I'm Paul Choi, and I cover the SMid-cap biotech sector here at the firm. It's my pleasure to have PTC Therapeutics here on stage with me, to my immediate left, Dr. Matthew Klein, CEO, and to my far left, Pierre Gravier, CFO. I think what we'll do is what we've done in prior sessions, we'll let Matt kick it off maybe with some high-level comments on sort of the strategic priorities for the balance of the year going into 2027. 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 and total revenue of $273 million, fueled by the continued success of the SEPHIENCE launch. We announced a launch in Japan late first quarter, which was great news and really helped accelerate the launch. We also shared positive top-line data from the long-term extension of the votoplam phase II PIVOT-HD study, the phase III trial is now underway and being enrolled by Novartis, and we continue to have nice revenue contributions from our more mature products. We look forward to an exciting rest of 2026 into 2027.
Clearly, with the SEPHIENCE launch, the continued momentum of the launch is going to be an important part of the story, we are also looking forward to a number of advances in our R&D portfolio. We've aligned with the FDA on the design for the next vatiquinone Friedreich's ataxia 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 . It's an open-label natural history comparator to provide additional data to support NDA resubmission. We also look forward to initiating a phase II-A study of PTC844, which is our next -generation DHODH compound. We've already initiated the phase I study of PTC612, which is our NLRP3 compound, we look forward to continuing to enroll that phase I 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 and myotonic dystrophy, as well as other disorders. We look forward to announcing that compound and really continuing to lead the field of oral small molecule splicing. We also expect to have a development candidate from our ferroptosis platform for synuclein-targeted therapy that can be used in both Parkinson's disease and MSA. Really a lot going on at PTC, 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- and intermediate-term top-line revenue.
Great. Clearly a lot going on. Maybe we can start with the commercial piece , and I guess either you, Matt, or Pierre—how would you characterize the SEPHIENCE launch so far to date? How do you think about how it's done versus your internal expectation—
Yeah
...as we look at the sort of comps or historical comps in the category? 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 SEPHIENCE to the other PKU therapies, whether it be brand or generic or PALYNZIQ was really an appropriate comparator given the unique differentiated profile of SEPHIENCE. We knew coming into the launch that there were about 17,000 patients in the U.S., 58,000 in markets where we could commercialize globally, and despite there being approved therapies, there was really still a significant unmet need.
We know that SEPHIENCE, given its oral well-tolerated profile and the fact that it has a dual mechanism of action, one being a more bioavailable precursor for BH4, the second being an independent chaperone that can provide benefit for non-BH4 responsive or classical patients, means that for the first time we had a therapy that could address the full spectrum of PKU patients, regardless of age and regardless of severity. 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 or treatment experience, whether that be therapy naive, those who've 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. had already prescribed SEPHIENCE, 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. 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 SEPHIENCE.
With the approvals in Japan and Europe and Brazil, our ability to leverage early access programs in countries where we don't yet have pricing and reimbursement—really, we think that this is unique again in the category for being able to start within the first year and have a true global launch. 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 2026 reimbursement on board, and position us really in [inaudible] and beyond.
Right. As you think about sort of the other launch dynamics, Matt, you touched on utilization both in treatment-naive, treatment-experienced, and switch patients. Can you maybe help us understand to what degree this is given 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 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, 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. This is a bucket of folks who tend to have more severe disease, classic PKU. 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. Many thought that these individuals were, quote unquote, lost to follow-up, or it would be very hard to get them on drugs.
If you did get them on drugs, that was going to be sort of in the latter part of the launch, years down the line. That's not the case. We've had strong contributions from that segment. 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. 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 provided some nice numbers on where the patient growth and patient types are coming from. 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 and, on top of that, the ex-U.S. opportunity. Have you sort of internally rethought about the TAM? Then I had a follow-up on that.
Yeah, absolutely. I think in terms of market numbers or population sizes, there are about 17,000 patients in the U.S., and we've said there are about 58,000 globally in geographies where we believe we can get access and reimbursement. 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. I think we've been now very clear that we believe that this is overall a $ +2 billion global opportunity. We expect there to be strong growth in the U.S., 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 in the launch. I think at its peak, Translarna did something like $330 million. Again, I think we think the PKU profile is much stronger.
Translarna was never in Japan. The PKU population is much larger. When you just start thinking about that, you can see that the international contribution can be quite strong. Then in the U.S., given the differentiated profile, I think we could reach those $ +2 billion numbers with modest penetration for an effective differentiated rare disease therapy.
That's great. Since you mentioned the ex-U.S. markets, can you maybe give us sort of a current update on sort of 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. We'll start with Japan first because pricing and reimbursement are done there. Given that SEPHIENCE is an orphan product, that price is set for 10 years, and there's no referencing done when we get pricing in other countries. That list price is consistent with the U.S. list price. 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 acknowledgment by G-BA that they view phenylalanine, which was the primary endpoint of the clinical study, as holding value and as having clinical meaningfulness, which is really important because traditionally they do not regard biochemical or biomarker data as supportive of clinical meaningfulness, but 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 SEPHIENCE, or SEPHIENCE, then BH4, which allowed us to demonstrate within individuals the significant benefits of SEPHIENCE.
Overall in that study, we had a mean benefit or a mean reduction of 70% more with SEPHIENCE than for BH4. To be able to go and have payer discussions with a published manuscript that shows superiority over the other oral therapy really puts us in a good position for reimbursement. Those discussions are ongoing. We're also recently announcing that we have been able to get SEPHIENCE into the AP2 or early access program, in France. 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. That has a number of advantages, because being able to do early access at this point gets us patients on drugs in countries. That's a price set by us, pending agreement on pricing.
Also puts the drug in the hands of the influential KOLs who can then participate in the pricing and reimbursement discussions. 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 as we seek to get favorable pricing.
Okay, great. I want to talk about some of the real-world feedback that you're getting now that you guys are commercializing SEPHIENCE here. One of the things I picked up on social media was a patient talking about eating McDonald's Chicken McNuggets—of all things—for the first time. I want to maybe just on that point of diet liberalization or increased protein—
Yeah
...intake. What are you hearing in the field? I guess this is something I think you're still continuing to prosecute in the clinic and follow up on.
Absolutely.
Just what are you hearing from your sales force , and are you just thinking about the development of that aspect of the disease?
Yeah. Absolutely. I didn't see the McDonald's comment, but I assume that was a kid, but who knows?
Yeah.
I think the point that is super important is that these are individuals who have not had the benefit of having freedom to eat foods that other kids may eat or that their parents may eat, or that the parent could eat the same meal as what they're serving their child. We're continuing to hear these stories of real success, of SEPHIENCE success, of allowing folks to have some diet liberalization, and, in some cases, complete diet liberalization, and that's super important. We hear them on social media, see them on social media, and our field forces get feedback, and we also work very closely with the dietitians. All these centers of excellence have a multidisciplinary approach to the 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.
What we've worked very hard on is, as individuals start on SEPHIENCE, that there is a plan to gradually increase protein intake and also some guidance on the right protein to take from a nutrition standpoint. We're getting a lot of feedback from 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.
We also know that SEPHIENCE gets across the blood-brain barrier. We're hearing more and more about these types of benefits, decreased anxiety and increased executive functioning. 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. As you alluded to, Paul, we're continuing to gather real-world evidence because this data captures different types of benefits; obviously, we talk a lot about diet liberalization, but other things in terms of cognitive function and mood are incredibly important for generating market demand and market pull and also, obviously, adherence and compliance.
Great. I think all biotech CEOs have a healthy sense of paranoia when they think about competition. As you look at the competitive landscape.
Yeah
Who are you monitoring, who do you think of as potentially next to market here? What does that, I guess, in your mind, as you look at the market down the road, potentially do to pricing and share in your mind?
Yeah, absolutely. Look, I think there's been a lot of interest in the kidney-directed therapies that Genya Dana and Maze have. I think what we're hearing a lot from the physicians is a desire to combine those with SEPHIENCE to get even more benefits for patients. I think it's still, while those are in clinical trials, I still think there are some questions out there in terms of what the ultimate therapeutic window for these compounds is going to be.
Who are they going to be most useful in? Currently, all the clinical studies, including the phase III trial for SEPHIENCE, and I think Maze Therapeutics just announced a phase II study, are in adults. That's still leaving that huge pediatric—
Yeah
...and adolescent population without. The reasons for that, I'm not sure of, we'll see how that goes over time. I think from our standpoint, we're watching those, well, 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 SEPHIENCE, that SEPHIENCE would be first line, this could be added on to get even more benefit.
As a backbone.
Yeah, it makes perfect sense. This is a community for PKU patients, their daily life is a combo cocktail of therapies. That's how they live today. It's not surprising if you can have additional therapies that can get you even more benefit. That would be the ideal.
Great. Let's maybe turn to your pipeline and maybe start with the vatiquinone. Can you maybe walk us through your recent FDA dialogue and just sort of what drove the change in perspective at the agency to allow your new trial design, and maybe you could just remind us what your new trial design is here?
Yeah, absolutely. 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. 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.
Yeah.
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 in question now. In terms of efficacy, I think the FDA has long viewed the Friedreich's ataxia natural history database as being robust and reliable and able to provide a reliable comparator to what would happen without therapy. There are 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. We've worked with them on the protocol. They've reviewed our protocol and analysis plan. This is going to be about 120 individuals aged seven to 21, consistent with the primary analysis population of the MOVE-FA study. It's going to be a 24-month treatment period. There'll be a natural history comparator group from the robust and reliable Friedreich'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. 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 a 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 background or prior therapies, are patients allowed to be on CoQ10, vitamin E, or previously on SKYCLARYS? 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. In terms of SKYCLARYS, they can't be on active SKYCLARYS therapy. 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 really comes down to whatever they have in the trial is natural history subjects, right? The real issue with SKYCLARYS, which we still have the MOVE-FA extension going on, and we have patients on both therapies, which a lot of the physicians want. The registry doesn't have enough individuals who had enough SKYCLARYS experience that we could match. That's why we had to be very limiting in terms of how much SKYCLARYS you could have been exposed to.
In terms of the mechanics of the matching, what we've done is 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. The obvious things that you can imagine that are important in terms of influencing disease progression. Age, number of GAA repeats, baseline mFARS score so that you're aligning on disease severity. Those types of things will be influential in disease progression.
We specified already how we're going to do that, what the procedure's 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.
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 ahead of time.
Good. 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 SKYCLARYS, where do you think the biggest unmet need is outside of sort of the pediatric or younger patient population? Maybe just from a clinical benefit perspective as well.
Yeah, I think the pediatric and adolescent unmet need is significant, right? About a third of the patients are in that age group. If you think about a degenerative condition, it's quite clear you want to start treatment as soon as possible. I think we've talked a lot about the data from MOVE-FA 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. I think that is an important part of the population. When you look to the adult population, there are a number of individuals who've tried and not stayed on SKYCLARYS, and I think the ability to have therapeutic options is really, really important for patients.
I mentioned as well that there are a number of physicians who want to combine therapies and who also want to look at combining a therapy like vatiquinone with some of the frataxin -augmenting therapies. Why? Because even though the frataxin protein is deficient in Friedreich's ataxia, introducing a functional frataxin protein into a very broken cell will not work nearly as well as introducing a 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, giving exogenous frataxin will obviously work better. This has been shown pre-clinically in a number of different diseases. I would say kids and adolescents, don't underestimate the importance of having a therapy that's safe and well-tolerated and is unlikely to have significant monitoring requirements as a therapeutic option for folks.
Great. Maybe one more on Friedreich's ataxia before we move on is, with an open-label natural history trial design here, can you remind us if there is an interim look built in? How does that work? If positive, how do you think about timelines if you have after, let's say, half your patients enrolled?
Yeah
Whatever the case may be, for those who are monitored, how do you think about that?
We thought a lot about that. Whenever you start a trial like this, you have a high probability of success, given the nature of the study and given the fact that we were able to incorporate a lot of the learnings from our previous studies, what we have now is a very good understanding of the disease and the endpoint. Really, our last chance here, if you believe in the drug, you've designed 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. The plan is, at this point, not to have an interim.
I think it's also important, given the open-label nature of this, that we have the largest data package we can.
Right. There are always questions. That's part of the decision to go 24 months: you want to make sure that that placebo effect in Friedreich's ataxia, which is known to exist and known to be pretty fully washed out by month six, you want to have as long a time as possible to show that this effect can be credibly attributed to the therapy and nothing else. 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.
Sure.
Your program, which you've partnered with Novartis, has now transitioned to a phase III. Can you maybe just remind us of what are your obligations, I guess, at this point as the phase III now is underway? 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 votoplam program. We've talked a lot about it from the beginning, this was following the path established by VYVGART. How do you develop a small molecule splicing agent for a mostly CNS disease? Everything we've done along the way has followed that path. I think the PIVOT-HD data and 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 is 770 patients, approximately, with cUHDRS as the primary endpoint up to 36 months. There's an interim analysis that'll allow for an earlier look at a smaller number of patients. 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.
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. I think that really increases the technical success of this study. They're also 100% responsible for the funding of the study. According to the terms of the agreement, we still have about another $1.8 billion in sales and commercial milestones. Then there's the 40% profit share in the U.S. double-digit ex-U.S. royalties. We're set up for significant financial benefit here if successful. Again, I think both companies feel that if the long-term extension data play out in phase III, then we could have the first disease-modifying therapy for Huntington's disease, which would be incredibly exciting.
Great. Maybe in our remaining time, as we turn to your earlier stage pipeline, one of the things that I find most interesting is your NLRP3 program. A lot of interest in this area generated by the recent Vertex data.
Yes
As well as the acquisition there. So maybe just at a high level, could you point out some of the key differences and pros and cons for your asset versus Vertex?
Yeah, absolutely. Look, I think this is, as you mentioned, a hot area, inflammation and NLRP3. The NLRP3 inflammasome has been recognized as truly an important hub. The NLRP3 pathway, it's sort of a sensor of inflammation, then 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 NLRP3 inflammasome, and pulmonary pathology. What we've been able to do, and we shared this at our R&D day, is really show that PTC612 is differentiated from some of the other therapies. If you look at the Vertex compounds, the obesity compound, we've shown that in an IL-1 beta, I'd say we have 50%, 50% greater potency, and then over, we have almost 10x the potency of their other compounds.
I think from a potency standpoint, we believe that we have a really differentiated compound, and we believe we have a lot more specificity. Then its chemical structure's different as well. A lot of the existing NLRP3 therapies have a sulfonylurea backbone, which is responsible for some of the off-target toxicities.
Ours doesn't have that. Its chemical structure's a bit different. It's got a good potency profile, and it has, we believe, more specificity for NLRP3. We mentioned SAD/MAD type things, the 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 I on some of the potential biomarker effects we're having on inflammatory markers associated with the NLRP3 inflammasome.
Great. Pierre has been very patient here. As Matt and I have chatted on the pipeline, Pierre, I want to maybe ask you a little bit, you're having massive top-line growth from SEPHIENCE here. You've sort of maintained your OPEX guidance for the remainder of fiscal 2026, the company's sort of financial profile is in the midst of changing here to break even and eventually profitability. 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 2026 and going into 2027 and then the intermediate term?
Yes. First of all, we're very excited to be here. We worked really hard to get there. We're excited about, obviously, SEPHIENCE launch and continued growth in 2026 and beyond. We talked about a $ +2 billion opportunity, not guidance yet. That obviously bodes very well with our OPEX management, which we've been very disciplined over the years to reduce . Again, we're built globally. We talked about the launch, the commercial was ready right in Europe, in the U.S., in Japan, and in LATAM. Obviously, 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. Both our platforms on both splicing and ferroptosis are very important to us. HD is obviously partnered, so there's no OPEX for that. That's how we think about the future.
Obviously, we talked about a very strong cash balance where, for the right opportunity, we will be ready to deploy additional capital. We have a commercial team available that has demonstrated they can sell rare disease products globally. We think about how we can accelerate that top line in a very disciplined manner and make sure we create value for our shareholders in a creative manner.
Great. Pierre, it's no secret you have a banking background, you've obviously looked at shopping for assets, and I guess as 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 SEPHIENCE, how do you sort about the landscape as you look for potential BD on the early stage side versus something that might be closer to clinical trial completion or commercial stage?
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, are there ways that could complement our approach? Historically, we're a small molecule company, just if you look at modalities, we could look at other ways to enhance the portfolio. Again, it's all about how we can complement both commercial assets, which are important. Are there things that could fit rare neuro, rare metabolic, or neurometabolic? Again, we could build another adjacency as long as it's a rare disease, right? We're not going to start and do an oncology therapeutic area for us. That makes very little sense for us. Again, what are the areas where we are really the leader? Also, I will say beyond commercial, we have global regulatory capabilities.
We manage to push programs to the finish line that could be very attractive to companies. When we look at the landscape, if a company, I mentioned the fact that we want to create value for our shareholders, if a company trades at 20x sales, good for them. That's not going to be something we would be looking at. Are there ways where we can find value? Again, there's also that global commercial capability. We could take ex-U.S. rights, for example, and start to market in Europe, in Japan, and in LATAM, in a very efficient manner.
Great. We're coming up on time here, 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?
Clearly having a product like SEPHIENCE that can be a multi-billion dollar rare disease product. The fact that we have a robust pipeline with the leading oral HD therapy and development with none of the cost, a significant back end. The ability to innovate and have a steady flow of pipeline assets 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. The fact that we're, as you mentioned, Paul, going to be vectoring towards being cash flow breakeven and profitable in the near future. It's a pretty unique setup for a company, we're pretty proud of that.
Great. Okay, we're at time here. My thanks to Matt and Pierre for joining us, and thanks to PTC.
Thank you.
Thank you.