Healthcare Conference. I'm Tazeen Ahmad. I'm one of the senior mid-biotech analysts at the bank. It is my pleasure to have our next presenting company on stage with me, PTC Therapeutics. I've got several members from the management team. Specifically sitting next to me are Matthew Klein, who's Chief Executive Officer, Pierre Gravier, who's Chief Financial Officer, as well as Kylie O'Keefe, who is Chief Commercial Officer. Good morning, everyone.
Good morning.
Thanks for coming over from New Jersey.
Tazeen, thank you. Great to be here.
So maybe, Matt, you can, for the folks who may not be as familiar with PTC.
Just give us an overview of the company, sort of the main platform and some of the recent events that have occurred, and then we can go into some more detail around those.
Yeah, terrific. For those of you who don't know PTC, we are a global biopharmaceutical company that discovers, develops, and commercializes therapies for patients with rare disorders. We have a robust commercial portfolio with six products, five of which we market ourselves all over the world for a number of different rare diseases, and the sixth product is commercialized by Roche, from which we derive collaboration and royalty revenue. We have a robust R&D portfolio with programs in a number of different rare disorders, particularly with focus on neurology and metabolic disease. And we have two scientific platforms, one in splicing, being that we are the pioneers in small molecule splicing, and ferroptosis, where we're also pioneering therapies there. We're looking forward to a very exciting year.
We came into 2024 after making a number of changes to strengthen the company. We went through exercises to focus our R&D portfolio, as well as to rightsize the company so that we could reduce our operating expenses and put the company on a trajectory to cash flow breakeven. We recently, in Q1 earnings, reported an excellent revenue performance, beating consensus by over 20%, and are also making significant progress on a number of regulatory submissions this year. We're on schedule to submit four approval applications to the FDA, including one for our PKU program, which we've described as easily being a billion-dollar-plus peak revenue opportunity. We also have two important data readouts scheduled for this year, one for our Huntington's disease program, which is regarded as the leading Huntington's disease program right now, and also.
We, again, we're expecting data from that in the second quarter. In the fourth quarter, we're expecting top-line results from our ALS trial, which is a registration-directed study of our drug, utreloxastat, for ALS. So it's really shaping up to be an exciting year with a number of important milestones, all of which are on schedule.
Okay, perfect. Yeah, there's a lot going on. And one of the topics that, you know, every investor, I think, is interested in hearing more about is Translarna, which was your first approved drug. It's been approved in Europe now for multiple years, and based on a review that occurred, I can't even remember how many months ago, the most recent update was that CHMP had recommended removing the product from the market because of the results of a confirmatory study that occurred. I think everybody who follows PTC had made the assumption that that was the end of the story. But it appears it is not. The drug is still on the market. And maybe, Matt, can you just give us a quick summary of the saga that we all know as Translarna?
Yeah.
Because I think it's important, because it's meaningful revenue that people were assuming would go away, which if it doesn't go away, does, does have a meaningful impact for the company.
Absolutely, and quite frankly, it already has, in many ways. And so, as you pointed out, Tazeen, Translarna was the first-ever approved therapy for Duchenne muscular dystrophy. It received conditional marketing authorization in Europe in 2014.
Subject to confirmatory studies, the most recent of which was Study 041, which, while demonstrated statistical significance on all key endpoints in the overall ITT population, did not reach significance in the pre-specified primary subpopulation. So it's this very, I'd say, unique situation, which many of the. Well, I'm going to discuss with Translarna is also unique, but it was a unique situation in which a smaller subgroup didn't achieve significance, but the overall population did. And of course, the study also demonstrated, as expected, that Translarna continues to be safe and well-tolerated. The CHMP took a very strict view and said that since this was a confirmatory study and the primary analysis population, while being just a subgroup of the overall enrolled population, didn't reach significance, then we did not sufficiently confirm benefit and issued a negative opinion.
That negative opinion was issued following a reexamination in January. What typically happens is when the CHMP issues an opinion, it then only becomes official once the European Commission adopts it, and that typically happens within 67 days of that opinion.
Being issued. So as you pointed out, most expected that come the end of the first quarter, the product would be withdrawn from the market, and this would've been the first time that a product that was safe and well-tolerated was withdrawn. It went to the European Commission, and what we do know is that procedure to remove the drug was stopped. There was at least one country that voiced objection, which stops the procedure. And then a meeting of the Standing Committee of the European Commission is convened to discuss the matter. We know that that meeting was held the week of April 22. We have not heard anything further. So what does that mean? That means what, the drug remains on the market. The opinion of the CHMP was not adopted thus far.
We know that there was a tremendous outpouring of support to keep the drug on the market from patients and physicians all over Europe. So this very well may be a situation where you have patients and physicians saying, "Look, here's a drug that's safe. There's clear evidence of benefit." The review at CHMP may have been overly conservative, particularly given the unmet need for patients with nonsense mutation DMD in Europe. We are awaiting to hear what happens next. But for us, it's really been business as usual. We were commercializing the drug in Europe and many places all over the world. Physicians continue to prescribe it, patients continue to want it, and we continue to generate revenue.
Is there a situation where countries can order product and keep inventory in the event that something occurs officially, where it says, you know, after this day, you can no longer, you know, order more product or anything like that?
Yeah. So we were preparing different approaches to keeping the drug available by leveraging individual country processes, whether those be named patients or other access pathways. That still remains in the background, but of course, the benefit of where we are right now is just continue to commercialize as if.
Yeah.
Last, it was last year.
Yeah.
None of that has changed. We do have that as a backup plan, should it come to pass that this opinion gets adopted and the drug is withdrawn.
Can you give us a sense of what level of penetration you have in Europe, market-wise?
Yeah, sure. Kylie, do you want to talk of the dynamics?
Yeah, look, absolutely. I think one of the things we've talked a lot about, Tazeen, is our approach with Translarna preceding this situation was, Europe was definitely our first market, right? So we started in Europe, and we've been able to ensure across many of our European markets to have pretty deep penetration. What we then looked at is said, "Okay, to grow the business substantially, we need to continue geographic expansion." So we looked at a number of markets across the world that we started to geographically expand into. And I would say these have been probably our bigger growth driver markets, whereas Europe's been a bit more stable in that regard. We've still talked about definitely finding new patients in Europe.
Yeah.
We've still continued to penetrate. We're not at max penetration. We are at high penetration, but we're not at max penetration. We still continue to find new patients in Europe. We still continue to put new patients on drug. It's just where we see larger growth rates is some of these earlier markets that we've started to geographically expand into.
Okay. During the 10 years or so that this drug has been on the market, how has price impacted uptake, if at all?
Not at all. I think one of the things that, you know, traditionally is believed, especially in Europe, that over time, as you see, you know, a product enter the market at a certain price point, over time, that tends to degrade.
One of the things that we've been able to do across the board in all of our markets is hold a very tight pricing corridor, and this has been something that we've been heavily focused on to balance the business, and I think it's been something the team's done an incredible job of managing.
Okay. How have, have you managed the need for infrastructure in terms of, you know, marketing and sales?
One of the things we talked about, as this situation started to unfold is the fact that we have a PKU launch just around the corner.
And so what we had always intended to do, and we talked about this, was we built infrastructure around the world that was not only fit for delivering and executing on the business that we have today, but also being ready for tomorrow's business. And tomorrow at the time was PKU which is now today.
And so we did not touch the infrastructure. We've kept it whole, we've kept it in place, and this has obviously been a huge benefit as this situation has continued, and we, as Matt said, we have Translarna remaining on the market, and we don't see a situation changing any time soon. So from that perspective, with regards to that, the infrastructure is focused on ensuring that we meet the needs of patients and physicians for Translarna, as well as preparing for PKU.
Okay. So Matt, does the European Union have any obligation to tell you, the company, about what's going on at a certain point?
We assume that we will be, and we expect we'll be notified once there is a clear path. Now, what has to happen, the way it works in Europe is the CHMP, which is part of the European Medicines Agency, is responsible for the scientific and technical evaluation of a product. They do that evaluation, they then send that opinion over to this central body, the European Commission, that then makes a decision on that.
T o adopt it or not. So you can imagine in this period, it's entirely possible that there's ongoing discussions-
Between the two bodies about next steps and such, and we expect that once there's clarity, we'll hear. You know, I think for us, we did a lot of work last fall and winter to prepare for the withdrawal of the drug.
Yeah.
We strengthened our balance sheet and so that we're in a strong position to build forward all of our other programs this year. So this is really a situation where we're, it's pure upside, where we had set the company up for success based on what's coming with PKU, based on HD, based on all of our other programs, and had the cash to execute on all of those. And now we're in a situation where something we didn't expect is occurring, but it's upside, which is a great situation to be in.
So presumably, you've tried to figure out the different scenarios of outcomes here, you know, through consultants or experts who know the European regulatory system. Is there any kind of outline you could provide for us on what could happen? I mean, the obvious is they decide to keep it on the market as is or pull it from the market.
But is there in-between scenarios?
Certainly. Look, I don't think there's any expert here who can weigh in because we're in a bit of an unprecedented situation. The only precedent of which we're aware, where there was a CHMP opinion that the European Commission did not adopt, is the case of Glibera in 2012, which was the gene therapy being developed for lipoprotein lipase deficiency. What happened at that point was there was a negative opinion. It went to the European Commission. They did not adopt the opinion. It went back to the CHMP, and six months later, the CHMP changed their opinion to positive, which was adopted then by the European Commission. I think we have the range of options. I mean, certainly, the commission could decide we're just going to adopt a negative opinion, and it goes into effect.
But what could also happen is it could go back to CHMP. They could either look at changing the opinion to positive. They could say: "Look, this was a conditional authorization. We'll maintain that subject to additional work being done.
And you could imagine as those discussions happen, that even getting to that point could unfold over several months.
Could they ask you to do yet another confirmatory study?
They could, and you know, we will obviously welcome any opportunity to keep the drug on the market and make it available to boys in Europe.
Okay. If there continues to be business as usual, is there any change to your expectations for expenses this year, though?
No change for expenses. As Kylie said, the infrastructure is the infrastructure, and the important point, there's no change for expenses to maintain Translarna, and we weren't expecting any change to expenses to prepare for the PKU launch. So that's, that was all set.
Okay. So that's Europe. Let's talk about the U.S. and where you are with your application.
Yeah, absolutely. You know, as you're well aware, it's been a long history, and I know a lot of people are saying, "I wouldn't bet on this one because it's such a long history." But I think a lot has changed.
Yeah.
I think first of all, and I believe this is also supported by what's going on in Europe, Study 041 provides a very strong data set of efficacy, in an overall ITT population, which is something that really hasn't been seen for a genetically directed therapy for Duchenne muscular dystrophy. Having significance on walk test, North Star test, time function test, all of those is really, really important that the therapy is safe. We also have the STRIDE registry, which is a important source of confirmatory evidence showing long-term benefit in terms of delay or time to loss of ambulation. You know very well the long history between FDA and, and, and PTC on Translarna, and I think we can clearly say that in our recent discussions, there's really been a reset in the relationship.
I think the players have changed on both sides, and I think I'm happy that we've had very collaborative, productive discussions with the agency about how we can use these data to support a resubmission. And we made it very clear to the agency that we are not gonna go down this path if there's an RTF in.
In waiting for us, and the feedback we got from our pre-submission meeting was that if there's any questions left, they'd be a matter of review, which is what we needed to hear to move forward with the submission. We've also talked about just even looking at CDER's recent review of givinostat and looking at how they looked at the data for givinostat in terms of some of the analyses their statisticians did at FDA. And those were all things they asked for from us prior to advising us on the submission and doing all those analyses. They all support the robustness of our finding of significant benefit across these endpoints, and also some of the issues that the review teams have previously raised around real-world evidence, we're able to easily satisfy.
While there is a long history here, and it makes it difficult for anyone to be too bullish on the therapy getting approved by FDA, I think we're in a strong position to provide a dossier that has persuasive evidence of benefit and necessary confirmatory evidence, and the FDA has demonstrated to us their openness to review this package.
Which committee would be reviewing?
This is in neurology.
Of the people that PTC would have interacted with over the last several years, how many of them are the same?
So I, as you know, Billy Dunn, who oversaw the division, has left-
Teresa Buracchio is now the head of the Division of Neurology Products, and she is with Teresa. She's been in all of these meetings where we've had these discussions.
Okay.
The review teams obviously change, and things change over time.
Okay. So if I'm understanding you correctly, you don't think you would get an RTF, so you're gonna go ahead and apply. And assuming that, you don't get an RTF, they will accept the application, what are your chances, do you think, of getting a priority review?
Since it's an NDA resubmission, technically, it follows a priority review timeline.
Okay.
So there's two months to accept the filing, and then it should be six months from there. We're planning to resubmit the NDA mid-year. We would expect then, according to that timeline. to have an outcome in the first quarter of 2025.
Okay. And do you think an AdC om would be likely?
We don't know. We'll have to see what they decide. We welcome whatever comes in the review process.
Just remind us, what's new in the package that you would be submitting versus packages that they've seen before?
Study 041.
Okay.
I think being able to have a study in 359 boys, that in the ITT population, that was well controlled, standard steroid regimen, well stratified, and having P values less than 0.05 on all of the key endpoints, is quite frankly, something they've never seen in a DMD product before.
I think the safety of the product has always been there, and then they've never seen data from STRIDE, because STRIDE didn't start till after the last submission. So to be able to provide real-world evidence on over 280 boys over the course of five years of data collection and having a robust propensity-matched comparator natural history population against whom we show that we slow loss of ambulation by three and a half years, slow loss of pulmonary function by over a year and a half, is again, something they've never seen in DMD and something the FDA's increasingly said are things that they wanna see to try to understand what the benefits over the long- term could be in horrific diseases like DMD.
Okay. I asked about the AdC om because I'm curious. You did mention that for Europe, for example, there was a strong advocacy coming from physicians as well as parents of patients. We do know that there's, in general, a strong advocacy for DMD in the U.S. vis-à-vis parents of these boys. Why wouldn't an Ad Com be a good opportunity for these parents to voice to FDA why it would be important, presumably, to have access to this drug?
I think it would be an excellent opportunity. We're aware that over the years, even as recently as this past year, parents of children with nonsense mutation have let their voice be heard to have data even now. I think, you know, if you think about how long we've been looking at, we've been studying the struggle, we have over 150 boys in the United States who have been on Translarna for 10 years or more.
And many more who want to get on the drug. So we welcome any and all opportunities to be able to showcase these data and to be able to let the voice of the patient community be heard. So, you know, for us, if there's an AdC om, terrific. If there's not, fine. We're confident in the package and look forward to any opportunity we need to take to, to push this forward.
Okay. Is the company engaging right now with parents to let them know that this is, this is what's happening?
We established the field of DMD drug development.
Our connections with the patient community go back to the beginning.
So we are very well connected with the patient groups. We have always been. The early history of DMD is the history of PTC, and a lot of the early history of PTC is the history of DMD. So they are very engaged with us. We pride ourselves on those relationships that we build with the community. Of course, we also have commercialized EMFLAZA over the past several years- which continue to strengthen our ties with the patient community.
Okay. So maybe let's talk about EMFLAZA. So you recently lost exclusivity, but on your earnings call, your most recent earnings call, you've talked about not really experiencing impact from that yet. Is that a temporary situation, or do you expect that to continue? And what strategies has PTC undertaken in order to maintain share?
Do you want to talk a bit about that?
Yeah, absolutely. So as we've talked about, obviously loss of exclusivity wasn't a surprise to us. We knew it was coming, and so what we wanted to do was make sure that we built a robust plan to be able to do what we can to protect the business. So we put a number of different strategies in place. We focused on contracting with targeted payers. We focused on contracting and putting initiatives in place with specialty pharmacies, ensuring that we have as robust as possible patient support programs. We have a wonderful PTC Cares team that spend a lot of time with these DMD patients, making sure they're engaging with physicians, insurance companies, and limiting the burden that's on these patients and caregivers.
We have a disposition team that's engaging with the physicians and making sure that the understanding of the value of EMFLAZA exists, brand loyalty exists, and everything around that. So it's sort of a multi-pillared approach to be able to protect the business. So fast-forward, the other thing we also talked about is rare disease is quite unique when it comes to loss of exclusivity, and DMD is no different there. Particularly, there is a high number of Medicaid patients. So when generics tend to enter the marketplace at that 20%-30% discount to brand, that margin is eaten up very quickly when you lose, you know, $0 copay, when you lose access to the PTC Cares team or to any cares team, when you lose access to specialty pharmacy, you lose access to patient support programs and everything associated.
And so with just one generic entering the market, we had hopes, believed, that they would enter around that discount rate, and they did.
They entered around the 20%. So what we've seen is that brand loyalty remains. Even with a 20% discount, they still want to remain on EMFLAZA, and so these, these programs we've seen have had success. Looking forward, I think the team is, is working incredibly hard to keep each patient on brand, each patient on EMFLAZA. I think where you might start to see a shift in this situation is more generics entering the marketplace, because then you start to see deeper, deeper discounts occur, and it becomes a little bit harder to maintain patients on branded product.
Sure.
The team is gonna continue to work exceptionally hard to do that, and so far, we've only seen one.
So when you say you're contracting, part of that contracting, I'm assuming, is agreeing on a certain price point to keep that, to keep EMFLAZA the preferred option?
Not so much of a price point, but more of a discount point.
A discount point?
Yeah.
Right. And so you're saying that with one generic competitor, you've been able to maneuver around that pretty successfully, but there's a potential that as more generics come, that becomes more challenging to do over time?
Potentially, yeah.
Okay.
We'll continue to look at it. We still have some additional levers in place that we can pull if, if more generics enter, and we can look at that business. But where we stand today we've got the right measures in place.
Okay. There's a company called Catalyst Pharma, which, which has its own steroid branded, that got approved. Have you heard anything from your field force about any impact from that?
Limited at this point. I think it's very early days, so it's hard to comment too much.
Yeah.
I think there's a couple of factors at play here, and, you know, from what we've heard from physicians, if you look at their label and the data package in their label, it's based on a study in four to seven-year olds over 26 weeks. I think EMFLAZA has one of the most extensive data packages in DMD, outside, of course, Translarna. And from that perspective, it has 10 years of real-world evidence. There's long-term studies. It looks at a number of different motor benefits, safety benefits across the board. We also have the fact that EMFLAZA's in the AAN guidelines. We have the robust, you know.
Cares program we just talked about. So I think the real-world experience, the fact the team's been in the field for over five years, building those relationships, establishing themselves, we haven't seen a major impact at this point, particularly with, you know, if you look at the product profiles, there's no major benefit over EMFLAZA. And so from that perspective, early days, but we've not seen much.
Okay. That's good to know. So maybe let's go, in the few minutes we have left, touch upon PKU.
Sure
Because that's an area that you guys have been spending a lot of time preparing for. So, maybe can you just give us an overview of the PKU program, where it stands?
Then we can talk about your expectations for the upcoming launch.
Yeah, absolutely. So incredibly excited about the PKU program. Our drug, sepiapterin, had incredibly strong data from the phase III trial, where we had over 60% reduction in phenylalanine levels in all patients, a 69% reduction in the more severe classical patients. We were able to show that in patients who've been previously treated with KUVAN, that we had significantly greater, almost 50% greater reduction in phenylalanine levels with our drug compared to KUVAN. Strong safety profile, and then the data we're collecting now in the long-term extension study, including the Phe tolerance program, we're showing that patients are able to liberalize their diet.
We have now over 60% of the patients who are going through that protocol are able to have protein intake above what would be recommended for you and I, and still maintain control of phenylalanine levels, and that's really the holy grail for PKU patients. So this has generated a tremendous amount of interest across patients, across physicians, across the world. It's a population of over 58,000 patients, the vast majority of which, probably close to 90% of which, are not served by current therapies. So it's really a very large unmet medical need. We have submitted the European marketing authorization in March. We expect to get validation, and the procedure to start for the, approval review in the next week or so. We expect to submit the NDA, no later than the third quarter.
We had mentioned we're just waiting to complete a mouse study, which will be done in June, and.
Look to submit the package shortly after that. And so we are incredibly excited about this program, right? This will be our first global launch, as we're also preparing submissions for Japan and Brazil this year. And our teams are ready and waiting, and I'll let Kylie talk a little bit about the specifics of how we're thinking about the launch plan.
Yeah, absolutely. So you know, one of the things we've talked about is this is a well-trodden path in PKU, and in many senses, with such a strong data package that Matt just outlined, it's a good thing. So there's we're not going to look for patients, which is challenging and costly. You know, newborn screening is widespread across most major markets, so patients are identified at birth. Treatment centers are well known, and their multidisciplinary care teams engage with the patients very frequently. Because if you think about it, you've got geneticists, prescribers in the U.S., and then outside of the U.S. is pediatric metabolic specialists.
But in addition to that, you've got the dieticians, and they play an incredibly important role in these patients' treatment journeys and disease journeys because they need to manage that Phe-restricted diet on an ongoing basis, and that continues whether they're on therapy or off therapy. In addition to that, the disease is well documented, and it's clear from a market access point of view, what's needed to be able to establish treatment benefit and value. And then lastly, it's a well-coordinated and connected patient advocacy community. So across the board, the teams have been engaging for a number of years now with the key treatment centers of excellence, and, you know, many of those top physicians were investigators in our APHENITY study.
The teams have also been engaging with the patient advocacy groups and, being present in a number of these conferences, and we've really seen a very steep and quick pull coming from the market, in the sense of both physicians and patients wanting to see when they can get access to sepiapterin and really looking forward to, as Matt was saying, not just a Phe reduction, but a potential to liberalize that diet, because that means everything to these patients and physicians.
Okay. So, a couple of questions. Do you think you could get, similar to what I just asked about Translarna, a priority review because it's an unmet need area?
We certainly have all of the justification to support the priority review, and we'll request it as part of the application.
Yeah. Do you think something like this application would also necessitate an outcome?
I don't think so, because as you're in the realm of precedent, the FDA has been very clear on what and has a very clear understanding of the thresholds for approval for these drugs. You know, we clearly clear those bars.
Yeah. Okay, that's good to know. And then for the commercial prep, how big of a infrastructure do you need?
We have the infrastructure in place, so we're not planning to add infrastructure, and we have it across all elements of customer-facing teams. So we have it from, you know, key account managers that engage with the physicians, the patient engagement team that engage with the patients, and also the patient advocacy groups. We have our market access teams in place that engage with payers, and they've already begun those discussions to understand what's required from a payer point of view, and we have everything in hand.
Which is very reassuring. And then also our medical team, the MSLs, that also engage with physicians. So infrastructure's in place, we're not planning to add to it, and hence we're in a good position to really hit the ground running upon approval.
Okay. So, you know, if you get approved on a Tuesday, technically, you could launch on a Wednesday, basically. Unless you need to print out your labels or whatever.
Yes. Yeah, there's a few.
We're ready to go.
There's a few logistics to go alongside it, but yes, we will be as quick as we possibly can.
Okay.
Yeah.
What portion of the population do you think would be the earliest on onboarders?
It's a good question. I think you'll see it coming from across the board. That's what we've heard from physicians and patients alike. I think from our perspective, we always saw those that are therapy naive, who want to move quickly and see if they could get onto the therapy because they think the nobody wants to remain on a Phe-restricted diet alone. It's burdensome, and it doesn't give you the outcomes that you're looking for.
So, you know, therapy-naive, classical PKU, that have had nothing else, that have been able to access, I think that's one. Another that we thought through is those that have previously tried KUVAN but failed, not had a response. I, I think one that was a little bit of, not. I wouldn't say a surprise to us, but something that we didn't expect in the near term, but is something that physicians have pushed very hard with us on, is those that have had a response on KUVAN and are "controlled," quote-unquote, but could use additional control in the sense that, they could actually gain another 100 micromolar per liter Phe reduction and be able to potentially liberalize their diet.
That is a big deal, and we didn't think that that would be a near-term patient segment, but physicians have said loud and clear that that would also be a segment that they would look to, to start patients on.
Okay. So in aggregate, all those subgroups that you just mentioned, do you have a sense of what percentage of the total PKU population that would be?
So we think we have an ability to target the full PKU population. I think one of the things that we've talked about, and Matt alluded to it in his opening, is that we believe we can easily achieve north of $1 billion in market opportunity. So another way to answer that is, if you look at the 58,000 patients globally, as Matt said, there's less than 10% that are well controlled on current therapies. All it would take to sort of take PALYNZIQ -like pricing and achieve north of $1 billion is around 15% market penetration.
Okay.
Now, we're not aiming for that. We can do much better than that, but just to help frame how easy it is to get to that point.
Okay. And then last question is, the steepness of the launch, how should we be thinking about that?
Yeah, look, I think one of the things that PTC has done in the past and will do again with PKU is not only look at moving quickly in markets where we'll be securing pricing and reimbursement, but also name patient programs and early access programs. And with either an E.U. approval or a U.S. approval, there's a number of countries that we can move quickly with that, with Middle East, North Africa, Latin America, parts of Europe, etc. So we'll do that. But what I would say is in the U.S., if we look at that as a.
A standalone country, we're able to move very quickly. As I said, the framework for launch success is there, and from our point of view, it's about differentiating, and we think we have the package to do that. Obviously, when you look at ex-U.S., it takes a little more time because of individual country-by-country pricing and reimbursement negotiations, but we'll be able to move quickly with name patient and early access while we negotiate.
Okay. With that, we're out of time. Thanks, Kylie, for talking very fast.
No worries.
Answered the question. Thanks everybody for joining us today. We hope you enjoy the rest of the conference, and thanks again.
Thanks. Thanks.