NATIONAL HARBOR, Md.—Nearly two thirds of patients with end-stage renal disease (ESRD) experience a drop in hemoglobin (Hb) level after hospitalization, and it may take months after hospitalization for Hb levels to recover, researchers reported at the National Kidney Foundation 2012 Spring Clinical Meetings.

Given the frequency of hospitalizations in the ESRD population and the length of time for Hb recovery, hospitalizations are significant contributing factors to Hb variability in the ESRD population,?the investigators concluded in a poster presentation.

T. Christopher Bond, PhD, of DaVita Clinical Research in Minneapolis, Minn., and colleagues reviewed data from 176,199 hospitalizations (more than 30 days since the last hospitalization) among 138,762 HD patients. The median length of stay was five days. Pre- and post-hospitalization Hb test results were available for 156,353 hospitalizations. Of these, 66.7% were associated with a decrease in Hb from a mean of 11.87 g/dL pre-hospitalization to 10.55 g/dL post-hospitalization.

The time to recovery depended on starting Hb level. Patients within the target Hb range of 10-12 prior to hospitalization recovered to pre-hospitalization levels in a median and mean of 41 and 54.3 days, respectively. Patients with pre-hospitalization Hb levels below 10 recovered in a mean and mean of 29 and 4.7 days, respectively. Those with pre-hospitalization levels above 12 recovered more slowly, with a median and mean time to recovery of 85 and 180.5 days, respectively.

"These data, and evidence that a low proportion of hospitalized [hemodialysis] patients receive ESAs [erythropoiesis-stimulating agents], point to a need for new strategies to control anemia in these patients," the authors stated.

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NATIONAL HARBOR, Md.—Patients with chronic kidney disease (CKD) before undergoing surgical procedures are at increased risk of acute kidney injury (AKI) post-operatively, researchers reported at the National Kidney Foundation Spring Clinical Meetings.

A study of 255,188 general surgical cases revealed that the proportion of patients who experienced AKI increased as renal function decreased. Post-operative AKI developed in 0.42% of patients with normal kidney function, 0.58% of those with reduced kidney function (estimated glomerular filtration rate [eGFR] of 60-89 mL/min/1.73 m2), 2.3% of those with CKD stage 3 (eGFR 30-59), and 9% of subjects with CKD stage 4 (eGFR 15-29).

Patients with CKD stage 4 had a 34-fold increased risk of acute renal failure (ARF) than patients with normal kidney function, said investigator Linda W. Moore, RD, of The Methodist Hospital in Houston, who presented study findings.

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LOWVILLE ? Lewis County General Hospital officials plan to break ground today on a long-anticipated dialysis project, although construction is not expected to begin for at least another couple of months.

?This patient-focused project further demonstrates our attention to quality care and services for patients as a closer-to-home option for area residents,? Eric R. Burch, CEO at the county-owned hospital, said in a statement. ?We wouldn?t have been able to proceed with this important project without the support of our Board of Managers, Lewis County legislators, state and local officials and the community.?

A groundbreaking ceremony is slated for 9:30 a.m. just outside the main entrance at the hospital?s North State Street campus.

The state Department of Health this year approved a certificate of need for DaVita Inc., Buffalo, to operate a dialysis clinic on the hospital.

After meeting with DaVita representatives Wednesday, hospital officials are anticipating project completion by fall 2013.

The hospital is proposing to build a $1 million, 7,200-square-foot addition off the west side of the Medical Arts Building?s first floor and basement to accommodate the dialysis center. Upon completion of the building shell, the hospital would turn it over to DaVita, which would do the interior finish work.

Hospital officials expect to iron out lease details and undertake the bidding process over the next couple of months, which would allow construction to begin by late summer. The shell is expected to take about six months to complete, with interior work to take an additional six months.

Legislators in both 2006 and 2007 supported certificate of need submissions that would have allowed Renal Care of Northern New York, Watertown, to set up a dialysis operation here, but those plans never came to fruition.

Faxton St. Luke?s Healthcare, Utica, in fall 2009 submitted another certificate of need application. However, by the time the project received state approval, Faxton St. Luke?s officials had decided not to move forward, leaving local officials searching for another partner. They found one last year in DaVita, a company based in Denver that operates similar centers throughout the country.

Up to 30 Lewis County residents receive dialysis treatments in either Watertown or Utica, Mr. Burch has said. Dialysis patients typically undergo four-hour treatments three days each week.

The groundbreaking ceremony will be followed by the hospital?s annual Community Health Awareness Day from 10 a.m. to 1 p.m.

This year?s theme is ?Celebrating Health, Healing, & Service to the Community.?

There will be more than 45 vendors, and the event will include a number of free screenings, including blood sugar, blood pressure, cholesterol, hearing and skin, breast and colon cancer. Appointments for skin cancer checks must be made by calling 376-5151.

Attendees may sign up to participate in a cancer prevention study. The event also will include sleep lab tours, chiropractic screenings and participation by various hospital, fire and police departments. Children?s activities will be offered.

Paulie?s Meat & Seafood Market will hold a chicken barbecue from 11 a.m. to 1 p.m., with a portion of the proceeds to go toward the dialysis project.

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Question-and-Answer Session

Robyn Karnauskas – Deutsche Bank

Let those of you know, we do have a new app that you can ask questions anonymously, it’s yorn.com/hc, or shoot me an e-mail if you don’t want to raise your hand. But I thought maybe first to start off – I noticed today that it was announced that you’re not going to – and you’ve mentioned previously you’re not going to go forward with dry AMD. I was just curious about the Taligen compounds that you acquired that were in development for wet AMD, whether or not that is still on the table?

Vikas Sinha

So Taligen was acquired last year – early last year. As I mentioned, Robyn, that our focus is in ultra-rare life transforming diseases, and we’re continuing to learn more about TT30 and the other compounds. As we gain more experience with the drug, we will identify the indications as we go forward. At this time, it’s too early.

Robyn Karnauskas – Deutsche Bank

Any questions? Maybe you can turn a little bit to the acquisition of Enobia and asfotase alfa? So, you have three different types of patient populations, the newborns, sort of the adolescents and the adults. And you’re known for going after the patients that are really most likely to benefit. Which population do you think faces the most challenges? Which do you have the best understanding of the disease?

Vikas Sinha

Let me go to the chart. So when you look at the infants, when you start looking at 50% of the newborn patients die within a year, that’s the area we want to go first. It’s primarily because this is a highly unmet need that we want to address first before we go into juveniles and the adults, right, because this is where the problem is the most. If we can save these people, we’ll go into juvenile and adult. So the progress – the focus will really be on the infants first and juvenile and then adults.

Robyn Karnauskas – Deutsche Bank

In the adult data, it looked the drug has a sustained and really good benefit. I was just curious though if there is a – how the genetic differences between the baby form and the infant form and the adult form impacts how well your drug will do in these patients long-term?

Vikas Sinha

The trial results are very good in the pediatric and juvenile side. The adult side, I think we’re looking at redoing it with a patient population, as you mentioned, which are very severe and go after that, so another trial will be started very soon.

Robyn Karnauskas – Deutsche Bank

And what percentage of that population is it and do you think that the larger population might still benefit?

Vikas Sinha

We don’t have any specific data on that because it is such an ultra-rare disorder. That level of data is very tough to get until you really go into the market. Our objective really is to focus on bringing these life transforming therapies to these patients. And we’ll worry about the subset and the size of the market at that time.

Robyn Karnauskas – Deutsche Bank

And what percentage of the babies treated are still alive today?

Vikas Sinha

I truly don’t know the answer. I really don’t know. I can get back to you on that.

Robyn Karnauskas – Deutsche Bank

If you ask, I can repeat the question.

Unidentified Analyst

(Inaudible) when you acquired Taligen. So what actually changed between the acquisition and today that you decided not to pursue that opportunity?

Vikas Sinha

You’re talking about Taligen, right? Nothing has changed there. It’s just that the results from this morning definitely did not complement – didn’t see any impact on the dry AMD patients. We don’t know about wet AMD. That’s what I was trying to say.

Navdeep Singh – Deutsche Bank

Hey, Vikas, thanks for the presentation. Just a quick question on potential biosimilar competition, how protected do you think you are because your patent for Soliris does go away in the 2020 to 2021 timeframe? Just an overview of how protected you are.

Vikas Sinha

So, from a patent protection side, you’re right, we said that in 2020 to 2021, we’re protected. We are in environment where biosimilars are being discussed quite a lot, right, and the ability to go closer to the market and stay with the patients and bring that life transforming therapy there is what we are into it. And we haven’t talked about – much about what else is in the pipeline and you were just talking about several other compounds that we have in our pipeline which we have not fully discussed right now. It’s a bit too early.

Navdeep Singh – Deutsche Bank

So, you think that the loyalty that you’ve developed from your patient population will kind of protect you against anybody?

Vikas Sinha

Two things, there is one side is to create that loyalty in the market, second side is to continue to build a second line therapy behind that.

Navdeep Singh – Deutsche Bank

Okay.

Vikas Sinha

On the innovation front, right, because who knows about future?

Navdeep Singh – Deutsche Bank

Okay. And any update on the trial design for the myasthenia gravis studies, the pivotal studies?

Vikas Sinha

It’s still being discussed, so it’s too early to respond to that.

Navdeep Singh – Deutsche Bank

Okay.

Robyn Karnauskas – Deutsche Bank

I’ve a question on – on transplant actually. Thinrise is being developed a little bit in the same indication. I was wondering if you thought that the two products could be complementary.

Vikas Sinha

I just cannot comment on somebody else’s drug.

Robyn Karnauskas – Deutsche Bank

The next question on NMO.

Vikas Sinha

Yeah.

Robyn Karnauskas – Deutsche Bank

So a lot of our research suggests that so many patients are well controlled with RITUXAN, which is an off-label drug, which poses an interesting question. When you go in front of the FDA, you want to develop a drug for like the sickest population when a good chunk is being treated with an off-label drug, how much does that play into how you design this trial?

Vikas Sinha

That will be something you should really look at when the data comes out in Q4 this year. And to look at how many patients were on Rituximab or were not on the Rituximab.

Navdeep Singh – Deutsche Bank

Maybe a follow-up to that question. So, since the NMO trial is an open-label study, have you already made the decision to advance it into pivotal studies?

Vikas Sinha

We are keeping that channel parallel going.

Navdeep Singh – Deutsche Bank

Okay.

Vikas Sinha

As Steve had mentioned in the earnings call, we are talking to several physicians already – investigators already to start building a multinational trial.

Navdeep Singh – Deutsche Bank

Okay.

Vikas Sinha

Multi-center trial, because this one is specific to one center only.

Navdeep Singh – Deutsche Bank

I guess you’re committed to NMO going forward.

Vikas Sinha

Yeah, we just don’t want to lose the time.

Navdeep Singh – Deutsche Bank

Got it. Any other questions in the audience?

Robyn Karnauskas – Deutsche Bank

I have a quick financial question for you. Take a break from the clinical. But then we’ll go back to the clinical. So, everyone wants to be like Alexion. There is – countless numbers of companies said we want to be like Alexion in that you always seem to surprise investors and really manage the Street very well. So the question is as your company – as you start really penetrating new markets and you become a more mature company, how might your strategies change? How do you envision the company changing and what you communicate to the Street regarding your product?

Vikas Sinha

If I understand, your question is how would you manage going forward a multi-product, multi-indication company. That’s your question, right? Yeah, a very key role that we look at in managing our businesses, there is a view that one takes of the future, but you have to also consider the risk and opportunities that are around that opportunity or risk, right? And leveraging that risk and opportunity and aggressively managing that day-by-day, quarter-by-quarter is how we run the business.

And – it’s easier to do it in one indication, going into two, that was one of our biggest worries in Q4 was, when we expand our business in U.S. when we launch it, when you launch something new, people try to drop past one, right? So that was our big fear and that was very important for us in Q4, to how we structure ourselves, how we drive our business, that both the indications need to move forward. And that was a very big feeling internally between Q4 and Q1 that we saw both PNH and aHUS, both progressing with the – with PNH growth not slowing down, right? And, now replicating that into multiple indications and multiple drug, it’s all about how you manage the talent with the right level of objectives. And that’s something we’re tremendously focused on and building the talent level in the company.

Robyn Karnauskas – Deutsche Bank

And so when you think about reporting at some point you think you’ll break out aHUS with PNH and if so like how many years?

Vikas Sinha

I don’t think that is the intention right now, but we will see where the future takes us.

Robyn Karnauskas – Deutsche Bank

Okay. That’s helpful. And then also thinking about strategy, so one of the biggest fears in the investors is that they’ll wake up in the morning and price will be slashed for Soliris in Europe because Spain goes out of business, something like that. So, maybe, you can help us understand why you’re comfortable, and STEC-HUS, which may not be as orphan as HUS, will that influence pricing in Europe?

Vikas Sinha

So, a very good question, two questions here right. One is what do you think about the pricing, right, and future pricing, and what do you think about STEC-HUS which is a acute therapy and not a chronic therapy. The first one is pricing-wise, look, there is always pressures in pricing market. There is no question about it. What we have to really do as a company and as a strategy – I’ll just go back to the chart that I had put as a strategy of the company. So, key thing is to focus our strategy and what we know well and do well which is in devastating ultra-rare disorder, where very key part is, point number two, is to seek transformative impact on patient’s life.

The drug really has to work. If the drug doesn’t work in this market, you just can’t charge the price if it only works in 30%, 40% of the patients, right? It has to work in all the patients that take the drug and that is really key. The top two points are really key as we drive our indications and the strategy, because unless the drug makes that difference, you can’t sit across the table and ask for that premium pricing.

And going back to your STEC-HUS question, then, it’s an acute therapy, a lot in children, a lot in adult, there is a fair mix in the last – last study that have – the last crisis that happened in Germany. But a lot of other countries and then even in U.S. sometimes you see HUS as fairly a pediatric area where short-term duration, a less number of vials is not as – I wouldn’t believe that that would be as high in the pricing radar as you would imagine. Did I answer your question?

Robyn Karnauskas – Deutsche Bank

Well, sort of, so you won’t be charging as much, because your duration is very short?

Vikas Sinha

Yeah.

Robyn Karnauskas – Deutsche Bank

But the question is does it influence – these countries, you negotiate price upfront, so this drug will already be in the market. How do you negotiate like another indication that’s shorter, does it impact the price at all?

Vikas Sinha

As I mentioned, it has to be life transforming therapy. You have to be sitting across the table and talking about a life transforming therapy, when you want to charge a premium price. And Robyn, there is always a pressure, whether it’s a good time or a bad time, right? And if you go back and look at it when we launched the drug, 2007-2008, it was the biggest crisis in U.S. Now, we’re looking at Europe. So there will be some crisis somewhere else throughout the globe and we just have to learn to work around that.

Robyn Karnauskas – Deutsche Bank

The question for the webcast is, do you think you’ll have similar challenges in Brazil?

Vikas Sinha

In terms of challenges?

Unidentified Analyst

(Inaudible).

Vikas Sinha

Yeah, I wouldn’t comment country-by-country here. But there is no country you don’t have that challenge. So, whenever you have a single payer, you have to go and discuss and demonstrate the value of your drug.

Unidentified Analyst

(Inaudible) 750 million people that are left in your chart, Brazil and Latin America was about 40% of it. So it’s a significant chunk of the market that you have left to launch?

Vikas Sinha

Yes that number 295 million, if you look at it, if you add Brazil, Colombia, Argentina, and Mexico, that number will be very large, much larger than 295 million. What we tried to do was within that population base, how much are reimbursable population, that’s how that $295 million comes out. It is not the 190 million of the whole Brazilian population included there neither the full of Mexico is not included there. We’ve looked at which subsets of that population have insurance coverage.

Robyn Karnauskas – Deutsche Bank

Any last questions? Okay, great. Thank you very much.

Vikas Sinha

Thank you very much.

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