PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2025 Financial Results

Core Insights - PTC Therapeutics reported strong financial results for the fourth quarter and full year 2025, with product and royalty revenue of $831 million, exceeding guidance [1] - The global launch of Sephience™ has been successful, with significant uptake and revenue generation [1] - The company is approaching cash flow breakeven and has a robust financial position with $1.95 billion in cash as of December 31, 2025 [1] Corporate Highlights - Sephience has a strong start, with plans to expand its global footprint to 20-30 countries by the end of 2026 [1] - The company received 1,134 patient start forms in the US and has 946 patients on commercial therapy worldwide as of December 31, 2025 [1] - The FDA has shown openness to an Accelerated Approval pathway for the votoplam Huntington's disease program, with a global Phase 3 trial planned to initiate in 1H 2026 [1] Financial Highlights - Cash, cash equivalents, and marketable securities increased to $1,945.4 million from $1,139.7 million year-over-year [1] - Net income for full year 2025 was $682.6 million, a significant improvement from a net loss of $363.3 million in 2024 [1] - Total revenues for full year 2025 reached $1,730.7 million, compared to $806.8 million in 2024, driven by collaboration and license revenue of $998.4 million related to the votoplam agreement with Novartis [1][2] Revenue Breakdown - Total net product revenues for Q4 2025 were $184.0 million, compared to $150.1 million in Q4 2024 [2] - Emflaza® net product revenues decreased to $27.1 million in Q4 2025 from $50.5 million in Q4 2024 [1] - Translarna™ net product revenues also declined to $39.0 million in Q4 2025 from $89.1 million in Q4 2024 [1] 2026 Financial Guidance - The company projects non-GAAP R&D and SG&A expenses of $680 to $720 million for 2026, excluding estimated non-cash, stock-based compensation expense of $95 million [1] - Total product revenue is expected to be between $700 to $800 million, representing a 19% to 36% increase from 2025, primarily driven by Sephience [1]