PTCT · PTC Therapeutics, Inc.

PTC Therapeutics delivered a strong Q1 2026 earnings report, driven primarily by robust product revenue growth to $225.6 million, a 47% increase year-over-year, led by Sephience which generated $124.6 million in its continued global launch. This included $112 million in the US and $13 million ex-US, reflecting 36% quarter-over-quarter growth, sustained patient starts at ~140 per month in the US surpassing 1,500 cumulatively, and first commercial sales in Japan with expansion to up to 30 countries by year-end. Royalty revenue from Roche's Evrysdi also rose to $46.8 million on CHF 464 million sales. Offsetting factors included lower Translarna revenue at $59.0 million (impacted by a large prior-year Brazil order) and Emflaza at $21.5 million due to generic erosion. Operating expenses increased modestly with GAAP R&D at $100.9 million and SG&A at $86.2 million, reflecting ongoing investments. Despite a small GAAP net loss of $2.8 million (vs. $866.6 million income last year, due to prior collaboration revenue), CEO Matthew Klein highlighted the 'outstanding revenue performance' supporting a raised full-year product revenue guide to $750-850 million and total revenue to $1.08-1.18 billion, with expense guidance unchanged. Balance sheet remains solid with $1.89 billion in cash despite a slight decline. Key pipeline catalysts include positive April topline from votoplam's 24-month PIVOT-HD interim showing dose-dependent cUHDRS benefits (52% progression slowing at 10mg), no NfL spikes, supporting Novartis-led Phase 3 INVEST-HD (first patient dosed, triggering $50 million Q2 milestone to PTC) and regulatory discussions. FDA feedback enables Q3 vatiquinone open-label study initiation for Friedreich’s ataxia NDA resubmission using mFARS endpoint. A conference call at 4:30 p.m. ET on May 7 reviewed these results and updates.