PTC Therapeutics, Inc.

What they do

PTC discovers, develops and commercializes medicines for children and adults with rare disorders, focused on neurology and metabolism. Commercial products include Sephience for PKU, Translarna and Emflaza for DMD, Upstaza/Kebilidi for AADC deficiency, and Tegsedi and Waylivra in Latin America and the Caribbean. It also earns royalties on Evrysdi for spinal muscular atrophy under a collaboration with Roche and the SMA Foundation. Pipeline programs include votoplam for Huntington's disease, vatiquinone for Friedreich's ataxia, PTC303, PTC612 and PTC844.

Revenue drivers

• Sephience (sepiapterin) — PKU therapy approved in the US, EEA, Japan and Brazil; generated $151.3M in Q2 2026, the largest product line, with 1,647 patients on commercial therapy worldwide as of June 30, 2026.
• Royalty revenue (Evrysdi) — Royalty on Roche's risdiplam sales for SMA; $71.1M to PTC in Q2 2026 versus $57.6M in Q2 2025, based on Roche year-to-date sales of about 968 CHF million.
• Translarna (ataluren) — DMD product with conditional EEA approval not renewed in March 2025 but continued in some EU countries under Articles 117(3) and 5(1); Q2 2026 net sales of $42.2M, down from $59.5M a year earlier.
• Emflaza (deflazacort) and other products — Emflaza for DMD in the US generated $24.6M in Q2 2026 versus $36.4M in Q2 2025 due to generic erosion; Upstaza/Kebilidi and Tegsedi/Waylivra make up the remainder.

Recent performance

Q2 2026 total revenue was $361M, including $239M of product revenue and $309.9M of net product plus royalty revenue versus $175.9M a year earlier. Sephience revenue was $151.3M, up 21% sequentially, and a $50.0M Novartis milestone was recorded as collaboration revenue for the INVEST-HD first patient dosed. Translarna and Emflaza both declined year over year, to $42.2M and $24.6M respectively. Full-year 2025 revenue was $1.73B with net income of $683.0M and operating cash flow of $711.2M. At June 30, 2026, cash and equivalents were $1.05B, total liabilities were $3.42B and shareholder equity was negative $165.4M.

Strategy

PTC is prioritizing the global launch and geographic expansion of Sephience while managing the declining DMD franchise. It is advancing a pipeline that includes votoplam for Huntington's disease, where partner Novartis funds and runs the Phase 3 INVEST-HD study, and vatiquinone, with a PROVE-FA study for Friedreich's ataxia expected to initiate in Q3 2026. Earlier-stage programs include PTC303, a splicing candidate for Huntington's disease and myotonic dystrophy planning to enter the clinic in 2027, PTC612, an NLRP3 inhibitor already in Phase 1, and PTC844, a DHODH inhibitor expected to start Phase 2a in Q3 2026. The company describes its strategy as leveraging scientific expertise and global commercial infrastructure across rare neurology and metabolism.

Risks

• Translarna EEA authorization — The EC adopted the CHMP negative opinion in March 2025 not to renew Translarna's conditional marketing authorization, and continued EU commercial use depends on individual countries invoking Articles 117(3) and 5(1) of Directive 2001/83.
• US Translarna withdrawal — PTC withdrew the Translarna NDA resubmission for nmDMD in February 2026 and does not plan further US development for that indication.
• Emflaza generic erosion — Emflaza net sales fell to $24.6M in Q2 2026 from $36.4M a year earlier due to continued generic erosion.
• Pipeline and regulatory execution — The 10-K risk factors cite possible clinical trial delays, enrollment difficulties, adverse side effects, manufacturing challenges and failure to obtain regulatory approval for product candidates.

Outlook

Management raised full-year 2026 total revenue guidance to $1.18 to $1.28 billion, with product revenue guidance of $850 to $950 million. Vatiquinone's PROVE-FA study to support an NDA resubmission for Friedreich's ataxia is expected to initiate in Q3 2026, enrolling approximately 120 individuals age 7 to 21. PTC plans to engage with the FDA in the second half of 2026 on the PIVOT-HD 24-month votoplam results. The company reported a cash position of $2.2 billion as of June 30, 2026.