Sara Pellegrino - Director, Investor Relations John Crowley - Chairman, Chief Executive Officer Chip Baird - Chief Financial Officer Bradley Campbell - President and Chief Operating Officer.

Ritu Baral - Cowen and Company, LLC Anupam Rama - JP Morgan Chase & Co Mayank Mamtani - Leerink Partners.

Good day, ladies and gentlemen and welcome to the Amicus Therapeutics’ First Quarter 2015 Results Conference Call and Webcast. At this time, all participants are in a listen-only mode. Later, there we will be a question-and-answer session, and instructions will follow at that time.

[Operator instructions] As a reminder, today’s conference is being recorded. I would now like to turn the conference over to Sara Pellegrino, Director of Investor Relations. Ma'am, you may begin..

Thank you. Good evening and thank you for joining our conference call to discuss Amicus Therapeutics’ first quarter 2015 financial results. Speaking on today’s call, we have John Crowley, our Chairman and Chief Executive Officer; Chip Baird, our Chief Financial Officer; Bradley Campbell, our President and Chief Operating Officer and Dr.

Jay Barth, our Chief Medical Officer is on today’s call and available to participate in the Q&A session.

As a reminder, this conference call contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to business, operations and financial conditions of Amicus, including but not limited to, preclinical and clinical development of Amicus' candidate drug products, cash runway and the timing and reporting of results from clinical trials evaluating Amicus’ candidate drug products.

Words such as, but not limited to, look forward to, believe, expect, anticipate, estimate, intend, plan, would, should, and could and similar expressions or words identify forward-looking statements.

Although, Amicus believes the expectations reflected in such forward-looking statements are based upon reasonable assumptions, there can be no assurance that is expectations will be realized.

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 10-K for the year ended December 31, 2014.

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. Good evening and welcome everybody to our conference call. I just want to emphasis the momentum going on it Amicus right now as we build out initial commercial team and do so on an accelerated time line following our positive meetings with both European and United States regulators at the end of the first quarter.

So we are very busy at Amicus preparing for the global launch of Migalastat monotherapy which as you know is a precision medicine for people living with Fabry diseases we have a amenable genetic mutations.

We have accelerated our submission timeline in Europe which of courses moves up the potential EU launch and we are very pleased to report that we are on track to submit the MMA for migalastat. Again this will be our first ever marketing submission for Amicus so major milestone for this company. That is on track to be submitted in the second quarter.

So this quarter we also following our Type C meeting with FDA we are also as you know pursuing a U.S. approval pathway on the recommendation of the U.S. regulators under subpart H as we work towards the NDA submission in the second half, comment more on both of those efforts underway in a moment.

With respect to commercial and Brad Campbell as here as well so our last Brad to provide a little more color and background and this is a major investment and a major focus in the company today we are focusing first in Europe and we will very quickly turn the buildup of the team to here in the United States again in preparation for the migalastat launch.

We continue to recruit key leadership in Europe there is a very significant pool of talent there after several successful rare diseases companies being acquired by other companies so we are looking at that, but we are also looking at existing companies both in the rare diseases and also more proximately throughout biotechnology with some of the key European leadership.

You saw of course a week ago our announcement of a new member of our senior leadership team David Allsop, David coming to us from Biogen where he was mostly recently Senior Vice President of Europe and Canada.

David is a remarkable leader in 27 year Industry Veteran very instrumental and building Biogen’s international commercial organization and we are just so very pleased to have David on Board and certainly welcome him to Amicus and to my senior leadership team. David reporting directly to Brad Campbell our President and Chief Operating Officer.

We are committed to making migalastat available and accessible to patient as quickly as possible and to do that let me provide you with some updates on some key recent activities. Again just to briefly recap our regulatory meetings as we outlined on our March 19 conference call and with many of you in individual meetings over the past several weeks.

Again with Europe we’ve received in a personal meeting very positive feedback from the repertoires who were designated to this a project in Europe they commented specifically on this significant unmet need in Fabry as well as the [indiscernible] and the repertoires encouraged us to request accelerated assessment and we have done that.

So as far as the status update we requested accelerated assessment and with either accelerated assessment were standard review again we are in place to file the MAA over the coming weeks this quarter the second quarter of this year. In the United States we also had a very good Type C meeting with the U.S.

FDA again a face-to-face interaction based on the guidance from FDA from that meeting we will be pursuing a Subpart H pathway for accelerated approval in the U.S. which is for an unmet medical need based on a surrogate biomarker reasonably likely to predict clinical benefit that of course is the standard for approval.

The FDA indicated it will consider in addition to the previously agreed to endpoint of the GL3 reduction in the interstitial capillaries they will also consider cardiac mass as measured by left ventricular mass index by echocardiogram is a potential surrogate FDA also indicated additionally that they would consider stabilization of kidney function is measured by glomerular filtration rate.

So having now completed two Phase III studies successful in Fabry again both of these the largest randomized controlled studies ever conducted for Phase III in Fabry disease we continue to be very confident in the drug approval pathway in the U.S. The second part of the feedback from that meeting from the U.S.

FDA was their input us that we would of course under accelerated approval have the Phase IV commitment. So our team is working diligently on a number of different alternatives that was specifically suggested by the - there were specifically suggested by FDA within the context of a GI study.

Again we are in the GI division for review at FDA, so it’s not a surprise that the FDA GI division focused on the very serious and life limiting GI symptoms notably the diarrhea constant diarrhea and patients living with Fabry disease. So again all therapies under Subpart H have that post marketing commitment.

So we are actively preparing the new drug application the NDA submission for the second half of this year again much of that work overlaps with the very excellent and detailed work that our teams have done in preparation for the immanent filing of the MAA in Europe.

The next interaction with the FDA will be a pre-NDA meeting in the middle of this year to finalize the details of the NDA and to discuss the final plan for this Phase for post marketing setting.

So I will just comment I could not be more proud of Jay Barth and his team in clinical and regulatory here at Amicus they have been working tirelessly to finalize these marketing application to make them of a very highest quality and certainly that provides us a great path forward as we begin to prepare to market these drugs globally.

So we have been very conservative, just a brief update I’ll go now pivot briefly to I mentioned the updated guidance and Chip will outline in more detail.

As you know we have been conservative up till now in our commercial infrastructure, but now especially with a feedback the regulatory feedback we received at the end of the first quarter and now with David Allsop as our leader, business leader in Europe, we are ready to move forward with a full execution of our launch plan and that includes accelerating the timelines based on that regulatory feedback.

So with that we will be updating our full-year net cash spend guidance to account for the pre-commercial investments that have shifted from 2016 into 2015 as well as with the continued success in our Pompe program, increased manufacturing investment for our next generation Pompe ERT. So we’ll hear about that revised guidance in a moment from Chip.

Let me just comment briefly on the Pompe program. Again this is our next-generation enzyme replacement therapy for people living with Pompe disease that remains on track and will enter the clinic by the end of this year. We as you know we are manufacturing that drug through our partnership with WuXi Biotechnology in China.

In fact I will be in China all next week for meetings with their CEO and senior leadership on that program. Chip, I am very much looking forward to in part of our deepening relationship with our partners at WuXi.

This program in our investments as you know is build off the significant enthusiasm that we’ve had from this program based on our pre-clinicals results much of which was presented earlier this year at the World Meeting at Orlando in February.

And again what we have identified and designed and now scaled up to manufacturing is a novel uniquely glycosylated highly phosphorylated Pompe enzyme replacement therapy, what we refer to internally as ATB200 and that is combined with a chaperone.

So we believe that through our dedication to being an extraordinarily patient focused company on world-class science and clinical medicine we are laying the foundation to become a leading global biotechnology company with one product poised to - for its filing and hopefully to enter the market and our next generation product in Pompe, our second product ready to enter patients in the second half of this year.

Before I turn it over to Chip for the financial update. Let me just hand it over to Brad to add a little bit more color on some of the significant activities underway in building our commercial infrastructure and leadership..

Sure, thanks, John. Good evening everybody, so I will just touch on a couple of key themes here and not go into too much detail.

First and foremost we are continuing to build on our ten-year history of strong medical affairs and patient advocacy relationships with the community and we are redoubling our efforts to engage in that medical outreach going forward.

That builds upon I think the great new data that we have seen over the course of last year from our studies and continue to see from our extension studies and we have been take sort of an updated target product profile out to the community and the anticipation and support from the physicians, and payers, and patients all of our key stake holders continues to be strong and finally as John mentioned earlier we are building a top notch organization to ensure that we are successful in the launch of Migalastat.

David Allsop in particular as a strong leader as John mentioned with a wealth of experience, building successful teams and launching great products into areas of high unmet medical need and we are continuing to recruit a higher top talent in key functional areas as well as key territories in order to be sure that we have that team in place.

So we are making great process towards the successful launch and as John mentioned with the feedback from the regulatory agencies, is anything we are accelerating those efforts and things are going smoothly so far. So with that I will turn it over to Chip to highlight the financial details..

Chip I know you are traveling, so you are joining us remotely..

Sorry guys, Thank you. Good evening, start today’s financial discussion and then a few comments about our current cash position and guidance. Amicus continues to maintain a strong balance sheet. At March 31, 2015 we had $151.6 million in cash and cash equivalents compared to $169 million at the beginning of the year.

As indicated in our press release we issued earlier this afternoon, we are updating our full-year 2015, net cash guidance to $100 million to $110 million for 2015 and increase from a previous guidance range of $73 million to $83 million.

For updated guidance reflects additional commercial manufacturing investments are in our positive meetings with regulators in the Europe and in the U.S. prepare for the commercial launch on migalastat monotherapy. As well as manufacturing clinical supply of our ERT as we have that programs towards the clinic.

We expected current cash position to fund our current operating plan into the second half of 2016 and beyond several expected regulatory and data milestones. Turning to our first quarter 2015 financial results, I will be referencing tables one and two in the press release we issued earlier today.

Additional details on our financials can be found in our Form 10-Q which will be filed later this evening. Total operating expenses for the first quarter of 2015 increased to $24.1 million compared to $16.1 million for the first quarter of 2014.

The year-over-year increase was primarily due to increases in pre-clinical and clinical development costs on the monotherapy program and the Pompe ERT programs. Moving down the P&L, we had a non-operating loss of $200,000 in the first quarter 2015 compared to non-operating loss of $300,000 in the first quarter of 2014.

This change was primarily due to an increase in interest expense on outstanding debt, partially offset by an increase in interest income on our hired cash balance.

Net loss attributable to common stockholders in the first quarter of 2015 was $24.3 million or $0.25 per share compared to a net loss of $15.9 million or $0.25 per share in the first quarter of 2014. The change in the net loss was attributed to an increase in operating expenses and as of March 31, 2015 we had 96.4 million shares outstanding.

This summarizes our key financial results for the first quarter of 2015 as well as our updated full-year guidance. More information is available in the 10-Q and I’m happy to answer any additional questions during the Q&A session. But for now I will turn the call back to John..

Great. Thanks, Chip. So it was a great quarter for us, again continued momentum, we have got some great milestones ahead of us. I think there will be terrific for people living with Fabry disease as we look to the MAA submission this quarter and then the NDA submission in the second half of this year.

And again for people living with Pompe disease to know that they will have another potential therapeutic option enduring in the clinic in the second half of this 2015 on many levels is very, very rewarding. So with that operator we are happy to take any questions..

Thank you. [Operator Instructions] Our first question is from Ritu Baral of Cowen. You may begin..

Hi guys, thanks for taking the question.

Now we are approaching commercial launch can you give any more thoughts on sequential pricing both and especially if you were given some of the recent orphan drug launches and how their pricing has worked out?.

Yes, of course Ritu thanks for your question on pricing. We have done a lot of work on that with consultants and internally and are forming a point of view and let me turn it over to Brad to answer that questions specifically..

Thanks for too, we continue to believe as we’ve looked at the value of that migalastat brings and the orphan pricing landscape that the orphan pricing models still holds up quite robustly as long as you are brining real value to the community which we believe we are.

I think we are in a unique position to some extend compare to some recent launches in that. We don’t have to create a reimbursement environment for Fabry disease, we don’t have to convince payers that it’s worth - the disease is worth reimbursing for.

So that provides us with some advantage and as we’ve said even if you are to price at parity with the current enzyme replacement therapy somewhere in the $275,000 to $300,000 range. You are still bringing significant savings to payers in the avoidance of the infusion associated cost concomitant et cetera with the enzyme replacement therapy.

So we think we bring a lot of value with migalastat and we think we have a strong package to bring to payers and we think that the pricing opportunity is robust for this orphan disease..

And just a follow-up on the co-formulation study, the expanded co-formulation study that you mentioned, what detail do you have set, and what’s supposed to be the challenge around the potential co-formulation study later this year..

You mean in Pompe Ritu..

I am sorry. I believe you mentioned that Fabry co-formulation study..

Sure, so we of course have our Fabry monotheraphy precision medicine migalastat and that’s for up to half the patients living with Fabry.

For the other half or so of the patients we are developing our own proprietary next generation Fabry enzyme replacement therapy and that is co-formulated with migalastat and the benefit there of course is that the chaperone co-formulated with our ERT in one product is conferring substantial benefits in terms of keeping the enzyme folded in plasma more active, specifically more active when it gets to key compartments and key tissues of the disease.

So we’ve seen in pre-clinical studies that it lead to substantially greater reduction of substrate versus enzyme alone. So that’s the basis of the product and that is a product in development still that will be part of my discussions, when I am over to China, WuXi is also in the process development stage for that protein with us.

And I think we’ll have more to say in that second half of this year..

And the potential for co-administration in Fabry as well..

Yes, so with the co-administration again that’s the notion that you are taking our oral migalastat in combination with one of the currently marketed ERT is Fabrazyme or Replagal, we have done a study previously a couple of years ago, where we show in a single dose that you could significantly enhance the half-life and tissue distribution of the enzyme.

So it was a good proof-of-concept, single PK study we like to do that over a longer period of time we know through our pre-clinical studies that the co-administration over longer periods of time can confer significant benefits.

We are not visioning that as a product extension necessarily for migalastat monotherapy rather we see that as a longer-term study and a bridge for patients and physician to our next generation ERT..

And finally what related more co-administration work with [indiscernible] what sort of study may you have to….

Sure, it would be a longer term clinical study, so previously we’d only done a single dose study Ritu, this would be a longer-term study to understand the PK of the safety, the uptake of the enzyme and potentially other benefits that we may be able to measure over a period of time measure in months instead of that single dose.

But that’s still a development..

Is this potential dataset that could generate before a potential approval in Europe or the U.S.?.

Well, we think we are on such an accelerated timeline certainly in Europe that data is wouldn’t be available prior to approval in Europe, uncertain whether it would be available in time for U.S. I think we need to understand the U.S. filing timelines a little bit better for us..

Great. Thanks for taking the question..

You’re welcome..

Thank you. Our next question comes from the line of Anupam Rama with JPMorgan. You may begin..

Hi, guys, thanks so much for taking the question. Just maybe a quick modeling question for Chip on the increased spend. How should we think about the spend in terms of maybe a steady growth throughout 2015 or will it be more second half 2015 weighted and other differences between say the R&D and SG&A lines that we should be thinking about thanks..

Sure, thanks Anupam. I think you are right, it’s going to be more backend loaded versus, [indiscernible] in fact you we can see - in the first quarter here we spent about $18 million in cash.

So there will be ramping and as Bradley and John alluded to reports its really driven by some other 2016 commercial expenses in the 2015 as well as accelerating and continuing the investment in Pompe manufacturing. So those are the two principal drivers.

And I would say between R&D and SG&A are fairly equivated in terms of they’re driving upsize, the increase in the overall net cash spend for 2.15..

Great, thanks for taking the question..

Yes, thank you, Anupam and just to add a little more color to that in terms of any future financing. We think we’ve got good strength in our balance sheet today even with this increase spend. We are looking at 18 plus months of cash on hand today.

So we continue to have no immediate need for financing, we will be opportunistic, but we do think it’s important though for us to achieve one or more significant value creating milestones before we do any financing. So no immediate plans for financing..

Thanks for the additional color..

Thank you. Our next question comes from Joe Schwartz of Leerink Partners. You may begin..

Thanks. This Mayank covering for Joe. Quick question along the same teams on oral migalastat co-administered with currently marketed ERTs.

Can you talk a little bit about the longer-term Phase II study how big is that any value creating milestones that we can expect from that around some of the approvals that may come? And going back to the FDA meeting that you have is that calendared yet or no and to follow up to that what are some of the key topics that I know you said you will be discussing the Phase IV study design there but also the existing data points that you have other some teams that you plan to focus more which will help you set the stage up for the ad compound or which may happen closer to be approval.

Thanks..

Yes, that’s excellent question, there is quite a few of them in there. So let me try to piece them apart. In terms of FDA we have not yet requested the pre-NDA meeting. So to do that we need to finalize our plans for what we think is the best Phase for GI focus study.

And to the last part of your question yes absolutely if you look at the Fabry patient community although they die from cardiac disease and kidney disease as well as strokes.

What they complain of in terms of the most light limiting aspects of their disease is severe GI problems most notably diarrhea problems very, very severe and we’ve shown in one of our Phase III is that were it was measured a statistical significance in the improvement on a validated patient reported outcome measure of the GI symptom it was diarrhea Jay and with that end point compare to placebo we showed improvements again in patients statistically significant.

So we think we can design a study where we have a high confidence that we would succeed, but also it would be something very, very meaningful and also something that would support very rapid conversion of the Subpart H conditional approval to a full and final approval which would make it of course the only fully approved drug in the United States for people living with Fabry disease.

So I do think we can design a good steady Phase IV study the work on that is well underway and certainly would be helpful in a number of different ways for us. The first question that you had was around the co-administration.

And on co-administration again that is the study we think is very important further proof-of-concept of the significant benefits of our CHART platform technology the Chaperone-Advanced Replacement Therapy the use of a small molecule combined with enzyme therapy we think the ultimate benefit for people living with Fabry is going to be when combined and co-formulated with our own proprietary ERT, we do think it’s very important that physicians have experience with a longer-term Chaperone plus ERT and that we could build on our extensive pre-clinical data together with the data from that single dose Phase IIa PK study that we did with that migalasat plus either Fabrazyme or Replagal.

So we don’t have any specifics on patients numbers we think it would be a small study and it could support a very strong publication maybe even further additional intellectual property there is a lot of different ways in which that study could be beneficial but again with the notion that we want to move science forward to provide better alternatives for people living with Fabry.

We think that would be an important step forward..

And maybe I can squeeze in one last question..

Of course, yes..

Does that feed into in anyway your lifecycle management plans and also can you remind us on the patent situation on the IP for migalasat and will this in any way help or one of your next generation programs will facilitate with that?.

No so to the second part of that question we have very strong intellectual property protection in addition to the orphan exclusivity the seven years in the United States, 10 years in Europe we have a significant patent to stage around migalasat around the method in use and various extensions of that as well as different patents and patents pending around the dosing in novel characteristics of migalasat that take us out to the late 2020s on that patent around 2027, 2028 in the United States.

So I think we are in a strong position there. So we are not doing this study as part of lifecycle management we are doing this study to facilitate to better understanding the co-administration study a better understanding of the benefits of Chaperone plus ERT.

So that would lead we think to better data to better facilitate how we design our studies with our next generation ERT and that’s the program we think that certainly have substantial exclusive protections and quite a few trade secrets around that in terms of our unique abilities to make those enzymes therapies.

So I think we are in a very, very good place with migalastat monotherapy in a co-admin is a terrifically strong bridge to our next generation ERT, the co-formulated program..

Great, thanks..

You are very welcome. Thank you..

Thank you. And our next question comes from [David Lavalis with Janney Capital]. You may begin..

Thank you very much for taking my question.

Would you have any estimate on to when you might hear about whether or not there will be accelerated assessment in the Europe?.

We think we should hear right about the time that we are filing the MAA, those are separate activity as that and work streams were probably this quarter..

Okay.

And what is the process going to be like for obtaining reimbursement given the high cost in Europe as far as country-by-country, if you could just shed some light on that?.

Yes, go ahead Brad..

Yes, I mean it’s fairly standard as you said it is country-by-country in some cases it can go almost immediately upon approval from the central authorities, in other cases it’s a more drawn out process and so we’ll of course follow that process closely also based on global valued RCA which articulate the value in a payer friendly way and then you translate that to local valued RCA for each of those geographies that’s in Europe and then of course U.S.

and other geographies follow a different process. So those are the kinds of activities that are well underway now and that we are preparing for and we’ll proceed as quickly as possible in each of the territories to get migalastat available to as many patients as possible as quickly as we can, that’s the goal..

Thanks..

Thank you. Our next question is follow-up from Ritu Baral of Cowen. You may begin. Ritu, your line is open, please check your mute button..

Sorry about that. Thanks for taking the follow-up guys. Where are you as far as CMC package and any additional CMC work that we typically done in the U.S.

or Europe?.

That’s complete, so all of those sections of the filing are complete for CMC for pre-clinical we are with the MAA in the final quality check. So the overall document in the clinical section I believe is also complete to very, very nearly complete.

So I think we are in a very good place, so there is no gating data, there is no data we are waiting for, there is no other analysis of date. We’ve just being very, very thorough in this document..

In NDA expense?.

May we have under that conversation with FDA, but usually you do that when you are waiting for more data of course and we are really not waiting for any more data..

Okay, and last question. What is your current priority on some of the portfolios as we are looking for a Chief Commercial Officer regional head for U.S.

where are the priorities there as we expand the commercial?.

Right, so with the strength of David and his experience as ahead of international. David reports directly to Brad and at the appropriate time we would expect also to hire a head of the U.S.

and a par with David there are other key commercial hires ahead of global marketing, other executive level hires beginning with the international together with market access, reimbursement, patient efficacy, medical affairs and country managers.

All that’s underway we actually are in very advanced discussions and not yet ready to announce, but some senior executive hires in addition to David also that I believe will be in a position this quarter to announce as well. So I’ve been very, very pleased with two with the quality of candidates that have stepped forward.

We had abundance of high quality candidates from biotechnology including major rare disease companies as well as other companies with significant international experience who raised their hands for this opportunity. So we are very, very pleased with the quality of candidates that we are seeing for these positions..

Great. Thanks for taking the follow-up..

Very welcome..

Thank you. I am showing no further questions at this time. I would like to turn the call back over to John Crowley for closing remarks..

Great, operator that’s all I have. Thank you everybody for listening have a good evening..

Ladies and gentlemen this concludes today’s conference. Thank you for your participation have a wonderful day..