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.

开始日期2022-06-22

申办/合作机构

Capricor, Inc.

NCT04623671 / Completed临床2期

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.

开始日期2020-11-15

申办/合作机构

Capricor, Inc.

NCT04428476 / Active, not recruiting临床2期

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.

开始日期2020-07-20

申办/合作机构

Capricor, Inc.

100 项与 Deramiocel 相关的临床结果

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100 项与 Deramiocel 相关的转化医学

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100 项与 Deramiocel 相关的专利（医药）

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项与 Deramiocel 相关的文献（医药）

2023-01-02·Expert opinion on biological therapy

Biological and genetic therapies for the treatment of Duchenne muscular dystrophy

Review

作者: 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.

2022-03-01·Lancet (London, England)1区 · 医学

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

1区 · 医学

Article

作者: Collado, Jorge ; Johnson, Hannah ; 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 ; Baek, Jayoon ; Duke, Julie ; Marbán, Eduardo ; Henricson, Erik K ; Rogers, Russell G. ; Edmondson, Angie ; Vega, Melisa ; Rodriguez, Aixa ; Nicorici, Alina ; 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.

2020-07-01·Basic research in cardiology

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

Article

作者: Friedman, Oren ; Chen, Peter ; Marbán, Linda ; Singh, Siddharth ; Akhmerov, Akbarshakh ; Zaman, Tanzira ; Falk, Jeremy ; Ebinger, Joseph E ; 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.

112

项与 Deramiocel 相关的新闻（医药）

2024-11-25

·GlobeNewswire-Health Care

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.

细胞疗法孤儿药

2024-11-13

·GlobeNewswire-Health Care

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

临床3期疫苗临床结果细胞疗法

2024-11-13

·CPHI制药在线

这款创新药呼之欲出，外泌体赛道迎来割裂一刻

关注并星标CPHI制药在线近日，创新型生物技术公司Capricor Therapeutics宣布，已向FDA提交其在研细胞疗法Deramiocel（CAP-1002）的生物制品许可申请（BLA），用于治疗杜氏肌营养不良症（DMD）相关的心肌病，目前该领域还没有批准上市的药物。 Deramiocel是一种基于外泌体作用的异体细胞治疗产品，其通过分泌外泌体进行免疫调节、抗炎、抗纤维化，来减缓DMD疾病进展。而且，Deramiocel的外泌体中包含microRNA等活性物质，以此改变巨噬细胞等的基因表达，从而抑制DMD引起的全身炎症。目前，Deramiocel已获得FDA授予的再生医学先进疗法（RMAT）和孤儿药指定，用于治疗DMD。 Deramiocel的研发进展，使得外泌体这一新兴力量进入行业视野，或为医学领域带来革命性变革。不过与任何一种新兴力量一样，外泌体早期研发也充满了挫折和挑战，入局者甚至呈现割裂现象。来自诺奖级发现，优势几何？ 1983年，外泌体首次在体外培养的绵羊红细胞上清液中被发现，它是由细胞分泌的一种细胞外膜状脂质囊泡，大小在40 nm至100 nm之间，在细胞间物质运输和信号交流中发挥重要作用。人体的几乎所有细胞都会分泌外泌体，如血液、唾液、脑脊液等体液中都有外泌体的分布。 2013年，诺贝尔生理学或医学奖授予美国科学家James Rothman、Randy Schekman及德国科学家Thomas C. Südho，以表彰他们在细胞间囊泡运输调控机制领域作出突出贡献。诺奖的加持，使得“外泌体”这个名词闯入大众视角。外泌体可携带蛋白质、核酸、脂质和代谢物等，不仅能够发挥细胞间的通讯功能，还可以参与免疫应答、病毒感染、代谢等多种生理和病理过程。因此，外泌体具有多方面的临床应用前景，既可以作为药物递送工具，也可以作为疾病的治疗制剂，或是诊断疾病的一种新型生物标志物等。目前，全球超过2/3的外泌体企业主要开展在外泌体治疗相关的研究，包括外泌体治疗和外泌体递送。其中在外泌体递送领域，与常见的腺相关病毒（AAV）和脂质纳米颗粒（LNP）相比，外泌体由于是来自细胞的天然产物，因此具有优秀的低毒性和低免疫原性。其次，外泌体空间容量大，能够携带的有效成分类型更丰富，可包容多种药物分子结构。而且，外泌体具有一定的组织向性，可实现靶向递送。诸多优势，使得外泌体在药物递送上的开发前景不可估量。此次Capricor Therapeutics开发的Deramiocel是一种源于健康人类心脏的基质细胞。它们通过释放包含微RNA、非编码RNA和蛋白的外泌体，改善DMD患者肌肉瘢痕或纤维化以及心脏功能。研究显示，Deramiocel已在多项临床试验中降低了DMD对心脏的影响。BLA申请消息公布后，Capricor Therapeutics股价当日上涨52.43%。然而，外泌体的开发并非一帆风顺，没有Capricor Therapeutics的幸运，行业先驱Codiak BioSciences就碰到了很多难以解决的掣肘与挑战。先驱变先烈，外泌体距成功产业化之路还有多远？ 2015年，Codiak BioSciences成立，公司致力于开发生产临床级别外泌体用于治疗胰腺癌的技术，成为公认的外泌体先驱。成立当年，Codiak就完成了3100万美元A轮融资。此后，完成了6100万美元的B轮融资和总额达7650万美元的C轮融资。完成融资后，Codiak开始往工程化外泌体进军。公司开发了专有的engEx™技术平台，可以将小分子、核酸、抗体、酶等各种类型的生物活性分子整合到外泌体中，并将其靶向至特定的细胞与组织。旗下进展较快的两个产品exoIL-12和exoSTING相继进入临床。与前期相当顺利的发展历程相反，2022年，Codiak宣布停止ExoIL-12和ExoSTING的临床推进，同时裁员37%。次年，公司宣告破产。 ExoIL-12是一种经工程改造后在其表面表达促炎IL-12（白介素）的外泌体，其作用机制为在外泌体表面携带IL-12，将IL-12定向递送到肿瘤微环境中来增强IL-12的剂量，激活人体免疫系统对抗肿瘤的免疫反应，同时减少IL-12暴露造成的副作用。此前，基于IL-12的免疫疗法已在动物模型中取得成功，2020 年12月，公司公布了ExoIL-12针对健康人群的初步安全性数据，结果积极。在披露数据结果的同时，公司宣布将于2023年第一季度启动II期试验，但2022年8月，公司突然公告暂停exoIL-12的临床II期，具体原因未披露。 ExoSTING通过外泌体将STING（干扰素基因刺激因子）激动剂整合到外泌体腔内，同时在外泌体表面表达PTGFRN蛋白，PTGFRN的高水平表达可以将STING激动剂定向递送至肿瘤微环境中的抗原呈递细胞（APC），诱导干扰素基因的表达，局部激活人体的免疫应答以杀伤肿瘤细胞。 ExoSTING通过瘤内注射方式治疗头颈部鳞状细胞癌、三阴性乳腺癌、甲状腺间变性癌、皮肤鳞状细胞癌，2022年9月，完成27例志愿者参与的临床I/II期试验。数据结果支持将ExoSTING推进到II期临床，特别是在早期疾病中，与免疫治疗结合可能疗效增强。公司原计划2023年第一季度启动临床II期试验，但2022年8月，公司突然公告暂停ExoSTING项目。在公告暂停临床试验的同时，公司宣布裁员三分之一以上的员工。而且2023年3月，Codiak宣布根据《美国破产法》第 11 章自愿向美国特拉华州破产法院申请破产保护，并寻求出售资产。尽管外泌体先驱的突然离场令人唏嘘，但该赛道仍然吸引了众多MNC入局。制药巨头入场，外泌体成为药物递送新势力鉴于外泌体在药物递送方面所展现的亮眼优势，吸引了多家药企入局，无论是初创企业还是制药巨头，均有入局。据不完全统计，全球约有50家企业积极布局外泌体这一赛道，其中罗氏、礼来等多家制药巨头，以大手笔布局外泌体递送。例如罗氏与波士顿的PureTech Health公司合作，计划利用对方的乳源外泌体（milk-derived exosome）平台技术，为罗氏的反义寡核苷酸平台开发口服配方的核酸类药物，PureTech Health将获得超过10亿美元的里程金及特许权使用费。礼来与Evox Therapeutics达成协议，将利用Evox Therapeutics的DeliverEX外泌体递送技术，开发装载siRNA和反义寡核苷酸外泌体治疗药物，用于治疗神经系统疾病，合作金额达12.3亿美元。国内外泌体治疗技术平台也在来到台前，百趣生物、达尔文细胞生物、恩泽康泰、睿健医药和亿航生物已顺利完成融资。尽管入局者众多，但不得不承认，目前无论是学术界还是产业界，对于外泌体的认识还处于相对早期，在外泌体的认识和开发上还有较大的学习空间。随着首款药物即将获批上市，外泌体的关注度将不断提高，更多功能也将被发掘。无论是外泌体诊断的相关研究，还是外泌体载药递送上的探索，亦或是外泌体药物的开发，终将迎来爆发。参考： 1.https://www.capricor.com/investors/news-events/press-releases/detail/291/capricor-therapeutics-announces-intent-to-file-biologics 2.PureTech Health Announces Collaboration with Roche to Advance Technology for Oral Administration of Antisense Oligonucleotides END 【生物制药新锐说直播预告】来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~

细胞疗法孤儿药

100 项与 Deramiocel 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
杜氏肌营养不良症	申请上市	美国	Capricor Therapeutics, Inc.	2024-10-09
新型冠状病毒感染	临床2期	美国	Capricor, Inc.	2020-11-15
心脏衰竭	临床2期	美国	Medical University of South Carolina	2016-09-11
心肌疾病	临床2期	美国	Capricor, Inc.	2015-07-19
心肌梗塞	临床2期	美国	Capricor, Inc.	2012-11-13
左心室功能不全	临床2期	美国	Capricor, Inc.	2012-11-13
肌营养不良	药物发现	美国	Lonza Houston, Inc.	-

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04428476 (HOPE-2 OLE, GlobeNewswire) 人工标引	临床2期	杜氏肌营养不良症	-	Deramiocel	構醖齋網網衊構構廠糧(遞淵淵簾壓齋糧鏇醖構) = 獵憲窪獵艱築鏇鏇衊糧鏇艱鑰膚鬱廠積選餘築 (鏇齋鏇網遞鏇鹽餘衊憲 )	积极	2024-10-11
NCT02941705 (RegressHFpEF, CTgov)	临床2期	射血分数保留型心力衰竭 \| 纤维化 \| 舒张期心力衰竭	27	Allogeneic Derived Cells (RECIEVED CELLS)	憲願艱夢衊範齋簾獵構(醖艱願築夢艱積鑰鹽壓) = 鬱廠醖鹽鹽餘鏇鹹膚鏇襯膚鹹觸壓範網憲襯築 (積築遞願選衊廠製觸繭, 廠願獵願鹹齋淵鏇膚齋 ~ 願餘鬱選簾餘憲選築築) 更多	-	2024-07-25
				Placebo/Control Arm (CONTROL ARM)	憲願艱夢衊範齋簾獵構(醖艱願築夢艱積鑰鹽壓) = 積壓鑰鬱餘簾壓築範鬱襯膚鹹觸壓範網憲襯築 (積築遞願選衊廠製觸繭, 繭廠觸窪齋夢網顧鑰窪 ~ 襯顧築遞選網網遞簾淵) 更多
NCT04428476 (HOPE-2-OLE, Biospace) 人工标引	临床2期	杜氏肌营养不良症	20	CAP-1002	壓觸蓋鏇蓋膚簾艱獵餘(範蓋網製憲襯夢淵鬱簾) = 願壓築夢築網簾齋醖遞醖廠醖憲願範齋簾築製 (鹽餘鑰選膚簾夢醖遞醖 )	积极	2024-06-04
				External Comparator	壓觸蓋鏇蓋膚簾艱獵餘(範蓋網製憲襯夢淵鬱簾) = 膚鹽膚襯蓋顧齋衊襯淵醖廠醖憲願範齋簾築製 (鹽餘鑰選膚簾夢醖遞醖 )
NCT01458405 (ALLSTAR, CTgov)	临床1/2期	左心室功能不全 \| 心肌梗塞	135	placebo (Randomized Treatment Cohort: Placebo)	觸築鑰範觸糧遞壓範鹹(鹽膚遞築廠鏇鏇願築網) = 積積廠繭獵獵壓鹽構選醖鬱夢獵窪窪廠蓋鏇鏇 (構觸衊糧鹹憲膚膚築憲, 艱繭積鹹淵醖構膚獵鑰 ~ 範鬱觸衊餘淵簾繭鏇蓋) 更多	-	2024-04-09
				CAP-1002 (Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells)	觸築鑰範觸糧遞壓範鹹(鹽膚遞築廠鏇鏇願築網) = 膚鬱積顧構餘廠鹹鏇蓋醖鬱夢獵窪窪廠蓋鏇鏇 (構觸衊糧鹹憲膚膚築憲, 獵鏇簾蓋廠繭壓夢鑰鏇 ~ 鹽廠遞憲糧願膚鬱憲顧) 更多
NCT05126758 (HOPE-2-OLE, MDA2024) 人工标引	临床3期	杜氏肌营养不良症	12	CAP-1002	夢壓製繭選夢製壓選繭(觸選襯襯範鏇淵壓憲壓) = 壓築膚構構淵糧繭遞鹽築鬱獵範繭鹽蓋顧廠壓 (夢窪餘艱顧範廠醖顧築 ) 更多	积极	2024-03-03
NCT04428476 (Corporate Publications) 人工标引	临床2期	杜氏肌营养不良症	13	CAP-1002 (continuous CAP-1002)	網鏇齋糧壓觸醖願夢範(顧網艱鏇糧選鹹積襯蓋) = CAP-1002, when used over time, is slowing DMD’s devastating effects and may be disease modifying. 簾築網艱鑰糧憲積觸範 (製積網繭顧遞壓範構鹽 ) 更多	积极	2022-06-27
				CAP-1002 (from placebo to CAP-1002)
NCT03406780 (HOPE-2, Pubmed \| Lancet) 人工标引	临床2期	杜氏肌营养不良症	19	CAP-1002	壓願夢願餘衊構鏇獵顧(夢窪齋繭鏇餘範遞獵齋) = No other major adverse reactions were noted 鏇構膚築鏇膚窪廠壓遞 (鑰積願構憲衊選蓋簾鑰 ) 更多	积极	2022-03-12
				Placebo
NCT02485938 (HOPE-Duchenne, Pubmed \| Ann Neurol) 人工标引	临床1/2期	杜氏肌营养不良症	25	CAP-1002	襯壓範蓋築鹽蓋蓋廠獵(淵鬱衊鹹繭觸憲衊廠鏇) = Mid-distal PUL improved at 12 months in 8 of 9 lower functioning CAP-1002 patients, and no controls 願選膚衊夢窪壓廠遞選 (鏇製鬱襯蓋網獵鏇廠鑰 )	积极	2019-02-19
				usual care
NCT02293603 (Biospace) 人工标引	临床1期	扩张型心肌病 \| 心脏衰竭	-	CAP-1002	夢憲獵製願網選築廠壓(顧網製艱鑰顧鬱製蓋醖) = 夢範選醖鏇淵衊構鏇夢繭鏇鏇觸選繭膚簾繭糧 (夢糧積構鹽繭艱艱製觸 ) 更多	积极	2015-11-09