PTC Therapeutics at Goldman Sachs Conference: Strategic Growth Insights

On Tuesday, 10 June 2025, PTC Therapeutics (NASDAQ:PTCT) shared its strategic vision at the Goldman Sachs 46th Annual Global Healthcare Conference. The company highlighted a robust financial standing with over $2 billion in cash, positioning itself for significant growth. While the outlook is optimistic with upcoming product launches, challenges remain in meeting regulatory milestones and market penetration goals.

Key Takeaways

• PTC Therapeutics holds over $2 billion in cash, supporting its operational and strategic initiatives.
• Anticipated launches of Cefiance for PKU and vatiquinone for Friedreich’s ataxia are key growth drivers.
• The company aims for $2 billion in revenue, with significant contributions expected from Cefiance.
• PTC518 shows promise for Huntington’s disease, with potential for accelerated approval.
• The company is actively pursuing external opportunities to complement its internal R&D.

Financial Results

• Cash Position: Over $2 billion at the end of Q1, enabling planned launches and R&D advancements.
• Translarna Revenue: Expected to maintain 25-30% of European revenue, contributing 40% to overall 2024 revenue.
• Revenue Targets: Aiming for $2 billion in top-line revenue, with Cefiance projected to generate $1 billion in the U.S. alone.

Operational Updates

• PKU (Cefiance): FDA NDA review underway, with approval expected in six weeks. Targeting 85-90% unmet need, with premium pricing supported by payers. Early access program started in Germany.
• Friedreich’s Ataxia (Vatiquinone): FDA PDUFA date set for August 19, with a focus on treating both children and adults.
• Huntington’s Disease (PTC518): Positive Phase II data supports discussions for accelerated approval. The drug shows dose-dependent benefits and is positioned for broader market access with Novartis partnership.

Future Outlook

• PKU (Cefiance): Anticipated simultaneous launch in Europe and the U.S., driven by strong clinical data.
• Friedreich’s Ataxia (Vatiquinone): Initial focus on pediatric market penetration, expanding to adults.
• Huntington’s Disease (PTC518): Plans to expand into earlier-stage patients, leveraging strategic partnerships.
• Strategic Business Development: Actively seeking external opportunities to enhance growth, targeting $2 billion in revenue through internal and external initiatives.

Q&A Highlights

• PKU Market: Significant penetration expected in a market of 15,000-17,000 U.S. patients, supporting a $1 billion opportunity.
• Huntington’s Disease Competition: Confident in serving a broader market despite potential competition from gene therapies.
• Cash Usage: Funds allocated for operations, product launches, and strategic business development.

For more detailed insights, refer to the full transcript below.

Full transcript - Goldman Sachs 46th Annual Global Healthcare Conference:

Paul Choi, Analyst: Good morning, everyone. I’m Paul Choi, and I cover the biotechnology sector here at the firm. And it’s my pleasure to welcome PTC Therapeutics for our next session here. To my immediate left is CEO, Matt Klein, and to my far left, Pierre Grave, CFO. Maybe what we’ll do is turn it over to Matt to kick it off maybe with some high level comments just on sort of the course of the year ahead, and then maybe we’ll get into Q and A, a little bit about the commercial side, but what is obviously a very exciting pipeline stage period for the company.

Matt Klein, CEO, PTC Therapeutics: Yes. Thanks a lot, Paul. It’s great to be here. And indeed, we’re in a very exciting time for the company. We’re coming off an incredibly successful 2024, where we had outstanding execution across every part of the company.

We had four FDA NDA submissions, all of which were accepted for review with one approval coming last year and now three approval applications pending, including our Suppiance NDA from which we expect an approval within the next six weeks or so, so really coming up fast. We had another year of strong revenue performance and effective operating expense management that put us in a very strong cash position. We closed Q1 with over $2,000,000,000 in cash in the bank, and that really provides us a solid foundation to successfully complete the planned launches, continue to advance our R and D portfolios and also think about strategic business development to complement our existing R and D and commercial portfolios. So as we sit here today, we have a solid foundation in terms of cash on the balance sheet, a solid revenue opportunity forthcoming with PKU, potential upside scenarios, including the Friedreich ataxia program, potential approval in August, Translarna in The U. S.

As well as our PTC518 program, for which we announced a successful Phase II data package last month. So really a lot of exciting things going on and look forward to discussing.

Paul Choi, Analyst: Great. Maybe we can just start with sort of the existing commercial portfolio and talking a little bit about the sort of legacy products, for lack of a better term. And given the regulatory change with Translarna in Europe, maybe just as kick it off there, you’ve previously talked about maybe roughly a quarter of that business being maintained. How is that transition going since the decision sort of intra quarter? What updates can you give there?

And then maybe just how you’re thinking about that on the forward as you’re thinking about strategic planning and sort of the base case?

Matt Klein, CEO, PTC Therapeutics: Yes, absolutely. I think Translarna in Europe is again proving to be an unprecedented story. We had the adoption by the European Commission of the CHMP opinion at the March. So while in first quarter revenue was in line with our run rate for 2024, we did share that given that the European Commission invoked something called Article one one seven, which enables each individual country within Europe to make a decision to potentially continue to commercialize Translarna in spite of the lack of renewal of the marketing authorization. And this is something our team had been planning for.

And in fact, as we sit here today, more than half the countries in Europe have expressed a desire to continue to commercialize the drug. We’ve been importing drug into a number of countries. This has included providing continuity of transformative therapy for those who’ve previously been on drug and in some cases even discussion about starting new patients. So we’re again in these unprecedented waters that despite not having the marketing authorization, we are still able to commercialize the therapy. And so this, as we said, we believe can allow us to maintain about 25% to 30% of the European revenue, which represented about 40% of the overall Translarna revenue for 2024.

And so exactly as you framed it, Paul, these are sort of the legacy products that have been able to bridge us quite well to the future products, PKU and others. Mhmm. And we’re still in a position to garner meaningful revenue both in Europe and outside of Europe and other countries with Translarna as we continue to move forward.

Paul Choi, Analyst: Right. Maybe one more question on the topic before moving on is just how you think about the potential durability of that revenue in Europe. I think a lot of investors, they ask, what’s an analog here? I’m like, I really can’t think of one. And so I’m thinking, how does the investment community model this and think about it on

Matt Klein, CEO, PTC Therapeutics: this one?

Paul Choi, Analyst: Or can just take it out of the model altogether?

Matt Klein, CEO, PTC Therapeutics: Yes. Think there’s no analog. I think we’re comfortable with that 2530% for the foreseeable future, in particular because there’s really no therapy that can supplant Translarna even if you look at what may or may not happen with gene therapy in Europe, we know that many of the nonsense mutations that the Translarna patients have sit in exons that may be relatively contraindicated for the gene therapy. And when you also consider that PHMP, believe, is looking at a potential label of four to seven year olds. So again, so there’s no situation we see in the near future or intermediate term future where there’ll be a replacement for Translarna.

So

Paul Choi, Analyst: I that

would Maybe turning to some of your pending commercial stage assets and starting with Cefiance. I think this is the asset that investors primarily focus on as they think about what PTC looks like in the future here. Maybe you could frame for us, you’ve characterized it as potentially $1,000,000,000 opportunity, but the analog drugs of the PKU space have certainly not come up to that level of sales. So can you maybe just frame for us what is the unmet need, I guess, in the PKU community right now that’s not addressed by the currently available therapies?

Matt Klein, CEO, PTC Therapeutics: Yeah. I think SIFIANCE is a highly differentiated therapy. Its efficacy and safety package is highly differentiated from the current approved therapies, which is why the market opportunity far surpasses that which has been achieved with the current therapies. It’s estimated that roughly about ten percent to fifteen percent of the fifteen thousand to seventeen thousand patients in The U. S.

Are on these therapies, which leaves a significant unmet need of eighty five percent to ninety percent of the population. And so we’ve talked a lot about the significant opportunity. That’s based in part on the size of this population, the strength of our data which supports that we can address every key patient segment, including the more severe classical PKU patients. We’ve shared that we have plans to price this premium to Palynziq, which is supported by the payers. In fact, there was a note recently that came out on payers’ response, the net conclusion was as we said, there should be no barriers to getting, suppliance to all of these, patients.

And finally, our established commercial team. We have a, this is the team that has, successfully commercialized Emflaza, has continued to enjoy significant revenues post loss of exclusivity. They’ve been able to successfully navigate competitive and genericized markets. And that’s not only our field force teams, but also having things like our PTC Cares patient services team that ensures high levels of adherence to therapy, helps navigate through prior auths and re auths in DMD, which is far more complex than PKU. And now we’re seeing more and more research done by others which is supporting that numbers such as, you know, over 80 of physicians are are are going to or switch their pay or physicians think over eighty percent of their patients on current therapies, they’ll look to switch.

Over fifty percent say that they’re going to look to try all patients on PKU. So when you start now looking at what penetration could look like in a market of 15,000 to 17,000 patients with the potential net pricing we’ve talked about, the math to get to $1,000,000,000 alone in The United States is quite easy. And then when you layer on top of that, that we have a team in place that knows how to successfully commercialize rare disease therapies.

Paul Choi, Analyst: Great. Maybe can you remind us, like in terms of the treating community within PKU and endocrinologist, metabolic metabolic focused treatment centers versus sort of your rare neuro orphan call points, how much of that is overlapping and just how much is will require a bit of a new build out to hit these new call points?

Matt Klein, CEO, PTC Therapeutics: So there’s about there’s 103 PKU expert centers in The U. S, and a lot of those centers overlap with the centers that we have worked at before, which is what’s really important. While the specific physicians may differ, you’re in the same hospitals, same pharmacies, same things to navigate in terms of getting on formulary and getting all that is and we’ve done the work already to we’ve mapped those centers, mapped the KOLs, understanding the treatment teams. It’s very important in PKU to understand that not only are there physicians driving prescribing decisions, but nurse practitioners and also importantly dietitians. Patients who have PKU may not be on an existing therapy, yet they remain they maintain their connections with these specialty centers, in particular with the nutritionists and dietitians because then the mainstay of their therapy is is diet.

And so understanding those interactions at each center, understanding who we need to work with Mhmm. To drive prescriptions and manage patient care, we’ve been doing all that. I mean, is an advantage not only of having an experienced commercial team, but coming into a commercial landscape where there have been therapies. Because often what we’ll have to do when you’re to a therapeutic area is talk to centers and educate them about prescribing a commercial drug. You have to aggregate the patient communities.

In many cases you have to do patient finding. But we’re in a situation now where there’s newborn screening for PKU. So the patients are all identified. And there’s about three hundred to three fifty new patients identified every year, not by us, just by newborn screening. The centers are identified.

The Centers of Excellence are there. There’s an understanding and a culture of understanding about what it’s like to prescribe a therapy. When we talk to payers, they educate you. And importantly, they understand that you could tie value to phenylalanine levels so that when we’re able to come in and say, look look in our clinical trials at the high level of phenylalanine lowering we’re achieving, sixty nine percent on average of the class we gave you patients, Getting eighty four percent of patients to less than three sixty, which is the threshold for desired phenylalanine where you can start liberalizing to high. All those things are really, really important and facilitate our launch.

Paul Choi, Analyst: Great. I think in terms of time lines, Europe might be a little bit ahead. And so is the thinking that you’ll presumably launch ahead in Europe, just given the regulatory time line on the calendars as a little bit in advance of The U. S. PDUFA here?

Matt Klein, CEO, PTC Therapeutics: Yes. They’ll actually be very close in time. So the plan, we’re expecting to have the approval in Europe before the June. Our plan is to launch in Germany, taking advantage of that six months of free pricing. You a certain advance notice needed before you list the price in the Larotax in Germany, which is what kicks off your launch.

Paul Choi, Analyst: And your IRAG review and so

Matt Klein, CEO, PTC Therapeutics: And so I’ll then so it will probably then be in in, you know, close to the same time. And obviously, we’re thinking a lot about the free pricing level. What we’ve done in preparation in Germany where we think there’s about 8,000 patients, we’ve initiated an early access program. So we’ve already gotten a number of patients on drug in this period waiting for approval and launch. And we’ve gotten into a lot of the key centers in Germany.

And that’s really important because in Germany, once you’re approved and launched and list the price of Liotox, those patients on early access right away by law switch to commercial therapy. So we’re doing everything we can to make that launch in Germany as robust as possible as quickly as possible. Great.

Paul Choi, Analyst: Maybe in terms of the insurance preparations and your payer discussions, I’m just curious in terms of like the metrics and how payers will assess that. Clearly, the drug works very well in reducing fee levels. How does the diet liberalization sort of potentially figure in down the road after patients have been on therapy for future renewals and continuous approvals?

Matt Klein, CEO, PTC Therapeutics: Yeah, think our feedback is there will, of course, be some payers for some patients that will want to ensure that patients are having a response to the drug. Our understanding is that’s more going to be linked to Phe reduction and not diet liberalization. Diet liberalization is incredibly important for patient uptake and physician uptake because for patients, really, holy grail is to be able to get to a point where they have control over their phenylalanine levels so that they can start to liberalize what is a debilitating restricted diet. And so we fully expect that payers may, in certain cases, will wanna see evidence of phenylalanine reduction, not necessarily liberalization, but that your the blood test, which is objectively measured, is in line with what we saw in clinical trials. So we’re fully prepared for that.

Paul Choi, Analyst: Great. You’ve also talked about a little bit you you mentioned that you have some early access at at in in Germany. And just in terms of patient identification and potential pent up demand here with the early part of The U. S. Launch, should we think about the early launches potentially having a bit of a bolus just given awareness of Cephein’s in the PKU community?

And just is that how you would sort of guide the street to think about this an early built up demand?

Matt Klein, CEO, PTC Therapeutics: Yeah. There’s cert there certainly is a pent up demand, and and that comes from several things. Right? It comes from the fact that the data have been so strong that we’ve been able to show the diet liberalization. I just look on social media and you see the patient community generating their own buzz about the ability to liberalize their diet in a meaningful way.

And the fact that you now have physicians, again, said we’ve seen a number of these surveys saying that most physicians want to try all their patients on the drug. Now what that group looks like in the launch will differ center by center. We’ve had physicians say, look, we’re going to take our patients who are on existing therapies and just switch them because we know if you’ve been on saproteren, either branded or generic, time and time again, you’re going to have a much better response to Cefiance. That’s what the data suggests. So it’s very easy to take patients who are on a once a day therapy and switch them.

Others say, look, we’ve got a bunch of therapy naive or tried and failed patients tried existing therapies and failed them, who we want to get on a therapy right away. And so I think we will see a lot of patients come on in the phases of the launch. And that’s due to the pent up demand, due to the fact that these are centers that are used to the idea of having to prescribe drugs and the patients are identified, prevalent, and desiring a safe and effective therapy.

Paul Choi, Analyst: Great. I think one question that or understanding of the PKU outlook for you guys is just understanding how much of your IP is dependent on orphan or regulatory exclusivity versus other patent exclusivity. And I think it probably goes out further than people realize. And so can you maybe just remind us on what that IP landscape, both on the regulatory side and the patent side, looks like?

Matt Klein, CEO, PTC Therapeutics: Yes. So we’ll certainly have the orphan exclusivity of seven years, and we expect the pediatric add on for six months. But we also have patent protection. We’re guiding folks to 02/1939. We have 2038 on a very what we believe it will be a very durable and strong polymorph patent that we’ve been able to further support with additional IP.

And as is always the case, we’re continuing to expand the IP landscape to keep putting fences around what we have. So for today, we’re guiding to 39 on the back of patent protection to 38 in what we believe conservatively is one year of patent term extension based on Orange for post double patents.

Paul Choi, Analyst: Right. And one more for the investment community. Is this something they’ll be able to track through standard data sources like IQVIA? Will it be censored? Just how are we going to be able to sort of track the launch metrics?

Matt Klein, CEO, PTC Therapeutics: It’s going come through us. So we’re dealing with a closed specialty pharmacy network, so the prescriptions will prescription data will not be available. But when when we get to launch, when we get to approval in The US, we we will share the metrics that we’ll be sharing with everyone so we could track along and understand what uptake is like and what and what adherence is like.

Paul Choi, Analyst: And maybe just at a high level, what will be the key things we should look for? Number of physicians, new prescribers, switches, what just maybe maybe some round numb or round things that we should look for.

Matt Klein, CEO, PTC Therapeutics: Yeah. I think right now, as we’re thinking about it, it’s gonna be, you know, patients on drug. Right? And that’s something that we can we should follow. And I I look.

I think what we’ve talked a lot about is we expect, given the pent up demand and all the things that we’ve talked about so far, Paul, is that you’re gonna see a we expect a lot of patients to come on early, but we expect it to continue to grow. And we’ve talked a lot about these different patient segments. And again, I keep coming back to what we initially thought and what we’re seeing more and more surveys that are out there that there’s a majority of physicians wanting to try all of their patients. And a lot of that is coming from the physician’s own view of the drug, including recent data we put out on mutation data, which shows that if you look purely on a mutation basis, over seventy percent of the patients in the AFFINITY phase three trial technically have classical PKU. And if you see the level of phenylalanine reduction we’re having there and the fact that we’re having two thirds of the patients get to over thirty percent reduction, seventy five percent of patients get over fifteen percent reduction, it’s very easy to understand how this is a drug that can be a therapy for the majority of patients with PKU, and that’s a very large opportunity.

Paul Choi, Analyst: That’s great. Maybe switching gears to Friedreich’s ataxia. I think the investors are watching the Skyclaris launch and Biogen, which is going reasonably well. But maybe you can highlight for us what are the sort of the key differences mechanistically for vitiquinone versus Skyclaris to start? And then I have some commercial questions after that.

Matt Klein, CEO, PTC Therapeutics: Yeah, I think there’s several important differences between the two therapies. Now mechanistically, they’re different. Sclarus targets the Nrf2 response pathway, which is part of the downstream antioxidant response pathway. We’ve talked about how betuquinone targets 15 lipoxygenase, which is a key regulator of the oxidative stress and cell death pathway that’s been intimately linked to Friedreich’s ataxia pathology. But perhaps from a practical standpoint and a commercial standpoint, what’s more important is the fact that vatiquinone would be a therapy for both children and adults.

There’s a large volume of safety and tolerability data accumulated in pediatric patients with vatiquinone down to less than one year of age. And then of course the MOVE FA trial was really focused in pediatric and young adult patients. And so to be able to now have efficacy data along with safety data will allow patisiran to be a patient a therapy for all patients with Friedreich’s ataxia regardless of age and regardless of disease stage. So as we think towards moving into a commercial landscape, I think, you know, clearly the pediatric populations, we see about six thousand patients with Friedreich’s ataxia in The US, about a third of them are pediatric, you’re looking at the only therapy available from those patients. And then of course if you look to the experience with Glyclaris, which has had a lot of patient starts but also patients who have challenges based on the tolerability profile, the monitoring requirements, we believe that Vedicuanole could also be a very meaningful treatment option for adults.

Paul Choi, Analyst: Okay, great. Maybe parsing the commercial strategy for the two populations between the adult population and the pediatric population. As you think about the sort of the selling message for those two distinct populations, Is it your thinking that you’ll be able to primarily get adult patients who are treatment naive or treatment inexperienced versus SKYYCLARIS experience? Or how sort of what’s the sort of low hanging fruit in your mind?

Matt Klein, CEO, PTC Therapeutics: Yes. So the low hanging fruit for sure is the pediatric population. And the dynamics of the pediatric population differ quite a bit from the adult population. The pediatric FA patients, as we said, is about a of the population. They tend to be clustered at a small number of specialty centers that are pediatric neurology centers.

We’ve worked with all of these centers. They’ve been in our trials. Many of them have extensive experience with the drug. And again, if you read what the physicians are saying in different surveys of them, these doctors are saying they will look to get all of their pediatric and adolescent patients on metiquinone if approved. So that’s really good.

That’s the low hanging fruit. Then you talk about a migration into the adult population, and we see that in two different buckets. One, as you said, is the sort of tried but didn’t stay on Sclicalaris due to monitoring issues, lipid level issues, LFT problems, or other tolerability issues. And we see that as also sort of the bucket of patients that we can easily access. And then I think you have those adults who are therapy or Sclaris naive.

I think we can penetrate as well given what we expect to be the lack of a monitoring requirement, the lack of potentially need to go on a lipid lowering therapy Mhmm. Or having LFT abnormality. So I think that there’s a very large opportunity for the drug. If you have unlike PKU where we believe we can we would look at all of those segments pretty much at the time of launch, I think for Friedreich’s ataxia, we think it’s going to be sort of the kids It’s a significant unmet need. One of the interesting dynamics in the community, as always happens when a when a a drug is approved, is there’s an increase in diagnosis of the disease.

Right? There’s just heightened

Paul Choi, Analyst: disease awareness.

Matt Klein, CEO, PTC Therapeutics: And this has happened in Friedreich’s ataxia with the approval of Sconclaris. However, the patients getting diagnosed are younger. Again, not surprisingly, typically what you’ll see is the age of diagnosis move lower and lower as there’s heightened disease awareness and motivation to get a diagnosis. And so if anything, the approval of Skyclaris has heightened the unmet need in the younger patients, increased the numbers and increased the desire not only of patients but of physicians to be able to have a therapy that they can give to give to younger patients. Because, of course, if you’re trying to slow progression of the disease, it’s best to start as soon as possible.

Okay.

Paul Choi, Analyst: Great. And then maybe just in terms of your latest thinking on pricing, there is a commercial analog out there in terms of Skyclaris. Is that sort of a reasonable benchmark as investors model out this opportunity both in the pediatric and adult segments?

Matt Klein, CEO, PTC Therapeutics: Yes, I think so. Okay.

Paul Choi, Analyst: Okay, great. Maybe one more on vutiquinone, which is just that its regulatory time frame is a little bit behind that of Cefianz. And so just given that we still have several months for that to go, are there any other regulatory interactions that are going to happen along the way beyond perhaps regular way of label potential label discussions? Any other material meetings of note?

Matt Klein, CEO, PTC Therapeutics: Yes. So we so our PDUFA date is August 19, as you indicated, a bit after suppliance. We’ve had the mid cycle meeting. That went well. It was, at the mid cycle meeting that the agency shared with us that they do not expect, to hold an AdCom, so that was an important update.

We still will have a late cycle meeting and then, of course, labeling discussions still to come. Okay,

Paul Choi, Analyst: great. I want to talk and maybe switch gears to your recent PTC518 Huntington’s update. Maybe, Matt, you could just sort of recap for us the latest set of data you presented from that program and just sort of then some follow-up questions on just sort of the development path there.

Matt Klein, CEO, PTC Therapeutics: Yes, absolutely. So I think as we look at the PTC518 data package and the Phase II PIVOT H2 trial, it ticked all the boxes it had to. We achieved the primary endpoint. We demonstrated that the drug is working the way it needs to work with dose dependent continued dose dependent reduction in huntingtin protein levels in the cells. The drug continues to be safe and well tolerated, which is very, very important as we go out to longer terms.

We had continued confirmation of CNS exposure, incredibly important for the drug a drug that’s working the way it works. We know it’s going to where it’s supposed to go. We also saw in the data readout continued evidence in the short term at twelve months of dose dependent effects on stage two patients on CUHDRS, total motor score, and cognitive function very important. And then importantly in this update, we had the group of patients out to two years. And at twenty four months, patients who’ve gotten PTC518, not only is it safe and well tolerated, but we’re seeing dose dependent benefit on the CUHDRS disease rating scale relative to natural history as well as benefit on the total functional capacity and cognitive scales relative to natural history that was statistically significant.

And we saw out a two years dose dependent benefit lowering on neurofilament light chain. The natural history of Huntington’s disease is to see gradual increases, and we’re seeing decreases now out at twenty four months. So that’s really important. We also learned about study populations. And this was one of the objectives of the PIVOT-eighty two study to try to figure out in which group of patients do we think would be the optimal future efficacy trial population.

And what we clearly saw is what we suspected is that likely the stage two patients are those that are moving at the appropriate rate and that they’d be best suited to population in whom we could capture significant effect. So when you take everything as when you take the data package as a whole, we achieved all the objectives for for phase two, which positions us well to move forward with an efficacy study. We believe that the data also can support further discussions on the potential for accelerated approval. I think we’ve long talked about the potential surrogate endpoint of lowering Huntington protein. I think the recent correspondence from Unicure on their conversations with FDA about a potential intermediate clinical endpoint approach using long term CUH DRS versus natural history data and Nf L.

I think we now with this dataset are in play for that option as well. So we think our optionality in terms of what we could talk about supporting accelerated approval has gone up.

Paul Choi, Analyst: Yes. I mean it certainly seems like to your point, bringing out UniCure’s recent disclosures just on the regulatory landscape, still seems supportive of a mix of biomarker and quasi functional or some sort of hybrid endpoint in the space. So I guess in terms of next steps, when would you present any additional detailed data from your recent update at a medical meeting? Are there plans to do that over the course of 2025? And then sort of when would you and your partner Novartis potentially have regulatory interactions on a Phase III design?

Matt Klein, CEO, PTC Therapeutics: So the teams are working on that right now on the interaction standpoint. I think what we’ve said all along, even before the readout, was that we’d be doing these things in parallel, moving forward with the Phase III trial and having discussions about accelerated approval. And the plan would be to have an FDA meeting where both things would be discussed, of an end of Phase II meeting that could talk about the data package that would be needed to support accelerated approval, knowing that we’re continuing to harvest data from the open label extension of PIVOT HD, and then, of course, what does that efficacy trial look like and trying to move as quickly as possible on both fronts. So the teams are actively working on that right now. I think we also all have in mind as well that the significant opportunity in HD is in stage two patients and earlier, right?

And also trying to think about how do we get into stage one patients and then even earlier stage zero patients where there’s and and the undiagnosed patients, which is that that population is multiples of the of the identified patient now. So there’s a lot of thought going into how we migrate earlier and earlier, which is what the patients want and what the physicians want. In terms of presentations and medical meetings, I think the teams are still looking at that. There’s some HD meetings in the fall. I think that would be a good opportunity to give updates.

Paul Choi, Analyst: Okay. Great. Maybe just on your last point, Matt, of finding and identifying or potentially including earlier stage patients, how are these patients typically identified versus like the more symptomatic stage II and stage III patients that were in PIVOT HD? I’m sure most doctors don’t typically scan for CAG repeats, but just kinda how are these patients potentially identified, and how could this expand the pool for you?

Matt Klein, CEO, PTC Therapeutics: Yeah. So if you think about Huntington’s disease is is pretty unique. Right? A hundred percent of the patients have a genetic defect, And it’s also uniquely autosomal dominant, which means that if your parent has it, you have a fifty percent chance of passing that on to your children. So there is then a lot of work in the community to get genetically diagnosed before you start having symptoms.

Of course, that has a downside if there’s no therapy available, then you’ve just basically found out that you have a fatal disease that will have an onset at a certain amount of time. So a lot of this is going to be walking hand in hand with having a therapy available. As we think about stage one patients, which there’s a large pool of as well, again, when we talk about a clinical trial, it’s very hard to have an efficacy trial of patients who are not progressing a lot clinically. But that’s a group that we could think about a biomarker strategy or something to be able to get into to be able to show that we’re slowing progression. I think when you think about Huntington lowering and the potential of Huntington protein lowering, it really is one of these things where the sooner you can get to patients and forestall the onset of clinical symptoms, the better.

That’s why I think really the the that large population is is a holy grail, if you will. Course we want to provide benefit to the prevalent patients who are rapidly progressing in stage two and stage three, but there’s also a very large opportunity of earlier stage patients.

Pierre Grave, CFO, PTC Therapeutics: And that’s why the partnership with Novartis is highly valuable because they canit will require a lot of work to unlock the full spectrum of the opportunity Mhmm. And a lot of investments. Nobody’s you know, I can think of a billion reason why they wanna unlock the full patient population. Once you get a drug approved, of course, this is where diagnosis rate you know, you should expect that to go up because right now, even if you know your dad or mom has or add HD because it’s fake also, you know, you don’t wanna know. Most patients don’t wanna know.

They wanna have a laugh. Why would you wanna know if there’s no cure? If at some point you can slow down the progress, we will expect this to increase. And this is where, again, Novartis is truly committed. They are designed to do this.

Right? Large pharma, bone crushing machine, they you know, this is their their DNA.

Paul Choi, Analyst: One last one on PTC518, is that Unicure, I think, is probably in a position to file their application early next year, so they’ll be to market. How do you think, I guess, about the commercial market post their approval and your approval evolving? Will the market be segmented out depending on it? Their drug is obviously involved not a trivial procedure, right, and so And just how do you think about that?

Matt Klein, CEO, PTC Therapeutics: Yes. I I I’m not sure we’re very worried or the Novartis team would be very worried about that, about being to market. As you as you pointed out, it’s a very large ten, fifteen hour, I’ve heard sixteen hour surgical procedure. Right? It’s clearly that that alone is gonna slow uptake.

Right? There’s only a certain amount of centers and surgeons who could do that. You’re asking a neurosurgeon to take their entire or his entire day to do that procedure. And probably there’s an opportunity cost that they’re gonna be very aware of and the centers are gonna be very aware of. You’re also using up sixteen hours of MRI time, so there’s a huge opportunity cost for a center in terms of being able to do, you know, 10 or 15 different scans versus just one patient all day.

So that’s gonna play into it. And then the other part is is also the the patients. Right? Is I think most people realize there’s probably a finite durability of a gene therapy. So this idea of getting into the larger patient buckets that may be earlier stage with minimal symptomology or no symptomology, that’s a large part of this market that is unlikely to be one served by the gene therapy because, of by the time they get to the stage of disease where they’re going to be fulminantly symptomatic and needing a therapy, the gene therapy pump may not be working anymore.

Paul Choi, Analyst: Great. We are almost out of time, so I want to ask maybe Pierre one question just on the balance sheet, which is one of the largest cash balance sheets among the mid cap biotechs here in The U. S, a little bit of debt as well. But just I guess as you think about your uses of cash here on the forward, how are you sort of rank ordering internal development versus maybe looking externally?

Pierre Grave, CFO, PTC Therapeutics: Yeah. So we’re in a very strong position, as you mentioned, to be enough cash. And, you know, this gives us a number of you know, quite frankly, we have the flexibility to do everything and not look over our shoulder and try to raise capital, especially in this tough environment. So this gets us to all our operation, the launch of PKU, of Atiquinone, Transplant U. S.

And our own pipeline. And the extra cash will be used for BD activities, for instance. We’re actually looking at a number of opportunities. We have to turn our own cars over the summer. And as we mentioned, if you zoom out, the goal is to get to 2,000,000,000 top line.

We believe PKU would get us to $1,000,000,000 The U. S. Alone, let’s say half of that ex U. S. And then FA or Transana US will be more than enough to get us there.

And we’ll accelerate all of that with BD, and we have the global infrastructure and the know how to push products to the finish line. So that’s how we’re thinking about our capital. Okay.

Paul Choi, Analyst: Great. We’re out of time now. So my my thanks to Matt and Pierre for joining us, and we’ll end the session on that note. Thank you very much. Thanks, Paul.

Thank you.

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