aibioPTC raises 2026 product revenue guidance after first quarter
Tickers PTCT
PTC reported first quarter 2026 total revenue of $273 million and a net loss of $2.8 million, and raised full-year product revenue guidance to $750 to $850 million.
Key facts
- Company
- PTC Therapeutics, Inc. (NASDAQ: PTCT)
- News type
- Company Updates
- Primary source
- PTC Therapeutics, Inc. 8-K (2026-05-07)
On May 7, 2026, PTC Therapeutics, Inc. reported a corporate update and financial results for the first quarter ended March 31, 2026. Total revenue was $273 million, including $226 million of product revenue. The company reported a net loss of $2.8 million for the quarter, compared with net income of $866.6 million for the first quarter of 2025. The release was furnished in an 8-K.
Sales and the Sephience launch
Sephience (sepiapterin) net product revenue was $124.6 million for the first quarter, representing 36% growth compared with the fourth quarter of 2025. The company said first quarter Sephience revenue included $112 million in the United States and $13 million outside the country. As of March 31, 2026, 1,244 total patients were on commercial therapy worldwide. The company said patient start forms in the United States have run at about 140 per month over the past several months, surpassing 1,500 in the quarter.
PTC recorded first commercial sales of Sephience in Japan in the first quarter and expects revenue from up to 30 countries by the end of 2026.
Translarna (ataluren) net product revenue was $59.0 million, compared with $86.2 million in the first quarter of 2025. The company said the first quarter figure includes a large government purchase order from Brazil, so it may not repeat. Emflaza (deflazacort) net product revenue was $21.5 million, compared with $47.8 million a year earlier, which the company attributed to continued generic erosion. Total net product revenue across the commercial portfolio was $225.6 million, a 47% increase from $153.4 million in the first quarter of 2025.
Roche reported Evrysdi (risdiplam) sales of about 464 CHF million, which resulted in royalty revenue of $46.8 million to PTC for the quarter, compared with $36.4 million a year earlier. Net product revenue and royalty revenue together totaled $272.4 million.
Guidance and costs
The company raised its full-year 2026 product revenue guidance to $750 to $850 million and expects total revenue of $1.08 to $1.18 billion. GAAP research and development and selling, general and administrative expense guidance remains $775 to $815 million. Non-GAAP guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million.
For the quarter, GAAP research and development expense was $100.9 million, compared with $109.0 million a year earlier. Non-GAAP research and development expense was $89.7 million. GAAP selling, general and administrative expense was $86.2 million, compared with $81.0 million, and non-GAAP expense was $73.9 million.
Chief Executive Officer Matthew B. Klein, M.D., said, "The Sephience launch continues to be strong, with sustained momentum in the US and growing momentum internationally as more countries contribute to the global launch."
Pipeline updates
In April 2026, PTC reported positive topline results from the 24-month interim analysis of the PIVOT-HD extension study of votoplam. The company said participants in Stage 2 who received 10 mg showed 52% slowing of disease progression on the cUHDRS scale relative to a natural history cohort, not a placebo group. The release gives no p-value or patient count for that figure. Novartis initiated the global Phase 3 INVEST-HD study, and the first patient dosed triggered a $50 million milestone payment from Novartis to PTC in the second quarter.
Following a Type C meeting with the FDA in April 2026, PTC expects to start an open-label vatiquinone study using a matched natural history control in the third quarter of 2026. The primary endpoint will be the change in mFARS from baseline to 24 months. An open-label design with a historical control is a weaker test than a randomized trial.
Balance sheet
Cash, cash equivalents and marketable securities were $1,892.5 million on March 31, 2026, compared with $1,945.4 million on December 31, 2025. The company had 82,882,024 shares issued and outstanding on March 31, 2026.
The company's annual report describes its rare disease focus, including Sephience for phenylketonuria, Translarna and Emflaza for Duchenne muscular dystrophy, Kebilidi for AADC deficiency, and Evrysdi for spinal muscular atrophy.