PTC Raises 2026 Product Revenue Guidance Again

The Tip Desk · July 31, 2026 · 3 min read

Second-quarter total revenue reached $361 million as Sephience posted $151.3 million and the company lifted full-year product guidance to $850 million to $950 million.

PTC Therapeutics (PTCT) raised full-year 2026 product revenue guidance for a second consecutive quarter, lifting the range to $850 million to $950 million from $750 million to $850 million, and increased expected total revenue to $1.18 billion to $1.28 billion.

The global biopharmaceutical company reported second-quarter total revenue of $361 million, including $239 million of product revenue, versus $273 million total and $226 million of product revenue in the first quarter. Chief Executive Officer Matthew B. Klein said the teams delivered another strong quarter, enabling the company to again raise 2026 product revenue guidance.

Sephience (sepiapterin) remained the commercial engine. Net product revenue was $151.3 million in the second quarter, up 21% from $125 million in the first quarter, after that first-quarter figure had risen 36% from $92 million in the fourth quarter of 2025. Sephience sales came primarily from the United States, with increasing contributions internationally. Patients on commercial therapy worldwide rose to 1,647 as of June 30, 2026, from 1,244 as of March 31.

Total net product revenue across the commercial portfolio was $238.8 million in the second quarter, compared with $118.3 million a year earlier, an increase of more than 100%. Translarna (ataluren) net product revenue was $42.2 million. Emflaza (deflazacort) net product revenue was $24.6 million, which the company attributed to continued generic erosion. Royalty revenue from Roche’s Evrysdi (risdiplam) sales was $71.1 million.

Collaboration and license revenue was $50.6 million. PTC recorded a $50.0 million development milestone from Novartis for the first patient dosed in the ongoing INVEST-HD Phase 3 study of votoplam for Huntington’s disease. PIVOT-HD 24-month results showed dose-dependent disease slowing on the cUHDRS scale in Stage 2 participants, reaching 52% at the 10 mg dose level. INVEST-HD is being conducted and funded by Novartis, with a target enrollment of approximately 770 individuals with early symptomatic disease. PTC plans to engage with the FDA in the second half of 2026 to discuss the PIVOT-HD 24-month results.

Net income was $83.5 million, or $0.92 a share on a diluted basis, compared with a net loss of $64.8 million, or $0.83 a share, a year earlier. Income from operations was $149.0 million, versus an operating loss of $35.0 million in the year-ago quarter. GAAP research and development expense was $99.2 million. GAAP selling, general and administrative expense was $80.6 million.

In June, PTC issued $550.0 million of senior convertible notes due in 2031 at a 0% coupon and a conversion price representing a 40% premium over the stock’s closing price at the time of the deal. Concurrently, it repurchased the majority of its 1.5% senior convertible notes due in September 2026. Cash, cash equivalents and marketable securities were $2,229.1 million on June 30, 2026, compared with $1,945.4 million on December 31, 2025. Shares issued and outstanding were 83,327,286.

The company named PTC303 as the development candidate for its MSH3 splicing program targeting Huntington’s disease and myotonic dystrophy, with a plan to enter the clinic in 2027. It initiated a Phase 1 study of PTC612, an oral NLRP3 inhibitor, in the second quarter. A Phase 2a study of PTC844, a next-generation DHODH inhibitor, is expected to initiate in the third quarter of 2026. The vatiquinone PROVE-FA study to support an NDA resubmission for Friedreich’s ataxia is expected to initiate in the third quarter and will enroll approximately 120 individuals aged 7 to 21.

GAAP R&D and SG&A expense guidance for the full year remains $775 million to $815 million. Non-GAAP R&D and SG&A expense guidance remains $680 million to $720 million, excluding an estimated $95 million of non-cash stock-based compensation.