PTC Therapeutics, Inc. (PTCT)
NASDAQ: PTCT · Real-Time Price · USD
70.26
+1.04 (1.50%)
At close: Apr 28, 2026, 4:00 PM EDT
71.20
+0.94 (1.34%)
After-hours: Apr 28, 2026, 7:54 PM EDT
← View all transcripts

43rd Annual J.P. Morgan Healthcare Conference 2025

Jan 13, 2025

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Okay, thank you. Good morning, everybody. I'm Eric Joseph, Senior Biotech Analyst with J.P. Morgan. Our next presenting company is PTC Therapeutics, and CEO Matt Klein is going to be talking us through the story. There's a Q&A after the presentation. Just raise your hand, and we'll get a mic over to you. And for folks that are tuning in via the webcast, feel free to submit a question via the portal, and yeah, we'll pick them up. With that, Matt, thanks for joining us.

Matthew Klein
CEO, PTC Therapeutics

Thank you, Eric, and good morning. Last year, I stood on this stage and talked about how PTC had made a number of significant changes and shared an ambitious 2024 plan for building the PTC of the future. Now, I realize a lot of people didn't believe that we could achieve all that we said we would and so quickly transform the company, but we did, and I can proudly report that the future is now. This morning, I'll review a number of our 2024 accomplishments and share our plans for 2025 and beyond. Before I get started, I refer you to our forward-looking statement slide, as well as to our SEC filings for a description of risks and uncertainties. 2024 was a year of outstanding execution across every part of the company.

We achieved all clinical and regulatory milestones on time, including submitting four approval applications to FDA, setting us up for the possibility of having four U.S. commercial launches within 12 months. We had another year of outstanding revenue performance exceeding guidance, which, along with our effective OpEx management, gives us line of sight to being cash flow break-even in the near future. We built a strong cash position, $2 billion pro forma, to adequately fund our planned commercial and R&D activities and allow us to engage in meaningful business development to accelerate revenue growth, and through our efforts to focus our R&D pipeline on our highly differentiated scientific platforms, including splicing, we're preparing the next wave of innovative PTC products. Let me dig deeper into these 2024 accomplishments.

We submitted four approval applications to the FDA: one for our AADC gene therapy, sepiapterin for PKU, Translarna for nonsense mutation DMD, and vatiquinone for Friedreich's ataxia. Our AADC gene therapy, Kebbilidi, was approved in November and became the first-ever direct-to-brain administered gene therapy approved by FDA. The sepiapterin and Translarna files were accepted for review, and those reviews are ongoing. And for Friedreich's ataxia, we expect to hear from the FDA in February about the filing decision. 2024 was another year of outstanding revenue performance, with total unaudited revenue of $814 million, exceeding our revenue guidance. This revenue performance was driven by our inline products, including our DMD franchise, in spite of significant headwinds for both Translarna and Emflaza. This revenue performance is a testament to our commercial team's ability to effectively execute around the globe, even in genericized and competitive markets.

With this revenue performance, effective OpEx management, our rapid and robust monetization of the priority review voucher received as part of the Kebbilidi approval, we closed 2024 with over $1.1 billion in cash. This strong cash position enables several important things. It allows us to reach cash flow break-even without additional capital. It also supports all of our planned commercial activities, including our commercial launches, and allows us to invest in our R&D programs, and it gives us capital to participate in business development activities that can complement both our commercial and R&D portfolios. In addition, when we consider the uncertainties in 2025 on a more macro level, having this capital allows PTC to control its own destiny. In addition, in 2024, we continued to build out our highly differentiated research platforms, including splicing and inflammation and ferroptosis.

We now have a number of preclinical programs that will look to advance towards the clinic in 2025. In addition to being a source of PTC-developed and commercialized therapies, we plan to leverage these platforms as a source of strategic partnerships for non-core therapeutic areas. I'm incredibly proud of all that we accomplished in 2024, and we're just getting started. In 2025, we expect another year of strong execution and success. We have a number of potential value-creating milestones expected, including the global launch of sepiapterin, the data readout from the PIVOT-HD study of PTC518, and a number of regulatory decisions both in and outside of the U.S. We're providing a wide initial revenue guidance for 2025 of $600 million-$800 million, given the number of pending regulatory actions that could impact revenue.

For OpEx, we're providing guidance of $730-$760 million, including expenses associated with the ramp-up of commercial material in preparation for our sepiapterin launch, as well as costs associated with ongoing clinical studies. I'll now share our 2025 plans for our key programs, starting with our sepiapterin PKU program. We're preparing for the global launch of sepiapterin in 2025, and our teams and the PKU community couldn't be more excited for this launch. As we've discussed, despite there being two approved therapies for PKU, there remains a significant unmet need for PKU patients, as the vast majority of patients are not well served by available therapies. Sepiapterin has demonstrated the potential to address this unmet need. Sepiapterin has a dual mechanism of action. First, as a bioavailable and highly potent cofactor that can provide greater lowering of phenylalanine than administering BH4 itself.

The second mechanism of action is as a chaperone that allows for treatment benefit in patients who are considered non-responsive to BH4 therapy. This potential for benefit across the full spectrum of PKU patients has been confirmed in our clinical studies. In our phase 3 APHINITY trial, we demonstrated highly statistically significant and clinically meaningful benefit across the full spectrum of PKU patients, including classical PKU patients, those with the most severe form of disease. Over 80% of patients reach targeted levels of phenylalanine of less than 360 micromolar per liter, and 22% of patients achieve normalization of phenylalanine levels, something unheard of in clinical trials. In our food tolerance protocol, as part of the ongoing APHINITY extension study, we continue to demonstrate that patients are able to liberalize their diets and have greater protein intake than would be recommended for someone without PKU while still maintaining control of phenylalanine.

These findings of feed tolerance benefit are incredibly meaningful to patients. While these clinical data speak volumes about the potential of sepiapterin to meet the unmet need of the PKU community, the words of physicians and patients themselves strongly substantiate this potential. We've heard from a number of physicians, such as Dr. Ania Muntau, how they're willing and ready to trial all of their patients, including those on current therapies, on sepiapterin once it's available. The potential of sepiapterin to provide meaningful benefit to classical PKU patients, those most severe patients, was recently highlighted by Dr. Harding in a commentary that accompanied the publication of the phase 3 APHINITY results in The Lancet. Perhaps most meaningful are the words of patients themselves.

Time and time again, we see anecdotes on social media from patients about being able to liberalize their diets and enjoy meat, fish, pizza, and other foods that they were never able to enjoy before while still maintaining Phe control. From a commercial standpoint, the clinical data support the potential for sepiapterin to address all key PKU population segments, including patients who have failed current therapies, patients who are not well controlled or tolerating current therapies, and therapy-naive patients, including those with classical PKU. When one considers the size of the PKU population, the strength of our clinical data, the experience of our commercial teams, and our ability to price sepiapterin at a premium to Palynziq, we believe the revenue opportunity for sepiapterin exceeds $1 billion, and we believe we could reach that level in the U.S. alone.

Our customer-facing teams are in place, and launch plans are well underway. We've mapped centers of excellence and key opinion leaders. We're actively engaged with PKU centers, including nurse practitioners and dieticians, who play an important role in patient care, diet management, and prescribing decisions. We've had ongoing discussions with payers, and we remain close to the patient community, which is well aggregated and working with us to ensure that we can meet the needs of patients as sepiapterin becomes available. Turning to the vatiquinone program for Friedreich's ataxia, in December, we submitted the NDA for vatiquinone for the treatment of children and adults with Friedreich's ataxia. If approved, vatiquinone would be the first therapy for pediatric FA patients. The NDA includes evidence of efficacy from the placebo-controlled MOVE-FA study, as well as the results of two long-term studies that demonstrate benefit in slowing the long-term progression of disease.

We expect to hear from FDA about the filing acceptance in February, and if priority review is granted, we could have an approval decision by August of 2025. Here are some of the data included in the NDA supporting vatiquinone efficacy. In the 72-week placebo-controlled MOVE-FA study, statistically significant benefit was demonstrated on the upright stability scale, the most relevant and meaningful measure of benefit for the pediatric and young adult patients enrolled in the study. This slowing of decline on upright stability translates into a meaningful delay in time to loss of ambulation. In addition, the benefits on upright stability were supported by significant findings of benefit on the physical component of the Modified Fatigue Scale , as well as the evidence of benefit on the one-minute walk distance. In addition, the NDA includes results from two long-term studies, both of which met their pre-specified primary endpoints.

The first study includes data from the long-term extension of the MOVE-FA study, in which patients treated with vatiquinone for three years were compared with a matched natural history population from the robust and well-respected FA natural history database. In this analysis, following three years of vatiquinone treatment, there was a 50% delay in disease progression, a result that was not only highly statistically significant but clearly clinically meaningful. In the second study, we analyzed data collected from a study years earlier in adults, both ambulatory and non-ambulatory, with Friedreich's ataxia. In this analysis, following 24 months of treatment, there was a 4.8-point benefit on the MFARS scale, signifying again a significant and highly important delay in disease progression.

When we take the placebo-controlled results and the long-term study results together, we clearly see that vatiquinone provides meaningful short and long-term impact on disease progression for patients of all ages and disease stages. Despite the approval of Skyclarys, there remains a significant commercial opportunity for vatiquinone. In the United States, there are approximately 6,000 patients, about a third of whom are pediatric and for whom there's no approved therapy. In addition, there's a large number of adults in the U.S. and outside of the U.S. who are not currently on Skyclarys treatment for any number of reasons. When one considers the safety and efficacy data of vatiquinone, particularly in children, it's clear that vatiquinone could meet the unmet need of the pediatric population and become the treatment for patients under the age of 16 and provide a safe, effective, and well-tolerated alternative therapy for adult FA patients.

Our teams have begun launch preparations, and we continue to work closely with the FA patient community, with whom we've enjoyed a long-term relationship. We're again working closely with the community to be ready to meet all the needs of patients as we move closer to a potential launch. Turning to our PTC518-HD program, in the second quarter of this year, we plan to provide the complete 12-month results from the PIVOT-HD study. PTC518 comes from our splicing platform and follows the successful discovery and development for Evrisdi, which is now the largest global SMA therapy. PTC518 is a highly differentiated huntingtin lowering therapy. It's orally bioavailable, highly selective and specific for its HTT target, achieves excellent and broad CNS exposure, and allows for uniform huntingtin lowering in every region of the brain.

In addition, in our clinical studies, PTC518 has been shown to be safe and well tolerated without evidence of NfL spikes. It's this unique combination of characteristics that makes PTC518 one of the most promising, if not the most promising, disease-modifying therapy in development for Huntington's disease. In June of last year, we shared the interim results from the PIVOT-HD study on the first approximately 30 patients who completed 12 months, and this data readout met all key objectives. We demonstrated dose-dependent and durable lowering of mutant huntingtin protein in blood and demonstrated dose-dependent lowering of CSF mutant huntingtin protein levels in line with what was recorded peripherally. In addition, we demonstrated dose-dependent benefit on several clinical scales, including the Total Motor Scale and the cUHDRS, and again, importantly, PTC518 was shown to be safe and well tolerated.

In the second quarter of this year, we plan to share the results from all patients at the 12-month time point. These results will include safety and tolerability data, biomarker data, including huntingtin protein and other biomarkers, as well as data on clinical scales, and include both stage two and stage three patients. Based on our discussions with FDA, the results of this study could support huntingtin lowering as a surrogate endpoint for accelerated approval. In December, we had a type C meeting with FDA, and there was alignment with the agency on the potential of huntingtin lowering to be a surrogate endpoint, including the scientific rationale, and FDA asked that we provide additional data, such as those we're going to be reporting out from the PIVOT-HD study, that show a link between changes in huntingtin lowering and clinical scales.

Late last year, we announced our new collaboration with Novartis for the development and commercialization of PTC518, and I'm proud to report, as of this past Saturday, that deal has officially closed. As part of the agreement, Novartis will assume all development and manufacturing and commercialization of PTC518 following the completion of the placebo-controlled portion of the PIVOT-HD study in the first half of this year, and per the agreement, PTC will receive $1 billion upfront, up to $1.9 billion in development, regulatory, and sales milestones. PTC will have a 40% U.S. profit share, double-digit tiered royalties on ex-U.S. sales, and Novartis will fund all future development activities, including the phase three trial. PTC is proud to have pioneered the field of splicing therapies, and we're well positioned to continue to lead it.

The teams responsible for the successful discovery and development of SMA and HD programs have only gotten smarter. We've made a number of key learnings that have expanded the set of potential druggable splicing targets, and we've streamlined key processes that are part of the discovery plan. One such advancement is PTSeq. PTSeq is a proprietary screening engine that allows for rapid and reliable identification of potential hits for specific splicing targets. PTSeq has significantly accelerated elements of our preclinical timelines. We now have a number of active splicing programs, both CNS and non-CNS indications, that will look to bring forward closer to the clinic in 2025. We've also made significant progress on our inflammation and ferroptosis platform. This platform focuses on targets that are key to the inflammation and oxidative stress pathways known to underpin a number of different diseases.

We have several active programs targeting both CNS and non-CNS indications that we'll work on in 2025, including a phase 2-ready DHODH inhibitor, which we'll be developing for neuroinflammatory conditions. We have an NLRP3 program that we're going to be moving towards IND-enabling studies in 2025, as well as preclinical programs targeting alpha-synuclein and NRF2 activation that we'll look to bring forward in 2025. We are proud to have built PTC into a strong, innovative, and well-capitalized company. As we look to 2025 and beyond, we have line of sight to being cash flow breakeven, and we've set as an objective having a path to reaching $2 billion of top-line revenue, which we could reach with our current programs pending 2025 regulatory decisions. In addition, with the PTC518 collaboration agreement with Novartis, we are well positioned to enjoy potential significant financial upside based on PTC518 commercial success.

From an R&D standpoint, we have two highly differentiated and validated scientific platforms that can be a continuous source of innovative therapies for PTC and possible strategic partners. And as we build the company forward, we'll continue to use business development to accelerate revenue growth to that $2 billion mark and beyond. In conclusion, we entered 2024 with an ambitious agenda to position PTC for future success. With the many outstanding achievements of 2024 and our demonstrated ability to effectively execute across every part of the business, I can confidently state that the future is bright and the future is now. Thank you.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

We have some time for questions, and so if you have any, just raise your hand and we'll get a mic over to you.

But just picking up on sepiapterin and PKU with the approval and approaching approval decision and approaching launch, you commented on your pricing expectation here, anticipating payer support for a price point, a premium to Palynziq. Can you just talk a little bit about sort of just the feedback from payers, a little bit about sort of the value proposition that you're taking to payers that gives you confidence in being able to support that premium price point that you anticipate?

Matthew Klein
CEO, PTC Therapeutics

Yeah, I think one of the things that's really important, Eric, with PKU is the clear understanding that the name of the game is lowering phenylalanine levels and doing so in a way that's safe and well tolerated for patients.

And so when we can show payers the data that we have showing incredibly strong phenylalanine levels across the full spectrum of patients, as well as our Phe tolerance data, there's no question that we're able to really achieve superior results in terms of Phe lowering. And then when you also consider the tolerability profile of sepiapterin and its suitability for both pediatric and adult patients, it's clearly a highly differentiated therapy that, based on its safety and efficacy profile, would support pricing at a premium to existing therapies, including Palynziq.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

And once in the market, I guess how to think about where new starts on drug will sort of originate? Is there sort of a low-hanging fruit opportunity that you expect to target first?

Matthew Klein
CEO, PTC Therapeutics

It's a great question, and I smile a bit because the short answer is yes.

The longer answer is that low-hanging fruit varies depending on which physician you talk to. We've had a number of physicians, such as Dr. Muntau, who is one of the leading global KOLs for PKU, who said, "Look, I'm going to try all my patients on sepiapterin." Based on the mechanism and the data we have, those patients who, for example, are well served on Kuvan, if you're getting a benefit on Kuvan, again, based on mechanism and our data, you're going to have a much more superior result with sepiapterin. So for some docs, the low-hanging fruit's switching existing patients towards a potentially more efficacious and tolerable therapy. Others, of course, are keen to try those more severe patients, classical PKU patients, who have not been served by existing therapies, given how significant their high levels of phenylalanine are.

And then there's patients who fail current therapies who have documented Kuvan failures or generic BH4 failures who then could be easily transitioned to sepiapterin, where rapid exposure and blood tests can show superiority in terms of lowering phenylalanine, which could support reimbursement. So the short answer is there's a lot of sources of low-hanging fruit. I think the important point here is that our data support our ability to address all patient segments, and it may differ based on physician to physician in terms of which patients they target first, but our teams are ready, and look, we're looking to get to any patient we can possibly help, which is obviously, we believe, a large portion of the population.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Question over here.

Speaker 3

Yeah, I just wanted to ask about your thoughts on the accelerated approval using HTT as a surrogate endpoint and how you're thinking about that relative to the FDA's views on, for example, uniQure's therapy. Thank you.

Matthew Klein
CEO, PTC Therapeutics

Yeah, I think the important point here is the FDA has shown a willingness to leverage the accelerated approval pathway for neurodegenerative diseases for what I think are obvious reasons. And when we think about Huntington's disease, we're in a unique situation where we actually know what causes the disease in all patients. It's a mutation in the Huntington gene. And so when we go to have discussions with the FDA about what would be a reasonable surrogate endpoint, there's certainly a wealth of scientific literature that supports that lowering huntingtin protein has benefit.

This has been shown in preclinical models and also very nicely shown in an epidemiologic study in patients who make less huntingtin protein and have a delayed disease onset, so in our discussions with FDA, there was clear alignment on the scientific rationale. A simple ask was for us to provide some more data from the large readout we're going to have in a few months that shows that as huntingtin is moving, there's some relationship with some of the clinical scores, so I think there's some misconception sometimes that there's a B path for accelerated approval for Huntington's disease, and I think from a regulatory standpoint, it's a path, a path in which you can provide the necessary evidence to support an endpoint to serve as a surrogate for accelerated approval.

And the conversations we had with the agency were certainly encouraging that huntingtin lowering could be one of those endpoints.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

I guess in the analyses you're proposing to the agency that would help support the relationship between mutant huntingtin lowering and clinical scales, I guess, are there certain particular clinical scales that the agency wants you to focus on, demonstrate improvement, and relate those to mutant huntingtin lowering? Just maybe a little bit of a sense of the analyses that you're going in with leading up to the additional 12-month readout later this or this half.

Matthew Klein
CEO, PTC Therapeutics

So the agency was not prescriptive in terms of the specific analyses, but we know that the kinds of clinical measures we're using in PIVOT-HD are certainly appropriate to do that.

In the written feedback, they used words like associations, just showed that things were associated, that there's movement in one that predicts movement in the other. So again, there was not a prescriptive analysis plan that was needed. It was more looking at the data holistically and saying, "Can we show some relationships between what we're observing in HTT lowering and changes in clinical scales?" Which makes sense, right? The idea of accelerated approval is likely to predict clinical benefit, not definitive evidence of clinical benefit with statistically significant clinical scales, but show that there's a relationship here that supports that as we're changing or as we're lowering huntingtin protein, we can believe that there'll ultimately, in the long term, be a clinically meaningful benefit to patients.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Okay. You had the opportunity to look at mutant huntingtin lowering beyond 12 months in PIVOT-HD trial.

You'll have some cohort of patients be able to. So I guess there's two kind of buckets of longitudinal follow-up that you'll get, right?

Matthew Klein
CEO, PTC Therapeutics

Exactly right, right. So in PIVOT-HD, it was placebo-controlled, placebo 5 milligram, 10 milligram. All patients are eligible to continue on. The placebo patients will get randomized to 5 or 10, and we'll continue to harvest data over time. And as you allude to, I will continue to flesh out that relationship between Huntington changes and longer-term clinical effects.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

So just coming back to PKU, oh boy. I guess, sorry, you're not alone in this space, right? Obviously, there's a well-known competitor that we alluded to earlier that also plans to sort of seize a growth opportunity through label expansion initiatives and so forth. I mean, how receptive are clinicians today to potentially seeing Palynziq move into the younger adolescent population?

Does that kind of present as a risk factor to your launch?

Matthew Klein
CEO, PTC Therapeutics

So we don't talk much with physicians about that, and so I can't speak about their views on Palynziq. We understand the benefits of a well-tolerated oral therapy that's now been well-studied in pediatric and adolescent patients. We believe having a safe and highly effective therapy will be something that is and is something that's quite welcome by the PKU community, not only in pediatric patients all the way down to newborns, but in adolescent patients, and I'd also say in adult patients.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

And where would you say sort of sepiapterin and awareness is across the PKU treating community?

Matthew Klein
CEO, PTC Therapeutics

I think there's a great deal of awareness.

Look, I think one of the things here that's exciting is we're moving into a, as you said, it's a competitive landscape, but at PTC, we're used to pioneering fields, and this is one where we're coming to where all the essential pillars for success for a commercial launch are in place, right? There's newborn screening, centers of excellence. Their payers understand what it takes to call a therapy effective, and it's a well-aggregated patient community. And we've got incredibly experienced commercial teams that have demonstrated the ability to execute in every possible scenario in every corner of the planet. And that includes having very close relationships with both the patients and physician communities, which we have enjoyed. In fact, we often say commercialization starts in development.

In designing the APHINITY placebo-controlled study, we made sure we had a broad reach to centers around the world so we could start building those relationships with physicians and patient communities globally so that when we come to this day where not only do we have launch preparations well underway in the U.S., but outside of the U.S. and in Europe, where we expect to see CHMP opinion in the second quarter, there's a great deal of awareness and, in fact, a great deal of market pull from the patients and the physicians who really desperately need a safe and effective therapy that can lower phenylalanine and allow for diet liberalization, which is what we've been able to show we can do with our data.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

And maybe just I think we have time for one or two more questions.

One on vatiquinone and Friedreich's ataxia, just the scope of the approval that is kind of on the table with the recent filing. I believe it would support accelerated approval, if I have that correct, I guess. And if that's the case, how to think about what a confirmatory study would look like with that agent.

Matthew Klein
CEO, PTC Therapeutics

Yeah. So based on our discussions with the agency, this is an application for full approval. And that's based on the feedback we've gotten from FDA that they see upright stability now as an efficacy endpoint. I think one of the concepts that's become clearer over the time of us doing the study and since we completed the study is that when you look at that disease rating scale, different parts have different relevance based on the age of the patients and the stage of the patients.

And in fact, it was an FDA-funded study that confirmed that if you're going to look to do a study in pediatric and young adult patients, it's the upright stability scale, which is the preferred measurement of clinical efficacy. And since FDA believes that upright stability itself is a clinical efficacy measurement, it therefore was suggested that we pursue a full approval. And then, of course, we have the confirmatory evidence from the two long-term studies that includes adult patients as well, which allows us to have a data package that supports safety and efficacy in the unmet pediatric population, but also evidence of safety and benefit in the full spectrum, full age spectrum, and disease severity spectrum.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

With potentially two product launches in the second half, I guess how to think about the sizing of the organization on the sales side, MSL sizing and so forth to support both launches? Obviously, you have a presence currently in DMD. I guess how much expansion is needed to support these two upcoming launches? Yeah.

Matthew Klein
CEO, PTC Therapeutics

We're ready for four potential launches in 12 months, right? We have the Kebbilidi launch, the sepiapterin launch, the vatiquinone launch, and the potential for a Translarna launch in the U.S. And our teams are ready and couldn't be more excited.

Look, in terms of commercial infrastructure, there's the core elements, market access, patient engagement, even some of the other work that we do in terms of patient care services, which is an incredibly, I'd say, secret weapon we have in our success with Emflaza so far, which will be incredibly important to getting patients on drug and having adherence for any of those launches. They're in place. And so there will be incremental scaling that's needed in terms of the sales force and MSL support forces. But the key components are there and scale quite easily. So there'll be just some necessary additions to, again, the customer-facing teams in terms of sales and MSL support. But we look forward to that opportunity of having the four launches in 12 months. And look, our teams are ready.

I think if you look at the over $200 million of revenue in Emflaza last year, I think that's a testament to our team's ability to execute in a competitive market, in a genericized market, along with significant headwinds. So we look forward to having this opportunity to bring all these therapies to patients.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Okay. Well, great. I can clearly sense that we're between this presentation and lunch, so I think we'll leave it there for time. Thanks everybody for joining the session. Thanks for the presentation, Matt.

Speaker 4

Like between six inches and a foot.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Okay. Can you hear me here? This is good, right?

Speaker 4

You can stand back a little bit.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

A little bit further back?

Speaker 4

All the way back and get a hold.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Yeah. Okay. This is great. I can see the whole slide too, which is actually fantastic.

Speaker 4

I have my notes here if you need those.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Yeah, yeah, yeah. It's just like a few little things here to remind myself.

Speaker 4

Yeah, that's awesome.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Yeah. Yeah.

Speaker 4

Oh, nice. Okay. This is good. Yeah.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Okay. This is great. And then where, oh yeah, sorry. Let's do that.

So I'm going to talk about this slide, talk about this slide.

Speaker 4

It should play automatically.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Oh, good.

Speaker 4

Yeah. Hey, it looks pretty good.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

I actually thought that I had to push another button for this, so I just push it once.

Speaker 4

I meant to tell you that when I tested it earlier.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Yeah. Oh, okay.

Speaker 5

That makes it so much easier that you, right.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Yeah.

Speaker 5

And then don't go to the next one because that's the appendix, I think.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Oh. Can we retitle it just in case and just say thank you on it instead of appendix?

Speaker 4

Sure.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Can we just do that? Just in case I click it?

Speaker 4

Yeah.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

I'll try to leave it up though, Tracy, because I think that's a great summary slide.

Speaker 4

Because I have to push another volume, so you have to go downstairs.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

This would be great. Don't worry about it. I won't do it then. It's fine. If you go there, next slide is appendix, but I'm just going to keep it on this because this is a great ending slide, right? Yeah.

Speaker 5

Can you go back to the video one more time? I want to just make sure it plays all the way through and it looks okay.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Sorry.

Speaker 4

All right. It's good. You have to go one more back and then go forward again. There you go.

Speaker 6

See, that looks really good.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

That's just all it's real.

Speaker 6

I know, right?

Speaker 4

Yeah. And then it goes, yeah.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Perfect. Right.

Speaker 4

Okay.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

I'll probably still be talking a little bit as it's going, so I'll wait and I'll just pause and then, yeah. Then I go to there.

Speaker 5

And the webcast is just automatic?

Speaker 7

Yes. Yeah.

Matthew Klein
CEO, PTC Therapeutics

Yeah. Basically, we will start right on time at 5:15 P.M. Brian should be here. Brian's really good.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Is Brian going to sit here or is he going to sit in the front?

Matthew Klein
CEO, PTC Therapeutics

No, he's sitting here. Usually, he does sit up here.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

That's fine.

Matthew Klein
CEO, PTC Therapeutics

In the past year that I've worked with him, he does sit up here. You're going to have your clocks here, your clocks there, 20-minute segments for a total of 40. I will change the clock. It'll run down from 20 to 0 and then, this is roughly running around 18 when I'm doing it, so it should be okay.

So what I'll tell Brian to do is just do questions with you initially until I can switch the timer back to 20 again so I can not come back to Mike.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Yeah. Yeah. Perfect. And I'm just going to stand here, right? Like if Brian's sitting there, I'll just stand here.

Matthew Klein
CEO, PTC Therapeutics

You can interrupt and sit if you'd like. Those mics work just fine too. So it's really up to your comfort level and what you want to do.

Speaker 5

I would stand. I think it's,

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

I think it's better. Yeah. It looks better.

Speaker 5

Yeah. It looks a lot better. Yeah. Yeah.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Yeah. Yeah. Okay. That's great. This is actually very smooth and very well done, guys. So that's good.

Speaker 4

I feel like we've done this before.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

No, it's not your first rodeo, I know, so. But I wasn't sure, so.

Speaker 5

Yeah. That's great.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

That's a good setup.

Speaker 4

It's always better to test.

Speaker 5

Oh, absolutely.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Always. Yeah.

Speaker 5

Good. Okay. And if you can go down and change that. So if we change that slide downstairs.

Speaker 7

Yeah. He's going to push a new volume up to me here and it'll be volume two.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

And if I click it accidentally and it goes to appendix, it doesn't matter. I'll just go and I'll flick it back to the main thing. So don't worry about it. Yeah. That's not going to be the end of the world.

Matthew Klein
CEO, PTC Therapeutics

I wouldn't worry about it.

Speaker 4

You can see a preview right there too.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Yeah. And I'm not going to. Yeah. Honestly, that's nothing to worry about, I think. Yeah.

Matthew Klein
CEO, PTC Therapeutics

Thank you. And I didn't get your name.

Speaker 5

Thank you. I'm Sophie.

Eric Joseph
Senior Biotech Analyst, J.P. Morgan

Hi, Sophie. Oh.

Matthew Klein
CEO, PTC Therapeutics

Thank you. My daughter Sophia.

Powered by