A Phase III Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating Desidustat’s Efficacy and Safety in Non-Dialysis Chronic Kidney Disease Patients with Anemia

INTRODUCTION: This phase III trial assessed desidustat's efficacy and safety compared with placebo for the treatment of anemia in non-dialysis-dependent chronic kidney disease (NDD-CKD). METHODS: A multicenter, randomized, double-blind trial enrolled 152 NDD-CKD patients with anemia (101 desidustat, 51 placebo). PRIMARY ENDPOINT: hemoglobin (Hb) change from baseline to weeks 7-9. Secondary endpoints included Hb response rate (≥100 g/L), time to target Hb, and target Hb maintenance (100-120 g/L). Safety evaluated treatment-emergent adverse events (TEAEs). Exploratory endpoints analyzed Hb dynamics (weeks 21-25) and serum hepcidin levels (baseline-week 53). RESULTS: After 9 weeks of treatment, desidustat increased Hb level from 89.18 g/L to 105.66 g/L, whereas placebo decreased from 89.41 g/L to 88.51 g/L, with a mean difference of 17.52 g/L (95% confidence interval [CI]: 14.35-20.68) in the full analysis set. And in the per protocol set, desidustat increased Hb level from 88.86 g/L to 105.62 g/L, and placebo declined from 89.37 g/L to 87.57 g/L, with a mean difference of 18.39 g/L (95% CI: 15.39-21.39). Hb response rates were 85.15% vs. 23.53% (p < 0.001). Median time to target Hb was 30 days with desidustat vs. 0 days with placebo (p < 0.001). By week 9, desidustat maintained Hb within target range 51.68% of the time vs. 10.24% for placebo (p < 0.001). TEAE incidence was comparable between groups. During weeks 21-25, Hb rose further (desidustat: +21.26 g/L; placebo: +26.63 g/L). Serum hepcidin decreased sharply in desidustat by week 9 (-67.43 ng/mL vs. -6.38 ng/mL; placebo), though placebo showed delayed reduction by week 25 - levels equalized by week 53. CONCLUSION: Desidustat significantly improved Hb levels and accelerated anemia correction in NDD-CKD patients versus placebo, with comparable safety.