FDA clears Cellenkos ALS drug for trial

amyotrophic lateral sclerosis ALS

Cellenkos, Inc., a clinical stage biotech company, has 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, 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.”

About amyotrophic lateral sclerosis

ALS is a rare and fatal neurological disease that primarily involves the nerve cells (neurons) responsible for the muscles that control limb movement as well as critical functions such as speech, swallowing, and breathing. Approximately 5,000 new patients are diagnosed with ALS every year and there are over 31,000 patients living with ALS in the U.S. Most patients with ALS die from respiratory failure, usually within three to five years after first onset of symptoms. Currently there is no cure and no effective treatment to halt or reverse the progression of this disease.

About Cellenkos’ CK0803

CK0803 is a novel allogeneic cell therapy product that contains robust, activated Treg cells that specifically carry neurotropic detection signals on its cell surface which allows for them to seek and travel to inflammatory pockets inside the central nervous system. 

Derived from clinical-grade umbilical cord blood units and manufactured using Cellenkos’ CRANE process, multiple doses of CK0803 can be generated from a single manufacturing campaign that can be stored in ready to use cryobags at subzero temperature for long period of time to be made readily available for patient use. 

CK0803 does not require any HLA matching and does not require immune suppression or lymphodepletion prior to its administration. CK0803 can be administered by intravenous infusion in the outpatient setting.

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