United Therapeutics Corporation (UTHR) Earnings Call Transcript & Summary

Good afternoon, everyone. My name is Will Pickering. I cover U.S. biotech at Bernstein. Very pleased to be joined this afternoon by the team from United Therapeutics. I will pass it over to them for some opening remarks, and then we'll have a fireside chat after that.

Yes. Thanks, Will. So to start today, we may make some forward-looking statements, and I encourage you to read our latest public filings for any risks and uncertainties associated with those statements. But now I'll pass it off to our CEO, Dr. Martine Rothblatt, for some opening remarks.

Thank you, Harry. That was the best forward-looking statement preface I have ever heard in 20 years of public company life. I love it. They should all adopt that one, right? Well, I'm really pleased today to be joined by our Chief Financial Officer, James Edgemond, in the center here, who oversees vast swaths of United Therapeutics, including all of the financials, the P&L decisions, pricing, reimbursement and so forth. He's also overall in charge of our current accelerated share repurchase program underway. Thank you so much for inviting us to be here at Bernstein. It's a great pleasure and for doing this town hall. I thought I might start with just some introductory remarks about like who is United Therapeutics and what are we all about and then launch into a kind of fireside chat Q&A up here on stage. And everybody, I guess, is free to ask questions, too. So United Therapeutics, we make medicines, organs and devices. The medicines we make, which are approved by the FDA, there's a lot of them. I'll just highlight some of them. There's Remodulin for late-stage pulmonary hypertension, Tyvaso for interstitial lung disease. Orenitram for early-stage pulmonary hypertension, Unituxin for pediatric neuroblastoma. We make bioengineered lungs that allow for extended transplant assessment. We make different types of inhalation devices, such as nebulizers and dry powder inhalers. So all of those things are developed and being made to treat all types of fatal illnesses. Most of our patients that have all of these illnesses, at the end of the day, they're going to require a lung transplant to survive for the long term. So we've also focused on being able to make lungs that are autologized in a way that they won't reject the lungs and won't have to take immunosuppressants. So we autologize lungs with different types of technologies. We've created master cell banks that are HLA matched, so the patient can get a recellularized lung that matches their immunology. We have developed xenografts that are thymasized, which means that the recipient receives the donor animal thymus along with the organ that helps to retune the patient's immune system to recognize the xenograft as themselves. And then finally, we make truly autologous lungs by taking a blood draw from a patient several months ahead of time and then reprogramming some of their cells back into inducible pluripotent stem cells, growing those to the many billions and then differentiating those cells into -- for the lung, endothelial, epithelial stromal cells, different type of airway cells for the kidney, tubular epithelial cells and podocytes and all of these recellularized organs look to the patient just like themselves. So they would not require any immunosuppressant. In the meantime, our research and development is focused on prolonging the patient's time period until they would need to have a transplant. And that R&D is focused particularly on pulmonary fibrosis and pulmonary hypertension. With regard to pulmonary fibrosis, we have 3 Phase III trials for a new medicine to treat pulmonary fibrosis. One of those trials just read out last month. And in fact, in all the decades of treating pulmonary fibrosis, no drug ever demonstrated a better endpoint measure than the Tyvaso medicine that we read out last month with nearly 100 milliliter increase in oxygen compared to placebo at the end of the clinical trial. So all of the key opinion leaders in the field are of one mind that this is the best medicine ever to be seen in pulmonary fibrosis. We have another trial going on right now for another type of pulmonary fibrosis called proliferative pulmonary fibrosis. And now shifting over to pulmonary hypertension. We have a Phase III trial that will read out in just a few months for what promises to be the only and the best once-daily pill to treat pulmonary hypertension called ralinepag. And this Phase III trial will read out in about the first quarter of next year. And hopefully, it will be approved by the FDA in the middle of '27. I feel quite confident that, that will become the dominant treatment for patients with pulmonary hypertension who are about 50,000 in the U.S. alone. So while we're doing all of these different type of R&D activities, we also are finally attuned to always protecting a proprietary space, so that we can continue to innovate and have additional positive cash flow to innovate. And one of the main techniques that we've developed to maintain a proprietary space, we call it technology reproprietorization. And what that means is we take a molecule, which is proven safe and proven effective, but before it goes generic, we reinvent that molecule with a device to turn it into a drug device combination product. And we make that device one that is novel, better than anything that's out there so that, that device has its own intellectual property protection, usually runs for something like 2 decades. That, for example, is the case with our product -- our best-selling product, Tyvaso DPI, which took treprostinil, a molecule that was proven safe and effective, was becoming generic. We reproprietorized it into this Tyvaso DPI dry powder inhaler product. It's now become our best-selling medicine. We're very excited to be here at the Bernstein Investors Conference. While we are a public benefit company, there is no stakeholders' interest that is more important to us than the investor stakeholders. So to attune to that, we try to do everything we can, and we've been successful to keep both revenues and profits growing at a double-digit rate year after year after year, I think now about 12 consecutive quarters of record growth in both profits and revenues. Another thing which is very much attuned to our shareholders is a wise deployment of capital. So we use a lot of our capital for internal R&D, all those projects I just got done talking about. We use other capital for in-licensing or business development, buying new products in like this best one pill a day that I was talking about. And finally, when there's excess capital, we also return it to investors through share buybacks. And today, there's been literally millions of UTHR shares that have been repurchased as another way to return capital to shareholders. We know shareholders are also very attuned to what's around the corner, what's new, what's exciting, what's going to generate growth. So we have a huge slate of new product development activities, most of which are actually right now in stealth mode and will be coming out of stealth mode in the middle of next year. At the same time that we file for regulatory approval for pulmonary fibrosis and for one pill a day for pulmonary hypertension, we'll be bringing 6 new products out of stealth mode that promise multiple decades of additional IP-protected proprietary technology going after the different pulmonary and respiratory diseases that we tackle. I'd like to wrap up before starting the town, the fireside chat portion by noting that a lot of what we are able to do and do uniquely compared to other biotech companies is 10 years ago, we stood up a computational biology laboratory. We call it CLIMB, the Computational Laboratory for In Silico Molecular Biology. The purpose of this laboratory is to build a highly detailed digital analog of the human lung. Why the human lung? Because that's our main focus in pulmonary hypotension -- pulmonary hypertension. We've been poured into this model all of the available data that we have from 20-plus years of conducting clinical trials in pulmonary hypertension, all peer-reviewed literature published on pulmonary hypertension, all other labels published by the FDA. And we did the same thing for pulmonary fibrosis, including blending in very well done, but not as comprehensive as ours, models of pulmonary fibrosis that have been developed in Europe. So we now have what must be the most complex and complete digital models of the human lung, both for pulmonary hypertension, pulmonary fibrosis. We also use this for our lung manufacturing. And this has allowed us to see kind of ahead of the curve to see which potential business development products would probably fail. And so we better not spend like $1 billion or $2 billion on buying those and which ones that would probably succeed. And I was really excited to see that many people ask us, well, why are you guys always so positive about treprostinil and pulmonary fibrosis. And of course, now everybody is. But when we ran the digital model, it estimated that the endpoint, the key endpoint is forced vital capacity would be compared to placebo, about 129 milliliters of oxygen, and we demonstrated, as I mentioned, just short of 100. This, while it's not exact, it's remarkably close given that the other products are down there at low double digits of improvement of milliliters of mercury. So this is clearly some kind of a proof of concept that our molecular biology and AI-powered efforts are paying good dividends for us. We've run those models that give us similar confidence in the ralinepag trial that will be unblinding soon. And we're going to continue to deploy this model for all of these stealth new product development opportunities that will be in healing next year. So with that overview of the company, happy to turn it over to you for the fireside chat.

Excellent. Thanks so much for that -- those remarks. Perhaps we could start the Q&A with IPF. And you mentioned the TETON 2 study. Perhaps you could share a little bit more about what were some of the top takeaways from that data and what to be on the lookout for this weekend at your ERS presentation.

Sure. The top takeaway from the IPF unblinding was that was really wow. People have never seen these kind of results from a pulmonary fibrosis study. And for this weekend, which is the conduct of the European Respiratory Society meeting, there will be the scientific team that led the study called the TETON 2 study, sharing some data about the secondary endpoints and some of the other subgroup analyses that have been done. This team is -- these presentations are going to be led by one of the country's greatest pulmonary fibrosis experts, and he will share what he has seen amongst his patients and what he's seen in the data and why he believes that this data, both the primary endpoint data and the important secondary endpoint data, things like quality of life, what this is going to mean to his patients who suffer from pulmonary fibrosis.

Great. And how would you frame expectations for the TETON 1 study that you'll have reading out towards the beginning of next year?

Yes, it's a very great question. And just to make sure I level set so everybody is on the same page here. The TETON 2 study was done outside of North America and the TETON 1 study was done in Canada and the U.S. Other than that, the studies are identical, identical in size, identical in inclusion, exclusion criteria. The demographics are very, very closely comparable. No material differences in any of the demographics. One question that has come up at times is whether or not there might be people who have pulmonary hypertension as well as pulmonary fibrosis in the European study because our Tyvaso medicine for pulmonary hypertension is not approved in Europe, but it is approved in the U.S. so that maybe that might mean that there might be fewer patients with pulmonary hypertension in the U.S. study because they would already be treated. I think that's really fetching for smoke on something like that. First of all, the data that we have data that tells you whether or not a person likely has or doesn't have pulmonary hypertension. It's things like DLCO level and use of oxygen. And by all of these measures, the patients in both the TETON 1 study in North America and the TETON 2 study in Europe do not have pulmonary hypertension. So I think it's very unlikely that there's any excess pulmonary hypertension creep, if you will, in the TETON 2 study as compared to the TETON 1 study. But that's the kind of data that people who are following the ERS can expect the doctors and the scientists to go into detail on.

Great. And could you speak to the time line for filing an IPF and any potential ways you're considering to expedite that?

Yes. So we are working as hard as possible to file this as soon as possible. For anybody that knows about pulmonary hypertension -- I mean about pulmonary fibrosis, it is a bad, bad disease. The end stage, as I mentioned earlier, is lung transplant. So to be able to have a drug available to these patients as soon as possible is not only is it a business imperative, I personally feel it's a moral imperative. So we will go absolutely as quickly as possible. Our time frame for the filing would be not later than the middle of next year, okay? So that gives us just enough time to collect all the data from both of the studies, compile it together into the supplemental NDA for the FDA and file that in the -- not later than the middle of next year. And then based on a standard FDA review period, we would expect to have approval not later than June of '27. By the way, our United Therapeutics will be 3 decades old on June 26 of '27. So I've given everybody huge incentives that I want to have like 3 new drugs approved by one for each decade. And definitely, the IPF drug is one and the ralinepag drug is the second. Third one is kind of in the stealth mode right now. So that will be -- you need to have a surprise for a birthday. So that will be the surprise. Now there are some avenues that could allow us to be approved sooner, which is, of course, fine. So these avenues involve one-on-one discussions with the FDA, and our team is engaged in those discussions. So if there is an avenue, which I would say, frankly speaking, is on the order of at least 50-50. It's not like a long shot. I think it's a reasonable avenue, to be approved sooner than June 27, we'll go for that.

And what will IPF mean for the way that you organize your field force?

Yes. It's going to be transformative. If I was looking into an ivory a glass ball here, I would say that in all likelihood, there will end up being more UT sales reps on IPF than on pulmonary hypertension. And one of the reasons for that is both diseases are underdiagnosed, but I think pulmonary fibrosis is even more underdiagnosed. And secondly, just in terms of actual diagnosed patients, there are twice as many pulmonary fibrosis patients as there are pulmonary hypertension patients. And that's not even adding in the somewhat similar patients with proliferative pulmonary fibrosis. So we are already staffing up a new sales force. Fortunately, there's a lot of common touch points between the interstitial lung disease, doctors who I mentioned at the beginning of my remarks that we have a medicine for interstitial lung disease. So we see those doctors and the pulmonary fibrosis patients. So the top members of the commercialization team who are engaged in ILD, they will be the super seeds, if you will, for the IPF sales force, allows them a promotion opportunity and allows them to grow their leadership and hire in a lot of new people to be on the pulmonary fibrosis team. And then separately, we'll hire additional people to fill in for those people on the ILD team.

Great. And could you recap the IP for Tyvaso and what IPF could mean for that?

Yes. So the -- so first of all, the -- for IPF, we have what's called an orphan drug exclusivity because we did all the hard work. We were the first ones to have this kind of dramatically improved result for IPF. So the rules are such that in the United States, we have 7 years of orphan drug exclusivity once it's approved, of course, by the FDA. And then in Europe, we have 10 years of orphan drug exclusivity. And in Japan, there would also be 10 years of orphan drug exclusivity. So that's very, very significant right there. In addition to that, I talked earlier in my introductory remarks about our business strategy of technology reproprietization. So we have reproprietorized Tyvaso with the TD-300 nebulizer, which is a proprietary device to United Therapeutics. That is not easy to make, and it's very difficult to make in a way that you have a dispersion of the drug in the nebulized form just the right way throughout the pulmonary bed. So we own the software, the hardware and the manufacturing rights for that device. Based on our enthusiasm for IPF, we have actually stood up a second manufacturer as well. So we have 2 manufacturers making TD-300 devices. And then as I mentioned briefly, we've also extended our drug device reproprietorization into another type of device called Tyvaso DPI. That's not -- was not used in this particular trial. However, I would hasten to note that we have been planning for the success of our product in IPF. And hence, we sized our DPI production for 75,000 patients. So we size it for that population level by -- we have a partnership with a company called MannKind, and they have a production facility of a proprietary DPI device with patent that goes out to 2042, I believe, that they make in Danbury, Connecticut, and that has a 25,000 patient population. We are now completing construction of a separate clone of that facility basically in Research Triangle Park, North Carolina, that will have a capacity for 50,000 patients. So you add the 50,000 and the 25,000, so we're able to satisfy 75,000 patients. I think that, that would represent -- well, numerically, it does represent the preponderance of the IPF population and would still enable us to treat all of the ILD population as well.

And that facility in North Carolina, that will be -- when are you going to be completing that one?

Yes. So we're going to be completing this, and this will not come as a huge surprise to you. But I've got everybody super motivated to complete it on June 26, '27 for our 3-decade birthday. So we'll have 3 giant candles outside the facility. And I really hope to have you there for the 3-decade anniversary. We'll have a big party. And that's the -- you kind of got the surprise out of me, but it's an approval of TETON, approval of ralinepag and the actual goal is to have 3,000 DPI, GMP commercially ready DPI devices out of that facility already inspected and approved by the FDA.

Excellent. Maybe shifting over towards PAH and PH-ILD. You've had a competitor in the market for a few months now. Maybe just talk about what you're actually seeing in the field and what gives you confidence in Tyvaso's continued leadership.

Yes. So the competitor has been out there and what we are seeing is that some doctors are trying their device. We have not seen it have any impact on our sales. I think the reason for that is there are 50,000 people with pulmonary hypertension. There are 30,000 people with ILD. So there's a total of 80,000 people that might be able to benefit from these kind of devices. Despite the fact that we've had this double-digit growth year after year, I guess we're in probably our third year since ILD approval, something like that. Despite that, round numbers, we're at about 10,000 patients ourselves, starting obviously just with a few patients in the clinical trial. So there's a huge gap between our 10,000 and the 80,000 that can avail themselves of this device. So I think it's -- they're out there drumming up the bushes to raise more interest and excitement about DPI, and it helps them and it helps us at the same time.

And could you comment on formulary placement and work that you're doing to secure that for 2026?

So that activity, we have a whole -- formulary replacement is kind of a specialized skill that actually, I am not really that smart about that. But I'm smart enough to hire really smart people about it. So the smart people have done just an extraordinary job of getting formulary replacement for our medicines. There is -- we've long had a policy that if you can't afford our medicines, we'll give it to you for free. And despite that fact, more than 95% of all of the patients have their medicines covered by managed care markets in some way or another. In terms of which one the payer goes to first, second and third, that if you were going by the data, I think people would choose the United Therapeutics products because we have the most scientific data published about our products for pulmonary hypertension. So we've not seen anything adverse in any kind of formulary placement that would put any of our products at a disadvantage.

And what's your view on the competitive threat from TPIP?

I don't really have too much of a view on it because it's not something that we've seen kind of hardly any data on. There was some data released that I'm not like a statistician, but I send it to our biostatisticians. And they really could not really draw any clear lines from what they saw in that data. So -- at this point in time, I would say it is another way to inhale another version of treprostinil with very, very little patient exposure. And I would say you have to compare that to the enormous baseline of current ways to inhale Tyvaso, all of the data that's backing all of these current ways plus the continuous reproprietorization into newer and newer, more and more cooler, I would say, drug device combination products and all the data that's coming out on them. So it's something for us to monitor, but I don't think it's anything that we feel would change UT's leadership position in pulmonary hypertension or pulmonary fibrosis.

Understood. You've mentioned ralinepag a few times. Maybe we could shift in that direction and start by perhaps just sort of framing the overall opportunity for that drug in the context of the current treatment landscape.

Yes. Ralinepag is a really amazing drug, and I'm really especially excited about it because my daughter does have pulmonary hypertension. And the patients, they don't like taking multiple drugs and using multiple devices. One pill a day is a dream situation. The problem was that there was no drug that had both the pharmacokinetics and the pharmacodynamics in terms of the potency of activating the prostacyclin receptors within the pulmonary vascular lining until ralinepag. And frankly, that's the reason we spent over $1 billion for it, because we never spent that much money for an in-licensed product before. But we did because the scientific data was indisputable that this was the most potent prostacyclin receptor, yet had a completely knife and clean safety profile. So we've executed the study using the same endpoints that were used by the current drugs like Uptravi, selexipag, combination of tadalafil and ambrisentan. And we use the same protocol, which is to show that the patients on your drug have fewer death and less morbidity than the group of patients on the background drugs and just on a placebo version of your drug. So that study is completely enrolled right now. And we will get the readout of those results, as I mentioned, at the beginning of next year. We'll file for the -- with the FDA no later than the middle of next year and hope for product launch in the middle of '27. There is some data that you can review online right now that shows the open-label 6-minute walk distance of the patients who have exited the study. And it is best-in-class of all those other drugs I just got down talking about, a best-in-class improvement in 6-minute walk, which has been retained more than a year after they left the study. So I think that's an indication that this drug will pretty much sweep the market, will become the 1 pill a day that will be first go-to drug for patients to both nip their pulmonary hypertension in the bud and if it is already well advanced to try to roll it back.

And what would success in that study potentially mean for Tyvaso's growth arc in PAH?

I think it could probably clip it because why would you really breathe something when you could just take a pill? I mean most people would just prefer to take a pill. Now Tyvaso, I think, will have a very good life in what's called interstitial lung disease because this is a different type of disease where if you end up with a better heart function, which you'll get from ralinepag, but still a compromised lung function, you get what they call V/Q mismatch, and it is -- it actually will make you worse. So for that particular type of pulmonary hypertension called interstitial lung disease, I don't think that's the place where you would want to use ralinepag. And in fact, we did not test it in that area anyway. But with regard to straight up, what's called like Group 1, World Health Organization Group 1, pulmonary hypertension, there's always going to be some patients that that first, they're already on Tyvaso. It's keeping them alive. They don't want to leave it. God bless them. I hear that. I can understand that. There'll be some patients who they would just prefer to inhale something. They may not -- they may get a GI problem or something from taking the pill. That's good. But by the 80/20 rule, I would say I'd expect 80% of the pulmonary hypertension market to be on ralinepag and 20% on other stuff.

Great. Great. For your xeno transplantation program, this is just incredibly exciting when I read about this, the science behind it. But could you share a bit more about your work in that space, what you've achieved so far and sort of what you're on the cusp of achieving in kidney?

Sure. So what we've done is we've demonstrated to the FDA that our xeno kidneys are safe and probably effective. And therefore, they've allowed us to go into a clinical trial. So they've authorized 2 clinical trials for us for 2 different types of xenokidney products. One of them is a xenokidney with a 10 gene edits to prevent acute rejection of the kidney and for long-term kidney function to be maintained with what's called conventional immunosuppression, meaning the same immunosuppression they would get if they got a kidney from down the hall from another donor, a human donor. And then the other product that the FDA authorized us to go into clinical trials with is called the thymokidney. So in the thymokidney, UT has created quite an amazing product here that when the donor pig is less than 4 weeks out old, we remove its thymus, okay, which is kind of behind the chest cavity here. And we manipulate that thymus in such a way that we are then able with a syringe to implant that thymus in an empty section of the kidney called the kidney capsid. Every kidney has this kidney capsid. It's a highly vascularized, but empty space. Then over the succeeding 8 weeks, that thymus becomes revascularized in the kidney, unlike what was previously by nature, vascularized in the chest wall. And therefore, it's able to perform the job that a thymus does, which is to educate T cells and separate cell and [ dot ] cell and positive and negative selection. Then when it's -- the pig is about 16 weeks old, we explant the kidneys to transplant them into the patient. But the patient is now getting not just the kidney, they're getting the kidney and the thymus. So it's a fine we call the thymokidney. And the benefit of that is without making so many genetic modifications, we are just making one genetic modification, we're able to educate the recipient's immune system to recognize that xenokidney as itself. And our hypothesis to be validated in the clinical trial is those patients will require a much reduced load of immunosuppression. And in the longer term, we may be able to wean themselves off of all immunosuppression completely. That was, in fact, the the theory of my mentor, Dr. Tom Starzl, who is the father of liver transplantation, that if you were to do this, after a few years, the patient would not need any immunosuppression at all because their immune system would completely accept that kidney. So this administration FDA is awesome. It's probably the best FDA that certainly as a drug developer, I've ever seen. And their attitude toward xeno is admirable and laudable, and we are very grateful to them allowing us to conduct these 2 registration level xeno trials starting like -- we expect the first transplant next month. Now the way this will go, they said that each product needs 50 patient transplants, no placebo here, nothing like that. There's further brilliant, no comparison to historical controls. That's something we could have done, but wisely didn't do. So basically at 12 weeks, we need to show that the patients are healthy with the xenografts. So that's going to come by very quickly. And so there's every likelihood that we should be able to complete this clinical trial, both clinical trials by the end of '28 and file in '29 and have a commercialized xenokidneys in 2030. In anticipation of that, we have built one, and we are now finishing 2 other xenokidney production plants. The one we finished is in Virginia and the ones that we will be finishing next year, one is in -- by the Mayo Rochester campus and the other is in -- by Texas Medical District in Houston -- Houston Medical District outside Houston. So those 3 centers will collectively be able to produce approximately 600 xeno kidneys per year plus an additional 300 xeno hearts per year. We are also right now working day and night, the xeno team is to file with the FDA INDs for also a xeno heart clinical trial. And hopefully, we'll get that filed by Christmas. So that in '26, we'll actually have 3 xenoclinical trials going with a production capacity of around 1,000 xenografts per year.

Great. And what was your approach to disclosure be during that trial? Is it something you'd wait until the end or you share some updates periodically.

Yes. I think everything that would be material to an investor, we would disclose. So you have a great hand in that. Whatever -- folks like you say is material. That's kind of the word because your clients are going to do what you say pretty much, not completely, but you know what I mean. So whatever is material, I think like people would be material -- just I'm a lawyer, but I'd say like the first patient in is material. I would say the FDA decided you could proceed with the full 50 cohort that's material. And then when you finish the enrollment, that's material.

Great. Great. And maybe -- we're kind of running short on time. So I was just going to ask about capital allocation, how you're thinking about that through the end of the decade and especially just in light of the TETON results.

Sure. If I may kindly ask my Chief Financial Officer, James Edgemond to opine on it.

Yes, I think I'll run through it. So as you know, and we've talked before, we have a capital allocation kind of waterfall between internal research and development, which Martine outlined a lot of the programs and projects that we continue to work on. The second element is corporate development, those business development opportunities. And third is the return to shareholders. And currently, we're doing that through a $1 billion accelerated share repurchase. So that framework is something that we historically have followed, and we will consistently follow that on a going-forward basis. And what gave rise, for example, to the ASR currently in play that we started August 1 was really feedback from shareholders, but also a continual review of our capital allocation priorities, which led us then to decide to do a $1 billion share repurchase.

Excellent. Well, thank you all so much for joining us.

Thank you, Will. We appreciate it.

Thank you.