Cellenkos^® Inc., a clinical stage biotechnology company focused on developing allogeneic, off-the-shelf, T regulatory cell therapies for treatment of rare inflammatory diseases and autoimmune disorders, announced today that it has dosed the first patient in a Phase 1/1b study evaluating CK0803 for treatment of amyotrophic lateral sclerosis (ALS) (under National Clinical Trial registry NCT05695521). CK0803 is a neurotrophic, allogeneic, umbilical cord blood-derived T regulatory (Treg) cell therapy that preferentially homes towards central nervous system, developed by using Cellenkos' proprietary CRANE^TM technology, to generate disease-specific products. The dosing of this first patient marks the beginning of a Phase 1 Safety Run-in Study of 6 patients, to be followed by a Phase 1b Randomized, Double Blind, Placebo Control Trial of CK0803 in an additional 60 patients with ALS. The treatment will include four weekly infusions followed by five monthly infusions.

Regulatory T Cells for Amyotrophic Lateral Sclerosis (REGALS) is a multicenter study with a lead site at Columbia University, led by principal investigator Neil Shneider, M.D., PhD., Associate Professor of Neurology and director of Columbia's Eleanor and Lou Gehrig ALS Center. The primary objective of the Phase 1 study is to establish safety and tolerability of multiple doses of CK0803 in ALS patients. In addition, the Phase 1b study will provide preliminary efficacy data of CK0803 in ALS using the primary endpoint of combined assessment of function and survival (CAFS) that ranks patients' clinical outcomes based on survival time and change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score, and secondary endpoint of longitudinal measurement of neurofilament light chain levels in serum and cerebrospinal fluid (CSF). 

"The dosing of the first patient marks an exciting milestone for our company. The initiation of this study of CK0803 is an important achievement that brings us closer to delivering a potential new treatment which may have a life-changing impact for ALS," said Tara Sadeghi, Chief Operating Officer of Cellenkos Inc. "ALS is a devastating disease with no cure, and we believe that CK0803 has the potential to provide a much-needed treatment option for patients. We are enthusiastic to move forward with this trial and to further explore the potential of CK0803 in ALS and other neurodegenerative diseases."

"We are excited to participate in this important study of this novel allogeneic Treg cell product in patients with ALS," said Dr. Shneider. "Promising data from six ALS patients previously treated with allogeneic cord blood Treg cells through an expanded access program at our Center supports the clinical development of CK0803, and we are grateful to Cellenkos for their commitment to applying their technology to ALS".