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-1002Latest Information Update: 29 Feb 2024
At a glance
- Originator Capricor
- Developer Capricor; Capricor Therapeutics; Medical University of South Carolina
- Class Exosome therapies; Stem cell therapies
- Mechanism of Action Cell replacements
-
Orphan Drug Status
Yes - Duchenne muscular dystrophy
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
- Available For Licensing Yes - Duchenne muscular dystrophy
Highest Development Phases
- Phase III Duchenne muscular dystrophy
- Phase II COVID 2019 infections
- Phase I/II Cardiomyopathies
- Discontinued Heart failure; Myocardial infarction
Most Recent Events
- 14 Nov 2023 Capricor plans a meeting with the US FDA in the scheduled for late March of 2024.
- 10 Oct 2023 Updated efficacy data from the phase II HOPE-2-OLE trial in Duchenne Muscular Dystrophy released by Capricor Therapeutics
- 29 Sep 2023 Capricor Therapeutics plans a follow-up Type B meeting with the US FDA to discuss proposed path towards submission of a potential BLA of CAP 1002 and further discuss its ongoing HOPE-3 clinical trial for Duchenne muscular dystrophy, in early 2023
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), COVID-2019 infections 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 for COVID-2019 infections, cardiomyopathy, and Duchenne muscular dystrophy in the US. Clinical development has been discontinued in heart failure and myocardial infarction.
Capricor Inc merged with Nile Therapeutics in November 2013 to form Capricor Therapeutics [1] .
Company Agreements
In January 2022, Capricor Therapeutics and Nippon Shinyaku entered into an commercialization and distribution agreement for CAP 1002 for the treatment of Duchenne muscular dystrophy (DMD) in the US. Under the terms of the agreement, Nippon Shinyaku will distribute the product, while Capricor will supply it and receive a double-digit share of revenue, as well as development and sales-based milestone payments. The upfront payment was $30 million, with potential milestone payments of up to $705 million. In February 2023, Capricor Therapeutics announced that the partnership was extended to include exclusive commercialization and distribution in Japan. Capricor will be responsible for clinical development, Nippon Shinyaku for distribution, and Capricor will receive an upfront payment of $12 million, potential milestone payments of up to $89 million, and a double-digit share of revenue. If approved, Capricor will hold the Marketing Authorization in Japan. [2] [3] [4]
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. [5] [6] [7]
In July 2017, Capricor announced that Janssen 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. [8] [9] [9]
In January 2021, Capricor and Johns Hopkins University entered into a nonexclusive License Agreement, or the JHU Serology License Agreement, effective January 6, 2021, which provides for the grant of a non-exclusive, world-wide, non-royalty-bearing license by JHU to Capricor to develop and commercialize licensed products under the licensed patent rights for COVID-19. The JHU Serology License Agreement is due to expire July 2022 and at this time, Capricor does not intend to convert it to an exclusive license or extend the JHU Serology License Agreement [10] .
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 February 2022, Capricor Therapeutics completed the phase II INSPIRE trial that evaluated the safety and efficacy of CAP 1002 in patients with COVID-2019 infections (NCT04623671; CAP-1002-COVID-19-02). The randomised, double-blind, placebo-controlled trial was initiated in November 2020 and enrolled 63 patients in the US [11] . Earlier, in August 2020, the US FDA accepted the IND application for the trial. The submission of the application to the US FDA happened in the same month [12] [13] [14] [15] . In December 2020, Capricor Therapeutics announced that the independent Data and Safety Monitoring Board (DSMB) completed a safety review for the trial and recommended to continue the trial as designed. Additionally, an independent prespecified review of the safety data was conducted on an initial group of INSPIRE patients and the study was continuing according to the study protocol [16] . In March 2022, the company released the efficacy and safety data from the trial [17] .
In October 2020, Capricor Therapeutics completed an expanded access programme that evaluated the safety and afficacy of intravenous delivery of CAP 1002 in patients with COVID-2019 infection (NCT04338347). Earlier, in May 2020, the US FDA approved the Investigational New Drug (IND) application for the expanded access programme (EAP) 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. The company released the results in April 2020. In April 2021, the company released updated results from the expanded access programme [18] [19] [20] [21] [22] .
Cardiovascular disorders
As of March 2022, Capricr decided to discontinue the use of CAP 1002 in heart failure (the DYNAMIC Trial) and post myocardial infarction (MI) with cardiac dysfunction (the ALLSTAR trial) based on the decision to focus on Duchenne Muscular Dystrophy study [10] . In March 2022, Capricor completed enrollment in its phase II REGRESS trial [10] . 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 [23] .
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) [24] . 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 [25] [26] [27] [28] . 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 [29] . 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 [30] . Positive phase I data were reported in March, July, September and November 2014 [31] [32] [33] [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 [34] [32] [35] [36] .
In March 2022, Capricor has terminated the phase Ia/Ib DYNAMIC (Dilated cardiomYopathy iNtervention with Allogeneic MyocardIally-regenerative Cells) trial based on the decision to focus on Duchenne Muscular Dystrophy study [10] . The trial was initiated 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 [37] . 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 [38] [39] [26] [40] [41] .
Duchenne muscular dystrophy (DMD)
In February 2024, Capricor Therapeutics announced that it has been granted an in-person Type-B meeting with the U.S. Food & Drug Administration (FDA) scheduled for late March. In the meeting with the FDA, the Company intends to discuss its proposed chemistry, manufacturing and controls (CMC) plans for commercial launch, if approved which may enable opportunities to expedite the pathway to a Biologics License Application (BLA) submission [42] [43] . In September 2023, Capricor Therapeutics announced positive Type-B clinical meeting with the US FDA on the design and execution of phase III HOPE-3 trial in Duchenne muscular dystrophy (“DMD”). Feedback from the FDA on trial design and timeline confirms CAP-1002’s path towards a future Biologics License Application (“BLA”) submission [44] [45] [46] . Earlier, in May 2023, Capricor announced that it has submitted a request for a Type-B clinical meeting with the US FDA [47] . As of March 2023, Capricor announced that the Company had met with the US FDA in a Type-B CMC meeting to discuss the manufacturing plans in anticipation of a potential BLA application, Regenerative Medicine Advanced Therapy (RMAT) designation. In the meeting, Capricor and the FDA discussed the plans with respect to commercial manufacturing activities, including the potency assay and other product release criteria to support commercialization. The Company is currently awaiting the meeting minutes from the FDA and plans to request a follow-on Type B clinical meeting with the FDA and expect to have further clarity following that meeting on the topic. If Capricor were to receive market approval for CAP 1002 by the US FDA, the Company would be eligible to receive a Priority Review Voucher based on its designation as a rare pediatric disease [48] .
In May 2022, Capricor initiated a phase III HOPE-3 study to evaluate the safety and efficacy of CAP 1002 in patients with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function (NCT05126758; CAP-1002-DMD-04). The double-blind, randomised trial intends to enrol approximately 102 patients in the US [49] . In November 2021, the US FDA cleared the protocol for the phase III HOPE-3 trial which was submitted by Capricor Therapeutics in August 2021 [50] [51] [52] . In July 2022, Capricor announced commencement of dosing in the trial [53] . In November 2023, the company announced that cohort A has completed its targeted enrollment of 61 patients and expect to complete enrollment for cohort B of approximately 44 patients in the second quarter of 2024. Reported a positive outcome from the interim futility analysis for Cohort A which triggered the first milestone payment of $US10.0 million under our U.S. Distribution and Commercialization Agreement with Nippon Shinyaku [44] [54] [47] .
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 [19] .
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 [51] [55] .
In July 2020, Capricor Therapeutics initiated the phase II HOPE-2 open label extension (OLE) study to assess the safety and efficacy of four intravenous administrations of CAP 1002 in patients with Duchenne muscular dystrophy (NCT04428476; CAP-1002-DMD-02-OLE). Patients who completed the one-year follow-up in the HOPE-2 trial were eligible to receive CAP 1002 in the open label study. The trial intends to enrol approximately 13 patients in the US. In June 2022, efficacy data from the trial were released by the company [56] [57] . In July 2023, clinical data from the trial was released by the company [58] . In October 2023, the company released updated efficacy data from the trial [59] .
In April 2020, Capricor Therapeutics had 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 20 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 [19] [60] [61] [62] [63] [64] [65] [66] [67] [68] [69] [70] [71] . In November 2020, the company announced the release of final 12-month positive data from the trial at the International World Muscle Society Virtual Congress 2020 [72] . In May 2020, Capricor Therapeutics released final therapeutics and safety data form a phase I/II trial [73] . The final efficacy and safety data from the trial were released by Capricor Therapeutics and presented at World Muscle Society Virtual Congress (WMS-2021). The trial met primary efficacy endpoint of mid performance of upper limb (PUL) v1.2 [74] . In January 2023, clinical data from the trial was released by the company [75] .
In December 2018, Capricor met the US FDA 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 [65] . The meeting was a part of expedited review process under Regenerative Medicine Advanced Therapy (RMAT)designation [76] . 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 [77] .
The US FDA, in July 2017, granted a Rare Pediatric Disease Designation to CAP 1002 for treatment of DMD [78] .
In April 2015, CAP 1002 was granted orphan drug designation by the US FDA for the treatment of Duchenne muscular dystrophy associated cardiomyopathy [79] .
In March 2019, Capricor completed a phase II HOPE-OLE trial that assessed the safety and efficacy of two intravenous administrations of CAP-1002, each separated by three months (NCT06304064; CAP-1002-DMD-03). This open-label extension trial of the HOPE-Duchenne study was initiated in June 2018 and enrolled 8 participants in the US.
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 [73] [80] [81] [82] [83] [67] [84] [85] [86] [87] [88] [26] [89] [90] [30] [91] [92] [34] [93] [94] [95] [96] [97] .
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 [98] .
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 [99] [100] [101] .
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 [88] .
Financing information
In December 2023, interim futility analysis from the HOPE-3 trial, triggers first milestone payment under the Company’s U.S. Commercialization and Distribution Agreement with Nippon Shinyaku [102] . Earlier in January 2022, Capricor announced additional funding for the planned HOPE-3 trial which was secured through the commercialisation and distribution agreement with Nippon Shinyaku [50] .
In September 2023, Capricor Therapeutics entered into definitive agreements with Nippon Shinyaku and funds associated with Highbridge Capital Management for the issuance and sale of 4,935,621 shares of its common stock in a registered direct offering. The gross proceeds from this offering are expected to be approximately $US23 million. The company intends to use the net proceeds on research and development related to its product candidates [103] .
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 [104] .
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 [91] .
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 [105] .
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 [34] [106] .
Drug Properties & Chemical Synopsis
- Route of administration Intracoronary, IV
- Formulation Infusion, unspecified
- Class Exosome therapies, Stem cell therapies
- Target Cell
- Mechanism of Action Cell replacements
-
WHO ATC code
V03A-X (Other therapeutic products)
-
EPhMRA code
V3X (All Other Therapeutic Products)
Biomarkers Sourced From Trials
| Indication | Biomarker Function | Biomarker Name | Number of Trials |
|---|---|---|---|
|
cardiomyopathies |
Eligibility Criteria |
tissue factor pathway inhibitor (lipoprotein-associated coagulation inhibitor) Cystatin C |
|
|
cardiomyopathies |
Outcome Measure |
syndecan binding protein 2 Osteopontin Interleukin-10 (IL-10) IL1RL1 Galectin-3 (Gal-3) |
|
|
Chronic heart failure |
Detailed Description |
BNP |
|
|
Chronic heart failure |
Eligibility Criteria |
BNP |
|
|
cOVID 2019 infections |
Detailed Description |
Estrogen receptor alpha (ER alpha) Cardiac Troponin T C-reactive protein (CRP) BNP |
|
|
cOVID 2019 infections |
Eligibility Criteria |
Tumor necrosis factor alpha (TNF-alpha) myoglobin Interleukin-6 (IL-6) Interleukin-10 (IL-10) Interleukin 1 Beta (IL-1β) Interleukin 1 alpha (IL-1α) Ferritin Cardiac Troponin T Cardiac Troponin I C-reactive protein (CRP) |
|
|
dilated cardiomyopathy |
Outcome Measure |
Cardiac Troponin I |
|
|
Duchenne muscular dystrophy |
Eligibility Criteria |
tissue factor pathway inhibitor (lipoprotein-associated coagulation inhibitor) Cystatin C |
|
|
Duchenne muscular dystrophy |
Outcome Measure |
syndecan binding protein 2 Osteopontin Interleukin-10 (IL-10) IL1RL1 Galectin-3 (Gal-3) |
|
|
left ventricular dysfunction |
Eligibility Criteria |
CKM CKB Cardiac Troponin I |
|
|
myocardial infarction |
Eligibility Criteria |
CKM CKB Cardiac Troponin I |
|
|
respiratory tract infections |
Detailed Description |
Estrogen receptor alpha (ER alpha) Cardiac Troponin T C-reactive protein (CRP) BNP |
|
|
respiratory tract infections |
Eligibility Criteria |
myoglobin Interleukin-6 (IL-6) Cardiac Troponin T Cardiac Troponin I C-reactive protein (CRP) |
|
|
SARS-CoV-2 acute respiratory disease |
Eligibility Criteria |
Tumor necrosis factor alpha (TNF-alpha) Interleukin-6 (IL-6) Interleukin-10 (IL-10) Interleukin 1 Beta (IL-1β) Interleukin 1 alpha (IL-1α) Ferritin C-reactive protein (CRP) |
Biomarker
| Drug Name | Biomarker Name | Biomarker Function |
|---|---|---|
| CAP 1002 | BNP | Detailed Description, Eligibility Criteria |
| bone morphogenetic protein receptor, type II (serine/threonine kinase) | Detailed Description | |
| C-reactive protein (CRP) | Detailed Description, Eligibility Criteria | |
| Cardiac Troponin I | Eligibility Criteria, Outcome Measure | |
| Cardiac Troponin T | Detailed Description, Eligibility Criteria | |
| CKB | Eligibility Criteria | |
| CKM | Eligibility Criteria | |
| Cystatin C | Eligibility Criteria | |
| dsDNA Auto-antibodies | Detailed Description | |
| Estrogen receptor alpha (ER alpha) | Detailed Description | |
| Ferritin | Eligibility Criteria | |
| Galectin-3 (Gal-3) | Outcome Measure | |
| IL1RL1 | Outcome Measure | |
| Interleukin 1 alpha (IL-1α) | Eligibility Criteria | |
| Interleukin 1 Beta (IL-1β) | Eligibility Criteria | |
| Interleukin-10 (IL-10) | Eligibility Criteria, Outcome Measure | |
| Interleukin-6 (IL-6) | Eligibility Criteria | |
| myoglobin | Eligibility Criteria | |
| Osteopontin | Outcome Measure | |
| phenylalanine hydroxylase | Detailed Description | |
| RNA binding region (RNP1, RRM) containing 3 | Detailed Description | |
| syndecan binding protein 2 | Outcome Measure | |
| T-cell surface antigen CD4 | Eligibility Criteria | |
| tissue factor pathway inhibitor (lipoprotein-associated coagulation inhibitor) | Eligibility Criteria | |
| Tumor necrosis factor alpha (TNF-alpha) | Eligibility Criteria |
Development Status
Summary Table
| Indication | Qualifier | Patient Segment | Phase | Countries | Route / Formulation | Developers | Event Date |
|---|---|---|---|---|---|---|---|
| COVID 2019 infections | - | - | Phase II | USA | IV / Infusion | Capricor Therapeutics | 15 Nov 2020 |
| Cardiomyopathies | in patients with Duchenne Muscular Dystrophy (DMD)-related cardiomyopathy | In adolescents, In adults, In the elderly | Phase I/II | USA | Intracoronary / unspecified | Capricor | 01 Aug 2015 |
| Duchenne muscular dystrophy | - | In adolescents, In adults, In children, In the elderly | Phase III | USA | IV / Infusion | Capricor | 20 May 2022 |
| Heart failure | in patients with regression-heart failure with preserved ejection fraction | In adults, In the elderly | Discontinued (II) | USA | Intracoronary / Infusion | Medical University of South Carolina | 22 Mar 2022 |
| Myocardial infarction | - | - | Discontinued (II) | Canada, USA | Intracoronary / Infusion | Capricor Therapeutics | 22 Mar 2022 |
Priority Development Status
| Type | Region | Indication |
|---|---|---|
| Regenerative Medicine Advanced Therapy | USA | Duchenne muscular dystrophy |
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 |
| Nippon Shinyaku | Market Licensee | Japan, 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 III | - | 24 Sep 2021 |
Scientific Summary
Pharmacokinetics
COVID-2019 infections
Results from the from the compassionate care programme for CAP 1002 showed that the treatment caused a decrease in the white blood cell count, IL-6 levels, C-reactive protein, and/or reduced reliance on supplemental oxygen. The patients had received IV infusions of 150 million allogeneic cardiosphere-derived cells CAP 1002 [18] [22] .
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 [39] [88] [86] [41] .
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) [34] [29] . 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 [31] .
Duchenne muscular dystrophy
Phase II:
The final safety data obtained from a phase II HOPE-2 trial indicated that CAP 1002 was generally safe and well tolerated with an exception of two hypersensitivity reactions that were relieved using routine pre-medication regimen. There were no serious safety signals were identified [74] . 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 [73] [61] [63] [71] .
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 [84] [97] .
COVID-2019 infections
Phase II
In the phase II INSPIRE trial, CAP 1002 was safe, well tolerated and consistent with the historically observed safety profile of the therapy [17] [11] .
Results from the compassionate care program, reported no adverse events related to the administration of CAP 1002 [20] [22] .
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 [98] .
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 [94] .
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) [29] .
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 [24] [32] [33] [28] .
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) [38] . 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 [39] . 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 [86] [41] .
Duchenne muscular dystrophy (DMD)
Phase II
The phase II HOPE-2 open label extension trial met its primary endpoint at the one-year timepoint on the PUL v2.0 scale (p=0.02). Updated results measured by the Performance of the Upper Limb (PUL 2.0) showed that mean PUL 2.0 decline after 24-months of treatment with CAP 1002 was 2.8 points versus a 7.7-point decline on average observed over 24-months in the placebo patient group (delta change=4.9 points, p=0.021). The average rate of decline in CAP 1002-treated patients showed an attenuation of disease progression by approximately 64%. Additionally, CAP 1002 revealed clinically meaningful improvements in ameliorating cardiac function. The data showed that cardiac function, as measured by left ventricular ejection fraction (LVEF%) by MRI at the 24-month timepoint, improved in 67% of patients, compared to a steady decline in a comparable natural history population [59] . Earlier results from the trial demonstrated statistically significant benefit (p=0.021) after two years of treatment in the Performance of the Upper Limb (PUL v2.0) scale. Six of nine patients showed improvements in heart function with CAP-1002 treatment compared to their final assessment at the end of the HOPE-2 study [58] . Previously updated results showed statistically significant improvements (p=0.02) on the performance of the Upper Limb scale after 18 months. The OLE phase showed significant slowness in progression of the disease. Previously, treatment with CAP 1002 after 12 months demonstrated statistically significant improvements on the Performance of the Upper Limb (PUL version 2.0) scale for patients on CAP 1002 testing three different hypotheses of treatment benefit during the open label extension. The open-label extension treatment difference vs. HOPE-2 treatment difference was found to be 3.8 (0.5, 7.0) (p=0.023). The open-label extension vs. HOPE-2 placebo group difference was 2.3 (0.5, 4.1) (p=0.015). T open-label extension vs. HOPE-2 placebo group off-treatment (Placebo GAP) was observed to be 2.8 (0.8, 4.8) (p=0.006) [75] [56] [57] .
Phase
II: Updated results from the phase II HOPE-2 trial with CAP 1002 met primary efficacy endpoints in PUL 1.2 mid-level dimension favoring the treatment over placebo with change difference 2.6 (p=0.014). Cardiac MRI assessments showed improvements in heart function and structure with CAP 1002 treatment. Left ventricular ejection fraction (LVEF) decreased in the placebo group over time, but improved in the CAP 1002 group, showing a 107% slowing of progression of cardiac disease (p=0.002) [75] . The final 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 = 0.04) with a mean change of 1.8 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 = 0.02) with a mean change of 3.2 points over placebo patients, improvements in cardiac function as measured by ejection fraction was 4% (p = 0.002) and reduction in the biomarker CK-MB, an enzyme that is only released when there is cardiac muscle cell damage compared to placebo (p = 0.02) was -2.2%. The LV End Diastolic Volume, Indexed mL/m2 was-12.4‡ with p-value 0.03 and LV End Systolic Volume, Indexed mL/m2 was -4.2‡, p-value 0.01. One-Year results demonstrated CAP 1002 slowed decline by 71% (mid-level PUL v1.2) [74] . 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) [73] [19] [61] [63] [71] [72] .
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 [81] . 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 [82] [84] [97] .
COVID-2019 infections
Phase II
In the phase II INSPIRE trial, Overall mortality was observed to be 20%, with 6 deaths in the placebo group and 5 deaths in the CAP 1002 group (n=11; p=NS) [17] [11] .
Updated results from the from the compassionate care programme for CAP 1002 showed that of the seven patients hospitalized with severe COVID-2019 infection, six were on ventilator support. All patients were treated with CAP 1002. Four of the seven patients were fully discharged and three died between one- and two-months post-treatment. Earlier results from the compassionate care programme had demonstrated that 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 [18] [20] [22] .
Future Events
| Expected Date | Event Type | Description | Updated |
|---|---|---|---|
| 31 Dec 2025 | Regulatory Status | Capricor Therapeutics plans to file BLA for CAP 1002 in Duchenne muscular dystrophy in 2025 [45] | 06 Oct 2023 |
| 31 Mar 2024 | Regulatory Status | Capricor plans a meeting with the US FDA in the scheduled for late March of 2024. [54] | 29 Feb 2024 |
| 31 Jul 2023 | Regulatory Status | Capricor Therapeutics plans a follow-up Type B meeting with the US FDA to discuss proposed path towards submission of a potential BLA of CAP 1002 and further discuss its ongoing HOPE-3 clinical trial for Duchenne muscular dystrophy, in early 2023 [45] | 06 Oct 2023 |
| 31 Dec 2022 | Regulatory Status | Capricor Therapeutics requests and plans to meet with the US FDA to discuss next steps on pathway to approval of CAP 1002 for Duchenne muscular dystrophy, in 2022 [107] | 22 Mar 2023 |
| 30 Jun 2022 | Trial Update | Capricor Therapeutics plans pivotal phase III HOPE-3 trial for Duchenne muscular dystrophy in March 2022 and plans to initiate recruitment in the second quarter of 2022 (3649186) (NCT05126758) (700341458) [50] | 30 May 2022 |
| 30 Sep 2021 | Regulatory Status | Capricor Therapeutics plans to announce next steps of CAP 1002 for Duchenne muscular dystrophy, in the third quarter of 2021 [108] | 20 May 2021 |
| 15 Nov 2020 | Trial Update | Capricor Therapeutics plans the phase II INSPIRE trial in COVID-2019 infections in 2021 (700326520) [109] | 20 Jun 2022 |
| 31 Jul 2020 | Trial Update | Capricor plans a phase II open label extension of the HOPE-2 trial for Duchenne Muscular Dystrophy in USA, in July 2020 (700323145), (NCT04428476) | 19 Aug 2020 |
| 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) [110] | 03 May 2018 |
Development History
| Event Date | Update Type | Comment |
|---|---|---|
| 14 Nov 2023 | Regulatory Status | Capricor plans a meeting with the US FDA in the scheduled for late March of 2024. [54] Updated 29 Feb 2024 |
| 10 Oct 2023 | Scientific Update | Updated efficacy data from the phase II HOPE-2-OLE trial in Duchenne Muscular Dystrophy released by Capricor Therapeutics [59] Updated 13 Oct 2023 |
| 29 Sep 2023 | Regulatory Status | Capricor Therapeutics plans a follow-up Type B meeting with the US FDA to discuss proposed path towards submission of a potential BLA of CAP 1002 and further discuss its ongoing HOPE-3 clinical trial for Duchenne muscular dystrophy, in early 2023 [45] Updated 06 Oct 2023 |
| 29 Sep 2023 | Regulatory Status | Capricor Therapeutics plans to file BLA for CAP 1002 in Duchenne muscular dystrophy in 2025 [45] Updated 06 Oct 2023 |
| 29 Sep 2023 | Regulatory Status | Capricor Therapeutics plans to file BLA for CAP 1002 in Duchenne muscular dystrophy [45] Updated 06 Oct 2023 |
| 29 Sep 2023 | Regulatory Status | Capricor Therapeutics conducts positive Type-B clinical meeting with the US FDA for phase III HOPE-3 trial in Duchenne muscular dystrophy [45] Updated 06 Oct 2023 |
| 03 Jul 2023 | Scientific Update | Updated efficacy data from a phase II HOPE-2 trial was released by Capricor Therapeutics [58] Updated 04 Jul 2023 |
| 11 May 2023 | Regulatory Status | Capricor Therapeutics submits a request for a Type-B clinical meeting with the US FDA for CAP 1002 for Duchenne muscular dystrophy [47] Updated 17 May 2023 |
| 22 Mar 2023 | Regulatory Status | Capricor Therapeutics met with the US FDA in a Type-B CMC meeting to discuss next plans that support commercialization of CAP 1002 for treatment of Duchenne muscular dystrophy [48] Updated 22 Mar 2023 |
| 16 Feb 2023 | Licensing Status | CAP 1002 licensed to Nippon Shinyaku in Japan [4] Updated 24 Feb 2023 |
| 25 Jan 2023 | Scientific Update | Efficacy data from a phase II HOPE-2 and HOPE 2 OLE trial in Duchenne muscular dystrophy released by Capricor Therapeutics [75] Updated 30 Jan 2023 |
| 16 Nov 2022 | Regulatory Status | Capricor Therapeutics plans to submit BLA for CAP 1002 for the treatment of Duchenne muscular dystrophy Updated 16 Nov 2022 |
| 10 Aug 2022 | Regulatory Status | Capricor Therapeutics requests and plans to meet with the US FDA to discuss next steps on pathway to approval of CAP 1002 for Duchenne muscular dystrophy, in 2022 [107] Updated 22 Mar 2023 |
| 27 Jun 2022 | Scientific Update | Efficacy data from a phase II HOPE-2 open label extension (OLE) trial in Duchenne muscular dystrophy released by Capricor Therapeutics [56] Updated 01 Jul 2022 |
| 20 May 2022 | Phase Change - III | Phase-III clinical trials in Duchenne muscular dystrophy (In adolescents, In children, In the elderly, In adults) in USA (IV) (NCT05126758) [49] Updated 30 May 2022 |
| 28 Mar 2022 | Scientific Update | Efficacy and safety data from a phase II trial in COVID-2019 infections released by Capricor Therapeutics [17] Updated 31 Mar 2022 |
| 22 Mar 2022 | Phase Change - Discontinued(II) | Discontinued - Phase-II for Heart failure (In the elderly, In adults) in USA (Intracoronary) [10] Updated 22 Mar 2022 |
| 22 Mar 2022 | Phase Change - Discontinued(II) | Discontinued - Phase-II for Myocardial infarction in Canada (Intracoronary) [10] Updated 22 Mar 2022 |
| 22 Mar 2022 | Phase Change - Discontinued(II) | Discontinued - Phase-II for Myocardial infarction in USA (Intracoronary) [10] Updated 22 Mar 2022 |
| 22 Mar 2022 | Trial Update | Capricor Therapeutics completes enrolment in its phase II REGRESS trial for Heart failure in the US [10] (NCT02941705) Updated 22 Mar 2022 |
| 10 Mar 2022 | Trial Update | Capricor Therapeutics plans pivotal phase III HOPE-3 trial for Duchenne muscular dystrophy in March 2022 and plans to initiate recruitment in the second quarter of 2022 [10] (NCT05126758) [50] Updated 30 May 2022 |
| 04 Feb 2022 | Trial Update | Capricor completes the phase II INSPIRE trial in COVID-2019 infections in USA (IV) (NCT04623671) Updated 13 Apr 2022 |
| 30 Jan 2022 | Licensing Status | CAP 1002 licensed to Nippon Shinyaku in USA [2] [3] Updated 30 Jan 2022 |
| 09 Nov 2021 | Trial Update | Capricor Therapeutics completes enrolment in the phase II INSPIRE trial for COVID-2019 infections in USA [12] Updated 17 Nov 2021 |
| 29 Sep 2021 | Licensing Status | CAP 1002 is available for licensing as of 24 Sep 2021 [74] Updated 29 Sep 2021 |
| 24 Sep 2021 | Scientific Update | Final 12-month positive efficacy and safety data from the phase II HOPE-2 trial in Duchenne muscular dystrophy presented at World Muscle Society Virtual Congress (WMS-2021) [74] Updated 29 Sep 2021 |
| 12 Aug 2021 | Regulatory Status | Capricor Therapeutics submits phase III protocol to US FDA for Duchenne muscular dystrophy [51] Updated 20 Aug 2021 |
| 30 Jun 2021 | Biomarker Update | Biomarkers information updated Updated 17 Sep 2021 |
| 13 May 2021 | Regulatory Status | Capricor Therapeutics plans to announce next steps of CAP 1002 for Duchenne muscular dystrophy, in the third quarter of 2021 [108] Updated 20 May 2021 |
| 13 May 2021 | Regulatory Status | Capricor Therapeutics requests to meet with the US FDA to discuss next steps on pathway to approval of CAP 1002 for Duchenne muscular dystrophy [108] Updated 20 May 2021 |
| 14 Apr 2021 | Scientific Update | Updated efficacy and pharmacokinetics data from a clinical trial in COVID-2019 infections in USA released by Capricor Therapeutics [18] Updated 14 Apr 2021 |
| 14 Apr 2021 | Trial Update | Capricor plans phase III HOPE-3 trial for Duchenne Muscular Dystrophy in USA [18] Updated 14 Apr 2021 |
| 06 Jan 2021 | Licensing Status | Capricor and Johns Hopkins University enters into a nonexclusive license agreement or JHU Serology License Agreement for for COVID-2019 infections and is due to expire in July 2022 [10] Updated 25 Mar 2022 |
| 15 Nov 2020 | Phase Change - II | Phase-II clinical trials in COVID-2019 infections in USA (IV) (NCT04623671) Updated 25 Nov 2020 |
| 12 Nov 2020 | Trial Update | Capricor Therapeutics plans the phase II INSPIRE trial in COVID-2019 infections in 2021 [109] Updated 20 Jun 2022 |
| 07 Oct 2020 | Trial Update | Capricor completed clinical trials in COVID-2019 infections in USA (Parenteral) - compassionate use Updated 20 Oct 2020 |
| 01 Oct 2020 | Scientific Update | Capricor presented the final 12-month positive data from the phase II HOPE-2 trial at the International World Muscle Society Virtual Congress 2020 [72] Updated 15 Mar 2021 |
| 25 Aug 2020 | Regulatory Status | US FDA accepts IND for CAP 1002 for phase II clinical trial in COVID-2019 infections [15] Updated 27 Aug 2020 |
| 07 Aug 2020 | Regulatory Status | Capricor Therapeutics submits an IND application to the US FDA for phase II trial in COVID-2019 infections before August 2020 [14] Updated 11 Aug 2020 |
| 20 Jul 2020 | Trial Update | Capricor initiates the phase II HOPE-2-OLE trial for Duchenne Muscular Dystrophy in USA (IV) (NCT04428476) Updated 19 Aug 2020 |
| 17 Jun 2020 | Trial Update | Capricor plans a phase II open label extension of the HOPE-2 trial for Duchenne Muscular Dystrophy in USA, in July 2020 , (NCT04428476) Updated 19 Aug 2020 |
| 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 [19] 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 [19] Updated 20 May 2020 |
| 13 May 2020 | Regulatory Status | Capricor plans to meet the US FDA to discuss pathway to approval for CAP 1002 [73] [14] 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 [73] Updated 31 May 2020 |
| 29 Apr 2020 | Scientific Update | Safety and efficacy data from clinical trial in COVID-2019 infections released by Capricor Therapeutics [20] Updated 04 May 2020 |
| 29 Apr 2020 | Trial Update | Capricor Therapeutics plans a randomized, placebo-controlled trial for moderate and severe COVID-2019 infections [20] 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) [60] Updated 20 Apr 2020 |
| 03 Apr 2020 | Phase Change - Clinical | Clinical trials in COVID-2019 infections in USA (Parenteral) - compassionate use [21] (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 [21] 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 [61] Updated 14 Oct 2019 |
| 30 Sep 2019 | Trial Update | Capricor Therapeutics plans a phase III trial for Duchenne muscular dystrophy [55] 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 [63] 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 Mar 2019 | Trial Update | Capricor completes a phase I/II trial HOPE-OLE trial in Cardiomyopathy (In adolescents, In adults, In the elderly, In patients with Duchenne Muscular Dystrophy related cardiomyopathy) in USA (Intracoronary) (NCT06304064) Updated 15 Mar 2024 |
| 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 [64] 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 [64] 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 [65] 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 [65] Updated 29 Jan 2019 |
| 21 Jun 2018 | Trial Update | Capricor initiates enrolment in a phase I/II trial HOPE-OLE trial in Cardiomyopathy (In adolescents, In adults, In the elderly, In patients with Duchenne Muscular Dystrophy related cardiomyopathy) in USA (Intracoronary) (NCT06304064) Updated 15 Mar 2024 |
| 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) [70] 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 [77] 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 [6] 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 [67] Updated 30 Nov 2017 |
| 29 Nov 2017 | Regulatory Status | The US FDA approves IND application for CAP 1002 in Duchenne muscular dystrophy [67] 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) [81] Updated 22 Nov 2017 |
| 08 Nov 2017 | Regulatory Status | Capricor files an IND application with the FDA in USA for Duchenne muscular dystrophy [68] 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 [82] 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) [110] 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 the elderly, 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) [69] Updated 04 Aug 2017 |
| 18 Jul 2017 | Regulatory Status | CAP 1002 receives Rare Pediatric Disease Designation from the US FDA for Duchenne muscular dystrophy [78] Updated 20 Jul 2017 |
| 06 Jul 2017 | Licensing Status | Janssen Biotech terminates its options agreement for CAP 1002 [9] Updated 07 Jul 2017 |
| 12 May 2017 | Trial Update | Interim efficacy data from a phase II trial in Myocardial infarction released by Capricor Therapeutics [24] 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 [84] 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 [86] Updated 11 Nov 2016 |
| 05 Oct 2016 | Trial Update | Capricor Therapeutics completes enrolment in the phase II ALLSTAR trial for Myocardial infarction in USA [25] 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 [96] Updated 12 Sep 2016 |
| 15 Aug 2016 | Scientific Update | Twelve month efficacy data from the DYNAMIC trial in Cardiomyopathy released by Capricor Therapeutics [38] 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) [88] 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) [39] 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 [92] 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 [34] 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 [31] 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) [94] 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 [32] 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 [33] 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 [5] Updated 18 Jan 2018 |
| 28 Mar 2014 | Phase Change - II | Phase-II clinical trials in Myocardial infarction in Canada (Intracoronary) after March 2014 [25] 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 [29] 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 [27] 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
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Nippon Shinyaku and Capricor Therapeutics Enter into an Exclusive Partnership for Commercialization and Distribution of CAP-1002 for the Treatment* of Duchenne Muscular Dystrophy in US.
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Capricor Therapeutics and Nippon Shinyaku Enter Partnership for Exclusive Commercialization and Distribution of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy in Japan.
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Capricor Therapeutics Enters Into Exclusive License With Cedars-Sinai Medical Center for Exosome-Related IP Portfolio.
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Capricor Therapeutics and Janssen Biotech, Inc. Enter Into Collaboration Agreement and Exclusive License Option.
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Capricor Therapeutics Retains Full Rights to CAP-1002 as Janssen Biotech, Inc. Decides Not to Exercise Option.
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Capricor Completes Enrollment of Initial Phase of DYNAMIC Clinical Trial of Cardiosphere-Derived Cell Therapy for Treatment of Advanced Heart Failure.
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Capricor Therapeutics Announces Manufacturing Scale-Up of CAP-1002 Production at New San Diego Facility as it Continues Plans Toward Commercialization.
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Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial
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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
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Capricor Announces Positive Top-Line Final Results from HOPE-2 Study in Patients with Duchenne Muscular Dystrophy Treated with Lead Candidate CAP-1002.
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Capricor Therapeutics Announces Positive 18-Month Results from Ongoing HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy Patients.
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Capricor Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for Duchenne Muscular Dystrophy Therapy.
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Capricor Receives Rare Pediatric Disease Designation from FDA for CAP-1002 for Patients with Duchenne Muscular Dystrophy.
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Capricor Granted FDA Orphan Drug Designation for Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy.
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Significant Improvements Reported in Duchenne Muscular Dystrophy Patients Treated with Capricor's Investigational Cell Therapy.
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Capricor Therapeutics Presents Positive Six-Month Results in Duchenne Muscular Dystrophy at World Muscle Society International Congress.
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Capricor Announces 12-Month Results from HOPE-1 Trial in Duchenne Muscular Dystrophy to be Presented at AHA Late-Breaking Session.
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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.
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Capricor Therapeutics and CureDuchenne to Host Webinar on November 3, 2015 at 3:00 p.m. ET.
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Capricor Therapeutics Announces DSMB Recommends Continuation of HOPE-Duchenne Clinical Trial.
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Capricor Awarded $3.4 Million Grant from California Institute for Regenerative Medicine.
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Capricor Therapeutics Announces Positive Pre-Clinical Data for Cardiosphere-Derived Cells (CDCs) on Duchenne Muscular Dystrophy Cardiomyopathy.
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Capricor Announces Plans to Pursue Clinical Program for the Treatment of Duchenne Muscular Dystrophy With Cardiosphere-Derived Cells (CDCs).
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Capricor Therapeutics Completes Enrollment in Randomized HOPE Clinical Trial in Duchenne Muscular Dystrophy.
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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
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Capricor Announces New Pre-Clinical Study Finds Repeat Doses of CAP-1002 Lead to Enhanced Exercise Capacity in Duchenne Muscular Dystrophy Disease Model.
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Capricor to Present on Exosomes at the Plenary Session of the International Society for Cellular Therapy in Montreal.
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New Published Study Reports Cardiosphere-Derived Cells Improved Skeletal and Cardiac Muscle Function in Mouse Model of Duchenne Muscular Dystrophy.
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Capricor Therapeutics Reports Intravenous Administration of CAP-1002 Shown to be Effective in Preclinical Models of Duchenne Muscular Dystrophy.
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Capricor Therapeutics Announces Continuation of Phase 3 HOPE-3 Trial of CAP-1002 in Duchenne Muscular Dystrophy Based on Completion of Interim Futility Analysis.
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Capricor Therapeutics Announces $23 Million Registered Direct Offering.
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Capricor Therapeutics Provides Corporate Update and Reports First Quarter 2016 Financial Results.
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Cure Duchenne Ventures LLC To Provide $1 Million to Capricor Therapeutics to Advance Promising Research to Treat Heart Disease Associated with Duchenne Muscular Dystrophy.
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Capricor Therapeutics Announces $10 Million Private Placement of Common Stock.
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Capricor Therapeutics Reports Second Quarter 2022 Financial Results and Provides Corporate Update.
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Capricor Therapeutics Reports First Quarter 2021 Financial Results and Provides Corporate Update.
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Capricor Therapeutics Reports Third Quarter 2020 Financial Results and Provides Corporate Update.
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Capricor Therapeutics Advances Clinical Development Program in Duchenne Muscular Dystrophy with Naming of Principal Investigator.
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