PTC Therapeutics at William Blair Conference: Strategic Growth and Innovation

On Tuesday, 03 June 2025, PTC Therapeutics (NASDAQ:PTCT) presented at the 45th Annual William Blair Growth Stock Conference. The company highlighted its strong financial performance in 2024 and outlined its strategic vision for 2025 and beyond. While PTC has achieved significant regulatory milestones and robust revenue growth, challenges remain in maintaining market momentum and reaching ambitious future targets.

Key Takeaways

• PTC Therapeutics reported $807 million in revenue for 2024, surpassing guidance.
• The company’s cash position stands at over $2 billion as of Q1 2025.
• PTC aims to launch SUFIANCE for PKU with a market potential exceeding $1 billion in the US.
• Strategic partnership with Novartis for PTC518 includes a $1 billion upfront payment.
• The company targets $2 billion in top-line revenue and anticipates cash flow breakeven soon.

Financial Results

PTC Therapeutics reported strong financial results for 2024, with revenue reaching $807 million, exceeding the company’s guidance. As of the end of Q1 2025, PTC holds over $2 billion in cash, providing a solid foundation for future investments and growth. The company expects to achieve cash flow breakeven in the near future, driven by effective operational management and strategic partnerships.

Operational Updates

PTC has made significant progress in regulatory milestones, with four FDA approval applications submitted in 2024. The company’s gene therapy, Cability, was approved in November 2024. The New Drug Application (NDA) for SUFIANCE, targeting phenylketonuria (PKU), has been accepted with a PDUFA date of July 29. Additionally, the NDA for Vatiquinone, aimed at treating Friedreich’s ataxia, is under priority review, with a PDUFA date of August 19.

Future Outlook

Looking ahead, PTC Therapeutics is focused on launching SUFIANCE for PKU in the US, with a significant market opportunity. The company is also advancing Vatiquinone for Friedreich’s ataxia and PTC518 for Huntington’s disease, supported by a collaboration with Novartis. PTC’s research and development efforts are centered on splicing, ferroptosis, and inflammation platforms, with strategic partnerships to expand its therapeutic reach.

Q&A Highlights

The conference call addressed the commercialization strategy for SUFIANCE, emphasizing the importance of newborn screening and payer engagement. PTC’s experienced commercial team is prepared to manage step edits efficiently. Despite the non-renewal of Translarna’s authorization in Europe, PTC expects to maintain 25% to 35% of European revenue through alternative mechanisms.

For a detailed understanding, readers are encouraged to refer to the full transcript provided below.

Full transcript - 45th Annual William Blair Growth Stock Conference:

Sammy, Introducer, William Blair: Started, I’m obligated to direct everyone to WilliamBlair.com for a full list of disclosures. I’m very pleased to introduce doctor Matt Klein, the CEO of PTC Therapeutics, who’s going to provide an overview of the company. Thanks, Matt.

Matt Klein, CEO, PTC Therapeutics: Thank you, Sammy. It’s great to be here and be able to provide this update on PTC Therapeutics. Before I get started, I refer you to our forward looking statements, including those listed in our most recent, 10 k, which you can find on our website. For those not familiar with PTC, we were built on a patient focused mission to use innovative science to develop important therapies for patients who desperately need them. Over the past two decades, we’ve built a global biopharmaceutical company that discovers, develops, and commercializes transformative therapies for patients with rare diseases.

We’re coming off a very successful 2024 where we had strong execution across in 2025 and beyond. We achieved all development regulatory milestones on time, including the submission of four approval applications to the FDA, setting us up with the potential to have four commercial launches in The United States within twelve months. We again had a year of outstanding revenue performance, which along with effective OpEx management gives us line of sight to being cash flow breakeven in the near future. We’ve established a strong cash position with over $2,000,000,000 on the balance sheet as as of the end of q one twenty twenty five, which enables us to succeed in all of our commercial launches, continue to develop our r and d programs, as well as participate in meaningful business development opportunities. And finally, through our efforts of focusing our r and d activities on our highly differentiated innovative scientific platforms, including splicing, we’re preparing the next wave of transformative PTC therapies.

Let me dig deeper into some of these important accomplishments. As I mentioned, we submitted four approval applications to the FDA in 2024, all of which were accepted for review. We had our AADC gene therapy BLA, NDAs for sepiaterin for children and adults with PKU, Translarna for patients with nonsense mutation DMD, and vatiquinone for children and adults with Friedreich’s ataxia. Our gene therapy, Cability, was approved in November, and it became the first ever approved gene therapy that is surgically administered directly into the brain. For sepiaterin, our NDA was accepted, and we have a PDUFA date of July 29, so next month.

And for vitiquinone and Friedreich’s ataxia, that NDA was accepted with priority review, and we have a PDUFA date of 08/19/2024. In addition, we submitted a number of regulatory applications outside of The United States for sepiaterin in preparation for our global launch for this PKU product, which I’ll talk a little bit more about in the presentation. We had another strong year of revenue performance in 2024 with total revenue of $807,000,000 exceeding our revenue guidance. This revenue performance was driven by our in line products and is a testament to our customer facing team’s ability to successfully execute around the globe in both competitive and genericized markets. This is incredibly important as we gear up for the next wave of commercial launches.

And through our revenue performance, our effective OpEx management, the rapid and robust monetization of the priority review voucher we received with the approval of Cability Gene Therapy and business development deals, we’ve built an over $2,000,000,000 balance in cash, which it does several important things for us. That cash balance will enable us to get to cash flow breakeven without the need for additional capital. It will allow us to successfully execute on our commercial launches as well as advance our r and d programs and will also enable us to participate in business development activities to complement both our commercial and r and d portfolios. In addition, in this time of macro uncertainties, this cash balance provides us with significant insulation to pursue our priorities for 2025. And in 2025, we are planning for a number of potential value creating milestones, several of which we’ve already achieved.

We had the vadiquinone NDA accepted with priority review in February. We recently received a positive opinion for our PKU drug, Sefiance, in Europe, with an EU approval expected by the end of this month. We recently read out the successful phase two PIVOT HD study from our PTC five one eight Huntington’s disease program, and we still have a number of important milestones to go for the remainder of the year, including the upcoming, Suffiance launch for our PKU product. Let me dig a little deeper into some of our key programs starting with our PKU program. As we’ve talked about, despite there being two approved therapies for PKU, there still remains a significant unmet need for the majority of children and adults with PKU.

Based on the data we’ve generated to date, it’s clear that SUFIANCE can meet this significant unmet medical need. Suffiance has a dual mechanism of action. First, as a cofactor for the enzyme implicated in pet in PKU known as phenylalanine hydroxylase, and it’s a more effective and bioavailable cofactor than giving b h four alone. It has a second activity, which is as a chaperone to stabilize the defective enzyme configuration, which allows us to not only have an a better effect in patients who may be responsive to BH four, but also to provide significant benefit to patients with non BH four responsive mutations, which tend to be the more severe PKU patients. This ability to address the full spectrum of PKU patients was substantiated in our phase three affinity trial in which we demonstrated significant decrease in phenylalanine levels across the full spectrum of PKU patients.

We were able to bring over eighty four percent of patients within guideline levels of phenylalanine of less than 360 micromolar per liter. We were even able to normalize phenylalanine levels for twenty two percent of patients, something not really ever seen in clinical trials of PKU therapies. In the long term extension where we did a phe tolerance substudy, We demonstrated that over ninety seven percent of patients in this substudy were able to liberalize their diet, and two thirds of patients were able to take protein levels beyond the recommended daily allowance of protein intake for someone not affected by PKU. This ability to liberalize diet and allow patients to take protein levels that are recommended for someone without PKU is incredibly meaningful to PKU patients and supports why Suffiance is such a potentially important therapy for patients. Finally and importantly, in all of our studies, Suffiance has been demonstrated to be safe and well tolerated.

While these data speak volumes about the potential for SUFIANCE to meet the significant unmet need for PKU patients, it’s the words of the patients and and physicians themselves that strongly substantiate the promise of this therapy. Anya Muntau, who’s a global PKU key opinion leader, has shared that she’s excited about the opportunity to offer the drug to all of her patients. And we’ve heard this from a number of physicians interested in providing the drug to those already on therapies as well as the vast majority of patients who currently are not on any therapy. Doctor Carrie Harding recently wrote about how impressive the results we’ve obtained in our clinical trials are in classical PKU patients. Those are the most severe patients who desperately need an effective therapy.

And time and time again, we see anecdotes on social media from patients talking about their ability to take have hamburgers and pizza and liberalize their diets in ways they never could before. Again, it’s this ability to provide the patients the opportunity to liberalize their diets, which is so, so important and speaks so strongly to the potential for to meet the unmet need of PKU patients. Our global commercial teams are in place, and we’re well positioned for the launch. A lot of time has been spent in building out our US team. We’ve done all the essential things ready to launch this product.

We’ve mapped a hundred and three centers of excellence in The United States for PKU. We understand who the prescribers are. We’ve also been understanding the dynamics at these centers where you have both physician’s assistants, nurse practitioners, and nutritionists participating in prescription decisions. We’ve we understand that a lot of these centers have generated waiting lists, in some cases, with hundreds of patients who are anxious to get on drug as soon as possible. We’ve also set up an opt in program so that we can keep patients, families, and physicians up to date as we move closer and closer to launch.

We’ve had a number of payer, and access discussions. We understand, importantly, that payers understand the value of lowering phenylalanine, and we’ve been able to understand that based on the data we have that we’ll be able to price this drug at a premium to existing therapies, specifically Palynziq. And finally, we continue to work closely with the patient community given this is a close knit community, and we wanna be able to support patients throughout their journey and as we transition to making commercial product available. The data that we’ve collected to date strongly support that we can access all key patient segments, including those patients who have never been on a therapy before, and this tends to be those more severe classic patients and whom we’ve demonstrated strong efficacy. Patients who failed on current therapies for one reason or another and strongly want to be on a therapy that can allow them to have lower phenylalanine and potentially liberalize their diet.

And finally, patients who were on current therapies but not well controlled or even maybe well controlled but wanna do even better in terms of phenylalanine lowering and diet liberalization. When you consider the size of the PKU population, globally 58,000, about 17,000 in The US, the strength of our data, the ability to price at a premium to Palynziq, as well as a successful track record of our commercial teams, we see this as a significant market opportunity of up to and above beyond a billion dollars even in The United States alone. And so we’re incredibly excited, with the PDUFA date of July 29, to be able to get ready to potentially launch this drug in The US and meet the needs of so many patients. Next, I’m gonna discuss our Friedreich’s ataxia program with Vetiquinone. If approved, Vetiquinone would be the first ever therapy for children and adolescents under the age of 16 with Friedreich’s ataxia.

The NDA, which was accepted with priority review in February, is based on the findings of efficacy from the MoveFA placebo controlled trial as well as data collected in two long term studies. As I mentioned, that NDA was accepted with priority review, and we have an FDA action date of August 19. We’re also getting ready to look at other regulatory submissions outside of The US where there’s a number of Friedreich’s ataxia patients. Let me review some of the key data in the NDA. First, from the seventy two week MoveFA placebo controlled study, we demonstrated significant benefit on the upright stability scale.

The upright stability scale is the most sensitive and meaningful measurement of disease progression in the pediatric and adolescent patients that were enrolled in the Move FA study. This significant slowing of forty two percent over seventy two weeks full delay in risk of loss of ambulation. In addition, the findings on upright stability were supported by benefit observed on the physical component of the modified fatigue scale as well as the one minute walk test. In addition to the placebo controlled data, the NDA also included results of two different long term studies. The first was long term study based on the pay patients enrolled in the Move FA study.

In the analysis of this pay in this of these patients treated for three years with vutiquinone, we demonstrated a fifty percent slowing in disease progression. We also analyzed the long term data from an earlier placebo controlled study done in ambulatory and nonambulatory adult patients. And in this analysis, patients treated for twenty four months had a 4.8 benefit point benefit on the disease rating scale, which equates to roughly two year slowing of disease progression. So taken together, these data demonstrate that fituquinone provides important benefit in terms of slowing of disease progression in all FA patients, ambulatory and nonambulatory, and of all age groups, including pediatric, adolescent, and adults. Despite there being an approved therapy for Friedreich ataxia, it’s only for patients older than 16 years of age, and there remains a significant commercial opportunity for Vetiquinone.

First, there’s about six thousand patients with Friedreich ataxia in The US, about a third of whom are pediatric patients for whom there is not an approved therapy. The safety and efficacy data we’ve collected on vatiquinone to date, including in young children, support not only that vatiquinone could provide a safe and effective disease modifying therapy for pediatric and adolescent patients not eligible for the current therapy, but given that there are a large number of adults not on the approved therapy, betiquinone can absolutely provide a treatment option for adult patients as well. Our launch preparations are well underway in The United States. We’re working closely with the patient commit patient advocacy group with whom we’ve worked for a number of years. We know where the pediatric adolescent adult patients are mainly treated.

We have the relationships with these centers. And, again, we’re in excellent position for successful launch if the drug’s approved, this coming August. Now I’d like to turn to our PTC five one eight Huntington’s disease program from which we recently shared the successful phase two pivot HD study results, last month. PTC five one eight comes from PTC’s leading splicing platform and follows on the heels of the of the successful discovery and development of Avrizdy, which is now the global leading SMA therapy. PTC five eighteen is a highly differentiated HTT lowering agent.

It’s orally bioavailable. It broadly biodistributes through every region of the brain. We’ve been able to demonstrate dose dependent and durable lowering of huntingtin mRNA and protein. It has a favorable safety profile and in clinical studies has no evidence of Nf L, treatment related spikes. And we’ve been able to demonstrate thus far dose dependent effects of PTC five one eight on a number of different clinical scales.

It’s this combination of unique characteristics of PTC five one eight and the data we’ve collected to date that supports this as really a leading and incredibly promising therapy for patients with Huntington’s disease. As I mentioned, we recently shared the results of our successful phase two PIVOT HD study, which achieved all the key objectives of this study. The trial met its primary endpoint of Huntington lowering at week twelve with durable findings out to twelve months. We had favorable and dose dependent trends on a number of clinical scores in stage two subjects, and we demonstrated long term benefit at month twenty four on a number of clinical scales relative to natural history along with evidence of dose dependent NFL lowering in these patients. As background, what should happen in Huntington’s disease is that NFL should be increasing over time, and our ability to show dose dependent lowering at twenty four months really supports the biological effects of neuroprotection.

And importantly, our phase two study confirmed, that that PTC five one eight is safe, and well tolerated with no treatment related serious adverse events or NFL spikes. We’re now working to advance PTC five one eight, and are looking at the design of an efficacy trial as well as the potential for accelerated approval based on these data and the data that we continue to collect in the open label extension study of PIVOT HD. In December of twenty twenty four, we announced our licensing, collaboration with Novartis for the development and commercialization of PTC five one eight. This agreement included a a billion dollar upfront payment upon deal closure, which occurred in January of this year, up to 1,900,000,000.0 in development, regulatory, and sales milestones, a 40% US profit share with double digit tiered royalties ex US. And Novartis is to fund all further development of PTC five one eight following the completion of the of the PIVOT HD study.

And so now, as I mentioned, as we think about next steps, the Novartis and PTC teams are working closely together on outlining the next regulatory and development plans for this program. In addition to all the advances we’ve made in our development and commercial portfolios, we’ve also worked hard to focus our research activities on our highly differentiated scientific platforms of splicing and pherocytosis and inflammation. We focused our resource our research efforts on these platforms where, quite simply, we are the leaders in these fields and wanna be able to use this unique expertise we have in these scientific areas to continue to develop highly innovative therapies. Let me start with splicing. We’re incredibly proud to have pioneered the field of splicing molecules and are well positioned to continue to lead it.

A first approved therapy when, really, the the only approved splicing modulator is of RISD, which came from the PTC labs, and PTC five one eight, the second compound from our pi platform, is advancing well in the in the Huntington’s disease program as I described. The teams that worked on the discovery and early development of these molecules have only gotten smarter over time. We’re continuing to leverage the learnings from these previous development programs so that we could discover more therapies and advance them faster. One example of this is PTC, which is a proprietary screening engine that now allows us to much more rapidly and reliably identify molecules that can hit specific splicing targets. The effect of PTC and this high throughput system is that we’ve significantly shortened the time of preclinical development for splicing molecules.

We now have a number of active preclinical programs for CNS disorders as well as non CNS disorders that we look forward, to advancing this year and sharing more, as progress is made. We also are looking to not only have our splicing platform be a source of PTC discovered and developed therapies, but we also realize the potential of splicing to develop meaningful therapies for other indications outside of PTC’s expertise, such as large neurodegenerative disease and oncology. And, therefore, we’re also looking to use splicing platform as a source of strategic partnerships for these non core therapeutic areas. We’ve also made significant progress in our inflammation and ferroptosis platform. Again, this is a a platform or an area of science where PTC has unique, expertise.

The platform focuses inflammation and cell death targets that are known to be important in both CNS and non CNS disease pathologies. Accordingly, we have a number of active preclinical programs in CNS and non CNS therapeutic areas that we’re looking to advance this year. For example, we have a phase two ready DHODH inhibitor program that we’re gonna look to develop for neuroinflammatory disorders. We have an NLRP three, directed in inflammation program, which is entering IND enabling, studies. We have a preclinical pheroptosis program targeting alpha synuclein for Parkinson’s disease as well as an n r f two activation program for a number of CNS and non CNS disorders.

In conclusion, we’re incredibly proud to have built PTC into a strong, innovative, and well capitalized company. We have line of sight to being cash flow breakeven in the near future, and we have set as an objective going beyond that to reaching $2,000,000,000 in top line revenue, which we can achieve with the current programs pending regulatory decisions in 2025. From an r and d pipeline perspective, we have two highly differentiated and innovative platforms that can be a continued source of development products over the next several years. And finally, with the cash balance we have at hand, we’re we’re in a strong position to continue to use business development strategically to further support growth to that $2,000,000,000 top line mark and beyond. Thank you.

Sammy, Introducer, William Blair: Thanks, Matt. Looks like we have about five minutes left, so, maybe we can take a couple of questions before we head to the breakout. Traditionally, it can be challenging to commercialize rare disease drugs, because there isn’t a huge population that can be, challenging to identify. So can you, describe a bit some factors about the PKU population that makes you more confident that this will be a strong launch and then how you’re kind of thinking about early adopters?

Matt Klein, CEO, PTC Therapeutics: Yeah. Absolutely. I I I think this is one where all the key pillars for commercial success are in place. Because there have been previous therapies, they’ve just not met the needs of the majority of the population. So there’s, for PKU, there’s newborn screening.

So the patients are all identified. You know, the the patient identification work we often have to do for rare disease launches, we don’t have to do. The patients are all identified. There are centers of excellence already practicing. And as I mentioned, we’ve mapped the hundred and three centers in The US.

Payers understand the disease. So when we sit down and have discussions about PKU and treating PKU, payers understand that value can be tied to phenylalanine lowering. That’s so, so important. We don’t have to educate about the patient journey and what all this means. It’s it’s very well understood.

And then the other part is we’ve got an experienced commercial team. We’ve got a team that in The US that has been selling Emflaza for the past eight years and has been highly successful, in a very challenging, you know, genericizing competitive market. So, you know, we’re well positioned to move, over and now launch PKU. And it’s not just the teams in the field. It’s also, for example, our patient services team, which is an incredibly important group that helps not only work with patients and remind them and families about, you know, time for renewal of your prescription and that’s responsible for the high rate of adherence we have for our therapies.

But they all this group also works with the physician physician’s offices to help walk through prior authorizations and step edits where needed. You know? So it’s really this sort of white glove service that assures that every patient that can be prescribed the drug gets prescribed the drug, stays on the drug, and that we can help navigate any barriers they may be to gain to gaining access. So, again, we’re we’re incredibly excited about this opportunity. And you had also asked about who we see as sort of the initial target populations.

What’s interesting is we hear different things from different physicians. We talked about those those big market segments, the patients who are on therapies, patients who are therapy naive, who who desperately want a therapy that can lower phenylalanine and allow for diet liberalization and those who may have tried and failed. What we’re hearing is there’s some physicians who say, look. Like doctor doctor mentioned in my presentation, I’m gonna try all my patients on the drug. They may start with the patients who, for example, may be on an existing therapy.

These are patients who are already in the clinic on a drug, and it’s a matter of changing out once one once a day drug for another one that we’ve demonstrated if you have an effect with with with, saproteren, either branded or generic, you’re gonna have a much better effect with with Suffiance. Others say, look. Those folks are on drugs. We’ll look to a drug already. We’ll look to switch them, but I wanna get these classical.

These more severe patients who don’t have any therapy, I wanna get them on a drug first. So we’re hearing different things from different places, but I think the important thing we keep hearing is there’s a lot of pent up demand at these centers. And the physicians, a number of physicians have said, look. We’re just gonna try all of our patients, on the drug, and and we’re there to support that large effort.

Sammy, Introducer, William Blair: And and then you mentioned step edits, with there being an available therapy that is generic. How are you kind of thinking about if patients are gonna make what required to do that step edit through that generic therapy and how long that might take?

Matt Klein, CEO, PTC Therapeutics: Yeah. I think this is, again, a a benefit of PKU in a way, which is within one to two weeks, you can step through. You you take the drug four to two weeks. You reach steady state, and it’s a blood test to demonstrate what your phenylalanine level is on that generic therapy. And then, of course, one to two weeks later, you can show the much better lowering in phenylalanine that you would get with Cefiance.

Our teams are very used to this. Again, this is the Imflaza team. This is a group that’s dealt with step edits to prednisone for Emflaza in DMD, and that takes many, many, many months. And, of course, there’s no hard objective endpoints like a blood test for DMD. So I think our teams say, okay.

There’s step edits. We we can manage this. We manage prednisone, and it’s gonna be a much shorter time, much more objective, quick, way to step through. Yeah. Sure.

So we had previously shared that the European Commission in May adopted I’m sorry. Late April adopted the negative opinion of for Translarna, which led to the nonrenewal of the authorization. Under most circumstances, that would mean that the therapy would be fully off the market. We as we’ve shared, we have a number of countries in Europe that are have through a mechanism that the European Commission is allowing, who remain on commercial therapy. So we are, in fact, still commercializing the drug in Europe despite it not being authorized, and that has the approval of the European, government.

We’ve said that we expect using this these mechanisms to maintain about 25 to 35% of European revenue, going forward for the near future. In The US, the NDA was accepted. It’s under review. Clinical site inspections have been completed. We’re still in back and forth with the agency on information requests.

Sammy, Introducer, William Blair: And it looks like we are at time, but we are going to head up to Burnaby for the breakout session next. Thank you,

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