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spk09: Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics First Quarter 2022 Financial Results Conference Call and Webcast. At this time, all participants are in a listen-only mode. Later, we will conduct a question and answer session. An instruction will follow at that time. If anyone should require assistance during the conference, please press star and then zero on your touchtone telephone. As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Fonin, Executive Director of Investor Relations. You may begin.
spk10: 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 Queamy, Chief Financial Officer, and Dr. Jeff Costelli, Chief Development Officer. Joining for Q&A will have Dr. Mitchell Coleman, Chief Medical Officer, and Sebastian Martel, Chief Business Officer. As referenced on slide two, we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as well as our plans and prospects. Our forward-looking statements should not be regarded as representation by us that any 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?
spk05: 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 three primary objectives. One, continue to advance Gallifold to as many patients in as many geographies as possible. Two, secure approvals for and launch ATGAA and 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, Gellifold 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 Gallifold 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 Gallifold has been strengthened by the issuance of eight new patents this year. Together, there are now 35 Orange Book listed issued patents related to Gallifold 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 Gallifold in 2022, And with this strengthened IP protection, believe that it has a long runway into the next decade and beyond, and that Gallipold 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 ATGAA, 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 ATGAA's approvability and its potential to become the next standard of care in Pompe disease. We continue to expect a manufacturing inspection at the WUSHI facility for the biologic ATB200 again, the biologic component of ATGAA, 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 WUSHI have completed all preparatory work, including internal mock inspections, and are fully prepared. Today, we continue to expect the agency 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 Applications, or MAA, for ATGAA. 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 WUSHI manufacturing site. Our global launch plans continue to accelerate, including pre-launch 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 US 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 Gallifold and prepare for the global launch of ATGAA. 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 Infebre 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 ATGAA, and by making judicious strategic investments in next-generation therapies Infebre and Pompe and in core science and platform technologies in the field of genetic medicine. We are rapidly approaching two pivotal inflection points for amicus. First, the global launch of a second rare disease medicine with ATGAA. And secondly, profitability. These are feats that we believe will enable amicus to truly enter the upper echelon of biotechnology companies. Ahead on slide six, 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 Gallifold to more people living with Febre disease with a minimal variance in existing and new markets. We look to achieve double-digit global product revenue growth of 15 to 20 percent 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 ATGAA regulatory filings and initiating the anticipated launch of ATGAA 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 ATGAA. 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 Gallifold performance. Bradley.
spk03: Great. Thanks, John. Good morning, everyone. Let me start by walking you through in more detail our Galfold performance for the quarter. On slide seven, 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 eight, 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 two graphs depicted on this slide, Q1 results follow the revenue pattern we've continued to observe, where due to a variety of factors, including timing of orders as well as pay reauthorizations 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 Gallifold 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, US, UK, 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 on 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 Gallifold generally stay on Gallifold. And importantly, the value of Gallifold 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. a relentless focus remains on ensuring access to Gallifold for anyone who needs it. Within the table on the right side of the slide, we've provided a three-year historical snapshot of the average proportion of Gallifold sales that occur each quarter during a given year, and we expect a similar trend to occur this year. Now moving to slide nine, what we've seen so far this year is that Gallifold 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 in 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's 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 Gallifold has the potential to surpass $500 million in annual revenue over the next few years through three primary growth drivers that we continue to talk about. Continuing to penetrate into existing markets, continued to expand into new geographies and broadening the labels for Gallifold. In the longer term, we remain confident in that billion-dollar 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 IT coverage into the late 2030s, 19 of which provide protection through 2038, including the latest Composition of Matter patent, all of which give us opportunity to provide access to Gallifold globally for many years to come. With that, let me turn the call now over to Dr. Jeff Costelli, our Chief Development Officer, to highlight our ongoing GALIFOLD initiatives across our clinical regulatory medical teams as well as our APGAA program. Jeff?
spk04: 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 GALIFOLD. We continue to look to broaden the global labels within the adolescent 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 and metabolism reports, as well as presented at the 2022 World Symposium in February. These long-term data highlighted stable renal function during treatment up to eight and a half years on Gallifold, 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 Fibrate outcomes measured by rates of kidney, cardiac, and CNS events, which we believe compare favorably to similar studies in the medical literature. Third, Amicus has met 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 Gallipol 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 4 studies that we continue to move forward. And last, as Riley 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 Gallifold as an important driver of the long-term success. Moving on to slide 13 in our ATGA 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 and 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 endpoint from our Phase III study. As a reminder, PROPEL was a double-blind randomized study assessing the efficacy and safety of ATGA in both treatment-naive and ERT-experienced patients against the approved therapy, aglucosidase-alpha. PROPEL is the only controlled trial to date that's 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 two most recognized endpoints in Pompeii, six-minute walk distance and FDC, shown here on the slide, favored ATGA over aglucosidase alpha in the overall population. We believe this consistency of effect across the key disease manifestations of Pompeii illustrates the potential impact of ATGA for patients. On slide 15, we call out the ERT experience population. where 95 participants were in the standard of care for more than seven and a half years on average. Generally, this is associated with the decline phase of disease for most patients. And we actually saw an increase in six-minute walk in these patients and stabilization in their FEC, which achieved nominal statistical superiority on both endpoints versus agglucosidase alpha 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 ATGA, recently at the 2022 NDA Clinical and Scientific Conference a few months ago, the amicus team presented positive long-term data from the Phase 1-2 study of ATGA. 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 three years. The results have been shared with the global regulatory authorities in the US 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 ATGA 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 ATGA program. First, as John mentioned, on the regulatory progress, last year the U.S. FDA accepted for review the BLA for sipaglucosidase-alpha and the NDA for miglisat, the two components of ATGA. And the agency set a PDUCE action date of May 29, 2022 for the NDA and 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 WUSHI 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 MEA 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 has indicated in writing that based on prior manufacturing inspections of this facility, An inspection is not required for ATGA at WUSHI to support the MAA. We are now very excited to also have multiple expanded access programs in place globally, including in the UK, Germany, Japan, and other countries. This includes the EAMS framework that we announced in June, in which ATGA 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 needs faced by the Pompe community and permits eligible adults living with late onset Pompe disease who have received Alka-Costas Alpha for at least two years to switch to ATGA, granting access prior to marketing in the UK. We're seeing significant enthusiasm for ATGA under the EMS mechanism with multiple physicians having requested access across all the leading Pompe centers in the UK. and multiple patients now receiving this novel two-component treatment. Since the positive scientific opinion, interest, and momentum for ATGA 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 ATGA 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 ATGA. Brad?
spk03: Sorry about that. Thanks, Jeff. Just on slide 18, as Jeff mentioned, as we talk through our launch preparations for ATGA, we are poised for another successful product launch building on the successes of our Gallifold franchise, although unlike when we launched Gallifold, when we were hiring the commercial organization and supportive 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 will launch ATGAA 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 ATGA to people living with Pompe disease around the world. From the team, the medical education, the now-published Phase III data, and 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 very strong position for a second rapid and successful launch for Amicus. And with that, let me turn the call now over to Daphne Queeney, our Chief Financial Officer, to review our financial results, guidance, and outlook. Daphne.
spk11: 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 Gallifold 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 Bradley highlighted, given 69% of Gallifold revenue is generated outside the US, we see significant FX exposure to our reported revenue numbers. Applying average April 2022 exchange rates, the FX impact on 2022 full year Gallifold 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 gap 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 ATGAA, 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 30 cents per share, as compared to a net loss of 65.7 million, or 25 cents 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 Gallifold launch and ATGAA clinical studies and pre-launch activities. As we highlighted previously, this guidance also includes an approximate $70 million related to certain non-recurring costs for the manufacturing of ATGAA to 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 Gallifold, securing approvals and launching ATGAA 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 Gallifold 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.
spk05: 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.
spk09: Thank you. Ladies and gentlemen, if you have questions, please press star and then the number one key on your touchtone telephone. At this time, we ask that you only ask one question. If you have any additional questions, please enter back into the queue. If your question has been answered or you wish to remove yourself from the queue, please press the pound key. Thank you. Your first question comes from from . Your line is now open.
spk14: Good morning, everyone. Thanks for taking the question. John, since you mentioned that labeling discussions for ACGAA have already started, can you talk to, you know, the tenor the tenor of those discussions around the breadth of the label, and then, you know, as you're having those discussions overall, including on manufacturing, I guess, what's negotiable on that WUSHI 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? Thanks.
spk05: Sure, Ritu. Good morning. Thank you. So I'll take both parts of those. With respect to the labeling 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' interests 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 spirited discussions, and again, we're very confident in the approvability of ATGAA with the label that will support extraordinary success for patients and for the product commercially. You know, 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 29th would be something that would be able to be achieved in this timeframe.
spk14: Great. Thanks.
spk05: Sure, Ritu. Thank you.
spk09: Your next question comes from Ani Panramah. of JP Morgan, you may now state your question.
spk06: Hey, guys. Thanks so much for taking the question. 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? Thanks so much.
spk05: Sure, thanks. I won't comment on any ongoing discussions with the FDA. You're correct, Anupam, 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 three or four 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 29th 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.
spk06: Thanks so much for taking our question.
spk05: Of course, Anupam. Thank you.
spk09: Our next question comes from Ellie Merle from UBS. Your line is open.
spk12: Hey, guys. Thanks for taking the question. Maybe just on sort of Pompeii launch preparation, maybe first if you could give, you know, the latest color in terms of how the EAP is going in the UK 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 Neo-GAA and how you're thinking about sort of uptake in the ERT switch patients as well as potentially, you know, switches from Neo-GAA and any initial thoughts there. Thanks.
spk05: Sure. Thank you, Ellie. Good morning. Bradley, do you want to handle both of those on the EAMS and EAP as well as our launch expectations in the United States?
spk03: Sure. Thanks, John. Thanks, Kelly, for the question. So first for expanded access, yeah, 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 the Early Access Medicine Scheme in the UK. And that's important because both ATGA and Neo-GAA are available through that scheme. And what we've heard is very positive feedback towards ATGAA. We know that all of the key centers that prescribe Pompe medication have asked for and are now receiving the EAMS, sorry, access to ATGA through the EAMS program. So that's, I think, really important to see how much support and excitement there is for ATGA through that program. We would expect that number to continue to climb, and the U.K. is actually a pretty significant Pompe program. market and so we think that's a great opportunity for patients to get access to ATGA while the UK is reviewing the application and obviously gives us a great view into how the community sees ATGA 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 ATGA, but if you just focus on the two segments you talked about, experienced patients who are on myozyme and experienced patients who perhaps have gone to NeoGA, from our perspective, I think, you know, any Pompe patient who falls within the label would be indicated for APGA. We believe that the data show that our product has significant improvement, in particular in switch patients, and would encourage folks to re-familiarize themselves with the longer-term data from the studies on the OGAA in 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 ATJ could have utility in both of those populations. And we think the data are our friend there. And you'll see over time that we have the chance to be, we think, standard of care in all Pompe patients.
spk12: Great. Thanks.
spk09: Your next question comes from Joe Schwartz from SVB Securities. Your line is now open.
spk07: Hi, everyone. This is Beth Feinscott 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. Thanks.
spk05: Great, thanks, Beth, and good morning. 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?
spk04: Sure, yeah. You know, 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 involved longer term. Just for whatever reason, these patients show some potential initial mixed 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 weaning of effect. And I think what was really exciting about that Phase I-II data that we just presented was, you know, after three years, those improvements we saw in those Phase I-II patients have been maintained. And, again, that was in switch patients, naive patients. So we were excited by that data. You know, we got very good reception to that data as well. And then, yeah, this year we will continue to, you know, we have patients we've been following now in that Phase I-II that are now on the five-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. You know, the patients after one year continuing on ITGA or that lumagime arm that switched to ATGA, you know, we'll have at least one year of data in all of those patients. We don't have specific, you know, dates and conferences we can announce yet, but we do plan to have that data disclosed here this year. But we don't have necessarily obligations to include that data in the submissions in the U.S. or the EU. You know, at some point, you know, we may add that longer-term data, but for the initial review and approval, we don't need to add additional data.
spk05: Great. Thanks, Jeff. Thank you, Beth.
spk09: Your next question comes from Kristen Kluska from Cantor Fitzgerald. Your line is now open. Hi.
spk02: Good morning, everybody. Thanks for taking my question. I wanted to ask, outside of all the additional findings that you've presented and published on ATGAA since the Propel readout, what else gives you confidence heading into this decision, but then also in the role that ATGAA could place in the market? So, for example, thoughts around the agency commentary on the unmet need to the EAMS, next by the label, 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 FDC and the understanding around mortality risk.
spk05: Great. Thank you, Kristen. Good morning. And actually made our job easier by highlighting a number of the different elements that we think will go into making this a differentiated presentation. medicine for people. Again, I'll just highlight that, you know, 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, lumozyme. They had switched to ATGAA. And again, the vast majority of people who we see taking ATGAA for the first number of years there are going to be people who are currently on enzyme replacement therapy, whether that be myozyme or lumozyme 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 six-minute walk, on forced vital capacity, and virtually all secondary endpoints, You know, this is a very sophisticated patient community and physician scientist community, and they will be data-driven. So they'll look at the data from that study, and again, they'll 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 of 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 in 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 ATGAA 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 important we think, positive for ATGAA 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 ATGAA. Jeff, I don't know if you've got any other comments to add.
spk04: No, I think, John, you covered it well. It's really just that consistency of the fact across parameters that we measured and patients that we treated, you know, all the different endpoints across the key manifestations, either nominally significantly or numerically favored, you know, ATG over aglucosidase alpha. And then across naive patients, experienced patients, non-ambulatory patients from phase 1 to patients with higher or lower baseline severities, We've just seen a really consistent effect, and I think that's really the key, that that consistency adds into the weight and the meaningfulness of the treatment.
spk05: No, it's an important point, Jeff, too. And again, Kristen, I'd probably summarize it that when you look across all the data that we've had in now more than six 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 a billion dollars. And with increased diagnosis rates and the ability to affect and positively affect, 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. You know, we think this is a market with the 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, you know, when you step back then and look at amicus with Gallifold as 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, you know, with a billion-dollar revenue potential for Gallifold with what we believe will be an approved product in Pompe disease that we think has the potential to become the standard of care for at least split the market with the existing entrenched other competitor in the field. You know, that's another potential billion-dollar revenue product, also with significant intellectual property protection, including composition of matter on ATGAA, extending well into the 2030s. That, combined with the financial strength of the company, we think is pretty distinguished, particularly in this volatile market.
spk02: Thank you, John. Thank you, Kristen.
spk09: Our next question comes from . You may now state your question.
spk08: Hey, good morning, guys. Thanks for taking our question. On the labeling discussion, I was kind of curious, you know, in terms of differentiating ATGAA 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. and perhaps that could accentuate some of the profiles that you were just alluding to. Thank you very much.
spk05: Yeah, thanks, Dagan. 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 1-2 study, but they've not asked, for instance, for the Propel study for any additional data cuts.
spk08: Thank you.
spk05: Thank you.
spk09: Your next question comes from Salveen Richter of Goldman Sachs. Your line is now open.
spk13: Hey, guys. Thanks for taking the question. This is Elizabeth on for Salveen. Maybe going back to some of the initial questions, how do you expect to message around the WUJI 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. Thank you.
spk05: 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 29th from the agency, and we expect both the NDA and the BLA to We expect action by July 29th, 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 we've had tremendous manufacturing history here with WUSHI, 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. You know, 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 WUSHI 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, you know, 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 inspections. You know, 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 29th 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 competent in the approvability of ATGAA 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.
spk13: Great. Thanks for the color.
spk05: Great. Thank you, Elizabeth.
spk09: Your next question comes from Yoon Zong of BTIG. Your line is now open.
spk01: Hi. Good morning. Thanks very much for taking the questions. 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 ATGA launch? And also, given that it's still an IV infusion, not like the oral for Gallifo, Do you expect that you still have to face some type of COVID-related challenges when you launch ATGA?
spk05: Yeah, sure, Bradley. I'll go ahead and take that over to you, particularly to highlight the operating leverage that we have at Amicus.
spk03: Yeah, thanks, John. So just as a reminder, what we've characterized is we have about 150 50 global field personnel between sales, medical, market access, et cetera. That organization is effectively the organization we'll use to launch ATGA, so we get significant leverage out of the Gallifold commercial organization, commercial and medical organization, I should say. What we said is we would intend to add perhaps less than a dozen SDEs globally to support the launch. primarily in direct marketing, uh, direct medical, and then in the United States, some market market access folks in particular in support of our patient assistance hub. So highly levered, leverageable, uh, organization to be able to support the launch of APGA in terms of your second question as relates to COVID impacting the uptake of APGA at this point, based on what we know from Gallifold, based on where we see, the global market's evolving, we don't expect any significant impact on ATGA 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 significant impact by COVID on our ATGA launch.
spk04: Great. Thank you.
spk09: At this time, I would now turn the conference back to Mr. John Crawley, Chairman and CEO, for closing remarks.
spk05: 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 Gallifold, ATGAA, our pipeline, and the financial strength for the company. So we will continue to press on, onward. Thank you. Have a great day.
spk09: This concludes today's conference call. Thank you and have a great day.
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