Good day, ladies and gentlemen. And welcome to the Amicus First Quarter 2014 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, today’s meeting is being recorded. I would now like to introduce your host for the conference, Ms. Sara Pellegrino, Director of Investor Relations. Ms. Pellegrino, please go ahead.

Thank you. Good evening. And thank you everyone for joining Amicus Therapeutics conference call to discuss first quarter 2014 financial results. Speaking on today’s call we have John Crowley, Chairman and Chief Executive Officer; Chip Baird, Chief Financial Officer; Jay Barth, Chief Medical Officer; and Bradley Campbell, Chief Operating Officer. Today’s prepared remarks coincide with the slide presentation that is now available on our corporate website at www.amicusrx.com. These slides are located in the Investor section under Events and Presentations right below the webcast link to today’s call. Starting on slide two, you will find the reference to our Safe Harbor statements. 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, 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 its 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 Form 10-K for the year ended December 31, 2013. All forward-looking statements are qualified in their entirety by this cautionary statement and Amicus undertakes no obligation to revise or update this presentation to reflect events or circumstances after the date hereof. And with that, it is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer of Amicus.

Great. Thank you, Sara, and good evening, everybody. We, of course, had a very important update call on our Fabry Study 011 result last week. So through this call I will shortly turn it over to Dr. Jay Barth, our Chief Medical Officer. We see there are number of new callers in who may not have had a chance to review the data with us live on that conference call last week. So in a much more expedited fashion we will go through a high-level overview of that data and then I will turn the call over to Brad Campbell and to Chip as well to go throughout some of the operational update. Brad in particular will take us through the great progress we have been making with our next-generation enzyme replacement therapy. Let me begin though by just reiterating what we stated last week and again, what was in the press release just issued after market close today. That last week really was we believe momentous -- occasion for Amicus as we announced the very positive 12 and 24-month data from the first of our two Phase 3 studies for Fabry Monotherapy, our drug migalastat for Fabry disease. We had a chance as we indicated on that call last week to review that data with a handful of investigators under great confidence prior to the data being made public and since then I had a chance to present the data within even larger number of investigators and key opinion leaders. And again, the results have been greeted with great enthusiasm in the investigator and physician and science community. We will continue to describe these data to that community and of course, both in the early fall, hopefully, present this data at the major science conference. We have also had…

Thank you. I'm very pleased to have the opportunity to review the data with you briefly from study 011, starting with slide five. These are the key results from the study in patients with amenable mutation and now with new data out to 12 months and there are two important comparisons that I would want to draw your attention to. First is from baseline to six months and these are at a double-blind placebo controlled phase of the study. The patients who are on placebo are in the red dashed line, patients receiving Migalastat in the solid blue line. And you could see a statistically significant decrease in kidney IC GL-3 in the patients who are receiving Migalastat and the P value for that comparison 0.008. And these patients continuing to follow blue line had a durable effect. They maintained this effect over the 12 months of the study period. The second comparison and this is the primary analysis of these new data are the patients who are on placebo for six months red dashed line and then switch to Migalastat between month 6 and month 12, the blue dashed line. These patients experienced the same decreasing kidney IC GL-3 as those who had gotten Migalastat in the first six months. And the P-value for the comparison of patients who switched from placebo to Migalastat P 0.013. And seeing the same effect in both these groups -- really confirms the activity of the drug. So there are multiple statistically significant and durable effects that demonstrates Migalastat activity in kidney IC GL-3. The same effects were seen in turning at slide six. Same effect in the other substrate of disease, which John mentioned Lyso -- Plasma Lyso-GB3 which is really very important emerging biomarker in Fabry Disease. We have the same…

Great. Thank you Jay. It was an excellent overview. I will now turn the call over to Brad. Brad, I think you’ve got a number of milestones highlighting here on the slide #10, shifting now from migalastat monotherapy to a focus on our next-generation Pompe ERTs but we -- all our three-and-three strategy, one ERT into the clinic every year for the next years beginning this year with our next generation separate ERT. So Brad, I’ll turn it to you.

Sure. Great. Thanks John and good evening, everybody. I’ll just add a little bit of color to the milestones that were listed on the slide here. The Fabry as John mentioned, we do remain on track there to start our clinical development in the second half of this year. And this will be a product for roughly half of the population who don’t have addressable mutations with Migalastat as a model therapy. This will of course support our vision that eventually all patients with Fabry disease may take Amicus product as their only treatment from their Fabry. We also continue to make great progress in Pompe and development of our next generation enzyme replacement therapy there. To address what we see is really the key three major challenges in enzyme replacement therapy today. That is activity and stability uptake and targeting and tolerability and immunogenecity. Recall that we’ve already made good progress in initial pre-clinical studies of our proprietary GAA cell line AT-B200 that is unique carbohydrate structure. We’ve shown in initial pre-clinical studies that we can demonstrate superior uptake in activity compared to current standard of care, which we believe may also be further optimized through co-formulation with our pharmacological chaperones AT2220 to further improve stability and tolerability and also by applying the company’s peptide tagging technology for better targeting. Manufacturing scale-up activities and preclinical studies continued to move forward on schedule and we expect to make selection of our final drug candidate for IND-enabling tox in the second half of this year to support clinical studies, which we expect might begin in 2015. And then finally, we do continue to move forward with our MPS I program. We continue to advanced preclinical studies, looking to develop proprietary recombinant human rhIDUA enzyme as the next-generation therapy for MPS 1. So, great progress towards our 3-in-3 strategy and each of these milestones remain on track.

Great. Thanks, Brad for the overview. I'll turn it over to Chip now for a discussion on the financials.

Great. Thanks John. Good evening everyone. I’ll start today’s vast discussion with a few comments about our current cash position and guidance, which is shown on Slide #11. As indicated in this afternoon's press release, Amicus continues to maintain a strong balance sheet. At March 31st, we had $71.6 million in cash and cash equivalents which compares to $82 million at the end of last year. In terms of additional sources of funding, $10 million remains available under the debt facility we entered into in the fourth quarter of last year. We also have an at-the-market or ATM equity facility in place, which became effective March 4, 2014, and covers the offering about $240 million of our common stock that may be issued and sold under sales agreement with Cowen. We believe it’s a financially prudent practice to have both our effective shelf and an ATM facility in place. Today, we haven't sold many shares under the ATM agreement. And as previously guided, we expect full year 2014 net cash spend to total between $54 million and $59 million. We believe that our current cash position is sufficient to fund our operating plan into the second half of 2015. Turning to our first quarter 2014 financial results on slide 12, I will also be referencing Tables 1 and 2 in the press release which we issued earlier today and additional details on the financials can be found in our quarterly report on Form 10-Q, which will be filed later this evening. We recorded modest revenues of $456,000 in the first quarter of 2014, in conjunction with the Biogen collaboration which continues to progress very well. Total operating expenses for the first quarter of 2014 decreased to $16.1 million as compared to $17.3 million in the first quarter of 2013. The…

Great. Thanks Chip. So, we will shortly here get to the Q&A. I just want to summarize by stating that I think we're in a really good position in Amicus. We are just so happy to see the results and as I stated in the beginning, we've had a chance over the last two weeks with the investigators, with some key opinion leaders and now with leaders in the patient community to take them through the data on this 011 Study and specifically to describe in more detail what we are doing in the Study 012, where we expect those results from the 18-month primary endpoint here in Q3 and as Jay described with our respective strategies for the U.S. and the EU. When you think about that, we can be in a position in the second half of this year to have hopefully a successful Phase 3 Monotherapy program and a program that is unpartnered. It is an Amicus program with very modest royalties in most geographies. That is our program that we could drive forward. We absolutely could commercialize the program. There is a significant amount of potential partner interests that we are considering. Although, we said to those potential partners and to the broader public here that our bar for partnering would be very high. And with that, also in the second half of the year, it is going to be a huge occasion for Amicus to finally put our next-generation ERT that we've been working on for several years into the clinic beginning with our next-generation enzyme replacement therapy in February. So as we think about Fabry disease, currently a billion dollar market for the existing therapies, we will have two, we hope product offerings for patients and the idea would be to build a franchise in Fabry disease with our late stage Monotherapy program together with our ERT program. Monotherapy, of course for people with amenable mutation, the use of the pharmacogenomic aspect of the monotherapy to determine those patients in advance and for the roughly other half of the population to be able to provide an enzyme replacement therapy that we believe has the potential to be a superior therapy. Of course, we will put that in the clinic and test the hypothesis in man. But a very exciting time as we head into the middle part in the second half of the year. And with that operator, we are happy to take any questions.

Thank you. (Operator Instructions) So the first question is from Ritu Baral of Canaccord. Please go ahead.

Hi. Thanks for taking the question. As we look forward to the Q3 data, can you help give us an idea of what you consider, sort of non-inferiority and as we look at the data that comes out and what are the ranges or sort of the ranges of variability for the different renal function measures that you outlined in the slide, so any different threshold?

Sure. I will let Dr. Barth take the second half of that question as we think about some of those ranges, Ritu. But to just remind folks on the upfront that this is the study that begin enrolling in the early part of 2012, right after we had finished the Study 011. And at the J.P. Morgan Conference in January of 2012, people asked me what kept me up at night? We had a great conference with some of our new ERTs and then proof-of-concept data. And I said what kept me up at night was the enrollment of Study 012 that we had to convince patients and their physicians to completely give up ERT and for those patients with amenable mutations to switch to migalastat as their only treatment for Fabry disease. We were remarkably successful in enrolling that study. I think it speaks to the level of interest in patient and physicians communities to try a new therapy and to experiment at that point with our molecule migalastat. It was designed to be a 50% study we enrolled in less than a year. We overenrolled the study at 60 patients. It is a 1.5 to 1 randomization, so that we took 36 of those patients completely off of ERT switching them to migalastat as their only treatment for Fabry, 24 remained as the control arm on ERT. The primary endpoint is looking at kidney function by glomerular filtration rate at 18 months compared to baseline when they entered the study. It is a non-inferiority study using descriptive statistics showing that the switched patients perform as well in their renal function as the patients remain on ERT, which is one of the reasons why the Study 011 with the maintenance of the patient’s kidney function that we saw in that study makes us very hopeful that the data that we will see in 012 will be similar in nature. So with that background and overview, Jay, if you want to comment a little further on the end points in that study.

Sure. We will be looking at renal function in 012 in the same way as we looked at in 011 with the measured GFR is my exo with two measurements of eGFR, CKD-EPI and MDRD. And all of them will be factored into the descriptive statistics that will be used to judge non-inferiority. We have the mGFR prioritize that is the traditional and gold standard method for measuring renal function, but the eGFR measurements will certainly factor into, I think, anybody’s looking at the data to compare migalastat to ERT. And as it is based on descriptive statistics, there isn’t a strict non-inferiority margin established. Of course that’s because of the size of the patient population that it will not be possible to do statistically powered non-inferiority study in this disease. But we do have the approach with the EU and I mean discussed with EMA of using as I said the descriptive statistics in order to look at this data to determine that the results from migalastat are the same are non-inferior to ERT.

And is that -- is the final sort of delta going to dependent part on any sort of standard deviation, or is there sort of a pre-agreed, I guess, measure of non-inferiority -- delta of non-inferiority?

I think, Ritu, rather than getting into that level of detail quite here, I think we reserve that for when the data comes out. But suffice to say what we need to show in the descriptive statistics is that the aero bars essentially overlap between the two groups. There is some degree of variability patient to patient and time point to time point, but there is a good degree of consistency among these patients. So, I think, Jay, feel free to add any color, but I think generally showing the overlap at the aero bars with the feedback that we got from the Europeans which is I think an achievable bar based on the knowledge that we have from the literature from our Phase II studies and now very importantly from the Phase III study.

Thank you. Sorry I just -- I have one quick follow-up to that. Is the delta between when you guys have the data in-house and when you release the data, will that be sort of a similar time lag as the 011 Study or can it…

Yeah. Well, I mean, there was very little time lag just to give you some indication. We received that data on 011 in mid April and we had a predetermined sequester team off-side or clinical team led by Jay -- Dr. Barth here, Ritu, who took about a week in confidence to go through the data and then to share with our senior team and it was within the 48 hour period from that point when we received the data to when it we shared with key opinion leaders in confidence and within another few days until we made it public. So it was a very tight from receipt of data, internal review, external validation under confidentiality with investigators, review with our board, drafting all these documents that was all-in less than a two week period. So we would expect a similar turnaround here.

What about time between database lock and sort of deliverable data to Amicus?

Again that will all be the database lock will take place in the third quarter as well. We stated I think publicly that the last patient out of that study is later in the second quarter, so that will trigger a series of events internally into CRO to lock the database, turn it around pretty quickly and then get it out to 3Q, but I can’t give any timeframe for when those results would be public then to say in Q3.

Got it. That’s very helpful. Thanks for taking the question, guys.

You are very welcome.

(Operator Instructions) Our next questionnaire is Anupam Rama of JPMorgan. Please go ahead.

Hey, guys. Thanks for taking the question. Just on the Pompe 3-in-3 strategy you kind of outlined sort of the next steps for AT-B200, but I was just looking you give us your latest thoughts on AT2220, the chaperon and what are sort of the next steps in determining which chaperon you might take forward? Thanks.

Yes, [Rich] (ph), I will ask Brad to comment on that.

Yes, I think right now the progress on AT2220 is essentially fully developed at this point to use as a chaperon. And so the biggest question that we’re looking to answer in those preclinical studies is remember we’ve got the base (indiscernible) which had the superior we think unique superior carbohydrate structure. Then you have plus or minus the peptide tag, the variant idea to tag for further improvements in targeting. And then you have plus or minus the chaperon and what we’re really looking for from these preclinical studies is to confirm how much additional benefit you get from each of those two platform technologies that we can bring to bear. So that the preclinical work that’s underway now and we will be able to give final guidance on that in the second half of year as those studies play out.

I will just add a little more color to that to be perfectly transparent. We are in the process of final preclinical studies as Brad indicated and certainly includes in several of those arms the addition of the AT220 chaperon. We have one other chaperon that we are testing that we have been working with for number of years that has different characteristics, whether it’s better than AT2220 we will find out here very shortly. The good news is though that if we choose that molecule, it's ready for the clinic as well, so there's no further delay or no delay whatsoever.

Great. Thanks for taking my question.

You are welcome.

The next questioner is Kim Lee with Janney Capital. Please go ahead.

Good afternoon. Thanks for taking my question. I have a, I guess, hypothetical question here. Given we now know the results of Study 011, past 6 months and 12 month data, 12 month being positive, 6 months been negative. Worst-case scenario, should Study 012 data come back negative? How do you see your conversations with FDA going and kind of, can you walk us through some of big potential possibilities of how on approval could still occur? Thanks.

So, I guess, an 011 is the endpoints, a pre-specified analysis of the 12 months. That was a biochemical marker as surrogate, a very important one and we think, one likely to predict clinical benefit. The real beauty of the 011 data though is that since it ran out to 24 months, we were able to follow these patients for 18 to 24 months depending on a cohort they were in and follow their kidney function and that's where we think this study is very significant. And that kidney function remaining stable really helps to validate the clinical significance of that surrogate marker, but again that study was originally intended for approval under Subpart H. We may use that as one basis together with Study 012 for full approval. To your hypothetical -- I might still might getting it to hypothetical but in the absence of seeing any data from 012 of course, if that showed that it was significantly different than the ERTs, it would give us pause for concern and to understand why that would be because it would be inconsistent not with the biochemical end -- well, it would be inconsistent with the biochemical endpoints but much more significantly would be inconsistent with the data that we see on the kidney endpoint and Study 011. So we really have to work to reconcile that. So without getting further and hypothetical, if Study 012 is not successful, we’d certainly have to understand why and we have some pause for concern for moving forward. But the converse is also true if Study 012 is positive, we think that that combined with Study 011 would present a very, very strong dataset in the largest and what would then be the second largest studies ever conducted in Fabry disease. Taking together, we think it would be a very, very powerful dataset. So, I’d much prefer to dwell on that hypothetical, Kim, than your prior example but both valid for considerations.

Yeah. No, it’s completely understood. And just to clarify expecting the study 012 to be positive, you would considerably go for full outline approval instead of conditional approval, is that correct?

Still to be determined but I think, it absolutely be a pathway in the United States.

Great. Thanks so much.

Thank you.

The next questioner is Joseph Schwartz of Leerink Partners. Please proceed.

Hi, thanks very much. I was wondering…

Hi Joe.

Hi there. I was wondering if -- would you talk a little bit about the Fabry co-formulation program?

Yeah. Absolutely.

In particular, what should we be looking for out of the Phase 1 PK study, is there any key the analysis that’s possible there. What are you trying to achieve? I mean, how would you choose a dose going into patients in 2015? And then have you settled on the enzyme component of that strategy or it sounds like from your press release that you're leaving the door open to -- for a little bit longer to choose the agents that be complying with IV migalastat?

Okay. I was just furious writing down our notes, Joe. I think that was a couple of points to make there. So just I think, what you’re referring to first is the IV migalastat study which is ongoing right now. That is to determine the PK characteristics of the IV version of migalastat for the co-formulation. And we don’t yet have that full data set. We would expect to share that as part of the development strategy. I think, most likely to the midpoint of the year, maybe third quarter. But again that help us choose the fixed dose of chaperone that will go with the ERT combination. And your question there was PK, as well as PD.

Yeah. How are you thinking about choosing the dose in healthy volunteers, what you’re looking for, is it just, what would be adequate exposure, I guess, that could drive future development?

Sure. Again, we have significant experience with oral migalastat and we’re trying to compare that with our oral experience as well as the IV and in oral experience in animals. And Jay, would you comment further on what would we expect to see and what would be the measures in this ongoing study?

I think that’s exactly that’s you’re saying. The healthy volunteer PK study in migalastat really have lots of data from the oral studies co-administration to know what plasma exposure we’re targeting. So it's really a steady design to achieve the optimal target plasma exposure with the IV formulation. And running through ascending doses in order to determine the PK of the IV formulation of migalastat looking at those data and then making sure we are selecting the dose that puts the migalastat in the target plasma concentration. So I think that’s going to fairly straight-forward and the study is proceeding well. And I think those data will be very informative in the design the dose selection for the co-formulation study.

And again, Joe, to answer the later part of your question. So with that data here in the next few months, we will know what is the exact dose of the chaperone. The ERT has already completed manufacture and is ready pending that last piece of information to put in man. We will put that in patients, Fabry patients, a next-generation Fabry ERT in the Phase 1-2 study in the second half of this year. So that will be very big milestone for us. So that will go in. That will be the Fabrazyme biosimilar combined with chaperone to make the next-generation ERT. That ERT, of course, was sourced from GSK and their partner JCR. Again, the Fabrazyme biosimilar, we have identified several potential sources there are in or in advance discussion with several parties to source a long-term supply. Originally envisioned that the commercial supply for the base ERT, we think, however, that will be able to how that programs sets so that we can make it available for our Phase 3, so that we don't have to do any of the bioequivalent studies at BLA filing. We can actually do it as we bridge from Phase 2 to Phase 3. So we don't have a final contract for that set, but we’re in pretty advance discussions to secure that long-term ERT component of the supply.

Okay. Excellent. Thanks very much.

You’re welcome.

I’m showing no further questions in the queue and I would like to turn the conference back for any further remarks.

No. Just thank you all this evening. Thank you for the many conversations especially with the analyst over the last week. As I indicated, we’ve had a chance over the last two weeks now to talk a lot about our monotherapy migalastat program with physicians, scientists and the patient community. We over the next couple of weeks are going to engage in a concerted effort to visit with investors and analysts to explain more of the data and specifically, our strategy for Fabry franchise based on migalastat monotherapy. What we see is defining success with the 012 study and very specifically then getting into the broad franchise strategy of monotherapy and the addition of the second product, the next-generation ERT. So, anybody who would like to be added to that schedule that’s just coming together, please feel free to reach out to Sara, and certainly, through any of the excellent analysts on the call. And that’s all I have, Operator. Thank you very much.

Thank you. Ladies and gentlemen, thank you for participating in today's program. This does conclude the meeting and you may all disconnect. Everyone have a wonderful evening. Thank you.