- Based on the completion of its End-of-Phase 2 Meeting with the FDA and the Scientific Advice Process with the European Medicines Agency (EMA), Akebia Therapeutics (NASDAQ:AKBA +0.9%) has set the key elements of its Phase 3 program for lead product candidate vadadustat (AKB-6548) for the treatment of patients with anemia related to non-dialysis-dependent chronic kidney disease (NDD-CKD).
- The program, called PRO2TECT, includes two separate studies that will enroll ~3,100 subjects across 500 sites globally. The correction study will enroll anemia patients not currently being treated with recombinant erythropoiesis-stimulating agents (rESAs) while the conversion study will enroll patients currently receiving rESA therapy who will be converted to either vadadustat or active control with the goal of maintaining baseline hemoglobin levels. Both trials will include a 1:1 randomization and an open-label, active-control, non-inferiority design. Efficacy endpoints will include hemoglobin response.
- The Phase 3 program will be finalized after the company presents its completed Phase 2 results to the both the FDA and EMA.
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Vadadustat is an inhibitor of hypoxia inducible factor (HIF)-prolyl hydroxylase, an enzyme that promotes the breakdown of HIF proteins. HIF is the primary regulator of the production of red blood cells. The body's natural response to anemia is to increase the level of HIF proteins.
- Previously: Akebia's lead product candidate successful in mid-stage study; shares up 59% AH (Sept. 8)