Novel Monoclonal Antibody Fast Tracked for Anemia of Nondialysis Dependent CKD

The Food and Drug Administration (FDA) has granted Fast Track designation to DISC-0974 for the treatment of anemia in patients with nondialysis dependent chronic kidney disease (NDD-CKD).

DISC-0974 is an investigational monoclonal antibody against hemojuvelin, a bone morphogenetic protein-signaling co-receptor. It is designed to suppress hepcidin production and increase serum iron levels in patients with anemia of inflammation. 

In a phase 1 study involving healthy volunteers (ClinicalTrials.gov Identifier: NCT04999527), inhibition of hemojuvelin with DISC-0974 led to rapid, dose-dependent and sustained decreases in serum hepcidin. This effect corresponded to increases in serum iron and improvements in markers of erythropoiesis including statistically significant increases in hemoglobin (Hb). 

“Receiving Fast Track designation highlights the unmet need for the millions of NDD-CKD patients with anemia, as well as the potential of DISC-0974 to address this need,” said John Quisel, JD, PhD, President and CEO of Disc. “We believe DISC-0974 could be a transformative therapy for these patients and are excited to share additional results from our ongoing phase 1b/2 study in NDD-CKD patients with anemia this year.”

The phase 1b/2 study (ClinicalTrials.gov Identifier: NCT05745883) is currently assessing the safety, tolerability, and efficacy of DISC-0974 in NDD-CKD patients with anemia. Eligible patients include those with stage II-V CKD not receiving dialysis or concurrent treatment with erythropoietin-stimulating agents, and with baseline Hb less than 11.0g/dL for males and Hb less than 10.5g/dL for females.