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spk07: Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Third 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, and instructions will follow at that time. 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.
spk01: Thank you, Shannon. Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics third quarter 2022 financial results and corporate highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer, Stephanie Creamy, Chief Financial Officer, Sebastian Martel, Chief Business Officer, and Dr. Jessica Stelly, Chief Development Officer. Joining for Q&A is Dr. Mitchell Goldman, Chief Medical Officer, and Ellen Rosenberg, Chief Legal 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 of our plans will be achieved. Any or all of the forward-looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. We are cautioned not to place undue reliance on any forward-looking statements which speak only to the dates 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 quarterly report on Form 10-Q for the quarter-ended September 30, 2022, filed this morning with the Securities and Exchange Commission. At this time, it's my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Bradley.
spk08: Great. Thank you, Andrew, and welcome, everybody, to our third quarter 2022 results conference call. I'm really pleased today to review the continued progress made across our business this year. And as we did in this morning's press release, let me highlight several key accomplishments for the quarter. First and foremost, Gallifold continues its strong performance and remains the cornerstone of our success. We continue to be very pleased with the uptake of Gallifold globally, despite significant foreign currency headwinds we've seen this year. And the first three quarters of the year represented 8% reported revenue growth or 16% on an operational basis. In the third quarter, our growth and key performance indicators are meeting our objectives in all geographies. And as Sebastian will highlight, we continue to see strong trends in a number of our metrics, including new patient starts and in-person visits between our field team and our physicians. So we were reiterating our guidance that 15 to 20% growth in Gallifold revenues and constant exchange rates. We're also pleased to announce that the Gallifold intellectual property estate continues to grow this quarter. This year, we've strengthened our IP estate through the issuance of 19 new patents. There are now 46 orange book listed issued patents related to Gallifold in the United States, 30 of which provide protection to 2038 and beyond. and importantly includes five composition of matter patents. We see this patent portfolio, along with our orphan drug exclusivities, providing broad and long-term intellectual property rights well into the late 2030s with its novel precision medicine. As we've discussed throughout the year and as anticipated, we saw three abbreviated new drug application or ANDA filers and have received their paragraph four certifications. Given our strong and innovative intellectual property estate, we believe we are well positioned to address any potential generic challenges to our patents. And we announced this morning, later today, we intend to file infringement lawsuits against three antifilers who are requesting approval to market a generic version of Gallifold. We intend to continue to protect and enforce our broad intellectual property rights. And looking ahead, we expect continued growth for Gallifold this year and remain confident that with our strong IP protection, it has a long runway well into the next decade. Second, we continue to make progress on our global regulatory filings and commercial planning for ATGAA, our novel next-generation therapy for Pompe disease. As we just announced last week, the U.S. Food and Drug Administration recently deferred action on the biologics licensing application, or BLA, for sipaglucosidase alpha, the biological component of ATGA, which we also call ATB200. The agency cited that due to restrictions on travel related to COVID-19, it was unable to conduct the required inspection of the Wuxi Biologics manufacturing site in China during the review cycle and is deferring action in the application until the inspection is complete. That being said, the good news is that per FDA guidance, a deferral in such circumstances can only be given, quote, provided that no deficiencies have been identified and the application otherwise satisfies the requirements for approval, end quote. So as we've been saying, we believe this is a matter of when, not if, APGA is approved. Additionally, the company is now actively engaged in the FDA on this step, and at the agency's direction, the company has requested a Type A meeting with the Office of Pharmaceutical Quality to discuss and agree upon plans and logistics for the pre-approval inspections. We at Amicus, along with our manufacturing partner, Wuxi Biologics, are ready for the inspection of the manufacturing facility in China. And as soon as we have more clarity, we will provide an update on when we might expect an approval in the United States. As a reminder, we continue to expect the two components of APGA will be approved together. Importantly, in Europe, we are very far along now in the review by the EMA on the marketing authorization application for APGA. To provide a little more color there, We are announcing today that we are included now in the agenda for an oral explanation in November. And thus, the CHMP opinion is expected at the December meeting, setting us up for a commercial launch in 2023. As a reminder, the EMA has indicated that it does not require an inspection of the WUSHI manufacturing site as a condition of their approval. We're also extremely pleased with the level of interest and participation we're seeing in our expanded access programs globally. We now have programs in place in the United States, United Kingdom, France, Germany, and Japan, with a growing number of patients participating in each. In fact, across all of our ongoing clinical studies and access programs, there are now nearly 190 patients on ATGA today, which we believe represents more than 5% of the total treated Pompe patients around the world. We expect this number to continue to grow as we approach approvals in the United States and Europe. In anticipation of these approvals in the near term, our global launch plans continue to move ahead, including our pre-launch activities, targeted investments, and a handful of additional personnel to support the launch and ongoing investments in building launch inventory. We are close now to reaching this much anticipated milestone of ATGA getting across the regulatory finish line and providing another treatment option for people living with Pompe disease, both in the United States and in Europe, and with further regulatory applications planned in the months ahead. And third, Amicus has maintained a strong financial position as we continue to execute on the global expansion of Gallifold and prepare for the global launch of ATGA. Despite all the headwinds with FX and the delays with ATGA approval in the United States, we will continue on our path to profitability. Our goal is to achieve non-GAAP profitability in the second half of 2023. This is dependent on a number of factors, including the timing of approvals and the launch of ATGA, But based on the current operating plans and projections, we believe this will happen in the second half next year. Additionally, today we entered into an at-the-market or ATM equity offering of up to $250 million. We see this as good financial housekeeping measure, and we have no present intention to use the ATM this year. In 2023 and beyond, we will be judicious about any use of the ATM, and any proceeds will go towards ensuring continued commercial expansion of Gallifold and maximizing anticipated launches of APGA. I'll emphasize that we're sharply focused on our three primary objectives. Number one, continuing to advance Gallifold to as many patients in as many geographies as possible. Number two, securing approvals for and launch of APGA globally. And number three, ensuring the financial strength of Amicus. On slide five, we see that we're well on our way towards achieving our key strategic priorities for this year. including number one, continuing to drive Gallifold to more people living with febrile disease with amenable variants and existing in new markets. As I mentioned, we're on track to achieve our guidance of double digit global product revenue growth of 15 to 20% at constant exchange rates. This reflects the strong momentum and demand behind this precision medicine globally. We remain steadfast in our commitment to advancing APGA regulatory approvals and the anticipated launch of APGA, leveraging our seasoned global commercial, medical, and market access teams and our experience across all areas needed for a successful and effective drug launch, we are fully prepared for and anticipate a successful launch at ATGA. We continue to judiciously invest in the advancement of our best-in-class next-generation genetic medicines and capabilities, as well as our next-generation chaperone for Fabry disease. And again, we'll continue to maintain a strong financial position as we carefully manage our expenses and our investments in the business. With that, let me now hand the call over to Sebastian Martel, our Chief Business Officer, who'll give further highlights on the Gallifold performance for the quarter.
spk12: Sebastian? Thank you, Bradley. So good morning to everyone on the call. I will start by providing you with more details on our Gallifold performance for the quarter. On slide seven, for the third quarter of 2022, Gallifold reported revenue reached $81.6 million, driven by strong new patient accruals partly upset by significant foreign currency headwinds. The geographic breakdown of revenue during the quarter consisted of $51 million, or 63% of revenue, generated outside of the US, and the remaining $30 million, or 37%, coming from within the US. When ignoring FX impact, this is in line with a two-thirds to one-third split that we expect as we continue to grow both parts of the business. It leads to see continued strong patient growth in countries like the USA, Japan, Canada, Spain, Portugal, and Poland, just to name a few. Turning to slide eight, our results in the first nine months of the year highlight the strength of our global commercial efforts. The business continues to be incredibly resilient with patients added in all major markets and an operational growth rate of 16.2% over the same period in 2021 at constant exchange rate. the negative impact on foreign currency was 8.3% in the period. As a result, GAFO reported revenue growth with 7.9% in the first three quarters of the year. To add a bit of color on our geographical performance, in the first nine months of the year, our sales in the US grew 16.8%, while our ex-US sales grew 16% at constant exchange rates. On a year-to-year basis, GalaFold continues to be the fastest-growing product in 2022 for Fabry disease globally and the greatest contributor to the global Fabry market growth. I'm pleased to report that our monthly net patient trends continue to show positive signals. Indeed, the three-month trend is the highest in the last two years. And if you look at the growth in net patients on GalaFold globally, which is perhaps the 12th measure of the underlying business, we see greater than 19% growth in patients from GalaFold at the end of Q3 this year versus the same period last year. All indications of the continued and growing demand for GalaFold. We ended the third quarter with about half of the global market share of treated amenable patients. And while the global mix remains about 55% switch and 45% naive in many geographies, we're seeing a stronger uptake in naive populations. So while we are achieving high market shares in countries where we've been, of course, the longest, there's plenty of opportunity still to continue to switch patients over to Galapagos and continue to grow the market as we penetrate into the diagnosed and treated, as well as the newly diagnosed segments. All of that is underpinned by the impressive compliance and adherence rate that we continue to see exceeding 90%, reiterating our belief that those patients who go on Galapagos generally stay on Gallifold. We continue to expect nonlinear quarterly growth due to uneven ordering patterns and ethics fluctuations. Importantly, the value of Gallifold continues to be recognized by payers as we have a very strong track record of successfully negotiating and renegotiating reimbursement outside of the US. Our relentless commitment remains on ensuring access to Gallifold for anyone who needs it. On slide nine, What we've seen so far this year is that Gallup Overtake continues to track very well. And we're seeing growth across all our major markets, as well as most of our smaller markets. We're trying to achieve a full year revenue guidance of 15 to 20% growth at constant exchange rates. Altogether, we view this as a great place to be three quarters of the way through the year. Moving to slide 10. We know that GalaFold has the potential to surpass $500 million in nano-revenue over the next few years through three key growth drivers. First, continuing to penetrate into existing markets. Second, expanding into new geographies. And third, broadening the label. I'm pleased to share that we're making continued progress on expanding into new markets. Just to name a few examples, we recently received marketing authorization in Turkey We've submitted the marketing authorization application in Hong Kong earlier this year, and we're about to submit in New Zealand. We've also successfully renegotiated pricing and reimbursement agreements recently for Gala Falls in Poland and Spain. In the longer term, we continue to see significant growth in the fabric market globally, driven by diagnosis of patients through a variety of measures, including high-risk screenings, newborn screening, and other diagnostic initiatives. which we continue to support and invest in as well. And finally, we have orphan exclusivity in the U.S. and Europe, in addition to our now 46 orange book listed patents that give us IP coverage into the late 2030s, 30 of which provide protections to 2038 and beyond, including five composition of matter patents, all of which gives us opportunity to provide access to GalaFold globally for a long time to come. Moving on to ATGA, on slide 11, we outline launch preparations as we are poised for another successful product launch. Unlike when we launched GalaFold, where we were hiring and building the commercial infrastructure from scratch, we now have a presence in over 40 countries around the world, including all the major markets. That team, that same team, will be largely the one involved in launching ATGA, with only a handful of new FTEs needed. We have experience. across all areas that are needed for a successful drug launch, in regulatory, commercial, supply chain, experience with payers, reimbursement and access, and in addition, and perhaps most importantly, we have key relationships with physicians. We're very confident in our world-class organization, and we can leverage their experience and relationships to deliver ATGA to people living with Pompe disease around the world. From the team, the medical education, the published phase three data, and the highly regarded Lancet Neurology Journal, our experience with reimbursement and access around the world, and again, all the strategic planning that we're doing together with building inventory with our partners at Uchi Biologics, we believe we're in a very strong position for a second successful launch for Amicus. With that, let me now hand the call over to Dr. Jessica Philly, our Chief Development Officer to highlight our ATGA program and pipeline updates.
spk10: Thank you, Sebastian, and good morning, everyone. On slide 13, we'll start with our ATGA program. Pompe is a severe and fatal neuromuscular disease and one of the most prevalent lysosomal disorders, and we recognize that Pompe poses a range of health challenges for people affected by the disease, and having therapeutic choices is crucial. Multiple publications in natural history studies highlight the initial benefits of treatment generally being followed by continued long-term decline for many individuals. On slide 14, we present a summary of the primary and key secondary endpoints from our phase three study. As a reminder, PROPEL was a double-blind, randomized study assessing the efficacy and safety of ATGA in adult treatment naive and ERT-experienced participants with late-onset Pompe disease, or LOPD. against the approved therapy, Alglucosidase Alpha. Propel is the only controlled clinical trial to date that included both the ERT-experienced patients and ERT-naive, with the experienced patients representing one of the sets of patients with the greatest clinical unmet needs. Endpoints across motor function, muscle strength, pulmonary function, patient-reported outcomes, and biomarkers, including the two most recognized endpoints in Pompe, six-minute walk distance and FDC, shown here in the slide, favored ATGA over adlucosidase alpha in the overall population. We believe this consistency of effect across the key disease manifestations of Pompe illustrates the potential impact of ATGA for patients. Additionally, in the ERT experience population, where 95 participants were on the standard of care for more than seven and a half years on average, generally associated with continued progression for most patients at this point in treatment, we actually saw an increase in six-minute walk distance and stabilization in FTC after switching to ATGA, which achieved nominal statistical superiority on both endpoints versus the current treatment and showed a clinically meaningful outcome never before seen in this population. Moving to slide 15, as part of the growing body of evidence supporting ATGA, the AMICUS team presented additional positive long-term data from the Phase 1-2 study of ATGA at the 2022 World Muscle Society Conference. As seen here in the slide, these latest data continue to represent very meaningful and durable improvements in functional outcomes, as well as persistent reductions in key biomarkers of muscle damage and disease substrate. Compared with what is known about the natural history of both untreated and ERT-experienced Pompe patients, the observed durable improvements give great hope that ATGA has potential to become the new global standard of care for people living with LOPD. On slide 16, we show key results from an indirect treatment comparison of Pompe ERTs that was also recently presented at the World Muscle Society Conference. This analysis used published data from the Phase I-II and Phase III studies for the three ERTs currently available or under regulatory review for late-onset Pompe. A multi-level network meta-regression accounting for effects of study-level covariates was performed using individual patient-level data from the Propel study and available aggregate data from the other studies. The forest plots shown here on the slide represent the estimated relative effects in 95% credible intervals of each treatment comparison in the base case analysis in which all covariates were set to the target population of the Propel trial. These results suggest ciproquacacidase plus miglistat may potentially have a differentiated clinical profile versus the other ERTs, particularly for individuals with some level of previous ERT treatment. On slide 17, we have highlighted key updates on the ATGA program. First, on the regulatory progress, as shared previously, last year, the U.S. FDA accepted for review the BLA for sipic glucosidase-alpha and the NDA for miglostat, the two components of ATGA. And as Bradley summarized earlier, following the recent FDA deferred action, which was due solely to COVID-related inspection delays, the company is now actively engaged with the FDA and at the agency's direction has requested a Type A meeting with the Office of Pharmaceutical Quality to develop plans and logistics for the pre-approval inspection. We continue to expect that the two components of ACGA will be approved together And once we have more clarity, we'll be able to get more color on estimated approval timing. We've also shared previously that the MAA has been submitted to the European Medicines Agency and is now in the later stages of review. Following an upcoming oral explanation in November, the CHMP opinion is expected at the December meeting. Of note, the EMA has indicated in writing that based on the extensive and prior manufacturing inspections of the WUSHI facility, that an inspection is not required prior to ATGA approval. We now have multiple expanded access programs in place, including in the US, UK, Germany, France, Japan, and other countries. This includes the EAMS framework, of which we have previously announced that ATGA was granted a positive scientific opinion through the early access to medicine scheme by the UK's MHRA. We are seeing significant enthusiasm for ATGA under the EAMS mechanism, with multiple physicians having requested access across the leading Pompe centers in the UK and dozens of patients now receiving ATGA through this program. With this growth in our access programs, as Bradley noted, we are pleased to report that approximately 190 patients worldwide are now being treated with ATGA across 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 up to 18 years of age living with LOPD and expect to expand into patients with infantile onset Pompe disease later this year. Importantly, in response to the many requests for treatment that we continue to receive for children living with LOPD and IOPD, our expanded access programs continue to increase. With that, I would like now to turn the call over to Daphne Queenie, our Chief Financial Officer, to review our financial results, guidance, and outlook. Daphne?
spk04: Thank you, Jeff, and good morning, everyone. Our financial overview begins on slide 20 with an overview of our third quarter revenue performance and FX impacts. For the third quarter, we achieved total revenue of $81.7 million, which is a 3% increase over the same period in 2021. This includes operational revenue growth of 14% offset by a negative currency impact of 11%. Given a majority of Gallifold revenue is generated outside the U.S., we see significant FX exposure to our reported revenue numbers. The Euro, British Pound, and Japanese Yen are the currencies we are most exposed to, and on a year-to-date basis, these have declined 14%, 18%, and 20%, respectively. Applying average October 2022 exchange rates, the FX impact on 2022 full-year Gallifold reported sales would be a negative impact of approximately 9% or $28.5 million. Slide 21 outlines our income statement for the third quarter ending September 30, 2022. Cost of goods sold is a percentage of net sales with 16% in the quarter as compared to 15% for the prior year period. Total gap operating expenses were 102.1 million in the third quarter as compared to 110.2 million in the third quarter of 2021. The decrease reflects the reprioritization of the gene therapy portfolio. On a non-gap basis, total operating expenses were 85.5 million in the third quarter as compared to 93.6 million in the third quarter of 2021. We define non-GAAP operating expense as research and development, 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 third quarter of 2022 was 33.3 million, or 12 cents per share, as compared to a net loss of 50.3 million, or 19 cents per share, for the prior year period. Driven by the revenue growth of Gallifold and expense management, we continue to make progress towards our path to profitability in the second half of next year. At September 30, 2022, we had approximately 281 million shares outstanding. We are updating our full year 2022 non-GAAP operating expense guidance from $470 million to $485 million to $430 million to $440 million. driven by prudent expense management while maintaining ATGAA manufacturing and prelaunch activities. Importantly, in 2023 and beyond, we continue to expect non-GAAP operating expense levels to decline the low levels we saw in 2021. Turning now to slide 22, we continue to operate from a position of financial strength, and our goal remains to achieve non-GAAP profitability in the second half of 2023. as defined in our press release. Profitability is dependent on a number of factors, including the timing of approvals and launch of ATGAA. 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. As a good financial housekeeping measure, today we entered into an at-the-market or ATM equity program of up to $250 million. We have no current intention to use the ATM this year. In 2023 and beyond, we will be strategic and judicious about any use of the ATM. A few comments about our cash position and 2022 financial guidance. Cash, cash equivalents, and marketable securities were $354.7 million at September 30, 2022, compared to $482.5 million at December 31, 2021. Our full-year revenue, GalaFold revenue guidance is $350 million to $365 million at constant foreign currency exchange rates, in addition to our non-GAAP operating expense guidance of $430 million to $440 million. And with that, let me turn the call back to Bradley for closing remarks.
spk08: Great. Thanks, Daphne, Jeff, Sebastian, for highlighting all the great progress of the quarter. Thanks to everybody at Amicus and all of our employees around the world who work so tirelessly for people living with rare diseases. With that, operator, we can now open the call to questions.
spk07: Ladies and gentlemen, if you have a question, please press star and then 1-1 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. Your first question comes from the line of Ritu Baral with Cowan. Your line is now open.
spk03: Good morning, guys. Thanks for taking the question. Good morning, Ritu. Good morning. It's basically on the timeline of ATGAA approval. I guess you outlined today that you're going to request a Type A meeting. Can you tell us when you plan on requesting the type A meeting? Do you need to put a briefing book like usual together in advance, or can this be done more ad hoc and what those timelines might be? And then I have a quick follow-up.
spk08: Sure. Great question. So the request actually has already gone in, so that's already been submitted, and that was at the direction of the agency. I think that's important information. This is really the appropriate vehicle for us to have formal conversations, and we were careful to highlight it with the Office of Pharmaceutical Quality. You know, we had talked when we put the press release out last week about why we felt confident this was really an active step to get to a solution around conducting the inspection. I think having this formal vehicle really helps put a fine point on that. In terms of when we might have an update, and then I'll come to your next question, In terms of when we might have an update, we still hope to have an update in the coming weeks. And as I mentioned last week, as soon as we have more clarity, we can provide some expectations on when we might see an approval. But again, we think this is a very important step that we're having this discussion and the request indeed already went into the agency.
spk03: Got it. And my quick follow-up is on Europe. Have you responded to the 180-day questions and were there any surprises or differential content from U.S. review issues for the EMA review. Thanks.
spk08: Yeah, thanks, Ritu. Jeff, do you just want to talk to the progress we're making in Europe and where we are with the oral explanation and the expectation for December?
spk10: Yeah, thanks, Brian. Thanks, Ritu. So we are in very late stage review with CHMP. There have been no, I would say, surprises in terms of the remaining Topics being discussed, the oil explanation coming up here shortly, and then we still have a lot of confidence that we have an approvable filing, and we expect that opinion at the December meeting. But very consistent so far with a lot of the U.S. review and questions.
spk03: Great.
spk07: Thanks. Thank you. Our next question comes from the line of Anupam Rama with J.P. Morgan. Your line is now open.
spk15: Hey, guys. Thanks so much for taking the question. If I remember correctly, you guys do provide sort of forward year guidance at an investor conference in January with the CHMP decision on the horizon. Would you be in a place to give some, you know, guidance and metrics around ATGAA, or should we just be thinking it'll be focused on Gallifold and, you know, expenses? Thanks so much.
spk08: Yeah, thanks, Ana, Tom. It's a great question. I think we will be, as you said, be able to give some color on Gallifold and expenses. Let us get a little bit more certainty around the timing of the approvals, which, as you said, we should have more clarity here relatively soon, especially in Europe and hopefully in the U.S. as well. We'll certainly give some metrics to follow in terms of the launch and the launch progress. But stay tuned on exactly what those look like. But we'll do our best to provide the street with some sense for how to make sure we're tracking the progress of the launch.
spk15: Thanks so much for taking our question.
spk08: Thanks, Anil.
spk07: Thank you. Our next question comes from the line of Joseph Schwartz with SVB Securities. Your line is now open.
spk11: Hi, thanks so much. I guess I'll ask on the inspection situation as well. I guess, what is your understanding for why the FDA has not been receptive to a hybrid inspection to this point? Is an in-person inspection the only path forward? And what plans and logistics will you propose to the FDA in order to get WUSHI, the process there, inspected? Do you have any reason to believe that they'll be receptive to any particular solutions
spk08: Yeah, thanks, Joe. So a couple things. So first of all, I can't speak on behalf of the agency, but we have said all along that despite what we believe are multiple types of inspections the agency could use to satisfy this PLI for APGA, they have always signaled to us that they would like to inspect in person. Now, whether that's in person from Maryland or in person from a hybrid team, I don't know, but they have always said that they'd like to inspect in person. Specifically, what we're working on now is exactly that, which is coming up with logistics and a plan to enable the agency to inspect the Wuxi facility in China in person. And I think the biggest difference here, you know, versus some of the commentary we've given over the course of the summer as we approached this latest PDUFA date, is that now we have active dialogue and now formal dialogue through this type A meeting request. And again, that was at the agency's direction. And so I think what's changed here is number one, we are actively discussing the logistics in particular, how to navigate the COVID situation on the ground in China. And that it was at the agency's request that we have this formal meeting and they are now actively engaged in that dialogue. So we feel, you know, a high degree of confidence that we now have an engaged discussion and that we have specifics of a plan and logistics that are being discussed. And our hope here, again, is that in the coming weeks we can finalize that and then we can provide more clarity externally on timing for when an approval might happen.
spk11: Okay, thanks. And then what are you able to do during this time where you're waiting for FDA approval to get in front of Pompeii physicians and patients in a compliant manner in order to ensure that you can really hit the ground running and not lose too much ground to competition once you're approved in the U.S.?
spk08: Yeah, I think what Jeff highlighted on the call is really the most important way to do that is really through medical education at ongoing medical conferences. So we had a great presence at the World Muscle Society, presented a host of new data there. I don't know, Jeff, do you want to remind us just the high level of the Pompe data in particular that was presented there? and then kind of the general medical education, publications, posters, abstracts, et cetera.
spk10: Yeah, thanks, Brian and I, Joe. So, you know, as I summarized briefly in the call, we've had a very active presence at World Muscle. We presented the four-year long-term data from the Phase I-II trial. We presented some new indirect treatment comparisons from available data across different ERT products. And, you know, we continue for, you know, to keep planning upcoming conferences, you know, World LDN, and then other meetings early next year to continue to have lots of new, you know, evidence presented and data presented on ATGA. And, of course, we're having appropriate disease education, and our teams are both the sales team, medical team are all, you know, ready to go with materials and messages. So we can do everything that you typically would do, you know, prior to and we're going to continue to, you know, get ready for the launch, and we are ready in many ways, but we'll be even more ready with extra time.
spk08: The only other thing I would add there, Joe, is, you know, I think this is a highly anticipated development program and regulatory process, and so the good news here is I think the community, physicians, patients, et cetera, are well aware that APGA is going through this process, and so I think there's a lot of anticipation as well in addition to the medical education activities that Jeff highlighted.
spk11: Thanks for taking my questions. Thanks, Joe.
spk07: Thank you. Our next question comes from the line of Kazin Ahmad with Bank of America. Your line is now open.
spk02: Hi, good morning. Thanks so much for taking my question. Just want to get your thoughts. I know you're not talking about the specifics of what a label language could look like, but in the event that you do get the specification about the switch data patients, How do you think that would impact your ability to gain traction against your key competitor who's been marketing really without that language? And longer term, how should we think about the split between U.S. and ex-U.S. sales for ATGAA? Thank you.
spk08: Sure. Maybe I'll turn to Sebastian in a moment on the split between the two, between the geographies in terms of the sales. But in terms of the first question around the ability for us to be successful with ATGA and what some of the label scenarios might suggest. I think the most important thing is the differentiated data set that Jeff highlighted on the call. We are the only manufacturer to have studied in an active control setting That switch population and as we've said before that makes up, you know, probably 90% of the opportunity over the next few years And so, you know I think that differentiated data set and hopefully over time the long-term data that you know We saw from the phase 1 2 study as we build that body of evidence. I think that gives us a really differentiated product especially in that switch population and for us to build on. The other thing I would remind people is that, you know, while I know we've spent a lot of time around what could be scenarios in the United States, obviously we have multiple jurisdictions that we're seeking approval in. And just because one jurisdiction has one, you know, label doesn't mean all jurisdictions will have that label. So I think it'll be really important to see how that evolves over the, as we see, you know, some of these anticipated approvals in the coming months. So I think people should watch out for that as well. And then the last thing I'll add and then turn it over to Sebastian. Again, you know, we do intend to continue to build the body of evidence in many Pompeii populations. And so there may be an opportunity down the road, of course, if for some reason you have one specific population in your label to add to that over time. and potentially expand those labels. So with that, maybe Sebastian, just talk a little bit about the distribution of the opportunity between the U.S. and some of the other major markets around the world.
spk12: Yeah, thank you, Brian. You know, there are a couple of ways to look at that. You can either look at it from a patient standpoint or look at it from a sales standpoint. So I'll start with the breakdown of sales across the main geographies. The U.S. represents about 40% of sales. Europe, about 35% of sales, and rest of the world, roughly 25%. Now, the picture is slightly different when you look at actual patients, mostly because of differences in pricing across the Antarctic Ocean. So, Europe accounts for more than 40% of patients currently treated with COMPONPE disease, while the U.S. represents around 25% of patients only. Europe represents a very large opportunity from a patient percentage standpoint.
spk00: Great. Thanks, Sudarshan.
spk07: Thank you. Our next question comes from the line of Ellie Merle with UBS. Your line is now open.
spk06: Hi, this is Sarah on for Ellie. Thanks for taking our question. I think two quick ones from us. In terms of the type A meeting with the FDA, I guess anything we know historically about, you know, now that the request is submitted, sort of how long it might take to start conversations and the duration maybe of that process. And then looking at XUS growth in Gallifolds, What are the key regions that you see driving growth in 23 and sort of maybe cadence of expanding geographically over 2023 as well? Thanks.
spk08: Sure. Thanks, Sarah. So on your first question is related to the Type A meeting. As I mentioned, the request has already gone in. So that process has already started. Our hope is that technically there's a formal clock that we can meet with them as soon as possible. And again, as soon as we have more color there, we'll provide that update. And in terms of, you know, sort of how long the process will take, again, our hope is that it can be fairly expedient. Let us get through those discussions, and as soon as we can, we'll provide more color. And then on the XUS growth opportunities for Gallifold, you know, one thing I'll just say at a high level, then maybe, Sebastian, you can provide some specific color. You know, Sebastian talked on the call how Gallifold is actually the largest contributor to February revenue growth around the world, which I think is pretty incredible given the fact that, you know, we're restricted to the amenable population. The other thing I'll say is that, you know, as we look at the numbers and as you kind of look at the metrics we've given, what we're seeing is really a continued building of momentum coming out of what was probably the, you know, depths of the COVID impact. And so as Sebastian highlighted on the call, you know, seeing the three-month net new patient numbers being the largest they've been for the last two years, I think that gives us great confidence that we are seeing kind of resumed growth and momentum, which is so important for that key franchise. But Sebastian, maybe talk a little bit about, you know, what are the geographic growth drivers for next year for Gelfold?
spk12: Yeah, so as I highlighted earlier, What we've seen this year is actually pretty much the same kind of growth rate in the U.S. and ex-U.S., so we were at 16.8% in the U.S. and 16% ex-U.S. When you start looking at, you know, specific countries, you know, we continue to see a W growth rate in countries where we've even been launched for the longest. And that's explained in those markets where we've been the longest on the market. That's explained by the growing participation we have to put naive patients on treatments. There's also countries where we've launched more recently, and there you see a much larger proportion of switches initially. But overall, we continue to see double growth rate in most markets where we're currently launched. we're seeing very strong growth in some rest of world markets. So, you know, if I take some Latin markets, for example, Colombia, Brazil, in Europe, the Polish market has also seen, you know, great expansion for GalaFold. And as I highlighted, you know, we continue to launch in or to register at least in markets where GalaFold has not been launched yet. So I mentioned specifically Turkey, Turkey is quite a sizable market from a total number of patients. The actual number of treated patients in Turkey is close to any of each of the European top five markets. There are still a small number of markets where we've got approval and are working through the pricing and reimbursement process. So these will be new launches in 2023. But again, you know, the core markets and I would say the top 10 markets continue to show very significant growth.
spk08: Great. Thanks, Sebastian.
spk07: Thank you. Our next question comes from the line of Dagon Ha with Stifel. Your line is now open.
spk14: Hey, good morning, guys. Thanks for taking the question. I'll stick with one. Just going back to ATGAA, there have been some rumblings on unverified reports of China maybe reopening, given the impact of the tech sector and that economy. I guess if you can speak to two different scenarios. One is, what's sort of your guesses to the timeline towards them reopening? And two, in a bear case where they don't, What's the real hypothetical here, given that the U.S. Chamber of Commerce was still able to inspect Wuxi in the whole zero COVID policy era? What should we be thinking about in that case? Thanks so much.
spk08: Yeah, thanks, Dagon. Two things. So first of all, I think we're hearing the same thing you're hearing, which is, you know, perhaps at the margins, things are getting better. There's still some hope that, you know, there might be a very different or more permissive policy around COVID. That being said, the plan we're working on right now actually does not require any change to the official COVID policy. So we're working on plans with the agency that would allow them to conduct the inspection in the current environment. And so, you know, maybe that is the bare case, but I think we're being conservative there. And again, the intent is that this is something that can happen within a finite amount of time. I know that the key question is exactly what that amount of time looks like, and I wish we had more color there, but we are actively working on it. Again, we feel like in the coming weeks we'll have some more line of sight there, and then we can provide a better update. But again, this is assuming that the current COVID travel and quarantine restrictions remain in place, and we're working to execute on a plan that will allow the agency to inspect in those conditions.
spk14: Great. Thanks so much. Thanks.
spk07: Thank you. Our next question comes from the line of Salveen Richter with Goldman Sachs. Your line is open.
spk05: Good morning. Thanks for taking my question. I know with regard to the launcher in Pompeii, there was a question about cadence, but could you speak to how you think the launch will play out in terms of the competitive dynamics with Sanofi and how you think patients will be triaged between the drugs?
spk08: Yeah. So, you know, first and foremost, I think from a European perspective, as Sanofi went through the review process and the appeal process around the new active substance, I think that significantly shortened the time between their launch and ours. So I think that that gives us I think good confidence that effectively we'll be launching roughly contemporaneously based on the timelines that we're on. And so that I think is a very different, excuse me, different scenario. As relates to the U.S., as we've mentioned, I think what's important about APGA is we have a very differentiated data set. And if you look at a number of the market research polls and a number of the sell side research that's been published out there, I think you do see an alignment around the populations that were studied. And so some alignment around ATGA in the switch population and the other products in the naive population. So I think that data set that Jeff highlighted on the call today is really the key for us, that we can show an improvement in patients who switch from enzyme replacement therapy on both six-minute walk and four-spinal capacity. And we think those are the data that are really going to be compelling for physicians and look forward to having the opportunity to get out there and promote the product on the other side of an approval.
spk07: Thank you. Our next question comes from the line of Yun Zong with BTIG. Your line is now open. Yun Zong, your line is open. Please check your mute button.
spk13: Hi, thank you. Sorry, I didn't hear my name. Hi, Yun. Yeah, good morning. Thank you very much for taking the question. So my question is on ATGAA. Can you remind us if the launch in Europe is going to be supported by the island side solely or is that going to be supported by Wuxi side as well? And also, what was your plan in terms of how you were going to coordinate between the two sides? And given that you had this issue on inspection, have that changed your plan in terms of how you are going to coordinate between the two sides going forward?
spk08: Yeah, great question. So just as a reminder, as you suggested, we do have the current manufacturing facility in Wuxi City in China, and that's where we've been manufacturing ATP-200, which is the biologic, throughout the course of the development. That will support the initial launch in both the United States and Europe. However, Wuxi has also now completed the build-out, and we're now actually in engineering runs for manufacturing of ATP. B200 at a site in Dundalk, Ireland. That site has actually more capacity than the site in China. And so our anticipation is that material from that site will enter into the commercial supply chain sometime in the 24 timeframe. And that was always the intention. But once that site comes on board, it's likely that it will supply the majority of product for both Europe and US and rest of world for that matter. with likely a minority of product coming out of China. So we are not too far away here from having dual site manufacturing, which is critically important, but also increasing the capacity. And the intention was for that capacity to come online into the commercial supply chain when you're starting to really get into the meat of the global launch expansion, which would again be in kind of that 24 timeline. So hopefully that gives some clarity in terms of the plans there. And it And it hasn't really changed in the context of the situation right now with the agency and the inspection.
spk13: Great. Thank you.
spk07: Thank you. Our next question comes from the line of Gil Blum with Needham & Company. Your line is now open.
spk09: Hi. Good morning, everyone. Grads on your progress. Maybe a quick one. Thank you. Do you think that any of those 190 patients on a TKA could potentially convert to commercial product once it's approved? Thank you.
spk08: Yeah, it's a great question, and the answer very much is yes. As soon as we get regulatory approvals, then we look to convert our clinical trial or expanded access patients as quickly as possible. What we've given is just kind of rough numbers in terms of the breakout of those patients. So probably about half of the 190 are in Europe with a big portion of those coming from the UK, Germany, and the EU5. The UK with the EAMS program is now the largest number of patients on ATGA today. And then likewise in the United States, we've said that 20 or so patients in the US are in our clinical trials and other ongoing access programs. And so again, there too, that would be a population we would look to switch very quickly. With Gallifold, the goal was to be able to switch patients from either development drug or expanded access drug within 90 days. And we would have a similar goal here.
spk09: Thank you.
spk07: Thank you. That was your last question. This concludes today's conference call. Thank you, and have a great day.
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