Cellenkos, Inc. Announces FDA Clearance to Initiate Phase 2 Clinical Trial of CK0801 (Allogeneic Cord Blood-Derived Tregs) for Aplastic Anemia

Phase 2 multicenter, open-label study (NCT07499102) will evaluate the safety and clinical efficacy of CK0801 Tregs in transfusion-dependent aplastic anemia.

HOUSTON, April 27, 2026 /PRNewswire/ -- Cellenkos^®, Inc., a clinical-stage biotechnology company, developing allogeneic, tissue-targeted regulatory T cell (Treg) therapies, today announced the receipt of U.S. Food and Drug Administration (FDA) clearance to initiate CK0801 Phase 2 clinical trial, for the treatment of aplastic anemia.

Aplastic anemia, a rare and life-threatening blood disorder, continues to present a significant challenge for thousands of patients who fall outside the reach of current standard treatments. Aplastic Anemia develops because of an autoimmune attack on the bone marrow, due to unchecked immune imbalance, resulting from exposure to radiation, toxic chemicals, certain medications, and specific viral infections, though in many cases the cause remains unknown. Characterized by bone marrow's failure to produce essential red blood cells, white blood cells, and platelets, the condition can be fatal within just three months if left untreated. While medical advancements such as intensive immunosuppressive therapy and allogeneic bone marrow transplantation have saved many lives, a stark "unmet need" remains, especially for older Americans > 65 years of age, and patients with minority ethnic background who have difficulty finding a matching donor. These patients are often forced into a lifelong cycle of chronic blood and platelet transfusions. This dependency, coupled with frequent hospitalizations due to severe infections, leads to a drastically diminished quality of life.

"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."

About the CK0801 Phase 2 Study

The Phase 2 multicenter, open-label study (NCT07499102) is designed to evaluate the safety and clinical activity of CK0801 in patients with aplastic anemia

• Target Population: Adults (≥18 years) with aplastic anemia 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.
• Primary Endpoint: 30% reduction in transfusion requirements at Day 180.

How can CK0801 treat Aplastic Anemia?

In aplastic anemia, body's own regulatory T cells — that normally keep the immune system under check — are often decreased in number and impaired in function, allowing for the un-opposed autoimmune attack on the bone marrow. Derived from healthy, allogeneic, cord blood, CK0801 is manufactured using Cellenkos' proprietary CRANE^® technology to generate "Supercharged Tregs" that can overcome the inflammatory chaos, by driving Four Rs:

1. 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.
2. Reset Immune system. Support the recovery of the patient's own regulatory T cell function and retrains them to start working well for long term protection
3. Restore homeostasis: Foster a protected microenvironment in which stem cells may regenerate and produce healthy blood cells
4. Reduce transfusion burden: Potentially enable patients to achieve durable transfusion independence in responders.

"FDA clearance to advance CK0801 into the Phase 2 trial is a pivotal milestone for Cellenkos and, more importantly, for patients living with aplastic anemia," 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. We look forward to further evaluating CK0801 as a potentially non-toxic, transformative and curative therapy that can bring meaningful improvements in patient lives"

CK0801 is designed to act, in effect, as an immunological "peacekeeper" — calming the immune attack on the bone marrow and creating conditions intended to support hematopoietic recovery, regardless of the initial trigger of the disease.

CK0801 Phase 1 Trial Outcomes

Phase 1 results for CK0801 in bone marrow failure syndromes were published in NEJM Evidence (2024). In that study of 9 enrolled patients (4 with aplastic anemia, 4 with myelofibrosis, and 1 with hypoplastic myelodysplastic syndrome), CK0801 was administered intravenously through peripheral line, in the outpatient setting, without lymphodepleting chemotherapy or interleukin-2 (IL-2) supplementation. No infusion reactions, no dose-limiting toxicities, and no Grade 3 or 4 severe adverse reactions attributable to CK0801 were reported. Among aplastic anemia patients, at 12 months assessment, 3 of 4 achieved a partial response, and 2 of 3 patients who were transfusion-dependent at baseline achieved durable transfusion independence.

About CK0801

CK0801 is manufactured from a single allogeneic umbilical cord blood unit (CBU) 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, reflecting the significant unmet medical need in this patient population. CK0801 remains investigational and has not been approved by the FDA for any indication.

About Cellenkos, Inc.

Cellenkos^®, Inc. is a clinical-stage biotechnology company developing allogeneic, tissue-targeted regulatory T cell (Treg) therapies for autoimmune diseases and inflammatory disorders. Cellenkos was granted US patent (US12472182B2) on November 18, 2025, for their technology covering "Compositions comprising regulatory t cells 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.

For more information, please visit www.cellenkosinc.com.

Media and Investor Contact: [email protected]

Forward-Looking Statements

This press release contains forward-looking statements regarding the clinical development of CK0801. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected.

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