Cellenkos has received the US Food and Drug Administration (FDA) clearance to begin its Phase II clinical trial of CK0801 to treat aplastic anaemia.
The open-label, multi-centre trial will evaluate CK0801’s safety and clinical activity in adults with this condition.
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It will include patients who are refractory to at least one prior therapy, intolerant to standard treatments, or transfusion-dependent for red blood cells and/or platelets.
The primary endpoint is a 30% reduction in transfusion requirements by day 180.
CK0801 has been developed with Cellenkos’ CRANE technology. The therapy seeks to resolve bone marrow inflammation, reset and restore immune homeostasis, and ultimately reduce patients’ reliance on transfusions.
Aplastic anaemia is characterised by bone marrow’s failure to produce essential blood components, potentially leading to death in as little as three months without intervention.
Cellenkos chief operating officer Tara Sadeghi said: “FDA clearance to advance CK0801 into the Phase II trial is a pivotal milestone for Cellenkos and, more importantly, for patients living with aplastic anaemia.
“Our Phase I experience with CK0801 showed a favourable safety profile and early signals of durable clinical activity, with follow-up of up to three and a half 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 I findings were published in the New England Journal of Medicine (NEJM) Evidence in 2024. It enrolled nine patients with bone marrow failure syndromes. CK0801 was administered intravenously in the outpatient setting without additional conditioning regimens.
No dose-limiting toxicities or severe adverse reactions attributable to CK0801 were observed.
In February 2024, Cellenkos received approval to advance to the second cohort of patients in its clinical trial of CK0803 for ALS treatment.
