Sara Pellegrino - Director, Investor Relations John Crowley - Chairman, Chief Executive Officer Chip Baird - Chief Financial Officer Bradley Campbell - President and Chief Operating Officer Jay Barth - Chief Medical Officer Hung Do - Chief Science Officer.

Ritu Baral - Cowen Anupam Rama - JPMorgan Joseph Schwartz - Leerink Partners David Lebowitz - Janney Capital.

Good day, ladies and gentlemen and welcome to the Amicus’ Second Quarter Results Conference Call and Webcast. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. [Operator Instructions]. As a reminder, this conference call is being recorded.

I would now like to introduce your host for today’s conference, Sara Pellegrino, Director of Investor Relations. Please begin..

Good evening and thank you for joining our conference call to discuss Amicus Therapeutics’ second quarter 2015 corporate highlights program update and financial results.

Speaking on today’s call, we have John Crowley, Chairman and Chief Executive Officer; Chip Baird, Chief Financial Officer; Bradley Campbell, President and Chief Operating Officer, Dr. Jay Barth, our Chief Medical Officer and Dr. Hung Do, our Chief Science Officer.

The slide deck to accompany this call is also available on our corporate website at www.amicusrx.com in the investors section.

As a reminder, this conference call contains forward-looking statements about our future expectations regarding our business, operations and financial conditions including but not limited to, preclinical and clinical development, cash runway, regulatory timelines, the potential success of our product candidates and amenable patient population.

Actual results could differ materially from those projected in Amicus’ forward-looking statements due to numerous known and unknown risks and uncertainties, including the Risk Factors described in our Annual Report on Form 10-K for the year ended December 31, 2014, which is available from the SEC and on our website.

All forward-looking statements are qualified in their entirety by this cautionary statement and Amicus undertakes no obligation to revise or update this conference call to reflect events or circumstances after the date hereof.

At this time, it is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics..

Great. Thank you, Sara. And good evening everybody. It’s a pleasure to be here joined by our executive team. Certainly a pleasure to host this conference call coming off what was our most significant and most value creating quarter in Amicus history.

For those of you who are able to access the document that we put up online, we have some slides to reference as well. And I’ll just begin by noting some of the key achievements and program highlights from a business perspective in the last quarter. This is on slide 4 if you’re looking at the deck.

Really four things and there are many, many good things happened at Amicus in the second quarter. Four we think were particularly transformational.

First, of course we had the name accepted our trade name and announced for Galafold Migalastat hydrochloride now known as Galafold, our precision medicine personalized medicine for Fabry disease monotherapy. As you know we submitted the MAA to Europe under accelerated assessment. It was also validated by the European regulatory authorities.

We are also pleased to announce on this call that the FDA has accepted our pre-meeting NDA meeting request. And that’s now been scheduled for later in the third quarter. The briefing document for that is now complete and Jay Barth, our Chief Medical Officer will go into some more detail about that in the call.

We think that’s a great advancement here very recently for the program. And we continue to make great advancement across the global regulatory processes in other geographies as well.

And some terrific new additions to the Amicus commercial team, I’ll highlight some of those and Brad Campbell will speak to some of those as well, so, tremendous progress for Galafold in Fabry disease. Secondly our Pompe program now, very, very close to the clinic, our next generation enzyme replacement therapy regimen for Pompe disease.

We’re pleased to announce on this call that we have now completed the first campaign GMP manufacturing production run. It was very-very successful we’ll take you through some of the details both from a material quantity standpoint as well as for the quality, the very high quality of the material.

And that’s now completed its manufacturing including all the purification steps. So, we’re very pleased that that went well. Our second campaign is underway. But the first material for patients is already ready for the clinic. As well as the IND-enabling studies that Dr. Hung Do, our Chief Science Officer will comment on those through the call.

And we are also going to describe for you here today our clinical strategy as we move forward in Pompe disease, Dr. Barth, Jay will take us through that as well.

The third area to highlight from our second quarter and now as we sit here today is that we are very well capitalized to continue to build on our vision of creating one of the world’s great new biotechnology companies focused in the rare diseases.

Chip of course will take us through the financial performance in the second quarter including the $258.8 million follow-on public offering that we offered at $13.25 a share back in early June that leaves us with a very, very strong cash position.

And finally, the fourth point that I’ll highlight here as part of our success in the last quarter has been building an absolute world-class international commercial leadership team. The key elements of that leadership team are in-place and eagerly preparing for the hopeful launch of Galafold. And again, Brad will take us through more of that.

With that as my introduction, if you can, if you have the slides, you can flip forward to slide number 6. I’ll turn the call over now to Jay Barth, our Chief Medical Officer. And Jay, I’ll ask you to speak to the global regulatory strategy for Galafold..

Thanks John. Things in our regulatory strategy have really been proceeding very well, getting us closer to bringing Galafold to patients. Starting with the U.S. the FDA as John mentioned has accepted our request for the pre-NDA meeting which is scheduled. And the briefing document is finalized. And we submitted shortly to the FDA.

Important goal of the pre-NDA meeting is the discussion of the Phase IV component of our program. And the proposed plan includes a study of GI symptoms in Fabry patients as well as supportive data from the registry that we’re planning post marketing.

On the European side, the MAA is under review under accelerated assessment which of course is now shortened to timelines the review times go from 210 to 150 days. And therefore we’re expecting the CHMP opinion by the end of the year. And that’s proceeding well.

Finally, in the rest of the world, we have started the process of starting the rest of the world regulatory activities following on the MAA and the NDA submissions. So, on all fronts we are moving forward with our regulatory strategy as planned. And I’ll now turn it over to Brad, to discuss the Fabry market..

Great, thanks Jay. Good evening everybody. So, as we know on slide 7, and the enzyme replacement therapy market for the treatment of Fabry disease is a large market over $1 billion in sales in 2014, that’s based on products that are priced at about $275,000 to $300,000 per patient per year.

And we continue to see robust growth in that market fuelled primarily by an increase in diagnosis rates that continues to be shown in the United States, Western Europe and rest of the world and bringing new patients on therapy. If you look to slide 8, this really shows how we look at the Galafold commercial opportunity.

We have the schematic on the right that we’ve looked that before, which shows our three key patient segments, patients in the center there, the blue segment who are the ERT treated market that’s $1 billion in sales, which we think represents about 4,000 to 5,000 patients.

And then the next outer ring which represents the diagnosed but untreated population which is another significant population about as large as the treated patient market today, and then finally the outer ring which represents what we think is a significant under-diagnosed population or undiagnosed population, which I’ll talk through in a minute.

But importantly, we have studied both switch and naïve patients who represent a cross-section of phenotypes throughout these patient segments both males and females. And we have a range of genotype from classic to late onset.

So we think the data package that we studied with Galafold over the course of its development supports application across these patient segments.

Moving on to slide 9, as you’ll recall there have been a number of newborn screening studies over the course of the last decade, beginning in Italy moving through various geographies in Asia, in Europe and in United States, which continue to show that rather than the historic published incidents of 140,000 to 150,000 births with the GLA mutation.

These studies continue to show significantly higher portion of patients with GLA mutations looking like one in a 1,000 to one in 4,000 so almost 10 fold greater than the historical published incidents.

And even if you were to look at those what we call index patients who discovered to those higher screening populations are newborn screening populations. You may not choose to treat that patient you choose to follow them through their disease course.

But certainly based on the fact that this is an excellent disease, we’ve shown to those literatures that brings another three to five additional patients to that family screening opportunity. And what these literature reports have shown that high proportion of those patients have amendable mutations, ranging from 75% to even 100%.

So, as that market grows, as we find additional patients through newborn screening and other screening populations, we expect those to have a significant portion of amendable mutations.

Moving on to slide 10, importantly as we have generated all this data over the course of 2014, we were able to go back out to the market with an updated target product profile interacted with our key stakeholders, with physicians, with patients, with payers, and present that target product profile to get their receptivity to what the new value package that we’re bringing with Galafold would represent.

And I think in each of those key patient segments that we talked about and diagnosed in treated patients who would have to switch off of their current standard of care in diagnosed untreated patients and in new patients, physicians clearly show that a large majority of patients across those segments, 65% to 75%, based on our current target product profile, they would look to switch their patients or put their patients on to Galafold.

So, very clearly a very high and positive response to the value that we think we can bring to the market with Galafold as an oral therapeutic for Fabry patients. In a similar vein on slide 11, we go through some of the payer feedback.

And here too, I think very important recognition by the payers who said both based on the clinical trial data so that the data that we show through 011 and 012 studies, and also through the improvement in the drug administration reducing that burden of treatment going from every other weak infusions with enzyme replacement therapy to and every other day small molecule pill with Galafold.

Payers very clearly said that if you were to model a parity pricing again that $275,000 to $300,000 per patient per year, very strong support for reimbursement for Galafold. So very good feedback from both payers and physicians in terms of the target product profile and the value package that we would bring with Galafold.

So, it’s very encouraging feedback from the market. And importantly too on slide 12, as John mentioned, we continue to be impressed with the level of quality, experience and patient centric focus of our key commercial hires.

We now have leaders in place in all of our key functional areas internationally as well as our key regions who will be able to lead the launch of Galafold successfully.

And this of course builds upon the strong history that we’ve established with a very broad global footprint in terms of our clinical trial activities but also a strong history of patient advocacy, medical outreach etcetera.

So, a very strong momentum leading into the second half of this year, a very strong team in place and great feedback from the market so far. So, with that, I will turn it over to John, to walk us through the Pompe program..

Great, thank you Brad. Thanks for the great overview, both to Jay and to Brad. And hopefully you sense not only the great confidence we have in the regulatory strategy and where we are in the process, both in Europe and the United States and also looking to rest of world.

But the more and more we’ve reached out to physicians, to patient groups, the more we’ve looked at much of the work done to understand how many people are living with this very devastating disease, Fabry disease, the more we are condensed. This is indeed one of the largest human genetic disorders in the world today.

And one for which we’re very excited to have our product Galafold with the potential to offer significant potential ways to treat this disease that may be potentially differentiated from existing therapies but also to use that as the cornerstone for a broad Fabry franchise where we’re also of course building our own proprietary next-generation enzyme replacement product.

We’re not going to talk about that in this call, I would expect in the months to come we’ll have much more to say about the success we’ve had scientifically and from some early manufacturing in our next generation ERT.

But again with Galafold and the Fabry, the opportunity to help people with Fabry is something we’re very, very excited about here at Amicus. Let me now go ahead and shift to our Pompe program.

This is one as you know for several years has been in development here at Amicus, really accelerated in the fall of 2013 with the acquisition of Callidus Biopharma and Hung Do, now our Chief Science Officer is the founder of that company helping to lead that effort.

I’ll remind you on slide 14 from a disease overview standpoint this is a devastating lysosomal storage disorder, Pompe disease. There while there is an existing improved enzyme replacement product, we still think that this is a devastating disease even for patients on ERT today.

Our goal here as with all of our programs, just either be first-in-class or best-in-class. Let me describe for you a little bit more and you can reference again on the slide on slide 15. What’s the problem that we’re trying to solve for here? So, we think there are three significant deficiencies in the current enzyme therapy.

The first is activity and stability. The second relates to tolerability and immunogenicity. And the third relates uptake and targeting. Those first two we addressed with the addition of our chart platform technology which is the chaperone advanced replacement therapy.

A specific small molecule designed to bind to and stabilize our proprietary enzyme replacement therapy. We think that is shown in preclinical models and in some early human proof-of-concept studies with an existing ERT, the potential to address both of these problems.

We also know through our own design of the proprietary cell-line that we use or base enzyme therapy the ATB200, we think we have shown dramatic improvement in preclinical models in uptake and targeting with our uniquely engineered GAA cell-line that has optimized levels of Nano 6 phosphate.

And very importantly what we believe to be very appropriate levels of glycosylation and the right sugar structures and carbohydrates. This was our belief and we acquired Callidus a year and half ago.

Much of the work for the last six or seven quarters has focused on taking this base enzyme together with the addition of the small molecule chaperone and developing a drug rapidly for patients that would we believe have the potential to become best-in-class.

To do that we’ve worked with our partners at WuXi in China at designing this cell-line, and if you look on the next slide on slide 16, you can see much of the key activities over that last year and half, really culminating in the second quarter of this year as we’ve now completed the GMP run.

I can’t emphasize enough how much of our team’s effort and technical operations and in science has focused on ensuring that we not only design the right cell-lines and drug regimen but that we’re able to scale it efficiently and effectively and to have a drug product that we think is very highly differentiated.

So, with that we’ve completed of course the Master Cell Banking in 2013. Throughout 2014 we scaled from the five leader scale all the way up to the clinical production scale which is 250 leaders. Our first GMP batch again was just completed in the second quarter of this year.

And we’re very, very happy that it continued to have very high levels of Nano 6 phosphate, continued to have the right and proper glycosylation. It’s also a very high yielding cell-line. So I think in every way we achieved manufacturing success. So the material for our first clinical studies is now complete and ready for patients.

We now have additional GMP manufacturing runs underway for further long-term clinical supply. And we’re now also working on finalizing the very last parts of the IND-enabling toxicology studies that are nearing completion here.

Let me Hung, if I can turn it to you and if you can just comment on some of the cost studies and the good news that we’ve had there and the progress. And then if you can just comment a little bit more on slide 17, we also showed some additional data on preclinical proof-of-concept which gives us further reason to be excited by this program..

Sure, thank you John. Hello everyone. Yes, so let me just speak a little bit to the - what we have learned so far on our IND-enabling toxicity studies. We are nearly complete with these studies. We actually have tested our molecule ATB200 alone as well as ATB200 has been co-administered with our chaperone AT2221.

To date we have seen no over-toxicities with any of the trails that have been performed. And I think that we are in the final stages of testing. We are doing the final pathology test right now with study reports pending, and those are expected by Q4 of this year..

So, so far so good, everything looks very good with the TOC..

Everything looks fine, we have not seen any signs of, any issues at this point..

Great, excellent. So continue, to have a clear fact of the clinic very good..

Right. And importantly I think that one thing that we have learned from our preclinical studies is that we actually have now a much better understanding of those that want to go into the clinic for our chaperone. There was a lot of work to determine this particular dose.

We actually have now selected our final dose for our enzyme as well as for our smart molecule chaperone enhancer. And that provides the optimal stabilization of the enzyme in blood as John alluded to earlier as well as more improved uptake and activity in the tissues.

And one thing I just want to remind everyone why we are so excited about this particular approach is that, if you look on slide number 17, one thing that we have come to find is that our enzyme is significantly better targeted and provides much better effect of clearing glycogen accumulated glycogen in the muscle tissues.

As shown on this figure, the glycogen clearance of our enzyme with the chaperone is significantly better than this current standard of care. It reduces the glycogen levels not nearly to wild type but certainly close.

But more I guess encouraging from our standpoint is that it also appears to have a robust effect in terms of improving the muscle physiology as shown in the lower panels. What is shown in the map 1 screening slides is that, map 1 is a protein component of these vesicles which are known to accumulate in Pompe disease.

The fact that our enzyme product is able to clear these particular accumulated vesicles to nearly wild type levels is certainly very encouraging. And we think that we can replicate this combination of enzyme with chaperone in the clinic to maximize the effect.

And with that, let me turn it over to our - back to Jay to give an overview of our clinical strategy..

Yes, this will be really important this evening, and this will be the first time that we are discussing our Pompe clinical development plan and strategy.

I think you’ll find something that we’ve worked on throughout this year we’ve embedded with patients, with our patient advisory board we embedded in sessions with key opinion leaders around the world. We believe that’s a smart and very rapid path into and through the clinic with what we believe to be a very differentiated product.

So, Jay, I’ll let you describe it more fully..

The clinical development plan begins the first study as an observational study. We will collect longitudinal data in patients on the current standard of care ERT. That study will be starting in the next few weeks.

The next study which will start by yearend is Phase I/II study of the next generation ERT ATB200 plus chaperone in both naïve and ERT switch patients. And we expect to have data from that study in 2016. And then, third in the middle of 2016 we’ll be starting our Phase III pivotal study, which will be of ERT switch patients.

Now we’ll be meeting with U.S. and European regulators in the coming months to discuss this plan with them. And we’ll be able to provide additional details after we have those interactions.

But I’m happy to be able to share with you at a high level the overview of the plans that we think is both robust and streamlined to get us through to regulatory submissions..

Great, we’re happy to discuss this further in the Q&A as well. But it’s a program we’re very excited about. It’s invented with some very, very experienced and smart people. And we think this could offer great benefit for people living with Pompe, so it’s something we’re very excited about, something that will generate some very important data for us.

And certainly very past path then by mid next year into the Phase III study for Pompe, so more to follow on that. Chip, let me turn it to you for the 2Q ‘15 financial results and guidance..

Great, thanks John. Good evening everyone. I’ll start today’s financial discussion with a few comments on our current cash position and financial guidance beginning on slide 20. Amicus continues to maintain a very strong balance sheet.

At June 30, of this year we had $361 million of cash and cash equivalents compared to $169 million at the end of last year. Our balance sheet was significantly strengthened during the second quarter with an over-subscribed follow-on financing raising $258 million in gross proceeds.

We expect this cash position including the proceeds from the offering to fund our operating plan into 2017. Turning to our second quarter 2015 financial results on slide 21, I’ll be referencing tables 1 and the 2 in the press release we issued earlier today and additional details will be found on our Form 10-Q which will be filed later this evening.

Total operating expenses for the second quarter of 2015 increased to $26.9 million compared to $14.7 million for the second quarter of 2014, the year-over-year increase was primarily due to increases in preclinical and clinical development costs on Fabry monotherapy program and the Pompe ERT program as well as investment in pre-launch activities for Galafold.

Net loss attributable to common stockholders in the second quarter was $27.1 million or $0.27 per share compared to a net loss of $14.6 million or $0.22 per share in the second quarter of 2014.

The amount of net loss is primarily attributed to an increase in operating expenses and as of June 30, 2015 we had approximately 118 million shares outstanding. I’ll also note that during the second quarter we paid off the outstanding balance of our term loan and currently have no debt on the balance sheet.

As a reminder in December 2013 we did a debt financing, we did $15 million of aggregate principle amount with an 8.5% interest rate. From January 2014, until now we have been making interest only payments. And in June, we paid off the outstanding balance.

This summarizes our key financials for the second quarter as well as our updated full-year 2015 guidance. More information on our financials will be available in the 10-Q which will be online later this evening. I’m happy to address questions during the Q&A. But for now I’ll turn it back to John..

Great, thank you Chip. So, I’ll just finish up on the last slide here that, talks about all of the milestones ahead of us. Certainly a great first half of the year for Amicus and we hope an equally great and perhaps greater second half of the year. A lot of work ahead of us still.

If you look at the Fabry franchise, the first two milestones for Galafold, again we have now accepted by the FDA or pre-NDA meeting request. And that meeting will occur later in 3Q. We then immediately hope to be able to file our NDA based on that meeting.

And specifically with agreements on the sales for plan, much of the work on that NDA submission is ongoing now, so, important milestones ahead for Galafold. But also too, we continue to invest significant resources in developing our own proprietary next generation enzyme replacement therapy co-formulated with our own proprietary chaperone.

To advance that program in the second half of this year, in the coming months, we will initiate a Phase II co-administration study of chaperone plus existing enzyme therapies for further proof-of-concept also important to facilitate our own Fabry next generation ERT of clinical program as well.

And that cell-line continues to, its development and early manufacturing and we’ll have more to say about that and some specific data this year during the second half of this year.

Again, on the right hand of that slide, then focusing on some of the key milestones ahead, quite a few of them for our Pompe program, as you can see a lot of activity in Pompe, a lot of excitement now that we have what we believe to be this very high quality and highly differentiated product, ready to enter into patients.

Continued discussions with regulatory officials as Jay indicated in the third quarter. We will hear in the next month or two begin first patient in, in the Pompe observational study. We will, in addition to the pre-IND and MHRA meetings in the third quarter.

And we will also then have final completion of the IND-enabling TOC studies very early in the fourth quarter. And in the fourth quarter, we will begin our Phase I/II study in people both ERT experienced and naïve patients in Pompe disease, again leading into what we hope will be a rapid transition to a Phase III program in 2016.

So, a lot of work still ahead of us. The mood at Amicus continues to be very, very positive. And we continue to welcome extraordinarily capable and passionate new leaders to the company both here and with our significant European operations led by our senior vice president of international data at Allsop.

And all of this continues to build toward our mission and our vision of building one of the next great biotechnology companies in this industry focused on the rare diseases. And to that end we’re very excited now to have our program ready for commercial launch. We hope with Galafold as that part of the pipeline advances.

We’re very excited that by the end of this year we’ll have another clinical candidate in patients with our Pompe program. We also continue to invest and work diligently in research and development. Science continues to be a very strong core competence together with clinical research here at Amicus.

And we will continue to advance our pipeline and will have much more to say about that in the second half of this year, in research and development and the science increases with our preclinical programs, again looking to develop products for devastating rare diseases where we can be first-in-class or best-in-class.

And with that, we also continue to be very, very active on the business development front looking at products, programs and technologies that will enhance our existing programs or add to our pipeline to help us fulfill this vision. With that operator, we’re happy to take any questions..

[Operator Instructions]. And our first question comes from Ritu Baral with Cowen. Please go ahead..

Hi guys, thanks for taking the question. First question I have is on Galafold in Europe. Have you, can you give us a little more color on the depth and breadth of your payer work in Europe, I know you have the quote from the U.K.

but have you had preliminary conversations with different companies on how they view the benefit of the data package produced? And also, have there been any changes in Fabrazyme and Replagal prices are assessed recently in Europe potentially in anticipation of a Galafold launch?.

To your question, it has two parts Ritu. I’ll let Brad take up both of those..

Yes, so, thanks Ritu for the question. So, from a market research perspective what we showed you there was I think a snapshot of the work that we had done with over 20 different payers through the major markets in Europe, rest of the world and the United States, so that was a high-level snapshot that was reflective of those conversations.

I won’t speak to the formal conversation that will happen with regulatory authorities and reimbursement authorities around the potential approval of the product.

But I think rest assured that the feedback we’ve gotten from payers through that market research process was very supportive based on the target profile that we showed them from the data package we generated over the course of last year.

And I think to the acknowledgement that it was both the data that they saw but also I think the value of that bringing in, oral small molecule into that space would be perceived well from that group as well.

The second part of your question, remind me was?.

If there been any changes?.

So, from what we understand there has not been price taken in the replacement therapy in the Europe, in the major European markets. There was, a couple of years ago some look at the number of enzyme replacement therapies in terms of whether or not they were bringing the value of their prices were commanding.

But at this point from our perspective there hasn’t been any major change in terms of reimbursement for enzyme replacement therapies..

One of the areas Ritu that we’ve done quite a bit of diligence on is, understanding the pricing of enzyme replacement therapy around the world. And of course there is, distinctions by geography and certainly given that enzyme therapy is a price in a milligram per kilogram basis, differences in patients.

And I think we have a very clear view of the dynamics of pricing on the enzyme therapy in the Fabry market space.

And maybe Brad, if you just want to comment about where we see enzyme therapy priced today?.

Yes, from our understanding from the research we’ve done and again this is a global average, it’s of course a week-based product. And as John has mentioned, there are differences by geography. But in general that average looks like it’s around $275,000 to $300,000 per patient per year.

And from what we understand there is not much difference between Fabrazyme and Replagal, so that’s - you could consider that a global average price..

But it hasn’t gotten easier for patients to get the enzyme in what might otherwise be restricted access?.

The dynamics in anticipation of Galafold, I will say that one of the advantages that we have, of course there is competition that’s always going to be a challenge.

But one of the advantages that we have over other new product launches is that we don’t have to go out and convince payers that Fabry disease is a life-threatening progressive disease that it’s worthy of approving a treatment and a treatment that would command an orphan pricing level.

So, that reimbursement very clearly exists, those payers very clearly acknowledge the unmet medical need in the Fabry space. And the regulators frankly based on granting us accelerated assessment clearly acknowledge that that unmet need still exits.

So we don’t have to build that reimbursement environment which I think gives us an advantage over other new products coming into this space..

Got it. And last question and just going to slide 18 where you outlined the post clinical development plan. What data will you get out of the Phase I/II before the start of the pivotal, and that will help them from the pivotal, I mean, obviously you’re not going to be completely done.

What do you need, what information do you need out of the Phase I/II to best design the pivotal?.

Yes, I’ll let Jay answer that question primarily. I can tell you Ritu in working with the doctors and thinking about again this notion of a smart and rapid path into and through the clinic. What we heard was the doctors want to see safety they want to understand the pharmacokinetics of the next generation enzyme therapy.

That primarily will inform the ability to switch patients. And they’d like to see that in both naïve and ERT experienced patients. So, that will be important data to gather. Jay, I’ll let you comment further..

The safety data is critical of course. And it’s hard to be more specific yet until we have the regulatory interactions. But clearly things like the end-point that we study in the Phase I/II study will inform us further on the Phase III study.

And the inclusion of both naïve and the ERT switch patients in particular, that is the switch patients will give us data that will help us in the Phase III study in the switch patients..

At this point, I think the plans that we’re going to submit to the regulators, I think we’ll get - be well-received by them. But and we’re very eager to have those meetings. We’ll be able to provide more details after we have those meetings on specifics..

Got it. Thanks for taking the questions..

Thank you..

Your next questioner is Anupam Rama from JPMorgan. Please proceed..

Hi guys, congrats on all the progress and thanks so much for taking the question. I just had a quick question on slide 10, when you talk to physician and KOL, maybe could you provide a little bit more color on what are the drivers of switching to from ERT to Migalastat.

Is it the oral convenience that’s driving it, the LVMI data, the GI data or is it the totality of the data? And then conversely, what is the strategy for those physicians that are sort of on the left side of this slide in terms of the strategy of trying to be - convince them to become early adopters ahead of the launch? Thanks..

Yes, thank you Anupam, great questions. And I’ll let Brad provide the answers..

Yes, so great questions Anupam. I think a couple of things, without question the totality of the data weighs on our favor. One of the upsides of having such a long development program is you have almost eight or nine years now of experience with Galafold in Fabry patients.

And over that period of time we’ve seen a consistent safety profile, we’ve seen very strong evidence both in naïve and in switch patients for sub-straight reduction for stabilization of kidney function, foreign impact on left ventricular mass, of course it all depends on what label you end up getting in those markets.

But at least the totality of that data package which goes into the target product profile was very well received by physicians. There is no question that that is a very important part of the value proposition.

That being said, we’re also I think very pleased and not surprised to understand that there is value ascribed as well to this being an oral therapeutic into an injectible and infused market where again patients are getting infusions every other week of their life, one day out of their lives, every other week to dedicate to their Fabry infusion.

So, that’s a very important part of the value proposition as well. From how you move patients from left to right on this, excuse me, how do you move physicians from left to right on this graph, I think part of it is certainly experience.

And so as more patients, as more physicians get experienced they would be more comfortable in looking to their peers and moving across to the other side of that market.

I think that there are also of course these significant efforts working with key opinion leaders to help us to continue to work through congresses, we’ll work to publications, etcetera just to get the information out there. So, I think it’s a broad gestalt of activities that we’ll be doing now as appropriate.

And then of course once we get our labels then we’ll be able to promote on specifically to those labels. So lots of activities to help move that from left to right and I think a great data package to support that..

Yes, I mean, starting with this as our baseline in this status already several months dated as well, so it doesn’t incorporate a lot of the work we’ve been doing in the last handful of months. But this is a great baseline to start from even in all these three different segmentations of that patient population.

So, to Brad’s point to the ones in the left, I think it’s more work, more conversations the doctors who do not have experience in the clinic generally with the drug.

So we’re already beginning those conversations and this is as much about science education as it is about medical and clinical practice given the very unique mechanism of action of this drug that’s just very, very differentiated from enzyme replacement therapy but also differentiated from any other drug and development today in Fabry..

Great. Thanks so much for taking our question..

And the next questioner is Joseph Schwartz with Leerink Partners. Please proceed..

Great, thanks so much. I was wondering first of all on your pre-NDA meeting.

How do you expect the focus of that meeting to differ if at all from previous interactions you’ve had with the FDA like the Type-C meeting? And how much of the NDA is actually already in the works at Amicus? And do you think that you might communicate with The Street if the outcome of the, how the outcome of the FDA meeting, the pre-NDA meeting might differ from your expectations.

It seems like you’re doing a great job anticipating what the FDA would like you to focus on.

I’m just wondering about your communication strategy afterwards given you’ve got second half of ‘15 guidance for NDA filing, I’m just wondering if that might be refined further after you’ve actually worked down with the FDA again?.

Great, yes, I think I heard three questions there, Joe. So I’ll ask Jay to address then the first on the FDA meeting coming up, the pre-NDA meeting the nature of it. Second, how much we have done on the NDA submission itself, the percent of progress if you will.

And third, the communication strategy, so Jay, maybe if you’ll take those first two and then we’ll take the third..

Yes. The pre-NDA meeting, it’s really going to be based on requests that we got that the Type-C meeting in the discussion. So it’s a follow-on with regard to the content of the NDA, the under sub part H.

And specifically it’s going to be forward focused on the proposed design for the Phase IV commitments, confirmatory studies that are necessary part of sub-part H.

So, we’re picking up where we left off at the Type-C meeting and providing more details to the FDA with regard to our plan which and they have requested a GI study, what that study would look like as well as additional supportive data that we’d be able to provide out of the registry.

And we are quite far along in the NDA, I can’t give a specific percentage but from the moment that the MAA went in, we turned around and started converting that as it were to an NDA. So, we’re very much on track for submission this year and following the pre-NDA meeting..

Great, that’s super helpful. Thanks.

And then maybe one on Pompe, is this your final scale for production of ATB200? And then actually I wanted to follow-up with that with some questions on your trial design?.

Yes, of course, Joe, yes. So this is the scale sufficient to go into the clinic. This will be our clinical scale. We do anticipate moving from 250 to 1,000 leaders at some point in the next year for the last of our clinical supply and also importantly our early commercial supply. We want to make that transition for patients.

We think those drugs will be the same. But we want to do that in the final stages of the clinic. Importantly we have that material to move forward. Technically, just to let you know, if the 1,000 leader working volume, 2,000 leader bioreactor technically..

Okay, that’s helpful. It’s encouraging you’re doing that sooner rather than later.

Can you give us any more details on the Pompe observational study in terms of the size and what data you’re going to focus on gathering and then also maybe the doses end-points and size of the Phase I/II study and when we might be able to see data reported from that trial?.

And Jay, maybe why don’t you take the observational study part first please?.

The observational study, we’ll be looking at multiple outcome measures of the types that have been studied in Pompe that is on standard of care. We’ll be coming out with the sample size shortly when the study is initiated. But it’s certainly something that is doable and going to be recruited in a relatively short time.

And we will gain the important longitudinal data once we follow that patient period of time that in four months for the remainder of the program.

And I could turn it over for?.

Yes, Phase I/II, yes, and again I think we can having not yet embedded this fully with the regulators, I probably don’t want to release all that, so just the general thinking on the Phase I/II Jay, the purpose in general study design..

Yes. We will, I mean, in any Phase I/II study in this realm safety is the first step that you’re looking at. We don’t anticipate any safety issues whatsoever based on what’s done pre-clinically and ERTs. But of course it’s the necessary step.

And we will cover a range of doses, as doses increase as you monitor safety towards an anticipated end-dose of 20 milligram per kilogram for the enzyme.

And then these patients will be studied for a period of time for safety, for pharmacokinetics as well as activity of the drug informing us even further in this population as I had mentioned before for the Phase III study that is planned to start by middle of next year..

Okay, great. Thank you for taking my questions..

Thank you. Great questions. Thank you..

And the next question is from David Lebowitz with Janney Capital. Please go ahead..

Thank you very much for taking my question and it’s great to see the progress.

Would you be able to I guess explain why the Phase III in Pompe is examining the drug in switch patients whereas the Phase I/II is looking at naïve and ERT switch?.

Sure, I think there are, different learning from those two different populations. Jay, I’ll let you comment a little further..

We feel that the naïve patients will be studied adequately in the Phase I/II study that we would gain as much data as is needed on both safety and activity of the drug in that population. And therefore there won’t be a need to study naïve patients further in the Phase III study.

Whereas, as it’s designed now to get complete information on the ERT switch based ERT experienced patients, would acquire that additional Phase III study. So, in the program as a whole we’re going to be studying both populations adequately, it’s just divided up between the two studies in that way..

I think if you look forward David, I think it’s important for labeling purposes for this drug that we have appropriately studied naïve patients. And to do that sooner rather than later makes sense we believe.

Secondly also in the immunology of this disease, as you know the current enzyme therapy has significant issues around tolerability, immunogenicity, all patients are known to zero convert and develop antibodies.

We’re particularly interested in studying patients who have never seen enzyme replacement therapy to see what the tolerability and immune response would be with this new regimen that we’ve developed..

Well, thank you for that.

Would you be able to provide an update on the Migalastat ERT co-admin trial as well as the Next-Gen ERT in Fabry?.

Sure, so the Next-Gen ERT again is our owned proprietary enzyme therapy that we’re making again also with WuXi now that we’re developing the cell lines here and together with our partners at WuXi, co-formulated with migalastat that continues the science progress and work both here at Amicus and having been transferred now to WuXi at WuXi as well and some of the early stages of manufacturing.

I will have more of an update on that in the months to come but that continues to be a very exciting and very fast-moving program for us.

In terms of the co-administration study, again remember there the intent is to replicate what we did in a single-dose study that both Fabrazyme and Replagal with the oral co-administration of our chaperone given every other week infusion just prior to the infusion, again the belief being in the early data in the clinic showing that you can extent the half-life increase the tissue uptake.

And in preclinical studies not only increase uptake into key target organs of disease until much better substrate reduction. So, we’re going to, having done that a couple of years ago with single-dose studies we’re going to do that with multiple dose studies.

We don’t believe that the right path and the best path for patients, is going to be to extend that to a full drug development program. We think this Phase II will be very important in understanding the long-term safety and efficacy of combining chaperone and ERT.

We also think it will provide very important information that will facilitate our path into and through the clinic for the co-formulated next-generation ERP that we develop..

Thank you very much for taking my question..

Of course..

And I am showing no further questions in the queue. I would like to turn the conference back to Mr. John Crowley for any final remarks..

Great, thank you operator. I think that’s all we have. It was an excellent call, an excellent quarter. And we look forward to continued good news ahead. Thank you so much. Take care..

Thank you all for joining us today. This does conclude the conference. And you may all disconnect. Everyone have a wonderful day..