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 Capricor Therapeutics, Inc.

0 Patents (Medical) associated with Capricor Therapeutics, Inc.

Literatures (Medical) associated with Capricor Therapeutics, Inc.

15 Jun 2023·Microbiology spectrum

Exosome-Based Multivalent Vaccine: Achieving Potent Immunization, Broadened Reactivity, and Strong T-Cell Responses with Nanograms of Proteins.

Article

Author: Young, Meredith ; Cacciottolo, Mafalda ; Mora, Ciana L ; Li, Yujia ; Chin, Esther R ; Nice, Justin B ; Adachi, Stephanie Y ; Sun, Minghao ; LeClaire, Michael J ; Twaddle, Ryan ; Angeles, Jenna ; Elliott, Kristi

Currently approved vaccines against severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) have focused solely on the spike protein to provide immunity. The first vaccines were developed rapidly using spike mRNA delivered by lipid nanoparticles but required ultralow-temperature storage and have had limited immunity against variations in spike. Subsequently, protein-based vaccines were developed, which offer broader immunity but require significant time for development and the use of an adjuvant to boost the immune response. Here, exosomes were used to deliver a bivalent protein-based vaccine in which two independent viral proteins were used. Exosomes were engineered to express either SARS-CoV-2 delta spike (Stealth X-Spike [STX-S]) or the more conserved nucleocapsid (Stealth X-Nucleocapsid [STX-N]) protein on the surface. When administered as a single product (STX-S or STX-N) or in combination (STX-S+N), both STX-S and STX-N induced strong immunization with the production of potent humoral and cellular immune responses. Interestingly, these results were obtained with the administration of only nanograms of protein and without an adjuvant. In two independent animal models (mouse and rabbit), the administration of nanograms of the STX-S+N vaccine resulted in increased antibody production, potent neutralizing antibodies with cross-reactivity to other variants of spike, and strong T-cell responses. Importantly, no competition of immune responses was observed, allowing the delivery of nucleocapsid with spike to offer improved SARS-CoV-2 immunity. These data show that the StealthX exosome platform has the enormous potential to revolutionize vaccinology by combining the advantages of mRNA and recombinant protein vaccines into a superior, rapidly generated, low-dose vaccine resulting in potent, broader immunity. IMPORTANCE The pandemic emergency has brought to light the need for a new generation of rapidly developed vaccines that induce longer-lasting, potent, and broader immune responses. While the mRNA vaccines played a critical role during the emergency in reducing SARS-CoV-2 hospitalization rates and deaths, more efficient approaches are needed. A multivalent, protein-based vaccine delivered by exosomes could meet this urgent need due to the high speed of development, manufacturability, and the ability to produce a strong antibody response, with neutralizing antibodies and a strong T-cell response able to broadly combat viral infection with a minimum number of injections.

01 Apr 2021·Nanomedicine: Nanotechnology, Biology and MedicineQ2 · MEDICINE

Small molecule inhibitors and culture conditions enhance therapeutic cell and EV potency via activation of beta-catenin and suppression of THY1

Q2 · MEDICINE

Article

Author: Ibrahim, Ahmed Gamal-Eldin ; Marbán, Linda ; Jones-Ungerleider, K Candis ; Ciullo, Alessandra ; Li, Chang ; Peck, Kiel ; Marbán, Eduardo

Primary cell therapy continues to face significant hurdles to therapeutic translation including the inherent variations that exist from donor to donor, batch to batch, and scale-up driven modifications to the manufacturing process. Cardiosphere-derived cells (CDCs) are stromal/progenitor cells with clinically demonstrated tissue reparative capabilities. Mechanistic investigations have identified canonical Wnt/β-catenin signaling as a therapeutic potency marker, and THY1 (CD90) expression as inversely correlated with potency. Here we demonstrate that the cardiosphere formation process increases β-catenin levels and enriches for therapeutic miR content in the extracellular vesicles of these cells, namely miR-146a and miR-22. We further find that loss of potency is correlated with impaired cardiosphere formation. Finally, our data show that small GSK3β inhibitors including CHIR, and BIO and "pro-canonical Wnt" culturing conditions can rescue β-catenin signaling and reduce CD90 expression. These findings identify strategies that could be used to maintain CDC potency and therapeutic consistency.

17 Nov 2020·Circulation

Abstract 14478: Left Ventricular (LV) Determinants of Left Atrial (LA) Remodeling in Ischemic Cardiomyopathy (iCM): From the Allogeneic Heart Stem Cells to Achieve Myocardial Regeneration (ALLSTAR) Double Blind Placebo Controlled Trial

Author: Ac Lima, Joao ; Henry, Timothy D ; Makkar, Raj ; Ambale-Venkatesh, Bharath ; Varadarajan, Vinithra ; Marban, Linda ; Ostovaneh, Mohammad R ; Kapoor, Karan ; Marban, Eduardo

Introduction: ALLSTAR, a trial of cardiosphere-derived cells in iCM, phenotyped the subjects by cardiac magnetic resonance (CMR) at baseline, and 6- and 12- months post-randomization. The overall CMR dataset provides an opportunity to study temporal remodeling of the LA in the context of iCM-related LV alterations. Hypothesis: We hypothesize that LA volumetric and functional remodeling in iCM are coupled to LV determinants. Methods: LA volumes (LAV; maximum and minimum) were obtained from 4- and 2-chamber cine CMR images. LA functional parameters included total, passive and active LA emptying fractions and global longitudinal strain (GLS). LV parameters included end-diastolic and end-systolic volumes (indexed to body surface area; LVEDVi and LVESVi), as well as LV ejection fraction (EF), infarct size, GLS and end-diastolic mass. Six- and 12-month differences from the baseline CMR study were calculated for LA and LV parameters. Linear regression models were used to evaluate univariate associations between LA and LV parameters (at baseline, 6-months, 12-months and their differences [Δ]), independent of treatment effect (which is the subject of ongoing analysis). Results: 101 participants had 6-month follow up CMR studies; 57 had 12-month studies. Baseline means(SD): age 53.9(10.9) years; 87(86.1%) male; LVEF 39.7%(7.0%); infarct size 22.7%(5.4%). In univariate analyses at 6 months, ΔLVEDVi and ΔLVESVi were both significantly associated with ΔLAVimax and ΔLAVimin, but not with LA functional parameters (Table). At 12 months, ΔLVEDVi remained significantly associated with ΔLAVimax, and ΔLVESVi with ΔLAVimin. No association with LA functional parameters was seen. LVEF, infarct size, GLS and end-diastolic mass were not associated with any volumetric or functional LA parameters (not shown). Conclusions: In conclusion, in this cohort of iCM, pathologic dilatation of the LV appears coupled to volumetric LA remodeling, but not other functional LA parameters.

126

News (Medical) associated with Capricor Therapeutics, Inc.

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 Jan 2025

·endpts.com

Hutchmed’s divestiture; Xgene reports Phase 2b win for non-opioid pain drug

Plus, news about Hookipa and Capricor Therapeutics: Hutchmed divests 45% stake in Chinese company: Hutchmed will get about $608 million in cash by divesting Shanghai Hutchison Pharmaceuticals Limited, which it described as “a non-core, non-consolidated joint venture.” The move will allow Hutchmed to focus more on its drug development business. — Max Gelman Xgene’s non-opioid pain drug clears Phase 2b: The biotech’s XG005 met both the primary and key secondary endpoints in the placebo-controlled test of people who had bunion surgery. Around 42% of patients given the high 1250 mg dose didn’t need rescue pain medication. The drug is also being evaluated for cancer-related bone pain and chronic osteoarthritis pain. — Ayisha Sharma Hookipa to buy Poolbeg Pharma: Per the deal, Hookipa is acquiring Poolbeg and raising $30 million in a private placement. Hookipa will continue to trade on the Nasdaq while Poolbeg’s listing from the London Stock Exchange will be removed. The focus of the combined company will be on two immunotherapy programs: HB-700 and POLB 001. — Max Gelman Capricor records $10M milestone: Nippon Shinyaku made the payment after Capricor completed a submission to the FDA for its experimental cell therapy for patients with Duchenne muscular dystrophy cardiomyopathy. — Jaimy Lee

Phase 2Cell TherapyClinical ResultAcquisitionImmunotherapy

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

100 Deals associated with Capricor Therapeutics, Inc.

100 Translational Medicine associated with Capricor Therapeutics, Inc.

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Pipeline Snapshot as of 02 Feb 2025

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

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Current Projects

Drug(Targets)	Indications	Global Highest Phase

Deramiocel	Muscular Dystrophy, Duchenne More	NDA/BLA
Engineered exosomes (Capricor Therapeutics)	Shock, Hemorrhagic More	Preclinical
CAP-2003	Muscular Dystrophy, Duchenne More	Preclinical
Exosome COVID-19 mRNA vaccine (Capricor) ( SARS-CoV-2 S protein )	COVID-19 More	Preclinical
Exosome VLP vaccine (Capricor) ( SARS-CoV-2 S protein )	COVID-19 More	Discontinued

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