PTC Therapeutics Q2 2025 Earnings Call Transcript

Quartr
Provided by Quartr
August 7, 2025

Key Takeaways

  • Positive Sentiment: PTC’s SUFIANCE received EU and FDA approvals with a broad label for all PKU subtypes and age groups, positioning it to exceed $1 billion in U.S. revenue and become the new standard of care.
  • Positive Sentiment: Second quarter total revenue reached $179 million, including $96 million from the DMD franchise, marking another solid quarter of financial performance.
  • Neutral Sentiment: After the nonrenewal of Translarna’s EU marketing authorization, PTC leveraged Article 117 agreements to supply paid product in about half of European countries, maintaining roughly 25% of prior EU revenue through 2025.
  • Positive Sentiment: PTC will pay approximately $225 million upfront to acquire the annual global net‐sales payment obligation on SUFIANCE from Scenta, unlocking ~90% of the royalty and representing an accretive use of cash.
  • Negative Sentiment: Revenue for Emflaza continued to erode due to additional generic entrants, though PTC’s support programs have helped sustain patient starts and brand loyalty.
AI Generated. May Contain Errors.
Earnings Conference Call
PTC Therapeutics Q2 2025
00:00 / 00:00

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Operator

Ladies and gentlemen, thank you for standing by. Welcome to PTC Therapeutics Second Quarter twenty twenty five Earnings Conference Call. All participants are in listen only mode. After the presentation, there will be a question and answer session. Today's call is being recorded.

Operator

I would now like to turn the call over to Ellen Cavallari, Head of Investor Relations. Please go ahead.

Ellen Cavaleri
Ellen Cavaleri
Head of Investor Realtions at PTC Therapeutics

Good afternoon, and thank you for joining us to discuss PTC Therapeutics second quarter twenty twenty five corporate update and financial results. I'm joined today by our Chief Executive Officer, Doctor. Matthew Klein our Chief Business Officer, Eric Powell and our Chief Financial Officer, Pierre Fabien. 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.

Ellen Cavaleri
Ellen Cavaleri
Head of Investor Realtions at PTC Therapeutics

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 ks 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 a reconciliation of GAAP to non GAAP are available in today's earnings release. I will now pass the call over to our CEO, Doctor. Matthew Klein.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Thank you all for joining today. I'm pleased to share results from another strong quarter highlighted by the first approvals for SUFIANCE for the treatment of children and adults with PKU. We expect SUFIANCE to be the foundational product for PTC's sustainable growth and path to profitability. In the second quarter, we again had solid revenue performance with total revenue of $179,000,000 with continued contributions from our DMD franchise, including in Europe. Following the nonrenewal of the Translarna conditional marketing authorization, we have reached agreements with about half of European countries based on the unprecedented use of Article 117 to provide paid Translarna product.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

For the remainder of 2025, we expect to be able to maintain approximately 25% of European revenue from prior to authorization nonrenewal. The highlight of the quarter was the EU approval of Suffiance in late June with a broad label inclusive of all disease subtypes and age groups. And last week, we announced FDA approval of SUFIANCE with similar broad labeling for patients aged one month and above. Based on the strong data package and the significant unmet need for PKU patients, Suffiance is positioned to become the new standard of care for children and adults living with PKU. As we have discussed, we believe the SUFIANCE revenue opportunity in The U.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

S. Exceeds $1,000,000,000 and our global customer facing teams are excited to bring the therapy to all those that could benefit. We initiated the European launch in Germany in mid July and are leveraging early access mechanisms in other European countries, while formal pricing and reimbursement discussions proceed. In The U. S, we plan to ship the first commercial drug to patients within the next two weeks and look forward to a robust early launch.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Eric will provide further details on the Sefiance launches shortly. Given the potential significant revenue opportunity for Sefiance, PTC has reached an agreement to purchase the annual global net sales payment obligation of 8% to 12% that was part of the acquisition of Scenta Pharmaceuticals in 2020. PTC will pay the participating Scenta rights holders approximately $225,000,000 upfront and additional future sales milestones for approximately 90% of our net sales payment obligation. We view this transaction as a constructive use of our cash reserves given the expected value creation based on the transaction terms. Now with the FDA approval of CFIANCE, we have two NDAs that remain under FDA review for vutiquinone and Translarna.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

For the vutiquinone NDA for Friedreich's ataxia, a late cycle meeting was held in July. At that meeting, FDA shared that the application is still under active review and confirmed they did not plan to hold an advisory committee meeting. Turning to the PPC-five eighteen for vodoplan Huntington's disease program. Following the positive Phase II PIVOT HD study results, we continue to collaborate with Novartis on next steps for vodoplan and aim to meet with FDA in the fourth quarter to discuss the Phase III trial study design and potential accelerated approval pathway. Finally, we remain in a very strong financial position, closing the quarter with approximately 1,990,000,000 in cash, allowing us to fully support all planned commercial and R and D initiatives, engage in strategic business development activities and achieve cash flow breakeven without the need for additional capital.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

I will now turn the call over to Eric to discuss our commercial performance and our SUFIANCE global launch. Eric?

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Thanks, Matt. Our global customer facing teams performed well again this quarter, achieving $118,000,000 in second quarter revenue from our marketed products, and we are excited to have initiated the global launch of Sefiance following approvals in both Europe and The U. S. We will review details of our Sefiance launch efforts shortly. We generated $96,000,000 revenue in the second quarter from our global DMD franchise.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

As Matt mentioned, despite the nonrenewal of the EU conditional license, we have continued supplying paid Translarna to several European countries, leveraging mechanisms specific to each country in accordance with Article 117. Outside of Europe, we continue to generate Translarna revenue, including in Latin America, the Commonwealth Of Independent States, The Middle East and North Africa. Our experienced U. S. Neurology team is ready to bring Translarna to nonsense mutation DMD patients following potential FDA approval.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Now turning to Emflaza. As expected, with additional generic entrants, we have seen continued market erosion. However, we continue to see meaningful revenue, and our PTC Cares team has done an outstanding job ensuring new patient starts and maintaining high levels of brand loyalty for Emflaza with the DMD community. Shifting to TEGSEDI and WAYLIVRA in Latin America, we continue to identify and treat new patients in the region and have received group purchase orders in Brazil. For AVSTAZA and KABILITY, we are pleased that new AADC patients have been treated across multiple regions, and our commercial efforts remain focused on where patients are identified, including those countries with AADC deficiency founder effects.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

We expect a steady cadence of AADC patients to be treated in The U. S, Europe, Asia Pacific and Latin America throughout 2025. Turning now to SUFIANCE for PKU. Our world class commercial team is in place to successfully launch SUFIANCE following The U. S.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

And European approvals. We are well positioned to leverage our core launch capabilities in rare disease with more than a decade of commercial experience to drive early and rapid science adoption. We initiated the first launch in Germany in mid July and engaged key PKU centers in the country. We are very pleased with the initial feedback from health care providers, and the first patients have already received commercial therapy. Our teams in Europe have identified other key markets where paid early access programs are available for Sefiance, and we'll leverage them as soon as possible.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

We are thrilled with the recent FDA approval of Sefiance, which we believe is well positioned to redefine the standard of care for PKU. In The U. S, our dedicated team is already calling on health care providers in 104 PKU centers of excellence, who account for more than eighty percent of PKU clients and treat the highest concentration of U. S. Patients, including those diagnosed at birth, children, adolescents and adults.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

The clinical data support the ability of Sefiance to address the full spectrum of the approximately seventeen thousand patients in The U. S. In terms of sequencing, our initial focus is on the patients who recently failed or are not well controlled on existing therapies and those who could be switched from existing oral therapies who are seeking greater fee reduction. We will then progress to treatment naive patients who could benefit from a new effective treatment. Our peer meetings continue to be productive, following presentations of the clinical data and the value proposition of Sefiance from our market access and medical affairs teams.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

We have actively engaged with key commercial, Medicaid and Medicare payers covering over two twenty million lives and have received positive feedback on access and coverage of Suffiance with minimal restrictions. We are equally excited about the anticipated regulatory approvals of Suffiance in Japan and Brazil before the end of the year, building on the launch momentum that has already begun in The U. S. And Europe. Initial feedback from health care providers worldwide is highly positive, and we look forward to continuing to provide updates on the global trajectory in the next several quarters.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

In addition to our team's focus on the Cefiance launch, we have also been preparing for the potential launch of etiquinone in The U. S. Our experienced teams in neurology are ready to launch the product and address the significant unmet need for both children 16 who currently have no approved therapy as well as adults with FA who may benefit from a well tolerated and effective therapy. With that, I will now turn the call over to Pierre for a financial update. Pierre?

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Thanks, Eric. I will begin by reiterating our excitement for the approval of Cefiance, a pivotal milestone both for patients and for PTC. As we discussed, Cefiance has the potential to become the standard of care for PKU and will serve as the cornerstone product driving our path to profitability. Today, we announced the purchase of Sefiant's annual global net sale payment obligation owed to Censa. This strategic transaction is accretive based on the terms we negotiated and underscores our confidence in the market opportunity.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

I'll now share the financial highlights of our 2025. Beginning with top line results. Total products, collaboration and royalty revenue for the second quarter was 179,000,000 including DMD franchise revenue of $96,000,000 Starting with the DMD franchise. Transflarma net product revenue in the quarter was $59,000,000 and Emflaza net product revenue was 36,000,000 For Emflaza, Roche achieved second quarter global revenue of approximately US559 million dollars resulting in royalty revenue of $58,000,000 for PTC. For the 2025, non GAAP R and D expense was $104,000,000 excluding $9,000,000 in noncash stock based compensation expense compared to $123,000,000 for the 2024, excluding $9,000,000 in noncash stock based compensation expense.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Non GAAP SG and A expense was $76,000,000 for the 2025, excluding $10,000,000 in noncash stock based compensation expense, compared to $60,000,000 for the 2024, excluding $10,000,000 in noncash stock based compensation expense. Cash, cash equivalents and marketable securities totaled $1,989,000,000 dollars as of 06/30/2025, compared to $1,140,000,000 dollars as of 12/31/2024. Our strong financial position provides us with the necessary resources to seamlessly execute on our strategy, successfully launch all our new commercial products globally, achieve all our anticipated milestones as well as advance our novel R and D efforts and accelerate our trajectory towards cash flow breakeven and profitability. Furthermore, this strong foundation provides us with the ability to explore business development opportunities to enhance our commercial portfolio and pipeline for long term growth. I will now turn the call over to the operator for Q and A. Operator?

Operator

Thank you. And now we're going to take our first question. And it comes from the line of Kristen Kluska from Cantor Fitzgerald. Your line is open. Please ask your question.

Kristen Kluska
Equity Research Analyst at Cantor Fitzgerald

Hi, everyone. Congrats on a great quarter and thanks for taking my questions. I have two. The first is just on Huntington's. What is going to be on your wish list related to the trial design you and your partners will talk with the FDA?

Kristen Kluska
Equity Research Analyst at Cantor Fitzgerald

And will you have any additional data to share with them at that time? And then for Translarna in Europe, under this Article 117, which I'm less familiar with, do you have to renew this every year? Should we be expecting this 25% revenues on a go forward basis? Thank you so much.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Thank you very much for the questions, Christian. Christian, on the first question, so look, I think this is exactly as we said we hope to be doing with our partner Novartis, which is once we completed the readout of PIVOT HD, engage with the FDA to discuss two things. One, the design of the efficacy trial based on some of the key learnings that we talked about from PIVOT HD as well as discuss pathways to accelerated approval, whether that's on the existing data we've shared thus far with PIVOT HD or the additional data that we could continue to collect as the open label extension is ongoing. As we talked about, we would fully expect the efficacy trial to be a large trial, as has been done previously with Huntington's disease. And as you know, there's a fairly finite universe of endpoints and certain factors that could go into discussion. So I would say our wish list is to come away with alignment for what the key elements of that efficacy trial will be, and obviously, we have confidence that that should happen, as well as clarity on the data that we'll need, whether it's the data we have at hand now showing long term CUHDRS changes, Nf L, some of the other biomarker changes to support accelerated approval, or whether there's going to be additional data that we could use once we get further into the open label extension.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

On your second question, would Article 117 is something that was referenced in the European Commission's adoption of the CHMP opinion. And actually, there's two articles. There's Article 117 and Article five, which together which reference specific things in the European Commission doctrines. And what they allowed together is individual countries to allow Translarna to still be commercially available despite the fact that the license has not been renewed. So, it's basically an umbrella or a directive that each individual country can elect to leverage or not based on individual country by country mechanisms that they have.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

And as we said, we've seen about half the countries leverage that availability. And that's, again, based on a lot of the feedback we've gotten from patients and physicians who have clearly communicated the benefits they've observed with Translarna, as well as the lack of alternative therapies. And so in countries where possible, we've been able to provide paid drug. Other countries have elected not to do it. And so we're about half now.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

We've said that we expect to be able to maintain about 25% revenue through the rest of 2025. And I think it's going to we're going to see how different countries, different contracts and things play out over time. 01/2017 doesn't need to be renewed. It will be at the discretion of individual countries about renewing. And in some countries, for example, Italy has publicized they're allowing for six months and that will be revisited.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Other countries have not given a timeline. So it's going be very variable, which is why we've said we expect the 25% for the remainder of the year. But look, this is all an incredible upside given the context of this situation when you consider that the license wasn't renewed. And again, this is really based on a lot of the feedback from the patients and the physicians about the clear perceived benefits of Translarna. And we're, one, happy to be able to still harvest revenue from Europe, but two, really happy to be able to still provide this therapy to patients who really need it.

Operator

Thank you. Thank you. Now we're going to take our next question. And the question comes from the line of Tazeen Ahmad from Bank of America Securities. Your line is open. Please ask your question.

Tazeen Ahmad
Tazeen Ahmad
MD - US Equity Research at Bank of America

Hi, guys. Good afternoon. Thanks for taking my question. For Stephanie and Matt, can you give us clarity on the metrics that we should expect to see in the early innings of the launch presumably on the 3Q call? And then can you clarify if you've already started receiving scripts?

Tazeen Ahmad
Tazeen Ahmad
MD - US Equity Research at Bank of America

And if so, do you have any kind of sense on what types of patients are receiving scripts first?

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Thank you for the question, this is Zazeen. I think it's early days, as you say, and I think we're happy with how things have gone and in particular what we've seen in the public, a lot of social media and a lot of patients talking about how happy they are. This is data they've wished for and hoped for. And just a lot of positive feedback, which, one, is incredibly gratifying and two, is really consistent with our understanding of the significant unmet need that suffice can fill. Eric, do you want to go into a little more detail on Tazeen's questions on the early on the metrics we claim to share in early dynamics?

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Yeah. Thanks for the question, Tazeen. I mean, as we said earlier, the metrics we're going to continually provide on a quarterly basis will be prescriptions, patient start forms, the number of commercial patients that are currently available on treatment. We'll also provide information regarding health care provider, as well as payer sort of prescribing dynamics and coverage dynamics. We'll also provide you some color on the rollout in the international flavor of Suffice, which countries we'll actually be bringing on board.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

There's So, a number of key metrics here, but more importantly, think you're going to see this. It's still early days, but you're going to see the metrics really centered around the number of prescriptions and number of health care providers who have prescribed.

Tazeen Ahmad
Tazeen Ahmad
MD - US Equity Research at Bank of America

Thanks. On the scripts received so far, if any?

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Yeah. And just to provide you some we're thrilled that we've already gotten prescriptions in The US. We have patient start forms that came in on the very first day, And the feedback from health care providers has been excellent so far. As I mentioned, we also have our first patients on commercial therapy in Europe already. The feedback from physicians has been very positive.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Our teams have been promoting the benefits of Sophias immediately. The characteristics that we've seen and again, it's very early days, it kind of matches up very closely to what we've seen. Physicians are looking to bring in patients who are poorly controlled or have failed. Many of them have interest in switching patients to get better feed control, and we've also seen prescriptions for naive patients.

Tazeen Ahmad
Tazeen Ahmad
MD - US Equity Research at Bank of America

Okay. Thanks, guys.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Sure.

Operator

Thank you. Now we'll go and take our next question. And this comes from the line of Brian Chen from JPMorgan. Your line is open. Please ask your question.

Brian Cheng
Brian Cheng
Senior Biotech Analyst at J.P. Morgan

Hey, guys. Thanks for taking our questions this afternoon. Two from us. It's probably still in the early days to understand how contracting and the eventual net pricing for Subsciences. But I know that you already have been in touch with a sizable portion of commercial payers out there.

Brian Cheng
Brian Cheng
Senior Biotech Analyst at J.P. Morgan

So just curious if you have some feedback that can help us to think through the level of contracting? And then I have a quick follow-up. Thank you.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Sure.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Eric, go ahead and take it away whenever you're calling.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Sure. Yes. Thanks, Brian, for the question. We've been having really good meetings with payers so far.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

As I mentioned, it's been really dozens of payers and quite a good mix between both commercial, Medicaid and Medicare. We continue to have these meetings now post launch. And I think really more than anything else, it's going as well as what we expected. We see right now that the clinical profile of Suffiance is being very well received. Payers already see that it's highly differentiated, and there's a high willingness to cover the product.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Importantly, very minimal restrictions, prior authorizations to the label, and only a few have said that we would implement step edits. So overall, restrictions. Regarding your question around contracting, we haven't gotten it's still early days, we haven't gotten into that at this point in time. We don't necessarily see the need at this point in time either to contract with payers. And as I said, we will be providing metrics around gross to net further down the line as we get the payer mix, which we anticipate at this point in time to be 65% commercial and approximately 35% to be Medicaid or Medicare.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

So at this point in time, it's a bit early, But overall, I think it's going as well as we expected it to go.

Brian Cheng
Brian Cheng
Senior Biotech Analyst at J.P. Morgan

Great. And then maybe one for PA. Is there any meaningful inventory build that we should think through? And also, how should we also think about any changes in terms of the SG and A line that we should expect for the Zafion's launch? Thanks for taking our questions.

Pierre Gravier
CFO at PTC Therapeutics

Yes. Thanks for the question, Brian. As we mentioned, we are leveraging our existing infrastructure, so there will be no additional OpEx. And then in terms of inventory, everything is ready, right, just to be clear. So, you know, all the patients have what they need.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Yeah. And I would just add I can add to a little more color to that, Pierre. In the context of inventory, we plan to ship to patients sometime around mid August. And clearly, we were working with two specialty pharmacies that will carry just in time inventory levels. So, it's not really something that we're looking at in terms of building. But what we are doing right now is anticipating that demand based on the number of start forms that we've already started to accumulate.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

As we've mentioned, there has been incredible excitement around the launch of Sefiance, the community now is really poised to begin that. So, we'll be monitoring that very closely. But again, Brian, we're not really going to be building inventory at all. We're just going to be managing accordingly with our specialty pharmacies.

Brian Cheng
Brian Cheng
Senior Biotech Analyst at J.P. Morgan

Great. Thanks, guys.

Operator

Thank you. Now we'll go and take our next question. And it comes from the line of Judah Frommeth from Morgan Stanley. Your line is open. Please ask your question.

Judah Frommer
Judah Frommer
Executive Director - Senior Equity Research Analyst at Morgan Stanley

Yes. Hi, guys. Thanks for taking the questions and congrats on the update. I guess, can you provide a little more color on the decision to allocate capital to these prior sense of shareholders? Is there anything you can share in terms of kind of hurdles for your decisions to do that on kind of a returns basis relative to maybe allocating that capital somewhere else, business development on potentially earlier stage assets or just investing further in your pipeline? Yes.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Thanks for the question, Judah. As we talked about, we have accumulated and built significant cash reserves. We closed the second quarter with almost $2,000,000,000 still in cash on the balance sheet. And what we said we're going to use that for, we're going to deploy it strategically. We're going to support our commercial programs, our R and D program pipeline programs as well as be thoughtful about business development and corporate development.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

And I would put this under their heading of a strategic deployment of our capital. Pierre, do you want to give a little bit more detail on the thinking around this and why it was a strategic deployment of capital?

Pierre Gravier
CFO at PTC Therapeutics

Yes, so we see. Look, given the revenue, total share of Defiance, our ability to achieve $1,000,000,000 plus of revenues in The U. S.

Pierre Gravier
CFO at PTC Therapeutics

Alone, This is a thoughtful use of our cash. We said that we will be disciplined. We will focus on creative value transaction, and that's exactly how we thought about it. And this is a very high return on capital. So that's how we thought about it.

Pierre Gravier
CFO at PTC Therapeutics

Furthermore, as Matt mentioned, we have a very strong financial position that will not preclude us for additional opportunity for BD or pipeline investments on R and D efforts. We have a lot of firepower remaining.

Judah Frommer
Judah Frommer
Executive Director - Senior Equity Research Analyst at Morgan Stanley

Okay, great. And then just on Huntington's, is there anything you can share on interactions you've had with Novartis since providing the update?

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Hey, Jude, I would say that the teams have worked very well together. There's a clear shared sense of urgency in getting this program forward and getting a therapy to patients that could be beneficial. There's a shared enthusiasm for the mechanism of Huntington lowering and the positive attributes of boto plan or PTC518 being an oral small molecule splicing agent. And I think what we've done over the past few weeks is really take two very aligned teams, work together and make sure that we take the next necessary important step, which is meeting with the FDA to align on the efficacy trial design, as well as understanding with the data we have at hand and what additional data we could have, what the potential pathway for accelerated approval looks like. Thanks.

Operator

Thank you. Now we're going to take our next question. And this comes from the line of Kelly Shi from Jefferies. Your line is open. Please ask your question.

Kelly Shi
Kelly Shi
SVP & Senior Research Analyst - Biotechnology at Jefferies

Congrats on the progress and thanks for taking my questions. Maybe first on PKU launch, specifically on how to timely capture those who are under care and the recently failed other therapies. How soon could they get C5s? And will there be some wait time between for logistical reasons? Also on the sales guidance for full year 2025 remain the same from Q1 from $650,000,000 to $800,000,000 Curious if this number include any revenues from TKO launch? And also I have a follow-up. Thanks.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Thank you for the questions, Kelly. I'll start and then pass to Eric and Pierre. First, we've talked about the ability to act to provide benefit to the full spectrum of PKU patients. But in terms of sequencing that there are a relatively large number of patients who are at these specialty centers, 104 specialty centers that we talked about, who are either on existing therapy, oral therapies and can certainly benefit from one that could provide greater lowering and see greater diet liberalization. Others who've recently tried and failed, and others who may be in recent contact with the centers and therapy naive.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

So, it's that group of patients that we've talked about that we see is the first and that we would be targeting in terms of sequence. And then, of course, those who may be in less contact with the center as time goes on. But there's a large number, as we shared on the call last week, somewhere 7,000 patients sticking to that first wave of sequence. Eric, do you want to talk a little bit about wait times and how quickly patients can get therapy?

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Yeah, Kelly. Thanks for the question. I think, really, that's going to depend. Again, it's very early days, and part of that is going to be based on the patient's profile. If they've already been controlled on current therapies and would like to switch to SUFIANCE, then it may take a little bit longer.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

However, most of these patients that Matt described are either poorly controlled or they failed, and some of them are looking for better feed control. If there's documentation, usually that will go much quicker through the payer. So for us, we anticipate that that first wave will already have previous documentation, and we'll be able to, if you will, address many of the prior authorizations, and if required, some of the step edits very quickly. We have a lot of experience with that over the last eight and a half years. Our teams have been dealing with that with prednisone and Emflaza.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

And the good news here is, suffice and the activity of suffice and fee can be measured very quickly. So, we can get to the point of prescription and dispense relatively quickly, particularly in that group that Matt mentioned, that initial wave of close to 7,000 patients.

Kelly Shi
Kelly Shi
SVP & Senior Research Analyst - Biotechnology at Jefferies

For the color. Maybe just quickly, I say, could you share any comments on your latest engagements with the regulatory agency and also your confidence level for the PDUFA given it's only twelve days away? Thank you.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Yes. Thanks. Sorry, I can answer that, and then I can pass it to Dier to answer your question about guidance because I don't think we got to that one. So on SA, we held a late cycle meeting a few weeks ago. It was a very constructive meeting.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

We were told by the agency that they're still actively reviewing the application. And so we're just waiting for any additional information and questions that we can address as they continue their review despite it being so close to PDUFA. Pierre, did you just want to comment on guidance and the inputs?

Pierre Gravier
CFO at PTC Therapeutics

Yes. Guidance six fifty to 800, we discussed, the bulk of it is our existing product portfolio and obviously includes new product launch as well. There are still some uncertainties as you can imagine on Emflaza, for instance, that's probably how you derive the $650,000,000 at the bottom end and the 800 will be dependent on how fast we ramp up and there's upside potential there as well.

Kelly Shi
Kelly Shi
SVP & Senior Research Analyst - Biotechnology at Jefferies

Thanks very much.

Operator

Thank you. Now we're going to take our next question. And this comes from the line of Brian Abrahams from RBC Capital Markets. Your line is open. Please ask your question.

Kevin Meli
Kevin Meli
Equity Research Associate at RBC Capital Markets

Hi, this is Kevin on for Brian. Thanks for taking our questions. I just had a couple on CFIANCE in the EU. Just maybe can you talk about what uptake is expected in other EU countries with early access that you've identified? And sort of more generally, we should think about the EU opportunity?

Kevin Meli
Kevin Meli
Equity Research Associate at RBC Capital Markets

And then I believe you mentioned no G and A impact given these launches. Does that I'm assuming that also applies to the EU as well? Thanks so much.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Kevin, thanks for the questions. I'll just tackle the second one first and let everyone talk about European dynamics. No, we don't expect any OpEx changes for Subiacenza. It's all currently covered. As Pierre said, we're leveraging our existing infrastructure.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Similarly, for vatiquinone, if approved and launched there, the OpEx is already baked in as we'll be leveraging our existing neurology commercial infrastructure in The U. S. Eric, do want talk a bit about what we're seeing or expecting in Europe beyond the Germany access program?

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Yeah. Thanks for the question, Kevin. The European opportunity will be very significant. And of course, Germany is the second largest market in the world. So for us, it's incredibly important to get off to a really good start and establish, if you will, the pricing corridor.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

We're going to be leveraging a number of key markets in Europe that have early access programs and inpatient programs. Those are typically the Southern European markets, as well as Central and Eastern European markets, and there are some in the North. We would anticipate somewhere between five to about up to 10 markets that could potentially contribute during the course of this year and through the first half of next year. And the European opportunity will be incredibly important if we can maintain and we will maintain a very narrow pricing corridor. But in addition to that, we also expect approval in Japan and Brazil, and coming on board there will also add some of the momentum that we've built from The U.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

S. And Europe. So, these are two also incredibly important markets to our growth in the future.

Operator

Excuse me. Any further questions?

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

No. Thank you.

Operator

Thank you. Now we're going to take our next question. And the question comes from the line of Ellie Merle from UBS. Your line is open. Please ask your question.

Tejas Wein
Tejas Wein
Associate Director - Biotechnology Equity Research at UBS Group

Hi. This is Tejas on for Ellie. Thanks for taking our question. I know you mentioned you had some scripts coming in. Have you guys seen any approvals yet for coverage?

Tejas Wein
Tejas Wein
Associate Director - Biotechnology Equity Research at UBS Group

I know it's ahead of any drug getting shipped, but just anything anecdotal. And then a little bit on the ex US opportunities. How does the distribution of patients work in some of these countries? Are they concentrated at major centers? Or are they a bit more spread out through these countries?

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Thanks for the questions to ask. On the first one, it's still early days to answer that question, so it's nothing more to add on that. But I just want to talk a little about distribution of patients in Europe.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Yeah. And just going back on the insurance, we're in the process. We're just in a few days. And so as you can understand, we're going through the process of insurance verification in The US. That process, price was listed, insurance verification, and then co pay assistance, that takes a few days for all that to happen.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

But as we progress, we'll be providing a little bit more color there. In terms of Matrix Centers of Excellence, in fact, I think Germany is a great example where we were able, through our compassionate use program, to target more than half of the centers in Germany that actually oversee close to 8,000 patients. And with that, our compassionate use program was actually rolled out and has been incredibly important in converting of those patients immediately. Most of these patients are seen in centers of excellence, very much like The US, where you have a patient that's diagnosed at birth. So, they're actually moved immediately into that center and followed throughout adolescence and then their adulthood.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

So we see in Europe a very high concentration in the major cities, and it's really not as diffuse. It's actually far more centralized.

Tejas Wein
Tejas Wein
Associate Director - Biotechnology Equity Research at UBS Group

I guess just with those compassionate use programs, just around the world, how many patients are on them, including in The U. S?

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

And how fast do you think you could convert those? This was a very specific early access program in Germany that we launched because for two reasons. One, with pricing with listing of the pricing and laryngotics submission in the German launch, those patients pretty quickly turn right over to commercial. And also, it was an opportunity for us to get the drug in the hands of those physicians at the concentrated centers. So we did not institute a global early access program.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

This was a specific decision made in Germany, our global capacities program. This was a specific decision made in Germany due to the dynamics of the launch listing of the price and the six months of repricing you get as soon as you issue a launch.

Operator

Thank you. Now we're going to take our next question. And the question comes from line of Geoff Meacham from Citigroup. Your line is open. Please ask your question.

Analyst

Hey, guys. Thanks. This is Jarvi on for Jeff. Two questions. Just to your earlier comment on the early scripts coming in, could you provide some color on the cadence and types of patients coming in?

Analyst

Have these patients lined up more with their scheduled visits? Or have these early patients been coming in voluntarily for a medication switch? And then second question, based on your latest conversations with FDA on vutiquinone, you know, what has been your sense on the agency's stance on a broad label for all age groups? And is there a possibility that agency could proceed demonstration of benefit on certain MFAR subgroups to be more appropriate for, you know, certain portions of the patient population? And on that topic of labeling, have those discussions be done? Thanks.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Thanks for the questions, Jeremy. On the first question, look, it's still early days. And what we can tell you is that there's no rules or patterns we see other than we know there are a lot of patients who are really interested in getting on drug really quickly. We're seeing a lot in social media, patients saying we're writing our doctors right away. We're trying to get we're going to get in as quickly as possible.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

And again, a lot of the prescription decisions and prescription writing at these centers can be MDs, could be the doctors, could be the nurse practitioners. And so, it's really kind of a mix. We've also talked about a lot of centers, particularly in The U. S. Having weightless patients.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

So, they're working off that as well. So, there's no clear rules, but exactly as we expected, you're seeing a broad swath of patients wanting to get access to drug and physicians wanting to provide the drug for all those different segments, as Eric has talked about. On vutiquinone, we would expect, based on our conversations, that the label will be inclusive of all age groups, while the Move FA study was focused in pediatric and adolescent patients for whom there's an unmet need without approved therapies now. We have data in adults in that study as well that is consistent with what we've seen in the younger patients, as well as data from our longer term extension studies that were provided in terms of confirmatory evidence, including data from an earlier study where we see in adults, both ambulatory and non ambulatory, significant effect in terms of slowing of disease progression over years. So, taken together, the data package clearly supports benefits and certainly safety in the full age spectrum and full spectrum of disease severity

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

We have not gone formally into labeling negotiations yet at this point.

Analyst

Awesome. Thanks, Matt.

Operator

Thank you. Now we're going to take our next question. And the question comes from the line of Yun Lee from Trieste. Your line is open. Please ask your question.

Joon Lee
Joon Lee
MD & Senior Biotech Analyst at Truist Securities

Hi. Thanks for the updates and for taking our questions. When you talk to the FDA for the Huntington's, my guess is that it'll be with C. DER, but CBER is also looking at AMG one hundred thirty for Huntington's and is expected to render a decision on accelerated approval path this quarter. So, the same disease indication with the decision by CBER, is that any sort of regulatory precedent that may impact SEDAR and program?

Joon Lee
Joon Lee
MD & Senior Biotech Analyst at Truist Securities

And also quickly, do you still owe royalties to Suratore? Thank you.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Thanks for the questions, Jun. On the first question, we're obviously watching with great interest the FDA interactions that CBER is having around the gene therapy. Look, we've talked a lot about, know, there's potential benefits of the gene therapy administered to one part of the brain through direct administration to lower HCT if there's benefits there.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

We think that leads to incredibly well to lowering huntingtin protein throughout the whole brain and to be able to do that in a durable way, and also allowing for dose titration and monitoring peripherally of HTT lowering. But when we start from a development standpoint, are very good read throughs. And certainly on the regulatory side, I think there's been increasing desire by the agencies to have alignment, particularly when it comes to their views on rare disease, whether it's in CBER or CDERT. Obviously, there's been a lot of changes in FDA recently. But I think that that concept and that desire to have alignment remains.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

So, with that in mind, we're clearly very interested in understanding what the pathway could look like. As we shared the PIVOT HD readout in May, we were very happy to demonstrate the statistically significant benefit out to two years relative to a well matched natural history cohort from the enroll HD database, as well as the signals of dose dependent longer than a fell out of twenty four months. So, obviously, we're going be very keen to see the FDA's position with regard to using the natural history comparator with CH2 address. Obviously, we have the additional data shown by the peripheral Huntington lowering biomarker data as well, which is clearly an important important piece of evidence that we're having a favorable effect on what matters most of the disease, toxic huntingtin protein. Then, Pierre, do you want to comment on the remaining royalties for Shibatory?

Pierre Gravier
CFO at PTC Therapeutics

Yes. We still owe low single digit royalty to Shibatory.

Joon Lee
Joon Lee
MD & Senior Biotech Analyst at Truist Securities

Thank you.

Operator

Thank you. Now we're going to take our next question, and it comes from the line of Sami Kovind from William Blair. Your line is open. Please ask your question.

Sami Corwin
Research Analyst at William Blair

Hey, thanks for taking my questions and congrats on the progress. I was curious if you could provide an update on the Translarna review in The U. S. I think the last we heard there have been some clinical site inspections. And then given your strong balance sheet and revenue projections, what are your thoughts on additional BD opportunities or early pipeline investments?

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Thanks for the question, Sami. On Translarna, that NDA remains under active review. As we mentioned, that we had had the clinical site inspections completed in the spring. We have got IRs in the early part of the summer that we were able to easily address. And so, obviously, the PDUFA date, it's hard to know exactly when the agency will reach an action, but we have had back and forth in terms of IRs in addition to the inspections as we previously talked about.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

In terms of the balance sheet and the BD, clearly, as we still remain with significant firepower and look to strategic BD. Pierre, do want just talk a little bit about how we've been thinking about the potential BD opportunities and timings?

Pierre Gravier
CFO at PTC Therapeutics

Yes. As Matt mentioned, we have a significant financial position and we're actually looking at the BD opportunities. You know, we have a global infrastructure. We know how to get drug approved globally and we know how to obviously commercialize this drug worldwide. And so these are, you know, so we're looking at assets that we could cut it in our existing infrastructure.

Pierre Gravier
CFO at PTC Therapeutics

Furthermore, we're also looking at pipeline assets that will complement our R and D portfolio. And that's how we're thinking about BD opportunities. And we demonstrated today that we will be disciplined and that we will do transactions that create value for our shareholders.

Sami Corwin
Research Analyst at William Blair

Kind of follow-up question on the BT. Is there a sweet spot in terms of the stage of clinical development that you're looking at?

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Yes, I think we're looking pretty broadly, Sami. I think a lot certainly on the commercial side, we'll see what we have in terms of all the regulatory decisions, and that will dictate whether we're busy doing multiple launches or whether we'll have capacity, commercial capacity with our existing infrastructure to bring something and to continue to develop to drive top line revenue. We have a number of things coming from our research platforms coming into the clinic. So, it's going to be a matter of looking for an opportunity that can complement what we have already very nicely. But clearly, it will be in our sweet spot of rare disease.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

It could be CNS, could be non CNS. I think it's going be something that would have to be the right opportunity that we felt could complement our existing programs and expertise.

Kristen Kluska
Equity Research Analyst at Cantor Fitzgerald

Thanks. Congrats again.

Operator

Thank you. Now we're going to take our next question. And it comes from the line of Joseph Thong from TD Cowen. Your line is open. Please ask your question.

Joseph Thome
Managing Director, Senior Biotechnology Analyst at TD Cowen

Hi. This is Peyton on for Joe. So kind of asking a little bit about the metoclitone scenarios. Saying that it is approved on the round of aduva date, how quickly could you launch therapy? And is there a particular population that you would go after?

Joseph Thome
Managing Director, Senior Biotechnology Analyst at TD Cowen

And then kind of along with that, would you then move it to try and get a registration in Europe? And then in the case that you do not get it approved, would you be open to running another trial? If you can walk us through that as well, that'd be great.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

The thing that our focus right now is on potential success. So I would say that our teams are in position and ready and the infrastructure is built and we'd be ready to launch this right away. As we talked about with there out being any approved drugs for patients 16, we clearly see that as the first place that we would go. We are proud of having a longstanding collaboration with pediatric ataxia centers in The U. S, many that treat pediatric patients and treat pediatric and adult patients.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

So, I think it would be relatively it would be a very exciting opportunity for us, and we'd be ready to go by day one.

Joseph Thome
Managing Director, Senior Biotechnology Analyst at TD Cowen

Then if there wasn't success, would you consider running another trial, or would you shelter the program?

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Yes. As I said, we're thinking about success right now. We believe this is a we believe that vutiquinone provides, as the data show, an effective therapy, not only for children who need a safe and effective therapy, but also for adults who would benefit from having a safe, well tolerated and effective treatment option. That's what we believe the data showed, which is what supported the NDA submission. And you can expect that we have a commitment to patients that we will always look to try to support.

Joseph Thome
Managing Director, Senior Biotechnology Analyst at TD Cowen

Great. Thank you so much.

Operator

Thank you. And now we're going to take our next question. And it comes from the line of Luke Herman from Baird.

Luke Herrmann
Research Analyst at Baird

Just two quick regulatory ones for me. First, a follow-up on the vatiquinone review. Has FDA given you any sort of indication around when labeling discussions could potentially get underway? And then second, on Translarna, again, has FDA given you sort of runway or timeline for when an action date could be put in place?

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Yes. So, Luke, on your first question, there was no formal timetable. As we said, late cycle meeting was just a few weeks ago, and there were still some questions about we were talking to them about at the time, and no specific timeline for next steps was given. As you know, I think things are quite busy at FDA these days. And in terms of the Translarna, there will not be a PDUFA date issued, just given the particulars of that application that it was a resubmission of an NDA that was received a CRL following a submission following RTF.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

And it's just been the legacy of this going back many, many years, it is not going to be as part of the PDUFA program. So PDUFA date will not be given.

Operator

And now we'll proceed with our next question, and it comes from the line of Paul Choi from Goldman Sachs. Your line is open. Please ask your question.

Daniel Ni
Daniel Ni
Equity Research Analyst at Goldman Sachs

Hi, this is Daniel on for Paul. Thanks for taking our question. We're curious about like what's the pricing assumption after the six month free drug program ends in Germany?

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Thank you. Thanks for the question, Daniel.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Eric, do want to just talk about our global pricing strategy?

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Yes. Thanks for the question, Daniel. Look, right now, we're working with we're working to provide a very narrow pricing corridor. We've already announced the price in The U. S.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

And in Germany. They're very close to each other. In fact, we anticipate the launch of Japan and other European markets to maintain that very narrow pricing quarter. Regarding your specific question, it's a process in Germany. The AMNOG process provides you free pricing.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

And then after that, six months later, we'll be in negotiations following medical benefit assessments. And we'll be supporting that with all of the clinical data to help differentiate for all the same reasons why patients in Germany should actually obtain suffice, particularly those who are poorly controlled, who have failed. And I think the real world data over the next six months and the support from our key opinion leaders and centers are going to help us with that benefit assessment. So stay tuned. This is a process that will take at least twelve months during the whole process for us to work.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

And I think we're very optimistic about maintaining a very close and narrow pricing corridor.

Operator

Thank you. Now we're going to take our next question. And the question comes from the line of Gina Wang from Barclays. Your line is open. Please ask your question.

Gena Wang
Gena Wang
MD - Biotech Equity Research at Barclays

Thank you for taking my questions. I have one regarding Cefiance. Just want to confirm, there is no IP protection and it will be rely on the orphan designation market exclusivity in U. S, Europe and Japan. And I missed the beginning part of the call, just apologize if already asked.

Gena Wang
Gena Wang
MD - Biotech Equity Research at Barclays

So should we start to see revenue contribution in 3Q twenty twenty five? And then second question was very quickly regarding the ventuquintal in FAE. I know you mentioned that late cycle review was discussed a few weeks ago. I'm just wondering if you can provide a little bit more color what was discussed during that late cycle review and then any label discussion come up during the discussion or afterwards?

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Thank you, Gina. Let me correct the first question. We guided IP 02/1939. We have a polymorph patent that goes out to 2038 that we believe will provide it's a composition matter of a polymorph that we believe will support protection from 2038 and then conservatively estimating at least a year of patent term extension beyond that to 02/1939. And of course, we're continuing to work to expand as we always do the IP portfolio to see if we can take protection even further beyond 02/1939.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

And that applies not only to The U. S, the 2039 guidance, but in key markets globally. We have we will expect there to be revenue in Q3 from SAVIANCE as we have already begun delivering commercial product in Germany and we've gotten patient start forms in The U. S. And expect to be able to ship SUFIANCE to patients in the next couple of weeks.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

We've said that there is in the wide guidance that there is some new product revenue baked in there And that upper end of the guidance that stays 8 100 is allowing for the potential of upside from new products. In terms of the Tickranil and the late cycle meeting was a relatively brief meeting where we discussed the mainly focus on discussion of the evidence, where they are with their review, and their assurances that they're actually still working through the review. Not surprisingly, one of the main questions in the review is the fact that upright stability wasn't prespecified as the primary endpoint. Would that support persuasiveness of evidence of effect, which along with the confirmatory evidence could support approval? Of course, acknowledged and well aware of their recent guidance that others have received that upright stability can itself be an efficacy endpoint.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

It will be used as a primary endpoint, for example, in the Sclarus pediatric trial, which I think supports the FDA's evolving understanding that upright stability is the most relevant measurement of efficacy in ambulatory pediatric and adolescent patients. As we said, we've not had any labeling discussions beyond anything that was discussed in the late cycle meeting at this time. Thank

Operator

you. Now we're going to take our next question. And it comes from the line of Joseph Schwartz from Leerink Partners. Your line is open. Please ask your question.

Jenny Gonzalez-Armenta
Equity Research Associate at Leerink Partners

Hey, guys. This is Jenny on for Joe. Thank you for taking our questions. I just have a few follow ups on vatiquinone. First, has the FDA given any indication that the review has been delayed and or may be delayed due to staffing issues?

Jenny Gonzalez-Armenta
Equity Research Associate at Leerink Partners

We know it's kind of crazy over there. And then if approved, should we be looking at the Skyclaris launch as a template? And if not, how should we be thinking about that? And do you see any potential for combinations of Skyclaris and the Ticlinone in FA patients? Thanks.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Yes. Thanks for the question, Jenny. There was no discussion of not meeting timelines. I'm not sure there would be one, even if there was a chance that could happen. Think we all understand that there's been a lot of changes over at FDA and a lot of work.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

And certainly, we've seen examples as recently as this week of programs in the neurology division maybe not getting meeting certain timelines. But look, we look forward to continuing to work collaboratively with the division and getting to what we hope will be a positive outcome on time, and if not on time, when the time can be. As we talked about, we see the launch dynamics being quite different for a number of reasons. I can let Eric go into that. Want to talk a bit about it?

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Yeah, I think we have an experienced team already that have been calling for the last eight and a half years in pediatric neurology. So, we've already profiled all the key centers specific to FA. And we know the prescribers. We know through claims data where they are and the high unmet needs, plus the clinical differentiation. So we're already very well poised to launch and begin the process like we have with Suffiance to get patient start forms on day one.

Eric Pauwels
Eric Pauwels
Chief Business Officer at PTC Therapeutics

Our teams not only are experienced in this, but we're looking at a number of different things, which include not only the pediatric, but a number of those patients who are adults who have failed on Skyclaris, And some of them were poorly controlled, and there's still quite a bit of naive patients. So, all in all, I think the opportunity is very significant, and our team is experienced and ready to go on day one.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

And then to your other just to add to that also, you know, we would see we wouldn't expect there to be any monitoring. And obviously, the pediatric patient has patients have a much different dynamic than adults do. So, I think we would see the launch dynamics quite different for that reason. In terms of combination therapy, look, I think that's something that people talk about. I think those close to the FA community have always believed rightfully so that phrygic ataxia is going to be optimally managed with a cocktail of therapies like any other complex disorder.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

So, we certainly imagine that they'd be interested in looking at combining both reticulitis scleros.

Operator

Excuse me, Jenny. Any further questions?

Jenny Gonzalez-Armenta
Equity Research Associate at Leerink Partners

Nope. That's it. Thank you.

Operator

Thank you so much. Dear speakers, there are no further questions. I would now like to hand the conference over to your speaker, Chief Executive Officer, Doctor. Matthew Klein. Please go ahead.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

Thank you all again for joining the call today. We're excited to have had another strong quarter. We're still seeing continued contributions, meaningful contributions from our DMD franchise, which is great because we now can have that in hand as we now embark on the future, which is the launch of Suppliance. I'm incredibly excited how things are going in the early days and all indicators we're seeing from everywhere in the world. That this will meet our expectations of being our foundational product for building PTC going forward.

Matthew Klein
Matthew Klein
CEO at PTC Therapeutics

So thank you all again, and have a great evening.

Operator

This concludes today's conference call. Thank you for participating. You may now all disconnect. Have a nice day.

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