United Therapeutics Corporation (UTHR) Earnings Call Transcript & Summary

January 6, 2022

NASDAQ US Health Care Biotechnology special 47 min

Earnings Call Speaker Segments

Dewey Steadman

executive
#1

Good morning, everyone. I'm Dewey Steadman with United Therapeutics. And I'm happy today to present or to host or at least introduce Jessica Fye from JPMorgan who will be hosting our fireside chat with the management team today. Before we start, I just want to remind you that we may make forward-looking statements today, and any risks and uncertainties are listed in our SEC filings, including Forms 10-K and 10-Q. And with that, I'll turn it over to Jess.

Jessica Fye

analyst
#2

Great. Thanks, Dewey, and welcome, everyone. Happy New Year. I'm Jess Fye. I'm a biotech analyst at JPMorgan. Delighted to be joined today by United Therapeutics senior management team, including the company's Chairperson and CEO, Dr. Martine Rothblatt, as well as the company's President and COO, Michael Benkowitz. So thank you so much for spending this time with us this morning.

Jessica Fye

analyst
#3

Martine, I wanted to start out talking about your goals for the year ahead. So maybe with that, you can outline some of the recent progress of United Therapeutics. And then just share what you're looking forward to this year.

Martine Rothblatt

executive
#4

Okay. Thanks, Jess, and Happy New Year to you and to everybody else on the call. So with regard to 2021, I think we accomplished a lot of really great goals. I think first and foremost is we had achieved our goal of approval of Tyvaso for Group III pulmonary hypertension. That's the first approval ever for Group III pulmonary hypertension. That was huge. Secondly, we got bud approval, totally queued up for imminent approval of our DPI form of Tyvaso for not only Group III, but also Group I and Group III. So that's really huge. We experienced back to double-digit patient growth and put us strongly on track for wrapping up the year, this coming year with 6,000 Tyvaso patients. And that was a goal we set at the beginning of '21 to have the double-digit patient growth and we definitely experienced it. And we started a Phase III trial in pulmonary fibrosis. First time, we've ever been in that indication. And a whole lot of additional pipeline R&D. So it's just a huge number of things that were accomplished in '21. With regard to the '22 Jess, I think it's going to be a year of fulfillment for UT. And I would spell fulfillment as like FULLfillment -- Fullfillment. And the reason I use that mnemonic is because in '22, we expect to have full enrollment of our COPD Phase III trial. We expect to have full enrollment of our outcomes trial of ralinepag. We expect to have full enrollment of our full approval -- I'm sorry, full approval of our DPI product, full commercial launch of our Remunity product. And then a full achievement of the 6,000 patient goal that we had set out for ourselves for Tyvaso. So it's a whole lot of fulfillment in '22.

Jessica Fye

analyst
#5

Okay. Great. Maybe to now look farther ahead, I also wanted to spend some time talking about your 2025 goal, your 25 by '25 goal. So 25,000 patients is a locking number, clearly, one you see is achievable. I guess I want to understand how you accomplish that and what we envision the business looking like when you do? So I guess, first, just thinking about the baseline where we are now, how many patients are on Tyvaso, Remodulin and Orenitram today?

Martine Rothblatt

executive
#6

Yes. Great question. And 1 that the whole team at United Therapeutics is really focused on, and we do feel confident in our ability to achieve 25,000 patients by 2025. So right now, we are north of 10,000 patients. So roughly speaking, 40% of the way there. And the basic breakdown of the numbers that you asked for, and how we would get from these numbers to the 25,000 patient numbers goes like this. So right now, we have something north of 4,000 patients on Remodulin. And we expect to more than double that by 2025. Principally, through the launch of Remunity and then after Remunity RemoPro, which is our painless form of Remodulin. And that's one of the products that I mentioned at the beginning is in our active pipeline R&D that will go into human trials very soon within the next 2 or 3 months. Then, the next building block toward 25 is our oral products. Right now, we have north of 3,000 patients on our oral products, and we expect to double that to over 6,000 patients by 2025. And that's going to be accomplished principally through the launch of ralinepag, but also through continued organic growth in LatAm. And then finally, the third major building block just to get us to 25,000 is Tyvaso. So right now, we are somewhere north of 4,000 patients on Tyvaso. We expect to triple that by 2025 through the organic growth of Tyvaso in WHO Group I, Group III, the launch of the DPI product this year, in Group I and Group III. And then the subsequent launches of our COPD product. I mentioned at the beginning that we expect to have full enrollment of the COPD Phase III this year as well as the launch of our IPF product. And I mentioned that, that Phase III trial is underway. So with all of that Phase III activity and with all of those launched products and just about launch products and imminently launched products, I feel it is quite achievable to close the 60% gap between the 10,000 that we're at right now and the 25,000 goal by '25.

Jessica Fye

analyst
#7

Okay. Great. So we talked about kind of the near term, talked about the long term a little bit. I want to drill down on Tyvaso. Now your largest product after the very successful early start to the PH-ILD launch. I guess, first, you've talked about adding these 3,000 patients by the end of '22. Can you tell us how many patients were added in the fourth quarter? And just to confirm, do you still expect to meet that 3,000 patient add target?

Martine Rothblatt

executive
#8

Yes. Thanks, Jess. Just so I don't like hog all the time from my co-keynoter or firesider. Mike, would you like to jump in and talk for a while?

Michael Benkowitz

executive
#9

Sure. Happy to do so. Yes. So Jess, I think as you know, we -- and as Martine mentioned, at the beginning of '21, we set out the goal for Tyvaso to double the number of patients from 3,000 to 6,000 by the end of '22. I was kind of want to remind folks of sort of some of the cautionary language around that, which is that you're probably not going to be a linear path to get there, but we feel confident that we will get there. And then obviously, with COVID still in green, there's a little bit of a risk factor out there, but we haven't knock on wood until now, not really experienced any impact as a result of COVID. So at the end of -- in terms of how we're progressing towards that goal at the end of Q3, we announced that we were at approximately 4,000 patients on Tyvaso. As Martine said, we're north of that now. I think we'll probably wait until our earnings call to provide a final number on a number of patients on Tyvaso as of the end of the quarter, we're still kind of cutting through some of that data. But the bottom line is, we do feel very confident that we will be able to achieve the doubling of the 6,000 patients by the end of 2022.

Jessica Fye

analyst
#10

Okay. Great. And I guess related to that, can you walk us through where things stand with CMS reimbursement for Tyvaso in PH-ILD? And tell us maybe how you plan to communicate that to investors once it's received?

Michael Benkowitz

executive
#11

Sure. So we started the process for CMS reimbursement really weeks after we received approval back in April, submitted our application in May. And really, we're sort of at the mercy of the government in terms of receiving the policy update. I think the guidance or the requirements that they have, they have up until a year after receiving the application to make a decision. So that kind of puts an outer bound of, I guess, it will be May of this year. I think we were optimistically thinking that, that process would go fairly quickly in '21 and that we would receive approval shortly after our application and try out not to be the case. They opened their comment period. I think at the end of Q3, that wrapped up in the middle of November, at least in terms of what we saw from comments, really no issue there. So we feel extremely confident that we will get the policy -- we will get a positive coverage decision. We just can't predict when. So as of now, we're still waiting for CMS to update the policy. But as I said, we certainly expect that, that will happen here between now and certainly may at the outer band.

Jessica Fye

analyst
#12

And to the extent you have some government pay patients currently receiving free Tyvaso, when could they start being reimbursed?

Michael Benkowitz

executive
#13

So because you -- I think you want to kind of think about it sort of bifurcate, and I'll come back specifically to the current patient -- the current patient that are receiving free drug. So once the policy update is completed and it's effective, any new patients coming in with CMS will become commercial paying patients under Medicare. With respect to patients that are currently in our patient assistance program, the law says that for CMS patients once they enter your patient assistance program, they have to stay in for the calendar year. So those patients that are currently in our patient assistance program, given that the calendar rolled over to January, if you're able to get a coverage decision tomorrow. Unfortunately, from a revenue standpoint, those patients need to stay in patient assistance through the balance of '22 then they roll into commercial in '23. But at the end of the day, as Martine said, we're really kind of focused on the long term and our 25 by '25 goal, which is why we opened up our patient assistance program to these patients because we feel it's the right thing to do to help these patients with this therapy, particularly given that there's no other alternative. But just from a down-the-road revenue standpoint, it's certainly much easier. We've already have them on therapy and the system to convert them over.

Jessica Fye

analyst
#14

Got it. And you've alluded a couple of times to maybe some anecdotal reports of warehousing dynamic where some physicians are waiting for CMS reimbursement to kick in before going through the process of initiating a patient on Tyvaso. Can you elaborate a little bit on that?

Michael Benkowitz

executive
#15

Yes. I mean I would -- I think it's important -- I think the key word there is anecdotal because it's really hard for us to quantify what that looks like, I think, but just -- and having conversations with physicians, particularly ILD treaters that are new to Tyvaso, I think they have been going through the process of understanding how they're going to screen, how they're going to diagnose, where are they going to send patients to get a right heart cath. They don't have that capability within their community clinic or their institution and then there's the whole process of going through the referral process and all the things you have to do to actually start a patient on therapy. So we have heard for a number of physicians that are new to Tyvaso that despite the fact that they have the ability -- the patients have the ability to apply for patient assistance, I guess taking the cath at least for resistance, which is -- they're just going to wait for CMS coverage. So rather than have to go through the process of approving or applying for cath and then going back and applying for insurance coverage they said they're waiting. I'm hoping that's going to start to kind of breakdown a little bit after the first year, given that the CMS coverage has taken a little bit longer than expected, but we'll see. But in any event, we do think that there is some -- there are some patients that are just sitting there that I think will kind of come through the system once, once we have the CMS coverage.

Jessica Fye

analyst
#16

Can you give us a breakdown of how you see the number of PAH versus PH-ILD patients currently on Tyvaso and how you think about that changing over time?

Michael Benkowitz

executive
#17

Yes. I mean it's hard for me to give you specifics just because of the way the data comes in to us. I mean I think what you can sort of think about it in terms of round numbers as we were at 3,000 patients at the beginning of the year before approval. So assume those are all Group I. As we said, we added about 1,000 plus now through the course of 2022. I think the vast, vast majority of those are probably PH-ILD, not all of them. I mean, I think we're just continuing to kind of grow, grow Tyvaso in PAH, but assume most of those. So it's probably somewhere around 75/25 right now between the Group 1 and Group 3, that's rough. Again, the data is a little messy when it comes in, but that is just sort of my estimation. I think as we move forward, certainly, that tilts more towards, I think, Group III, right, just given the fact that you've got a 30,000 patient population, no treatment, no other treatment option besides Tyvaso. Not to mention, I think DPI certainly plays into that and provide a catalyst for even more patients coming on board. And so I think that will start to tell more -- that mix will start to tell more towards PH-ILD. And I think, the majority will be in PH-ILD, but again, I think with DPI, there's still an opportunity for us to see some growth with Tyvaso in Group I as well.

Jessica Fye

analyst
#18

Okay. Got it. And how are you thinking about the Tyvaso franchise outside of the U.S.? What do you need to do to get approved in Europe? And would you potentially pursue a partner?

Michael Benkowitz

executive
#19

So we have pursued a partner. We have engaged with a partner. In fact, we -- just before the holiday signed agreement with our current European partner Ferrer to commercialize Tyvaso for PH-ILD in the European markets. So the terms of that is it's a 50-50 revenue share between Ferrer and us plus we'll get a payment for COGS to make the product. In terms of the process and what needs to happen, hopefully, we'll have more information to share as we get into Q2. We're actually going through the EMA scientific advice process right now to understand what exactly they're going to require, what steps we need to go through and what the timing looks like for approval for PH-ILD.

Jessica Fye

analyst
#20

Okay. Got it. And I think there were some headlines over the holidays on this, but what's the latest status of the Tyvaso litigation with Liquidia?

Michael Benkowitz

executive
#21

Do you want to me keep going, Martine?

Martine Rothblatt

executive
#22

Sure. I'll give you a break now. Jess, we really don't get into the details of litigation for all the obvious reasons, lawyers don't want to try their cases in public. So we're just not going to comment on the status of the litigation other than I can say that there are multiple patents which we feel are being infringed. And these patents are being litigated through a mix of fora, the patent office fora, the IPR process, the federal court system. So there's just like a kind of layers and layers of patent disputes going on. And I think that they will go on for quite some time to come.

Jessica Fye

analyst
#23

Okay. Maybe switching to Tyvaso, DPI. Can you talk about how you see sales of Tyvaso or that franchise evolving with the introduction of the DPI?

Martine Rothblatt

executive
#24

Yes. I think that DPI will be popular. There's always a lot of excitement over a product that is an improvement on the existing product. On the other hand, what we see in pulmonary hypertension, is that there is tremendous loyalty to the legacy products. And I'll tell you that I find it surprising myself that I need patience every so often, who are still on intravenous Flolan and for those listeners who are newer to the story, that was the very first product approved by the FDA back in the 1990s. And this product has a very short half-life of like 1 to 2 minutes. So if the flow is interrupted, the patient is at essentially immediate risk of fatal rebound hypertension. In addition, it is -- it requires an indwelling Hickman catheter which has shown itself to be prone to septicemic infection. And in fact, that's on the label of the product and then finally, the pump is a 1990s legacy pump, which is big and bulky and needs to be surrounded with ice. And I'm like, why he is still on Flovent, but of course, I'm very respectful to the patient and their decisions and what not, but when I asked them that question, because I'm not like pushing our products, I'm just kind of thinking of the patient, why are you still doing this? They express that sentiment that this product has saved my life. This product -- I'm alive because of this product. And because of that, I think there's an inherent human nature aspect of loyalty to whatever has saved your life and has become like in some ways, your closest friend. In fact, many people name their pumps and whatnot. So it's the same sort of story with the inhaled product. There are many, many patients. I mean, actually, as Mike just said, you know, thousands of patients to whom they feel that the Tyvaso product with its Optineb nebulizer, if not per label having saved their life has at least been life-changing for them and has given them back a great deal of their life. So I think for patients who have had this very positive experience with the Tyvaso, Optineb, there would be a natural reluctance to just like chuck it off to the side and grab the new kid on the block. For newly diagnosed patients, I think those newly diagnosed patients would definitely preferentially along the percentage splits that Mike referred to earlier, although he was talking Group III, Group I, I would say something like that 75-25 could also very likely be the case with Tyvaso versus the legacy Optineb. So the long and short answer to your question, Jess, is that I do not believe the legacy Optineb is like going away. As with all of United Therapeutics products, we are like obsessed on supply chain. We were obsessed on supply chain country and world was obsessed on supply chain. So we have a multiyear inventory of our drugs, of all of the devices. All of our devices and our drugs, they're made in the United States. We store them at multiple different locations that are hundreds of miles away from each other. So I think that you're going to see continued use of Tyvaso nebulizer, Optineb in addition to Tyvaso DPI.

Jessica Fye

analyst
#25

Got it. Bit of a specific question, but now that you have resubmitted the NDA as of December, any inclination yet as to whether you expect FDA to classify it as a Class I or Class II response?

Martine Rothblatt

executive
#26

We don't know at the moment. We're waiting for like a bureaucratic step at the FDA. So we'll -- which we should know about within a month or so.

Jessica Fye

analyst
#27

And can you tell us if the resubmission was based on resolving the inspection with the third-party vendor? Or did you move that function they were providing elsewhere in order to resubmit?

Martine Rothblatt

executive
#28

Yes. We did move that function, Jess. And I really have to give a huge, huge kudos to our manufacturing, quality assurance and regulatory teams that we were able to successfully move that entire process within 69 days. And I know the people who aren't like -- I mean I totally geek out on all this, but a lot of people aren't this deep, they're kind of type, okay, you move to process. It's so much vastly more complicated than you just move the process. These are highly regulatorily controlled processes for the assays and tests. And everybody is very busy right now. Everybody is short staffed. But we -- these are multipart processes, but our team successfully accomplished this and I'd go out on a limb, and I would say it would be extremely rare in our wonderful pharmaceutical industry that these type of tests could go from like 0 to everything is checked out and done and submitted to the FDA in 69 days through Thanksgiving and all of that sort of stuff, although we did it.

Jessica Fye

analyst
#29

Right. We've just got an investor question popping up here that's relevant to this topic. Do you need any inspection at the facility that you moved this function to?

Martine Rothblatt

executive
#30

I don't know a specific answer to that level of question, but if it's required, I can assure you that we are on it like peanut butter on bread.

Jessica Fye

analyst
#31

Great. Maybe last one on Tyvaso DPI. I'm curious how you think about emerging competition like Insmed's TPIP, which is early, but might have the potential for less frequent dosing?

Martine Rothblatt

executive
#32

Sure. Mike, do you want me to keep talking or do you want to talk?

Michael Benkowitz

executive
#33

I mean, I can take it if you want. Sure. I mean, I think from -- from our standpoint, there's always a lot of, I think, emerging things in the space that we're certainly keeping our eye on. But a lot of this, I think, is still pretty early. And so I think I tell these things get a little bit further along, we tend not to -- not to get too excited -- too excited about it, because it's a long row to hoe. Stepping back, I think what I would just sort of remind everybody is that it's -- we have pretty big markets that we're playing in, right? Take Group I, which is the most competitive market that we play in, right? Roughly 50,000 patients diagnosed with Group I PAH, you still only have about 1/4 of those patients that are on process cycle therapy. And so there's really, I think, a lot of room for growth, not only with our products, but even with other products that come into the market. So I think the patient size there, the patient population is there to support multiple players. When you move into Group III, you look at the PH-ILD, there, like I said before, you get 30,000 patients, no competition yet, but we've got exclusivity through 2024, so we have had that market to ourselves for a few years and an opportunity to really I think kind of establish ourself as a premier product in that space. If our PERFECT study is positive for Tyvaso PH-COPD, we would expect, again, another probably a 3-year exclusivity for that patient population, which we estimate to be about 100,000 patients. And then finally, if we get into -- if the TETON study and IPF is successful, that's another 100,000 patients and we believe we'll get orphan designation there for 7 years. So I think it's certainly interesting and we're keeping an eye on emerging technology, but I think we're really kind of focused on the markets we're in, and the markets we're going into. And then we feel like we've got a lot of runway from an exclusivity standpoint ahead of us there.

Jessica Fye

analyst
#34

Okay. Makes sense. Maybe switching to the Remodulin franchise. What's the feedback you're hearing from physicians and patients using Remunity? And Martine, at the beginning, you kind of alluded to full launch for Remunity this year. Can you just elaborate a little bit on what that means relative to what was happening in '21?

Michael Benkowitz

executive
#35

Yes, I'll take that one, Martine. So I think overall, the feedback from the physicians and the patients that have had experience with Remunity has been quite positive. And if you just kind of recall and just to remind everybody, the differentiators relative to the MS3, I mean, start with the technology. I mean, the MS3 is, I guess, 25, almost 30 years old right now. It's still a great comp and works really well. But one of the things that Remunity does is, it brings 21st technology, 2 of a pump that was designed specifically for use with Remodulin. And so one of the kind of cool things if you want to kick out, there's actually no motors in the pump. It uses acoustic volume sensing technology to control the Remodulin flow rates without the use of a motor. So tech wise, it's pretty cool. But then I think from a patient standpoint, the things that they really appreciate about the technology are the size. I mean, the form factor, the profile is really, really small. It's much more discrete than what they're are used to with the MS3 or certainly the CAD legacy in the case of IV patients. It's prefilled. So patients are getting prefilled cassettes shipped to them every couple of weeks, so all they have to do is really kind of snap out their old cartridge, snap in a new cartridge, they're good to go. They do that every 2 to 3 days. And then I think the other thing that patients really appreciate is the fact that it's water resistance that they're out having to go through, a big rig of a rule if they want to take a shower or jump the pool, kind of only cover things up and deal with those issues like they are with the MS3. So I think there are lot of attributes, a lot of things that patients and physicians are really excited about. One of the things that Jess, you and I have talked about, and I think on prior calls is that, logistically, this is probably one of most complicated launches we had. Just because of the fact that you have specialty pharmacy having to fill the cassettes and they're having the packaged cassettes and then shift those to patients. And so when we launched in 2021, we went through sort of a kind of a phase launch because we wanted to make sure happen is that those logistical issues worked as smoothly and seamlessly as we needed them to, because we wanted to have the patients, we wanted the patients to have a great experience and love the pump as much as we do. So that process worked well. We learned some things along the way. So one of the things that we've learned along the way is that -- I guess the other feature about the pump that I didn't mention is that it has a wider rate of notifications and alerts. So from a safety standpoint, it's really great because if something goes wrong, the patient is going to know. It turns out that I think some of those alerts are more sensitive than what we were expecting, not to the point of creating a safety issue, and I think for patients that had started on Remunity for the most part, not enough for them to say, "I don't like this pop, I'm going to go back to the old one" but there was enough noise around that to where we were like, "hey, you know what, we need to maybe kind of tweak the settings on these alerts" because it was -- I would put it in a category of annoyance. And so again, from our standpoint, we want our customers to have an amazing experience. Our patients have an amazing experience with this pump. And so we kind of slowed down bringing patients on to Remunity until we kind of changed the settings on the pump around these alarms. That process is ongoing. We believe with our partner, DEKA, we fix that. And so we kind of here, we get probably to the middle of the first quarter. We'll kind of move back into bringing patient back on at the same rate or faster than we were in 2021.

Jessica Fye

analyst
#36

Okay. And I guess looking ahead, understanding there's probably a mix of new patient starts and switches to Remunity, what makes someone eligible or likely to be switched from Remodulin and its current form, current pump onto Remunity and then why?

Michael Benkowitz

executive
#37

Yes. I mean, I think from a -- just from an eligibility standpoint, whether it's existing patients or new patients, I mean, I would say both are technically eligible to start on Remunity. I would refer back to what Martine said or explained about some of the loyalty that patients have to their existing devices when we were talking about the Tyvaso nebulizer versus Tyvaso DPI. And I think that's that dynamic exist here with Remodulin, maybe more so. Because, again, you're dealing with sicker patients, typically, if you're on Remodulin, you're typically a sicker patient and maybe if you're on an oral or you're on inhaled prostacytline. And so if a patient is feeling like they have their disease under control, they're managing that. I think the reluctance to switch to a new technology, maybe greater. Certainly, with newer patients, I think they're going to be much more inclined to start on Remunity just because of all the features I described. We won't see patients transition. We have seen patients transition from the MS3 over to Remunity. And I think that will continue. I think that that transition will just play out over a longer period of time. But ultimately, we expect the vast, vast majority of patients to be on Remunity over the long term. And then certainly, with the next version, the next 2 versions, when you have the manufacturing filled version of Remunity and then eventually RemoPro, which Martine mentioned earlier, we would expect all patients to be on Remunity.

Jessica Fye

analyst
#38

Maybe it's a good time to turn to RemoPro. So what exactly is the status there? And how much larger the patient population do you think it could capture compare to Remodulin.

Martine Rothblatt

executive
#39

Yes. Thanks, Jess. So the status is that we take RemoPro into human testing in, like I said, within the next couple of months. And then we will do a formal bioequivalence study, very analogous to what we did with DPI and the Optineb. And I believe all of that activity is consistent with us being able to file for RemoPro approval either in late '23 or worse case early '24 and then have a full year or more than a full year of product launch to help contribute to our 25 by '25 goals. The potential of it per our 25 by '25 goals is to contribute to more doubling of our current 4,000 Remodulin patients up toward 8,000. So it would end up really carrying a lot of the water in terms of that roughly 30%, 33% contribution to the 25 by '25. Another thing to mention in that regard is, we're pretty also obsessive about continuing to improve our products. And the way we look at things at United Therapeutics is that like in manufacturing, we say the process is the product, it's something similar to that from the patient standpoint. There is the API, then there is the device to deliver the API, then the whole supply chain to get the device deliver the API. So for example, Mike was talking about what we're doing to make sure that our patients have an awesome experience with Remunity, but one complicated logistics that he mentioned was the need for the pharmacies to fill the cassettes. So we are right now midstream in a process to implement a machine, what we call a machine fill process of filling the cassettes that would eliminate the need for the pharmacies to be in the middle. And there -- oh, I guess it's been over a year now, we signed an agreement with Philips to act, to use their technology to build an automated Remunity machine fill plant down in our Jacksonville facility. So that RemoPro product will be able to be delivered through a complete streamlined supply chain with the -- all of the filling of the Remunity cassette being done at our own factory plant where we also are making the product itself in conjunction with this very nice Philips technology. So all of that combined, I think, puts us on very solid ground for Remunity being a product together with RemoPro that has really long legs. The technology and the patents behind the Remunity delivery system, including the Philips technology for the machine fill version of it, go way into the late 2030s. So this product, as seen by the patient is something that from the United Therapeutics standpoint is all part of patent-protected technology going out to the late 2030s and provides a vital benefit to the patients as the -- I think, the best way to parenterally deliver treprostinil.

Jessica Fye

analyst
#40

Got it. Let me sticking with pipeline opportunities, you partially answered this one earlier, but can you just provide a status update on both the TETON and PERFECT studies? Talk about how enrollment is going and when we could see data?

Martine Rothblatt

executive
#41

Yes. So that's part of our goals for '22 is to have full enrollment of the PERFECT study. And I believe we should reach just about 50% enrollment on the TETON study. So those are things that we're doing in '22. I would say that's probably -- that's probably like my personal #1 goal and responsibility just as Mike's on the commercialization side. So we are doing a really good job of it. I think that we are progressed in terms of PERFECT, with the enrollment, the randomization, blah, blah, blah, all that kind of stuff to achieve full enrollment by the end of this year. The study has an endpoint, a 6-minute block endpoint, standard pulmonary hypertension endpoint. So that's good because we won't have to wait forever for the data. The last patient is in -- by the end of this year. I think it's quite reasonable to expect data in the middle of '23. And then the team would work very hard to get the NDA filed before Christmas so that we could launch that product in '24. And I know we probably sound like a broken record here and contribute to our 25 by '25 goal in '25. But that is what we're all focused on. All of these multi-faceted activities, all feed into that 25 by '25 goal. With regard to TETON, we're often running. We're building a lot of new relationships. We were not previously dealing with the pulmonologists that treat just pulmonary fibrosis. So we are making a lot of those solid relationships. We're randomizing patients. In fact, things are going so well that we decided to launch an international complement to the U.S. study, as Mike said, we formed a -- -- I wouldn't say we formed, we expanded our long standing good partnership with Ferrer. So we're really excited about the worldwide potential in pulmonary fibrosis. And so we launched a second study, TETON 2 with a top CRO that in fact, had just been deeply involved in another IPF study. So really have super relationships throughout the world. And I think that will allow us to like jump out of the starting block faster than ever on TETON 2.

Jessica Fye

analyst
#42

Okay. Great. Maybe one more question on the pipeline before we wrap up because we're running out of time here. For the PERFECT study, curious how similar or different the PH that COPD patients get is from the PH that ILD patients get and whether the increased results read across to success in PH COPD and why or maybe why not?

Martine Rothblatt

executive
#43

So the way -- like it's a great question, it's scientifically a very interesting question. But of course, the only real answer to that will be to see the outcome of the PERFECT study to see to what extent those results are correlative with the ones from, say, increase in ILD. But I will say that the reason we launched this study was because of the Phase II work that we had funded that showed the largest improvements in 6-minute block that we had ever seen were in the Group III patients with ILD and then separately in the Group III patients with COPD. So exactly why that is, what exactly is the mechanism of action. You're getting kind of a little bit into the black box of biology there that I don't think anybody really knows. I could talk about this pathway and that pathway, but I think I would just be like talking. And biology is going to do its own thing. But as an empiricist, I would say the Phase II data was like you would be an idiot not to go ahead and develop this into Phase III. So we did develop it in the Phase III. The patient uptake has been good. The physician uptake has been excellent. When physicians are excited that is a good positive sign that that they understand and believe in the mechanism of action. More than that, it's not too long to wait now just about 11 more months and will be fully enrolled.

Jessica Fye

analyst
#44

Great. So maybe just to wrap up, we kind of talked about near-term business opportunities, we talked about longer-term pipeline opportunities. I was hoping you could tie it all together and talk about your long-term vision for the company and what we should be looking forward to?

Martine Rothblatt

executive
#45

Thanks, Jess. It's always very interesting to be asked that type of a question, because that also is very much of a black box like which directions will a company go into. It would have been hard to predict a few years ago that we would be so committed to the IPF indication as we are now. But we were led there by the data that came out of the research that we were doing in other fields. But the way that Mike and I really run the company is that our goal is for looking, let's say, 5 or 10 years down the road, to have thousands of people working at United Therapeutics. And as many of them as possible feel that they are having the absolute best career development experience of their life, working on therapies and medicines that can save the lives of thousands of other people. And that's the vision that we work toward. Right now, we're at 1,000 employees. I think several years down the road, we'll be at several thousand employees. Right now, we're one of the places that people love to work out more than any of our peers and is reflected in all kinds of surveys and whatnot. We want to be able to scale that to having thousands of people, feeling they are having their best career development experience, creating medicines to help save the lives of thousands of other people.

Jessica Fye

analyst
#46

Great. Well, we'll leave it there. So thank you so much, Martine and Michael and James and Dewey for joining us today, and Happy New Year, everyone.

Martine Rothblatt

executive
#47

Happy New Year to you.

Michael Benkowitz

executive
#48

Thanks, Jess.

For developers and AI pipelines

Programmatic access to United Therapeutics Corporation earnings transcripts and 32,000+ others is available through the EarningsCalls.dev REST API. Plans from $24.99/month — full transcripts, speaker segments, full-text search, and the recently-added /api/v1/transcripts/recent polling endpoint for ETL pipelines.