PTC Therapeutics, Inc.

Ladies and gentlemen, thank you for standing by. Welcome to PTC Therapeutics Second Quarter 2025 Earnings Conference Call. All participants are in listen only mode. After the presentation, there will be the question and answer session. Today's call is being recorded. I would now like to turn the call over to Ellen Cavalleri, Head of Invest Relations. Please go ahead.

Good afternoon, and thank you for joining us to discuss PTC Therapeutics Second Quarter 2025 Corporate Update and Financial Results. I'm 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 10Q and annual report on Form 10K 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 the 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.

Thank you all for joining today. I'm pleased to share results from another strong quarter highlighted by the first approvals for suffice for the treatment of children and adults with PKU. We expect suffice 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 million, with continued contributions from our DMV franchise, including in Europe. Following the non-renewal 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 products. For the remainder of 2025, we expect to be able to maintain approximately 25% of European revenue from prior to authorization non-renewal. The highlight of the quarter was the EU approval of suffice in late June with a broad label inclusive of all disease subtypes and age groups. And last week, we announced FDA approval of suffice with similar broad labeling for patients age one month and above. Based on the strong suffice data package and the significant unmet need for PKU patients, suffice is positioned to become the new standard of care for children and adults living with PKU. As we have discussed, we believe the suffice revenue opportunity in the US exceeds one billion, 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 mid-July and are leveraging early access mechanisms in other European countries while formal pricing and reimbursement discussions proceed. In the US, we plan to ship the first commercial drug to patients within the next two weeks and look forward to a robust early launch. Eric will provide further details on the suffice launches shortly. Given the potential significant revenue opportunity for suffice, 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 Sensa Pharmaceuticals in 2020. PTC will pay the participating Sensa rights holders approximately 225 million 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 suffice, we have two NDAs that remain under FDA review. For Veticranone and Tranwarna. For the Veticranone NDA for Phrygitux Taxia, 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 PTC 518 for Vodoplam Huntington's disease program, following the positive phase two pivot HD study results, we continue to collaborate with Novartis to develop our next steps for Vodoplam and aim to meet with FDA in the fourth quarter to discuss the phase three trial study design and potential accelerated approval pathway. Finally, we remain in a very strong financial position, closing the quarter with approximately 1.99 billion in cash, allowing us to fully support all planned commercial and R&D initiatives, engage in strategic business development activities and achieve cashflow breakeven without the need for additional capital. I will now turn the call over to Eric to discuss our commercial performance and our Safiance Global Launch. Eric?

Thanks, Matt. Our global customer-facing teams performed well again this quarter, achieving 118 million in second quarter revenue from our marketed products. And we are excited to have initiated the global launch of Safiance following approvals in both Europe and the US. We will review details of our Safiance launch efforts shortly. We generated 96 million revenue in the second quarter from our global DMV franchise. As Matt mentioned, despite the non-renewal of the EU conditional license, we have continued supplying paid trends larnet to several European countries, leveraging mechanisms specific to each country in accordance with article 117. Outside of Europe, we continue to generate trans larnet revenue, including in Latin America, the Commonwealth of Independent States, the Middle East and North Africa. Our experienced US neurology team is ready to bring trans larnet to nonsense mutation DMV patients following potential FDA approval. Now turning to implausible, as expected with additional generic entrance, 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 implausible with the DMV community. Shifting to Teg Ceti and Wey Libre in Latin America, we continue to identify and treat new patients in the region and have received group purchase orders in Brazil. For Obstaza and Kibility, 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. We expect a steady cadence of AADC patients to be treated in the US, Europe, Asia Pacific, and Latin America throughout 2025. Turning now to suffiance for PKU. Our world-class commercial team is in place to successfully launch the fines following the US 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 suffiance 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 healthcare 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 suffiance and will leverage them as soon as possible. We are thrilled with the recent FDA approval of suffiance, which we believe is well positioned to redefine the standard of care for PKU. In the US, our dedicated team is already calling on healthcare providers in 104 PKU centers of excellence who account for more than 80% of PKU clients and treat the highest concentration of US patients, including those diagnosed at birth, children, adolescents, and adults. The clinical data support the ability of suffiance to address the full spectrum of the approximately 17,000 patients in the US. 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 suffiance from our market access and medical affairs teams. We have actively engaged with key commercial, Medicaid, and Medicare payers covering over 220 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 US and Europe. Initial feedback from healthcare providers worldwide is highly positive and we look forward to continuing to provide updates on the suffiance global trajectory in the next several quarters. In addition to our team's focus on the suffiance launch, we have also been preparing for the potential launch of the Tiquinone in the US. Our experienced teams in neurology are ready to launch the product and address the significant unmet need for both children under 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?

That is Eric. I will begin by reiterating our excitement for the approval of the science, the pivotal milestone both for patients and for PTC. As we discussed, suffiance is 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 suffiance annual global net cell payment obligation owed to SAMHSA. This strategic transaction is actually based on the terms we negotiated and underscores our confidence in the market opportunity. I'll now share the financial highlights of our second quarter of 2025. Beginning with top line results, total product collaboration and royalty revenue for the second quarter was 179 million, including DMD franchise revenue of 96 million. Starting with the DMD franchise, Tramflana net product revenue in the quarter was 59 million and Mflaza net product revenue was 36 million. For every ZD, Roche achieved second quarter global revenue of approximately 559 million US dollars, resulting in royalty revenue of 58 million for PTC. For the second quarter of 2025, non-GAAP R&D expense was 104 million, excluding 9 million in non-cash stock based composition expense, compared to 123 million for the second quarter of 2024, excluding 9 million in non-cash stock based composition expense. Non-GAAP S&E expense was 76 million for the second quarter of 2025, excluding 10 million in non-cash stock based composition expense, compared to 60 million for the second quarter of 2024, excluding 10 million in non-cash stock based composition expense. Cash, cash equivalent for marketable securities total, 1,989 million as of June 30, 2025, compared to 1,140 million as of December 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&D efforts and accelerate our trajectory towards cashflow 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&A. Operator.

Thank you, dear participants. As a reminder, if you wish to ask a question, please press star, one, one on your telephone keypad and wait for your name to be announced. To withdraw a question, please press star, one, one again. Please, then, I will compile the Q&A Q-disk. We'll take a few moments. And now we're going to take our first question. And it comes to the line of Kristen Kluska from Cantor Fidgerald. Your line is open. Please ask your question.

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 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.

Thank you very much for the questions, Kristen. Kristen, 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 complete the readout of PivotHD, 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 PivotHD, as well as discuss pathways to accelerated approval, whether that's on the existing data we've shared thus far with PivotHD 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. 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 at FL, some of the other biomarker changes to support accelerated approval, or whether there are gonna be additional data that we could use once we get further into the open label extension. On your second question, 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 5, which together, which reference specific things in the European Commission doctrines. And what they allowed together is individual countries to allow TransVarna to still be commercially available, despite the fact that the license has not been renewed. So it's basically a umbrella or a directive that each individual country can elect to leverage or not based on individual -by-country mechanisms that they have. And as we said, we seem that half the countries leverage that availability. And that's again, based on a lot of the feedback that they've gotten from patients and physicians who have clearly communicated the benefits they've observed with TransVarna, 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. We've said that we expect to be able to maintain about 25% revenue through the rest of 2025. And I think we're gonna see how different countries, different contracts and things play out over time. 117 doesn't need to be renewed. It'll 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 then it'll be revisited. Other countries have not given it a timeline. So it's gonna 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 positions about the clear perceived benefits of trans-Larder. 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.

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

Hi guys, good afternoon. Thanks for taking my question. For Stephian, 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 three Q call? And then can you clarify if you've already started receiving scripts? And if so, do you have any kind of sense on what types of patients are receiving scripts first? Thanks.

Thank you for the question, Tazin. 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 the day that 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 wanna go into a little more detail on Tazin's two questions on the early, the metrics we plan to share in early dynamic?

Yeah, thanks for the question, Tazin. I mean, as we said earlier, that the metrics we're gonna 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 healthcare provider as well as payer sort of prescribing dynamics and coverage dynamics. We'll also provide you some color on the rollout and the international flavor of suffice, which countries we'll actually be bringing on board. So there's a number of key metrics here, but more importantly, I think you're gonna see this, it's still early days, but you're gonna see the metrics really centered around the number of prescriptions and number of healthcare providers who have prescribed.

Thanks, and on the script with you so far, if any.

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 healthcare 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. Our teams have been promoting the benefits of suffice 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 interests in switching patients to get better feet control, and we've also seen prescriptions for naive patients.

Okay, thanks guys.

Thank you. Now we're going to take our next question. And this comes to the line of Brian Chen from JP Morgan. Your line is open, please ask your question.

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 sub-science is. But I know that you already have been in touch with a sizable portion of commercial payers out there. So just curious if you have some feedback that can help us to think through the level of contracting and then have a quick follow-up, thank you.

Yeah, sure. Thank you for the question, Brian. Yeah, go ahead. Thanks for the question. Eric, go

ahead and take it away. Whenever you're calling, it comes to mind. Yeah, thanks Brian for the question. We've been having really good meetings with payers so far. 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 sub-science is being very well received. Payers already see that it's highly differentiated and there's a high willingness to cover the product. Importantly, very minimal restrictions, prior authorizations to the label. And only a few have said that we implement step edits. So overall minimal 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. 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.

Great. And then maybe one for PA. Is there any meaningful inventory bill that we should think through? And also, how should we also think about any changes in terms of the SG&A line that we should expect for the Defiance launch? And thanks for taking our questions.

Thanks for the question, Brian. As we mentioned, we are leveraging our existing infrastructure. So there will be no additional OPEC.

And then in terms of inventory,

everything is ready, right, just to be clear. So, you know, all the patients have what they need.

Yeah, and I'll just add, I can add 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. As we've mentioned, there has been incredible excitement around the launch of Defiance. And the community now is really poised to begin that. So we'll be monitoring that very closely. But again, Brian, we're not really in the gonna be building inventory at all. We're just gonna be managing accordingly with our specialty pharmacies. Great, thanks, guys.

Thank you. Now we're gonna take our next question. And it comes from, oh, Judah from Mogan's stand. Yolana is open, please ask your question.

Yeah, 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? Thank you.

And thanks for the question, Judah. As we talked about, we have accumulated and built significant cash reserves. We closed the second quarter with almost two billion still in cash on the balance sheet. And what we said, we're gonna use that for. We're gonna deploy it strategically. We're gonna support our commercial programs, our R&D pipeline programs, as well as be thoughtful about business development and corporate development. And I would put this under the heading of a strategic deployment of our capital. Pierre, do you wanna give a little bit more detail on the thinking around this and why it was a strategic deployment of capital?

Yeah, absolutely. Given the revenue potential of the funds, and our ability to achieve a billion dollar plus of revenues in the US 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. Furthermore, as Matt mentioned, we have a very strong financial position that will not preclude us for additional opportunity for BDE or pipeline investments on our R&D efforts. We have a lot of firepower remaining.

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?

Andrew, 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 Vodoplam 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 improvement looks like.

Thanks.

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

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 recently failed other therapies. How soon could they get a safe device? And will there be some wait time between for logistic reasons? Also on the sales guidance for full year 25 remain the same from Q1 from 650 to $800 million. Curious if this number include any revenues from PKU launch and also have a follow up. Thanks.

Thank you for the questions, Kelly. I'll start and then pass to Eric and Pierre. So first, we've talked about the ability 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 feed greater diet liberalization. Others who've recently tried and failed and others who may be in recent contact with the centers and therapy naive. So it's that group of patients that we've talked about that we see as 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 centers time was on, but there's a large number as we shared on the call last week, somewhere around 7,000 patients fit into that first wave of the sequence. Eric, do you wanna talk a little bit about wait times and how quickly patients can get therapy?

Yeah, Kelly, thanks for the question. I think really that's gonna 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 suffice, then it may take a little bit longer. However, most of these patients that Matt described are either poorly controlled or they've failed. And some of them are looking for better fee control. If there's documentation, usually that will go much quicker through the pair. 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, we have been dealing with that with prednisone and influenza. 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.

Thanks for the color. Thanks 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 producer given it's only 12 days away? Thank you.

Yeah, thanks Kelly. I can answer that and then I can pass it to Deirdre to answer your question about guidance because I don't think we got to that one. So on FA, we held the late cycle meeting a few weeks ago. It was a very constructive meeting. 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 the DUPFA. Pierre, did you just want to comment on guidance and the inputs?

Yeah, guidance, 662-800. As 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 MFLAZA for instance, that's probably how you derive the 650 at the bottom end and the 800 will be dependent on how fast we ramp up and has upside potential there as well.

Thanks very much. Thank you. Now we're going to take our next question and it comes to the line of Brian Abrahams from RBC Capital Markets. Your line is open, please ask your question.

Hi, this is Kevin on for Brian. Thanks for taking our questions. I just had a couple on suffiance 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 more generally how we should think about the EU opportunity? And then I believe you mentioned no GNA impact given these launches. I'm assuming that also applies to the EU as well. Thanks so much.

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 suffiance. It's all currently covered. As Pierre said, we're leveraging our existing infrastructure. Similarly for the Ticuanone, if approved and launched there, the OPEX is already baked in as we'll be leveraging our existing neurology commercial infrastructure in the US. Eric, do you want to talk about what we're seeing or expecting in Europe beyond the Germany early access product?

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. We're gonna be leveraging a number of key markets in Europe that have early access programs and then patient 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, we'll also add some of the momentum that we've built from the US and Europe. So these are two also incredibly important markets to our

growth in the future.

Excuse me, any further questions? No, thank you. Thank you.

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

Hi, this is Tejasan for Ellie. Thanks for taking your question. I know you mentioned you had some scripts coming in. Have you guys seen any approvals yet for coverage? 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? Thanks.

Thanks for the questions Tejasan. 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.

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 or in the US. That process, price was listed, insurance verification, and then copay assistance. That takes a few days for all that to happen. But as we progress, we'll be providing a little bit more color there. In terms of major 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 some 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. 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.

I guess just with those compassionate use programs, just around the world, how many patients are on them, including in the US, and how fast do you think you can convert those?

Yeah, this was a very specific early access program in Germany that we launched for two reasons. One, with the pricing, with listing of the pricing of the lower tax and issuing 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. This was a specific decision made in Germany, or a global compassionate use 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 free pricing you get as soon as you initiate launch.

Thank you.

Now

we're going to take

our next question. And the question comes from Jeff Meitem from CT Group. Your line is open. Please ask your question.

Hey guys, thanks. This is Jarway 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? 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 the ticlonone, what has been your sense on the agency's stance on a broad label for all age groups and is there a possibility the age group agency could proceed demonstration and benefit on certain MFAR subgroups to be more appropriate for certain portions of the patient population? And on that topic of labeling, have those discussions begun. Thanks.

Thanks for the questions, Jarway. 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 yet other than we know that a lot of patients who are really interested in getting on drugs really quickly, we're seeing a lot in social media of patients saying, we're writing our doctors right away. We're gonna get in as quickly as possible. 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 US having weightless patients. So there can be 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 drugs and physicians wanting to provide a drug for all those different segments as Eric has talked about. On particular known, we would expect based on our conversations that the labeled 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 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 a full age spectrum and full spectrum of disease severity for FDA. We have not gone formally into labeling negotiations yet at this point. Thanks,

Matt.

Thank you. Now we're going to take our next question. And the question comes to the line of Yun Li from TWIST. Your line is open, please ask your question.

Hi, thanks for the update, Sam, for taking our questions. When you talk to the FDA for the Huntington's, my guess is that it'll be with C. dir, but CBER is also looking at AMC 130 for Huntington's and is expected to render a decision on accelerated approval path this quarter. So given the same disease indication with a decision by CBER or set any sort of regulatory precedent that may impact C. dir and your program. And also quickly, do you still owe royalties to C. uratory? Thank you.

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, 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. We think that reads through incredibly well to lowering Huntington protein throughout the whole brain and to be able to do that in a durable way and also allowing for no citration and monitoring peripherally of HCT lowering. So we've always thought from a development standpoint, there 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 CDER. Obviously there've been a lot of changes in FDA recently, but I think that that concept and that desire to have alignment remains. So with that in mind, we're clearly very interested in understanding what the pathway could look like as we shared at the PivotHD 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 benefit out to 24 months. So obviously we're gonna 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 benefit being able to find the peripheral Huntington lowering biomarker data as well, which is clearly an important piece of evidence that we're having a favorable effect on what matters most to the disease that toxic Huntington protein. And then up here, do you wanna comment on remaining royalties for Chiratory?

Yes, we still owe low single

digit royalty to Chiratory. Thank you.

Thank you.

Now we're going to take our next question. And it comes to the line of Sammy Corwin from William Blair, your line is open. Please ask your question.

Hey, thanks for taking my questions and congrats on the progress. I was curious if you could provide an update on the TransLARN review in the US. I think the last we heard, there had 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? Thank you.

Thanks for the question, Sammy. On TransLARN, that MBA 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 without the BDUFA data, 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. In terms of the balance sheet and the BD, clearly as we still remain with significant file power, but do you think we need to hear, just talk a little bit about how we've been thinking about the potential BD opportunities and findings.

Yes, as Matt mentioned, we have a significant financial position and we're actually looking at BD opportunities. 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, so we're looking at assets that we could cast it in our existing infrastructure. Furthermore, we're also looking at pipeline assets that will complement our R&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.

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

Yeah, I think we're looking pretty broadly, Sammy. I think, you know, a lot, you know, certainly on the commercial side, we'll see what we have in terms of all the regulatory decisions and that'll dictate whether we're busy doing multiple launches or whether we have, you know, a certain capacity, a commercial capacity with our existing infrastructure to bring something in 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 gonna be a matter of looking for an opportunity that can be complement what we have already very nicely, but clearly it'll be in our sweet spot of rare disease, could be CNS, could be non-CNS. I think it's gonna be something that would have to be the right opportunity that we felt could complement our existing programs. And expertise.

Thank you, Kira.

Thank you. Now we're going to take our next question. And it comes to land of Joseph Thome from TD Cowans. Your line is open, please ask your question.

Hi, this is Peyton for Joe. So kind of asking a little bit about the bad tickle tone scenarios, saying that it is approved around the Biduvidate, how quickly could you launch therapy? And is there a particular population that you would go after? 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 could walk us through that as well, that'd be great.

So Peyton, 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 their out being any approved drugs for patients under 16, we clearly see that as the first place that we would go. We are proud of having a long standing collaboration with the Pre-Gytaxia centers in the US, many that treat pediatric patients and treat pediatric and adult patients. So I think it would be relatively, it would be a very exciting opportunity for us and we'd be ready to go day one.

And then if it wasn't success, would you consider running another trial or would you shelter the program?

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

Thank you so much.

Thank you. And now we're going to take our next question. And it comes to line of Luke Herman from BIT. Your line is open, please ask your question.

Hi guys, thanks for taking the question. Just two quick regulatory ones for me. First, a follow up on the Vitoquinone 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?

Yeah, so Luke, on your first question, there was no formal timetable, as we said, the late cycle meeting was just a few weeks ago, and there's still some questions about, that we were talking to him 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, and they will not be a PEDUFA data 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 an RTF, and just the legacy of this going back many, many years, it is not gonna be as part of the PEDUFA program, so the PEDUFA date will not be given.

Okay, thank you so much.

Thank you. Now we'll proceed with our next question, and it comes to the line of Paul Choi from Goldman Sachs. Your line is open, please ask your question.

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

Thanks for the question, Daniel. Eric, do you wanna just talk about our global pricing strategies?

Yeah, 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 US and in Germany. They're very close to each other. In fact, we anticipate with the launch of Japan and other European markets to maintain that very narrow pricing corridor. Regarding your specific question, it's a process in Germany. The MNOG process provides you free pricing, and then after that, six months later, we'll be in negotiations following a medical benefit assessment, 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 gonna help us with that benefit assessment. So stay tuned. This is a process that will take at least 12 months during the whole process for us to work, and I think we're very optimistic about maintaining a very close and narrow pricing

corridor.

Thank you. Now we're going

to take our next question, and the question comes from Gina Wang from Buckley. Your line is open. Please ask your question.

Thank you for taking my question. I have a one regarding the suffience. Just want to confirm that there is no IP protection, and it will be rely on the often-designation market exclusivity in US, Europe, and Japan. And I missed the beginning part of the call. Just apologize if already asked. So should we start to see revenue contribution in 3Q25? And then second question is very quickly regarding the Antiquino in FAA. I know you mentioned that late cycle review was discussed a few weeks ago, and 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?

Thank you, Gina. Let me

correct the first question. We have guided IP to 2039. We have a polymorph patent that goes out to 2038 that we believe will provide a composition of matter or polymorph that we believe can support protection from 2038, and then conservatively estimating at least a year of patent term extension beyond that to 2039. 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 2039. And that applies not only to the US, the 2039 guidance, but to the key markets globally. We will expect there to be revenue in Q3 from Suffiance as we have already begun delivering commercial product in Germany, and we've gotten patient start forms in US and expect to be able to ship Suffiance to patients in the next couple of weeks. We've said that there's in the wide guidance that there is some new product revenue based in there, and that upper end of the guidance that Stacey's 100 is allowing for the potential of upside for new products. In terms of the ticker known the late cycle meeting was a relatively brief meeting where we discussed, we mainly focused on discussion of the evidence where they are in their view 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 pre-specified as a primary endpoint with that support persuasiveness of evidence of effect, which along with the confirmatory evidence could support approval. Of course, they acknowledged and were all aware of their recent guidance that others have received that upright stability can itself be an efficacy endpoint and it will be used as a primary endpoint. For example, in the Cochlearis 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 you. Now we're going to take over next question. And it comes to the line of Joseph Schwartz from Laring Partners. Your line is open. Please ask your question.

Hey guys, this is Jenny on for Joe. Thank you for taking our questions. I just have a few follow-ups on Veticuonone. First, has the FDA given any indication that the review has been delayed and or may be delayed due to staffing issues? We know it's kind of crazy over there. And then if approved, should we be looking at the SkyClearis launch as a template? And if not, how should we be thinking about that? And do you see any potential for combinations of SkyClearis and Veticuonone in FA patients? Thanks.

Yeah, thanks for the questions, 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. I think we all understand that there's been a lot of changes over at FDA and a lot of work. And certainly we've seen examples as recently as this week of programs in the neurology division, maybe not getting 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. Yet want to talk a little bit about.

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-end met needs, plus the clinical differentiation. So we're already very well poised to launch and begin the process like we have with Sufiance to get patients start forms on day one. Our teams not only are experienced in this, but we're looking at a number of different things which includes not only the pediatric, but a number of those patients who are adults who have failed on SkyClarus. And some of them were poorly controlled and they're 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.

And then to your other, just to add to that also, we wouldn't expect there to be any monitoring and obviously the pediatric 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 Phages of Taxis is gonna be optically managed with a cocktail of therapies like any other complex disorder. So we certainly imagine that they'd be interested in looking at combining both reticulones

like Clarus.

Excuse me, Jenny, any further questions?

I know that's it, thank you.

Thank you so much. Dear speakers, I don't know if there are any further questions. I would now like to hand the conference over to your speaker. Chief Executive Officer, Dr. Matthew Klein. Please go ahead.

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 DMV franchise, which is great because we now can have that in hand as we now embark on the future, which is the launch of Suppliants. 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. So thank you all again and have a great evening.

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