Travere Therapeutics, Inc. (TVTX) May 13, 2026 Conference Transcript

Welcome to this, our second day of our Bank of America annual Las Vegas healthcare conference. On a very toasty day, I am pleased to invite up here on stage with me, Trevier Therapeutics, Eric Dubé, President and CEO, and Peter Herma, Chief Commercial Officer. Eric, Peter, thank you so much for joining us. Thanks for hosting us. Pleasure to be here. Maybe let's start broadly for investors who may be somewhat new to the story. Eric, can you provide a brief background on Trevere and Filspari?

Yeah, absolutely. And you can go on to our website for a pretty extensive corporate deck with background information as well as forward-looking statements. Trevere Therapeutics is a commercial stage rare disease company. We've been focused on developing innovative therapies for rare kidney diseases that really have been devastating for both IJ nephropathy and FSGS communities. And we're very pleased to really pave the way for innovation and hope for these communities. We also have a phase three program in classical homocystinuria with a pegylated enzyme replacement therapy, pegged abatinase, that is now currently enrolling phase three. And we're really a company that's excited about bringing now the first FDA approved therapy to the FSGS community. This is a really devastating, rare kidney disease. We received FDA approval 30 days ago, and I'm sure we'll be talking about some of the dynamics of that launch. But this has been really a passion of our company and really one that we've stuck through because we know how important a therapy is for these patients that have just a rapid progression to kidney failure.

So obviously, I think recent attention has understandably been centered on this approval in FSGS. So recognizing the launch is still early, what are you seeing so far in terms of initial demand? And how would you characterize early physician awareness of this monumental approval?

Yeah, I would say overall, there has been overwhelmingly positive feedback from the community. To Eric's earlier point, this is the most progressive glomerular disease. and there were no approved therapies so far so this was highly anticipated by physicians i just came back from the national kidney foundation conference last week with over 3 000 participants and the feedback was highly consistent and overwhelmingly positive you know the fsgs label specifies patients without nephrotic syndrome i think there was some concern here initially but why might it not be that big a deal yeah let me start with saying like it's for a broad patient population it's it's treatment of proteinuria in fsds patients broadly independent of etiology that are not currently in a nephrotic syndrome state and nephrotic syndrome is a dynamic state it's not a static state patient can be in a nephrotic syndrome at one point but with treatment of, for example, diuretics or with steroids, patients stabilized with having remaining high proteinuria, the patient still remains at high risk, and that's where Phil Spary comes in, which is very consistent to how physicians treat today and also very consistent to the Global Guidelines Cadego that was published in 2021.

How challenging do you think it's going to be to educate physicians about sort of managing the system and making sure that patients are in compliance but still, you know, able to receive treatment?

Yeah, I think from a physician perspective, I think there's two elements, right? There's the patient component, there's the physician component. From the physician component, physicians understand it's a common injury pathway. It's really about a polycytopathy independent of etiology that needs to be treated. Physicians understand as well that this is not competing with a steroid, but it's complementary to, and it's more long-term kidney protection. From a patient perspective, this is a highly activated patient community. Many are also connected with NefCure, the patient advocacy organization. So far, I'm expecting that we will see high compliance and persistence, as what we saw with IGA nephropathy, also given the patient services that we are providing to this patient community.

In terms of early feedback, have physicians given you a sense of where they intend to use Filspari relative to ACE-ARBs or the SGLT2 inhibitors?

Yeah, well, first of all, Filspari has a component of inutensin inhibition. So it's per definition replacing ACE inhibitors or ARBs. Or for incident patients, they could go to Filspari directly. As I mentioned before, this is the first approved medicine. So I think Filspari will be really like an inkyl therapy. And to my earlier point, it's about the podocytopathy. So it's hard to say right now, is there a particular subtype where it's used more than others? I think most importantly is that physicians understand the common injury pathway that needs to be treated. And that's what you do with Filspari.

You know, you've guided to faster uptake in FSGS versus IGAN. But what does that mean and sort of how are you measuring sort of your metrics for a successful launch?

Yeah, there's a couple of things to take into account. I mean, if you look at the addressable patient population that we anticipate is more than 30,000 at launch, I'm actually expecting that market to grow quite quickly over time with more biopsies, in particular in secondary FSDS as well. If you compare it to IGA nephropathy, where we anticipate about 70,000 patients addressable today. So you have, let's say, roughly half the patient population. But then FSDS is regarded as the most progressive low malior disease with the highest unmet need. So with IGA nephropathy, we really had to establish the urgency to intervene earlier. That's not needed with FSGS. And on top of that, you build upon three years of brand equity. This is basically the same prescriber phase. Physicians are very familiar with the profile of Phil Spari. Many have already experienced with IgA nephropathy.

So taking those components in consideration, even though it's a small patient population, we do anticipate a faster uptake. in general fsgs patients are generally seen once a quarter so it does that impact the cadence of the launch at all in terms of expectations or is there just over such overall awareness of this drug coming in that that it might not matter as much yeah i think it matters within within context of and often i get a question like do you expect a bolus is there like a high amount of patients that will be treated right away.

And how I answer that in the past is I don't really anticipate that given the frequency that those patients are being seen by the nephrologist in average every three months, it can be more frequently, particularly when they are in a nephrotic state. I mean, sometimes they're in the hospital as well being treated, sometimes with IV steroids as well. But overall, I'm expecting a steady linear update.

Yeah, I will avoid the word bolus moving forward, gentlemen, for sure. With significant prescriber overlap between the two conditions, I guess how much incremental education is necessary both around Filspari and maybe to navigate the REMS reimbursement program? What's needed there?

Like I said earlier, it's a large overlap. It's basically like nephrologists that are focused on glomerular disease, treating both IgA as well as FSCI. So the profile of Filspari is well understood. The nephroprotective nature of the drug, I think, is well understood. The injury in FSDS is slightly different than in IGA. IGA is more about a mesangium disease, while FSDS is more about a potocytopathy. So that's an education. The indication that we just talked about brought FSDS not actively in nephrotic syndrome. That's an educational element as well. And then there's an important dosing component as well. IgA nephropathy, the indicated dose is two weeks, 200 milligrams, and optitrate is to 400 milligrams. With FSDS, it's 400 milligrams for two weeks, and then optitrate to 800 milligrams. So I think those are the three elements that you still need to educate nephrologists on.

You know, you've mentioned that payer feedback has been generally very, very positive. We've talked about the halo effect, and we'll get into that a little bit more when we talk about IGAN. but is it possible that there's a similar sort of positive payer halo effect there that somehow that facilitates use in IGAM?

Well, I've been nuanced at first. I mean, it's four weeks in the longs. I wouldn't say like all the payers are re-immersing from the GAM goal, but what we are seeing, and I mentioned that last week in the earnings poll, is that the initial first pass approval rate for FSDS is higher than what we saw for IGN property. So if there is a halo effect, it's really on Phil Spirey, it's already included in most of the formularies and payer plans. And payers review both at the product level as well as an indication level. So when you have the product already in the payer plans, it's easier to build upon that and to extend that with the indication. and what before it's being uh evaluated by the pnt committees often what you see is that authorization criteria are applied for the other indication and that's i think explains why we see a higher first pass approval rate so far but it's four weeks in launch i just want to nuance uh because like the payer plans and pt committees that's really happening in the next in the next few months well if anyone's going to hold you to that it's jackie on the team jackie so uh looking ahead there are additional therapies advancing for fsgs so how important is phil spari's first to market advantage in establishing share yeah well i think first of all it is really important um but i think even more importantly is like we have quite a long leeway till the next potential product comes to the market and they're not competing i mean it's complementary to what Phil Spirey's mechanism is. I think we're in a very strong position for all the reasons that I just mentioned, to create a very strong position in FSDS. And I think it's great for patients that other mechanistic approaches will be evolving over time. And I think this is encouraging as well, new innovation for FSDS patients. This is definitely a patient community that needs new innovation. So I'm actually encouraged by that.

As these programs mature, should we think about them primarily as potential combination partners, competitive threats, or more of an opportunity to expand the market?

Yeah, I think IGA is a good proxy for that. Are you talking about specifically FSDS or?

Yeah, FSDS.

I mean, if you look at IGA nephropathy, I think that's typically a market that needs to be developed. When we launched three years ago, as I mentioned earlier, that market really needed to be established because historically physicians saw that as a relatively benign disease. and they felt like even with high proteinuria, patients were relatively stable. With the investment that we made in more scientific evidence in the radar data set that was then adopted by the Global Guideline Committee, KDGO led to a much more rigorous and more ambitious treatment target. That's step one, but then you have to create noise as well in the education and more companies being in the market will help to really develop that market. And that's exactly what we're seeing in IGA nephropathy. More mechanistic approaches are coming in. More companies bring firepower to educate the nephrology community. And that's actually what you're seeing right now. That market is rapidly growing and will continue to grow because we're still scratching the surface there.

Yeah, I mean, I think if I can add, Jackie, one of the most important things that I think we all should be focused on as an industry is reaching these patients sooner. I think what we've seen within kidney disease, particularly if we think about the concept of each kidney has about a million nephrons. Once those die, for example, in FSGS, the damage really at the podocyte, the filtering cell, that's it. They're gone. So that's what leads to the progressive nature of these diseases and particularly FSGS. So the idea of being able to reach these patients earlier in their disease, diagnosing them, biopsying them earlier, that really is going to have the greatest meaningful impact on these patients' outlook. So for us to be able to do that is one thing. We're certainly going to be doing that as part of our education campaign and reaching these patients sooner. But that's where, as we have more therapies coming in, more companies coming in, we hopefully will be able to reach them sooner. We're seeing early signs of that in IG nephropathy, and the data that we've generated in first-line use in IG nephropathy clearly show that you have a better response the earlier you reach. That really is, I think, what we should be striving for as an industry.

You know, if you think about how you got here, the parasol working group was obviously very important in sort of establishing protein areas as a really good surrogate endpoint for the disease. And so I'm curious, you know, Eric, as you've kind of made the rounds, has that message sort of been taken up amongst the greater community that the idea of, you know, the willingness to intervene earlier to maintain this proteinuria prevented from getting worse?

Yeah, I mean, I think that that's certainly going to be the approach. We were very encouraged to see the consistent feedback that FDA gave to another company within this space that proteinuria can be used as an endpoint. And I think what we would expect to see is being able to reach patients earlier. The awareness is going to be there. I think the incentive for nephrologists to want to biopsy patients earlier, because that's not that that is a risky procedure. It is a painful procedure. And so I think that's going to help also be able to reach these patients. Hopefully, we'll have more clinical trials within this space. We would expect that now that we have approval of Filspari, that that likely will be seen as the foundational therapy in other clinical trials. So I think that this space should evolve. And, I mean, just look at what we've seen in the IG nephropathy space over the last five years. It's been tremendous. And, you know, let's hope that in the foreseeable future that a patient that's diagnosed with IGAN should never have to see kidney failure. That, I think, with all of the options that we have in innovation coming and how quickly something like Filspari has been adopted within clinical practice, there's no reason to believe we can't see that within FSGS, particularly with something like Filspari helping to extend these patients to be able to get to, for example, a clinical trial if they do progress.

Maybe this is too philosophical, but as you think about how IGAN has changed with the Cadego guidelines moving towards a much more aggressive approach, is that something that's really, I think, resonating with nephrologists and their willingness to intervene in both IGAN and FSGS earlier? Peter, you want to take that?

Yeah, I think that's certainly the case. This is a progressive disease. I mentioned earlier, like often physicians historically thought about proteinuria, well, my proteinuria is stable, let's say 1.5 gram per gram. But the reality that patient is at high risk of progression, it's not a patient that is stable at all. And again, you need to have the rigor of evidence to show that based on the investments we have made, and I think we really spearheaded that early on, that evidence is existing now and you want to build upon that base.

I think one of the easiest ways that we've been able to look at that is what is the median proteinuria level, UPC level at initiation of Filspari. We've seen that continue to evolve to earlier levels that still signify patients at risk, but it's encouraging to see that physicians are adopting Filspari, and we would expect to see with other therapies coming, other classes of therapies that address the immune side of the disease, that we're going to see an acceleration in the adoption of innovation. and hopefully be able to reach these patients sooner with a combination to be able to hopefully ideally prevent them from ever reaching kidney failure.

Let's take that question or that topic to just a deeper level. How much of what you've seen over the past two years in terms of IGANS growth is driven by earlier line use?

It's hard to quantify that, but I think to Eric's earlier point, what we had commented on earlier is that our in the accelerated approval phase we had a proteinuria target of uh 1.5 with the full approval that that target uh was uh eliminated from the indication and from that moment we started to see that the the median proteinuria uh baseline treatment started going to the left to lower levels and that's continuing the case so it's hard to quantify that like how much is driven by the market development but it's it's how the market is developing overall to prevent progression early on and with lower proteinuria targets and that's exactly what we're seeing do you think that same sort of bent towards being much more aggressive earlier is going to help open up the market to branded combination approaches earlier well i think that's exactly right i think the conceptually the treatment paradigm remains the same and what i mean was that it's it's a two-pronged approach historically physicians were treating with a nephroprotective treatment being ras inhibition and then going upstream uh impacting the the immune mediation with steroids but now you have superior options phil spari versus asian arms and phil spari is the only treatment that has shown superiority for a maximally dosed active competitor. And what you see in the other category is that B-cells, complement inhibitors, are replacing the historical steroids. So you have more efficacious and safe treatments in both categories that would allow you to go to the much lower and more rigorous treatment targets.

And when I'm speaking to the guideline committee members, they always tell me as well, we have to take an example for example to to rheumatology where you treat much earlier and much more aggressively and as a nephrology community we have to go in the same direction and that's exactly what you're seeing right now i guess for for both of you what are your expectations as far as phil spari's ability to become foundational in some of the treatments especially as you know the newer sort of more immunologically focused uh assets the april baths come into play Well, I mean, I think, you know, when we look at the guidelines and we look at what thought leaders in the space are saying, it already is foundational that, you know, the positioning within the global guidelines is very clear, you know, and I think reflecting the superior nature of what has historically been foundational ACE inhibitors or ARBs is there. We also have very strong data in combination with SGLT2s, which have demonstrated benefit in IJ nephropathy. And that's what we hear from many physicians saying that really is the ideal combination for foundation. And the guidelines clearly say that it should be used. You know, patients should have a combination targeting the immune system. But I think, you know, perhaps most compellingly is how are physicians changing their behavior now? And Peter can talk about our uptake, given that we now have innovation within the immune space.

Yeah, and one thing to add on that is you have to realize once a patient is being referred to the nephrologist, the vast majority of those patients are already in stage 3. That means that half of their kidney function has already been lost. Half of their nephrons died. And so the first thing a nephrologist wants to do, a nephrologist, is to protect those remaining nephrons. So that's why the nephroprotective treatment is really the foundational component. And then simultaneously, you also want to go upstream to minimize the production of GDIGA. So I think it's a hand-in-hand approach. But again, those patients have already lost half of their kidney function to start there. That's why we call Phil Spary foundational.

I think that's a great segue into some comments you made at the last earnings call that the combined opportunity between FSGS and IGEN could be upwards of $3 billion. What goes into those assumptions, and what are the key drivers here?

Yeah, so if we look at the number of patients that in the U.S. are eligible for Filspari and IG nephropathy, there are about 70,000 patients, and we believe that that's going to continue to grow as we've talked about the physicians really helping to identify these patients earlier. There's growing awareness about this. And I think there's a greater sense of urgency that patients that may be seen as stable or earlier in their disease still are at significant risk over 10 years. And these are, you know, patients that oftentimes are in their 20s or 30s. So, you know, they've got an extended period of time that we've got to preserve their kidneys. So we do believe that that addressable number of patients is going to grow. And with the introduction of other therapies such as B-cell inhibitors, we don't anticipate that that's going to impede our growth. When you look at the last two quarters, when we do have multiple treatment options available for these patients, we've seen our highest number of patients being prescribed Filspari. So that adoption of innovation on both sides of the treatment guidelines is only accelerating. We anticipate that that's going to continue. When we look at the other component of growth for Filspari over time, it's FSGS. These patients have historically been treated with long periods of immunosuppressant and steroids. These patients really have been desperate for something that can be one that is FDA approved, but in many ways is an option that's better suited for them. And that's really where Filspari plays. There's over 30,000 patients today that are addressable, but we believe that with greater awareness, earlier detection, earlier biopsy, that those numbers are going to grow, just as we're starting to see early signs of that in FSGS. And it will be a number of years before other therapies are likely to be approved for FSGS. We are hopeful that these patients have treatment options that could be used with Phil Spari. But at this point, Phil Spari is the option. And as Peter mentioned, the reception from the nephrologist community has been overwhelmingly positive. And we do believe that the uptake, you know, is a sustainable one to be able to reach those numbers. But fundamental to us is a speed of reaching these patients because we know that time matters for these patients and their kidneys.

Maybe just to switch gears a little bit, you obviously have a late-stage development program, as you mentioned earlier, and PEG-debatinase. Could you describe a little bit about the opportunity here? And then, you know, as you think about top-line data next year, you know, what sort of the benchmarks we should be looking for?

Sure. Sure. Well, so classical homocystine or HCU is a genetic metabolic disease. It is part of newborn screening because there is a much older therapy that is approved. It's a betaine for these patients. It's not all patients respond to this and don't tolerate it that well, but that's what allowed it to be part of newborn screening. And unfortunately, as part of newborn screening, half of newborns that have HCU are missed. So the estimates that we have today of about 35,000 patients that are diagnosed, being seen by a specialist, and are not controlled, we believe that that's an underestimate because so many patients may not be diagnosed until later in childhood or early adulthood. For those patients that are not well controlled, you see a toxic accumulation of homocysteine that leads to dislocation of their lens and eye problems, bone malformation and growth of bones. 25% of these patients have an ischemic event by the time they turn 16 and half of them by the time they turn 30. And unfortunately for many of these patients, that is fatal. So there really is a real need beyond how these patients are being treated, which is with a very strict limitation of protein in their diet, a really expensive and terrible tasting protein powder. And for some patients, about half of the patients, they're responsive to high doses of vitamin B6, which is a cofactor for any remaining enzyme that the patients have to be able to metabolize the amino acids in their diet. And so really where pegdebatanase fits in is to replace that native CBS enzyme. It is a pegylated humanized enzyme replacement therapy. And what we hear from patients and from their physicians is really the goal is twofold. One, reduce total homocysteine to safer levels. And the guidelines currently say that the level should be below 100 micromolar. And that leads to your question around what does good look like in a clinical trial. The second goal is really driven by the experience of patients. They want to be able to have at least moments in their life to be able to eat with their friends, with their family, rather than feeling like they have to strictly control, you know, all of the protein. A typical patient will say, I'm able to have 10 grams of protein a day. I'd encourage you, you know, with our focus in our culture of, you know, high protein, everything, that is a reminder for these patients of how difficult their condition is. And many of them say they never get used to it. So we really have two goals in our phase three program. The first from a regulatory standpoint and from a clinical standpoint is to see a reduction in their total homocysteine. In our phase one, two study at the target dose that we brought into phase three, we saw 67% reduction in total homocysteine. 100% of patients in that, at that dose were able to get below the guideline goal. And 50% of them will be able to get even lower at below 50 micromolar. So our goal is to be able to replicate that. And the trial design and timeline and the endpoint is similar in phase three. So we have every confidence that we'll be able to manage that. And one of the key risks in a study like this is patients eating some of the higher protein. Part of what we've done in this trial design is to make sure that we stabilize their diet before they're randomized. The second goal in our phase three program is a sub-study to look at, are we able to protocolize an increase in protein intake for those patients that are able to get control of their total homocysteine? So that less from a regulatory standpoint, but it is incredibly important for this community that's looking for something, not just to manage the risk, but also manage day-to-day living.

Maybe we have about a couple minutes left, but just curious in terms of the overall health of the business. Obviously, Pilspar is doing extremely well, healthy balance sheet. What's the appetite for further business development, whether it's internal or external?

Yeah. Well, I think, you know, first, we're really excited about the three opportunities for sustainable growth moving forward in IG nephropathy, FSGS, and HCU. But we are looking at beyond that. We will be focusing our business development efforts within rare disease, particularly within areas that we really can bring expertise and experience, so rare kidney, rare metabolic disease, and then really looking at proof of concept so we can leverage the clinical development and regulatory experience that we have, as well as the very strong commercial expertise that Peter's team brings. And that's our approach to business development.

Any sort of timelines investors should be sort of thinking about here?

So hard to be able to pinpoint that on business development. What I would say is that our teams are absolutely focused on executing strongly on the launch and in enrolling our Harmony study with Peg de Batonace. But we do have folks that are actively looking for what's next for us.

Well, perfect. I think that maybe just squeeze in one more. You know, look, it's been a fantastic year for you in terms of, again, patient starts, the approval in FSGS. How do you benchmark success second half of this year?

Well, I think, you know, what I would say is that we're going to continue to report on the number of new patients that are prescribed Filspari. So that's the most important measure for us is reaching these patients that really need something better for their kidneys. The other aspect is we are absolutely focused on enrolling our phase three program. So we have guided to top line data with PEG-debatinase in the second half of next year. While we won't be providing enrollment updates, we will certainly remind that that is a key target for us going into next year.

Wonderful. Eric, Peter, thank you so much for joining us. Great. Thank you.