial primary completion date: Dec 2025 ➔ Jun 2025

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

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