Cellenkos to Present CK0804 Phase 1b Myelofibrosis Data at 2024 ASH Meeting

Cellenkos Inc., a clinical-stage biotechnology company specializing in T regulatory (Treg) cell therapies for inflammatory diseases and autoimmune disorders, announced that its CK0804 Phase 1b study data in myelofibrosis will be presented at the 66th Annual Meeting & Exposition of the American Society of Hematology (ASH) in December 2024 in San Diego, California.

The presentation will cover the safety and efficacy data from Cellenkos' Phase 1b trial of CK0804 in myelofibrosis patients with suboptimal responses to prior therapies, including ruxolitinib. CK0804 is a non-HLA matched, CXCR4-enriched Treg cell therapy administered in fixed doses of 100 million cells every 28 days for six doses. The therapy has shown early potential in addressing this high unmet medical need.

The study data reveals significant improvements in symptom burden, spleen volume reduction, blood transfusion dependence, and systemic inflammatory cytokine levels for patients treated with CK0804. Notably, CK0804 was well-tolerated, with no significant drug-related adverse events reported, except for one patient with a sulfa drug allergy who experienced an infusion reaction.

Nine patients with a median of two prior lines of treatment were included in the trial. All reported improvements in fatigue and overall symptom burden, with four out of six evaluable patients showing reductions in spleen volume. Two patients who were transfusion-dependent exhibited a decreased need for transfusions by the end of the treatment period. Longitudinal data indicates sustained benefits, including improved hemoglobin levels and inflammatory markers.

Following the initial success of the Phase 1b trial, the Data Safety Monitoring Board approved an expansion cohort to further explore the safety and efficacy of CK0804. This expansion includes an induction phase of four weekly doses, followed by five monthly doses, with active participant enrollment ongoing.

Myelofibrosis is a rare, chronic blood cancer that leads to scar tissue formation in the bone marrow, disrupting normal blood cell production. Patients often experience debilitating symptoms such as fatigue, spleen enlargement, and night sweats. Approximately 16,000 to 18,500 people in the U.S. are living with myelofibrosis, and those who do not respond adequately to current treatments face limited options and a poor prognosis. Inflammation is a key driver of disease progression.

CK0804 is an investigational, allogeneic, off-the-shelf Treg cell therapy that leverages the CXCR4/CXCL12 axis to suppress inflammatory cytokines implicated in myelofibrosis pathogenesis. Derived from clinical-grade umbilical cord blood and manufactured using Cellenkos' proprietary CRANE process, CK0804 does not require HLA matching, making it an ideal option for patients needing prompt intervention. The therapy is administered intravenously and can be infused in an outpatient setting.

Cellenkos, based in Houston, Texas, is focused on developing off-the-shelf Treg cell therapies for rare inflammatory diseases and autoimmune disorders. The company's portfolio includes four clinical-stage candidates: CK0801 for aplastic anemia, CK0802 for COVID-19 ARDS, CK0803 for amyotrophic lateral sclerosis (ALS), and CK0804 for myelofibrosis. Cellenkos' Treg therapies, derived from umbilical cord blood, are designed to provide powerful anti-inflammatory effects and long-lasting immune modulation without donor matching. The company is dedicated to advancing these therapies to improve the lives of patients with rare and underserved conditions.