RegulationOrphan Drug (US), Orphan Drug (EU), Regenerative Medicine Advanced Therapy (US), Advanced Therapy Medicinal Products (EU), Rare Pediatric Disease (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: Collado, Jorge ; Johnson, Hannah ; Harmelink, Matthew M ; Connolly, Anne ; Muntoni, Francesco ; Furlong, Pat ; Anderson, Kristan ; Newton, Andrew ; Abedi, Mehrdad ; Dayan, Jonathan G. ; Pyzik, Erika ; Ruckdeschel Smith, Rachel ; Marbán, Linda ; Hor, Kan N ; Williams, Paula ; 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 ; Tian, Cuixia ; Wezel, Germaine ; Duke, Julie ; Baek, Jayoon ; Marbán, Eduardo ; Henricson, Erik K ; Rogers, Russell G. ; Edmondson, Angie ; Vega, Melisa ; Rodriguez, Aixa ; Nicorici, Alina ; Mayer, Oscar H ; Evans, Maya ; Rogy, Siegfried ; Hogan, Nathaniel

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 ; 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.

100

News (Medical) associated with Deramiocel

02 Jan 2025

·pipelinereview.com

Capricor Therapeutics Completes Submission of Biologics License Application to the U.S. FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy

-If approved, deramiocel would be first approved therapy for Duchenne muscular dystrophy cardiomyopathy- -BLA submission triggers $10 million milestone payment to Capricor from Nippon Shinyaku- SAN DIEGO, CA, USA I January 02, 2025 I Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the completion of the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking full approval for deramiocel, an investigational cell therapy, to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. “The submission of the BLA marks a pivotal step for Capricor and those impacted by DMD. This BLA is the culmination of a body of work that has been focused on bringing this potentially transformational therapy to those patients in need,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “We believe that the strength of this application is that deramiocel has shown in multiple clinical trials attenuation of the cardiac implications of DMD. We look forward to working with the FDA throughout the review process to support this potential approval.” The full submission of the rolling BLA was completed as the Company had previously guided in late December 2024 and is supported by Capricor’s existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to natural history data from an FDA funded and published dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression. Capricor has requested a priority review, which, if granted, would reduce the review timeline from the standard 10-month to a priority 6-month review from the date the submission is accepted by the FDA. In conjunction with this achievement, Capricor will receive a milestone payment of $10 million from its distribution partner, Nippon Shinyaku Co., Ltd., under the terms of its U.S. Commercialization and Distribution Agreement. Deramiocel for the treatment of DMD, has received Orphan Drug Designation from the FDA and European Medicines Agency (EMA). The regulatory pathway for deramiocel is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) in the U.S. and the Advanced Therapy Medicinal Product (ATMP) Designation in the European region. In addition, if Capricor were to receive FDA marketing approval for deramiocel regarding the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation. About Deramiocel 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. About Duchenne Muscular Dystrophy 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. 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 in late-stage 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 . SOURCE: Capricor Therapeutics

Cell TherapyDrug ApprovalOrphan DrugPhase 2Priority Review

02 Dec 2024

·www.globenewswire.com

Capricor Therapeutics to Present at Upcoming Investor Conferences

SAN DIEGO, Dec. 02, 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 the Company is scheduled to present at the following upcoming investor conferences. Event:Piper Sandler 36th Annual Healthcare Conference (New York, NY)Presentation:Tuesday, December 3, 2024 from 11:30-11:55 a.m. ETFormat:Fireside chat, industry panel and one-on-one meetingsWebcast LinkClick here Event:Oppenheimer Movers in Rare Disease Summit (New York, NY)Presentation:Thursday, December 12, 2024 from 10:00-10:50 a.m. ETFormat:Fireside chat and one-on-one meetings The live webcast of the Piper Sandler fireside chat can be accessed on the “News & Events” page in the Investor section of Capricor’s website at http://capricor.com/news/events/. 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 September 30, 2024, as filed with the Securities and Exchange Commission on November 14, 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

Phase 3Cell Therapy

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

100 Deals associated with Deramiocel

R&D Status

10 top R&D records.

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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 with normal ejection fraction	Phase 2	US	Medical University of South Carolina	12 Jul 2017
Heart Failure, Diastolic	Phase 2	US	Medical University of South Carolina	12 Jul 2017
Cardiomyopathies	Phase 2	US	Capricor, Inc.	07 Jan 2016
Tabes Dorsalis	Phase 2	US	Capricor, Inc.	07 Jan 2016
Myocardial Infarction	Phase 2	US	Capricor, Inc.	13 Nov 2012
Ventricular Dysfunction, Left	Phase 2	US	Capricor, Inc.	13 Nov 2012
Cardiomyopathy, Dilated	Phase 1	US	Capricor, Inc.	01 Nov 2014
Heart Failure	Phase 1	US	Capricor, Inc.	01 Nov 2014

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04428476 (HOPE-2 OLE, GlobeNewswire) Manual	Phase 2	Muscular Dystrophy, Duchenne	-	Deramiocel	fuppnxqkpe(zwduubonse) = gmzrapvvub wdjpzswzjh (sutmztpqgi )	Positive	11 Oct 2024
NCT02941705 (RegressHFpEF, CTgov)	Phase 2	Heart failure with normal ejection fraction \| Heart Failure, Diastolic	27	Allogeneic Derived Cells (RECIEVED CELLS)	bpdwbppzhd(lqxvbkyvse) = mddwpflivc tcybzcbmvr (gpysaksqew, pbgrhxutxy - ogbatecome) View more	-	25 Jul 2024
				Placebo/Control Arm (CONTROL ARM)	bpdwbppzhd(lqxvbkyvse) = dwvtfpqzfq tcybzcbmvr (gpysaksqew, uaoplsdkfy - mxgmynyaee) View more
NCT04428476 (HOPE-2-OLE, Biospace) Manual	Phase 2	Muscular Dystrophy, Duchenne	20	CAP-1002	gogrwlhigi(hyehzmtlvi) = ghlnhsauuy gobrglnytd (aataftenbd )	Positive	04 Jun 2024
				External Comparator	gogrwlhigi(hyehzmtlvi) = fuxmbwuseq gobrglnytd (aataftenbd )
NCT01458405 (ALLSTAR, CTgov)	Phase 1/2	Ventricular Dysfunction, Left \| Myocardial Infarction	135	placebo (Randomized Treatment Cohort: Placebo)	auzgwxbdkl(yqdcwmhkha) = hlgkipupog clataeqwwn (xijtzrrkgt, fmasalfczz - hmdyogkduz) View more	-	09 Apr 2024
				CAP-1002 (Randomized Treatment Cohort: CAP-1002 Allogeneic Cardiosphere-Derived Cells)	auzgwxbdkl(yqdcwmhkha) = izsmuxnywt clataeqwwn (xijtzrrkgt, nxixmebcwp - phcrlnifuz) View more
NCT05126758 (HOPE-2-OLE, MDA2024) Manual	Phase 3	Muscular Dystrophy, Duchenne	12	CAP-1002	naouatsnkz(kzdclfwkeo) = bkkttnuocc ukydrapsxk (krforsimkh ) View more	Positive	03 Mar 2024
NCT04428476 (Corporate Publications) Manual	Phase 2	Muscular Dystrophy, Duchenne	13	CAP-1002 (continuous CAP-1002)	zndenoemnm(tjijfghrnc) = CAP-1002, when used over time, is slowing DMD’s devastating effects and may be disease modifying. fhbrvenkip (lwdultjyfq ) 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	zfylcqexbv(kbyudfzvsc) = No other major adverse reactions were noted ujvqzwsujs (hybmykgtos ) View more	Positive	12 Mar 2022
				Placebo
NCT02485938 (HOPE-Duchenne, Pubmed \| Ann Neurol) Manual	Phase 1/2	Muscular Dystrophy, Duchenne	25	CAP-1002	ederhxziqu(elfmxmygyw) = Mid-distal PUL improved at 12 months in 8 of 9 lower functioning CAP-1002 patients, and no controls yffdovfvrm (rwgquggcks )	Positive	19 Feb 2019
				usual care
NCT02293603 (Biospace) Manual	Phase 1	Cardiomyopathy, Dilated \| Heart Failure	-	CAP-1002	wqcfjcdqmg(krdgyootth) = iemhilwzrw voqwmdyikm (oevnfmepmo ) View more	Positive	09 Nov 2015

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