原始出處紀錄
決策級主要結果 (Topline)
PTCT：votoplam 在 HD 中的 PIVOT-HD 24個月正面頂線數據
PTC 公布 PIVOT-HD 延伸研究 24個月期中分析的正面頂線結果，顯示 votoplam 在 cUHDRS 上有劑量依賴性益處，10 mg 劑量可減緩 52% 疾病進展，且安全性持續良好。這些數據支持由 Novartis 主導的全球 Phase 3 INVEST-HD 研究持續進行，首位患者給藥將在 Q2 2026 觸發 PTC 獲得 5000萬美元里程碑付款，PTC 與 Novartis 現將就潛在法規互動進行協調。
關鍵事實
來源摘錄
EX-99.1 2 tmb-20260507xex99d1.htm EX-99.1 Exhibit 99.1 PTC Therapeutics Provides Corporate Update and Reports First Quarter 2026 Financial Results – First quarter 2026 total revenue of $273 million, including $226 million of product revenue, supporting full-year 2026 guidance raise – – Global Sephience™ (sepiapterin) launch momentum continues with first quarter 2026 revenue of $125 million, representing 36% quarter-over-quarter growth – – Positive topline results from 24-month interim analysis of PIVOT-HD extension study of votoplam, supporting ongoing global Phase 3 INVEST-HD study and potential regulatory interactions – – Open-label vatiquinone registration study to be initiated in Q3 2026 based on FDA feedback – WARREN, N.J., May 7, 2026 – PTC Therapeutics, Inc., (NAS