Amicus Therapeutics Inc

NASDAQ:FOLD

Earnings Call Transcript 2018-Q2

[Operator]

Good day, ladies and gentlemen, and welcome to the Amicus Therapeutics Second Quarter 2018 Results Conference Call and Webcast. As a reminder, today's conference call is being recorded

I would now like to turn the call over to Mr. Andrew Faughnan, Associate Director, Investor Relations. Sir, you may begin.

[Andrew Faughnan, Associate Director, Investor Relations]

Thank you, Styler [ph]. Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics’ second quarter 2018 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, Chief Science Officer, and Jeff Castelli, Chief Portfolio Officer are also available here to participate in the Q&A session.

On this call, as referenced on Slide two, we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as well as our plans and prospects. Our forward-looking statements should not be regarded as representation by us that any of our plans will be achieved.

Any or all of the forward-looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward-looking 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 statements on Slide 2 of our second quarter 2018 results slide deck, as well as the Forward-looking Statements and Risk Factors section of our Annual Report on 10-K for the year ended December 31, 2017 as well as our quarterly report on Form 10-Q for the quarter ended June 30, 2018 to be filed 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. John?

[John Crowley, Chairman and Chief Executive Officer]

Great. Thanks Andrew and welcome everyone to our call this morning where I’ll begin the discussion on slide three. I am very pleased to report that the second quarter of 2018 reflects successful execution across our key strategic priorities that we have outlined at the beginning of the year, all of which we are on track to achieve.

To highlight, the first priority we laid out earlier this year was to double revenue for our oral Fabry precision medicine Galafold this year. I am pleased to say that we are set to exceed this objective in 2018 and we continue to provide, as we continue to provide access to this oral precision medicine to more people than both current in new geographies around the world.

Based on the success of Galafold in the first half, we are raising our global revenue guidance for the year to a range of $80 million to $90 million from the previous $75 million to $85 million. Now Brad will talk more about the launch details and demographics in a moment.

Our second priority outlined earlier this year was to secure approvals of Galafold or migalastat in Japan and in the United States. Our J-NDA as you know was approved in Japan in March ahead of schedule and I am excited to say initial patients in Japan are now being treated with commercial Galafold, the first oral precision medicine for Fabry patients with amenable mutations.

In the United States, an NDA is currently under priority review with the U.S. FDA and we very much look forward to our upcoming PDUFA date scheduled for August 13th now less than a week away.

Our U.S. launch preparations are now complete and our U.S. leadership team is now fully launch ready. We are very excited about the days ahead for Galafold here in the United States.

Our third priority is to further advance our novel Pompe treatment paradigm, a combination of our own proprietary enzyme replacement therapy combined with the pharmacological chaperone, a treatment regimen that we now refer to as AT-GAA. This program continues to advance at a rapid pace. We are pleased with the progress made across our clinical development program here and did manufacturing as we continued towards the goal of making AT-GAA available to the Pompe community globally as quickly as possible while continuing to build a compelling body of clinical data. I’ll provide more detail on these key clinical and manufacturing activities as well as where we are in the ongoing regulatory discussions later during this call. We continue to believe that this important Pompe program will be a future source of significant value for people living with Pompe disease and for our shareholders.

Fourth, we are committed to developing a pipeline of first and to our best-in-class medicine that has the potential to deliver significant benefits for people living with rare metabolic diseases and to shift the treatment paradigms for each disease in which we work. Specifically we are extraordinarily focused on assembling a portfolio of technologies, programs and partnerships in the gene therapy space. Our goal beginning in the second half of this year now is a bold one, to build one of the most robust gene therapy pipelines in the field of rare metabolic disorders.

We strongly believe that Amicus has the opportunity to improve upon and accelerate current gene therapy efforts in a number of ways which I’ll highlight later in the call.

We are actively engaged in licensing and product acquisition discussions on a number of gene therapy technologies and programs that we believe would be an excellent strategic fit in the Amicus portfolio and would further leverage our scientific and global development expertise.

And finally, with over $550 million of cash on hand, our balance sheet remains in a position of strength. With a broad range of successes so far this year and anticipation of the opportunities, data and potential new programs that are to come in the months ahead, I’m also pleased to announce that we will be hosting our first ever analyst day in New York City. It will be held at the Harvard Club on October 10th and I’ll have more details on this in the weeks ahead, so please stay tuned.

Let me now go ahead and turn the call over to Brad Campbell, our President and Chief Operating Officer to highlight the Galafold launch and the upcoming plans to expand access to important precision medicine for even more people living with Fabry disease who have amenable mutations or amenable variance. Brad, please?

[Bradley Campbell, President and Chief Operating Officer]

Thanks, John good morning. I’ll begin on slide five with a snapshot of the Galafold launch through June 30. We are very pleased with how the first half of 2018 has progressed for Galafold.

Our second quarter revenue was $21.3 million, which is a year-over-year increase of approximately 200% from second quarter 2017, and which was ahead of our internal forecast.

The success internationally continues to be driven by strong adoption in our existing markets in switch patients and now also an increasing numbers of diagnosed untreated patients coming on Galafold as well.

We’ve also continued successfully opening up new markets like Japan, Canada and others, which is driven by the strong value proposition from Galafold as well as our successful pricing and access strategy and the hard work of our global team.

And finally, we continue to see very high rate of compliance and adherence to this oral precision medicine. With that strong momentum in our existing markets, and the anticipated contribution from Japan, the U.S. and other expected new launch countries, we’re now raising our full-year global revenue guidance to $80 million to $90 million.

As a reminder, while this guidance now includes revenues from Japan and the United States, we do expect relatively limited contributions in 2018 from both of these countries, given it will only be a partial launch here for both.

In 2019, we do expect significant contribution from both Japan and the United States. These expectations are consistent with the trends in our launch curves [ph] in Germany, U.K., France and other large countries we reached [ph] several quarters of launch data and experience to inform our outlook.

The remainder of this slide highlights our key commercial metrics including now seven regulatory approvals, the most recent thing Japan of course, as well as pricing reimbursement in 19 countries and pending approvals in Taiwan and the United States.

On slide six, let me just drill a little bit more detail into our progress in Japan. As a reminder, Japan is the second largest Fabry market in the world with more than 850 people diagnosed with Fabry disease, and thanks to the hard work of our teams globally Galafold is now the first new treatment option for Fabry patients in Japan in well over a decade. And again, we are very pleased to say that following the launch in late May, initial patients are now being treated with commercial Galafold.

And I’ll say that one particularly inspiring event was our July launch symposium in Tokyo. It was attended by members of the patient advocacy community, the media and well over a 100 Japanese physicians, including the original inventor of Migalastat. It was really a great event and the turnout confirms the continued excitement and need for an oral precision medicine like Galafold.

With a highly experienced and passionate team there, we continue to believe that the high treatment rate, the lack of home infusions and the excitement for this new treatment option positions us well for success.

Now moving to slide seven, with our U.S PDUFA date just days away, let me update you with where we are in the U.S. As a reminder, the FDA accepted the company’s new drug application for Migalastat under Subpart H priority review with a six month PDUFA date of August 13, 2018.

An important part of any Subpart H agent approval, we do expect to further build the body of evidence through a confirmatory phase 4 study in our ongoing registry. In preparation for the potential launch, I am pleased to say that Amicus is now fully launch ready. The team is hired, trained and in place across the U.S, which includes sales, our internal patient support team and a full team of medical science liaisons.

As we highlighted in our first quarter call, this [ph] Amicus team to support the planned U.S. launch brings great experience, passion from leading rare [ph] disease and other biotech companies and they are all eager to introduce Galafold to the U.S. community where more than 3000 Fabry patients are currently diagnosed roughly half of them are treated to that.

In summary, we’ve made great progress so far this year and now with our global team of patient focused and passionate entrepreneurs in place, we look forward to continued success towards our goal of providing Galafold to as many patients around the world as quickly as possible and we are eagerly anticipating our August 13, PDUFA day.

With that, I’ll turn the call over to Chip to provide an update on our financials. Chip?

[Chip Baird, Chief Financial Officer]

Thanks, Bradley. Good morning everyone. Our financial overview begins on slide nine with our income statement for the three months ending June 30, 2018. For the second quarter of 2018, we recorded Galafold revenue of $21.3 million, continuing a strong trend of quarter-on-quarter growth.

Cost of goods sold, includes manufacturing costs as well as royalties associated with the sales of our product. Cost of goods sold as a percent of net sales was 14.7% for the three months ending June 30, 2018, as compared to 14.8% for the year ago period.

We continue to make significant investments in R&D as we continue on our vision at building one of the leading global rare disease biotech companies. During the second quarter of 2018, we increased our investment in R&D recording 34.7 million in R&D expense as compared to 32 million for the prior year period.

Moving down the income statement, total selling, general and administrative expense for the second quarter of 2018 was 29.2 million as compared to 19.3 million for the prior year period. The increase here represents the expanded geographic scope of the ongoing Galafold commercial launch including launch preparations for Japan and the United States.

Net loss for the second quarter of 2018 was $61.8 million or $0.33 per share, compared to a net loss of $48.1 million or $0.34 per share for the prior year period. And as of June 30, 2018 we have approximately 189.1 million shares outstanding.

Moving on to Slide 10, a few comments about our current cash position and 2018 financial guidance, cash, cash equivalents and marketable securities totaled $553 million on June 30, 2018 compared to 359 million at December 31, 2017.

Our balance sheet was strengthened in February with the successful completion of a follow-on public offering. Total net cash spend for the second quarter of 2018 was 51.

Looking at the remainder of the year, we are raising our full year top [Indiscernible] revenue guidance to $80 million to $90 million to reflect the continued success globally.

In addition, we are updating our full-year cash spend guidance to 220 million to 250 million, a 10 million reduction on both the high and low end of the range due to both an increase in expected revenue as well as expenses that continue to trend favorably to budget.

With our current cash position and continued ramp we see from the Galafold launch, we have sufficient capital to fund ongoing Fabry and Pompe program operations into at least 2021. As we have noted in the past, potential future business development collaborations, pipeline expansion and investment in biologics manufacturing capabilities could impact our future capital requirements.

This summarizes our key financials for the second quarter. Additional details can be found in our quarterly 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.

[John Crowley, Chairman and Chief Executive Officer]

Great, thanks, Chip. And let me now highlight the progress with our Pompe program. Focusing on what we’ve achieved across our manufacturing, clinical and regulatory milestones this year and please reference slide.

As we have highlighted previously, we are very excited about this program, the data presented at the World Meeting in February demonstrated positive effects in nearly all 19 patients in the Phase 1/2 study including on multiple measures of muscle strength and on all key biomarkers of disease. We have not seen responses of this magnitude and published literature and also with such consistency of response. Moreover, our data show that these clinically meaningful improvements have been persistent with many patients seeing further strength improvements overtime.

Importantly, as of today, all 19 patients continue to receive AT-GAA in the extension study. And today, we are pleased to announce that the 18-month data from this study will be presented via a podium presentation at the 23rd International Annual Congress of the World Muscle Society in October of this year.

Our responsibility now is to advance this program so that AT-GAA is available to as many Pompe patients as quickly as possible. On the regulatory front, we have been engaged in iterative discussion with both the European medicines agency, EMA in Europe as well as the U.S. FDA surrounding a pivotal study for full approval as well as our manufacturing activities and existing and future clinical data sets to help us to define the best and fastest path forward for AT-GAA.

Following meetings in the second quarter in Europe, we intend to continue a dialogue on the potential pathway for conditional approval with the EMA authorities in 2019; with further data on both functional measures and on safety and I’ll speak to you again in a moment.

In the United States, ongoing interactions with the FDA include a scheduled type C meeting and we look forward to providing an update later this quarter after written receipt of minutes. As in Europe, we expect this to be an ongoing iterative discussion with regulators on the path toward approval.

We continue to believe that the regulatory path in the U.S. and in Europe will include a series of further discussion as the program advances and as additional data are collected. Our commitment remains the same as it always has, since we initiated the development of our Pompe cell lines.

And again as to deliver this promising new treatment AT-GAA to as many people living with Pompe as quickly as we can, and through these regulatory interactions we will endeavor to do just that.

Let me highlight few of the key items ahead for the important Pompe program. First, as we noted a few months ago, we are adding up to 10 additional ambulatory ERT-switch patients to our ongoing Phase 1/2 clinical study.

These ambulatory ERT-switch patients designated as Cohort 4 are similar to the patients in Cohort 1. Data from these Cohort 4 patients are expected in the first half of 2019. The data will include six-minute walk test, muscle strength assessment, pulmonary assessment and biomarker evaluations on all patients.

Second, we were advancing a retrospective study known as POM-002 on the natural history of Pompe disease and up to 100 ERT treated Pompe patients to help provide context for the AT-GAA results that we have seen in our Phase 1/2 study.

We expect to complete this natural history setting in the second half of 2018. Third, we are also running a prospective observational study which we refer to as POM-003 or the STRIDE study to assess safety and functional outcome in patients currently treated with Standard of Care ERT.

We intend this observational study to serve as a potential running for a pivotal study expected to start in the second half of 2018. And finally again as mentioned earlier we plan to present the 18 months data from the Phase 1/2 clinical study at the World Muscle Society this October.

Before I hand the call over for a moment to Brad to provide some updates on Pompe manufacturing, I just want – I cannot overstate enough [ph] the importance of having now successfully scaled manufacturing from the 250 liter bioreactor scale to the 1000 liter commercial scale and now to have it released and ready for a pivotal clinical study. I am tremendously proud of our Amicus technical operations team led by Dr. Enrique Diloné, a quality teams here at Amicus as well as our partners at WuXi Biologics.

With that, let me go ahead and turn the call over for a moment to Brad to highlight some of these key Pompe manufacturing accomplishments.

[Bradley Campbell, President and Chief Operating Officer]

Thanks John. We have indeed come a long way in just a few years time and we're proud to say that this progress has now let to number one, the release for the first time of 1000 liter GMP material for initiation of a pivotal study. And number two, securing feedback from Germany regulatory authorities indicating agreement with our strategy to demonstrate comparability of the ATB200 1000 liter GMP and 250 GMP material which is in line with FDA's feedback earlier this year, as with John, I applaud the efforts of our team.

Now, we're heavily focused on ongoing manufacturing to supply ATB200 for our clinical studies as well as ongoing process developed work for our PDQ run. We anticipate an update we anticipate an update on the progress here with these manufacturing activities in the first half of 2019.

With that, we've also been very mindful putting in place a long term manufacturing strategy to serve the needs of the Pompe communities. We continue to believe that is a strategic imperative to have capacity and capabilities to manufacture our own biologic products here in the United States.

With that overview, I'll now turn the call back to John.

[John Crowley, Chairman and Chief Executive Officer]

Thanks Brad. Before we close I'd like to just take a moment to highlight our pipeline strategy on slide 14. With all of the great progress in Gene therapy and gene editing broadly, we continue to believe that making smart investments across the range of platforms and technologies we'll advance our mission at Amicus that delivering next generation therapies for patients living with rare metabolic disorders.

As I mentioned at the beginning of the call, we believe that Amicus has unique client, typical expertise and capabilities that can build upon and accelerate existing gene therapy programs.

Specifically, under Dr. Hung Do's dealership we have build significant expertise in protein engineering, secretion and trafficking which enables us to optimize the type of proteins that has produced and how that protein is traffic to disease relevant tissue.

Amicus also has proven expertise in the clinical development of novel treatment for rare metabolic disorders which can be leveraged to accelerate novel gene therapy of purchase.

We continue to diligence to range of gene therapy and gene editing approaches and believe that these technologies will be an important part of the future of the Amicus pipeline.

In closing on slide 15, I'd like to once again reiterate our significant progress we had made in 2018, as we continue to build a leading global biotechnology company focused on rare metabolic disorders and as we continue to advance our five-year vision to treat 5000 patients with an Amicus medicine by the year 2023.

Throughout 2018 and along the way this 2023 goal, we are well-positioned to create significant value per patients and for shareholders alike. And again let me just reiterate our enthusiasm about the upcoming PDUFA date for Galafold and now less than a week away, and again to emphasize that our U.S. launch preparations are now complete. Our US leadership team is fully lunch ready and it is a very very exciting days ahead for Galafold and hopefully for Fabry patients here in the United States.

With that operator, we're happy to take any questions.

[Operator]

[Operator Instructions] Our first question comes from Anupam Rama with JPMorgan. Your line is now open.

[Anupam Rama, JPMorgan]

Hey, guys. Congrats on the progress and thanks for thanking the questions. I know you guys have said that your launch ready here in the U.S. and just wondering what your market research suggest on the ramp curve here in the U.S. relative to say, Japan and Europe, and if you're planning key opinion leader, physicians symposiums, here's as well. Thanks so much.

[John Crowley, Chairman and Chief Executive Officer]

Thanks, Anupam, let's Brad go ahead and fill that.

[Bradley Campbell, President and Chief Operating Officer]

Sure. And Anupam, I just miss the last piece of your comment there, but as related to the U.S. I'll just give a high-level overview – of course get more detail in anticipation of PDUFA date coming in about week's time. The U.S., we think we'll be obviously a large contributor to the overall commercial market. It's about 27% of the global market today. In terms of a launch curve, we'll just state that, we continue to think that it's an exciting opportunity here.

Remember there are about 3000 patients diagnosed today, about half of them are treated today and so the dynamics we've seen playout in our other key markets I think would be similar here, but again we'll provide more detail on that in the coming days. And I'm sorry I miss the second piece of your call. I think it was about symposia et cetera here in the U.S. Of course we have major Scientific at Congresses in the fall and in the first part of the year, next year and we would expect to have a credit there as we have historically.

[Anupam Rama, JPMorgan]

Thanks so much for taking my questions.

[John Crowley, Chairman and Chief Executive Officer]

Yes. Thank you, Anupam. Again we look forward to speaking to you and everybody here as we approach the PDUFA date shortly. Thank you.

[Operator]

Our next question comes from Ritu Baral with Cowen. Your line is now open.

[Ritu Baral, Cowen]

Good morning, guys. Thanks for taking the question. Brad, again you mentioned that your launch ready in the U.S. Can you tell us a little more about the size and organization of the U.S. salesforce including risk managers, distribution hub, what's the plan is? Also can you give us an update on how you guys see the potential of competitive dynamic in the U.S. [Indiscernible]?

[John Crowley, Chairman and Chief Executive Officer]

Yes. Ritu, thanks for the question. Again, we'll keep it a little high-level here in anticipation of more detail for the anticipated PDUFA date. But just to remind you the few things we've shared before. We will use an efficient commercial team here as we have in other markets. I think what we've said historically is it in the dozen of folks to make up the full team. You would notice on the call here today that we indicated that we do have an internal patient support teams, so rather than an external hub, its an internal hub, we believe that the first call should always be to Amicus employee, so that gives you a little bit of color there.

I think fundamentally it relates to the competition. We continue to believe the disease as a competition here. I think we've been successful in sharing Galafold with other key markets and see a great uptick there. And I think that philosophy is focused on the value that Galafold can bring has been successful for us. But again we'll have more detail on that with the anticipated PDUFA date coming up here.

[Ritu Baral, Cowen]

What is the overall status of patient awareness of their nutrition pipe in the U.S.? Is that something you've been working on? Is that something that's maybe an important part of [Indiscernible]?

[John Crowley, Chairman and Chief Executive Officer]

Yes. I think awareness of pharmacokinetics and the work we've really done over the last decade to educate community on the importance of that, and its relatively new concept in Fabry, but of course really important for us. Our expectation is that there is high awareness in physician and patient communities as well. Again we'll have more details to talk about that in the coming days.

[Ritu Baral, Cowen]

Got it. And just last question on, John, as we think about pipeline expansions and new technologies, how are we discuss the potential for in more detail of gene therapy that different thing that you had considered. How do you think about the manufacturing investment necessary for gene therapies given your current cash balance and then the FDA guidance that really says, launch of the commercial value just soon as possible starting with these programs?

[John Crowley, Chairman and Chief Executive Officer]

Yes. We think that FDA guidance actually plays well into what's in to Amicus's experience in manufacturing in proteins specifically. So with Pompe of course we made the decision very early on to scale our Pompe cell lines from the clinical scale 250 liters to the commercial scale, in fact every pivotal trial patient will be treated with the commercial scale which fits very well into how we think about mitigating manufacturing risk going forward.

And I think that philosophy will certainly carry through to the gene therapy work that we have expect to enter here shortly for all of the programs and technologies that we are actively diligent thing with two manufacturing as a very key part of those diligence effort. We would expect that any program that would be at or near the clinic or in the clinic shortly would have qualified manufacturing both from a quality standpoint and a capacity standpoint.

In the meantime for each of the programs that we expect to in license or to begin manufacturing not just in the near time, but over the long term is an important part of what we're considering. So I think as these deals come to fruition in the second half of the year we'll be able to articulate for each one of them a very clear manufacturing plan and path forward.

[Ritu Baral, Cowen]

But is it fair to say that new strategy remains preference while doing manufacturing in-house rather than any contract sales?

[John Crowley, Chairman and Chief Executive Officer]

I think that’s to be determined, I think it will vary on a program by program basis.

[Analyst]

Got it. And thanks for taking the questions.?

[Bradley Campbell, President and Chief Operating Officer]

Of course, we too, thank you there.

[Operator]

[Operator Instructions] Our next question comes from Tazeen Ahmad with BOA. Your lines are now open.

[Tazeen Ahmad, BOA]

Hi. Good morning, guys. Thanks for taking my questions. Just wanted to follow-up on just to set expectations correctly, John, what do you expect to be able to provide in terms of the feedback from your FDA meeting this quarter? Is it your expectation that you have more definitive feedback from the agency on a potential path for accelerate approval from Pompe? Or are you viewing this next meeting as a catch-up with the agency and that you would just – you like kind of expect to have multiple meetings with them going forward?

[John Crowley, Chairman and Chief Executive Officer]

Well, we certainly do expect multiple meetings going forward as data continues to evolve, but we also expect this type C meeting to be definitive in the questions and the areas that we're focused on. Again, two areas of focus where the type C meeting; one is the design with the pivotal study going forward to gain alignment on that. And secondly, to talk to the U.S. FDA about a path for fastest approval including the potential for accelerated approval in the United States. And we very much expect to have clarity on that in the third quarter.

[Tazeen Ahmad, BOA]

Okay. So, it will be much more definitive than we can keep talking about and let's see what happens?

[John Crowley, Chairman and Chief Executive Officer]

Absolutely, yes.

[Tazeen Ahmad, BOA]

Okay, great. And then on the 18 months data that you're planning on presenting later this year, can you give us an idea of how many patients that would include?

[John Crowley, Chairman and Chief Executive Officer]

I'll turn it to Jay Barth, our Chief Medical Officer for the World Muscle Society, Jay, for the data on 18 months, but we expect in on how many patients?

[Jay Barth, Chief Medical Officer]

As you recall there are about 10 patients data from the Cohort 1 and that's really what we're focused on and that's what we expect to present on primarily. There's also in that ERT ambulatory patients as well we'll represent additional data on other cohort of patients, the naïve patients as well as ERT experienced non-ambulatory patients as we've done before.

[Tazeen Ahmad, BOA]

And on vis-à-vis, go ahead, Jay.

[Jay Barth, Chief Medical Officer]

It will be the full range of data just for a longer period of time similar to what we saw at the World Meeting earlier this year just for most patients out to 18 months.

[Tazeen Ahmad, BOA]

Okay. And so, what would be your expectation for good data? Is it just simply continuation of trends seen before?

[Jay Barth, Chief Medical Officer]

We haven't seen the data but our hope would be that there was significant gains and function in muscle strength that we observed at the 12 month period – nine or 12 months period would be at least maintain or in some patients perhaps further extended out to 18 month. That would be pretty profound to be able to see that. That's our hope biologically we know how this medicine works. Now we have to see how it works in the longer term. So we'll see where we are.

[Tazeen Ahmad, BOA]

Okay. Thanks John, and then maybe one for Brad trying to making feel like you being left out. Can you give us an idea of how you're thinking about range of pricing for the U.S. for Galafold?

[Bradley Campbell, President and Chief Operating Officer]

Yes. We'll have more to say hopefully here in a few days, Tazeen, but I think in general you'd expected to be similar to our pricing strategy globally -- replacement therapy which reflects the value of the product but also save the system, the infusion associated cost.

[Tazeen Ahmad, BOA]

So, wouldn't necessarily be a meaningful premium to Europe to the price that you're getting in Europe?

[Bradley Campbell, President and Chief Operating Officer]

Again, just from a pricing range perspective, globally the prices range is roughly between $200,000, $300,000 per patient per year. Europe does tend to be more in the middle or the bottom of that range. U.S. tends to be higher in that range just from an average pricing perspective and patient weight perspective, but again we'll give a little bit more clarity on that in the coming day.

[Tazeen Ahmad, BOA]

Okay. Thank you.

[Bradley Campbell, President and Chief Operating Officer]

Thank you, Tazeen.

[Operator]

Our next question comes from Joseph Schwartz with Leerink Partners. Your line is now open.

[Joseph Schwartz, Leerink Partners]

Thanks. Good morning. So, where do you see the best opportunity to obtain or source gene therapy technology? And is your gene therapy business development focused on new patient areas? Or do you think it could cannibalize your approved products or pipeline candidates?

[John Crowley, Chairman and Chief Executive Officer]

Yes. Thanks Joe. Maybe just – let me just go back and talk a little bit about this strategy that we've undertaken. So about a year ago we begin to look seriously at the gene therapy space. We added senior members of our team to help us in that effort together with some consultants and experts around the world.

We began by looking at about a 100 different rare metabolic disorders, so we began with the diseases agnostic to technology. From there we focus in on about 15 of those rare metabolic disorders where we thought there were significant unmet medical need and where we thought broadly that a gene therapy approach could be technical feasible and particularly where we thought we could in some cases bring again that Amicus expertise particular Hung [ph] our Science Organization expertise in protein engineering, trafficking, secretion.

We then set about to go discover the landscape, but what was available in the gene therapy space. We've look at academic centers, we look at private companies and public companies. We've gone under CDA with numerous organizations over the last nine months and we're now to the point that we've made recommendations on which of these programs which technologies we think would be the best fit. We are not disclosing any of those 15 or so diseases with the exception of Pompe and Fabry. We've said that we are actively looking at gene therapies for Pompe and Fabry. We think that fits in the Amicus mission statement where we wrote in 2005 that we have a duty to obsolete our own technologies and also our pledge to cure where we promised patients, where we have a marketed or approved product we will invest a portion of the proceed and revenue from that product into continued research and development on that specific disease until there is a cure.

So, we are looking at a range of technologies. Joe, we're looking at various AAVs. We're looking at [Indiscernible]. We're looking at areas that where we can combine technologies with what we have at Amicus and at our expertise. So I'd stay tuned for what's ahead over the second half of the year. I think it will be an exciting time for Amicus, for patients and for shareholders.

[Joseph Schwartz, Leerink Partners]

Okay, thanks. That’s helpful. And how is your commercial presence set up in Japan and how extensively are Japanese Fabry patient’s genotypes relative to those in Europe and U.S. and is there any different propensity, sense of Japanese patients with Fabry to switch as compared to those in the U.S. and Europe?

[John Crowley, Chairman and Chief Executive Officer]

Thanks Joe. Let me just address a few of those. Again, from a team set of perspective, we have KK [ph] established in Japan, so we have a full Japanese entity for their regulatory requirements. Again, very efficiently sized team in the dozen, low dozens there as well, which we think is sufficient to successfully execute there as we’ve done in other key markets, Germany, U.K. etcetera.

In terms of the Japanese market dynamics, we did provide a little bit of color on this but let me just remind you of a fear to get to your point there, so a couple of things that are unique about Japan, a relatively high treatment rate, so we’ve said that we estimate over 700 of those 850 diagnosed patients around treatment today. And if you look at the update on all their markets I think we’ve had very good success in switch patients.

A couple of other dynamics to keep in mind there is that there is a very high awareness I think of Galafold in Japan, in part because as you might have heard the anecdote in my comments in the call, Galafold was actually invented in Japan and came out of University there in Japan and then through Mount Sinai so there’s some kind of inventor I think pride there, which is great. And then you also have a no home infusion, so patients have to go into hospitals or in fusion clinics to get around in the replacement therapy.

So all those things, we think could provide a very strong foundation for commercial success and we are very much looking forward to that in the coming months.

[Joseph Schwartz, Leerink Partners]

Thanks again.

[Operator]

Our next question comes from Mike Ulz with Baird. Your line is now open.

[Michael Ulz]

Hey guys, thanks for taking the question. Just with respect to the phase 3 design from the Pompe program, you recently received clarity from the European regulators and I’m just curious if you still get the sense that the FDA will largely be aligned there or could we see potential differences and some of the requirements between the agencies?

[John Crowley, Chairman and Chief Executive Officer]

I think Mike we need to engage in those discussions and not speculate on what may or may not be acceptable to FDA, so let us have that conversation and once we’ve got the written minutes back from FDA we’ll be able to provide more color on that.

[Michael Ulz]

Got it. And then maybe just another quick question here. Just with respect to the Pompe natural history data which I think is the 02 study, you mentioned completing this study in the second half of 2018, does that mean we could potentially see some of the data from that study, or should we be more thinking 1Q or first half of next year? Thanks.

[John Crowley, Chairman and Chief Executive Officer]

We’ll complete that natural and important natural history study, still to be determined when we’ll share it publicly, whether that’s towards the end of this year or whether that’s in the first half of 2019, potentially at a science congress. You know multiple purposes for that data, including for regulatory uses as well.

So we have to be mindful of that. So let us finish this study and I think as we get closer to the end of 2018, we’ll be able to provide an update on when you guys in the external world will be able to see that important data.

[Michael Ulz]

Great, thanks.

[John Crowley, Chairman and Chief Executive Officer]

Thank you, Mike.

[Operator]

At this time, I’m showing no further questions.

[John Crowley, Chairman and Chief Executive Officer]

Great, thank you operator. Thank you everybody for listening. It was an excellent quarter and a great start first half of the year now. And I expect that we’ll be speaking in the days ahead. Thank you.

[Operator]

Ladies and gentlemen, thank you for your participation in today’s conference. This does conclude the program. You may now disconnect. Everyone have a great day.