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Acorda Therapeutics, Inc. (ACOR)

April 15, 2013 8:30 am ET

Executives

Jeff Macdonald

Ron Cohen - Founder, Chief Executive Officer, President and Director

Andrew R. Blight - Chief Scientific Officer

Analysts

Joel D. Sendek - Stifel, Nicolaus & Co., Inc., Research Division

Michael J. Yee - RBC Capital Markets, LLC, Research Division

Mark J. Schoenebaum - ISI Group Inc., Research Division

Christopher J. Raymond - Robert W. Baird & Co. Incorporated, Research Division

Geoffrey C. Meacham - JP Morgan Chase & Co, Research Division

Philip Nadeau - Cowen and Company, LLC, Research Division

Marko K. Kozul - Leerink Swann LLC, Research Division

Raghuram Selvaraju - Aegis Capital Corporation, Research Division

Operator

Thank you for holding. Welcome to the Acorda Therapeutics Conference Call. [Operator Instructions] Please be advised that this call is being taped at the company's request. Now I'd like to introduce your host for today's call, Jeff Macdonald, Senior Director of Corporate Communications, Acorda Therapeutics. Please go ahead.

Jeff Macdonald

Good morning, everyone, and welcome. With me today are Dr. Ron Cohen, our President and Chief Executive Officer; and Dr. Andrew Blight, our Chief Scientific Officer.

Before we begin, let me remind you that the presentation includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts regarding management's expectations, beliefs, goals, plans or prospects should be considered forward-looking. These statements are subject to risks and uncertainties that could cause actual results to differ from -- materially including our ability to successfully market and sell AMPYRA in the U.S.; third-party payers, including governmental agencies, may not reimburse the use of AMPYRA or our other products at acceptable rates, or at all, and may impose restrictive prior authorization requirements that limit or block prescription; the risks of unfavorable results from future studies on -- of AMPYRA or our other research and development programs, including Diazepam Nasal Spray or any other acquired or in-license programs; we may not be able to complete development of, obtain regulatory approval for or successfully market Diazepam Nasal Spray or other products under development; the occurrence of adverse safety events with our products, delays in obtaining or failure to obtain regulatory approval of, or successfully market FAMPYRA outside the U.S. and our dependence on our collaboration partner, Biogen Idec, in connection therewith; competition including the impact of generic competition on ZANAFLEX CAPSULES revenues; failure to protect our intellectual property or to defend against the intellectual property claims of others or to obtain third-party intellectual-property licenses needed to -- for the commercialization of our products; failure to comply with regulatory requirements could result in adverse action by regulatory agencies and the ability to obtain additional financing to support our operations. These and other risks are described in greater detail in Acorda Therapeutics' filings with the SEC. Acorda may not actually achieve the goals or plans described in its forward-looking statements, and investors should not place undue reliance on these statements. Forward-looking statements made in this presentation are made only as of the date hereof, and Acorda disclaims any intent or obligation to update any forward-looking statements as a result of developments occurring after the date of this presentation. I'll now turn the call over to our CEO, Ron Cohen

Ron Cohen

Thanks, Jeff. Good morning, everyone. This morning, we announced the top line results from 2 proof-of-concept studies, which exploit the uses of dalfampridine extended release tablets, also known as AMPYRA in treating people with respectfully, post-stroke deficits and cerebral palsy. I'm going to summarize the key findings, and then Andy Blight, our Chief Scientific Officer, will provide you with more detail and then we'll open the call for your questions. The primary endpoint of both of these studies was safety and tolerability. The safety profile in both studies was consistent with what we saw in the MS clinical trials and in our post-marketing experience in MS. We saw promising efficacy signals in the post-stroke deficits trial. I'm pleased to report that after we completed the analysis, we plan to proceed to design a clinical development program for dalfampridine ER in this patient population and to discuss this with the FDA.

With respect to the CP study, efficacy data suggested potential treatment activity on measures of walking and hand strength. However, these are still being analyzed to determine if they are sufficiently robust to warrant further clinical studies. Given that this study was smaller than in the post-stroke study with just 20 participants, we'll need to wait for these analyses before we can draw further conclusions. Once we've had a chance to fully analyze the data from both trials, we'll be disclosing additional information at medical meetings and in publications.

Now I'm going to turn the call over to Andy, so he can take us through the studies, beginning with post-stroke deficits. Andy?

Andrew R. Blight

Thanks, Ron. As a reminder, this was a double-blind randomized crossover study, in which the participants received 14 days of dalfampridine extended release, 10 milligrams twice a day and 14 days of placebo, with 1 week washout period in between, where everyone was treated with placebo. There were 2 groups, one which started on dalfampridine-ER and then went to placebo, and the second that started on placebo and then received dalfampridine-ER. This was designed as a proof-of-concept study, first to confirm that the drug is well tolerated and safe in the study population, and second, to examine the number -- a number of potential efficacy measures. The focus of the efficacy measures was on walking as measured by the Timed 25-Foot Walk, which was also the primary measurement tool used in the MS development program. In addition to walking, we also looked at other measures of upper and lower extremity function, as well as more global measures. Our initial look at the data has focus on safety and the Timed Walk data, and we will need more time to look at the full extent of the data set.

The safety findings were encouraging. We did not see any novel signals that would differ from the experience with the drug in MS clinical trials and extensive post-marketing experience. The most frequent adverse events seen at a higher frequency in the dalfampridine treatment periods were dizziness and insomnia. With respect to serious adverse events, there were 3 participants in the study who experienced a seizure. One of these was a participant who was in the placebo arm for 5 days and had not previously been exposed to dalfampridine-ER. One had been on dalfampridine for 6 days. The third had a seizure 8 days into the dalfampridine-ER period after intentionally overdosing on the study drug in what the investigator reported as a suicide attempt that followed a family tragedy. All 3 participants recovered fully from these events.

The frequency of seizure is known to be elevated in the post-stroke population. It is important to understand that the design of this crossover study was to have every patient treated with dalfampridine-ER in 1 period, and placebo in the other. The order in which they received the 2 treatments was randomly assigned. The results you're looking at compares the patients while they were on dalfampridine versus while they were on placebo, irrespective of the sequence using the full crossover design.

The overall results of the study showed that the improvement in walking speed while participants were on dalfampridine-ER was statistically greater than the improvement while on placebo. But we can also analyze the first period of treatment as though it were a simple parallel group design and look at the number of people in each treatment arm who improved by more than a certain percentage of their baseline walking speed. When we do this, we see a distribution of change in walking speed in this population that looks very similar to that seen in the MS population as you see illustrated within the AMPYRA prescribing information. Although the study was too small to show statistically -- statistical significance on this kind of measure, the trend for improvement of dalfampridine-ER, no matter what threshold percent improvement in walking speed we use, is clear.

As we mentioned in our press release this morning, participants also showed a positive change on the Functional Independence Measure or FIM scale during treatment with dalfampridine compared to placebo. This is a widely used measure of independence in daily functions. This was a small but statistically significant signal in a short duration study that we will be analyzing in more detail as we move forward.

In summary, we saw a clear signal that there was a drug-related improvement in walking speed. The safety profile was consistent with our previous experience in MS. We're in the process of fully analyzing the data set and plan to share those data with the FDA, as we develop the parameters for the next clinical trial. At this point, it's too early to provide any detail on what the next trial design might look like.

Moving on to cerebral palsy, as a reminder again, this was a double-blind randomized crossover study in which in this case, 24 participants received 7 days of dalfampridine-ER and 7 days of placebo with a 1 week washout period in between, during which everyone was given placebo. There were 2 groups, one which started on dalfampridine and then went to placebo, and the second that started on placebo and then received the dalfampridine-ER. This was designed as a proof-of-concept study, first to confirm that the drug is well tolerated and safe in this study population, and second, to examine a number of potential efficacy measures across a range of deficits on lower and upper extremity function.

With respect to safety, the findings were encouraging as again, they were consistent with what was seen in MS clinical trials and post-marketing experience. The most frequent adverse events occurring at a higher frequency in the dalfampridine treatment periods were headache, fatigue and insomnia. There were no serious adverse events reported in this study. The efficacy data suggested treatment activity on measures of walking and hand strength, but further analysis is needed to determine if these are sufficiently robust to warrant moving forward with development in this indication. We should have a clearer picture on this in the near future. Now I'll turn the call back over to Ron.

Ron Cohen

Thanks, Andy. And we're going to open the call up now for your questions. Operator?

Question-and-Answer Session

Operator

[Operator Instructions] And your first question comes from the line of Joel Sendek from Stifel.

Joel D. Sendek - Stifel, Nicolaus & Co., Inc., Research Division

The data look good. Two questions I have. I think you kind of answered but I just want to make crystal clear here. So the other endpoints like manual dexterity, motor and sensory function, you just haven't gotten around to analyzing that yet, is that correct? So we shouldn't read into whether that -- those endpoints were met or not?

Andrew R. Blight

We haven't analyzed them in great detail at this point because we really focused on the walking data in stroke. As we said, we saw some evidence of activity in the CPs trial looking at those endpoints. In stroke, we haven't really dealt in great detail into whatever signals there might be in that data.

Joel D. Sendek - Stifel, Nicolaus & Co., Inc., Research Division

Okay. So you just don't know then?

Andrew R. Blight

That is correct. Yes.

Ron Cohen

Correct.

Joel D. Sendek - Stifel, Nicolaus & Co., Inc., Research Division

Okay. And then my other question has to do with the slide you had on the Timed 25-Foot Walk results, the graph there. It looked like the speed was about double as opposed to placebo and it seems to me that was a lot better than you had, the speed improvement that you had -- the walking speed improvement that you had in MS, is that correct? Am I remembering that correctly?

Andrew R. Blight

No. It's in the same ballpark. I mean, this is obviously a small trial, and so -- to project from the individual numbers here to what we might see in a larger trial is obviously difficult, but this is not outside the range of what we would have expected in MS.

Ron Cohen

Yes. It's -- Joel, it's also worth to everyone remembering, this was a crossover design. Again, it was designed for proof-of-concept and signal detection, rather than as a robust Phase III trial, the way the MS studies work. So one of the issues that we're exploring as we continue to analyze the data, is the classic issue of floating baseline that you potentially can get in a crossover study, and that can change the relative magnitudes as well. So we're looking at all that but fundamentally, we don't think it's outside the realm of what we're used to in MS.

Operator

Your next question comes from the line of Michael Yee, RBC Capital Markets.

Michael J. Yee - RBC Capital Markets, LLC, Research Division

Two questions, one was, you look at the entire group, I guess, with the functional endpoint as well as the walking speed, can you say anything about the consistency within patients? In other words, were the people who walked the fastest, also the people who had the greatest functional scores? Have you looked into anything like that? And then I don't know how much you can say about your thinking about the next study, but why or why not would you not go into a Phase III? And can you talk a little bit about, would you design that similarly as you did the MS, is that the initial thinking?

Andrew R. Blight

Yes. We haven't gone into patient-by-patient analysis at this point but that's something that we obviously need to do it as we move forward. And in terms of future trial design, it's really too early to talk about those details, because we really have to have a full understanding and then take this to FDA and see what we'd consider to be the best way moving forward for a pivotal program.

Ron Cohen

Yes. I mean, I think the good news from our perspective is that this is something we had said prospectively when we were first launching these studies, was that we were looking for a signal and frankly, if we have seen trends, we would have been happy. The fact that we got a statistic-significant result on a couple of these outcome measures, particularly, the walking, is very encouraging to us. And we had said early on, that if we could pick one thing that could come out positive, it would be the walking measure because we have a history with it and we have a very solid grounding in what to do with that. Now having said that, it's a different indication, we still have to analyze all the data, as Andy pointed out, so it's premature to say that the study would look exactly like MS or not. We just have to get a better understanding of the full data set and then design it and then, of course, come to an agreement with the FDA.

Michael J. Yee - RBC Capital Markets, LLC, Research Division

Let me push a little harder, Ron, I mean why would you not, what will be different here than MS? I mean, why -- do you have enough patients, do you think you have the right dose?

Ron Cohen

Well, let me push back a little harder here, Mike, and say that we haven't analyzed all the data, and as scientists -- what got us here is, I think, a ruthless adherence to analysis in science, starting with our theory and then preclinical studies, as you know. So we're not going to deviate from that now. We have to do our homework. And we have to look at all the data because putting it as simply as I can, we don't know what we don't know yet because we haven't finished the analysis. And it's possible that it could look very much like the MS study. It's possible that when we complete the analysis, we see something that might alter that a bit. So we just don't know yet.

Operator

Your next question comes from the line of Mark Schoenebaum from ISI Group.

Mark J. Schoenebaum - ISI Group Inc., Research Division

I had 8 4-part questions. The first is, can you give us, and I'd like you to answer them as a responder now, can you give us the baseline walking -- the first one, can you give us the baseline walking speed so that we can better interpret Slide 7 which has the overall walking speed change from baseline, please?

Ron Cohen

Yes.

Andrew R. Blight

It's in the same ballpark as the MS trial.

Mark J. Schoenebaum - ISI Group Inc., Research Division

Roughly the same as because the only thing [indiscernible] was in one of the pivotal trials is, between -- it was 2.28 in the placebo arm, and 2.21 for dalfampridine in the 204 trial, is that a rough proxy we should be using here, too?

Ron Cohen

It's roughly in that ballpark. We don't have the exact in front of us.

Mark J. Schoenebaum - ISI Group Inc., Research Division

Okay. Fair enough. And Ron, when you spoke on a call, I don't know, a couple of quarters ago, you talked about -- and I may, this is just a quote I read this morning, so I may be misquoting here or misrepresenting what you were trying to say, which is why I'm asking about it, but you mentioned something like a 20% improvement being clinically meaningful. Could you expand on that, am I right or wrong? What was the calculation you were referring to?

Ron Cohen

Yes. This is very important for everyone to understand. So the Timed 25-Foot Walk, what we actually showed in our studies, which validated what was in the literature to a certain degree is that an approximately 18% to 20% improvement in Timed 25-Foot Walk speed, correlates very well with various other measures of clinical meaningfulness, clinical improvement, so it's felt to be a clinically meaningful improvement. Now what's important to remember is that, that is not what we showed in the MS studies in direct group comparisons, which is what we have here in this crossover study. That is what we showed in Timed Walk responders as defined in the responder analysis that the FDA accepted and used to approve the drug in MS. So when you looked at the subset of actual walking responders in MS, which was somewhere between 35% and 43% depending on the study, they had an average 25% improvement in walking speed, but when you looked at the total group comparison, that dropped by 2/3, because it was being diluted by the nonresponders. So you can't -- it's not apples-to-apples when you're looking at it here. Andy, do you have anything you want to add?

Mark J. Schoenebaum - ISI Group Inc., Research Division

What is the percent here?

Andrew R. Blight

Yes. The thing is that I mean, as Ron said, the 20% is considered to be clinically significant, which is why in the label for AMPYRA, you see the improvements by percentage.

Ron Cohen

Yes.

Andrew R. Blight

It's like 10%, 20%, 30%, that from the FDA's point of view is a very important indicator of how many people are really benefiting from the treatment in this way. It's a bit complicated obviously, because there is spontaneous variability but that is the real measure of the effect.

Ron Cohen

So what you...

Andrew R. Blight

So the group differences are diluted by those who don't respond, so...

Ron Cohen

Yes. But what you want to do is then look at that, the ski slope graph that Andy showed earlier because that gives you a better handle on it. We couldn't do the type of responder analysis here that we did in the MS trials. That responder analysis required 9 different visits over 3 months so it's not possible here. But we can do the secondary responder analysis that's also seen in the label for AMPYRA. So if you take a look, you'll see that. And then that slide, we showed, which shows the average percent increase in walking speed from baseline according to threshold. So greater than 0%, greater than 10%, 20%, 30%, 40%, 50%, and what you see in that graph for this study, is that the percent of the dalfampridine group versus placebo group, who respond with greater than 0%, 10%, 20%, 30%, 40%, at every one of those thresholds is bigger than for a placebo.

Mark J. Schoenebaum - ISI Group Inc., Research Division

So when you 8% to 20% clinically meaningful, you're talking about patients who have a 20% change from baseline and those patients they're experiencing clinically meaningful improvement? You got to make sure I understand the number.

Andrew R. Blight

Yes.

Mark J. Schoenebaum - ISI Group Inc., Research Division

I'm trying to put a sentence around the number, so that I can understand it.

Ron Cohen

That's fundamentally correct.

Mark J. Schoenebaum - ISI Group Inc., Research Division

Okay. And my next question was, if you'll bear -- if you'll tolerate it, was it looks like you enrolled 83 patients but only 78, the end is 78 in the efficacy slides, what's -- who were the 5 missing? Who were the 5 possibly missing patients from the difference between those enrolled and those actually in the efficacy slides?

Andrew R. Blight

The efficacy analysis was done on a modified intent to treat which is the standard way of doing things, and that means that a patient to be included must have taken at least 1 dose of the study medication and have 1 measurement post-treatment. And so those 5 patients were patients that dropped out for 1 reason and another before they had received any post-treatment measurement. So there's no data to work from for [indiscernible]

Mark J. Schoenebaum - ISI Group Inc., Research Division

Okay. So you just -- those 5 patients got no drug? Okay, fair enough.

Operator

Your next question comes from the line of Chris Raymond, Robert Baird.

Christopher J. Raymond - Robert W. Baird & Co. Incorporated, Research Division

Just a couple of questions. So also along the lines of patient numbers, and I guess, I don't know if I was maybe not paying attention but I think just a couple of months ago, you guys had 66 as the end in this trial, and just kind of wondering at what point did you decide to upsize it and what was the -- why was that decision taken?

Ron Cohen

Well, there are a couple of things to say about that. And/or -- we wanted 66, and we wanted them in a particular distribution. So we wanted enough so that in the first half of the study, we could analyze the study as a parallel group study with a total of 66 completers of the study, okay? So obviously, we had to account for dropouts as the study went on, so we had to enroll more. In addition, because of the way the randomization works is not perfect, in order to ensure that we had a certain number of dalfampridine takers in the first part versus placebo, we also had to take more in just to account for the randomization. And then the third element is, you never hit it or rarely do you hit it exactly because by that time you realize you're there and you tell the centers, "Okay, stop." You generally will have some additional patients in the queue, and you don't cut them off at that point. So you take all that together and that's why there was a bit of an overshoot.

Christopher J. Raymond - Robert W. Baird & Co. Incorporated, Research Division

Okay. And also just -- and I'm sorry if I'm perhaps displaying my ignorance here, but can you just talk about the -- in terms of the comparison, are patients measured at baseline -- is the comparison in the baseline from when they enroll in the trial or is it from when they cross over to the drug? So I guess, where I'm getting at in this question is if 2/3 of the patients started on placebo and then crossed to the drug, is there a provision in there to ensure that there's no training effect? And I guess, that you answer that if basically, if the baseline is from the time that they start the drug?

Andrew R. Blight

Yes. This is one of the issues with crossover designs and one of the reasons they have obviously, mixed blessing is that you do have the potential for baselines to change between 2 periods. And that's one of the reasons also that we looked at the first period by itself, because that's more like a straightforward parallel group design. So in this study, we used the baseline prior to the period of treatment. So the first baseline for the first period was at the beginning of the study but the baseline for the second period was the measurement before they were put on whatever they were on in the second period. And that means that you have the potential for things like training effects, carryover effects to affect that second period, so that second period data is never as reliable, particularly for the group that was on drugs first, because you can have carryover effects. So that's what comes with the territory with the crossover design.

Operator

The next question comes from the line of Geoff Meacham, JP Morgan.

Geoffrey C. Meacham - JP Morgan Chase & Co, Research Division

One on the CP side and then I've got a couple on stroke. So do you guys feel like the AMPYRA exposure was enough to see a signal? I don't know if you correlated efficacy with PK in the study.

Andrew R. Blight

That's one of the things we have yet to do, is to look at the relationship patient-by-patient with exposure, but we don't have any data on that at this point. But I mean, these trials we're using, the current approved dose for MS, that's the 10 milligrams twice-a-day.

Geoffrey C. Meacham - JP Morgan Chase & Co, Research Division

Got you. But does the 7-day or the 1 week of exposure, do you feel like that may not be enough relative to some earlier studies in MS or even the post-stroke study?

Ron Cohen

It's one of the things we are looking at as we analyze the whole data set. Obviously, we had 2 weeks in the post-stroke trial and we had 1 week on treatment in the CP study, so that is also something we need to look at.

Geoffrey C. Meacham - JP Morgan Chase & Co, Research Division

Okay. And then on the post-stroke side, when you look at -- what you guys have done in MS after the Timed 25-Foot Walk test, you later showed some data on the -- for a 6-minute walk distance, do you feel like that would be in future studies for a post-stroke? In the population that you're studying, could that be a reasonable endpoint or do you feel like the Timed 25-Foot Walk test would be the best proxy?

Ron Cohen

You should just come and join us here in our conversations, because you're right on the same issues that we've been talking about. Yes, so.

Geoffrey C. Meacham - JP Morgan Chase & Co, Research Division

Invitation accepted, Ron.

Ron Cohen

We don't pay as well here. It's a very good question that we are already beginning to think about. The 6-minute walk data in the low-dose, high-dose study from the MS trial was quite impressive. That was in MS, of course, and we have no data in stroke but given these results, it's something that we would have to consider among a number of ways to go.

Geoffrey C. Meacham - JP Morgan Chase & Co, Research Division

Okay. And then the final one would be when you look at the trial population in the post-stroke population, obviously, there looked like there is some confounding issues on the seizure side, but what is your assessment about how frequent this naturally occurs in this population that you guys have segmented?

Ron Cohen

The literature does show an elevated risk of seizure in people who are post-stroke. We've seen the published reports showing a few percent of people post-stroke have seizures within a year and/or -- and 5 years after their strokes. So there is an elevated risk and obviously, you mitigate that as you do in MS as much as you can. One of the reasons that we didn't take people within the first 6 months of stroke was not just for the efficacy issues but also the safety issues, because the incidents of seizure declines markedly 6 months and beyond after stroke. So there are ways to mitigate that. In this study, clearly, we had -- if you exclude the suicide attempt, which was 14 pills, it actually was remarkable that, that patient did as well as they did, given the massive overdose. They had a seizure and then recovered fully. But if you'd look exclusive of the deliberate overdose, you had an equal number in placebo and drug, 1 in the placebo, 1 in the drug group.

Operator

The next question comes from the line of Phil Nadeau from Cowen.

Philip Nadeau - Cowen and Company, LLC, Research Division

Ron, was there a baseline requirement for walking in upper or lower bound in Timed 25-Foot Walk in [indiscernible] to the study?

Andrew R. Blight

Well, there was no quantitative bound, no. The patients just have to show a reasonable amount of disability that would allow them to be -- to show some potential benefit of the trial.

Philip Nadeau - Cowen and Company, LLC, Research Division

Okay. And just so I can understand Slide 8 a bit more, are you -- is the reason that there's 52 patients on placebo and 26 on drug, in that slide because this was just a period 1 data, is that correct?

Andrew R. Blight

Yes, correct. That's right. And there was an unequal randomization in order to have more patients who went from placebo to drug. As I said, if you go from drug to placebo, you have the potential issue of carryover effect.

Philip Nadeau - Cowen and Company, LLC, Research Division

And so do you -- understanding that there's maybe some confounding features in the period 2 data, do you have the similar numbers for period 2 that you can share with us?

Andrew R. Blight

We don't have them here today, no.

Philip Nadeau - Cowen and Company, LLC, Research Division

Okay. And then you guys have had a lot of discussions with the FDA about responder analysis and what the FDA thinks is meaningful or could support approval. Here in this Slide 8, you show about a 22% of patients have a greater than 20% improvement when on drug compared to 10% of placebo patients. Do you have a sense of what magnitude in responders the FDA is looking for or thinks is approval, is like a doubling kind of based on your MS experience, reasonable or just maybe [indiscernible]

Ron Cohen

Yes, so first of all, I think this calls for maybe, a reframing of how people are looking at these data. The first point and the most important one is, this is a very small study, so it's very hard to extrapolate from this to what we might see in a larger study and also, a study of longer duration. So the fact that we see this is very encouraging, particularly considering that it is such a small number of patients for a short period of time and you still see a pattern that looks very similar to what we saw in MS. But it's not really possible to extrapolate from the specific quantities that you see in the graph. Now having said that, at the end of the day, our belief is that if you have significantly more responders in the drug group than you do in the placebo group, you are clearly helping some subset of patients who otherwise couldn't get help and that should be approvable.

Philip Nadeau - Cowen and Company, LLC, Research Division

Okay. That's fair enough. And my last question is, I'm a little less familiar with the stroke literature -- is there a precedent for Phase III trials for symptomatic improvement in post-stroke patients? And if so, what is the typical design that's been conducted in the past?

Ron Cohen

To our knowledge, there is no other product that's approved for that kind of an indication. And so we don't believe there is a history for that. It's very analogous to what we encountered when we first were doing MS, which is there was no drug on the market that had been approved to improve function, so we have to develop that methodology in collaboration with the FDA who turned out to be very open and flexible to coming up with logical ways of showing it. The fact that we're dealing again with walking here, in our view, is a significant positive because we're not tilling brand-new ground, we're going through -- into an area that the FDA is now quite familiar with together with us, and we believe that, that's a very good basis for the conversation.

Philip Nadeau - Cowen and Company, LLC, Research Division

Okay. The FDA division here is the same as in MS. I know I should know that but...?

Ron Cohen

Yes.

Operator

The next question comes from the line of Marko Kozul from Leerink Swann.

Marko K. Kozul - Leerink Swann LLC, Research Division

I want to ask if you can help us think about what these data suggest on how to narrow down an addressable post-stroke down-set patient population? We start with an influence of 800,000 strokes per year, what do these data suggest could be a realistic number of patients that might be treated with AMPYRA?

Ron Cohen

Yes. So Marko, the correct population to focus on is not the incidence which is 800,000 or so new cases a year, but rather, the prevalence, which is about 7 million people currently living in the U.S. post-stroke or after having had a stroke. The estimates that have been published are that about 1/2 of those, let's say 3.5 million of those, have ongoing sensory motor deficits and ambulation difficulties, specifically. So it's really about 3.5 million people, and then there is an additional increment to that every year based on the 800,000 new strokes every year, which presumably is more than the death rate in the prevalence population, but 3.5 million or so is the right number to be focusing on.

Marko K. Kozul - Leerink Swann LLC, Research Division

So Ron, in the study, it looks like you're looking at patients that are 6 months out from the original [indiscernible], so are you suggesting that the drug might possibly be used in other patients beyond 6 months? So this is why I focused on incidence rather than prevalence?

Andrew R. Blight

Yes. The 6 months was a threshold. So the patients have to be at least 6 months, but there were patients who obviously had much longer periods living with stroke out there and I'm not sure what the maximum was but it's in years, not in months. And there's no real reason based upon our understanding of mechanism to think that this is age-related.

Ron Cohen

Right. So in other words, the minimum in this population, in this study, was 6 months out. There were patients who were many, many years out.

Operator

Your next question comes from the line of Ram Selvaraju from Aegis Capital.

Raghuram Selvaraju - Aegis Capital Corporation, Research Division

Regarding the post-stroke context, can you give us an idea of -- in your discussions with the regulators so far, as such as they might be, what sort of time line might be considered appropriate for demonstrating a sustainable clinical, meaningful benefit in this population? How long would you potentially need to go, for example, in a registration? Just give us an idea of what we should be thinking about.

Andrew R. Blight

Yes, the FDA typically requires 3 months for a chronic treatment of this kind. The European regulators generally look at more like 6 months, but can be persuaded at times to go with 3 months. But FDA has really set that as a standard for chronic treatments.

Raghuram Selvaraju - Aegis Capital Corporation, Research Division

And what about long-term follow-up? Are you likely to be required to do the same sort of long-term follow-up in the post-stroke population as you did in the MS population?

Andrew R. Blight

I'm sorry, the long-term follow-up in what sense?

Raghuram Selvaraju - Aegis Capital Corporation, Research Division

In the sense of carrying on, dosing patients in the open-label setting to ascertain whether or not there's likely to be any long-term seizure risk, that is elevated upon a beyond a baseline in stroke?

Ron Cohen

Yes, Ram, we have not had those conversations yet with FDA so we just can't say. Obviously, we have a fairly large database now of extension studies in MS and post-market experience with MS. We have to discuss with them the extent to which they will take cover from that, and allow us to do post-market surveillance in stroke, or how long they would want open-label to go, we don't know that yet.

Raghuram Selvaraju - Aegis Capital Corporation, Research Division

Okay. And then the last question is from a practical standpoint, in the post-stroke population, which obviously as you said earlier, with the prevalence of 7 million people in the United States, is very large even when compared to the MS patient population for which AMPYRA is currently indicated. Can you give us an idea of practically, whether it is possible to market AMPYRA in this larger patient population using the same specialty distribution system that you currently have set up? How scalable is that, just practically speaking?

Ron Cohen

We -- the short answer is yes, we can handle it. It remains to be seen the extent to which we might have to add to our commercial infrastructure, but fundamentally, these patients are also taken care of by neurologists, and we would expect that our neurology, especially sales force, would be able to make the same calls, maybe expand their call list. I guess, this comes under the heading of, a nice problem to have.

Raghuram Selvaraju - Aegis Capital Corporation, Research Division

Yes, I would agree. And then the last question is, pertaining to what was being discussed earlier regarding the difference in this context between -- if we look at the post-stroke data and what we have achieved with AMPYRA in the MS population, where of course you used a responder analysis, whether indeed at this juncture, we can say that in the post-stroke context, there is an equal likelihood of everyone to respond to this drug and obviously, not everybody is going to have the same benefit, but we would expect to see some benefit in everybody. Is that a fair statement or not?

Ron Cohen

I'm just trying to understand, Ram, you're -- on what basis are you opining that maybe we would see an effect in everybody?

Raghuram Selvaraju - Aegis Capital Corporation, Research Division

Basically, because if -- looking at the data, I mean, you didn't do a responder analysis-based design here, correct?

Ron Cohen

Right.

Andrew R. Blight

This is a misconception that's very widespread and it's an unfortunate one, but the concept is that in MS, we had to use a responder analysis to show an effect, that is not really the case. We saw statistically significant differences between groups in MS just as we have here. The reason for looking at responders was to get a better sense of the clinical impact of the effective treatment on those patients who are really benefiting. It's very unusual for a drug to produce the same effect in everybody that it's given to. Usually most drugs are only really effective in a subpopulation of those who are treated, so I don't think we expect this to be different in this case.

Raghuram Selvaraju - Aegis Capital Corporation, Research Division

Yes. So basically, what I was asking was, do you anticipate based on the data from this admittedly small exploratory study that the average percent of people who respond to the drug is likely to be significantly higher than what you see in MS, or is it that you expect it to be about the same?

Ron Cohen

Yes. I think there's no way to know that right now based on this figure. Just you say, it's a small exploratory study and I think that's going to be beyond the ability of this study to give us the answer. We're going to have to wait for the bigger study. If you look at the sort of the ski slope graph, that might be indicative, it might not. But all we can say at this point is we would expect that a reasonable subset based on the fact we are able to show a statistically significant improvement in the timed walk in a relatively small population, we would expect that there will be a reasonably good subset who are actually responders but at this point, we can't possibly say it's equal to less than or greater than the MS population.

Raghuram Selvaraju - Aegis Capital Corporation, Research Division

And did you report, or did you measure specifically what percentage of individuals responded to the drug in this -- in the post-stroke study? And what was that number?

Andrew R. Blight

No, we had no way to determine that here because it was not based on any sort of prolonged treatment or responder analysis of the kind we used in MS. So you just have to rely on the overall appearance of the data, to say it doesn't look like it's remarkably different from what we would have expected in MS.

Operator

I would now like to turn the call over to Ron Cohen for closing remarks.

Ron Cohen

All right. Well, thank you very much everyone for joining us and we will be updating you as we get more information, and have a great week.

Operator

Thank you for your participation in today's conference. This concludes the presentation. You may now disconnect. Have a great day.

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