UNITED THERAPEUTICS Corp (UTHR) September 2026 Conference Transcript

get started. My name is Ben Burnett, biotech analyst at Wells Fargo, and I'm pleased to be here with the United Therapeutics management team. We have Martine Rothblatt, CEO, and James Edgman, CFO. So thank you all for being here.

Thanks, Ben.

I think maybe just to kind of kick us off, just kind of start by giving us a quick overview of sort of the business and some of the near-term events that we should be focused on.

Sure, Ben. Glad to do so. United Therapeutics is right now at a major inflection point where we are rolling out 14 new products over the next few years that cumulatively will provide us, we believe, somewhere between $50 to $100 billion a year in pharma revenue, moving us to the very top tier of pharma companies. And I'll maybe march through these new products, Ben, quickly. So the first one that I think most people are aware is the NDA that we submitted a few months ago and now has its PDUFA timing, which is nebulized hyvaso for idiopathic pulmonary fibrosis. In its pivotal trial, it produced far and away the best clinical trial data ever from any registration study for pulmonary fibrosis. Coming right behind that is our development of that same product for progressive pulmonary fibrosis or PPF which is a three times larger indication and we just announced about a month ago or so that we had fully enrolled that study already so that's on its way for its 12-month endpoint measurement. Then right behind those two products we have our dry powder inhaler for both IPF and for PPF. Then right behind those four products, and all of these coming at a very rapid clip of more than one new product introduction per year, we have a coughless product called Coughless Tresmai for both ILD, PPF, and IPF. and that product is a truly amazing one in terms of its convenience and its ability to get rid of the number one side effect that patients in these indications have. Coming right behind that product is a once daily inhalation product called RALPi, which we're developing again in three different indications, ILD, IPF, and PPF. So right there, Ben, is 10 new products over the next few years and there's even more. The one that I have to say, people often ask me which is my favorite, probably my favorite, is the Tresmai product for PHCOPD. This is a very debilitating condition. It's a small proportion of people with COPD for whom their pulmonary hypertension is all out of proportion, and they are the people with the severest disease and the rapid decline into fatality. Our coughless Tresmai for PHCOPD can address a market of somewhere between 400 and 800,000 patients in the U.S. alone. And now moving over to pulmonary hypertension, we already filed an NDA, have a Pidufidate for our Generaldi product, once daily pill for pulmonary hypertension. Again, produce the best clinical trial data of any product in pulmonary hypertension ever. That one will be queued up for FDA approval in June of next year. And then right on its heels, we have what we call triple generality, which is that once daily pill, together with the two background medications, we demonstrated its efficacy over PD-5 and ETRA. That takes us up to 12 new products. And then last but not least, we've got two very, very exciting products to roll out on top of all of that. We have a triprostanol-iloprost combination product that can be used as a PRN, meaning that you just use this product as necessary during the course of the day. And it doesn't matter if your background therapy is one of our therapies or one of our competitors' therapies. this would be the first, you know, quote-unquote rescue inhaler for pulmonary hypertension. So you add all of these 14 products up, you're looking at a half million patients with the PHCOPD, you're looking at 400,000 patients with the pulmonary fibrosis type of conditions, another 100,000 patients with the pulmonary hypertension conditions. So it's a million addressable patients. All millions should be captured by one product or another. But even with a 50% capture rate, we're looking at $50 billion in pharmaceutical revenue over the next few years. And that's really the reason, Ben, that we are excited to announce at this conference, at this Wells Fargo conference, that we have done an accelerated share repurchase with a half billion dollars, bringing up our total repurchases just in the past near term to upwards of $4 billion. And the reason for that is it just makes no sense to us at all that the company is at a market cap of around $20 billion when based on produced clinical trial revenues, filed NDAs, best results in the industry, multiple moats in terms of intellectual property protection to the 2040s, composition of matter protection for Relenopeg to the 2040s. We have a super clear straight shot at $50 to $100 billion in pharmaceutical revenue. Okay.

That's great. Well, let's start with, so a lot of areas to dig into. Let's start with PAH and PHILD. So your products are out there. there's other products that are in development or on the market now. So I guess the question is, how do you see the PAH market evolving with your products and competitors, and where do you see that kind of end state?

I see the market evolving to a place where the physician will ask themselves, do I want to slow my patient's rate of decline, or do I want to provide them actual clinical improvement. Only one product out of the 12, 13, 14, I've actually lost track of products approved in pulmonary hypertension, has shown in its phase three clinical trial data actual probability of having a clinical improvement, and that's this product we call Generaldi, Relenopeg, with the trade name Generali. Furthermore, that product is the easiest to take, just one pill once a day. So I think physicians are going to move their patients from whatever other therapies they're on into just one pill once a day to actually get a clinical improvement in their patients. Now, some physicians will say, well, my patient has been on a background PD-5 or ETRA for a long time. And that's why right on the heels of Generaldi's approval, we plan to file for approval of what we call triple Generaldi, which is a single pill in which the Generaldi has been blended together in a very specific technical way with a PD5 and an ETRA. So you get three mechanisms of action, endothelium receptor antagonist, phosphodiesterase-5 inhibition, and the superprostacycline activity that Generaldi offers. One pill, once a day, three MOAs. I think, you know, more than 80% of all the pH patients are going to be on that, certainly by the end of this decade.

Okay, that's fantastic. What are the timelines for that, the triple?

So that one, the NDA, we filed for generality, and we received an acceptance of filing from the FDA and a PDUFA date, best of my recollection, June 27th of next year. And then coming right on the heels of that would be a filing of triple generality. And we think because the basic generality would have already been approved, that we would have a rapid turnaround from the FDA on the triple generality, not more than 12 to 24 months later. So well in time for those. First, we would get the physicians that say, well, this pill demonstrate improvement even in patients who were not on background therapy. So I'm going to go ahead and take all my newly diagnosed patients and put them in generality. But for other physicians that say, well, you know, the pill, the generality was also proven very effective in patients on background therapy, I'd say to them, look, in addition to your, say, one ambrocentin pill that you're taking and maybe your one adcerco pill, I want you to just take a third pill of generality. So for them, it'll be three pills, but just once a day, which is a tremendous relief. and knowing that those would be the only patients in pulmonary hypertension that have a probability of clinical improvement, that's the holy grail. I mean, frankly, Ben, I started this patient, this business, to save my daughter's life who has pulmonary hypertension. That's well known on the web. And I feel like for the first time ever, like hope is better than ever that she'll actually be able to improve instead of just getting weaker and weaker.

That's amazing. Very cool. Well, let me turn now to the inhalable franchise. So with Tyveso, we've seen some decline kind of quarter over quarter. Do you envision the soft mist inhaler in that format, maybe returning that sort of franchise back to growth?

I absolutely do, Bill. Ben, sorry about that. The soft mist inhaler is a truly remarkable product. And it will allow a patient with just four puffs a day. And when I say four puffs a day, I mean like one puff four times a day. So it's vastly easier for the patient to take, to be able to get their treprosinol medicine down into the deep lung without the cough. So as a coughless product, we call it Tresmai for a treprosinol soft mist inhaler. And I think that this product is going to kind of sweep the floors with the competition. I mean, I, you know, just to be very blunt, it's such a more convenient product. Now, our goal is to first get this product approved in ILD and then to move this product into IPF, which is, frankly, about probably a four times, maybe even a five times larger market. and then move it into PPF, which, as you know, we just completely enrolled our nebulized product in that PPF franchise, which is around 300,000 patients. So the forecasts are so great for this Coughless product that we've actually had to open up two separate manufacturing sites to be sure that we have an adequate clinical trial supply for the entire market.

Okay, that's fantastic. And actually, I'm glad you mentioned that because I think, you know, we're all looking forward to the type-based nebulizer and the IPF opportunity. But not to put the cart before the horse too much, but we're also kind of looking at what are the other formats that could kind of go that direction. What have you said about the path to getting some of these other formats, such as the DPI and such as the soft-miss inhaler, into IPF? What kind of studies would you need to run?

Yeah, so they all run, Ben, a kind of a somewhat similar algorithm. And that algorithm is that what we call at UT, United Therapeutics, we have a kind of mantra inside the company. And if you ask anybody working in the company, any of the 2,000 of us, say, what's the main mantra of the company? Everyone will say, approve and then improve, approve and then improve. And, you know, it's not a litmus test, but it makes so much logical sense to them that they get it. So, first, you get treprosinol nebulized approved for IPF. Now, why that one first? Well, treprosinol is already approved in ILD, and nebulized treprosinol is already approved in ILD. The FDA is very familiar with it. It already demonstrated efficacy in this patient population in the INCREASE trial, back before the Teton trial. The FDA is familiar with the molecule, familiar with the device. We presented it to them. They said, yeah, this is like, you know, 12-month endpoint, go for it. And we went for it. Then why next? How can you improve it? Well, one way to improve it is instead of having a kind of large nebulizer, why not a little DPI that you can stick in your pocket? You've seen the mankind DPI. It's like a whistle, really, about the size of a whistle. So we went to the FDA. Now, why the DPI next? They had already approved it for IOD. They felt very comfortable with its safety and with its efficacy. So we said to them, you know, we'd like to next develop this into IPF. Now the FDA then makes an analysis. Okay, well what type of bridging study or efficacy study should we need in this new indication? What type of safety data? And different parts of the FDA will ordinarily look at things based on their own area of competency. So you're gonna have the cardiorenal division look at things one way, and the pulmonary division look at things another way. United Therapeutics respects the FDA and all of their competencies, so we meet with them, and we take the guidance, whether it's like a smaller bioequivalency or a bigger, whatever it is that they want, we do the development that each part of the FDA wants us to do. And I think that the track record with our Tyveso DPI has shown that it's so similar with nebulized DPI that we're going to have a very fast track route for that Tyveso DPI into first IPF, and then after we could get a nebulized Tyveso approval in PPF, then PPF. Then the next thing is the SMI. FDA said, well, that's something new. We didn't previously approve an SMI, but we did previously approve treprosinol. And now we want to see, should we do the Tresmai as a bioequivalence, a bridging? What data do we need? And again, we have these discussions with the FDA. And the reason that UT is always able to do the shortest path for each new drug device combination is we're very careful to change just one thing at a time. We don't change like multiple things at a time, just change one thing at a time. And that's why I can say to you confidently that I believe all of those products that we just reviewed will all be in the marketplace this decade, this 2020s, and we'll be making major contributions to taking us towards that $50 billion of pharmaceutical revenue before the end of this decade.

Okay, well, that's great. And maybe we could talk a little bit about the IPF opportunity specifically. So I guess the question is, what do you envision that commercial opportunity kind of being? And I think the context that a lot of us have here is we've watched JazzCade, which is sort of a new launch into IPF. And it's been a while since we've seen a new launch in IPF. But I guess, you know, looking at that and kind of the adoption curve, like what should we expect with Typeso and IPF?

Yeah, JazzCade was an awesome introduction to the space. And by the way, we have now JASCADE patients and our open-label extension patients of nebulized TIVASO. So we are already showing the ability to enroll patients and show a kind of a combinatorial benefit of TIVASO on top of JASCADE, as we showed beyond a doubt with natenamide and perfenadone. So the key thing to look at here is what's the size of the market, as you said, Ben, and there's some bouncing around, but people estimated something like 100 to 150,000 patients in the United States with IPF. And what are some of the limiting factors in addressing that population. Well, with nintenamide and perfenidone, there were some pretty challenging GI side effects that caused a very large amount of dropouts. So you have to factor all of that in. And then we have to kind of, we're in a casino here, right? Some kind of a casino. So we have to put a bet on the table in that, you know, we can't just snap our fingers and have thousands of patient drug patient doses available, we have to spend money to build up a launch inventory based on how many patients we think we're going to ramp to during the first year. It could take a few months to build up products. So we try to say, okay, we're getting ready to launch. Things are looking great for the FDA approval. How do we want to launch with? And that gets your question, right? So we put a bet. We actually made a $100 million bet. We bet that we would need 10,000 patient starter kits for the first year of product launch. So that's what we're looking for, for the first year. And I think that's, you know, not dissimilar from, you know, Jascade is looking at. And I think it's not unreasonable in a patient population of around 100,000 patients to be able to capture something like 10% of that population in the first year, especially when you have the amazing data that we have in Teton 1 and 2 of 100 milliliters of oxygen improvement from baseline.

Very cool. And on PPF, were you surprised at how fast that study enrolled? Blown away, blown away.

Ben, I have never seen in the pharmaceutical industry that a company enrolls a trial faster than like their business people thought it was going to be enrolled. And then even their clinical people. So it's usually like you say, hey, we're falling below the forecast we gave to the street for enrollment. This time, the clinical drug development people said multiple times, Martin, we have to move the schedule to the left. It's like, what? I didn't know clinical trial schedules could move to the left. I thought they only moved to the right. So it was astonishing. But the physicians were just pushing patients on the drug after they saw the remarkable effect that nebulized tyvaso had on their IPF patients. And they know these poor PPF patients, I mean, mean survival, just ask your AI, it's three to five years. I mean, it's horrible. So they're really pushing their patients on the PPF study. So I was blown away, surprised, Ben.

And so what does that mean from the commercial perspective? Like if you look at some of these drugs that have both an IPF and a PPF approval, you know, you see a disproportionate amount of drug sales from, I believe, IPF, but the numbers of patients that have PPF are a lot higher. So what do you see as sort of the addressable kind of PPF market, and how does that compare to your thoughts on IPF?

Yeah, so I think the starting point is that it's very important that things be on label to be able to be accessible to the patients. First of all, you know, physicians will not even be all that well-educated about things that are not on label. Secondly, it's going to be difficult, if not impossible, to get reimbursement for a diagnosis which is not on label. So the the FDA themselves required us to do a separate study for IPF and PPF because the pulmonary division feels that there are different clinical aspects of the IPF and PPF patient population that requires different clinical data sets for each patient population. So I think they did the right thing requiring a separate clinical trial for PPF. I'm very hopeful that we're going to have superb results. I will say that my hope, in addition to based on the physicians rapidly enrolling the study, is based on our digital AI lung model where we have run this patient population as we did our previous IPF population and shown a high likelihood of successful results. It's also based on the fact that in our phase two trial, the increase trial that we did, there were PPF patients in that trial who did just as well as the IPF patients. Still, the FDA made us do a separate IPF trial. And frankly, I was nervous about doing a parallel PPF trial before it was proven in IPF. So, in retrospect, you could say I was too cautious, and I would say guilty as charged. But nevertheless, that's the way it happened, and now I feel very confident about the results in the PPF trial, and I think finally this population will get the drug they need. With the FDA approval, it'll be on-label specifically for PPF, and insurers will not balk because their diagnosis will match what the label is for.

Okay. Excellent. I want to turn now to Rolanipeg, and you gave some helpful comments early on about the efficacy profile, the oral, but the format, the DPI format that you're developing, I guess, number one, is it fair to assume that that is going to be developed, you know, kind of along the same sort of pattern as Tyveso in terms of the number of indications and sort of the path forward?

Yes, Ben. So we're going to follow the same, what you could almost call the UT algorithm, which is approve and then improve. So the approve is PO once daily, which, as I mentioned, we have the PDUFA date for that coming next year. And then the improve is how can we get this to patients that really need to take their therapy via inhalation route so that they don't get into a realm of VQ mismatch between cardio output and inhalation. So that improve requires an inhalation formulation. So we work very hard with our great partners, Mankind, And we developed like an amazing inhalation formulation, which with once daily pharmacokinetics will allow us to improve on Tyveso DPI because the Tyveso DPI, we got it approved first for ILD, next we'll get it approved for IPF. But now we want to improve on that by having it once daily instead of four times a day. So the same approve and then improve algorithm. So we're going to apply the RALPi as a once daily inhalation solution for ILD, IPF, and PPF. So that'll be three more labels there. We are so bullish on this one, Ben, that once again, to say like we kind of put our money where our mouth is, especially in this beautiful Encore Resort that you have here, that we have built out a 50,000-patient annual production facility dedicated for Raald Pai. That's the once-daily inhalation formulation of Herlenipeg. It will have its ribbon-cutting October of this year. And thereafter, it will ramp up during calendar year 27 to be FDA approved as a site of production. You know, you have to have your safety efficacy and your production all approved by the FDA. So we should have it approved by the, all that, you know, in the bag by the FDA by the end of 27, you know, could possibly roll into 28, and thereafter we'd be able to launch a once-daily inhalation therapy with the best-in-the-industry pharmacokinetics. Very important, but not everybody understands the best-in-the-industry pharmacodynamics, because the fact is Rolenepeg is a better molecule than troposinol. We prove that in our outcome study, and we will prove that also in our studies for the inhalation version of the product. So you have better pharmacokinetics, better pharmacodynamics, and I think that this RALDPI is just going to sweep through the IPF and the PPF landscape. And I will probably end up outstripping the 50,000 production capacity of this plant, but that'll be a high-class problem.

Well, the follow-up I had, you just touched on this a little bit, is the hypothesis is that this will be more efficacious than troposinol, and is that based on the oral experience?

It is. That is my strong belief that the changes in the molecular structure between relenopeg and troposinol dictate a better antifibrotic activity for relenopeg even than troposinol.

Okay. In other formats, would you explore like soft mist with relenopeg at some point?

Approve then improve.

Yep. Okay.

Approve then improve. We're like robots. We just approve and improve.

Very cool. OK, I want to also talk about you. You've given out a four billion kind of revenue guidance. What are the puts and takes around that?

Well, I think the main put and take is it should be looked at as like the first step forward to a much higher pharma revenue run rate with these 14 new products that we're really first announcing at this Wells Fargo conference. We've not really shared the full panoply of the products. So these will be coming one after another very rapidly, all in the next handful of years, all with tremendous protection against kind of copycat or worse than copycat concepts. For example, in the pulmonary fibrosis and progressive pulmonary fibrosis, we have patent protection out to the 2040s. with whole patent families there. With regard to all the great features of Relenopeg that you just elucidated, Ben, we have composition of matter patents out also until the 2040s. With regard to idiopathic pulmonary fibrosis, which is where the big breakout from pulmonary hypertension begins, we have, upon approval, orphan drug exclusivity into the 2030s. So there are a lot of moats protecting these new type of devices, these SMIs, they have their own intellectual property protection. So with all of this, this $4 billion revenue run rate growing on the growing traction of our products in ILD and in PAH will be the next step toward an inflection that I talked about at the beginning of my talk here today, up to tens of billions of dollars per year of revenue from first the IPF market and then the PPF market and then the soft mist inhaler into the ILD market and into the IPF market.

Great. And if there are any questions in the audience, I want to make sure we have time to work that in. Just let us know. We'll work on the conversation. I want to ask just about the spending side of this. So as you think about the expansion into all these different opportunities and the improve kind of part of that equation, I guess, how do you see kind of the R&D and SG&A kind of trajectory over sort of the medium to long term?

Well, fortunately, we have our chief financial officer, James Edsman, to comment on that.

Yeah, thanks, Ben. It's a great question in light of what Martine actually just outlined in terms of products and product developments. So, as you know, what others know, we actually apply a budget algorithm to our spending model that says we don't spend larger or more than a percentage of prior year revenues. And historically, we've talked about 50%. We're going to tweak that a little bit going forward to raise it to 55% of prior year revenue on just cash operating budgets. So it doesn't include business development or facilities. One, to be able to support what Martine outlined is the vision going forward. So we're going to tweak that budget algorithm a little bit. And why we do that is we want to have good visibility into what we're giving folks within the organization from a budget perspective. but we really want to be good financial stewards of investors' money. And we think it's very important to have good budgets, to have good clarity, and it helps you make great decisions. For example, one of the areas you might touch on is corporate or business development. With this huge cadre of things ahead of us, we don't feel the need that we need to rush out and do a corporate development or a business development opportunity unless it makes real sense. folks within UT are extremely busy, and we want to be thoughtful of how we allocate this budget algorithm within the organization. So again, good financial discipline going into, again, planning for 27. We're going to tweak up our number a little bit just to make sure we have enough money to accomplish these great goals, but we're still going to be very good executors from a financial discipline perspective going forward.

James, can I add a coda to that? Sure. Is that okay, Ben? So I'm really glad that James highlighted the fact that it doesn't include the capital expenditures on the facilities, because one of the biggest dilemmas that I'll say I face as CEO of United Therapeutics right now is we are on the cusp of a revolution in a much larger field of medicine, even than IPF and PPF and COPD, and that is organ transplantation. We have three INDs approved by the FDA to create an unlimited supply of transplantable kidneys and transplantable hearts from our genetically modified herds of pigs. And we're marching very well through this, and we expect to have the first of these three INDs resulting in a BLA before the end of this decade. That's only like three years from now. Now, when you talk about an unlimited supply of transplantable organs, unfortunately, it's a lot of CapEx, you know, because building these facilities, there are 500,000 Americans in dialysis. So to build a facility that can provide 500,000 kidneys a year, it costs tens of billions of dollars to build that type of facility. and I have a lot of people asking me how long until I can get a xenokidney for my relative they are on dialysis how long until I could get a xenohart because a half million Americans a year die of end-stage heart disease so the build-out of our xenotransplantation facilities is going to involve tens of billions of dollars and when you come to a CapEx type of question like your question, that has to be taken into consideration.

And just one follow-up on that, what is the kind of cadence you would expect to have sort of clinical data presented from that program?

Yeah, so the xenotransplantation team led by Dr. Peterson, they expect to be sharing the first stage of clinical trial data, which is the data that the FDA allows you to go to the end of the registration study. It's like a stopping point. They expect to share that data at the end of this year, 2027. And then the final clinical trial data from the whole cohort of the registration study would be shared at the end of 2028. And then we would submit that data to the FDA in at the beginning of 2029.

Okay. So end of next year, potentially initial data. Yeah. That's great.

It's like after being far away for a long time, it's suddenly like right here.

Awesome. Well, thank you so much. I think we're out of time, but appreciate the conversation. Thank you, Ben. Thanks, Ben.