Cellenkos' CK0801 Receives FDA Orphan Drug Status for Aplastic Anemia Treatment

Cellenkos Inc., a biotechnology firm headquartered in Houston, has announced a significant milestone in the development of its innovative cell therapy product, CK0801. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to CK0801, which is intended for the treatment of Aplastic Anemia, a rare and life-threatening disorder characterized by the failure of the bone marrow to produce adequate blood cells. This designation is crucial as it offers various incentives to the company, such as tax credits and exemption from certain FDA fees during clinical trials, along with the possibility of obtaining seven years of market exclusivity after the drug's approval.

Aplastic Anemia is a rare condition affecting between 1.0 to 2.3 individuals per million in the United States, with approximately 5,000 people currently living with the disorder. This grant of Orphan Drug Designation underscores the importance of bringing new, innovative treatments to patients who suffer from this debilitating disease. Dr. Simrit Parmar, the founder of Cellenkos and a faculty member at Texas A&M University’s College of Medicine, emphasized the transformative potential of CK0801. The goal is to offer a new treatment that can reduce the reliance on blood and platelet transfusions for those who have not responded to existing therapies.

CK0801 is an allogeneic T regulatory (Treg) cell therapy designed to be used in treating inflammatory diseases and autoimmune disorders. The promising results from a Phase 1 clinical trial were published in NEJM Evidence, demonstrating that CK0801 can lead to stable transfusion independence for up to 3.5 years in patients with Aplastic Anemia. The trial involved nine participants with bone marrow failure syndromes, showing that a single infusion led to a 67% overall response rate after one year. Among the patients with Aplastic Anemia, 75% achieved partial responses, and some became transfusion-independent within months of treatment, maintaining this independence for as long as 41 months.

The therapy also showcased a strong safety profile, with no severe adverse reactions or dose-related toxicities observed. The treatment does not require additional conditioning regimens or interleukin-2 supplementation, making it a less invasive option for patients. CK0801 appears to increase circulating Treg cells, promoting their expansion within the bone marrow, which may help modulate the immune system and enhance bone marrow function.

Beyond Aplastic Anemia, the Phase 1 study also investigated the potential of CK0801 in other bone marrow failure syndromes, such as Myelofibrosis and Myelodysplastic Syndromes. Patients with these conditions experienced significant improvements, including symptom relief, transfusion independence, and, in one instance, complete remission following an allogeneic stem cell transplant.

Aplastic Anemia is a severe condition that results in insufficient production of blood cells by the bone marrow, leading to significant health issues like bleeding, infections, and fatigue. Current treatment options, which include immunosuppressive therapy, growth factors, and stem cell transplants, often fall short in effectiveness for many patients. CK0801 aims to restore immune balance and enhance bone marrow function through the use of Treg cells, offering a potentially groundbreaking treatment for those affected by this rare disease.

Cellenkos Inc. is dedicated to advancing Treg cell therapies, derived from umbilical cord blood, to treat rare inflammatory diseases and autoimmune disorders. These therapies are designed to deliver powerful anti-inflammatory effects and sustained immune modulation without necessitating donor matching, with the ultimate goal of improving the lives of patients with rare and underserved medical conditions.