RegulationRare Pediatric Disease (US), Orphan Drug (EU), Regenerative Medicine Advanced Therapy (US), Advanced Therapy Medicinal Products (EU), Orphan Drug (US)

Clinical Trials associated with Deramiocel

NCT05126758 / RecruitingPhase 3

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy

HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during the first 12-months of the study. All participants will be eligible to receive 4 doses of CAP-1002 for an additional 12 months as part of an open-label extended assessment period.

Start Date22 Jun 2022

Sponsor / Collaborator

Capricor, Inc.

NCT04623671 / CompletedPhase 2

A Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Intravenous Infusion of CAP-1002 in Patients With COVID-19 (INSPIRE)

This is a randomized, double-blind, placebo-controlled, Pilot, Phase 2 Exploratory study that will enroll subjects with a clinical diagnosis of COVID-19 confirmed by laboratory testing and who are in severe or critical condition as indicated by life-support measures.

Start Date15 Nov 2020

Sponsor / Collaborator

Capricor, Inc.

NCT04428476 / Active, not recruitingPhase 2

Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial

This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of twenty intravenous administrations of deramiocel, each separated by three months. Subjects will undergo a targeted screening during a 30-day screening period, eligible subjects will then undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of deramiocel.
Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, and 60. Safety and efficacy assessments will be conducted prior to deramiocel administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57 trial visits, unless otherwise indicated.
All deramiocel infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day, if medically cleared by the site Investigator.

Start Date20 Jul 2020

Sponsor / Collaborator

Capricor, Inc.

100 Clinical Results associated with Deramiocel

100 Translational Medicine associated with Deramiocel

100 Patents (Medical) associated with Deramiocel

Literatures (Medical) associated with Deramiocel

02 Jan 2023·Expert opinion on biological therapy

Biological and genetic therapies for the treatment of Duchenne muscular dystrophy

Review

Author: Wilton-Clark, Harry ; Yokota, Toshifumi

INTRODUCTION:

Duchenne muscular dystrophy is a lethal genetic disease which currently has no cure, and poor standard treatment options largely focused on symptom relief. The development of multiple biological and genetic therapies is underway across various stages of clinical progress which could markedly affect how DMD patients are treated in the future.

AREAS COVERED:

The purpose of this review is to provide an introduction to the different therapeutic modalities currently being studied, as well as a brief description of their progress to date and relative advantages and disadvantages for the treatment of DMD. This review discusses exon skipping therapy, microdystrophin therapy, stop codon readthrough therapy, CRISPR-based gene editing, cell-based therapy, and utrophin upregulation. Secondary therapies addressing nonspecific symptoms of DMD were excluded.

EXPERT OPINION:

Despite the vast potential held by gene replacement therapy options such as microdystrophin production and utrophin upregulation, safety risks inherent to the adeno-associated virus delivery vector might hamper the clinical viability of these approaches until further improvements can be made. Of the mutation-specific therapies, exon skipping therapy remains the most extensively validated and explored option, and the cell-based CAP-1002 therapy may prove to be a suitable adjunct therapy filling the urgent need for cardiac-specific therapies.

01 Mar 2022·Lancet (London, England)Q1 · MEDICINE

Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

Q1 · MEDICINE

Article

Author: Johnson, Hannah ; Collado, Jorge ; Harmelink, Matthew M ; Connolly, Anne ; Muntoni, Francesco ; Furlong, Pat ; Anderson, Kristan ; Abedi, Mehrdad ; Newton, Andrew ; Dayan, Jonathan G. ; Pyzik, Erika ; Ruckdeschel Smith, Rachel ; Marbán, Linda ; Williams, Paula ; Hor, Kan N ; Varadhachary, Arun S ; Ascheim, Deborah D ; Jhawar, Sanjay ; Prasad, Poonam ; Chouteau, Wendy ; Finkel, Richard S ; Hendrix, Suzanne ; Kusmik, Kyle ; Janas, Joanne ; Wells, Julie ; Eagle, Michelle ; Sarwary, Omaid ; Brown, Michelle ; Surampudi, Prasanth ; Van Eyk, Jennifer ; Joyce, Nanette C. ; Giordano, Anthony F. ; Filar, Lauri ; Goude, Erica ; Butterfield, Russell ; Rybalsky, Irina ; McDonald, Craig M ; Anthonisen, Colleen ; Taylor, Michael D ; Rehborg, Rebecca ; Civitello, Matthew ; Wezel, Germaine ; Tian, Cuixia ; Duke, Julie ; Baek, Jayoon ; Marbán, Eduardo ; Henricson, Erik K ; Rogers, Russell G. ; Edmondson, Angie ; Vega, Melisa ; Nicorici, Alina ; Rodriguez, Aixa ; Mayer, Oscar H ; Evans, Maya ; Hogan, Nathaniel ; Rogy, Siegfried

BACKGROUND:

Cardiosphere-derived cells (CDCs) ameliorate skeletal and cardiac muscle deterioration in experimental models of Duchenne muscular dystrophy. The HOPE-2 trial examined the safety and efficacy of sequential intravenous infusions of human allogeneic CDCs in late-stage Duchenne muscular dystrophy.

METHODS:

In this multicentre, randomised, double-blind, placebo-controlled, phase 2 trial, patients with Duchenne muscular dystrophy, aged 10 years or older with moderate upper limb impairment, were enrolled at seven centres in the USA. Patients were randomly assigned (1:1) using stratified permuted blocks to receive CAP-1002 (1·5 × 10⁸ CDCs) or placebo intravenously every 3 months for a total of four infusions. Clinicians, caregivers, patients, and clinical operations personnel were fully masked to treatment groups. The primary outcome was the change in mid-level elbow Performance of Upper Limb version 1.2 (PUL 1.2) score at 12 months, assessed in the intention-to-treat population. Safety was assessed in all individuals who received an investigational product. This trial is registered with ClinicalTrials.gov, NCT03406780.

FINDINGS:

Between March 1, 2018, and March 31, 2020, 26 male patients with Duchenne muscular dystrophy were enrolled, of whom eight were randomly assigned to the CAP-1002 group and 12 to the placebo group (six were not randomised due to screening failure). In patients who had a post-treatment PUL 1.2 assessment (eight in the CAP-1002 group and 11 in the placebo group), the mean 12-month change from baseline in mid-level elbow PUL1.2 favoured CAP-1002 over placebo (percentile difference 36·2, 95% CI 12·7-59·7; difference of 2·6 points; p=0·014). Infusion-related hypersensitivity reactions without long-term sequelae were observed in three patients, with one patient discontinuing therapy due to a severe allergic reaction. No other major adverse reactions were noted, and no deaths occurred.

INTERPRETATION:

CAP-1002 cell therapy appears to be safe and effective in reducing deterioration of upper limb function in patients with late-stage Duchenne muscular dystrophy. Various measures of cardiac function and structure were also improved in the CAP-1002 group compared with the placebo group. Longer-term extension studies are needed to confirm the therapeutic durability and safety of CAP-1002 beyond 12 months for the treatment of skeletal myopathy and cardiomyopathy in Duchenne muscular dystrophy.

FUNDING:

Capricor Therapeutics.

01 Jul 2020·Basic research in cardiology

Allogeneic cardiosphere-derived cells (CAP-1002) in critically ill COVID-19 patients: compassionate-use case series

Article

Author: Friedman, Oren ; Chen, Peter ; Marbán, Linda ; Singh, Siddharth ; Akhmerov, Akbarshakh ; Zaman, Tanzira ; Ebinger, Joseph E ; Falk, Jeremy ; Marbán, Eduardo ; Gheorghiu, Mitch ; Makkar, Raj R ; Chakravarty, Tarun

Abstract:

There are no definitive therapies for patients with severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) infection. Therefore, new therapeutic strategies are needed to improve clinical outcomes, particularly in patients with severe disease. This case series explores the safety and effectiveness of intravenous allogeneic cardiosphere-derived cells (CDCs), formulated as CAP-1002, in critically ill patients with confirmed coronavirus disease 2019 (COVID-19). Adverse reactions to CAP-1002, clinical status on the World Health Organization (WHO) ordinal scale, and changes in pro-inflammatory biomarkers and leukocyte counts were analyzed. All patients (n = 6; age range 19–75 years, 1 female) required ventilatory support (invasive mechanical ventilation, n = 5) with PaO2/FiO2 ranging from 69 to 198. No adverse events related to CAP-1002 administration were observed. Four patients (67%) were weaned from respiratory support and discharged from the hospital. One patient remains mechanically ventilated as of April 28th, 2020; all survive. A contemporaneous control group of critically ill COVID-19 patients (n = 34) at our institution showed 18% overall mortality at a similar stage of hospitalization. Ferritin was elevated in all patients at baseline (range of all patients 605.43–2991.52 ng/ml) and decreased in 5/6 patients (range of all patients 252.89–1029.90 ng/ml). Absolute lymphocyte counts were low in 5/6 patients at baseline (range 0.26–0.82 × 103/µl) but had increased in three of these five patients at last follow-up (range 0.23–1.02 × 103/µl). In this series of six critically ill COVID-19 patients, intravenous infusion of CAP-1002 was well tolerated and associated with resolution of critical illness in 4 patients. This series demonstrates the apparent safety of CAP-1002 in COVID-19. While this initial experience is promising, efficacy will need to be further assessed in a randomized controlled trial.

News (Medical) associated with Deramiocel

25 Nov 2024

·www.globenewswire.com

Capricor Therapeutics Granted Orphan Drug and ATMP Status for Deramiocel by European Medicines Agency

Nov. 20, 2024 -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today 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). The Orphan Drug designation provides Capricor with several benefits that support the development of deramiocel in Europe, including market exclusivity for 10 years if approval is granted and substantially reduced regulatory fees. The ATMP designation provides substantial regulatory support to assist in the development of cell-based therapies. The additional support can potentially reduce time to market, streamline development, and open up access to critical resources, making it an invaluable tool for companies working on innovative therapies. “Receiving the Orphan Drug and ATMP designations from the EMA are significant steps forward as we work to bring deramiocel to DMD patients worldwide,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “With these designations from the EMA, along with Orphan Drug and RMAT designations from the U.S Food and Drug Administration (FDA), we have the opportunity to secure market exclusivity for deramiocel in two of the world’s key markets. These milestones reflect our commitment to advancing novel, transformative treatments for patients suffering from DMD.” Recently, Capricor announced that it had initiated its rolling Biologics License Application (BLA) submission process with the U.S. FDA seeking full approval of deramiocel to treat all patients diagnosed with DMD-cardiomyopathy. Capricor expects to complete its full BLA submission by the end of 2024. Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a population of stromal cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory, antifibrotic and regenerative actions in dystrophinopathy and heart failure. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials. Deramiocel for the treatment of DMD has previously received Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation (RMAT) from the U.S. FDA. In addition, if deramiocel is approved, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on our previous receipt of a rare pediatric disease designation. Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000-20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. In DMD patients, heart muscle cells progressively die and are replaced with scar tissue. This cardiomyopathy eventually leads to heart failure, which is currently the leading cause of death among those with DMD. Treatment options are limited and there is no cure. Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also harnessing the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter. The content above comes from the network. if any infringement, please contact us to modify.

Cell TherapyOrphan Drug

13 Nov 2024

·www.globenewswire.com

Capricor Therapeutics Reports Third Quarter 2024 Financial Results and Provides Corporate Update

Announced intent to file a biologics license application (BLA) for deramiocel to treat all patients with DMD-cardiomyopathy Rolling BLA submission initiated in October 2024 with full submission expected by year-end 2024Company anticipates potential PDUFA date in second half of 2025Announced signing of binding term sheet with Nippon Shinyaku for European expansion and commercialization of deramiocel; potential milestones from combined agreements would total approximately $1.5 billion payable to Capricor Reported positive long-term data from HOPE-2 OLE trial at 2024 World Muscle Society CongressStealthX™ exosome platform expanded to include PMO loading and targeting for the treatment of DMD presented at AAEV Annual MeetingCompleted public offering of common stock for gross proceeds of approximately $86 million; cash runway is expected to support current planned operations into 2027Capricor to host conference call and webcast today at 4:30 p.m. ET SAN DIEGO, Nov. 13, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced its financial results for the third quarter ended September 30, 2024 and provided a corporate update. “This has been a transformational quarter for Capricor as we move towards potential commercialization of deramiocel for the treatment of DMD. We have commenced the submission of our BLA which we expect to be complete by year end and we have significantly strengthened our balance sheet in order to scale up manufacturing as we anticipate a strong launch, pending FDA approval,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “In addition, we continue to advance our proprietary StealthX™ platform technology as part of our long-term strategy to leverage exosomes as vehicles for targeted delivery of payloads for therapeutic development. We continue to explore partnership opportunities and other non-dilutive sources of funding to advance this program.” Recent Updates and Upcoming Milestones Deramiocel DMD Program: Deramiocel is an investigational cell therapy in late-stage development for the treatment of DMD. Deramiocel for the treatment of DMD has received Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation (RMAT) from the FDA in the U.S. In addition, if deramiocel is approved, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on our previous receipt of a rare pediatric disease designation. Based on FDA feedback and following Capricor’s recent pre-BLA meeting in August, Capricor initiated the rolling BLA submission in October of 2024 seeking full approval of deramiocel for the treatment of DMD-cardiomyopathy with full submission expected to be complete by year end 2024. The BLA submission will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2 open label extension (OLE) trials compared to patient-level natural history data. To support potential label expansion to treat DMD, Capricor plans to provide clinical data on skeletal muscle myopathy by combining Cohorts A and B of the Phase 3 HOPE-3 clinical trial to serve as a post-approval study and does not intend to unblind Cohort A at this time, which was originally planned to occur in the fourth quarter of 2024. Furthermore, if required, the HOPE 3 study may also potentially support ex-U.S. marketing authorizations. Currently, Capricor has initiated regulatory activities in Europe and Japan and will be working with the various regional health authorities to develop the most efficient path forward for regulatory approval of deramiocel in these regions. Capricor is actively working towards expansion of our commercial manufacturing capacity and throughput. In addition, Capricor is exploring expansion opportunities for its GMP production for additional capacity that may be necessary to meet product demand in the U.S. and other regions. Capricor signed a binding term sheet with Nippon Shinyaku for European expansion and commercialization of deramiocel for the treatment of DMD. The potential transaction covered by the term sheet would be similar to the existing Commercialization and Distribution Agreements with Nippon Shinyaku in the U.S. and Japan with an opportunity for increased global product reach. Subject to finalization of the Definitive Agreement, Capricor will receive an upfront payment of $20 million along with potential additional development and sales-based milestone payments to Capricor of up to $715 million and a double-digit share of product revenue. In September 2024, Nippon Shinyaku purchased approximately $15 million of Capricor common stock at a 20% premium to the 60-day volume weighted average price of Capricor’s common stock.Capricor presented positive 3-year safety and efficacy results from its ongoing HOPE-2 OLE in a late-breaking poster presentation at the 29th Annual Congress of the World Muscle Society. The 3-year data from HOPE-2 OLE demonstrated improvements in multiple measures of cardiac function, including left ventricular ejection fraction (LVEF%), as well as indexed volumes, which are considered highly relevant in terms of predicting long-term cardiac outcomes.In addition to the cardiac data, patients demonstrated a statistically and clinically relevant benefit (+3.7 points, p< 0.001) in the PUL v2.0 total score when compared to an external comparator dataset of similar DMD patients. StealthX™Exosome Platform: Exosomes are membrane-bound extracellular vesicles and contain lipids, proteins and nucleic acids. They act as messengers to regulate the functions of neighboring or distant cells. Capricor is developing our engineered exosome technology using our proprietary StealthX™ platform focused on the areas of targeted therapeutics and vaccines to potentially treat and prevent a diverse array of diseases. The Company’s proprietary StealthX™ exosome-based vaccine (StealthX™ vaccine) for the prevention of SARS-CoV-2 was selected to be part of Project NextGen, an initiative by the U.S. Department of Health and Human Services to advance a pipeline of new, innovative vaccines. Under the terms of the initiative, Capricor will supply its vaccine candidate for clinical use and NIAID's Division of Microbiology and Infectious Diseases will conduct the trial.Currently, manufacturing is underway for our StealthX™ vaccine with current plans to deliver it to NIAID in the first quarter of 2025.NIAID plans to initiate the trial in the first quarter of 2025, and Capricor expects that it will have preliminary data available in the second quarter of 2025, subject to FDA approval of the NIAID’s IND.If NIAID finds that our StealthX™ vaccine meets its criteria for safety and efficacy, they may consider funding our program for a Phase 2 study. Capricor presented preclinical data at the 2024 American Association of Extracellular Vesicles (AAEV) Annual Meeting highlighting a potential exosome-based approach by delivering phosphorodiamidate morpholino oligomers (PMOs) for the treatment of DMD. Third Quarter 2024 Financial Results Cash position: Cash, cash equivalents and marketable securities totaled approximately $85.0 million as of September 30, 2024 compared to $39.5 million as of December 31, 2023. In the third quarter of 2024, Capricor raised approximately $52.2 million in net proceeds through issuances of common stock at an average price of approximately $9.84 per share under its at-the-market (ATM) Program. Effective October 1, 2024, the ATM Program was closed and terminated. Additionally, on October 18, 2024, the Company completed a public offering of 5,073,800 shares of common stock and received gross proceeds of approximately $86.3 million before deducting underwriting discounts, commissions and offering expenses of approximately $5.5 million, for net proceeds of approximately $80.8 million. Revenues: Revenues for the third quarter of 2024 were approximately $2.3 million compared to $6.2 million for the third quarter of 2023. Additionally, revenues for the nine months ended September 30, 2024 and 2023 were approximately $11.1 million and $13.1 million, respectively. Capricor’s primary source of revenue was from the ratable recognition of the $40.0 million (upfront and milestone payments) in accordance with its U.S. Commercialization and Distribution Agreement with Nippon Shinyaku. Expenses: Total operating expenses for the third quarter of 2024 were approximately $15.3 million compared to $13.1 million for the third quarter of 2023. Total operating expenses for the nine months ended September 30, 2024 and 2023 were approximately $46.0 million and $35.9 million, respectively. Net loss: The Company reported a net loss of approximately $12.6 million, or $0.38 per share, for the third quarter of 2024, compared to a net loss of $6.4 million, or $0.25 per share, for the third quarter of 2024. Capricor reported a net loss of approximately $33.4 million, or $1.04 per share, and $21.5 million, or $0.85 per share, for the nine months ended September 30, 2024 and 2023, respectively. Financial Outlook: The Company believes that based on the current operating plan and financial resources, our available cash, cash equivalents and marketable securities will be sufficient to cover anticipated expenses and capital requirements into 2027. This expectation excludes any additional potential milestone payments under the Commercialization and Distribution Agreements with Nippon Shinyaku, as well as any strategic use of capital not currently in the Company’s base-case planning assumptions. Upcoming Events The Company plans to present at the following upcoming events: B. Riley Securities NextGen Tissue Delivery Modalities Summit, November 14, 2024, VirtualPiper Sandler 36th Annual Healthcare Conference, December 3-5, 2024, New York, NYOppenheimer Movers in Rare Disease Summit, December 12, 2024, New York, NY Conference Call and Webcast To participate in the conference call, please dial 1-800-717-1738 (Domestic/Toll-Free) or 1-646-307-1865 (International) and reference the conference ID: 68076. Participants can use guest dial-in numbers above and be answered by an operator or click the Call me™ link for instant telephone access to the event. To participate via a webcast, please click here. A replay of the webcast will be available following the conclusion of the live broadcast and will be accessible on the Company’s website. About Capricor Therapeutics Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also harnessing the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter. Cautionary Note Regarding Forward-Looking Statements Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; potential future agreements; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, as filed with the Securities and Exchange Commission on August 8, 2024. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements. Capricor has entered into an agreement for the exclusive commercialization and distribution of deramiocel (CAP-1002) for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation. For more information, please contact: Capricor Media Contact:Raquel ConaKCSA Strategic Communications rcona@kcsa.com212.896.1204 Capricor Company Contact:AJ Bergmann, Chief Financial Officerabergmann@capricor.com858.727.1755 CAPRICOR THERAPEUTICS, INC.CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS(UNAUDITED) Three months ended September 30, Nine months ended September 30, 2024 2023 2024 2023 REVENUE Revenue$2,261,642 $6,185,814 $11,139,956 $13,089,977 TOTAL REVENUE 2,261,642 6,185,814 11,139,956 13,089,977 OPERATING EXPENSES Research and development 11,807,867 10,028,964 35,413,649 26,507,872 General and administrative 3,463,655 3,021,450 10,593,308 9,378,672 TOTAL OPERATING EXPENSES 15,271,522 13,050,414 46,006,957 35,886,544 LOSS FROM OPERATIONS (13,009,880) (6,864,600) (34,867,001) (22,796,567) OTHER INCOME (EXPENSE) Investment income 453,152 479,380 1,516,418 1,276,502 Loss on disposal of fixed assets - (5,388) - (5,388) TOTAL OTHER INCOME (EXPENSE) 453,152 473,992 1,516,418 1,271,114 NET LOSS (12,556,728) (6,390,608) (33,350,583) (21,525,453) OTHER COMPREHENSIVE INCOME (LOSS) Net unrealized gain (loss) on marketable securities (58,766) (66,485) (139,592) 7,964 COMPREHENSIVE LOSS$(12,615,494) $(6,457,093) $(33,490,175) $(21,517,489) Net loss per share, basic and diluted$(0.38) $(0.25) $(1.04) $(0.85)Weighted average number of shares, basic and diluted 33,090,063 25,817,676 32,099,181 25,468,880 CAPRICOR THERAPEUTICS, INC.SUMMARY BALANCE SHEETS September 30, 2024 (unaudited) December 31, 2023Cash, cash equivalents and marketable securities $85,028,624 $39,487,703 Total assets $92,951,613 $58,734,327 Total liabilities $24,686,654 $36,132,860 Total stockholders' equity - 40,332,392 and 31,148,320 common shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively 68,264,959 22,601,467 Total liabilities and stockholders' equity $92,951,613 $58,734,327

Phase 3VaccineClinical ResultCell Therapy

12 Nov 2024

·www.globenewswire.com

Capricor Therapeutics to Present Exosome Platform Updates at 2024 American Association of Extracellular Vesicles Annual Meeting

-Abstract Highlights New Preclinical Data on Exosome-Based Delivery of PMOs for the Treatment of DMD-SAN DIEGO, Nov. 12, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that an abstract featuring new preclinical data highlighting the therapeutic potential of its StealthX™ exosome platform technology is being presented at the 2024 American Association of Extracellular Vesicles Annual Meeting taking place in Houston, Texas from November 10-13, 2024. The findings highlight a potential exosome-based approach for the treatment of Duchenne muscular dystrophy (DMD) by delivering phosphorodiamidate morpholino oligomers (PMOs). “We continue to build and characterize our StealthX™ exosome platform and this study further supports that endeavor as we provide a novel approach for exosome-based PMO delivery for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “This study further demonstrates the potential for an exosome-based therapeutic approach to exhibit better cellular uptake and promote higher exon skipping efficiency. Furthermore, our platform continues to support the concept of exosomes as a potential suitable delivery vehicle for a variety of payloads and we continue to support our goal to leverage partnership opportunities to further advance this technology.” The abstract will be made available on the publications section of the Capricor website. About Capricor Therapeutics Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also harnessing the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter. Cautionary Note Regarding Forward-Looking Statements Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; potential future agreements; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, as filed with the Securities and Exchange Commission on August 8, 2024. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements. Capricor has entered into an agreement for the exclusive commercialization and distribution of deramiocel (CAP-1002) for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation. For more information, please contact: Capricor Media Contact:Raquel ConaKCSA Strategic Communications rcona@kcsa.com212.896.1204 Capricor Company Contact:AJ Bergmann, Chief Financial Officerabergmann@capricor.com858.727.1755

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Indication	Highest Phase	Country/Location	Organization	Date
Muscular Dystrophy, Duchenne	NDA/BLA	US	Capricor Therapeutics, Inc.	09 Oct 2024
COVID-19	Phase 2	US	Capricor, Inc.	15 Nov 2020
Heart Failure	Phase 2	US	Medical University of South Carolina	11 Sep 2016
Cardiomyopathies	Phase 2	US	Capricor, Inc.	19 Jul 2015
Myocardial Infarction	Phase 2	US	Capricor, Inc.	13 Nov 2012
Ventricular Dysfunction, Left	Phase 2	US	Capricor, Inc.	13 Nov 2012
Muscular Dystrophies	Discovery	US	Lonza Houston, Inc.	-

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04428476 (HOPE-2 OLE, GlobeNewswire) Manual	Phase 2	Muscular Dystrophy, Duchenne	-	Deramiocel	azfyfckltm(cbloaxrkhi) = lswkzrrtgn jmgvzniola (lxoqinguvx )	Positive	11 Oct 2024
NCT02941705 (RegressHFpEF, CTgov)	Phase 2	Heart failure with normal ejection fraction \| Fibrosis \| Heart Failure, Diastolic	27	Allogeneic Derived Cells (RECIEVED CELLS)	oxizrmsixa(ggesjdrhdy) = avawdemyak imlepfrpbu (bmkekrqjhj, pjzwppxeni - yyvewowjnx) View more	-	25 Jul 2024
				Placebo/Control Arm (CONTROL ARM)	oxizrmsixa(ggesjdrhdy) = mmgxhzqwdb imlepfrpbu (bmkekrqjhj, aqhbwlhygi - ofszisfefu) View more
NCT04428476 (HOPE-2-OLE, Biospace) Manual	Phase 2	Muscular Dystrophy, Duchenne	20	CAP-1002	qatrptiwpf(ipunwfussl) = szabbkuucp lxchimivxj (ehiesdlcze )	Positive	04 Jun 2024
				External Comparator	qatrptiwpf(ipunwfussl) = fmiwfbndmf lxchimivxj (ehiesdlcze )
NCT01458405 (ALLSTAR, CTgov)	Phase 1/2	Ventricular Dysfunction, Left \| Myocardial Infarction	135	placebo (Randomized Treatment Cohort: Placebo)	uvlnqquxju(vovqedywfz) = cngclrprgq hsqkfuacni (zalmowgedw, bhpsfkzbkz - dzzftfjhwf) View more	-	09 Apr 2024
				CAP-1002 (Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells)	uvlnqquxju(vovqedywfz) = redzbxqzsy hsqkfuacni (zalmowgedw, sfohshwsjx - xoiqnzpsnb) View more
NCT05126758 (HOPE-2-OLE, MDA2024) Manual	Phase 3	Muscular Dystrophy, Duchenne	12	CAP-1002	lvjpefpgdr(zutujsonqd) = lbbbnepkty haanzrvwuk (xsxvibhxja ) View more	Positive	03 Mar 2024
NCT04428476 (Corporate Publications) Manual	Phase 2	Muscular Dystrophy, Duchenne	13	CAP-1002 (continuous CAP-1002)	uaqfawjxnw(afhrwmsibk) = CAP-1002, when used over time, is slowing DMD’s devastating effects and may be disease modifying. zwzffexhae (xubebqzmlg ) View more	Positive	27 Jun 2022
				CAP-1002 (from placebo to CAP-1002)
NCT03406780 (HOPE-2, Pubmed \| Lancet) Manual	Phase 2	Muscular Dystrophy, Duchenne	19	CAP-1002	zduyqijvsp(zovsdfjnuk) = No other major adverse reactions were noted txnvpzchjq (rjatacyfxn ) View more	Positive	12 Mar 2022
				Placebo
NCT02485938 (HOPE-Duchenne, Pubmed \| Ann Neurol) Manual	Phase 1/2	Muscular Dystrophy, Duchenne	25	CAP-1002	jwjyjvqblg(qwlrvoihzs) = Mid-distal PUL improved at 12 months in 8 of 9 lower functioning CAP-1002 patients, and no controls dusqwaaqui (syzdwhcrjd )	Positive	19 Feb 2019
				usual care
NCT02293603 (Biospace) Manual	Phase 1	Cardiomyopathy, Dilated \| Heart Failure	-	CAP-1002	dbfxlrqgud(mprjrbrira) = sxhzbdrhst uodgsevuet (wpohmnlidn ) View more	Positive	09 Nov 2015

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