PTC Therapeutics narrows 2025 revenue guidance to $750M–$800M as Sephience launch accelerates

Earnings Call Insights: PTC Therapeutics (PTCT) Q3 2025

MANAGEMENT VIEW

* CEO Matthew Klein opened the call by highlighting "the initiation of the Sephience global launch in Europe and the United States" and described Sephience as "the foundational product for PTC's sustainable growth and near-term path to profitability." Total third quarter revenue was $211 million, which included the first revenue from the Sephience launch and ongoing contributions from the DMD franchise. Klein stated, "we are narrowing our 2025 full year revenue guidance to $750 million to $800 million, the upper end of the initial revenue guidance range."
* Sephience generated $19.6 million in revenue as of September 30, with $14.4 million from the U.S. and $5.2 million ex U.S. The company received 521 patient start forms from U.S. centers by the end of the quarter. Klein noted the broad demand, saying, "we are seeing demand from all key patient segments," indicating uptake among adults, various disease severities, and different treatment histories.
* At the International Metabolism Meeting, Klein reported, "Sephience treated resulted in an average of 70% greater reduction in phenylalanine levels compared to BH4," and additional benefits were observed in cognitive function and diet liberalization.
* Chief Business Officer Eric Pauwels shared that Sephience had 341 patients on commercial therapy globally by quarter-end, and that U.S. payer engagement was "overwhelmingly positive," with more than 35 payers covering approximately 250 million lives. Pauwels added, "U.S. payers continue to recognize the highly differentiated profile and strong value proposition of Sephience."
* Chief Financial Officer Pierre Gravier stated, "Total revenue for the third quarter was $211 million. Starting with Sephience. Net product revenue in the quarter was $19.6 million, as of September 30." Gravier also highlighted a cash position of $1.68 billion at quarter-end and detailed the company's purchase of 90% of the Sephience global net sales payment obligation for $225 million upfront.

OUTLOOK

* The company narrowed its 2025 full year revenue guidance to $750 million to $800 million, attributing the upper end confidence to early Sephience results and stable DMD franchise performance. Pierre Gravier said, "this is the upper end of our initial guidance, which highlights our confidence in our ability to execute on our launch and all our products."
* Klein indicated that guidance was previously wide due to uncertainty around Emflaza's performance amid generic competition, but consistent results allowed for the range to narrow.
* No explicit EPS or revenues guidance was compared to analyst estimates.

FINANCIAL RESULTS

* Third quarter revenue was $211 million, including $19.6 million from Sephience, $86 million from the DMD franchise (Translarna at $51 million and Emflaza at $35 million), and $71 million in royalty revenue from Evrysdi.
* Non-GAAP R&D expense for Q3 was $91 million, excluding $9 million in noncash stock-based compensation. Non-GAAP SG&A expense was $74 million, excluding $10 million in noncash stock-based compensation.
* The cash, cash equivalents, and marketable securities position was $1.688 billion as of September 30, 2025.
* The quarter included the strategic purchase of the majority of Sephience milestone obligations for $225 million upfront.

Q&A

* Kristen Kluska, Cantor Fitzgerald: Questioned confidence in Sephience's durability beyond the initial launch. Klein responded, "we're consistently hearing...great response" from physicians and patients, citing examples of severe patients responding well and initial adherence being positive.
* Tazeen Ahmad, BofA Securities: Asked about magnitude of Phe reduction sought by doctors and payers, as well as expectations for patient retention. Klein described varying expectations for Phe reduction and cited positive feedback on cognitive and mood improvements. Pauwels added, "robust efficacy is seen within days, days and weeks. It's rapid acting."
* Brian Abrahams, RBC Capital Markets: Inquired about the timeline from start form to prescription and patient conversion in Q4. Klein and Pauwels explained that processing is progressing well, with commercial payers providing fast approvals and government plans expected to take longer.
* Lut Ming Cheng, JPMorgan: Requested color on the sustained momentum of start forms. Klein said rates were "pretty consistent" and noted ongoing monitoring as the quarter progresses.
* Judah Frommer, Morgan Stanley: Sought explanation for narrowed guidance and Sephience penetration in centers of excellence. Gravier attributed confidence to Emflaza stability and Sephience uptake; Pauwels reported "100% awareness of Sephience in each one of these centers."
* Additional questions covered patient demographics, international market dynamics, pricing, refill rates, and response tracking, with management consistently noting it is early in the launch and more detailed metrics will follow as data matures.

SENTIMENT ANALYSIS

* Analysts generally expressed positive and congratulatory tones regarding the Sephience launch, frequently referencing the strong start and seeking clarity on sustainability and broader adoption.
* Management maintained a confident and upbeat tone in prepared remarks and responses, with Klein stating, "we're excited about the performance for the quarter and the strong start to the Sephience launch." During Q&A, management was measured but optimistic, emphasizing early positive signals and ongoing data collection, and occasionally cautioning that it is "early days."
* Compared to last quarter, both analysts and management maintained a positive outlook, with increased focus on Sephience results and launch execution.

QUARTER-OVER-QUARTER COMPARISON

* The current quarter shifted from anticipation and preparation for Sephience's launch to active reporting on early commercial performance and patient uptake. Last quarter, focus was on regulatory approvals and the expected market potential.
* Guidance was previously $650 million to $800 million and is now narrowed to $750 million to $800 million, reflecting increased visibility and confidence in both Sephience and Emflaza stability.
* Management tone evolved from preparatory optimism to confident execution, supported by explicit revenue contributions from Sephience and broad engagement with payers and physicians.
* Analysts this quarter probed more on launch durability, payer dynamics, and patient retention, while last quarter focused on launch preparations and initial market entry.

RISKS AND CONCERNS

* Management acknowledged it is still "early days" for Sephience, emphasizing the need to monitor patient retention and real-world efficacy as the launch matures.
* Payer mix and final coverage policies, especially for government payers, remain areas to watch, with expectations for stabilization as plans finalize their policies in Q4.
* International markets present reimbursement and access timelines, particularly in Europe and anticipated launches in Japan and Brazil.
* Analysts asked about patient retention, refill rates, and the potential for competition-driven market erosion, which management addressed by citing early refill momentum and broad initial uptake, but did not provide long-term retention data.

FINAL TAKEAWAY

PTC Therapeutics reported a strong start to Sephience’s global launch, which contributed to narrowing 2025 revenue guidance to $750 million to $800 million. Management highlighted robust early uptake, positive payer engagement, and broad patient interest across disease severities and geographies. While early results are promising, the company emphasized continued data collection and monitoring as the launch progresses, with confidence that Sephience will serve as the cornerstone for sustainable growth and near-term profitability.

Read the full Earnings Call Transcript [https://seekingalpha.com/symbol/ptct/earnings/transcripts]

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