United Therapeutics Corp (UTHR) 2012 Q2 法說會逐字稿

Good morning. My name is Sayed and I will be your conference operator today. At this time I would like to welcome everyone to United Therapeutics Corporation's second quarter conference call. All lines have been placed on mute to prevent any background noise. After the speakers' remarks there will be a question-and-answer session. (Operator Instructions)

Remarks today concerning United Therapeutics will include forward-looking statements which represent United Therapeutics' expectations or beliefs regarding future events based on current assumptions. United Therapeutics cautions that such statements involve risks and uncertainties that may cause actual results to differ materially from those in the forward-looking statements. Consequently, all such forward-looking statements are qualified by the cautionary language and risk factors set forth in United Therapeutics' periodic and other reports filed with the SEC. There can be no assurance that actual results, events, or developments referenced in such forward-looking statements will occur or be realized. United Therapeutics assumes no obligation to update these forward-looking statements to reflect actual results, changes and assumptions, or changes in the factors affecting such forward-looking statements.

At this time, I would like to hand the conference over to Dr. Martine Rothblatt. You may begin your conference.

Thank you very much, Operator. We here at United Therapeutics are glad to welcome everybody, as we move into the middle of summer, to our second quarter 2012 financial results.

I'm on the phone today with our Chief Financial Officer, John Ferrari and our President and Chief Operating Officer, Dr. Roger Jeffs. The three of us will be able to answer your questions in any of the normal areas of business operations. So I'm just going to give a brief introduction and then ask the operator to open up the lines for questions.

I'm pleased with our second quarter results, which reflect the continued strength of our core business. I'm also pleased that our Board authorized an additional $100 million share repurchase program to return yet further value to our shareholders. So that is the summary quotation for the press release, which we issued today.

The total revenues for the quarter were $225.6 million. Earnings per share were $1.37 per basic, $1.34 per diluted. And the operating metric which we think best reflects the real business operations of the Company, earnings before noncash charges, grew to $2.42 per basic share, $2.37 per diluted share. So, across the board, very positive financial results.

Take a look at our product revenue. In terms of Remodulin, grew to $110 million from $104 million, a matching quarter last year to this year. That's pretty remarkable, given that the product has been approved now for 10 years and has not taken a price increase for at least 2 years. So it's really nice to see continued strength in that core product 10 years out.

Tyvaso grew to $81 million in this quarter, up from $61.8 million the second quarter of 2011. That product is our flagship inhalation product that only has to be inhaled four times a day for just two minutes per inhalation session, compared to the only competitive alternative which needs to be inhaled for six-to-nine times a day, 10 to 15 minutes per inhalation session. So, a very dramatic difference between those two products, no doubt underlying the continued growth of Tyvaso.

And then our third product, Adcirca, grew to $30 million in the three months ending June 30th, up from $16.8 million in the three months ending June 30, 2011. This is our first oral product for the treatment of pulmonary hypertension. It's a real benefit to the patients, because they only need to take these pills one time a day, compared to the only competitive alternative in the phosphodiesterase 5 inhibitor class known as sildenafil, which needs to be taken three times a day.

So, across the board, what you see is, if you add up these revenues, you get to $225 million. And what you really see is a Company that is dedicated to doing the best it can for the pulmonary hypertension patients and the physicians, producing our best in class therapy in the parenteral class and the inhaled class and in the oral class. And the therapeutic market is rewarding us with continued revenue growth in each of these areas.

We then manage our expenses in a disciplined fashion, leading to similarly strong, strong growth in the earnings before noncash charges.

So, with that introduction, I'd now like to open the phones to any questions.

(Operator Instructions) Liana Moussatos, Wedbush Securities

Thank you for taking my question and congratulations on another strong quarter.

Thank you.

What's the status and outlook for Remodulin and Tyvaso sales outside of the US? Is Remodulin still about 15% sales still in Europe?

Thanks, Liana, nice to hear your voice this morning. Thanks for following us these years. We do look forward to continued growth in Remodulin and Tyvaso outside the US. You're very much up-to-date on the figures. We've been in the range of around 15%, ex-US, for Remodulin.

But I think we're reaching an inflection point on that, a very positive inflection point and that inflection point is the result of three different events outside the US. First of all, we just recently achieved, after years of perseverance and persistence, approval for IV Remodulin in Europe. And I think this is going to be the same game-changer in Europe that it was for the -- in the US.

Just yesterday, it's interesting, John Ferrari and I were talking about the early years of Remodulin in the US and how in the first two or three years when we only had sub-q, growth was, frankly, fairly modest in the 2002 to 2005 timeframe. And then there was something about the IV approval. Not only did it create a whole new area of revenue among IV, but it also somehow had the effect of supercharging the sub-q growth as well and I think we're going to see the same thing happen in Europe with the approval.

In Europe, it takes another half year to nine months to get all of your reimbursement approvals for the different countries. Those will be dropping into place during the balance of this year. So, I think 2013 is the first full year of IV revenues that we'll see in Europe and I believe it's going to have the same kind of transformative effect for Remodulin in Europe that it had had here in the US. So that's one positive inflection.

The second one is we are feeling very confident that we are now within 12 months of receiving Remodulin approval in China, in the People's Republic of China. And as we all know from reading the newspapers and magazines and whatnot, the Chinese market for pharmaceuticals is growing very, very rapidly.

I, for example, just as an anecdote, I was surprised to read in The Economist last week that China is now the number one market in the world for luxury goods. And you know you wouldn't really think that, but more Rolls and Bentleys are sold in China than in any other country and on and on with other luxury goods.

Well, in the somewhat somber but nevertheless realistic sense, something like Remodulin is a luxury good for people with pulmonary hypertension, because it is an advanced therapy. It's a pricy therapy. But we think that the fact that over the past several years we've built up a great network of relationships with all of the main pulmonary arterial hypertension prescribers in China.

Those were amongst our lead enrollers in our oral Treprostinil trial, so we know the prescribers, their centers, very well. We have a great reputation with all of them, because of all of the open-label continuation patients that we've kept going on oral Treprostinil over in China. And I think it augers very, very well for the take-up of IV and sub-q Remodulin in China.

It's, of course, also crucial that we were, I think, wise not to try to be an expert on distributing drugs in China ourselves, which would be I think a fool's errand, and instead we linked up with the number one drug distributor in China, Lee's Pharma. And they have definitely been preparing the ground for the launch of the drug and I think they're excited about a fast take-up.

Then last but not least, the third transformative event for Remodulin ex-US comes from Japan. Japan is a pretty mature parenteral prostacyclin market. The best data we have is that upwards of $100 million a year in sales are being booked for epoprostenol, which is the trade name for Flolan over in Japan.

And we've had to do the normal and customary things one has to do to get a drug approved in Japan. We had to, for example, do a special substudy, if you will, just in patients of Japanese ancestry. That's one of the requirements. We did that. Very pleased with the outcomes and now we're in the regulatory process.

We're looking at a 2013 approval in Japan, launch in 2014. And there I think we could reasonably expect the same experience that we had here in the US where we took 80% of the market from Flolan, because of the startlingly better convenience and safety advantages of Remodulin compared to Flolan.

With regard to the sub-q, there's no risk of septicemic infections through an open intravenous line and with regard to line interruptions, you have the long half-life associated with Remodulin not giving rise to a risk of abrupt rebound hypertension that you have with Flolan with just a couple minutes half-life. So these are all very important advantages of Remodulin.

So, I think the bottom line answer to your question, Liana, is that the future prospects of Remodulin ex-US are very good.

Thank you.

Next question, Operator?

Mark Schoenebaum, ISI Group.

Hey, guys, this is Salim stepping in for Mark. Just a question around the Gleevec panel. So Gleevec has a panel coming up, I guess, in September. Oral Remodulin didn't seem to make it into that panel and just wondering if you have any thoughts on that. Thanks.

I'm going to ask Dr. Jeffs, who's our expert in all these matters, to please answer your question.

Sure, Martine and thanks for the question, Salim. So the panel for Gleevec was just announced and I believe it was on September 14th. There're a couple of things, so let me just remind newer callers about our filing date and then our own PDUFA date.

So we filed oral Treprostinil on December 27th of 2011 and that makes our PDUFA date October 27, 2012. There are a couple, maybe three, points that would make us believe that we will not be invited to this advisory panel, which is the last advisory panel of the year for the cardio-renal group to our knowledge.

So, firstly, would be that typically at the pre-NDA meeting, which we held in the third quarter of 2011, if there was an advisory panel type issue, the division would typically tell you that there was a potential, even then, for a panel.

Secondly, there's a 55-business day notice period that the FDA would normally give to a sponsor to notify them of the need to prepare themselves for the panel. Well, we've just recently passed that 55-day notice period.

And thirdly, I think we should all remember that Treprostinil is already approved, both the sub-q, intravenous and inhaled routes, so the division has a lot of comfort, knowledge and experience with Treprostinil and there's probably no specific issue for them in terms of knowledge related to Treprostinil.

So, for those three reasons, we don't think we will be asked to participate in that panel. Having said that, you can never say never and if we were asked to participate we would gladly share our data publicly, because we remain very confident in the filing based on its meeting all of the 1998 guidance for effectiveness. So, we don't anticipate participating in the panel, but, if we did, we would confidently do that.

Great. Thank you.

Thank you. Thanks, Roger. Next call please?

Salveen Richter, Canaccord.

(Technical difficulty) my question. Just heading into this PDUFA date of October 27th, maybe you can just walk through expectations and how we should think about, if the drug is approved, real-world use here on approval?

Sure. Why don't we -- well, I'll bifurcate the question, because Dr. Jeffs could perhaps provide some color on the strength of our package that was submitted to the FDA last year and I can provide, perhaps, a little color on the second part of your question.

So, we really are not trying to count our chickens before they're hatched, in terms of talking at all about the market forecast of oral Treprostinil before we have it approved. So there's not much that I can say to you about that. Suffice to point out that over the past 10 years or so of drugs being approved for the pulmonary hypertension space, three molecules have, recurrently, shown very strong therapeutic results -- the prostacyclin class of molecules, the endothelin inhibitor class of molecules, and the PDE-5 inhibitor class of molecules.

There is actually nothing in Phase I, II, or III trials other than these three classes of drugs that have shown any type of strong silver bullet effect on pulmonary hypertension. So it's a virtual certainty that the next 5 to 10 years of treatment for pulmonary hypertension is going to revolve around the prostacyclin molecule, the endothelin molecule and the PDE-5 inhibitor molecule.

We're really excited about the fact that no matter what stage the patient is at, we'll have a formulation of the prostacyclin molecule that seems appropriate for that stage. For example, at the end stage of pulmonary hypertension, the patient needs, if I could borrow a phrase from one of the real founders of the field, Dr. Lewis Rubin, the patient needs to be "bathed in prostacyclin". Of course he means the blood bathed in prostacyclin with a continuous infusion of the drug. And of course, that it what Remodulin is all about

And we're also very excited that our implantable pump program with Medtronic is moving forward, and within the next couple of years that will provide yet another and ultimately most convenient way for a patient to get that continuous flux of prostacyclin that Dr. Rubin is referring to.

Then, you have this kind of intermediate stage of patients that the disease has really taken hold in the lungs and you need a kind of a direct battle of the disease in the distal pulmonary arterials right near the alveoli where the disease takes hold. Tyvaso is the perfect way to get prostacyclin to those distal pulmonary arterials.

Some people ask -- well, can't you do a surgery for this disease? And unfortunately, the disease is not near the heart. It's near the alveoli. It's in millions of little pulmonary arterials and there's no surgery that can be done for that.

And then, ultimately, with FDA approval, we would have a pill form of prostacyclin so that the drug could be really -- the disease could be addressed at its earliest inception, when it was diagnosed at very early stages. So, I think we really are on a very solid track with our prostacyclin franchise here and, as mentioned at the beginning of the call, we also have the best in class drug of the PDE-5 class as well.

Roger, would you like to give a little bit of color on the NDA package on oral Tre?

Yes, certainly, Martine. So maybe just I'll back up and speak to sort of the strength of the package and the sort of the anchoring position for the NDA.

So, as we've said previously, oral Treprostinil is an alternate dosage form of Treprostinil for which there is a myriad of safety experience in thousands of patients, both from the sub-q, IV and inhaled routes. So there's a lot of comfort, if you will, by the Agency with this molecular entity.

Our FREEDOM-M trial, which was in naive patients, showed a highly significant improvement in 6-minute walk distance, which was the primary endpoint, with a 23-meter improvement in the P-value of 0.0125. So that's, in fact, the best efficacy result we've had with any dosage form of Treprostinil and something we're quite proud of.

A couple of other things with regard to the safety. We now have patients -- and it's almost 400 patients -- in long-term open-label extension, with the longest duration of exposure of about 5.6 years and many, many patients over 4.0 years of exposure, which I think is self-evident in speaking to the clinical utility of this agent. Because if it didn't work and it wasn't durable, then patients would not remain on the therapy in this open-label extension trial.

I think the other thing that we've done recently that gives us great enthusiasm and confidence for the potential of the molecule in the market is, under very strict and tight control by a few investigators, we have begun transitioning patients who are on Remodulin to oral Treprostinil and we've done four such transitions at this point.

These patients have been on Remodulin for anywhere from two to upwards of eight years. They're on sub-q forms. They were having some site pain and they were at doses ranging anywhere from 25 nanograms per kilogram per minute to 75 nanograms per kilogram per minute and we've been able to transition these patients off of parenteral therapy to twice-daily oral therapy in the course of two-to-three days.

The patients who've been on this trial one-to-three months, they seem to be doing well. They are delighted that they don't have the rigors of parenteral therapy to manage. They are now swimming, hiking and increasing their activities and their quality of life seems to be greatly improved.

So that's an anecdote, if you will, but I think it shows that this agent is Treprostinil. It can work analogously to the other approved forms of Treprostinil, such as Remodulin and we remain quite confident in the data package that we've submitted.

So that's a little bit of a primer on where we are. We won't comment on the dialog we've had with the FDA about the package for oral Treprostinil at this time and we'll wait until the decision date and the conclusion of those discussions before we go public.

Thanks, Roger. That's a terrific answer. And it's important, I think, maybe just if I could just put a little exclamation mark at the end of your answer that the IP life on that agent goes out until 2026/2027 timeframe and so it's got very, very strong IP.

Next question, please?

Phil Nadeau, Cowen & Co.

Hey, Phil.

Hey, good morning, Martine, how are you?

Good, good.

Roger, just some follow-ups to the comments you just made on the strength of the FREEDOM-M package and just kind of two related questions, first on the P-value. When you were submitting Tyvaso for approval, at that time you were suggesting that a single study could suffice if the P-value was less than 0.01. I realize that you're very close to that, but technically you're not under that. So what's your thinking there?

And then, second, on 6-minute walk distance as a primary endpoint itself, we've heard from some consultants that the FDA is kind of gradually moving away from 6-minute walk distance as an endpoint, primary endpoint, and looking more towards (inaudible) endpoint. Actelion has done or at least some correspondence between a primary 6-minute walk distance endpoint and supported secondary endpoints, which I don't believe you had in the FREEDOM-M.

So what's United Therapeutics' thinking on the strength of the endpoint as well?

Great, great questions, Phil. So, on the P-value, as an alternative dosage form, I think there's a kinetic difference, if you will, between Tyvaso and oral Treprostinil. So Tyvaso, if you think about it, is really a bolus dose and is given, therefore, as an immediate release and doesn't really mimic parenteral therapy.

We specifically designed oral Treprostinil to have a twice-daily sustained release kinetic profile so that it would more closely mimic Remodulin, so it's really an alternative dosage form to Remodulin.

As an alternative dosage form, the strength of evidence is usually a single trial with a P-value less than 0.05 and that was obviously a conversation we had with the Agency at our pre-NDA meeting and at that point, we presented the data and they accepted the filing for review. I think, had they wanted a stronger strength of evidence, they would have rejected the filing at that time.

So Tyvaso, because its kinetic profile was quite different, more of an immediate release with not providing itself as a [comparative] or an alternative to Remodulin, the strength of evidence requirement for a single study submission was more in the 0,0- -- less than 0.01 range, which we obviously achieved.

Further, we have very supportive, although not statistically -- didn't meet the 0.05 standard of statistical significance, but closely approximated that -- two large trials in combination so we can show the drug is safe and certainly directionally effective when used in combination. So there's a lot of supportive data other than just FREEDOM-M that ports with the FREEDOM-C and FREEDOM-C-squared trial.

So that's kind of the rationale of why we filed on FREEDOM-M based on a 0.0125 highly significant finding.

In terms of the FDA view of what are acceptable endpoints, so there's a lot of chatter, I understand, around morbidity and mortality trials. And I think they're important to do and we certainly are taking on the FREEDOM-E event trial, or FREEDOM-EV trial, ourselves, which is provide long-term morbidity/mortality data for oral Treprostinil.

Now, I want to be clear that's not a mandated study. That's an independent study that we're doing on our own so that we can maximize the market potential of oral Treprostinil over the 2026 patent life expiry of this product.

So, I think 6-minute walk, on its own, is approvable. It's served as the primary endpoint and surrogate for all of the approved therapies to date. I think, even with the result that Actelion had with macitentan, where they showed a long-term benefit event, macitentan in terms of reducing clinical worsening or event, what was interesting is they did not announce 6-minute walk distance change other than to say that it was less than 0.05.

So that was a combination study. And my assumption, therefore, is that they've seen a walk distance that's probably small but significant as the less than 0.05 level and a more significant effect of the drug over the long-term at the less than 0.01 level.

So, I think that tells me two things. One, walk does predict long-term outcome and actually validates that 6-minute walk is a good surrogate for morbidity/mortality type things. And I think 6-minute walk, therefore, remains a good surrogate for clinical trials and approval at this time.

Now, in the future, will the FDA be a little bit more rigorous in their demands? Possibly. I think we've found in Europe that they've moved more towards a morbidly/mortality census and way of thinking and that is why we have decided not to file in Europe.

But for where we stand currently, based on our package, our interaction with the FDA both at pre-NDA and during the course of this review, 6-minute walk distance and the result that we have certainly is reviewable and portends for approval, based on a successful review.

Thank you.

Great, thank you, Roger, excellent response, very comprehensive. So, thank you very, very, very much. Next, operator, we have time for one more question, so next question.

Michael Yee, RBC Capital Markets

Oh, good. Hi, Mike.

Hi, this is actually [Jon Chang] in for Michael Yee.

Okay. Oh, tell Mike I said hi.

Yes, sure, I'll do it. I wanted to ask with regards to Tyvaso, what were the net patient adds for this quarter and what are your views on continued market penetration? Thank you.

Yes. We don't ever tally up the number of precise patients, in part because it's just too laborious of an exercise, too fraught with error. And we don't want to ever get into a situation where we've accidently said one number and we're five patients off and somebody complains about it.

So, but you can always get, especially in cases like this where inventory is flat quarter-to-quarter, you can always get a close approximation by just dividing the revenues by the average price that we get for Tyvaso, which to a round approximation is around 1.25. So, if you just divide the Tyvaso revenues by that you'll be able to get a pretty close approximation in the patient count.

The forward prospects are very good. If you take a look at the growth curve for Tyvaso, if you just plotted it on a graph and you plotted Remodulin, as a crazy plotter like I am can't help myself from doing, and you extend those lines it's predictable, if everything follows it's curve, that Tyvaso sales will actually exceed Remodulin sales within the next two years or so.

It wouldn't really be surprising, because Remodulin is a therapy which has found its best take-up among New York Heart Association Class 4 patients, which represent perhaps 10% of all of the pulmonary hypertension patients. Tyvaso has found its best take-up among New York Heart Association Class 3 patients, which are about 40% of the patients.

And since the price of Tyvaso and Remodulin are pretty comparable, just the normal course of arithmetic and trends here are that Tyvaso will continue to grow and end up even with greater revenues than Remodulin.

So things look very good on the Tyvaso front. We are always doing things to improve the therapy. It's got Orphan Drug Exclusivity approval. We recently have expanded some of our advanced QA/QC storage space for Tyvaso, because we are shipping out so many inhalers and disposables that we needed a more advanced space and all of our inventory and manufacturing is under SAP control, SAP controlled inventory management system.

So we're continuing to invest significantly in the Tyvaso franchise, because we expect this franchise to go strong for a decade or more, just like Remodulin.

Well, everybody, thank you so much for your questions this morning. Liana, thank you for your congratulations earlier.

And just to reiterate what I mentioned at the beginning of the call, for those of you who may have come in late, revenues up, cash profits up, every component, every one of our products up and we continue to build on this strong core business.

We also had a chance to talk, answer some questions about pipeline, the oral Treprostinil being the most exciting near-term portion of our pipeline. So that's all looking very positive and strong as well.

Thanks for your interest and support of United Therapeutics. The IR team and myself will be at BMO Capital Markets next week for some one-on-ones and we look forward to seeing you at other healthcare conferences during the next few months.

Thank you, Operator.

You're welcome. And thank you for participating in today's United Therapeutics Corporation second quarter earnings conference call. This call will be available for replay beginning at 11.30 a.m. Eastern time today through 11.59 p.m. Eastern time on Friday, August 3rd. The conference ID number for the replay is 96430669. The number to dial for the replay is 855-859-2056 or 404-537-3406. Thank you for your participation. You may now disconnect and have a wonderful day.