Core Insights - PTC Therapeutics reported strong revenue performance of $190 million for the first quarter of 2025, with a significant cash position exceeding $2 billion as of March 31, 2025, supporting ongoing commercial and R&D activities [1][8]. Financial Performance - Total net product and royalty revenue for Q1 2025 was $190 million, a decrease from $208.8 million in Q1 2024 [5]. - Revenue from the DMD franchise was $134 million, with Translarna™ contributing $86 million and Emflaza® contributing $48 million [5]. - Collaboration and license revenue included $986.2 million from the PTC518 agreement with Novartis, which closed in January 2025 [5][7]. - Net income for Q1 2025 was $866.6 million, compared to a net loss of $91.6 million in Q1 2024 [8]. Regulatory and Clinical Updates - Positive CHMP opinion for Sephience™ (sepiapterin) received on April 25, 2025, with an expected European Commission adoption in approximately two months [5]. - NDA for Sephience is under review by the FDA, with a target action date of July 29, 2025 [5]. - NDA for vatiquinone accepted and granted Priority Review by the FDA, with a target action date of August 19, 2025 [5]. - Phase 2 PIVOT-HD study for PTC518 met primary endpoint of dose-dependent blood HTT lowering at Week 12, with favorable safety and tolerability [5]. Guidance and Future Outlook - PTC anticipates full-year 2025 revenue between $650 million and $800 million, including in-line products and potential new launches [8]. - Full-year 2025 GAAP R&D and SG&A expenses are projected to be between $805 million and $835 million [8]. - Non-GAAP R&D and SG&A expenses are expected to be between $730 million and $760 million, excluding estimated non-cash, stock-based compensation of $75 million [8].