First Patient With ALS Dosed With Treg Cell Therapy


CK0803 is being evaluated for safety and tolerability in the REGALS study.

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The phase 1 REGALS study (NCT05695521) evaluating the safety and tolerability of the regulatory T-cell (Treg) therapy CK0803 (Cellenkos) has dosed its first patient with amyotrophic lateral sclerosis (ALS).1

"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," said Tara Sadeghi, Chief Operating Officer of Cellenkos Inc.1 "ALS is a devastating disease with no cure, and 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."

The multicenter REGALS study, which stands for Regulatory T Cells for ALS, has its lead site at Columbie University. The study is primarily assessing safety and tolerability of multiple doses of CK083 in patients with ALS. The first, safety run-in part of the study will be conducted with 6 participants. The phase 1b part will also assess preliminary efficacy of CK0803 with measures including combined assessment of function and survival, change in the ALS Functional Rating Scale-Revised score, and a secondary endpoint of longitudinal measurement of neurofilament light chain levels in serum and cerebrospinal fluid in 60 participants that will receive 4 weekly infusions followed by 5 monthly infusions. CK0803 is an allogeneic, neutrophic, umbilical cord blood-derived Treg cell therapy developed with Cellenkos’ proprietary CRANE technology.

"We are excited to participate in this important study of this novel allogeneic Treg cell product in patients with ALS," principal investigator Neil Shneider, MD, PhD, associate professor, neurology and director, Eleanor and Lou Gehrig ALS Center, Columbia, said in a statement.1 "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, and we are grateful to Cellenkos for their commitment to applying their technology to ALS".

READ MORE: Stacy Lindborg, PhD, on Addressing Unmet Needs in ALS With Cell Therapy

Earlier this year, Cellenkos, along with its collaborator Incyte, dosed the first patient in the LIMBER-TREG108 trial (NCT05423691) evaluating another of the company’s Treg therapies, CK0804, in people with myelofibrosis. This study isstarting with an open-label safety run-in of 9 patients and will progress into an expansion cohort of 15 additional patients.2 The study’s primary endpoint is treatment-limiting toxicities, as defined by severe (grade 3 or 4) infusion-related toxicity within 24 hours of treatment that does not resolve with standard of care treatment within 72 hours, or treatment-related death within 28 days. Secondary outcome measures include overall response rate, rate of anemia response, rate of spleen response, and the percentage of patients with symptom reductions of at least 50% as measured by the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score.

"We are pleased that the first patient first dose milestone in the LIMBER-TREG108 study – one of several studies in our LIMBER clinical program evaluating multiple monotherapy and combination strategies to improve and expand treatments for patients with myeloproliferative neoplasms – has been achieved. We look forward to the results of the study, and to continuing our partnership toward important scientific advances for these patients," Ekaterine Asatiani, MD, Division Vice President and Head, Early Development, Incyte, added to the statement.2

1. Cellenkos Announces First Patient Dosed with CK0803 Cell Therapy for Treatment of Amyotrophic Lateral Sclerosis. News release. Cellenkos. May 2, 2023.
2. Cellenkos announces first patient dosed with CK0804 cell therapy in LIMBER-TREG108 clinical trial. News release. Cellenkos. January 6, 2023.
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