FDA Clears ALS Clinical Study of Cellenkos Treg Cell Therapy CK0803

Biotech plans to launch 2 Phase 1 trials testing therapy's safety, efficacy

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by Patricia Inácio, PhD |

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Cellenkos cleared for CK0803 study | ALS News Today | illustration of medicine bottle labeled clinical trials

Cellenkos will soon launch two Phase 1 clinical trials to investigate the safety and potential efficacy of its regulatory T-cell-based — Treg cell — therapy CK0803 for amyotrophic lateral sclerosis (ALS).

The biotech’s announcement follows the approval, by the U.S. Food and Drug Administration (FDA), of its investigational new drug (IND) application, which sought clearance to initiate clinical testing of CK0803 in ALS.

“We are thrilled to have received IND clearance for our CK0803 program in ALS,” Tara Sadeghi, chief operating officer of Cellenkos, said in a company press release.

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The first planned Phase 1 trial is designed to investigate the safety and tolerability of multiple doses of CK0803, administered as intravenous or into-the-bloodstream infusions, in people with ALS.

Then, a placebo-controlled Phase 1b trial will follow. This study will continue to evaluate the cell therapy’s safety, while also examining its impact on the combined assessment of function and survival (CAFS), a joint assessment of survival and disease progression.

Regulatory T-cells, also known as Tregs, are immune cells that keep the immune system in check and help dampen excessive inflammatory responses. In so doing, they help maintain a healthy immune balance.

Previous research suggested people with ALS have higher levels of pro-inflammatory immune T-cells accompanied by a reduction in Treg levels. Also, the remaining Tregs are less effective at suppressing immune system responses, which is associated with worse outcomes.

This suggests that boosting Treg numbers and function may slow ALS progression and lessen disease severity.

CK0803 is a donor-derived (allogeneic) cell therapy composed of activated Tregs designed to travel to sites of inflammation in the central nervous system, composed of the brain and spinal cord. Once there, the cells are expected to restore a so-called immune equilibrium.

The Tregs in CK0803 are specifically derived from umbilical cord blood, representing an “off-the-shelf” product that can potentially be used in numerous patients without the need to match patients and donors.

The cells are produced with Cellenkos’ proprietary CRANE technology, which enables multiple doses of CK0803 to be manufactured in a single run. Moreover, the cells can be frozen and stored for long periods of time, which would make them readily available for patients as needed.

“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, in New York, and the lead investigator for the CK0803 clinical 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,” he added.

No information was provided on a timeline for the trials.

“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,” Sadeghi said.