Cellenkos Unveils Phase 1b Data on CK0804 T-Regulatory Cell Therapy for Myelofibrosis at ASH 2024

Cellenkos, Inc., a biotechnology firm specializing in developing off-the-shelf, allogeneic T regulatory (Treg) cell therapies, has recently presented promising Phase 1b clinical trial results for its investigational therapy, CK0804. The findings were shared at the 66th Annual Meeting & Exposition of the American Society of Hematology (ASH) held in San Diego. CK0804 is being explored as a potential therapy for patients with myelofibrosis, particularly those who have not responded well to standard treatments such as JAK inhibitors.

Dr. Simrit Parmar, the founder of Cellenkos, emphasized the need for new therapeutic alternatives for myelofibrosis patients with inadequate responses to existing treatments. CK0804, enriched with CXCR4-enriched Treg cells, is designed to target bone marrow, addressing the inflammatory pathways that contribute to myelofibrosis. The trial results demonstrated a favorable safety profile and encouraging signs of efficacy in the patient cohort, prompting further evaluation through an expansion cohort.

The trial, titled "A Phase 1b, Open-Label Study of Add-on Therapy with CK0804 in Participants with Myelofibrosis and Suboptimal Response to Ruxolitinib," involved six infusions of CXCR4-enriched Treg cells administered every four weeks to nine patients. The treatment was well-tolerated, requiring no lymphodepletion or HLA matching, as the CK0804 Tregs were cryopreserved and thawed for infusion.

Key outcomes from the study included a notable reduction in spleen volume for four out of six evaluable patients, observed through MRI scans. Additionally, two patients saw a decrease in their monthly transfusion needs, and all participants reported improvements in symptoms such as reduced fatigue and early satiety. Biomarker analysis indicated a reduction in TGFβ levels, a pro-inflammatory cytokine, correlating with the overall clinical response. Further analysis suggested a promising decline in systemic inflammation.

In light of these encouraging results, Cellenkos plans to continue the study with an expansion cohort to explore a refined CK0804 dosing regimen. This expansion aims to address the heightened inflammation levels observed in myelofibrosis patients with poor responses to ruxolitinib, and patient enrollment is currently underway.

Cellenkos is dedicated to developing allogeneic Treg therapies, potentially treating a wide array of inflammatory and autoimmune disorders that require sustained immune modulation. Beyond CK0804, the company is advancing another candidate, CK0801, for aplastic anemia, which has shown positive efficacy, including achieving transfusion independence in some patients.

Myelofibrosis is a rare, progressive blood cancer characterized by scar tissue formation in the bone marrow, impairing normal blood cell production. Sufferers often experience severe symptoms like fatigue, spleen enlargement, and night sweats. With approximately 16,000 to 18,500 affected individuals in the U.S., those who do not respond well to current treatments face limited alternatives and a bleak prognosis. Inflammation plays a crucial role in the disease's development and progression.

CK0804 is an investigational therapy that utilizes the CXCR4/CXCL12 axis to mitigate inflammatory cytokines involved in myelofibrosis. Derived from clinical-grade umbilical cord blood and produced through Cellenkos' CRANE® process, CK0804 does not require HLA matching, offering a timely therapeutic option for patients in need. Administered intravenously, the therapy is suitable for outpatient settings.

Based in Houston, Texas, Cellenkos is committed to advancing Treg cell therapies derived from umbilical cord blood, aiming to deliver potent anti-inflammatory effects and lasting immune modulation without the need for donor matching. The company is focused on enhancing the lives of patients with rare and underserved conditions through the development of these innovative therapies.