Physician-investigators at Johns Hopkins University Hospital report on the promising data of treatment of COVID-19 induced acute respiratory distress syndrome with allogeneic, cord blood-derived T-regulatory (Treg) cell therapy (manufactured by Cellenkos), published in a peer-reviewed journal of Annals of Internal Medicine. Both patients were critically ill and intubated (one on ECMO). Both had failed Tociluzimab (Actemra, Roche) and had a multiorgan failure. Patients received cell therapy under FDA Emergency Use IND for up to 3 doses. Clinical improvement was evident within 48 hours of the first infusion and correlated with concurrent dampening of the cytokine storm as demonstrated by a rapid decline in peripheral biomarkers including lactate, C-reactive protein, and Ferritin as well as decreased blood levels of inflammatory cytokines including IL-6, IFNϒ and TNFα.

"We are excited by these early data in very sick patients suffering from COVID-19 ARDS. We recognize that there are several factors at play but believe that the temporal relationship between Treg infusions and patient recovery cannot be ignored," said Dr. Douglas Gladstone, principal investigator at Johns Hopkins. "We look forward to evaluating this promising therapy in the FDA-approved randomized, double-blinded, placebo-controlled clinical trial of cryopreserved, allogeneic, off-the-shelf, cord blood derived T-regulatory cells (CK0802) in intubated patients suffering from COVID-19 ARDS. Planned correlative assays during this trial will provide insights into the mechanism of action of CK0802 and its relation to clinical outcomes."

The multicenter clinical trial is set to launch in Q3 2020, with patients assigned to treatment with multiple doses of CK0802 or placebo, with two co-primary outcomes of safety (no severe toxicity) and efficacy (alive and extubated at day 28). CK0802 will be manufactured by Cellenkos and shipped to the clinical site to be infused at the patient's bedside.

"We are very encouraged by these early clinical observations and remain fully committed to bringing forward this promising, potentially life-saving therapy into the market. We believe that our product will materially change the fatal outcome of COVID-19, allowing for the needed (necessary) time to develop an effective COVID-19 vaccine," said Tara Sadeghi, VP, Clinical Operations at Cellenkos. "Our company-owned cleanroom facility allows us to have full control of the manufacturing processes, supply chain, and distribution logistics. We are confident that we can deliver on this multicenter clinical trial."

Since 2017, the company has owned and operated an independent ISO-7 cleanroom manufacturing facility in Houston, engaged in process development and manufacture of clinical cell therapy products, including testing and quality control. Staffed with experienced personnel, the facility is equipped to support product supply for the clinical trials. Cellenkos already holds two FDA INDs for inflammatory bone marrow failure syndromes and demyelinating polyneuropathy. Cellenkos' CK0801 cell therapy product has demonstrated a high degree of safety in bone marrow failure syndromes, in the first two dose level cohorts completed to date at The University of Texas MD Anderson Cancer Center (NCT03773393).

Cellenkos Treg platform aims to develop tailored T-regulatory (Treg) cell therapeutics for various underserved inflammatory diseases and autoimmune disorders. The technology allows for varying degrees of immune responses against antigens of choice as well as immunotherapies for the treatment of cancer and rare diseases.

CK0802 is a novel allogeneic, off the shelf, cell therapy product consisting of Treg cells derived from clinical-grade umbilical cord blood units and manufactured using Cellenkos' proprietary process. The product is cryopreserved and readily available off-the-shelf, without any requirement for HLA matching, and is infused intravenously. One manufacturing campaign can generate multiple doses for infusion into several different patients. The multi-center trial of CK0802 will examine safety and efficacy in the treatment of COVID-19 ARDS. It is a cryopreserved product that will be manufactured by Cellenkos and shipped to the participating clinical site, where it can be thawed and infused at the patient's bedside.

Treg cells in CK0802 express lung homing markers on their cell surfaces. Once the cells reach the tissue, Treg cells are believed to disarm and dampen the cytokine storm by engaging with antigen-presenting cells including the pneumocytes that line the alveolar epithelium and drive the inflammatory reaction. Rather than indiscriminate therapy with a drug such as an inhibitor of a single cytokine such as IL-6, the T-regulatory cells can potentially calm inflammation exactly where it is most active, without causing a more general "global" immunosuppression that would be harmful in a virally infected patient.