Amicus Therapeutics Inc (FOLD) 2018 Q1 法說會逐字稿

Good day, ladies and gentleman, and welcome to the Amicus Therapeutics First Quarter 2018 Results Conference Call and Webcast. (Operator Instructions) As a reminder, today's conference is being recorded. I would now like to turn the call over to Ms. Sara Pellegrino, Vice President, Investor Relations and Corporate Communications. Ma'am, you may begin.

Good morning. Thank you, for joining our conference call to discuss Amicus Therapeutics First 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 are also available in here 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 statements, 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 or 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 first 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 March 31, 2018, to be filed tomorrow with the Securities and Exchange Commission. At this time, it is my pleasure to turn the call over to John F. Crowley, Chairman and Chief Executive Officer of Amicus.

Great. Thank you, Sara, and good morning, everybody, and welcome to our call.

I'll begin on Slide 3. I am pleased to report that the first quarter of 2018 reflects successful execution across our 5 key strategic priorities that we outlined at the beginning of the year, all of which we are well on track to achieve. Our first priority is to double revenue for our oral Fabry precision medicine Galafold in 2018 to a range of $75 million to $85 million. We are now highly confident that we can achieve the upper end of this guidance range. This does not include the contribution of any revenue from Japan or the United States. Brad will talk more about the launch details and demographics in a moment, but I could not be more pleased with the progress we're making to provide access to this important oral precision medicine to more people in both current and new geographies around the world.

Our second priority is to secure approvals of Galafold or migalastat in Japan and in the United States. As you know, our J-NDA was already approved in Japan in March ahead of schedule. And our Amicus team in Japan is prepared to launch this medicine in the second quarter.

In the United States, an NDA is currently under priority review with the U.S. FDA, and we look forward to our PDUFA date in mid-August. In parallel, our U.S. launch preparations are well underway, subject of course to FDA approval of migalastat, and Brad will provide more details on the outstanding team that we're putting in place for the U.S. launch in a few moments.

Our third priority is to further advance our novel Pompe treatment paradigm, which is a combination of our own proprietary enzyme replacement therapy, combined with a pharmacological chaperone, a treatment regimen that we now designate as AT-GAA. This program continues to advance at a rapid pace. We're excited by the progress made across our clinical development program here, and in manufacturing, as we continue 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 regulatory discussion 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 as well as to our active shareholders.

For our fourth priority, we are committed to developing a pipeline of first and/or 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.

We continue to focus on our internal science and technology programs, as well as external opportunities within the rare metabolic disorders. We are actively engaged in licensing discussions on a number of gene therapy and gene editing technologies and programs that we believe would be an excellent strategic fit in the Amicus portfolio and that they further leverage our global capabilities.

And fifth, and final strategic priority that we are focused on now with $600 million of cash on hand. We now have the strongest balance sheet in our company history to fund our key value driver in Fabry and Pompe, and to invest in the expansion of our rare metabolic disease pipeline and technologies.

So with that introduction, let me go ahead and hand the call over now to Brad Campbell, our President and COO to highlight the Galafold launch, and upcoming plans to expand access to the important precision medicine Galafold for even more people living with Fabry disease with amenable mutations. Brad, please.

Thanks, John. Good morning, everyone.

I'll begin my remarks on Slide 5 with a snapshot of the Galafold launch as of March 31. And let me just reiterate, we are very pleased with how the year has started out with hundreds of patients around the world now taking Galafold as their treatment for Fabry disease and on our way to hundreds more this year.

First quarter revenue was $16.7 million, which is a year-over-year increase of approximately 300% from first quarter '17, and this was slightly ahead of our internal forecast for this quarter. And as John mentioned, we're now highly confident in our ability to achieve the high end of our guidance range of $75 million to $85 million based on the current and anticipated increase in patient and physician adoption, continued penetration in our existing markets, expansion into new markets, successful negotiation of drug pricing and market access, and what we're seeing as continued very high rates of compliance and adherence to this oral precision medicine.

The remaining metrics on this slide support the strength of the launch as well as future growth opportunities, and I'll just touch on 2 of them here. Number one, as John mentioned, we have secured an additional approval in Japan, bringing our total to 7 regulatory approvals around the world as well as pricing and reimbursement now in 18 countries.

And our EU label now includes 348 amenable mutations, which is an expansion from the original 269 amenable mutations at the time of approval and reflects several updates to incorporate new amenable mutations into the label.

Turning now to Slide 6, you can really see here our expanding global footprint, which continues to be a key success factor for us, in addition to the 7 approvals highlighted on the previous slide, we have 2 pending, of course, in the United States but as well as Taiwan, with more approvals and more launches anticipated in the future.

And on the next Slide 7, we highlight what we think are the key success factors that we expect to drive future growth as we expand the launch. I won't review these in detail as we've touched on most of them already, but we do believe these lay the very strong foundation to support our upcoming launch in Japan and potential approval and launch in the United States, which are so important, given the opportunity to impact such a large portion of global Fabry population. We estimate nearly 40% of diagnosed patients are in those 2 markets.

And finally, on Slide 8, let me just drill down a little bit more into our launch planning in those 2 key markets. In both United States and Japan, we continue to have a strong presence at key congresses where we're working closely with key opinion leaders on disease awareness and appropriate medical education. We've expanded our speciality distribution system to support both countries, and in the U.S., we're building what we believe will be a world-class patient services system. Specifically in the U.S., the leadership team is now in place, and we're very pleased to announce that [Mike Keavany] is our new Senior Vice President of the U.S. business. He has over 30 years in the industry with the majority of time focused in rare diseases, including senior roles with great companies like [Kastle, Sobi, CSL Behring] and others. He has demonstrated success in building and leading teams, has numerous rare disease drug launches, and he has a track record of driving performance with high integrity. And most importantly, he brings that strong patient focus, which is so key to helping us drive our mission to bring new medicines to patients living with rare and orphan diseases.

We've also now hired a majority of the commercial team under Mike to support the planned U.S. launch. And again, they all bring great experiences from leading rare disease in biotechnology companies. And I can tell you all of them are very eager with the opportunity to potentially introduce Galafold to the U.S. market where more than 3,000 patients are currently diagnosed and roughly half of whom are untreated today.

Turning now to Japan, following the March approval of our J-NDA, we're now in the final stages of pricing and reimbursement process and continue to anticipate launching this quarter. We've built an equally qualified team in Japan. Again, we're so enthusiastic and ready to introduce Galafold to this important country where more than 850 people are diagnosed with Fabry disease, and where we continue to believe that a number of market dynamics including the lack of any home infusions as well as high proportion of treated patients among others will position an oral precision medicine like Galafold for great success.

So in summary, we've made great progress so far this year, and we're very much looking forward to continued success across our goal of providing Galafold to as many patients as quickly as possible around the world. With that, let me turn the call back to John, who can provide an update on Pompe regulatory and clinical activities.

Great. Thank you, Brad. Let me add now as Brad indicated, go ahead and highlight the significant progress with our important Pompe program. Focusing on what we've achieved since the beginning of the year across manufacturing, clinical and regulatory activities.

Let me begin first by reiterating why we're so excited about this program. At the WORLD meeting in February, we reported additional positive 9- and 12-month results from the extension study for the 19 patients who are enrolled across the 3 cohorts of this program. Those include 10 patients in Cohort 1, who are ambulatory and who switched from the approved ERT standard of care.

Cohort 2 included 4 patients who are wheelchair-bound and on full-time ventilator support, who also switched from the ERT standard of care.

And Cohort 3 included 5 ambulatory patients who had never received any enzyme replacement therapy. These patients are all adults living with Pompe disease. The data demonstrated overwhelmingly positive effects in nearly all of these patients including on multiple measures of muscle strength and on all key biomarkers of disease. We have not seen in published literature responses of this magnitude and with such consistency of response. Moreover, our data show these clinically meaningful approaches have been persistent, with many patients actually seeing further strength improvements over time.

The Pompe experts, many of whom are investigators in the study across our 16 global sites are uniformly positive on these data. And importantly, as of today, all 19 patients continue to receive AT-GAA in the extension study. In fact, the very first patients enrolled in this study just crossed the 2-year mark on treatment in April, and we look forward to reviewing and releasing further data from this extension study for all 19 patients at a major scientific meeting this coming fall. Our responsibility now is to advance this program so that AT-GAA is available to as many Pompe patients as quickly as possible. To do so, there are 10 key activities for this program listed on Slide 10 and that we are focused on in 2018. And we have made great progress thus far on all of these key activities.

On today's call, let me turn first to regulatory activities on Slide 11. And I'll provide an update from our series of ongoing regulatory interactions, with both the European Medicines Agency, EMA, in Europe, as well as the U.S. FDA. Since the fourth quarter of last year, we have been engaged in discussions surrounding a registration-directed study or pivotal study for full approval as well as manufacturing activities and clinical data set to help us to find the best and fastest pathway forward for AT-GAA.

Building on these initial interactions as well as the cascade of data from our ongoing Phase II extension studies, these regulatory discussions have provided valuable initial feedback. The next generation -- interactions, rather, will include formal in-person meetings that have been scheduled with both agencies.

As mentioned in our press release issued this morning, we are pursuing formal scientific advice with the European Medicines Agency. The scientific advice meeting with the EMA is scheduled in the second quarter, this quarter of 2018, and we expect to provide an update on the results from that meeting by the end of this second quarter.

In the U.S., ongoing interactions on this program will include a Type C meeting scheduled to occur in third quarter 2018, and we expect to provide an FDA update in 3Q after we see written meetings -- minutes from the FDA.

The goal of these formal meetings and series of interactions is twofold. First, we seek to gain alignment with the agencies on the design of a pivotal study for full approval as well as other supplemental studies in Pompe patients. We have already begun a non-interventional study of ambulatory Pompe adults on currently approved ERT. We expect these patients to roll into the pivotal study. We seek regulatory alignment with FDA and EMA on the overall design for this pivotal study, which we expect will be a study involving patients switching from ERT as well as gaining alignment on the primary endpoint, key secondary endpoint, study duration and the statistical analysis plan.

As this may ultimately be the pathway to approval, we have proposed a pivotal study design that we believe has a very high likelihood of success in a relatively short period of time and with a reasonable number of patients. We will provide more detail on the pivotal study design once there is regulatory alignment. With regulatory agreement, we expect to begin the pivotal study in the second half of this year.

Now we also have smaller supportive studies that we plan to commence, including studies in pediatric populations as well as studying further ERT-naïve patients. As Brad will note in a few moments, we have had great success at the larger 1,000-liter bioreactor scale, and will have significant quantities of drug product available for further studies very shortly.

The second goal of the interactions with both the EMA and FDA is grounded in the notion, we believe, that the current data set is very compelling and that it demonstrates preliminary safety and functional data that seem to address an unmet medical need in the market in the Pompe community today. Pompe patients desperately need newer and better treatment options as soon as possible. Despite an available therapy, many patients with Pompe continue to decline and ultimately die from their disease. As such, we have an obligation to discuss with regulators whether we may pursue a pathway that includes a conditional approval in Europe and/or an accelerated approval in the United States.

We have been encouraged by many of the world's leading experts in Pompe disease to consider these faster preliminary paths to patients, while simultaneously engaging in robust clinical studies to continue to build the most compelling data sets possible. Several of these Pompe experts have been engaged with us in the regulatory process to date and are also expected to participate in the regulatory meetings ahead. We've also been encouraged by the level of support in the patient community, and we have certainly welcomed their continued input into the designs of our further studies and the sharing of their perspectives on the extent of the unmet need with the regulators.

While we hope that we are successful in defining a conditional or accelerated approval pathway soon based on currently available data, the regulators may ultimately conclude that our current data, while compelling, is not sufficient to justify additional or accelerated consideration at this time. For example, we may need to provide additional data from our ongoing studies as well as new data from the addition of further patients into the Phase II extension study to support conditional or accelerated pathways.

In any path to approval, these additional data, as well as the data being collected on the natural history of Pompe patients on currently-approved ERT will be important and will be part of any regulatory submissions. Importantly, we do not view the outcome of these upcoming meetings as binary events. We continue to believe that the evolving regulatory path here will include a series of further iterative discussions with regulators as the program advances and as additional data are collected. Our commitment remains the same as it always has been. Since we initiated the development of our Pompe cell line, and that's to deliver this promising new treatment, again that we designate AT-GAA, to as many people living with Pompe disease as quickly as we can. And through these regulatory interactions we will endeavor to do just that, and we also hope to build a great body of data ahead.

Turning now to Slide 12, I will briefly review some of the key clinical activities currently underway. First, our ongoing Phase I/II clinical study, again including 19 current Pompe patients treated for up to 12 months with enrollment of up to 10 additional patients, now underway. These are ambulatory, ERT-switch patients designated as Cohort 4 of this Phase I/II study. They are similar to the patients in Cohort 1. Doing this will allow us to double the number of patients for whom we have data in the ambulatory ERT-experience population.

We had previously indicated that we would enroll 4 to 6 additional patients in this Cohort 4, but we are announcing this morning that we are expanding that now to a total of up to 10 patients based on continued manufacturing success at the 250 liter bioreactor scale and the availability of additional GMP material. Data from those new Cohort 4 patients who are currently enrolling are expected in the first half of 2019, likely at a major scientific meeting. The data will include muscle strength assessments, pulmonary assessments and biomarker evaluations on all 10 patients. Importantly these patients will begin the study at the 20 milligram per kilogram dose of our ERT plus high-dose chaperone. This is consistent with what we believe to be the optimal dosing. So that's one key part of our activities. Secondly, we are advancing 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 AT-GAA results that we've seen thus far in our Phase I/II clinical study. We have collected significant data from key centers of excellence in Pompe disease worldwide to date, and we expect to share that data 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 outcomes in patients currently treated with standard of care ERT. We intend this observation study to serve as a potential run in for a registration study or pivotal study again expected to start in the second half of 2018. And also later this year we plan to present the 18-month data from the Phase I/II clinical study at a science meeting, likely in the fall, where we hope to see continued consistency and durability of effect.

So as I mentioned, we've had great success in the manufacturing here of our novel ERT. I cannot overstate enough the importance of having successfully scaled our manufacturing from the 250-liter scale to the 1,000-liter scale, which is now also our commercial scale. To be producing drug product now at this larger scale and to be doing so while maintaining the critically important carbohydrate characteristics of this ERT is a major technical achievement. By doing this, it has greatly -- it greatly derisks the program, it advances our time line, and it gives us flexibility to supply AT-GAA to many more Pompe patients in clinical studies and ultimately as a commercial product.

I will note I'm tremendously proud of our Amicus technical operations team, and our quality team, our tech ops team led by Dr. Enrique Diloné here at Amicus as well as our partners at WuXi Biologics.

Let me go ahead now and turn it over to Brad to highlight some of these key manufacturing accomplishments that are so critical to the continued success of this program. Brad, go ahead please.

Great. Thanks, John. We really have come a long way in successfully scaling up from our 5-liter bench scale to a 1,000-liter GMP runs in just 4 years, as highlighted on Slide 13. The team has been laser focused all along the way of maintaining these key quality attributes of ATB200. And as previously announced, analytical and in vivo comparability studies have been completed between the 250-liter and 1,000-liter engineering batches. And we've reached agreement with the FDA that material from those engineering batches is comparable.

We've also reached agreement with the agency on the comparability testing strategy for the GMP material, and we have now successfully completed multiple GMP runs at a 1,000-liter scale, with drug product released for packaging and labeling. All of this progress is significantly de-risking towards our next major milestones from a manufacturing perspective, including the release of the 1,000-liter GMP material for initiation of the registration-directed study later this year as well as securing final regulatory agreement on the comparability between 1,000-liter GMP and 250-liter GMP material. But as critical as the regulatory process is in the immediate term, we've also been very mindful of putting in place a long-term manufacturing strategy to serve the needs of the Pompe community going forward.

On Slide 14, I overview a few of those activities. First since our acquisition of Callidus at the end of 2013, we've worked side-by-side with WuXi Biologics to develop the optimal Pompe cell line and ultimately manufacturing process that we have today. Now that we've successfully scaled up to the 1,000-liter GMP scale at our current site, we've also secured additional capacity of WuXi in China, as well as a second source of supply to their plant, new state-of-the-art facility in Ireland, which they just announced at the end of April. And for the long term, we believe that it's a strategic imperative to have capacity and capabilities to manufacture our own biologics products in the United States. By building a dedicated facility to complement our own supply from WuXi, we can ensure we'll have capacity and capability at multiple sites to always meet the supply needs of the Pompe community, which, as you know, is particularly important in this space.

So again, important progress in the manufacturing front with much more to come. And finally, let me just touch on Slide 16 here, where we overview again our pipeline strategy. We continue to believe that we can leverage our great science, clinical and regulatory and now increasingly commercial capabilities.

We're committed to developing new therapies for rare and metabolic diseases, including potentially bringing in novel technologies in gene therapy and gene editing with the goal of having a product in the clinic sometime in 2019.

With that, let me conclude, and I'll turn it over to Chip to review our first quarter '18 financial results. Chip?

Thanks, Bradley. Good morning, everyone.

Our financial overview begins on Slide 18 with our income statement for the 3 months ending March 31, 2018.

For the first quarter of 2018, we recorded Galafold revenue of $16.7 million, continuing a very strong quarter-on-quarter growth trajectory. Cost of goods sold includes manufacturing cost as well as royalties associated with sales of our product. Cost of goods sold as a percentage of net sales was 15.7% for the 3 months ending March 31, 2018 as compared to 18.6% for the year-ago period.

We continue to make significant investments in R&D as we continue on our vision of building one of the leading global rare disease biotech companies. During the first quarter of 2018, we significantly increased our investment in R&D, recording $40.8 million in R&D expense, as compared to $30.9 million for the prior-year period. The increase in R&D expenses was due to a $6.2 million increase in manufacturing and clinical development costs related to AT-GAA, and a $3.5 million increase in personnel costs. Moving down the income statement, total selling, general and administrative expenses for the first quarter of '18 was $27.3 million as compared to $19.1 million for the prior-year period. The increase represents the expanded geographic scope of the ongoing Galafold commercial launch, including launch preparations for Japan and the United States.

Net loss for the first quarter of 2018 was $49.9 million or $0.28 per share compared to a net loss of $55 million or $0.39 per share for the prior-year period.

As of April 30, 2018, we had approximately 188.5 million shares outstanding, which includes the shares issued in conjunction with our February 2018 follow-on equity financing.

Moving to Slide 19, a few comments about our current cash position and financial guidance. Cash, cash equivalents and marketable securities totaled $605.2 million on March 31, 2018 compared to $358.6 million at December 31, 2017.

As mentioned, our balance sheet was strengthened in February with the successful completion of a follow-on public offering, which totaled $294.6 million in net proceeds. Total net cash spent for the first quarter of 2018 was $48 million, which is tracking well within our 2018 full year cash spend guidance of between $230 million to $260 million. With our current cash position and the continued ramp we see with the Galafold launch, we have sufficient capital to fund ongoing Fabry and Pompe program operations into at least 2021. As we've noted in the past, potential future business development collaborations, pipeline expansion and investment in biologics manufacturing capabilities could impact our future capital requirements.

That summarizes our key financials for the first quarter of 2018. Additional details can be found in our quarterly 10-Q report, which will be filed tomorrow. I'm happy to address any questions during the Q&A, but for now, we'll turn back to John.

Great. Thanks, Chip. So this has been a great start for the year. You reference Slide 20. I like to again just reiterate the significant progress we've made against those 5-key strategic priorities we outlined in January for 2018. We are continuing to build a leading global biotech company focused on rare, metabolic diseases, and we continue to advance our 5-year vision to create 5,000 or more patients with Amicus medicine. Throughout 2018 and along the way to this 2023 goal, we believe we are well positioned to create value for patients and shareholders alike. And with that, operator, that will conclude our opening comments, and we're happy to take any questions.

(Operator Instructions) And our first question comes from the line of Anupam Rama from JP Morgan.

Maybe just a quick one on the Pompe regulatory update you guys gave. You guys have previously talked about harmony across potentially a global strategy, maybe a clarification point. Should we still be thinking about like a single, global registration strategy or potentially 2 different registration type of strategies in the U.S. and EU? And maybe a quick second question, in what time frame do you guys expect to make a decision on the Fabry co-formulation and how tight is this to business development? What are some of the push and pull levers you're thinking about for moving forward with the Fabry co-formulation or looking externally for some options, or can we even think about a dual strategy here?

First, with respect to Pompe, it's our absolute goal and our belief that we can align the United States and Europe again on both of -- certainly on the first point of the registration directed or pivotal study. We really hope to only have one pivotal study going forward. Secondly, in terms of our looking for the potential for an accelerated or a conditional approval pathway, in either geography, of course, it would be optimal to have alignment there, but ultimately that will be determined by the regulatory feedback we receive in the next couple of weeks, in the month or 2 ahead from both agencies. You can see it, a divergence. Historically you have seen the agencies diverge there, but again, our goal is to get alignment as close as we can.

Second -- secondly, with respect to Fabry, co-form again, that's our own proprietary novel enzyme replacement therapy we've developed for the other half or more of Fabry patients living with non-amenable mutations and it is co-formulated with our chaperone, again to enhance activity and stability of the protein. In preclinical studies, we saw significantly improved absorption into key spell of disease and significantly more reduction of the key substrate. As we've indicated before, we've completed all preclinical proof-of-concept that has moved to WuXi for initial manufacturing. We are -- we're waiting to pull the trigger on advanced manufacturing of that program in a timeline for moving it into the clinic. I think it is dependent in large measure on the work that we're doing on the business development front. I continue to believe, Anupam, that we have a commitment to obsolete our own technologies, and to develop therapies (inaudible) for the diseases that we work in. We're very excited about a range of the technologies and programs that we're seeing. Fabry and Pompe are key strategic priorities for us, for gene therapy, and or gene editing technologies. So I would expect in the second half of this year that we'll make a decision based on where we are in those business development activities. Whether we indeed move forward the ERT into the clinic or whether we leapfrog it with a gene therapy approach. I think we'll have an important update on that in the second half of the year.

And our next question comes from the line of Tazeen Ahmad from Bank of America.

So John, just a follow-up to the presentation that you had made back in February at the WORLD conference, and had spoken with analysts there. At that point, you'd said that you would be adding patients to the study. Can you give us an update on how many patients have been added thus far? And then secondly, in terms of your timeline for update, you've gotten the initial data back in October, and I think the original plan was to have some clarity by the end of the first half of the year. This FDA meeting has been scheduled for 3Q. Is that timing based on FDA or on your desire to potentially collect additional data before having that formal meeting?

Sure. I'll comment on the first question with respect to patients. The enrollment of Cohort 4, Tazeen, is ongoing. We expect that to be complete over the next couple of months here, it's very important for us. We had a lot of requests from a number of centers around the world to participate significant patient demand. We wanted to make sure that was appropriately balanced and not heavily weighted to any 1 or 2 or 3 sites. So because of that, we're making sure patients are put in that appropriately. But we're really -- there is more than enough patient demand for access to AT-GAA, so that we'll be able to provide that data in the first half of 2019, again hopefully at a major science meeting.

We're not going to give -- we don't traditionally give specific target objectives for small cohorts like these. We'll let you know when it's completed its enrollment, but again, I feel in a really good place, and I think it will add substantially to the body of data that we have. And with respect to the timing, again, we've engaged in what really are a series of collaborative discussions with regulators. They provided valuable feedback that's informed a lot of the work that we're doing. Certainly, the briefing documents and the design of our pivotal study as well as our approach in discussing conditional or accelerated approval pathways. The timing of the meeting, again, that will move forward into a Type C meeting, that was on the FDA's time line. And again, it's very early in 3Q, but I think important. And again, I want to reiterate, I do see this -- while those meetings will be highly constructive and important, I do see this in the months and quarters ahead, [a continued] collaborative engagement, a series of iterative discussions with the regulators as we move this medicine to more and patients and ultimately to approval.

Okay. And then maybe one on the path for U.S. approval for Fabry. Can you comment at all on discussions that have been occurring with the agency and anything new that's popped up? Just want to make sure that there is no remaining questions about GI.

Yes. We're not going to comment on ongoing discussions with the FDA. I'll just remind everybody that in February, they accepted the NDA filing. They gave it priority review. That PDUFA date is August 13. And we are deep in the review process with FDA, and again, we continue to remain committed to get that to patients as soon as hopefully it is approved.

And our next question comes from the line of Joseph Schwartz from Leerink Partners.

So you mentioned that the evolving regulatory path for Pompe in the U.S. will include a series of further iterative discussions based on your interactions to date. Are you finding that a filing for accelerated approval is a tougher sell to the FDA for any reasons in particular? And since these iterative discussions are expected to occur as additional data are collected, do you expect further iterative discussions to occur after the FDA's answer in the third quarter?

Again, during the third quarter, we expect following the Type C meeting to get feedback from the FDA. Again number one, on the design of the pivotal study; and number two, on what a pathway toward accelerated approval would look like if that is an option. I wouldn't say -- you began your question by characterizing that it's a tougher road with FDA. I wouldn't read anything into that. I wouldn't give any color as to where the FDA's mindset is. We've not had those face-to-face meetings and discussions with FDA, those will take place shortly. Again, we're focused on everything that we can control in terms of making the drug, getting it to as many patients, expanding the studies, and I think again we have an obligation based on the strength of the data, the expert view, the patient needs and perspective to continue that dialogue with the regulators both in the U.S. and in Europe to try to determine the best and fastest pathway.

Okay. Great. And then just on the business development commentary. I was wondering if you could expand a little bit more because I heard you suggest that maybe gene therapy could be better suited to address the challenges in Fabry. And that gene therapy might potentially leapfrog your ERT combo and Fabry. Are there -- is this because of anything you're seeing in the clinic or are the challenges in the Fabry different? I'm just -- than what you've been able to achieve in Pompe, for example. I'm just trying to understand how you see the world at this juncture.

Yes. I think for us, Joe, it really is part of a longer-term commitment to patients than to our portfolio. Even when we started Amicus, we built in part of our mission statement that we have a duty to obsolete our own technology. We, back in February, made a commitment in our Healing Beyond Disease initiative to all patients with rare diseases that for any Amicus product that's approved, we would devote a significant portion of cash flow to not just investment broadly in R&D but in that exact disease until there's a cure. I think like a lot of folks were very excited about what we're seeing, increasing in the gene therapy and gene editing fields. And this may be a long-term investment strategy for us for all of our diseases, but I think particularly for Fabry and for Pompe. We have -- we're looking at or actively engaged in negotiations and discussions on a range of platform technologies and products in the gene therapy space. What's clear to us is that once we begin with a disease target, we then want to scour the world [in diligence] what we think could be the best technical approach. Very clear there is no one platform technology that's best for all rare metabolic diseases, not even for all lysosomal storage disorders. So I'm really excited about some of what we're seeing. Again, we're very actively involved in a range of discussions, activities, and I would -- I'm very hopeful that we'll be able to build a program in Amicus leveraging those global capabilities that we now have in the second half of the year. So I think stay tuned for where we're at there.

And our next question comes from the line of Mike Ulz from Robert.

Just wanted to ask a follow-up on the Pompe program, and maybe in terms of your current thinking related to any potential pivotal trial design. And I know you don't want to make too many comments, but maybe you can just give us a rough idea of the patient numbers you're considering or potentially arranged there. And then in terms of duration, in your prepared remarks, you mentioned potential for shorter duration there. Does that mean we should be thinking more 6-month primary endpoint or more 12 months, any color you can provide there?

Yes. Mike, again that's part of the ongoing discussions. I think we've had a really smart plan that we've developed for that study that would ultimately be for full approval. And in patient numbers, I don't want to give you specifics, but you're in the dozens of patients. We hope [we're within] a hundred patients for instance, but part of that is still to be determined. With respect to duration, we're looking at a number of different ways to cut that, but I think I'll reserve that until we have got alignment from both regulatory agencies.

And our next question comes from the line of Ritu Baral from Cowen.

John, can you maybe give us a little more clarity about what you'll go into your Q3 meeting with FDA with? Specifically, at that point, even if it's not publicly released, what sort of follow up on the Phase I/II patients you'll have? What preliminary data from Cohort 4 you might have? And will POM-002 be largely complete and ready to show the agency?

Ritu, I'd rather not, given that we're just in the midst of these discussions [to give] any indications to what the nature of those are. What I can assure you is that any data that we've collected to date on patients treated with our products, in whatever Cohort they may be, together with any data that we may have been building on this natural history study, would all be available for regulators in the months ahead, and certainly, I assure you that we, together with the experts, are focused on building the most compelling body of data that we have today, and frankly in the months and quarters ahead. So lots of activities ongoing, and with that we'll move forward. I hope that's helpful.

Okay. Have you started treating patients in Cohort 4 yet?

We have. We've begun enrolling patients.

Okay. And then just a quick follow-up. How are you looking at the changes within the FDA divisions, specifically the G.I. division, and the structure, and the head, et cetera both for migalastat and for AT-GAA?

Yes, I'm not going to comment on specific FDA leadership changes except to state that these have been and continue to be ongoing collaborative discussions. There has been strong continuity with the people at the agency who have been reviewing both our Fabry and Pompe programs. So again, I think we've got great confidence [in the] people around the table at FDA.

And you don't expect the people around the table to change in the near term?

We don't expect it, no.

Would you expect a new interim head to be in place by the time of your Q3 meeting for AT-GAA?

I don't expect so. Jay, I don't know if you want to comment on anything that the FDA would have publicly released about any changes at the division chief level?

We don't have any information about that or insight into that, but I would reiterate what you said, John, that all the discussions that we've had to date and continue to have with the same group of individuals within the agency and division have been very collaborative, and we have a very good working relationship with them, so I'm sure that will continue.

Okay. Last question, I want to switch gears to migalastat. On the prelaunch effort, assuming approval, can you talk a little bit about maybe genotyping programs, or payer discussions that you've had, and any changes and thoughts on how you think of potential pricing for migalastat in the U.S.?

Brad, I'll let you go ahead and field those, please.

Yes. Thanks, Ritu. Good questions. So you may remember that going back quite a way is actually -- the research that we've seen, and as we've been out in the market, we continue to believe that genotyping in the U.S. is upwards of 90% or more. So it's a well-characterized population, which is great. In terms of sort of launch strategy, et cetera. I think we laid out in the slides here what we think are the foundation of our success in our launch strategy outside the U.S. and we'll deploy that here as well. What we have noted is that there is a significant number of diagnosed untreated patients here in the United States. And so we think that continues to be an opportunity as well as a significant number of potential switch patients. So again, I think the demographics here lay themselves out well for a successful launch based on our other markets. And from a pricing and reimbursement perspective, you know, I think we'll continue to reiterate our strategy, which is we believe our medicines must be fairly priced and broadly accessible. And we should expect at this point that we would stay with our parity pricing strategy, which we think reflects the value of the product, and afford some savings back in the system in terms of avoiding a huge [associated] cost. Of course that will depend on the label, et cetera, et cetera. So more to say in the months to come, but for now, I think we are well set up with our strategy to be successful here in the U.S.

Brad, are you still -- are you also expecting sort of a switch patient-led adoption in the U.S. like we saw in Europe?

Yes. I think that is safe to assume at this point. Of course, we'll know more as we get into the launch. Perhaps the one big difference, of course, in the United States is there -- outside of the U.S., there is really not an ability to engage with patients directly. It really has to go through physicians, whereas in the United States, there's a difference there. So it's a slightly different dynamic, but by and large, I think you should expect the same launch strategy as we've seen.

Our next question comes from the line of Salveen Richter from Goldman Sachs.

Salveen, we can't hear you. Okay.

And I'm showing no further questions at this time. I would now like to turn the call back to Mr. John Crowley for closing remarks.

Great. Thank you, operator. And I'll just remind everybody at the JP Morgan Conference at the beginning of the year, we set out our goals for the year, including the 5 key strategic priorities. And just remind everybody we'll make great progress along all 5 of those. And look forward to continued interactions and welcome any follow-up phone calls or questions. Thanks for the continued support. Have a great day.

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