Amicus Therapeutics Inc (FOLD) 2022 Q1 法說會逐字稿

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics First Quarter 2022 Financial Results Conference Call and webcast. (Operator Instructions) As a reminder, this conference call is being recorded.

I would now like to turn the conference over to your host, Mr. Andrew Faughnan, Executive Director of Investor Relations, you may begin.

Thank you, Charlotte. Good morning, everyone. Thank you for joining our conference call today to discuss Amicus Therapeutics' first quarter 2022 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; Daphne Quimi, Chief Financial Officer; and Dr. Jeff Castelli, Chief Development Officer. Joining for Q&A will have Dr. Mitch Goldman, Chief Medical Officer; and Sebastien Martel, Chief Business Officer.

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 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 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 and Risk Factors section of our annual report on our Form 10-K for the year ended 2021 and for the quarter ended March 31, 2022, to be filed later today with the Securities and Exchange Commission.

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

Great. Thank you, Andrew, and welcome, everyone, to our first quarter 2022 results conference call. I'm pleased to highlight the successful start to the year across our business and reiterate the Amicus team's focus on our business priorities, despite, as we all know, this tumultuous stock market environment across the biotech sector.

Let me begin by emphasizing that we are sharply focused on 3 primary objectives: one, continue to advance Galafold to as many patients in as many geographies as possible; two, secure approvals for and launch AT-GAA in Pompe disease globally; three, ensure the financial strength of Amicus including the path to profitability without the need for any further equity-based financing.

As we did in this morning's press release, let me elaborate on several key accomplishments. First, Galafold continues its strong performance and remains the cornerstone of our success. With $78.7 million in first quarter revenue, a nearly 19% increase from a year ago, we continue to be very pleased with the uptake of Galafold globally. Its growth thus far this year is meeting or exceeding our objectives in all key geographies. We are excited to announce also that the intellectual property portfolio estate for Galafold has been strengthened by the issuance of 8 new patents this year.

Together, there are now 35 orange book listed issued patents related to Galafold in the United States, which provide protection through 2038, including the very important and recently issued composition of matter patent. This portfolio will provide broad and long-term intellectual property rights well into the late 2030s for this novel precision medicine. We expect significant growth for Galafold in 2022 and with this strengthened IP protection, believe that it has a long runway into the next decade and beyond and that Galafold continues to have the potential to achieve $1 billion in peak revenue.

Second, we continue to make great progress on our global regulatory filings for AT-GAA, our novel next-generation therapy for Pompe disease. The U.S. and EU regulatory reviews are progressing very well, and we are extremely pleased with the level of engagement from the regulatory agencies. We now have recently completed the late cycle review with the U.S. FDA and are now in labeling discussions with the agency.

We remain confident in AT-GAA's approvability and its potential to become the next standard of care in Pompe disease. We continue to expect the manufacturing inspection at the WuXi facility for the biologic ATB200, again the biologic component of AT-GAA and that this will be needed for FDA approval. We are carefully watching the COVID-19 situation in China and recognize that it could impact the potential timing of an inspection. We are in communication with the agency regarding the best way to support an inspection. Amicus also continues to evaluate all options with respect to the inspection requirement. We and our partners at WuXi have completed all preparatory work, including internal mock inspections and are fully prepared. Today, we continue to expect the agency to improve -- to approve the applications together by the July 29 action date.

In the EU, we are actively engaged with the EMA on the Marketing Authorization Application, or MAA, for AT-GAA. We continue to expect a positive CHMP opinion later this year and commercial launch in 2023.

Of note, importantly, the EMA has indicated that it will not require a new inspection of the WuXi manufacturing site. Our global launch plans continue to accelerate, including prelaunch activities, targeted investments in additional personnel to support the launch and significant investments in launch inventory preparations. We are now closer to having another potential treatment option for people living with Pompe disease, both in the United States and in Europe, with further regulatory applications planned in the months ahead. Of note also, both the EMA and the U.S. FDA have approved the trade names for this novel new treatment paradigm for people living with Pompe. So we expect one global brand name, which, of course, we will not disclose until approval.

And third and importantly, Amicus has maintained a strong financial position and remains committed to achieving profitability in 2023 as we continue to execute on the global expansion of Galafold, and prepare for the global launch of AT-GAA. We have no need for any equity or equity-linked financings to achieve profitability.

In order to accomplish this, we will concentrate the vast majority of our efforts and investments in our priority growth franchises in Fabry disease and Pompe disease. And we'll do this through the continued global growth and commercialization of Galafold through advancing the activities to secure global regulatory approvals and commercial launch for AT-GAA and by making judicious strategic investments in next-generation therapies in Fabry and Pompe and in core science and platform technologies in the field of genetic medicine. We are rapidly approaching 2 pivotal inflection points for Amicus. First, the global launch of a second rare disease medicine with AT-GAA. And secondly, profitability. These are feats that we believe will enable Amicus to truly enter the upper echelon of biotechnology companies.

Ahead on Slide 6 and as we laid out last month, we are focused on achieving our key strategic priorities for the year. These include; one, we will continue to drive Galafold to more people living with Fabry disease with a minimal variance in existing and new markets. We look to achieve double-digit global product revenue growth of 15% to 20% and with revenue of $350 million to $365 million at constant exchange rates. This reflects the strong momentum and demand behind this precision medicine globally. Second, we remain steadfast in our commitment to advancing AT-GAA regulatory filings and initiating the anticipated launch of AT-GAA in the United States, leveraging our seasoned global commercial team and experience across all areas for an effective drug launch, we are fully prepared and anticipate a successful launch of AT-GAA. Third, we are strategically advancing our best-in-class next-generation genetic medicines and capabilities.

And finally, again, we continue to maintain a strong financial position as we carefully manage our expenses and investments. and we remain fully funded through all major milestones.

With that introduction, let me go ahead now and turn the call over to Bradley Campbell, our President and Chief Operating Officer, to further highlight the Galafold performance. Bradley?

Great. Thanks, John. Good morning, everyone. Let me start by walking you through in more detail, our Galafold performance for the quarter on Slide 7. For the first quarter, again, total product revenue grew 18.5% to $78.7 million globally. This is driven by strong new patient accruals and continued sustained patient compliance and adherence rates. The geographic breakdown of revenue during the quarter was consistent with what we've seen historically with $55 million or 69% of revenue generated outside the United States and the remaining $24 million or 31% coming from within the United States, and this is in line with the roughly 70-30 split that we expect as we continue to grow both parts of the business.

Now turning to Slide 8. Q1 results highlight the strength of the global commercial efforts. This business continues to be incredibly resilient with patients added in all major markets and an operational growth rate of 23.5% over the same quarter last year at constant exchange rates. As you can see in the 2 graphs depicted on this slide, Q1 results follow the revenue pattern we've continued to observe were due to a variety of factors, including timing of orders as well as payer re-authorizations in the United States. The rate of growth is typically nonlinear and a smaller percentage of annual revenue falls into Q1. Here, we've also called out several of the drivers and metrics which will lay the foundation for growth this year and beyond.

We ended the first quarter with a little under half of the global market share of treated amenable patients. And while the global mix remains about 55% switch patients and 45% naive patients, in many geographies, we're starting to see stronger uptake in naive populations, which is in line with our strategy from launch. So we're achieving high market shares in countries where we've been approved the longest, but there's still plenty of opportunity to continue to switch patients over to Galafold and to continue to grow the market as we penetrate into the diagnosed untreated and newly diagnosed segments as well.

Within our core business, the EU, U.S., U.K. and Japan, we continue to see growth coming out of these markets. And in fact, in Europe, where we've been on the market the longest, we're now at about an 80% to 90% market share of switch patients. The growth there is now being driven by bringing in diagnosed, untreated patients and newly diagnosed patients. Whereas in Japan and the United States, the overall patient mix is still about 50-50 switch versus naive. Although there, we're evolving more towards naive patients as well.

On the other hand, if you look at the newer markets coming in Latin American countries like Brazil, Chile, Argentina and Colombia, the Asia Pacific countries outside of Japan, the Gulf states as well as countries in the Middle East and North Africa, including Turkey, where we're hoping to get approval later this year. We see the majority of net new patients are switch patients, and we expect that to continue throughout the next several years.

All of that is underpinned again by impressive compliance and adherence rates that continue to exceed 90%, reiterating our belief that those patients who go on Galafold generally stay on Galafold. And importantly, the value of Galafold continues to be recognized by payers, with the vast majority of insurance reauthorizations granted in 2022 by U.S. payers within the first quarter and a very strong track record of successfully negotiating and renegotiating reimbursement outside the United States.

Our relentless focus remains on ensuring access to Galafold for anyone who needs it. Within the table on the right side of the slide, we've provided a 3-year historical snapshot of the average portion of Galafold sales that occur each quarter during a given year, and we expect a similar trend to occur this year.

Now moving to Slide 9. What we've seen so far this year is that Galafold uptake continues to track very well and we're seeing growth in demand across all of our major markets as well as most of our smaller markets as well. We're on track to achieve our full year revenue guidance of $350 million to $365 million at constant exchange rates and Daphne will provide a little bit more color on FX later in the call.

Encouragingly, we continue to see signs of improvement in terms of patient access in the COVID environment. We recently conducted a survey of a number of Fabry physicians around the world and only about 40% to 50% of Fabry physicians still feel that COVID is impacting the diagnosis of new patients and initiation of treatment. But the majority said they're starting to prioritize Fabry care again, and we've actually seen an almost tenfold increase in the number of in-person physician interactions with our field sales and medical team from 150 face-to-face interactions in January, February of 2021 to over 1,000 in the same period, January, February 2022. We think that's a great sign for things to continue to unlock. And altogether, we view this as a very strong start to the year.

Moving to Slide 10, looking forward a little bit. We know that Galafold has the potential to surpass $500 million in annual revenue over the next few years through 3 primary growth drivers that we continue to talk about; continuing to penetrate into existing markets, continue to expand into new geographies and broadening the labels for Galafold. In the longer term, we remain confident in that $1 billion peak revenue opportunity as we continue to see significant growth in the Fabry market globally, driven by diagnosing patients through a variety of measures, including high-risk screening, newborn screening and other diagnostic initiatives, which we continue to support and invest in as well. And finally, as John touched on, we have orphan exclusivity in the U.S. and Europe, in addition to our now 35 orange book listed patents that give us IP coverage into the late 2030s, 19 of which provide protection through 2038, including the latest composition of matter patents, all of which give us opportunity to provide access to Galafold globally for many years to come.

With that, let me turn the call now over to Dr. Jeff Castelli, our Chief Development Officer, to highlight our ongoing Galafold initiatives across our clinical regulatory medical teams as well as our AT-GAA program. Jeff?

Thanks, Bradley, and good morning, everyone. Starting here on Slide 11, we highlight the ongoing work we're doing to build the body of evidence around Galafold. First, we continue to look to broaden the global labels within the asset and pediatric populations as well as adding additional variants or mutations to the label. Second, our medical team continues to work diligently on our publications and medical conference schedule. Long-term data was published last year in the molecular genetics in the capitalism reports as well as presented at the 2022 WORLDSymposium in February. These long-term data highlighted stable renal function during treatment up to 8.5 years on Galafold irrespective of treatment status, gender or phenotype and inclusive of classic males.

We have also presented at conferences and recently submitted a publication looking at Fabry outcomes, measured by rates of kidney, cardiac and CNS events, which we believe compare favorably to similar studies in the medical literature. Third, Amicus significance over 500 patients in a large global registry as part of our post-marketing commitments and is exploring ways to leverage this wealth of long-term data to add to the growing body of evidence for Galafold through publications as well as towards providing confirmatory evidence to support our remaining post-marketing requirements. Additionally, we have multiple other ongoing and planned Phase IV studies that we continue to move forward.

And last, as Bradley mentioned, we continue to add to our IP coverage and now have 35 orange book listed patents. We see all of this continued work on evidence generation for Galafold as an important driver of the long-term success.

Moving on to Slide 13 on our AT-GAA program. It's important to recognize that Pompe disease continues to pose a range of health challenges for people affected by the disease and having therapeutic choices is crucial. Pompe is a severe and fatal neuromuscular disease and one of the most prevalent lysosomal disorders. Multiple publications in natural history studies continue to highlight the initial benefit of treatment being followed by continued long-term decline on key measures of disease for many patients.

Here on Slide 14, we present a summary of the primary and key secondary endpoints from our Phase III study. As a reminder, PROPEL was a double-blind randomized study assessing the efficacy and safety of AT-GAA in both treatment naive and ERT-experienced patients against the approved therapy alglucosidase alfa. PROPEL is the only controlled trial to date that included ERT-experienced patients, which represent one of the greatest set of patients with unmet needs. Endpoints across motor function, muscle strength, pulmonary function, patient-reported outcomes and biomarkers, including the 2 most recognized endpoints in Pompe, 6-minute walk distance and FVC shown here on the slide, favored AT-GAA over alglucosidase alfa in the overall population. We believe this consistency of effect across the key disease manifestations of Pompe, illustrates the potential impact of AT-GAA for patients.

On Slide 15, we call out the ERT-experience population where 95 participants who are on the standard of care for more than 7.5 years on average. Generally, this is associated with the decline phase of disease for most patients and we actually saw an increase in 6-minute walk in these patients and stabilization in their FVC, which achieved nominal statistical superiority on both endpoints versus alglucosidase alfa, and showed a clinically meaningful outcome never before seen in this population.

Moving on to Slide 16, adding to the overall body of data behind AT-GAA. Recently at the 2022 MDA Clinical & Scientific Conference a few months ago, the Amicus team presented positive long-term data from the Phase I/II study of AT-GAA.

As seen on Slide 16, these latest data continue to represent very meaningful and importantly, durable improvement in functional outcomes as well as persistent reductions in key biomarkers of muscle damage and disease substrate out to 3 years. The results have been shared with the Global Regulatory Authorities in the U.S. and EU as part of their ongoing reviews and compared to what is known about the natural history of both untreated and ERT-experienced Pompe patients. These durable results give great hope that AT-GAA indeed has the potential to become the new global standard of care for people living with Pompe.

Moving on to Slide 17, we have highlighted the key updates on the AT-GAA program. First, as John mentioned on the regulatory progress. Last year, the U.S. FDA accepted [for review], the BLA for cipaglucosidase alfa and the NDA for miglustat, the 2 components of AT-GAA and the agency set a PDUFA action date of May 29, 2022, for the NDA and on July 29, 2022, for the BLA. We expect these filings will be approved together by the July 29 action date, again, dependent on the ability for FDA to conduct an inspection at the WuXi manufacturing facility. And again, we're working with the FDA to consider alternatives to a full on-site inspection. We've also shared that the MAA has been submitted to the European Medicines Agency and is under review. The CHMP opinion is expected later this year.

Of note, as John also mentioned the EMA is indicated in writing that based on prior manufacturing inspections of this facility, an inspection is not required for AT-GAA at WuXi to support the MAA. We are now very excited to also have multiple expanded access programs in place globally, including in the U.K., Germany, Japan and other countries. This includes the EAMS framework that we announced in June, in which AT-GAA was granted a positive scientific opinion through the early access to medicine scheme by the UK's MHRA. This positive opinion recognizes the high unmet medical need based by the Pompe community, and permits eligible adults living with late-onset Pompe disease who have received alglucosidase alfa for at least 2 years to switch to AT-GAA, granting access prior to marketing in the U.K. We're seeing significant enthusiasm for AT-GAA under the EAMS mechanism with multiple physicians having requested access across all the leading Pompe centers in the U.K. and multiple patients now receiving this novel 2-component treatment.

Since the positive scientific opinion interest and momentum for AT-GAA has grown, and we are pleased to be able to provide access to those who are eligible. And we continue to report that well more than 150 patients worldwide continue to be treated with AT-GAA across all our clinical extension studies and expanded access programs.

And for the younger Pompe community, we continue to enroll the ongoing open-label study in children with late-onset Pompe disease up to 18 years of age and look to expand into patients with infantile-onset Pompe as soon as possible.

And finally, in response to the many requests for expanded access that we received for children living with IOPD, our expanded access programs for both those living with infantile- and late-onset Pompe continue to add patients for multiple individuals as appropriate.

And with that, let me hand the call back over to Bradley to further discuss our launch preparations for AT-GAA. Brad?

Sorry about that. Thanks, Jeff. Just on Slide 18, as Jeff mentioned, as we talked through our launch preparations for AT-GAA. We are poised for another successful product launch, building on the success of our Galafold franchise, although unlike when we launched Galafold, when we are hiring the commercial organization and support of infrastructure from scratch, we now have a presence in over 40 countries around the world, including all the major markets. And that team will be largely the same that we launch AT-GAA with only a small handful of new FTEs needed. We have experience across all areas that are needed for successful drug launch, regulatory, commercial, supply chain, experience with payers, reimbursement and access, in addition and most importantly, the key relationships with the physicians and medical community around the world.

We're very confident in our world-class commercial organization that we can leverage with their experience and relationships and deliver AT-GAA to people living with Pompe disease around the world. From the team, the medical education, the now published Phase III data in the highly regarded Lancet Neurology, our experience and focus on reimbursement and access. And again, all the strategic planning that we're doing together with building inventory with our partners at WuXi Biologics. We believe we're in a very strong position for a second, rapid and successful launch for Amicus.

And with that, let me turn the call now over to Daphne Quimi, our Chief Financial Officer, to review our financial results, guidance and outlook. Daphne?

Thank you, Bradley, and good morning, everyone. Our financial overview begins on Slide 20 with an overview of our Q1 revenue performance and FX impacts. For the first quarter, we achieved Galafold revenue of $78.7 million, which is a 19% increase over the same period in 2021. This includes a year-over-year operational growth of 24% and a negative currency impact of 5%. As broadly highlighted, given 69% of Galafold revenue is generated outside the U.S., we see significant FX exposure to our reported revenue numbers. Applying average April 2022 exchange rates, the FX impact on 2022 full year Galafold reported sales would be a negative impact of approximately 6% to our reported numbers for the whole year.

Slide 21 outlines our income statement for the first quarter ending March 31, 2022. Cost of goods sold as a percentage of net sales was 10.4% in the year as compared to 10.2% for the prior year period. Total GAAP operating expenses were $146.5 million in the first quarter as compared to $112.9 million in the first quarter of 2021. The increase reflected expenses related to the reprioritization of the gene therapy portfolio, manufacturing and marketing costs to support the global launch of AT-GAA and increased stock compensation expense.

On a non-GAAP basis, total operating expenses were $109 million in the first quarter as compared to $90.5 million in the first quarter of 2021. We define non-GAAP operating expense as research and development and SG&A expenses, excluding share-based compensation expense, loss on impairment of assets, changes in fair value of contingent consideration and depreciation.

Net loss for the first quarter of 2022 was $85.3 million or $0.30 per share as compared to a net loss of $65.7 million or $0.25 per share for the prior year period. As of March 31, 2022, we had approximately 280 million shares outstanding. This year, we expect total non-GAAP operating expense to be within the range of $470 million to $485 million, driven by continued investment in the global Galafold launch and AT-GAA clinical studies and prelaunch activities.

As we highlighted previously, this guidance also includes an approximate $70 million related to certain nonrecurring costs for the manufacturing of AT-GAA, support the global launch as well as committed obligations for the gene therapy portfolio. Importantly, in 2023 and beyond, we would expect non-GAAP operating expense levels to decline to a similar level as in 2021.

Turning now to Slide 22. We continue to operate from a position of financial strength and remain on track to be self-sustainable without the need for any future dilutive financing and expect to achieve profitability in 2023 as we defined in our press release. We will focus the majority of our investments on our core value-driving franchises in Fabry disease and Pompe disease by continuing to deliver on the global growth of Galafold, securing approvals and launching AT-GAA globally as well as driving efficiencies, cost savings and careful expense management.

A few comments about our cash position and 2022 financial guidance. Cash, cash equivalents and marketable securities were $411.2 million at March 31, 2022, as compared to $482.5 million at December 31, 2021. Our full year Galafold revenue guidance is $350 million to $365 million before any currency impact in addition to our non-GAAP operating expense guidance of $470 million to $485 million.

And with that, let me turn the call back over to John for closing remarks.

Great. Thank you, Daphne and Jeff and Bradley as well. So as you can see, we have been relentlessly focused on performance across the business, driven by our team of global entrepreneurs, passionate people who have led and will continue to lead us on our patient focused mission.

And with that, operator, we're happy to take questions.

(Operator Instructions) Your first question comes from Ritu Baral from Cowen.

John, since you mentioned that labeling discussions for AT-GAA have already started. Can you talk to the tenor of those discussions around the breadth of the label? And then as you're having those discussions overall, including on manufacturing, I guess, what's negotiable on that WuXi inspection? Has there been prior inspections, so there's only elements, I guess, to focus on? Can you give us a little more clarity around that?

Sure, Ritu. So I'll take both parts of those. With respect to the label negotiations, obviously, we take it as a very good sign that we're moving through negotiations on the label and discussions with the FDA. I'm not going to talk about exactly what we're discussing with the FDA. Obviously, the things that we care about. So for instance, making sure that we highlight the important data in the switch population and other matters, we feel really good about the tone and tenor of those negotiations, and we continue to believe that the label will support a launch that will provide access to people living with Pompe disease that would be in patients' interest and also commercially very viable. But again, we think it's very good and continued with the practice that we've had with the FDA, of the spirit of discussions. And again, we're very confident in the approvability of AT-GAA with a label that will support extraordinary success for patients and for the product commercially.

With respect to manufacturing, the FDA has indicated all along that they're going to have to do an on-site inspection of the WuXi Biologics facility in WuXi City, in China. We've completed with WuXi all of the preparatory work necessary to facilitate that inspection. We've completed, for instance, in March, a mock inspection with our auditors internally that went very well. We continue to look at all options with the FDA around other ways in which they may conduct the inspection. And right now, we continue to expect that the PDUFA action date of July 29 would be something that would be able to be achieved in this time frame.

Your next question comes from Anupam Rama of JPMorgan.

Maybe following up on Ritu's question here about the FDA review of the WuXi facility in China. Could you expand a little bit on the strategies that you're kind of exploring? You've previously talked about maybe FDA employees in the region, helping conduct the review. What other strategies are being employed here?

Sure. I won't comment on any ongoing discussions with the FDA. Are you correct on a pump that the FDA does have FDA-designated employees in China? The WuXi City facility is not currently under lockdown. A lot of the complexity here has to deal with Shanghai itself, again, to remind everybody, WuXi is about 3 or 4 hours North of Shanghai. So we continue to expect that we will need a full on-site inspection by the FDA. That's what they've indicated to us, and we expect that, that could be completed by the July 29, PDUFA action date.

In parallel, we are also evaluating all other options, remote interactive options and other ways in which the inspection could be conducted, ways in which they've done it with other sponsors as well. So we're fully prepared for all potential contingencies.

Our next question comes from Ellie Merle from UBS.

Maybe just on sort of Pompe launch preparation. Maybe first, if you could give the latest color in terms of how the EAP is going in the U.K.? And maybe any color in terms of uptake and physician feedback there? And then just thinking towards the U.S., maybe just if you could give color on your expectations for uptake in patients that have already, maybe started on neoGAA and how you're thinking about sort of uptake in the ERT-switch patients as well as potentially switches from neoGAA and any initial thoughts there?

Sure. Bradley, do you want to handle both of those on the EAMS and EAPs as well as our launch expectations in the United States?

Sure. Thanks, John. Thanks, Ellie, for the questions. So first, for expanded access, yes, we've continued to provide expanded access through a number of mechanisms, probably the most visible one or the most important one is EAMS at this point, which is their Early Access Medicine Scheme in the U.K. And that's important because both AT-GAA and neoGAA are available through that scheme. And what we've heard is very positive feedback towards AT-GAA. We know that all of the key centers that prescribe Pompe medication have apps for and are now receiving the EAMS -- sorry, access to AT-GAA through the EAMS program. So that's, I think, really important to see how much support and excitement there is for AT-GAA through that program. We would expect that number to continue to climb.

And it's -- the U.K. is actually a pretty significant Pompe market. And so we think that's a great opportunity for patients to get access to AT-GAA while the U.K. is reviewing the application. And obviously gives us a great view into how the community sees AT-GAA as a potential treatment for Pompe. And again, we have other mechanisms in other countries around the world as well, where we see strong uptake.

In the United States, to your point around opportunities for uptake for sure. We think the switch population, as we've noted all along, is the clearest unmet medical need. We are, as John alluded to, continuing to negotiate what the indication would be for AT-GAA. But if you just focus on the 2 segments, you talked about experienced patients with -- who are on Myozyme and experienced patients who perhaps have gone to neoGAA. From our perspective, I think any Pompe patient who falls within the label would be indicated for AT-GAA. We believe that the data show that our product has significant improvement, in particular, in switch patients and would encourage folks to refamiliarize themselves with the longer-term data from the studies on the neoGAA and the Phase I/II and then the publicly available data in the Phase III to see how those patients perform over time, but we're very confident that AT-GAA could have utility in both of those populations. And we think the data are a friend there, and you'll see over time that we have good chance to be, we think, standard of care in all Pompe patients.

Your next question comes from Joe Schwartz from SVB Securities.

This is Beth Feindt-Scott, on for Joe. I was just wondering if you could share any physician feedback you received on the 36-month data you presented at the MDA conference this year and also building off that, I believe you said you plan to have open-label extension data from the PROPEL trial later this year. We were just wondering if you'll be able to share that open-label extension data from PROPEL with regulators ahead of the approval decision.

Great. Jeff, do you want to take both of those on the physician feedback on the 36-month data that we shared at WORLD as well as any color on the open-label PROPEL study extension data?

Sure. Yes. One of the biggest unmet needs in Pompe is really durability of treatment effect. I think that's something we see now in every study that's booked longer term, just for whatever reason, these patients shows some potential initial mix benefit, but then they generally tend to decrease longer term. And I think we've seen that now with multiple treatments that the long-term data seems to show some leaning of effect. And I think what was really exciting about that Phase I/II data that we just presented was after 3 years, those improvements we saw in the Phase I/II patients have been maintained. And again, that was in switch patients, naive patients. So we were excited by that data. We got very good reception to that data as well.

And then yes, this year, we will continue to -- we have patients we've been following now in that Phase I/II that are now off that 5-plus years of treatment. So we'll have later data cuts even of that Phase I/II population. And in addition, we'll have data cuts from our ongoing PROPEL extension, the patients after 1 year continuing on AT-GAA or that Lumizyme arm that switched to AT-GAA, we'll have at least 1 year of data in all of those patients.

We don't have specific dates and conferences we can announce yet, but we do plan to have that data disclosed here this year. But we don't have necessary obligations to include that data in the submissions in the U.S. or the EU. At some point, we may add that longer-term data. But for the initial review and approval, we don't need to add additional data.

Great. Thanks, Jeff.

Your next question comes from Kristen Kluska from Cantor Fitzgerald.

I wanted to ask outside of all the additional findings that you've presented and published on AT-GAA since the PROPEL readout, what else gives you confidence heading into this decision, but then also in the role that AT-GAA could place in the market. So for example, at around the agency commentary on the unmet need to the EAMS, next Myozyme labeled. And then also publications around the different endpoints. So for example, I know in the past, you really emphasized some of the newer work on SBC and the understanding around mortality risk.

Great. And actually made our job easier by highlighting a number of the different elements that we think will go into making this a differentiated medicine for people. Again, I'll just highlight that this was -- the PROPEL study was the largest study ever conducted in any lysosomal storage disease. And nearly 80% of the patients in that study had switched from the standard of care from Myozyme/Lumizyme. They had switched to AT-GAA. And again, the vast majority of people who we see taking AT-GAA for the first number of years, there are going to be people who are currently on enzyme replacement therapy, whether that be Myozyme or Lumizyme, or any of those who may already be switching to the neo product. And we would expect and hope for a label to support that.

So again, the data that we have in that very large study, particularly in that switch patient population, again, showing the nominal statistical significance on 6-minute walk on forced vital capacity in virtually all secondary endpoints. This is a very sophisticated patient community and physician scientist community, and they will be data driven. So they look at the data from that study. And again, to look at the data that Jeff just talked to, the long-term durability data that we see from our Phase I/II and in the months ahead, the data that we'll show, we hope, with the long-term durability patients in the PROPEL study.

So we think all of the data from all the clinical studies together with the real-world experience of physicians, now with multiple uses in patients ranging from infants all the way through adults and the EAMS study. We would expect, again, to Bradley's point, significant numbers of patients in the United Kingdom to switch from existing standard of care enzyme from the approved Myozyme to AT-GAA over the coming months and into 2023 as we then look to the full approval.

So I think all of that builds the totality that's overwhelmingly, we think, positive for AT-GAA and presents a very, very strong risk benefit profile for physicians. We know doctors and as we're engaged appropriately with physicians, patient community are extremely excited for the potential option of AT-GAA. So Jeff, I don't know if you've got any other comments to add.

No. I think, John, you covered it well. It's really just that consistency of effect across parameters that we measured in patients that we treated and all the different end points across the key manifestations either nominally, significantly or numerically favored AT-GAA over [cost] itself and then across naive patients, experienced patients, non-ambulatory patients from Phase I/II, patients with higher or lower baseline severities. We've just seen a really consistent effect. And I think that's really the key that consistency adds into the weight and the meaningfulness of the treatment.

No, it's an important point, Jeff, too. And again, Kristen, I'd probably summarize that, that when you look across all the data that we've had and now more than 6 years of clinical research to look at the magnitude, the consistency and the durability of that data, we think are particularly impressive and will bode very well. And again, this is a market where you had enzyme replacement sales last year, global revenue with one product of over $1 billion. And with increased diagnosis rates and the ability to effect and positively effect, we hope the lives of many people living with Pompe and finding many more new patients broadly, for us and for others in the market.

We think this is a market with a potential for more than $2 billion in global revenue. And we think we have the potential for at least half of that by the end of this decade. So when you step back then and look at Amicus with Galafold is the cornerstone of our success, where we are today and where we'll take it with the length of that intellectual property protection well into the 2030s with $1 billion revenue potential for Galafold with what we believe will be an approved product in Pompe disease that we think has the potential to become the standard of care or at least split the market with the existing entrenched at other competitor in the field, that's another potential $1 billion revenue product also with significant intellectual property protection, including composition of matter on AT-GAA, extending well into the 2030s. That combined with the financial strength of the company, we think, is pretty distinguished particularly in this volatile market. So...

Our next question comes from Dae Gon Ha of Stifel.

On the labeling discussion, I was kind of curious in terms of differentiating AT-GAA from neoGAA, wondering if you can comment on whether FDA has asked for any additional data cuts. We've seen some of that happen recently -- then perhaps that could accentuate some of the profile that you were just alluding to.

Yes. Thanks, Dae Gon. They've not asked for any additional data cuts. We have supplied them with all the data. So for instance, they've seen the 36-month data from the Phase I/II study, but they've not asked for instance, for the PROPEL study for any additional data cuts.

Your next question comes from Salveen Richter of Golden Goldman Sachs.

This is Elizabeth on for Salveen. Maybe going back to some of the initial questions. How do you expect to message around the WuXi inspection? And should we expect you guys to communicate when that takes place to the Street? And then maybe you could just remind us of what we should expect to learn from the first FDA review date.

Sure. Let me take that first part again, to be perfectly clear, and we've had this discussion with the agency. We expect no action on May 29 from the agency, and we expect both the NDA and the BLA. We expect action by July 29, which, of course, we hope to be approval. With respect to -- look, there's a lot of activities going on right now with labeling discussion, all of our internal launch preparations. But in addition, of course, the important final CMC parts. Important to note that this has -- we've had tremendous manufacturing history here with WuXi, and we think that bodes quite well. And we've been very successful in all of our discussions and much of the focus in the BLA review has been on the CMC section, which we fully expected.

Dozens and dozens of information requests from the agency going all the way back to September into the early part of this year, we believe we've satisfied the FDA's questions with respect to the CMC section, and we do have this important WuXi inspection. Again, a lot of different ways, we think that could be satisfied. We're to the point now we're not going to be providing the Street with -- I mean, literally daily or week-to-week, we're having our exchanges with the agency. We're not going to provide the blow-by-blow including updates on manufacturing, inspection, when did it take place? When is it scheduled, how did it go? What did they ask? We're not going to provide that color. We're now into the final stretches coming up to the July 29 PDUFA date.

So continue to expect the medicine to be approved by the PDUFA date. And obviously, if there are any changes to that, we would provide that update to the Street. But again, we remain very, very confident in the approvability of AT-GAA and are fully prepared for the launch in the United States. And again, we're in active discussions with the EMA on the MAA and expect the CHMP opinion before the end of the year with launch in Europe in 2023.

Your next question comes from Yun Zhong of BTIG.

Can you remind us the size of the sales force that you have in the U.S. and in Europe? Did you -- or do you plan to increase the size in preparation for the AT-GAA launch? And also given that it's still an IV infusion, not like the oral for Galafold, do you expect that you still have to face some type of, call it, related challenges when you launch AT-GAA?

Yes, sure, Bradley. I'll go ahead and take that over to you, particularly to highlight the operating leverage that we have at Amicus.

Yes. Thanks, John. So just as a reminder, what we've characterized is we have about 150 global field personnel between sales, medical, market access, et cetera. That organization is effectively the organization we'll use to launch AT-GAA. So we get significant leverage out of the Galafold commercial organization -- commercial and medical organization, I should say. What we said is we would intend to add perhaps less than a dozen FTEs globally to support the launch, primarily in direct marketing, direct medical and then in the United States, some market access folks, in particular in support of our patient assistance hub. So highly leverageable organization to be able to support the launch of AT-GAA.

In terms of your second question as it relates to COVID impacting the uptake of AT-GAA. At this point, based on what we know from Galafold, based on where we see the global markets evolving, we don't expect any significant impact on AT-GAA uptake. Of course, we'll continue to monitor that. But I think the trends we're seeing now give us good confidence that we're moving into a place where patient care is largely able to move forward, perhaps in a slightly evolved way. Perhaps more hybrid interactions between our field force and physicians. But at this point, we don't see a significant impact by COVID on our AT-GAA launch.

At this time, I would now like to turn the conference back to Mr. John Crowley, Chairman and CEO, for closing remarks.

Great. Thank you, operator. Thank you, everybody, for listening, for the great questions. As you can see, we're sharply focused on all of these strategic priorities for Galafold, AT-GAA, our pipeline and the financial strength of the company. So we will continue to press on onward. Thank you. Have a great day.

This concludes today's conference call. Thank you, and have a great day.