Question · Q4 2025
Michael asked about the definition of success for the VOCAL study, specifically regarding TIW non-inferiority versus ESAs and potential superiority endpoints, the importance of the RBC sub-study, and the status of AKB-097's IND reactivation and protocol changes.
Answer
Senior Vice President, Chief Research and Development Officer Steven K. Burke stated that the 350-patient VOCAL study aims to establish Vafseo's safety and effectiveness against ESAs (Mircera) for DaVita, with a primary endpoint of non-inferiority for hemoglobin control. He expects superiority on some hemoglobin-related safety endpoints (less rapid rises, high hemoglobins, dose adjustments). Steven K. Burke emphasized the RBC sub-study's importance in differentiating Vafseo's mechanism, noting that Vafseo-influenced red blood cells are different (bigger, more hemoglobin, uniform distribution of widths), which helps physicians understand clinical benefits like reduced death and hospitalization. Regarding AKB-097, he confirmed they are reworking the protocol for simplicity, but it remains fundamentally the same as FDA-agreed with Q32 Bio, and the IND will be activated upon resubmission of the finalized protocol.
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