Alexion Pharmaceuticals' CEO Discusses FDA Approval of Soliris as a Treatment for Patients with aHUS - Conference Call

Alexion Pharmaceuticals, Inc (NASDAQ:ALXN)

FDA Approval of Soliris as a Treatment for Patients with aHUS Call Transcript

September 26, 2011, 10:00 am ET


Leonard Bell - CEO

Tom Dubin - SVP, Chief Legal Officer

David Hallal - SVP, Global Commercial Operations

Vikas Sinha - SVP, CFO


Salveen Richter - Collins Stewart

Sapna Srivastava - Goldman Sachs

Eric Schmidt - Cowen and Company

Geoff Porges - Bernstein

Robin Karnauskas - Deutsche Bank

Matthew Luchini - Piper Jaffray

[Andrew] - UBS

Jonathan Eckard - Leerink Swan

Marko Kozul - ThinkEquity

[Brian Kwon] - Citigroup


Good day, everyone. Welcome to today's Alexion Pharmaceuticals conference call. Today's call is being recorded. At this time, for opening remarks and introductions, I'd like to turn the conference over to Dr. Leonard Bell. Please go ahead.

Leonard Bell

Thank you, Operator. Good morning and thank you for joining us on today's call to discuss the FDA approval of Soliris as a treatment for patients with aHUS. I am joined today by David Hallal, Senior Vice President, Global Commercial Operations, and Vikas Sinha, Senior Vice President and Chief Financial Officer.

Also with us today are Stephen Squinto, Executive Vice President and Head of R&D, and Tom Dubin, Senior Vice President and Chief Legal Officer. We are also extremely pleased to welcome our Alexion team working around the world.

I will begin today's call by discussing our broad label for all patients with aHUS. David will then outline our plan to provide Soliris to the first US patients with aHUS as we continue to serve more patients with PNH.

Finally, Vikas will discuss some key financial matters, including Friday's upward revision guidance resulting from our improved outlook in PNH.

Before we begin, Tom will apprise you of our potential to make forward-looking statements. Tom?

Tom Dubin

Thanks, Lenny. During this call, we may make forward-looking statements such as medical benefits of Soliris, commercial potential and plans for the commercial launch of Soliris and aHUS, plans for further clinical trials of Soliris and aHUS and operations, reimbursement, price approval and funding processes in different territories.

Forward-looking statements are subject to factors and may cause our results and plans to differ from those expected including decisions of regulatory authorities regarding approval or limitations on the marketing of Soliris for various indications; the possibility that results of clinical trials are not predictive of the safety and efficacy of Soliris in broader patient populations in the disease study or in other diseases; the risk that third parties won't agree to license any necessary intellectual property to us on reasonable terms or at all; the possibility that initial results of commercialization are not predictive of future results; the risk that third-party pairs will not continue to reimburse for the use of Soliris at acceptable rates or at all; and a variety of other risks set forth from time to time in our filings with the SEC, including our 10-Q for the quarter ended June 30, 2011.

We do not intend to update any of these forward-looking statements after this call except when the duty arises under law.

Before beginning this call, I want to point out that we will be limiting today's question-and-answer period to the subject of our aHUS approval in the US and positive opinions in the [CHMD].

A more complete update of Alexion's operations, research pipeline and financial performance will be provided in our third-quarter conference call, which we expect to hold in the second half of October. Thank you. Lenny?

Leonard Bell

Thanks, Tom. Friday was truly a historic day for patients and families suffering with atypical hemolytic uremic syndrome, or aHUS. The FDA approval of Soliris as the first treatment for patients with aHUS brings life-transforming hope that children and adults with this severe, debilitating and life-threatening ultra rare disorder.

With the approval of Soliris with a broad label, this unprecedented hope is now extended to patients with aHUS of all ages and all clinical profiles in the United States.

The FDA approval of a second indication for Soliris opens a new era for patients with aHUS. The approval is likewise a milestone in Alexion's mission to provide innovative and life-transforming therapies to patients with severe and ultra rare disorders.

As we continue to serve more patients with PNH in more countries, we are now also fully prepared to begin making Soliris immediately available to aHUS patients in the United States.

On Friday, we are also very pleased to announce the positive opinion by the [CHMP] recommending the granting of a marketing authorization for Soliris as a treatment for children and adults with aHUS in Europe, a key milestone for patient access.

Approval by the European Commission is expected in approximately two months. Following country-by-country reimbursement processes, this would enable us to start serving patients in the first major European countries in the first half of 2012 with additional major European countries commencing through mid-2013.

Turning back to the United States, we have received a broad and strong label that will help us accomplish our key goals on behalf of patients with aHUS. The label supports building a common understanding among physicians that aHUS is a disorder of uncontrolled complement activation and identifies the dramatic clinical benefits of terminal complement inhibition with Soliris in patients with this ultra rare and life-threatening disorder.

Most importantly, our strong label will support broad and unrestricted access to Soliris for all patients with aHUS in the United States. Soliris is indicated for the treatment of patients with aHUS to inhibit complement-mediated thrombotic microangiopathy, for TMA, thus the new product indication statement clearly identifies the disease mechanism of aHUS and ties it directly to the therapeutic action of Soliris.

By way of background, in patients with aHUS, genetic mutations result in a lifelong deficiency in one or more complement regulatory proteins leading to permanent, uncontrolled and excessive complement activation.

This uncontrolled complement activation in turn causes activation of platelets, white blood cells and endothelial cells resulting in clots in small blood vessels throughout the body, the condition known as systemic TMA.

In patients with aHUS, complement-mediated TMA causes progressive failure of vital organs leading to stroke, heart attack, kidney failure and death. The importance of inhibiting complement-mediate TMA in patients with aHUS is clear. Currently, within one year of diagnosis more than 50% of all patients with aHUS die, require kidney dialysis or have permanent renal damage despite the intensive use of currently available supportive care.

We are especially pleased to have achieved a new aHUS indication for patients of all ages, explicitly including children. I'll particularly note the expanded label confirms that the benefit of Soliris in children is similar to the benefit in adults.

Our accelerated approval is based on two prospective studies in 37 adult patients and one retrospective study that included 19 pediatric patients. Importantly, the label clearly establishes the effectiveness of Soliris to reduce TMA and improve renal function across a broad patient population.

Prospective clinical trials are ongoing with the same endpoints as the already successfully completed trials. In a disease defined by uncontrolled complement activation, the product label confirms that all patients treated with Soliris demonstrate a reduction in terminal complement activity.

This underscores the fundamental role of uncontrolled complement activation in aHUS and the highly efficient nature of Soliris treatment.

Next, the label notes that the TMA process is responsible for thrombosis, renal impairments, seizures and angina in patients with aHUS and that Soliris reduced complement-mediated TMA activity.

Additionally, the label states that patients treated with Soliris demonstrate important reductions in TMA, elimination of plasma exchange and plasma infusion, improved kidney function, reduction in need for dialysis and absence of any new dialysis while on Soliris. These clinical benefits are life transforming for children and adults with aHUS.

The expanded product label also confirms that aHUS patients do not require an identified genetic mutation for diagnosis in treatment and that there's also no requirement for any supportive care prior to initiation of Soliris therapy.

Additionally, Soliris was well tolerated in all three studies. The most frequently reported adverse events were generally mild to moderate hypertension, upper respiratory track infection in diarrhea.

The safety profile of Soliris is essentially unchanged in the expanded aHUS indication from the PNH indication. As with PNH, patients receiving Soliris for aHUS must be vaccinated against the long understood risk of meningococcal infection and patients should also be monitored for early signs of meningococcal infection.

Patients with aHUS, like patients with PNH since approval in 2007, will receive Soliris through a risk evaluation and management strategy or REMS to help ensure safe use.

I'd like to point out that there are multiple aspects of the aHUS label for Soliris that specifically support broad patient access and reimbursement. First, aHUS is defined as a disease of uncontrolled complement activation, thus establishing that all patients can benefit from complement inhibition with Soliris.

Second, Soliris is highly efficient. All patients treated with Soliris demonstrate a reduction in terminal complement activity. Importantly, the label specifically includes patients of any age, children as well as adults.

Next, the label has no requirement for any supportive care prior to commencing Soliris therapy. Additionally, the label confirms that not all patients with aHUS have an identified genetic mutation and that patients had equally strong responses to Soliris irrespective whether a specific mutation was identified or not.

Finally, the label strongly affirms the robust efficacy of Soliris, demonstrating the impact of sustained treatment to improve renal function, thus minimizing the progression to renal failure, dialysis, transplant and [inaudible].

In 2007, when Alexion received FDA approval for Soliris for the treatment of patients with PNH, we stated our objective that every patient with PNH who can benefit from Soliris will have access to Soliris. We are now also affirming the same objective for patients with aHUS in the United States that every patient with aHUS who can benefit from Soliris will have access to Soliris.

As the key component of this objective, the registered nurses in our one-source treatment support program will now serve patients with aHUS, just as they have done for patients with PNH since 2007.

Our Alexion case managers working here in Connecticut will provide personalized support to United States patients with aHUS and their physicians by helping them coordinate care and secure reimbursement.

Turning to our going-forward expectations in aHUS, we anticipate that over time Soliris will transform the lives of increasing numbers of patients worldwide. However, I would like to point out that in the early stages of our operations to serve patients with aHUS, we expect that the use of Soliris will at first grow only gradually.

In this regard, as we have noted in the past, the prevalence of patients with aHUS appears to be lower than that of PNH. This lower prevalence likely results in part from the current natural history of aHUS.

Unfortunately, more than one-half of patients not treated with Soliris are known to advance to dialysis, permanent kidney damage or die within the first year of following diagnosis. This is true despite plasma exchange or other supportive care.

The lower prevalence of aHUS is reflected in the substantially lower number of patients in our clinical trials with aHUS compared to PNH.

While there were 195 patients worldwide in the PNH registration studies, there were only 37 patients worldwide in the perspective aHUS studies. Further, like in PNH, only a small minority of patients in our aHUS clinical studies are in the US. For these reasons we expect that the use of Soliris in aHUS will initially grow only gradually.

We are eager for this new opportunity to serve children and adults with aHUS worldwide beginning here in the US. I'll now turn the call over to David to provide more insight into our initiatives as we immediately start to serve patients with aHUS in the United States. David?

David Hallal

Thanks, Lenny. With FDA approval for Soliris in aHUS, we are now immediately prepared to make Soliris available to patients with this ultra rare, devastating and life-threatening disease.

As we begin to serve patients with aHUS in the United States, I note that, as it relates to PNH, we are mindful that even in the countries where we have been operating the longest, the majority of patients with PNH still lack accurate diagnoses, let alone appropriate treatment.

We remain focused on the need to continue accelerating our multinational efforts to serve patients with PNH globally. As with PNH,m our aHUS operations are based on four strategic imperatives.

Our execution on each of these four imperatives is strongly supported by the broad label for Soliris as a treatment for all patients with aHUS without limitations based on age, clinical profiles, identifiable genetic mutations or past histories of supportive care.

As with PNH, the new aHUS label includes compelling clinical data and life-transforming benefits to patients. Our first imperative is to help physicians identify patients with aHUS.

We will accomplish this through medical education based on the latest information regarding the signs and symptoms of the disease. The goal of our disease awareness initiatives is to help practitioners to rapidly and accurately diagnose their patients who are suffering with aHUS.

Second, we will educate physicians and patients on the progressive and life-threatening nature of aHUS and the compelling clinical benefits of Soliris so they can make better informed treatment decisions without delay.

Third, as we have done since 2007 on behalf of patients with PNH, we are now beginning to create access to Soliris for all patients with aHUS through the personalized patient services provided by our one-source treatment support program.

Our nurse case managers are now providing reimbursement assistance to US patients who participate in our aHUS clinical studies and we anticipate a smooth transition extending through the fourth quarter.

Finally, our four imperative is to support appropriate utilization of Soliris, helping patients maintain treatment for their lifelong genetic disease.

Turning now to our going-forward expectations with regard to aHUS, we anticipate that over time Soliris will transform the lives of increasing numbers of patients with aHUS worldwide.

However, the prevalence of aHUS appears to be lower than that of PNH. This lower prevalence likely results in part from the current natural history of aHUS whereby, unfortunately, more than half of patients not treated with Soliris are known to advance to dialysis, permanent kidney damage or die within the first year of diagnosis. This is true despite current supportive care.

The lower prevalence is reflected in the substantially lower number of patients in our clinical trials with aHUS compared to PNH. While there were 195 patients worldwide in our PNH registration studies, there were only 37 patients worldwide in our prospective aHUS studies.

Further, like in PNH, only a small minority of patients in our aHUS clinical studies are in the United States. For these reasons, we expect the use of Soliris in aHUS will grow only gradually in the early stages of our operations.

Moreover, the total combined number of patient who might receive Soliris for PNH or aHUS in any country is still expected to be small. aHUS and PNH are each so rare that together the combined population of patients is below the standard number of patients generally recognized for a single ultra rare disorder.

As we expand our capabilities to serve the PNH and aHUS communities, we will use our single experienced US field team to lead our initiatives in patient identification and disease education.

We are approaching these efforts with great urgency knowing that aHUS catastrophic clinical consequences often develop rapidly and unpredictably.

We have expanded our US team with individuals who have experience in rare disorders and relevant medical specialties. Both new and existing field representatives are now fully cross trained in both PNH and aHUS and the specific role that complement inhibition with Soliris has in each ultra rare disorder.

A great importance with an expanded field team, each member will be able to tightly manage a smaller geographic territory.

In the past, for example, many members of our field team had territories that included several states. Now, they can spend less time traveling and more time with both adult and pediatric nephrologists and hematologists.

There is an additional benefit to having a single field team focused on PNH and aHUS. Our early assessment has demonstrated that in addition to be managed by nephrologists, patients with aHUS also may be seen and managed by hematologists, thus we expect significant additional synergies and having the same field representative call on hematologists to discuss two separate ultra rare disorders in which the same underlying cause of the disease, uncontrolled complement activation, results in severe morbidities and high mortality.

Like with our field team, we will serve patients with PNH and aHUS with a single, highly-experienced on-source team of registered nurses who are now cross trained in both disorders.

As they have done since 2007 for patients with PNH, they are now prepared to support access and appropriate utilization of Soliris for patients with aHUS. The are experienced in navigating through the often complex processes to obtain reimbursement and, like with PNH, we expect that once a physician has made the decision to treat an aHUS patient with Soliris, nearly all patients will work with our case managers to obtain access to this life-transforming therapy.

As we continue to deliver on our objective to patients with PNH, we are also affirming the same objective for patients with aHUS in the United States, that every patient with aHUS who can benefit from Soliris will have access to Soliris.

While we anticipate that patients with aHUS will obtain timely and efficient access, we have also extended our patient assistance programs, which have been in place since 2007 to now include patients with aHUS who were either underinsured or have no access to insurance.

As we being to serve patients with aHUS in the United States, we are likewise on track in the EU following Friday's positive [CHMP] recommendation and we anticipate EC approval in approximately two months. This would enable us to start serving patients in the first major European countries in the first half of 2012 with additional major European countries commencing through mid-2013.

In addition, our global regulatory team expects to begin discussion with the Japanese authorities within the next several months with an eye toward outlining a regulatory pathway in Japan.

For now, of course, we are highly focused on our activities to serve patients with aHUS in the United States and the ongoing expansion of our global PNH operations and I will provide you with updates on future quarterly calls. Now, I'll turn the call over to Vikas, who will review some key financial matters. Vikas?

Vikas Sinha

Thanks, David. We are pleased to now serve patients with two different severe and ultra rare disorders, PNH and aHUS. I would like to provide some clarity regarding these two different indications.

First, I would like to discuss the continued strength in our global rollout of Soliris in PNH which resulted in Friday's upward revisions in 2011 guidance. The revision results from strong growth in our core PNH territories of US, Western Europe and Japan plus the addition of public funding for PNH in most of Canada this past summer.

Based on this performance in PNH we have raised our guidance for 2011 revenues and non-GAAP EPS. Revenue guidance has been raised from the previous range of $745 million to $755 million now to the higher range of $760 million to $768 million.

2011 guidance for non-GAAP EPS has been ranged from the previous range of $1.10 to $1.15 now to the higher range of $1.15 to $1.20. Other items of guidance including non-GAAP operating expenses are unchanged from our July guidance.

Turning now to aHUS, I would like to point out that the impact of our new aHUS indication is expected to be modest in 2011 and is included in our 2011 guidance. I note that aHUS and PNH has [disting dynamex] for dosing. I'll remind you that unlike PNH, aHUS is a disease in which dosing will be based on the patient's weight.

Currently, we anticipate that a significant proportion of patients will be children or infants where the recommended dose is substantially lower than the dose for adults.

As Lenny and David noted, we expect that in the initial stages of our aHUS operations, the use of Soliris in aHUS will at first grow only gradually and, thus, incremental quarterly contributions from aHUS will increase slowly.

Longer term, of course, we expect to serve increasing number of patients with aHUS over time, as a global rollout in the new indication proceeds.

As in PNH, we are committed to serving patients with aHUS in more countries around the world in the months and years ahead. At this point, I'll turn the call back to Lenny. Lenny?

Leonard Bell

Thanks, Vikas. With our second FDA approval, we are now focused on a new mission of enormous personal significance to everyone here at Alexion. During the past several years, the aHUS community has shared many heartbreaking stories with us.

Before today, too many patients, including children, have been identified too late, provided ineffective supportive care and have suffered through severe and life threatening morbidities.

We are inspired by the tremendous dedication and courage of patients, families, researchers and physicians coping with this disease. As we join these remarkable individuals and their efforts, our commitment is to work diligently and rapidly to make life changing, positive outcomes the new standard for every patient with aHUS.

We will now be happy to take questions regarding the FDA approval in the United States or the positive opinion from the [CHMP]. Operator?

Question-and-Answer Session


(Operator Instructions). Your first question comes from the line of Salveen Richter - Collins Stewart.

Salveen Richter - Collins Stewart

Just wanted to know whether the patients in the pediatric and adult safety studies will continue to be monitored for the full two years or whether they'll be switched to commercial patients at 26 weeks?

Leonard Bell


Stephen Squinto

Yes, no, I think after the 26 week period we'd expect them to be converted over to commercial on a country-by-country basis as we obtain reimbursement first in those countries. So as we point out, some countries in Europe that won't be until mid or the latter part of 2013.

Salveen Richter - Collins Stewart

Can you just maybe elaborate on the break out on these trials of patients in the US versus Europe?

[Leonard Bell]

Generally, I think we've commented before that like in PNH probably about only a quarter or maybe even less of the patients in the trials are in the United States and the balance are in Europe.


Your next question comes from the line of Sapna Srivastava - Goldman Sachs.

Sapna Srivastava - Goldman Sachs

Just a quick question; one was an assignment treatment duration. I mean, the expectation is going to be life long therapy. Can you help us understand that and whether that's the right assumption. Also just some clarification; on the label it basically speaks about the prospective study where five children discontinued drug and had repercussions and four reinstated on drug.

Is the response in these patients similar who have been discontinued drug and then given retreatment as the patients who have been chronically on treatment?

Leonard Bell

So the first comment obviously is that we do currently anticipate, given our understanding of the medical community as well as the approved label in the United States and what we anticipate to be the label in Europe, chronic lifelong treatment.

The reason for that is that increasingly I think people are understanding -- and the labels reflect this -- that a patient of uncontrolled complement activation due to genetic mutations, irrespective of whether the mutations have been identified or not.

Additionally, I just would comment that in the US approved label and we anticipate this being very similar if not even stronger, for example, in Europe, the -- it does actually cite that patients who were inadvertently or not inadvertently discontinued therapy had very severe medical complications related to their TMA.

I think that the label doesn't really say there were five children. It says prospective versus retrospective, so it could have been adults as well, as you're pointing out. I think that the -- four of those individuals had drug restarted. One individual as I recall, unfortunately, they weren't able to get there fast enough.

So I think a very -- not a potential complication but a very real complication that I think underscores actually that these individuals all have lifelong uncontrolled complement activation and, therefore, have a permanent, clear risk of severe catastrophic outcomes that could occur quite suddenly.


Your next question comes from the line of Eric Schmidt - Cowen and Company.

Eric Schmidt - Cowen and Company

Just on the strong Q3 sales performance, Vikas, is that -- you mentioned the core territories in Canada. Could you quantify how much of an impact Canada may have had? Secondly, could you address whether you'll be breaking out sales in the two indications on a financial basis going forward?

Leonard Bell

I would actually, again, just [look at] your very short comment regarding our guidance because we'd really like this to focus on the going-forward clinical impact on patients. Vikas, do you want to respond?

Vikas Sinha

Yes, so Canada had a small impact but I think the guidance change is primarily focused on all three territories doing extremely well: US, Western Europe and Japan. What was the second question?

Leonard Bell

Do you expect to break out?

Vikas Sinha

No, we wouldn't break the aHUS and PNH indications.

Eric Schmidt - Cowen and Company

Circling that, maybe on the good news in aHUS, perhaps, Lenny, you could just -- I know you've been precommercial for some time in the US. Could you talk about what the most important thing you've learned about the marketplace is in that period of time?

Leonard Bell

So I thought I [should say] precommercial. [It was back] in like '92. But you're right. There was that interlude here of 2007 forward.

I think that there are important lessons that I'd let David describe. But some of the important lessons are that, first of all, there's very, very low awareness of aHUS just as there was of PNH throughout the medical community, almost irrespective of country or location.

Second, actually while there's very low awareness, there's probably even lower levels of understanding of that the disease is largely due to uncontrol of complement activation. I think what we learned from that as well is that because it's a blood disease where uncontrolled complement activation occurs throughout all the blood vessels and the organs that this has effects in all organs, including, as indicated in the label, in the heart and the brain as well, of course, as the kidneys.

Because of all these factors -- the genetic mutation, as Dr. [Paser] noted, of course, in his press release -- it's a chronic disease in all patients. All this type of information we understand is primarily going to be the objective from a disease education perspective. I think David, though, can provide more insight into lessons learned.

David Hallal

Just quickly, Eric, we're, as I mentioned on the call, we're running a similar playbook that we've been running for PNH since 2007. Just as Lenny indicated, because the disease is ultra rare, very few physicians are well educated about the disease. So we've spent a significant amount of time developing a disease education and awareness platform.

I think one thing that's maybe a bit different here in the early stages for aHUS versus PNH is we're dedicating more time to diagnostics earlier on making sure that physicians are well aware of the patients at higher likelihood for having the disease.

So and then I think one other thing about the marketplace, as I alluded to on the call, unlike PNH where we were primarily centered on hematologists and hematologists, oncologists patients with aHUS may be on both sides, the adult and pediatric nephrology audience as well as the adult and pediatric hematology audience.

So, yes, I think we've got some more places to go to educate the physicians earlier on here.


Your next question comes from the line of Geoff Porges - Bernstein.

Geoff Porges - Bernstein

Quickly, the question about where the patients are expected to come from, you were thinking that these patients are going to primarily be newly diagnosed patients or would you anticipate that patients who are either on plasma exchange today or on -- or actually already on dialysis might be candidates for treatment?

Then secondly, also on pricing, Lenny, could you talk a little bit about adult pricing is significantly above the PNH level? Would you expect that you're going to have to offer some concessions on pricing in Europe as you launch the product on a country-by-country basis?

Leonard Bell

David, do you want to talk about where you see patients seeking treatment?

David Hallal

Yes, so first, Geoff, we're thrilled, as we mentioned on the call, that Soliris is available for all patients with aHUS really without limitations based on age, clinical profiles, identifiable genetic mutations or past histories of supportive care.

With that said, we also highlighted that the prevalence of patients with aHUS clearly appears to be lower than that of PNH based upon one year from diagnosis half of patients either dying, advancing to permanent kidney damage or being on dialysis.

So early on we would expect that more patients would be likely newly diagnosed as they present with the early signs and symptoms of uncontrolled complement activation and thrombotic microangiopathy. We don't have necessarily that base of prevalent patients that we had.

Leonard Bell

So clearly what we don't really see then is any -- as we did see in PNH, we don't see any likelihood or any possibility of what others would describe as pent up demand where there's a large number of patients waiting to obtain treatment. Unfortunately, given the severity of the disease, that's not an option, which makes us all the more urgent in our actions.

In regard to your questions, Geoff, regarding reimbursement, I guess the point I'd make and reiterate one of David's points he made earlier in the call is that those PNH and aHUS are devastating and life threatening with very near-term mortality but also ultra rare, extremely rare, very rare disorders where even as David described when one combines the number of patients potentially it's still -- even that combined number is ultra rare, very rare, extremely rare, however you want to address it.

I also want to point out that there's a very compelling view, certainly in the United States in regard to our label as recognizing right away that it's a life threatening disease and ultra rare disease and we anticipate a very similar recognition in our label in Europe as well.

Last, I would describe actually that certainly overall from a government's perspective, we know each and every government around the world is keenly interested in certainly helping those amongst us who, over the longest term, benefit potentially from early intervention in a genetic disease. Of course, that would be children who also fortunately would require a much less treatment in terms of the amount.

The duration would be long, of course, and that would also provide to the governments around the world that additional benefit of saving children's long-term lives for what obviously on a per-patient basis would be substantially less than would be required to save the life long term for an adult.


Your next question comes from the line of Robyn Karnauskas - Deutsche Bank.

Robin Karnauskas - Deutsche Bank

So, Vikas, you mentioned that the dosing in children is much less. I was wondering if you could help us understand, since they may be a majority of the original patients how much lower the dosing is in pediatrics.

Then second, are there any specific countries in Europe that have a higher incidence of aHUS?

Leonard Bell

I guess the first point that we'd make actually is that for children compared to for adults with aHUS, the dosing, as you point out, could really be markedly less and for the smallest children, for example, the dosing really would be less than one-quarter of the adult dose. It's probably, quite frankly, around a fifth or less even.

So it's markedly lower and that would then reflect for the smallest children. It would reflect a vial consumption and requirements that would be 75% to 80%, 85% reduced from that of a typical adults with aHUS.

Certainly, as regards to comparison to PNH, the children also would require somewhere between a third to a quarter of the adult PNH test, so, therefore, indicating again substantially fewer vials that would be required to support the smallest among us of babies and infants and toddlers for this life threatening disease.

Robin Karnauskas - Deutsche Bank

You said that most would be children, correct? Have you -- this is for modeling reasons. Help us understand maybe how to think about it like our [inaudible] ends around the percentage of early patients that might be kids.

Leonard Bell

Yes, we actually really didn't say. We actually think that a significant proportion of the patients will be children. Certainly that's not true in PNH. It's not significant at all in terms of the number of patients for children who are afflicted with PNH.

But we haven't really provided guidance there but we are drawing your attention to it since it'll help you understand that a significant proportion of patients require 80% less vials. That's probably important to consider.


Your next question comes from the line of Ian Somaiya - Piper Jaffray.

Matthew Luchini - Piper Jaffray

First, I was wondering if you could comment on the measure of efficacy you were able to provide to the FDA and EMA to gain approval for pediatriac aHUS. Relatedly, how many of the patients are still alive?

Leonard Bell

Is there a part D or E or F or that's it? I just want to make sure we understand where we're going here.

Matthew Luchini - Piper Jaffray

That’s it for that and then I just had a question as it relates to the presence in Europe.

Leonard Bell

Yes, so actually it's important to note that in the package insert, first of all, the indication statement clearly identified that the effectiveness of Soliris is based upon the inhibition of TMA and the improvement in kidney function. So that's an extremely important guidance for what one can anticipate.

Second, as you point out, in several positions within the US package insert, it states unequivocally that there are similar benefits and similar safety and efficacy in [children] and adults and certainly the results that were obtained and in the package insert indicate those improvements, again, as reflected by the FDA's own press releases.

Those benefits really impact reduction in TMA as well as long-term, sustained improvements in kidney function. Then, finally, the answer to your question is, yes, all patients who were in the clinical trials and particularly including all pediatric patients have remained alive with treatment.

Matthew Luchini - Piper Jaffray

My second question, going back to what you were talking about earlier with the rollout in Europe through the beginning of 2013, can you provide any update on the number of countries where you've established a sales organization and just give us some color, compare that to the number of countries where you're actually selling Soliris?

Leonard Bell

Go ahead, David.

David Hallal

I think just as we've done in the US we are assessing where the physicians are that are most likely to have experience with aHUS and we're leveraging our single sales teams that exist in all of those major countries to expand them as needed so they can support both PNH and aHUS.

Leonard Bell

So generally, to answer your question, in Europe, virtually all the countries where we're selling Soliris with our own sales force in place in the countries.


Your next question comes from the line of Matthew Roden - UBS.

[Andrew] - UBS

I guess my question is on the new refocusing of the sales force. Is it specifically in regard to the aHUS launch or is it related to a newer strategy in accelerating PNH sales as well?

David Hallal

So obviously our objective continues to be since the majority of patients with PNH we still are pretty confident in the countries where we've been operating the longest and the US would certainly qualify for that lack in accurate diagnosis let alone appropriate treatment.

So clearly when we look to expand the sales team we look to accelerate our PNH operations but we also feel like we've adequately expanded to also facilitate our efforts now with aHUS.


Your next question comes from the line of Howard Liang - Leerink Swan.

Jonathan Eckard - Leerink Swan

My first question was going to be on the size of the current aHUS registries, but based on your comments it doesn't seem like that would be particularly important because a lot of those patients may not be eligible for Soliris.

But have you seen changing in dynamics within the existing registries both in the US and Europe with regards to the number of patients they've been enrolling in the last say 12 months versus the prior years?

David Hallal

We have a longstanding policy that we don't comment on supposed patient registries that analyst identify since we can't really verify what's in them or what's out of them, nor can the anlyst.

Jonathan Eckard - Leerink Swan

Then second question was regarding Japan. I'm sorry if I missed it but are there any known regulatory requirements for Japanese approval that would differ for the European or American regulators with regards to this type of data package?

Leonard Bell

Well, I think for approval in Japan one needs to go through the [PMBA] and then eventually the Ministry of Health. I think there is a pretty straightforward pathway and I think as we said on the call earlier we will be meeting with the Japanese regulatory authorities later to discuss the results of our program and on the approval in the US and Europe.


Your next question comes from the line of Marko Kozul - ThinkEquity.

Marko Kozul - ThinkEquity

I was wondering if you have any estimates on the proportion or numbers of physicians in the US that might be treating both disease, PNH and aHUS.

Leonard Bell

That would be with Soliris. Is that right?

Marko Kozul - ThinkEquity

That's correct, yes.

Leonard Bell


David Hallal

Yes, so in our efforts to understand a bit more about aHUS in the US, again, I think I touched upon this on the call that we clearly have identified that hematologists as well as nephrologists may be seeing and managing patients with aHUS.

To that end, we would clearly be focused on educating those physicians on both ultra rare disorders. So we have a sense that there might be a few. Remember, these are ultra rare diseases but maybe a few are managing patients today with both diseases but not too many given the current number of patients that we would expect to be out there.


Your next question comes from the line of Lucy Lu - Citigroup.

[Brian Kwon] - Citigroup

You mentioned that probably the most [inaudible] will be most of the aHUS patients will be newly diagnosed. I was just wondering what the company plans to do for the patients who are already on dialysis or on plasma therapy.

Leondard Bell

That is an excellent question. I'd point out that as described in the label in the US as well as news from the FDA on Friday that the two trials -- there was what we call the 03 trial, which are patients who are receiving chronic plasma exchange or plasma infusion, which, of course, is in our label, and those patients also -- some of them actually were also receiving chronic dialysis.

We certainly are aware that in some of the trials as is indicated actually in our label, some of the patients actually have gone on to transplantation and received eculizumab or Soliris post transplantation.

So I think all of that information to help physicians understand the backdrop that patients with plasma exchange or plasma infusion have shown elimination of TMA with Soliris have maintained their kidney function. In addition to that, actually no new patients require dialysis as well as is pointed out in the label patients went on to receive transplantation, kidney transplantation and receive eculizumab as well.

So we think all of that information is extremely helpful and then we would anticipate physicians being aware of that.


With no further questions in the queue, I'd like to turn the conference back to Dr. Bell for any additional or closing remarks.

Leonard Bell

Thank you, Operator. Have a good rest of the day if it's still day time.


Ladies and gentlemen, that does conclude today's conference. We thank you for your participation.

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