PTC Therapeutics Q1 Earnings Call Highlights

Key Points

• PTC Therapeutics posted a record first quarter, with total revenue of $273 million and product revenue of $226 million, and it raised its full-year 2026 guidance to $750 million-$850 million in product revenue and $1.08 billion-$1.18 billion in total revenue.
• Sephience was the main growth driver, generating $125 million in global sales in the quarter and attracting 1,244 commercial patients worldwide as PTC expands launches into more countries, including Japan and Brazil.
• The company also highlighted pipeline progress in Huntington’s disease and Friedreich’s ataxia, while ending the quarter with $1.89 billion in cash and equivalents to support its commercial and R&D plans.

PTC Therapeutics NASDAQ: PTCT reported a record first quarter for product revenue and raised its 2026 revenue outlook, citing strong early demand for Sephience, its treatment for phenylketonuria, or PKU.

Chief Executive Officer Dr. Matthew Klein said the company is “off to a terrific start to 2026,” with first-quarter total revenue of $273 million, including $226 million in product revenue. PTC raised its full-year 2026 product revenue guidance to a range of $750 million to $850 million and projected total revenue of $1.08 billion to $1.18 billion.

Sephience Launch Drives Record Product Revenue

Sephience generated $125 million in global revenue during the quarter, up 36% from the fourth quarter of 2025. U.S. revenue accounted for $112 million, while international revenue contributed $13 million.

Klein said the company had 1,244 commercial patients globally on Sephience as of March 31. In the U.S., PTC surpassed 1,500 patient start forms during the quarter, with prescription starts averaging about 140 per month in recent months. He said the company expects that pace to continue “for the foreseeable future.”

Chief Business Officer Eric Pauwels said PTC has generated more than 2,200 prescriptions worldwide since the initial U.S. and Germany launches last summer. He said more than 90% of U.S. PKU centers of excellence have prescribed Sephience, and adoption has spanned age groups, disease severity and treatment history, including treatment-naive patients and patients who previously failed other therapies.

PTC said discontinuation rates remain in the low double digits and refill rates remain strong. Pauwels said U.S. payer dynamics are favorable, with commercial and government policies in place covering more than two-thirds of the U.S. population and “very few limitations,” including limited step edits.

Internationally, PTC recorded its first Sephience sale in Japan in late March, ahead of schedule. Pauwels said pricing and reimbursement in Japan were finalized in the first quarter and are locked in for the 10-year orphan exclusivity period. The company also received regulatory approval in Brazil and is pursuing access through named patient programs while pricing is finalized. PTC expects commercial patients in up to 30 countries by year-end.

Company Points to Multi-Billion-Dollar Opportunity

Klein said PTC remains confident in a global commercial opportunity for Sephience of more than $2 billion, pointing to the product’s efficacy and safety profile and its dual mechanism of action. He said the company has received positive feedback from patients, families and healthcare providers, including reports of lower phenylalanine levels and broader diet liberalization.

In response to analyst questions, Klein said patients are staying on therapy for multiple reasons, including the ability to liberalize diet, and some have reported improvements in anxiety, cognition and “brain fog.” He said PTC is working to capture these observations through real-world evidence presentations and publications.

PTC said it does not specifically track Sephience revenue by mild, moderate or classical PKU categories. Klein said up to one-third of patients are treatment naive, a group he said tends to include more classical PKU patients who may not have been expected to benefit from existing therapies.

DMD Franchise and Royalty Revenue Contribute

Chief Financial Officer Pierre Gravier said first-quarter net product revenue across PTC’s commercial portfolio was $226 million, compared with $153 million in the first quarter of 2025, representing 47% growth.

The DMD franchise generated $81 million in revenue during the quarter. Translarna revenue was $59 million and included a large one-time government purchase order in Brazil. EMFLAZA revenue was $22 million, which Pauwels said came despite erosion from multiple generics and was supported by brand loyalty and patient services.

PTC also recorded $47 million in royalty revenue from Evrysdi, based on Roche’s first-quarter global revenue of approximately $585 million. Gravier noted that PTC continues to report Evrysdi royalty revenue on its financial statements, but there are no cash proceeds to the company.

Pipeline Updates Include Huntington’s and Friedreich’s Ataxia

Klein highlighted recent data from the PIVOT-HD long-term extension study of votoplam in Huntington’s disease. He said a 24-month interim analysis showed dose-dependent slowing of disease progression on COHDRS, including an average 52% slowing relative to a matched natural history cohort at the 10-milligram dose in participants with Stage 2 disease. He said the safety profile remains favorable across doses and disease stages.

The data support the ongoing phase 3 INVEST-HD study, which is funded and led by Novartis. Klein said that study is enrolling about 770 individuals with early symptomatic disease, randomized three-to-two to receive votoplam 10 milligrams or placebo, and includes an interim analysis.

For vatiquinone in Friedreich’s ataxia, Klein said PTC held a Type C meeting with the FDA in April to discuss a new trial intended to support a potential NDA resubmission. The company plans an open-label study with a matched natural history control group from the FACOMS Disease Registry. The trial is expected to enroll about 120 patients ages 7 to 21, with change in mFARS from baseline to 24 months as the primary endpoint.

Klein said PTC expects to begin the study within the next few months. He added that concomitant use of SKYCLARYS will not be allowed because the study must align closely with the natural history comparator, though the protocol will include provisions for some patients with short prior exposure who wash out.

Expenses and Cash Position

PTC reported non-GAAP research and development expense of $90 million for the quarter, excluding $11 million in non-cash stock-based compensation, compared with $100 million a year earlier excluding $9 million in stock-based compensation.

Non-GAAP selling, general and administrative expense was $74 million, excluding $12 million in stock-based compensation, compared with $72 million in the prior-year quarter excluding $9 million in stock-based compensation.

Cash, cash equivalents and marketable securities totaled $1.89 billion as of March 31, down from $1.95 billion at the end of 2025. Gravier said the company’s financial position supports its commercial and R&D portfolios and provides flexibility for “strategic and disciplined” business development.

Klein said PTC is evaluating business development opportunities that could leverage its global rare disease commercial infrastructure while maintaining a strong financial position.

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.

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