Sara Pellegrino - Director, Investor Relations John Crowley - Chairman of the Board, Chief Executive Officer Hung Do - Senior Vice President - Discovery Biology Jay Barth - Chief Medical Officer Bradley Campbell - Chief Operating Officer Chip Baird - Chief Financial Officer.

Ritu Baral - Cowen Anupam Rama - JPMorgan Joseph Schwartz - Leerink Partners.

Good day, ladies and gentlemen. Welcome to the Amicus 3Q 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 is being recorded.

I would like to introduce your host for today's conference, Director of Investor Relations. Ms. Sara Pellegrino. Ma'am, you may begin..

Thank you, Vince. Good evening, and thank you for joining our conference call to discuss our third quarter 2014 financial results. Speaking on today’s call, we have John Crowley, our Chairman and Chief Executive Officer; Chip Baird, our Chief Financial Officer; Jay Barth, our Chief Medical Officer; and Bradley Campbell, our Chief Operating Officer.

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 Investor section under Events and Presentations, right below the webcast link to today’s call. On Slide 2, you will find the reference to our Safe Harbor.

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

Great. Thank you, Sara. Good evening, everybody and it certainly good to be back full-time here at Amicus after my several months leave for a military reserve duty, and I find Amicus in an even better position than when I left the company at the end of this summer, so I am very excited with the future here in Amicus.

I will only make some very brief opening comments, since we have a lot of substitute materials to work our way through with the senior team that I have here and I will draw your attention to Slide number 3 in the third quarter investment highlights.

There are a lot of great rare disease companies that are out there today doing many great things in the field and I think Amicus, in that field, is very uniquely positioned.

If you look on slide three, we state that we have strength of our clinical program and the breadth of technology programs with the potential to create significant value for shareholders and patients living with lysosomal storage disorders. I think that has never been truer. Obviously, we were very pleased and excited with Migalastat monotherapy.

Again, an unpartnered post Phase 3 program preparing for its regulatory filings outside the United States as well as here in the United States, again, the first oral therapy with unique mechanism of action, very distinct from enzyme replacement therapy for Fabry patients with the amenable mutations. Now, again, with two positive Phase 3 studies.

As Jay will highlight here, we are on track for an EMA pre-submission meeting this quarter, so very, very valuable program for us and I think will offer the potential for great benefit for people living with Fabry, but beyond a pre-commercial global asset in a rare disease, we also have a terrific pipeline and we will talk more about that, because we have had some terrific advances in the pipeline that will go through with that 3-in-3 strategy, the notion of three new next-generation enzyme replacement therapies into the clinic over the next three years in Fabry, Pompe and MPS and we will spend a bit of time today on our Pompe program as that met some great milestones over the past quarter.

Again, the notion that we not only have a pre-commercial asset, we have an expanding pipeline and a series of great proprietary technologies, so that is where we find ourselves at Amicus today. We have more data and we highlight this in the press release that just came out.

Additional long-term Phase 3 data, very important data that will be presented at the American Society of Nephrology coming up here in a couple of weeks, we of course had some additional long-term Phase 3 data presented in October at the ASHG, The American Society of Human Genetics that give us further confidence in Migalastat as a monotherapy and Jay will highlights some of that.

I have got Dr. Hung Do and Brad Campbell, who will talk more about our next-generation Pompe ERTs. It has been almost a year since our acquisition of Callidus and Hung rejoining the company in a senior role in the science organization and that program could not have gone better over the last year.

We will talk specifically about some good success we had in the scale up of that as well as a very recent first regulatory interaction with the European authorities that Jay will provide an update on that and Bradley as well. Across the board, I think we are in a very, very good position.

With that, let me turn the call now over to Jay and he will take us through the next couple of slides focused on Fabry monotherapy..

Thanks, John. I want to remain everybody in Slide 4 of the two studies that have been conducted, Phase 3 studies that are part of our global registration plan. Having a robust data set at this point as we do really puts us in very good position for global approvals of Migalastat monotherapy, for the patients with the amenable mutation.

Just to remind everyone, first Phase 3 study shown here is the Facets Study, and this is studying with 67 patients who naïve to ERT were enrolled. The first six months were placebo-controlled. We have those data and the primary endpoint was the reduction in substrate (Inaudible) in the kidney, looking a kidney GL-3 at 6 and 12 months.

The secondary endpoint was the kidney function, which was measured both, by estimated eGFR and measure GFR, mGFR at 12 and 24 months. These are the data that we had first presented before and I will be discussing a little more of that shortly.

I am on the slide of the ATTRACT Study, our second Phase 3 study and this was a study that enrolled 60 ERT experienced patients and 36 were switched Migalastat, 24 remained on ERT.

The primary endpoint that we looked at here was comparability of Migalastat ERT based on the eGFR and the mGFR over 18 months and versus we had mentioned previously that study did meet the primary endpoints of showing comparability to ERT on both measures of renal function. On Slide 5, we have some specific data that further validates the assay.

The GLP HEK assay that we developed to identify patients who have amenable mutations, very briefly, but you are looking at our individual patient's data for their interstitial capillary GL3 and amenable patients are in blue non-amenable patients are in red.

We can see looking at this slide, that the amenable patients had decreases from baseline in the GL3 and the non-amenable patients had no change or increases in their GL3, so clearly the assay is effective in distinguishing patients who have amenable mutations and this is a key part of our regulatory strategy as well, so having these data are able to support that point in a very strong way.

In terms of the functional outcome, -- to the Slide 6, the data we presented ASHG, in October for a long-term kidney function, so even now since patients had continued from study 011, the FACETS Study into an extension study an open-label extension and patients are continuing as we speak.

We can measure their kidney function over a longer and longer period of time and at the time of reaction. We could report on data for 32 months, almost three years of renal function data. The longer term data, is even more meaningful for kidney function decline is a major cause of morbidity and mortality in these patients.

One can see from the results for eGFR by two methods, CKD-EPI and MDRD, that the change in GFR was negligible over a 32-month period. The stability of the kidney function compares very well to what's in the literature for Fabry patients. As Dr.

(Inaudible) who's a nephrologist expert in the field of Fabry disease says you can see that if were ever to look at patients with similar levels of proteinuria, you can see that the Migalastat-treated patients are more stable than untreated comparative patients from the literature.

These data are very strong for the long-term kidney function and that study open-label extension is continuing as we gather more and more data to support that. The next slide, Slide 7, shows highlights what is upcoming and these are the study 12 results, very high levels as mentioned.

Previously, which we are able to present the data in some details and it includes of course the kidney function data as well as new data on cardiac endpoints as well as other important secondary endpoint.

It is going to be a poster presentation and you can see the information there, at the American Society of Nephrology Meeting in Philadelphia, and it will be presented by Dr (Inaudible) who is investigator and expert, who has been working with us for years. We are very excited to able to share that data with you in just a little over a week.

The next slide, Slide 8, really puts together the patient experience that we have at this point with Migalastat, which is very extensive in the rare disease settings. There are 97 patients today who are on Migalastat is their only treatment for Fabry disease.

The total patient years of therapies 377, very substantial and the maximum years on therapy right now is over 8.5 years and continuing, so patients have been on this treatment - only specific Fabry disease for a very long time.

The fact that the retention rate in to next study is 96% very high, it shows the patients satisfaction with the treatment and their response to the treatment, so all these data supports the clinical trial data from our Phase 3 data and our regulatory strategy. That's what is highlighted on the next Slide, Slide 9, which shows in both, U.S.

and Europe how we are pursuing the fastest path to have approval from Migalastat in these regions. Starting on the right side, in Europe, the centralized procedure is underway. As John has mentioned, a pre-submission meeting is occurring this quarter. The regulatory pathway is very clear.

I think it is primarily our non-inferiority of profitability to ERT in Study 012, which is has been demonstrated by using both of the co-primary endpoints and these all leading toward the MMA submission in 2015, so that is on track.

In the U.S., we now have, as I said the robust data set, from both of the Phase 3 study as well as the long-term clinical trial data eight-plus years and the extension study and all of these data across all the endpoints are going to be discussed in the meeting with the FDA in early 2015, where we will be discussing the regulatory path in the U.S, so that is very much underway as well..

Great. Yes. Thank you, Jay. Again, just to emphasize the importance of the presentation next week at the ASN meeting with the cardiac disease being increasingly known to be a cause of mortality in Fabry in a complications something, you know, the data I think will be very, very, very relevant, so we will be eager to share that.

Let me turn the conversation now over to Dr. Hung Do. We are going to talk about our next generation enzyme replacement therapy for Pompe disease that is a program that is showing great progress.

Hung will review for us some of the unique characteristics of ATB200, which is our next-generation ERT and Pompe, distinguished from some of the work we've done comparing that to Lumizyme, both in the analytical and in-vitro setting as well as the in-vivo settings, so I will turn it to Hung and then Brad will highlight some of the advancements this quarter in that program that keep us very much on track..

Great. Thank you, John. As a reminder to everyone, Amicus developed our own propriety replacement enzyme therapy for Pompe disease designated as ATB200 as alluded to by John. ATB200 was developed and was up in glycosylation for improved drug targeting. It has distinct glycosylation patterns from any existing ER rhGAA ERT to-date.

It contains significantly higher amounts of mannose-6 phosphate for binding the intended mannose-6 phosphate receptor. This receptor is known to be utilized for uptake in delivery of this enzyme to target cells. As shown on Slide 11, this difference in glycosylation and significantly better receptor binding is showing these particular graphs.

We performed multiple animal studies to evaluate ATB200 relative to Lumizyme. Based on a number of different studies, our data indicate that ATB200 is significantly better for improved uptake in target muscle tissues as well as the corresponding glycogen clearance. These data are highlighted on Slide 12.

When in combination with the pharmacological chaperone we see that the reduction clearance is further enhanced. We believe that our next-generation Pompe ERT has significant advantages over the current ERT and we believe these data up to this point suggest that it should be a superior treatment for Pompe disease..

Great. Thank you, Hung. That was an excellent summary and I am sure we will have some follow-up question. Let me turn the call over to Brad and on the next slide, Slide 13, Brad if you would highlight the 3-in-3 strategy and the pathway to the clinical and begin with a discussion building on Hung's presentation, where we are with that Pompe program..

Great, thanks John. As John mentioned on Slide 13, we do go through some of the key milestone that we have achieved and expect to achieve over the course of the year. I would like to expand a couple of them here, because I think it shows the great progress we have made on this exciting program.

In the third quarter, we did in fact complete our manufacturing scale up from the pilot clinical preclinical scale that we had at the begin of the year all the way to the scale that will be used to support both, our IND-enabling tox studies as well as our clinical studies and during that process we are able to maintain those key quality attributes in particular the mannose-6 phosphate content that Hung alluded to which is so key to the superior attributes of this program.

We also got to seek scientific advice and in fact recently had a very positive meeting with the EMA, where we gained initial feedback on the program going forward. I will share a couple of those details with you here now.

In particular they did acknowledge that there remains a significant unmet need in Pompe disease and they were supportive of our proposed clinical trial enabling tox program, so that means we are on track to start those IND-enabling studies at the end of this year.

Importantly also, and this is a question that we have gotten quite a bit, they did acknowledge that given the rarity of Pompe disease and the proof-of-concept that we have already demonstrated that our clinical study will evaluate, the effect of the combination therapy rather than needing to show the individual molecules the chaperone and the enzyme replacement therapy and their impact, so very positive and very important feedback from the European agencies.

We will seek the U.S. regulatory feedback as well. Most likely sometime in 2015, we will be a further guidance on that as we get closer, but I think very strong progress in the Pompe program and very exciting for patients and for shareholders as well.

On the Fabry next generation enzymes replacement therapy program, again there too we have made great progress.

We did complete our manufacturing activities, we also reported earlier that we had completed our IV Migalastat study in healthy volunteers, where we look to learn more about the dosage and selection to use in the next study in Fabry patients.

However, we would reiterated at this time that we are holding the start of that study in Fabry patients pending the outcome of several business development discussions, we do plan on providing an update here in the fourth quarter on our next-generation ERT development strategy and we will provide guidance there later in the quarter.

Again, great progress in both of our key programs, we continue to make good progress on our earlier enzymes replacement therapy programs as well and I look forward to providing further updates as they come.

John?.

Great. Thanks Brad, excellent. Chip, I will turn it to you for the financials..

Great. Thanks John. Good evening, everyone. I will start today’s financial discussion with a few comments about our current cash position and guidance. This is shown on Slide 14. As indicated in this afternoon's press release, Amicus continues to maintain a very strong balance sheet.

At September 30, 2014, we had $85.2 million in cash and cash equivalents. This compares to $82 million at the end of last year. As a reminder, we significantly strengthened the balance sheet in the second quarter through the successful execution of a $40 million after market equity financing.

We begin sales under the ATM in May and sold final block of shares in early July. In terms of additional sources of funding, we still have $10 million remaining available under the debt facility we entered into in the fourth quarter of 2013. 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 current operating plan into 2016. Turning to our third quarter 2014 financial results from Slide 15, I will be referencing tables 1 and 2 in the press release we issued earlier this afternoon.

Additional details will be found in our quarterly report on form 10-Q, which will be filed later this evening. After the quarter, we recorded modest revenues of $293,000 compared to $39,000 in the year ago period. Our revenues were recorded in conjunction with the Biogen collaboration, which as we disclosed last quarter, has since concluded.

Our total operating expenses for the third quarter of 2014 increased to a little over $17 million compared to $15.2 million for the third quarter of 2013. The year-over-year increase here was primarily due to increases in our research and development costs.

Net loss attributable to common stockholders in the third quarter of 2014 was $17.1 million compared to net loss of $14.6 million in the third quarter of last year. Net loss per share of $0.22 in the third quarter was narrower, than the net loss per share of $0.29 in the year ago period.

The narrow net loss net loss per share versus the year ago period is attributed to an increase in the share count compared to last year period. As of September 30, 2014, we had approximately 79.3 million shares outstanding and this compares to 49.6 million shares outstanding on September 30, 2013.

This summarizes our key financials for the third quarter of 2014 as well as our 2014 full year guidance. I am happy to address any questions on the financials during the Q&A session. For now, I will turn it back to John..

Great. Thanks, Chip. I have again never seen Amicus in a better position. We have never been busier and there is a real palpable sense of excitement around the company as these programs advance. With that, operator, I will turn it back and we are happy to take questions..

Yes, sir. (Operator Instructions). Our first question comes from Ritu Baral of Cowen. Your line is open..

Hi guys. Thanks for taking the question. I apologize if you addressed it. I scrambled on when Jay was on Slide four or five, but how do you look at the data package that you have right now in terms of how FDA might interpret this in the path forward in the U.S.

What do you have right now that you think the agency would find most compelling and what may come out on the 15th that has a high level of meaningfulness for the agency?.

Yes. I will just comment briefly and then ask Jay to comment. You know, Ritu, when we last met with the FDA in the second quarter of 2013, we had a much more limited data set and we have today. In that meeting, the FDA said that they would need to see the 012 Study result in addition to 011. We have now done that and done that successfully.

They also said they would consider the totality of the data. Considering where we were, we see that as great progress and we think it will form the foundation of a very strong filing with the US FDA.

We have, we are more advanced and I think there is a more clear path with the Europeans and we have gone down that path immediately after getting the 012 data in the third quarter.

As Jay indicated, we will have that meeting this quarter, the pre-submission meeting with the European authorities, but we would expect that to follow very quickly in the first quarter, with discussions with the U.S. FDA on the path there, so we have a lot more data.

It is much stronger data than the interim data set that we presented in the second quarter of 2013.

When it comes to showing the 012 data set, we feel very comfortable that that will support filing and this whole notion of the totality of the data certainly 011, 012 a long-term Phase 2 study data, as well as now this important data on the secondary endpoints including the cardiac outcomes that would be part of the filing.

Jay, see if you have any color to fill in?.

I would love to go into more detail about that, but some of - we will have to wait (Inaudible) but as John captured totality of the data has only grow over time from what we had discussed initially. First the focus has been and still is on the substrate reduction and stability of renal function. Those are very critical.

With additional data that we been supplemented what we had mentioned before in terms of other endpoints, cardiac included really makes the benefit risk assessment clearly weighted toward the benefit to patients and I think that really puts us in a good place when we talked to FDA.

Agree? Is that clear, Ritu?.

Got it. Yes. I guess, I will ask this question again after the 15th when we have got the full data set.

As far as your business development discussions, what is important for us to keep in mind in the deals that you may be pursuing? What do you want from those and where do you want assets to take you?.

Again, here I just comment briefly and then ask Brad to add more color, right. You characterized the deals we are pursuing, I would actually characterize it as deal and partners that are pursuing and it is a nice place to be in after 011.

Certainly 012, a number multiple parties all very capable organizations approached us about partnering and commercializing Migalastat either globally or ex-U.S. Our intention right now is that Amicus has the capabilities to build the global infrastructure to commercialize Migalastat.

Until somebody can convince us that we can do it better partnered with them than we can do alone in terms of our mission to deliver the drug to patients and to create maximize shareholder value. That's where we are. We are entertaining those discussions.

Some of them are more intense, of course, but right now we have to go it alone strategy that we think we have the capabilities and the experience globally to sell that drug. With that, we have also had people interested in a broader Fabry franchises that would include the combination therapy and a next generation enzymes therapy in Fabry.

We had people approach us with broader interest including Pompe and the platform. Pompe is one we really feel strongly that we need to take that into the clinic, so we have been less open to those overtures.

Obviously, an extraordinary deal with something we would consider, but again with the standard that somebody would have to convince us that it builds enormous shareholder value and gets the drug to patients sooner and better than we can do alone. Brad, I will let you if is there anything to add..

No. I think that was a very complete response, John. I think that is exactly how we are thinking about. The good news is, as John said, we have a lot of options that table.

The bar is high for doing any those deals, but the key will be whether we can increase the value for shareholders and for patients and that is the yardstick we used to evaluate, we have in front of us..

I find, Ritu, you are either popular in this business or you are not. There is not a lot of middle ground and it is nice to be in the popular category..

Got it. Thanks for taking the question, guys..

Okay. Thank you..

Thank you. Our next question comes from Anupam Rama of JPMorgan. Your line is open..

Hey, guys. Thanks for taking the question..

Welcome back to JPMorgan, Anupam. Hi..

Thanks. Thanks a lot. I had a question on the 3-in-3 strategy. It sounds like we are going to get an update on Pompe disease and Fabry disease by the end of the year.

I was wondering, by the end of the year, if we might get an update on MPS I as well just in terms of what you guys have been doing on that program, maybe some pre-clinical data sometime in 2015 on what an enzyme kind of looks like. Just wondering what is going on behind the scenes on that program. Thanks..

Yes. I would probably look to your conference in January is when we would have a more robust discussion around that program.

There is some important work going on right now in the company, but we have got so much news between now and the end of the year in our monotherapy Migalastat program and in the Fabry and Pompe next-generation ERT programs, I think we will hold the MPS data to the early part of the year..

Okay. Great. Also, can you give us an impression of what you hear from physicians about enzyme replacement therapy in Fabry disease in terms of cardiac improvement, the literature we have looked at seems a little bit mixed. I just wanted to hear some perspective from you guys..

Yes.

Jay, please?.

I think you characterized it well. For the literature on the arcane Cardiac, there is not a lot of data to support it. There may be some effect of the ERT on the heart, but it's not a good really ideal treatment for the cardiac aspects of the disease.

That is something we feel is a need that needs to be met that there's room for improvement on what ERT can offer in the realm of the cardiac involvement with Fabry disease. .

Thanks for taking my questions..

Thank you. Our next question comes from Joseph Schwartz of Leerink Partners. Your line is open..

Hello, Joe. If you are talking, we can't hear. I am sorry..

…muted, but anyway congrats on the progress. I was wondering, first of all, if we could talk a little bit more about the Pompe ERT program for ATB200, in particular, I know it has been a challenge for other companies, so just curious about how you are feeling about the scale-up and expression levels of that enzyme.

Then what are you thinking about at this point for the design and endpoints from the Phase 1/2 study to start next year?.

Yes. Just on that latter point, Joe. We are not yet prepared to talk about the clinical aspects of that program.

The EU meeting that Jay just got back from a few days ago was focused on pre-clinical CMC toxicology and we get great feedback and I was particularly struck by just how explicit the European regulatory authorities were in noting the unmet need on multiple levels with the existing standard-of-care, so we are not yet ready to talk about our clinical strategy.

We were still working with some experts on that, but Brad if you talk about how well the scale up has gone and the characteristics of that..

Sure. Joe, it is a great question. As you might recall, John and myself and Hung, were all at Genzyme, so we lived some of the early days and Dr. Do….

End nights..

…were there. They know as I am previously working on enzymes replacement therapy for Pompe, so lots of experience here in that process and understand full well the challenge there. I think part of, it just backing it with the benefit of experience.

I can tell you that while it is taking a lot of hard work and a lot of long nights and a lot of trips back and forth to China, we have been able to successfully scale up to the scale necessary to support our clinical development in the early years here, so that went very well as I mentioned during the call.

We were able to maintain the carbohydrate structure that is so critical to the uptake of Pompe enzyme replacement therapy and that both, in terms of carbohydrate structure and also the M6P content specifically, so those are things that we knew to look out for and design the processes specifically to be able to control that and we were able to successfully do it, but so not easy for sure.

I think built on years of experience and also the technology and know-how Dr Do and some of his colleagues brought along with Callidus was certainly good starting point, so we feel like we are in a good position and we are on track to start the studies this year..

Yes. We feel that was the greatest risk this year, was choosing the right cell line.

We knew by the second quarter that we had an excellent cell line, but we also knew from the experience that we had to scale it, so we just focused like the laser and put the best people around it and I think we were very, very pleased with this manufacturer reports we got in the last couple of weeks that we are now at scale and we have done it successfully.

You are right. I think it is distinct from other experiences over time with other people who have sought to make this enzyme at larger scale, so I think we have got a very, very special product here and we are eager to get that in to patient..

Okay, sounds encouraging.

Could I also ask, I know you have got the data coming in a couple of weeks, but I wanted to - and don't want to steal your thunder, but can you talk a little bit about the cardiac disease measures that you have captured just broadly what they are and if there is natural history data there that you can point to, to further differentiate Migalastat monotherapy?.

Yes. No. Of course, Jay, I will let you comment on..

I could talk about the methodology that was used in assessing the patients. We had very rigorous echocardiography that was performed and done in that centralized procedure in a blinded fashion, so we know that the quality of the data was high. We looked at the number of parameters.

The most important one in is diseases is left ventricular mass, because cardiac hypertrophy is a characteristic finding in these patients, so the left ventricular mass or LVMI, left ventricular mass index are really the important parameters to focus on. I think that's kind of data that we will be able to discuss at the time.

The natural history is quite well characterized in patients in that they have gradual progress increase in their cardiac size in their left ventricular mass, so that is a well recognized phenomenon in Fabry patients and if you really can't interpret our data in light of what is known in this natural history for the cardiac disease in Fabry..

Okay. Thanks very much. .

You are very welcome..

Thank you. At this time, I am showing no further questions. I would turn the back to you Mr. Crowley for any closing remarks..

Great. Thank you, operator. Thank you all for joining. We have a lot more news to share yet here before the end of the year, so stay tune. Thank you very much..

Ladies and gentlemen, thank you for your participation in today's conference. This concludes the program. You may now disconnect. Everyone, have a great day..

You too..