Cellenkos’ CK0801 Treg Therapy Receives FDA Orphan Drug Designation for Aplastic Anemia
HOUSTON, April 14, 2025 /PRNewswire/ — Cellenkos, Inc., a clinical-stage biotechnology company developing off-the-shelf Treg cell therapies, today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to CK0801 for the treatment of Aplastic Anemia (AA).
Orphan Drug Designation supports therapies for rare diseases affecting fewer than 200,000 people in the U.S., offering incentives like tax credits, fee exemptions, and potential market exclusivity. Aplastic Anemia, with an incidence of 1.0-2.3 per million, affects an estimated 5,000 patients in the U.S.
“This designation underscores CK0801’s potential to transform treatment for Aplastic Anemia patients,” said Dr. Simrit Parmar, Founder of Cellenkos. “We aim to provide a therapy that reduces transfusion dependence in patients unresponsive to standard care.”
Published in NEJM Evidence, Phase 1 trial results demonstrated CK0801’s ability to achieve durable transfusion independence for up to 3.5 years in AA patients, with an excellent safety profile and outpatient administration. The trial showed a 67% overall response rate across bone marrow failure syndromes, with 75% of AA patients achieving partial responses and two becoming transfusion-independent.
Key Study Findings:
- Durable Transfusion Independence: Two of three transfusion-dependent AA patients achieved complete and durable independence for up to 3.5 years.
- Safety: CK0801 was well-tolerated, with no infusion reactions or severe adverse events, and requires no conditioning chemotherapy.
- Immunologic Impact: CK0801 increased circulating Tregs and promoted bone marrow Treg expansion.
The Phase 1 study also showed promising results in Myelofibrosis and Myelodysplastic Syndromes, with improvements in symptoms and transfusion independence.
About Aplastic Anemia:
Aplastic Anemia is a rare and serious bone marrow failure disorder. CK0801 offers a novel, non-immunosuppressive approach to restore immune balance and improve bone marrow function.
Cellenkos is advancing CK0801 towards a registration trial to support regulatory approval for transfusion-dependent Aplastic Anemia.
About Cellenkos, Inc.:
Cellenkos is a clinical-stage biotechnology company focused on developing off-the-shelf Treg cell therapies for inflammatory and autoimmune diseases.
Source:
https://www.prnewswire.com/news-releases/cellenkos-announces-us-fda-orphan-drug-designation-granted-to-ck0801-allogeneic-cord-blood-derived-t-regulatory-cell-product-for-treatment-of-aplastic-anemia-302427571.html
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