PTC Therapeutics Q3 Earnings Call Highlights

Key Points

• PTC raised its 2026 outlook after reporting third-quarter revenue of $358 million, including $276 million in product revenue. The company now expects $960 million–$990 million in product revenue, $1.29 billion–$1.32 billion in total revenue, and cash-flow breakeven during 2026.
• Sephience was the main growth driver, generating $182 million in third-quarter revenue, up more than 20% sequentially, with over 2,000 patients actively receiving treatment worldwide. PTC estimates peak sales could exceed $2 billion as it expands U.S. adoption and pursues reimbursement decisions in major European markets.
• PTC acquired the Fabry disease gene therapy candidate ST-920 for $111 million upfront and plans to submit a rolling application in the fourth quarter of 2026, potentially supporting a 2027 launch. The company ended the quarter with $2.13 billion in cash, equivalents and marketable securities.

PTC Therapeutics NASDAQ: PTCT reported third-quarter 2026 total revenue of $358 million, including $276 million in net product revenue, as sales of phenylketonuria treatment Sephience continued to expand in the United States and internationally.

Chief Executive Officer Matthew Klein said the company raised its full-year product revenue outlook to $960 million to $990 million and now expects total 2026 revenue of $1.29 billion to $1.32 billion. The revised product revenue outlook represents year-over-year growth of 64% to 69%, according to the company. PTC also said it expects to reach cash-flow breakeven during 2026.

Sephience Launch Drives Product Growth

Sephience generated $182 million in third-quarter revenue, rising more than 20% from the second quarter, according to Chief Business Officer Eric Pauwels. The company said more than 2,000 patients worldwide were actively receiving the treatment as of Sept. 30.

Klein described the product’s first year of launch as a success but said the company remains in the “early innings” of expanding use. PTC estimates that roughly 58,000 people with PKU live in its target markets globally, while about 17,000 are in the U.S. The company said the vast majority of those patients have not yet tried Sephience.

In the U.S., the company cited continued demand among therapy-naive patients, patients who previously tried and failed other therapies, and individuals switching from branded or generic Kuvan. PTC said all U.S. centers of excellence are now prescribing Sephience, and its commercial organization is focusing on deeper engagement at individual treatment centers.

Pauwels said more than one-quarter of actively treated Sephience patients in the third quarter were therapy-naive adults, including some who had returned to care after years without treatment. The company is also targeting patients switching from Kuvan, citing extension-study data from AMPLIPHY that PTC said showed further reductions in phenylalanine levels and increased protein intake among responding patients after they switched.

Management said adherence remained strong, while cumulative discontinuations were in the low 20% range. Klein said the company expects adherence among patients switching from Kuvan to be higher than levels observed during the early launch, when Sephience was used more frequently by therapy-naive and more severe patients.

Internationally, PTC highlighted growth in Japan as well as patient starts in the Middle East, North Africa, Latin America and Canada. The company is negotiating pricing and reimbursement in Germany, Spain, Italy and France. Pauwels said PTC expects a German reimbursement decision “fairly soon,” while negotiations in France, Italy and Spain are anticipated to conclude in the first half of 2027.

Klein said the company expects the U.S. to account for the majority of Sephience revenue at peak, although it expects the patient population outside the U.S. to be larger. PTC continues to project a peak sales opportunity of more than $2 billion for Sephience.

Mature Brands and Royalty Revenue

PTC’s Duchenne muscular dystrophy franchise generated $74 million in third-quarter revenue. Translarna contributed $49 million, including a government purchase order from Russia, while Emflaza contributed $25 million.

Management said Translarna sales were also supported by markets in Europe, Latin America and the Middle East and North Africa. However, Klein said PTC does not expect additional large government purchase orders from Brazil or Russia in the fourth quarter.

For Emflaza, the company said brand loyalty remained strong despite multiple generic competitors. Klein noted that a new generic entrant has reduced prices in the market, which affected the company’s expectations for the product.

Roche reported approximately $607 million in global third-quarter sales of Evrysdi, resulting in $82 million of royalty revenue for PTC.

Fabry Asset Acquisition and Pipeline Updates

During the quarter, PTC acquired ST-920, a BLA-stage gene therapy candidate for Fabry disease, for an upfront payment of $111 million and up to $100 million in contingent milestones tied to U.S. regulatory approval. The company said ST-920 is a one-time administered AAV gene therapy designed to deliver a humanized copy of the GLA gene.

PTC plans to complete a rolling biologics license application submission seeking accelerated approval in the fourth quarter of 2026. If approved, the company said ST-920 could launch commercially in 2027. Klein said PTC believes the acquisition could become accretive in 2027.

Pauwels said PTC is preparing for a U.S. launch by mapping claims data and engaging with centers of excellence and patient advocacy groups. He said the company sees more than 11,000 Fabry patients in the U.S. and believes a number of them remain untreated.

Elsewhere in the pipeline, the company said an FDA Type C meeting on its votoplam Huntington’s disease program has been rescheduled for the first quarter of 2027 because of FDA scheduling. Klein said the change was a calendar matter and not related to the program’s data package. Enrollment continues in the global Phase III INVEST-HD trial, which is planned to enroll approximately 770 early symptomatic patients.

PTC also initiated the global PROVE-FA study of vatiquinone in Friedreich’s ataxia. The single-arm study is intended to support resubmission of a new drug application and is expected to enroll about 120 participants ages 7 through 21.

The company further said it started a Phase IIa study of PTC844, an oral DHODH inhibitor, in adults with rheumatoid arthritis, while its Phase I study of oral NLRP3 inhibitor PTC612 is progressing. PTC303, an oral small-molecule splicing candidate targeting MSH3, remains on track to enter the clinic in 2027.

Expenses and Cash Position

Third-quarter non-GAAP research and development expense was $95 million, compared with $91 million a year earlier. Non-GAAP selling, general and administrative expense declined to $67 million from $74 million in the prior-year quarter.

PTC ended the quarter with $2.13 billion in cash, cash equivalents and marketable securities, compared with $1.95 billion at the end of 2025. Chief Financial Officer Pierre Gravier said the company intends to remain disciplined in evaluating additional business-development opportunities while using its global commercial infrastructure and financial flexibility to support long-term growth.

About PTC Therapeutics (NASDAQ:PTCT)

PTC Therapeutics, Inc is a biopharmaceutical company focused on discovering, developing and commercializing medicines for rare diseases. Its research programs include small-molecule treatments, gene therapies and other approaches designed to address conditions with limited or no available treatment options.

The company's commercial portfolio has included Emflaza (deflazacort), a corticosteroid approved in the United States to treat Duchenne muscular dystrophy, and Upstaza (eladocagene exuparvovec), a gene therapy authorized in the European Union for aromatic L-amino acid decarboxylase deficiency.

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