Clinical Trials And Safety ConcernsThe company disclosed three additional cases of hepatocellular injury for mitapivat in the open label portions of the ENERGIZE and ENERGIZE-T trials.
Market CompetitionShare weakness is likely also driven by PKR activator competitor readout from Novo Nordisk, which demonstrated comparable performance in VOC reduction and hemoglobin response.
Regulatory ChallengesThe withdrawal of Oxbryta increases the likelihood that regulators will need to see a definitive clinical benefit as a condition for approval, raising the regulatory bar.