First ALS patient dosed in Cellenkos’ trial of CK0803

Phase 1/1b trial will test the potential cell-based therapy for safety, tolerability

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by Andrea Lobo |

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Cellenkos has dosed the first patient in the Phase 1/1b clinical trial that’s evaluating its regulatory T-cell-based therapy CK0803 for the treatment of amyotrophic lateral sclerosis (ALS).

The patient will be one of six participants included in the Phase 1 run-in period of the REGALS clinical trial (NCT05695521), which will examine the therapy’s safety and tolerability before an additional 60 patients are added to the randomized Phase 1b part.

“The dosing of the first patient marks an exciting milestone for our company. The initiation of this study of CK0803 is an important achievement that brings us closer to delivering a potential new treatment which may have a life-changing impact for ALS,” Tara Sadeghi, chief operating officer at Cellenkos, said in a press release.

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Regulatory T-cells (Tregs) are immune cells that act as a so-called peacekeeper, helping to suppress excessive inflammatory responses, and contributing to maintain a healthy equilibrium of the immune system.

Previous studies have indicated that ALS patients have higher levels of inflammatory T-cells, but their Tregs are found at lower levels and also are less effective at dampening immune responses. This suggests that increasing Treg numbers and boosting their function may slow ALS disease progression and ease symptoms.

CK0803 is a donor-derived Treg-based treatment product that’s designed to travel preferentially to sites of inflammation in the central nervous system (the brain and spinal cord), where they can help to balance the immune response.

The cells in CK0803 are derived specifically from umbilical cord blood. This means it can be used as an off-the-shelf therapy in multiple patients without the need to match patients and donors.

CRANE technology

Cellenkos uses its proprietary CRANE technology to produce its Treg products, in which the collected cells are enriched and differentiated to become tailored to a treat a specific disease.

The technology also enables the production of multiple doses of CK0803 from a single umbilical cord blood unit. And the final product can be frozen and stored for long periods, being readily available for patients when needed in an outpatient setting.

“Promising data from six ALS patients previously treated with allogeneic cord blood Treg cells through an expanded access program at our Center supports the clinical development of CK0803,” said Neil Shneider, MD, PhD, principal investigator for the CK0803 clinical study.

”We are grateful to Cellenkos for their commitment to applying their technology to ALS,” added Shneider, who also is an associate professor of neurology and director of Columbia University’s Eleanor and Lou Gehrig ALS Center in New York.

The recently started clinical trial follows an approval by the U.S. Food and Drug Administration allowing Cellenkos to begin clinical testing of CK0803 in ALS patients.

Participants will receive the investigational cell-based therapy via into-the-vein (intravenous) infusion. The first four infusions will be given weekly, and additional infusions will be administered monthly for the next five months.

Two trial phases

The initial run-in part of REGALS will assess the safety and tolerability of multiple doses of CK0803. Then the placebo-controlled Phase 1b trial step will continue to examine safety and establish some preliminary signs of efficacy.

That phase’s main goal is to assess CK0803’s effect on the combined assessment of function and survival, a combined measure of survival and disease progression. Secondary measures include changes in neurofilament light chain levels, a marker of nerve damage, in the blood and spinal fluid.

Top-line data is expected by May 2026 and the trial is estimated to end by late 2027.

“We believe that CK0803 has the potential to provide a much-needed treatment option for patients. We are enthusiastic to move forward with this trial and to further explore the potential of CK0803 in ALS and other neurodegenerative diseases,” Sadeghi said.