Travere Therapeutics at Jefferies Conference: Strategic Insights on Rare Disease Focus

On Wednesday, 04 June 2025, Travere Therapeutics (NASDAQ:TVTX) presented at the Jefferies Global Healthcare Conference 2025. The company highlighted its strategic advancements in treating rare kidney diseases and classical homocystinuria (HCU). While the company expressed optimism about its upcoming FDA advisory committee meeting, it also acknowledged challenges such as the competitive landscape and regulatory hurdles.

Key Takeaways

• Travere Therapeutics is making progress with Filspari for rare kidney diseases, particularly FSGS and IgA nephropathy.
• The company is preparing for an FDA advisory committee meeting regarding Filspari’s sNDA for FSGS.
• Travere anticipates extending its cash runway into 2028, supported by a strong cash position and expected milestone payments.
• The entry of Novartis’ atrasentan is seen as complementary, potentially expanding the endothelin class.
• Travere is pursuing international expansion, with efforts underway in Europe and Japan.

Financial Results

• Travere ended Q1 with a cash position of $322 million.
• The company expects to receive a $17.5 million milestone payment from CSLV-four for full approval in Europe.
• The cash runway is projected to extend into 2028 and beyond, providing financial stability for ongoing operations.

Operational Updates

• The Filspari launch for IgA nephropathy has exceeded 700 new patient start forms per quarter over the last two quarters, with high compliance and persistence rates.
• Travere is working with partners to expand Filspari’s availability in Europe and Japan, with pivotal trial data expected in the second half of the year.
• The FDA has accepted the sNDA for Filspari in FSGS, with a PDUFA date set for January 13th of next year. The focus will be on proteinuria as a surrogate endpoint.
• The company is exploring modifications to the REMS for Filspari, aiming to reduce testing frequency or potentially remove it altogether.
• The HCU program is on track to restart Phase III enrollment next year.

Future Outlook

• Travere expects Filspari to become a foundational therapy for FSGS, addressing an unmet need for over 30,000 patients in the US.
• The company is confident in Filspari’s clinical profile and anticipates it will complement existing therapies in the IgA nephropathy market.
• Travere is focused on expanding its pipeline, particularly in rare renal diseases, with a disciplined approach to upcoming catalysts.

Q&A Highlights

• The FDA advisory committee meeting will likely focus on the use of proteinuria as a surrogate endpoint for FSGS approval.
• Travere anticipates that Filspari’s indication statement for FSGS will be similar to that for IgA nephropathy.
• The company views Novartis’ atrasentan as a complement to Filspari, with Tarpeo and Fabholta seen as therapies for more severe patients.
• Payer access for Filspari has improved, and Travere expects similar policies for Filspari and atrasentan over time.
• Data on the combination therapy of Filspari and SGLT2 inhibitors will be presented, highlighting additive benefits and safety.

Readers are encouraged to refer to the full transcript for a detailed understanding of Travere Therapeutics’ strategic direction and insights.

Full transcript - Jefferies Global Healthcare Conference 2025:

Farzin Hak, Biotech Analyst, Jefferies: Hello everyone. My name is Farzin Hak, one of the biotech analysts at Jefferies in Moriw Baycroft’s team. I’m happy to introduce Eric Dubay, CEO of Trivia Therapeutics. This is a fireside chat format. Thank you for joining us today, Eric.

Eric Dubay, CEO, Trivia Therapeutics: Well Farzan and Jeffries, thanks so much for hosting us.

Farzin Hak, Biotech Analyst, Jefferies: So for those who are new to the story, can you provide a one minute overview of your programs and strategic focus?

Eric Dubay, CEO, Trivia Therapeutics: Yes. So Trivia Therapeutics is focused on rare disease. And our focus currently is providing hope to three rare disease communities by really transforming and upgrading the standards of care. We are in the midst of a launch of Filspari, an IgA nephropathy, with the aim to become the new foundational therapy within that disease. We also have a file with FDA for FSGS, another rare kidney disease, with a PDUFA date of January thirteenth of next year.

And we also have a program that is in phase three for classical homocystinuria or HCU. We’re currently working through manufacturing scale up and we’ll be initiating our enrollment in that Phase three next year.

Farzin Hak, Biotech Analyst, Jefferies: Great intro. So let’s start with the FSGS, and that’s where the investor focus is at. Your supplemental NDA was accepted and you have a PDUFA schedule with an AdCom plant. So since the NDA acceptance and the AdCom request, have you gotten any additional insights from the FDA on why the AdCom was requested and what the AdCom is intended to discuss?

Eric Dubay, CEO, Trivia Therapeutics: So we’ve not heard specifically the rationale that FDA has for hosting an advisory committee. In the acceptance of our SNDA for FSGS, they indicated that they’re currently planning for an advisory committee. And we can only surmise that as FDA typically will call advisory committees where there are questions around the endpoint, questions around the clinical trial when an endpoint is not met, or around the safety benefit risk of a therapy. Now, we’re in a rather unique position where there was an independent public private initiative called PARISOL that worked to evaluate what the best endpoints in FSGS was, given the heterogeneity in this disease. And what that group reported out last fall was that eGFR was not feasible for FSGS, given the heterogeneity of this disease.

It would be very difficult to power and to have a phase three program within two years. What they did find is that levels of proteinuria independently predict a patient’s risk over time of reaching kidney failure. And so even though our phase three, which is the only phase three ever to be completed within FSGS, was designed with eGFR as the primary endpoint for approval. We now know that that would be infeasible. But we do have a very robust data set with proteinuria demonstrating a positive treatment benefit versus an active control irbesartan in that trial.

So we imagine that the advisory committee will be focused on the evidence to support proteinuria, but importantly, looking at the evidence specifically with Vilspari because this Parasol group was looking at registry data. We did not submit our phase three data to Parasol because we wanted to retain the independence of that analysis and the independent validity within our phase three program. So we look forward to, and we will be very well prepared for that advisory committee.

Farzin Hak, Biotech Analyst, Jefferies: Great. Just curious, have there been any personal changes in the FTS cardio renal division handling or application?

Eric Dubay, CEO, Trivia Therapeutics: So we’re not aware of any changes within personnel of cardio renal specifically. We certainly see the same headlines around FDA staffing changes that many of you see. And importantly, the the lead reviewer and the division director, Eliza Thompson, is with the FDA. And in fact, she recently became the division director, whereas previously she was the deputy director. So we can’t foresee what will happen in the future, but we are very pleased with the the continuity of our reviewer staff over the years.

Farzin Hak, Biotech Analyst, Jefferies: Got it. So the parasol, as you mentioned, it says a clear framework for using the proteinuria as an endpoint to support full approval in FSGS. But based on the recent interactions or other actions you have seen from FDA make recently support, what is the direction they’re really heading into?

Eric Dubay, CEO, Trivia Therapeutics: Well, I think, you know, as as I as I mentioned in the first part, PARISOL really was designed to better understand what are the endpoints for clinical trials and for regulatory purposes in FSGS that ultimately predict a patient’s risk of kidney failure. It’s really difficult within a rare disease to be able to power for hard outcomes like kidney failure. What we’ve seen from Parasol, and FDA was part of Parasol, is that proteinuria can, in fact, predict longer term risk of kidney failure. And in fact, for patients that are able to get below certain thresholds of proteinuria, which are consistent also with our phase three pre specified criteria for proteinuria control, patients are able to reduce their risk of kidney failure over eight years by eighty five to ninety four percent. And so it really is a profound independent predictor.

And that was what we believe led to the recommendation of the parasol group that proteinuria can be used as a validated surrogate endpoint for approval. Now we’ve been very encouraged that the FDA provided consistent feedback to another sponsor with a phase three in FSGS that proteinuria can be used for full approval. And certainly, we look forward to the ongoing review of our program.

Farzin Hak, Biotech Analyst, Jefferies: Got it. From the for the AdCom coming up, has FDA given you any guidance yet? And can you book in potential discussion

Eric Dubay, CEO, Trivia Therapeutics: So FDA has not provided us guidance specifically. We expect that we’ll learn more throughout the review of our sNDA. And typically, we’ll learn more about an IADCOM, whether it’s scheduled, when it’s scheduled, etcetera, later in the review. What I can say right now is twofold. One is the review of our sNDA is going along according to our expectations and is very consistent in terms of the types of questions that we received when we had a file under review for the same medicine for IgA nephropathy.

We can also surmise from our Type C meeting where we discussed the submission of this sNDA, where FDA said they recognize now that with the use of ProNeuria and the recommendations from Parasol that we can submit an sNDA for this indication and that their review will likely focus on understanding our Phase III results in the context of parasol. Since that meeting, we have presented data, most recently at the NKF meeting in April, from our Phase III program showing that patients who are able to achieve partial and complete remission have differential levels of kidney failure. If you’re able to achieve those levels of proteinuria control, you have much lower levels of kidney failure within a two year period of our clinical trial. But we also have demonstrated and published within the New England Journal of Medicine the fact that patients consistently across all of these thresholds of proteinuria control, patients on sparsentan are able to achieve far greater rates than active control max dosed irbesartan. So a very consistent result within our trial, and it results consistent with Parasol.

So we do look forward to those discussions, and we expect that that could be an area of focus for the advisory committee.

Farzin Hak, Biotech Analyst, Jefferies: Got it. EMA has been less involved in the Parasol project. How does that shape your view on the EU regulatory path with CSL Wi Fi partner?

Eric Dubay, CEO, Trivia Therapeutics: Sure. So the EMA has been involved in Parasol. They were part, not to the same degree as FDA. And the approach to changing or defining endpoints is different between FDA and EMA. With that said, we have confidence that EMA is considering what this looks like, given the significant unmet need within FSGS.

Certainly our partners at CSLV4 are actively engaged in helping to find a pathway with EMA.

Farzin Hak, Biotech Analyst, Jefferies: And then just clarifying, the seven year market exclusivity for orphan drug, should that trigger upon approval? And this would provide exclusivity into 02/1933?

Eric Dubay, CEO, Trivia Therapeutics: That’s right. So we expect, given that we have orphan drug designation for FSGS, that we would get a term of exclusivity that runs in parallel, and that would be seven years from approval. So assuming approval in January of next year, excuse me, that would get us into 02/1933. But we also expect to receive a six month pediatric exclusivity that would extend beyond that.

Farzin Hak, Biotech Analyst, Jefferies: Okay. And then what are your base case assumptions for what the FSGS market opportunity would look like? And what do you expect the fill spray label to look like for FSGS in that context?

Eric Dubay, CEO, Trivia Therapeutics: If we if we think about the opportunity, I I think it’s important to keep in mind what these patients face. So the average patient with FSGS will face kidney failure within five to ten years. These patients oftentimes have degrees of proteinuria that are higher than any of the other rare kidney diseases, higher than chronic kidney disease, and is so severe that oftentimes they have symptoms of edema, facial edema, etcetera. This is really a significant burden for the patients, for their family, and for the health system. There’s nothing that is approved for these patients.

What’s worse is, given the heterogeneity of this patient population, most of these patients do not respond to the most commonly used therapies, immunosuppressants like steroids and tacrolimus. And so there is a desperate need for something that treats the underlying condition within the kidney. What we see with Filspari is a consistent benefit in reducing proteinuria of around fifty percent and the only medicine that we’ve seen in a clinical trial to get these patients into complete remission. And so when we look at what the opportunity is, it’s really to address and become the new foundational therapy by directly addressing the over activation of angiotensin and endothelin. When we look at patients with primary and genetic FSGS, there are over thirty thousand of these patients within The US that are under the care of a nephrologist, that are diagnosed, and that are not already in dialysis or in kidney failure.

So this represents a significant opportunity for us to really change the lives and the outlook for these patients. We believe that based on the feedback we’ve received from nephrologists and from market research that we’ve done, that there will be a very rapid uptake. And that’s based not only on the unmet need, but also in the high recognition that these patients need to lower their proteinuria. This is different than IgA nephropathy, where nephrologists are trained historically that IgA nephropathy is a slowly progressing disease that represents a low risk of kidney failure within a patient’s lifetime. It’s clearly not the case, and it’s certainly not the case with FSGS.

And so we expect there to be rapid uptake. There also are no therapies on the near term horizon for other treatment options. And so Filspiri could be the only therapy that’s available for these patients for a number of years. And at double the target dose for adult patients, this represents a significant opportunity for us to transform not only the community but also for us to transform Trevere. With regard to, I think you asked about the indication statement, our planning assumption is similar to what we have indication for IgA nephropathy.

It’s for the treatment of IgA nephropathy. And we would expect that to be similar. And that would allow us to reach those 30,000 plus patients. And we believe that that addressable population will increase over time as there’s greater recognition and greater incentive to biopsy patients earlier, which is currently the standard way of diagnosing FSGS.

Farzin Hak, Biotech Analyst, Jefferies: Of the thirty ks patients, all of them are biopsy proven?

Eric Dubay, CEO, Trivia Therapeutics: So the majority of them are biopsy proven. When we did our analysis of estimating the addressable number of patients, we looked at biopsy confirmed, which is the standard. There is an increased awareness and use of genetic testing, but that’s still very early, and that still represents a small proportion and the smallest proportion of of FSGS. And so while there may be an increase, we believe that biopsies will still be the standard by which these patients will be diagnosed. Got

Farzin Hak, Biotech Analyst, Jefferies: Okay. Switching gears a bit to the filspirin launch in IGAN. So the PSF growth was sustained going from 4Q to 1Q for the last two quarters. So how are you setting commercial expectations heading into the second half of this year with regards to PSFs as well as persistency?

Eric Dubay, CEO, Trivia Therapeutics: Sure. So we are very pleased with the uptake, particularly the increased demand since we received full approval in September. Over the last two quarters, we exceeded around 700 new patient start forms per quarter. And our Chief Commercial Officer, Peter, indicated that that should be the new baseline as we move forward. Certainly, there are a number of new treatment options that are or will become available, but we believe that the unique clinical profile and positioning of Filspari will allow us to compete very effectively as we continue to reach more patients.

The important aspect of our Filspari business is that the compliance and persistence rate is and continues to be very high. So that average number of 700 patient start forms that we’ve seen over the last two quarters is added on to a very solid base of patients that continue to retain their therapy. And so we expect that the revenue growth will continue. Our goal is to continue to reach more patients and continue to increase the breadth and depth prescribing by nephrologists because the overwhelming majority of patients with IgA nephropathy, ninety percent of them, are on an ACE or an ARB and are not at the goal of proteinuria. So these patients continue to progress and continue to be at risk, and we really are offering something better with upgrading their ACE or ARB to Filspari.

Farzin Hak, Biotech Analyst, Jefferies: Got it. A little bit more on the commercial dynamics. What do you expect with the Novartis’ atrasentan on Venrafia market entry? That is coming in without REMS, but has a more limited UPCL label.

Eric Dubay, CEO, Trivia Therapeutics: Yeah. So we are very confident in the clinical profile of Filspari. So not only do we have full approval across the broad indication of IgA nephropathy, whereas atrasentan under accelerated approval is limited to those patients at greatest risk of 1.5 grams of protein and above, but we also have two year data. That two year data reflects not only a sustained reduction in proteinuria. It reflects a differential rate of achieving complete remission of proteinuria as well as an accumulation of benefit of eGFR the longer a patient is on therapy.

And so the clinical profile really reflects what nephrologists are looking for in the treatment of a kidney directed therapy, a foundational therapy like Filspari. But we certainly are expecting that Novartis will continue to grow the endothelin class by reaching more patients. And so we expect they will have an uptake. We’re very pleased that we have another therapy that’s talking about the importance of not just angiotensin blockade, but endothelin blockade. And we believe that that’s going to continue to drive, with our efforts, the growth of a far better foundational therapy than patients have historically been treated.

And with regard to the REMS, they did launch with no REMS. We’ve not seen the REMS be a barrier. We have had greatest uptake of a rare kidney launch over the last five years, and that is with a REMS. We also have a PDUFA date coming up in August to modify the REMS from monthly testing to quarterly testing. And we also will have our pregnancy testing REMS removed.

So we believe that that will become much easier for patients and will level the playing field in many ways. But importantly, the dynamic we expect to see with Atrasentan is a continued expansion of the endothelin class, and we certainly will continue to grow our business along with them.

Farzin Hak, Biotech Analyst, Jefferies: What about the degree of Tarpeo and Fabholta use you’re seeing in real world prescribing patterns?

Eric Dubay, CEO, Trivia Therapeutics: Well, I think with Tarpeo and with Fabholta, those represent a very different part of the treatment algorithm. The global guidelines, the CADIGO guidelines for IgA nephropathy, very clearly spell out that patients with proteinuria should have combination therapy. They should have therapy that directly addresses the damage in the kidney. That’s where Filspari or SGLT2s fit in. And with our head to head data showing superiority over RAS inhibitors, we believe and we’re very confident in our ability to become the new foundational therapy along with SGLT2 combination.

In fact, this month, the ERA meeting, we’re presenting data on combination of Filspari plus SGLT2s, demonstrating an additive benefit and a clear safety profile that should continue to support that being the new foundational therapy moving forward. The QADIGO guidelines on the second part of the treatment algorithm talk about needing to address the over activation of the immune system within IgA nephropathy. That’s where Tarpeo and steroids and Fabholta fit in. And what we’ve seen today is that those therapies are largely focused on more severe patients. We’ve seen them be used in combination with Vilspari, and we don’t see them as competitors.

We see them as complementary, and that’s certainly how physicians see them, but certainly reserved more to higher proteinuria level patients.

Farzin Hak, Biotech Analyst, Jefferies: Got it. Since you have the full approval label, how many of the payer policies have updated since then? And do you expect the payer policies to look similar or different between filspari and atrasentan?

Eric Dubay, CEO, Trivia Therapeutics: Yeah. We’ve been very pleased with our payer access. And since full approval in September, it’s only gotten better, particularly with regard to either removing or lowering the proteinuria thresholds for a physician to prescribe. And so we see a very broad access that is continuing to improve. It’s very early for us to look at the atrasentan approvals.

What I would say is that we would expect over time that they’re gonna look similar. And many of them will likely limit the access given the accelerated approval and the limited indication.

Farzin Hak, Biotech Analyst, Jefferies: Okay. And then on the REMS, you mentioned briefly, so you submitted additional data to the FDA. What is the latest thought on whether they could potentially remove it completely given that atresinant didn’t get REMS?

Eric Dubay, CEO, Trivia Therapeutics: Yes. So our our plan all along since approval in February of twenty twenty three was ultimately to remove the REMS. We’ve discussed that with FDA, and our approach has been a two step approach, which we believe allows for the fastest, most efficient access for patients. The first would be, as we’ve submitted the sNDA, to modify the REMS from monthly testing in the first year to quarterly. We do believe that there is an opportunity to fully remove.

That may happen this time, or it may happen later. We’ve said that we’ll take every opportunity, but that was not the original request of this SNDA. But certainly, we’ll take that opportunity to have that discussion. We are confident that we will have this shift to quarterly given the increased exposure that we’ve had since the submission and that we still have no cases of drug induced liver injury, no cases of Hy’s Law. And based on the increased exposure, we’ve been able to rule out one case of DELI in one thousand, which is an important threshold.

So we’ll continue to provide FDA with those safety updates. And if ultimately it’s not removed now, our plan was to go back with a second step with broader exposure and ultimately get this removed. Makes sense. There’s obviously a

Farzin Hak, Biotech Analyst, Jefferies: lot of data coming up in the near term in the IgAN treatment paradigm. So how do you see the competitive field developing as well as feel sparingly play a role in the near and longer term?

Eric Dubay, CEO, Trivia Therapeutics: Yeah. I think within the broader rare kidney disease, which has, you know, suffered historically from very little innovation, it is an incredibly exciting time We can foresee within five years that the majority of patients will never have to face kidney failure because of the increased innovation and the treatment options that are there. If we think about what the QADIGO guidelines recommend and and our understanding of this disease, a patient should be on therapy that is nephroprotective and directly targets the kidney. The best option that we’ve seen based on the clinical data is a combination of Filspari and SGLT2s.

With regard to addressing the immune system, the data that we’ve seen recently from the aproblis or B cell targeted therapies are really exciting and very aligned with our expectations that we’ve had over time. We expect that they’re gonna replace the role traditionally that steroids have played and will be used likely chronically for these patients, but only in combination with a foundational targeted kidney targeted therapy. In fact, all of those trials looking at immune targeted and B cell therapies are on top of kidney directed therapy. So we don’t see them as competitive. And, certainly, they’re very aligned with the QADIGO guidelines saying that patients with proteinuria should be should be treated with a combination.

Farzin Hak, Biotech Analyst, Jefferies: Yeah. I agree on the the combo aspect, but the payer policies maybe will push back on the prohibitive cost of combining the two drugs.

Eric Dubay, CEO, Trivia Therapeutics: Well, I think we still need to see how those therapies are priced, and we need to see what the longer term benefit and risk is. That’s how payers will typically make these decisions. What we can speak to is our access and our pricing and our benefits. And when we engage with payers, we’re very confident going in and saying that we have head to head data showing superiority over the other treatment option of kidney directed therapy, which is ACE inhibitor and ARB. We also have two studies demonstrating additive benefits when you combine with an SGLT2.

I can’t speak for companies that have immune targeted therapies, but we do know that those are you know, those that are available can and are being used on top of Filspari. And that’s something that they’ll need to discuss with the payers. I think this is another aspect of having the lead time that we have, being able to demonstrate the benefits and talking about the long term two year data on eGFR, particularly superior to active control, whereas it’s gonna be some time for any of those companies to have eGFR data long term to be able to discuss with payers.

Farzin Hak, Biotech Analyst, Jefferies: Makes sense. Are there any other Filspiry clinical or potential ex ex US regulatory updates that we should be aware of, either for FSGS or IGAN in the next six to twelve months?

Eric Dubay, CEO, Trivia Therapeutics: Yeah. So we’ve been very pleased with the progress within Europe. So our partner, CSLV four and Entrevir, have been able to convert approval in Europe and The UK to full approval in IgA nephropathy. CSLV four has launched in a number of countries, and we’ve been very pleased with the uptake thus far. We expect that that will continue, and patients in Europe will be able to receive Filspari in more countries over time.

We also expect data from a pivotal trial from our partner, Rinalis, that is developing Filspari within Japan and parts of Asia Pacific in the second half of this year. So we are on track in being able to help provide Vilspari to patients globally in the years to come.

Farzin Hak, Biotech Analyst, Jefferies: How big is the market opportunity in Japan?

Eric Dubay, CEO, Trivia Therapeutics: So in Japan, the estimates are continuing to be refined. It is not a rare disease in Japan. It is much more common in many parts of Asia. And we’ll we can certainly provide updates on that as we move forward and as our team in Rinalis refines their estimates for launch.

Farzin Hak, Biotech Analyst, Jefferies: Okay. We have four minutes left. For the HCU program, do you have any updates on the expected timeline to restart the enrollment?

Eric Dubay, CEO, Trivia Therapeutics: So we are on track with our peggedabatinase program to initiate enrollment next year. And we’ve made very good progress, and we’re we’re exactly where we would hope to be this year.

Farzin Hak, Biotech Analyst, Jefferies: And then looking ahead, can you talk more on the pipeline expansion and BD opportunities this year?

Eric Dubay, CEO, Trivia Therapeutics: Yeah. So we certainly would like to expand our pipeline, And our focus will continue to be in rare disease with a particular focus in rare renal disease. We believe that we have quite a bit of expertise and experience in clinical development and in working with regulators and in commercial launch. We’re going to continue to be very disciplined. And as you can imagine, with a PDUFA date in January and an advisory committee and a continued launch, our primary focus is there.

But in parallel, we’ll continue to survey the landscape.

Farzin Hak, Biotech Analyst, Jefferies: And then what is your cash position and runway assumptions? And then highlight key updates that we should be looking forward to.

Eric Dubay, CEO, Trivia Therapeutics: So our cash position at the end of Q1 was $322,000,000 And we expect this quarter to receive a $17,500,000 milestone payment for the full approval in Europe from CSLV-four as well as future milestone payments later this year for market access and commercial milestones. And that, along with our growing revenues from our launch, we expect to be in a very good position. We’ve guided that our cash runway is into 2028 and beyond. With regard to catalysts for the rest of the year, certainly, the continued uptake of the Filsperi launch and IGAN will be important from a quarterly standpoint. The REMS modification PDUFA on April 28, as well as the advisory committee, which is yet to be scheduled, but certainly will be very important as we prepare for the potential approval and launch in FSGS, and then ultimately being able to reach the HCU community by reinitiating the Phase III enrollment sometime next year.

Farzin Hak, Biotech Analyst, Jefferies: Thank you, Eric.

Eric Dubay, CEO, Trivia Therapeutics: Okay. Thank you very much.

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