Martine Rothblatt - Founder, Chairman & CEO Andrew Fisher - Deputy General Counsel and Chief Strategy Officer.

Alethia Young - Crédit Suisse AG Evan Seigerman - Barclays PLC Santhosh Palani - Cowen and Company Jessica Fye - JPMorgan Chase & Co. Hartaj Singh - Oppenheimer & Co..

Good morning. My name is Andrew, and I will be your conference operator today. At this time, I would like to welcome everyone to the United Therapeutics Corporation 2017 Third Quarter Financial Results. [Operator Instructions].

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 Form 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 the U.S. Generally Accepted Accounting Principles.

Reconciliations 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 approval labeling or to provide all available information regarding the products, their risks or related clinical trial results.

Anyone seeking information regarding 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 with your conference..

Good morning, everyone, and thank you for taking the time to join United Therapeutics' Third Quarter Earnings Call. I'd like to take the opening few minutes to provide some updates of 6 key areas of our focus, patients, physicians, payers, products, pipeline and profit. Let's start with patients.

Earlier this week, we announced the FDA's approval of our new inhalation device for Tyvaso. This is a very cool device and it addresses several reasons for patient's discontinuation on Tyvaso. Thus, we think this greater ease of use will result in increased revenue from Tyvaso. Also regarding patients.

This past quarter, I gave a speech in Boston at a McKinsey & Company-sponsored event for recruitment of MBAs and finance people.

And I was blown away afterwards when a handsome, healthy-looking third-year MBA in attendance came up to me and said, "This medicine makes my life possible." I looked at him curiously and he then reached into his pocket and pulled out an Rx bottle that said Orenitram on it. I talked with him a bit, learned who his physicians were, whom I knew.

And I'm going to tell you that this is happening more and more, upfront and early prescription of Orenitram to make patients' lives better. In the PAH space, it takes time for things to catch on, and I can tell you Orenitram is definitely catching on. So let's move on to physicians.

There is tremendous excitement over the coming launch of the RemUnity and RemoSynch system. Physicians naturally want to make things as easy for patients on Remodulin as possible, and these products will definitely do that for subcu and IV patients successively.

I feel quite confident that physicians will insist with payers on keeping the Remodulin patients on branded Remodulins both because of the fragility of the patients and for new patients because of the major advantages of a pre-filled pump in the case of RemUnity and in the importance of skin-puncturing catheter in the case of RemoSynch.

Excitement is so high that physicians had no trouble at all rapidly and fully enrolling our recent safety trial of RemUnity. So our third P is for payers, and we are very attentive to our business relationship with them.

I've taken note that Orenitram's competitor, Uptravi, has not increased very much in number of patients, having just barely twice as many patients as we do, notwithstanding less frequent dosing and very aggressive promotion.

I think payer concerns is a big factor here as Uptravi is tens of thousands of dollars more expensive than Orenitram but has never shown itself to be any more effective.

We also expect to save the payers a lot of money with the upcoming launch of RemUnity because its pump system will be a small fraction of the cost of the pump system that is used today for parenteral treatments of pulmonary hypertension such as Flolan, Veletri and generics. Now the next P is for products.

And here, I'd like to draw attention to the fact that all of our products are part of the United Therapeutics' continuum of care system.

This enables patients to move seamlessly from Orenitram to Tyvaso to Remodulin, always expecting the treprostinil pharmacodynamic profile, and always enabling nursing staff to work easily with team members who have knowledge of all 3 products.

I do believe this continuum of care system is in part responsible for the outstanding 2 quarters in a row of record 20%-plus Orenitram product growth. Indeed, the continuum of care story will get even stronger when the FREEDOM EV combination therapy trial unblinds in 2018. Well, perhaps the most exciting P to speak about today is pipeline.

We have 2 new chemical entities queued up, Tysuberaprost, a uniquely potent oral prostacyclin analog now completing its Phase III trial called BEAT in combination with Tyvaso; and RemoPro, a novel resynthesis of treprostinil into a form of treprostinil without the subcutaneous pain profile of Remodulin.

We expect both of these MPEs to launch soon after the next-step launches of RemUnity, RemoSynch and Orenitram combination therapy post FREEDOM EV is unblinded.

In addition, we expect new product launches of Orenitram into the 50,000-patient heart failure with preserved ejection fraction market, following success of its current SOUTHPAW Phase III trial; and another launch of Tyvaso into the 30,0000-patient interstitial lung disease market following success of its current INCREASE Phase III trial.

Also noteworthy is our Phase III study of dinutuximab in small cell lung cancer, a study called DISTINCT; and our Phase III study of engineered stem cells in resistant pulmonary hypertension Phase III study called SAPPHIRE.

This SAPPHIRE study uses an engineered cell product that is made from the patient's own cells which are engineered at a central location to include certain genes. And then are sent back to the patient at their own hospital. Once they're infused back in the patient's body, these genes continue to act like a little medicine factory.

The engineered cells appear to continue doing their magic for quite some time after they're delivered, giving hope to a once-a-quarter or maybe even once-a-year therapy for pulmonary hypertension.

So all told, the pipeline here at UT in the late stage has 7 late-stage new product candidates based upon 5 Phase III studies addressing both virgin markets for prostacyclin therapy and markets characterized by a high level of unmet medical need.

Now let me talk a bit about our earlier stage pipeline because it is truly exciting as well, with MDI, that's metered dose inhaler, versions of our denisil and treprostinil being developed for p.r.n. use in pulmonary hypertension. With this p.r.n.

product, every pulmonary hypertension patient, no matter what therapy they may be on, could be able to benefit from a prescription of one of our product to help them get through particularly arduous parts of their day. Also exciting in our early stage pipeline are our organ-manufacturing activities.

We've recently signed organ manufacturing research agreement with the Transplantation Department for the University of Maryland for our xeno heart, the University of Alabama for our xeno kidney and Columbia-Presbyterian for our xeno lung.

We've also actually manufactured about a dozen successfully transplanted lungs this year from declined brain-dead donor allografts using our ex-vivo lung perfusion procedure.

And perhaps most amazing of all though, is that as of now, we have ramped up our lung manufacturing via decell and recell lines to reliably produce about 300 lung samples per year. It's an assembly line, so we're looking forward to extend into recellularization, achieving normal gas exchange and finally shipment to transplantation.

The sixth P in our lineup is for profit. And here, too, we have had an outstanding quarter. Net income increased 70% to $276 million this quarter compared to $162 million last year.

Even making adjustments for noncash items, profitability remained strong because we leverage our R&D and S&M spending across multiple different products and pipeline development. For example, while some other companies might need a separate sales force for each product, we can leverage one sales force to promote 3 products as a continuum of care.

While some companies might even need separate clinical development teams for each Phase III trial, we are able to leverage one innovative clinical development and regulatory approval organization to support multiple pivotal studies in parallel.

It is this high efficiency of UT, as reflected also in our metric of $2 million in revenue per employee, that enables us to maintain strong profitability year-to-year. So thank you for your attention during these introductory remarks.

I would now open the line for questions to our Chief Financial Officer, James Edgemond; or regarding IP or litigation matters, to our Deputy General Counsel, Andy Fisher; or otherwise, for myself. Operator, you can open the lines..

[Operator Instructions]. Our first question comes from the line of Alethia Young with Credit Suisse..

Just a little bit more detail, can we get maybe, on Orenitram. And like, I know you're saying that you've seen kind of nice revenue growth over the past couple of quarters.

So maybe just explain, maybe is it kind of a slow and steady that's kind of driving prescriptions up? I mean a kind of word-of-mouth? Or is there some kind of turning point that maybe we missed that we should be thinking about?.

Yes, thanks, Alethia, for the question. It's -- I think with Orenitram, it's slow and steady. I mentioned in my introductory remarks that things take a while to get started in PAH. So once they do, you really see the growth.

I recently asked my sales and marketing team to give me data on the performance from launch of all of the different oral medications for pulmonary hypertension. And this goes back quite a few years now.

And I was actually fairly astounded that things like Letairis, which is the most-prescribed ECRA in pulmonary hypertension today with, I think, like over 10,000 patients on it, it began very modestly. A few hundred the first year. 1,000, 3,000. I mean, almost linearly.

Now admittedly, it's been out getting close to 10 years, but it's now pumping over $1 billion a year in revenue. So things definitely do take a while.

I don't think the sharp eyes at Crédit Suisse missed anything, but I would point that our launch was complicated by the fact that right after we launched as a monotherapy with an exercise label, Uptravi was launched with a combination therapy and morbidity and mortality label.

So I have been giving people a heads up for a couple of years that, that was definitely going to slow the initial take-up of Orenitram. But I'm meeting more and more people who their first oral prostacyclin analog is Orenitram, and they are looking great. So -- and it's also worthy of noting that Orenitram is the only true prostacyclin analog.

So some physicians may have cycled their patients through Uptravi, but pretty quickly, are getting them over to Orenitram. So I do believe that you are going to see continued growth in Orenitram year-after-year.

And certainly, an adjuvant-type of data point to look to is in just next year, 2018, when the unblind the FREEDOM EV study that would then provide Orenitram with both a morbidity and mortality endpoint as well as good data in combination therapy. So that's a major exciting news flow event for 2018..

Our next question comes on the line of Geoff Meacham with Barclays..

This is Evan Seigerman on for Geoff. With the RemoSynch and RemUnity pump, what is the update with the FDA for RemoSynch? And how do you view both of these pumps in the marketplace? They kind of do similar things, potentially..

Yes, thanks, Evan, for those good questions about the Unity and the RemoSynch pump. So to clear up any confusion right up front, I actually think that they do 2 very different things, that the Unity pump is for subcutaneous drug delivery, whereas the RemoSynch pump is for intravenous drug delivery.

And the pulmonary hypertension Remodulin market is pretty much split right down the middle between subcu and IV and has been for a number of years. So I don't think that there's going to be any cross-cannibalization, if that's even a word, between those 2 pumps because one will be for the IV market and the other one will be for the subcu market.

The FDA is doing a great and diligent job of processing all of the information. It's just like if you're running yourself in a race, you're going to run faster than if you're like in one of those races where 2 people have to tie their legs together -- and like, a three-legged race across the finish line.

So because RemoSynch is like a three-leg race here between the FDA, United Therapeutics and Medtronics, things take a little bit slower. But the end result is going to be awesome for the patients.

I -- those of us who are healthy, we can't even imagine what it's like to walk around every day of your life with a catheter that punctures through your skin. And then to be able to say, "Okay, well, you don't have to have the catheter puncturing through your skin.

You don't have to be freaked out about getting septicemia through infection of the wound. Instead, your drug will be delivered automatically like clockwork without even thinking about it, 24/7, being implantable device. By the way, this implantable device has been in hundreds of thousands of patients, has a tremendous, excellent safety record.

And depending on the dose of Remodulin you're on, once every month, 2 months or possibly even 3 months, a specialist nurse will come to your house and refill your RemoSynch pump for you." So it's a major, major breakthrough, Evan, and doctors are super excited about it. I think the FDA is going to justify it.

They feel very, very proud of themselves and of our whole industry when that first-ever continuous drug delivery device in the cardiovascular space is approved..

And our next question comes from the line of Chris Shibutani with Cowen & Company..

This is Santhosh calling for Chris. I'm just wondering.

Can you comment on how clinical study experience has been so far with RemUnity, RemoPro and dinutiximab in small cell lung cancer? It will be helpful for us to know, like, how many patients have you actually treated so far with these 3 agents? And when do you expect to disclose clinical data?.

Yes, thanks for the question, Santhosh, it's a very good one. And it's a question that led us to move these therapies, both of RemUnity and on dinutuximab, forward.

The reason that we moved them forward is that the active pharmaceutical ingredient in each of these drugs and in each of their clinical studies has already had a lot of exposure in patient.

For example with regard to RemUnity, the active pharmaceutical ingredient is exactly the same API as is in Remodulin, which has been used by thousands and thousands of patients. So the amount of exposure that is needed in this clinical trial is therefore quite modest.

And I mentioned in my introductory remarks that the trial for it was very rapidly and very -- I mean, completely enrolled by the physicians in under a year. So it's just went like wildfire. With regard to dinutuximab, it's a similar and a little bit of a different situations.

The API or active pharmaceutical agreement -- ingredient, dinutiximab, is the same exact one in our approved therapy for neuroblastoma called Unituxin.

And there was so much evidence that API would be effective for other TD2-characterized cancers that the experts at Practitioner in Oncology recommended that we take it into small cell lung cancer patients who had unfortunately had a progressing of their cancer notwithstanding some of the new therapies and older therapies that have been approved.

So that is now enrolling at, I don't know the exact number, Santhosh, but it's about -- it's over 100 hospitals throughout the world. And I think one thing which is greatly accelerating its enrollment is that this is an API that there is quite a bit of experience for us. It's a chimeric antibody, so it can have a side effect profile.

Has to be -- patients have to be monitored very closely. But we feel confident that this study will be able to be enrolled. And as shown on our website, we are planning to recount this therapy in our near-term set of product launches..

Our next question comes in the line of Jessica Fye with JPMorgan..

Hoping you could help us understand the way to think about the Adcirca generic erosion. And also any, just, more refined timing on FREEDOM EV, more specific than just 2018..

Yes, Jessica. I'm going to turn it over to Andy Fisher, our Deputy General Counsel, to talk about the Adcirca situation because it involves a patent expiration, maybe a patent extension. It's like an IP situation that he is best able to address.

Andy?.

Sure, thanks, Martine. Hi, Jessica. So as we've disclosed in our public filings for many quarters running, the key patent on Adcirca is set to expire next month.

There has been some uncertainty because of the existence of a couple of later-expiring patents listed in the Orange Book for Adcirca which were challenged several years ago in an IPR proceeding, and that proceeding remains pending to the extent that there's actually still an appeal pending.

But the patents, I believe, were invalidated in the initial determination by the Patent Trial and Appeal Board. So we've always guided The Street toward focusing on the expiration of this patent next month.

There are various circumstances, including the pendency of that IPR, as well as some other regulatory issues that could cause generic entry for Adcirca to slip by some number of months. But those remain unclear and uncertain. So we remain sort of focused on that November timeline.

If it ends up being longer because of some of these other things that remain pending, great.

But otherwise, we've also retooled our agreement with Lily, which we've previously disclosed, to account for sort of the post-generic-entry existence of our relationship and modify the license agreement such that we'll continue to take point on selling Adcirca even post generic entry. So hopefully, that answers your question..

Our next question comes from the line of Hartaj Singh with Oppenheimer..

Just a quick question on the organ transplantation business. We had not been paying a lot of attention to it, Martine, but it seems to be kind of coming up a little bit more in discussions with clients. So if you could give any sort of insight as to where you are with some of the technical hurdles there.

And where you could see yourself being a year or 2 years from now?.

Sure. Thanks for the question, Hartaj. It is a -- the purpose of the organ transplantation business is that there's just, like, a yawning, huge unmet medical need between the number of organ transplants that can be done through the United Network for Organ Sharing system today and the number of patients who die of end-stage organ disease.

And for example, just in our kind of orphan area of pulmonary hypertension, only about 1 patient's life is going to be able to be saved with a lung transplant for every 10 people who end up dying with pulmonary hypertension despite the fact that an organ transplant is a complete cure for hypertension, it has never recurred in any patient post-transplant.

But there's so many other people who need lung transplants, the people with cystic fibrosis, people with pulmonary fibrosis, not to mention the gigantic number of people with various forms of late-stage COPD, obstructive lung disease, emphysema, what have you.

So there's just a huge need there, and I think it would be belaboring the obvious to talk about the need in the other major organs, heart, kidney, lungs and liver.

So we've been able to develop a suite of technology which we're moving the entire suite of them forward at the same time from the nearest safe technology that I mentioned on the call, where we seed organs that have been donated but declined by all transplant centers throughout the country.

And so these are essentially dead organs, just about being a sense of biowaste. We cool them down, we fly them to Silver Spring, Maryland. And we put them through a procedure that aims to bring them back to life. Remarkably, about half the time, we do bring them back to life.

And we've established a nationwide high-speed real-time data and video network that then allows transplanting, wherever they are in the country, to look at the revived organ, to look at realtime bronchoscopic imagery down the lungs and decide if they want it or not. And then when they want it, we cool the lung back down, we fly it.

And in every single one of those instances, the patient has left the hospital healthy with this lung that otherwise would have been completely thrown away. Just to give you a sense of the numbers, Hartaj, 80% of the donated lungs from brain-dead donors are in fact declined and thrown away.

So this is an opportunity to double, maybe triple, the number of lung transplants done in the U.S. So we're doing that right now, week in, week out, and already saved over a dozen people this year. In parallel with that, we've got efforts in the field of xeno transplantation, I referred to in the call there.

Our partnership with Synthetic Genomics has allowed us to modify the pig genome so that its organs will not be acutely rejected, and hopefully, will have any rejection be well-managed by conventional immunosuppression. So that project is going on very well with regard to kidney, hearts and lungs.

And in addition to that, I also mentioned in my introductory remarks, we have a ability to actually manufacture human-cellularized lungs using what's called a decell/recell procedure. And there, we set up a whole assembly line.

I mentioned the tremendous success that we have now in automating the process of decellularizing and recellularizing these lungs.

So hopefully, between our allogenic decell/recell; our xenogeneic kidney, heart and lungs; and our recovered donated organs, we'll be able to make a significant increase in the number of lives that could be saved from end-stage organ disease. Hartaj, thank you so much for your question. And operator, you can wrap up the call..

Thank you for your participated in today's United Therapeutics Corporation Conference Call. A rebroadcast will be available for replay for 1 week by dialing 1 855-859-2056 with international callers dialing 1 404-537-3406, and using the access code of 97086326..