United Therapeutics Corporation
Q1 2017 Earnings Call Transcript

Published: 26 Apr 2017

[Operator:]

Good morning. My name is Chanel, and I will be your conference operator today. At this time, I would like to welcome everyone to the United Therapeutics Corporation 2017 First Quarter Financial Results. All lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question-and-answer session. Remarks today concerning United Therapeutics will include forward-looking statements representing the company's expectations or beliefs regarding future events. The company cautions that these statements involve risks and uncertainties that may cause actual results to differ materially. Please see the company's latest SEC filings, including Forms 10-K and 10-Q for additional information on these risks and uncertainties. The company assumes 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 accepted accounting principles. Reconciliation of non-GAAP financial measures to the most directly comparable U.S. GAAP financial measures can be found in our earnings release available on our website at www.unither.com. Finally, please note that today's remarks may include reporting on the progress and results of clinical trials or other developments with respect to the company's products. These remarks are intended solely to educate investors about the company and are not intended to promote the company's products, to suggest that they are safe and effective for any use other than what is consistent with their FDA approved labeling, or to provide all available information regarding the products, their risks, or related clinical trial results. Anyone seeking information regarding the use of one of the company's products should consult the full prescribing information for the product, available on the company's website at www.unither.com. Thank you. Dr. Rothblatt, you may begin your conference.

[Martine A. Rothblatt:]

Thank you, operator. Good morning, everybody. Welcome to United Therapeutics' first quarter 2017 earnings call. My name is Martine Rothblatt. I'm the Chairman and CEO, and I'm joined on the call by our Chief Financial Officer James Edgemond and our Chief Strategy Officer Andrew Fisher, who is also known as our IP guru. I've got a few introductory remarks, and then we'll open up the lines for questions that can be directed to Andy, James or myself. This past quarter, we saw a continuation of the transient, temporary phenomenon of too few patients coming onto our therapies to offset the loss of our patients to either transplantation or death. This phenomenon is the result of patients remaining longer on AMBITION therapy than they have in the past, plus some diversion to Uptravi therapy, while duration on our true prostacyclin therapies, which are generally used after an initial challenge with either oral PDE-5 or oral ETRA therapies, has remained largely unchanged. I say this is a transient and temporary phenomena because the numbers of patients who need to be on our therapy are building up on the AMBITION protocol like an ever-larger backlog, and when they do break through to needing true prostacyclin therapy, which will be in the coming quarters, we expect to see a significant jump in Orenitram, Tyvaso and Remodulin revenues. Indeed, in the past quarter, the first quarter of this year, we saw for the first time since Uptravi's launch our decline of patients have stopped, stabilized and has begun growing again. So that's a huge good sign of what we can expect to see in the quarters to come. Now, whether these patients who are now moving onto United Therapeutics' therapies go onto Orenitram first and then Tyvaso, or Tyvaso first and then Remodulin, or straight to Remodulin, this is all simply a consequence of their doctor's judgment as to how much prostacyclin they require and in what form. It is true that because Orenitram and Remodulin are priced in a dosage fashion, a dosage-sensitive fashion, when they first come on to Orenitram or Remodulin, the average revenues for that patient is going to be a lot lower than when they first go onto Tyvaso, which has a steady dosing prescribed in its label. But that kind of stuff works itself out within two or three quarters. So to some extent I would say the financial results here seem, in any given quarter, are really an echo of patient trends six months in the past, and that's an important thing to keep in mind. Meanwhile, we at UT are concentrating on our core long-term interest in curing end-stage lung diseases and this occupies a much greater percent of our attention than the fluctuations in quarterly results because we can see from the trends of patients needing to move off of front-line therapies onto our therapies, the quarterly results are going to get better and better in the coming quarters as more and more patients move onto our therapies. Meanwhile, we're quite confident that these long-term interests will provide the greatest value yet to shareholders, and indeed, within our internal business plan, our net present value assessment is in excess of $15 billion. And this assessment comes from three segments of our pipeline in addition to our current revenues. We label these segments our nearer-term pipeline, medium-term pipeline, and longer-term pipeline. So let me say a few words about each of these three pipelines. The nearer-term pipeline covers product launches for the four-year period, 2018 to 2021. And while there are a number of items in this pipeline, in the interest of time, I'll focus on just the most significant launches in this period, which are a replacement product for all parenteral prostacyclin therapies, called RemoPro, a morbidity-mortality label for Orenitram, and a novel, brand-new oral prostacyclin – oral prostacyclin called Esuberaprost. These products are expected to double our current level of sales because, first, we believe they will eliminate the two factors that have limited Remodulin acceptance, namely, a kind of Hobson's choice between subcutaneous pain or intravenous delivery. RemoPro will blow that choice out of the water. Second, we believe they will eliminate the label efficacy distinction between Uptravi and Orenitram. We are shooting for a morbidity-mortality label for Orenitram. And third, we believe they result in a doubling of duration of time on Tyvaso therapy because of its coupling with Esuberaprost, which has a complementary pharmacokinetic as well as pharmacodynamic profile. So those are the highlights of the short-term pipeline or nearer-term pipeline. Let's now talk about the medium-term pipeline. This covers product launches for the four-year period, 2022 to 2025. Again, there are numerous products in this pipeline, but I'll just, in the interest time, focus on the most significant. The most significant ones, I think, are extending Remodulin into idiopathic pulmonary fibrosis via our new EPITOPE [08

[Operator:]

Thank you. And our first question comes from the line of Geoff Meacham of Barclays. Your line is now open.

[Evan Seigerman:]

Hi, all. This is Evan, on for Geoff. Thanks for taking the question. Just one about the interim look on the FREEDOM-EV study. What do you view as a reasonable bar for commercial viability and differentiation versus the competition? And does it matter if a patient is on one or more therapies? So basically, what's the role of combination therapy going forward?

[Martine A. Rothblatt:]

Thanks, Evan. Two insightful questions, indeed. So first of all, we've got an incredible biostatistics team here at United Therapeutics, and we assessed carefully the question of the interim work on the FREEDOM-EV study. And once we came to the conclusion that we were more likely than not to succeed with that interim look – of course there's no guarantee or assurance – but that we were more likely to succeed or not, we always ask ourselves at this company what's the right thing to do? And the right thing to do is, of course, avail patients of a therapy that could reduce their morbidity and mortality as soon as possible. So since the statistics dictated a greater than 50% likelihood of success, we chose to go for the interim look and that will occur later this year. The bar, I believe, is to show a reduction in morbidity and mortality which would then put it on par from a label sense in terms of efficacy, for sure, with Uptravi. Now, with regard to the second question, I think everybody would love for there to be a silver bullet for pulmonary hypertension, and Lord knows we continue to search for that. It would take way more time than I would have this morning to go into some of the very interesting, early, early-stage pipeline activities that we're looking at about new molecular entities that are capable of addressing two of the five pathways of pulmonary hypertension that have been left unaddressed despite 20 years of drug development. There are these five pathways that have been well-known for a generation

[Evan Seigerman:]

Great. Thanks for taking the questions.

[Martine A. Rothblatt:]

Sure thing.

[Operator:]

Thank you. And our next question comes from the line of Martin Auster of UBS. Your line is now open.

[Mark Connolly:]

Hi. This is Mark Connolly on for Marty. Thanks for taking my call. So following up with FREEDOM-EV, we noticed in the queue that some approval timelines were adjusted from previous guidance. And can you comment specifically on FREEDOM-EV, which was pushed to 2020 from 2019 and what that might mean related to the interim analysis planned this summer?

[Martine A. Rothblatt:]

Yeah, Mark, the guidance we'd give is that we have a group of projects that we call in the nearer-term guideline, in the nearer-term pipeline. And the only guidance that we're providing are that these products which are in the nearer-term pipeline will be launched in the four-year period 2018, 2019, 2020, and 2021. I don't have the whole list of them, but certainly FREEDOM-EV, which we go by the product name OreniPlus, because it's Orenitram plus background therapy. That's the study design, okay, that's something that will be launched in the 2018 to 2021 pipeline. And it's just too arbitrary to be able to parse the particular launch timeframe within a quarter or even within the break of an annual year. So with regard to the launch of OreniPlus, that would be nearer-term, sometime between 2018 and 2021.

[Mark Connolly:]

Okay, great. Thanks for taking my question.

[Martine A. Rothblatt:]

Sure thing, Mark.

[Operator:]

Thank you. And our next question comes from the line of Terence Flynn of Goldman Sachs. Your line is now open.

[Terence Flynn:]

Hi. Thanks for taking the question. Martine, just based on your pipeline review during your prepared remarks, is it fair to assume that you're content with your pipeline as it currently stands? Or should we expect more collaborations along the lines of the bio-printing agreement? Or would you even perhaps consider any mid-stage clinical assets outside of PAH? Thanks.

[Martine A. Rothblatt:]

Yes, Terence, we're not content. We at United Therapeutics looked at ourselves as like a restless engine, and we are always on the prowl for new opportunities. In fact, we find a lot of companies want to work with us and even would like to be acquired by us because the culture and the atmosphere at UT is so awesome. I know you're like a big statistics guy, like one stat figure is that the voluntary turnover at United Therapeutics is way less than it is at other biotech companies and pharma companies in our peer group. So people definitely want to be acquired, and we're interested in acquiring new technologies, new therapies. We're interested in both early-stage assets, such as those that are active in the thromboxane and serotonin pathways. We're interested in more advanced methods of current pathways. During the past quarter, we closed a deal with a terrific company named Respira, who was referred to us by one of the leading pulmonary hypertension physicians, Dr. Adaani Frost, down in Houston, and we're really excited about this IBAR [26

[Operator:]

Thank you, and our next question comes from Liana Moussatos of Wedbush Technology. Your line is now open.

[Liana Moussatos:]

Thank you for taking my question. What are the next steps to get the 3D print transplant organs to the market?

[Martine A. Rothblatt:]

Yeah, Liana, thank you for that question. It's a step-by-step process and what I like about it is that it's now an engineering process. It isn't something that any longer requires fundamental breakthroughs in physics, something that doesn't require new inventions. It requires a step-by-step improvement of the engineering of our current technologies. So for example, today we have literally an assembly line of porcine lung decellularized scaffolds. And when I say an assembly line, we are decellularizing over 500 scaffolds a year, so that would be, once cellularized, that would increase the supply of lung transplants 25% in the entire country right now. We also have a parallel assembly line in another lab where we recellularize these scaffolds with allogenic cells, that is with human cells. So when the final organ is completed, it looks to the patient recipient just like a human lung, even though at its collagen skeleton, if you will, these are proteins that were originated in the – by instructions from a pig's DNA. Over time they will get completely replaced by human DNA-directed collagen. It will be a completely human lung. So these same technologies of scaffold management and recellularization of these scaffolds are exactly the same technologies that will apply to the 3D printed scaffold. Of course the great benefit of the 3D printed scaffold is that we can make these in any size and shape that is necessary for different patients' chest size. And another great benefit is that we can scale up the number of scaffolds far greater than we can do with the porcine decellularization process. And finally, it looks like it will be much less expensive as well. So the process is to begin printing the scaffolds with our partners at 3D Systems, basically branch by branch. There are, roughly speaking, 15 to 20 branches of vessels and airways in the human lung. So we print these branch by branch. They go down to finer and finer resolutions. As we're printing them in parallel, we are cellularizing them. And when we cellularize the reprinted branches, just as when we recellularize the decellularized scaffolds, we test for gas exchange. And in fact, it's a magical thing, Liana, to watch. We hook up the scaffolds to, basically, a kind of an ECMO machine, and the scaffolds themselves breathe in the cells that they're being recellularized with until finally, in this little bioreactor, the lungs are able to breathe on their own. And it's virtually an embryonic type of process. So it is the – the step is going through the xeno process with the de-cell/re-cell while the 3D folks are building up their 3D printing and then mapping over our recellularization technology from the de-cell/re-cell onto the 3D printing. Finally, on yet a third parallel track, there's some remarkable work being done with successfully teasing out precise cell lines from IPF cells. And the types of cell lines that we're most interested in differ by organ. For the lung there are different types of epithelial cells and a general family of endothelial cells to the predominant cell lines. We at UT have mastered the technology to expand these cells now into the tens of billions of cells and which is, by the way, the amount that you need to recellularize an organ. So the IPF cell development activity at UT will ultimately be mapped onto the 3D Systems-printed scaffold so that rather than having a scaffold recellularized with allogenic cells, as we're doing today and as we would do as an interim step, the 3D scaffold will be cellularized by the intended patient's own cells. And that means, of course, that patient will not be obligated to take any immunosuppressants, which is the major factor keeping most people off of transplant lists. It will be a huge benefit to the patients and to the health care system as well. Now, there will always be people who cannot wait to have a 3D-printed lung, cannot wait to have their IPF cells expanded and regenerated. We have a lot of interest from, for example, people who are in first-responder type of situations, military type of situations, acute lung injury type of situations, where what you need is a very rapid transplant. And in those situations, the work in our xenotransplantation group is a perfect solution. We plan to move organ transplantation from a paradigm of scarcity where it is right now with a viciously managed transplant list that keeps 99% of the people in need of an organ off the list, to a paradigm of abundance in which organs can be constantly produced and available for people who need them as a result of car accidents or fire or other trauma, as well as having those custom designed for patients that can wait two or three months for their transplant. So I believe there is enough demand, Liana, for all of these different organ transplantation technologies we're developing. And I really look forward in the 2020s to our biological products being a harbinger of a paradigm of abundance in the field of transplantation science.

[Liana Moussatos:]

Thank you.

[Operator:]

Thank you. And our next question comes from the line of Alethia Young of Credit Suisse. Your line is now open. Alethia Young - Credit Suisse Securities (USA) LLC (Broker) Hey, thanks for taking my question. Just one on the timing of how you're thinking about when revenues might return back to some of the advanced therapies. I know if you look at AMBITION it seems like kind of the rate in which people had events was very slow in the combination arm, so I just wondered if you could talk about maybe what you're seeing in the real world and how that applies to your business maybe over the rest of this year. Thanks.

[Martine A. Rothblatt:]

Sure, I'm glad to. And this will be the last question, operator. So it's not a kind of a thing that you can be super precise about because we're in the realm of human affairs and there's thousands of patients on these AMBITION therapies. But what can be said, largely because we're dealing with relatively large numbers of people, is that about 15%, say one out of every seven patients or so, who are on combination therapies experience a morbidity or mortality event each year. Now, that does not ipso facto automatically mean that the patient is going to be immediately moved onto an advanced therapy. I think in the best case, at the largest treatment hospitals, that would ordinarily be what one would do, because it definitely is a bad sign to be suffering these morbidity events, which are things like requiring hospitalization, dropping in New York Heart Association functional class, significant decrement in six-minute walk distance, or significant deterioration in cardio-hemodynamics. But there are thousands of patients with PAH. They're seen all over the country. Many people live in rural areas. Not everybody can, like, get switched to a new therapy right away. So in real life, sometimes the rate of patients moving onto the more advanced therapies may be a little bit slower. On the other hand, in clinical trials, patients are monitored very intensively and are basically under a kind of, I would say, a subliminal encouragement to remain on therapy, whereas, what's usually the case in the real world is patients do worse on therapies than they do in clinical trials. So that's a factor kind of tending to move patients more to our therapies more rapidly. I have a chart that was prepared for me. It was very interesting. It showed that from the launch of Uptravi the number of patients on United Therapeutics declined gradually, a small slope, but declining until the fourth quarter of last year when it flattened. And then the first quarter this year, it's an upward slope. So obviously, as a CEO of a company, I'm happy to see that more patients are clearly coming onto our therapies and that was had been a temporary decline in the history of our company since the launch of Uptravi has pretty clearly been reversed. Now, I've heard there was just a big International Society of Heart and Lung meeting in San Diego, and staff came back and told me that the reports from physicians are that Uptravi is not working as well as they thought. Well, as I mentioned, it's usually the case that in the real world, things don't perform as well as they do in a clinical trial. Some people were referring to it as prostacyclin-lite, like L-I-T-E. I mean, that could be just a – that could be a good and a bad thing because prostacyclin does have side effects. And perhaps it's good to have something with lighter side effects. But if you need prostacyclin therapy, it's maybe not the time to have a lite one. It's the one time to have the real one. And so I think these are the kind of factors that are underlying this bottoming out and then upturn in the number of patients on our therapies. Whether the revenues will reflect that within one quarter or two quarters lag, it's really, really hard to predict. Remodulin and Orenitram in particular, patients start at low doses because of this side effect profile I mentioned and ramp up gradually. So you're always going to see a several-month lag between patients and revenue, patient growth and revenue growth, uptick. For Tyvaso, there is less of a lag. There is some argument that the patients are basically holding on to the orals too long and then they need to go directly to Remodulin. And we're really agnostic. All of our sales reps know that their job is to educate physicians on Orenitram, Tyvaso, and Remodulin. Once patients are on their therapeutically ideal dose, we are really revenue-agnostic in terms of whether they're on Orenitram, Tyvaso, Remodulin. All three of those drugs were designed to be pretty much priced equivalent to each other because they all have the same active pharmaceutical ingredient. So the bottom line is that the prospects for UT are super good. There's never been a larger backlog of patients who require our therapies, and therefore the prospects for continued growth and significant revenue growth across all three of our therapies have never been better. Thank you very much. Operator, you can conclude the call now.

[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 1-855-859-2056, with international callers dialing 1-404-537-3406 and using access code 7268729.