United Therapeutics Corp

NASDAQ:UTHR

Earnings Call Transcript 2021-Q1

[Operator]

Good morning, and welcome to the United Therapeutics Corporation First Quarter 2021 Earnings Call. My name is Phyllis, and I will be your conference operator today. [Operator Instructions] I will now turn the conference call over to Mr. Dewey Steadman, Head of Investor Relations at the United Therapeutics.

[Dewey Steadman, Head, IR]

Good morning. It's my pleasure to welcome you to the United Therapeutics Corporation first quarter 2021 earnings call. Accompanying me on today's call are Dr. Martine Rothblatt, our Chairperson and Chief Executive Officer; Mr. Michael Benkowitz, our President and Chief Operating Officer; Mr. James Edgemond, our Chief Financial Officer and Treasurer; and Dr. Leigh Peterson, our Senior Vice President of Product Development.

Remarks today will include forward-looking statements representing our expectations or beliefs regarding future events. These statements involve risks and uncertainties that may cause actual results to differ materially. Our latest SEC filings, including Forms 10-K and 10-Q, contain additional information on these risks and uncertainties. We assume no obligation to update forward-looking statements.

Today's remarks may also include financial measures that were not prepared in accordance with U.S. generally accounting - generally accepted accounting principles. Reconciliations of our non-GAAP financial measures to the most directly comparable GAAP financial measures can be found in our earnings release available on our website at www.unither.com.

Today's remarks may discuss the progress and results of clinical trials or other developments with respect to our products. And these remarks are intended early to educate investors and are not intended to serve as the basis for medical decision-making or to suggest that any products are safe and effective for any unapproved or investigational uses. Full prescribing information for the products are available on our website.

Now I'll turn the call over to Dr. Rothblatt for an overview of our first quarter 2021 financial results and business activities of United Therapeutics. Dr. Rothblatt?

[Martine Rothblatt, Chairperson and CEO]

Thank you, Dewey. Good morning, everybody. Thanks for joining us on our first quarter 2021 earnings call.

I'm going to start off with a sort of 30,000-foot overview. And then after that, I'll open the line for any questions. If there are business questions, I'll refer them to our President, Michael Benkowitz. If there are financial questions, I'll refer them to our Chief Financial Officer, James Edgemond. And any scientific questions, I'll refer to our Senior Vice President, Dr. Leigh Peterson. I may even pass the odd legal question to Dewey.

Well, I think from a 30,000-foot viewpoint, the main quantitative goal at United Therapeutics is what we call 25/25. This means increasing the number of patients that we are helping with our main medicine from over 8,000 patients today to over 25,000 patients by 2025.

This quantitative metric is an especially important one to call out today because today is World Pulmonary Hypertension Day. It's the one day in the whole calendar that countries throughout the world have agreed to focus their thoughts, attention and for many patients and families' prayers on the hopes for getting better therapies and treatments for people with pulmonary hypertension.

We expect about half of the roughly 18,000-patient growth that we will require to meet our 25/25 goal will come from patients with what's called WHO, or World Health Organization Group 1, pulmonary arterial hypertension, and about half of that 18,000-patient growth will come from patients with World Health Organization, or WHO Group three pulmonary hypertension.

Let me start by breaking down how I believe we'll get our 9,000-patient growth in the Group one category, and then I'll talk briefly about the second 9,000-patient growth in the Group three category. So for the half of the growth we expect in WHO Group I, we expect that to come in roughly equal thirds from our Remodulin product, our Tyvaso product, and our Orenitram product. We think we'll be able to gain about 3,000 new patients on Remodulin, particularly due to the convenience and patient friendliness of our newly approved Remunity Pump.

We think we'll be able to gain about 3,000 new Group one patients on Tyvaso because of the convenience and patient friendliness of our new Dreamboat dry powder inhaler, which we have filed for approval and we hope to be approved in December of this year. This is a finger-sized device compared to the large liquid nebulizer that we use today.

And then finally, we think we'll be able to gain about 3,000 new patients between now and 2025 on Orenitram because of the constantly growing appreciation of the efficacy of Orenitram in reducing morbidity and mortality.

Now please keep in mind that there are about, in the U.S. alone, 45,000 patients in WHO Group one being treated with non-United Therapeutics drug and about 15% progress in their disease annually per the Kaplan-Meier curve of their trials. So overall, there are some 50,000-plus patients being treated for pulmonary hypertension, about 8,000 of them currently being treated by our drug and the balance somewhere between 40,000 and 45,000, probably closer to 45,000 being treated with non-United Therapeutics drugs. So it is very achievable. There is plenty of market space. There's plenty of help-the-patient space available for us to grow in WHO Group one from the current 8,000 patients we're treating to something like 16,000 patients that we expect to help by 2025.

Now I mentioned that our total goal is to help 25,000 patients with pulmonary hypertension by the year 2025. And the second half of that, achieving that 25/25 goal comes from penetration of WHO Group three by Tyvaso for our FDA approval that we achieved just last month. Now within WHO Group 3, there are something between 30,000 and 40,000 patients with pulmonary hypertension. We are told to actually by prescribers that the actual number is quite a bit larger than even 40,000. It's somewhat similar to the situation with WHO Group 1. You'll see various accounts that there are 40,000 patients, 50,000 patients.

We are told by prescribers that actually, there are quite a much larger number of patients with WHO Group one pulmonary hypertension. And it is a difficult disease to really quantify and identify because it's a disease that is identified by excluding numerous other conditions whose symptom limit those of Group one pulmonary hypertension. Anyway, back to the Group three pulmonary hypertension, there, whether the number is 30,000, 40,000 or larger, we are the only drug now approved for this IPF market segment of IPF patients with pulmonary hypertension.

And this portion of patients, also called ILD-PH patients, are actually the ones who have the most rapid decline. In other words, if you have the interstitial lung disease and you have pulmonary hypertension, the statistics show that your decline to death is much more rapid than the patients with interstitial lung disease that do not have pulmonary hypertension. So given that there are well over 30,000 patients who need treatment and that our goal by 2025 is to capture at least 9,000 of those patients, I think it's a very achievable goal in terms of comparison to the capturable market size.

So in addition to the 25/25 goals that we are highly focused at and is our number one quantitative priority, let me also mention a few of what I would kind of call free options in our pipeline. And by free options, I mean, we are doing these things. I believe they have a very high probability of success because they are in either late phase clinical trials, Phase III clinical trials, where statistically the odds of success are quite high or they are developments that we would call bio comparable therapeutic, meaning that they are comparable to things that are already approved by the FDA. So let's talk about some of these "free options" in our pipeline. First of all, there is a Phase III study of Tyvaso in COPD-associated pulmonary hypertension.

The name of this Phase III trial was called our PERFECT study, and it should complete its enrollment by the end of next year. Second, we have a Phase III trial of a drug called ralinepag in Group one pulmonary hypertension. This study is also doing very well, and I think it should probably complete its enrollment also by the end of next year. In fact, it's just under 1/3 enrolled at this point in time in the lead of the ralinepag trials called the outcome - ADVANCE OUTCOMES trial.

Next up, we have yet another Phase III trial. This one of Tyvaso in interstitial - I'm sorry, in idiopathic pulmonary fibrosis, IPF, that should be completed in this enrollment or I would say the first half of '23. This is very interesting for United Therapeutics because it is actually the first time that we have developed non-oncologic treatment outside of around having anything to do with pulmonary hypertension and yet using a drug, Tyvaso, which has just been approved in an adjacent therapeutic class to the target of this trial.

So let me unpack that for a moment. The approval that we just have in an adjacent therapeutic class is the approval the FDA granted us just this month based on our increased trials, which was in patients with pulmonary fibrosis as well as pulmonary hypertension. So they had kind of that double strike, and that's what I mean by an adjacent therapeutic class to pulmonary fibrosis without pulmonary hypertension.

And in that adjacent therapeutic class, we were able to show improvement in the key therapeutic clinical markers of pulmonary fibrosis. That key indication is called forced vital capacity or just for short FVC. And we're showed - be able to - we were able to show improvement in that, that was shown and talked about also in our New England Journal of Medicine publication on the trial. And so now we're approved in the adjacent class.

So we are now taking that same drug that's proven safe and effective in IPF with pulmonary hypertension and developing it in IPF without pulmonary hypertension. So again, I think the odds are very strong in our success, and it's a well-designed study.

By the way, we call that study, the TETON study since the head of our clinical and product development program for the pulmonary fibrosis activity, Dr. Leigh Peterson, hails from the Grand Teton State of Wyoming.

Third, we have another Phase III study, a pivotal study, a registration study of our eNOS gene therapy treatment for pulmonary hypertension. And this one should also complete its enrollment by the end of next year. And then last but not least at all, in fact, in many ways, close to first, is our bio comparable once-daily form of Orenitram and our bio comparable less painful forms of Remodulin, both of which should be launched by the key quantitative date of 2025.

So to wrap up from this high-level overview, we've got an awful lot in the market. It's a market which is very capturable by us and much larger than the number of patients we're currently helping plus a lot of new products to bring to the market. So I think if I was a parent of a patient with pulmonary hypertension, as I am, and there are tens of thousands of others - hundreds of thousands of others throughout the world, on this World PH Day, I would be very thankful and very hopeful about a bright and optimistic future.

With that set of introductory remarks, operator, if you could be so kind to open the lines, and I'll be happy to field the questions.

[Operator]

[Operator Instructions] Your first question comes from the line of Eun Yang with Jefferies.

[Eun Yang, Jefferies]

Thank you. I have a couple of questions. One is on Group three PH-ILD. So we heard from physicians that they see their patients every three to six months. And I know, Martine just mentioned that Group three is a more difficult disease to diagnose and quantify compared to PAH. But is it like aside from targeting physicians, do you plan to target patient so that patients are actually seeking treatment with Tyvaso?

And the second question is on subcutaneous Remodulin. I understand that you may be limited to what you can say, but in 10-Q, the trial with Sandoz and Liquidia, you don't expect to begin before mid-2022 at the earliest. Does that mean that Liquidia could not - cannot launch the product until the trial is over? Thank you.

[Martine Rothblatt, Chairperson and CEO]

Thank you for your question, Eun. Nice to hear your voice this morning. Due to the shortage of time on the call, we're only going to be able to allow one question per inquisitor, if I could say that. So I'm going to just take the first question and refer it to Michael because it's really in the domain of a business question. Mike, can you help Eun with understanding what our strategy is with regard to ILD-PH?

[Michael Benkowitz, President and COO]

Sure. So let me - before I get to the specific question around the patient, maybe give a little bit of color around the PH-ILD launch. So Martine, I think, gave a great overview of sort of our 5-year vision of growing from roughly 8,000 total patients to 25,000 patients and roughly half of those being Tyvaso patients for various indications.

So with respect to the PH-ILD piece of that, I just want to reiterate what we've been saying since January. And that with this launch, we expect the number of patients on Tyvaso to approximately double by the end of next year. And the other thing that we've been saying is that it's probably not going to be a straight line from here to that doubling.

There'll be a little bit of a ramp and then something looks a little bit more hockey stick-ish as we get later into the year and certainly into next year. And that's due to various things around the fact that, and Eun touched on this, we have a new group of ILD treaters. So they're having to go through the process of getting their patients in, screen those patients, diagnose those patients and then clear the - get that cleared with payers.

So that process is ongoing. I will say, in the first three to four weeks of the launch, it's very early days. We're incredibly pleased with what we've seen in terms of referral activity, in terms of the number of referrals that have come in. We actually have patients that have started since the launch on Tyvaso.

And I'll also say that in the last week, I've been out meeting with six of the major ILD centers in the western half of the U.S. And I will say you to a person, to a center, all of those physicians are extremely excited about having a treatment option for these very sick patients. And again, all have said that we have - there is a meaningful unmet need here that we're going to be able to address with Tyvaso.

So we have been - since last summer, we're building relationships with these new treaters, educating them on the disease. Now we're able to talk more openly about Tyvaso, the benefits of Tyvaso, how you get a patient started now that we have the approval.

And then the second point to that, coming back around to Eun's question is, yes, of course, we are - we have multiple patient engagement and education efforts ongoing. Obviously, with FDA guidelines and other restrictions on being able to communicate with patients, we have to be a little bit more careful in those engagements for very good reasons. But we do have a, I would say, very robust patient engagement and education effort ongoing.

[Martine Rothblatt, Chairperson and CEO]

Awesome, Mike. Thanks so much. Operator, next question.

[Operator]

Your next question comes from the line of Liana Moussatos with Wedbush Securities.

[Liana Moussatos, Wedbush Securities]

Thank you for taking my question and congratulations on all the progress. How are you going to commercialize Tyvaso solution versus Tyvaso DPI at the end of the year, presuming approval?

[Martine Rothblatt, Chairperson and CEO]

Thanks so much, Liana. So great to hear your voice again as well. So that's, again, squarely a business question. So Mike, can you help describe the differential launch plans?

[Michael Benkowitz, President and COO]

Sure. So we're really excited about the prospect of bringing - being able to bring the DPI to market. We think that it certainly provides a more convenient, easier way to deliver Tyvaso. But if you kind of step back and really kind of look at the history of United Therapeutics and what our objective and missions has been or have been, and that is the right patient options. Patients with options - different ways to take our medicines in a way that is - meets kind of where they are in their disease, and that's most convenient to them. So we started with Remodulin and then we developed two versions of Remodulin.

We developed Tyvaso, which was an easier way to take treprostinil inhaled, and then eventually got to Orenitram, which is obviously the simplest version of all of them. And so I look at DPI as really kind of fitting in. It's fitting kind of into that strategy of providing patient options. And so I would expect that with the DPI, you're going to have a significant number or majority of patients that are going to want to transition to the dry powder inhaler, and that's fantastic. You will have other patients that, for various reasons, may choose to stay on the nebulizer. And that's great.

We're going to continue to support that product. It's a great product. There is going to - I do think that there is going to be a patient type that is maybe better suited for that product over the DPI. Don't hold me to this. I think when the dust settles, you're probably looking at something of around a 70-30 split between dry powder and nebulizer. So we're going to continue to provide both options, support both options. And I think they're both great delivery devices for the right patient.

[Martine Rothblatt, Chairperson and CEO]

Perfect. Thanks so much, Mike. Operator, next question, please.

[Operator]

Your next question comes from the line of Hartaj Singh with Oppenheimer & Company.

[Hartaj Singh, Oppenheimer & Company]

Great, thank you for the question and great work. Got a question on Orenitram. So since the approval of the FREEDOM-EV, the addition to the label, the study, FREEDOM-EV study, Martine, it has - the growth trajectory of the product has definitely increased substantially in the last two years. Johnson & Johnson with Uptravi, the old Actelion drug, indicates about 20% on average growth every quarter. Some of that from market share, some of that from an increase of the overall market size. Can you give us color there and also help walk us through how you see Orenitram adding those additional 3,000 patients, as you had mentioned earlier in your prepared commentary? Thank you for the question.

[Martine Rothblatt, Chairperson and CEO]

Great. Hartaj, so awesome to hear your voice this morning as well. Very interesting question. Very much in the range of what we call business and market strategy. So Mike, if I can ask you to provide Hartaj with some more insight into the business ramp plans for Orenitram?

[Michael Benkowitz, President and COO]

Yes. Happy to do that. Yes. So I think with Orenitram, I think you have to kind of, again, kind of step back and look at the timing of the launch and kind of where that kind of fit with COVID, right? So I will say, at a high level, I think as a company, we continue to be very pleased with the physician reaction to the EV data. I think our underlying patient trends continue to be very solid.

And I would say, in particular, I think, in late Q1 and even heading into Q2, if you look at referrals and starts, we've seen actually a nice uptick over the prior months. I will say, I mean, hindsight to 2020, as I kind of look back over the last 18 months and think about when we received the label expansion, which was October of 2019, when the publication came out, which was in March, like literally two days, I think, before the world shut down, to your point, Hartaj, we did see a nice uptick in that kind of initial 4 to 6-month period.

And then it did get sort of muted. So we had a nice ramp and then it just kind of plateaued a little bit there for a little while. And I think, obviously, as I look at it, I think it's a function of kind of COVID shutting down, the lack of access that we had to physicians and just impacting our ability to really kind of get out and continue to communicate the EV data.

I mean we certainly had some access during COVID virtually, but nowhere near the axis that we had pre-COVID. So I think that we're starting to come out of this a little bit. We're getting that access again. We're able to get in and have really, I think, fruitful discussions with physicians and really kind of talk about the Orenitram value proposition. I think the doctors are seeing that.

And like I said, I think we're starting to see that uptick. And so I think as we look into the future, I think, long-term, as Martine said, I think we do have a lot of runway there with Orenitram as we continue to educate the physicians on the value proposition. I also think with some of the data that we've talked about in prior calls around this idea of treating patients to pressure.

So putting - starting them on Remodulin, really trying to drive down their pulmonary arterial pressure and then switching them over to Orenitram, I think that is continuing to get a little bit of - certainly a lot of attention by physicians. And I think over the long-term, that's going to give us, I think, another - sort of another leverage point with Orenitram. And then I think the third one that we see in sort of the latter half of the 25/25 plan that Martine laid out is OreniPro, the once daily.

So that provides, as we're talking about Tyvaso, a more convenient way to take Orenitram and potentially with a better tolerability and an ability to dose faster. So maybe some additional attributes there. So I think it's really sort of a combination of building on and restarting - build on the momentum we've seen in the last quarter and continuing to leverage the EV data, leverage this treat to pressure idea that's getting some attention and then the once-daily formulation that's coming in kind of the back end of the 25/25 time frame.

[Martine Rothblatt, Chairperson and CEO]

Excellent, Mike. Thank you so much. Operator, I believe we have time for one last question.

[Operator]

Your next question comes from the line of Joe Thome with Cowen & Company.

[Joe Thome, Cowen & Company]

Good morning and thank you for taking my question. Just a quick one on the TETON program. As you start thinking about adding more trials here and expanding Tyvaso's opportunity, is it possible to study the DPI alone in some of these trials, maybe the increased adherence or ease of use would maybe drive better treatment outcomes? Or do you have to start with the nebulized version and maybe do a PK study to show equivalents there?

[Martine Rothblatt, Chairperson and CEO]

Yes. Just that is, I would say, that was close to a scientific question as we've got so far this morning. I'd like to bounce that one over to Dr. Peterson to discuss.

[Leigh Peterson, SVP, Product Development]

Yes. Thank you for the call or the question. So we have been told or actually we have, in writing, that once we receive approval for Tyvaso for new indications and once we receive approval for the Tyvaso DPI for PAH, really, then the approval will automatically be granted for the additional indications for DPI as they come along.

So given that, it really depends on what we decided at the time with regard to supply, ease of use, where patients are with wanting to be on our DPI versus the inhaler, the Tyvaso inhaler, we don't exactly know. I mean we have studied the DPI - the Tyvaso DPI in - for human factors in all different indications, whether it's PAH or PH-ILD or even some ILDs without diagnosed PH.

So as far as that goes, they're quite similar. But there is some question, and I can't say one way or the other, that some patients with different indications might actually prefer a nebulized form. It might be a little bit easier for them to take and get a good dose versus the dry powder inhaler because they actually breathe in for the dry powder inhaler versus the nebulizer is a little bit more passive. So we're going to - as soon as we continue to do additional studies, we'll investigate that. But right now, TETON one is already in the work. The sites are open, and we have a protocol. We should have the enrollment any day now, and that's with Tyvaso.

[Martine Rothblatt, Chairperson and CEO]

Thanks so much, Dr. Peterson. Really appreciate it. Well, we really have been happy to have an opportunity to share our business, scientific and our financial progress with everybody during this first quarter earnings call. We've been happy to share with everybody on this World Pulmonary Hypertension Day that by the end of next year, we expect to be able to double the number of patients that we have on Tyvaso.

And by 2025, we expect to be able to help roughly three times as many patients with our treprostinil suite of products that we're currently helping today. It is a fact that based on the Kaplan-Meier curves of all of the main approved therapies that about 15% to 20% of the patients progress in their disease signs and symptoms each year. And just to make the math easy at 50,000 patients, 15% a year progressing is 7,500 a year, 20% progressing is 10,000 a year.

So whether the number of patients needing a new therapy is 1,500 a year or - I'm sorry, is 7,500 a year or 10,000 a year, both of those numbers are well above the growth that United Therapeutics requires to go from 8,000 patients that we're helping today to 25,000 patients that we will be helping by 2025.

So I believe that the goal is realistic. The need is real. And our capabilities are strong, impressive and ever growing. Thank you so much, everybody, for joining us on this call and look forward to seeing you at least virtually at an upcoming healthcare conference. Operator, you can wrap it up.

[Operator]

Thank you for participating in today’s United Therapeutics Corporation Conference Call. A rebroadcast will be available for replay for one week by dialing 1800-585-8367 with international callers dialing 1416-621-4642 and using access code 3044748.