Okay, we'll continue with the next session. I'm Paul Choi, and I cover the spit-cap biotech sector here at the firm. It's my pleasure to have PTC Therapeutics here and on stage with me to my immediate left, Dr. Matthew Klein, CEO, and to my far left, Pierre Gavier, CFO. I think what we'll do is what we've done in prior sessions, and we'll let maybe Matt kick it off maybe with some high-level comments on sort of the strategic priorities for the balance of the year and going into 2027, and then we'll go into Q&A after that.

Sounds great, Paul. Thanks so much for having us. For us, 2026 so far has been really successful. We had a really strong first quarter with record product revenue of $226 million, total revenue of $273 million, fueled by the continued success of this Affiance launch. We announced launch in Japan late first quarter, which was great news and really helped accelerate launch. We also shared positive top-line data from the long-term extension of the Vodoplank Phase 2 Pivot HD study. The Phase 3 trial is now underway and being enrolled by Novartis and continue to have nice revenue contributions from our more mature products. We look forward to an exciting rest of 2026 into 2027. Clearly, this Affiance launch, the continued momentum of the launch is going to be an important part of the story. But we also are looking forward to a number of advances in our R&D portfolio. We've aligned with the FDA on the design for the next vetequinone-phrederic-zotaxia study and expect to initiate the what we're calling PROVE-FA trial in the third quarter, which I know we'll probably talk a little bit about as open-label natural history comparator to provide additional data to support NDA resubmission. We also look forward to initiating a Phase 2a study of PTC 844, which is our next-generation DHO-DH compound. We've already initiated the Phase 1 study of PTC 612, which is our NLRP 3 compound, and we look forward to continuing to enroll that Phase 1 healthy volunteer study as well to collect data in the second half of the year. We expect to announce a development candidate for our MSH3 splicing program. We're really excited about this program. MSH3 is an important target for regulating somatic expansion, which is important in Huntington's disease, myotonic dystrophy, as well as other disorders, and we look forward to announcing that compound and really continuing to lead the field of oral small molecule splicing. And we also expect to have a development candidate from our ferreptosis platform for synuclein-targeted therapy that can be used in both Parkinson's disease as well as MSA. So really a lot going on at PTC, and also, as we've mentioned, we'll continue to look at business development opportunities given our strong cash balance and our desire to look at ways to augment the PKU portfolio as well as drive near an intermediate-term top-line revenue.

Great. Clearly a lot going on, so maybe we can start with the commercial piece, and I guess either for you, Matt, or Pierre, how would you characterize the Suffiance launch so far to date? How do you think about how it's done versus your internal expectations as we look at the sort of comps or historical comps in the category, and then I had a follow-up question after that.

Yeah, absolutely. Look, we're really excited with how the launch has gone to date and for what we see as continued growth. We expect to have continued growth in the U.S. as well as later in the year contributions ex-U.S. given our global footprint and already the number of global approvals that we have. Look, we were very clear from the beginning that we did not think comparing suffiance to the other therapies, PKU therapies, whether QVAN branded a generic or Palinzeek was really an appropriate comparator given the unique differentiated profile suffiance. We knew coming into the launch that there's about 17,000 patients in the U.S., 58,000 in markets where we can commercialize globally, and despite there being approved therapies, there was really still a significant unmet need. We know that suffiance, given its oral, well-tolerated profile, the fact that it has a dual mechanism of action, one as a precursor, a more bioavailable precursor for BH4, the second as an independent and chaperone that can provide benefit for non-BH4-responsive or classical patients, that we, for the first time, had a therapy that could address the full spectrum of PKU patients regardless of age and regardless of severity. And the story of the launch thus far really is a story of breadth, seeing broad uptake across all different segments, no matter whether you're talking about age segments of kids and adults, treatment experience, whether that be therapy naive, those who tried and failed other therapies, or switches from existing therapies. We shared our Q1 earnings that at that point in time, we had over 90% of the centers of excellence in the U.S. have already prescribed suffiance, and that we soon expect to be able to get that number to 100%, which again, after only two full quarters of launch, is really a significant milestone. We also talked about our team as being quite experienced in selling rare disease drugs not only in the U.S., but also globally. We have a robust global commercial infrastructure, and we've talked a lot about how that team did really well with Translarna, which I think everyone could acknowledge had a much more challenging profile than something like Suffiance. And with the approvals in Japan and Europe and Brazil and our ability to leverage early access programs in countries where we don't yet have pricing and reimbursement, And really, we think that this is unique again in the category for being able to be start within the first year and have a true global launch. And so overall, I think we're really pleased with how things have gone. We've been very clear that we expect continued growth in the U.S., accelerated growth outside the U.S., which we expect to really start having an impact in the latter part of 2020 on board and position us really.

As you think about sort of the other launch dynamic that you touched, you know, on utilization both in treatment naive, treatment experience, and switch patients, can you maybe help us understand, you know, to what degree is this, given the sort of that there hasn't been a new PKU drug in a while, how much of this is just sort of bolus driven versus how much, you know, do you think this is just sort of market expansion in the PKU category?

Yeah, absolutely. Look, when we came into this launch, a lot of folks believed, and in fact, a lot of prescribers said that they thought the early part of the launch would be driven by switches. That's actually not been the case. One of the, I think, surprises early on is about 30% of patients are from that therapy-naive bucket. And this is a bucket of folks who tend to have more severe disease, classical PKUs. The reason they're therapy-naive is because they were never tried on other therapies because there wasn't a belief that they would work or that it would be well-tolerated. And many thought that these individuals were, quote-unquote, lost follow-up. It would be very hard to get them on drug. And if you did get them on drug, that was going to be sort of a ladder in the latter part of the launch years down the line. That's not the case. And so we've had strong contributions from that segment and probably the largest segment contributing is those who've tried and failed previous therapies with a smaller number of switches, which we expect to grow over time. So I think what we're really seeing is still steady, strong demand from a market or from a patient population that has real needs for a safe and effective therapy.

Great. You've probably had some nice numbers on where the patient growth and patient types are coming from. But either for you or for Pierre, have you sort of, I guess, based on this early launch trajectory, rethought the TAM here in PKU? I mean, $1 billion plus, and on top of that, the ex-US opportunity. Have you sort of internally rethought about the TAM, and then I had a follow-up on that?

Yeah, absolutely. So I think in terms of market numbers or population sizes, about 17,000 patients in the U.S., and we've said about 58,000 globally in geographies where we believe we can get access and reimbursement. And when you think about the fact that this is a therapy that has the potential to serve virtually all of those patients, we see this as a large market opportunity, and I think we've been now very clear that we believe that this is overall a $2 billion-plus global opportunity. We expect there to be strong growth in the U.S., and then again, the ex-U.S. contribution. We've talked a lot about the importance of the international contribution here and being able to have that early and launch it. I think at its peak, Translarnia did something like 330 million. Again, we think the PKU profile is much stronger. Translarnia was never in Japan. The PKU population is much larger. So when you just start thinking about that, you can see that the international contribution can be quite strong. And then in the U.S., given the differentiated profile, you know, I think we could reach those $2 billion-plus numbers with modest penetration for an effective differentiated rare disease therapy.

Since you mentioned the ex-U.S. markets, can you maybe give us sort of a current update on sort of where reimbursement is, where reimbursement is? We've, I guess, in the past talked about, like, Amnog in Germany. You recently launched in Japan. Just sort of those larger markets, maybe just give us an update there.

Yeah, absolutely. So we'll start with Japan first because pricing reimbursement is done there. And given that Suppliance is an orphan product, that price is set for 10 years, and there's no referencing done when we get pricing in other countries. So that list price is consistent with the U.S. list price, so that's a great place to be in terms of having that locked in for the next 10 years. The negotiations are ongoing in Germany. As you know, it's a long process and a lot of back and forth. We were very encouraged by the early feedback we've received and the rating we received and the acknowledgement by GBA that they view phenylalanine, which was the primary endpoint of the clinical study, as holding value, as having clinical meaningfulness, which is really important because traditionally they do not regard biochemical or biomarker data as support of clinical meaningfulness, and they do in this case, and they were very explicit in the initial feedback. The other thing that's really helped us is being able to have the results of the Amplify study available to be in the dossier, the pricing reimbursement dossier. Amplify is a head-to-head study where we took individuals, they were randomized to either get BH4, then suffiance, or suffiance, and then BH4, which allowed us to demonstrate within individuals the significant benefits of suffiance. And so overall in that study, we had a mean benefit or mean reduction of 70% more with suffiance than for BH4. So to be able to go and have pair discussions with a published manuscript that shows superiority over the other oral therapy really puts us in a good position for reimbursement. So those discussions are ongoing. We've also recently announced that we have been able to get suffiance into the AP2 early access program in France, so we'll be beginning pricing and reimbursement discussions there, as well as in Italy and other countries. We've mentioned that while we're going through the often lengthy pricing and reimbursement discussions, we've been able to, as we're doing in France, leverage early access programs. And that has a number of advantages, because being able to do early access at this point gets us patients on drug in countries, that's a price set by us pending agreement on pricing, and also puts the drug in the hands of the influential KOLs who can then participate in the pricing and reimbursement discussion. It's very common in a number of the countries that the ministries of health who make decisions around pricing and reimbursement will consult with expert physicians. Clearly, if they've had the drug in their hand and seen the benefits of that drug, that will certainly help a favorable price.

I want to talk about some of the real-world feedback that you're getting now that you guys are commercializing suffiance here. And, you know, one of the things I picked up on social media was a patient talking about eating McDonald's chicken McNuggets, you know, of all things, for the first time. And so I want to, you know, maybe just on that point of diet liberalization or increased protein intake, you know, what are you hearing in the field? And I guess, you know, this is something, you know, I think you're still continuing to prosecute in the clinic and follow up. And just, you know, how are you thinking, you know, what are you hearing from your sales force and just, you know, thinking about development of that aspect of the disease?

I didn't see the McDonald's comment, but I assume that was a kid, but who knows. But I think the point, which is super important, is that these are individuals who have not had the benefit of having freedom to eat food that other kids may eat or that their parents may eat or that the parent can eat the same meal as that they're serving their child. And we're continuing to hear these stories of really success, of suffiant success, of allowing folks to have some diet liberalization in cases, complete diet liberalization. And that's super important. We hear them on social media, see them on social media, our field forces get feedback, and we also work very closely with the dieticians. All these centers of excellence have a multidisciplinary approach to care of the PKU patient, which not surprisingly includes dieticians. Why? Because in the absence of a therapy, the mainstay is this highly restrictive diet. And what we've worked very hard on is as individuals start on suffiance, that there is a plan to gradually increase protein intake and also some guidance on the right protein to take from a nutrition standpoint. So we're getting a lot of feedback from the dieticians as well as how well this is going. We've also been hearing a lot about another benefit, which is super interesting, which is improvements in mood and cognitive function. We know that PKU can have significant effects on cognition, clarity of thought, And we also know that suffiance gets across the blood-brain barrier. And so we're hearing more and more about these types of benefits, decreased anxiety, increased executive functioning. And we shared some of these data that we collected from our Open Label Extension of the affinity trial where we're able to show significant improvements on those points in the QOL assessment. But as you alluded to, Paul, we're continuing to gather real-world evidence because these data, which capture different types of benefit, obviously we talk a lot about dialyberization, but other things in terms of how I'm going to function and mood are incredibly important for generating market demand, market pull, and also obviously adherence to compliance.

I think all biotech CEOs have a healthy sense of paranoia when they think about competition. And so as you sort of look at the competitive landscape, Maybe, you know, who are you monitoring? You know, who do you think of as potentially next to market here? And, you know, what does that, I guess, in your mind, as you look at the market down the road, potentially due to pricing and sort of share in your mind?

Absolutely. Look, I think there's been a lot of interest in the kidney-directed therapies that Janana and Mays have. You know, and I think what we're hearing a lot from the physicians is a desire to combine those with suffiance to get even more benefits for patients. I think it's still, while those are in clinical trials, I still think there's some questions out there in terms of what's the ultimate therapeutic window for these compounds going to be, who are they going to be most useful in. Currently, all the clinical studies, I mean, the phase three trial for Janata, I think Mays just announced a phase two study, are in adults. So that's still leaving that huge pediatric and adolescent population without, and the reasons for that I'm not sure of, and we'll see how that goes over time. So I think from our standpoint, we're watching those. We'll have to see what the safety and efficacy are, but I think importantly what we're hearing a lot is the desire to have combo therapies with suffiants. And suffiants would be first line, and then this could be added on to get even more benefit.

As a backbone.

Yeah, and it makes perfect sense. This is a community that PKU patients, their daily life is combo cocktail therapy. That's how they live today. And so it's not surprising if you can have additional therapies that can get you even more benefit. That would be the ideal.

Let's maybe turn to your pipeline and maybe starting with the tiquinone. Can you maybe walk us through your recent FDA dialogue and just sort of what drove the sort of change in perspective at the agency to allow your new trial design? And maybe you can just remind us what your new trial design is here.

Yeah, absolutely. So, look, we were clearly disappointed by the CRL last summer and believed that we had evidence of significant benefit in the younger patients who currently don't have a therapy. And we had discussions with the FDA. An interesting part here was they were the ones who suggested that if, well, one, they said they'd like additional data. They had suggested that we could do an open-label natural history comparator study to get that additional data to support NDA resubmission. Quite frankly, that was a little surprising to us in a good way, and I think it potentially reflects the fact that, one, the safety profile of the drug is very well characterized. It's safe and very well tolerated, so that's not at the question now. And in terms of efficacy, I think the FDA has long viewed the Friedrich's Texture Natural History Database as being robust and reliable and able to provide a reliable comparator to what would happen outside of therapy without therapy, and there's a lot of reasons for that. The clinical trial sites tend to be the same sites who contribute to the Natural History Registry, so it's the same teams doing the same assessments on the same patients. I think also if you look at our MOVE-FA study, the placebo group's progression looked a lot like natural history, which also gives you confidence that you're seeing those two groups aligned and therefore supports the fact that the natural history group can provide a reliable comparator in an open-label fashion. So we've worked with them on the protocol. They've reviewed our protocol and analysis plan, and this is going to be about 120 individuals age 7 to 21 consistent with the primary analysis population of the MOVE-FA study. It's going to be a 24-month treatment period. There will be a natural history comparative group from the robust and reliable Friedrich's Ataxia Natural History Registry. We have a statistical analysis plan that is already specifying what the matching criteria are going to be, what is the matching model going to be, and how we're going to account for any potential differences between those key characteristics that are most influential on disease progression. We believe we can get this study started in the third quarter, and obviously what we've heard so far is a lot of excitement from the community to be able to enroll in a study and get access to the drug without having to worry about being put on a placebo and therefore delaying access to a drug that could slow progression, which is particularly important for younger ambulatory individuals.

Great. Maybe two mechanical questions. Can you maybe remind us in terms of, like, background or prior therapies? Are patients allowed to be on CoQ, 10S, vitamin E, or previously on SkyClarus? And then just on the matching dynamic, is that locked in as they're randomized, or how does that process evolve during mid-trial or after treatment is initiated?

Yeah, very good question. So we're fairly, in terms of SkyClarus, they can't on active Skyclaris therapy, that we do have certain provisions if they were on it for a limited amount of time because there were a number of individuals who tried and dropped off. It comes down to whatever they have in the trial, there's natural history subjects, right? So the real issue with Skyclaris, which we still have the MOVE-FA extension going on, and we have patients on both therapies, which a lot of the physicians want, but the registry doesn't have enough individuals who had enough Skyclaris experience that we could match. That's why we had to be very limiting in terms of how much scyclarus you could have been exposed to. In terms of the mechanics of the matching, so what we've done, we will do the query of the registry once everything is done and the trial is enrolled. However, we did pre-specify exactly what are going to be the characteristics on which we're going to match. That was the key part. And the obvious things that you can imagine that are important in terms of influencing disease progression. So age, number of GAA repeats, baseline MFAR score, so that you're aligning the disease severity. So those types of things that will be influential of disease progression, we specified already how we're going to do that, what the procedure is going to be for querying the registry, and then once we do that, what are the details of the propensity match model, including all the coding and all of that, has already been submitted before we start the trial so that there's integrity there in terms of sort of a fixed process. Exactly right. Given the open-label nature, we wanted to make sure that all of that was fixed and aligned on ahead of time.

Great. As you think about your learnings from your previous MOVE-FA trial and just sort of what the real-world experience has been with SkyClarus, where do you think the biggest sort of unmet need is outside of sort of the pediatric or younger patient population, maybe just from a clinical benefit perspective as well?

So, look, I think the pediatric and adolescent unmet need is significant, right? About a third of the patients are in that age group. And if you think about a degenerative condition, it's quite clear you want to start treatment as soon as possible. And I think we've talked a lot about the data from MoveFA and being able to show the significant benefit in the short-term on upright stability, which has those items around balance and gait, which are clearly important to younger individuals who can still ambulate. So I think that that is an important part of the population. But then when you look to the adult population, there's a number of individuals who've tried and not stayed on Slyclaris. And I think the ability to have therapeutic options is really, really important for patients. I mentioned as well that there's a number of physicians who want to combine therapies, who also want to look at combining a therapy like verticlin on some of the frataxin-augmenting therapies. Why? Because even And though the frataxin protein is deficient in Friedrich's ataxia, introducing a functional frataxin protein into a very broken cell will not work nearly as well as introducing frataxin protein into a cell where you can turn down the cell stress and inflammation. If you get the genetic machinery and other cellular machinery to be less inflamed and work better, then giving exogenous frataxin will obviously work better. And this has been shown preclinically in a number of different diseases. So I would say kids and adolescents, and then don't underestimate the importance of having a therapy that's safe, well-tolerated, is unlikely to have significant monitor requirements as a therapeutic option for folks.

Great. Maybe one more on Friedrich's ataxia before we move on. With an open-label natural history trial design here, can you remind us, is there an interim look built in? How does that work? If positive, how do you think about sort of timelines? if you have after, let's say, half your patients in a room or whatever the case may be, or monitored? How do you think about that?

We thought a lot about that because whenever you start a trial like this and you have a high probability of success given the nature of the study, given the fact that we were able to incorporate a lot of the learnings from our previous studies, and what we have now is a very good understanding of the disease and the endpoint. but then we see a last chance here and if you believe in the drug you design your trial well we believe what made the most sense is let's not have an interim let's run it straight through 24 months with the full population because that's what's going to give us the highest probability of success and so the plan is at this point not to have an interim and I think it's also important given the open label nature of it that we have the largest data package we can, right? Because there's always questions. That's part of the decision to go 24 months was because you want to make sure that that placebo effect in phrygics ataxia, which is known to exist and known to be pretty fully washed out by month six, went up as long as time as possible to show that this is a credible, this effect can be credibly attributed to the therapy and nothing else. And so when you put all that together, we said, look, high probability of success. Let's get as many patients treated for the full duration and go with the strongest data package and get this across the line and approved.

Okay, great. I want to maybe touch on Huntington's for a moment. Your program, which you partnered with Novartis, has now transitioned to a Phase 3. Can you maybe just remind us of what are your obligations, I guess, at this point as the Phase 3 now is underway, and just how should we think about updates from that program now that the asset is more or less fully in Novartis' hands, given it is potentially a very big value driver for you in the future?

Yeah, absolutely. Look, we're incredibly excited about the VOTA plan program, and we've talked a lot about, you know, from the beginning, this was following the path established by RISD. How do you develop a small molecule splicing agent for a CNS, mostly CNS disease? And everything we've done along the way has followed that path. I think the pivot HD data, the long-term extension data, give both PTC and Novartis confidence that the Phase III trial is very well designed, includes early symptomatic patients, the target enrollment, 770 patients approximately, with CUHUDRS as the primary endpoint up to 36 months. There's an interim analysis that will allow for an earlier look at a smaller number of patients. And, you know, this is fully in the hands of Novartis in terms of the technical execution. They're responsible for the enrollment and the conduct of the trial. And I have to say, as proud as I am of our team and the capabilities of the PTC team, Novartis was built to do a 770 individual study. And I think that really increases the technical success of this study. They're also 100% responsible for the funding of the study, and then according to the terms of the agreement, we still have about another $1.8 billion in sales and commercial milestones, and then there's the 40% profit share in the U.S. double-digit XUS4LTs. So we're set up for significant financial benefit here, if successful. And again, I think both companies feel that if the long-term extension data play out in phase three, then we could have the first disease-modifying therapy for Huntington's disease, which would be incredibly exciting.

Maybe in our remaining time, as we turn to your earlier stage pipeline, one of the things that I find most interesting is your NLRP program. A lot of interest in this area generated by the recent Ventex data as well as the acquisition there. And so maybe just at a high level, could you point out some of the key differences and pros and cons for your asset versus Ventex?

Yeah, absolutely. Look, I think this is, as you mentioned, a hot area. Inflammation in NLRP3, the NLRP3 inflammasome has been recognized as really an important hub. The NLRP3 pathway, it's sort of a sensor of inflammation that propagates a further inflammatory response. We've been clear that we're interested early at this point in looking at pulmonary disorders where there's a very nice link between NLRP3, the NLP3 inflammasome, and pulmonary pathology. What we've been able to do, and we shared this at our R&D day, is really show that PTC 6.2 is differentiated from some of the other therapies. If you look at the Ventix compounds, the obesity compound, we've shown that in an IL-1 beta assay, we have 50%, 5, 0% greater potency, and then over, you know, we have almost 10x potency of their other compounds. So I think from a potency standpoint, we believe that we have a really differentiated compound. We believe we have a lot more specificity. And then its chemical structure is different as well. A lot of the existing NLP3 therapies have a sulfonylurea backbone, which is responsible for some of the off-target toxicities. Ours doesn't have that. So its chemical structure is a bit different. It's got a good potency profile, and it has a, we believe, more specificity for an LRP3. We mentioned the MAD-type things in drug exposure, but we'll also be having an MAD cohort of individuals with a metabolic syndrome biochemistry that could give us a read in Phase 1 on some of the potential biomarker effects we're having on inflammatory markers associated with the NLRP-3 inflammasome.

Pierre has been very patient here. Matt and I have chatted on the pipeline, but Pierre, I want to maybe ask you a little bit, you know, you're having massive top-line growth from suffiance here. You've sort of maintained your OPEX guidance for the remainder of fiscal 26, and so the company's, you know, sort of financial profile is in the midst of changing here to break even and eventually profitability. And so as you think about the shape and sort of velocity of the company's margin profile change, what would you sort of tell the street to how to think about the balance of 26 and going into 27 and the intermediate term?

So first of all, we're very excited to be here. We work really hard to get there. We're excited about WCC finance launch and continued growth in 26 and beyond. We talked about a $2 billion plus opportunity. That's guidance yet. And, you know, that obviously bodes very well with our OPEX management, which we've been very disciplined over the years to reduce that. And, again, we're built, you know, globally. We talked about the launch. Commercial was ready, right, in Europe, in the U.S., in Japan, in LATAM. And, obviously, you know, R&D, we're going to be very disciplined. We have a number of new products, obviously, coming in the pipeline, which we talked about a little bit. and both our platforms on both splicing and ferroptosis are very important to us. And then, you know, HD is obviously a partner, so there's no OPEX for that. So that's how we think about the future. And obviously we talked about a very strong cash balance where, you know, for the right opportunity, we would be ready to deploy additional capital. We have a commercial team available that demonstrated they can sell rare disease products globally. And so we think about how can we accelerate that top-line discipline matter and making sure we create value for our shareholders in a creative manner.

Pierre, it's no secret you have a banking background, and so you've obviously looked at shopping for assets, and I guess if you sort of think about the core competencies of the company, historically it's been in things like splicing and so forth, you have a strong launch going on with the suppliers, but how do you sort of think about the landscape as you look for potential BD on the early stage side versus something that might be closer to, you know, clinical trial completion or commercial stage?

So the number one thing that is very important is we have flexibility, right? We don't have a rush to do one way or the other. And are there ways that could complement our approach historically? We're a small molecule company. So just if you look at modalities, but we could look at other ways to enhance the portfolio again it's all about how can we complement both commercial assets which are important are there things that could fit rare neuro rare metabolic or neuro metabolic but again we could build another adjacencies as long as it's rare disease right we're not going to start and do an oncology therapeutic area for us that makes you know very little sense for us so again it's what are the areas where we are you know we are really the leader and also i would say beyond commercial we have global regulatory capabilities we manage to you know push programs to the finish line that could be very attractive to to companies and so when we look at the landscape you know if a company i mentioned the fact that we want to create value for our shareholders if a company trades at you know 20x sales good for them uh that's not going to be something we will be looking at but are are there ways where we can find value and again there's also that the global commercial capabilities. We could take ex-U.S. rights, for example, and start to market in Europe, in Japan, in LATAM in a very efficient manner.

We're coming up on time here, so maybe one to close with you, Matt, which is if you were to direct investors to one or two things that you think are really key over the next 12 to 18 months, what would you highlight?

Yeah, so clearly having a product like Suffiance that can be a multibillion-dollar rare disease product, The fact that we have a robust pipeline with the leading oral HD therapy in development with none of the costs but a significant back end, the ability to innovate and have a steady flow of pipeline assets, and then sitting in a strong cash position with over $1.9 billion gives us flexibility to continue to augment, as Pierre said, both the R&D and commercial pipelines. And the fact that we're, as you mentioned, Paul, going to be vectoring towards being cash flow break-even and profitable in the near future. It's a pretty unique setup for a company, and we're pretty proud of that.

Okay, we're at time here. So my thanks to Matt and Pierre for joining us, and thanks to PTC.