other material
confidence high
sentiment positive
materiality 0.85
PTC Therapeutics reports positive PIVOT-HD extension data: 52% slowing of HD progression at 10 mg votoplam
PTC THERAPEUTICS, INC.
- Stage 2 HD participants showed 52% (10 mg) and 28% (5 mg) slowing on cUHDRS vs natural history at 24 months.
- Mean NfL levels remained below baseline at 24 months for both doses, contrasting with natural history increases.
- Safety data at Month 24 consistent with prior favorable safety profile; no treatment-related NfL increases.
- Novartis has initiated global Phase 3 INVEST-HD study of votoplam (10 mg vs placebo, n≈770, primary endpoint cUHDRS at 36 months).