Oct 29, 2026 earnings call — Sephience global launch and new Fabry gene therapy
Raised full-year product revenue guidance to $960 million to $990 million and total revenue to $1.29 billion to $1.32 billion.
Revenue $0.4B (est $0.3B) · EPS $-0.62 (est $0.14)
Key quotes
"Beginning with Sephience, we are now 1 year into the global launch, which by all measures has been a great success and all signs point to Sephience being a blockbuster rare disease product."
"ST-920 is a onetime administered AAV gene therapy that provides a humanized copy of the GLA gene, enabling the body to produce sustained physiological levels of the alpha-Gal A enzyme deficient in the disease."
"Due to FDA scheduling, the Type C meeting to discuss the votoplam data package and a potential path to accelerated approval is now planned for the first quarter of 2027."
Analyst Q&A 14
Sephience launch stage and growth
Q (Kristen Kluska · Cantor): Where they are in the Sephience launch in the U.S. and abroad, and how growth should be thought about; also asked about FDA webinars and rare disease trial tailoring.
A: Management said they are at the 1-year launch mark and have delivered more than expected, but remain in the early innings with many patients yet to try Sephience; they expect steady growth in the U.S. and room to expand ex U.S. They view FDA emphasis on rare disease flexibility as positive and expect it to help their pipeline discussions and programs.
"I think by everyone's expectations, one, we've delivered far more than was expected." — Matthew Klein (CEO)
U.S. vs ex-U.S. sales split
Q (Tazeen Ahmad · BofA): How they think sales will split between U.S. and ex U.S., and whether the U.S. launch is slowing.
A: Management said most of the current >2,000 active patients and most revenue remain in the U.S., and they expect the majority of peak revenue to come from the U.S. while ex U.S. will contribute strongly as more countries gain access. They do not see the U.S. launch slowing but expect steady, durable quarter-over-quarter growth after an initial acceleration phase.
"The majority of those remain U.S. patients, the revenue, the majority of which still comes from the U.S." — Matthew Klein (CEO)
Guidance drivers and BD appetite
Q (Benjamin Burnett · Wells Fargo): What the increased top-line guidance includes (Sephience, DMD franchise, other project revenue) and their appetite for future business development.
A: They said the guidance raise is driven largely by conviction in Sephience; the DMD franchise has outperformed earlier but Emflaza faces generic pressure and Translarna had an unusual one-time Russian order not expected to repeat. On BD, they said they have $2 billion cash, global commercial capacity, will be disciplined, and want to create shareholder value with selective deals.
"Clearly, a lot of that comes from our confidence in Sephience and the continued growth of Sephience as we move into the fourth quarter." — Matthew Klein (CEO)
Seasonality and patient adherence
Q (Barclays): Whether summer seasonality affected Q3 and if Q4 could accelerate into fall; plus current persistency and dropout rates.
A: They said the launch is global so summer seasonality had some regional impact but overall they still saw steady growth and new patient adds across geographies. On adherence they reported continued strong adherence with about low 20% cumulative discontinuations to date and suggested discontinuations are nearing a steady state.
"we've been around low 20% cumulative discontinuations to date." — Matthew Klein (CEO)
Pricing talks in Europe
Q (Lut Ming Cheng · JPMorgan): Update on pricing negotiations in Germany and timing in France, Spain, Italy; and impact of holidays on decisions and Q4/1Q dynamics.
A: They said negotiations with Germany's GBA are active and a decision is expected fairly soon despite some summer holiday delays; launch in Germany continues well with centers of excellence and switches occurring. For France, Italy and Spain they are engaged and expect pricing and reimbursement finalizations in the first half of 2027; U.S. case teams are preparing for insurance resets and feel well ahead of that process.
"We're still in active negotiations with GBA on the pricing and reimbursement in Germany, and we expect the decision to come out actually fairly soon, fairly shortly." — Eric Pauwels
ST-920 market prep and patient targets
Q (Eric Joseph · Citi): How they expect a ST-920 (Fabry) launch to go on market access, pricing, and who early adopters might be if approved.
A: They said they are preparing market access workstreams now and must first clearly segment patients (naive, newly diagnosed, or on enzyme replacement). They highlighted STAAR study evidence of stability, durability and quality-of-life benefits, patient advocacy excitement, and potential cost savings versus enzyme replacement that could shape early adopters and access discussions.
"we have to first really clearly understand the patient segment." — Eric Pauwels
FDA meeting timing for votoplam
Q (Faisal Khurshid · Jefferies): Any context on FDA scheduling for the votoplam meeting and whether the timing means anything specific.
A: They said the meeting timing is driven by PDUFA scheduling and FDA coordination and that delays reflect FDA scheduling logistics, not a substantive signal. They remain focused on preparing to share votoplam data and see the FDA timing as routine scheduling.
"this is simply a matter of FDA scheduling." — Matthew Klein (CEO)
Why some patients still on Kuvan
Q (Joseph Thome · TD Cowen): Why some patients remain on Kuvan instead of switching to Sephience, and how many prior-treatment failures are still under care versus lost to follow-up.
A: They said reasons vary: center visit frequency, throughput limits, and payer discussions can delay switching, but access and payer issues are typically timing matters their teams manage. They also noted many patients are connected via dietitians and social networks; social media success stories drive market pull and bring patients back to clinic.
"access questions and payer questions become a when, not an if." — Matthew Klein (CEO)
Votoplam trial design and ST-920 review
Q (Maxwell Skor · Morgan Stanley): Would accelerated approval change INVEST-HD design or enrollment, and any chance of priority review for ST-920?
A: They said INVEST-HD was designed to potentially serve as a confirmatory study for accelerated approval and includes features (interim analysis, global sites) that mitigate enrollment impact if U.S. accelerated approval occurs. For ST-920 they requested priority review and noted it has RMAT designation, so priority review is possible.
"that Phase III study when it was set up was really set up to have the potential to serve as a confirmatory study in the context of an accelerated approval." — Matthew Klein (CEO)
Sephience dosing and Fabry OpEx/accresivity
Q (Asim Rana · UBS): Whether average dose per patient changed since launch, and how to think about OpEx to support the Fabry program and how quickly ST-920 could be accretive.
A: They said average weight-based dosing has stayed around 45 kilogram to 50 kilogram as expected, with newborns now starting therapy. On Fabry OpEx they expect only incremental spend because the commercial infrastructure exists, and they believe the $111 million ST-920 acquisition could be accretive in 2027.
"we expected the average weight to be somewhere in the 45 kilogram to 50 kilogram ballpark." — Matthew Klein (CEO)
Leading indicators for U.S. penetration
Q (Kevin Meli · RBC): What leading indicators show they can reach additional U.S. target populations and are any quantifiable.
A: They listed indicators such as start forms, direct prescriber and patient community interactions, inbound demand to PTC Cares, and internal center-by-center and prescriber-level data. They also cited Ambassador, peer-to-peer, and advisory programs working with centers of excellence to identify unmet needs and drive deeper penetration of the 17,000 eligible U.S. patients.
"we have 17,000 patients in the U.S. that are eligible for this, for Sephience, and we have still quite a ways to go." — Eric Pauwels
PIVOT-HD longer-term data disclosure
Q (Basma Radwan Ibrahim · Leerink Partners): Whether they plan to disclose 30- or 36-month PIVOT-HD data with Novartis.
A: They said disclosure timing and decisions about PIVOT-HD longer-term data are for Novartis to decide and that their focus is on preparing for the FDA discussion on existing data and enrolling INVEST-HD.
"Yes, I think that's going to be Novartis' call." — Matthew Klein (CEO)
Adherence and discontinuation trends
Q (Luke Herrmann · Baird): Whether renewal rate remains near 90% and if discontinuations could fall as more stable Kuvan switch patients are treated.
A: They said prescription renewal rates remain high and cumulative discontinuations are near a steady low-20% level; they expect switch patients (previously adherent on Kuvan) to have even higher adherence, supported by data showing greater response and diet liberalization after switching. They expect adherence to improve as more switch patients come on.
"we expect the adherence rate in the switch patients to be much higher than what we've observed to date." — Matthew Klein (CEO)
Experience with Kuvan switchers
Q (Alexander Nackenoff · Truist): More color on who is switching to Sephience and whether early prescribers were testing hard patients before switching stable Kuvan patients.
A: They said switch patients show compelling intra-subject AMPLIPHY data with significantly greater phenylalanine reduction and about 100% greater increase in natural protein on average, leading to quality-of-life benefits; physician and patient enthusiasm plus social and meeting presentations are driving sustained switch interest across prescribers regardless of how experienced they are with Sephience.
"These are folks who very much understand the biochemical basis of the fact that their patients are quite simply going to do better as the data are continuing to show, and there's a great deal of enthusiasm to do this." — Matthew Klein (CEO)