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Alexion Pharmaceuticals, Inc. (ALXN)

June 12, 2013 11:40 am ET

Executives

Leonard Bell - Co-Founder, Chief Executive Officer, Treasurer and Director

Analysts

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Thanks. Good morning, everyone. Thanks for joining us. I'm Terence Flynn, I'm one of the biotech analysts here at Goldman Sachs. We're very pleased to have Alexion with us this morning. And joining us from the company is Doctor Lenny Bell, CEO. Thanks, Lenny, for your time. Before we get started, I'm required to make certain disclosures in public appearances about Goldman Sachs' relationships with companies that we discuss, the disclosures relate to investment banking relationships, compensation received, or 1% or more ownership. I'm prepared to read disclosures for any issuer now or at the end of this call if anyone would like me to. However, these disclosures are available in our most recent reports available to U.S. clients on our firm portals. In addition, updates to those disclosures are available by ticker on the firm's public website at www.gs.com/research/hedge.html. Thanks a lot, Lenny.

Leonard Bell

Geez. You're using that every 0.5 hours. Thanks, Terence. It's a pleasure to be here.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Maybe to start off, I think maybe I could place would be just, you guys had a number of additions down the senior management team, and maybe if you could just provide us some perspective on those people? Why now? And the path forward here?

Leonard Bell

Great. Thanks, Terence. Yes, as Alexion is growing over the last 5, 6, 7 years from essentially a development stage company to a U.S. commercial company to now a multinational, in one indication, and now a second indication in 30, 40 countries around the world. There's been a fair amount of growth in organizational structure and talent. And in fact, over the last 2 years, 5 of the 8 people who currently report to me weren't employed with Alexion 2 years ago. So Chief Human Resource Officer, Clare Carmichael, as the first; and Frank Wright, who came in and to run our international operations in Switzerland; and John Moriarty, our General Counsel; and then the 2 most recent would be Martin Mackay, previously Head of Research and Development at Pfizer and then AstraZeneca, and now, Global Head of R&D at Alexion, reporting directly to me; and of course, Saqib Islam is with me today. I actually would note that today is Saqib's birthday also. So it's an important note. We'd come back to that later in the discussion, I'm sure. But Saqib came. We've known each other for well over a decade, and came now as heading Strategic Development and Corporate Development as well as managing all the risk and opportunities across the company. So it's really an indication of evolution over the last 2 years of the need for a growing talent in the most senior level men, obviously, throughout the corporation, not only at a corporate level, but also of course, regionally and across all the countries that we operate in.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

So in terms of looking forward, are there anything we should expect differently on either, I guess, R&D side and/or BD given the recent additions?

Leonard Bell

Yes, I think qualitatively I would expect much of the same, which is that we have sharpened and maintained our focus on identifying those disorders than the most severe and devastating. We only seek to invest either internally or externally in treatments that will provide a large and transformative impact on patients' lives. And the rarity is really a secondary kind of assessment on our part. It's really the type of disorder, the severity and the transformative nature of the treatment. And so that focus, I think will stay very much the same, but I think one can also see that they're important engines for growth, are not only a strategic development, but of course internal R&D and they're tightly related as it turns out. So I would expect us to do more of the same, but maybe at a faster pace both on internally and otherwise.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Okay. Great. And then maybe one other strategic question, just -- there's been a tremendous amount of discussion on drug pricing given the number of austerity measures across the globe, you guys obviously are at the front lines of some of these discussions. Maybe give us your perspective on the future of drug pricing, tie that back to innovation and I'll start there.

Leonard Bell

Yes, we're sort of an unusual situation. As it turns out, we chose at an opportune time to launch a multinational company, 2007, 2008. Looking back, it probably would've been an easier time. So we actually launched a transformative treatment across much of the developed world at a time that the road is going through the greatest recession that it had in previous half-century or more. So I think that certainly, we've been breadth under fire for sure. And now, 5, 6 years later, if anything, I think it's eased up a bit from compared to what it was then throughout the world. Many economies, obviously, have not regained their footing. For certain, we obviously feel for that and all the countries that we operate and feel mostly for the citizens there, of course. But as we look at that, at the same time, I think that there is generally a sharpening across different cultures and different health care systems where appropriately admire [ph] you at least. Our payers are looking to distinguish between those that are really transformative and just sort of me-too type treatments and they're looking to separate that out and where they invest in terms of sponsoring innovation.

Innovation, it's a term that's thrown around a lot. It's sort of like the weather. People kind of report it, but often they don't really impact it very much. And I think that governments throughout the world are starting to identify that. Even in the United States for example, they came up with a new term for regulatory perspective. Breakthrough Therapy designation which for the first time, require actually the treatment effect as opposed to just the disorder severity, that should have a treatment effect as well, which is kind of important policy change. So we do see, over time, that most economies will look to distinguish between me-too less innovative third product for patient who's been treated with 2 other products already that none of us have major effect versus those that kind have an effect in every single patient and are transformative. The biggest change actually, I would say, is probably in Germany, where they actually looked to adopt the system that looks distinguished on a qualitative way between me-too products where they're not anxious to give strong pricing reimbursement for appropriately my view versus actually truly transformative treatments where they anticipate doing that.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Would -- If you look across the different systems in the world, which would you say does the best job at kind of making that distinction and driving true value?

Leonard Bell

That's a tough one. So I think that the reality is that speaking and interacting with governments around the world, everyone's beset by the same problems and I don't think anyone's really found that magic bullet. It's like most experiences we have in life that it's always too much or too little, and the sides, which sides you like to error on. I think that -- and it's a cultural issue, which side they seek to error on it, the governments don't error by mistake, they error intentionally. So those governments that have made decisions to ration care so that patients who could benefit don't get access, I think there's a societal cause of that. And so I don't there's a suicidal cost to that. So I don't anyone has really found the magic bullet per se.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Okay, and then maybe one other thing as we look forward. Someone impacts just the introduction of potentially biosimilars in the U.S., there's some debate about how this will actually impact the branded market. Obviously, it will depend on a number of players, but if you look forward here, how do you see that playing out on the U.S. side of things and what are you guys doing to plan for that in the event that there is one day a solar specimen?

Leonard Bell

Yes, so it's a matter of policy. In United States, there's both Federal policy and law and the regulation and then there's also increase in state laws and regulations and distinguishing between something substitutable versus not substitutable. We think that the way it currently stands, I think that there is a good protection for biologic, highly innovative treatments in the United States. We would anticipate that given the strength of that in terms of being a manufacturing job base, I think that will be actually be sustained. We see those distinctions in and that's -- was that a substitutable drug which is a very big difference versus something that's just similar. We should have used difference in value quite frankly. And so we see the strong line in the sands that have been drawn between substitutable and non-substitutable as extremely important from a policy perspective. We're excited about continuing to not only provide Soliris, but of course, to provide improvements as they come along where we're tremendously incented of course to buy the very best outcomes we can for patients here in the United States, but around the rest of the world, where we operate as well, and we are keenly focused on doing that over the next short period of time.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

And how about in the life cycle management side for Soliris, is there anything there that you can talk to that's in development or planning stages?

Leonard Bell

Yes, absolutely, there's at list, 3 molecules that are distinct and in development currently, that we think will provide opportunities for the patients that currently don't have and I would anticipate that in all likelihood, we'd probably, over in a relatively near period of time go forward more than one at a time, again with the overall objective of try to provide the very best outcome to as many patients with devastating disorders as possible.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Okay. And now as we move on to Soliris, from my perspective, looks like the aHUS launch is obviously off to a very strong start in the U.S., a little earlier in Europe, but maybe just walk us through some of the drivers here that success in the U.S. and then maybe applying that to the European side of the business and what about some perspective on kind of how you see this playing out longer term?

Leonard Bell

Sure, well, we have established a strong presence and appreciation in hematology community by virtue of Soliris' activity of PNH. That's established a strong image and flavor for what Soliris can do at a severe and life-threatening cognitive [ph] disorder, and I think if anything has been amplified in the United States and also, increasingly now outside in terms of what Soliris can provide to patients with aHUS. And I say, if anything, it's going to amplified because aHUS is, if anything, somewhat more higher velocity, severity than PNH is typically. And I think that by virtue of the effectiveness of Soliris and the well-tolerated aspect of it, is that it can really stop a rapidly progressive disorder and maintain it for the lifetime of the patient where they're constantly susceptible to this genetic account activation. But I think that sort of transition to something that could be much more rapidly progressive to a patient is a key piece of how we work to operate. We find, in the United States, perhaps the greatest threat to patients with aHUS is the difficulty of rapidly recognizing it in the hospital setting exactly what it is, soon enough, until there is a really just such rapidly severe damage to the kidney or to other vital organs like the brain or the heart or the gut, that's really our biggest challenge, actually, is the shortened of time or patients can -- physicians, excuse me, and other health care providers will recognize that this is a rapid course, they need intervene that this is rabid course. There's a publication electronically in early with very promising, there's publication electronically very recently in last few days really and the one premier medical journals, which really focused on the effectiveness of Soliris and diverse aHUS population. I also made the point that earliest treatment is best for these patients by virtue of providing data and how much better the outcome could be. So we think that's really the key of learning actually is, the obstacle to optimal care is early intervention for these patients. They don't have, unfortunately, a lot of time. We can dog carry forward as we look to transition increasingly. Our growing businesses now in Germany, the Netherlands, and next month, starting in Belgium, already a small business starting in England, and then probably in the fall, starting in France as well. And Italy, currently underway as well, Spain. We think those are really probably the key learnings. I think that there's increasing depreciation that aHUS has underlying multitude of different genetic causes or mechanisms, and like any genetic disorder, the genes don't -- the gene activation doesn't go away. It is what it is, so the life long aspect, we think is increasingly appreciated, it's just the need to intervene earlier and something that's raising by the medical staff is really what we see is the biggest opportunity in improved care. Here in the U.S. so I could confidently the same in European countries.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

And what's the average age of treatment initiation for aHUS routed to PNH, I mean, how big of a difference is there? And then any commentary on just treatment duration, I think that was the other thing raised in the New England Journal Medicine publication?

Leonard Bell

Yes, so typically most patients are diagnosed with PNH in their 30s. And even today, unfortunately, with all the activities and education and so forth, it's a little disappointing to know that how difficult it is to change care and patients are getting care more quickly, but PNH is probably not til several years later actually that they start treatment usually, there's a spread of course, but probably mid to later 30s. In the case of aHUS, it's a little bit distinguished that in our experience, most of the children are typically seen by pediatrics or nephrologists and the adults are seen somewhat similarly between nephrologists in United States, very much so by hematologist actually. And so, we see obviously in the adult population, ages are typically actually still a little bit younger, so maybe high 20s, low 30s. But we definitely see patients in their 50s or 60s. And the pediatric population, it's really much closer to 6 to 8.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

I And I guess I'm -- know in terms of [indiscernible]

Leonard Bell

I'm sorry for the duration of the bucket. So it's interesting that between the period of time when we had discussions with the European Medicines Agency regarding Soliris for PNH which was 2007, and the like time for Soliris and aHUS which is 2011, there have become additional regulations from the EMA to speak in the European version packages, I think sort off thought in SERT which is called SMPC, to talk about recommendations for duration of treatment, so it's now required part of the pathogens here in Europe. And the pathogens right then in Europe actually states that Soliris is recommended to be treatment for the patient's lifetime, unless there are some safety consideration, so it's pretty explosive, actually. Id doesn't say maybe, it doesn't say you can think about it, this is what we recommend. And the reason for that is very important. In severe [indiscernible] disorders, a little bit of leakage could cause a lot of problems, so there's a little bit of [indiscernible] activation that occurs in these patients be it PNH or aHUS, we'll talk about the aHUS but it's equally applicable to PNH. You can get a severe disruption in the case of aHUS -- PNH is blowing up red cells and actively inputs within clotting. In the case of aHUS, it's like damaged in field of layer and destroying vital organs like the kidney, brain, heart, gut and so forth. And the observation is that when you stop Soliris in the package in certain Europe, there is indications both of PNH and we're talking about aHUS in particular, that significant proportion of patients went on to a very severe outcomes that in some cases, were not reversible. So they went into kidney failure and cardiorespiratory arrest, required dialysis, a lot of severe outcomes that occurred because of that little bit of activation occurred afterwards. And the new internal medication publication that you referenced actually also made comments likewise like that since these physicians are of course are aware of this. And I think it's a very powerful thing, particularly, and a very rare disorder, that's a very devastating disorder for regulators provide that type of guidance, and then perhaps even more valuable in some ways for the physician than more common disorders and the reason for these physicians are typically less familiar obviously with more rare disorders and particularly that common issue of rarity, less knowledge and high severity that's a good idea to have that recommendation of lifetime treatment. And so we certainly in Europe, obviously, we endorse that very much.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

And do you see anything different right now in terms of PNH versus aHUS in terms of what I saw early in aHUS but in terms of that treatment duration, and anything you can do to kind of bring that message on the physicians?

Leonard Bell

Absolutely, and in fact, actually, I think it's that part up front about physicians recognizing as described in this recent publication that earliest treatment is the best outcome. It's actually that's probably the biggest piece and the reason for it is obviously, very disappointing for physicians to treat something too late when it's too late to be done about it so they'll obviously to treat for a short period of time, so I think the focus on that disease education and now there's a publication as well that says that says that not only is the disease go quickly, but the publications says earliest treatment is the very best. I think that's actually the biggest approach towards duration of treatment actually.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Okay. And then any update in terms of the U.K.? I know that you guys have gone back and forth, you're getting revenue right now, minute by minute. Anything you can say there in terms of next steps and what to look for, I know there's going to be review by NICE at some point, but we're just loving the latest update.

Leonard Bell

We love all the governments around the world equally, no one more and no one less than other one for sure. And in England, every 2 or 3 years, over the last 6, 7 years, we've created new process for evaluating the treatments, so the process that Soliris was evaluated in for PNH was a new process. After that, they changed the process again. This process that Soliris was a value for aHUS was that new process, came out with a [indiscernible] deposit of recommendation describing actually that -- really, it's quite remarkable for a government reporting any government around the world would never seen this. Describing enough the drill will save patients' lives both children as you point out and adults, of 25% of patients have immediate mortality. The politicians then decided that they didn't want to go forward and accept that recommendation, then they delayed any decision-making, they created an adhoc process, unfortunately, the pressure they're under by citizens who are patients, and to provide some sort of limited access for patients in a various sort of convoluted way. And at the same time, they've said publicly that they're going to look to create better access in a near term. We understand that they've had a series of meetings to sort of provide a broader access policy. So both the policy of that will be more broadly understandable, but also providing access to a broader patient population. So broader both ways in those case. And we're very encouraged, frankly, that we now see that patients are starting to get access to Soliris, it's a relatively limited number, in a typical way that happens often in England, it's often based upon where the individual lives and who they know, which is not a very democratic way to access if you have a life threatening disorder. And so over the next few months, we anticipate this becoming public, hopefully, it's shorter than that, the new access policy. And we think that not only the access policy itself will be broader, but I think if they make it publicly available, that will be helpful for patients as well. And patients are getting access. Soliris is being reimbursed, and those numbers are growing overtime and I think that will continue to go all the way going forward.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

And so in terms of the patients who are getting access now with those big patients in larger cities who live near academic medical centers or something, or what's the -- how does that play out in terms of who's getting access right now?

Leonard Bell

It's a bit more haphazard than that. We spent a lot of our time around the world focused on equity. It's a very important principle, it's not recognized universally by all governments, even in the United States sometimes, but good people make good decisions and equity is extremely important. So, I mean, it's unfortunate, what -- I mean there's no good answer, because any answers in my view where there's lack of equity I think are bad answers, but with a haphazard it makes it a little worse.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

And so the final process sometime NICE will put out some guidance, and then they would review Soliris for aHUS, and then that would provide access for a broader set of patients?

Leonard Bell

I don't really know that we would be broader actually. I actually -- I don't know that I believe that actually. I actually think that from what I know now, you think -- I think they're likely to provide a official publicly known clinical access policy that's relatively brought anyway before hand. I think that will happen over the next few months.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Okay, so you think it happens before the actual review?

Leonard Bell

I do. Yes. And I think it's a policy issue for sure whether it's very hard to see how this will happen, but anything's possible I suppose. I don't think they would restrict the policy from there, but people do funny things sometimes. But I mean, that would be very surprising, I think.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

And any insights on what NICE is actually going to use as a benchmark, because I know quality is very difficult to apply the orphan drugs. So any insight in terms of what factors they might choose to make these decisions?

Leonard Bell

My impression the last half dozen years or so is that unlike many countries, England tends to have specific criteria in regulations that's used for valuation. And in our experience, they just don't follow them. So it's hard for me to judge necessarily what actually they will use because that's a combination of the two. But I would anticipate it's somehow trying to understand the transformative impact, understand how limited the patient population is and so forth, and I would imagine at some quality aspect of it, how that's weighted, I think no one really knows. It's a very unusual setting because I think most people anticipate it will be very, very similar, if not closer to that, then exactly AGNSS did, which was a highly defined process actually. So I don't think anyone really anticipates it being different than the same menu, frankly, as well, friendly comp is roughly going to be the same.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Any question from the audience before we move on with the pipeline? Okay. Maybe just to start on, if we look at maybe the third, we'll start with asfotase alfa there isn't much time left, but you guys recently received Breakthrough designation there, you mentioned you're opening remarks that you think this is interesting move on the FDA to provide this designation for not just Soliris but other drugs, maybe provide some perspective there on what the new nuances of the Breakthrough designation are and then maybe the path forward here up until the filing, what are the gaiting steps for asfotase alfa?

Leonard Bell

Having had a lot of intimate experience with Soliris and PNH and U.S. and Europe, almost simultaneously in March -- February, March and April, 2007, and then a little bit less underneath but enough that I have swallowed the taste for aHUS in both the Europe and U.S., it's actually a pretty important change. And the reason for that is there are a number of mechanisms by which the U.S. drug regulatory policy her policy seeks to accelerate development, and to me, it's a subtle change, but it's a pretty big one actually to move this from a situation where you look at how awful the disease is. And again, in my view, it's a relatively modest criteria that you used for how devastating, but there's important criteria, right. So this will be essentially the prior review or fast track types of things, which are not exactly the same, but pretty similar in terms of criteria, and then to add on top of that, a treatment effect requirement, which is soft and qualitative as opposed to quantitative, I think it's a very big policy change actually. And it's very consistent which is why I mentioned it, it's very consistent with the approach over time to sort of separate out. So the number of treatments get prior to the review and so forth, it's a lot. So all is going to stopwrite, but at the end of the day, there's a difference. And which is a little bit like, as I mentioned, some of the European systems, not from a regulatory perspective per se, but from a reimbursement perspective. So I don't think that's irrelevant, I think it's probably an important policy change, actually, in the United States, I welcome it, I think it's a very positive thing to do. So I do think overall, regulators or the economy can kind of cover everything that people want to invest in or quite frankly, do I personally think they should. Now, I think that, that addition of treatment benefit is extremely important from our view perspective, in my experience, with Soliris and PHS and aHUS and the reason for it is in the formal business, it's highly motivating for review, these are very good people who are looking to do good things. And to institutionalize that, by saying this is something I wanted to carve out your time for, and make available more time, not just because you're a good person but because this is the rule I have. I think it's extremely important. I think that, hard to say, in any particular draw, what the impact would be, but I think what they've said publicly, which as Mark MacKay said on a variety of discussions, with regulators over the last month or 2 about this, before and after draining Alexion, but about exactly the Alexion face that essentially, on the manufacturing end, this should look to sort of facilitate that, on a number of things that they can look to evaluate and address together earlier, so I it's a very positive, it's actually -- but I think if the treatment effect, because that's generally have FDA reviewers who work, it's a very unpleasant expense having had this earlier at Alexione, maybe 10, 15 thing years ago, to try and have that conversation with regulators about our burning [ph] modest effect, and on the other hand, whether they're going in, as was the case with Soliris and PNH with 10, 15, 0s [indiscernible] decimal point in the first integer, it's a very different kind of experience. And I think we're trying to institutionalize that.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Okay, great. And I guess, so the second piece in terms of timelines here for asfotase alfa, where is it getting...

Leonard Bell

5, 10 years thing. Just kidding.

Yes. So yes, I apologize for Essentially. So essentially We, in the Breakthrough designation, I point out also does before you go past that longest lead of Breakthrough Therapy designation, they talk it none onset of disease, which is very important at about sort of essentially onset of children, and also disease in children, which actually obviously means that patience may get treated after their children. no longer children are older, which is extremely important and I think that we have an infantile onset, natural history study. That we think that data will be good, it's completed. Then the hope that become available there they say, there's a variety of presentations looking at different demographics of its back in and if it's an offer would different bases 70 pieces, and now that tracks with other markers, for example, which will become available this year. We don't yet know where to do the juvenile randomized control study, but we're planning us if we do, we need to know that. Manufactories is progress, we're looking to luck and we anticipate filing probably sometime around or somewhat before mid-year next year.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Ended to the juvenile trial that would be as much trial you guys have said.

Leonard Bell

I think so.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

And then any sense of how big that day would be, 15, 20 patients?

Leonard Bell

Yes, maybe 30, 40. Something like 30, 40.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

And so that if -- and when will we have clarity if that piece will be required or not?

Leonard Bell

Probably sometime in the fall. Let me share that with you.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

And then the other data you mentioned regarding HPP at that, provision for HPP data that have generated at Alexion, or is this someone's more mature data from some of the Novi databases. What's the...

Leonard Bell

It's supposed -- a lot of it's actually additional data that's been generated.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

And that's from a kind of a worldwide sample for you...

Leonard Bell

Across sections, yes.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Okay. And so that will be a supplement, I guess, to the Natural History studies?

Leonard Bell

It's actually not meant from a regulatory perspective. It's more giving inside to [indiscernible] what the community looks like and so forth. The patient population segmentation so forth.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

And is that...

Leonard Bell

Well, it's not a dating issue from regulatory perspective in life.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Will that give us, I guess, a better sense of potentially market size when we see that data or...?

Leonard Bell

Hopefully not. That would be a mistake, no?

Probably not. Just give you a flavor of different types of patients presentations, how severe and of the once you see how this segment.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

And I guess, so those are 2 main buckets. So that's the peds and then the juveniles. But then as you look forward to [indiscernible] patient population, I know you said you think you have some more work to do there in terms of thinking about identifying the best segment of the population to maybe take this therapy forward and when might we hear more about that? And what have you guys been doing on that front to address that.

Leonard Bell

That actually is sitting on the phone as well, and the issue then in terms as you're describing which I agree with is that we're not focused on developing treatment for patients with mild rare disorders, we're really focused on developing, transforming the treatments for patients with severe disorders that happened to be aware. And so we recognize that when we first looked at the opportunity a year or 2 ago, which is that they hadn't really focused it on those types of adults with that level of severity and that's where we intent to focus our efforts for sure. And that really will be looking at those adult on-set patients who just got very severe disorders and we expect that after we get data from the current trial underway that they have started beforehand then we'll get additional 12 month data sometime, probably late summer or fall, and I think that will be informative to how we shape the next study in adults. The only thing I tried to mention is that I know we said in our last earnings call is that we had also started a treatment study in Japan, hypophosphatasia, and that's a particularly important, it's been well described in the west how there are some particular virial mutations that are present in Canada and elsewhere. As it turns out that there is different mutations, but of equal very high virial and severity and disorder in children in Japan. And so we would anticipate as opposed to the 1 or 2 year delays we've had in aHUS and 2 or 3 delays we've had in PNH, we've actually looking to file in Japan in 2014 as well for hypophosphatasia and asfotase alfa.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Okay, great. May we just in the last minute, another piece of data, I think people are focused on ears, Soliris for transplant. We're expecting some Phase II data later this year, maybe just highlight for us what you guys view as the benchmark for success there, in that setting. What would be viewed as a positive outcome in your view?

Leonard Bell

Yes, sure. There's 2 different studies that we've started, one, is a randomized living-donor trial, where it's a multinational trial and patients sort of the -- primarily, what's important is 9 week and about immediate rejection. The challenge that we run into, of course, is that positions understandably are somewhat resistant to putting patients into non-active [indiscernible]. Having the opportunity for [indiscernible] helps a little bit there, but still it's a challenge. That's actually started picking up in Rome at a little bit over the next month or 2 which we're enthused by. We anticipate the complete enrollment in the study by year end, that will be very important study, because it's a randomized trial for sure. The disease-donor trial, which actually enroll at a much faster pace than we have anticipated internally. That was a single ARM study. Again, a multinational study that already completed enrollment and we think that data we presented publicly and scientific sayings for the 2nd half of the year, that study also not only looked at antibody-mediated rejections of primary endpoint, but has had as its key secondary endpoint laid graph function. And we think taking the deceased-donor data for AMR, and looking at where we are in the living-donor will give us a view towards what we think the regulatory strategy will be. We believe that the diseased-donor study is probably going to be sufficient for hypothesis generating to go going into a registration delayed graph function trial which we expect to do right around year end. So we go forward ahead then with probably 3 indications living donor-AMR, deceased donor-AMR, and delayed graft function, being registration extra.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Okay great, well, I think we're out of time.

Leonard Bell

Thanks very much, Terence. Again, Saqib's birthday, if anybody wants to wish her a happy birthday.

Terence C. Flynn - Goldman Sachs Group Inc., Research Division

Happy birthday, Saqib.

Leonard Bell

Thank you very much.

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Source: Alexion Pharmaceuticals, Inc. Presents at Goldman Sachs 34th Annual Global Healthcare Conference, Jun-12-2013 08:40 AM
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