Amicus Therapeutics Inc

NASDAQ:FOLD

Earnings Call Transcript 2018-Q1

[Operator]

Good day, ladies and gentlemen, and welcome to the Amicus Therapeutics First Quarter 2018 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 be given at that time. [Operator Instructions] As a reminder, today's conference is being recorded.

I now like to turn the call over to Ms. Sara Pellegrino, Vice President of Investor Relations and Corporate Communications. Ma'am, you may begin.

[Sara Pellegrino]

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 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 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 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 to 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.

[John Crowley, Chairman and Chief Executive Officer]

Great. Thank you Sara and good morning, everybody and welcome to our call. I will being on Slide 3. I'm pleased to report that the first quarter of 2018 reflects it's excellent execution across our five key strategic priorities that we outlined at the beginning of the year all of which we are well on track to re-queue.

Our first priority is to double revenue for our oral a very 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 demographic in a moment, but I could not be more pleased with to the progress we are 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 approval 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 [Indiscernible] team in Japan is prepared to launch this medicine in the second quarter.

In United States, an NDA currently is under priority to 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 to quarter two FDA approval of Migalastat and as Brad will provide more detail on the outstanding team that were 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 as a combination of our own proprietary enzyme replacement therapy combined with a pharmacological chapter a treatment regimen that we now designated AT-GAA.

This program continues to advance at a rapid pace, we are 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 communities globally as quickly as possible while continuing to build the compelling body of clinical data.

I will 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 Amicus shareholders.

For our fourth priority we are committed to developing a pipeline of first and/or best-in-class medicine that have the potential to deliver significant benefits for people living with rare metabolic diseases and to shift the treatment paradigm for each disease in which we work.

We continue to focus on our internal science and technology program as well as external opportunities within the rare metabolic disorders. We are actively engaged in licensing discussion on a number of gene therapy and gene editing technology and programs that we believe would be an excellent strategic fit in the Amicus portfolio and that may 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 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 technology.

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 important precision medicine Galafold for treating more people living with Fabry disease with amenable mutation.

Brad, please.

[Bradley Campbell, President and Chief Operating Officer]

Thanks, John. Good morning everyone. I will begin my remarks on Slide 5 with the snapshot of the Galafold launch as of March 31st, 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 of 2017 and this was slightly ahead of our internal forecast for this quarter.

And as John mentioned, we are 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 market, expansion into new markets, successful negotiation of drug pricing and market access and what we are seeing is continued very high rates of compliance and adherence to this oral precision medicine.

The remaining metrics on the slide support the strength of the launch as well as future growth opportunities and I will just touch on two of them here. Number one, as John mentioned, we secured an additional approval in Japan, bringing our total to seven 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 seven approvals highlighted on the previous slide, we have two 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 have touched on most of them already, but we do believe these lay the very strong foundations to support our upcoming launch in Japan and potential approval in launch in Untied States, which are so important given the opportunities to impact such a large portion of the global Fabry population, we estimate nearly 40% of diagnosed patients are in those two markets.

And finally on Slide 8, let me just drill down a little bit more into our launch planning of those two key markets. In both the United States and Japan we continue to have a strong presence at key congresses where we are working closely with key opinion leaders on disease awareness and appropriate medical education. We have expanded our special distribution systems to support both countries and in U.S. we are 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 are very pleased to announce that Mike [Harvoni] (Ph) 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 at great companies like [Capital, Stoby, CSO Bearing] (Ph) and others.

He has demonstrated success in building and leading teams as 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 the rare and orphan diseases.

We have 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 and 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 3000 patients are currently diagnosed and roughly half of whom are untreated today.

Turning now to Japan following with the March approval of our J-NDA we are now in the final stage of the pricing and reimbursement progress and continue to anticipate launching this quarter.

We have built an equally qualified team in Japan again who are so enthusiastic and ready to introduce Galafold to this important country where more than 850 people are diagnosed with that Fabry disease and where we continue to believe that a number 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 to a great success.

So in summary, we have made great progress so far this year and we are 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 and regulatory and clinical activities.

[John Crowley, Chairman and Chief Executive Officer]

Great, thank you Brad. Let me now [indiscernible] go ahead and highlight the significant progress with our important Pompe program focusing on what we have achieved since the beginning of the year across manufacturing clinical and regulatory activities.

Let me begin first by reiterating why we are so excited about this program at the World Meeting in February we have reported additional positive nine and 12 months results from the extension study for the 19 patients who are enrolled across the three cohort of this program. Those include 10 patients in Cohort 1 for ambulatory and to switch from the approved ERT standard of care.

Cohort 2 included four patients who are real chair bound and on full time ventilator support who also switch from the ERT standard of care. And Cohort 3, included five ambulatory patients who had never received any enzyme replacement therapy. These patients are all adults leaving with Pompe disease.

The data demonstrated overwhelmingly positive effect in nearly all of these patients, including on multiple measures of muscle strength and on all key biomarkers of disease. We have not seen an published literature responses of this magnitude and with such consistency of response. More over our data show these clinically meaningful improvements have been persistent with many patients actually seeing further strength improvements overtime.

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 very first patient enrolled in this study just crossed the two 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 the program so that At-GAA is available to as many Pompe patients as quickly as possible. To do so, there are ten 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 regulator activities on Slide 11, and I will 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 with pivotal study for full approval, as well as manufacturing activities and clinical data set to help us define the best and fastest pathway forward for AT-GAA.

Building on these initial interactions as well as the cascaded data from our ongoing Phase 2 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 of 2018 and we expect to provide an FDA update in 3Q after we see written 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 in 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.

We also have smaller supportive studies that we plan to commence, including studies in pediatric populations as well as study in further ERT naïve patients. As Brad will note in a few moments, we have had great success at the larger 1000L bioreactor scale and we will have significant quantity of the drug product available for further studies very shortly.

The second goal of the interactions with both 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 and the Pompe community today.

Pompe patients desperately needs newer and better treatment options as soon as possible. Despite the available therapy, many patients with Pompe continues 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 have also been encouraged by the level of support in the patients and we have certainly welcomed their continued input into the designs of our further studies and the sharing of their perspective on the extent of the unmet need with the regulators.

While we hope that we are successful in defining a conditional or accelerated approval pathways soon based on currently available data, the regulators may ultimately conclude that our current data while compelling is not sufficient to justify conditional 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 2 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 do the outcome of these upcoming meetings as binary event, we continue to believe that the evolving regulatory paths here will include a series of further [different] (Ph) 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 [indiscernible] and that's just delivered 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 be able to create 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 1/2 clinical study again, including 19 current Pompe patients to [indiscernible] 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 1/2 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 enrolled four to six 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 250L 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 assessment, pulmonary assessment and biomarker evaluation on all ten patients. Importantly, these patient 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 have seen thus far in our Phase 1/2 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 outcome in patients currently treated with standard of care ERT.

We intend this observational 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 18 month data from the Phase 1/2 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 have had great success in the manufacturing year of our novel ERT. I cannot overstate enough the importance of having successfully scaled our manufacturing from this 250L scale to the 1000L 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 greatly de-risks the program, it advances our timeline 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 that I am 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 the key manufacturing accomplishments that are so critical to the continued success of this program. Brad go ahead please.

[Bradley Campbell, President and Chief Operating Officer]

Great, thanks John. We really have come a long way and successfully scaling up from 5L bench scale to a 1000L GMP runs in just four years as highlight on Slide 13. The team have been laser focus all along the way of maintaining these - all attribute to ATB200 and as previously announced analytical and in-vivo comparability studies have been completed between the 250L and 1000L engineering batches and we have reached agreement with the FDA that material from those engineering batches is comparable.

We have also reached agreement with the agency and the comparability testing strategy for the GMP material and we have now successfully completed multiple family GMP runs at 1000L scale with the drug product release for packaging and labeling.

All of this progress is significantly de-risking towards our next major milestones for manufacturing perspective, including the release of the 1000L GMP material for initiation on the registration-directed study later this year, as well as securing final regulatory agreement on comparability between the 1000L GMP and 250L GMP material.

What is critical is the regulatory process is in the immediate term, we have 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 have 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 have successfully skilled up to the 1000L GMP scale at our current site we have also secured additional capacity of WuXi in China as well as the second source of supply through their plants and 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 facilities to compliment our own supply from WuXi we can ensure we will have capacity and capability at multiple sites to always meet the supply needs of the Pompe community which as we know is particularly important in this space. So again, important progress for 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 greater science, clinical and regulatory and now increasingly commercial capabilities. We are 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 will turn it over to Chip to review our first quarter 2018 financial results. Chip.

[Chip Baird, Chief Financial Officer]

Thanks Bradley, good morning everyone. Our financial overview begins on Slide 18, with our income statement for the three months ending March 31, 2018. 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 three 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 at 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 from prior year period. The increase in R&D expenses was due to a 6.2 million increase in manufacturing and clinical development costs related AT-GAA and a 3.5 million increase in personnel costs.

Moving down the income statement, total selling, general and administrative expense for the first quarter of 2018 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 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 spend for the first quarter of 2018 was 48 million was is tracking well within our 2018 full-year cash spend guidance of between $230 million to $260 million.

With our current cash position and continued ramp we see from 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.

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 it back to John.

[John Crowley, Chairman and Chief Executive Officer]

Great, thanks, Chip. So, this has been a great start to the year, you referenced Slide 20. I would like to again just reiterate the significant progress we have made against those five 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 five year vision to treat 5,000 or more patients within Amicus medicine. Throughout 2018 and along the way to 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 are happy to take any questions.

[Operator]

Yes sir. [Operator Instructions] And our first question comes from the line of Anupam Rama from JPMorgan. You may begin.

[Anupam Rama, JPMorgan]

Hey guys, thanks so much for taking the question. Maybe just a quick one on the Pompe regulator 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 two different registration type of strategies, in the U.S. and EU. And maybe a quick second question, in what timeframe do you guys expect to make a decision on the Fabry co-formulation and how tied is this to business development, what are some of the push and pull levers you are 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. Thanks so much.

[John Crowley, Chairman and Chief Executive Officer]

Yes, no thank you Anupam. 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 direct to 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 a month or two ahead from both agencies. You can see a divergence historically you have seeing, the agencies diverge there, but again our goal is to get alignment as close as we can.

Secondly, with respect to Fabry co-form again that's our own proprietary novel enzyme replacement therapy we have developed or together half or more of Fabry patients living with non-amenable mutations and it is co-formulated with our chaperone again to enhance the activity and stability of the protein.

In preclinical study as we saw significantly improved absorption into key cell of disease and significantly more reduction of the key substrate. As we have indicated before we have completed all preclinical proof-of-concept that has moved to WuXi for initial manufacturing, we are waiting to pull the trigger on advance manufacturing of that programming and a timeline for moving it into the clinic.

I think it is dependent in large measure on the work that we are 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 toward a secure for the diseases that we work and we are very excited about a range of this technologies and programs that we are 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 will 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 lead progress with a gene therapy approach. I think we'll have a important update on that in the second half of the year.

[Anupam Rama, JPMorgan]

Great. Thanks so much for taking our questions.

[John Crowley, Chairman and Chief Executive Officer]

Thanks Anupam.

[Operator]

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

[Tazeen Ahmad, Bank of America]

Hi good morning guys, thanks for taking my question. So John just a follow-up to the presentation that you have made back in February at the World Conference and have spoken with analysts there. At that point you had said that you would be adding patients to the study. Can you give us an update on how many patients has been added thus far. And then secondly, in terms of your timeline for update, you have 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.

[John Crowley, Chairman and Chief Executive Officer]

Sure I will comment on this first question with respect to patients. The enrollment to 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 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 one or two or three sites.

So because of that we are making sure patients have put in that appropriately, but really there is more than enough patient demand for access to AT-GAA so that we will be able to provide that data in the first half of 2019, again hopefully at a major science meeting. We are not going to give, we don’t traditionally give specific target objectives for small cohorts like these. We will let you know when it’s completed its enrollment, but again I feel at 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 have engaged in - that really are series of collaborative distributions with regulators that provided valuable feedback that’s informed a lot of the work that we are doing, certainly the briefing document and the design of our pivotal study as well as our approach in discussing conditional or accelerated approval pathways.

The timing of the meet and again that will move forward into a Type C meeting, that was on the FDA’s timeline and again [indiscernible] in 3Q, but I think important, and again I want to reiterate, while those meetings will be highly constructive and important, I do see this in the months and quarters ahead, continued collaborative engagement, a series of iterative discussions with the regulators as we move these medicines to more and more patients and ultimately to [indiscernible].

[Tazeen Ahmad, Bank of America]

Thanks John 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 now remaining questions about GI?

[John Crowley, Chairman and Chief Executive Officer]

Yes we are not going to comment on ongoing discussions with the FDA, I will just remind everybody that in February, they accepted the NDA filing, they gave it priority review, that PDUFA date is August 13th 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.

[Tazeen Ahmad, Bank of America]

Okay thanks.

[John Crowley, Chairman and Chief Executive Officer]

Sure.

[Operator]

Thank you. And our next question comes from the line of Joseph Schwartz from Leerink Partners. You may begin.

[Joseph Schwartz, Leerink Partners]

Great. Thanks very much and congrats on all the progress. So, you have 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 a filing for accelerated approval is a tougher cell 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 third quarter?

[John Crowley, Chairman and Chief Executive Officer]

Well 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 towards accelerated approval would look like if that is an option. Again 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 have not had those face-to-face meetings and discussions with FDA, those will take place shortly.

Again we are focused on everything that we can control in terms of making the drug, getting it to as many patients, expanding the study, 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.

[Joseph Schwartz, Leerink Partners]

Okay, great thanks 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 to be better suited to address the challenges in Fabry and then a gene therapy might potentially leapfrog your - combo in Fabry, is this because of anything you are seeing in the clinic or are the challenges in Fabry different 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.

[John Crowley, Chairman and Chief Executive Officer]

Yes, I think for us Joe, it really is part of a longer term commitments to patients and to our portfolio. Even when we started Amicus, we built in part of our mission statements that we have a duty to obsolete our own technology, we back in February made a commitments in our human beyond disease initiative to all patients with the rare diseases that's for any Amicus products 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 is a cure.

I think like a lot of folks we are very excited about what we are seeing increasing in the gene therapy and gene editing field and this maybe a long-term investment strategy for us for all of our diseases, but I think particularly for Fabry and for Pompe we are looking at or actively engaged in negotiations and discussions on a range of platform technologies and products in the gene therapy space.

What is clear to us is that once we begin with a disease target we then want to carry to world and diligence what we think could be the best technical approach, very clear, there is no one platform technology that's best for all of their metabolic diseases, not even for all life, it's almost - disorder.

So I'm really excited about some of what we are seeing, again we are very actively involved in a range of discussions, activities and I'm very hopeful that we will be able to build a programs 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 are at there.

[Joseph Schwartz, Leerink Partners]

Thanks John, good luck.

[John Crowley, Chairman and Chief Executive Officer]

Thank you Joe.

[Operator]

Our next question comes from the line of Mike Ulz from Robert.

[Michael Ulz, Robert W. Baird]

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

[John Crowley, Chairman and Chief Executive Officer]

Yes Mike. Again, that's part of the ongoing discussions, I think we have had a really smart plan that we have developed for that study that would ultimately be for full approval and in patient numbers I don’t want to give specifics, but you are in the dozens of patient ,we hope there are even a 100 patients for instance, but part of that is still to be determined. With respect to duration, we are looking at a number of different way to cut that but I think I will reserve that until we have got alignments from both regulatory agencies.

[Michael Ulz, Robert W. Baird]

Okay, great. Thanks.

[Operator]

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

[Ritu Baral, Cowen and Company]

Hey guys thanks for taking the question. John can you maybe give us some little more clarity about what you will 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 1/2 patients you will have, what preliminary data from Cohort 4, you might have, and will Pom-002 be largely complete and ready to show to the agency?

[John Crowley, Chairman and Chief Executive Officer]

Yes Ritu, whether or not given that we are just in the midst of these discussion, give any indication as to what the nature of those are, what I can assure you is that any data that we have collected to-date on patients treated with our product in whatever cohort they maybe together with any data that we may have building on this natural history study would all be available to regulators in the months ahead.

And certainly 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 will move forward. I hope that’s helpful.

[Ritu Baral, Cowen and Company]

Have you started treating patients in Cohort 4 yet?

[John Crowley, Chairman and Chief Executive Officer]

We have. We have begun enrolling patients.

[Ritu Baral, Cowen and Company]

Okay. And then just a quick follow-up, how are you looking at the changes within the FDA division, specifically the GI division and the structure and ahead et cetera., both for Migalastat and for AT-GAA?

[John Crowley, Chairman and Chief Executive Officer]

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

[Ritu Baral, Cowen and Company]

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

[John Crowley, Chairman and Chief Executive Officer]

We don’t expect it. No.

[Ritu Baral, Cowen and Company]

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

[John Crowley, Chairman and Chief Executive Officer]

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.

[Jay Barth, Chief Medical Officer]

Yes. 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 have had to-date and continue to have with 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.

[Ritu Baral, Cowen and Company]

Okay. Last question and I will 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 have had and any changes and thoughts on how you think of potential pricing for Migalastat in the U.S.?

[John Crowley, Chairman and Chief Executive Officer]

Great and Brad why don’t you go ahead and fill those please?

[Bradley Campbell, President and Chief Operating Officer]

Yes, thanks Ritu for your questions. So you may remember that going back quite a way is actually the research that we have seen and as we have 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 operation which is great.

In terms of writing sort of launch strategy et cetera, I think we have laid out in the slides here what we think are the foundation of our success and our launch strategy outside of the U.S. and we will 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 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 I think we will continue to reiterate our strategy which is we believe our medicines must be fairly priced and broadly acceptable and we just expect at this point that we will stay with our parity pricing strategy which we think reflects the value of the products and afford some savings back to the system in terms of avoiding used and associated and cost. Of course that will depend on the label et cetera, et cetera, so more to say on 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.

[Ritu Baral, Cowen and Company]

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

[Bradley Campbell, President and Chief Operating Officer]

Yes, I think that is safe to assume at this point, off course we will no more as we get into the launch. Perhaps the one big difference off course in the United States is outside of the U.S. there is really not an ability to engage with patients directly, I would really have to go through physicians where in the United States there is a difference there. So it's a slightly different dynamics, but by and large I think you should expect the same launch strategy as we have seen.

[Ritu Baral, Cowen and Company]

Got it. That’s helpful. Thanks for taking the questions guys.

[Operator]

Our next question comes from the line of Sylvan Richard from Goldman Sachs [Operator Instructions]. 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.

[John Crowley, Chairman and Chief Executive Officer]

Great, thank you operator, and I will just remind everybody at the J.P. Morgan conference beginning of the year, we have set out our goals for the year and including the five key strategic priorities and just remind everybody we will make great progress along all five of those and look forward to continued interactions and welcome any follow-up phone calls or questions. Thank you for the continued support. Have a great day.

[Operator]

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