Seeking Alpha
We cover over 5K calls/quarter
Profile| Send Message| ()  

Regeneron Pharmaceuticals, Inc. (REGN)

February 26, 2013 1:30 pm ET

Executives

Bill Sasiela

Analysts

Yaron Werber - Citigroup Inc, Research Division

Edward A. Tenthoff - Piper Jaffray Companies, Research Division

Yaron Werber - Citigroup Inc, Research Division

I think we'll go ahead and get started. Good afternoon, everybody and thanks once again for joining the Citi Global Healthcare Conference. It's a great pleasure to introduce the next dyslipidemia panel, [indiscernible] Today we have with us John Seibel, [ph] who is [indiscernible] endocrinologist and Medical Director of Health Insight. Welcome, thanks for being here. And also, Dr. Bill Sasiela, who's the program leader of the PCSK9 program of Regeneron

Thanks for coming, really appreciate it.

So really what we wanted to do is focus on we're going to spend a lot of time talking about PCSK9 therapies and then also touch on some of the recent FH drugs advancement to market, and also some of the drugs facing [indiscernible] as well. So maybe Dr. Seibel, [ph] let's start with you and give us a little bit of a sense, right now sort of on everyday practice, the LDL goal of 70, how many, what percentage of your population gets to goal? And of those who don't, why don't they reach the goal?

Unknown Executive

I think we have to clarify that a little bit. The LDL goal that we aim for in the general population is less than 100. If you've already had a coronary event or a diabetes, we will aim for 70 as our goal. And I would say not a lot of people reach that goal for various reasons. One, it's not pushed often enough that they need to get down to 70 by their physician. And 2 is that the media has put such a scare in the patients about statins that they're very fearful of taking them, even though you explain to them that the bad side effects are about 1 to 2 in a million, whereas they saves over 100,000 heart attacks a year. So it takes some effort to get people to increase their dose high enough to get the LDL down to less than 70.

Yaron Werber - Citigroup Inc, Research Division

And when you say risk, how do you guys define risk in practice to actually get patients to below 70?

Unknown Executive

Well, risk, it's in the eyes of the beholder, I guess. Your risk of having something might be quite low but if you've got it, it's 100%. And that's the whole problem. But here, when we're talking about statins, we're talking about a risk of having a serious side effect of about 1% or less and much less than 1%. Now other risk we're talking about maybe a 2%, 3%, 5% risk as far as muscle weakness might be concerned or muscle pain might be concerned. But that's a relatively low risk when you get right down to it, it's probably lower than your risk of taking an aspirin every day.

Yaron Werber - Citigroup Inc, Research Division

Okay. And so maybe just to clarify, the kind of risk I am referring to is cardiovascular risk.

Unknown Executive

Okay. The cardiovascular risk. Well, the cardiovascular risk is actually pretty high if your LDL is elevated. If we can get the LDL down, we decrease that risk significantly. But if you have diabetes, for instance, your risk is extremely high. If you've got had a previous heart attack or any cardiovascular event, your risk is extremely high.

Yaron Werber - Citigroup Inc, Research Division

So the patients who, just so we understand, the patients who you managed sort of to 100 are what, patients without a family history? I mean, just give us the profile of that patient versus the patients you manage to below 70.

Unknown Executive

Well, I think most patients that we managed with hyperlipidemia do have a family history of it, okay. It's not the familial type that we talked about but they have a family history of hyperlipidemia for various reasons. It may be related to another illness that they have, such as diabetes and which runs very heavily in their family or hypertension, or some other illness, obesity, that will contribute to the hyperlipidemia. So I would say that most of the patients have some family history of it. There's a few that don't. I guess, sometimes they're a little bit more scary than the others because it seems that those are the ones who start to do everything right and then go on and have a heart attack anyway.

Unknown Analyst

When you have your patients either the 100 or 70 and you said a large proportion of them, at least the 70, the ones with the goal of 70, don't reach because the doctors don't push, how many don't reach because if the drugs are able, either they're not able to tolerate them. So if we break down out of the 100% who don't reach their goal, how many are close and not close enough? How many are kind of intermediate, and how many are just not even close to being there?

Unknown Executive

Well, first of all, I think the goal of 100 is not that hard to achieve. And we can usually achieve that. And that's the majority of patients. The goal of 70 is a little harder to achieve. But we are able to get that if we can push the drugs in most cases. The problem is that we might have 5% to 7% of people who are, have some problem with statins such as muscle pain, and they don't want to take it any longer. The percentage that get close, it's hard to say, especially on the 70. I would say we probably only get about 30%, 40% down to 70%, and then the rest are close to it if we get them there.

Yaron Werber - Citigroup Inc, Research Division

So there's not a huge portion that aren't even close to where they would be where you probably just stick them with their current therapy if they're at 90, 95, but if they're still, like, 120, 150 even on a statin, tolerating it that, that patient population that you see is not very large.

Unknown Executive

Yes. Certainly, we're going to push that group as hard as we can. But even the 90, 95, we really try to get them all the way down to 70, if they've had a previous event.

Yaron Werber - Citigroup Inc, Research Division

Okay. Just so I understand, so you said that 30% or 40% of the patients you want to get to 70 actually get to 70, only 30% to 40%.

Unknown Executive

Right. But as I said, there's a lot of reasons for that. And part of that reason is the perception that they're going to have trouble by taking more statins and they don't do it. You can prescribe it to them, and they won't do it. They'll take it part of the time, but not all the time.

Yaron Werber - Citigroup Inc, Research Division

And in addition, when you say 5% to 7%, of those 5% to 7% of statin intolerant. And then as you optimize statin therapy, what percentage of the population is actually on Zetia also?

Unknown Executive

What percentage of?

Yaron Werber - Citigroup Inc, Research Division

Zetia.

Unknown Executive

On Zetia?

Yaron Werber - Citigroup Inc, Research Division

Yes, as you try to get them to 70?

Unknown Executive

Overall, I would say very few people are on Zetia anymore, maybe 10% at the most. The problem with Zetia is that when they did the simvastatin Vytorin study and it showed no difference in the outcome, the cholesterol dropped down to very low levels, which was very good. But the outcome was the same as if they used simvastatin. And so people felt that, really, Zetia although it will lower their levels, really isn't doing much as far as health are concerned. It's more of a feel-good thing.

Yaron Werber - Citigroup Inc, Research Division

And what else are you adding? Are you adding -- are you still using niacin?

Unknown Executive

I use niacin, but very few people take them. And the reason again is they don't like the side effects of the niacin, which really are not bad side effects. But they find them annoying and they stop the niacin. I'd say very few of my patients remain on it long term.

Yaron Werber - Citigroup Inc, Research Division

So the patient that you are trying to get to below 70 is what? Maybe can you describe that patient? They're not happy with stopping at 100 and we try to get them to below 70?

Unknown Executive

The ones who doesn't get down or the one I do get down? Usually, the ones that try to get down are patients with diabetes or patients who've had a cardiovascular event, and we push fairly hard to get them down to 70. But as I said, the patient just doesn't always take the medicines the way they're supposed to.

Yaron Werber - Citigroup Inc, Research Division

With regards to how the medical community, whether it be -- well, we know what the physicians want to do. You want to push them but with regards to the payors, how do they view that population with the goal of reaching 70, if you think about previous risk? And how -- like in the risk benefit of really trying to push them to get down, now, obviously, one of the hurdles is the tolerability of the statins. Is the risk benefit of that patient population something that payors would say, "I'm going to save money if I get more people down there." Or are there events with people who are not all the way to 70 don't justify it, maybe kind of broad changes of treatment that kind of really get them down.

Unknown Executive

In my experience, the payors never think long term. They only think short term. They think of their pharmacy budget separate from everything else. It could save them money by spending a little more on the pharmacy budget, or going over the budget, but they can't do that. So they don't care. And in the long run, it ends up costing them more.

Yaron Werber - Citigroup Inc, Research Division

And you also have -- on your background, you were also Medical Director of, I guess, the local Medicare Part B program, is that correct?

Unknown Executive

Yes, Mexico Medicare Part B.

Yaron Werber - Citigroup Inc, Research Division

And I mean I guess obviously you've got some insights from the policies you probably saw or have to follow through there. Have you been seeing a change? I mean, I know you're not doing that anymore. But have you been seeing a change in how some of the payors, including Medicare, are approaching some of these with the pharmaco economic approach, or is it still kind of stumbling?

Unknown Executive

Well, I think Medicare is actually the best as far as how they approach it. I think they tend to approach, from the standpoint of, they like to cover things that have been proven effective and will benefit the patient. The problem there is everything's gone to a Medicare Part D program now, and that's carried by somebody else, whether it be a hospital or insurance company or whoever. And they don't look at it the same way. And you only have to offer 2 of that category so, they'll offer the cheapest too. And then it hardly get one of the others covered. This is another problem that we run into in trying to get somebody down to 70. We might be able to do it with a product that's a little bit more expensive than what patients may have been using, and we can't get it covered. And the patient will not put out the money for the extra cost, which sometimes can be substantial.

Yaron Werber - Citigroup Inc, Research Division

So maybe just one more question, we'd like to see, so to understand them, sorry if I'm confused. But of the patients who you want to get to 70, the patients with high risk, with a history, what percent do you actually get to 70? So imagine you have 100 patients who you want to get to 70, what percent...

Unknown Executive

.

I probably only get 30% to 40% down to 70.

Yaron Werber - Citigroup Inc, Research Division

So that's the 30% to 40% okay. So maybe a question for Bill. Let's kind of shift focus and talk about PCSK9 therapies. The magnitude of cholesterol reduction has been phenomenally good. Safety profile, nice wide therapeutic window. One of the sort of the controversial points, if it's a controversy, really has to do with now we're beginning to talk about very, very low levels of LDL. And let's say, in the clinical study, you're going to be able to, in some of the patients, you're going to be able to get to goal, to 70. So are you going to try to even get them lower, and then the question is that original ratio, the percent reduction to actually outcomes, does it still hold true when you really get into those really super low LDL levels? What is the data showing? What gives you confidence?

Bill Sasiela

Well, yes, I think there's been 3 recent study, large cardiovascular end point trials that have been done to look at the benefits of lowering LDL. And they're a nice set of studies to look at. There's Jupiter, PROVE IT and TNT. Nice because they have a different diverse set of patient populations. JUPITER was primary prevention. PROVE IT was a study in recent ACS and TNT was a more of a chronic secondary prevention patient population. In the main treatment arms, the more aggressive treatment arm, they got patients down to LDLs between 77 and 55 milligrams per deciliter in the 3 studies. So that right there gives you strong confidence that getting to those ranges is going to be beneficial. On top of that, each of those 3 studies have done post-op [ph] analysis of the achieved LDL levels within the more aggressively treated cohorts. And in every case, they've given an indication that even greater reductions in LDL will provide even greater benefits. So I think one of the best examples to look at was the JUPITER study, where they had over 4,000 of the rosuvastatin patients achieve an LDL below 50 milligrams per deciliter there was clearly a difference in the cardiovascular end point rate in those who got below 50 and those above 50 on the same drug. And of course, the placebo rate was even higher than those 2 cohorts. So all the evidence gives you indication that the -- that we can continue to push down the rates of cardiovascular disease if we can push down LDL even lower.

Yaron Werber - Citigroup Inc, Research Division

And what percentage of the patients actually got to below 50?

Bill Sasiela

Of the rosuvastatin patients, it was roughly 50%.

Yaron Werber - Citigroup Inc, Research Division

About 50%.

Bill Sasiela

But they started out very low at base line. So this is a patient population that got to -- they started off at around 108 milligram per deciliter. So it's a very aggressive trial in the sense that you took primary prevention patients who had just some risk factors for cardiovascular disease, and baseline LDL of 108, and they showed significant benefit in cardiovascular disease.

Yaron Werber - Citigroup Inc, Research Division

And then the primary prevention over on obese or ratably heavy diabetic population?

Bill Sasiela

Well, one of the key things they were selecting for was high CRP and there's very much an association between high CRP and obesity in metabolic syndromes. So I don't remember specifically, but I won't be surprised if that was over-selected for.

Yaron Werber - Citigroup Inc, Research Division

Do you recall what the tolerability was for [indiscernible] at the high dose?

Bill Sasiela

Well, this is using a monitor, I think, in that study, but I don't recall offhand. But yes, I mean, it's probably going to be typical statins in that they are going to be, as Dr. Seibel [ph] mentioned, there are those percentage of patients who get skeletal muscle adverse events, who get liver enzyme elevations, and they typically fall in the single-digit percent range, typically in the clinical trials.

Unknown Executive

As I recall the tolerability was better with rosuvastatin than it was with the other statins.

Yaron Werber - Citigroup Inc, Research Division

And then what percentage, I don't know if you recall at PROVE IT, what percentage of patients got to below 50 or below 70?

Bill Sasiela

It was a relatively smaller number. I know that the mean LDL in 12% rosuvastatin was 62 and so a little bit higher than in the JUPITER trial. And a smaller number of patients overall because it was a 4,000 patient study versus a greater 15,000 or 18,000, whatever JUPITER was. But still saw trends going down to even below 40 milligrams per deciliter, small number of patients, though.

Yaron Werber - Citigroup Inc, Research Division

And what was the, in both studies, what was the cardiovascular rate, the event rate?

Bill Sasiela

It depends obviously on what you use as your pooled primary efficacy parameter. So it's usually a composite endpoint. In the PROVE IT trial, you were dealing with ACS patients. You were talking about death MI. But even including things such as revascularization, and so the event rate there was on the order of 20%, I think 26% in the pravastatin arm. In the JUPITER arm it's primary prevention, so it was on the lower side. And obviously, they had -- they may have had a slightly different composite primary endpoint. But I want to say that they're -- maybe their rate of endpoint at the end of trial is somewhere in the 6% to 8% range.

Yaron Werber - Citigroup Inc, Research Division

Okay. And secondary prevention in TNT?

Bill Sasiela

TNT, that was probably about 2% a little bit more than 2% per year. So a 5-year trial, they're around 9%, 10% at the end of 5 years, depending on which arm you were looking at, whether it's 12 to 10 or 12 to 80.

Yaron Werber - Citigroup Inc, Research Division

So as you guys design the outcome study, the 18,000 patients, ACS outcome study, what did you use sort of your comparison to ultimately decide how to power it and decide what the event rate is going to be?

Bill Sasiela

Well, the comparison is really to standard of care. And so standard of care right now is aggressive doses of statins and so that's what we're going up against. It's basically going to be to add our PCSK9 antibodies on top of aggressive doses of statins, compare that to aggressive doses of statins alone. And have an incremental difference to push the LDLs even incrementally lower than what you can get with statins alone. And particularly focus on the patients who are above 70% with statins who can't get to goal. And see what's the difference in cardiovascular events are.

Yaron Werber - Citigroup Inc, Research Division

And the enrollment criteria is a patient who is at high risk, who's got -- and what's the cut-off in LDL? That's basically is going to be greater than 70. Despite being even on high dose?

Bill Sasiela

Correct.

Yaron Werber - Citigroup Inc, Research Division

Despite. You have to be on high dose to get in.

Bill Sasiela

Unless you can't tolerate it. Unless you show inability to tolerate statin, you can back down the dose. But yes, otherwise, you're going to be on a high dose of statins.

Yaron Werber - Citigroup Inc, Research Division

And so the data that you use then to help you power the study, I don't know if you can share with us roughly what are you expecting from the comparison arm in cardiovascular outcomes?

Bill Sasiela

Well, we've got a design paper coming out. So I don't want to steal the thunder from that. We'll talk more about what the assumptions are there, the powering, when that comes out in due time.

Yaron Werber - Citigroup Inc, Research Division

Okay. And can we talk about the data that's going to come later on this year is going to be more data from FH in the second half of this year?

Bill Sasiela

That's going to be -- the trial is going to be in a monotherapy -- some monotherapy study. It's going to be in a more general hypercholesterolemics.

Yaron Werber - Citigroup Inc, Research Division

It's going to be the 100 patients, hypercholesterolemia.

Unknown Executive

We do have Phase II data obviously in FH patients.

Yaron Werber - Citigroup Inc, Research Division

Okay. So as you look at 2013, that's the mono study would be the data coming up this year?

Unknown Executive

Correct. Second half of this year is what we're expecting for the monotherapy.

Unknown Analyst

And then what are you expecting in terms of data release next year?

Bill Sasiela

It will be over the course of 2014 in some of the other Phase 3 studies. And we haven't specifically announced or mapped out what conferences, what studies at what point.

Unknown Analyst

Okay. And the statin tolerance study, the 250-patient study, is that on track for next year?

Bill Sasiela

I can't -- again, we haven't mapped out specific studies in specific time points yet.

Unknown Analyst

Okay. Part of what I'm trying to go with this is trying to understand, I mean, ultimately, it sounds to us that you've done, I think, you've said you're going to file in '15 and be on the market in '16?

Bill Sasiela

If all-- yes. If all goes to plan that would be the idea.

Unknown Analyst

And so the thought is that the initial label, I don't know if you can help us understand, what data would you have in hand by then? Is it going to be a label including mono NHS and statin tolerance?

Bill Sasiela

The studies that we're talking about that are in the pack outside the outcomes trial, obviously, would take longer, it would be part of the package that would go in. So I mean the way you can kind of look at it and the way we've kind of organized our trials, you can think about generally 3 main populations. And it was nicely kind of laid by Dr. Seibel [ph] ahead of time. You think about your general hypercholesterolemic patient who's not at goal on a statin. And those are primarily going to be high-risk patients, simply because they have the most, having the lowest goals, have the most trouble getting to goal. We've got studies looking at the FH patient population, which obviously has a high met medical need because of the High baseline levels and they again have, they get the kitchen sink thrown at them and still have difficulty getting to goal. And then of course, the patient population and the numbers that Dr. Seibel [ph] mentioned, John, [ph] with what's out there in the literature to date around statin intolerance. So the patient populations that need a statin, but can't tolerate statins and therefore, have very little in terms of therapeutic options to get to goal because of that.

Unknown Analyst

Okay. And so how do you think about, I guess, what I was trying to understand a little bit, is there a way to get some kind of a fast track designation with FDA based on the statin intolerance, maybe based on FH that would provide for a shorter review?

Bill Sasiela

My general experience in this area is that there's a tendency not to want to give fast track for something like FH, general FH, because of the potential that it will be used beyond that, so, and we think that we can get all these trials in, in roughly the same time, and so might as well get everything at once.

Unknown Analyst

Okay. And then the -- when you guys are thinking about outcomes, I mean, did you think it's a question of is it all-in from start to finish sort of a 5-year study or...

Bill Sasiela

Roughly that, yes. I think we've projected something along those lines in terms of all-in. But obviously, it will depend on ultimately on enrollment and how the events rate accumulate. What drives the completion of any event trial is the accumulation of the primary endpoint.

Unknown Analyst

So that will be data in '17, '18 probably, on a 5-year then.

Bill Sasiela

Yes, yes.

Unknown Analyst

With regards to your outcomes trials, that's something where it was designed based on feedback with payors about what we'd be looking for? Or I mean, outcomes like with cardiovascular outcomes is usually pretty convincing end point. I guess, in the kind of your question that way back when which was -- in the patient population that's already above 70, but maybe the net differences of change in cholesterol may not be large. How or what's the analysis you can do to kind of articulate the benefits of the drug it's bringing? I mean it could just purely be -- get to correlate it with anything on the cholesterol side?

Bill Sasiela

Well, you can certainly, I mean, I'll answer it from a broader perspective. I mean, there's been a lot of really good work done by the cholesterol trial as collaboration out of Oxford that have looked at the completed statin trials. And I think they number now 26, 27 different large trials. And there's pretty solid data to show that for every millimole reduction in cholesterol, which translates to about a 38.6 milligrams per deciliter reduction, you'll see a 20% to 22% reduction in major cardiovascular events. So that's probably a pretty good way in general to think about any given trial, any given therapy, a way to try to engage what to expect in terms of a relative or absolute risk reduction in any given trial with any given therapy.

Yaron Werber - Citigroup Inc, Research Division

Okay. So if you're talking about, let's say, let's go back to the outcome study. So if you’re starting with let’s say with 100, actually 70, I guess the question is, let's say, you can -- the question is, I guess, with that kind of a level do you have any sense as to what kind of cholesterol reduction you can achieve?

Bill Sasiela

Well, I mean, obviously, everybody's in the minimum, they're going to be at 70. Obviously, they will be 70 and above. If you look at other patient populations like that, you get a sense of the baseline LDL maybe in the 90 plus milligram per deciliter range. And then you can apply reductions with statins and with our PCSK9 monoclonal antibody, are baseline independent, which means the percent reductions are going to be the same regardless of what the starting LDL is. So you can get a sense of what to expect.

Yaron Werber - Citigroup Inc, Research Division

Can you just remind us in the Phase II, what was sort of the average cholesterol reduction?

Bill Sasiela

In the Phase II, obviously dose dependent. And it ranged from roughly a 40% reduction with a 50-milligram dose administered every 2 weeks, up to roughly 68% to 70% plus reduction with our 150-milligram dose every 2 weeks.

Yaron Werber - Citigroup Inc, Research Division

And the dose you're taking forward is...

Bill Sasiela

Two doses, 75 milligrams every 2 weeks and the 150 every 2 weeks.

Yaron Werber - Citigroup Inc, Research Division

Okay. So just -- I mean, if you put it together, it sounds like you're going to be looking for roughly a 50% to 70% reduction likely, of Starting base is less than 90 to 100. These are going to get pretty material. So if the historical data holds true, we should be looking at maybe to upwards of 30% decrease in event rate?

Bill Sasiela

You'd have to go back and do the calculations, it's probably not so straightforward. I think, again, when we -- we'll give a sense of what everything might look like in the design paper.

Yaron Werber - Citigroup Inc, Research Division

Okay. The question, the 18,000 patients, I mean, where I'm going with this is the trial looks like it's -- obviously, it's highly powered, but the question is it just absolutely massively overpowered just given the reduction in cholesterol? You need 18,000 patients [indiscernible]

Bill Sasiela

Again, I think with our design paper we'll come out with the specifics on this. But I would call it conservatively powered..

Yaron Werber - Citigroup Inc, Research Division

Okay. And maybe, Dr. Seibel, [ph] just 2 questions for you. When the data comes out and when the drug gets to the market, like in 2016, at that point, you're not going to have outcomes. I mean, you're going to have data on patients with higher risk because you mentioned data with statin intolerant, which is 5% of the market, but what I'm trying to go with this is, do you think the clinicians are going to want see outcomes to really use it broadly? Or is LDL reduction going to be enough?

Unknown Executive

;o

I think that the outcomes are probably the most important thing. And if the outcomes are very good and physicians are going to use it very broadly. The big problem that we're going to be running into is we now have a situation with the Obama Care, where everything has a computer that has to meet meaningful news and on there, when using pathways as you go through it, and when you get to drugs, it runs you through the drug but the generics first. And then you get the drug. So if you're going along the pathway and you choose a different course, you have to justify why. If you're using this already tell me they can cut down their efficiency as much as 1/3, so they're seeing less patients, which has cost you, now of course, more money. Then we also have the idea of bundling payment. And you've got ACOs you have to deal with it, as well as managed care and everybody else. And in that, you're going to be one payment made for a disease process. And again, you're looking at you're bound to be at the cheapest medical care possible. And that's what we're looking at in the future. So I think it's going to be much more difficult to sell your medicines. Right now, many medicines doctors don't know even know about because they have no exposure to them. Patients definitely don't know about them because they don't have any exposure. It's not in their formulary where they work. And we're going to see more and more of this.

Unknown Analyst

Ted, you've got a question?

Edward A. Tenthoff - Piper Jaffray Companies, Research Division

I'll just shout. Two questions on the PCSK9. Firstly, in the trial, what kind of dropout rates are you assuming, given the need for chronic injection? So what are you factoring in, a 50% dropout at 12 months? Just trying to fit it on top of Yaron's question about the size of it. And then second, to what extent is the negative [indiscernible] data? And we'll see what the outcome in PROVE IT is, but to what extent if PROVE IT is also negative, does that damage the surrogacy of LDL and therefore the FDA may actually require outcomes in order to approve the agent full stop.

Bill Sasiela

So with regards to the discontinuation rate, we do obviously calculate that into any sort of our trials and our assumptions. Off the top of my head, I don't recall whether there's anything we -- what we've built in for the outcomes trial in terms of that. But we do build that in for every trial. Just to add to that, I would say that our compliance and our adherence to therapy in Phase II and in some of the open label extension trials that obviously go out longer, have been quite well. So I think, even though this is an injectable therapy, which is relatively new to this whole area has been very well received. In regards to the impact of some of the recent or future trials, in regards to the class and the perception on LDL, [indiscernible], I don't think see there being too much -- we'll have to see, obviously, how the data pans out with the HPS2-THRIVE, which should be announced in a couple of weeks in the American College of Cardiology. I think the perception around that is going to be more towards HDL, which has been taking some hits lately in terms of both the 2 niacin trials, AIM-HIGH and this one, CATT trials. So I don't expect that to have much of an impact on the perception around LDL. The improvement trial potentially could, and it's going to depend upon the outcome and what we see and what the differential is in LDL. I think part of the problem that they're going to wrestle with the improvement trial potentially is what kind of LDL differential, what it was between the 2 treatment arms. The treatment arm allowed the possibility, for example, for titration between symba 40 and symba 80, the higher dose of symba, which could narrow the treatment gap in LDL.

Edward A. Tenthoff - Piper Jaffray Companies, Research Division

So just to clarify. I'm not sure I understand why the [indiscernible] data is irrelevant. Yes, I know there's an impact on HDL, but also, those could be a negative impact on LDL. And it didn't translate into an outcome. So why is it less relevant for you? I mean, obviously, it's a multifactorial impact but it still is an LDL impact there.

Bill Sasiela

Yes, I'll be curious to see what the LDL differential tends to be. With that dose of niacin, it tends to be fairly small. And maybe just a matter if you're not able to pick up the differential in terms of having an impact on events. Some of these -- the moderate to higher doses of niacin, you may only be talking about a 10%, 15% difference in LDL, which may translate to a single-digit difference or low double-digit difference absolute in LDL. So it could be a very small difference that it could just be very difficult to see, and that may be the same issue that's facing the PROVE IT trial. You've got a drug that only provides 15% to 20% reduction in LDL by itself. If you've got any differential titration statins between the 2 arms that will narrow that difference, you're starting at an LDL that's below 100, so the absolute difference gets even smaller. And I think that maybe, if we see anything neutral come out of the improvement trial, that may be some of the reasons behind it.

Unknown Analyst

So Dr. Seibel, [ph] I guess, there's been some new development in the familial hypocholesterolemia, I mean, we know that the market here is very select. But based on what you know about the new agents, are there any in particular that you find, either the efficacy of the profile to be interesting, somewhat problematic, challenging? Or is it just a high need, and we just need more drugs in the space?

Unknown Executive

Well, I think they're all interesting, new mechanisms of [indiscernible]

Bill Sasiela

[indiscernible]

Unknown Executive

Yes. It looks very interesting and seems to really do a good job. The problem is that it's going to be on a room protocol, and that's going to slow down, the slow growth of products that's only used in about 200,000 people in the United States. When physicians see that, they tend to back off a little bit because they're worried about themselves getting sued somewhere along the line.

Yaron Werber - Citigroup Inc, Research Division

And I know it's kind of off-topic, but with regards to some of the omega-3s, so I know this is for triglycerides, do you fold this into your current therapy for, I don't know what percentage of patients, and with regards to Lovaza versus the new approved placebo what do you see is the biggest difference between the 2?

Unknown Executive

Well, I do use fish oil, even though recent studies tend to cast doubt on whether or not they actually prevent any events. Lovaza we try to use quite often, but usually it's turned down by the managed care company because it's too expensive. And I just tell the patient go buy fish oil. So that's what I say, if I could treat patients with everything I wanted, I wouldn't have 30% to 40% down at 70, and if 70% to 80% down at 70%. But I can't treat them the way I want to because the managed care won't pay for it. But -- the newer drugs is coming on the market to decrease triglycerides really look good and some very interesting, but again, they're going to have to have some outcome studies to show that they really do something, especially after the recent fish oil outcome.

Yaron Werber - Citigroup Inc, Research Division

So I think those trials are underway. But if there was an outcome measure that did have a meaningful benefit and some aspects of events, cardiovascular or stroke or even sometimes, the cross formation, fish oil seem to have effect on multiple different things in the blood. Is that something that, really, you think would change the landscape of how people think about these agents?

Unknown Executive

Well, fish oil is difficult to get patients to take on a regular basis. And some of these new drugs that come out, I think, would be easier to get them to take if we can get them covered. And I think it will also add added hope to physicians that this is going to be a much more powerful medication than just fish oil.

Yaron Werber - Citigroup Inc, Research Division

Very good. And do you have any question? Well, I was just going to say, is there anything else that you've seen? I mean, of course, nothing is not in the public domain or any kind of mechanisms that you're particularly attracted with? Or is there anything kind of emerging in Europe you're excited about?

Unknown Executive

Nothing other than what we've talked about.

Yaron Werber - Citigroup Inc, Research Division

All right. Well, that's good enough.

Unknown Executive

They are exciting.

Yaron Werber - Citigroup Inc, Research Division

Very good. Well, thank you very much. And then thank you to everybody for participating.

Unknown Analyst

Great. Thanks so much for coming. We appreciate it. Thank you.

Copyright policy: All transcripts on this site are the copyright of Seeking Alpha. However, we view them as an important resource for bloggers and journalists, and are excited to contribute to the democratization of financial information on the Internet. (Until now investors have had to pay thousands of dollars in subscription fees for transcripts.) So our reproduction policy is as follows: You may quote up to 400 words of any transcript on the condition that you attribute the transcript to Seeking Alpha and either link to the original transcript or to www.SeekingAlpha.com. All other use is prohibited.

THE INFORMATION CONTAINED HERE IS A TEXTUAL REPRESENTATION OF THE APPLICABLE COMPANY'S CONFERENCE CALL, CONFERENCE PRESENTATION OR OTHER AUDIO PRESENTATION, AND WHILE EFFORTS ARE MADE TO PROVIDE AN ACCURATE TRANSCRIPTION, THERE MAY BE MATERIAL ERRORS, OMISSIONS, OR INACCURACIES IN THE REPORTING OF THE SUBSTANCE OF THE AUDIO PRESENTATIONS. IN NO WAY DOES SEEKING ALPHA ASSUME ANY RESPONSIBILITY FOR ANY INVESTMENT OR OTHER DECISIONS MADE BASED UPON THE INFORMATION PROVIDED ON THIS WEB SITE OR IN ANY TRANSCRIPT. USERS ARE ADVISED TO REVIEW THE APPLICABLE COMPANY'S AUDIO PRESENTATION ITSELF AND THE APPLICABLE COMPANY'S SEC FILINGS BEFORE MAKING ANY INVESTMENT OR OTHER DECISIONS.

If you have any additional questions about our online transcripts, please contact us at: transcripts@seekingalpha.com. Thank you!

Source: Regeneron Pharmaceuticals' Management Presents at Citi 2013 Global Healthcare Conference (Transcript)
This Transcript
All Transcripts