Type: Cardiac cell therapy consisting of allogeneic cardiosphere-derived cells. The cells are designed to function by releasing exosomes that are taken up largely by macrophages and T-cells and begin a cycle of repair.
2021 Status: Lonza has agreed to partner with Capricor in developing CAP-1002 for treating complications arising from COVID-19 as well as Duchenne Muscular Dystrophy (DMD), Capricor said January 12. The agreement, whose value was not disclosed, aims to expand Capricor’s manufacturing capacity for potential late-stage clinical trials and commercialization. Operations will begin with a tech-transfer to Lonza’s Houston center of excellence, where Lonza will perform process development activities for late-clinical and commercial-scale GMP manufacturing of CAP-1002.
2020 Status: Capricor said November 24 that the first two patients have been dosed in its Phase II INSPIRE trial (NCT04338347) assessing CAP-1002 in patients with severe COVID-19. INSPIRE is a randomized, double-blind, placebo-controlled study that will enroll up to 60 patients who have a confirmed diagnosis of SARS-CoV-2 and require supplemental oxygen. The study is being conducted at multiple sites in the United States and will enroll up to 60 patients.
In August, Capricor won FDA acceptance of its IND application for the INSPIRE trial. Its primary objectives are to determine the safety and effectiveness of intravenously infused CAP-1002 for improving clinical outcomes in severe to critical patients with COVID-19. Eligible subjects will be randomized to either the CAP-1002 or placebo group (1:1 ratio) and undergo baseline safety and efficacy assessments approximately one to three days prior to the administration of CAP-1002.
CAP-1002 has been studied in preclinical models of myocardial ischemia, myocarditis, heart failure, muscular dystrophy and pulmonary hypertension. Potential targets studied include pro-inflammatory pathways (TNF-α, interferon γ, IL-1, and IL-6) and anti-inflammatory pathways (regulatory T cells and IL-10).
Capricor has cited positive data from initial emergency use individual patient compassionate care cases: “Our ability to leverage the CAP-1002 program to treat the early to mid-stages of the cytokine storm in COVID-19 patients may reduce the need for ventilatory support and may potentially not only save lives but also reduce long-term morbidity that can occur after severe respiratory compromise,” stated Linda Marbán, PhD, Capricor’s president and CEO.
In April, Capricor said that all six critically ill COVID-19 patients suffering from acute respiratory distress syndrome (ARDS)—all but one of them on ventilators— were safely treated with CAP-1002 under compassionate use in the course of a month. Four of the six patients treated had been discharged. The fifth patient remained on mechanical ventilation and the sixth patient was receiving supplemental oxygen and was currently clinically stable as of the announcement date.
Five male patients and one female patient, all between ages 19 and 75, received IV infusions of CAP-1002 consisting of 150 million allogeneic cardiosphere-derived cells. Details of the compassionate use study were published May 5 in Basic Research in Cardiology. The paper is believed to be the first peer-reviewed report on the use of a cell therapy in critically ill COVID-19 patients, stated co-author Eduardo Marbán, MD, PhD, executive director of the Smidt Heart Institute at Cedars-Sinai, where the study took place.
Based on study results, the FDA approved the company’s expanded access protocol to treat up to 20 additional COVID-19 patients.
The patient outcomes, while encouraging, didn’t prove that CAP-1002 is safe and effective for treating COVID-19, investigators emphasized, as the study wasn’t a clinical trial with a control group. A large-scale study is now in late-stage planning.
Capricor has cited previously published preclinical data showing that CAP-1002 mitigated the release of anti-inflammatory cytokines as well as macrophage activation in a number of models of inflammation including sepsis and autoimmune diseases.
Capricor in May announced positive topline 12-month results from the Phase II HOPE-2 trial assessing CAP-1002 in advanced stages of Duchenne muscular dystrophy. The 12-month data from HOPE-2 showed statistically meaningful improvements in the PUL 2.0 in CAP-1002 treated patients (p=0.05) with a mean change of 2.4 points over placebo patients.
COVID-19: 300 Candidates and Counting
To navigate through the >300 potential therapeutic and vaccine options for COVID-19, GEN has grouped the candidates into four broad categories based on their developmental and (where applicable) clinical progress:
● FRONT RUNNER – the most promising therapeutics/vaccines based on clinical progress, favorable data or both.
● DEFINITELY MAYBE – earlier phases with promising partners, or more advanced candidates in development that have generated uneven data
● KEEPING AN EYE ON… – interesting technology, attracting notable partners, or both, but preliminary data.
● TOO SOON TO TELL – longshots pending additional experimental and/or clinical data.
GEN has also tagged the most common treatment types: