2023 Potential Key Value-Creating Milestones: Results from the placebo-controlled portion of APHENITY, the Phase 3 registration-directed clinical trial of sepiapterin in patients with PKU, are expected in the first quarter of 2023. Results from MIT-E, the Phase 2/3 registration-directed clinical trial of vatiquinone in mitochondrial disease associated seizures, are expected in the first quarter of 2023. Results from MOVE-FA, the Phase 3 registration-directed clinical trial of vatiquinone in Friedreich ataxia, are expected in the second quarter of 2023. Results from the 12-week portion of PIVOT-HD, the Phase 2 study of PTC518 in Huntington’s disease, are expected in the second quarter of 2023. Submission of a Biologics License Application to the FDA for Upstaza is expected in the first half of 2023. A Phase 2/3 clinical trial of unesbulin in diffuse intrinsic pontine glioblastoma is expected to initiate in the fourth quarter of 2023.
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