Sara Pellegrino – Senior Director, Investor Relations John Crowley – Chairman and Chief Executive Officer Bradley Campbell – President and Chief Operating Officer Chip Baird – Chief Financial Officer Dipal Doshi – Chief Business Officer and General Manager-Scioderm Jay Barth – Chief Medical Officer.

Ritu Baral – Cowen & Company Anupam Rama – JP Morgan Tazeen Ahmad – Bank of America Joseph Schwartz – Leerink Partners Mike Ulz – Robert W. Baird.

Good day, ladies and gentlemen and welcome to Amicus Therapeutics’ Full Year 2016 Financial Results and Corporate Update. At this time, all participant lines are in a listen-only mode to reduce background noise, but later, we will be holding a question-and-answer session after the prepared remarks and instructions will follow at that time.

[Operator Instructions] As a reminder today’s conference call is being recorded. I would now like to introduce your first speaker for today conference call over to Sara Pellegrino, Senior Director, Investor Relations. You have the floor..

Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics’ full-year 2016 financial results and corporate highlights and program updates. Speaking on today's call, we have John Crowley, Chairman and Chief Executive Officer; Bradley Campbell, President and Chief Operating Officer; and Chip Baird, Chief Financial Officer. Dr.

Jay Barth, Chief Medical Officer; and Dr. Hung Do, our Chief Science Officer; and Dipal Doshi, our Chief Business Officer and General Manager of Scioderm are also here and available to participate in the Q&A Session.

On this call, we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as wells 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 statement, which speak only as of the date hereof.

All forward-looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof.

For a full discussion of such forward-looking statements and the risks and uncertainties that may impact them, we refer you to the forward-looking statement on Slide 2 of our full year results slide deck, the forward-looking statements and risk factor sections of our Annual Report on Form 10-K for the year ended December 31, 2016, 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 Therapeutics..

Great, thank you, Sara, and good morning, everybody. Before I highlight our corporate update, let me just take a moment to recognize that today, yesterday in this week we celebrate the 10th anniversary of World Rare Disease Day.

We’ve had at Amicus a series of events over the last week to celebrate bringing patients and their families in, hearing of their journeys and being able to celebrate in their hopes and dreams as well.

Also as many of you may have seen and in fact and still here in Washington DC, as many of you may have seen I was with my family last night and we were honored to be special guest of President Trump’s for his first joint address to Congress.

And I will say before we get into the Amicus just as a dad how proud I was to have my daughter featured and her courage, and character and strength for. Those of you were parents you can certainly appreciate your heart bursting with pride, especially knowing everything that Megan has lived through in her 20 years and now being a sophomore in college.

It was a wonderful event for our family. But also too, I think it was wonderful that the President highlighted the needs of the community in rare diseases. As we all know there are more than 7,000 rare diseases that together effect more than 30 million people, just here in the United States. Fewer than 400 of those diseases have any medicines.

So with that there remains tremendous need.

I also have the honor and privilege yesterday to meet with the President and the Vice President in the Oval office to discuss the needs of the rare disease community and the important of healing and innovation, the importance of emerging growth biotechnology companies and what we need to do in my view to move forward medicines for as many patients as quickly as possible.

It was also an honor to meet, as well with the President and Vice President following their speech. So as I have done for many years, I will continue to advocate for individuals living with rare diseases and for the importance of patient-focused drug development.

And I think that was very clear in the President’s message last night that patient-focused medicine needs to at the core of all new medicine development. So we will incorporate the patient perspective as early as possible in all of our processes.

I am very pleased that the White House has chosen to focus on the cause of rare diseases and as Chairman and CEO of Amicus, I think that we represent one of the leaders in biotech innovation for the rare disease community of that I’m most proud.

Turning now to our Amicus corporate highlights, I’ll being on Slide 4 by highlighting the significant milestones that we achieved here at Amicus during the full-year 2016. As I outlined early this year at the JP Morgan Conference there are a couple of key accomplishments that we think lay a very significant foundation for the coming year.

So first, we got the drug approved, our first drug, our precession medicine, Galafold for Fabry disease, which we have launched independently in the EU on a commercial basis.

In deed it’s a rare accomplishment in biotech to move from an idea to molecule, to clinical development and regulatory approval and on to the market as the first new treatment option for Fabry patients in now more than a decade.

So we couldn’t be more pleased with the initial launch success and Brad in a moment will take us through some additional metrics we have for that launch in Europe.

Secondly, during 2016, our novel Pompe treatment paradigm, our own recombinant protein regime entered the clinic and we reported positive preliminary data from this clinical study in Pompe patients.

Third, we made significant progress to open new sites and have now nearly completed enrollment in our very important Phase 3 study, for EB or epidermolysis bullosa, a devastating rare disease, which is a connective tissue disorder and disorder in of the skin.

So finally, we ended 2016 with an exceptionally strong balance sheet with $331 million in cash and we anticipate that to fund our ongoing programs through several key inflection points. Turn now to Slide 5 please and you look at the five key strategic priorities. So we had some good accomplishments in 2016.

We believe these now strongly position us for what we hope to be our most successful year to date for patients, for shareholders, for all of our key stakeholders at Amicus in 2017. To get there, we are laser-focused on five key strategic priorities that appear on this slide. Our first priority is to continue the international Galafold roll out.

Again Brad will review the launch metrics in more detail but I'm really pleased to say that we have now 75 patients on reimburse drug. Almost all exclusively from our initial launch country Germany and the vast majority of these patients continue to be patient switching from enzyme replacement therapy.

The launch so far is exceeding our expectations and we are very confident on our ability to treat and to get to our target number of 300 patients with Galafold by the end of this year.

Second, we plan to submit our Japanese new drug application or J-NDA, which remains on track for the first half of 2017 in this second largest global Fabry market in Japan. Third, we hope to establish definitive proof-of-concept in the clinic for our highly differentiated treatment approach for Pompe disease.

Fourth, we hope to successfully complete our Phase 3 EB program. And fifth, we will remain diligent in maintaining a strong balance sheet. So very clear, I hope, those are our five key strategic priorities.

We believe we are well-positioned for success as a commercial company with a robust pipeline today, arguably one of the most robust in all of the rare diseases. And that we are on track to building a leading global biotech company focused on the rare and orphan diseases.

So with that as introduction and backdrop, let me turn the call now over to Brad to highlight the Galafold launch.

Brad?.

Thanks John. Good morning everybody. I'll begin on Slide 7 with an update on some of the launch metrics that we've been providing each quarter since launch. And reminder this is all as of February 28.

So first as you know European launches are really a country-by-country process and with the initial EMA approval last year we saw immediate success with our launch in Germany. And throughout the year we secured expanded access in a handful of additional markets, as well.

I’ll go into more detail on this continued success in Germany in a moment, but these initial markets have delivered a consistent five to ten patients per month in the nine months since launch and again as of February 28, we have 75 patients on reimbursed Galafold. So a great start to launch.

However, we have an ambitious goal to put 300 patients on reimbursed Galafold by the end of 2017. In order to do that we need to secure reimbursement and launch in additional countries throughout Europe and continue to pursue Expanded Access Programs outside of Europe as well.

The 2017 is really about opening up new commercial markets and continuing to succeed in the launch in those markets. So I’m pleased here today to highlight the momentum we've made towards that goal. First and foremost, in the last few weeks even we've secured pricing and reimbursement in four additional markets, which brings the total now to ten.

And importantly, we have now launched in three of the top five EU countries on a commercial basis in Germany and the UK and through our ATU in France. And we have additional EU5 launch which is forthcoming. We've also added one additional ERT market in the recent days and expect more throughout the year.

During the weeks and months to come we should expect the patient rate to begin to increase towards our goal of 300. Our commercial patient demographics continue to be very encouraging as well. The majority of patients continue to be switch patients. But we're also seeing nice early uptake in the naïve segments.

And we see a good mix of males and females classically patients laid on for mutations. And we're really seeing great utilization across the spectrum of Fabry patients with amenable genetic mutations who meet our EMA level.

With this great momentum on the pricing and reimbursement front, we continue to believe the success in Germany is a strong predictor of our potential success as we launch in these additional commercial markets and is fast now in order to support that launch we have an Amicus footprint either directly or indirectly through partners on the ground in 27 countries around the world.

Let me turn to Slide 8 now to focus on the German metrics. And the important numbers that we've been following since the launch. First, we estimate that after only nine months of launch we have a 33% market share of treated patients in Germany. So great early uptake in that key market.

As a reminder, all of these patients and their physicians are newly experienced with Galafold because we did not have any clinical sites in Germany during the clinical development program. And now we have 16 unique prescribers which speaks to the broad interest and experience thus far for majority of the top KOLs in that country.

Again we think the reporting these indicators in Germany provides a sense of our early launch success. And as we have said before, we do not expect to provide this country-level granularity as other EU five countries and other countries throughout Europe begin to meaningfully contribute to the patient numbers.

Turning to Slide 9, let me quickly comment on our global regulatory strategy to reach even more Fabry patients. The EU approval provides a stepping stone to access approximately 75% of the global Fabry market.

Europe of course is an important region that represents 34% of the global ERT market in 2015, a significant portion of which comes from the EU five where again we've seen such strong momentum to date.

Outside the EU we have received approval in Switzerland and successfully completed six additional regulatory submissions with more to come, including as John mentioned, our Japanese submission which remains on track for the first half of this year. In the U.S. we've defined a pathway for full approval.

The protocol for the GI study is nearly complete and we're continuing our detailed feasibility assessment. We continue to work collaboratively with the FDA to determine the most expeditious approach to making migalastat available to U.S. patients including through our planned, intermediate Expanded Access Program.

And as I work with FDA discussions progress we may refine or modify our approach and timeline. And finally, over the remainder of the year we continue to expect additional Expanded Access Programs in multiple geographies around the world.

To wrap things up we continue to believe that strong loss momentum our success in the country-by-country pricing and reimbursement processes our regulatory progress, all reflects the strength of our data and European label with significant value of our precession medicine approach, the unmet need that remains in the Fabry disease community and also the important and strong relationships that our team is building in the filed.

We remain committed to providing broad access to Galafold for all Fabry patients with amenable mutations and confident in reaching our goal of 300 commercial patients by year end.

We're also committed to the broader Fabry community and designing a novel proprietary Fabry enzyme replacement therapy co-formulated with migalastat to enhance that ERT activity for patients who do not have amenable mutations.

In the years ahead is our hope and vision that patients and physicians may have two Amicus treatment options, driven entirely by their genotype. With that, let me turn the call back over to John, to provide further update on our clinical pipeline..

Great, thank you Brad So let me go ahead and turn to our Pompe program. This is the intersection of our expertise and in the designing re-competent enzymes. Our patient focused and clinical program that's designed to make a meaningful difference in the lives of patients.

This is really based on extraordinary science and in many respects, I think, this program showcases Amicus as great abilities in science and in clinical development and really believe that this is biotech at its best. So what we've developed is a highly differentiated approach from those that exist today.

We began with the same base protein that others have developed which is a very recombinant enzyme rhGAA. And we do three things to make it very different. The first two important differences are designed for efficient targeting.

First, we produce our protein with high levels of mannose-6 phosphate or M6P, which is essential for targeting an uptake of the enzyme. And we also focus on all of the carbohydrates and optimized mixture glycans. Third, we also have the oral co-administration of an Amicus chaperone, AT2221.

This helps to provide a scaffold for the enzyme to remain stable in plasma until it is delivered into muscle cells. So our treatment regimen of the enzyme with chaperone, which we designate ATB200/AT2221 is very distinct from what others have developed. Turn out to Slide 12 please. What we call the Pompe puzzle.

So I will outline significant learnings and key questions remaining about this very complex and devastating neuromuscular disease. It is a disease increasingly linked to other key muscular dystrophies such as limb girdle and Duchenne muscular dystrophy.

And it's this link, as well as our scientific findings at Amicus, which are being recognized among leaders in the field, including the Muscular Dystrophy Association which is quoted here on this slide to the right, based on the data that our Chief Science Officer, Dr. Hung Do presented at the WORLD Symposium.

So again, what do we know? For the last three and a half years our extensive preclinical studies fill in the light blue pieces of this puzzle. In animals the uptake of our treatment regimen into muscles was significantly greater.

And glycogen, which is the substrate that builds up in people living with Pompe was dramatically more reduced than the current standard-of-care. And again at that WORLD Symposium at San Diego, just two weeks ago, we shared important new scientific findings into the role of protein miss trafficking on muscle weakness and disrepair in Pompe disease.

We also presented new pre-clinical data showing significant reversal of muscle damage and improved muscle function and strength in animals receiving the ATB200/AT2221 combination.

Indeed these are significant, new data further differentiating our treatment regimen in animals in which we hope to replicate in the clinic in patients living with Pompe disease.

What we've seen so far in the clinic, in the initial patients treated with our drug approach is depicted in the dark blue puzzle pieces here, which I'll highlight in a moment.

The key remaining question this year, the one remaining important piece of the puzzle, the gold piece is can our regimen, our treatment regimen potentially improve clinical outcomes? Do Pompe patients walk further? Do they breathe better on our treatment? Do we see other signs of meaningful muscle improvement – muscle strength improvement? This is what we're looking to answer as part of the upcoming cascade of data in the quarters ahead.

Let me turn to Slide 13 and just comment for a moment on the preliminary data summary. Here we've summarized the results in the clinic in our initial Pompe patients who were switched from the standard-of-care ERT on to our regimen, as well as the first two treatment naïve patients in the study.

So as you see we've seen excellent safety and tolerability. The PK profile continues to be distinct and consistent with our previously reported pre-clinical data.

The improvements in kidney biomarkers of muscle damage, as well as biomarkers of substrate over the first 18 weeks suggest that ATB200/AT2221 is having a positive effect on muscles in patients who had previously been stable on ERT for several years, as well as in treatment naïve patients.

So very importantly though I want to highlight to our knowledge there are no published data for other Pompe ERTs either approved, or in development that have shown these types of reductions in biomarkers in patients previously treated with the standard-of-care.

We recently had the opportunity to share all of our latest pre-clinical and clinical data with leading Pompe experts during the WORLD Symposium. And their feedback and their level of enthusiasm was just remarkable. We could not be more pleased with the data to-date and we're very excited to see more data from this study.

So on Slide 14 we provide just a snapshot of what's next in the data cascade anticipated in the second and third quarters of the year. So we will have more of the same important measures of safety, PK and biomarkers in additional patients.

We will continue to track biomarkers in all patients throughout the extension phase and we will report functional measures, including six-minute walk test and pulmonary function tests among others from the extension phase of the study.

In the meantime we continue to make significant investments and are scaling up our manufacturing to the 1,000 leaders scale so that we can begin a registration study as soon as possible with our commercial scale product.

We believe that the strength of our science and clinical program, the positive preliminary data shown to date and key investments in manufacture underscore just how important we believe this program is for people living with Pompe, how important it is to Amicus as a company and certainly for all of our shareholders.

Slide 16, we talk a little bit about EB program here, before I turn it over to call – to Chip – the call to Chip, let me just go through an update on this important Phase 3 study for SD-101 for EB on Slide 16.

So significant momentum in this program, we have seen in the last couple of months a major uptick in enrollment, which is typical in rare diseases as you get to the close of enrollment periods. We've seen that as a result of our team's tremendous effort, in particular to activate new sites around the world.

Patient participation continues to be positive with more than 95% of patients completing the initial 12-week treatment period and continuing in the ongoing extension study.

As you know we have elevated the endpoint of time to wound closure and we are near final with our Statistical Analysis Plan or SAP, which is designed to optimize as study for success. We expect to complete enrollment in the coming weeks and top line data are on track for mid-2017.

So for people living with EB, we have the potential to offer a first-in-class treatment and we look forward to completing this study for this devastating disease. Chip let me go ahead and turn it now to you for the financial overview, please..

Great, thanks John. And good morning everyone. I will start today’s session with our financial results on Slide 18, beginning with our full-year income statement. In 2016 we recorded revenue of $5 million.

2016 revenue was derived from product sales in the third and fourth quarters of the year and represents our first product sales in our Company's history and marks our transition to a commercial stage company.

Moving down the income statement, total R&D expenses for the year increased to $104.8 million, as compared to $76.9 million for the full-year of 2015. The increase here is primarily due to investment in Pompe manufacturing scale up, as well as investment in our Phase 3 EB clinical development.

Total selling, general and administrative expenses for the full-year 2016 were $71.2 million, compared to $74.3 million in the full-year 2015. The increase here was primarily due to investment in pre-commercial and commercialization activities related to the international Galafold launch.

Net loss for the year was $200 million or $1.49 per share, compared to a net loss of $132 million or $1.20 per share for the full-year 2015. Water loss is attributed to an increase in total operating expenses. And as of December 31, 2016, we had approximately 142.7 million shares outstanding.

Moving on a Slide 19, a few comments on our current cash position and 2017 financial guidance. Cash, cash equivalents and marketable securities totaled $330.4 million at December 31, 2016.

We strengthened the balance sheet significantly in 2016 through $100 million at-the-market or ATM equity facility in addition to a $250 million convertible debt offering completed in the fourth quarter.

In terms of expense guidance, we expect full-year 2017 net operating cash spend of between $175 million to $200 million and full-year 2017 total net cash spend, including third-party milestone payments and capital expenditures of between $200 million and $225 million.

Our current cash position at the beginning of year is projected to fund operations into the second half of 2018 and importantly through several key catalysts that John outlined previously in the call. So this summarizes our key financials for full-year 2016.

Additional details will be found on our annual report on Form 10-K, which will be filed later today. And I'm also happy to address any questions during the Q&A, but for now I'll turn it back to John..

Great, thank you Chip. And before we go to the Q&A let me just conclude on Slide 21 with a summary of our anticipated milestones in 2017 again across our three lead programs.

The Galafold international launch for the regulatory submissions and studies to support our global Fabry strategy, a continuation of the important Pompe data cascade, a successful completion of our Phase 3 EB Study and the careful management of our balance sheet. Turn now please to Slide 22.

And here you can see we have a very big and a very bold vision for Amicus.

If we measure our success as the number of lives that we impact with our medicine what does that look like today, what does that look like in the years ahead? At the end of 2016 there were approximately 250 patients living with Fabry, Pompe and EB who were being treated with an Amicus product either on reimbursed Galafold, or enrolled clinical studies.

With significant advancements for our three lead programs if we look ahead to the end of 2018, less than two years from now, we believe that we have the potential to impact more than 800 people living with any of those three diseases.

And if we fast forward to 2023, we think that upwards of 5,000 people living with Fabry, Pompe and EB may be taking an Amicus product, the vast majority of them in a commercial setting.

So if we can get to 800 lives affected and especially if we can get to 5,000, we would be sitting alongside some of the most successful and larger rare disease companies in the biotech industry.

And that's where we need to be, that's our vision, we want to maximize our impact on patients to drive shareholder value so that we build one of the top global biotechnology companies focused on delivering significant benefits to patients with rare, devastating diseases for many years to come.

I am completely committed to that as part of my life's mission and my career. I think in many ways last night was a real inflection point for the entire rear disease community. No president since Ronald Reagan has taken so public and so strong a stance in support of the rear disease community.

And as you recall Ronald Reagan in January of 1984 signed into law the Orphan Drug Act.

And when he did that Ronald Reagan said in the Oval Office as he signed that with Democrats and Republicans at his side and patient advocates, he said that I only wish that with the stroke of this pen that I could also decree that the pain and suffering of people living with rare diseases would also cease.

It didn't cease with a stroke of that pen, but there has been much success over the last 30 plus years, but there is much, much more work to do. So with that operator I'll turn it over to Q&A..

Ladies and gentlemen the question-and-answer queue is now open. [Operator Instructions] Our first question will be coming from the line of Ritu Baral from Cowen & Company. Your line is open..

Hi guys, thanks for taking the question. I wanted to get your latest thoughts on the U.S. primary end point for Galafold and its Phase 3. You mentioned that there's been some additional communication with FDA and previously mentioned that we should look to the IBS D guideline.

What has that recent communication been and what are your latest thoughts?.

Hi Ritu, soI'm not going to comment on ongoing discussions and communications with FDA around this study. I will say that we will continue to work collaboratively with the FDA. We are continued to focus on making Migalastat available to as many U.S. patients. We continue to do or detailed feasibility assessment.

And as we indicated, the protocol is now near complete. So relative to all of those activities, and I will say obviously we continue to significantly engage the patient community. We have heard from patients that they would like access to Galafold as quickly as possible in the United States. That remains our mission.

Obviously, we've got our planned intermediate EAP as part of that commitment to the patient community. So we continue to work. And again we may refine or modify our approach and timelines, but that's the plan going forward as we sit here today..

And just a quick follow up on Galafold, now that you're commercially available in the U.K. Can we think of the uptake trend in the U.K.

as similar to Germany? Or would it be different for some reason reimbursement wise or lay of the land Fabry in the UK wise?.

Yeah, well the one big difference in the U.K. is that there are a number of clinical patients’ clinical trial participants who would be converting over we think fairly rapidly to Galafold in addition to new both the switch and treatment naïve patients. Brad you live in the U.K., so why don’t you add some color to that please..

Yeah, Ritu, I think we continue to think that Germany is a great indicator of where we have full reimbursement, how the launch may go. The UK is slightly smaller than Germany of course, but still a very substantial market and the second largest in the EU-5.

And so, we would expect over the sort of days, weeks, months to come that the rate of patients coming out of Galafold will increase driven in large part by countries like the UK and as I mentioned additional EU-5 and additional countries in Europe.

So more to come, of course, but we very much hope and expect that that those launches will look like the experience we have to date when we have full reimbursement..

Got it, I'll get back in the queue. Thanks..

Thank you, Ritu..

Our next question comes from the line of Anupam Rama from JP Morgan. Your line is open..

Hey, guys, thanks so much for taking the question. Just a couple for Brad, as you think about the 14 countries that you have reimbursement and pricing negotiations ongoing. Could you maybe give us some color on timeline for when some of those countries may come online in 2017? And just a quick one on the 16 unique prescribes in Germany.

How many of them are repeat prescribers? Thanks so much..

So haven't given quite such specific detail on the prescription habits in Germany although I will say that that many of their – if not all of them have multiple prescriptions written to date. And so we think that the experience has been strong and we would hope and expect that to continue.

As relates to your first question in terms of timing, I think my comments on the EU-5 provide a good sense for majority of the opportunity in Europe, but we would certainly expect.

And as I said we do expect an additional EU-5 country to launch here in relatively short order and again that will be an important part of changing that rate of patients coming on. But, of course, we'll also see additional countries as you mentioned of that that sort of remaining 14 to come onboard as well.

I don't want to give too many specifics there, but it would probably continue to be kind lumpy and with countries coming onboard kind of in the months and weeks to come.

So we will continue to give guidance as we give these updates on additional numbers of countries where we've had reimbursement success and I would expect that number to continue over each of these updates..

Great, thanks so much for taking our question..

Yeah, thank you, Anupam..

Our next question comes from the line of Tazeen Ahmad from Bank of America. Your line is open..

Hi, good morning. Thanks for taking my question. Maybe one question on EB as you approach potential data.

What are your expectations for what you would believe to be good result because you did make some important changes in your pivotal trial versus you had in Phase 2? And as we try to think about how to interpret data when it does come out what should we be looking for? Thanks..

Yeah, no, thank you, Tazeen. Jay, I will ask you to comment on you know what would be a successful study here..

We're still near finalized with the SAP. So, you know, I can't define right now exactly what is that is for this study, but it will be based on the primary endpoints and we've talked about having both primary endpoint, the time to wound closure as well as the proportions of wounds closed.

And I think for a study of this kind that when we see the results and if we have achieved statistical significance for the primary endpoint then that would be success for this study..

Can I just follow up maybe on one thing you just said? In terms of determining the appropriate time and proportion of wounds that were closed can you talk a little bit about how you refined that?.

How we refined that or….

Relative to the previous study....

Well the SAP originally before we elevated the time to wound closure just had the proportion of wounds closed at the time of the end point. And we have through the process of revising the SAP elevated, the time to wound closure to be also a primary endpoint. That’s in the Phase 3 study.

In the Phase 2b study, we looked at those endpoints as well and they look very encouraging, but to maximize the probability of success in the Phase 3, two key changes from the Phase 2 were to increase the target wound size at baseline of the size criteria in order to minimize placebo response.

And we also have increased the sample size in the Phase 3 study up to 150 patients, again to maximize the probability of success. There are some other elements as well.

But everything that was learned from the Phase 2b was applied to the Phase 3 and now we feel quite final that we have a statistical analysis plan that’s really going to optimize the study and the analysis of the results..

Okay, thank you..

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

Great, thanks very much. John, it was great to see you recognized for your many contributions to the rare disease community, last night.

I was wondering if you could share any insights from your interactions with the President on the changes that are likely on both the regulatory and reimbursement fronts for rare disease drug developers or biopharma in general?.

Yeah, Joe, I won't comment on specifics of my two visits with the President privately yesterday except that I did communicate our journey in the rare disease as we talked broadly about what are rare diseases, what the needs of the community are and again broadly the need to get as many medicines to as many patients as quickly as possible.

And the President was very, very supportive of that message. I did talk specifically about the need to work collaboratively with the FDA that there are strong leaders at the FDA like Dr. Woodcock and that we want to have a voice in these programs and these policies as they move forward.

A number of senior White House policy advisors were also involved in the discussions. So I again I think very, very good, but I won't comment specifically on anything that the President said other than being very, very supportive of these efforts..

Okay. Well that's helpful. Thank you.

And then as a follow up, I was wondering if you could walk us through your Pompe program, data cascade and some more detail so that we can get a clearer picture of the time points when you will report some clinical measures related to walking and pulmonary function?.

Right, Joe. So we would expect that we would have probably at least two more data releases in the second quarter and in the third quarter. Again, we'll have data on the important measures, biomarkers as well as safety measures in both of those releases.

And we would expect throughout the cascade without giving any specific guidance as to timing that this will also of course include measures of the muscle strength and potential functional improvement in patients.

Again remember, Cohort 1, we have the ERT experienced ambulatory switch patients, Cohort 2 or the non-ambulatory ERT experienced and the ERT treatment naïve we will begin seeing more data from all of those cohorts in the months ahead.

And then by the end of Q3 we will have the complete data in all of these patient groups and cohorts, including extension study data on functional improvements.

So over the next two to six months we'll continue to see very, very important data in this study, we continue to be very, very bullish on this program and its ability to positively affect patients..

Okay great. Thank you for taking my question..

No thank you Jim..

Our next question comes from the line of Mike Ulz from Robert W. Baird. Your line is open..

Hi guys thanks for taking the question.

Maybe if I can just ask a quick follow-up on the prior question, in terms of the Pompe program, do you have a sense of how long you might need to treat patients that start to see functional benefits?.

We're not going to comment on that right now Mike, I think let's start to release the data in the months ahead on clinical outcomes in patients as we have observed them both the ERT treatment naïve and the ERT experienced. And once we have the data out there we can go ahead and comment on that..

Okay. And then maybe just another question you've got it's a filing in Japan in the first half maybe you can give us a sense of the remaining steps there to get that filed.

And then if you can remind us of the patient numbers in Japan and how we should think about pricing?.

Great yes Jay let you take the first part of that about all of the ongoing activities to make sure we can get that in as soon as possible the filing and then Brad maybe you could take the second part on the opportunity and how many people we think we can help with Fabry in Japan..

I’ll start, the clinical regulatory teams and all the associated functions are very, I think they’re extremely hard at work right now putting all the final pieces together of the mission of the JMVA and that is still is on target for the first half of this year..

Great. And on the second part of your question, Mike in terms of the size of the market and market dynamics, et cetera, so remember Japan is the second largest Fabry market it represented about 13% of the $1.5 billion in sales in 2015.

And it is based on the market research that we've done is consistent with that 30% to 50% of patients having amenable mutations.

There are about 700 patients that are treated in Japan and a couple of other important points there, remember migalastat came out of technology that was discovered in Japan, so there's some element of excitement, I think there we've been working with the top KOLs in Japan and so we think we have anticipation and support for migalastat.

In this physician community we had a number of patients in our Phase 3 studies and in fact were treated in Japan at some of those key centers. So initial, early experience.

And so we really see this as an important market, and are excited that we're on track and look forward to the opportunity to continue those regulatory discussions and eventually launch and commercialize that..

Great thanks guys..

Great, thank you..

Our next question comes from….

Please go ahead. I'm sorry operator..

No problem. Our next question comes from the line of Salvin Richard from Goldman Sachs. Your line is open..

Hi congrats on all the progress and thanks for taking my questions. This is Garry [ph] on the line. So just up first regarding the EB program, can you update us on some of the pre-commercial preparations that you guys are doing for the launch? How many sales persons do you anticipate will be needed in the U.S.

and EU? And lastly, when will we hear about plans for potential Galafold and ERT co-formulation programs? Thank you..

Sure so yes I'll just comment, we are focused on obtaining the data and we are also focused on all the manufacturing activities necessary to support the CMC filings. So remember Garry [ph] we actually have a rolling NDA in this program that's already begun. We've completed the preclinical module submissions to FDA.

Next are the CMC sections and that's a key focus. Once we have the data we will then complete hopefully with strong data. The clinical sections to have a completed NDA ready for review. Again as a reminder also although the program has breakthrough therapy designation also two, we have one harmonized Phase 3 program agreed to by both U.S.

and European regulators. So we would expect to very quickly then be working on filing submissions with the European Union, as well. We are doing quite a bit of work to understand more about EB where are the patients. And also the number of patients, the key treatment centers, so that work is ongoing with our Amicus commercial and marketing teams.

Dipal Doshi, our Chief Business Officer, is leading those activities together with Brad Campbell in our commercial organization. We have not yet begun to invest in a specific sales force in the United States, until we see this data though.

We do think though, given the patient needs, and how rapidly we can get the medicine to patients and we think with our experience in Europe in building an effective sales organization that we could replicate that hopefully very quickly in the United States. So does that answer the question that Garry [ph]..

Yes, that was great..

Great thank you. And in terms of our next-generation Fabry program, that is our own proprietary ERT product co-formulated Galafold, we would expect to have some additional preclinical data that will be very important to see in the months ahead.

And with that the cell line has been transferred to manufacturing and we're working on a number of those scale up issues in the very early stages, but we'll have more data on that I’d expect probably mid-year or so. And probably an update on timing as well..

Great, thanks guys. Congrats on all progress..

Great thank you..

Our next question comes from the line of Ritu Baral from Cowen & Company. Your line is open..

Thanks for taking the follow-up. I want to actually ask a flip side of Joe’s question on Pompe, specifically, some of the safety markers that we may see on in your Pompe news flow.

When do you think that we would see a meaningful naïve patient antibody titers? When will that data mature? What's the meaningful difference? And John in your own experience when – what are the most problematic aspects of the current gold standard treatment that we’re talking infusion site reactions as you guys have reported? Are there other metrics that we should be keeping an eye on that you might report going forward?.

Sure so important to look at tolerability. The current standard-of-care in many patients is not well-tolerated, has a black box warning for instance. So thankfully, to date, I think, we reported 150 plus infusions that were update in San Diego a few weeks ago that we have had no infusion-associated reactions.

So we'll continue to observe that to see if the drug is well-tolerated. Again remember, a key part of the addition of the Chaperone is the whole notion of keeping our protein folded in plasma. There is nothing more immunogenic than an unfolded, aggregated protein, especially one given at high dose like it is in Pompe.

So we think very important for the chaperone to be there, but we'll continue to study those and look for those observations in the clinic. We think the greatest limitation of the current standard-of-care is it's inefficient targeting to muscle.

If you can get the protein, we believe into muscles and use that to break down glycogen and promote as Hung showed in pre-clinical models a few weeks ago, promote the proper trafficking of these enzymes, we think that you could potentially have a significant benefit as we've seen in preclinical studies.

So that is the goal to get as much enzyme and as active or form with as low of an immune response in patients, particularly on the tolerability side as possible. So that really incorporates everything that we've tried to design in this drug to address the problems of targeting an uptake loss of activity, and tolerability and immunogenicity.

Specific – we are measuring anti-bodies, we will have that data in one of the next two data cascades. Again we're looking at antibodies not just in titers, we're looking at quality and type if you will.

So we think it's a part of the emerging story, but equally too we need to see, do we continue to have an impact on these important biomarkers of disease and does that translate into functional benefit? So again it’s a very complex disease, very complex treatment regimen that we've developed, but it's intentionally so to try to address a lot of these limitations..

Great, thanks for taking the follow-up..

Of course, thank you..

Ladies and gentlemen that's all the questions that we have in the queue at this time. So I'd like to turn the call back over to management for closing comments..

Great, now, thank you operator, that's all I have. Thank you all for listening. And we have much more work to do, but we think we're off to a great start in 2017. Everybody have a great day. Thank you..

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