Good day, ladies and gentlemen, and welcome to Amicus Therapeutics Full-year 2018 Financial Results and Corporate Update Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session. Instructions will follow at that time. [Operator Instructions]. As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Sara Pellegrino, Vice President of Investor Relations and Corporate Communications. You may begin.

': ': On this call, as referenced on slide two, we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as well as our plans and prospects. Our forward-looking statements should not be regarded as representation by us that any of our plans will be achieved. Any or all of the forward-looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward-looking statements, which speak only to the date hereof. All forward-looking statements are qualified in their entirety by the cautionary statement and we undertake no obligation to revise or update this presentation or conference call to reflect events or circumstances after the date hereof. For a full discussion of such forward-looking statements and risks and uncertainties that may impact them, we refer you to the forward-looking statements on slide two of our full-year 2018 results slide deck as well as the forward-looking statements and risk factors section of our annual report on Form 10-K for the year ended December 31, 2018, to be filed later today with the Securities and Exchange Commission. At this time, it is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer of Amicus. John?

Great. Thank you, Sara. Good morning and welcome everyone to our full-year 2018 results conference call. This is the third year we are holding our full-year results on the last day of February on Global Rare Disease Day, a true reflection of our dual mission of making a positive and meaningful impact for people living with rare diseases, as well as our important mission to drive shareholder value for all Amicus shareholders. ': Indeed, Amicus strives every day to bridge these gaps through the commercialization and development of our portfolio of medicines for rare metabolic disorders, as well as our strength in patient advocacy, our corporate citizenship and our other support services. And beyond just the medicines we develop and deliver, our Healing Beyond Disease initiative brings team Amicus into the rare disease communities to help bridge these gaps in very unique and extraordinary ways. ': Indeed, Amicus strives every day to bridge these gaps through the commercialization and development of our portfolio of medicines for rare metabolic disorders, as well as our strength in patient advocacy, our corporate citizenship and our other support services. And beyond just the medicines we develop and deliver, our Healing Beyond Disease initiative brings team Amicus into the rare disease communities to help bridge these gaps in very unique and extraordinary ways. ': ': ': The first is the extraordinary success we have had with the launch of our precision medicine Galafold for Fabry disease. At the end of last year, more than 650 people living with Fabry disease with amenable GLA variants, also referred to as genetic mutations, were taking this precision medicine as their only treatment for Fabry disease. ': ': We are also even more confident now with our pathway to drive Galafold sales toward $500 million by 2023 and to…

Thanks, John. Good morning, everyone. Let me begin on slide seven with a snapshot of the Galafold launch through December 31. ': Our full-year revenue in 2018 was $91.2 million, which is a year-over-year increase of approximately 147% from fiscal year 2017. And as we move through our first quarter, we continue to see exceptionally strong momentum in new markets, including the United States and Japan, as well as in our more mature markets. Let me provide a little qualitative color in the different regions around the world. We do continue to see growth across the EU 5, and Europe more broadly. And in more mature markets such as Germany and the UK, we see an increasing proportion of previously untreated patients come on to Galafold. This is right in line with our strategy for initial adoption in switch patients followed by uptake in previously untreated patients. ': We have obtained broad reimbursement coverage and continue to make great progress with payers, experiencing shorter and shorter times toward moving from prescription to patients receiving drug. And finally, across all markets, we continue to see an exceptionally high rate of compliance and adherence to this oral treatment option. ': This would be a fantastic achievement for Amicus and will represent a meaningful impact on how Fabry disease is treated around the world. On slide nine, we continue to believe that Fabry may be one of the most under and misdiagnosed human genetic disorders in the world, with continued growth expected in the Fabry population through newborn screening and other diagnostic initiatives. We believe we can get to, as John mentioned, $500 million in potential sales by 2023, with a $1 billion plus addressable market opportunity in 2028. With that, let me hand the call back to John to provide an update on our pipeline programs.

': ': ': First, by definition, to receive a breakthrough therapy designation, there must be preliminary clinical evidence indicating that the drug may demonstrate substantial improvement on a clinically-significant endpoint over available therapies. And in the case of Pompe disease, we believe significant unmet need remains despite an approved therapy. For AT-GAA, the BTD is based on clinical efficacy results from the ongoing Phase I/II clinical study, including improvements in six-minute walk distance in late onset Pompe patients in comparison to natural history of treated patients. And now, with this BTD for AT-GAA, it will facilitate multi-disciplinary, comprehensive discussions of the AT-GAA development program with the FDA, including planned clinical trials and plans for expediting manufacturing development strategy. ': This BTD, together with our results from the ongoing Phase I/II study and the ongoing PROPEL pivotal study, support our strategy to advance AT-GAA as quickly as possible with a potential to become the new standard of care for people living with Pompe disease. In addition to the more frequent and intense dialog with the FDA on this program and also all of the initiatives that may advance this medicine to Pompe patients as quickly as possible with this BTD, we also believe that the BTD represents an important acknowledgment of the great unmet need that exists for patients with Pompe disease, despite an approved ERT. And also, the significance of the data that we have shown to-date as compared to the natural history of people on the ERT standard of care. The baseline remains that the pivotal PROPEL study will be the basis to support full approval of AT-GAA. We remain laser-focused on enrolling the PROPEL study and doing what we need to do to further build out our body of data for AT-GAA. Based on regulatory feedback from both…

Thank you, John. And good morning, everyone. Our financial overview begins on slide 25 with our income statement for the 12-month period ending December 31, 2018. For the full-year 2018, we recorded Galafold revenue of $91.2 million, a 147% increase over 2017. Cost of goods sold includes manufacturing costs as well as royalties associated with sales of our product. Cost of goods sold as a percentage of net sales was 15.8% for the full year ended December 31, 2018 as compared to 16.9% for the prior full-year period. We continue to make significant investments in R&D and manufacturing, with the ongoing pivotal study and commercial scale-up in our Pompe program, as well as the expansion of our gene therapy portfolio and capabilities. During the full year of 2018, we recorded $270.9 million in R&D expense, which, as a reminder, includes the upfront payment of $100 million for the Celenex asset acquisition. This compares to $149.3 million for the prior full-year period. Total selling, general and administrative expense for the full year of 2018 was $127.2 million as compared to $88.7 million for the prior full-year period. The increase represents the expanded geographic scope of the ongoing Galafold commercial launch, including initial launch activities in Japan and the United States. Net loss for the full-year 2018 was $349 million or $1.88 per share compared to a net loss of $284 million or $1.85 per share for the prior full-year period. At December 31, 2018, we had approximately 189.4 million shares outstanding. Moving onto slide 26, a few comments about our current cash position and 2019 financial guidance. Cash, cash equivalents and marketable securities totaled $504.2 million on December 31, 2018 compared to $358.6 million at December 31, 2017. In addition, net cash spent was $196.3 million for the full year of 2018, which was below full-year 2018 net cash spend guidance of $200 million to $225 million. Looking at the remainder of 2019, we are reaffirming our full-year Galafold revenue guidance of $160 million to $180 million. Taking into account our anticipated investments as well as anticipated Galafold revenue, we expect to have approximately $300 million in cash on the balance sheet at the end of 2019. This projected year-end cash balance reflects all ongoing investments in our operations, including Pompe manufacturing scale-up, as well as facilities, including our new Global Research and Gene Therapy Center of Excellence in Philadelphia. With our current cash position and the continued revenue from sales of Galafold, we have sufficient capital to fund ongoing operations into at least mid-2021. As we have noted in the past, potential business development collaborations, pipeline expansion and investment in manufacturing capabilities could impact our future capital requirements. This summarizes our key financials for full-year 2018. Additional details can be found in our annual report on Form 10-K, which will be filed later today. ':

': As you can see on this slide, we are well on our way to achieving our 2023 vision to treat 5,000 patients. ':

Thank you. [Operator Instructions]. And our first question comes from Anupam Rama from JPMorgan. Your line is now open.

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Sorry. Hello. Can you hear me?

Hi. We can hear you now. Yes.

Sorry. Sorry about that.

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': If you remember, we had shown data out to two years in the first two patients treated. One patient who looked – was quite older when she received it and she looked to have generally stabilized on the Hamburg scale. And then the other patient, who is her younger sister, who received it when she was quite young, I think she was about two-and-a-half old or so, she actually completely stabilized over that couple-of-year period. We think that data is incredibly exciting. And it is part of what gives us great hope that this gene therapy has the potential to really change the trajectory of the disease. ': If you remember, we had shown data out to two years in the first two patients treated. One patient who looked – was quite older when she received it and she looked to have generally stabilized on the Hamburg scale. And then the other patient, who is her younger sister, who received it when she was quite young, I think she was about two-and-a-half old or so, she actually completely stabilized over that couple-of-year period. We think that data is incredibly exciting. And it is part of what gives us great hope that this gene therapy has the potential to really change the trajectory of the disease. ': If you remember, we had shown data out to two years in the first two patients treated. One patient who looked – was quite older when she received it and she looked to have generally stabilized on the Hamburg scale. And then the other patient, who is her younger sister, who received it when she was quite young, I think she was about two-and-a-half old or so, she actually completely stabilized over that couple-of-year period. We think that data is incredibly exciting. And it is part of what gives us great hope that this gene therapy has the potential to really change the trajectory of the disease. ': Jay, Jeff anything to add? ': Jay, Jeff anything to add?

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Yeah, Just to be clear, Anupam, we want to see striking results, so that when we look at the data, when we see the evaluations on the patients, when we look at videos internally at least, it is night and day from what you would expect from a course of this just awful, awful disease. ': ':

Awesome. Thanks for taking the question, guys.

Great. Thank you, Anupam.

Thank you. And our next question comes from Tazeen Ahmad from Bank of America. Your line is now open.

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Yeah. Thanks, Tazeen, for the questions. What I think would be most helpful is to first highlight what we see as the big growth drivers over the next few years, which I think gets you to the 2023 time point, and share a little bit of the sort of distribution of those patients. And then, I can talk a little bit about the long-term market growth we see, the $1 billion plus market opportunity over the next decade. ': ': ': ': ': ': So, hopefully, that gives you a flavor of how we see the market progressing over time and our opportunities within that market.

Okay. Great. Thanks, Brad.

Thanks, Tazeen.

Thank you. And our next question comes from Irina Margine from Cowen. Your line is now open.

Hi, guys. Good morning. And thank you for taking my questions. I was wondering, just to follow up on the previous question, maybe if you could comment on the magnitude of update you are seeing with Galafold among naïve patients in the US? And how that might differ from dynamic in the EU? Is willingness to treat in anyway difference between the different geographies? Thank you.

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Yes, yes.

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Thanks so much, guys.

Thank you.

Thank you. And our next question comes from Mike Ulz from Baird. Your line is now open.

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Great, thank you.

Thank you, Mike.

Thank you. And our next question comes from Mohit Bansal from Citi. Your line is now open.

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Very helpful. Thank you very much.

Thank you, Mohit. Have a great day. Operator Thank you. And our next question comes from Whitney Ijem from Guggenheim. Your line is now open.

Hi. Good morning. First, I just wanted to follow up on CLN6. For the mid-year update, just to clarify, will we get data out to two years only or will we get additional follow-up for the patients that have it beyond two years?

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Okay, got it. And then, are there specific medical meetings that we should have on our radar for that update mid-year?

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Okay. And then, just last quick one, back on Galafold and kind of longer-term market opportunity, I think you had a slide in one of your decks earlier this year talking about, I guess, the rate of misdiagnosis in different categories based on screening studies. And two that were of particular note were MS and IBS. So, is FOLD sort of – or is Amicus doing any sort of active outreach or screening in those settings to find patients?

Yeah, very much. Brad?

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Great, thanks.

Thanks, Whitney.

Thank you. And our next question comes from Kristen Kluska from Cantor Fitzgerald. Your line is now open.

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': In terms of the breakout, expect, of those 200 people at our Global Research and Gene Therapy Center of Excellence, the vast majority of those will be scientists. We also expect to have some members of our technical operations team in Philadelphia working primarily on the early stage process development, getting that science ready for manufacturer and, ultimately, then hand off either to a CMO or to our later stage process development in our own Amicus manufacturing team. ': In terms of the breakout, expect, of those 200 people at our Global Research and Gene Therapy Center of Excellence, the vast majority of those will be scientists. We also expect to have some members of our technical operations team in Philadelphia working primarily on the early stage process development, getting that science ready for manufacturer and, ultimately, then hand off either to a CMO or to our later stage process development in our own Amicus manufacturing team. ': In terms of the breakout, expect, of those 200 people at our Global Research and Gene Therapy Center of Excellence, the vast majority of those will be scientists. We also expect to have some members of our technical operations team in Philadelphia working primarily on the early stage process development, getting that science ready for manufacturer and, ultimately, then hand off either to a CMO or to our later stage process development in our own Amicus manufacturing team. ': ': ': ':

Great. Thank you, John.

Thanks, Kristen. Have a good day.

Thank you. And our next question comes from Salveen Richter from Goldman Sachs. Your line is now open.

Yes. Hi. Thanks for taking the questions. [indiscernible] for Salveen. Congrats on all the progress. Very encouraging. I had a couple of questions. One of them, for the AT-GAA program, what is the CMC requirements that are still sort of in process of being completed for the Phase III and what is the work that you basically expect to do there in the next couple of years?

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': And along with that, we showed in the naïve patients, who have not been treated before, a quite robust increase in their FEC and their pulmonary function tests. And I really think that shows the ability of AT-GAA to have a pretty profound effect on their function. And we saw that also in the motor function. Of course, the six-minute walk increasing in both groups. So, I think all the results are pretty much as we expected and as good as they could be for these different populations. ': And along with that, we showed in the naïve patients, who have not been treated before, a quite robust increase in their FEC and their pulmonary function tests. And I really think that shows the ability of AT-GAA to have a pretty profound effect on their function. And we saw that also in the motor function. Of course, the six-minute walk increasing in both groups. So, I think all the results are pretty much as we expected and as good as they could be for these different populations. ': And along with that, we showed in the naïve patients, who have not been treated before, a quite robust increase in their FEC and their pulmonary function tests. And I really think that shows the ability of AT-GAA to have a pretty profound effect on their function. And we saw that also in the motor function. Of course, the six-minute walk increasing in both groups. So, I think all the results are pretty much as we expected and as good as they could be for these different populations. ': And along with that, we showed in the naïve patients, who have not been treated before, a quite robust increase in their FEC and their pulmonary function tests. And I really think that shows the ability of AT-GAA to have a pretty profound effect on their function. And we saw that also in the motor function. Of course, the six-minute walk increasing in both groups. So, I think all the results are pretty much as we expected and as good as they could be for these different populations.

Got it. Thank you. Very helpful. Would the ERT-naïve basically data will be a better comparison with the natural history? Is that the thinking?

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Got it. Thank you.

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Great. Thank you, operator. Thank you everybody for listening. Have a great day.

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