Q2 2025 PTC Therapeutics Inc Earnings Call
Ladies and gentlemen, thank you for standing by. Welcome to PTC Therapeutics second quarter 2025 earnings conference call or participants are in listen-only mode. After the presentation that will be the question and answer session. Today's call is being recorded.
I would now like to turn the call over to Alan Cavallari, Head of Investor 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 of Pierre. Gabier today's call will include forward-looking statements based on our current expectations. These statements are subject to certain risks and uncertainties in actual results May differ materially. Please review the slide posted on our investor relations website in conjunction with the calls, which contains information about our board listening 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 and non-gaap financial measures and a Reconciliation of gaap to non-gaap are available in. Today's earnings release Alma, 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 safian for the treatment of children and adults with PKU, we expect Defiance 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 transactional marketing authorization, we have reached agreements with about half of European countries. Based on the unprecedented use of article 1117 to provide paid trans longing a product
For the remainder of 2025, we expect to be able to maintain approximately 25% of European revenue from prior to authorization non-renewal.
Based on the strong Supply and data package and the significant unmet need for PKU patients. So bias is positioned to become the new standard of care for children, and adults living with PKU.
As we have discussed, we believe the safian revenue opportunity. In the US exceeds 1 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 and mid July and are leveraging Early Access mechanisms in other European countries, while formal pricing and reimbursement, discussions for seed.
In the US, we plan to ship the first commercial drug to patients within the next 2 weeks, and look forward to a robust early launch.
Eric will provide further details on the supply and launches shortly.
Given the potential significant revenue opportunity for Safian, PTC has reached an agreement to purchase the annual global net sales payment obligation on 8% to 12%. That was part of the acquisition of Sense Pharmaceuticals in 2020.
PTC will pay the participating rights holders approximately $225 million upfront, with additional future sales milestones for approximately 90% of our net sales payment obligations.
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 safian, we have 2 ndas that remain under FDA review for a particular own and Trend Lana.
For the particular, Don NDA for fic's taxia, a late cycle meeting was held in July at that meeting FDA, shared that the application is still under active review and confirm, they did not plan to hold an advisory committee meeting.
Turning to the PTC. 518 for Voda plan, Huntington's disease program, following the positive Phase 2, pivot HD study results. We continue to collaborate with novardis on next steps for voter plan and aim to meet with FDA in the fourth quarter to discuss the phase 3 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 cash flow break even without the need for additional capital.
I will now turn the call over to Eric to discuss our commercial performance and our supply and 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 safian following approvals in both Europe and the US.
We will review details of our safian launched efforts shortly.
We generated 96 million Revenue in the second quarter from our Global DMD franchise. As Matt mentioned, despite the non-renewal of the EU conditional license. We have continued supplying paid Trends Lara to several European countries, leveraging mechanisms specific to each country in accordance with article 1117.
outside of Europe, we continue to generate transa 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 Lorna to nonsense, mutation DMD patients, following potential, FDA approval.
Now, turning to imp Plaza.
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 foreign Plaza with the DMD community.
Shifting to Tech City and way Libra and Latin America, we continue to identify and treat new patients in the region and have received group, purchase orders in Brazil.
For aasa and capability.
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.
Europe, Asia, Pacific and Latin America throughout 2025.
Turning now to safian for PKU.
Our world-class commercial team is in place to successfully launched the finance.
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 suffices adoption.
We initiated the first launched 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 safian and will leverage them as soon as possible.
We are thrilled with the recent FDA approval of sein 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, adolescence and adults.
The clinical data support the ability of safian 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 fear reduction.
We will then progress to treatment naive patients.
Who could benefit from a new effective treatment?
Our prayer meetings continue to be productive following presentations of the clinical data and the value proposition of Safian 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 safian with minimal restrictions.
We are equally excited about the anticipated regulatory approvals of safian 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 surveillance, Global trajectory in the next several quarters.
in addition to our team's focus on the safest launch, we have also been preparing for the potential launch of aquinus
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 safian.
People thought my stone both for patients and for PTC.
As we discussed the science of the potential to become the standard of care for PKU, and will serve as the Cornerstone product driving a path to profitability.
Today, we announced the purchase of the finance annual global net sale payment, obligation or to Senza.
The Strategic transaction is a creative based on the terms. We negotiated and underscores are confidence in the market opportunity.
I'll now share the financial highlights of our second quarter of 2025,
Beginning with start line results, total products collaboration and royalty revenue for the second quarter was 179 million including the MD franchise revenue of 96 million.
Starting with the DMD franchise.
Trump SLA. Net product review in a quarter was 59 million and in Plaza, net product Revenue was 36 million.
For every ISD.
Row 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 compensation expense.
Compared to 123 million for the second quarter of 2024.
Excluding 9 million in non-cash stock based composition expense.
Non-GAAP as DNA, the expense was $76 million for the second quarter of 2025, excluding $10 million in non-cash stock-based compensation expense.
compared to 60 million for the second quarter of 2024.
Excluding $10 million in non-cash stock-based compensation expenses.
Cash, cash equivalents, and marketable securities totaled $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 to successfully launch, all our new commercial product globally, achieve all our anticipated Milestones, as well as advanced our novel R&D efforts and accelerate, our trajectory towards cash flow, break even and profitability.
Furthermore, the 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 1 1 on your telephone keypad and wait for your name. To be announced to withdraw a question. Please press star, 1 1 again, please tell our compiler, the Q&A queue. This will take a few moments.
And now we're going to take our first question.
And it comes to line of Christian cusca from Canto, feral to your line is open. Please ask your question.
Hi everyone. Congrats on a great quarter and thanks for taking my questions. I have 2. 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 FTA. And will you have any additional data to share with them at that time? And then for Trans Learner, in Europe under this article 1117, 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. Christian, uh, Christian on the first question. So look, I think this is exactly as we had uh said we hope to be doing with our partner of artists which is once we complete the completed, uh, the readout of pivot HD engage with the FDA to discuss 2 things 1, the design of the efficacy trial uh, 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, we would fully expect to advocacy trials, to be a, you know, a large trial. Um, as has been done previously with Huntington's disease. And as you know, there's a fairly finite uh, Universe of endpoints in certain factors that could go into discussion. So I would say, I wish list is to Come Away with alignment for what that the key elements of that efficacy trial will be and it will
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, uh, showing long term C hdrs changes at FL, some of the other biomarker changes, uh, to support accelerated approval or whether they're going to be additional data that we could use, once we get further into the openly, look extension.
Uh, on your second question, uh, I would, uh, r117 is something that was in referenced in the European commission's. Adoption of the chmp opinion and it actually there's 2 articles, there's article 117 and Article 5, uh, which together which reference specific things in the Europe. European commission doctrines. And what they allowed to together is individual countries to
Physicians who have clearly uh, communicated the benefits. They've observed with transplant as well as the lack of alternative therapies. Uh, and so in countries where possible we've been able to provide page rug uh other countries uh have elected not to do. And so we're about half now. Uh we've said that we expect the able to maintain about 25% revenues for the rest of 2025 and I think it's going to we're going to see how different countries different contracts and things plan out over time 1. 177 doesn't mean need to be renewed. It will be at the discretion of individual countries uh about renewing. And in some countries, they're for example, Italy is publicized. They're allowing for 6 months and then it'll be Revisited. Other countries have not given it a timeline. So it's going to be very variable which is why we've said we expect the 25% for the remainder of the year. Uh, but look, this is all incredible upside given the context of this situation. Uh, when you consider the license wasn't renewed, uh, and again, it's this is really based on a lot of the feedback from the patients and the positions about the clear
Received benefits of transart. And, and we're, you know, 1 happy to be able to still, uh, Harvest revenue from Europe, but to really happy to be able to still provide this therapy, to, to patients, who really need it.
Thank you.
Thank you.
Now, we're going to take our next question.
Um the question comes from line of tazin, Ahmed from Bank of America Securities, your line is open. Please ask your question.
Hi guys. Good afternoon. Thanks for taking my question. Um, for safian. 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, on the 3Q 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? Are receiving scripts first? Thanks,
Thank you for the question. Mrs. I think, uh, it's early days as you say, and I think we're happy with how how things have gone and, and particularly what we've seen in the public, a lot of social media, and a lot of, uh, patients talking about how happy they are. Uh, this is the day that they wish for and hope for. Uh, and just a lot of positive feedback, which you know, 1 is incredibly gratifying and 2 is, is really consistent with our understanding of the significant unmet need that. Uh, safian can fill Eric, do you want to go into a little more detail on to the MC questions on the early on the metrics? We plan to share in early Dynamics.
Yeah, thanks for the question, tazeen. I mean, as we said earlier that the metrics we're going to continually provide uh on a on a quarterly basis will be prescriptions uh patients start forms the number of commercial patients that are currently available, uh, on treatment. Um, we'll we'll also provide information regarding health care provider as well as payer sort of uh prescribing Dynamics and coverage Dynamics. Um, we'll also provide you some color on the roll out and the international, uh, flavor of safian where we which countries will actually be bringing on board. So there's a number of key metrics here, uh, but more importantly, I think you're going to see this as it's still early days but you're going to see um the metrics really centered around the number of prescriptions and number of of healthcare providers who have prescribed?
Thanks but on the um scripts received so far if any yeah and that just to just to provide you some, we're thrilled that we've already gotten prescriptions in the US. We have patients start forms that came in on the very first day, uh, and we, um, the the feedback from Healthcare Providers has been excellent. So far, as I mentioned, uh, we also have our first patients on Commercial therapy, uh, in Europe already.
Uh, the feedback from Physicians has been very positive. Uh, our teams have been promoting the benefits of suffice immediately, uh, the, the characteristics that we see in and against very early days, it's kind of matches up very closely to what we've seen. Physicians are looking to, uh, bring in patients, who are poorly controlled or have failed. Um, many of them have, uh, interests in switching patients to get better fee control. And we've also seen prescriptions for naive patients,
Okay. Thanks guys. Sure.
Thank you.
Now, we're going to take our next question.
And this comes around or Brian Chen from JP Morgan, your line is open, please ask your question.
Uh, and I have a quick follow-up. Thank you.
Yeah, sure. Yeah. Go ahead. Thanks for the question. Eric. Go ahead and take it away whenever call. Yeah, thanks. Brian for the question. We've been having a really good um meetings with payers so far. As I mentioned, it's been really dozens of pairs in a quite a good mix between both commercial Medicaid and Medicare. Uh we continue to have these meetings now post launch and I think really more than anything else. It's going as well as as what we expected. Um we see right now that the clinical profile
Of the science is being very well received the payer's already see that. It's highly differentiated. And there's a high, willingness, to cover the product importantly. Very minimal restrictions prior authorizations to, uh, the label and only a few have 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 pair mixed, 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, uh, I think it's going as well as we expected to go great, and I'm
Um, maybe 1 for pa. Um, is there any meaningful inventory bill that we should, um, think through? And also, um, how should we also think about any changes in terms of the sgna line, um, you know, that we should expect for the safian launch and thanks for taking our questions?
Hey, thanks for the question, Brian. Uh, as we mentioned, you know, we are leveraging, our existing infrastructure. So there will be no additional uh, Opex.
And then in terms of inventory, everything is ready, right? Just to be clear. Uh, so you know, uh all the patients, uh, have have what they need.
Yeah, and I'll just add I can add to a little more color to that Pierre. Um in the context of inventory, we plan to ship to patients sometime around mid August uh and clearly we were working with 2 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 as we've mentioned. There has been incredible excitement around the launch of safian and the community. Now is really poised uh, to uh, to begin that. So we'll be monitoring that very closely. But again, uh, Brian, we're not really in the, in the going to be building inventory.
Inventory at all. We're just going to be managing accordingly with our with our specialty pharmacies.
Great. Thanks guys.
Thank you.
Now, we're going to take our next question.
And it comes from Judah from a... from, um, Mongan is open. Please ask your question.
Yeah. Hi guys. Thanks for taking the questions and congrats on the update. Um, I guess can can you provide a little more color on on the decision to allocate Capital, uh, to these prior centers shareholders? Is there anything you can share in terms of kind of, you know, hurdles for for your decisions to do that on on kind of a returns basis relative to maybe allocating that Capital somewhere else Business Development on on potentially earlier stage assets or just investing, um, further in your pipeline. Thank you.
Yeah, thanks for the question Judah. So as we talked about we have we have accumulated and built, you know, significant cash reserves as we close the second quarter with almost 2 billion, still in cash on the balance sheet and what we said we're going to use that for uh, we're going to deploy it to strategically. We're going to support our commercial programs. Our R&D program pipeline programs as well as the thoughtful about business development and corporate development. And I would put this under their heading of a strategic, uh, deployment of our Capitol Pierre. Do you want to give a little bit more detail on on the thinking around this and, and why it was a strategic deployment of capital?
Position that will not preclude us from additional opportunities for BD or pipeline investments or on 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 Nevada since providing the update?
Adrian, I would say that the teams have worked very well together. There's a clear shared sense of uh, urgency and getting this program forward and and getting a therapy to patients. Uh, that could be beneficial. There's a share enthusiasm for the mechanism of huntingtin lowering and the positive attributes of of voter plan or PTC, 518 being and all an oral small molecule splicing agent. Uh, and I think what we what we've done over the past few weeks is really uh Take 2 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, uh, looks like
Thanks.
Thank you.
Now, we're going to take our next question.
And it comes to line of, uh, Kelly from Jeffrey. Your line is open. Please ask your question.
Uh congrats on the progress and thanks for taking my questions. Maybe first, I'm picking you launched specifically on how to timely capture those who are under care and the recently failed, other therapies also could they uh get a safe eyes and will there be some weight time between for logistical reasons? Also on the sales guidance for 4 year? 25 remaining, same from q1 uh from 650 to 800 million dollars. Curious. If this number included, any revenues from pko launch and also have a follow-up. Thanks.
Thank you for the questions Kelly. I'll start and then, uh, pass to to Eric and Pierre. First, what we, we've talked about the ability to act to provide benefits to the full spectrum of of, of PKU patients. But in terms of sequencing that there are relatively large number of patients, uh, who are at these specialty centers, 104 specialty centers that we talked about who were either on existing therapy or all therapies in, certainly benefit from 1, that could provide greater lowering and fee greater diet liberalization others, you've recently, tried and failed, uh, and others, you may be in recent contact with the centers and therapy naive. So, it's that group of patients, uh, that we've talked about that. We see is the first, um, and that we would be targeting in terms of sequence. And then, of course, those who may be in less contact with the center's time moves on, but there's
A large number as we shared on the, on the call, uh, last week, somewhere around, you know, 7,000 patients, fit into that first wave of of the sequence. Um, Eric do you want to talk a little bit about weight times and how quickly patients can get can get, uh, therapy?
Exactly. 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 uh on current therapies and would like to switch to an uh to safian. 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 um 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 8 and a half years. Our teams we have been dealing with that with pre-done and Plaza and the good news here is safian and the activities to find and and fee can be measured very quickly uh 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 caller.
Thanks for the caller, maybe just quickly, uh, uh, ah, could you share any comments on your list is the engagements with? Uh um uh uh the regulatory agency and also your confidence level. Uh for the for DOA, given its a only 12 days away, thank you.
Uh, despite it being so close to Padua. Uh yeah. You just want to comment on guidance and the inputs.
Yeah, guidance 662800. 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 a platform for instance, that's probably uh, the how you derive the 650 at the bottom end and the the 800 will be uh dependent on how fast we ramp up and as of type potential there as well. Thanks very much.
Thank you.
Now, we're going to take our next question.
And it comes to learn of brand Abrahams from RBC Capital markets. Your line is open. Please ask a question.
Hi. This is Kevin on for Brian, thanks for taking our questions. I just had a couple on uh supplies in the EU. Uh, just maybe can you talk about? You know, what, uptake is expected in in other EU countries with Early Access that you've identified and sort of more? Generally how we should think about the EU opportunity and then I believe you mentioned, no DNA, um, uh, impact, given these launches does that, I'm assuming that also applies to the EU as well. Thanks so much.
Kevin, thanks for the questions. I, I'll just tackle the second 1 first and let everyone talk about European Dynamics. Uh, no. We don't expect any Opex changes. Uh, for safian, it's all currently covered. As Pierre said, we're leveraging our existing infrastructure. Similarly for Vatican, own, if approved and launched their the Opex is already baked in. As we'll be leveraging. Our existing neurology commercial infrastructure in the US, Eric, you want to talk about what we're seeing are expecting in Europe Beyond uh the Germany uh, Early Access program.
Yeah. Um you know thanks for the question. Kevin the uh 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 quarter.
We're going to be leveraging a number of key markets uh 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 5 to about up to 10 markets, that could potentially contribute during the course of this year. And through the first half of next year, um, 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 moment.
Momentum that we've built from the US and Europe. So these are 2 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 the line of LML from UBS. Your line is open, please ask your question.
Hi, this is Ted. Just on for Ellie. Thanks for taking your question. Uh, I know you mentioned, you had some scripts coming in. Uh, 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 xus opportunities. How does the distribution of patients work in some of these countries? Are they concentrated at Major centres or are they a bit more spread out uh, through these countries? Thanks.
Thanks for the questions to us. Uh on on the first 1, it's it's still early. It's still early days to answer that question. So it's uh uh nothing more to add on that but I just want to talk a little bit about distribution of of patients in Europe.
Immediately into that Center and followed throughout adolescence and then their adulthood. So we see in Europe, a very high concentration
um in the major cities. Uh and it's really not as diffuse, uh it's actually far more centralized.
I guess, just with those compassionate youth programs, just around the world. How many patients are on the men including in the US? And how fast do you think you could convert this?
Yeah, this was, this was a very specific Early Access program in Germany that we launched. Uh, because for 2 Reasons, 1 with the pricing with listing of the pricing large tax initiating, the German launch. Those patients pretty quickly, uh, turn right over to Commercial and it also it was an opportunity for us to get in. Get the drug in the hands of those physicians at the concentrated Center. So we did not Institute a global Early Access program. This was a specific decision made in Germany uh or a global commercial use program. This is specific specific decision made in Germany due to the Dynamics of the launch listing of the price and the 6 months of free pricing, you get as soon as you issue the plunge.
Thank you.
Now, we're going to take our next question.
And the question comes down of Joe M. From City Group, your line is open, please ask your question.
Hey guys. Thanks, this is jar weigh on for Jeff uh 2 questions just to your earlier, comment on the early scripts coming in. Uh, could you provide some color on the uh, the Cadence and types of patients coming in? You know, I have these patients lined up more with their scheduled visits, or have these early patients been coming and voluntarily for a medication switch. And then second question, uh, based on your your latest conversations with FDA on the twin. You know, what has been your scent on the agency stance on a broad label for all age groups. And uh, is your possibility, the age agency?
um, could proceed demonstration and benefit on certain nfar 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,
Yep. Yep.
Thanks for the questions, your way on on the first question. Look. It's it's still a it's still early days. Uh and what we can tell you is that there's no you know, rules or or patterns we see you at others and we know that a lot of patients who are really interested in getting on drug really quickly uh you know we're seeing a lot in social media patients saying we're writing our doctors right away. We're trying to we're going to get in as quickly as possible uh and again a lot of the prescription decisions and prescription writing at the center
The MDS created, doctors could be inspect conditioners and so it it's it's really kind of a mix. Uh, We've also talked about a lot of centers particularly in us. Having weight lists, a patient. So there's, you know there 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, you know, wanting to get access to drug and Physicians wanting to provide the drug for all those different segments. As Eric has talked about, uh, on particular on, we would expect, uh, based on our conversations that the, uh, lab, a label would be inclusive of of all age groups, uh, while the move FFA study, uh, was was focused in uh Pediatric and Adolescent patients for whom there's an unmet need without approved therapy now. Um, we have data in adults in that study as well that are consistent with what we've seen in the uh younger patients. As well as data from our longer term extension studies that were provided uh in terms of confirmatory evidence.
Uh, and including data from an earlier study where we see in uh, adults book mandatory and non-android toy, significant significant effects in terms of slowing of disease progression over over years. So, taken together the data package clearly supports uh, benefits and certainly safety in the full age, spectrum and full spectrum of disease. Severity for FDA. Oh, we have not uh Gone formerly into labeling negotiation yet at this point,
Awesome. Thanks, man.
Thank you.
Now, we're going to take our next question.
And the question comes line of Julie from twist. Your line is open, please ask your question.
That the impacts heater and uh your your program and also quickly. Um do you still owe royalties to seereiter? Thank you.
Thanks for the questions, June, uh, on the first question. Uh, we're we're obviously watching with great interest, uh, the FDA interactions uh, that, uh, Sievers having around the gene therapy. Look, we've, we've talked a lot about, uh, you know, if there's potential benefits of the gene therapy administered, uh, to 1 part of the brain, uh, through direct Administration to lower HCT. If there's benefits there, we think that reads through incredibly well to lowering huntingtin protein, uh, throughout the whole brain, uh, and to be able to do that in a durable way. And and also allowing for those titration and monitoring periphery of, of at lowering. So there's always thought from a development standpoint, there are very good read throughs and certainly on the regulatory side, I think there's been an increasing, uh, uh, Desire by the agencies to have alignment, particularly, when it comes to their views on, uh, rare disease, whether it's in CBA or Cedar. Uh, obviously, the middle how to change is an FDA recently. But I think because that concept
In that desire to have alignment, uh, remains so, uh, with that in mind, we're clearly very interested in understanding how what the pathway could look like as we shared at the pivot HD readout in uh in May uh we were you know, very happy to demonstrate the statistically significant benefit out to 2 years relative to a well-matched. Uh Natural History cohort from the role. Each enroll HD database, as well as the uh, signals of dose dependent longer than a fellow out of 24 months. So you know, obviously we're going to be very keen to see uh uh the fda's um position with regard to using the natural history comparator with C2 address. Uh obviously we have the additional uh, initial benefit of being able to find the peripheral huntingtin lowering biomarker data as well, which is clearly, uh, an important uh,
Important piece of evidence that we're having a favorable effect on what matters most to the disease, that toxic huntingtin protein. Uh, and then up here, do you want to comment on remaining royalties, uh, where it should be toward?
Yes, we still owe low single digit warranty to Shiva toy.
Thank you.
Thank you.
Now, we're going to take our next question.
And it comes to an end of semicolon 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 trans learner review in the U.S. I think the last we heard there have been some clinical site inspections. 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 Sandy uh on trans Lauren that NBA remains under active review. As we mentioned that we had had the clinical site inspections completed in the spring. Uh we had got uh IRS in the early part of the summer, um, that we were able to see, uh, easily address. And so, you know, obviously, without the Bupa date, it's hard to know exactly when, uh, the agency will reach in action. But uh, we have head back and forth in terms of IRS, in addition to, uh, the inspections as we previously talked about, uh, in terms of the balance sheet we need, I know clearly as you know, we still remain with significant Firepower and looked at to strategically be here. Join just went talk a little bit about how we've been thinking about the potential, be the opportunity to use in timing.
Yes, uh, Osman mentioned we have a significant financial position and are actively looking at, uh, billy opportunities. You know, we have a global infrastructure; we know how to get drugs approved globally, and we know how to commercialize this drug, um, worldwide. And so these are, you know, we're looking at assets that we could cast in, uh, our existing infrastructure. Furthermore, we're also looking at pipeline assets that will complement our R&D portfolio. Uh, and that's how we're thinking about the opportunities. We have demonstrated to date that we will be disciplined and that we will do transactions that create value for our shareholders.
Um kind of follow a question on the BT. 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 on you know a lot a lot um
In certainly, in the commercial side, we'll see what we have. In terms of all the regulatory decisions and how to dictate whether we're busy doing multiple launches or whether we have, we'll have, uh,
Something in the continued to develop, uh, to drive Topline Revenue. We have a number of things coming from our research platforms coming in into the clinic. So it's going to be a matter of looking for an opportunity that can be complement, what we have already, uh, very nicely. But clearly, it'll be in our sweet spot of of, of rare disease could be CNS. Could be non CNS. I think it's going to be something that would have to be the the right opportunity that we felt could could complement our existing programs and and expertise.
I think the graph again.
Thank you.
Now, we're going to take our next question.
And this comes to land of Joseph, Tom from TD coins, your line is open, please ask a question.
Hi. This is uh, Payton on for Joe. Um, so kind of asking a little bit about the bad, tickle tone scenarios, um, saying that it is approved on around the budget date. 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, um, to try and get a registration in Europe? And then in the case that you do not get it. Um approved would you be open to running another trial? Um, if you could walk us through that as well, that'd be great.
The date and 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 uh, any approved drugs for patients under 16. We clearly see that as the first place, uh, that we would go, we we are are proud of having a long-standing collaboration with the project at taxia centers in the US, uh, many that uh, that treat pediatric patients and treat pediatric and adult patients. So I think it would be relatively uh it would be very exciting opportunity for us and we'd be ready to go day 1.
And, uh, and if you if it wasn't success, would you would you consider running another trial? Or would you, uh, shelter the program?
Yeah, as I said we're solving we think about success right now, we, we believe that this is a, we believe that particular know provides as a data show a an effective therapy. Uh, not only for children, we need a safe and effective therapy. Uh, but also for adults, uh, who would benefit from having a safe. Well, tolerated and effective treatment option. Uh, that's uh, that's what we believe the data is showing which is what support of the NBA submissions. Uh, and we, you know, we we can expect that we have a commitment to patients that we will always look to try to uh support
Great, 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, just uh, thanks for taking the question. Just 2 quick regulatory ones for me. Um, first to follow up on the quinone, 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 giving you uh, sort of Runway or timeline for when an action date could be put in place.
Yeah, so look on your first question of the, the there was no formal timetable, as we said, the midside, the late cycle meeting was just a few weeks ago. And, uh, there's still some questions about, uh, that we, we were talking to him about at the time, uh, and no specific timeline. For next steps was was given as you know I think things are quite busy at FDA these days. Um and in terms of uh the transa uh the uh and the AJ will not be a Pua data issued uh just given the particulars of that application. Uh that it was a resubmission uh of an NDA that was received with crl following a submission, uh,
Following an RTF anyway, just in the legacy of this going back to many years. It is not going to be as part of the PUFA program, so if AUA date will not be given.
Okay, thank you so much.
Thank you.
And I will proceed with our next question, and it comes to an end of Paul Choi from Goldman Sachs. Your line is open. Please ask your question.
Hi, this is Danielle on for Paul. Thanks for taking our question. We are curious about what the pricing assumption will be after the 6-month free drug program ends in Germany. Thank you.
Thanks for the question. Daniel Eric want to talk about our Global pricing strategies?
That very narrow pricing quarter regarding your specific. Question is a process in Germany? Yeah, I'm not process provides you free pricing and then after that, 6 months later, we'll be in negotiations following, um, um, 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, uh in Germany should actually, uh, obtain suffice, particularly those who are poorly controlled, who have failed. Uh, and I think the, the real world data over the next 6 months and the support from our key opinion, leaders and centers are going to help us with, uh, that that, um, uh, benefit assessment. So, uh, stay tuned. Uh, 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 quarter.
Thank you.
Now, we're going to take our next question.
And the question comes land of Jina Hwang from Buckley. Your line is open. Please ask your question.
Thank you for taking my questions. Uh, I have a 1 regarding, uh, Sophia's. Uh, just want to confirm. There is no IP protection and it will be rely on the often designation Market exclusivity in US, Europe and Japan. Uh, and I missed the beginning part of the call just, uh, apologize. It's already asked. So shall we start to see Revenue contribution in uh uh, 3Q 25? And then second question is a very quickly regarding the Antiquity in fa. Uh, I know you mentioned that late cycle review was discussed, uh, 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, uh, during the discussion or or afterwards
Thank you Gina. Uh, let me correct. The first question we have, we have guided it to 2039. Uh, we have a polymorph patent that goes after 2038 that we believe will provide if a composition of the matter, or a polymorph that we believe will support protection from 2038, and then conservatively estimating, uh, at least a year of patent term extension beyond that to 2079. Uh, and of course, we're continuing to work to expand as we always do. The IP portfolio to see if we can take protection. How do you even further Beyond 2039 and that applies not only to the US uh the 2039 guidance but to the key markets,
Globally. Uh, we have, uh, we will expect every Revenue in Q3, uh, from safian as we have already begun, uh, delivering commercial product in Germany, and we've gotten patients, start forms in the US and expect to be able to ship Supply and so patients in the next couple weeks. Um, we've said that there's, uh, in the, in the wide guidance that there is some new product Revenue based in there. And that upper end of the guidance, that stays 800 is allowing for the potential of upside, uh, from new products, uh, in terms of the tickling on The Late cycle meeting, uh, was a relatively brief meeting, uh, that we where we discussed, uh, the mainly focused on discussion of the evidence where they are with your view and their assurances, that they're actually still working through the review. Uh, not surprisingly 1 of the main questions of the review, uh, is
The fact that upright stability wasn't pre-specified, as the primary endpoint, with that support, persuasiveness of, of, of evidence of effect, which along with the confirmatory evidence could support approval. Uh, of course, uh, they acknowledged and were all aware of their recent, um,
Guidance, that others have received that upright stability, can itself be an efficacy endpoint and it has it will be used as a primary endpoint. For example, in the Scott Claris, pediatric trial, which I think supports the fds, uh, evolving understanding that upright, stability is the most relevant of measurement of efficacy, uh, in uh, ambulatory, uh, pediatric, uh, and Adolescent
Some patients as we said, we've not had any labeling discussions. Uh, beyond anything that was discussed in the uh late cycle meeting at this time.
Thank you.
Now, we're going to take our next question.
And it comes to an end of Joseph. Schwartz from Ling Partners. Your line is open. Please ask your question.
Claris launched as a template. And if not, how should we be thinking about that? And do you see any potential for combinations of Sky? Claris in particular, on in FA patience? Thanks.
Yeah, thanks for the question Jenny. I there was no discussion of of not meeting timelines. I'm not sure. They would be 1 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 and a lot of work. Uh, and certainly we've seen examples as recently as this week of programs from the neurology to division, maybe not getting meeting certain timelines. But, but we we, um, we look forward to continuing to work collaboratively with, with the division and, and getting to what we hope will be a positive outcome on time and if not on time, what what, what, what, what, when the time when the time can be, uh, as we talked about, we see the launch Dynamics being quite different, uh, for a number of reasons, I can let Eric go into that. Um,
You want to talk a bit about?
Yeah, I think we, we have an experienced team already in, uh, that have been calling for the last 8 and a half years in pediatric neurology. So, we've already profiled all the key centers, uh, specific, uh, to, uh, FAA. And we know the prescribers, we know, through claims data, where they are and the high-end needs plus the clinical differentiation. So we're already very well poised to launch and begin the process. Like we have with safian to to, to get patients, start forms on day 1, um, our our teams not only
Are experiencing this but we're looking at a number of different things which include not only the Pediatric, but a number of those patients and who are adults, who have failed on Sky Claris 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 in our team is experienced and ready to go on day 1.
And then to your other, just add that also, you know, we, we would see some we don't wouldn't expect there to be any monitoring. Uh, and, uh, you know, obviously the Pediatric patient has much patience. Have a much different Dynamic than adults do. So I think we would see the launch Dynamics quite different for that reason. Uh, in terms of combination therapy, look, I think that it's something that people talk about we've, I think, um, those close to the fa Community, have always believed rightfully, so that Fergus attack is going to be optimally managed with a cocktail of therapies like any other complex Disorder. So I, you know, we certainly imagine that they'd be interested in looking at combining both of the two Claris.
Excuse me, Journey. Any further questions?
Uh no, that's it. Thank you.
Thank you so much.
For the questions. I would not like to handle conference over to your speaker, chief executive officer Dr. Matthew
Klein. Please go ahead.
Thank you. All again, for, for joining the call. Today, we're excited to have had another strong quarter. Uh, we're still seeing continued contributions meaningful contributions from our DMD franchise, uh, which is great because we now can have that in hand as let me now embarked on on the future, which is the launch of supplies. I'm incredibly excited. How things are going in the early days? And, uh, 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, but thank you all again, uh, and have a great evening.
This concludes today's conference call, thank you for participating. You may now all disconnect have a nice day.