Good day, ladies and gentlemen, and welcome to the Amicus Q2 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 now like to introduce your host for today's conference, Ms. Sara Pellegrino, Senior Director of Investor Relations. Ma'am, you may begin.

Thank you. Good morning. And thank you for joining our conference call to discuss Amicus Therapeutics' second quarter 2017 financial results and corporate highlights. 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 are also here and available to participate in the Q&A session. On this call, as referenced on Slide 2, 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 statement, which speak only to 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 second quarter 2017 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, as well as our quarterly report on Form 10-Q for the quarter ended June 30, 2017 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, everyone. So I'm pleased to host this morning's call and let's begin on Slide 3. In the beginning of the year at the JP Morgan Conference, we outlined five key strategic priorities for Amicus in 2017. In the first half of 2017, we have made significant progress across all five of these priorities, which we believe, creates a strong foundation as we approach several important milestones in the second half of this year. First, we are executing very successfully with our International Galafold Launch. Galafold of course is our oral precision medicine for people living with Fabry disease, who have amenable mutation. Growth in patient and physician adoption has picked up significantly over the past three months and we continue to obtain pricing and reimbursement across the country-by-country process in the EU. Brad will review the launch metrics in more detail in a moment, but I am pleased to say that we now have 179 patients on reimbursed Galafold as of July 31. Of significance too, we also have seen extremely high rates of compliance and adherence to this oral precision medicine. This momentum reflects continued strength in our initial launch country, Germany, and more recently from several additional countries, notably France, Italy and the United Kingdom. We believe that this successful launch execution is an extension of and an embodiment of the science-driven patient-centric culture here at Amicus. Our second priority for the year is to complete additional global marketing submissions for migalastat, including our Japanese New Drug Application, or NDA, which we submitted on schedule during the second quarter of 2017, as well now as our NDA to the U.S. FDA, which is targeted for the fourth quarter of this year. Third, we believe that we are well on our way…

Thanks John. Good morning everybody. I'll begin on Slide 6 with an update on our launch metrics all as of July 31. The first half of 2017 has been all about opening new markets and continuing to drive successful launches in the markets that are open. And as of today, we continue to do well on both fronts. First let me highlight the patient numbers which continue to be the most important metric for our early launch. As John mentioned, there were 179 patients on reimbursed Galafold as of July 31. During the first quarter, most of that growth came from our initial launch country, Germany. In the second quarter and throughout July, we continued to see strong uptake in Germany where more than 50% of treated amenable patients are now on Galafold. However as we anticipated, we are now seeing a significant number of new patients from our recent launch countries, including France, the U.K. and Italy, as well as a number of smaller and mid-sized markets throughout Europe. In terms of the other important metrics that we've tracked, we've secured pricing and reimbursement in 12 countries, including four of the top five EU markets, in addition to several of the mid-sized EU countries as well. We are in the midst of pricing and reimbursement discussions in additional 13 countries and we have an Amicus footprint either directly or indirectly through partners on the ground in 27 countries, as we prepare to expand the launch into additional geographies. We have an ambitious but achievable goal to treat 300 patients with reimbursed Galafold by the end of 2017, and given where we are today, we believe we are right on track to hit that goal. As we now have a number of countries contributing to the launch, we will not…

Thanks Bradley. Good morning everyone. Our financial overview begins on Slide 9 with our income statement for the second quarter. And in the second quarter of 2017, we recorded revenue of $7.2 million, representing a sequential increase of 72% over the total product revenue of $4.2 million to the quarter - in the first quarter of 2017. Total product revenue for both periods represented commercial sales of Galafold, as well as Expanded Access Programs. Moving down the income statement, total R&D expense in the second quarter of 2017 increased to $32 million, as compared to $18.3 million for the second quarter of 2016. The increase here is primarily due to investment in Pompe manufacturing scale-up, as well as investment in Pompe phase 1/2 study that's ongoing and the phase 3 EB clinical study. Total selling, general and administrative expense for the second quarter of 2017 was $19.3 million, which was essentially identical to the $19.3 million we spent on SG&A in the second quarter of 2016. This steady rate of spending reflects our continued focus on carefully managing SG&A expenses. Net loss for the period was $48.2 million, or $0.34 per share, compared to a net loss of $51.1 million, or $0.40 per share, for the second quarter of 2016. The narrower loss here is attributed to an increase in net product sales versus the prior period. And as of July 25, 2017, we have approximately 164.6 million shares outstanding which includes shares issued during the recent follow-on offering. Moving onto Slide 10, a few comments on our current cash position and 2017 financial guidance. Cash, cash equivalents and marketable securities, totaled $227.2 million on June 30, compared to $330.4 million at December 31, 2016. Our balance sheet was further strengthened in July with a $258 million follow-on public offering. Total net cash spend for the first half of 2017 was $103.2 million, so we are tracking well against our full-year 2017 expense guidance. I'll note that even with the positive recent update on FDA's status for Galafold and our ability to now file an NDA in 2017, we continue to expect full-year net operating cash spend of between $175 million to $200 million, and full-year total net cash spend including third-party milestone payments and capital expenditures of between $200 million and $225 million. And with the proceeds from our offering, our current cash position is now expected to fund operations into at least the second half of 2019, and as John highlighted, through several key catalysts. So this summarizes our key financials for the second quarter of 2017. Additional details can be found on our Annual Report 10-K currently on file with the SEC, as well as our form 10-Q which will be filed later today. I'm happy to address any questions during the Q&A. But for now we'll turn it back to John.

Thank you, Chip. So again, a lot of work ahead of us and hopefully much more value for shareholders ahead and for patients too. The second half of this year will be a very important period for us here at Amicus as we continue to execute the Galafold launch as well as our global regulatory strategy for migalastat. And as we approach significant clinical data milestones, including top-line data from our phase 3 EB study later in 3Q followed by a complete data from our Pompe phase 1/2 study. In closing on Slide 13, we believe that our successful execution in the first half of 2017 has advanced our very bold vision here at Amicus, and that is to build one of the world's leading global biotechnology companies focused on rare devastating diseases and to measure our success towards that goal by the number of lives that we impact with our medicine. At the end of 2016, there were approximately 250 patients living with Fabry, Pompe or EB, who were being treated with an Amicus medicine, either on reimbursed Galafold or enrolled in one of our ongoing clinical studies. During the first half of this year, to give you a sense of the momentum in the ongoing - with the ongoing Galafold launch, in addition to patient enrolment in our EB and Pompe clinical studies, there are now approximately 350 patients being treated with an Amicus medicine today. We believe that with continued advancement of our three lead programs, that by the end of 2018, we have the potential to impact more than 800 people with an Amicus medicine. That would be a significant achievement for the patient community and is incredibly gratifying frankly for me here and for our entire Amicus team. And if fast-forward to 2023, which may seem like a long time from now but only six years, and we think that upwards of 5,000 patients may be taking an Amicus medicine, the vast majority of them in a post-approval setting. That would put us on par with the leading global rare disease companies and would continue to fulfill our patient-focused mission. So with that operator, and that summary of our vision ahead and the work ahead for us in the second half of this year, I'm happy to open it up to Q&A.

[Operator Instructions]. And our first question comes from Ritu Baral from Cowen. Your line is now open.

Good morning, guys. Thanks for taking the question. What are you guys expecting to release with the Q3 Pompe data release? Do you know the number of patients? Will it be full cohorts? And what are your expectations for the data? What would qualify a success on the endpoints six-minute walk function, PFTs, etcetera?

So with that dataset, Ritu, we would expect the full data from the 20 patients - as a reminder, we have three different cohorts in that study. We had ERT experience switch patients in cohort one, ERT - who are ambulatory. We have ERT non-ambulatory switch patients in cohort two and treatment naïve ambulatory patients in cohort three. We've seen some data now for each of those cohorts to remind everybody back in May we showed data of 10 patients, so half of the study set at six months. It included safety data, important biomarker data. It also included muscle function data on those 10 patients at six months. So fast-forward to the end of this quarter, Ritu, we would expect data on all of those patients. So similarly to the first 10 patients, where we think we had early proof-of-concept to continue to see the same magnitude of improvements. Obviously the safety profile continues to see extreme - continuing to see very low infusing associated reactions, we think would be very differentiating factor. And in muscle outcome too, we think what we saw previously of those trends were to continue with the next cohort, we think that would bode very favorably for this experimental treatment regimen for Pompe patients. In addition Jay comment, but I believe we'll have data on some of those first cohort patients at one year as well.

That's right. There will be long-term functional data really starting to collect that in patients that have been on treatment, as we've said, for as long as 48 weeks for long as patient and continuing.

Would you…

Right, so we haven't seen that. We haven't seen that but we'll share that. Yes. I'm sorry, go ahead.

Would you expect additional improvement between the month six and the month 12 time point?

Yes, we don't know. We haven't seen. It would be wonderful if patients continued to improve. That would be a very high bar indeed, given the level of improvement that we saw in month zero through month six. But to see that they were - that the affected month six was persistent and durable, we think that would be very important to see. And obviously if they continued improvements that would certainly be a really important dataset as well.

And how should we think about the secondary efficacy measures? There was a lot of discussion around the pulmonary function tests upon first release. Now that there will be more patients and longer follow-up, what should our expectations be around that?

Yes, Jay, I'll let you comment.

In the data that we reported previously, we saw very good trends that interim analyses in the motor function test and they were supported by the pulmonary function test directionally in the cohort and we hope to see the same going forward. And I think the motor function tests are important. The pulmonary function tests serve a supportive role as well in assessing the treatment regimen.

Got it. And last - sorry, go ahead.

No, you go ahead, Ritu.

The last very quick question on the Galafold launch. Can you talk to the progress that you've made on the clinical patient conversion to commercial in the U.K., France and Italy? Will that be a continuing tailwind, or is that - have all those patients been captured already?

They have not all been captured. But I'll let Brad comment.

Yes, we are probably two-thirds to three-quarters away through, so there is still a little bit left there in those markets and a handful of those others. But we make good progress so far and - but I would say the progress that we've made is certainly not just that but a reflection of the quality of the team and the quality of the strategy and of the product.

Great. Thanks for taking all the questions guys.

Yes, of course. Thank you, Ritu.

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

Hi, good morning. Thanks for taking my questions. John, just so that I'm clear, did you say that we were definitely going to get the top-line EB data before Pompe in 3Q?

Yes, that's our expectations, Tazeen.

And then in terms of what you think the range of pricing could be for EB. I know that we are still a little bit away from doing that. But in terms of market research, what have you found about what kind of level insurance providers would be willing to pay for a drug, which really doesn't have any options right now? And then I have a follow-up.

Yes, so Tazeen we are not yet prepared to talk about pricing. I'll just state that the Amicus pricing philosophy has always been that our medicines be fairly priced and broadly accessible. And that philosophy will certainly underline how we approach pricing for EB. We think it's important - let's get the datasets, let's look at the strength of the data hopefully, and with all the market work that we are doing right now, after that we'll be prepared to talk about them more specifically how we view pricing.

Okay. And then for Brad, can you talk a little bit about compliance, not so much discontinuation but what kind of feedback are you getting about how compliant patients are with their therapy? And then for the upcoming countries that you are expecting to launch in, I guess, specifically for Japan, what are the addressable populations? How well identified is Fabry in that market?

Sure. Thanks Tazeen. On the first question, as it relates to compliance, as John highlighted in his call, we have seen incredibly high compliance and adherence rates both - so both very few drop-offs and for patients who are on drug. They are adhering to their regimen incredibly well. And that's no accident. So in part - of course it's a rare chronic disease, so there is always some added, I think, compliance factor there. But we've put together a very robust set of patient services, support services, and it's different in the country-by-country basis. Some countries allow more. Some countries allow us. We work very closely through our specialty distributor with the pharmacies or the hospitals, where the patients receive their medicine, and very closely with the physicians themselves. So both at the patient level, we are allowed. At the distribution level and with the physician level, we worked very closely from very early on to educate the importance of the regimen and also ensure that we are having high compliance. And so far it's been very high, which is great. And we'll continue to focus on that as a key driver going forward. As it relates to your second question, which was Japan, correct, in terms of the market environment there?

Yes, that's right.

So there are about 700 patients treated today in Japan. It's a higher-than-average treatment rate, so it's more penetrated from a treatment perspective. From an amenability perspective, it is in the 30% to 50% range that we have discussed in other markets, so typical from that perspective. It's a very high - there is a very high awareness of the chaperone and as you may recall, as we've mentioned before, the technology came out of Japan, so there is some receptivity there. And importantly too, we think that there is no home healthcare in Japan. So we think that in addition of course to providing all the benefits that we believe that the product can and the data that we've shown so far with a very favorable safety profile, we also think that the reduction in the infusion - the burdens of the [indiscernible] will be particularly meaningful in that market. So we think - obviously it's a very important market. It's the third largest - the second largest individual country market in the world. It also has a number of demographics that we think will be receptive for our launch there.

Okay, thanks. And then last question on U.S. Galafold. You're planning to submit by year-end. What needs to be done basically between now and then? Has FDA asked you for anything minor to submit between now and then?

No. To remind everybody the FDA has said, Tazeen, that we do not need to generate any additional clinical data, so it is literally pulling together all the magnitude of detail required to the highest quality submission to U.S. FDA and that will be completed in the fourth quarter. Our regulatory teams, I assure you, are hard at work.

I'm sure. Thank you very much.

Thank you.

And our next question comes from Anupam Rama from J.P. Morgan. Your line is now open.

Hello?

Your line is now open. If your phone is on mute, please unmute it.

Maybe operator, we can - if Anupam is not coming through, we could try to work that technical issue out and go to the next caller in the meantime.

Yes. And our next question comes from Joseph Schwartz from Leerink Partners. Your line is now open.

Thanks very much and congrats on all the progress. For EB, can you remind us whether you are having the physicians choose target ones that are in frictional and non-frictional zones of the body and how that's being controlled if at all? And in addition to your co-primary endpoints of target wound closure and time to wound closure, what do you think - how much do you think regulators appreciate that this disease is not a single wound in the way the drug is being used and measured and secondary endpoints - could help secondary endpoints like pain and itching in body surface area improvement?

Of course, thank you for both questions. Jay, I'll put this over to you.

For the selection of the target one by the investigators, that is at their discretion. Of course it's selected at baseline and then that is the wound that's followed for the remainder of the study. And for the target wound, the FDA had said that the success of the study based on the time to wound closure is based on the target wound as long as that - outcome for that primary endpoint has statistical significant less than or equal to 0.05 of p-value. That success with the study, irrespective of what's happening with other endpoints. Other secondary endpoints you mentioned some pain, itching and wound burden overall are supportive and potentially can enhance the overall effect that's demonstrated for the drug. But the success of the study is based solely on what the outcome is for the target wound.

Okay. And then turning to Pompe, where are you in the process of talking to regulators about what the next steps should be for designing a study that could support regulatory submission?

Yes, Joe, so as a matter of policy we are not going to comment on ongoing regulatory discussions. We think they will be ongoing for some time. The most important thing right now for us is to get this additional clinical data. That will certainly help to shape the view of the regulators in terms of next steps. For this program - but I assure you, we are in deeply engaged in regulatory strategy and planning, discussion, engagement with the top key opinion leaders around the world, who will help us. And the regulators frankly form an opinion around the fastest way to get this drug to as many patients as quickly as possible toward a marketing approval.

Okay. And then while I'm on the subject of your best bio-better ERTs. Can you give us an update on your Fabry ERT cell line development and when would you anticipate entry into the clinic?

Right, so we expect in the second half of this year to have some very important preclinical further proof-of-concept data that we think will further distinguish this treatment paradigm. And to remind everybody, Joe, this is our own biologics that under Hung's leadership was developed in our R&D organization and that it incorporates in the biologics formulation, migalastat, as a stabilizing agent for the ERT. Again the notion that it would keep it - we believe it would keep it more stable in blood, keep it folded, potentially make it less immunogenic, and once targeted then through the carbohydrates or the protein keeping it in the lysosome in a more active form. And what we've seen to-date are substantially higher levels of uptake of the enzyme to target tissues and organs and cell types, and significantly better breakdown of the GLT3 substrate. So we'll have more of that in the second half of the year. We are in the process of a final decision on manufacturing timelines and strategies for this. But I would expect all of this in the second half of the year to be very clear, including a timeline to the clinic.

Great. Thanks for taking my questions.

Thank you, Joe.

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

Hi guys. Thanks for taking the question. Maybe if I can just follow-up on Pompe program here and if you could reminder us the current status of large-scale manufacturing there and when could product be available for start of a phase 3?

Right, so we continue to make great progress, Mike. Remember we are operating at the 250 liter scale under GMP. That's the scale that's been producing the material for our phase 1/2 study. We are now in the process of moving from engineering batches that have been successful to full GMP batches at the 1,000 liter scale. That will be an ongoing activity through the second half of this year. And we would expect that if it was successful, the material to be available in the first half of the year to mid-2018 for the start of a phase 3 study.

Okay.

I'm very, very pleased with the quality and then also the productivity of the cell lines at 1,000 liters with additional work from our team and the team at Wuxi Biologics, we've seen some terrific success, all the while ensuring that we maintain optimized glycosylation and high levels of mannose-6-phosphate.

Okay. Great, thanks.

Yes, believe me, there is one thing that keeps me up at night is Pompe manufacturing and knocked on lots of woods, so far so good.

And at this time I'm showing no further questions.

Great. Well, thank you operator. Thanks everybody for listening. Again, we've got an enormous amount of work ahead of us here in the second half of the year. It's been a great start of course to 2017. But I do believe finely worth a point where we have a terrific portfolio of medicines, a tremendous team and a very strong balance sheet to help us achieve our vision and continue to execute. Thank you. Have a great day.

Ladies and gentlemen, thank you for your participation in today's conference. And this does conclude the program. You may all disconnect. Everyone have a great day.