Cellenkos Approved to Proceed with CK0803 Neurotrophic T regulatory Cell therapy to Treat Second Cohort in ALS Trial

• DSMB approval to proceed to second cohort of REGALS trial (T Regulatory Cells for ALS).
• Ongoing Phase 1/1b study evaluating safety and efficacy of CK0803 (Neurotrophic T regulatory cells)

HOUSTON, Feb. 19, 2024 /PRNewswire/ -- Cellenkos^® today announced encouraging safety data from its innovative CK0803 neurotrophic T regulatory (Treg) cell therapy, being developed to help treat individuals with Amyotrophic Lateral Sclerosis (ALS). The company can now begin treating the second group of patients in the trial (clinicaltrials.gov: NCT05695521)

The company was given the green light to move ahead following a review of safety data by the Data Safety Monitoring Board (DSMB). The DSMB found none of the participants experienced any serious adverse events or dangerous side effects from the therapy.

"We are very excited and encouraged by this development," says Tara Sadeghi, Chief Operating Officer of Cellenkos. "This recommendation from the DSMB is an important step in bringing this potential disease modifying therapy to the patients and takes us closer to our ultimate goal of curing ALS."

CK0803 is a neurotrophic, allogeneic, umbilical cord blood-derived T regulatory (Treg) cell therapy that preferentially homes towards inflamed microglia, developed by using Cellenkos' proprietary CRANE^® technology, to generate disease-specific products. The completion of cohort 1 dosing allows for the continued enrollment on the Phase 1 Safety Run-in Study, to be followed by a Phase 1b Randomized, Double Blind, Placebo Control Trial of CK0803 in 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 including Columbia University, New York; Michael E. DeBakey VA Medical Center, Houston and Baylor College of Medicine, Houston. The primary objective of this study is to establish safety and tolerability of multiple doses of CK0803 in ALS patients. This study will also 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). 

About Amyotrophic Lateral Sclerosis (ALS)
According to the National Institute of Neurological Disorders and Stroke, amyotrophic lateral sclerosis (ALS) is a group of rare neurological diseases that mainly involve the nerve cells (neurons) responsible for controlling voluntary muscle movement. In ALS, both the upper motor neurons and the lower motor neurons degenerate or die and stop sending messages to the muscles. Unable to function, the muscles gradually weaken and waste away (atrophy). Eventually, the brain loses its ability to initiate and control voluntary movements. The disease is progressive, meaning the symptoms get worse over time. Majority of ALS cases (90 percent or more) are considered sporadic. This means the disease seems to occur at random with no clearly associated risk factors and no family history of the disease. In a small (approximately 10%) of cases, ALS is caused by a mutation in a known ALS-associated gene.

Most people with ALS eventually die from respiratory failure, usually within 3 to 5 years from when the symptoms first appear. However, about 10 percent of people with ALS survive for 10 or more years. Currently, there is no cure for ALS and no effective treatment to halt or reverse the progression of the disease.

About CK0803
CK0803 is a novel allogeneic cell therapy product consisting of T regulatory cells, with a high cell surface expression of CD11a, that leverage CXCR3/CXCL10 axis to engage with the inflamed microglia. CK0803 is derived from clinical-grade umbilical cord blood units and manufactured using Cellenkos' proprietary CRANE^® process. There is no requirement for HLA or ABO matching of CK0803 to the recipient. Multiple doses of CK0803 can be manufactured from a single umbilical cord blood unit, where the final cryopreserved product is readily available for use, at the point of care, which makes it an ideal therapy that can be infused intravenously, in the outpatient setting. 

About Cellenkos^®, Inc. 
Cellenkos is a clinical-stage biotechnology company located in Houston, Texas, USA, dedicated to the development and commercialization of the allogeneic, "off-the-shelf" cell-based products for the treatment of rare inflammatory diseases and autoimmune disorders. Cellenkos' T regulatory cells (Tregs) are derived from umbilical cord blood and as such are naïve, bonafide suppressor cells that do not require HLA or ABO matching and resolve inflammation through multiple direct and indirect mechanisms. Cellenkos' proprietary CRANE^® process allows for tailoring of Tregs to become disease specific. Cellenkos' current clinical portfolio includes CK0801 (bone marrow failure); CK0802 (COVID ARDS); CK0803 (ALS) and CK0804 (myelofibrosis). For more information, please visit www.cellenkosinc.com. 

Contact: 
Stacy Minor
[email protected] 

SOURCE Cellenkos, Inc.