- Safety profile established and encouraging efficacy signals demonstrated by reductions in spleen volume and symptom burden scores
- Data underscore the vast potential of allogeneic, non-HLA matched, T regulatory cell therapy program in addressing diseases driven by dysregulated inflammation
HOUSTON, Dec. 10, 2024 (GLOBE NEWSWIRE) -- Cellenkos, Inc., a clinical-stage biotechnology company focused on developing allogeneic, off-the-shelf, T regulatory (Treg) cell therapies for inflammatory diseases of high unmet needs and autoimmune disorders, presented new Phase 1b results highlighting the safety and efficacy of CK0804 for patients with myelofibrosis who did not respond to standard of care during the 66th Annual Meeting & Exposition of the American Society of Hematology (ASH), taking place in San Diego, CA, held from December 7 – 10, 2024.
“There is a significant need for new therapeutic options for patients living with myelofibrosis who have suboptimal responses to approved JAK inhibitors,” said Simrit Parmar, MD, Founder of Cellenkos. “CK0804 is a CXCR4-enriched Treg cell therapy product candidate engineered to home to the bone marrow and offers a differentiated approach to address dysregulated inflammatory pathways driving this debilitating disease. We are greatly encouraged by the safety profile and early signs of efficacy observed in this patient cohort and look forward to continuing our evaluation of the clinical potential of CK0804 in our planned expansion cohort.”
Oral Presentation Details:
Title: A Phase 1b, Open-Label Study of Add-on Therapy with CK0804 in Participants with Myelofibrosis and Suboptimal Response to Ruxolitinib
Abstract Number: 999
Oral Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Advancing MPN Care: Innovative Therapies and Clinical Breakthroughs in Myelofibrosis
Date and Time: Monday, December 9, 2024. 5:00 p.m. PST
Key Highlights
- Treatment Regimen: Six CXCR4-enriched Treg infusions (106 cells/infusion) were administrated every four weeks in the outpatient setting and were well tolerated (n=9)
- Conditioning: No lymphodepletion was administered. No HLA matching was performed. All CK0804 Tregs were cryopreserved and thawed and infused by the bedside.
- Spleen Volume Reduction: Four out of six evaluable patients showed spleen volume reduction when measured by MRI
- Transfusion Requirements: Two patients experienced a reduced need for monthly transfusions at the end of the study period
- Symptom Improvement: All treated patients reported overall improvement in symptom burden, including diminished fatigue and early satiety
- Biomarker Analysis: A reduction in levels of TGFβ, a pro-inflammatory cytokine, was observed and correlated with overall clinical response
- Systemic Inflammation: Additional analyses of multiple plasma inflammatory cytokines suggest a promising decline in systemic inflammation
Following these encouraging results, Cellenkos is advancing this Phase 1 study with the addition of an expansion cohort to evaluate a modified CK0804 dosing regimen to account for the high level of inflammation observed in myelofibrosis patients with suboptimal responses to ruxolitinib. This expansion cohort is now enrolling patients.
Cellenkos’ allogeneic, off-the-shelf, Treg therapies hold potential to treat a broad range of inflammatory and autoimmune conditions where long-term immune modulation is anticipated to durably modify the course of disease. In addition to CK0804, Cellenkos’ CK0801 candidate for aplastic anemia has shown promising efficacy, including transfusion independence in treated patients.
About Myelofibrosis
Myelofibrosis is a rare, chronic, and progressive blood cancer that causes scar tissue to form in the bone marrow, disrupting the production of normal blood cells. Patients with myelofibrosis 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 fail to respond adequately to current treatments including ruxolitinib, face limited options and a poor prognosis. Inflammation is a key driver for disease pathogenesis and progression in myelofibrosis.
About CK0804
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. CXCR4 enriched Tregs home faster to bone marrow compared to unmanipulated Tregs. Derived from clinical-grade umbilical cord blood and manufactured using Cellenkos' proprietary CRANE® process, CK0804 does not require HLA matching, making it an ideal therapeutic option for patients in need of prompt intervention. The therapy is administered intravenously and can be infused in an outpatient setting.
About Cellenkos, Inc.
Cellenkos is a clinical-stage biotechnology company based in Houston, Texas, focused on developing off-the-shelf Treg cell therapies for rare inflammatory diseases and autoimmune disorders. Cellenkos' Treg therapies, derived from umbilical cord blood, are designed to provide powerful anti-inflammatory effects and long-lasting immune modulation without the need for donor matching. The company is committed to advancing the development of these promising therapies to improve the lives of patients with rare and underserved conditions.
For more information, please visit www.cellenkosinc.com and follow us on LinkedIn and X.
Contact:
Media
Jason Braco, Ph.D.
LifeSci Communications
jbraco@lifescicomms.com
