Cellenkos Inc. announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its investigational product, CK0804, for treatment of myelofibrosis, a rare blood cancer with an annual incidence of one to three new cases per 100,000 people per year and an estimated US prevalence of approximately of 25,000 patients.

CK0804 is composed of CXCR4hi Tregs that preferentially home to its ligand CXCL12, which is overexpressed in the bone marrow and at the sites of extramedullary hematopoiesis including spleen, in myelofibrosis. Upon arrival in the target tissue, CK0804 Tregs engage with Antigen Presenting Cells (APCs), undergo in-vivo proliferation, secrete the suppressor cytokine, IL-10 and resolve inflammation in a non-MHC dependent manner, while regulating PDGF-driven pathways involved disease remodeling.

"Receiving Orphan Drug Designation is an important milestone in the clinical development of CK0804 for myelofibrosis and underscores our commitment to advance CK0804 into phase-II trials to address the unmet need for patients who have not responded to currently available therapies. The observed increase in IL-10 and decreases in TGFβ levels in CK0804 responders, together with reductions in pathogenic monocytes in plasma and bone marrow, support the disease modifying potential of CK0804 Tregs as a distinct and differentiated therapeutic class in myelofibrosis," said Dr Simrit Parmar, MD and Founder, Cellenkos.