All right, I think we are live. Good afternoon, everyone. Thanks for joining us on the second day of our fifth annual Guggenheim Genomic Medicines and Rare Disease Day. I'm Vamil Divan, one of the senior analysts here at Guggenheim. Arsene is also on the line from the Guggenheim side. We got the full team here from the Travere Therapeutics side for our next session. Thanks so much to all of you for joining us. We have Eric Dube, who's the President and CEO. We have Chris Cline, the CFO, and Peter Heerma, the CCO, along with Naomi Eichenbaum from Investor Relations. Obviously a lot to get to, so I'm just gonna jump right in. Before I do that, for those of you listening in, if you have any questions that you want me to ask, please feel free to email me at vamil.divan@guggenheimpartners.com, and I'll work them into the conversation. I thought what we could start with, we obviously this weekend just had some additional data released at WCN. Maybe if you guys just wanna share, I guess I'll turn this to Eric, if you wanna share your thoughts on the Lancet publication, key takeaways that you took from the data and the confidence it provides you as you know, as we wait for the two year data in the fourth quarter of this year. Sure. Well, Vamil, thanks to you and to Guggenheim for hosting us today. We certainly are excited about the information that we have thus far in the year. It's a very important year for us at Travere, but probably more importantly for the rare kidney community. With the World Congress of Nephrology just completing, we were very excited to be able to provide some additional data from our ongoing PROTECT trial, as well as a simultaneous publication in the Lancet. You know, I think I'll certainly caveat this, that this is an ongoing trial, and this is an interim look at the data. What we've seen so far and what was published really does, you know, give us further encouragement, confidence in what we expect and hope to see, at the two year endpoint of this important trial. You know, certainly what we saw was a very rapid and sustained improvement in proteinuria for these patients, and that really is reflected in our FILSPARI label. We also were able to provide some additional information, in that response of proteinuria reduction. For example, partial remission, over 70% of patients on sparsentan were able to achieve partial remission of less than 1 g/ day, compared to about 44% of patients on an active control irbesartan, and that difference was highly significant. We also saw at a more rigorous measurement of remission, complete remission, those patients that were able to achieve less than 0.3 g/ day of proteinuria, was 21% of patients on sparsentan compared to 8% on irbesartan. You know, this was also very exciting to be able to see this early in the trial and really a sustained reduction in proteinuria, at least up until the point of this analysis. We also were very encouraged to see early trends in outcomes, hard outcomes for these patients, which we know are very important for nephrologists. These are, you know, again, preliminary numbers, but we did see, you know, numeric difference in the reduction of eGFR, kidney failure, and mortality compared to active control irbesartan. You know, that, I think the response at the early response at the meeting was very, you know, positive from the nephrology KOLs and community. One of the questions that we've received quite a bit around the mechanism of action for sparsentan is, you know, are the benefits that we see on proteinuria really tied to a reduction in blood pressure? We were pleased to be able to show the blood pressure data, and there was a very small difference between groups. Essentially, you know, it doesn't, what we believe, explain the difference in proteinuria, meaning that it's not just hemodynamic alone. It's likely well beyond that. You know, our hypothesis based on some of the preclinical data is that it really is based on structural changes that are going on in the kidney. More to come there, but really, directionally, confidence building. Then finally, you know, really around safety, it's important to be able to demonstrate the safety, especially with a known mechanism like endothelin blockade that has for other agents been associated with edema, heart failure, or hepatotoxicity. We continue to see a profile emerge with sparsentan where there are levels of edema, but no cases of severe edema, no cases of heart failure, no cases of hepatotoxicity, or edema-related discontinuations. We also were able to provide some body weight change data in this presentation and manuscript. I think it provides further information in this ongoing trial, and we're well-poised and eager to be able to complete the trial this year and provide additional information at the two year measurement of safety and efficacy in the fourth quarter of this year. Okay, that's great. Then you touched on sort of the early feedback on the data. Kind of ties into my next question, is just sort of the early feedback on the launch, 'cause you obviously got the approval about six weeks ago. Maybe you can just sort of share, you know, your latest thoughts on how the launch is progressing. I know obviously you guys commented how you got your first script filled pretty quickly after the approval. How are things sort of six weeks out, and what's the feedback been from, you know, physicians and patients, both on the sort of process to get on therapy, but also just the efficacy and safety of the product? Yeah. I think, certainly Peter can cover all of those questions. To give a high-level overview, we've been very pleased with the launch performance to date. I've been very impressed with how Peter's team has been able to execute on all levels to ensure that we get out very quickly. We know that these patients have been waiting for very long. We know that time matters for these patients. You know, every launch really is founded in strong execution, and I think Peter's team has been able to demonstrate that from not just day one, but hour one. Peter, do you wanna talk about the early response so far? Yeah, absolutely. Thanks for hosting us today. Couple of things. You already referenced that early prescription that we got, like, basically within eight hours after the approval. I think that speaks to the unmet need, but also the amount of patients that are being seen by nephrologists. I think the team that we have put in place, and we have already a foundation, commercial foundation in nephrology with our cystinuria product, THIOLA, which we have expanded. We have over 80 commercial people in the field right now that have a very good understanding of nephrology, that knows nephrology very well. Our initial focus is to be consistently targeting 6,000 nephrologists that cover about 85% of the rapid progressing IgA nephropathy. To Eric's point, like the early indicators of how the launch is going, we're very pleased with the initial feedback and receptivity of the market. We see scripts coming in. We see repeat scripts. We see a willingness from the payer to cover us. I cannot go into numbers today, but we will be covering that at the next earnings call, where we will provide additional color on what we are seeing with regards to patient start forms, net revenue, as well as progress we are making with the payers with regards to covering FILSPARI so far. One of the questions that we had a lot of initial response from the investor community was like, "Well, what does the REMS mean for initial uptake?" I think having been in the market now for six weeks, once we have the opportunity to really sit down with the nephrologist and talk about the efficacy data as well as the safety data, often the question we're getting is like, "Why do you have a REMS program in the first place?" Once the physicians understand, like, what the implications of REMS are and what the burden, the workload is for them, and that's basically an attestation, that burden kind of like falls away. So far we don't really see too much of a burden from the REMS implication. I think overall the receptivity has been very solid from the nephrology community. Patients are there, the unmet need is being acknowledged, and our field force has a good understanding to have that connectivity with the nephrologist. Okay, that's great. Maybe one quick follow-up on that is just in terms of the patients that are starting therapy, can you talk about sort of, I assume they're all sort of ACE and ARBs, you know, as been discussed before, getting on FILSPARI, but are most of them also on SGLT2s? Also maybe if you can touch on Tarpeyo. Have they already tried Tarpeyo? Just a sense of kinda where the patients are coming from. Yeah, it's a great question. First of all, we're focusing on the nephrologist. All those patients are under the care of the nephrologist, have had the confirmatory biopsy as well. All those patients have been on ACE and ARBs. That's like 90% of the patient population in the first place, I would say even higher. SGLT2 is an interesting one. I mean, certainly we see an increase in uptake of SGLT2. It depends on what physician you're talking with. FILSPARI is certainly seen as the potential for a new foundational care since it has the ARB component, but it adds then also the endothelin component. It blocks two bad actors in the pathophysiology of the disease. Physicians understand, like, where FILSPARI is more early upfront in the treatment paradigm, earlier than, for example, steroids like for example, Tarpeyo. SGLT2, as to what I mentioned earlier, you see a more rapid uptake over the last year and a half with SGLT2, and physicians see the combination of FILSPARI with SGLT2 as the new standard of care because it allows for a non-immune suppressive treatment paradigm for those patients to regress the progression of disease and stabilize those patients. I don't have insights yet on how many patients are or are not on SGLT2. We're still sort of building that evidence as well in our open label extension trial where we allow for SGLT2. So far, very pleased with where FILSPARI sits and how physicians react on where they see the positioning of FILSPARI, which is kind of like upfront in the treatment paradigm. Okay, that's great. What about on the payer side? Maybe you can just talk a little bit on what the sort of initial reaction's been these first few weeks from, in terms of getting patients reimbursed for the therapy. Yeah, absolutely. At the earnings call, we already highlighted that we have had substantial interaction with payers already prior to launch. We had a field force, an account management field force that were active in the field already six months prior to launch, to really educate payers with regards to cost associated with disease, IgA nephropathy, to make that more upfront in their understanding as well as the humanistic burden of IgA nephropathy. Now we're building upon those interactions with the actual data and the label. As we are in the process to get onto the calendar of PDUFA agendas, and inclusion in the policies in the formulary. Right now it's mainly on a medical exemption, and we're seeing good progress so far. I mean, I was talking about early scripts initially. We see repeat scripts as well, and we see claims coming through through the payer. So far, early days, but very pleased with the initial feedback that we are receiving from the payers and the actions they are taking. Okay. One question I got as a quick follow-up here, you touched on the SGLT2s and use there. Just when would we see some more formal presentation of safety data as a combination? I don't know what's the latest you've said there? Yeah. We have provided. Yeah. We Yeah. We did explain it- Oh, go ahead, Peter. Go ahead. Yeah, no, we have provided information from a drug interaction study in healthy volunteers that we believe now demonstrates that it is safe to combine sparsentan and SGLT2s. That really allows us to now look at the combination of sparsentan and SGLT2. We have initiated two studies that will be going on this year, and we'll have data next year. That will be looking at the addition of an SGLT2 on top of those patients in an open label extension for a PROTECT trial, so on top of sparsentan. Then we're doing a separate study looking at the opposite, where we're looking at what the benefit and the safety is of looking at the addition of sparsentan on top of those patients that are on stable SGLT2. We believe that's gonna be important looking at it both ways because we know that there are a segment of patients that are already on SGLT2 or will be referred to a nephrologist already being treated with an SGLT2. You can really expect to see more of these data sometime next year when we complete those two studies. Okay. Great. I do wanna talk a little bit more about the competitive landscape. We touched on Tarpeyo briefly there. There's obviously atrasentan in development, as another ERA, and then other mechanisms, you know, the APRIL, you know, BAFF approaches and others. Maybe you can just talk about how you see this sort of field evolving. We've seen some, you know, great progress in the last couple of years with two approvals. What do you see the role of, you know, ERAs and sparsentan specifically, say, if we look out two, three years from now or maybe four years from now when there might be a couple other players in the space? Yeah. Peter? Yeah. I think it's a very exciting time, in particular for patients. These are the patient population that is on a rapid path on progression that basically have seen no innovation in the last 30, 40 years. I think for the patient really wins with, like, all the new modalities that are in development. To my earlier point where we see FILSPARI is really, like, upfront in the treatment paradigm. I mean, it blocks angiotensin as well as endothelin. Both are known as bad actors in the progression of disease and in the pathophysiology. I think all new modalities that come to market is complementary to FILSPARI. we... I think the two, at least two-year window that we have before new modalities come to the market allows us to create this foundational space that I think we are able to get to based on the mechanistic approach. New modalities could then bring patients to proteinuria levels even lower than what. Okay. All right, great. Just kinda conscious of the time. There's obviously a lot more we could discuss on the launch and on IgAN, but a lot of focus from investors on FSGS also, with data coming here pretty soon. The first question is that one which we're getting. I don't know if you can comment any further beyond the second quarter, in terms of the eGFR data from FSGS, or where do you stand in terms of the process of releasing that? The other question we've gotten along those lines is what should we sort of expect in the top line release, when it comes? We are on track to be able to provide the two year confirmatory endpoint data this quarter. We've not narrowed down more specifically within the quarter, but we are on track to be able to provide that. You can expect that we would issue a release to announce the top-line data that should include enough of the eGFR data at two years as well as, you know, some high-level statement around safety to be able to give a good enough view around the confirmatory endpoint as well as, you know, what that means for regulatory pathway. We'll evaluate what level of detail for additional results, but I would not expect that we will go into much detail in a release. We do believe that this is a very important and highly anticipated trial. It really is the largest controlled trial done in FSGS, we do wanna make sure that we reserve enough of the results to be able to have a top-tier journal as well as to present this information at a medical meeting. You know, our goal is to be able to provide clarity on the results and clarity on the regulatory pathway and timing. We'll look to do that. We recognize that it's a large data set. This is a complex disease or disorder and, you know, we'll look to be able to provide that information very quickly in a medical setting. Okay. I have one quick follow-up. I don't know if you can answer this, but someone's asking if you can talk about sort of last patient visit or anything along those lines to give people a better sense on timing. I don't know if you. Yep care to comment. We've completed that. We're well beyond the last patient last visit and, you know, that was obviously a very important one for us to be able to now collect the data, clean up the data, QC, QA, and start the analysis. You know, nothing further in terms of what that would mean for timing this quarter. Okay. Just in terms of... We, you know, we had a panel yesterday as part of this conference on the renal, you know, market with a couple of KOLs and talking about sort of what level of eGFR, you know, benefit would be needed in FSGS. Maybe can you just talk about this or what you, what do you think you need to show in this trial, either from a statistical perspective or, and/or a clinical perspective to have really sort of meaningful data in this, in this indication? Sure. Well, let's start first with what is clinically meaningful for these patients. If we think about FSGS as the most rapidly progressing of the glomerular diseases, it is, as Peter mentioned, a rare disease with high unmet need. For us to be able to look at how we can slow the progression of these patients to delay their progression to kidney failure, really what we see is that it should be around 1 milliliter per minute per year difference than active control. We have designed and powered the trial to be able to demonstrate at the 90% level of power a low single-digit difference in annual slope. We would well be... You know, if we, if we achieve that, we certainly would have a clinically meaningful benefit that then would translate into, you know, a slowing of the progression to kidney failure. That really is consistent with what we hear from nephrologists, it's consistent largely with how guidelines reflect eGFR slope. We believe that in demonstrating a difference and of a magnitude of one or above, that should be in line with what regulators would be looking for. That's really what we would expect. We'll look to be able to provide that information later this quarter. Okay. All right, great. Maybe just on the commercial side, I think maybe because of some of the other developments in IgAN, there's been a lot more focus on the commercial opportunity there. I don't know, Eric or Peter, if one of you can just... How do you see the sort of commercial opportunity in FSGS, either just on its own or relative to what, you know, what we're seeing in IgAN? Peter, would you like to take that? Yeah, absolutely. Well, FSGS clearly has a very high unmet need as well. I mean, it's an even faster progressing patient population. I was just seeing in market research last night that over 70% of those patients have higher proteinuria levels than 1.5 g/g, so a very sick patient population on a fast path of progression, and less competitive as well. We're speaking about the new modalities in development in IgA nephropathy. We see less of clinical development in FSGS. Given that it's a sicker patient population, we also have a higher dose. It's a more proteinuria disease in general, so there's a dosing difference as well in FSGS. Overall, we see that as a very significant patient population, high unmet need, and certainly a strong opportunity for FILSPARI to penetrate as well. Okay. We only have a couple of minutes left, so I just don't wanna ignore pegtibatinase, where you also have some data coming up here. Similar sort of question, I think, a couple questions on this. One, sort of what should we expect with the update, you know, in the middle of the year here? Kinda maybe you can just sort of frame some sense of the commercial potential. Again, I think that's an area where maybe there's been a little bit less work done, in terms of how to think about the market opportunity. Sure. We're also very excited about the opportunity with pegtibatinase. This is a community, the HCU community that has also a high unmet need for better therapies. As a pegylated enzyme replacement therapy, we believe pegtibatinase is well poised to be able to, you know, really become the new standard of care for these patients. We would expect that in the middle of this year we'll be able to provide an update on the highest dose cohort in our ongoing COMPOSE study, which is phase I/II. We'll look to provide information on there. We obviously have had positive data from cohort 5. We wanna be able to see what incremental benefit we can have at a higher dose. We'll also look to provide regulatory clarity given that we're in the midst of engaging with regulators around the design of a phase III. We, based on where we are today, we're still in position to be able to start that phase III later this year. Those are the types of updates that we'll look to provide in the middle of this year, is really what does that phase III look like. With regard to the population and the opportunity, we believe that based on, you know, conservative estimates, there are about 3,500 patients in the U.S. and similar numbers in Europe that are addressable. There are pockets of patients with classical homocystinuria in other parts of the world, but this is a, I would say, a conservative estimate for U.S. and Europe based on what we know now. We do know that many patients go undiagnosed or undetected despite newborn screening. There has been quite a bit of research in the last few years that reflects that there may be a broader number of patients. You know, we will continue to explore how we might be able to better define and reach those patients. Conservatively, the 3,500 addressable population, we believe that this is meaningful. Again, we would expect that pegtibatinase would become the new standard of care based on, you know, the profile that we've seen thus far. Okay. All right, great. In just in our last minute here, I guess maybe we can get Chris, in here for just a second. In turn, you did the raise, earlier this year. If you can just talk about sort of your current financial position and your cash runway, you know, going forward here. Yeah, sure. Thanks for the question, Vamil. Really, I would say that we're in a very strong position from a financial perspective. We ended the year with $450 million in cash and equivalents in the balance sheet. As you highlighted, we did a recent equity offering, which had gross proceeds of $230 million. When we look at that in aggregate, we believe that that can, you know, support our operations into 2025. When you think about that, it includes a number of these great milestones we've been talking about today. That includes the FILSPARI launch that's ongoing in IgA nephropathy, potential European approval for IgA nephropathy, as well as the DUPLEX study data, potential U.S. and approval and European approvals there as well, in investing in the launch, and then moving pegtibatinase into phase III. We're really in a great position to continue to support these growth opportunities. Okay, great. Unfortunately, you know, we're at the end of the 25 minutes here. It goes quick. You know, definitely appreciate all of you joining us, you know, for the conference. Hope it's been a productive conference. I'm sure we'll be in touch soon with all this, all this data and information coming in the next few weeks here. Thank you Vamil. Thanks so much. Thank you very much. Thank you. Thanks.