Cellenkos Announces US FDA Orphan Drug Designation Granted to CK0801 (Allogeneic Cord Blood derived T regulatory Cell Product) for Treatment of Aplastic Anemia

• Orphan Drug Designation granted for Aplastic Anemia
• CK0801 is planned for Registration trial in H2 2025

HOUSTON, April 14, 2025 /PRNewswire/ -- Cellenkos^® Inc., a clinical-stage biotechnology company focused on developing allogeneic, off-the-shelf, T regulatory (Treg) cell therapies for inflammatory disease areas of high unmet needs and autoimmune disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its novel cell therapy product, CK0801.

This designation supports the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S, and qualifies a company for incentives, including tax credits, exemptions from certain FDA fees for clinical trials, and the potential for seven years of market exclusivity following drug approval. Aplastic Anemia has an incidence of 1.0-2.3 per million in the U.S and a projected prevalence of 5000 patients.

"Receiving Orphan Drug Designation for CK0801 in Aplastic Anemia underscores the importance of bringing novel, transformative treatment options to patients suffering from this rare disease", said Dr. Simrit Parmar, MD, Founder of Cellenkos. Dr Parmar is also faculty at The College of Medicine at Texas A&M University. "We aim to deliver a transformative therapeutic that can reduce the burden of blood and platelet transfusions in patients with Aplastic Anemia who have failed to respond to standard-of-care treatment."

The results of the Phase 1 clinical trial were published last year in NEJM Evidence, demonstrating that CK0801, an allogeneic T regulatory (Treg) cell therapy, leads to durable transfusion independence up to 3.5 years in patients with Aplastic Anemia, a rare and life-threatening bone marrow failure disorder. The trial (NCT03773393) also highlights the excellent safety profile of CK0801 and its potential to offer a novel, non-immunosuppressive treatment for patients who have not responded to conventional therapies. Specifically, patients were able to receive CK0801 infusions in an outpatient setting, intravenously through a peripheral intravenous line without any need for hospitalization and without any conditioning chemotherapy. The study, which included 9 patients with bone marrow failure syndromes, found that a single infusion of CK0801 Tregs resulted in a 67% overall response rate at the one-year follow-up. Notably, patients with Aplastic Anemia showed remarkable outcomes, with 75% of patients achieving partial responses, including two individuals who became transfusion-independent within months of treatment. These patients remained transfusion-independent for up to 41 months, marking a significant milestone in the treatment of Aplastic Anemia. CK0801, Cellenkos' lead asset, is advancing toward the filing of a registration trial intended to support regulatory approval for the treatment of transfusion- dependent Aplastic Anemia.

Key Study Findings

• Durable Transfusion Independence: Two out of three transfusion dependent patients with Aplastic Anemia achieved complete and durable transfusion independence, lasting up to 3.5 years.
• Safety Profile: CK0801 was well tolerated with no infusion reactions or severe adverse events, and no dose-limiting toxicities were observed. CK0801 does not require any additional conditioning regimen and/ or IL-2 supplementation
• Immunologic Impact: Infusion of CK0801 increased circulating Tregs and promoted Treg expansion within bone marrow, a key finding suggesting the therapy's potential to modulate the immune system and improve bone marrow function in patients with aplastic anemia.

Broader Potential Across Bone Marrow Failure Syndromes
Beyond Aplastic Anemia, the Phase 1 study also included patients with Myelofibrosis and Myelodysplastic Syndromes. In these patients, CK0801 induced meaningful improvements, including symptom relief, transfusion independence, and in one case, complete remission following an allogeneic stem cell transplant.

About Aplastic Anemia
Aplastic Anemia (AA) is a rare and serious condition where the bone marrow fails to produce sufficient blood cells, leading to severe complications such as bleeding, infection, and fatigue. Current treatments, including immunosuppressive therapy, growth factors and stem cell transplantation, are often inadequate or unsuitable for many patients. CK0801 offers a promising new approach by using Tregs to restore immune balance and promote bone marrow function.

About Cellenkos, Inc.
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.

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

Contact:
For Cellenkos: [email protected]

SOURCE Cellenkos, Inc.