A phase 1 and phase 1b study of CK0803 will soon be initiated following the FDA’s IND clearance.
The FDA has cleared Cellenkos’ investigational new drug application (IND) for CK0803, a T regulatory (Treg) cell therapy for the potential treatment of amyotrophic lateral sclerosis (ALS).1
Following the IND clearance, Cellenkos is planning to soon initiate a phase 1 safety study of CK0803. This will be followed by a phase 1b double-blind, placebo-controlled, randomized trial.
"This is an exciting opportunity to apply a promising, allogeneic, off-the-shelf, regulatory T cell therapeutic to the treatment of ALS," principal investigator Neil Shneider, MD, PhD, Director, Eleanor and Lou Gehrig ALS Center, Columbia University, said in a statement.1 "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."
The upcoming phase 1 study will primarily evaluate safety and tolerability of multiple doses of CK0803 administered intravenously in patients with ALS. The phase 1b study will further evaluate safety and will also look at clinical outcomes based on survival time and change in ALS Functional Rating Scale-Revised scores to assess CK0803’s impact on function and survival of patients with ALS.
CK0803 is a neurotropic, allogeneic, umbilical cord blood-derived Treg cell therapy developed using Cellenkos’ CRANE technology platform. CRANE, which stands for Cord Blood T-Regulatory Cells; Activated And Enriched, is designed to allow for cell enrichment and differentiation for specific indications. The Treg cells express neurotropic detection signals that allow them to migrate to inflammatory pockets inside the central nervous system. The CRANE platform also allows readily-available, off-the-shelf, use of the therapy. Additionally, CK0803 does not require HLA matching, immune suppression, or lymphodepletion prior to administration, which can be done in the outpatient setting.
"We are thrilled to have received IND clearance for our CK0803 program in ALS," Tara Sadeghi, chief operating officer, Cellenkos, added to the statement.1 "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."
Cellenkos also recently announced news on another of their candidates developed with the CRANE platform, CK0804. This Treg cell therapy received IND clearance in June 2022 to be evaluated as an add-on therapy to ruxolitinib in patients with myelofibrosis that had a suboptimal response to ruxolitinib alone in a phase 1b, open-label study.2 The study, named LIMBER-TREG108, will examine the safety of monthly doses of CK0804 as an add-on therapy. The trial is being launched in partnership with Incyte as part of their LIMBER initiative (Leading in MPNs Beyond Ruxolitinib).
"We are thrilled to have received IND clearance for our CK0804 program in myelofibrosis, marking our fourth IND Clearance as a company and our second hematological malignancy program to reach clinical development," Sadeghi said in earlier statement.2 "Clearance of this IND is a testament to the quality preclinical data supporting CK0804 through our internal R&D and our ongoing partnership with the University of Texas at MD Anderson Cancer Center as well as the strong chemistry, manufacturing, and control (CMC) and cGMP manufacturing capabilities we have developed internally... We are excited by the promise of CK0804 to offer a potentially transformative treatment for myelofibrosis patients."