Cellenkos Receives FDA Clearance for Phase 2 Trial of CK0801 Allogeneic Treg Therapy in Aplastic Anemia

Cellenkos (搜索)搜索该公司, Inc., a clinical-stage biotechnology company developing allogeneic regulatory T cell (Treg) therapies, announced receipt of U.S. Food and Drug Administration (FDA) clearance to initiate a Phase 2 clinical trial of CK0801查看药品详情 for the treatment of aplastic anemia (搜索)搜索该疾病. The multicenter, open-label study (NCT07499102) will evaluate the safety and clinical efficacy of the cord blood-derived Treg therapy in transfusion-dependent patients with this rare blood disorder.

Addressing Critical Unmet Medical Need

Aplastic anemia (搜索)搜索该疾病 represents a significant therapeutic challenge, particularly for patients who fall outside the reach of current standard treatments. The rare and life-threatening blood disorder develops from an autoimmune attack on the bone marrow (搜索)搜索该靶点 due to unchecked immune imbalance, resulting from exposure to radiation, toxic chemicals, certain medications, and specific viral infections, though the cause remains unknown in many cases.

The condition is characterized by bone marrow (搜索)搜索该靶点 failure to produce essential red blood cells, white blood cells, and platelets, and can be fatal within three months if left untreated. While intensive immunosuppressive therapy and allogeneic bone marrow transplantation have saved many lives, a stark unmet need remains, especially for older Americans over 65 years of age and patients with minority ethnic backgrounds who have difficulty finding matching donors.

"The reality for many aplastic anemia (搜索)搜索该疾病 patients is a persistent state of medical fragility," said Simrit Parmar, MD, MSCI, Founder of Cellenkos (搜索)搜索该公司 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."

Phase 2 Trial Design and Endpoints

The Phase 2 study is designed to evaluate the safety and clinical activity of CK0801查看药品详情 in adults (≥18 years) with aplastic anemia (搜索)搜索该疾病 who have failed at least one prior line of therapy or are intolerant to standard treatments and are dependent on red blood cell and/or platelet transfusions. The primary endpoint is a 30% reduction in transfusion requirements at Day 180.

Mechanism of Action: "Supercharged Tregs"

In aplastic anemia (搜索)搜索该疾病, the body's own regulatory T cells (搜索)搜索该靶点 that normally keep the immune system in check are often decreased in number and impaired in function, allowing unopposed autoimmune attack on the bone marrow (搜索)搜索该靶点. CK0801查看药品详情 is derived from healthy, allogeneic cord blood and manufactured using Cellenkos (搜索)搜索该公司' proprietary CRANE technology to generate "Supercharged Tregs" designed to overcome inflammatory chaos through four key mechanisms:

• Resolve bone marrow inflammation: Decreases harmful monocytes and cytotoxic CD8+ T cells and interrupts the vicious cycle of immune-mediated destruction of hematopoietic stem cells
• Reset immune system: Support recovery of the patient's own regulatory T cell function and retrain them for long-term protection
• Restore homeostasis: Foster a protected microenvironment in which stem cells may regenerate and produce healthy blood cells
• Reduce transfusion burden: Potentially enable patients to achieve durable transfusion independence in responders

CK0801查看药品详情 is designed to act as an immunological "peacekeeper," calming the immune attack on the bone marrow (搜索)搜索该靶点 and creating conditions intended to support hematopoietic recovery, regardless of the initial trigger of the disease.

Promising Phase 1 Results

Phase 1 results for CK0801查看药品详情 in bone marrow (搜索)搜索该靶点 failure syndromes were published in NEJM Evidence (2024). The study enrolled 9 patients (4 with aplastic anemia (搜索)搜索该疾病, 4 with myelofibrosis (搜索)搜索该疾病, and 1 with hypoplastic myelodysplastic syndrome (搜索)搜索该疾病), with CK0801 administered intravenously through peripheral line in the outpatient setting, without lymphodepleting chemotherapy or interleukin-2 (IL-2) supplementation.

The safety profile was favorable, with no infusion reactions, no dose-limiting toxicities, and no Grade 3 or 4 severe adverse reactions attributable to CK0801查看药品详情 reported. Among aplastic anemia (搜索)搜索该疾病 patients, at 12-month assessment, 3 of 4 achieved a partial response, and 2 of 3 patients who were transfusion-dependent at baseline achieved durable transfusion independence.

"FDA clearance to advance CK0801查看药品详情 into the Phase 2 trial is a pivotal milestone for Cellenkos (搜索)搜索该公司 and, more importantly, for patients living with aplastic anemia (搜索)搜索该疾病," said Tara Sadeghi, Chief Operating Officer, Cellenkos. "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."

Manufacturing and Regulatory Status

CK0801查看药品详情 is manufactured from a single allogeneic umbilical cord blood unit collected and banked from a healthy normal donor who has met FDA requirements for screening and testing for transmissible disease, in compliance with 21 CFR Part 1271, Subpart C. The FDA previously granted Orphan Drug Designation to CK0801 for the treatment of aplastic anemia (搜索)搜索该疾病, reflecting the significant unmet medical need in this patient population.

Cellenkos (搜索)搜索该公司 was granted US patent (US12472182B2) on November 18, 2025, for their technology covering "Compositions comprising regulatory t cells (搜索)搜索该靶点 and methods of" producing and using them. Using its proprietary CRANE platform, the company is developing a pipeline of "off-the-shelf" Treg cell product candidates designed to suppress pathological inflammation and support tissue repair and regeneration.