Overview
- AEMPS has authorized a Phase I study of AP-2 at Hospital Universitario de La Princesa in Madrid, with first dosing planned for April.
- Approximately 70–72 healthy volunteers will receive escalating doses to assess safety and pharmacokinetics.
- AP-2 inhibits CK1 to prevent pathological phosphorylation of TDP-43 and aims to restore its nuclear function, with preclinical data showing reversal of TDP-43 anomalies in cell and transgenic animal models.
- Patient dosing is planned to begin in January 2027 in a Phase Ib study if early safety results permit.
- The trial is funded by Molefy Pharma, a CSIC spin-off majority-backed by Grupo Arquimea, and the candidate holds EMA orphan designation granted in October 2025.