Cellenkos Announces FDA Clearance for Trial of CK0802 for COVID-19 ARDS

Cellenkos announced the US Food & Drug Administration (FDA) has cleared the way to initiate a Phase 1 clinical trial of CK0802 (Cryopreserved Cord Blood Derived T-Regulatory Cells) for treatment of COVID-19 associated acute respiratory distress syndrome (ARDS). The trial is designed as a randomized, double-blinded, placebo-controlled study to assess safety and preliminary efficacy in this hospitalized patient population.

"We appreciate FDA's expedited review of our plans to evaluate CK0802 in critically ill, intubated patients suffering from ARDS, a deadly complication of COVID-19", said Elizabeth J Read, MD, Chief Technology Officer, Cellenkos. "Preliminary observations in two intubated COVID-19 ARDS patients, who received cryopreserved cord blood T-regulatory cells under FDA Emergency Use Authorization after failing tociluzumab, were promising. In the forthcoming Phase 1 randomized trial, CK0802 will be assessed for both toxicity and 28-day treatment success, as co-primary outcomes."

"Use of allogeneic, off-the-shelf cord blood-derived T-regulatory cells has emerged as a promising therapeutic strategy for the treatment of inflammatory disorders, specifically in terms of interrupting and arresting the cytokine storm unleashed by COVID-19 infection," said Dr Siddhartha Mukherjee, MD, PhD, Columbia University, New York, scientific advisor and collaborator on the multi-center clinical trial. "Rather than indiscriminate therapy with a drug such as an inhibitor of 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. Planned correlative assays during the clinical trial will provide insights into the mechanism of action of CK0802 and its relation to clinical outcomes."

"This is a great milestone for our company and highlights our core strength in rapid scale up and manufacture of novel cellular products", said Tara Sadeghi, VP, Clinical Operations at Cellenkos Inc. "CK0802 is an off-the-shelf, allogeneic T-regulatory cell product that is cryopreserved, shipped to the clinical site and infused at the patient's bedside. We are very excited to bring forward this promising, potentially life-saving therapy and look forward to contribute to the growing therapeutic armamentarium for COVID-19."

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 (NCT03773393).

Cellenkos Treg platform aims to develop T-regulatory (Treg) cell therapy for various underserved inflammatory diseases and autoimmune disorders. The technology can be tailored to induce varying degrees of immune responses against antigens of choice, potentially providing potent prophylactic vaccines for the prevention of infectious diseases, such as rabies, as well as immunotherapies for the treatment of cancer. The technology can also be adapted to avoid such immune activation for purposes of molecular therapies, thereby providing potential new therapeutic modalities for patients suffering from rare diseases.

CK0802 is a novel allogenic 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 result in multiple doses of cryopreserved product that can be shipped to the clinical site, where it can be stored for an extended period or made available for immediate treatment, as needed.

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 damp down the cytokine storm by engaging with antigen-presenting cells including the pneumocytes that line the alveolar epithelium and drive the inflammatory reaction.