Cellenkos Presented Results of Phase 1b Data for CK0804 T-Regulatory Cell Therapy in Myelofibrosis at ASH 2024

Innovative Cellular Therapy Demonstrated Safety and Potential Clinical Benefit at ASH 2024

Cellenkos, 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.

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.” – said Simrit Parmar, MD, Founder of Cellenkos.

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.

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

A Phase Ib, Open-Label Study of Add on Therapy with CK0804 in Participants with Myelofibrosis and Suboptimal Response to Ruxolitinib

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

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.

Further Reading:

Highlights from ASH24 Day 1

Highlights from ASH24 Day 2

Highlights from ASH24 Day 3

Highights from ASH24 Day 4

Best of ASH24

Jacobio Pharma Presented Preliminary Results from a Phase I Trial of Jab-8263 at ASH 2024