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Edited Transcript of FOLD earnings conference call or presentation 8-Nov-17 1:30pm GMT

Thomson Reuters StreetEvents

Q3 2017 Amicus Therapeutics Inc Earnings Call

Cranbury Nov 14, 2017 (Thomson StreetEvents) -- Edited Transcript of Amicus Therapeutics Inc earnings conference call or presentation Wednesday, November 8, 2017 at 1:30:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Bradley L. Campbell

Amicus Therapeutics, Inc. - President and COO

* John F. Crowley

Amicus Therapeutics, Inc. - Chairman and CEO

* Sara Pellegrino

Amicus Therapeutics, Inc. - Senior Director of IR

* William D. Baird

Amicus Therapeutics, Inc. - CFO

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Conference Call Participants

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* Anupam Rama

JP Morgan Chase & Co, Research Division - VP and Analyst

* Joseph Patrick Schwartz

Leerink Partners LLC, Research Division - MD, Biotechnology

* Kerry Tang

Goldman Sachs Group Inc., Research Division - Research Analyst

* Michael Eric Ulz

Robert W. Baird & Co. Incorporated, Research Division - Senior Research Analyst

* Tazeen Ahmad

BofA Merrill Lynch, Research Division - VP

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Presentation

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Operator [1]

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Good day, ladies and gentlemen, and welcome to the Amicus Therapeutics third quarter results conference call and webcast. (Operator Instructions) As a reminder, this call is being recorded.

I would now like to introduce your host for today's conference, Sara Pellegrino, Senior Director of Investor Relations. Please go ahead, ma'am.

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Sara Pellegrino, Amicus Therapeutics, Inc. - Senior Director of IR [2]

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Thank you. Good morning, and thank you for joining our conference call to discuss Amicus Therapeutics Third 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 take undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof.

For a full description of such forward-looking statements and the risks and uncertainties that may impact them, we refer you to the forward-looking statements on Slide 2 of our third quarter 2017 results slide deck, the Forward-looking Statements and Risk Factors section of our annual report on 10-K for the year ended December 31, 2016, as well as our quarterly report on Form 10-Q for the quarter ended September 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.

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John F. Crowley, Amicus Therapeutics, Inc. - Chairman and CEO [3]

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Great. Thank you, Sara, and good morning, everybody. Let's begin on Slide 3. So 2017 has, indeed, been a remarkable year at Amicus, and we continue to make significant progress across our 4 priorities for our key programs in Fabry and Pompe disease. We believe that we've created a strong foundation for further value creation throughout this year and in the years to come, which will enable us to deliver on our mission for patients.

Let's begin with the Galafold launch. First, we are executing very successfully with our international launch of Galafold, which is our oral precision medicine as an every-other-day capsule for people living with Fabry disease who have a amenable mutations. Growth in patient and physician adoption has picked up significantly during 2017. As of October 31, in our first full year of launch, we now have more than 260 patients in 13 different countries treated with reimbursed Galafold. This growth in new patients, in addition to geographic expansion, reflects the strength of the Galafold label and the data set, the success in relationships of our world-class global Amicus team and our broader commitment to access for as many Fabry patients with amenable mutations as quickly as possible throughout the world. While Brad will review the launch here in more detail in a moment, I'll also add that we are seeing extremely high rates of compliance and adherence with this oral precision medicine.

Our second priority for the year is to complete additional global regulatory submissions for Galafold or migalastat. We have made significant progress there in several key geographies, including Japan, where we have submitted our Japanese new drug application, or J-NDA, during the second quarter. In addition, during the third quarter, we received 3 new regulatory approvals for migalastat in Israel, Canada and Australia. And in the United States, our NDA submission for the U.S. FDA is on track for -- before the end of this year.

Turning now to Pompe disease. Our Pompe disease program continues to advance, including our ongoing collaborative discussions with regulators, to determine the best and fastest pathway for our highly differentiated treatment approach, ATB200/AT2221 for this severe, fatal neuromuscular disease with significant unmet medical needs. The data cascade for this novel Pompe treatment paradigm, especially the latest data set announced in early October at the World Muscle Society meeting in France, has continued to exceed our expectations in terms of the consistency, durability and magnitude of effects across patients and across functional outcomes, key disease biomarkers and safety.

Later on this call, we will review the data highlights and provide a few key updates related to the initiation of important new retrospective and prospective studies, as well as our significant progress and success with manufacturing of the protein. As a reminder, ATB200/AT2221 is an important experimental therapy at Amicus that leverages our biological expertise and our proprietary CHART technology to develop chaperone-enzyme replacement therapy, or ERT, combinations that may benefit patients with lysosomal storage disorders, such as Pompe disease.

Let me comment, before turning it over to Brad, just briefly about our financial strength. Our fourth priority, of course, is to remain diligent in maintaining a strong balance sheet through our continued focus on successful execution for our key drivers in Fabry and Pompe disease, creating, we believe, a meaningful difference for patients and significant value for patients and shareholders.

With more than $425 million on the balance sheet, we expect to end the year with more than 18 months of cash and with runway into at least the second half of 2019.

So with that introduction, let me go ahead and turn the call over now to our President and Chief Operating Officer, Brad Campbell. Brad?

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Bradley L. Campbell, Amicus Therapeutics, Inc. - President and COO [4]

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Thanks a lot, John. Good morning, everybody. Let's begin on Slide 5 with an update on our launch metrics, all as of October 31. This year continues to be about opening new countries and driving successful launches in the countries that are open. And during the third quarter, the launch curve steepened significantly, reflecting growth in patient and physician adoption across our key countries.

First, as John mentioned, we now have over 260 patients on reimbursed Galafold as of October 31. This growth in patients reflects significant adoption across our launched countries, including Germany, France, the U.K. and Italy, as well as a number of the smaller and midsized countries throughout Europe, including Ireland where, just last week, Galafold became the fastest orphan drug to complete the Irish pricing and reimbursement process, which we think reflects the overall value that Galafold can bring as a new treatment option for Fabry in these markets.

Before I move on to some of the launch detail, let me make one quick comment on the patient update. You've noticed that this quarter, we've moved to providing a round number of more than 260 patients. As we're into the multiple hundreds of patients from over a dozen countries, these numbers can change on a daily basis, and as a result, we'll be speaking in round numbers from here through the end of the year.

Moving on to pricing reimbursement. We've continued to have a successful year through this pricing reimbursement process, which are really important metrics for tracking the Galafold launch. We've secured pricing now in 13 countries, including 4 of the top 5 EU markets. And in addition to several of the midsized EU countries, with discussions underway in an additional 12 countries, we also continue to have an Amicus footprint, either directly or indirectly, through partners on the ground in 27 countries around the world as we prepare to expand the launch into additional geographies.

Based on the current patient numbers as well as our track record of continuing to be successful in our pricing and reimbursement discussions, we're extremely pleased and proud that we remain on track to achieve our goal to treat 300 patients with reimbursed Galafold by the end of 2017.

From a commercial patient demographics perspective, consistent with last quarter, with more than a dozen countries now contributing to the launch, and we won't provide any specific country-level detail on today's call, but I will continue to provide some general detail on consistent strength of some of the additional patient demographics that are important metrics to track through the launch. We continue to see adoption from our 3 key patient segments, including patient switching from infused enzyme replacement therapy, which continues to represent the bulk of our patients in these early months of launch, a growing portion of patients who were previously diagnosed but not treated and a number of newly diagnosed patients who have started on Galafold as their first-ever treatment for Fabry disease. These 3 key patient segments continue to include a balanced mix of males and females, classic mutations and late-onset mutations throughout our launch countries.

From a utilization perspective, we're seeing great uptake, in line with our launch strategy, as well as a very high rate of compliance with the oral every-other-day dosing regimen across the spectrum of Fabry patients with amenable genetic mutations that's specified in our label.

If you look outside of Europe, we'll turn to Slide 6, I'll highlight a few of our key global regulatory milestones. As reminder, our strategy is to maximize the access to Galafold for Fabry patients who have amenable mutations as quickly as possible in as many geographies as possible.

Outside of Europe, as John mentioned, we have secured 4 additional regulatory approvals for Galafold: in Australia, Canada, Israel and Switzerland. We have 2 expanded access programs underway and expect to open additional expanded access programs. We have approvals pending in several countries, including Japan. And in the U.S., as John mentioned, we're on track to submit our NDA by year end.

So in closing, significant growth in our EU launch and global regulatory progress. This reflects the strength of our data, the significant value of our oral precision medicine and the unmet need in Fabry patients. Our long-term goal, of course, is for all Fabry patients and physicians to have an Amicus treatment option driven entirely by their patient's genotype. For now, we continue to commit -- to be committed to broad access to Galafold for all Fabry patients with amenable mutations. And for those patients with nonamenable mutations, we continue our preclinical development of a novel, proprietary Fabry enzyme replacement therapy co-formulated with migalastat to enhance that ERT activity.

With that, let me turn the call back to John to summarize our recent data, updates and provide milestones related to our Pompe disease program. Thank you.

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John F. Crowley, Amicus Therapeutics, Inc. - Chairman and CEO [5]

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Thanks, Brad. So let's begin with a brief summary of our Pompe clinical data cascade, which, as I mentioned earlier, has exceeded our expectations in terms of magnitude, durability and consistency across all 3 patient cohorts. As a reminder, on Slide 8, we enrolled ambulatory ERT-switch patients who had previously been on standard of care, nonambulatory ERT-switch patients who had previously also been on standard of care and ERT-naive patients who had not previously been treated for their Pompe disease.

On Slide 9, you can see the breadth of available data on safety, functional outcomes and biomarkers that are now available across each of the 3 patient cohorts at both 6 and 9 months.

Turning to Slide 10, let's focus on the 6 key takeaways from the clinical data that we've generated to-date in our Pompe clinical study. First, in terms of safety, we are pleased with a very low number of infusion-associated reactions, less than 1% of 400-plus total infusions to-date since the beginning of this study.

Second, we continue to see increases in the mean 6-minute walk distance for both the ERT-switch population as well as the ERT-naive population. The 6-minute walk distance is the most important endpoint of motor function in Pompe clinical studies for ambulatory patients. And very importantly, this affect in both patient cohorts was durable out to 9 months.

Third, all 4 of the nonambulatory Pompe patients who are in a wheelchair with available data have shown improvements in muscle strength test, following 6 months of treatment.

Fourth, muscle function across all 3 cohorts improved in 16 out of 18 patients and was stable in the other 2 patients at 6 months. And muscle function improved in 10 out of 10 patients with available data at 9 months.

Fifth, pulmonary function generally improved or remained stable based on the primary measure used, which was forced vital capacity, or FVC.

And sixth, I would like to point out the magnitude, consistency and durability of improvement in the key biomarkers of muscle damage and disease substrate for up to 58 weeks.

So overall, it is very exciting for us to see the consistent and durable improvement in muscle function and key biomarkers as well as stabilization or improvement in respiratory function. In over the past month, we've had the opportunity to share and discuss these results with many stakeholders, including our clinical investigators. The feedback has been very positive across the board, and we have received many requests for access to the -- this investigational therapy. In view of our broad Amicus commitment to patient access and given our success in manufacturing, we are committed to providing access to as many people as possible living with Pompe a soon as we can, including considering adding more patients to our existing clinical studies.

In the meantime, we continue to advance this important Pompe disease program forward. And on today's call, on Slide 11, as you'll see, we provide key updates as we build a robust data set in Pompe patients. A few things to highlight here. We have initiated a retrospective natural history study of Pompe patients treated with approved standard of care ERT at leading global Pompe disease treatment centers. This study is called POM-002. Also, a prospective observational study has also been initiated in late-onset Pompe patients currently receiving approved standard of care ERT at leading global Pompe disease treatment centers. This study is referred to as POM-003. Both studies are intended to build upon that body of knowledge of the understanding of the natural course of Pompe disease on the current standard of care ERT. We are also in the midst of collaborative discussions with U.S. and EU regulators and continue to expect an update on the best and fastest regulatory pathway during the first half of 2018. Finally, we also plan to present additional clinical data from our ongoing Phase I/II clinical study at the WORLDSymposium in February in San Diego.

Just to comment briefly on manufacturing. On the manufacturing front, if you turn to Slide 12, you'll note that we have successfully completed all engineering runs and have now commenced GMP production of ATB200 at the large post-approval scale, 1,000 liters.

We have also completed analytical and in vivo preclinical comparability studies between the 250-liter and 1,000-liter scale engineering batches. Discussions with FDA on whether these studies are complete or if additional comparability studies are needed are still to be determined, but we are very pleased with the results of these comparability studies to-date.

So with that, let me now turn the call over to Chip, our Chief Financial Officer, to discuss the third quarter financial result. Chip, please.

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William D. Baird, Amicus Therapeutics, Inc. - CFO [6]

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Thanks, John. Good morning, everyone. Our financial overview begins on Slide 14 with our income statement for the third quarter. In the third quarter of 2017, we recorded revenue of $10.9 million, representing a sequential increase of 51.4% over the total revenue of $7.2 million recorded in the second quarter of 2017. Total revenue for both periods represents commercial sales of Galafold as well as reimbursed expanded access programs.

Moving down the income statement. Total R&D expense for the third quarter of 2017 increased to $40.6 million as compared to $32.5 million for the third quarter of 2016. The increase here in R&D expense included a $6.2 million increase in costs related to the SD-101 program and a $4.5 million increase in costs related to ATB200. The increase in SD-101 costs included accelerated recognition of costs related to the wind-down of the Phase III ESSENCE study. The increase in Pompe costs was due to investment in Pompe manufacturing scale-up as well as continued investment in the Phase I/II study.

Total selling, general and administrative expenses for the third quarter of 2017 were at $21.6 million as compared to $17.5 million for the third quarter of 2016. The increase here represents the expanded geographic scope of the ongoing Galafold commercial launch.

There were several noncash items impacting the income statement this quarter, stemming from the results of the Phase III ESSENCE study and our decision to discontinue further investment in and development of this program.

And again, all the items I'm about to describe are noncash charges. We recorded a $244 million gain in the fair value of contingent consideration, stemming from the fact that we will not owe any additional milestones to the former [side] shareholders. We recorded a $465 million loss on the impairment of in-process R&D related to SD-101. And finally, you'll see that we recorded $164.7 million income tax benefit related to the reduction in deferred tax liability, which was tied to SD-101.

Net loss for the period was $111.7 million or $0.69 per share compared to a net loss of $46.7 million or $0.33 per share for the third quarter of 2016. Net loss, excluding the impact of these noncash charges related to the ESSENCE study, was $65.6 million or $0.41 per share. And as of September 30, 2017, we had approximately 165.5 million shares outstanding.

Moving on to Slide 15, a few comments about our current cash position and 2017 financial guidance. Our cash, cash equivalents and marketable securities totaled $426.6 million on September 30 compared to $330.4 million at the end of last year. Our balance sheet was strengthened in July with a successful completion of a $258 million follow-on public offering. Total net cash spend for the 9 months ending September 30, 2017, was $147.3 million. So we are tracking well against our full year 2017 expense guidance. We continue to expect full year net operating cash spend of between $175 million and $200 million and full year total net cash spend, including third-party milestone payments and capital expenditures of between $200 million to $225 million.

As it stands today, our current cash position is now expected to fund operations through at least the second half of 2019 and, as John highlighted, through several key catalysts.

This summarizes our key financials for the third quarter. Additional details will be found in our annual report on 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, I'll turn it back to John.

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John F. Crowley, Amicus Therapeutics, Inc. - Chairman and CEO [7]

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Great. Thanks, Chip. So it was a very successful third quarter with respect to a number of key initiatives, including the Galafold launch, regulatory progress for migalastat and our very important Pompe disease program.

As outlined on Slide 17, we have a lot of work ahead of us for the remainder of the year and into 2018 that we hope will create even more value for shareholders and will continue to make a meaningful difference for patients.

With respect to our Fabry program, to summarize, we are on track to achieve our target of 300 patients on reimbursed Galafold and to submit the NDA to the U.S. FDA by year end. We also anticipate a decision on our new drug app -- I'm sorry, anticipate a decision on our new drug application in Japan in the first half of 2018.

With our Pompe program, we plan to present additional data from our Phase I/II clinical study in the first quarter of next year during the WORLDSymposium in February, and we are on track to provide a Pompe regulatory update in the first half of 2018.

So as we advance our key value drivers in Fabry and Pompe, we are well capitalized with cash runway until at least the second half of 2019 to continue to build one of the world-leading global biotechnology companies focused on rare devastating diseases.

With that, operator, happy to take any questions.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question is from Anupam Rama of JPMorgan.

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Anupam Rama, JP Morgan Chase & Co, Research Division - VP and Analyst [2]

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Just a quick one, in the press release, just thinking about the Fabry ERT cell line here in 1Q '18, how should we be thinking about that? And looking -- what should we be looking for within that preclinical data for potential differentiation relative to current ERT? And will that update, including preclinical chaperone combination data, that we should be thinking about?

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John F. Crowley, Amicus Therapeutics, Inc. - Chairman and CEO [3]

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Sure. Thanks, Anupam. So to remind everybody, Galafold is intended for use where its approved for patients with amenable mutations. For those patients with nonamenable mutations living with Fabry disease, which is half or more of the Fabry population, we have developed our own proprietary enzyme replacement therapy. That therapy is co-formulated. So in the manufacturing, we combine the chaperone, migalastat. And the purpose is to confer additional stability and enhance the activity of that protein, so that when it's properly targeted to the key organs and key tissues, it can act in a number of ways. First, we can get additional enzyme into the key tissues and key organs. And secondly, we can also reduce the substrate further, and we've seen that in preclinical studies that, I think, Anupam, is the further data that we'll have, the magnitude of the impact and the increase. We also believe that with the confirmational stability added with the chaperone that we have the potential to provide even more of the enzyme replacement therapy, potentially higher doses to patients as well, which, again, could lead to further enzyme uptake and further substrate reduction. So a very important program for us. It's part of our broader commitment to the Fabry community and part of our vision for that program, so that 1 day, potentially all patients living with Fabry disease may have 2 different options driven by their genotype for treatment of Fabry.

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Operator [4]

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Our next question is from Tazeen Ahmad of Bank of America.

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Tazeen Ahmad, BofA Merrill Lynch, Research Division - VP [5]

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John, I just wanted to get a sense from you on what data we should expect to see at the WORLD meeting? Specifically, I'm wondering, would we have 12-month data at that point for the majority of the patients that were enrolled into the study? And then I have a couple of follow-ups.

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John F. Crowley, Amicus Therapeutics, Inc. - Chairman and CEO [6]

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Yes. I think in the early part of 2018, we'll be able to articulate what data to expect at the WORLD meeting. With respect to the clinical data that you referred to, Tazeen, we'll certainly have more 9-month data on patients. If you recall, we have -- previously, in the update in early October at the symposium in France, we've provided data on 10 patients with functional measures at 9 months. We'll have more data at 9 months. I would also expect that we will have data on patients at 12 months. The exact number is not yet clear.

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Tazeen Ahmad, BofA Merrill Lynch, Research Division - VP [7]

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Okay. And then on the Pompe program, so let's say, in a blue sky scenario, if FDA were to agree to a potential expedited path for approval, would your current state of manufacturing allow you to move at that accelerated pace? Or could that be rate-limiting?

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John F. Crowley, Amicus Therapeutics, Inc. - Chairman and CEO [8]

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So I'll remind everybody that our current base plan is that we will have to do a Phase III study. However, we are committed to looking at the most expedited path for patients to make the drug available to as many people living with Pompe. If there were an expedited scenario, we have been working with our partners at WuXi to reserve sufficient capacity, so that in any scenario, we'd be able to supply a sufficient number of patients, including patients at a market launch. So engineering batches have now been completed. Now at the GMP scale material at the 1,000 liters scale, so having the quality of material, but then also having the quantity is very important to us, and we are laser-focused on manufacturing both with respect to quality of manufacturing as well as quantity and capacity. And there, I'm very, very comfortable that we've got a strong plan. So no, it will not be rate -- but no, we do not expect it to be rate-limiting.

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Tazeen Ahmad, BofA Merrill Lynch, Research Division - VP [9]

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And then maybe one for Brad. On Galafold in Europe, what's your sense now that you've been on the market for about a year, on the environment regarding pricing? So would you expect price to stay flat in Europe? Or could it be a bit lower next year, given, for example, with other drugs in Europe, it has been the case historically that you do see price erosion albeit at a slow pace over time?

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Bradley L. Campbell, Amicus Therapeutics, Inc. - President and COO [10]

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Yes, it's a good question. I think -- first of all, we continue to be focused on the front end of that phase, which is securing pricing reimbursement. And again, as we mentioned the call, with Ireland, we continue to see great response from the reimbursement authorities to our pricing strategy, which is parody enzyme replacement therapy, which, again, we thinks reflect -- we think reflects the value of the product but also brings some savings back to the system and avoiding any infusion-associated costs. So, so far, strategy has been very successful, we believe. Next year, we will continue to expand that list of geographies to secure pricing and reimbursement. We've alluded to 12 countries where we have pricing reimbursement discussions underway. You have some big markets, Japan and United States, that we're trying to secure approval in. Those tend to be in the higher end of the pricing range. So I would say, we're still on the front end of that curve. As you suggested in the long term, you tend to see price erosion in Europe. But for the foreseeable future, I think we're still in the front end of that process.

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Operator [11]

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Our next question is from Joseph Schwartz of Leerink Partners.

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Joseph Patrick Schwartz, Leerink Partners LLC, Research Division - MD, Biotechnology [12]

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So I was hoping to ask a little bit about the retrospective natural history study, POM-003, that you've initiated. In the context of your discussions with the FDA, how far along is this project? And could this data provide a viable comparator arm, as the FDA said, could suffice in certain cases? I know they asked Sanofi Genzyme to run another Phase III/IV for neoGAA. But I think arguably, that data was less strong than yours at a similar state. So how should we think about the retrospective natural history data, its ability to be compared with yours -- your data that you've generated in Pompe to date?

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John F. Crowley, Amicus Therapeutics, Inc. - Chairman and CEO [13]

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Great. Thank you, Joe, for the question. I just want to clarify, the POM-002 study, the retrospective study, that is POM-002, so 2 different studies. One retrospective and one observational and prospective. Both of which, we think, are intended to provide important supportive for a BLA. The 002 study, by retrospectively collecting data in patients who had previously been treated with the standard of care ERT, in addition to prospectively evaluating patients who are currently on ERT standard of care. Frankly, data from both studies could support a BLA, build that body of knowledge, and the observational study could also be available for either a base case Phase III study or any ongoing clinical development or post approval. So just to be clear, I -- we think these are studies we have planned for some time, obviously, strengthened our perspective with the data that we revealed a month or so ago at the conference on the public of Pompe data in France. So again, multiple purposes to these studies, and that we think very important data to build on the body of knowledge, these studies could have multiple regulatory purposes.

Great. Does that make sense, Joe? We may have lost Joe. Okay, hopefully, that's good.

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Operator [14]

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(Operator Instructions) Our next question is from Mike Ulz of Baird.

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Michael Eric Ulz, Robert W. Baird & Co. Incorporated, Research Division - Senior Research Analyst [15]

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Just thinking about the international launch of Galafold and, in particular, Japan with approval expected in the first half of next year, maybe can you just comment on the current status of launch preparations there and remaining steps as we start to get near to the launch?

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Bradley L. Campbell, Amicus Therapeutics, Inc. - President and COO [16]

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Yes. Thanks, Mike, for the question. Preparations in Japan continue to go really well. We have a very seasoned Japanese General Manager that we brought onboard, and he's busy, as you might expect, recruiting out a team. We have a K.K. established with all the formal requirements that go into that, which is one of the key rate-limiting steps for starting up the preparations for Japan. It's a market that we think has a lot of opportunity for migalastat, specifically. We've talked a little bit before about the experience in our clinical studies. The technology came out of Japan. The original chaperone Galafold technology came out of Japan and is a high level of reimbursement and treatment for patients with Fabry disease. So we think a lot of the demographics there lend themselves well. As you might expect, we're doing all of the necessary medical affairs activities that are appropriate at this point in time. We're hiring the team, and we will be ready and eager to go as soon as we can hear back from the Japanese FDA.

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Operator [17]

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Our next question is from Salveen Richter of Goldman Sachs.

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Kerry Tang, Goldman Sachs Group Inc., Research Division - Research Analyst [18]

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Congrats on all the launch progress. And this is Kerry on the line. I just have a few questions. First, with Galafold approved and the major EU and additional countries now, and I remember, most of the revenues came from Germany in the prior quarters, can you give us a breakdown and color of the contribution from different regions and whether we should see any further kind of sales inflection at some point in the future? And are you seeing a similar traction in other countries as you are in Germany with regards to kind of the ERT-switch population? And I just have a follow-up.

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Bradley L. Campbell, Amicus Therapeutics, Inc. - President and COO [19]

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Yes. We won't provide specific country-level demographics. We use Germany because, initially, during the launch, it was really the largest market. The only market that was open right out of the gate. At this point, I can say that it is balanced. That launch progress is balanced. As you might imagine, the EU 5 countries -- 4 of the EU 5 countries where we have pricing reimbursement are the lion's share of patients based on their population sizes. But we're seeing good uptake on a comparable basis in other markets, the Nordics, the middle European markets, the smaller markets, et cetera and some outside of Europe as well. And we would expect to continue to see those demographics play out in a comparable way. Again, the switch population continues to be the largest share of our commercial patients today. However, we're seeing good uptake in the other segments, as I mentioned on the call. From the -- from a driver's perspective, if you look into next year, obviously, the 2 largest markets that we hope to bring onboard are Japan and the United States where we're hoping to file that by the end of this year. But you have other important markets outside of Europe as well, Australia, Canada and others where we have regulatory approval and are seeking reimbursement approval. So I think lots of opportunities to continue to open up large markets and to continue to drive the launch.

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Kerry Tang, Goldman Sachs Group Inc., Research Division - Research Analyst [20]

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Okay. That's very helpful. And then just finally, on the Pompe program. We saw a time-dependent increase in drug-treatment effect at month 6 and 9, especially in the ERT-naive cohort. Do you anticipate this to kind of continue to increase or kind of plateau? And how should we think about that? And finally, will you be presenting antibody and cytokine assessments from the ongoing study at some point?

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John F. Crowley, Amicus Therapeutics, Inc. - Chairman and CEO [21]

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Sure, Kerry. In terms of how much stronger and what further improvements we can see in these patients, we don't know. And we'll be collecting that data. Biologically, there is no reason why these patients with residual muscle couldn't potentially continue to see improvements. And that's a key part of what we'll look at out past 9 months, is the effect persistent and durable and, in fact, is it even increasing, we don't know. In preclinical models, we're encouraged, certainly, by what we see in the longer term and in the reversal of the muscle damage, both on the pathology but then also in the functional measures. So we'll see as the data goes forward. In terms of antibody and cytokine data, we'll be able to comment in the early part of '18 and what we will be able to see at WORLD, specifically.

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Operator [22]

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Our next question is from Joseph Schwartz of Leerink Partners.

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Joseph Patrick Schwartz, Leerink Partners LLC, Research Division - MD, Biotechnology [23]

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So it's good to hear that you noted success with comparability studies and other engineering feats in Pompe. I was wondering, at what stage do you expect to get some clarity that the FDA signs off on the comparability and other aspects over the manufacturing review? Does that have to wait until after the -- an actual filing occurs? Or did they ever consider that in the context or in parallel of what they evaluate clinically?

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John F. Crowley, Amicus Therapeutics, Inc. - Chairman and CEO [24]

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Great. Thanks, Joe. So again, we've completed that comparability between the 250-liter scale, which is the scale, we've used to treat all patients to-date; and the 1,000-liter scale, which we expect to be our scale for any further studies; and our commercial scale as well. So we've completed that work, and it'll be part of our ongoing discussions with FDA. I would expect to have an update on that as well. The CMC work that's so very important in the first half of 2018 to be able to share the regulators' perspective. Yes, we're really pleased with the data that we've seen to-date on comparability. And again, that's both in vitro, lots of analytical data as well as a very robust in vivo study in the Pompe knockout animals, looking at enzyme uptake into key muscles and glycogen reduction. And again, very pleased with the comparability data that we have in hand.

Yes, and just to add some more color, as we actually began at risk about a year ago, the transition from the 250-liter to the 1,000-liter scale. We did that when we saw the very first biochemical data that was released in the fourth quarter of last year, together with a lot of preclinical work that Hung and his team had done, and obviously, we're very pleased that we made those investments and very pleased with the ongoing very strong collaboration with our partners at WuXi Biologics.

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Operator [25]

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And that does conclude our Q&A session for today. I'd like to turn the call back over to Mr. John Crowley for any further remarks.

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John F. Crowley, Amicus Therapeutics, Inc. - Chairman and CEO [26]

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Great. Thanks, operator. And thanks, everybody, for listening. It was certainly a very successful third quarter. We're off to a great start in the fourth quarter of this year, much more work to do and hopefully, laying the foundation for an even stronger 2018. Thanks, everybody. Have a good day.

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Operator [27]

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Ladies and gentlemen, thank you for participating in today's conference. This does conclude today's program, and you may all disconnect. Everyone, have a great day.