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On Tuesday, 10 June 2025, Vertex Pharmaceuticals (NASDAQ:VRTX) presented at the Goldman Sachs 46th Annual Global Healthcare Conference. The company outlined its strategic initiatives, focusing on innovation and market expansion. While Vertex reported positive momentum in its cystic fibrosis (CF) franchise and new product launches, it also acknowledged challenges, including the impact of an illegal copy of its medicine in Russia.
Key Takeaways
- Vertex raised its 2025 revenue guidance, expecting 8% growth at the midpoint, driven by CF treatments.
- The company is facing a $200 million revenue impact from an illegal drug copy in Russia.
- New launches, including AlifTrex and Jornavix, are progressing, with significant market expansion plans.
- Vertex is advancing in kidney disease and type 1 diabetes, with pivotal trials underway.
Financial Results
- 2025 Revenue Guidance:
- Lower end raised from $11.75 billion to $11.85 billion; upper end remains at $12 billion.
- Implies approximately 8% growth at the midpoint.
- Growth Drivers:
- Primarily driven by the CF franchise, including the AlifTrex launch and Trikafta expansion.
- Building momentum with Kashyvi and the new launch of Gernavix.
- OpEx:
- Guidance remains between $4.9 billion and $5.0 billion for the year.
- Expect a slight increase in Q2 related to Phase III program launch preparation.
- Impact of Russian Illegal Copy:
- Expected $200 million revenue impact, already accounted for in revised guidance.
- $100 million impact in Q1 ’24 to Q1 ’25, with the remaining $100 million mostly in Q3.
Operational Updates
- CF Franchise:
- AlifTrex: U.S. launch underway, approval in the UK, positive opinion in Europe.
- Trikafta: Expansion into lower age groups and ultra-rare mutations.
- Planning for VX-828 combination in CF patients before the end of the year.
- Kashyvi:
- Momentum building; number of patients initiating cell collection has doubled.
- Gernavix (Acute Pain):
- Broad physician writing across various types of physicians.
- Positive feedback from both physicians and patients.
- Secured an agreement with one of the three major PBMs for access; advanced conversations with the other two.
- Pipeline:
- POV in IgAN: Phase III interim analysis cohort enrolled; potential filing for accelerated approval in H1 next year (if positive).
- POV in membranous: Phase II/III study to begin in H2 this year.
- AMKD (enaxaplin): Phase III enrollment to complete this year; potential accelerated approval filing (if positive after 48 weeks).
Future Outlook
- Next Verticals after CF and Pain:
- Kashyvi: Expected to emerge into a multi-billion dollar product over time.
- POV (kidney disease): Renal diseases (IgAN, membranous, ADPKD) seen as a significant growth area.
- Type 1 diabetes: Potential long-term pillar with VX-880.
- Jornavix:
- Expanding into chronic pain (peripheral neuropathic pain).
- Diabetes Program (VX-880):
- Pivotal development with 50 patients to be enrolled and followed for a year.
- Potential initial approval with further expansion based on safety and tolerability of immunosuppression.
Q&A Highlights
- Alevtrac Transition:
- Slower pace compared to Trikafta due to smaller patient group and increased liver monitoring.
- Expects majority of patients to switch to Alevtrac over time.
- Policy Dynamics:
- Monitoring Medicare and Medicaid exposure within the CF franchise.
- Waiting for Gernavix to be added to the No Pain Act list.
- Pain Strategy:
- Focus on long-term value and cost-effectiveness with Gernavix reimbursement.
- Seeking a broad peripheral neuropathic pain label for Jornavix, meeting with the FDA this summer.
For more details, please refer to the full transcript provided below.
Full transcript - Goldman Sachs 46th Annual Global Healthcare Conference:
Unidentified speaker: Launch, we are now seeing the building of momentum as we build up that franchise. And then most recently, the launch of Gernavix in acute pain. We’re keenly focused on those three launches. As we also think about the next wave of innovation that’s either in or nearing Phase III development. There are three actually four programs that are already in Phase III development, and the one, POV in membranous is coming very soon.
And then as I look beyond that, we have programs like ADPKD in Phase II. We’re making rapid progress with the Nav 1.7, which could be used alone or in combination with our Nav one point in acute or chronic pain. And we’re also making progress, for example, on things like improved conditioning for Kashyri. So the pipeline is keeping us quite busy. And then as I look at the company and round it out, we’re also scaling and growing to get to patients in The Middle East, to get to patients in Asia with our growing portfolio.
And I really like what we’re doing with regard to our balance sheet and how we’re thinking about capital allocation. Lots going on across the company, not just in launches, not just in the pipeline.
Salveen: Maybe just to go forward on your comment on capital allocation. How does business development or that lever play a role in the company right now? We saw you do a decently sized acquisition recently, but is that what we should expect from you going forward?
Unidentified speaker: Yes. I’m really going to sound like a broken record, Salveen. We really stay close to our knitting. Our approach to capital is to invest heavily in innovation, be that internal or external. That doesn’t change.
We have a share buyback program and you saw us re up that a couple of two weeks ago. That’s fundamentally how we think about it. And as we think about going forward, the balance sheet is strong. We have the flexibility to do what we think is right. But our strategy and what we are prioritizing doesn’t change one bit.
Salveen: Great. I want to ask you about 2025 guidance. So you recently raised the lower end of your revenue guidance for 2025, implying about 8% growth at the midpoint. Walk us through in a little more detail the key growth drivers here, particularly in the context of these two launches, but also how you’re thinking about the OpEx aspects of your balance sheet.
Unidentified speaker: Yes, absolutely. So as you heard me discuss on the Q1 call, we are upping the lower end of our guidance from 11.75 to eleven point eight five and the upper end remains at 12. So we’re going from 11.75 to 12 to 11.85 to 12. And as you point out that implies an 8% growth at the midpoint. That comes from largely CF at this point.
It is our biggest franchise and it continues to grow. And it’s growing as we serve more patients with AlifTrex. The U. S. Launch is underway.
We have an approval in The UK and positive opinion in Europe with the CHMP. We’re waiting on those launches to go on. We’re also continuing with Trikafta down into lower age groups and into the expanded population, those with ultra rare mutations. And lastly, we have growth coming through geographic expansion, think Brazil. So that’s through the CF franchise.
Then we have Kashyvi. What we talked about is that this year, we are seeing building momentum. We have about ninety patients who’ve had a initiation of their cell collection. We have doubled that who have started the process. So they’ve been referred to a transplant center.
They’ve been entered into the Vertex system to start to set them up for a slot allocation. So clearly, there’s some growth coming from Kashyvi. And then there’s the new launch of Jirnavik. And what we’ve said there is the half of the year, you should see prescription growth. And then as we secure access and reimbursement, you should expect to see revenue growth in the half of the year.
As it pertains to OpEx, the guidance is 4,900,000,000.0 to $5,000,000,000 for the year, and we’re on track for that. And what we talked about was it was kind of evenly spread in the first half and second half of the year. You should expect a little bit of an uptick in Q2 as we get ready to launch that Phase III program and then spread out through the half of the year. So that’s sort of what the inside scoop and guidance both on revenue and OpEx looks like.
Salveen: Great. Policy dynamics, I know Vertex doesn’t really have as much exposure as some of your peers, but could you just touch on the policy environment and how you’re positioning yourselves around it with regard to not just CF where you have Medicare Part B and mostly Medicaid exposure, but the pipeline at large?
Unidentified speaker: Yeah. It’s a very fair question, and it’s a really tough question to address because we don’t have a lot of facts and we don’t have a lot of detail of what the policy considerations might be. But if you try to draw a picture, and I’ll keep this contained to how I see this for Vertex, maybe what we can talk about is what is the exposure in the various segments. Insofar as CF is our biggest franchise, I’ll speak more on that. But I’ll just take off the let me at least take the swipe at and a pass at Kashyabi.
Kashyabi is a type A drug. So it’s in its own sort of basket, I’ll put it aside. And Gernabix is just getting going, so I won’t comment too much on that. On the large franchise, which is CF, maybe the points that are helpful for you to know are we have about 9% exposure to Medicare, about 23% exposure to Medicaid. So whatever it is that we’re talking about, whether it’s changes to Medicaid or perhaps an MFN type implementation of a CMMI demo project, we’re talking about it in the setting of Medicare or Medicaid.
Beyond that, other than MFN as a construct, it’s very hard to know what we’re talking about beyond that. But if we’re looking for some clues, maybe we can go back to 2020 when in the Trump administration there also was an MFN construct. At that time, what it was was Medicare. It was part B, as in boy. And it was the top 50 drugs.
We have no Medicare B exposure because we don’t have any injectables. So there’s a lot of detail that still needs to be fleshed out. But what we are hearing from DC more is a focus on the bill that’s going through. And MFN is not in that bill. So that’s sort of where we are from our vantage point.
Salveen: Great. Let’s dig into Alevtrac with the launch recently. Maybe speak to the early launch progress with the drug, including reimbursement dynamics and physician feedback. But specifically,
Unidentified speaker: if
Salveen: we could silo look at the various silos across these different patient groups that you’re targeting.
Unidentified speaker: Yeah. So Lift Truck is the CFTR modulator that we have brought forward. David Altschuler and that San Diego Team has been busy at work. I’m going to tell you a little bit about the launch of Elliptrex. But while we’re still launching that and haven’t yet even gotten approval in the EU, we’re already planning for the next molecule to go into CF patients.
That’s the VX-eight twenty eight combination. And I do expect that that will be in CF patients before the end of this year. So with Liftrec, we’ve talked about the fact that there are three categories of patients that we see who may want to avail themselves of this medicine. The is patients who are just plain naive to CFTR modulators. In large part, those are patients with ultra rare mutations.
The Elliptrex label has even more mutations than the Trikafta label. So 31 more mutations, that translates to hundreds of patients more in The US, and it will translate to thousands more in Europe because there are more ultra rare mutation patients in Europe. So that’s the category. Unsurprisingly, the uptake there is the fastest because they’ve been waiting for medicine to treat the underlying cause of their disease. The category are patients with CF who are discontinued from either Trikafta or a previous medicine.
So they have CF, they’re known to the health care system, they have a doctor, and at some point they were on a CFTR modulator. But for whatever reason, either twice a day was inconvenient to them, or they could not tolerate it. Maybe they had a rash. Maybe they had another tolerability concern. They may want to come back onto medicine.
And we expect that if they do, they’re going to want to come back onto a left track. And then the category are the bulk of the patients. They’re on Trikafta, and patients love their Trikafta. And it is an amazing medicine. We’ve never forced switching, and we are not forcing switching here.
If patients want to be on Trikafta, it’s a Vertex medicine, it’s a terrific medicine, we’re very happy for them to be on there. That being said, CF patients are very highly educated group of patients. They know their specific mutation. They know that the malfunction is in a protein called the CFTR protein. And they know that the direct readout of the functioning of that protein is sweat chloride.
In that regard, the LiftRec medicine has even better sweat chloride results than Trikafta. And the doctors are very well educated on this. And so I do expect that over time, the majority of patients will switch to a left track because they’re going to want to avail themselves of the best available therapy.
Salveen: Could you just touch on why the transition hasn’t been at a faster pace than maybe we’ve seen in the past?
Unidentified speaker: And
Salveen: how much of that might be driven by the liver testing?
Unidentified speaker: Yeah, yeah. So really good question. I think one part of this is what is the expectation and how do we model the transition of patients? And it’s very fair to say, well, we’re going to model it based on what happened with Trikafta. But we’ve got to remember that when patients were moving to Trikafta, it was 2019.
The best available medicine before that was Symdeko. Symdeko was available for about sixty percent of CF patients. The next medicine, TRIKAFTA, was available for about ninety five percent of CF patients. So the thirty five percent of patients in our previously described category one, patients who just didn’t have anything for them. Now we’re talking about 31 mutations and a few 100 patients.
So that group of patients is smaller now that Trikafta is in the market and as we think about people moving to a Liftrex. For those who are on Trikafta and want to think about switching to a Liftrex, there is more monitoring. So as Salveen points out, in December of twenty twenty four, when Trikafta was approved for expanded label, more mutations, it also had increased liver monitoring. Because at that time, the agency was also looking at some post marketing data. It was about 165,000 patient years, which is an enormous number for an orphan disease, real world data.
And in that one hundred and sixty five thousand patient years, there were eleven cases of LFT abnormalities. And in that setting, the agency said, we want to change the liver monitoring from when you start on a CFTR modulator like TRIKAFTA, we want you to instead of doing quarterly and then annually, we wanna change that to once every six months for six months, once a month for six months, then quarterly, then annually. So there’s increased monitoring in those six months. And even though this comes from TRIKAFTA data, it was also applied to a Lift Truck. Now interestingly enough, it doesn’t actually affect the TRIKAFTA patient population because almost everybody who’s on who’s eligible for TRIKAFTA was already on TRIKAFTA.
And this monitoring change is about new patients. So it does have an impact on the LiftRec. And so people who want to make the switch have to think about, do I want to do monthly monitoring for six months? And if I want to do it, how do I do it? Do I have to go to my CF center?
Can I do it at home? Can I do it at a nearby lab? What does it entail? Is it just a blood test? Or is it simply is it more than that?
It is simply a blood test. And so I imagine these are exactly the conversations that are going on between patients and physicians as they think about when and if they want to make the transition from Tri to a Lift Truck. As I said, this is a very educated group of physicians and doctors. And I do think the majority of patients will want to switch to a Lift Truck over time.
Salveen: And in one quarter, was about $100,000,000 impact to revenue due to an illegal copy in Russia. Is there any read forward here for the second quarter onwards? Or is there even a broader dynamic that could play out globally?
Unidentified speaker: Yeah. What we talked about on the Q1 call and it is the way I see the world today there is a limited, isolated, only in Russia issue where there is an illegal copy being sold. It is about a $200,000,000 impact. We have accounted for that for the full year. We talked about $100,000,000 was going to be a Q1 ’twenty four to Q1 ’twenty five dynamic.
We talked about the other $100,000,000 being through the rest of the year. Most of that will be in Q3, but all of it will be in the half. We fully accounted for that 200,000,000 and that is accounted for in the revised upward guidance from 11.75 to 12 to 11.85 to 12. We are fighting vigorously for our patents in Russia, But we have made the decision to just take the revenue out. It’s a bit of a perfect storm in Russia.
The war has put economic pressure on the country. There’s a movement towards nationalizing and having manufacturing in country. And in that setting, this is one of the outputs is this illegal copy situation. I will also say that I don’t see this as being anything beyond Russia for the following reason. In all of the Western world, it is a mere 100% patent being upheld when it comes to composition of matter.
And that composition of matter patent is up to 2,037 for Trikafta. In Russia, the way the patent system and legal system works is unlike the rest of the Western world, there isn’t high barriers to this kind of illegal copy. In the Western world, there is the possibility of an immediate injunction, treble damages, etcetera. Those constructs don’t exist in Russia.
Salveen: Perfect. Shifting to pain here, can you walk us through what you’re seeing with the early launch of Joronevax in acute pain, including what you’re observing with scripts, physician feedback, and reimbursement, recognizing you had one of the three big payers on board?
Unidentified speaker: Yeah. I have high expectations from the Jornavix launch. And I will say that I’ve been very pleased with the way the few months have gone. It’s very early days. But what I see that makes me quite pleased is there is broad physician writing for Gernavix across physician types.
Its use is in surgical and nonsurgical settings. The feedback that we’re getting from physicians and patients and I’m actually surprised to be receiving direct feedback from patients. And I get a lot of letters from CF patients, so that makes sense to me. Small community, we’ve been in the space for a long time. It’s a disease where there’s very high advocacy and a lot of knowledge.
And pain in that way is a little bit more diffuse. But I’ve received personal letters from patients who talk about the improvement in their pain management. They might have gone through a left knee. Now they’re getting a right knee. The left knee was done with opioids.
The right knee was done with gernavics. And the feedback is positive. So everything I see on that side looks really good. The one big pushback that people talk to us about prior to the launch was, well, we have no concerns about the fact that opioids are medicines with safety and tolerability issues. We get that.
We need no explanation. We also don’t need any explanation for why non opioids are a good thing and a necessary thing. The thing that we believe is going to happen is payers are going to have a hard time paying for this because opioids are cheap. That is clearly not the case. We’ve had very productive conversations with the PBMs.
As you point out, there are three large PBMs in The U. S. And one of the three we already have an agreement with to make sure that their members have access to the drug. And it’s a very favorable condition for the patient, minimal to no utilization management, no prior auth so people can get the medicine they need. We’re in advanced conversations with the other two PBMs, and I feel very good about those conversations also leading to access for those patients.
So all in all, very early days, but I’m really happy.
Salveen: And how are these discussions, or how is your strategy progressing with regard to preserving long term value as you look to going into chronic pain? And if you could just touch on the No Pain Act in that context
Unidentified speaker: as definitely. So as Stuart and Duncan will tell you, we could get access on day one of launch. But that would not be the kind of value that we would be happy with, and it would not be the kind of value that you would be happy with. We are staunch advocates for our medicines. And we believe that the medicines should the price should reflect the value that the medicines bring in.
The cost effectiveness here is indisputable. We have done analyses. Others have done analyses. And it’s very clear what the benefits are. So we’re strong on that.
And that’s why we are going through the negotiations, making sure that the data are clear, and making sure that Geronavix is reimbursed at a value that we think is appropriate. And that is how those conversations are going. With regard to where are we with Medicare now and the No Pain Act. So you’ll remember December of twenty twenty three, the No Pain Act was passed. And it was implemented in January of twenty twenty five.
And in that the list of medicines that get added to the No Pain list were added. And essentially what this bill, what this act does is it directs CMS to provide an add on payment over and above the DRG in the hospital outpatient and surgical center settings so that there isn’t a disincentive for the use of a branded non opioid pain medicine because there is an extra payment, an additional payment over and above the DRG. So we are waiting for everyone to get into their seats with the new administration to have gernavics added onto the list. I’m 110% confident that it will get onto the list. It’s just a matter of the logistics of adding it to the list.
Salveen: And when you look at expansion here within chronic pain, you’re looking at going after peripheral neuropathic pain. Walk us through the strategy you’re pursuing with the FDA. Obviously, are meetings that are ongoing with regard to getting this on label given the DPN trial and post the recent LSR study.
Unidentified speaker: David, do you want to talk about where we are with getting to the agency and our aspiration for PNP as an indication?
David: Absolutely. So our goal is to get a broad peripheral neuropathic pain label. There are about ten million people in America who have peripheral neuropathic pain, two million of them are diabetic peripheral neuropathy, about four million lumbosacral radiculopathy and the other four million are things like small fiber neuropathy, trigeminal neurology, etcetera. Our goal is to work with the FDA and come up with a path which we don’t know that they’ll necessarily do because no one’s ever done it before, but we believe that it’s justified by the mechanism of action, the biology of the diseases. And so with our DPN study which is already the Phase III study is running, we had the positive Phase II study, and the LSR data we’re going to we’ve been working to design what we think is an optimal sort of LSR approach and come up with our own strategy.
And we’ll be meeting with the agency this summer to try and develop we’ll propose what we think is the best path to getting that broad PNP label. We need to work with them. And sort of the boundaries are they could say well the very traditional approach is do two DPN studies, do two LSR studies, etcetera. But we have maybe what you could consider a more innovative approach we’re going to try and propose to get that broad label. And once we work with them and figure out the solution we’ll share with you what it is.
Salveen: Could you just also touch on the distribution at this point as well as the progress you’ve had with P and C committees and getting in the hospital networks?
Unidentified speaker: Yeah. So going back to acute pain with Gernavix, you know that the distribution that we have discussed in terms of where pain prescriptions come from, let’s call it 50% in the fully retail segment, 50% in an institution affiliated segment. In the institution affiliated, 15, one-five, hospital. 35% is written in the hospital, but you take that script and you go to your local CVS or Walgreens and fill it in the retail channel. So now what we’re talking about is how is it going inside the hospital.
The big milestone to get use in hospital is to go through P and T committees to get that medicine added to the formulary. And where we are is P and T committees have a certain cadence. They work in a particular way. And it takes a particular amount of time. This is not a one day affair.
It’s a multi month process. So we had planned for that. But I’m actually pleased to see that some of the larger hospitals have already gone through the P and T process and have already added Geronavix to their formulary. And I expect that as we get into the half of the year, more of these committees will finish their work and make their decisions about getting Gernabix added to their formulary list. So good wins, maybe a little bit earlier than I had expected and a little ways to go through all of these various P and C committees.
As a reminder, we’re targeting about 2,000 hospitals that ladder up to about 150 IDNs.
Salveen: When you look across your portfolio at this point, what do you think will emerge as the next vertical after CF and pain? And in that context, run us through some of the key timelines for these programs over the next twelve months.
David: Yeah, yep.
Unidentified speaker: It’s a really great question. I think we need to make a little space for Kashyvi. It is a long patient journey. But I do believe that it is going to emerge into a multibillion dollar product over time. So I want to make a little bit of space for that.
I think the next big vertical after that is going to be PoV. And you can look at PoV in two ways. You could say the next big vertical is going to be kidney disease. And I think that’s fair, because we have PoV in IGAN. That has already enrolled its phase III interim analysis cohort.
We have to wait for thirty six weeks. If positive, we will be set up for a filing for accelerated approval in the first half of next year. PoV and membranous is going to start its Phase IIIII study in this second half of this year. And then there are more potential PoV indications that might emerge from the RUBI-three and RUBI-four basket studies. You can also look at it as renal is the next big pillar, because IgAN is a renal disease, membranous is a renal disease.
We have AMKD, enaxaplin. That phase three program will also complete its enrollment for potential accelerated approval this year. That program, we have to wait for forty eight weeks. If that’s positive, then we’d be set up for an accelerated approval filing there. And then there’s ADEPKD, autosomal dominant polycystic kidney disease, which is in phase two development.
So either way you look at it, it’s povy and kidney disease. And it’s quite interesting because people think this is happening because I happen to be a nephrologist. It is not. It is simply where the sandbox took us.
Salveen: And you will have some data at the upcoming diabetes meeting on your type one diabetes program. Do you want to just frame where you stand with the long term follow-up there and then the additional programs that are progressing?
Unidentified speaker: Yeah, definitely. Maybe I’ll ask David to talk about that. Interestingly enough, David is the endocrinologist in the room. And we often get asked what our favorite program is, and I won’t reveal it. I’ll just say I’m a nephrologist and he’s an endocrinologist.
David: Well, we love them all. But the type one diabetes is a terrible disease. There’s about four million people in The U. S. And Europe.
There’s about at least ten million diagnosed worldwide. And it is a disease where despite one hundred years of treatment with insulin not only is it that despite modern technology only about twenty five percent of people actually achieve the target glucose control but it’s just also a burden of every minute of every day having to manage your blood sugar. So the idea of a curative therapy where someone can have a one time treatment and no longer have to live that way is truly transformative. VX-eight 80 is the such program. It’s in pivotal development.
And it is, there’s sort of two components. One is the cells themselves, to be able to make manufacture cells that are human islets that can restore function. And the other part which I’ll get to a minute is the improved modulation of the immune system so the cells don’t get rejected because it’s an off the shelf product. It’s not autologous. The results for VX-eight eighty which is using standard immunosuppression are actually quite remarkable.
I think that if someone had said five years ago that we’d have we’ve described and there’ll be an updated ADA, but the last description 12 people treated. Nine out of the twelve had were off insulin with normal blood sugar and the other three had reduction or were stopping insulin as well. And that and also the original enrollment of this group was severe hypoglycemia which is life threatening and they didn’t have any more hypoglycemic events. So that’s now in pivotal development and we will we’ve agreed with the agency that we’ll enroll 50 people. We’ll complete that midyear and then we’ll follow them for a year.
So that’s for the potential approval. And then really it’s about reducing the sort of safety and tolerability of standard immunosuppression with tacrolimus and sirolimus which we’re using. And we have multiple approaches we’re working on for that. One is just using some of the innovation in immune modulation that’s out there but also hypo immune gene editing and devices. So when we look at it we see the initial approval could be not that far off having enrolled all of the patients But then the ability to expand it will really be just about one, demonstrating its value and two, just making increased safety and tolerability of immunosuppression.
So it may not yet be the pillar, but it’s going to be a big pillar once we get it really going.
Unidentified speaker: You can start process of making the pillar.
David: Exactly. The pillar is growing. But with 4,000,000 people and a cure, it could be a very big pillar down the road.
Unidentified speaker: And Salveen, just to mention, at the upcoming ADA, there is going to be an investor event. You can expect to see at least one year follow-up data for the 12 patients dosed in the study. That’s to say the Phase III before it was converted to Phase III III. And you should expect to see some of the steering committee members and practicing physicians also be present to address how they see this.
Salveen: Great. Well, with that, thank you so much.
David: Thank you.
Unidentified speaker: Salveen, thank you. Thank you.
David: Thanks, Salveen. Thanks, everyone.
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