Cellenkos To Begin Phase 2 Cord Blood-Derived Treg Trial for Aplastic Anemia
Following their successful Phase 1, they've received clearance to begin Phase 2, where they'll assess whether it can reduce transfusion needs by calming the immune attack on bone marrow.
Key Points
- Cellenkos has received FDA clearance to start a Phase 2 trial of CK0801 for transfusion-dependent aplastic anemia.
- The open-label, multicenter study will assess safety and whether the therapy can reduce red blood cell and platelet transfusion needs by Day 180.
- Phase 1 data cited by the company showed no infusion reactions or dose-limiting toxicities, with early signs of response in aplastic anemia patients.
Cellenkos is a Houston-based company working on allogeneic cell therapies for autoimmune and inflammatory conditions.
The company just received FDA clearance to begin a Phase 2 for CK0801, its allogeneic cord blood-derived regulatory T cell therapy for aplastic anemia. The multicenter, open-label study will evaluate safety and clinical activity in adults with aplastic anemia who have failed at least one prior line of therapy or cannot tolerate standard treatment and remain dependent on red blood cell and/or platelet transfusions.
For those unaware, aplastic anemia is a rare bone marrow failure disorder in which the immune system attacks the bone marrow, reducing production of red blood cells, white blood cells, and platelets. Patients who are not candidates for standard immunosuppressive therapy or allogeneic (donor derived) bone marrow transplant can face ongoing transfusions, infections, and repeated hospital care. The Phase 2 study’s primary endpoint is a 30% reduction in transfusion requirements at Day 180.
“The reality for many aplastic anemia patients is a persistent state of medical fragility,” said Simrit Parmar, MD, MSCI, Founder of Cellenkos and Adjunct Faculty at Texas A&M University School of Engineering Medicine (EnMed). “We are seeing a massive gap in care where traditional ‘gold standard’ treatments simply aren’t an option, leaving these individuals in a cycle of supportive care rather than a true recovery.”
CK0801 mechanism of action
CK0801 is made from a single allogeneic umbilical cord blood unit and consists of regulatory T cells, or Tregs. In aplastic anemia, these immune-regulating cells are often reduced in number and impaired in function, which allows the autoimmune attack on the bone marrow to continue. Cellenkos says CK0801 is designed to restore immune balance and support hematopoietic recovery.
The company describes four intended effects of CK0801:
- Resolve bone marrow inflammation by reducing harmful monocytes and cytotoxic CD8+ T cells
- Reset the immune system by supporting recovery of the patient’s own regulatory T cell function
- Restore homeostasis in the bone marrow microenvironment so blood-forming stem cells may recover
- Reduce transfusion burden, with the goal of durable transfusion independence in responders
Tara Sadeghi, Chief Operating Officer of Cellenkos said, “Our Phase 1 experience with CK0801, showed a favorable safety profile and early signals of durable clinical activity, with follow-up of up to 3.5 years of transfusion independence in individual patients. We look forward to further evaluating CK0801 as a potentially non-toxic, transformative and curative therapy that can bring meaningful improvements in patient lives”
Phase 1 data
The previous Phase 1 results were published in NEJM Evidence in 2024. The study enrolled nine patients with bone marrow failure syndromes, including four with aplastic anemia, four with myelofibrosis, and one with hypoplastic myelodysplastic syndrome. CK0801 was given by intravenous infusion in the outpatient setting, without lymphodepleting chemotherapy or interleukin-2 supplementation.
The company highlighted these results:
- No infusion reactions were reported
- No dose-limiting toxicities were reported
- No Grade 3 or 4 severe adverse reactions attributable to CK0801 were reported
- Among aplastic anemia patients, 3 of 4 achieved a partial response at 12 months
- Among the three aplastic anemia patients who were transfusion-dependent at baseline, two achieved durable transfusion independence
CK0801 has also received Orphan Drug Designation from the FDA for aplastic anemia, and they have candidates in the pipeline for ALS, Parkinson’s, ARDS, Lupus, and many more. You can see their pipeline on their homepage here.
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