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CAP 1002

Drug Profile

CAP 1002

Alternative Names: Allogeneic adult stem cell therapy for myocardial infarction - Capricor; Allogeneic cardiosphere-derived cells - Capricor; Allogeneic CDCs - Capricor; Allogenic derived cells - Capricor; CAP-1002

Latest Information Update: 01 Jun 2020

At a glance

  • Originator Capricor
  • Developer Capricor; Capricor Therapeutics; Medical University of South Carolina
  • Class Anti-inflammatories; Antifibrinolytics; Antifibrotics; Cardiovascular therapies; Stem cell therapies
  • Mechanism of Action Cell replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Duchenne muscular dystrophy
  • New Molecular Entity No
  • Available For Licensing Yes - Duchenne muscular dystrophy

Highest Development Phases

  • Phase II Duchenne muscular dystrophy; Heart failure; Myocardial infarction
  • Phase I/II Cardiomyopathies
  • Clinical Phase Unknown COVID 2019 infections

Most Recent Events

  • 14 May 2020 CAP 1002 is available for licensing as of 14 May 2020. http://capricor.com/company-overview/
  • 14 May 2020 The US FDA approves IND application for an expanded access programme for CAP 1002 in COVID-2019 infections
  • 14 May 2020 Final efficacy data from the phase II trial in Duchenne muscular dystrophy released by Capricor Therapeutics

Development Overview

Introduction

CAP 1002 is an allogeneic cardiosphere-derived stem cell (CDC) therapeutic, being developed by Capricor Therapeutics (formerly Capricor Inc), for the treatment of heart failure, myocardial infarction, cardiomyopathy, Duchenne muscular dystrophy (DMD) and related cardiomyopathy. The therapy contains multiple progenitor cells and is derived from adult donor heart tissue. The cells are multiplied in the laboratory using a specialised process, and then they are introduced directly into the patient's heart via intracoronary infusion at the time of standard cardiac catheterisation. These cells act by releasing exosomes which are immunomodulatory and exert anti-inflammatory, anti-fibrotic, and anti-apoptotic effects. The cells also stimulate cellular regeneration. In DMD, the product has been demonstrated to preserve and improve the structure and function of dystrophic skeletal muscle by ameliorating the myocyte damage induced by dystrophin mutations. Clinical development is underway for heart failure, myocardial infarction, cardiomyopathy and Duchenne muscular dystrophy in the US and Canada. CAP 1002 is also available for the treatment of COVID-19 infections under the compassionate use pathway.

Capricor Inc merged with Nile Therapeutics in November 2013 to form Capricor Therapeutics [1] .

As of May 2020, Capricor Therapeutics is seeking partnerships for the development of CAP 1002 [2]

Company Agreements

In January 2018, seven new patent applications were added to the existing licensing agreement signed by Capricor and Cedars-Sinai Medical Center related to cardiosphere-derived cells (CDCs) and CDC-derived extracellular vesicles, including exosomes. This expansion allows Capricor to develop exosome based therapies for the treatment of Duchenne muscular dystrophy, ventricular tachyarrhythmia, cancer and age-related disorders. Earlier, in May 2014, the companies entered into an exclusive license agreement for intellectual property (IP) related to the development of exosomes. Under the terms of the agreement, Capricor has been granted an exclusive world-wide license from Cedars-Sinai Medical Center, with the right to sublicense, IP related to exosomes originating from cardiosphere-derived cells. In the first quarter of 2020, CSMC notified Capricor that it was in breach of the agreement since it failed to file an IND by December 31, 2019 and that if not done so by April 19, 2020, the agreement shall immediately terminate. [3] [4] [5]

In July 2017, Capricor announced that Jansses Biotech had decided not to exercise its option to exclusively license CAP 1002 for development and commercialization in the field of cardiology. Following the decision, the collaboration agreement has been terminated and all rights to develop and commercialize CAP 1002 for any indication, either independently or in collaboration with third parties have been retained by Capricor. Capricor will also have an irrevocable, fully paid-up non-exclusive license under patents controlled by Janssen utilized in the production of the clinical trial materials manufactured pursuant to the Chemistry, Manufacturing, and Controls (CMC) development plan between Capricor and Janssen and a non-exclusive perpetual license to publish, disclose and use the information of Janssen that was utilized in the production of the clinical trial materials manufactured pursuant to the CMC development plan. No payments between Capricor and Janssen are required to be made in relation to this decision. In January 2014, Capricor Therapeutics Inc executed a collaboration agreement and exclusive license option with Janssen Biotech Inc. Under the terms of the agreement, Capricor and Janssen agreed to collaborate on the development of Capricor's cell therapy program for cardiovascular applications, including CAP-1002. Pursuant to the agreement, Capricor and Janssen collaborated on elements of cell manufacturing development. Capricor contributed to the costs of the manufacturing collaboration, and was to receive an upfront payment of $US12.5 million from Janssen. Under the terms of the agreement, Janssen gained the right to enter into an exclusive license agreement for CAP-1002 at any time until sixty days following delivery by Capricor of the six-month follow-up results from phase II of Capricor's ALLSTAR clinical trial for CAP-1002. Had Janssen exercised its option rights, Capricor would be eligible to receive up to $US325 million in additional payments. [6] [7]

Capricor licensed technology for the harvest and manufacture of the cardiosphere-derived stem cells (CDC) from the Johns Hopkins Medical School and the University of Rome. Capricor has a collaborative relationship with Johns Hopkins Medical School and Cedars-Sinai with respect to the research of CAP 1002.

Key Development Milestones

COVID-2019 infection

In May 2020, Capricor Therapeutics reported that the US FDA approved the Investigational New Drug (IND) application for an expanded access program to investigate the use of CAP 1002 in 20 additional patients with COVID-2019 infections. The IND was submitted in April 2020, based on the data from the six patients enrolled in the compassionate programme initiated by Capricor Therapeutics to provide CAP 1002, to patients with advanced COVID-19 under the compassionate use pathway (NCT04338347). The company released the results in April 2020 [2] [8] [9] [10] .

Cardiovascular disorders

In October 2016, Medical University of South Carolina initiated a feasibility phase IIa trial to assess the exploratory efficacy and safety of CAP 1002 intracoronary infusion in patients with heart failure with preserved ejection fraction (54823; NCT02941705). The efficacy endpoints will determine if CAP 1002 treatment affects clinical functional status (QOL scores), exercise tolerance (6 Minute Walk Test - 6MWT), exercise haemodynamics (supine exercise ergometry during right heart catheterisation), myocardial interstitial fibrosis (MRI with native T1 mapping and calculation of extracellular volume after gadolinium administration), macroscopic fibrosis by delayed gadolinium enhancement and diastolic function (catheterisation, echocardiography, BNP). The randomised, double-blind, parallel, placebo-controlled trial recruited 40 patients in the US [11] .

In April 2019, Capricor ceased the ongoing follow-up activities and terminated the ALLSTAR trial to focus resourcing on its active HOPE-2 trial. The Clinical data obtained from the phase I/II ALLSTAR trial showed lack of a clear difference in the change in scar size from baseline to six months between the active and control groups (1002-01; RC3HL103356-01; NCT01458405) [12] . The enrolment of 134 patients with myocardial infarction in the randomised, double-blind and placebo-controlled trial was completed in the US and Canada, in October 2016. The stem cells were delivered intracoronarily into a single coronary vessel, and the infarct size was assessed by MRI. ALLSTAR assessed the use of intracoronary CAP 1002 from 30 days to one year post-myocardial infarction. The study was intended as a proof-of-concept trial to validate the results of CADUCEUS trial from the CAP 1001 drug programme [see Adis insight drug profile 800034783] using an allogeneic product and simultaneously looking for efficacy in patients. The trial consisted of a 14-patient lead-in phase, and an additional enrolment of patients in the phase II portion. The trial was initiated in October 2012 [13] [14] [15] [16] . In December 2013, Capricor received notification from the National Heart Lung and Blood Institute (NHLBI) Gene and Cell Therapy (GST) Data Safety Monitoring Board (DSMB) that the phase I portion met its safety endpoints, and that the company initiated the phase II portion [17] . Capricor Therapeutics and the ALLSTAR DSMB decided to reduce the enrolment goal of the phase II portion from 300 to 120 patients, based on the statistical results, in March 2016 [18] . Positive phase I data were reported in March, July, September and November 2014 [19] [20] [21] [1] . The phase I portion of ALLSTAR was partly funded by a grant from the National Institutes of Health, and the phase II portion is partly funded by the California Institute for Regenerative Medicine (CIRM)of $US3 millions [22] [20] [23] [24] .

Capricor has initiated the phase Ia/Ib DYNAMIC (Dilated cardiomYopathy iNtervention with Allogeneic MyocardIally-regenerative Cells) trial to assess CAP 1002 in patients with advanced heart failure (CAP1002DYNAMIC; R44HL095203-04; NCT02293603). In January 2015, the company announced that the first three patients were administered with CAP 1002, through non-occlusive intracoronary infusion via 3 vessels, in the NIH-sponsored DYNAMIC trial, in the US. The phase Ia portion is an open-label, single centre study evaluating the safety of CAP 1002 in 14 patients. As at April 2015, enrolment was completed. The trial has been funded in part through a $US3 million National Institute of Health grant [25] . The phase Ib portion of the study is designed as a randomised, double-blind, placebo-controlled study, and will evaluate the safety and efficacy of CAP 1002 in 28 patients at up to four sites. CAP 1002 will be administered through non-occlusive intracoronary infusion via 3 vessels. The Cedars-Sinai Medical Center received approval in April 2013 to conduct this trial. In November 2015, Capricor presented positive results of the DYNAMIC trial at the 88th Annual Scientific Sessions of the American Heart Association (AHA - 2015). The results demonstrated that CAP 1002 was safe with no significant adverse cardiac effects at one month or at six months post infusion in patients with dilated cardiomyopathy. The data at six months as well as 12 months also demonstrated preliminary efficacy signal in various parameters including, subjective well-being, exercise capacity, ejection fraction and ventricular volumes [26] [27] [14] [28] [29] .

Duchenne muscular dystrophy (DMD)

In May 2020, Capricor Therapeutics reported that the company has requested an End-of-Phase 2 meeting with FDA to discuss next steps and a pathway to approval of a Biologics License Application for CAP 1002 in DMD [2] .

As of September 2019, Capricor Therapeutics completed the Type B End-of-phase II meeting with the US FDA and discussed data from the interim analysis of the phase II HOPE-2 trial [see below]. The final minutes from the meeting are awaited. Capricor Therapeutics plans to provide an update regarding the guidance received from the US FDA in Q4 2019 and plans to discuss additional data from the trial with the US FDA when available. An approval for the meeting was granted earlier in the same month [30] .

Capricor met the US FDA in December 2018, to discuss clinical trial design of HOPE-2 trial, surrogate or intermediate endpoints and manufacturing processes for CAP 1002. Following the discussion, the FDA advised Capricor to request an end of phase meeting to determine if HOPE-2 could serve as the registration study. The FDA also suggested to use performance of the upper limb (PUL) 2.0 mid-level test as the primary efficacy endpoint for HOPE-2 [31] . The meeting was a part of expedited review process under Regenerative Medicine Advanced Therapy (RMAT)designation [32] . In February 2018, the US FDA had granted CAP 1002 Regenerative Medicine Advanced Therapy designation (RMAT) to CAP 1002 for Duchenne muscular dystrophy, based on results from the HOPE-Duchenne trial [see below]. The FDA Office of Tissues and Advanced Therapies also stated that the FDA would work with Capricor and provide guidance on subsequent development activities of CAP 1002 [33] .

The US FDA, in July 2017, granted a Rare Pediatric Disease Designation to CAP 1002 for treatment of DMD [34] .

In April 2015, CAP 1002 was granted orphan drug designation by the US FDA for the treatment of Duchenne muscular dystrophy associated cardiomyopathy [35] .

In April 2020, Capricor Therapeutics completed the phase II HOPE-2 trial that evaluated the efficacy and safety of CAP 1002, in patients with Duchenne muscular dystrophy (CAP-1002-DMD-02; NCT03406780). The randomised, double-blind, placebo-controlled trial was initiated in April 2018, and enrolled 18 patients in the US. The company released the data from the trial in May 2020. Earlier, in July 2019, Capricor Therapeutics announced that its independent Data and Safety Monitoring Board (DSMB) completed a safety assessment and futility analysis review of the phase II HOPE-2 trial and recommended the continuation of the trial. In January 2019, the company lifted the voluntary hold on dosing that was put in December 2018, following a serious adverse event occurring in the form of anaphylaxis in a patient. To prevent future events, Capricor initiated a pre-medication strategy and is supported by the Data and Safety Monitoring Board and HOPE-2 clinical trial steering committee. In November 2017, the US FDA approved Capricor's IND application to initiate the study in Duchenne muscular dystrophy. In October 2019, Capricor Therapeutics released six-month interim data for the trial [2] [36] [37] [38] [39] [40] [31] [41] [42] [43] [44] [45] [46] .

In September 2017, Capricor Therapeutics completed the phase I/II HOPE-DUCHENNE (Halt cardiomyOPathy progrEssion in Duchenne) trial that evaluated the safety and tolerability of CAP 1002, in patients with cardiomyopathy secondary to Duchenne muscular dystrophy (HOPE-1; NCT02485938; CAP-1002-DMD-01). The open label trial was initiated in August 2015, and enrolled 25 patients in the US. Patients received CAP 1002 in the three main coronary arteries, to enable broad delivery across the myocardium. In June 2015, the US FDA cleared Capricor's IND application for development of CAP 1002 for the treatment of Duchenne muscular dystrophy-related cardiomyopathy. In April 2017, Capricor Therapeutics presented a top-line six-month data from the study. Based on the data, the company submitted an FDA meeting request to discuss these results and will apply for Regenerative Medicine Advanced Therapy (RMAT) designation for CAP 1002. In October 2017, the company released data demonstrating patients in advanced stages of the disease experienced meaningful improvements in cardiac and upper limb function after a single dose of CAP 1002. Updated results from the trial were presented at the 90th Annual Scientific Sessions of the American Heart Association (AHA-2017). The trial was partly funded by the California Institute for Regenerative Medicine. In January 2019, results, showing improvement in cardiac muscle function and reduction in cardiac scarring that were statistically-significant and sustained improvement of skeletal muscle functions in patients with duchenne muscular dystrophy, were published in the Journal of Neurology. In May 2020, Capricor Therapeutics released therapeutics and safety data form a phase I/II trial [47] [48] [49] [50] [51] [42] [52] [53] [54] [55] [56] [14] [57] [58] [18] [59] [60] [22] [61] [62] [63] [64] [65] .

In April 2018, Capricor Therapeutics released preclinical results which demonstrated that CAP 1002 yields an increase in exercise performance in a disease model of Duchenne muscular dystrophy [66] .

In preclinical studies conducted in mouse models of DMD, intravenous CAP 1002 was shown to increase both exercise capacity and diaphragm function. The drug predominantly distributed to the lungs after injection and was cleared within three weeks. Dosing of more than 2.5 times the human equivalent dose proposed for the upcoming HOPE-2 trial was observed to be safe in these studies. The cardiosphere-derived cells (CDCs) improved cardiac muscle function, walking abilities and survival in a mouse model of duchenne muscular dystrophy. CAP 1002 stimulated tissue repair and regeneration, helped in reducing inflammation, which then enabled new healthy muscle to form, in the mouse model of duchenne muscular dystrophy. Additionally, the CDCs improved muscle physiology and showed strong immunomodulatory activity, in a mouse model of DMD [67] [68] [69] .

Positive preclinical data from a murine model of DMD were presented in November 2014 at the 87th Annual Scientific Sessions of the American Heart Association (AHA-2014).

In February 2016, the company revealed that CAP 1002 demonstrated positive efficacy in mouse preclinical models of Duchenne muscular dystrophy [56] .

Financing information

In May 2016, Capricor announced that it has received approval for a grant award of $US3.4 million approx. from California Institute for Regenerative Medicine, to support the HOPE-Duchenne trial [70] .

In March 2016, Capricor received a grant of approximately $US3.38 millions from California Institute for Regenerative Medicine (CIRM) to support the development of phase I/II HOPE-Duchenne trial. The grant was awarded under the CIRM 2.0 program [59] .

In January 2015, Cure Duchenne Ventures invested $US1 million in Capricor Therapeutics to support a phase I/II trial in patients with heart disease associated with DMD [71] .

In January 2015, Capricor entered into a definitive agreement with H.C. Wainwright & Co and SC&H Capital for the sale of $US17 million of the company's common stock. The net proceeds will be used to fund the ALLSTAR and DYNAMIC trials of the product for the treatment of cardiovascular disorders. The proceeds will also be used to fund the clinical programme of CAP 002 for the treatment of Duchenne muscular dystrophy associated cardiomyopathy [22] [72] .

Drug Properties & Chemical Synopsis

  • Route of administration Intracoronary, IV, Parenteral
  • Formulation Infusion, unspecified
  • Class Anti-inflammatories, Antifibrinolytics, Antifibrotics, Cardiovascular therapies, Stem cell therapies
  • Target Cell
  • Mechanism of Action Cell replacements
  • WHO ATC code

    C01E (Other Cardiac Preparations)

    M09 (Other Drugs for Disorders of the Musculo-Skeletal System)

  • EPhMRA code

    C1X (All Other Cardiac Preparations)

    M5X (All Other Musculoskeletal Products)

Biomarkers Sourced From Trials

Indication Biomarker Function Biomarker Name Number of Trials

cardiomyopathies

not specified

Dystrophin

A1C

1

1

cardiomyopathies

Outcome Measure

Osteopontin

IL1RL1

IL10

Galectin-3

1

1

1

1

Chronic heart failure

Inclusion

BNP

1

dilated cardiomyopathy

Outcome Measure

troponin I, cardiac

CKM

CKB

1

1

1

dilated cardiomyopathy

Safety

troponin I, cardiac

CKM

1

1

Duchenne muscular dystrophy

not specified

Dystrophin

A1C

1

1

Duchenne muscular dystrophy

Outcome Measure

Osteopontin

IL1RL1

IL10

Galectin-3

1

1

1

1

Biomarker

Drug Name Biomarker Name Biomarker Function
CAP 1002 A1C not specified
Dystrophin not specified
Galectin-3 Outcome Measure
IL10 Outcome Measure
IL1RL1 Outcome Measure
Osteopontin Outcome Measure
For more detail, check out BiomarkerBase: the leading source of information about biomarkers used in drug development and diagnostic tests, tracking a comprehensive list of biomarker uses worldwide by over 800 companies

Development Status

Summary Table

Indication Qualifier Patient Segment Phase Countries Route / Formulation Developers Event Date
COVID 2019 infections under compassionate use - Clinical Phase Unknown USA Parenteral / unspecified Capricor Therapeutics 03 Apr 2020
Cardiomyopathies in patients with Duchenne Muscular Dystrophy (DMD)-related cardiomyopathy In adolescents, In adults, In children Phase I/II USA Intracoronary / unspecified Capricor 01 Aug 2015
Duchenne muscular dystrophy - In adolescents, In adults, In children, In the elderly Phase II USA IV / Infusion Capricor 04 Apr 2018
Heart failure in patients with regression-heart failure with preserved ejection fraction In adults, In the elderly Phase II USA Intracoronary / Infusion Medical University of South Carolina 01 Oct 2016
Myocardial infarction - - Phase II Canada, USA Intracoronary / Infusion Capricor Therapeutics 05 Oct 2016

Orphan Status

Indication Patient Segment Country Organisation Event Date
Duchenne muscular dystrophy - USA Capricor Therapeutics 22 Apr 2015

Commercial Information

Involved Organisations

Organisation Involvement Countries
Capricor Originator USA
Capricor Therapeutics Owner USA
Cedars-Sinai Medical Center Technology Provider USA
University of Rome Technology Provider Italy
National Institutes of Health (USA) Funder USA
CureDuchenne Ventures Funder USA
California Institute for Regenerative Medicine Funder USA
Medical University of South Carolina Collaborator USA
National Heart, Lung and Blood Institute Collaborator USA
Johns Hopkins University School of Medicine Collaborator USA

Licensing Availability

Licensing Organisation Available Indication Available Phase Region Date
Capricor Duchenne muscular dystrophy Phase II - 14 May 2020

Scientific Summary

Adverse Events

Heart failure

Phase I:

In the phase I DYNAMIC trial, intracoronary infusion of CAP 1002 was safe in patients (n=14), which were randomised to groups, ischemic or non-ischemic dilated cardiomyopathy with left ventricular ejection fraction (LVEF) of 35% or below, or New York Heart Association (NYHA) Class III or Ambulatory Class IV heart failure. No major adverse cardiac events were reported at one month or at six months, post-infusion. No cardiac event types of composite of five were recorded as the primary safety endpoint events [27] [56] [54] [29] .

Myocardial infarction

Phase I/II

The phase I portion of the phase I/II ALLSTAR trial met its primary endpoint relating to safety at one month post-infusion. Results showed that there were no acute myocarditis attributable to CAP 1002, no deaths due to ventricular tachycardia or ventricular fibrillation, no acute myocarditis, no sudden deaths and no major adverse cardiac events. The open-label, dose-escalation phase I cohort included 14 patients (9 with recent MI and 5 with chronic MI) [22] [17] . The trial also met its 12-month safety endpoints; at 12 months, no sudden deaths or deaths due to ventricular tachycardia or ventricular fibrillation had occurred. No major adverse cardiac events occurred, and no cases of acute myocarditis attributable to CAP 1002 occurred [19] .

Duchenne muscular dystrophy

Phase II:

Top line safety data obtained from a phase II HOPE-2 trial indicated that CAP 1002 was generally well tolerated with an exception of hypersensitivity reactions that were relieved using routine pre-medication regimen. In the interim result, administration of 40 infusions to patients showed one serious adverse event that required an overnight observation. Previously, two patients enrolled in the HOPE trial had experienced serious adverse event in the form of an immediate immune reaction [47] [37] [39] [46] .

Phase I/II:

In a phase I/II HOPE trial, CAP 1002 was safe and well tolerated over the initial six-month follow-up period. During and immediately following CAP 1002 infusion, no treated volunteers experienced a pre-specified composite safety endpoint, which included the major adverse cardiac events of death, myocardial infarction, or hospitalization for a cardiovascular event. There were no early study discontinuations due to adverse events. There was no significant difference in the incidence of treatment-emergent adverse events in either group [52] [65] .

COVID-2019 infections

Results from the compassionate care program, reported no adverse events related to the administration of CAP 1002 [8] [10] .

Pharmacodynamics

Summary

In a mouse model of Duchenne muscular dystrophy (the mdx mouse), CAP 1002 was associated with improvements in absolute force in soleus (leg) and in the diaphragm. After three administrations of CAP 1002, decreased fibrosis in skeletal and cardiac muscles was observed [66] .

Data from a preclinical study in mice with a muscular dystrophy (mdx mice) showed that intracoronary injection of CAP 1002 (105 cells total) markedly improved LV ejection fraction in treated mice compared with vehicle-treated controls. Additionally, mice receiving CAP 1002 exhibited higher maximal exercise capacity compared with mice in the vehicle group. These effects were associated with increased activation of the antioxidative Nrf2 pathway, attenuated infiltration of inflammatory cells, reduction of collagen I and III depositions and fibrosis and augmented heart cardiomyogenesis [62] .

Immunogenicity

Results from the phase I portion of the ALLSTAR trial of CAP 1002 in patients who had suffered a myocardial infarction (MI) showed no immunological responses to the agent. Antibody response monitoring revealed low levels of antibodies present in treated patients, indicating a minor immune response to the allogeneic cell therapy. The open-label, dose-escalation phase I cohort included 14 patients (9 with recent MI and 5 with chronic MI) [17] .

Therapeutic Trials

Myocardial infarction

Phase I/II

At six months, mean ejection fraction was increased to 44.1% from a mean baseline of 38.9% following treatment with CAP 1002 intracoronary infusion in patients who suffered a myocardial infarction > 30 days and < 12 months prior to treatment. A relative reduction in scar size of 20.7% was observed at 12-month follow-up. This showed a low probability of achieving statistically-significant difference in the 12-month primary efficacy endpoint of percent change from baseline infarct size as a percent of left ventricular mass. At six months, a near-statistically-significant (p = 0.05) reduction of mean end-diastolic volume, as well as a trend of reduction of mean end-systolic volume was seen. No notable difference between treatment groups with respect to the change in ejection fraction was observed. The open-label, dose-escalation phase I portion of the phase I/II ALLSTAR trial was designed to involve 14 patients [12] [20] [21] [16] .

Heart failure

Phase I

In the phase I DYNAMIC trial, intracoronary infusion of CAP 1002 exhibited efficacy. Fourteen patients were randomised to groups, ischemic or non-ischemic dilated cardiomyopathy with left ventricular ejection fraction (LVEF) of 35% or below, or New York Heart Association (NYHA) Class III or Ambulatory Class IV heart failure. At 12 months, the data demonstrated directional improvements from baseline in key efficacy measures, including assessments of functional status, cardiac function and dimensions, and quality-of-life. The left ventricular ejection fraction had significantly improved as compared to baseline (p = 0.02) [26] . At six months, multiple efficacy signals were w.r.t. median change ranged from baseline for ejection fraction (-20 to 65; p = 0.02), left ventricular end-diastolic volume (LVEDV) (-47 to 35; p = 0.03) and left ventricular end-systolic volume (LVESV) (-44 to 37; p = 0.08), 6 minute walk test (-13 to 113; p = 0.68), maximal oxygen uptake (VO2) (-25 to 141; p = 0.46) and Minnesota Living with Heart Failure (MLHFQ) (-98 to 79; p = 0.01) for six months. An improvement in NYHA class III occurred in one out of eight patients, post infusion at six months [27] . In a pooled-dose analyses (N = 10-13) done at six and 12 months following CAP 1002 infusion, measures of functional status and capacity, cardiac function and dimension, and quality-of-life broadly showed trends of improvement from baseline at both time points. Statistically-significant improvements in NYHA Class (p = 0.01) and in left ventricular ejection fraction (LVEF) (p = 0.02), as well as in the Minnesota Living with Heart Failure Questionnaire (MLHFQ) score (p = 0.01), were demonstrated at six months. The level of significance was maintained for LVEF improvement at twelve months (p = 0.02). Improvement was seen in eleven of twelve (92%) patients in terms of at least one NYHA Class at six months (p = 0.01), including all five patients who received the highest dose of CAP 1002. In this group, improvement was seen in three patients at six months by one class to Class II and in two patients by two classes to Class I. Further improvement and durability of the benefit of CAP 1002 on heart failure status for as long as 12 months following administration was indicated by two patients at Class II and three at Class I at 12 months [54] [29] .

Duchenne muscular dystrophy (DMD)

Phase II:

The results from the phase II HOPE-2 trial showed that treatment with CAP 1002 after 12 months demonstrated statistically significant outcome relative to placebo controls and a positive treatment in upper limb, cardiac and respiratory functions. The results showed that improvements in Performance of the Upper Limb (PUL) 2.0 was observed in CAP 1002 treated patients (p = 0.05) with a mean change of 2.4 points over placebo patients. Other significant outcomes of the treatment were improvements in the mid-level PUL 1.2 in the stem-cell therapy treated patients (p = 0.08) with a mean change of 2.8 points over placebo patients, improvements in cardiac function as measured by ejection fraction (p = 0.004) and reduction in the biomarker CK-MB, an enzyme that is only released when there is cardiac muscle cell damage compared to placebo (p = 0.006). At 12 month time point the upper limb function evaluated on the basis of mi-level PUL (version1.2), shoulder+Mid+Distal PUL for version 1.2 and 2.0 was found to be -2.1, -2.3 and -1.3 for CAP 1002 treated patients (n=8) and -4.9, -6.4 and -3.7 for placebo treated patients (n=12) respectively (p=0.08, 0.03 and 0.05 respectively). The interim results showed that the mean change from baseline (standard deviation, SD) of the PUL 2.0 at 3-month point were CAP 1002 [0.1 (1.07)], placebo [-0.4 (0.52)], p-value (0.0591) and at 6-month point were CAP 1002 [-0.2 (1.17)], placebo [-0.8 (0.75)], p-value (0.0389). The SD for tip to tip pinch strength at 3-month point and at 6-month point were CAP 1002 [0.9 (3.44)], placebo [1.9 (4.12)], p-value (0.9057) and CAP 1002 [3.3 (2.88)], placebo [-0.3 (1.51)], p-value (0.0674), respectively. The SD for grip strength at 3-month point and at 6-month point were CAP 1002 [-0.6 (3.15)], placebo [-0.8 (2.30)], p-value (0.5897) and CAP 1002 [0.8 (4.54)], placebo [-2.2 (1.83)], p-value (0.0389), respectively. The pulmonary assessments were analysed at 3 months, by measuring the inspiratory flow reserve (absolute), a reflection of diaphragmatic strength, that exhibited a statistically significant improvement (p=0.0473). Positive trends were also seen in peak expiratory flow (% predicted), at 3 months. The cardiac assessment analysed by Magnetic resonance imaging (MRI) at 6 months, demonstrated improvement in cardiac muscle function including systolic wall thickening and cardiac mass among those treated with CAP 1002 compared to placebo. As per the updated skeletal assessments data mean change from baseline (standard deviation, SD) of the PUL 2.0 at 3-month point for shoulder + mid + distal level were CAP 1002 [0.5 (1.69)], placebo [-1.2 (1.69)], p-value (0.0549); for mid + distal level were CAP 1002 [0.4 (1.30)], placebo [-0.4 (0.70)], p-value (0.1035); for mid level were CAP 1002 [0.1 (0.99)], placebo [-0.4 (0.52)], p-value (0.2202). As per the updated skeletal assessments data mean change from baseline (standard deviation, SD) of the PUL 2.0 at 6-month point for shoulder + mid + distal level were CAP 1002 [-0.3 (0.52)], placebo [-2.3 (1.49)], p-value (0.0299); for mid + distal level were CAP 1002 [0.2 (1.47)], placebo [-1.4 (0.92)], p-value (0.0177); for mid level were CAP 1002 [-0.2 (1.17)], placebo [-1.1 (0.99)], p-value (0.0612) [47] [2] [37] [39] [46] .

Phase I/II:

In a phase I/II HOPE trial, a statistically-significant increase in mean (standard deviation, or SD) change from baseline in inferior wall segments of the left ventricle was reported with CAP 1002 (+31.2% (47%)) and a mean 25.8% (46.7%) increase in thickening 12 months after treatment. The standard care arm showed a mean (-8.8% (27.7)) (p = 0.02) decrease and a mean 1.6% (37.9%) increase at 12 months in the systolic thickening of the inferior wall. The difference between the groups at six months achieved statistical significance (p=0.04; p=0.09 at 12 months). At the 12-month follow-up, patients treated with CAP 1002 had a mean 7.1% (10.3%) reduction in scar size, in contrast to a mean 4.8% (22.3%) increase in scar size in the usual care group, a difference that achieved statistical significance (p=0.03). Among the lower-functioning patients (baseline Mid-Distal PUL <55 out of 58), investigators reported sustained or improved motor function in 8/9 (89%) of the CAP 1002 treated patients as compared to 0/4 (0%) of the usual care participants, at 12 months (p=0.007). In a post-hoc analysis, 89% of the CAP 1002 treated patients who were more severely impaired demonstrated sustained or improved skeletal muscle function at 12 months, as compared to none of the participants in the control group [49] . Improvement in mean changes in anterior (+16.3 (46.5)) and lateral (+24.5 (51.2)) wall segments was reported after treatment with CAP 1002 as compared with standard care ((-14.1 (24.9)) (p=0.11) and (-4.5 (35.0)) (p=0.24), respectively). The mean (SD) percent change from baseline in observed scar size was -5.1 (8.5) in the CAP-1002 group (p=0.04) and -0.2 (11.5) in the usual care group (p=0.71) (p=0.09 for treatment group difference) at six months. Mean (SD) percent changes from baseline to six weeks and three months, respectively, in combined middle-plus-distal Performance of the Upper Limb test (PUL) dimension were +8.8 (15.0) and +8.9 (15.4) in the CAP 1002 group and -1.7 (3.7) and +0.8 (3.7) in the usual care group. Mid-distal PUL score increased at six weeks by = 10% (or maximum possible) in 42% of CAP-1002 participants compared to none of the usual-care participants (p=0.045). At three months, the group difference in response was 33% CAP 1002 vs. 10% usual care (p = 0.32). The trial was conducted in 25 males with DMD [50] [52] [65] .

COVID-2019 infections

Results from the compassionate care program for CAP 1002, demonstrated of the five patients on ventilator support, four patients no longer required ventilator support within just one to four days following the infusion. The fifth patient remained on mechanical ventilation and the sixth patient received supplemental oxygen and clinically stable. Following infusion, several patients showed improvements in biomarkers, such as ferritin, absolute lymphocyte counts and CRP. Five male patients and one female patient (between ages 19 and 75) suffering from COVID-19 received IV infusions of 150 million allogeneic cardiosphere-derived cells CAP 1002 [8] [10] .

Future Events

Expected Date Event Type Description Updated
31 Mar 2018 Trial Update Capricor Therapeutics plans the phase II HOPE-2 trial for Duchenne muscular dystrophy (In adults, In adolescents, In children, In the elderly) in USA in March 2018 (IV) (700279077) (NCT03406780) [73] 03 May 2018

Development History

Event Date Update Type Comment
14 May 2020 Licensing Status CAP 1002 is available for licensing as of 14 May 2020. http://capricor.com/company-overview/ Updated 20 May 2020
14 May 2020 Regulatory Status The US FDA approves IND application for an expanded access programme for CAP 1002 in COVID-2019 infections [2] Updated 20 May 2020
14 May 2020 Scientific Update Final efficacy data from the phase II trial in Duchenne muscular dystrophy released by Capricor Therapeutics [2] Updated 20 May 2020
13 May 2020 Regulatory Status Capricor plans to meet the US FDA to discuss pathway to approval for CAP 1002 [47] Updated 01 Jun 2020
13 May 2020 Scientific Update Top line efficacy and adverse event data from the phase II HOPE-2 trial in Duchenne muscular dystrophy released by Capricor Therapeutics [47] Updated 31 May 2020
29 Apr 2020 Scientific Update Safety and efficacy data from clinical trial in COVID-2019 infections released by Capricor Therapeutics [8] Updated 04 May 2020
29 Apr 2020 Trial Update Capricor Therapeutics plans a randomized, placebo-controlled trial for moderate and severe COVID-2019 infections [8] Updated 04 May 2020
14 Apr 2020 Trial Update Capricor Therapeutics completes the phase II HOPE-2 trial in Duchenne muscular dystrophy (In adults, In adolescents, In children, In the elderly) in USA (IV) (NCT03406780) [36] Updated 20 Apr 2020
03 Apr 2020 Phase Change - Clinical Clinical trials in COVID-2019 infections in USA (Parenteral) [9] (NCT04338347) Updated 09 Apr 2020
03 Apr 2020 Regulatory Status Capricor Therapeutics submits an expanded access Investigational New Drug (IND) application for COVID-2019 infections [9] Updated 09 Apr 2020
23 Mar 2020 Regulatory Status Capricor Therapeutics announces intention to launch CP 1002 for Duchenne muscular dystrophy Updated 23 Mar 2020
07 Oct 2019 Scientific Update Interim efficacy and adverse event data from the phase II HOPE-2 trial in Duchenne muscular dystrophy released by Capricor Therapeutics [37] Updated 14 Oct 2019
30 Sep 2019 Trial Update Capricor Therapeutics plans a phase III trial for Duchenne muscular dystrophy [30] Updated 20 Nov 2019
15 Jul 2019 Scientific Update Interim efficacy and adverse event data from a phase II HOPE-2 trial in Duchenne muscular dystrophy released by Capricor Therapeutics [39] Updated 17 Jul 2019
11 Apr 2019 Trial Update Capricor Therapeutics completes enrolment in the HOPE-2 phase II trial for Duchenne muscular dystrophy in USA (NCT03406780) Updated 17 Jul 2019
04 Apr 2019 Trial Update Capricor terminates the phase I/II ALLSTAR trial in Myocardial infartion in USA to focus resourcing on its active HOPE-2 programme (NCT01458405) Updated 16 Apr 2019
06 Feb 2019 Regulatory Status The US FDA and the Data and Safety Monitoring Board (DSMB) grants permission to resume enrolment in the phase II HOPE-2 study for Duchenne muscular dystrophy [40] Updated 08 Feb 2019
06 Feb 2019 Trial Update Capricor Therapeutics resumes dosing in patients in the phase II HOPE-2 trial for Duchenne muscular dystrophy [40] Updated 08 Feb 2019
22 Jan 2019 Trial Update Capricor Therapeutics lifts the voluntary clinical hold on the phase II HOPE-2 trial in Duchenne muscular dystrophy due to an anaphylaxis event occurring in a patient [31] Updated 29 Jan 2019
31 Dec 2018 Trial Update Capricor Therapeutics places voluntary clinical hold on the phase II HOPE-2 trial in Duchenne muscular dystrophy due to an anaphylaxis event occurring in a patient [31] Updated 29 Jan 2019
24 Jun 2018 Biomarker Update Biomarkers information updated Updated 31 Aug 2018
19 Apr 2018 Scientific Update Pharmacodynamics data from a preclinical study in Duchenne muscular dystrophy released by Capricor Therapeutics Updated 24 Apr 2018
04 Apr 2018 Phase Change - II Phase-II clinical trials in Duchenne muscular dystrophy (In children, In the elderly, In adolescents, In adults) in USA (IV) (NCT03406780) [45] Updated 03 May 2018
05 Feb 2018 Regulatory Status CAP 1002 receives Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for Duchenne muscular dystrophy [33] Updated 07 Feb 2018
16 Jan 2018 Licensing Status Capricor and Cedars-Sinai Medical Center expand the licensing agreement to include seven new patent applications related to CDC derived exosomes technology [4] Updated 18 Jan 2018
29 Nov 2017 Regulatory Status Capricor announces intention to apply for Regenerative Medicine Advanced Therapy Designation for CAP 1002 to the US FDA [42] Updated 30 Nov 2017
29 Nov 2017 Regulatory Status The US FDA approves IND application for CAP 1002 in Duchenne muscular dystrophy [42] Updated 30 Nov 2017
15 Nov 2017 Scientific Update Updated efficacy and safety data from the phase I/II HOPE trial in Duchenne muscular dystrophy presented at the 90th Annual Scientific Sessions of the American Heart Association (AHA-2017) [49] Updated 22 Nov 2017
08 Nov 2017 Regulatory Status Capricor files an IND application with the FDA in USA for Duchenne muscular dystrophy [43] Updated 14 Nov 2017
04 Oct 2017 Scientific Update Updated efficacy data from the phase I/II HOPE trial in Duchenne muscular dystrophy released by Capricor Therapeutics [50] Updated 10 Oct 2017
26 Sep 2017 Trial Update Capricor Therapeutics plans the phase II HOPE-2 trial for Duchenne muscular dystrophy (In adults, In adolescents, In children, In the elderly) in USA in March 2018 (IV) (NCT03406780) [73] Updated 03 May 2018
01 Sep 2017 Trial Update Capricor completes the HOPE-DUCHENNE phase I/II trial in Cardiomyopathy (In adolescents, In adults, In children, In patients with Duchenne Muscular Dystrophy related cardiomyopathy) in USA (Intracoronary) (NCT02485938) Updated 02 Aug 2018
28 Jul 2017 Regulatory Status Capricor announces intention to submit IND to US FDA for Duchenne muscular dystrophy (DMD) (IV) [44] Updated 04 Aug 2017
18 Jul 2017 Regulatory Status CAP 1002 receives Rare Pediatric Disease Designation from the US FDA for Duchenne muscular dystrophy [34] Updated 20 Jul 2017
06 Jul 2017 Licensing Status Janssen Biotech terminates its options agreement for CAP 1002 [7] Updated 07 Jul 2017
12 May 2017 Trial Update Interim efficacy data from a phase II trial in Myocardial infarction released by Capricor Therapeutics [12] Updated 26 May 2017
25 Apr 2017 Scientific Update Six month efficacy and adverse events data from the phase I/II HOPE trial in Duchenne muscular dystrophy released by Capricor Therapeutics [52] Updated 08 May 2017
10 Nov 2016 Trial Update Capricor Therapeutics plans a clinical trial for Duchenne muscular dystrophy in USA Updated 18 Nov 2016
31 Oct 2016 Scientific Update Updated twelve month efficacy and adverse events data from the DYNAMIC trial in Cardiomyopathy released by Capricor Therapeutics [54] Updated 11 Nov 2016
05 Oct 2016 Trial Update Capricor Therapeutics completes enrolment in the phase II ALLSTAR trial for Myocardial infarction in USA [13] Updated 06 Oct 2016
01 Oct 2016 Phase Change - II Phase-II clinical trials in Heart failure (In adults, In the elderly) in USA (Intracoronary) (NCT02941705) Updated 25 Oct 2016
07 Sep 2016 Trial Update Capricor completes enrolment in the phase I/II HOPE-Duchenne trial in Duchenne muscular dystrophy USA [64] Updated 12 Sep 2016
15 Aug 2016 Scientific Update Twelve month efficacy data from the DYNAMIC trial in Cardiomyopathy released by Capricor Therapeutics [26] Updated 19 Aug 2016
22 Feb 2016 Scientific Update Interim adverse events data from the phase I/II HOPE-DUCHENNE trial in Cardiomyopathy (In adolescents, In adults, In patients with Duchenne Muscular Dystrophy (DMD)-related cardiomyopathy) released by Capricor (Intracoronary) [56] Updated 24 Feb 2016
09 Nov 2015 Scientific Update Positive efficacy and adverse events data from the DYNAMIC trial in Cardiomyopathy presented at the 88th Annual Scientific Sessions of the American Heart Association (AHA-2015) [27] Updated 03 Dec 2015
01 Aug 2015 Phase Change - I/II Phase-I/II clinical trials in Cardiomyopathy (In adolescents, In adults, In children, In patients with Duchenne Muscular Dystrophy (DMD)-related cardiomyopathy) in USA (Intracoronary) (NCT02485938) Updated 31 Oct 2015
08 Jun 2015 Regulatory Status FDA approves IND application for CAP 1002 in Duchenne muscular dystrophy [60] Updated 10 Jun 2015
22 Apr 2015 Regulatory Status CAP 1002 receives Orphan Drug status for Duchenne muscular dystrophy associated cardiomyopathy in USA Updated 25 Apr 2015
19 Mar 2015 Trial Update Capricor plans the HOPE-DUCHENNE phase I trial for Duchenne muscular dystrophy associated cardiomyopathy [22] Updated 06 Apr 2015
12 Jan 2015 Phase Change - I Phase-I clinical trials in Heart failure (In patients with ischaemic or non-ischaemic Dialated cardiomyopathy) in USA (Intracoronary) (NCT02293603) Updated 13 Jan 2015
18 Nov 2014 Scientific Update Adverse events data from the phase I portion of the phase I/II ALLSTAR trial in Myocardial infarction released by Capricor Therapeutics [19] Updated 20 Nov 2014
18 Nov 2014 Scientific Update Preclinical pharmacodynamics data in Duchemme muscular dystrophy presented at the 87th Annual Scientific Sessions of the American Heart Association (AHA-2014) [62] Updated 20 Nov 2014
06 Oct 2014 Phase Change - Preclinical Preclinical trials in Duchenne muscular dystrophy in USA (Intracoronary) Updated 08 Oct 2014
18 Sep 2014 Scientific Update Efficacy data from the phase I part of the phase I/II ALLSTAR trial in Myocardial infarction released by Capricor [20] Updated 22 Sep 2014
16 Jul 2014 Scientific Update Preliminary efficacy data from the phase I part of the phase I/II ALLSTAR trial in Myocardial infarction released by Capricor [21] Updated 22 Jul 2014
06 May 2014 Licensing Status Capricor Therapeutics in-licenses CDC-derived exosomes technology and related patents from Cedars-Sinai Health System [3] Updated 18 Jan 2018
28 Mar 2014 Phase Change - II Phase-II clinical trials in Myocardial infarction in Canada (Intracoronary) after March 2014 [13] Updated 07 Oct 2016
28 Mar 2014 Phase Change - II Phase-II clinical trials in Myocardial infarction in USA (Intracoronary) Updated 07 Apr 2014
28 Mar 2014 Scientific Update Adverse events and immunogenicity data from the phase I portion of the ALLSTAR trial released by Capricor Therapeutics [17] Updated 07 Apr 2014
21 Nov 2013 Company Involvement Nile Therapeutics has merged with Capricor to form Capricor Therapeutics Updated 18 Dec 2013
26 Oct 2012 Phase Change - I/II Phase-I/II clinical trials in Myocardial infarction in USA (Intracoronary) Updated 29 Nov 2012
09 Jul 2012 Regulatory Status US FDA approves IND application for CAP 1002 in Myocardial infarction [15] Updated 10 Jul 2012
21 Oct 2011 Trial Update Capricor plans a phase II trial (ALLSTAR) for Myocardial infarction in USA (NCT01458405) Updated 10 Jul 2012
20 Oct 2009 Phase Change - Preclinical Preclinical trials in Myocardial infarction in USA (Intracoronary) Updated 10 Jul 2012

References

  1. Capricor and Nile Therapeutics Complete Merger to Form Capricor Therapeutics, Inc.

    Media Release
  2. Capricor Therapeutics Reports First Quarter 2020 Financial Results and Provides Corporate Update.

    Media Release
  3. Capricor Therapeutics Enters Into Exclusive License With Cedars-Sinai Medical Center for Exosome-Related IP Portfolio.

    Media Release
  4. Capricor Announces Licensing of Additional Patent Applications from Cedars-Sinai Medical Center for Cellular and Exosome-based Technologies.

    Media Release
  5. SEC filing of Capricor Therapeutics. Internet-Doc 2020;.

    Available from: URL: https://www.sec.gov/Archives/edgar/data/1133869/000110465920039272/tm205377d1_10k.htm
  6. Capricor Therapeutics and Janssen Biotech, Inc. Enter Into Collaboration Agreement and Exclusive License Option.

    Media Release
  7. Capricor Therapeutics Retains Full Rights to CAP-1002 as Janssen Biotech, Inc. Decides Not to Exercise Option.

    Media Release
  8. New Capricor Data Reports 100 Percent Survival in Critical COVID-19 Patients Treated with CAP-1002.

    Media Release
  9. Capricor Initiates Compassionate Use Program for Severe COVID-19 Patients using CAP-1002, its Novel Cell Therapy.

    Media Release
  10. A Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Intravenous Delivery of CAP-1002 in Patients With Severe COVID-19

    ctiprofile
  11. Regress-HFPEF: Regression of Fibrosis & Reversal of Diastolic Dysfunction in HFPEF Patients Treated With Allogeneic CDCs

    ctiprofile
  12. Capricor Therapeutics Provides Update on ALLSTAR Trial.

    Media Release
  13. Capricor Therapeutics Completes Enrollment in Phase II ALLSTAR Clinical Trial.

    Media Release
  14. Capricor Therapeutics Reports Third Quarter 2015 Business & Financial Highlights.

    Media Release
  15. Capricor Announces FDA Approval To Initiate ALLSTAR Trial of Allogeneic Stem Cell Therapy In Patients Following Heart Attack.

    Media Release
  16. Randomized, Double-Blind, Placebo-Controlled Phase I/II Study of the Safety and Efficacy of Intracoronary Delivery of Allogeneic Cardiosphere-Derived Cells in Patients With a Myocardial Infarction and Ischemic Left Ventricular Dysfunction

    ctiprofile
  17. Capricor Therapeutics to Present ALLSTAR Phase I Trial Data at ACC Annual Meeting 2014.

    Media Release
  18. Capricor Therapeutics Reports Fourth Quarter and Full Year 2015 Financial Results and Business Update.

    Media Release
  19. Capricor Therapeutics Announces Favorable ALLSTAR Phase I Safety Results.

    Media Release
  20. Capricor Presents Safety and Preliminary Efficacy Results From ALLSTAR Phase I Clinical Trial at TCT 2014.

    Media Release
  21. Capricor Announces Encouraging Results From Phase I ALLSTAR Trial; Phase II Trial Underway.

    Media Release
  22. Capricor Therapeutics Reports Fourth Quarter and Full Year 2014 Financial & Business Highlights.

    Media Release
  23. Capricor Therapeutics Cleared to Begin Phase II Portion of ALLSTAR Clinical Trial with CAP-1002 in Patients Following Heart Attack.

    Media Release
  24. Capricor and Nile Therapeutics Announce Merger Agreement.

    Media Release
  25. Capricor Therapeutics Initiates the DYNAMIC Clinical Trial for the Treatment of Advanced Heart Failure.

    Media Release
  26. Capricor Therapeutics Reports Second Quarter 2016 Financial Results and Provides Clinical Update.

    Media Release
  27. Capricor Therapeutics Presents Positive Data from the DYNAMIC Clinical Trial at 2015 American Heart Association Scientific Sessions.

    Media Release
  28. Capricor Completes Enrollment of Initial Phase of DYNAMIC Clinical Trial of Cardiosphere-Derived Cell Therapy for Treatment of Advanced Heart Failure.

    Media Release
  29. A Randomized, Double-blind, Placebo-controlled, Phase I Study of the Safety of Multi-vessel Intra-coronary Delivery of Allogeneic Human Cardiosphere-Derived Stem Cells in Patients With Dilated Cardiomyopathy (DCM)

    ctiprofile
  30. Capricor Therapeutics Reports Third Quarter 2019 Financial Results and Provides Recent Corporate Update.

    Media Release
  31. Capricor Announces Positive Outcomes from Comprehensive Multidisciplinary Meeting with FDA.

    Media Release
  32. Capricor to Meet with FDA under its RMAT Designation to Discuss HOPE-2 Clinical Trial.

    Media Release
  33. Capricor Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for Duchenne Muscular Dystrophy Therapy.

    Media Release
  34. Capricor Receives Rare Pediatric Disease Designation from FDA for CAP-1002 for Patients with Duchenne Muscular Dystrophy.

    Media Release
  35. Capricor Granted FDA Orphan Drug Designation for Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy.

    Media Release
  36. Capricor Therapeutics to Host Key Opinion Leader Call on Cardiac Complications of Duchenne Muscular Dystrophy.

    Media Release
  37. Capricor Presents Additional Positive Data from Ongoing HOPE-2 Study of CAP-1002 in Duchenne Muscular Dystrophy at World Muscle Society: Data Demonstrates Improved PUL 2.0 Performance at 6 Months.

    Media Release
  38. Capricor Therapeutics Announces Positive DSMB Safety and Futility Review and Continuation of its HOPE-2 Study in Duchenne Muscular Dystrophy.

    Media Release
  39. Capricor Therapeutics Announces Positive Results from its Interim Analysis in the HOPE-2 Trial to Treat Patients with Duchenne Muscular Dystrophy.

    Media Release
  40. Capricor Resumes Dosing of Enrolled Patients in HOPE-2 Clinical Trial for Duchenne Muscular Dystrophy.

    Media Release
  41. SoCal's Capricor Places Voluntary Hold on DMD Drug Following Allergic Reaction.

    Media Release
  42. Capricor Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application for CAP-1002.

    Media Release
  43. Capricor Therapeutics Reports Third Quarter 2017 Financial Results and Provides Update on Duchenne Muscular Dystrophy Development Program.

    Media Release
  44. Capricor Announces Results of FDA Meeting on Intravenous CAP-1002 for Duchenne Muscular Dystrophy.

    Media Release
  45. Capricor Announces Initiation of HOPE-2 Clinical Trial of CAP-1002 for Duchenne Muscular Dystrophy.

    Media Release
  46. A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Safety and Efficacy of Intravenous Delivery of Allogeneic Cardiosphere-Derived Cells in Subjects With Duchenne Muscular Dystrophy

    ctiprofile
  47. Capricor Announces Positive Top-Line Final Results from HOPE-2 Study in Patients with Duchenne Muscular Dystrophy Treated with Lead Candidate CAP-1002.

    Media Release
  48. Capricor Therapeutics Reports First Quarter 2019 Financial Results and Provides Corporate Update.

    Media Release
  49. Significant Improvements Reported in Duchenne Muscular Dystrophy Patients Treated with Capricor's Investigational Cell Therapy.

    Media Release
  50. Capricor Therapeutics Presents Positive Six-Month Results in Duchenne Muscular Dystrophy at World Muscle Society International Congress.

    Media Release
  51. Capricor Announces 12-Month Results from HOPE-1 Trial in Duchenne Muscular Dystrophy to be Presented at AHA Late-Breaking Session.

    Media Release
  52. Capricor Therapeutics Announces Positive Six-Month Results from the Randomized Phase I/II HOPE Clinical Trial in Duchenne Muscular Dystrophy.

    Media Release
  53. Capricor Therapeutics Reports Third Quarter 2016 Financial Results and Provides Corporate Update.

    Media Release
  54. Capricor Therapeutics Presents Positive 12-Month Results from DYNAMIC at TCT 2016.

    Media Release
  55. Capricor Therapeutics Provides Enrollment Update on HOPE Clinical Trial in Duchenne Muscular Dystrophy.

    Media Release
  56. Capricor Therapeutics Announces the First Patient with Duchenne Muscular Dystrophy-Related Cardiomyopathy Treated with CAP-1002 in the HOPE-Duchenne Phase I/II Clinical Trial.

    Media Release
  57. Capricor Therapeutics and CureDuchenne to Host Webinar on November 3, 2015 at 3:00 p.m. ET.

    Media Release
  58. Capricor Therapeutics Announces DSMB Recommends Continuation of HOPE-Duchenne Clinical Trial.

    Media Release
  59. Capricor Awarded $3.4 Million Grant from California Institute for Regenerative Medicine.

    Media Release
  60. Capricor Announces Receipt of FDA Clearance for the Phase I/II Study of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy Related Cardiomyopathy.

    Media Release
  61. Capricor Therapeutics Reports Third Quarter 2014 Financial & Business Highlights.

    Media Release
  62. Capricor Therapeutics Announces Positive Pre-Clinical Data for Cardiosphere-Derived Cells (CDCs) on Duchenne Muscular Dystrophy Cardiomyopathy.

    Media Release
  63. Capricor Announces Plans to Pursue Clinical Program for the Treatment of Duchenne Muscular Dystrophy With Cardiosphere-Derived Cells (CDCs).

    Media Release
  64. Capricor Therapeutics Completes Enrollment in Randomized HOPE Clinical Trial in Duchenne Muscular Dystrophy.

    Media Release
  65. A Randomized, Open-label Study of the Safety and Efficacy of Multi- Vessel Intracoronary Delivery of Allogeneic Cardiosphere-Derived Cells in Patients With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

    ctiprofile
  66. Capricor Announces New Pre-Clinical Study Finds Repeat Doses of CAP-1002 Lead to Enhanced Exercise Capacity in Duchenne Muscular Dystrophy Disease Model.

    Media Release
  67. Capricor to Present on Exosomes at the Plenary Session of the International Society for Cellular Therapy in Montreal.

    Media Release
  68. New Published Study Reports Cardiosphere-Derived Cells Improved Skeletal and Cardiac Muscle Function in Mouse Model of Duchenne Muscular Dystrophy.

    Media Release
  69. Capricor Therapeutics Reports Intravenous Administration of CAP-1002 Shown to be Effective in Preclinical Models of Duchenne Muscular Dystrophy.

    Media Release
  70. Capricor Therapeutics Provides Corporate Update and Reports First Quarter 2016 Financial Results.

    Media Release
  71. Cure Duchenne Ventures LLC To Provide $1 Million to Capricor Therapeutics to Advance Promising Research to Treat Heart Disease Associated with Duchenne Muscular Dystrophy.

    Media Release
  72. Capricor Therapeutics Announces $10 Million Private Placement of Common Stock.

    Media Release
  73. Capricor Therapeutics Advances Clinical Development Program in Duchenne Muscular Dystrophy with Naming of Principal Investigator.

    Media Release
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