PTC Therapeutics at Wells Fargo Conference: Strategic Growth and Challenges

On Thursday, 04 September 2025, PTC Therapeutics (NASDAQ:PTCT) presented at the Wells Fargo 20th Annual Healthcare Conference 2025. The company highlighted its strategic initiatives, including the launch of Sefinance for PKU, while addressing both growth prospects and existing challenges. The presentation underscored a robust cash position, yet acknowledged potential declines in legacy businesses.

Key Takeaways

• PTC Therapeutics is launching Sefinance for PKU, targeting a significant market opportunity in the U.S.
• The company has a strong cash position of $1.9 billion, supporting future growth initiatives.
• PTC anticipates declining legacy commercial brands but sees them overshadowed by new opportunities.
• A meeting with the FDA is planned for Q4 regarding Vortilan’s accelerated approval.
• The company is confident in achieving consensus revenue estimates of $6 million for Q3 and $22 million for Q4.

Financial Results

• PTC Therapeutics reported a robust cash position of $1.9 billion.
• The company is optimistic about meeting revenue estimates for the remainder of the year, projecting $6 million for Q3 and $22 million for Q4.
• While legacy commercial brands are expected to decline, they continue to generate cash flow.

Operational Updates

• The launch of Sefinance for PKU is a key focus, with the potential to significantly impact the PKU market, which includes an estimated 15,000 to 17,000 patients in the U.S.
• Sefinance differentiates itself with strong efficacy and a favorable safety profile compared to competitors like Kuvan and Palynziq.
• The company is targeting both in-clinic and out-of-clinic patients, utilizing social media to reach those "lost to follow-up."
• PTC has identified 104 centers of excellence in the U.S., treating approximately 80% of PKU patients.

Future Outlook

• PTC plans to meet with the FDA in Q4 to discuss Vortilan’s accelerated approval and the next trial design.
• The company remains focused on its splicing platform and plans to share more information during an R&D Day in Q4.
• PTC’s intellectual property estate for Sefinance is expected to last until 2039, supporting long-term growth.

Q&A Highlights

• During the Q&A, PTC emphasized its comfort with current revenue projections and its strategic focus on Sefinance’s market penetration.
• The company acknowledged the challenges posed by a recent CRL for 2k1 but remains committed to resolving these issues with the FDA.

PTC Therapeutics’ comprehensive strategy and robust financial position suggest potential for growth despite existing challenges. Readers can refer to the full transcript for more detailed insights.

Full transcript - Wells Fargo 20th Annual Healthcare Conference 2025:

Thiago Fauci, Biotech Analyst, Wells Fargo: All right. Thanks everyone for joining us. I’m Thiago Fauci. I’m a biotech analyst here at Wells Fargo. We have PTC Therapeutics joining us in our health care conference this year.

Thank you

Matt, PTC Therapeutics: so much for spending the time.

Thiago Fauci, Biotech Analyst, Wells Fargo: We have Matt and Pierre for a fireside chat. I usually like to give you guys some time just for quick intros, overall setup. Again, PTC has been changing substantially over the last eighteen months, two years. So, yeah, if you can give, like, the brief intro and then, again, a detailed discussion on supply and some aspects of the business.

Matt, PTC Therapeutics: Yes, absolutely. Thanks, Thiago. It’s great to be here. And as you said, we’re really at a point in time today where a lot of the work we’ve put in over the past one years point to two years to shed a lot of legacy challenges and position ourselves in future success has come to fruition. We recently had our global approvals of Suffiance begin, including both The U.

S. And Europe. And as we’ve talked about, we see that as a significant commercial opportunity for the company that can take us to cash flow breakeven and beyond in the near future. We closed the second quarter with over $1,900,000,000 in cash. So we come to this point in time where we can move towards cash flow breakeven with sufficient capital to bank, not only to get us there but execute on all of our commercial and R and D priorities as well as have firepower for potential BD activities as we see fit to complement both the commercial and R and D portfolios.

We’re also excited later in the year, we’ll have a R and D Day where we can share with people the excellent work we’ve been doing in advancing our research platforms, including our splicing platform, which has brought us Avrizd, Vodoplam and we’ve been doing a lot of great work to advance a number of promising therapies to the preclinical stages. So a lot going on, but most importantly, we sit here today ready in the midst of a launch of something that for a product that we believe will be tremendously successful, strong cash position and look forward to continuing to build the company into the future.

Thiago Fauci, Biotech Analyst, Wells Fargo: Fair enough. Let’s talk about the launch, and let’s start with bigger picture, right? I feel like the market’s understanding of the PKU opportunity has evolved more recently, and you do see some numbers being taken up and a little bit more optimism. But again, historically, there has been a lot of skepticism on the potential to increase treatment rates and how exactly that could happen. PKU has been a market where, from a prevalence perspective, it should be a huge market, but the therapies that have launched on that have had less than expected success, let’s What call it, like subpar are some of those factors that are still being overlooked?

And why will Saptia be different, I guess?

Matt, PTC Therapeutics: Yes. Think there have been a number of, let’s just say, misconceptions that are starting to get corrected. The first is mistaking the individual therapies that have previously been reapproved, their opportunity is reflective of the market opportunity in PKU, and I think that’s a mistake. Both we’ve talked about there’s about fifteen thousand to seventeen thousand patients in The United States with PKU. As you said, Tiago, that’s a significant commercial opportunity.

And what Sefiance brings is really being the first therapy that has a strong efficacy profile across the full spectrum of PKU patients as well as a very favorable safety and tolerability profile. If you look at the previously approved therapies, had first you had Kuvan which is an oral therapy which while a large proportion of patients were tried on Kuvan, not a lot stayed on it. And I think that was because there wasn’t a perception of meaningful efficacy. Now if you think about the life of a PKU patient, you have newborn screening, so you’re diagnosed early in life and you’re relegated to a highly restrictive diet, diet control. Those diets are hard to maintain, they don’t taste well, they’re incredibly stigmatizing and really can impact quality of life.

And so you can imagine if that’s the mainstay of treatment, you now introduce a drug on top of that. If you can’t change having to have that really difficult and challenging diet lifestyle, why would you stay on the therapy? And that’s very different than we see in other diseases, right? We think about other rare diseases, including a number of neurological neuromuscular diseases where we’ll have therapies the goal of the therapy is to try to slow progression. Patients often have nothing else that’s going to slow progression.

So you’ll stay on a therapy in the hopes that over one or two years maybe there’ll be some slowing of progression, not that you may notice it, but there’ll be the inclination is to stay on that therapy. It’s different in PKU because your day to day life is already highly restricted. And if therapy can’t change it, why would you so it’s the inverse of what you’re used to. So that was the challenge with Kuvan. So you have basically I don’t think peak penetration never got very high.

That’s why peak revenue globally was $500,000,000 Then you move to Palynziq, which was approved in 2017 and was approved adults only, a tolerability profile that I think is challenging. There’s a black box warning for anaphylaxis. Patients are encouraged to carry EpiPens. If you look at some of the trial data, I think there’s an eighty nine percent adverse event rate for arthralgias. And you’ll hear patients talk about a lot of pain in their joints, difficulty walking.

It often can be a lengthy titration period. We’ve heard up to two years. And so there’s an enormous burden for an injectable on the patient, also an enormous burden on the healthcare team going through titration. And that’s why you’ve not seen a lot of penetration with that product. So basically, you still have all the today, all the pillars of success for commercial success.

You’ve got newborn screening, there’s centers of excellence, there’s educated physicians on how to prescribe, there’s patients who desperately want safe, effective therapies that can change their life, there’s payers that understand PKU, understand that value can be tied to phenylalanine lowering. So yes, you have this very large market opportunity, and now we believe there’s finally a therapy that can provide benefit potentially to the full spectrum of patients, change feed levels. Our data show that patients can have a diet liberalization, which is really, really important. And the drug has a very favorable safety and tolerability profile. And that’s why we’ve been so bullish on the commercial opportunity.

Thiago Fauci, Biotech Analyst, Wells Fargo: Fair enough. And I guess one of the follow-up questions that we get often is, again, for patients that are in clinic versus out of clinic, and that’s a term that I think that Maurin introduced a while back, just trying to segment some of the market here. Because the bare argument will be this is mostly going to be a Palynziq Kuvann switch story, right? Because if patients were severe enough, they would be on some kind of treatment.

Matt, PTC Therapeutics: What’s mistaken about some of those assumptions? So I think there’s two fundamental flaws with those assumptions. First is that if a patient isn’t on the current therapy, means they don’t want to be on the therapy, not true. Patients want to be on a therapy. They want to be able to have something that’s safe and well tolerated and make a difference in their life.

The second fundamental assumption is it’s not 1981 where the only way a patient can hear about or get access or know about a therapy is because they go to their physician. It’s 2025 and what we’ve seen in social media is first of all a tremendous amount of interest in patients including ones that doctors will say, I lost a follow-up. They’re like, I’m right here. I’d like a drug too. And you see this in social media and there’s networking and aggregation and patient pull for a therapy.

And so you could be out of clinic and still very much want a therapy. And in fact, one of them there was a whole lot of social media around the time of approval and there were several posts talking about me and my friends, we’re emailing our clinic right now. We want to get back in. We want to get on a therapy. And sure, admittedly, there may be adult patients who are years removed from being in contact with the clinic, whereas a lot of patients are still tied to a clinic for nutrition support, there’s those who aren’t.

Certainly we wouldn’t say that that’s the low hanging fruit of the launch, but we have initiatives already in place and we’ve been doing a lot of work with the community to ultimately get those patients on therapy if they desire. And as I said, the misconception that patients don’t want to be on a therapy is a horrible misconception and misunderstanding of the burden of PKU as a disease. What patients want is a therapy that’s going to make a difference and be tolerated.

Thiago Fauci, Biotech Analyst, Wells Fargo: Okay. And again, you did indicate there are a few centers of excellence that treat PKU. Do you have a sense of the number of patients that are being treated and being seen in some of these core centers? How widespread is the PKU patient population? So our work well, so there’s about 104

Matt, PTC Therapeutics: or 104 centers of excellence in The U. S, and our research suggests that a little over eighty percent of PKU patients are tied to these centers. So treatment is really concentrated at these 104 centers. And our team has been working on this launch for over two years now, and we, of course, have done a lot of the work of mapping these centers, understanding the demographics of the patients there, understanding the clinic staff itself. I think one of the important things to understand about the PKU clinic is, yes, there’s a physician who’s very important, but patient management, prescription decisions are often driven by nurse practitioners as well as dietitians.

Because if you’re not on a therapy and the vast majority of patients are not on a therapy, their care, their standard of care is diet management and therefore dietitians become very, very important in counseling and management. And so a lot of the connection patients will have at the clinic is through the dietitians. And I think the other important aspect of this launch and the key to success of this launch is the experience of our teams. We have a very experienced teams in doing rare disease launch. The U.

S. Team was our Emflaza team, which clearly has a track record of success in a competitive and genericized market. And so they’ve been working on this for two years, getting ready for the launch, understanding the dynamics of each center, knowing what you have to do to sort of get patients in, support teams at the clinics to ensure that we’re getting patient start forms, getting patients on drug, and making sure that we can really keep that funnel wide in terms of the opportunity.

Thiago Fauci, Biotech Analyst, Wells Fargo: Got it. And that is the only one question that we hear all the time just related to the pace of new patient patient adds. Right? Like, even even from our survey, work that we’ve done, there there seems to be a huge intent to prescribe and expectations for a substantial uptake early on. So based on the the early days or, I guess, based on your expectations, is that gonna be as a slow grind, kind of more typical rare disease launch, or is there an initial bolus potentially for for some of these patients?

How can we think about some of those dynamics? And, again, I’m assuming that’s going to be across different patient segments, so I don’t know if how helpful it is to subsegment the market I think it’s

Matt, PTC Therapeutics: hard to I think we can subsegment the market further, but I don’t know if it’s helpful because one the things that we’ve heard and your research has shown and a lot of the KOL interviews have demonstrated is that the initial segments the physicians want to target are going to vary center to center. We certainly heard a number say, look, it’s so easy for me to just take my patients who are on Kuvan branded or generic and just switch one once a daily therapy for another. There’s not a titration to do, there’s not adverse event management to do, it’s a very easy prescription switch. Others say well I’ll get to them but we know we have severe patients who don’t have any therapy who would certainly benefit from an efficacious oral therapy, and I’m going to get those on first. We do expect it to be different.

But as I mentioned, the way we’re thinking about this is given that the data support the potential to benefit the full spectrum of patients, including those more severe patients, what we’re watching for is the breadth, right? And I said wide funnel. What I mean is that if we’re seeing centers have interest in prescribing for the full spectrum of patients, given the response rate we had in the trials, including for the severe patients, that’s when this opportunity could get very big. I think we will see bolus. I think that will be the case, but I also think you’ll have sort of this sustained bolus over time.

We heard the interview recently with the Director of the PKU Clinic at the Children’s Hospital of Philadelphia, and he said, Look, I’ve got 200 patients. I think over time, I’ll trial all 200 patients My goal is to try to get 100 on therapy by the end of the year. I’m putting in start forms for two to three patients a week. So when you hear that and he’s not alone, we’ve heard that from other centers that sort of suggests that you’re going to see initial strong interest, but it’s going to be sustained just because of if we’re realistic about throughput in the clinics and then getting folks from PSF to prescription.

So we see this as a strong sustained start. Got it. So again, before we go into a little bit more detail on

Thiago Fauci, Biotech Analyst, Wells Fargo: the near term launch dynamic, just thinking about payer feedback and what we’ve seen so far, again, of the pushbacks we used to get less so now, but it’s still like, hey, if you have Kuvan and it’s generic

Matt, PTC Therapeutics: Yeah.

Thiago Fauci, Biotech Analyst, Wells Fargo: This is better Kuvan. So why wouldn’t you step through it? Like, so are you seeing any step like, what exactly have you seen? Can you quantify number of policies or lives cover that might have some sort of limitation initially versus not? Yes.

Matt, PTC Therapeutics: So I think, again, that’s another one of those misconceptions that this is sort of Kuvan two point zero. And I think it’s so clear from the data that this is a highly differentiated therapy. The data also show that if you did have benefit on Kuvan, the benefit on Cefiance is much stronger. Thousand miles away from here in Japan at the International Metabolism Meeting. Our team shared data this week.

It was a small head to head study done to support pricing and payer discussions in Europe, about 40 patients crossover study with Cefiance versus Kuvan branded generic saproteren and basically showed a seventy percent greater reduction on average with Cefiance. So again, highly differentiated therapy. And then of course, when you consider the potential to benefit patients who have what’s been called these sort of non BH4 responsive mutations, most of them are classical, that tells you this is a different therapy. And I think the payers understand that. In our payer discussions, a lot of this was about are the payers viewing this as a the value proposition is there as a highly differentiated rare disease therapy, and the answer is yes.

It turns out that the majority of patients, to our understanding, have been tried on Coumadin in the So you have the potential already to have medical documentation of previous treatment with Kuvan. We’ve had some payers say that we understand as well that there’ll be patients with these non BH4 responsive mutations. There’s no reason to think they’d respond to Kuvan, so it makes no sense to step through that. Now we do expect there will be some who will have steps, but this is another way in which PK is a bit different than other diseases because you can try Kuvan if you have to do the step. It’s a few weeks and a blood test.

It’s very easy to tell what your level of response is, and then you can go ahead and demonstrate the superiority with suppliance. And just by way of contrast, our teams are used to some plaza where we have to step through the prednisone, and that’s six or nine months trying to show some benefit in DMD, which is a lot less objective than a blood test for phenylalanine. So in terms of having to do a step in the cases that’s required, it’s obviously relatively simple and straightforward.

Thiago Fauci, Biotech Analyst, Wells Fargo: Got it. And again, can you just recap what’s their mix? And again, I’m assuming because of that competitive dynamic, there’s you shouldn’t expect necessarily substantial gross to net discounts or things like that. So what is the mix? And do you expect that to change over time?

There has been a lot of focus on some other launches with a more dynamic payer mix going forward. So that is one of the questions we get early on.

Pierre, PTC Therapeutics: Yes. Twothree of the patients are commercial. Twothree is commercial, yes. So in midstream dynamic versus other rare disease, when you take Duchenne, it’s the complete opposite, At the start, we believe most of these patients will be commercial. And so we’ll give more guidance on gross to net at Q3, etcetera.

So you will see, I will believe, probably on the lower end than other rare disease and slowly Going to an average. Grow a little bit, but again, similar and typical rare disease gross to net.

Thiago Fauci, Biotech Analyst, Wells Fargo: Got it. Perfect. And again, thinking about, like, near term, like, patients converting to commercial patients, right, so, how many patients do you have in The U. S. That are still kind of on trial drug?

And some folks assume that it’s kind of overnight switch that you just started them and they start generating revenues. Like how should we think about the magnitude and pace of that conversion? Yes. We spend a lot of

Matt, PTC Therapeutics: time thinking about this. And because we get asked a lot about that. We and did you consider an expanded access? Do you have an expanded access? So maybe I’ll talk about what we did in Germany and then in The U.

S. Because it’s a great example of how we think about the problem and make sure that we have a strategy that’s aligned with the context of the situation. So in Germany, we initiated an early access program because in Germany, as soon as you list an LAROTAX and become commercial, those patients do convert to commercial almost right away. So in that case, it made a lot of sense to have a program where we have patients on drug because those would be then commercial patients very early in the launch. And so the objective of the early access program in Germany was twofold.

One was to say, okay, let’s get patients on drug. But the other was let’s get the drug in the hands of the key prescribers at the leading centers in Germany because while they may not have a lot of patients that they’re going to put into early access, they’ll sort of the inertia of being in motion. They’re already in the habit of treating the drug has been in their hand. They have experience with the drug and their enthusiasm will build so that when you are commercial, where we became commercial in July, we’re seeing these folks now come and get even more prescriptions. So that worked really well.

In The U. S, we don’t have a lot of patients left in the open label extension. I’d say probably fewer than 20. But we again looked at this question of how do you accelerate access. And as you said, Tiago, I think it’s a misconception.

It’s not easy to go OLE to it doesn’t happen overnight. And it’s similar as true for expanded access. It’s not like we have it’s not like we had to find do patient finding, right? It’s not newborn screening, so it’s not like you had to find them and warehouse the patients. It wasn’t like we were in an overly competitive space where we had to try to get them on drug.

That really wasn’t the case. And the third part is what we heard is in a number of centers has been waiting lists for patients, where larger centers in some cases had hundreds of patients on a waiting list to get on the drug. And the fourth is we had an opt in program, PTC Reimagines PKU. And what that did is we got over 1,000 people participating in that program that allowed us then to proactively reach out upon approval, our patient services team to reach out to help sort of provide this white glove service to get folks to centers and get on drugs. So we thought that that was a much more effective and efficient way of getting early prescriptions than relying solely on the OLE.

Thiago Fauci, Biotech Analyst, Wells Fargo: Got it. Again, like everything sounds pretty bullish, and I think this really is slowly trying to recognize that the billion dollars in The US alone, peak sales opportunity, again, takes you about 3,000 patients, possibly, like a little less, a little more. Yeah. About about 3,000. So, again, versus 15,000 Yep.

Total prevalence. Got it. And, again, twenty, twenty five consensus depending on how you do the math is, like, 300 patients on drug gets you there. Like, that seems overly conservative, but but I don’t wanna get too too too ahead of myself here. But what are some of the things that could actually imply a slightly slower ramp than folks are modeling or slightly faster ramp than folks

Matt, PTC Therapeutics: are modeling? Look, I think it’s early days. We’ve held out that at least $1,000,000,000 number as something we believe in. And as you pointed out, it’s fairly modest penetration certainly when one considers the rare disease opportunity in the landscape and the potential of the therapy. We’ve said consensus revenue for the rest of this year, think it’s $6,000,000 for Q3, 22,000,000 for Q4.

We said we’re very comfortable with those numbers. And look, we’re really focused, again, on I’ll come back to that term, the wide funnel. We’re very focused on making sure that centers are have the information they need to follow what the CHOP said, for example, the CHOP clinic had said, trial all of our patients on the drug. Because we believe with the response rate that we have in our trials, I think we had twothree of patients respond if you look even at the most severe patients, about half of them respond with greater than 30 reduction, more than that with greater than fifteen percent reduction. Now that’s going to get us to big numbers.

Exactly when we get there, look, we’ll see. I think we’ll have a much better grasp on trajectory as we close out 2025, move into 2026. And what I can say is right now, we haven’t had any surprises in these early days of launch. A lot of the things that we planned for have been coming And to I’ll also say what’s really encouraging to us is when we see the interviews done with the physicians and surveys done with the physicians and discussions with payers, time and time again, the feedback that’s coming from those interviews is consistent with everything we’ve said. So I think when you hear that, that’s really encouraging to us that the opportunity as we see it really is indeed the opportunity in front

Pierre, PTC Therapeutics: of us. And then also what is really encouraging is the feedback from patients. So if you look at social media or feedback we get, we have a few, like, mom who had a breakfast with her daughter for the first time in her life, for instance, right, the same breakfast. What we take for granted on day to day life, this is obviously transforming patients’ life. Right?

Thiago Fauci, Biotech Analyst, Wells Fargo: Yeah. No. That’s fair. Anyway, so you have Germany. You’ve mentioned a narrow pricing corridor of US, ex US.

I don’t know historically if there are any comps that might be helpful in trying to think of the magnitude of discount or not. But again, Cuba and ex U. S. Had had very limited success, right? So we also see some pushback on that.

Might have a more favorable pricing across some regions. What are some factors here on

Matt, PTC Therapeutics: the ex U. S. Opportunity? Yes. So a few comments.

Again, I think the comp to Kuvan is difficult. Just given historically, I believe it was partnered early on for the launch, and then BioMarin took it back and tried to do it. And that’s always a very difficult setup. Also outside The U. S, particularly in Europe, where medical foods and formulas are paid for, the payers in Europe want to understand what diet liberalization will be like.

That’s why having those data so early on for us in the European label are going to be very helpful in Europe. Plus now having the head to head study I mentioned, which we did to support payer discussions in Europe would be helpful. With that as the backdrop, what we said is, look, we are we always look to maintain a narrow pricing corridor. The price in Germany for the six month free pricing period is equivalent when you account for currency conversion with The U. S.

Price, right, which is $4.9 for U. S. Dollars for forty five kilo patient, which we think will be the average weight. We also said we’re going to be looking to commercialize the drug now in other countries in Europe that have main patient programs or early access schemes where, again, we can control the price and maintain that narrow corridor. We said the next and so we have seen prescriptions from other countries in Europe.

We expect to continue to see that. The next major market to come on board will be Japan. We expect approval in Japan in the fourth quarter with an anticipated launch in 2026. And even prices in Japan for, I think, Kuvan and Palynziq were the highest in the world. So they tend to be higher than The U.

S. Prices. So again, we don’t see any challenge to maintaining the pricing core than in our key markets as we move into the through this first year of launch.

Thiago Fauci, Biotech Analyst, Wells Fargo: Got it. Last check the box question, just the IP state around Sentia, if you could kind of remind us.

Matt, PTC Therapeutics: Yes. We’ve been guiding to IP to 2,039, and that’s based on the 2,038 composition amount of polymorph and patent term extension conservatively getting us to 2,039. We’re continuing to work to expand the IP portfolio. We’re continuing to do filing work and things that can extend the patent life even further and further strengthen the fence we have now.

Thiago Fauci, Biotech Analyst, Wells Fargo: Got it. Again, spent a ton of time with the financing. That’s commensurate with the imbalance that we get. But perhaps let’s just touch upon the rest of the business as well. Relative to the, let’s call it, legacy commercial side of the business, again, this perception of it’s going to be a slowly decrease over time on revenues because of Translarna and Plaza, some other fact, like, how should we think about the legacy commercial side of the business still generating cash flows, right?

So it’s not worth zero. No,

Pierre, PTC Therapeutics: it’s still generating cash flow. And again, I think it went beyond what we all expected. I mean, at Europe, right? I mean the license was removed, but we’re still studying and we said we could maintain 25% of European sales at peak and Flaza despite generics that happened last year in early twenty twenty four, you can see that it’s eroding. But again, in rare disease, there’s no gain from generic to drop the price 90 percent because it’s rare, right?

You don’t have the prevalence and the volume you will need. So you should expect those brands to again, to go down over time. And then again, it’s going be dwarfed by PKU.

Thiago Fauci, Biotech Analyst, Wells Fargo: Yes. No, that’s fair. And on the rest of the pipeline, again, you get a lot of questions about the plan. I think now with Novartis kind of taking over the program, it gets a little bit more challenging. What can you tell us about next steps and expect a news flow to the extent that you can?

You have really strong back end economics and milestone payments associated What’s with the latest? Yes. I would

Matt, PTC Therapeutics: say, long term, it’s still a very important part of the picture for PTC because of the economics, the 40% profit share in The U. S, the $1,900,000,000 in milestones in development and commercial milestones. The Novartis and PTC teams are working very closely on the next steps. It’s been a very productive collaboration. I think there’s like mindedness on both sides in terms of the potential for this therapy and the desire to move it forward as quickly as possible.

We said the next steps for the program will be a meeting with FDA in the fourth quarter with two objectives. One is to align with the agency on latest thinking around accelerated approval with the data we have, with the data that will be coming with the twenty four month readout as well as in light of the discussions that Unicure has been having with their potential path to accelerated approval. We think is that those discussions increase the optionality for vorticline.

Thiago Fauci, Biotech Analyst, Wells Fargo: Yes. We’ve got that question as well, like what’s the potential Because there seems to be some degree of flexibility. At least that’s how it’s being perceived. The data for photo planning and given the safety profile and all those things, I would argue stacks up favorably.

Am I over extrapolating here? Hard to read the tea leaves, but

Matt, PTC Therapeutics: I don’t think you are. I think it at all. I think one thing there’s been a lot of change in FDA, lot of uncertainty, and it’s hard to predict the FDA. But I think one thing in FDA that’s been fairly consistent and clearly a greater effort in the recent past is to make sure that when there’s a precedent for accelerated approval, it’s broadly applied. And so I think that’s why I say if there’s a path forward for UniCure using CUH DRS and natural history comparisons, that just puts another option on the table for vodipan, which we’ll have.

We will have 12 of placebo controlled data. And then that’s just again, we have that, we have NFL, we have the evidence of target engagement So a lot of different options to put together a package to show that we are likely to have long term benefit. I’d say then the second objective of the FDA meeting is to align on the next trial. Novartis has always been keen on getting this trial going, whether it stands to be the confirmatory trial or accelerated approval or Phase III, get it started.

And so there’s a desire to align on that design and get that trial started as quickly as possible.

Thiago Fauci, Biotech Analyst, Wells Fargo: Got it. Real briefly on two k one. Again, not the the outcome you were expecting. Anything to add there? Is there something to is there a way to establish the program?

Is this something that you may actually run another confirmatory trial given capital constraints, timing? How are you

Matt, PTC Therapeutics: thinking about it? Yes. Look, it was it’s a very disappointing outcome, think, certainly for the patients and certainly because a lot of the words of leadership at FDA was that in these circumstances where there’s significant unmet need for pediatric patients, which certainly is refriedrich ataxia, that we’re going to use common sense and not statistical formalism. And I think what was very clear to us in the CRL was that when it came down to it, this was statistical formalism. We always said that the main question of the review would be the acceptance of upright stability as an approvable endpoint since that portion of the disease rating scale itself wasn’t prespecified.

And it turns out that was the major point for the review. Was very hard for them to believe we had substantial evidence of effectiveness when you didn’t hit the prespecified primary endpoint. So a lot of what wasn’t supposed to be the case in 2025 seemed to be the case, okay? But what we take away from this is there’s still an unmet need. It’s not that the drug didn’t work.

This was a question of it wasn’t we cherry picked to try to find something. These data were right in front of us and very clearly an important effect on what matters most in these young ambulatory patients, which is time of loss of ambulation. So we’ll have the Type A meeting with FDA. We’ll have the discussion, clearly understand is there any path other than doing another RCT. If the answer is no, do another RCT.

We’ll take a look at it. Again, we believe the drug has effect. We believe it’s safe. We’ll take a look at what the trial costs and POS and the landscape just like you would do with any other program and make a decision at that point.

Thiago Fauci, Biotech Analyst, Wells Fargo: Got it. And again, you have the upcoming R and D Day. I’m not sure you can front run that, I see a lot of details. But perhaps more broadly, last question on capital allocation. Really strong balance sheet.

You guys have been creative, refinancing some of the old debt and now rebuying some of the royalties, signals confidence in the launch, so on and so forth. So what are some of the key priorities here? And in terms of BD and rebuilding the pipeline, any thoughts on doubling down on the internal splicing platform or other molecules you might have versus licensing different opportunities?

Pierre, PTC Therapeutics: Yes. So first of all, we closed the books with $1,990,000,000 So you pointed out very strong financial position. We’re very pleased, and we worked very hard to get there. And we’re in no rush to go rush and buy an opportunity just for the sake of it. First of all, that allows us to run with our commercial PKU launch, continue to develop our internal pipeline with splicing and information, and we’ll talk more about that in Q4 in an R and D Day.

And having said that, we’ll be opportunistic, right? It’s what we did with Stemsa, and we will always make sure that we’re disciplined, opportunistic. And when we look at opportunities, we create value

Thiago Fauci, Biotech Analyst, Wells Fargo: for shareholders. Got it. Perfect. Any last parting words like on the overall PTC setup? Think we cover everything, but just in case I missed anything.

Matt, PTC Therapeutics: You covered everything. Would just say, look, we’re incredibly excited to be where we are today, very well positioned for growth and success with Defiance. LOTOPLAM is still an important part of the picture, the splicing platform and the cash we have, we think we’re in a pretty unique position in terms of potential for future growth and success. Perfect. Thank you so much again for your time.

Pierre, PTC Therapeutics: Appreciate it. Thank you, Taylor. Thank you.

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