PTC Therapeutics, Inc.

PTC reported first-quarter 2026 total revenue of $273 million and total net product revenue of $226 million, versus $153 million in Q1 2025, a 47% increase. Sephience net product revenue was $125 million; DMD franchise revenue was $81 million, including Translarna at $59 million and Emflaza at $22 million. Royalty revenue from Evrysdi was $47 million, but management said there are no cash proceeds from that royalty. Non-GAAP R&D expense was $90 million, down from $100 million a year ago, and non-GAAP SG&A was $74 million, up from $72 million a year ago. Cash, cash equivalents and marketable securities were $1.89 billion at March 31, 2026, down from $1.95 billion at December 31, 2025. Management raised full-year 2026 product revenue guidance to $750 million-$850 million and total revenue guidance to $1.08 billion-$1.18 billion.

Matthew Klein framed the quarter as a strong start to 2026, driven by Sephience’s launch and supported by mature products. He emphasized broad uptake across age groups and disease severities, rapid international expansion, and high persistence, repeatedly pointing to the product’s differentiated efficacy and safety profile. He also sounded optimistic about votoplam after the positive PIVOT-HD extension readout, about the redesigned vatiquinone path, and about the company’s ability to use its cash and commercial footprint to support both internal programs and business development.

Pierre Gravier highlighted that product and royalty revenue totaled $273 million, with commercial net product revenue of $226 million, up 47% from $153 million in Q1 2025. He broke out Sephience at $125 million and the DMD franchise at $81 million, including a one-time Translarna government purchase order. On spending, he said non-GAAP R&D was $90 million and non-GAAP SG&A was $74 million. He closed by noting the $1.89 billion cash balance and said the balance sheet supports continued development and disciplined business development.

Analysts focused on Sephience launch dynamics, asking about the durability of the roughly 140 monthly U.S. start cadence, discontinuation reasons, payer friction, pricing abroad, and whether international uptake could resemble the U.S. launch. Management said the 140-per-month pace is a reasonable run rate, discontinuations are in the low double digits and mostly not driven by efficacy or safety, payer coverage is broadly in place with few step edits, and Japan has already locked pricing under 10-year orphan exclusivity. Questions on vatiquinone centered on the FDA-backed natural history design, how prior data and Skyclarys use would be handled, and whether the study starts from scratch; management said the design is meant to leverage the prior signal while aligning closely with FACOMS natural history data and excluding concomitant long-term Skyclarys use.