Welcome, everyone, to Jeffrey's 2026 Global Healthcare Conference. My name is Roger Song, senior, and it covers SMICAP Biotech. It's my great pleasure to have the five-side chat with our next company, United Therapeutics. We have James CFO and then Harry from IR. Good to see you, gentlemen.
Great, Roger. Thank you. Thanks for inviting Harry and I, and we appreciate the time. We've had great meetings this morning, so thank you.
Excellent. Excellent to hear. So I'm very happy to have you here as well. So maybe, James, if you want to do like a quick state of art for a user because you just give us a fantastic data at the ATS and then from Tidon and then the RolandoPag, I think user is in a very good position for the next stage of the growth. And then, yeah, give us some high level and then we can have a conversation.
Yeah, you bet, Roger. Harry's going to do real quickly our forward-looking statements as always, and then I'll do an overview.
Yeah, thanks for having us, Roger. Thanks for everybody in the room and online listening in as well. Today we may make some forward-looking statements, and for any risks and uncertainties associated with those statements, would encourage you to look at our latest SEC filings on Forms 10-K and 10-Q. Great.
Thanks, Harry. And thanks again, Roger. And for a quick overview, it's hard to imagine really another mid-cap biotech that has as many growth prospects kind of in its quiver as we do, and as an example, we just read out, as Roger alluded to, two clinical trials that were incredibly successful with two therapeutics, so in two different diseases that had significant growth prospects, and both of those trials had clinical results that were outstanding. They had p-values of .0003, of less than .0003. And those two trials, one was nebulized Tyveso in idiopathic pulmonary fibrosis. And it was the second of two trials that read out that had great clinical results. I'm sure we're going to get into some of those discussions soon. The other clinical trial was called Advanced Outcomes, and it was a therapy called Relenipag. So an oral, once a day, very efficacious therapeutic in pulmonary arterial hypertension. So these are two different therapeutic areas. We believe significant growth prospects. And as we'll talk about, we're on track to file both of those with the FDA by the end of the summer. So with Relenipag, it will be an NDA. So a new drug application, new chemical entity. And for Tyveso nebulized and idiopathic pulmonary fibrosis, that'll be an SNDA. But we are beyond excited. The product development teams, the site management teams, and everybody that supports these clinical trials did an outstanding job, and it yielded great results that we're incredibly excited about.
Excellent. All right. I think, James, you said it well. At the mid-cap company, you do have your very solid foundation commercial business, and then you have a next-level growth from those pipeline product. So maybe we'll focus on the commercial for a minute. So maybe you've been the soft guidance in terms of the growth for the year, and then towards the end of next year, 2027, $4 billion run rate and then double-digit growth. And when I look at our model and then also the other consensus from the sales model seems lower than that. Maybe it's a good thing because people want to be conservative and surprise people in a positive way. So how confident are the user management team about those guidance and then why you think the street is missing and that you can give us the positive?
Yeah, thanks, Roger. Good question. So as we've talked about recently on our last quarterly earnings calls, we still have the expectation, broadly speaking, to exit 2027 with a quarterly billion-dollar run rate. And we've made this assertion previously, and we still expect coming out of 2027, so next year. And that's the growth of our commercial business when we made this assertion. So it's the currently approved commercial products. Now, as we just talked about, Roger, we had two clinical trials we're going to file with the FDA later or by the end of the summer. So we could have some contribution from that next year, but our assertions were really based upon our expectation of growing the commercial business. And where that growth is going to come from is in the Tyveso franchise, specifically in Tyveso DPI and looking at growth within the PH ILD disease state. So we feel very confident with the investments that we've made, the investments we're making, so that when we end 2027, we'll be on that billion dollar run rate going forward.
Great. And then the interesting thing is, Tyveso has been entrenched as a great product, as a franchise for PH and PH ILD. You have other product in a similar class, and they're also launching pretty well. So in that dynamic, how you think about your scenario, base case scenario, can continue to support the growth projection that you are making?
Yeah, thanks, Roger. So from a, I think it's a Tyveso DPI kind of perspective you're thinking about. So as an organization, we continue to feel very comfortable about the growth prospects in Tivaso DPI and specifically in PHLD going forward. If you think about the product, the partnership that we have with mankind, the success we've had, the product profile, the delivery of the drug deep into the lungs, we continue to feel very confident about our own growth prospects as we look forward. We've had many successful and thousands of patients combined with prescribers, and we feel very confident in continuing that success going forward in Tyveso DPI, and frankly when we look across the commercial business. So we're pretty excited. We have conviction. And the other thing that we're doing, and this may come into play, Roger, when we talk about IPF, we're actually investing in the sales force. And Michael Bankowicz, who runs, he's the president and chief operating officer, runs sales and marketing we've accelerated the hiring of sales teams for IPF and Relendipag but those are not approved products so we're going to deploy those sales teams which it within the PAH and PHILD franchises so we're going to have more more individuals so feet on the street talking about our commercial business as we onboard those new sales teams member members who will pivot going forward into the new disease states. Excellent okay great I like the
confidence and the conviction there. And then, you know, STRI want to be, you know, our particularly also want to be a bit conservative. But on the other side is, you know, we look forward to the growth, as you mentioned, kind of amongst all the competition. And then let's move on to the IPF because this is very exciting for everybody. So I think you also mentioned on the last earning call is thinking about with the new product, new indication across different, you know, formulation and the presentation, you can double for the 4 billion run rate by 2027 in the coming years. So how much we should think about the contribution from IPF or maybe from other new product like a soft mist and then some new indications? Yeah, Raj, we're pretty excited.
One, about the results of the clinical trial, really for the patients that are dealing with idiopathic pulmonary fibrosis. And so there, from the standpoint of currently approved therapies, we feel that the clinical trial results, and it was pretty clear actually, that it will be a superior product and an inhaled product, which is different than the current orals that are approved on the market. But we think based upon the clinical results, and ultimately the FDA will determine the but we have a lot of conviction that this will be a pretty big revenue opportunity for us and run ramp. We haven't actually talked about, Roger, what the run ramp will be, so in time we'll actually give more information on that. But based upon the currently approved therapies and where we currently price Tyveso and just the opportunity of having a therapy on the market that has a different profile from the current adverse events profile or what patients are dealing with that we think this is going to be very successful. We haven't outlined what that growth ramp looks like, but we feel very confident that we will be able to at least double the revenue size from where we're currently at going forward. So exiting 27, as I talked about earlier, which would be the billion-dollar run rate, we've actually talked about doubling that revenue size going forward based upon the contributions, primarily of nebulized Tyveso and idiopathic pulmonary fibrosis, but there will also be contribution, and we'll talk about Relinnipeg-2 a little bit later going forward.
Excellent. Okay. And then maybe quickly circle up to the ATS data for the IPF T9-1, T9-2. I think the top-line efficacy looks fantastic, right? So 100-plus and then across pooled analysis and across different subgroup. it seems very impressive if you I have to say you know invest ask about it and then it's a you driven the discontinuation a little bit higher and then how you think about that and then also I think in the comment from the paper and then talk about the nebulizer fatigue because the four times a day so how you will respond to that and how this will feed into the real world
adoption for type vessel nebulizer yeah sure Roger it's a good question look Look, when you think about prostacycline-related adverse events, cough is a known AE there. But I think what's important to note when you look at the trial, the amount of discontinuations due to cough overall, especially severe cough, were quite low. Severe cough was a single-digit percentage, or maybe even a single-digit N. But as doctors and patients have experience with using the nebulizer and doctors help coach patients through using the nebulizer you kind of tend to see that improve over time so you know you had the the dosing schedule and you can see in the graphs the separation of the FEC up to about 8 to 16-ish weeks patients are titrating and once you kind of get past that and you can tolerate the cough and get to the higher effective dose you really see that separation in the benefit of the therapy. And again, in the real world setting, these things really can improve over time when doctors have experience using the product. You have patient support programs like our own. And that's even evident in the trial itself at ATS. Dr. Nathan spoke to his own practice and his experience with the drug in the trial. And he actually had a much lower overall discontinuation rate relative to the overall trial because he's somebody that knows how to coach patients through using the nebulizer due to his familiarity with the product.
Yeah, some education is needed, just like a PH, PHRD. I think, you know, when you launch Tyveso, you also see early on discontinuation due to different rhythm, but with more education, then those patients will be able to stay on treatment a little bit longer, and then with the education from the physician. Okay, got it. And then in terms of the pricing, I know, you know, PH, PHRD, the epidemiology or the prevalence is a little bit lower than the IPF. And then your price is a little bit premium. And then the current standard of care for IPF with, you know, not considered generic, but the brand of the drug is about $100,000 per year. So how do you think about the pricing for Tyveso in this population for IPF if you launch it?
Yeah, thanks, Roger. So Tyveso, as we know now, is an approved therapy. And we don't anticipate modifying the current price by adding IPF to the label for nebulized Tyveso, just in summary. And as I mentioned earlier, at some point, we'll be talking from our perspective more about the market size. Obviously, that would play into the pricing aspect. But at this point, we don't anticipate changing by adding Tyveso to the label.
yeah and honestly when i look at the the data you presented for the t down one t down to you you definitely can see the differentiation even some a lot of that advantage compared to the standard care i understand you know you probably will justify the price either the current price or you know slightly closer to the standard care but we'll stay tuned stay tuned yeah okay good and then i think uh james you alluded earlier you are hiring new sales rep and then maybe expanding the current indication, PHP, HRD, and then IPF is, you know, some synergy with the current market, but also you probably want to increase further the sales force, you know, given you want to double in the market size. And how should we think about the cadence of the sales expansion?
Yeah, thanks, Roger. So the cadence of expanding the sales teams, some of that is actually underway and has been completed. So when we think about the opportunity in the IPF market is pretty significant as we talked about. And so there's a lot of activities going on in the background all across United Therapeutics to make sure once it's approved we can actually hit the ground running. And as Roger mentioned, one of the items was the sales teams that I talked about previously. So Roger, that hiring is actually ongoing right now. We expect them to be in place, trained and up and running specifically for the PAH right now and the PHILD market, so this summer. So you'll see that kind of cost increase on a going forward basis. Now whether Michael looks at opportunities and resizes things and shifts things based upon actually how that pans out over time, that'll be a decision later on and that'll be kind of what Michael sees as investment opportunities, et cetera. But that cadence and those hirings are happening right now, have been happening. Folks internally have been pretty busy. And then beyond that, what we're doing is looking at what are the things that we can do now to prepare for the launch. So we've had questions around nebulizers, inventory bills, things of that nature. And across United Therapeutics, because we want to make sure we can serve and support those patients right out of the gate, we're doing anything and everything we can to get
things in place. Great. All right. Maybe we'll stay on Tyvato before we move on to the Relenopack, considering the pipeline. So you have another pivotal program for PPF for Tyvato. It's ongoing. And then where's the status right now? And then how you think about the T-Done 1 translation to PPF? You know, understand it's a different disease, but they have some synergy and the underlying pathology, and then particularly for the magnitude of the treatment effect you see for the IPF, T down one, T down two, and then how confident you are regarding the PPF at this point?
Yeah, so we're certainly not going to speculate on what the magnitude of the FEC delta might be in that trial, but you're right, we are highly confident. As you said, the sort of underlying fibrosis and progression of disease are pretty similar between IPF and PPF. So when you think about the success of what we saw in the TEACH-ON trials, combined with what you already see in the space where nirindamilas and nintetinib are approved for both indications, we're pretty confident that we can also see a similar treatment effect in PPF with inhaled treprosinol.
Can I add on one thing? So Harry nailed it. I think just a couple other things, I mean, the PPF trial is another clinical trial that the product development team is actually working on, and we've seen a little bit of a halo effect where with the positive results of nebulized Tyveso in idiopathic pulmonary fibrosis, we've seen enrollment in the PPF trial going very well. As Harry's mentioned in some prior discussions, you know, it was 75 percent enrolled at this point. There's a lot of great attention on it. Folks are working very hard. And the opportunity size, as we provided, and you see some of the disclosures in the 10-Q that we filed, it's twice as big as the IPF market when you look at the PPF market. So we just think there's tremendous opportunity. The team is focused on the clinical trial and to get things done right. And we just think going forward, that's just another one to add to the quiver, Roger, of prospects for us for continued growth on a going forward basis.
Excellent, okay, great. Maybe another question related to the Tyveso pipeline is the trust me, right? So you announced the soft mist two quarters ago, and then that's very interesting because you see some dramatic reduction on the cough, which is maybe the only kind of drawback for the current kind of formulation. And then where are you in terms of the clinical and the regulatory status? I believe you say something like you complete pivotal study, and then you are filing later this year, and then how much you can tell us about the
result. Yeah, that's right. So we'll be filing for the indications for which Tyveso is currently approved. That's PAH and PHLD filing later this year. And again, that's based on some healthy volunteer PK work that we've done. As for branching out into the other diseases, IPF and PPF, we're still working on what the clinical development path looks like.
Got it. And then have you completed the study and then ready for the filing or you are continuing to do the trial and then waiting for data before you can file?
We completed a first cohort of healthy volunteers in the second half of last year. And the second cohort is either nearly complete or still going on right now. But we're still on track for later this year filing.
Yeah, I think the important part, Roger, is that we're still committing to filing by the end of the year, which I think these underlying activities that we're doing will continue. We're just focused on meeting that deadline that we've committed to.
Renanopack. So that's a super proselytion, as Martin said. So, how should we think about the data will support early use and then even take over the current drug? We know we have some drug and, you know, going generic pretty soon. I think, you know, how are you going to position this once daily, oral, and then in the PHPHRD first?
Yeah. Look, we're really excited about the potential for Relinipag. The advanced outcomes data was a clear home run. what this company has been working to from its beginning in terms of a highly convenient, really efficacious product. The data, you know, it's clear that Relinopag, it's QD and really has a high potent receptor affinity. So that's what led to what you saw in the trial with, you know, really strong ability to delay the progression of the disease. And we think these factors position it really strongly in the prostacycline class and could potentially shift it to earlier usage in terms of time since diagnosis, still following an ERA and PDE5, but potentially capturing earlier usage from prostacycline IEPAH patients.
Yeah, Roger, it's, and you've seen the clinical results, they was really a home run, as Harry talked about, and this was something that Martine was really focused on when she started the company, which is a once-a-day therapy, and it's hard to imagine why a patient wouldn't be prescribed Relentapag after frontline therapy because of the once-a-day, very efficacious formulation. So we just think, in addition to what we've talked about in the IPF, which is a tremendous opportunity. We also think in the PAH opportunity that this is going to be a new chemical entity that will have far-reaching benefits to patients that are dealing with PAH. And we think a huge
opportunity from a revenue standpoint as well. Yep. Got it. And then in terms of the indication expansion, I think you want to make this super procycline across, you know, broader than the pH. So what's the biology or magnetic rationale to support that? Because so far, the receptor seems to be more focused on the pH versus treprosinol is broader into the
other indications. Yeah. So it's very clear from the advanced outcomes trial that relinopag is a highly potent member of the class of drugs that treprosinol is in. And it's clear from the Teton trials that triprosinol is very effective in managing the progression of IPF. So thereby, we expect that Relinopag could also see the same effects. And to your point, we're planning on
developing it in DPI formulation. Yeah. Okay. And then for DPI, it's interesting you choose DPI as the inhaled formulation for Relinopag rather than other formulation. And what's the rationale behind
that? Yeah, it's kind of right now the path of least resistance in terms of the formulation work as well as the DPI may better support one's daily dosing. Got it. Okay. One new indication
that you raised at the last earning called COPD. So it is a massive population, but on the other side, you may not address the entire COPD, like a more kind of enriched population. How much we know about that and I know last time I asked the question that you see and stay tuned and you will give us some more updates and any latest thoughts about this. Yeah we we've disclosed that we're
seeking a PHCOPD indication expansion for inhaled treprosinol and in this trial we're actually using the SMI relative to the last time that we pursued this indication and you know we really learned a lot in the first time that we ran a full clinical trial to pursue this indication and it's we we have a lot of learnings that we're applying in terms of design and patient selection and then of course you layer in the SMI we're confident that we can hopefully see a successful outcome this time got
it okay very good so this is a in the to process an arrow and then the prostagland kind of franchise for PHP HRD and then the related disease and then you have another transformative angle of the story, it's the, you know, transplant. So it is in clinical right now. You're, you know, transplant people, patient now. How should we know the strategy disclosing data and then how we're gonna, when we're gonna see the data?
Yeah, thanks Roger. So it's, so this is kind of another one from a longer term growth opportunity for United Therapeutics, where we're actually doing clinical trials for those new to the story In kidney using xeno organs, so genetically modified organs, and we have three clinical trials right now going on. One is in eukidney, which is a 10-gene modified, genetically modified kidney that we've kicked off. We've had transplants in. The second one is euthymokidney, which is using the thymus of a one-gene edited kidney or pig with using the kidney. And we announced a couple of Fridays ago that the FDA released Express, which is using a heart, so a genetically modified heart. So those clinical trials, as you mentioned, Roger, are ongoing. And we think ultimately this will be an opportunity to really put a huge dent in the enormous shortage of transplantable organs that are currently, that exist today. It is, if you think about it, we're sitting here and there's 500,000 people that are currently on dialysis in need of an organ, and so we're pursuing these opportunities to supply kidneys and hearts through these clinical trials if they're successful. From a disclosure standpoint, Roger, the approach we're probably going to take is, in some of the protocols for the trials, we're required to finish six transplants and provide data back to the FDA, if I'm talking about the eukidney, and so we'll have to see what kind of disclosure comes from that, but we can't today commit to a disclosure plan, but we think once we talk with the FDA, and we potentially resize the trials, right now it's up to 50 patients, that we will be able to communicate more about those clinical trials that are currently ongoing.
Time's up, and then thank you so much for being here, and I thank everyone. for listening and watching.