Sara Pellegrino - Senior Director, IR John Crowley - Chairman and Chief Executive Officer Bradley Campbell - President and Chief Operating Officer Chip Baird - Chief Financial Officer Jay Barth - Chief Medical Officer.

Anupam Rama - JPMorgan Tazeen Ahmad - Bank of America Ritu Baral - Cowen Mike Ulz - Robert W. Baird Joseph Schwartz - Leerink Partners.

Good day, ladies and gentlemen, and welcome to the Amicus Therapeutics 2017 Financial Results Conference Call and Webcast. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions] As a reminder, this conference is being recorded.

I would like to introduce your host for today's conference, Ms. Sara Pellegrino, Senior Director of Investor Relations. Ma'am, you may begin..

Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics full-year 2017 Financial Results and recent 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 Jeff Castelli, Chief Portfolio 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 to 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 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 full-year 2017 results slide deck, as well as the Forward-looking Statements and Risk Factors section of our Annual Report on Form 10-K for the year ended December 31, 2017 to be filed March 01, with the Securities and Exchange Commission.

At this time, it is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics..

Great. Thank you, Sara, and good morning, everybody. It’s a pleasure to host today's call. It is being held on Rare Disease Day.

It is the 11th anniversary of Rare Disease Day and as many of you know, it's a day that has traditionally taken place on the last day of February each year to raise awareness of the 7000 unique rare diseases that collectively impact an estimated 350 million people throughout the world.

Today more than 94 countries are participating in a variety of activities and initiatives in honor of Rare Disease Day.

At Amicus we are reminded every day of why we come to work and every day to fulfill our vision to deliver significant benefits to people living with rare diseases through a broader corporate responsibility to the rare disease communities that extend beyond our medicines.

So for today's call to begin the discussion, I'll turn your attention to Slide 3. 2017 was an extraordinary year for Amicus and we achieved four key strategic priorities across our two leading programs in Fabry and Pompe.

First, we exceeded our Galafold launch target with $36.9 million in global revenue and more than 310 Fabry patients with amenable mutations treated at year-end. Second, we completed our Japanese and U.S. regulatory submissions for Galafold. Three, we established definitive proof-of-concept for our novel treatment paradigm for Pompe disease.

And fourth, we ended the year with a strong balance sheet with $359 million of cash on hand.

Throughout 2017, we continued to build a leading global biotechnology company focused on improving the lives of patients, people living with rare metabolic disorders and we laid the groundwork for significant long-term value creation for patients and for our shareholders.

Turning to Slide 4, you can see in 2018 we are focused on five key strategic priorities. First, we expect doubling full-year 2018 global Galafold revenue in the range of $75 million to $85 million and with more than 360 patients on treatment today, we are highly confident in achieving this guidance.

Next, we are committed to broadening global access to Galafold through additional regulatory approvals. In Japan we continue to track toward a decision in the first half of the year, and just two weeks ago we announced that the U.S. FDA has accepted our NDA for Galafold under priority review with a six-month PDUFA target date of August 13, 2018.

Third, we are laser focused on achieving key clinical, regulatory, and manufacturing milestones to advance our novel Pompe treatment paradigm which we refer to as ATB200/AT2221 towards regulatory submissions and approvals. I'll highlight that later more on today's call.

We believe that this Pompe program has the potential to create significant value and our approach was further validated by the most recent data set presented at the World Symposium in San Diego in early February.

Fourth, we are committed to advancing and expanding our preclinical pipeline so that we can move at least one new medicine for a rare metabolic disorder into the clinic by the end of 2019.

As for our pipeline, we are focused on innovative next-generation therapies for the rare metabolic disorders that have the potential to obsolete current technologies and deliver significant benefits for patients.

And finally, we have maintained and significantly strengthened our balance sheet with an additional $300 million from our recent follow-on offering and so we are very well capitalized to invest in our current core programs in Fabry and Pompe and to expand our rare metabolic disease pipeline.

So with that introduction, I will turn the call over now to Brad Campbell, our President and COO to highlight our progress in delivering our precision medicine Galafold to more people in more geographies worldwide.

Brad?.

Great, thanks John. Good morning everybody. I'll begin on Slide 6 with a recap of our full-year 2017 Galafold revenue and 2018 revenue guidance.

2017 as you know was our first full year of launch and we focused on growth in patient and physician adoption as well securing final pricing and reimbursement in new markets through the country-by-country processes in Europe.

By year-end there were more than 310 patients on treatment with Galafold for their Fabry disease which exceeded our target of 300 and full-year revenue totaled $36.9 million.

Based on our initial success in 2017, we believe we can double our Galafold revenue in 2018 to $75 million to $85 million in sales which will set us on track for what we believe is a $500 million plus global peak sales opportunity for Galafold for people with Fabry who have amenable mutations.

Turning now to Slide 7, I'll provide an update on the great progress we've made so far in 2018 all as of today, February 28. Now that we've introduced revenue guidance for 2018, we'll focus on our somewhat streamlined set of regulatory and commercial metrics for you to track throughout the year.

As John mentioned in the introduction, in today's press release we reported that more than 360 patients are being treated with Galafold today. This reflects the continued strong momentum coming into this year with more than 50 new patients in the first two months of the year.

The mix continues to include a high proportion of switch patients, a growing number of naïve patients who were previously diagnosed but never treated, as well as a handful of newly diagnosed patients as well. We also see strong adoption across the spectrum of the disease including males and females as well as classic and late onset mutations.

And we continue to have a high rate of compliance with the oral every other day dosing regimen. Overall we've seen adoption among these patient segments which is very much in line in with our launch strategy and we expect this to continue throughout the year as we open up new markets and continue to penetrate into our existing markets.

As a reminder, looking ahead to next quarter, we do expect to move away from the patient numbers metric and instead highlight quarterly revenue as a measure of progress towards our revenue goal for the year.

From a pricing and reimbursement perspective, as an oral precision medicine with a strong clinical data set we believe Galafold offers a value proposition that continues to resonate with payers and we've now secured final pricing and reimbursement in 18 countries including all five of the top-5 EU markets and generally well ahead of industry averages.

We've already launched in new countries in 2018, and throughout the year we will continue to pursue pricing and reimbursement or new patient sales opportunities in additional markets in Europe, Asia-Pacific, and Latin and South America.

One of the highlights on this slide is that we do continue to broaden the EU label to incorporate new amenable mutations and we now have a total of 348 amenable mutations listed in the lookup table which is up from 269 of the original mutations listed at the time of approval.

Again, this is a great example of our precision medicine and proprietary approach to identifying amenable mutations as well as unique feature in our label to regularly submit new mutations for inclusion in the lookup table. And finally, from a regulatory perspective, if you turn to Slide 8, we have secured approvals in six target markets to date.

We are on track with our guidance for the timing of potential approvals in the U.S. and Japan, and we're well underway in launch preparations in those key markets. In summary, our focus in 2018 is to drive continued adoption and further global expansion and approvals.

We believe there is still significant growth in our existing markets as well as substantial opportunity this year to deliver Galafold to people with amenable mutations in new geographies including Canada, the U.S.

and Japan among others, all building the foundation for what we believe is a half a billion dollar peak sales opportunity for treating Fabry patients with amenable mutations.

But we also remain committed to the rest of the Fabry patient population and our strategy there continues to be advancing next-generation therapies for Fabry patients with non-amenable mutations our novel proprietary Fabry enzyme replacement therapy co-formulated with migalastat as well as other innovative technologies that we continue to evaluate.

With that, I'll hand the call back to John to provide an update on our Pompe program..

Thanks Brad. I am pleased now to highlight some substantial progress across our clinical manufacturing and regulatory activities for our novel Pompe treatment paradigm ATB200/AT2221.

Just a few weeks ago in San Diego during the World Symposium we had the opportunity to share our latest data set from the ongoing Phase 1/2 clinical study with the world's leading experts in Pompe disease.

We are extremely pleased with the physician feedback on the data in terms of the magnitude, durability and consistency of results on safety, on key functional measures and key biomarkers across all three patient cohorts, data now for up to 12 months of treatment in those patients.

Turning now to Slide 10, I'd like to highlight the manufacturing activities underway to supply the global Pompe community and that it is so critical for our future success with a few key important updates. First I'll comment on the agreement we reached with FDA on our comparability studies.

The FDA reviewed all of our analytical and in vivo comparability studies and agreed that number one, 250L GMP materials and the 1000L engineering batches are comparable.

Two, FDA agreed that our analytical methods are the right way to test for comparability and three the FDA also agreed to our strategy to establish comparability between the 250L scale and the 1000L GMP materials using our analytical testing methods.

From there and that meeting at the end of the fourth quarter in 2017 we have set out to repeat the same comparability experiments and processes we used with the engineering batches, only this time under GMP conditions.

I'm very happy that we can report for the first time on the call today that we have successfully completed the first campaign of ATB200 drug substance at the 1000L GMP scale and we're in the process of completing additional GMP campaigns to establish the supply of this important material for our registration directed study as well as for commercial purposes.

Turning to Slide 11 I'll focus on all of the activities that are within our control in 2018 to lay the foundation for success and to ensure the best and fastest path toward approvals. We have several clinical activities now underway to assemble a robust and high-quality clinical data package.

First, our ongoing Phase 1/2 clinical study includes 19 current Pompe patients treated for up to 12 months with planned enrollment of 4 to 6 additional patients and secondly we are conducting a retrospective study what we refer to as our POM-002 Study on the natural history of Pompe disease in up to 100 ERT treated Pompe patients to help provide context for the ATB200 results that we've had in our Phase 1/2 clinical studies.

Our third, we are also running a prospective observational study which we refer to is as POM-003 or the STRIDE study to assess safety and functional outcomes in patients currently treated with standard-of-care ERT. This observational study may also serve as a potential run-in for registration study expected to start in the second half of 2018.

As I've mentioned, we've made significant progress already with our manufacturing and our next steps are to continue to 1000L GMP manufacturing campaigns and work towards final FDA agreement on comparability between the 250L clinical material and the 1000L GMP batches.

We also plan to expand our capacity for additional manufacturing at the existing Wuxi facility in China as well as a second source supplier and in the next few years we believe the potential for our own Amicus Biologics facility likely based here in the United States.

And on Slide 12 we've summarized the same potential regulatory pathways that we outlined at the beginning of 2018. We continue to engage in collaborative discussions with FDA and EMA and we expect to provide an update in the second quarter of this year.

Before I hand the call over to Chip, I'd like to reiterate that we could not be more pleased with the advancement of this important Pompe program. We remain fully committed to providing access to this treatment paradigm to as many people as possible living with Pompe as soon as we possibly can.

With that, let me go ahead and turn the call over to Chip to discuss our 2017 financial results. Chip, go ahead please..

Thanks John and good morning everyone. Our financial overview begins on slide 14 with our income statement.

For full year 2017 we recorded Galafold revenue of $36.9 million for our first full year sales in Europe, we recorded sales in more than a dozen countries although we only have six countries where we had reimbursement in place at the beginning of 2017 which is why we expect to see continued uptake in the launch trajectory.

Cost of goods sold included manufacturing costs as well as royalties associated with sales of our products, cost of goods sold as a percentage of net sales was 16.9% for the year-end 2017 as compared to 16.8% for the year-end 2016.

During 2017 we significantly increased our investment in R&D recording $149.3 million of R&D expense for the full year 2017 as compared to $104.8 million for 2016.

The increase in R&D expense was due to the $29.3 million increase in manufacturing and clinical development cost related to the ATB200 and $5.9 million increase in the costs of the SD-101 program and Epidermolysis Bullosa. The increase in SD-101 costs included the accelerated recognition of costs then to wind down of the ESSENCE study.

Moving down the income statement, total selling, general and administrative expense for 2017 was $88.6 million as compared to $71.2 million for the full-year 2016. The increase here represents the expanded geographic scope of the ongoing Galafold commercial launch.

I'll note there were several non-cash items that impacted the income statement in 2017 stemming from the results of the Phase 3 ESSENCE study and our decision to discontinue further investment and development of SD-101 and again all the evidence I am are about to discusses are non-cash charges.

We reported $254.7 million gain and the fair value continued consideration stemming from the fact that we will not owe any additional milestones to the former [indiscernible] shareholders we recorded $465 million loss on the impairment of in-process R&D related to SD-101 and finally you will see that we recorded $164.7 million of income tax benefit related to reduction deferred tax liability with this tied to SD-101.

Net loss for the year was $284 million or $1.85 per share, compared to a net loss of $200 million or $1.49 per share for the full-year 2016. Net loss for 2017 excluding the impact of those non-cash charges related to the ESSENCE study was $237 million or $1.55 per share.

As of February 23, 2018 we had approximately 186.9 million shares outstanding which included the shares immediately issued in conjunction with our February 2018 follow-on equity financing.

Moving on to Slide 15, a few comments about our current cash position and 2018 financial guidance, cash, cash equivalent and marketable securities totaled $358.6 million at the end of 2017 compared to $330 million at the end of 2016.

Our balance sheet was further strengthened in February with the successful completion of a $300 million follow-on public offering. Total net cash spend for 2017 of $216.5 million was in line with our 2017 cash spend guidance of $200 million to $225 million.

For 2018 we continue to expect full year net cash spend of between $230 million and $260 million. With our year on cash, the proceeds of our recent equity offering and expected Galafold revenues, we have sufficient capital today to fund ongoing Fabry and Pompe program operations into at least 2021.

Potential future business development collaborations [indiscernible] and investment in Biologics manufacturing capabilities could impact our future capital volumes [ph]. This summarizes our key financials for 2017. Additional details can be found in our annual report 10-K which will be filed on March 1.

I'm happy to address any questions during the Q&A but for now we'll turn it back to John..

Thank you, Chip. 2017 was certainly a tremendous year for Amicus. We are on track with respect to our five key strategic priorities as we begin 2018.

As I highlighted in the beginning of the call and again here on Slide 16 we look to create even more value for shareholders and importantly to make a meaningful difference in the lives of patients living with rare diseases.

In closing on Slide 17, I'll just reiterate that we are sharply focused this year as part of our long-term vision to maximize our impact on patients treated with an Amicus medicine with the goal of more than 5000 patients and more than $1 billion in revenue by 2023.

Finally, turning your attention to Slide 19 if you'll indulge me, I'd like to highlight that today is Rare Disease Day and with that we have the launch of a very important new global Amicus employee driven initiative that we referred to with Healing Beyond Disease.

Healing Beyond Disease is our unique promise to further serve the needs of the rare disease community in extraordinary ways. It is at the heart of who we are and the company we are building. Our core purpose at Amicus is to make great medicines. Healing Beyond Disease is a long term commitment though to do even more than that.

It is core to our company beliefs, it is based on the notion that we are in this business to positively change the lives of people living with rare diseases in many ways.

It includes our philanthropic endeavors, investment in next generation treatments within our current therapeutic areas to focus and our emerging plans to make our medicines accessible to patients in every part of the world possible to heal beyond disease.

On slide 20 I will outline the five unique pillars of our Healing Beyond Disease Program which reflect our obligations beyond the therapies that we provide. These pillars leverage Amicus' very internal resources, existing philanthropy, drug development expertise and our passion to bring a different level of support to the rare disease community.

The five pillars of Healing Beyond Disease at Amicus are number one, time. We will evolve volunteerism companywide to further the commitment to the rare disease patient community with information and incentives and programs for employees which will include three days of paid leave for volunteer efforts annually. Secondly is talent.

We have developed a significant amount of expertise in the rare diseases over the years.

We would like to leverage this expertise within the company to provide mentorship to individuals impacted by rare disease and complementary consulting to emerging rare disease organizations and startup businesses to establish their mission of developing therapies for people living with rare diseases.

Third treasure, we’ll advance philanthropy for broader support of rare diseases by providing the opportunity for voluntary financial support directly from company employees in diseases in areas not related to any Amicus products or research that are within the rare and metabolic disorders.

Four, is the Amicus pledged to a cure and this is very unique. We will designate a portion from any Amicus marketed product drug sales to reinvest in that specific disease until that disease has a cure. This may be a many years or many decades obligation to the patient community and it's something that we feel very passionately about.

And finally building rare bridges, the fifth pillar of Healing Beyond Disease. We are committed to provide access in the near term and the long term across the globe to our medicines for people living with rare diseases in the developed and the developing world and we'll have more in the months ahead to say about some of these initiatives globally.

So with that, great recap of 2017, you've got a perspective now on the success we've had in 2018. Our key strategic priorities and also I think a more in-depth look into the company and the culture that we're building with the vision that we will become one of the world's leading global biotechnology companies, focused on rare metabolic disorders.

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

[Operator Instructions] And our first question comes from the line of Anupam Rama from JPMorgan. Your line is now open..

Hey guys, maybe just two questions here for me, just on the 50 new patients added on therapy, maybe you could give us a little bit on how it breaks down in countries beyond Germany given there’s some of the reimbursement progress in 2017? And then also on the Fabry front, you guys have talked about advancing the co-formulation in 2019, wondering what some of the gating factors there are getting - to get that into the clinic and some of the activities ongoing for that program throughout this year? Thanks so much..

Sure, let me have Brad take the first one the Galafold uptake and I’ll take the second one Anupam..

Excellent. Good morning, Anupam. So good question on distribution of the new patients and in terms of how the patients are distributed through the markets, I think you can safely assume that the EU5 represent the largest portion of patients and then the largest countries within that bucket of countries represent the largest pool of patients still.

But I will say that we are doing a great job of launching and getting Galafold to patients in other markets throughout Europe and increasingly now and in other markets where we've gotten approvals and reimbursement outside of Europe.

So, we feel very comfortable with the fact that the existing markets continue to grow and Germany, U.K., France others continue to be great opportunities there, but we would expect to see and we hope to see great launch progress in new markets as well..

I'll just comment Anupam on the co-form here, couple of things we're working on. First, are all the manufacturing clinical plans, the regulatory necessary to allow that to enter the clinic in 2019.

Secondly, the other gating factor for moving that into patients is our continued diligence, our new technologies particularly in the gene therapy and even now the gene editing space, evaluating you know are there potential therapies that could become part of the Amicus portfolio for patients living with Fabry disease, particularly those patients with non- amenable mutations for whom Galafold is not a suitable treatment, are there newer and potentially even better technologies beyond what we're developing for co-form? And we're completing that diligence right now and that will be an important part of the step forward in providing other options for people living with Fabry..

Great, thanks for taking the question guys..

Thank you..

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

Hi good morning guys. Thanks so much for taking my questions.

Maybe one for Brad first, can you talk to us about what you think the Japanese opportunity will be in terms of number of patients, what have you done so far to identify people that would be - have amenable mutations first of all and early on in your launch be put on therapy? And then secondly, historically, I think pricing in Japan tends to be more generous for orphan drugs even relative to the U.S., are you finding that is still the case?.

Yes, thanks Tazeen. Good questions, so Japan as we've said before is the second largest individual country in terms of Fabry patients and Fabry sales. There are 700 or so enzyme replacement therapy patients today in Japan, so a significant number of treated patients.

It's a little bit unique in that there's a high penetration of treatment in Japan, so there's a slightly lower proportion of diagnosed untreated patients, but we're doing all the things that we've done historically in our other markets in terms of getting ready for an anticipated launch their approval and launch there.

So we've done a great job now of working with physicians around amenability and so far our estimates suggest that it's very similar to our other markets which is somewhere between 35% and 50% of patients have amenable mutations we believe and we're also building the team there.

We’ve done a great job, hired some great leaders there and putting people in the field as well in the medical side and on the sales side. So we believe that we've got a great team and we're ready to go.

And the only other thing that we've highlighted before in terms of perhaps unique attributes of the Japanese market, first is the technology, the chaperone technology for migalastat came out of science from Japan.

So there's a little bit -- perhaps a little bit of a home grown mentality there and also it's unique in the sense that there are no home infusions in Japan, so the extent that the burden of treatment with enzyme replacement therapy versus the oral every other day delivery of migalastat could give us a significant advantage there as well.

So lots of great opportunity and we're very much well underway in terms of launch preparation there, and then remind me your second question Tazeen?.

On pricing..?.

Oh yes, pricing thank you. So from a pricing perspective, yes, you're right.

There are some unique mechanisms in Japan for slightly higher pricing than other markets, but remember we'll likely continue with our parity pricing strategy which means that we provide the therapy at the same price as enzyme replacement therapy, but then forego the infusion associated costs which again is part of the value proposition we believe that we bring with Galafold, and Japanese patients tend to be from an average weight perspective lighter than the average patients in our other markets, and so while it's a premium on a unit basis from a pricing perspective, it still sits well within that $200,000 to $300,000 per patient per year corridor that we've talked about historically..

Okay and in terms of timelines is there an accelerated path in Japan similar to U.S.

fast track?.

Well, what we've said is we are on track for an anticipated approval in the first half of this year and we are reiterating that guidance.

After you receive approval then there's about a two to three month reimbursement process, so as we get closer and obviously get to the approval then of course we'll have a better sense for when we can actually launch, but again we're reiterating that we're on track for the anticipated timing..

Okay and then lastly, your sales guidance for this year does or does not include sales for Japan?.

That isn't not including sales for Japan and the United States, although remember even though those are the two largest individual country markets, Japan and the U.S., having a portion of the year and in a launch year, we would be careful to overestimate the contribution of those markets this year.

As we go forward, if we believe that there's an opportunity to revise guidance we will certainly do that..

Okay, thanks..

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

Good morning guys. Thanks for taking the question. I want to just asked about your 2018 guidance and how we should think about that range in terms of what you're seeing as far as compliance, persistence and pricing for Galafold in Europe and rest of world.

I mean if you pull out the numbers, you barely need any patient growth to get to the top half of guidance, are there things about compliance and persistence we should be keeping in mind?.

I let Brad comment on compliance, persistence, adherence, but again I think we're very confident in reaching the guidance of that $75 million to $85 million Ritu.

As we see the market developing, as we’ve seen new patient starts, as we hopefully have approvals in Japan and the United States, we will certainly take a hard look at whether we need to revisit that, but for now we'll reaffirm the high confidence and the guidance of $75 million to $85 million.

Brad, do you want to comment on what we're seeing with continued strong trends in compliance?.

Sure and remember Ritu to your point in terms of new patients starts, so if you were to essentially take the rate of growth from last year which is increasing at an increasing rate, you'd get to sort of the middle of that range of compliance.

So I think that's a good baseline assumption to start with and of course we think we can potentially drive it higher. So we certainly expect new patients to contribute to that $75 million to $85 million of revenue.

As it relates to compliance and persistence, as I said in my remarks we continue to see a high rate of compliance and a very high rate of persistence as well above the averages that you would expect to see certainly with chronic oral therapies and even perhaps with infused therapies as well.

So we're very pleased with those rates and we think that reflects anecdotally of course the experience that patients and physicians are having with the product..

And then on your first GMP batch, can you talk about the yields that you've seen how is that process gone, does it give you first insight into potential cost of goods on ATB200?.

Yields were very good. I think there is potential still for further improvement as we get into the next series of manufacturing campaigns. It is sufficient for us to begin the registration directed study and again we have other campaigns already underway.

We have capacity now added into our planning with Wuxi in the near term for additional material that will be available for other studies and for commercial purposes.

So I think we'll be in a very good place in terms of both the quality and the quantity of material and it’s too premature to speculate now on what our commercial cost of goods sold will be..

Great and last question, just a little more detail on POM-003 the STRIDE study, did you mention, did I miss what your target enrollment is for that study and when you might start enrollment and also POM-002 the retrospective study when might we see that analysis?.

Yes, let me add Jay Barth is here Ritu, let me ask Jay to comment on both of those, Jay so the observational study 003 and then the historical study that we're doing, the 002..

Hi Ritu.

We are planning at this point for about a 100 patients in the POM-003 study, the prospective observational study for ERT treated patients and regarding the POM-002 your question was?.

Oh, when we might see the data and analysis?.

Thanks. We’re in the process of gathering that data right now. It will be over the next couple of quarters I would say, the next period of months, but we don't have a specific date yet for knowing when we'll have the results..

I think likely that we would share it externally in the second half of this year I think we've said before..

Got it.

And entry criteria for a 003 or for STRIDE, what's the plan for the type of patients that you will…?.

Yes, that's a pretty broad entry criteria. These are a representative sampling of patients who have been on the ERT treatment with the standard-of-care treatment who meet standard criteria for their functional measures, an entry into the study.

And why they trained [ph] we really want to capture a full heterogeneous population that's reflective of LOPD..

Got it. Thanks for taking all the questions..

Thank you..

Thank you..

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

Hey guys, thanks for taking the question.

Just with respect to the Pompe program in your ongoing discussions with regulators, I'm just curious to the extent that you can comment, are you finding that generally the FDA and EMEA are sort of aligned on their thinking there? Or there are notable difference is emerging in terms of what might be required for an accelerated path or conditional approval and then also with respect to sort of Phase 3 trial design?.

Yes, Michael just say that our goal is to have this uniform and purchase we can globally, beyond that I'm not going to comment on the ongoing discussions and the nature of any perspectives, except to say that we'll tell you the regulators have certainly acknowledged the extent of the unmet need in unmet medical need on the Pompe world, that's – questions..

Got it and then just maybe follow up on an earlier question with respect to the POM-002 and POM-003 studies, should we think about those the sort of rate limiting assuming you could potentially get to an accelerated path or is that not necessarily the way to think about it? Thanks..

I think, yes I think POM-002 is very important. You know, remember our Phase 1/2 study did not have any controls. It was a traditional Phase 1/2 open label study.

And while we understand the clinical perspectives of the experts while we understand what's published in the literature, we think having a rigorous control sample could further provide information about the nature of the improvements, the magnitude, the consistency of what we're seeing in patients in that Phase 1/2 study.

So that will be a very important data set for us over the next couple of months. At POM-003 we think in no way is rate limiting.

We see that as related to the long term development of the drug providing further evidence on how people do on this ERT standard of care, but very importantly the purpose of that study is to provide potential for patients for a run-in period into a registration directed study, so if we don't see that at all as rate limiting to any plans or any potential for an earlier approval..

Got it. Great, thanks guys..

Thank you..

[Operator Instructions] And the next question comes from the line of Joseph Schwartz from Leerink Partners. Your line is now open..

Thanks very much. Happy Rare Disease Day guys. Thanks for all your leadership in this area.

Can you provide us with any insight into how you feel about the strength of the analyses that you've been able to undertake and you presented to the FDA from the POM-002 study relative to the patients that you've treated with ATB200?.

Thank you, Joe. Yes, we're not going to comment on ongoing discussions or ongoing analyses and that POM-002 study it’s still underway and collecting information on those approximately 100 patients to build that natural history study.

But we can comment on is what we know from the clinical experience of the experts that have been shared with us and on the published literature and that is that we think the experience of patients in our Phase 1/2 study is remarkably different in terms again of the magnitude of response and the functional measures across cohorts whether patients are ambulatory or non-ambulatory, whether they've received the ERT before or whether they have not.

And so on a range of those measures together with all the biomarkers, both of the muscle damage and glycogen measured enhanced for, so there we think it is an extraordinary data set to-date. And we hope that the POM-002 natural history study will only strengthen and support for that, but we're, that's still ongoing..

Okay, could you help us envision the scenarios that could result from your collaborative discussions with the FDA? Is just one of two outcomes likely either yes you can file on this data or no you need to run an Phase 3 before you can file or do you think there's maybe some middle ground where they could say track these patients longer and then file for instance?.

I think there could be a range of outcomes for sure Joe. Our job is to do everything we can in our control to allow for the best and fastest path for approval for these patients, so we have more work to do on that front and we'll provide that update in the second quarter..

Okay and then just as a quick follow on in a similar vein I guess is the indication that you in the FDA settle on, do you think ultimately just all patients or known Pompe patients or is there any possibility that you could be granted a subset indication to start addressing those who are at the greatest need?.

Still to be determined, our goal is to get the drug approved for all Pompe patients. We now have experience in ERT treatment, naïve ERT experience, ambulatory and non-ambulatory. So we think we've got great experience to date.

We will add to that with the additional patients added to this Phase 1/2 extension study, additional switch patients and we believe in relatively short order we'll be able to build further data in other populations such as the paediatric population.

And so I think more to come on what the extent of any approval would be, but certainly to really to speculate on that..

Okay, thanks for taking my questions. Good luck..

Yes, of course Joe. Thank you..

Thank you. At this time, I'm showing no further questions..

Great, and if there's no further questions, thank you everybody for listening and thank you for the recognition for everybody, all 350 million people in the world living with rare diseases. Thank you. Have a great day..

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