PTC Therapeutics Q3 2026 Earnings Call Transcript
PTC Therapeutics (NASDAQ:PTCT) reported third-quarter financial results on Thursday. The transcript from the company’s third-quarter earnings call has been provided below.
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Summary
PTC Therapeutics reported record product revenue of $358 million for Q3 2026, including $182 million from Cephiance sales, leading to revised full-year revenue guidance of $1.29 to $1.32 billion.
Cephiance, a rare disease product, continues to show strong global growth, with over 2,000 active patients and a significant opportunity for expansion in both the U.S. and international markets.
The acquisition of the BLA-stage Fabry disease gene therapy asset ST920 is expected to complete its BLA submission in Q4 2026, with potential approval and launch in 2027.
R&D and SG&A expenses were well managed, with significant cash reserves of $2.13 billion as of September 30, 2026, supporting further strategic initiatives and business development opportunities.
The strategic focus remains on expanding Cephiance’s market penetration, particularly among therapy-naive adults and patients switching from other treatments, alongside preparations for the potential launch of ST920 for Fabry disease.
Full Transcript
OPERATOR
Ladies and gentlemen, thank you for standing by. Welcome to the PTC Therapeutics third quarter 2026 earnings conference call. All participants are in a listen-only mode. After the presentation there will be a question-and-answer session. Today’s conference is being recorded. I would now like to turn the conference over to Ellen Cavalieri, Head of Investor Relations. Please go ahead.
Ellen Cavalieri (Head of Investor Relations)
Good afternoon, and thank you for joining us to discuss PTC Therapeutics’ third quarter 2026 corporate update and financial results. I am joined today by our Chief Executive Officer Dr. Matthew Klein, our Chief Business Officer Eric Powell, and our Chief Financial Officer Pierre Gravier. Today’s call will include forward-looking statements based on our current expectations. These statements are subject to certain risks and uncertainties, and actual results may differ materially.
Please review the slide posted on our Investor Relations website in conjunction with the call, which contains information about our forward-looking statements, and our most recent quarterly report on Form 10-Q and annual report on Form 10-K filed with the SEC, as well as our other SEC filings, for a detailed description of applicable risks and uncertainties that could cause our actual performance and results to differ materially from those expressed or implied in these forward-looking statements.
Additionally, we will disclose certain non-GAAP information during this call. Information regarding our use of GAAP to non-GAAP financial measures and reconciliation of GAAP to non-GAAP are available in today’s earnings release. I will now pass the call over to our CEO, Dr. Matthew Klein.
Matthew Klein (Chief Executive Officer)
Thank you all for joining us today. I’m pleased to share the results of another quarter of record product revenue and solid execution across the company. In the third quarter we achieved total revenue of 358 million, including 276 million of product revenue, led by continued quarter-over-quarter growth of Cephiance sales in the U.S. and internationally. Based on this performance, we are raising our full-year 2026 product revenue guidance to 960 to 990 million, and now expect total revenue of 1.29 to 1.32 billion.
This updated guidance represents 64 to 69% in year-over-year product revenue growth. In addition, with our continued effective expense management, we expect to achieve our goal of reaching cash flow breakeven in 2026. Beginning with Cephiance, we are now one year into the global launch, which by all measures has been a great success, and all signs point to Cephiance being a blockbuster rare disease product. In the third quarter, worldwide Cephiance revenue was 182 million, led by consistent growth in the U.S. and continued expansion internationally, and as of September 30th we have over 2,000 active patients on Cephiance worldwide. In the U.S. we continue to see strong underlying demand across all patient segments.
Over the course of the third quarter we saw an uptick in branded and generic Kuvan switches, with continued penetration into the therapy-naive and tried-and-failed patient segments. Adherence remains strong, reinforcing the meaningful benefits patients are experiencing on therapy, including diet liberalization, cognitive function, and mood improvements. Internationally, we also had continued growth in the quarter, led by the strong launch in Japan as well as new patients granted access through named-patient programs across several regions. As we have discussed, we expect international revenue to continue to grow with acceleration points as we achieve access and reimbursement in additional countries over the course of 2027. In the first year of launch, we built a broad commercial patient base upon which we are well positioned to expand as we move forward into 2027 and beyond.
In the U.S. there remains a large number of patients still on branded or generic Kuvan who are candidates for switching, as well as a large number of patients not on any therapy that could benefit from Cephiance. Overall, the vast majority of the 17,000 patients in the U.S. have yet to try Cephiance. We see strong underlying demand across all patient segments fueled by the differentiated profile of Cephiance and the growing real-world evidence of meaningful Cephiance benefits.
Thus, while we are proud of all we have accomplished over the past year, we believe there is still a long way to go in the launch. Furthermore, our teams have implemented plans to address a number of the key launch learnings that will support continued success in the coming years. Internationally, we expect continued growth in 2027 as we expand into new markets in Europe, Latin America, the Middle East, Russia, and the Commonwealth of Independent States, all geographies where we have experienced teams with a track record of success. Overall, we remain confident in the 2 billion plus peak sales opportunity for Cephiance.
In the third quarter we acquired the BLA-stage Fabry disease gene therapy asset ST920. ST920 is a one-time 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. The data collected to date demonstrate that ST920 significantly increases alpha-gal A activity, decreases the lyso-Gb3 substrate that causes organ damage, and improves renal function and other aspects of disease.
In addition, the safety profile has been demonstrated to be favorable, with no serious adverse events related to hepatotoxicity or other immune reactions. We expect to complete the rolling BLA submission for accelerated approval in Q4 2026, with the potential for approval and commercial launch in 2027. Given the size of the Fabry population, our existing commercial infrastructure and experience working in the Fabry community, as well as the differentiated profile of ST920, we believe this is a potential meaningful growth global commercial opportunity.
Turning to the Vodaflam Huntington’s disease program, due to FDA scheduling the Type C meeting to discuss the Vodaflam data package and a potential path to accelerated approval is now planned for the first quarter of 2027. Enrollment continues in the Phase 3 InvestHD trial of Vodaflam, which is planned to enroll approximately 770 early symptomatic patients. The primary endpoint of the study is change in cUHDRS from baseline up to 36 months, with an interim analysis planned for efficacy and futility.
Excellent progress on enrollment has been made. With sites now open across the globe for the Friedrich’s ataxia program, we initiated the global PRUVFA trial in the third quarter. As expected, PRUVFA is a single-arm study with a well-matched natural history comparator arm intended to support NDA resubmission. The study design, protocol, and statistical plan have all been reviewed by FDA ahead of trial initiation. The study is expected to enroll approximately 120 individuals between 7 and 21 years of age, with the primary endpoint being the MFARS disease rating scale.
In the third quarter we also made good progress on our other pipeline programs. We initiated a Phase 2a study of PTC844, our next-generation oral DHODH inhibitor. As we shared at our R&D Day last December, PTC844 has a differentiated potency and selectivity profile that benchmarks very well against approved DHODH inhibitors. The Phase 2a trial is a 12-week PK/PD study being conducted in a cohort of adults with rheumatoid arthritis, with the objective of confirming the relationship between dose, DHODH inhibition, and favorable effects on markers of B-cell and T-cell immunity.
We plan to use the results of this study to help finalize which rare inflammatory disorders we will pursue for PTC844 development. The Phase 1 study of PTC612, our oral NLRP3 inhibitor, is progressing well. We have dosed several SAD and MAD cohorts, including a cohort with inflammation and cardiovascular disease that can provide early evidence of pharmacodynamic effect on a number of biomarkers. We believe PTC612’s potency, selectivity, and the potential for improved tolerability are all key points of differentiation. We plan to focus development efforts on rare pulmonary indications where NLRP3 pathology has been closely linked to disease.
Finally, we continue to advance PTC303, our splicing development candidate targeting MSH3. PTC303 is our next candidate from our oral small-molecule RNA splicing platform and is an example of how we are extending the reach of the platform into new and highly innovative areas of biology by targeting somatic expansion. PTC303 has potential application in a number of triplet repeat expansion disorders. As we have discussed, we plan to focus on DM1 as well as Huntington’s disease, where we believe PTC303 can offer a complementary approach to HTT lowering.
We remain on schedule for PTC303 to enter the clinic in 2027. In conclusion, I’m proud of our team’s continued strong execution as we move into the fourth quarter. I will now turn the call over to Eric for a commercial update.
Eric
Thanks, Matt. Our commercial team delivered another quarter of record product revenue, highlighted by the continued strength of the Cefiance global launch. Launch momentum remains robust, supported by steady growth in the US and increasing contributions from international markets. In the third quarter, Cefiance revenue was 182 million, representing over 20% growth from the second quarter of 2026. More than a year into the launch, we have built a strong foundation that reinforces our confidence in our ability to deliver sustained long-term growth for the brand.
Importantly, as Matt mentioned, more than 2,000 PKU patients are actively benefiting from Cefiance therapy today, providing a large, growing base for future growth in 2027 and beyond. In the US, underlying demand remains strong with a steady cadence of new patients and seamless reauthorizations each quarter since the launch. As a reminder, all US Centers of Excellence are now prescribing Cefiance and our field-based teams are now focused on how to penetrate deeper into each center. The teams are leveraging claims data and their prescriber interactions to tailor a center-specific approach as we build our base of knowledge, targeting key PKU patient segments and rolling out key programs to healthcare providers and patients.
Going forward, we have recognized the value of organic peer-to-peer sharing of real-world experience and have built a framework, including a new Ambassador program, leveraging identified Cefiance champions to share their positive experiences across multiple channels. The program is focused on the US initially and we’ll extend the concept globally where applicable. We continue to enhance our digital presence for both branded and unbranded activities, targeting messages across channels and media types to support and re-engage the PKU community through the web, social media, programmatic targeting, and live Navigating PKU events.
Internationally, we saw continued momentum from the launch in Japan and continued growth in new patient starts in the Middle East, North Africa, Latin America, and Canada. In Europe, we continue to actively negotiate pricing and reimbursement in Germany, Spain, Italy, and France with the objective of maintaining a narrow global pricing corridor. Our highly experienced market access teams are well positioned to accelerate the global rollout and continued expansion in 2027.
As Matt mentioned, Cefiance’s first year on the market has been a success by every measure and we are just getting started. Of the approximately 58,000 individuals with PKU in target markets worldwide, the vast majority have yet to try Cefiance. Building on our first-year learnings, we have three key priorities to expand the market, reach more patients, and accelerate growth. The first priority is to continue to unlock the substantial opportunity among therapy-naive adults. In Q3, more than one quarter of patients actively treated with Cefiance were therapy-naive adults, many returning to care after years without treatment. We now better understand their treatment goals and care journeys, enabling more targeted initiatives to reach this important patient segment.
The second priority is to expand adoption among patients switching from branded or generic Kuvan. Early in the launch, prescribers prioritized untreated or poorly controlled patients, including those with more challenging PKU, before broadening use among patients already on therapy. AMPLIFY supports switching to Cefiance. In the extension study, every responding patient achieved further Phe reductions and increased protein intake after switching.
Combined with favorable US payer policies and positive international health technology assessments, these data support continued expansion in this segment. The third priority is to build the real-world evidence supporting Cefiance’s potential across the full spectrum of PKU severity. Physician-led studies show encouraging results in patients with classical PKU, including those with non-BH4 responsive mutations, with Phe reductions and diet liberalization comparable to those seen in patients with less severe mutations.
Together with the opportunity for early treatment in newborns and children, as well as treatment-naive adults and patients switching from existing therapies, this growing body of evidence reinforces Cefiance’s potential to address some of the most severe forms of PKU and patients with the greatest unmet need. We are applying launch learnings and executing activities that support these three priorities to expand adoption, reach more patients across the PKU spectrum, and drive sustained growth across all PKU patient segments.
While our teams are focused on the continued success of Cefiance, we are also excited about the potential launch of our next commercial product, the ST920 Fabry disease asset. Many of our PTC leadership and customer-facing teams have significant experience in lysosomal storage disorders and especially Fabry disease. We are excited about the potential to deliver a one-time administered therapy that provides the body the ability to produce continuous physiological levels of humanized alpha-Gal to individuals living with Fabry disease.
We are advancing our US commercial preparation, including mapping claims data with Centers of Excellence, and importantly we believe the Fabry patient community will play a key role in treatment adoption, and our initial meetings suggest strong support for this innovative approach. In parallel, we are developing the long-term durability value proposition for payers and leveraging our established US market access capabilities as we advance towards completing our FDA submission by year end.
In parallel, we are preparing to bring ST920 to the Fabry community as quickly as possible, marking another potential significant milestone for PTC Therapeutics’ continued growth in rare diseases. Now turning to our mature brands, we continue to generate meaningful revenue in the third quarter from both Emflaza and Translarna. For Emflaza, we delivered 25 million of revenue and continue to see strong brand loyalty despite multiple generics in the market. For Translarna, revenue in the quarter was driven by ongoing sales in Europe, Latin America, the Middle East and North Africa, and another government order from Russia. In summary, our customer-facing teams delivered another strong quarter, including record product revenue performance, underscoring the strength of PTC Therapeutics’ global rare disease commercial capabilities.
We remain confident in our ability to grow and establish Cefiance globally as the standard of care for PKU. With that, I will now turn the call over to Pierre for a financial update.
Pierre Gravier (Chief Financial Officer)
Thank you, Eric. I will now share the financial highlights of our third quarter of 2026, beginning with top line results. Total products and royalty revenue for the third quarter was 358 million. Total net product revenue across the commercial portfolio was 276 million, compared to 131 million for the third quarter of 2025, representing over 100% growth. Third quarter 2026 product revenue includes Cefiance’s net product revenue of 182 million and DMD franchise revenue of 74 million.
Translarna net product revenue was 49 million, including a government purchase order from Russia, and Emflaza net product revenue was 25 million. For Evrysdi, Roche achieved third quarter global revenue of approximately 607 million US dollars, resulting in royalty revenue of 82 million for the third quarter of 2026. Non-GAAP R&D expense was 95 million dollars, excluding 11 million in non-cash stock-based compensation expense, compared to 91 million for the third quarter of 2025, excluding 9 million in non-cash stock-based compensation expense. Non-GAAP SG&A expense was 67 million for the third quarter of 2026, excluding 13 million in non-cash stock-based compensation expense, compared to 74 million for the third quarter of 2025, excluding 10 million in non-cash stock-based compensation expense.
Cash, cash equivalents, and marketable securities totaled 2.13 billion as of September 30, 2026, compared to 1.95 billion as of December 31, 2025. In the third quarter of 2026, we acquired ST920 for an upfront payment of 111 million and up to 100 million in contingent milestone payments tied to US regulatory approval. We view the acquisition as an opportunistic business development transaction that adds a BLA-stage asset with meaningful near-term revenue potential while leveraging our current infrastructure and supporting our broader financial objectives. This value-creative transaction also preserves significant financial flexibility to pursue additional business development opportunities and support our path to cash flow breakeven and long-term sustained profitability.
I will now turn the call over to the operator for Q&A.
OPERATOR
Thank you, ladies and gentlemen. If you have a question or a comment at this time, please press star 11 on your telephone. If your question has been answered or you wish to remove yourself from the queue, please press star 11 again. We will pause for a moment while we compile our Q&A roster. Our first question comes from Kristin Kleska with Cantor. Your line is open.
Kristin Kleska (Analyst)
Hi, good afternoon. Congrats on a strong quarter and thanks for taking the questions. I wanted to ask where you would characterize yourself in the stage of the launch of Cefiance, both in the US and ex-US, and how we should be thinking about the growth in both regions respectively.
Matthew Klein (Chief Executive Officer)
Hi Kristin, thanks for the question. So first let me just say how incredibly excited we are about now being at the one-year launch mark, and I think by everyone’s expectations, one, we’ve delivered far more than was expected, and I think we’ve really redefined what the opportunity can be in PKU globally for a well-differentiated therapy, and we’ve done a really great job in the first year. But I think we very much believe we’re still in the very early innings here.
We have a very long way to go. We talked about 2,000 patients on therapy out of 58,000 globally. You know, take the US — we’re thinking, you know, we probably have at least 85% of PKU patients not yet tried Cefiance, and clearly that number is much bigger outside the US. So in this first year, what we’ve really done is built a strong, broad patient base that has allowed us to really accelerate the launch, delivering strong revenue numbers in the first year, but also set the stage as we now move deeper and deeper into each of the patient segments that are now well defined and, as Eric mentioned, have tailored programs to leverage what we see as a very significant opportunity that’s both in the US and abroad. And look, we know we see a lot of people externally trying to track prescription data and predict where this launch is going.
When we look at the real data, what we see is strong underlying demand, consistent growth in the US, room for strong long-term durable growth in the US, the ability now to grow ex-US as we get into additional countries. And everything we see reaffirms our confidence that [we are] on that trajectory for the 2 billion dollar plus opportunity we’ve talked about.
Kristin Kleska (Analyst)
Okay, thanks. And last question for me is we’ve seen the FDA out with a lot of public webinars specifically focused on tailoring trials for rare disease therapies in particular. So I’m curious if you’ve thought about that and if you think there’s any read either for programs that are currently in late-stage development and/or as you start to think about your own pipeline and platforms as you progress that into the clinic. Thanks again.
Matthew Klein (Chief Executive Officer)
Yeah, absolutely. I think we could all agree it’s a really exciting time with what’s going on for rare disease at FDA. And I think this has gotten to the top of the agenda in every part of the FDA. I’ve personally been a part of a number of discussions and there’s been the public discussions where we’re hearing importance of CBER and CDER alignment for rare disease, showing appropriate flexibility, that what’s been the standard approach for diabetes and hypertension doesn’t apply to rare disease.
And we really need a bespoke approach for each therapy. And this is something that was clearly articulated again yesterday at the Rare Disease Framework Forum. So I think, not talking about anything specific, but for us in general, I think this is really great with upcoming discussions on Vodaflam, with the Fabry program and other important programs we have in the pipeline, being able to take part in this evolution of uniform alignment, consistency, predictability and a true understanding of the rare disease challenges. So I think overall it’s a great thing and hoping that it’s a high tide that raises all rare disease boats, if you will.
OPERATOR
Thank you. One moment for our next question. Our next question comes from Tazeen with Bank of America. Your line is open.
Tazeen (Analyst)
Hi. Thanks for taking my question on cefiance as it relates to the breakdown between U.S. and ex-U.S. You know, ex-U.S., you do have far more patients in total than you do in the U.S., but the price point is much lower. So I think it would be helpful to get a sense from you, Matt, about how you’re thinking about ultimately the split of contribution of sales coming from U.S. versus ex-U.S. And can you give us a little bit of color on—it’s only been four quarters or so, but is the U.S. launch slowing?
I think that’s what people want to know. Thanks.
Matthew Klein (Chief Executive Officer)
Yeah, absolutely. Thanks, Zazine. I think what we have currently is we said we have over 2,000 active patients now on therapy. The majority of those remain U.S. patients. The revenue, the majority of which still comes from the U.S.—I think that’s very clear in the numbers. And we believe as we continue to go in the launch that probably in the end the majority, when we get to peak, the majority will come from the U.S., but with strong contributions ex-U.S. And part of that, as we talked about, you mentioned the differential price points. 17,000 patients in the U.S. and as I said, probably over 85% of those haven’t tried suppliance yet. And what we’re seeing is very much what you had alluded to, I think, in your recent note in your recent survey about what the potential for durable sales momentum in the U.S., given what a lot of the KOLs you spoke with see as the potential peak penetration in those centers as we move on. And so that’s what we’re seeing. So I think we unquestionably had an acceleration phase in the first few months of the launch.
We really see ourselves now in a steady, durable growth phase. And we see that quarter over quarter. Actually, despite seasonality and some trends and the headwinds in the summer, we still delivered steady growth and we obviously see room for continued growth and increasing growth in some quarters. But overall steady growth in the U.S. And outside the U.S., as we talked about, there’ll be periods of acceleration as we get into pricing and reimbursement and gain access in new markets. The story of Q2 and Q3 was driven by the strong Japan launch and then as we move into 2027, bring more countries on board, into 2018 further, we see a lot more room for growth outside the U.S. So again, as we said, we still think we’re very, very early in the U.S. We got through an acceleration phase that gave us a very strong base, I think far larger than anyone would have imagined at this stage in the launch.
And we have strong adherence rates. And that’s really the key foundation from which we’re going to continue to grow this quarter over quarter, year over year, and get to those numbers that we talked about in terms of peak opportunity.
Tazeen (Analyst)
Okay, thanks, Matt. And maybe just to get a little bit more granular on it, when you say the majority when you’re at peak will be U.S., is the magnitude going to be closer to, I don’t know, 55/45 or is it closer to 70/30?
Matthew Klein (Chief Executive Officer)
I think so. Just to be—let me be careful. So the majority of the revenue will be U.S. The number of patients we expect will be larger outside the U.S. just given because you’re talking 17,000 versus, you know, 41,000 plus/minus in areas we think we can get access and pricing and reimbursement. It’s hard to have an exact number right now. Tazeen, you know, we believe it’s going to be the majority. A little bit is going to determine what we get outside the U.S. in terms of access and what comes through pricing and negotiation.
So the harder part for us to figure out is exactly what that amount is going to be outside the U.S., but again, nonetheless, we remain confident in what we’ve talked about, the $2 billion plus opportunity. We see ourselves on that trajectory to get there by our internal metrics. And again, we have a long way to go and look forward to continuing to deliver for patients.
Tazeen (Analyst)
Okay, thanks, Matt.
OPERATOR
Thank you. One moment for our next question. Our next question comes from Ben Burnett with Wells Fargo. Your line is open.
Ben Burnett (Analyst)
Excellent, thank you. Great to see the guidance increase. Just want to know if you can maybe talk about sort of what the new top-line guidance contemplates. I assume you’re getting some additional conviction around—sort of—but how much—what’s contemplated with regards to the DMD franchise and some of the other elements of the product revenue base?
Matthew Klein (Chief Executive Officer)
Hi Ben, thanks for the questions. So once again, we felt, based on the performance to date and what we see going forward, comfortable raising the guidance. Clearly a lot of that comes from our confidence in suffiance and the continued growth of cefiance as we move into the fourth quarter. The DMD franchise, as we’ve talked about throughout this year, has really been a story of it outperforming our expectations. And I think a lot of that credit goes to our teams around the world who have done an incredible job continuing to deliver for two very—let’s just say two products with a lot of headwinds.
As we move into the fourth quarter, what we’re seeing first on the Emflaza side, we’re now seeing a new generic entrant to the market that has brought down the price quite a bit. So we may very well now be getting closer to that—I’ll say race to the bottom—but we’re starting to see the impact of generics in the marketplace in generic pricing. So that’s obviously impacted our expectations for Emflaza. And on the Translarna side, again in the third quarter we had a Russian order, a Russian government order which was a bit unexpected.
But we do not expect in Q4 to have any additional large government purchase orders from Brazil or Russia. So that’s a lot to say that I think we are increasing guidance, majority of that driven by our enthusiasm for suffiance. Obviously we’ll have contributions from the other products, but we don’t expect the DMD franchise to contribute as it has throughout the earlier parts of 26.
Ben Burnett (Analyst)
Okay, that’s super helpful. And then I also wanted to ask, so I thought that the February deal was super interesting. What is the appetite going forward for additional BD?
Matthew Klein (Chief Executive Officer)
We have talked a lot about the fact that we are aware that we have over $2 billion in the bank and the capacity from a commercial standpoint to add products globally, and we see that as a priority for top-line growth here. Do you want to talk a little bit about how we’re looking at BD opportunities in general?
Pierre Gravier (Chief Financial Officer)
Yeah, absolutely. I mean, look, we have a global infrastructure with capacity and we demonstrated that we know how to commercialize rare disease drugs globally. And I think that’s a key strength of ours and very unique and very rare. And we will apply the same philosophy we mentioned all along. We want to be very disciplined. We have the full flexibility to operate with the $2 billion cash balance and we want to make sure that we create value for shareholders.
Ben Burnett (Analyst)
Okay, great. Thanks so much.
OPERATOR
Thank you. One moment for our next question. Our next question comes from Elena Morrow with Barclays. Your line is open.
Tejas (Analyst)
Hi, this is Tejas on for Ellie. Thanks for taking our question. Can you comment first just on any trends you’re seeing in the U.S. and ex-U.S. around summer seasonality and if that impacted Q, and maybe from what you’re seeing in 4Q, could there be an acceleration into the fall? And then just a quick second question. What’s the latest you’re seeing from current patients on persistency and dropout rates? Thanks.
Matthew Klein (Chief Executive Officer)
Hi, Teja. So on the first question, we did, as I mentioned previously, we did see some summer seasonality, but again, this is the benefit of a global launch. So while we saw maybe one part of the summer vacations in certain regions, other times we did. So I think overall, despite the varied seasonality, we saw a strong quarter. And again, I think this is the benefit of a global launch. And I know, Erin, do you want to talk a little bit just about how we are seeing patterns over the summer as we move forward and the global contribution?
Erin
Yeah, in terms of global contribution, first of all, again, there was some seasonality, but we saw steady and sustained growth. We saw even in the quarter new patient adds. And importantly, we also had a very strong patient base, as Matt mentioned, 2,000—more than 2,000—active patients with continuous refills and reauthorizations, and we’re seeing sustained prescriptions. Now of course, seasonality is only an effect of having patients potentially on holidays and on doctor’s offices, but we’ve seen that move back into a nice steady cadence.
And seasonality also can play out in different geographies. We also saw some of our first prescriptions actually in Latin America in the winter months. And so a lot of this is really part of our whole global learning and global understanding of the launch to date. And then on your second question regarding adherence, we’re still seeing very strong adherence. We had mentioned last quarter that we believe we’re reaching steady state in terms of discontinuations. And again this quarter we’ve been around low 20% cumulative discontinuations to date.
OPERATOR
Thank you. One moment for our next question. Our next question comes from Brian Cheng with JPMorgan. Your line is open.
Brian Cheng (Analyst)
Hey guys, thanks for taking our questions today. Maybe just on pricing negotiations here in next ex-U.S. regions, can you give us an update on the final pricing agreement that will come soon in Germany and also other countries like France and Spain? Thank you.
Matthew Klein (Chief Executive Officer)
Hi Brian. Let me let Eric give you an update on where we’re at with pricing discussions.
Eric
Yeah, Brian, thanks for the question. You know, we’re continuing to have those kinds of discussions. I wish I could tell you we have a final agreement. I think it’s interesting because the summer months literally created a little bit of the delays in the administration over at G-BA, but there’s nothing unexpected at this point. We’re still in active negotiations with G-BA on the pricing and reimbursement in Germany, and we expect the decision to come out actually fairly soon, fairly shortly, but we have nothing specific yet.
The launch, however, in Germany just continues with great progress. And we’ve seen centers of excellence right now, and they continue to see broad access. We’ve seen young patients and older patients, and we’re also seeing switches, which is an important and high-value driver for the business. So Germany will continue to be a major contributor with the narrow corridor that we’re establishing right now. France, Italy, Spain and Southern Europe. I would say first of all, in Southern Europe, we’re very pleased with the ongoing early access programs. Patients have been enrolling very quickly in those programs. There’s been high unmet needs. Pricing and negotiation in these areas, including HTA assessments, take a little bit longer. But we have engaged with pricing and reimbursement in all three countries, and we anticipate somewhere in the first half of 2027 for those to be finalized.
Brian Cheng (Analyst)
And then if I can squeeze in one more, from a quarter-to-quarter dynamics, how should we think about, you know, the number that we should project for 4Q and 1Q? You know, are there potential holiday season impacts and also insurance reset dynamics that we should account for?
Eric
Well, I think in the US our case managers and PTC Cares will be way ahead of that during the enrollment process. We’re already reaching out to make sure that every single patient we understand what their insurance coverage is. We’ll be able to start and begin to get ahead of any of the changes. And importantly, prepare all the programs in the US for the reset, if you will, in January. For us, this is not something new. We went through that in January.
There was already a big lesson that we learned from that. And now that we have thousands of patients that we’re actively working with, and new patients coming in, this is relatively routine for our case management teams to handle. So simple message is, we’re way ahead of that.
Brian Cheng (Analyst)
Great. Thank you guys.
OPERATOR
Thank you. One moment for our next question. Our next question comes from Eric Joseph with Citi. Your line is open.
Eric Joseph (Analyst)
Great. Thanks for taking the question. Just one on ST920. Assuming approval next year, can you orient us a little bit around how you expect that launch to go from the standpoint of facilitating market access and reimbursement, as well as how you might be thinking about pricing? And I guess also, maybe you can describe the type of patient who might be an early adopter here, assuming approval. Thanks.
Matthew Klein (Chief Executive Officer)
Hi, Eric. We’re obviously quite excited about the potential of bringing ST920 to individuals in February, not only in the US but worldwide. As we talked about, we’re just getting the final parts of the BLA together and look forward to that submission in Q4. As you can imagine, Eric and his team have already begun to start exploring a number of your questions. So let me turn it over to Eric to give you a bit of detail on what we’re doing now in terms of market preparation and access.
Eric
Yeah, thanks for the question, Eric. You know, we’re very excited about this opportunity because it’s a near-term opportunity, and we’re leveraging that commercial infrastructure on our teams, including myself and many others. We all have gene therapy experience, lysosomal storage disease, and a very significant amount of our teams actually have Fabry, direct Fabry experience. This market is incredibly large in the US alone, and that’s where our focus is immediately. With over 11,000 patients, what we’ve already learned is that the market itself is not well served, and I think there’s a lot of patients that are still going untreated. We have a number of key workstreams right now in the US to prepare for that. I’ll get to your question about market access, but we have to first really clearly understand the patient segment.
Are they naive, are they newly diagnosed, or are they on enzyme replacement therapy? As we go through that, we have to leverage the information from the STAR study, which shows stability, improvement in some cases in renal and cardiac functions, durability and efficacy going years out, tolerability, and the study even demonstrated quality-of-life benefits, which I think will drive what patients need. But in the end, I think the value proposition that you’re looking at will be segmented towards the right patient.
If you actually look at the costs associated with enzyme replacement therapy and care and others, we see that there is potential cost savings to the healthcare system, but more importantly, really enhanced quality-of-life improvement for patients. And I’ll just say that we’ve had two meetings already with patients, and these are patient advocacy groups for Fabry. And the community is incredibly excited about having a gene therapy that we can bring to the market.
And we already have significant engagement with that community right now to help support those efforts.
Eric Joseph (Analyst)
Great, thanks for taking the question.
OPERATOR
Thank you. One moment for our next question. Our next question comes from Faisal Kershaw. Jefferies, your line is open.
Faisal Kershaw (Analyst)
Hey guys, thank you so much for taking the question. I just wanted to ask for the US, when you’re kind of characterizing this steady, consistent growth trajectory that you expect, what are the key levers for that? Is the bigger lever therapy-naive, and what are the kind of major unlocks to achieve that? Then I have a follow-up as well. Thank you.
Matthew Klein (Chief Executive Officer)
Hi, Faisal. So, as we talked about, we are well positioned to continue to go deeper into all of the key segments. I think early in launch, as we’ve discussed, we had a larger number of patients come from the therapy-naive, which was really a pleasant surprise. Reaffirming that’s really a component of what we see as the addressable market, as well as those who tried and failed previous therapies. And then as we moved into Q2 and further into Q3, we’re beginning to see now more and more the switch dynamic come into play.
And as Eric highlighted in his comments, we’ve been able to look carefully at what we’ve done in the first year and now understand not only on a segment level, but on subsegment levels, center levels, what we need to do to continue to penetrate deeper and deeper into all of those segments. And when we talk about our enthusiasm and our conviction in the potential for significant growth over time in the U.S., it comes from the fact that we know there’s still a large, large number of patients in each of these segments, including somewhere between 2,000, 2,500 patients still on Kuvan branded and generic that we know are all candidates for suffiance.
We know that as our data continue to show, all those who switch over to suffiance do better in terms of lowering and/or protein, diet liberalization and other benefits. But we’ve also made a number of key learnings about those who were in the therapy-naive segment and how can we access them? What’s that patient journey like? And so what we’re really now—this is really the fun part of launch. We take the learnings from the first year, develop targeted plans so that we can leverage the opportunity, the significant opportunity we still have across all of those patient segments as we move forward.
Faisal Kershaw (Analyst)
Got it. That’s helpful. And then just one quick one. Is there any context you can provide on the FDA scheduling meeting for Vodoquan? Like, obviously we’ve had a little bit of a history of turbulence at the FDA, just trying to understand if this is like anything like that or if it’s just simply a calendar thing. Thank you.
Matthew Klein (Chief Executive Officer)
Yeah, absolutely. Look, you know, as everyone knows, there’s PDUFA guidelines that say if you request a Type C meeting, it’s typically granted within 75 days of the request. Clearly, given that both Novartis and PTC guided to this meeting occurring in 2H26, you could imagine that the request for the meeting was submitted at a time that typically would have resulted in that meeting being in the second half of 2026. And FDA gets the meeting request, and you know, sometimes on their end, Faisal, they have to coordinate a number of different people attending these meetings.
They’ve clearly been quite busy at a number of rare disease initiatives. So it’s a long way of saying this is simply a matter of FDA scheduling. There’s nothing to read in there. And we remain excited about the opportunity to share the Vodoquan data—why we believe we have a compelling package and hopefully leveraging the precedent of CUH DRs being the basis of an intermediate clinical endpoint accelerated approval application in HD. Thank you.
OPERATOR
Thank you. One moment for our next question. Our next question comes from Joseph Tomey with TD Cowen. Your line is open.
Joseph Tomey (Analyst)
Hi there. Good afternoon, and thank you for taking my questions. Maybe, Matt, on those patients that you mentioned that are still on Kuvan, maybe what’s the largest reason as to why they’re still on Kuvan and haven’t switched over yet? Is it just touch points? Is insurance a barrier for these patients at all, or kind of what’s going to be the key learning to get them to flip? And then in that patient segment that maybe has tried something else, you know, and it didn’t work for them, what proportion of those are still under care versus maybe have been lost to follow-up, and you have to do a little bit of extra digging to bring them back into the clinic? Thank you.
Matthew Klein (Chief Executive Officer)
Hi, Joe. Okay, I’ll take the first one first. And I think there’s not a one-size-fits-all answer here. Part of the answer is fine. These centers have a limited throughput. There’s only so many individual patients they’re going to see at any one time. And we’ve heard from a lot of the KOLs that a number of these individuals will come in maybe every three months, maybe every six months. And in the early days, the focus was getting those who weren’t on the therapy on the therapy.
Okay. Now we’re switching to a stage where there is more of an interest in getting the switches done. And in each center and for each patient, it may be different in terms of the time, and that may just be getting into the clinic in some cases. Clearly there’s additional payer discussions we need, but our teams have really been able to manage those situations really, really well. So access questions and payer questions become a when, not an if. And furthermore, I think what our teams are really working on are continuing to deliver the value proposition here. You have the AMPLIFY data, which again really reinforces the benefits that individuals have when they switch from branded or generic Kuvan to cephalance in terms of significant improvements. And I think one of the other take-home points here too is that even those who may be somewhat controlled or moderately well controlled on branded or generic Kuvan, when they switch over, they’re finding significant increase in diet liberalization, significant natural protein intake, which all agree is a super important thing for patients.
So again, the value proposition there—it’s a matter of time. We’re working through this and our teams again are developing targeted plans and making sure that we manage through that process of the switches. And it’ll take time. In terms of quantifying those who are in the tried-and-failed and are not currently under active care, it’s a little bit difficult. But what we’re learning, importantly, is they may not be, a lot of these folks may not be under active care in terms of seeing the physician or nurse practitioner. A lot of them are tied into dietitians in the centers. And we’ve also made a lot of important learnings about social media networks and how individuals PKU have been very well connected to each other and while they may not be coming to clinic, they talk to other members of the community. It’s a well aggregated community and we’ve had the opportunity to talk to and understand from a lot of individuals how these networks work.
And that’s why when folks post on social media, starting to buy in, having steak for the first time, pizza for the first time, feeling so much better, brain fog. All those things that appear on social media have incredible, incredible impact in terms of continuing to generate the market pull and getting folks to come back to clinic on their own because they want to enjoy the benefits that their peers, friends and folks they’ve known for years at PKU.
And so again, part of this is what we do, what do we do in terms and what our teams do in terms of the launch. We went through the first stage, we take the learnings, understand these networks, who are the influencers, who are the key parts, how do we support patients on the patient journey, all those different things that we can now bring to bear as we continue to grow throughout the next stage of launch. So great. Thank you very much.
OPERATOR
Thank you. One moment for our next question. Our next question comes from Maxwell Score with Morgan Stanley. Your line is open.
Maxwell Score (Analyst)
Great. Thank you very much for taking my questions regarding Vodaflam. Would an accelerated approval path change Invest HD phase three design or enrollment, given that patients may be less willing to accept placebo if a commercial drug is available? And also, is there any chance you receive priority review for ST920?
Matthew Klein (Chief Executive Officer)
Thank you. Hi Max. On your first question on Vodaflam, that phase three 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. It was also a very thoughtfully designed study, large study population to serve as a pivotal trial if needed. It was also designed to have an internal analysis so that if there was not an accelerated approval based on the Pivot HD data, there’s the opportunity to have success on the trial at an earlier time point in a smaller number of patients.
So all those different factors were built in. Also, I’ll point out this is a global trial and I, you know, it’s so, it’s so impressive to see the Novartis team get this trial up and running and really do an incredible job. A machine of getting study sites up all around the globe. So, you know, even if you had an accelerated approval in the US there’s still going to be study sites all over the world that are going to be open and enrolling where there wouldn’t be an accelerated approval. So cannibalizing potential enrollment really isn’t a consideration in that matter. In terms of the potential for priority review for ST920, clearly we requested, given several factors, given the fact that there really still is a significant unmet need and several, we think relative benefits to the differentiated profile ST920 versus other available therapies.
And furthermore, it also has RMAT designation. So the potential for priority review comes along with that RMAT designation.
Maxwell Score (Analyst)
Great. Thank you very much. Very helpful.
OPERATOR
Thank you. One moment for our next question. Our next question comes from Asim Rena with UBS. Your line is open.
Asim Rana (Analyst at UBS)
Congrats on the quarter and thanks for taking the questions. This is Asam Rana on for June Lee, just on Defiance. Can you remind us if the average dose per patient has moved up since the launch? And then on the Fabry program, how should we be thinking about OPEX to support the launch? Thank you.
Matthew Klein (Chief Executive Officer)
Sure. On the first question, the we said in the beginning we expected average weight. Since it’s weight based dosing, we expected the average weight to be somewhere in the 45 to 50 kilogram ballpark. It stayed there, it’s been pretty stable and that’s how it’s been. We get adults to come on but we also actually recently just had a number of newborns come on which is super exciting to see a newborn get screened for PKU and positive and then be able to go on to science right away and have optimal protein intake in those critical early stages of neurodevelopment.
And I think that the newborn segment, not only in the US but globally is one that we can now also think about starting to penetrate. The 300 plus incident individuals in the US each year and a large number of course internationally. But overall again that average weight stays about even in terms of OPEX. I think as we talked about, since we have the commercial infrastructure built, all those key components are there. We also have a number of experienced people, as Eric mentioned, in every part of the customer facing organization for Fabry it would really be an incremental, incremental spend and incremental build next year.
Asim Rana (Analyst at UBS)
Just a quick follow up. How quickly do you expect the dealer could become accretive for the Fabry program?
Pierre Gravier (Chief Financial Officer)
I’m sorry, could you repeat that?
Asim Rana (Analyst at UBS)
How quickly do you expect the ST920 deal could become accretive?
Pierre Gravier (Chief Financial Officer)
Look, I think one thing that is particularly attractive here in this opportunity is that for $111 million asset acquisition, we believe this could be accretive in 2027.
OPERATOR
Thank you, thank you. One moment for our next question. Our next question comes from Brian Abrahams with RBC Capital Markets. Your line is open.
Kevin
Hi, good evening team. This is Kevin on for Brian. Thank you for taking our questions. So maybe just back on cefiance, can you talk about maybe some leading indicators that you can be looking at in particular in the US to suggest that you would be likely to reach those additional targets populations that you talked about at your expected rate. You mentioned potential social media interactions earlier. Are there any other potential leading indicators you could be monitoring that are quantifiable to some degree?
Thank you.
Matthew Klein (Chief Executive Officer)
Yeah, absolutely Kevin. So as we talked about earlier, we know there’s a lot of folks trying to look for external leading indicators and of course being in the closest. So now we’re in really the real data are the ones that we are seeing in addition to what we see in terms of start forms coming in. Our significant extensive interactions not only with prescribers but also with the patient community and all the different local and national patient communities.
The feedback that we continue to get in terms of demand and interest in trying suffiance. Folks that proactively work at reach out to us, talk to our PTC Cares team. All of those are really important leading indicators to us as well as the overall population that we know is out there, as well as some of our own internal center by center data. We know it centers how many individuals there might be in each of these different segments either under care, who’ve been previously in care. We understand what the timeline might be to get them in. These are all the kinds of things that we follow very closely. As Eric mentioned, one of the important things we’re doing right now is having bespoke center level and even sometimes prescriber level data within each center that can also help us understand what is the potential prescribing base for that individual or that center, what’s there, how much is penetrated, what’s the long way we see that they still have to go.
And those are all the kinds of things we look at. Eric, anything you want to add to that and what gives us the confidence in what we’re seeing on the ground?
Eric
No, I think you covered it really well and I think we’re really highly focused on each one of these centers going deeper to understand what are the unmet needs with those therapy, naive adults and the switches. And I think the ambassador program that we discussed is incredibly important because these are three very important pillars. We have champions patients who have actually on all spectrums of the disease been able to share in many ways and engage with the community about what’s happening with their fee reduction and the consequences not only of diet liberalization, but also cognitive improvements and daily life.
And then we have obviously peer to peer connect and advisory panels. So we are working right now with programs that are plugging into these centers of excellence to really identify the unmet needs and how to bring patients back into those centers and then very importantly, make sure they have all the information going back out for the long term, support these programs that we talked about both web on web and face to face and all this are actively working with these centers.
And these are the ones that are really very important in defining as we penetrate further and further into this, what I think is still a very untapped market. We have 17,000 patients in the US that are eligible for this for suffiance and we have still quite a ways to go.
Kevin
Thank you.
OPERATOR
Thank you. One moment for our next question. Our next question comes from Basma Radwan with Leerink Partners. Your line is open.
Basma Radwan (Analyst at Leerink Partners)
Good afternoon. Thank you for taking our question and congrats on the quarter. We have a question on suffice in the FGS market, do you see similar dynamics to the US market? Meaning do you see the treatment naive segment and the prior treatment failures? They are the primary growth driver early in the launch that slowly shift to the switch segment afterwards or is it different and we do have a follow up, please.
Matthew Klein (Chief Executive Officer)
Yes. So the first markets that we’ve been in in terms of having full access have been Germany and Japan and most recently Japan. And that pattern is very much the same. This desire early on to get those who haven’t been on a therapy on Cephayons again with very positive feedback, including for those who have classical PKU more severe non BH4 mutations. And then as we’re moving further into the launch now, a shift is starting to look at getting individuals who are on what’s known as Bioptin in Japan.
There’s Kuvan here onto suffiance. And I would also comment that I think there’s probably in terms of proportion of population on Kuvan or BH4, I think it’s one of the largest proportions in Japan anywhere else internationally. So that gives us a lot of confidence as well that we have a long way to go over time in the Japan launch.
Basma Radwan (Analyst at Leerink Partners)
Great. Our follow up is about Voroplam. Have you disclosed whether you’re planning to disclose the 36 month data from the Pivot HD trial of Vodoplam in Huntington’s along with your partner Novartis, or is that not announced yet?
Matthew Klein (Chief Executive Officer)
Yeah, I think that’s going to be Novartis’s call. We’ve not had any discussions. Clearly all, all efforts right now are on getting ready for the FDA discussion on the existing data and of course getting the InvestHD study up and running. Well, it’s up and running, but getting it fully enrolled. And as we mentioned in the script, Novartis team is doing a really, really great job of getting that, getting sites up and getting folks into that study.
Basma Radwan (Analyst at Leerink Partners)
Great, thank you very much.
OPERATOR
Thank you. One moment for our next question. Our next question comes from Luke Herman with Baird. Your line is open.
Luke Herman (Analyst)
Thanks for the question. So just a follow up on suppliance discontinuations and adherence dynamics. I think you said it was 90% renewal rate with last quarter’s update and today I think you said 20% cumulative discontinuations again similar to last quarter. So is renewal rate holding up around 90% and I guess building on that Is there any reason to think discontinuation rate can actually come down as you pick up more switching patients who have reliably been on another drug for years?
Maybe. Thanks.
Matthew Klein (Chief Executive Officer)
Yeah, Luke, so the numbers are, as you said, we’re seeing very high prescription rates. And as we commented on discontinuations, we’re getting close to that steady state in the low 20% now. And as you alluded to and we talked about, what makes that number so impressive is that early in the launch, we had folks coming in who were therapy-naive, more severe patients. And to be able to have the success rate that we’ve had in those individuals is incredibly encouraging.
And also, as you highlighted, since all of our data show, and it makes sense from basic scientific biochemical principles, that if you’ve had a response to BH4—Kuvan branded or generic—you should have a better response to sepiapterin. And obviously someone who’s switching from branded or generic Kuvan has already demonstrated him or herself to be adherent to that therapy. So that’s a long way of saying we expect the adherence rate in the switch patients to be much higher than what we’ve observed to date.
Eric, any additional color you want to give on prescription patterns?
Eric
Yeah, and I think you covered it really well. And I think what’s really driving this at an individual center level and individual patient level is the switching. And the switching is very, very important because we’re trying to change the paradigm from just getting to a theoretical goal or an established goal to now normalization. And the data that Matt just described is that patients who respond to generic Kuvan or Kuvan will have X amount of response, 70% greater response, and many of them can potentially actually get to normalized levels.
So part of our goal is to really tailor some of that data and show that to many of the healthcare providers and patients that the goal isn’t to stay on medical foods and stay on Kuvan or anything else, but the goal is actually normalization below 120.
OPERATOR
Thank you. One moment for our next question. Our next question comes from Alex Neckinoff with Truist Securities. Your line is open.
Alex Neckinoff (Analyst)
Hey, guys, thanks for the question. Just wondering if you could expand a bit more on your experience with the Kuvan switchers thus far. Just looking for some color that could give us some confidence that the trends you’re seeing are sustainable. Just curious if these patients are coming from prescribers writing their first prescription for sepiapterin, or perhaps from prescribers who wanted to have more experience in the more difficult-to-treat patients, for example, before prescribing to the stable Kuvan population.
Thanks so much.
Matthew Klein (Chief Executive Officer)
Yep. Hi Alex. I think the success stories of the switch patients are many and are continuing to fuel patient demand to switch and physicians’ and other prescribers’ strong interest in switching. As Eric alluded to, this is really an enriched population, if you will. They are definitely going to be sapropterin responders and going to respond really well if they’ve been on BH4 and even at a small amount. We’re seeing significantly greater lower phenylalanine reduction. We saw that in the head-to-head AMPLIFY study. That’s really compelling data for any prescriber, right, to be able to have actual well-designed head-to-head intrasubject data showing the significant greater reductions, the significant increase in protein intake that we’re seeing.
I think some of our latest numbers are on the order of 100% greater increase in natural protein on average for those who switch. That translates to significant, significant benefits for individuals in quality of life, flexibility of diet, as well as just feeling better and getting the benefits of natural protein. And not only are our field teams out there talking about that, there’s patient-to-patient communication on social media, we’ve got presentations at every local, national, international, global meeting that continue to reinforce this message. And so it’s not a matter of a prescriber’s first sepiapterin prescription, their 50th prescription, or their hundredth. 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. As you could imagine, if you’re on a once-a-day therapy, whether you’re a prescriber, a patient, a parent, if you could switch to another once-a-day therapy that could have greater benefits, well, you know, that’s a pretty strong motivating factor for everyone.
Alex Neckinoff (Analyst)
Thanks so much.
OPERATOR
And I’m not showing any further questions at this time. I’d like to turn the call back over to CEO Dr. Matthew Klein for any further remarks.
Matthew Klein (Chief Executive Officer)
Thank you all again for joining the call this evening. I’m incredibly proud of our team’s outstanding performance again this quarter with record product revenue, continued strong momentum in the sepiapterin launch and, as we’ve alluded to, we see as being quite durable and feel we have a very, very long way to go as we continue on this trajectory to $2 billion plus opportunity. We also look forward to continuing to advancing our other programs that are in the pipeline and look forward to giving an update on those in January.
Thank you all.
OPERATOR
Thank you. Ladies and gentlemen, that concludes today’s presentation. We thank you for your participation. You may now disconnect and have a wonderful.
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