FDA clears Cellenkos IND application for potential ALS treatment

Following FDA clearance, Cellenkos will initiate Phase I and Phase Ib trials of CK0803, allogeneic regulatory T cells, in patients with ALS.

ALS neurons

Cellenkos has announced that the US Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application to initiate a Phase I safety study of CK0803, neurotrophic allogeneic regulatory T cell (Treg) cells, in patients with amyotrophic lateral sclerosis (ALS). The FDA has also approved a subsequent Phase Ib randomised, double blind, placebo control trial.

The primary objective of the upcoming Phase I study is to establish safety and tolerability of multiple doses of CK0803 administered intravenously in patients with ALS. The goal of the Phase Ib 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.

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. 

“We are thrilled to have received IND clearance for our CK0803 programme 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 programme to reach clinical development utilising our proprietary Treg cell therapies. We are excited by the promise of CK0803 and are honoured 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.”

“This is an exciting opportunity to apply a promising, allogeneic, off-the-shelf, Treg therapeutic to the treatment of ALS,” said Dr Neil Shneider, Director of the Eleanor and Lou Gehrig ALS Center at Columbia University and principal investigator for the CK0803 programme. “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.”

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