PTC518 Phase 2 PIVOT-HD meets primary endpoint with dose-dependent HTT lowering at Week 12

PTC THERAPEUTICS, INC.

Primary endpoint met: dose-dependent blood HTT protein reduction (p<0.0001) at Week 12.
5mg dose lowered HTT 23% in Stage 2/3; 10mg lowered 39% (Stage 2) and 36% (Stage 3).
Favorable dose-dependent trends on cUHDRS, TFC, SDMT in Stage 2 patients at 12 and 24 months.
No treatment-related SAEs or NfL spikes; safety and tolerability profile favorable.
At Month 24, dose-dependent NfL lowering: -8.9% (5mg, p=0.12) and -14% (10mg, p=0.03) vs natural history.

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