PTC Therapeutics Q4 Earnings Call Highlights

Key Points

• Sephience garnered approvals in the U.S., E.U. and Japan and drove early commercial momentum with $92 million in Q4 sales ($111 million since launch), 946 patients on therapy as of Dec. 31, 2025, broad age/severity uptake and favorable payer access.
• PTC reported full-year 2025 product and royalty revenue of $831 million, ended the year with $1.95 billion in cash after monetizing remaining Evrysdi royalties (sold to Royalty Pharma for $240 million upfront plus up to $60 million), and guided 2026 product revenue to $700–800 million with the potential to reach cash flow breakeven in 2026.
• Pipeline updates: Novartis is set to start the phase III INVEST‑HD trial of Votoplam in H1 2026 (placebo-controlled, 3:2 randomization, ~770 participants), while the FDA signaled Vatiquinone will likely need an additional study—possibly a single-arm trial using a natural history comparator—before NDA resubmission.

PTC Therapeutics NASDAQ: PTCT reported fourth-quarter and full-year 2025 results highlighted by the initial global approvals and early launch performance of Sephience for phenylketonuria (PKU), which management described as the company’s “foundational product” for near-term growth.

2025 results and Sephience launch metrics

Chief Executive Officer Dr. Matthew Klein said 2025 featured “many significant successes,” led by approvals of Sephience in the U.S., E.U., Japan and other countries “all within several months.” Chief Business Officer Eric Pauwels said the company’s customer-facing teams engaged PKU centers of excellence, healthcare providers, payers and advocacy groups to accelerate adoption following FDA and EMA approvals.

For the fourth quarter, PTC reported total net product and royalty revenue of $263 million. Full-year 2025 total net product and royalty revenue was $831 million, which exceeded the company’s prior guidance range of $750 million to $800 million.

Sephience contributed $92 million of revenue in the fourth quarter and $111 million worldwide since launch in 2025. Pauwels said fourth-quarter Sephience revenue was approximately $92 million, including $81 million in the U.S. and $11 million ex-U.S.

PTC also provided early utilization indicators for Sephience:

• 946 patients on commercial therapy worldwide as of Dec. 31, 2025
• 1,134 patient start forms received in the U.S. as of Dec. 31, 2025
• 80% of PKU centers of excellence in the U.S. had written prescriptions for one or more patients

Management said uptake has occurred across disease severities and age groups, including “therapy-naive adults,” with prescriptions written for patients ranging “from newborns to eighty-year-olds.” Klein and Pauwels also cited early indications of high refill rates and low discontinuation rates. When asked about discontinuations, Pauwels said dropouts have been “single digits,” generally due to patient decisions rather than safety or efficacy.

Commercial dynamics: payers, “lost to follow-up” adults, and international rollout

Executives emphasized favorable early reimbursement and payer access, crediting the AMPLIFY head-to-head study versus BH4. Pauwels said payers have been supporting “open Sephience access with very few barriers,” including “no step edits” and refills of six to 12 months before reauthorization. Later in Q&A, he said policies written to date have largely been prior authorizations “to label,” with limited or no step edits, and that some large commercial payers have moved to 12-month refills.

Management also discussed demand from adults described by physicians as “lost to follow-up.” Klein said the label was sometimes misinterpreted as a lack of interest in treatment. He attributed renewed engagement to patients remaining connected through dieticians or community networks and to “a lot of activity on social media” sharing perceived benefits such as dietary expansion, phenylalanine (Phe) control and feeling better.

Internationally, PTC said it expects to expand Sephience’s commercial footprint in 2026, with Klein projecting patients on commercial drug in 20 to 30 countries by year-end 2026. Pauwels said Germany has shown momentum and that reimbursement discussions in Europe are expected to occur mainly in the second half of 2026 as health technology assessment (HTA) and pricing processes progress. The company is also using paid early access programs in parts of Europe while pricing negotiations proceed.

In Japan, where PTC received approval in December, Pauwels said pricing and reimbursement are being finalized and the company anticipates first commercial sales in the second quarter, with more meaningful revenue expected in the second half. He noted there are “about 1,000 PKU patients in Japan” and cited 10 years of orphan drug exclusivity. Pauwels also said PTC received approval “this week” in Brazil and plans to use named patient programs; he cited “over 5,000 diagnosed patients” in Brazil and said more meaningful revenue would likely come toward the end of the year as those programs take time to process.

Other products and 2025 revenue composition

Chief Financial Officer Pierre Gravier said fourth-quarter revenue included contributions from the Duchenne muscular dystrophy (DMD) franchise and Evrysdi royalties. DMD franchise revenue in the quarter was $66 million, comprised of $39 million from Translarna and $27 million from Emflaza.

For Evrysdi, Gravier said Roche reported fourth-quarter global revenue of approximately $584 million, resulting in $79 million of royalty revenue to PTC. Full-year 2025 Evrysdi royalty revenue was $244 million.

On the DMD franchise outlook, management cited headwinds, including “a larger number of generics in the U.S. from Emflaza,” and noted it had continued to generate revenue from mature products while defending Translarna and Emflaza.

Separately, management addressed a question about a Translarna sales allowance in France. Gravier said it was a “one-time France-specific” issue tied to the country’s early access program, in which PTC previously set pricing; when the license was not renewed, France set its own price and charged for the difference.

Pipeline updates: Votoplam and Vatiquinone

PTC provided updates across its development portfolio, including Votoplam for Huntington’s disease and Vatiquinone for Friedreich’s ataxia (FA).

For Votoplam, Klein said an end-of-phase II meeting with the FDA occurred in the fourth quarter of 2025 with alignment on the phase III design. Novartis is expected to initiate the phase III trial, INVEST-HD, in the first half of 2026. Klein described INVEST-HD as a placebo-controlled study with a 3:2 randomization (Votoplam to placebo), targeting approximately 770 participants across more than 30 countries, with a primary endpoint of change in cUHDRS. He said an interim analysis is planned for efficacy and futility, and he indicated the trial could serve as a confirmatory study in the context of accelerated approval or as a registration trial. Klein also said results from the phase II PIVOT-HD extension study are expected in the first half of 2026 once participants cross the 24-month time point.

For Vatiquinone, Klein said the company held a Type C meeting with the FDA in December following a complete response letter (CRL) for the Vatiquinone NDA. According to Klein, the FDA indicated an additional study will be necessary to support an NDA resubmission and stated that the study could be a single-arm study with a natural history comparator. PTC plans to meet with the FDA in the second quarter to align on the open-label protocol and matching strategy for the natural history control arm. Klein cited the FA-COMS natural history database and noted regulatory precedent for using it.

In response to questions on endpoints, Klein said endpoint selection would depend on study duration; he said upright stability appeared most sensitive over 12 to 18 months in MOVE-FA, while effects over longer periods were captured across additional subscales, which could support using mFARS as a primary endpoint in a longer study.

Cash position, 2026 guidance, and break-even goal

Gravier said non-GAAP operating expenses for 2025 came in below the company’s guidance, with non-GAAP R&D and SG&A OpEx totaling $728 million for the year. He reported fourth-quarter 2025 non-GAAP R&D expense of $124 million and non-GAAP SG&A expense of $87 million, both excluding non-cash stock-based compensation.

The company ended 2025 with $1.95 billion in cash, cash equivalents and marketable securities, up from $1.14 billion at year-end 2024. Management attributed the increase to strong commercial execution, disciplined expense management, and the monetization of remaining Evrysdi royalties.

Gravier said PTC sold the remainder of its Evrysdi royalty to Royalty Pharma in December 2025 for $240 million upfront and up to $60 million in sales-based milestones, while retaining the right to receive a $150 million milestone from Roche based on a single-year Evrysdi sales threshold of $2.5 billion. He added that Evrysdi will continue to appear on financial statements under the prior accounting method, but there will be no cash proceeds to PTC.

For 2026, PTC guided to $700 million to $800 million in product revenue, representing 19% to 36% growth versus 2025 product revenue and excluding Evrysdi royalty revenue due to the sale. The company also guided to $680 million to $720 million in non-GAAP R&D and SG&A expense (excluding an estimated $95 million in non-cash stock-based compensation). Based on that framework, management said PTC has the potential to reach cash flow breakeven in 2026.

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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