Good day, ladies and gentlemen. And welcome to the Amicus Therapeutics First Quarter 2013 Results Conference Call. 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 call maybe recorded. I would now like to introduce your host for today’s conference, Sara Pellegrino, Director of Investor Relations. Ma'am, you may begin.

Good evening. And thank you for joining our conference call to discuss Amicus Therapeutics first quarter 2013 financial results. Speaking on today's call we have John Crowley, our Chairman and Chief Executive Officer; and Chip Baird, our Chief Financial Officer. Bradley Campbell, our Chief Business Officer; and David Lockhart, our Chief Scientific Officer are also on today's call and available to participate in the Q&A. Today’s prepared remarks coincide with the slide presentation that is now available on our corporate website at www.amicusrx.com. The slides are located in the Investors section under Events and Presentations right below the webcast link to today’s call. Turning to the slide two, you will find a reference to our Safe Harbor statement. This presentation 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, 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 this expectation 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, 2012. 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. 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. So, as you see on slide three, I’ll go to the first two sections here. The update on the migalastat monotherapy program in Fabry, as well as the chaperone-ERT combination program, and turn it over to Chip to go through the details of Q1 financial results, and then I’ll come and handle the milestones, and certainly, as we always do turn it over to the Q&A. Let me just begin by commenting that as you see the first quarter really was one where we focused on execution, primarily the clinical operations execution for migalastat monotherapy, as well as the regulatory strategy and interactions around migalastat monotherapy as well. You saw from the press release that we issued that we have a Type C meeting now scheduled and in person meeting with FDA this quarter, that meeting is not yet occurred, although the briefing documents have all been submitted to the agency. And again, with the CHART programs the company continues to evolve more and more in addition to our monotherapy program toward developing these programs, multiple program using the CHART or Chaperone Advance Replacement Therapy Platform Technology, the goal here of course to develop next-generation proprietary enzyme replacement products for the lysosomal diseases and potentially beyond. So that’s the overview of what we are going to cover. Let’s go ahead and please turn to slide four, and again this is here for reference I’ll be providing some more statistics around these studies as well. As we know that we have two Phase 3 studies for monotherapy in Fabry disease, Study 011 and Study 012. Study 011 is an ongoing Phase 3 study looking into the use of migalastat, given 150 milligram at the other day compared to placebo in patients with Fabry…

Great. Thanks John and good evening everyone. I’ll start today’s financial discussion with a few comments about our current cash position and guidance starting on Slide 6. As indicated in this afternoon’s press release, Amicus continues to maintain a very strong balance sheet and we’re on track to achieve our full-year operating expense guidance. At March 31st, we had $84.8 million in cash and cash equivalents compared to $99.1 million at the end of last year. We continue to estimate full year 2013 net cash spend between $52 million to $58 million and that’s net of anticipated cost sharing under the GSK collaboration. As a reminder, GSK is responsible for 60% of the global development cost from the migalastat for Fabry disease, 2013 and beyond. Our current cash position along with the anticipated GSK reimbursements, are projected to fund our current operating plan into the second half of 2014. Turning over slide seven, you will find a snapshot of our first quarter financial results, which you also appear in tables one and two of this press release we issued early today. Additional details can be found in our quarterly report on Form 10-Q, which will be filed later this evening. For the three points ending March 31, 2013, no revenue was reported whereas we recorded total revenue of $7.8 million in the first quarter of 2012. The year-over-year decrease is entirely due to a change in revenue recognition accounting under the expanded GSK collaboration. This accounting change does not impact cash and we will explain this in further detail on the next slide. Moving down to P&L, total operating expenses for the first quarter of 2013 totaled $17.3 million compared to $18.5 million in the first quarter of last year. The year-over-year decrease was primarily attributed to lower research and…

Great. Thank you, Chip. So on slide 9, I will go ahead and just summarize. Again, Q1 was a really important quarter for us to buckle down to work on the clinical executions, work on the regulatory strategy for monotherapy, to make sure all the rest of the pipeline advances. And I’m very pleased with the company’s performance in the first quarter and heading now into midyear. If you look on this slide, you can see the migalastat monotherapy. Milestones going forward are very importantly now after this Type C meeting. The first important piece of communication of course is going to be the topline study 011 12-months data in Q3 and then providing, if that looks positive we would move forward with an FDA meeting, Pre-NDA meeting to discuss the U.S. approval pathway in the second half of this year. I can tell you, getting even deeper into the data with all the work that we did to prepare these documents to the FDA, the briefing documents and preparing for the meeting, I feel even more confident that the data shows that the drug is having a positive benefit in the patients for whom it’s intended to benefit. So consistent with what we saw in Phase 2, we think that this could be potentially a very, very strong option for people with minimal mutations, living with Fabry disease. Pompe is again an increasingly important part of Amicus. The Pompe ERT co-administration program, the first data was released at that WORLD Conference back in -- the complete data I should say was released at the WORLD Conference back in February. We continue to move forward with that on target from a timeline perspective, working on finalizing protocols with lead investigators in the world in Pompe disease. And we expect to…

Thank you. (Operator Instructions) Our first question comes from the line of Anupam Rama [J.P. Morgan]. Your line is open.

Hey, guys. Thanks for taking the question. Just a quick question on Pompe program. Have you finished all the formulation runs for AT2220 and how many doses are you going to be studying in the repeat-dose study? Thanks.

Yeah. So we finished all that formulation work and check back in early to mid Q1. We did the aseptic fill/finish with the contractor that was completed successfully. So the formulation is in very good shape. It’s in its final top studies and so far so good. In terms of the protocol, we are just finalizing that now on the firm I would imagine. In the next month or two, we will be able to provide more clear guidance in terms of what that study is going to look like from a repeat-dose perspective. But I could tell you and we’ve shared this publically before that we are considering this to be a 12 to 24-week open label study. We are just finalizing a lot of details right now.

Okay. Thanks for taking my question.

You bet.

Our next question comes from the line of Joseph Schwartz with Leerink, Swann. Your line is open.

Great. Thanks very much. I was wondering if you could talk some more about the interactions with the FDA that you’ve had and they expect to have. And in particular, if you could give us some insight into what the main argument, do you think that might resonate with the FDA based on the existing data, first of all and then -- what would be positive data that you think would enhance your case from a 12-months end points?

Sure, Joe. I want to be somewhat sensitive since these discussions are going on in real time. But when we first got the data, we -- obviously we reviewed with the Board and we immediately released it and our partners at GSK. We didn’t have plans for Type C meeting prior to unveiling the stage to 12 months data. However, given the FDA’s guidance that they would consider the entirety of the data such that there is not one primary endpoint that would be just positive, our Board and I felt very strongly that it is better to interact with the agency as frequently and as in depth as possible to try to understand what that means. And I think a good part of that reflects the very changing regulatory environment especially in the rare and orphan diseases where there is still such unmet need. And the FDA, I think much to their credit working very hard to try and get figure out the right and best solutions to bring these medicines forward especially under PDUFA 5 and fiduciary. So with that we went ahead in early Q1, requested a Type C meeting. Again, the FDA didn’t have to grant it in the middle of an ongoing phase 3 study. They very quickly came back and said yeah, that’s fine and they agreed to make it an in-person meeting. That led to quite a bit of work and activity here for probably better part of six to eight weeks to draft that briefing document. In terms of -- I can’t release it, of course, within there, it’s very important that we maintain those confidences going into this FDA meeting. What I can tell you, though, is its not cherry picking or cutting the data. Even that point three cut-off that…

Okay. It’s very helpful. Thank you. Can I just as a quick follow-up as far as your plans to share with investors, the insights that you’re able to gain. Can you give us some more granularity on when and what form that might take?

Yeah. I think we need to go to that meeting. First, we need to digest the results with GSK. Our bias at Amicus is always to be as transparent and real time as possible. Of course, they need to be respectful of the GSK needs. So I think it’s unclear right now, Joe, whether we would be able to release those, the feedback from FDA and to provide further guidance on with any changes or addition to this statistical analysis plan could be in advance of the 12 months data but certainly it would come with at lease come with the top line’s 12 months data in Q3, so kind of stay tuned.

Thanks again.

Yeah.

Our next question comes from the line of Greg Wade with Wedbush. Your line is open.

Good afternoon and thanks for taking my questions as well. John, with respect to the -- are you closing with the FDA a new statistical analysis planned for the 12 month’s data?

We’re proposing a revised statistical analysis plan. So our original plan Greg had that primary endpoint being the six month responder yes/no the percent of patients, you show that 50% had greater reduction. That’s would have been in place for several years. If you recall we had a Type C meeting last summer, where we went through a number of different aspects of the program. And the FDA discussed with us potentially expanding that to look at the entirety of the data. We actually put in place additions to the statistical analysis planned back in the fall of last year. And now we’re looking for further additions to the statistical analysis plan. So this is somewhat unchartered territory. The advantage here for us is that the FDA has already characterized that six months as interim Stage 1 data. The FDA is viewing this as a 12-month study for safety and for efficacy. And again it’s somewhat unusual because they are looking at the entirety of the data on this risk benefit analysis. And what we’re trying to do again is to define as best we can what success is going to be when that 12-month data comes. So we can communicate certainly to investors but also to a number of key interested parties in putting physicians and patients and very importantly be able to set up the guidelines and the grounds and the framework for that pre-NDA meeting in the fall. Does this answer your question?

It does. I think we’re trying to get from this phone call is the parameters around which you’re looking to change things. So for example, if you are successful in convincing FDA to allow you to look at their continuous variable as the primary endpoint in the 12-month study. Well that would more likely because of the success of the six months study lead to potentially statistically significant result. There is also the flipside too which is the risk right. So on the continuous variable side, if those responders show less of the response at 12 months then that would suggest not a durable impact of therapy and obviously with this being a chronic disease, that’s not going to be good for business perspective. So I think it will be helpful to us to understand what your preference is with respect to this negotiation of the statistical analysis plan. So that we at this end can make an assessment of….

Sure.

What the risks are is that -- is what you end up getting?

Yeah. I’ll answer and then I’ll ask David to add just a little more color to it to make sure I capture it all accurately. So our preference and we think the better way to look at this is rather than a responder pure yes or no analysis to look at the overall burden of GL-3 in the cell and the reduction of that overtime and as such we think the continuous variable is going to be a more accurate and more telling way to see what the impact is actually going to be. So let me just comment that we -- there is not going to be one primary endpoint at 12 months. The FDA has already been very clear that given the nature of this disease, it will be the entirety of the data including the safety components. What we do though want to do is to emphasize the importance and elevate the importance of that already pre-specified secondary analysis. So it becomes a very important part of the FDA analysis at 12 months. Whether it’s just positive or not, it’s ultimately going to be seen at, I think, at pre-NDA meeting. But I think it certainly helped give you guys because I understand there is some confusion, Greg, in terms of how do we view success. I do agree with you that if we were to see the migalastat treated arm regress at 12 months that would give us some pause for concern as to whether or not this is having the effect that we saw in phase 2 and is persistent and durable. But I think we need to look at the data to make that judgment. David, anything to add please?

Hi, Greg, this is David.

Hey, David.

So just to follow up with what John said, so we’re past, there is now primary at this point. I mean, we’ve past it that was a six months. And so at this point our job is to put together the best view of the data and what it tells us about the treatment effect of the drug and about the safety of the drug. And so it is by its very nature a multi-component and so it will -- there are multiple part of the analyses. And there would be multiple analysis to from different ways of looking at the data in order to determine whether there really is a clear treatment effect in the patients with the amenable mutations and -- versus patients who were either on placebo for the first six months or who do not have amenable mutations. So it really -- there is no simple answer to saying what do you hope to see on the primary because there is no primary. We’ve have past the primary. But when it comes to the durability of the fact that the key is that for the first six months, half the patient were on drug and half the patients were on placebo. And then at six months to 12 months, everybody is on drug. So we have the chance to see whether the initial placebo group when they’re on drug for six months, whether that looks like what happened in the original group that was on drug for six months. And then we also get to see the patients who were on drug for the first six months, if they were on drug for subsequent six months whether they have maintained what they -- what we saw in the first six months and whether that got even larger. So I think exactly you said as in any study, there is a chance that the data won’t go in the direction you hope but we have reason to believe that with longer treatment things get better because that’s exactly what we saw in phase 2. So you’re exactly right there is always a chance that it won’t go as hoped, as with any study. But we think we have the right analysis in place in order to make that assessment. And we have reasonable confidence that there will be both a durable effect and potentially even a greater effect when the patients are on longer based on what we saw in phase 2.

Okay. And just lastly based upon the things you’re thinking about, do you think you’ll have to have sequence level efficacy results by genotype in order to support the use of the drug across the board in patients or will we use biochemistry or chemical analysis of the amenable mutation? Thanks.

John, do you want me to answer that.

Yeah. I’m sorry. David, go ahead, please.

So, Greg, so the selection of the patients that we -- that should go on drug will be -- we expected to be the same as what we’ve done in phase 3, where that decision is based on their mutation. So it is based on sequencing. It is based on the sequencing of the entire coding region of the GLA gene and the patient that have a mutation that we have determined in our cell-based assay to be responsive to the drug. Those are the patients that we believe should go on the drug. And those that do not have such mutation are ones that we think should not be on the drug and that’s exactly what we did in phase 3 as part of the inclusion and exclusion criteria. So we expect it will be consistent with what we did in phase 3 based on the mutation, based on DNA sequencing.

And in the phase 3 results that you have presently, you believe they’re supportive of identifying the right patients because the response rate looks like it doesn't quite match up with the number of patients that were included?

So the answer to that is yes. We do believe that and we have data from phase 2 as well that’s supportive of being able to identify the patients who are likely to respond based on their personal mutation. So, yes, everything is pointing in that direction, of course, that we will be getting more data as part of the phase 3 study and we will be getting more data for patients who for example on the first six months, they were on placebo and with -- what we now know to be non-amenable mutations who for the second six months will then go on drug and will see if that, whether the amenable group showed an effect where the non-amenable group didn’t.

Okay. Great. Thank you.

Operator. Yeah, go ahead.

Our next question comes from the line of Kim Lee with Janney Capital. Your line is open.

Good afternoon. Apologies if you already addressed this but I got out paunch, what exactly do you expect from this Type C meeting besides going over there the statistical plan? Thanks.

I think we actually did cover that in pretty good detail, Kim. So, I think that’s in the transcript but I will just kind of highlight that what we did was to prepare a very thorough briefing document based on the six months Stage 1 analysis, the interim data for the 011 Phase 3, and a more detailed analysis of all of our Phase 2 studies to add to this statistical analysis plan, focusing on a number of different element including this notion of the continues variable that was a pretty specified secondary endpoint. But to elevate the importance of that in the assessment of the drug from a risk benefit prospective in terms of declining the entirety of the data. So, I think it will be very important to have this good exchange with the regulators to be able to explain and lay out the data as we see it. We think again it shows very encouraging results that the drug is having a positive benefit in those patients with amenable mutation. And as best we can to frame, how we should look at the entirety of the data once this 12-month data is unveiled. Again, we and GSK are blinded to the data still. It’s a very unique and important aspect of this study to remember that the primary reason, maybe the only reason that we can add to the statistical analysis plan in an ongoing Phase 3 is that we have not lock the data base and we still remain blinded to that 12-month data, although remember of course all those patients samples were collected by the end of 2012.

Okay. And just as a follow-up and I appreciate you reviewing this.

That’s okay.

But also what time show will this be -- with the FDA after the Type C meeting or could thing still be flexible and move around and change as far as agreements go after you on blinded data?

It certainly could be subject to further analysis and interpretation after the 12-months data. We are trying before we get to the 12-months data to as best we can define what the key elements of analysis should be. So stay tuned we will see I think I’m very, very pleased with the FDA accepting this request for Type C meeting. They are making personal meeting down in Maryland and I think hopefully speaks to there interest in seeing therapies for Fabry advance.

Great. Thank you.

You’re welcome.

At this time, I’m not showing any further question in queue, I would now like to turn the call over to John Crowley for any further remarks.

Great. Operator, that’s all I have. Thank you, everybody for listening to the great questions and a lot more to come in the months ahead. So, thank you.

Ladies and gentlemen, thank you for participation in today’s conference. This does conclude today’s program. You may all disconnect and everyone have a great day.