PTC518 Ph2 PIVOT-HD shows mHTT lowering and clinical trends; FDA lifts partial clinical hold

PTC THERAPEUTICS, INC.

PTC518 lowered mHTT in blood by 22% (5mg) and 43% (10mg) at Month 12; similar CSF levels.
Total Motor Score worsening: 2.0 (5mg), 1.3 (10mg) vs 4.9 for placebo, suggesting slowed progression.
FDA lifted partial clinical hold on PTC518 program based on PIVOT-HD interim data.
Company received $250M from Royalty Pharma for first tranche of Evrysdi royalty sale under amended agreement.
Hopewell lease amended to significantly reduce space and rent; lease no longer considered material.

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