Good morning and welcome to the United Therapeutics Corporation's Third Quarter 2020 Earnings Call. My name is Kenzie and I will be your conference operator today. I will now turn the conference over to Mr. Dewey Steadman, Head of Investor Relations at United Therapeutics.

Good morning. It is my pleasure to the United Therapeutics Corporation Third Quarter 2020 Earnings Call. Accompanying me on today's call are Dr. Martine Rothblatt, our Chairman 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 Vice President of Product Development. Remarks today will include forward-looking statements representing our expectations or beliefs regarding future events. These statements involves risks and uncertainties that may cause actual results to differ materially. Our latest SEC filings including Form 10-K and 10-Q contain additional information on these risks and uncertainties. We assume no obligation to update these forward-looking statements. Today's remarks may also include financial measures that were not prepared in accordance with US generally accepted accounting principles or GAAP. Reconciliations of non-GAAP financial measures to the most directly comparable GAAP financial measures can be found on our earnings release available on our website at ir.unither.com. Today's remarks may discuss the progress and results of clinical trials or other developments with respect to our products. These remarks are intended solely 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 these products are available on our website. Now, I'll turn the call over to Dr. Rothblatt for an overview of the third quarter 2020 financial results and business activities of United Therapeutics. Martine?

Thank you, Dewey. Good morning, everybody, and welcome to our third quarter earnings call for 2020. I'm going to be joined on the call today by our President, Michael Benkowitz; our Chief Financial Officer, James Edgemond; and our Head of Product Development, Dr. Leigh Peterson. We really have all good news to report today. So it's going to be a very fun earnings call. Let me divide the good news into two categories: pulmonary hypertension and pulmonary fibrosis. Let's start with pulmonary hypertension. We had over the past quarter the highest number of patients on our treprostinil medicines ever. In addition to that, we've seen double-digit growth in the number of our patients on Orenitram and the number of patients on Tyvaso, that's double-digit growth year-to-year, quarter-to-quarter. On top of that, we've also had solid Remodulin performance. And we expect this solid Remodulin performance to be even further enhanced with two planned launches for 2021. First, the Remunity launch for the patient on subcutaneous forms of Remodulin; and secondly, the ISR or Implantable System for Remodulin launch for patients on the intravenous form of Remodulin. These new product launches are important, because most pulmonary hypertension patients declined Remodulin due to its grave difficulty of delivery. Indeed, most pulmonary hypertension patients die without ever having access to Remodulin therapy. Speaking of our pipeline for pulmonary hypertension, also of great significance is our accelerating progress on ralinepag. We expect both the ralinepag outcomes and the ralinepag capacity Phase 3 trials to be half enrolled in 2021 and fully enrolled by the end of 2022. Let me then to some of the good news related to pulmonary fibrosis. We remain on schedule to launch our Tyvaso product for IPF-associated pulmonary hypertension in April '21, subject to FDA approval on its PDUFA date. We…

Great. Thanks, Martine. Good morning, everyone. As Martine said, overall, we're extremely pleased with our reported revenue performance in the third quarter, highlighted by strong double-digit year-over-year growth at Orenitram, Tyvaso and Unituxin. And when you adjust our revenue growth to account for the Excess Order that occurred by one of our distributors in the third quarter of 2019, that growth is even stronger. On this adjusted basis, US Remodulin revenues grew year-over-year and 5% sequentially, and Tyvaso and Orenitram revenues grew by approximately 30% each year-over-year. We periodically caution that our revenues represent sales to our distributors and may not reflect underlying demand for our products. Happily, we can report that revenue growth we saw in Q3 is supported by growing demand in the US as reflected by the following highlights during the quarter. First, after experiencing some COVID-related softness in new patient start earlier during the pandemic, total treprostinil starts returned to pre-pandemic levels during the quarter. Second, as Martine noted, the number of patients utilizing one of our treprostinil products reached another all-time high. This is the fourth quarter in a row that we've achieved an active patient census record for our treprostinil products. We have a record number of patients benefiting from Orenitram, reflecting continuing and increasing adoption of this therapy by physicians following the FREEDOM-EV label expansion last year. We have more patients benefiting from Tyvaso than we have had in approximately five years prior to the commercial availability of oral prostacyclin class medicines. And finally, we are just shy of a record number of patients utilizing Remodulin. In addition to those highlights, we're seeing increases in average dose levels and a length of time our patients stay on our medicines, both of which positively impact our revenues. So we're very pleased all the way…

Thanks so much, Mike. Operator, I'm now happy to field the calls, and I'll direct them amongst Dr. Peterson, James Edgemond and Mike Benkowitz as appropriate.

Thank you. Our first question comes from the line of Hartaj Singh from Oppenheimer. Please go ahead. Your line is open.

Great. Thank you. And a really, really good quarter, Martine and team. So, thank you. Just a quick question on Tyvaso. We're seeing a definite uptick in the trend growth over the last few quarters, Martine, as you mentioned, year-on-year double-digit growth. Key opinion leader calls, we've noticed that there is a lot of overlap between the physicians treating PAH using Tyvaso there and physicians treating PAH ILD. Do you expect a lot of this enthusiasm to be occurring in those patients and then also Tyvaso uptick in that Group 3 patients occurring there? Thank you.

Thanks, Hartaj. Excellent question and good to hear your voice this morning. I think, the best person on this call to field the question would be Mike, because he is right in the middle of all of the details right now of actually assigning different regional sales managers and their teams amongst the different types of physicians those who treat patients with ILD that have never seen really pulmonary hypertension patient treatment options and those who are normal PAH treatment physicians that then in addition to that also treat patients with PAH associated with their ILD. So Mike would really have the best answers for that. And Mike, if you could provide some color on Hartaj's question.

Sure. Thanks, Hartaj, for the question. I think the short answer to the question is, it's hard to tell when physicians are writing or submitting referrals or prescriptions for Tyvaso, they're not indicating whether it's a PH-ILD patient or a PAH patient. So, it's a little hard to tell if there's some off-label use in that group. What I can tell you though about the treating community is, and as I said in my opening remarks, we do see some differences in that group. I mean, we - by our account in terms of those physicians that are treating PAH and those that are treating ILD, there is maybe only about a 20% to 25% overlap and there's sort of an inverse relationship between the number of PAH patients those doctors see and the number of ILD patients those doctors see. In other words, if you've got a doctor that sees a lot of PAH patients are seeing very few ILD patients and vice versa. So, what we can see on the referral form are the doctors that are writing the prescriptions. And so what we haven't seen is a lot of new prescribers, which tells us obviously that all of the vast, vast majority of these type Tyvaso prescriptions are coming in are being submitted by your typical PAH doctors. And given the just sort of the, I think, the underlying data that we see would lead me to believe that the vast, vast majority are for PAH patients, but again we don't have the visibility to know exactly whether they are PAH or PH-ILD.

Thanks so much, Mike. That's a great response. Hartaj, one little footnote I'd add to Mike's response is, it's very interesting to me just having studied this field for quite a bit that there are this very substantial quantity of patients in the Group 3, WHO Group 3 type of pulmonary hypertension, over 30,000 patients that for those patients, the systemic drugs are contraindicated. So here in the field of Group 1 WHO, we have upwards of 12 different medicines available to treat pulmonary hypertension. And there are just about as many Group 3 patients as Group 1 patients, but there are zero no approved medicines to treat those Group 3 patients. So it's a screaming unmet medical need. And we are just crossing every finger we could cross that on - in April on the PDUFA date that the FDA will give us clearance to translate the INCREASE clinical trial results into an approved treatment for those 30,000 patients that would be the only approved treatment for those patients. It was a great trial, the INCREASE trial, as you are well aware, Hartaj, we met all of our primary and secondary endpoints. So I think there is going to be a tremendous amount of excitement amongst the physicians that finally they have something to treat those 30,000 patients with. Thanks so much again. Operator, next question please.

Our next question comes from the line of Eun Yang with Jefferies. Please go ahead. Your line is open.

Thank you. For Tyvaso in new indication PH-ILD, can you comment on your regulatory filing ex-US? And then for TreT, it to use in PH-ILD, do you expect you would need to run another switching study similar to Phase 3 BREEZE? Thank you.

Yes. Thank you, Eun, for those very interesting questions and exploring some kind of areas that a lot of people overlook. So I'm glad you've asked those questions. At this point in time, we haven't made a firm decision with regard to the European filing schedule, mostly because we're just like laser focused on gaining FDA approval to deal with this huge unmet medical need that I just talked about, the 30,000 patients here. So we are kind of like a step-by-step strategy here, and first we want to be able to successfully get approval into the US, and then we'll consider what is the best approach to go as to Europe. The second part of your question is also quite insightful, because the Dreamboat TreT device is a much easier to use device than the Opti-Neb nebulizer that that we used in the INCREASE study and that we submitted for FDA approval on. And I think, most people feel that whatever the captured market would be for this nebulizer, that dry powder Dreamboat device could give you two, maybe three times larger market, because of its greater convenience. So, as you mentioned, Eun, we did - we do plan to file for approval of the Dreamboat device based on the current BREEZE study in the April - in April of 2021 and then that would take its normal FDA approval period. So as I mentioned in my opening remarks, hopefully, we can get that approved if not by the end of '21, then at the very beginning of '22. Now whether or not that Dreamboat TreT device is approved for just WHO Group 1, pulmonary hypertension, which is the population in which we did our BREEZE study or is also going to be approved for the WHO Group 3…

Our next question comes from the line of Marty Auster with Credit Suisse. Please go ahead. Your line is open.

Hey, everybody. Thanks for taking the question. Martine, will you indulge my two-decade tenure to let me ask two questions again?

Yes, Dr. Auster. One for being Marty and one for being doctor.

Thank you kindly. So, a couple of things I want to follow up on. First on TreT; you reported the healthy volunteer kind of part of that study was done. Can you comment at all on the what that data looks like in the equivalents to what you've seen with Tyvaso? And then can you also talk on TreT about what the supply means that you're anticipating would be and if you think you'll be able to kind of meet where the demand for that new device and that new system might be if you were approved on a first pass approval? The second question was on the xeno organ side. I think there is something like 20,000, 25,000 kidneys transplanted in the US each year. You probably know the number more specifically. But if you could, comment maybe on what the overall big picture opportunity? I know there is a very long wait list and I know there is lot of people that don't, they don't get served. What is the kind of addressable big picture market opportunity there and when do you need to think about pulling the trigger to make decisions on expanding and building out your DPF capabilities to kind of start thinking about how to meet some of that long-term demand? Thanks.

Okay. Thanks, Marty. Fascinating questions. Kind of things we could like talk in a coffee house for hours over, if we could talk in a coffee house. But with regard to the first question on BREEZE, the PK data, the buildup of TreT devices, Dr. Peterson, if you could, at least, talk about the PK side. And if you feel comfortable talking about the inventory side roll into that. And if not, I'll talk about that at the front end of xeno stuff.

Yes. Sure. Hi, everyone. Thank you all for calling in. Great to speak with you. So, you all know that the TreT study in healthy volunteers, that was - the primary objective of that was to show comparable PK between Tyvaso and TreT. And that has been completed and we're actually undergoing the data analysis right now. So I don't have the final output package to discuss with you. But I will tell you that - I mean, this was an open label study and we're seeing consistent results between the two medications. We're actually seeing - it appeared at least in early on that the TreT device actually seems to penetrate the lung a little bit better than Tyvaso with Opti-Neb, but that also might be because as you all might know, TreT, you get your entire dose from like one to two breaths. And so, you just need a tiny little cartridge. And if you breathe it in, and then you get the full dose, whereas with Tyvaso it can take like 9 or 10 or it takes several different breaths to - multiple breaths to get the full dose. And so, that can explain the lung penetration. But again, the final data are being analyzed literally as I'm speaking, and we will be able to get that up to you very shortly, but we definitely do not expect any surprises there.

Thanks, Dr. Peterson. Thank you very much. So with regard to the inventory, Marty, I think, we're going to be in good shape. In fact, just this week, at the request of our Head of Manufacturing, Pat Poisson, we allocated $5 million for inventory buildup for the TreT launch during 2021. So, he regularly visits the MannKind plant in Connecticut, where we do the manufacturing of everything. And I feel confident that we'll have adequate supply to meet our needs. As you probably recall from reviewing our proxy, how one of our companies for companywide objective is upon which everybody's bonus is based in the entire company is that we have to have a two-year inventory of all commercially launched products at the rate of the product take-up. So Mike Benkowitz and his commercial team, they would provide a forecast to manufacturing in terms of how many Dreamboat TreT patients we expect to garner in the first 12 months after launch, which would be mostly a 2022 thing. And then, so already in 2021, Pat Poisson and our manufacturing team are making sure that we have 2 times that amount of inventory built up for at the time of launch, so that we can crush that manufacturing and inventory milestone as we actually always have every year for as far back as I can remember. With regard to the xeno question, I think, your numbers are quite accurate, Marty. There are 20,000-plus kidney transplants done. If you get into what are called living related kidney transplants, where people designate a kidney to a relative or something like that, you get up into the 35,000 type of category. And the next number that comes up a lot is 100,000 people are waiting for kidney transplants and are currently on dialysis. And…

Our last question comes from the line of Joseph Thome with Cowen & Co. Please go ahead. Your line is open.

Hi there. Thank you for taking my question and congrats on the great quarter. Just a little bit on Tyvaso in ILD, can you comment a little bit on how well these patients are identified currently given that as you mentioned, there really aren't great treatment options for patients? Do you anticipate that the potential approval of Tyvaso in the indication will change this paradigm and is it your expectation that there are some PH-ILD patients sort of built up and waiting for launch? Thanks.

Sure. Let me suggest like we give you a two-part answer to that question, with the first part being given by Dr. Peterson, because she was ultimately in charge of the INCREASE study that produce the clinical result in the ILD patients, so she could speak most expertly about those results and which were reported at the abstract level at the ATS conference, and I believe are imminently going to be appearing in a peer-reviewed publication. So she could talk about kind of the medical thing of how well it worked in those patients. And then Mike Benkowitz, our President, who has spent a lot of time thinking about the market segmentation and the approach to the market, can speak more about the second part of your question. Dr. Peterson?

Yes, sure. So, as you know, Tyvaso works quite well in the patient population 2/3 population. And this is - this was ILD plus pulmonary hypertension. And in this population, for the INCREASE study, the patients actually did have data from a right heart caths, okay? So they had documented pulmonary hypertension. And up until now, since there was no medication for this group, the doctors didn't really if they suspected IPS, they - or even suspected pulmonary hypertension in this population, they didn't give the patients necessarily a right heart cath, because even if they did discover PAH, there was nothing to treat them, so why go through all of that invasive procedure in order to find that out. So, we've heard several times that now that there is a treatment, they will be performing those right heart caths to see the degree of pulmonary hypertension, because they do have - they presumably will have an approved treatment relatively soon. So, yes, the identification of this population will definitely be more robust since we have something to give them. And again, also with what Martine said about the publication, so we do expect to publish these results in a major medical journal very shortly, prior to the PDUFA date of April. So that's definitely will - what we've also heard from the docs is that once we have that published that gives them even additional confidence in looking for this - for pulmonary hypertension in this patient population. And yes, we'll likely - they have said they will either prescribe or certainly be waiting for the approval if they feel comfortable. So I hope that answers the question.

Thanks, Dr. Peterson. That was great answer. And Mike, would you like to add some color on that that we're getting from all of our medical sales teams?

Sure. I agree with everything Leigh said. I think the - there is a lot of pent-up demand. There is also, I think, an - there has been, I think, an under-diagnosis of PH-ILD for the reasons that Dr. Peterson stated, which is you have these physicians that may suspect pulmonary hypertension, that suspicion typically happens as the - obviously, as the symptoms become more severe and present themselves. But because there is no treatment out there, the doctors are putting the patients through very invasive procedure of the right heart cath. All of our interactions with physicians, be it advisory boards, healthcare - scientific presentations at healthcare conferences, interactions with our medical team suggest that now that there is a treatment available, these physicians suspect that significant percentages of their patient population have pulmonary hypertension and well start to really screen and diagnose those patients appropriately. So - and that's really a large part of what we're doing right now to prepare for the launch in April as we've got our medical teams out, educate - certainly the key opinion leaders in the field understand the disease and understand the pulmonary hypertension piece related to interstitial lung disease. But as you get into some of the community physicians, we're spending a lot of time educating them on PH-ILD, how do you screen for these patients, how do - What are the things that may lead you to suspect, okay, this patient has pulmonary hypertension and then how do you go about arranging for right heart cath to confirm the diagnosis?

Great. Thanks, Mike. Operator, there's time for one bonus question here. Do you have another person lined up?

I do. Our next question comes from the line of Liana Moussatos with Wedbush Securities. Please go ahead. Your line is open.

That's excellent. Okay. Hi, Liana. Good morning.

Good morning. Thank you. Since COVID continues to be a problem and infection rates are going up and it's affecting our industry, clinical trials and commercialization, can you just give us a brief overview of what has to happen and when do you think the timing of this could be resolved between now and maybe end of '21?

Yes. It's like one of these kind of 30,000 foot questions that cover everything. In our Company, Liana, we have a risk management group that reports in to our Chief Financial Officer, James Edgemond. And this risk management group ultimately has group meetings that involve as many as like 50, 60, 70 people at United Therapeutics and every week they issue a report regarding COVID-19 and how it affects all the different parts of the Company. So, James, maybe if you could, provide some color for, Liana's question in terms of the overall impact of COVID-19 on United Therapeutics and when we see - based on inputs we're getting from our risk management group, when we see some of those effects perhaps ameliorating.

Yes. Thank you, Martine. And Liana, good to hear your voice. So as Martine mentioned, internally way back to the beginning part of this year we organized a leadership team to make sure that across the organization we were communicating effectively and coordinating with each other with respect to risk management and the impacts around COVID-19 on all areas of the business, from human capital to manufacturing, to clinical trials, product development through specialty pharmaceutical distributors. And it's been a really effective tool for us to make sure that across the organization everybody is in tune to what impacts there are to the organization as a whole. And I think broadly speaking, everybody has stepped up, all the Unitarians, as we call ourselves internally, has stepped up to make sure that we have understood the various risks and the impacts not only internally, but to those of our patients and those importantly to the patients and individuals in the clinical trials, both that were enrolled, that continued to be enrolled, as well as those that were back to enter clinical trials that we may have shut down as we talked about and we've begun to reopen some of those based upon the opportunities and really the hotspots around the country or around the globe. So broadly speaking, I think, Martine is right, it's been a coordinated effort to really understand and prepare and execute to make sure as an organization we can continue again for the Unitarians or patients at large. From a standpoint of when we think things will get better, we hope for the best as everybody does in the near-term. But we're going to need to stay vigilant around all aspects of the organization to make sure that we can continue to perform as an organization. And so, I think, as things unfold and we all continue to get better educated and educated on the multiple aspects, we'll certainly continue to communicate in our public filings and on conference calls in various questions, but it's something that we'll continue to take seriously as everybody is doing to make sure the business at whole and at large continue to move forward. So, back to you, Martine.

Thanks so much, James. To wrap up, everybody on the earnings call, we've had a great quarter. I think this quarter and perhaps more generally this half of the year, will be looked at in retrospect as a key pivot point for United Therapeutics. This will be the time when we pivoted from being solely a pulmonary hypertension company to becoming both a pulmonary hypertension company, as well as a pulmonary fibrosis and other forms of interstitial lung disease company. You can see this pivot in us moving into a group of pulmonary hypertension patients WHO Group 3, as to whom pulmonary hypertension is not their primary problem, their primary problem is interstitial lung disease. It's of course gravely worsened by the pulmonary hypertension, but the source of their pulmonary hypertension is ultimately interstitial lung disease. So, for the first time, we're moving into a group of patients for whom pulmonary hypertension is not their number one problem, if you will. And secondly, for the first time, we're moving into a large Phase 3 study in which patients do not have pulmonary hypertension at all, they simply have this form of interstitial lung disease, pulmonary fibrosis. If you take a look at a diagram of all of the species and sub-species of interstitial lung disease, you'll see it is a very large and diverse population. And we were really pleased to see in the outcome of our INCREASE study that we actually were able to modify the disease. In other words, not only slow the rate of decline and forced vital capacity, but actually improve forced vital capacity in the patients with pulmonary fibrosis, as well as a couple of other subsets of interstitial lung disease. So, it's a very exciting time here at United Therapeutics. You always hope that you can be strong at your home base and then expand from that. And with this adjacency of interstitial lung disease and pulmonary hypertension, we have a very logical and solid basis from which to expand our footprint in providing more and better healthcare to ever larger numbers of people. Thank you so much for your time and attention this morning. Look forward to seeing you at upcoming healthcare conferences. Operator, you can wrap up.

Thank you for participating in today's United Therapeutics Corporation conference call. A rebroadcast will be available for replay for one week by dialing 1-800-585-8367 with the international callers dialing 1-416-621-4642 and using access code 8782637.