Good morning, ladies and gentlemen, and welcome to the Amicus Third Quarter 2019 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 call is being recorded. I would now like to turn the conference over to your host, Ms. Sara Pellegrino, Vice President of Investor Relations. You may begin.

Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics’ third quarter 2019 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 Daphne Quimi, Chief Financial Officer. Also joining for Q&A are Dr. Jay Barth, Chief Medical Officer; Dr. Hung Do, Chief Science Officer; and Dr. Jeff Castelli, Chief Portfolio Officer and Head of Gene Therapy. 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 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 Statements and Risk Factors section of our annual report on Form 10-K for the year ended December 31, 2018 filed with the Securities and Exchange Commission, the third quarter press release and materials from today’s call which will be available on our corporate website, and the quarterly report on Form 10-Q for the quarter ended September 30, 2019 to be filed following today’s SEC holiday tomorrow. At this time, it is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer at Amicus. John?

Great. Thanks, Sara. And welcome everyone to our third quarter 2019 results conference call. I’m pleased to host today’s conference call to highlight the tremendous efforts and progress that we’ve made at Amicus during the third quarter and now into the early fourth quarter, beginning on slide three. A month ago, at our Analyst Day in New York, we grounded everyone in the vision for what we are building, the next great global biotechnology company, poised to impact many thousands of people around the world living with rare diseases. To achieve this vision, we are continuing to grow our very successful Galafold base business, which Brad will detail in a moment. We are also investing in the advancement of our leading rare disease pipeline, including our Phase 3 Pompe biologic AT-GAA, as well as our robust gene therapy pipeline and portfolio. We’re also maintaining a very strong balance sheet and financial discipline to keep us fully funded, well into the first half of 2022 through all major upcoming milestones, with a clear line of sight to profitability and self sustainability. So, let me move now to slide number four. Again, it’s highlighted at the Amicus Analyst Day in October and again today. Despite the volatility of the financial markets during the third quarter, our Amicus team focused intensely on the execution of our plans, and the achievement of extraordinary results. For Amicus, the third quarter was another significant period of growth and execution across all of our science, clinical, regulatory and commercial efforts. Here are the four key takeaways. First, Galafold continues to be one of the most successful launches for a rare disease medicine ever and remains the cornerstone of our success, with $48.8 million in third quarter revenue. We’re also now treating more than 1,000 patients at a…

Thank you, John, and good morning, everyone. Our financial overview begins on slide six, with our income statement for the three-month period ending September 30, 2019. For the third quarter 2019, we achieved Galafold revenue of $48.8 million, which is an increase of 137% over the third quarter of 2018. This includes year-over-year operational revenue growth measured at constant currency exchange rate of 143%, offset by a negative currency impact of $1.3 million or 6%. Cost of goods sold includes manufacturing costs as well as royalties associated with the sale of our products. Cost of goods sold as a percentage of net sales was 11.5% in the third quarter of 2019, as compared to 20.9% for the prior year period. Cost of goods sold as a percent of revenue was favorable as Galafold’s revenue continues to grow in the United States where we do not owe royalties, as well as other countries where we are subject to lower royalties. 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 third quarter of 2019 we reported $58.9 million in R&D expense as compared to $138.2 million for the prior year period. The decrease is primarily due to an upfront payment of $100 million for the Celenex asset acquisition in 2018, partially offset by continued investments in the Pompe clinical study program and our gene therapy pipeline. Total selling, general and administrative expense for the third quarter of 2019 was $39.7 million as compared to $31.9 million for the prior year period. The increase represents the expanded geographic scope of the ongoing Galafold commercial launch, including launch activities in Japan and the United States. Net loss…

Great. Thank you, Daphne. Good morning, everyone. Let me provide more color on the continued growth for Galafold in the third quarter and on our achievement of 1,000 patients on therapy at the end of 3Q. Our mission at Amicus is to get our medicines to as many patients as quickly as possible. And with this key milestone, we are increasingly confident in the potential to drive Galafold adoption in thousands of additional patients along the path to $500 million in global sales in 2023 and $1 billion peak revenue opportunity. Let’s begin with the global snapshot on slide 11. Total third quarter revenue, as has been mentioned, was $48.8 million, again a year-over-year increase of 137%, driven by exceptionally strong momentum in new countries, including the U.S. and Japan as well as a steady growth in our earlier launch countries. Specifically, this reflects $33.4 million or 68% of revenue generated outside of the United States and remaining $15.4 million or 32% coming from within United States. We have pricing and reimbursement now secured in 27 countries around the world, including three new additions this quarter the South Korea, Greece and Argentina. We already have the commercial team to support these global approvals, and we will continue to pursue opportunities to get Galafold approved and delivered to patients in more countries around the world going forward. This ongoing geographic expansion will continue to be an important growth driver along with continuing to switch patients from enzyme replacement therapy and increasing uptake in the diagnosed untreated market, which I’ll highlight more in a moment. Turning now to slide 12, let me comment on the positive momentum across all key global commercial metrics that we’re focusing on this quarter. First, at the global level, we estimate Galafold now has approximately 30% global…

Great. Thanks, Bradley. So, as many of you know, we just completed a detailed review of our entire portfolio during our October Analyst Day. So, for today’s call, I will highlight a few new pieces of information and briefly review the key takeaways. Many of the details that we covered at the Analyst Day can be reviewed on the event webcast and transcript, which of course is available on our website. So, I’ll begin here on slide 16 with our crown jewel, AT-GAA for Pompe disease. To remind everybody again, this is the first ever second generation breakthrough therapy designation for any lysosomal storage disease therapy product, as well as the first and only BTD for Pompe disease ever. We’ve seen tremendous momentum from what we believe may be the next standard of care, again for a broader population of people living with Pompe disease, and for products, representing what we believe could be a potential $2 billion plus opportunity. There are three new important updates with respect to AT-GAA on today’s call. First, we are reporting on the call today that the PPQ runs have now begun at WuXi Biologics. These will serve as the foundation for a CMC module for a BLA submission, which we continue to anticipate will be for full approval based on the results from pivotal PROPEL study. Second, we continue to make great strides in the PROPEL study enrollment, and we have now enrolled the target 100 patients in this study. This is a great achievement. As mentioned at Analyst Day, we are now targeting approximately 120 patients for this study in order to meet the extraordinary demand among physicians and patients across global participating sites. And we remain on track to complete that enrollment by year-end. We also continue our supported studies including…

[Operator instructions] Our first question comes from Anupam Rama of JP Morgan. Your line is now open.

Hi, all. This is Tessa on this morning for Anupam. Thank you for taking our questions and for the update here. A few from us on the Batten’s program specifically. First, can you remind us of latest thinking about next steps for the CLN6 program, specifically with an eye towards regulatory strategy here? And then, secondly, for the CLN3 program with dosing complete, as noted in prior remarks, how should we be thinking about next program update for CLN3? Thanks so much.

Sure. Thank you, Tessa. So, for the questions for CLN6 and CLN3, Jay, I’ll let you comment on what we see as next steps there.

Sure. Hello. We of course have dosed the initial cohort of patients and have -- also say the additional patients will be dosed in the CLN6 study with the aim of then engaging the regulators with the data that we have to discuss steps forward. And that’s something that we’ll be able to disclose after we have had those interactions. But, we plan to move ahead with the clinical study that we have now and expand actually the cohort in that study to include additional patients. That’s for CLN6. For CLN3, we -- as John stated before, we have dose the initial cohort of patients in the low dose moving towards dosing additional patients in the higher dose cohort with the anticipation when additional material is available to dose additional patients, presumably at the higher dose that shows the same safety as we’ve seen in the low dose. And once again with the body of data that we get in the Phase 1/2 study, engage regulators with that and discuss what the regulatory path forward would be for CLN3 as well. And this is something that will happen over the next year.

So, Tessa, just one thing to add to that in addition to all the clinical work and regulatory discussions that Jay has highlighted, I’ll also highlight a significant amount of the effort in both of these programs now involves the technology transfer, which is well under way for both of these programs to a Brammer and Paragon, respectively. And a lot of the work again will focus on all of the CMC requirements that would be necessary to support any BLA filings here.

Thank you. And our next question comes from Ritu Baral of Cowen. Your line is now open.

Hey guys. This is [indiscernible] on for Ritu. Thanks for taking my question. Just two quick ones for me, one on the Batten’s program. I’m just wondering, how are things going in terms of building out natural history. Is that really being done by Nationwide’s or you guys are also considering on cohort? And the second thing is, I know you’re initiating PPQ runs at WuXi, would there be potentially a possibility for submitting a rolling BLA starting next year? Just wondering how you guys are thinking about that. Thanks.

Sure. I’ll ask Jeff Castelli, our Head of Gene Therapy to talk about the natural history, which we think is going to be very important as the basis of approval for these Batten’s programs. So, Jeff, where are we with both of those?

Sure. Good morning. So, I think, as you’ve seen at Analyst Day, we’ve already provided some of the initial natural history data. That has all come from Nationwide. They have an ongoing study continue to compile data in that trial. We also have identified several other sources of natural history data. So, collectively, we are very optimistic we can get in the ballpark of 50 patients or so for a natural history dataset, which we think will be very robust for doing any kind of natural matched comparisons.

Sure. And just to your questions again with the PPQ runs initiated and a heavy focus on the CMC section for AT-GAA, we think particularly with the breakthrough therapy designation with this program that there is a potential for a BLA, a rolling BLA submission to begin. But again, we’re not going to comment on ongoing discussions with regulators. Once we have clarity on the potential there, we’ll be able to provide an update as well.

Thank you. And our next question comes from Joseph Schwartz of SVB Leerink. Your line is now open.

Hi. Good morning, guys. Thanks for the update. And congrats on all the progress. So, two questions for me. This is Dae Gon dialing in for Joe. Maybe for Jay or Jeff, if we can maybe first touch upon Batten’s disease. So, when we saw the latest update at the Child Neurology Society presentation, like you mentioned in the prepared remarks, we saw some sub scores. Particularly, I was wondering about what your thoughts were with regards to the visual acuity that we saw in three out of the six. I think, it’s older patients that seem to progress. So, any thoughts on intravitreal administration of the CLN6 gene therapy? And then on the second part, with regards to the Pompe gene therapy, can you remind us, is this the same candidate you presented the preclinical data for at ASGCT? And, I understand that there is increasing competitive forces sort of coalescing in this area. But, any additional data that we can expect to emerge before you enter into the clinic or any specifics you can provide on this candidate? Thank you very much.

Yes. Jay, I’ll let you take the first part with the additional data for Batten that we showed in at the Child Neurology...

Yes. To bring everyone up to speed on what was presented at Child Neurology Society a couple of weeks ago that you’re referring to. It was both more granular data on the Hamburg Motor & Language Subscores, which -- both combined and separately, which showed stabilization, both in the combined motor and language, plus each component individually motor and language are very consistent with one another, showing that the effect of the gene therapy on the patients, most profoundly in the younger patients, but in almost all of the patients as well when compared to natural history matched comparison in terms of slowing the progression of the disease or frankly stabilizing it. That was in the motor and language. The additional new data were on the vision scale within the Hamburg and the seizure scale within the Hamburg. Both showed general stabilization or a -- following a progression of the disease, a caveat. And I think, we said at the time for the visual scores on the Hamburg, is that is just one. It’s the first data that we have on the vision available. But, it will be more data related to visual acuity and other visual tests that will be coming up in the future. So, we’ll share that when we have that analyzed as well; and on the seizure subscale as well showing stabilization of trajectory in all but the most advanced patients. That was what we had shown. I’ll let Jeff comment on the other question about intravitreal or other administration going on.

Yes. So, there actually is a published preclinical manuscript with our CLN6 gene therapy showing an improvement in the eye in the animal studies. So, we are hopeful that the initial intrathecal treatment can at least partially address the vision defects. And as we continue to see more data, we’ll assess if there could be a benefit of adding an additional route down the line. Right now, we’re optimistic that the intrathecal approach will have an improvement across all the different functions. And like we’ve seen, older patients may continue to climb some or as we hope to see really more effect in younger kids in terms of stabilization.

Yes, I’ll just comment on the second question then for the Pompe gene therapy data. And yes, this is the initial data that we presented at the gene therapy meeting in Washington back in April. Again, this was something that we and Jim Wilson and his team were incredibly pleased to see these results that we presented back in April. We continued preclinical proof-of-concept through the summer into the early fall and then we of course -- we had the additional data that we showed at Analyst Day that allowed us to declare this as the candidate to move forward here. Look, there are a lot of people now involved in Pompe gene therapy, which I think is excellent. I think, it offers a lot of promise to people living with Pompe. It kind of takes me back to 21 years ago when I read my first paper on Pompe gene therapy. And now after all these years to finally see people moving forward I think is a very good thing. What we’re doing at Amicus though is a very differentiated approach to a gene therapy here. And our goal is to provide a complete approach to addressing this disease through gene therapy through many steps that we’ve taken with Dr. Wilson and his team. These include the protein engineering to optimize secretion, expression and targeting to key tissues of disease that includes muscle, and very importantly CNS targeting to reach within motor neurons. Again, Pompe is a neuromuscular disease and a disease of motor neurons. So, those steps coupled with what we believe to be the optimal promoter in the right vector we think could lead to a highly differentiated program. It’s something we’re very eager now to move forward.

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

Hi, there. This is Keith on for Mohit. And thanks for taking our questions. And of course, congrats on the progress this quarter. I just wanted one quick question for you. Could you walk us through what the R&D and cash runway guidance reflects, as we approach the end of 2019 and then for the flat year-over-year guidance for 2020, what beyond the current programs is baked in, or should we expect higher spending in connection with expanded programs, particularly with respect to the agreement with Penn?

Sure. I think, we were pretty clear on the call, but we’re happy to repeat it. Daphne, do you want to tackle those?

Sure. So, what’s included in the R&D spend is -- are the key priorities that John discussed at the beginning of the call, which is to continue to move AT-GAA forward through approval and then focusing on our gene therapy pipeline. So, all of that spend is included in the cash runway guidance as well as the operating expense guidance that we’ve given.

Yes. Thank you, Daphne. I just want to be perfectly clear again. What we’re doing now is we’ve laid the foundation through very significant investments in R&D for our crown jewel for AT-GAA. And we’ve also made some early investments in gene therapy. With the R&D expenses, the significant amount that we’ve invested over the last couple of years, that total dollar amount, you should expect to remain relatively flat in the coming years. And AT-GAA is a very expensive program, the largest lysosomal study and the most expensive I’m sure, lysosomal study ever conducted, again to fund this crown jewel. That spending now is going to be able to be redeployed as AT-GAA moves from clinical development toward approval and commercialization. We can take the expenditures there, move them to multiple gene therapy programs. Depending on the gene therapy programs, we can fund 5 to 10 clinical stage gene therapy programs, including all the R&D necessary to support these programs for the AT-GAA program. In addition, on the SG&A side, we’ve enabled the very capable, highly capable commercial organization now globally to deliver Galafold successfully. It’s now preparing in the early stages for a launch for AT-GAA. When we launch AT-GAA, we think we need to hire maybe 20 additional people globally to support that so effectively remaining flat. So, highly leverageable infrastructure. So, with all of that, this year again, operating expenses, SG&A and R&D at $410 million to $420 million, I’m very confident will come in, in that range. And that range should be one that carries forward for at least the next three years through the end of 2022 to reflect the advancement of all these programs.

Thank you. [Operator Instructions] And our next question comes from Earl DeSouza of H.C. Wainwright. Your line is now open.

Hi. Good morning. Thank you. Most of our questions have been answered already. But, I had a quick housekeeping question. So, is there any significant room to lower your COGS from the 12% range, given the anticipated volume growth? Thanks.

Yes. The only thing we’d say is -- from a manufacturing perspective, it’s a pretty steady state process at this point and distribution as well. The one place where you see a little bit of movement, as Daphne highlighted, is in the royalty. Our royalties are limited to ex-U.S. geographies. And so, as the U.S. and non-royalty producing countries continue to be a higher contributions of sales, you might see that number of creep down a little bit. Really that’s just a function of the distribution of revenue between royalty and non-royalty. We should not expect further savings from a operating cost of goods perspective.

Thank you. And our next question comes from Yun Zhong of Janney. Your line is now open.

Hi. Thank you for taking the questions. So, the question is on the Pompe gene therapy program. I believe you’ve talked about the potential additional animal species for preclinical study and also alternative route of administration. So, I wonder, do you think IV is still the best way to deliver. And do you plan to study the candidate in for example non-human primate? And also, at what point do you plan to disclose what kind of AAV capsid that is? Thank you.

Sure. I’ll ask Jeff Castelli to field that.

Yes, sure. So, we are moving forward with all of our preclinical IND-enabling studies which will include primate studies. As we’ve mentioned before, we are definitely going to address the CNS aspects or look to address the CNS aspects. While we’ve seen in our studies that IV administration led to robust impact in the CNS, as we go through those primate studies, we might determine that we need to do a combination of IV plus intrathecal to make sure we address the CNS. And we will provide that data as we have it. But right now, we’re really excited by the overall construct and its potential whether it’s dosed IV alone or IV plus intrathecal.

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

Thanks for taking our questions. This is Andrea on for Salveen. My first one is, when you think about your goal of moving Galafold to use for mostly switch patients to predominantly treatment-naïve patients. Can you help us understand the gating factors to achieving this transition?

Yes, sure. And remember, we still have quite a bit of room left to grow from a switch perspective. U.S. and Japan, as an example are more recently launched countries. And our strategy has always been to focus on the switch patients first, because they are the ones that are already in the system, they’re getting treated every other week with an alternative therapy. And so, that’s the initial population and there is lots of switch patients still to move. That being said, in our more mature launch markets, we’re starting to see roughly equal rate of growth from switch and naïve patients. And I think, there are a number of things there. First of all, again, those patients are not coming in every other week for their infusions. So, you have to reach out to those patients through the physicians. I think, also, as experience builds with the product that gives physicians and patients more confidence to take a therapy -- a relatively new therapy as their first treatment for their Fabry disease; and obviously, also, a big factor of the diagnosis rates we’re seeing. We continue to see a 10% to 20% diagnosis rate in Fabry disease that’s driven by newborn screening, targeted population screening, other diagnostic initiatives. So, that will also contribute to that increasing rate of previously untreated patients coming on to Galafold.

Thank you. And at this time, I would now like to turn the conference back to Mr. John Crowley, Chairman and CEO for closing remarks.

Great. Thank you, operator. Thank you, all for listening, again a very, very successful quarter. And again, we remain focused on delivering on our mission for patients and for shareholders. Thank you for listening. Have a great day.

This concludes today’s conference call. Thank you and have a great day.