Shots:

• Astellas discontinue the dosing in the ASPIRO trial that evaluates AT132 in 24 participants (7 at the 1.3x1014 vg/kg dose and 17 at the 3.5x1014 vg/kg dose) with XLMTM. The participant has a history of intermittent cholestasis
• The discontinuation is due to serious AEs in a study that demonstrated abnormal LFTs in patients who received a low dose of AT132. Additionally- 3 patients develops progressive cholestatic hepatitis & liver failure who treated at 3.5x1014 vg/kg dose
• In Dec’20- FDA lifted the hold after reviewed the modifications to ASPIRO trial protocol including a reduction of dosing. AT132 has received RMAT- RPD designation- FTD & ODD from the FDA while PRIME & ODD from EMA

| Ref: Astellas | Image: Astellas