PTC Therapeutics (NASDAQ:PTCT) reported second-quarter total revenue of $361 million and raised its 2026 outlook, citing continued growth from its Sephience launch and greater durability than expected in its Duchenne muscular dystrophy franchise.
Chief Executive Officer Dr. Matthew Klein said the company generated $239 million in product revenue during the quarter and expects full-year product revenue of $850 million to $950 million. PTC now forecasts total 2026 revenue of $1.18 billion to $1.28 billion and said it remains positioned to potentially reach cash-flow breakeven this year.
Sephience Launch Drives Growth
In the U.S., PTC said all centers of excellence are now prescribing Sephience, along with additional centers that were not previously designated as centers of excellence. The company reported uptake across disease severities, age groups and treatment histories, including treatment-naive patients, patients who had not responded to prior therapies and those switching from existing treatment options.
Klein said the company is focused on increasing penetration within those centers after achieving broad early access. He added that the U.S. addressable phenylketonuria, or PKU, population is approximately 17,000 patients, compared with 58,000 patients across the company’s addressable global markets.
PTC said patient adherence remains high. Klein characterized discontinuations as approximately 20%, defined as going 60 days without a prescription renewal, and said renewal rates exceed 90%. The company said it has heard reports of reductions in phenylalanine levels, diet liberalization, and improvements in mood, cognition and quality of life among patients receiving Sephience.
Chief Business Officer Eric Pauwels said PTC’s customer-facing teams are emphasizing prescription fulfillment, reauthorizations, refills and patient support as the treated patient base grows. At the National PKU Alliance Conference, the company presented data from its AMPLIFY study comparing Sephience with BH4 and data involving patients switching to Sephience in pediatric and adult PKU populations.
International Expansion Builds
International sales are beginning to contribute more meaningfully, according to management. Japan began commercial sales following its first commercial sale in late March, and Pauwels said early uptake across Japanese centers of excellence has exceeded internal expectations. PTC said it has obtained Japanese pricing on par with the U.S. price, with that pricing locked in for 10 years.
The company is also using early-access and named-patient programs in several markets. PTC said it is recognizing revenue from these programs in France, Italy, Spain, Latin America and the Middle East.
- In France, PTC said the health technology assessment has been completed and its early-access program was approved during the second quarter. Pricing and reimbursement discussions are expected to conclude in 2027.
- In Italy and Spain, health technology assessment processes remain underway, with pricing and reimbursement talks expected to begin in the fourth quarter and potentially conclude in early 2027.
- In Germany, pricing and reimbursement negotiations remain ongoing and could be completed during the third or fourth quarter, according to Pauwels.
- In Brazil, Sephience received the highest therapeutic-benefit classification from the country’s drug-pricing regulator, Klein said.
PTC said it could have revenue-generating activity in up to 30 markets during 2026, while noting its longer-term strategy extends beyond that number. Management expects international revenue to become a more meaningful contributor in the second half of 2026 and through 2027. The company continues to characterize Sephience as a potential $2 billion-plus global commercial opportunity.
DMD Franchise and Royalty Revenue
PTC’s DMD franchise generated $67 million in second-quarter revenue, including $42 million from Translarna and $25 million from Emflaza. Translarna revenue included a government purchase order from Russia.
Management said the Duchenne franchise continues to face pressure from Emflaza generics and other market headwinds, but Klein said the business has shown more durability than the company had anticipated. He said the raised revenue outlook reflects confidence in Sephience as well as potential continuing contributions from the DMD franchise.
Roche reported approximately $628 million in global second-quarter sales of Evrysdi, resulting in $71 million in royalty revenue for PTC, Gravier said.
Pipeline Updates
PTC and partner Novartis are preparing to discuss votoplam’s 24-month Huntington’s disease data with the FDA during the second half of 2026. Klein said the discussions will include potential accelerated pathways, although the Novartis-funded phase III INVEST-HD trial remains the base case for approval.
PTC previously reported that votoplam showed dose-dependent slowing of disease progression on cUHDRS in the PIVOT-HD long-term extension study. In the 10-milligram stage 2 cohort, the company reported an average 52% slowing relative to natural history at month 24. The global INVEST-HD study is expected to enroll about 770 patients with early symptomatic Huntington’s disease and includes an interim analysis.
For vatiquinone in Friedreich’s ataxia, PTC said it finalized a protocol with the FDA for PROVE FA, an open-label study with a natural-history comparator arm intended to support a new drug application resubmission. The company expects to initiate the study in the third quarter. It plans to enroll approximately 120 patients ages 7 to 21 and assess change in modified Friedreich Ataxia Rating Scale scores through month 24.
The company also initiated a phase I trial of PTC612, an oral NLRP3 inhibitor, and expects data as early as late 2026 or early 2027. PTC plans to start a phase IIa PK/PD study of its DHODH inhibitor, PTC844, later in the third quarter. In addition, PTC selected PTC303 as a development candidate targeting MSH3, with plans to enter the clinic in 2027.
Cash Position and Expenses
Gravier said non-GAAP research and development expense declined to $89 million from $104 million a year earlier, excluding stock-based compensation. Non-GAAP selling, general and administrative expense fell to $68 million from $76 million on the same basis.
Cash, cash equivalents and marketable securities totaled $2.23 billion as of June 30, up from $1.95 billion at the end of 2025. During the quarter, PTC repurchased most of its 2026 convertible notes and issued new notes due in 2031 carrying a 0% coupon and a conversion price set at a 40% premium to the stock’s closing price at issuance.
Management said the balance sheet provides flexibility for business-development activity, particularly opportunities that could complement Sephience or leverage PTC’s global rare-disease commercial infrastructure. Gravier said the company intends to remain disciplined and does not plan to use all of its cash or significantly increase leverage for a single transaction.
About PTC Therapeutics (NASDAQ:PTCT)
PTC Therapeutics, Inc is a biopharmaceutical company focused on the discovery, development and commercialization of small molecule and biologic therapies for the treatment of rare genetic disorders. Since its founding in 1998, PTC has dedicated its efforts to addressing high unmet medical needs by targeting underlying genetic causes of disease. The company’s research platform emphasizes mechanisms such as nonsense suppression and RNA modulation, enabling the development of novel treatments for conditions with limited therapeutic options.
Among PTC’s approved products is Translarna (ataluren), a first-in-class therapy designed to treat nonsense mutation Duchenne muscular dystrophy in select markets.
