Cellenkos, Inc., a clinical stage biotech company that focuses on developing transformative cellular therapeutics for treatment of inflammatory disorders and autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application to initiate a Phase 1 safety study followed by a Phase 1b randomized, double blind, placebo control trial of CK0803, neurotrophic allogeneic Treg cells, in patients with amyotrophic lateral sclerosis (ALS).
The primary objective of the upcoming Phase 1 study is to establish safety and tolerability of multiple doses of CK0803 administered intravenously in patients with ALS. The goal of the Phase 1b, randomized, double-blinded, placebo-control trial is to provide further safety data and evaluate the impact of CK0803 on the 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.
“This is an exciting opportunity to apply a promising, allogeneic, off-the-shelf, regulatory T cell therapeutic to the treatment of ALS,” said Neil Shneider, MD, PhD, Director of the Eleanor and Lou Gehrig ALS Center at Columbia University and principal investigator for the CK0803 program. “Cellenkos has made an extraordinary commitment to ALS and I am pleased to partner with them on this important therapeutic trial. ALS patients and families need a reason to be hopeful.”
“We are thrilled to have received IND clearance for our CK0803 program in ALS,” said Tara Sadeghi, Chief Operating Officer of Cellenkos. “With the addition of this new milestone, we now mark our fifth IND clearance as a company and an important neurology program to reach clinical development utilizing our proprietary Treg cell therapies. We are excited by the promise of CK0803 and are honored to work together with Dr Neil Shneider, world renowned neurologist and a leader in the field of neuro-muscular disorders, to bring forward a potentially transformative treatment for ALS patients.”