Amicus Therapeutics, Inc.

Q1 2023 Earnings Conference Call


spk06: Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics first quarter 2023 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, Vice President of Investor Relations. You may begin.
spk03: Good morning. Thank you, Gigi. Thank you for joining our conference call to discuss Amicus Therapeutics' first quarter 2023 financial results and corporate highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer, Daphne Queeney, Chief Financial Officer, Sebastian Martel, Chief Business Officer, and Dr. Jeff Castelli, Chief Development Officer. Joining for Q&A is Dr. Mitchell Volman, Chief Medical Officer, and Alan Rosenberg, Chief Legal Officer. As referenced on slide two, we might 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 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 questionnaire 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 Form 10-K, for the year ended December 31st, 2022, and the quarterly report on Form 10-Q for the quarter ended March 31st, 2023, to be filed later today 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.
spk13: Great, thank you, Andrew, and welcome everyone to our first quarter 2023 conference call. I'm pleased to report a successful start to the year across our global business, As we did in this morning's press release, let me highlight several key points before I turn it over to the team to provide more detail. First, Gallifold continues its strong performance and remains the cornerstone of our success. We continue to be very pleased with the commercial uptake of Gallifold globally, with $86 million in first quarter revenue representing 14% growth from the first quarter last year on a constant currency basis. Strong trends continue across a number of our key performance indicators, including increasing demand through new patient starts for both switch and naive populations, steady growth of in-person visits between our field team and Fabry treaters, and sustained patient compliance and adherence rates of over 90%. We expect to see continued double-digit growth in 2023, fueled by significant demand for patients and physicians. And in fact, as Sebastian will highlight in a moment, This was the best first quarter performance for net new patient starts in the past four years. Second, we continue to make great progress on our global regulatory filings and commercial planning for ACGA, our novel two-component therapy for Pompe disease. Importantly, I'm very happy to report that the US FDA has recently completed the required pre-approval inspection of the Wuxi Biologics manufacturing site in China. We're very pleased with the outcome of the inspection and believe the comments and observations received by Wuxi at the close of the inspection are all addressable. We therefore continue to expect regulatory approval of ACGA in the United States in the third quarter of 2023. I'd also like to give a word of thanks to the FDA inspectors, the Wuxi team, and the Amicus team the collaborative and professional conduct leading up to and during the course of the inspection. The regulatory reviews in Europe and the UK also remain on track. In March, the European Commission granted approval of Opfolda for the use in combination with Opfolda for adults with late onset Pompe disease. And as we just announced, the CHMP also adopted a positive opinion recommending marketing authorization of Opfolda So we continue to anticipate the full approval of Pombility plus Afolda to occur in the third quarter this year. And upon approval, this will be the first two-component therapy available in the European Union for the treatment of adults living with late-onset Pompe disease. And finally, the UK regulatory process for APGA was initiated December of last year and is following a similar timeline as the US and Europe, with expected approval in the third quarter this year as well. As we've observed throughout 2022, we remain extremely pleased with the level of interest and participation we're seeing in our expanded access mechanisms for APGA globally. As we get through the first part of 2023 across all of our ongoing clinical studies and expanded access programs, there are now approximately 200 patients on APGA, which we believe represents more than 5% of the total treated Pompe patients around the world. And we have more patients in the queue, so we expect this number to continue to trickle upward as we approach launch. In anticipation of the regulatory approvals, our global launch planning progress continues as we eagerly anticipate the final approvals of ATGA. We very much look forward to providing a real choice in this therapeutic area, both in the United States and in Europe, with regulatory applications in additional countries 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. Importantly, Based on the latest assumptions on approvals and launch of APGA, our current operating plan, and continued careful management of expenses, we remain on track to achieve non-GAAP profitability, which we expect in the second half this year. And on slide four, as we laid out at the beginning of the year, we are laser-focused on achieving our key strategic priorities for 2023, including, number one, sustaining that double-digit growth revenue of 12% to 17% for Gallifold at constant exchange rates, delivering this important medicine to more people living with Fabry disease with amenable variants and existing in new markets. Second, securing regulatory approvals of ATGA by the FDA, EMA, and NHRA this year, as well as continuing to advance preparations for the anticipated launches. Number three, continue to judiciously invest in the advancement of our best-in-class next-generation Fabry and Pompe genetic medicines and capabilities, as well as our next-generation chaperone for Fabry disease Number four, as always, maintaining a strong financial position as we carefully manage our expenses and investments on our path to non-GAAP profitability. With that overview, let me now turn the call over to Sebastian Martel, our Chief Business Officer, to further highlight the Gallifold performance. Sebastian?
spk09: Thank you, Bradley. And good morning to everyone on the call. I'll start by providing you with more detail on our Gallifold performance for the quarter. As you can see on slide six, for the first quarter, 2023, Galliford reported revenue reached $86.1 million, driven primarily by strong patient demand. The geographic breakdown of revenue during the quarter consisted of $57 million, or two-thirds of revenue, generated outside of the U.S., and the remaining $29 million, or one-third, coming from within the U.S. That's in line with the fit that we expect as we continue to grow both parts of the business. As Bradley mentioned, we're pleased to see continued patient growth in countries across our leading markets. Now turning to slide seven, our results for the first quarter highlight the strength of our global commercial efforts. The demand for GalaPulse globally continues to be strong, with patients added in all major markets, delivering operational growth rate of 14% over the same period in 2022 at constant exchange rates. From a year-over-year perspective, the negative impact on foreign currencies was 4% in the period. As a result, GalaCold reported revenue growth versus the same period last year with 10% for the first quarter. GalaCold continues to be the fastest growing treatment for public disease globally. I'm pleased to report that our monthly net new patient trends continues to increase. And if you look at the growth in net patients on GalaCold globally, which is the truest measure of the underlying business, we see a high teen growth rate in patients on GalaFold at the end of Q1 this year versus the same period last year. So all indications are of the continued and growing demand for GalaFold. When we ended the first quarter, we had more than 55% of the global market share of treated amenable patients. And within the global mix, we're starting to see stronger uptake in naive populations. So while we're achieving high market shares in countries where we've been approved the longest, there's still plenty of opportunity to continue to switch patients over to GalaFold and continue to grow the market as we penetrate into the diagnosed and treated and newly diagnosed segments. All of that is underpinned by impressive compliance and adherence rates that continue to exceed 90%, reiterating our belief that those patients who go on GalaFold predominantly stay on GalaFold. Importantly, the value of GalaFold continues to be recognized by payers as we've got a very strong track record of successfully negotiating and renegotiating reimbursement outside of the U.S. Our relentless commitment remains on ensuring access to GalaFold for anyone who needs it. Based on the performance we've seen here in the first quarter of 2023, GalaFold uptake continues to track very well, and we're seeing those trends continue with growth across all our major markets as well as most of our smaller markets. Turning to slide eight, GAAP growth remains strong with $86.1 million in revenue in Q1. As mentioned on past calls, due to a variety of factors, including uneven ordering patterns and FX fluctuations, the rate of growth within the year is typically nonlinear, and we expect that to continue throughout 2023. In the table that you have on the right-hand side of the slide, we've provided a five-year historical snapshot of the percent of GalaFold sales that occur each quarter during a given year. We would expect a similar trend to occur this year. Now, moving on to slide nine, we know there's significant demand for GalaFold and that the segment of the global family market made of patients with amenable mutations has the potential to surpass $1 billion in annual revenue in around five years. We anticipate sustained growth throughout 2023 to be driven by several key co-drivers, continuing to penetrate into existing markets, further uptake into the diagnosed and treated population, expanding to newer geographies, and label extensions. As we just mentioned, all of these efforts are supported by selling compliance and adherence rates and positive reimbursement and access mechanisms around the world. I'm pleased to share that we're making continued progress on expanding into new markets and also on extending our label. To name a few examples, we recently received marketing authorization for GalaFold in Hong Kong. The extension of use to adolescent patients 12 years and above has recently been approved in Canada, and we've submitted the marketing authorization application in New Zealand, and we've now entered into pricing and reimbursement negotiations for GalaFold in Turkey. In the longer term, we continue to see significant growth in the public 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. Also, important to note here, we have often exclusivity in the U.S. and Europe. In addition to our now 49 orange booklisted patents, including eight compositions of matter patents, And 33 of those give us IP coverage into the late 2030s, which provides us the opportunity to provide access to Galeco globally for a long time to come. Now, moving on to slide 10. We're happy today to stand less than a quarter from the commercial launch of Mobility and Upholder. On that slide, we outline launch preparations as we're poised for another successful product launch. As you know, Amicus is the presence in over 40 countries around the world today, including all the major Fabri and Pompée markets. That team that will launch Pompility and Upholder has largely stayed the same with only a few additional FTEs. We're now fully recruited and trained to support the first wave of launch countries. I'm pleased to note that product was being positioned to support the first group of patients coming onto commercial supply. We have experience across all areas that are needed for a successful drug launch, regulatory, commercial, supply chain, experience with payers, reimbursement and access. In addition, and most importantly, the key relationships with physicians. We're confident in our world-class organization that we can leverage their experience and relationships and deliver ATGA to people living with competencies around the world. Our educational and promotional materials are ready. And training in our initial launch countries is now completed. Market mapping is also complete. From the team, the medical education and publications, our ability to market rare disease products successfully, our experience with reimbursement and access around the world, and again, all the strategic planning that we've been doing, together with building inventory with our partners at Wuxi Biologics, we believe we're in a very strong position for succumb to successful launch advantages. With that, let me now hand the call over to Jeff Castelli, Chief Development Officer, to highlight our ATGA program and pipeline updates. Jeff?
spk12: Thank you, Sebastian, and good morning, everyone. On slide 12, we'll start with our ATGA program. Pompe is a severe and fatal neuromuscular disease and one of the most prevalent lysosomal disorders. And multiple publications and natural history studies have shown that the initial benefits of treatment are often followed by continued long-term decline for many individuals. We recognize that Pompe disease continues to pose a range of health challenges for people affected by the disease, and having therapeutic choices is crucial. Moving on to slide 13, we remain very excited to be anticipating potential regulatory approvals in three of the largest Pompe markets in the third quarter of this year, as Bradley noted, On this slide, we summarize the status of the anticipated regulatory milestones by market. First, in the EU, POMBILITY was granted European Commission approval in March. In April, the CHMP adopted a positive opinion about FODA, and we expect an EC approval to occur in the third quarter of 2023. In the US, the FDA recently completed the required pre-approval inspection of the Wuxi Biologics manufacturing site in China. And we are very pleased with the outcome and believe that the comments and observations received by WUSHI at the closing inspection are all addressable. We continue to expect approval of both components of ATGA together in the third quarter of this year. And finally, the regulatory submission process for ATGA in the UK was initiated in December via the recognition procedure based on the CHMP opinion. And we're on track for an expected MHRA approval also in the third quarter. So lots of great momentum across the key markets. Moving on to slide 14, we highlight our ongoing clinical studies and multiple mechanisms of expanded access that support some of the early demand for ATGA. For the younger Pompe community, we continue to enroll the ongoing open-label ZIP study in children up to 18 years of age living with late-onset Pompe disease. They have now begun enrolling the open-label Rosella study for children living with infantile-onset Pompe disease. Importantly, in response to the multiple requests for treatment that we continue to receive for children living with LOPD and IOPD, our expanded access programs continue to enroll those patients. We have multiple expanded access programs in place, including in the US, UK, Germany, France, Japan, and other countries. This includes the Early Access to Medicine Scheme, or EAMS, in the UK, where we continue to see significant enthusiasm for ATGA. with physicians having requested access across all of the leading Pompe centers and dozens of patients now on treatment. Since the positive scientific opinions, interest, and momentum for ATGA has continued to grow, and we are pleased to be able to provide access to those who are eligible. With this growth in our access programs, as Bradley noted, we are pleased to report that there are approximately 200 patients now worldwide being treated with ATGA across our clinical extension studies and expanded access programs. An experience with ATGA is growing worldwide with approximately 75 centers currently participating in those different programs. Finally, as highlighted in the pipeline slide in the appendix for our earlier stage pipeline, we continue to focus on novel approaches for Fabry and Pompei, including gene therapy to deliver our engineered DLA and GA transgenes and a next-generation Fabry chaperone. With that, I would like to now turn the call over to Daphne Queeney, our Chief Financial Officer, to review our financial results, guidance, and outlook. Daphne?
spk05: Thank you, Jeff, and good morning, everyone. Our financial overview begins on slide 16 with our income statement for the first quarter ending March 31, 2023. For the first quarter, we achieved total revenue of $86.3 million, which is a 10% increase over the same period in 2022. This includes year-over-year operational revenue growth measured at constant currency exchange rates of 14%, impacted by a negative currency of 4%. Cost of goods sold is a percentage of net sales with 8% in the year as compared to 9.6% for the prior year period. Total gas operating expenses decreased to $117. million for the first quarter of 2023 as compared to $146.5 million in the first quarter of 2022. On a non-GAAP basis, total operating expenses decreased to $80.6 million for the first quarter of 2023 as compared to $109 million for the first quarter of 2022, reflecting decreased program spend. 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 2023 was 52.9 million, or 18 cents per share, as compared to a net loss of 85.3 million, or 30 cents per share for the prior year period. Driven by the revenue growth of Gallifold and careful expense management, we continue to make progress towards our path to non-GAAP profitability in the second half of this year. As of March 31, 2023, we had approximately 283 million shares outstanding. Turning now to slide 17, 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. Our full year 2023 non-GAAP operating expense guidance is $340 million to $360 million. The expected decrease in operating expense for 2023 as compared to 2022 will be achieved by continuing to drive efficiencies and prudent expense management. offset by continued investment in the global growth of Gallifold and pre-launch and launch activities for ATGAA. We anticipate operating expenses to be nonlinear this year due to these pre-launch and launch expenses, as well as certain non-recurring manufacturing costs that we expect will be incurred in the second and third quarters of this year. We also expect to see a larger portion of our operating expense to be allocated to G&A this year, as we align our resources to support the launch of ATGAA and the continued growth of Gallifold. A few comments about our cash position and 2023 financial guidance. Cash, cash equivalents, and marketable securities were $267.1 million at March 31, 2023, compared to $293.6 million at December 31, 2022. Our full-year Gallifold revenue guidance is revenue growth of 12% to 17% at constant exchange rates, in addition to our non-GAAP operating expense guidance of $340 million to $360 million. And with that, let me turn the call back over to Bradley for our closing remarks.
spk13: Great. Thank you, Daphne, Jeff, Sebastian, for the detail there. So as you can all see, we've been relentlessly focused on execution this year across the business. and have laid a strong foundation for what we think will be an important year for Amicus. As we approach this next phase of the company, we're confident we can continue to drive sustainable, long-term value and deliver life-changing therapies to people in need. Operator, we can now open the call to questions.
spk06: Thank you. Ladies and gentlemen, if you have a question, please press star 1-1 on your touchtone telephone. At this time, we request that you only ask one question. If you have any additional questions, please enter back into the queue. Thank you. One moment for our next question. Our first question comes from the line of Anupam Rama from JP Morgan.
spk10: Hi, thanks for taking the question. This is actually Malcolm Kuno on for Anupam. So looking past the third quarter approval, what observations have you learned from Sanofi's launch in Pompeii that you can perhaps apply to your own launch? And then how should we view your launch curve relative to that competition?
spk13: Yeah, thanks for the question. Maybe I'll start. Look, so, you know, we are laser focused, as we said, is getting through the approvals and launch. And I think for us, the most important thing is providing a new choice that we think is differentiated in particular with its two component mechanism of action. We I've watched, I think, Sanofi both in the United States and other markets around the world. And I think you could see, depending on when they've launched, how they've progressed. And for us, I think it's much more about continuing to demonstrate the value of ATGA to physicians and patients. I think we look at the expanded access programs and uptake where the product is available through those mechanisms. And we take great confidence from the way we see patients and physicians Using the medicine and, you know, from our perspective, the most important thing is looking at what we've demonstrated in the clinic that in particular, in the experience population, when patients switch from therapy, we see a significant increase in 6 minute walk distance as well as improvement in force vital capacity. We think that differentiation the only therapies that shown in a controlled study. that we can show those changes. We think that sets us up as a very differentiated therapy when we launch and we're just really eager to get over the finish line. To your second question in terms of how we kind of think about the launch uptake, it'll come as no surprise, but of course the first focus is to convert our existing clinical and expanding access patients over to commercial medicine, and we're prepared to do that as quickly as possible. We'll also be focusing, of course, on patients who we think we can switch the current standard of care over to ATGA. The goal this year, frankly, just given the fact that the approvals happen in the back half of the year, it takes time from a pricing and reimbursement perspective, the goal really is to maximize the number of patients on therapy by the end of the year. And that, we think, gives us a really strong run rate going into 2024. Thank you.
spk06: One moment for our next question. Our next question comes from the line of Ellie Merle from UBS.
spk01: Hey, guys. Thanks so much for taking the question. Just wondering if you could elaborate a little bit on the comments and observations received from the FDA inspections. and maybe just kind of what drives your confidence that all of these are addressable. And then just a question procedurally on how to think about the timeframe, like from inspection to approval. I guess just in situations like this with a deferred action, how should we think about the typical timeframe and maybe also just next steps from here in terms of the timelines for addressing the comments and observations? Thanks.
spk13: Yeah, thanks, Ellie, for the questions. You know, as we said previously leading up to the inspection, we're not going to provide details of the findings, but I will reiterate we're very pleased with the outcome. The color I can provide is that we think the feedback was straightforward and addressable. That's shared by Wuxi with all their experience with inspections, and that's why we feel very confident here reiterating the approval in the third quarter. I would say, too, there was a high level of engagement, professionalism between the inspectors, Wuxi and Amicus, And so all of this together makes us feel very confident in that timeline that we reiterated today. You're right, in terms of sort of what happens from here, we are in a little bit of an unusual situation. But typically what happens is, you know, as the FDA finalizes the inspection, which has now happened, then they finalize the inspection report. You know, that takes a period of time. And once the inspection report is finalized and provided to the review division, that's where you get that kind of 30 to 60 day timeline to approval. And so that gives us a lot of comfort that we're on track there. based on the fact that we received the deferred action letter, and again, we'll remind folks, at least for the regulation, that those can only be given when no other issues remain and the application otherwise satisfies the requirements for approval. That gives us great confidence that effectively it's really just getting through the final inspection report and then the final memos within the agency, and then we're on to approvals and launch.
spk06: Thank you. One moment for our next question. Our next question comes from the line of Joseph Schwartz from SVB Securities.
spk02: All right, thanks so much. I was wondering if you could talk a little bit about the market opportunity from Pompability and whether that has evolved or your strategy for launching it has evolved now that the launch has been delayed and a competitor has been on the market for a while longer than was initially expected.
spk13: Yeah, maybe, Sebastian, do you want to talk a little bit about kind of what we're seeing and why we're remaining confident in that market opportunity to Joe's question?
spk09: Yeah, so, Joe, you know, the overall market for Pompeii last year reached about $1.2 billion. You know, it's a market that has been growing at high single digits, low double digits for a number of years. Still, this is a disease that is under-diagnosed, so we expect the overall market growth rate to continue at least at the same rate. As you look at the various regions and our initial focus in the US, in the UK, in Europe, these are the three largest markets for Pompe disease. We estimate that there is around 1,300 patients treated for Pompe in Europe, another couple of hundreds in the UK, and north of 800 patients in the US. I think we've seen repeatedly, you know, through all the outcomes that we've done and market research assessments that physicians react very well to the product profile of ATGA, and in particular to the switch data that we've generated from the Propel study. Obviously, the vast majority of the opportunity lies in this switch opportunity. The number of newly diagnosed naive patients for Pompe is a much smaller number on a global basis, maybe 100 to 200 patients per year, compared to an overall market of actually treated patients somewhere between 3,500 and 4,000. So we'll be very much focused on ensuring that we provide easy access to ATGA. And as Bradley alluded to, I think that the experience we're seeing through the early access programs bodes well for the launch of ATGA.
spk06: Thank you. One moment for our next question. Our next question comes from the line of Ritu Bharal from TD Cowan.
spk04: Hey, guys. This is Athena on for Ritu. Thanks for taking the question. Just another follow-up on the WSU inspection. Has every building as part of the entire supply chain been inspected at this point? And do you anticipate that any of the inspection findings could be getting for approval? Thank you.
spk13: Yeah, thanks for the question, Athena. So the important part of this inspection, so Wuxi has been inspected, that site in China has been inspected multiple times by multiple regulators for multiple products, all successful. So that's a good thing. This particular inspection was focused on the drug substance and drug product manufacturing facilities for our product in particular. And so, you know, I think that's the difference here. Now that that's been complete, there are no other inspections by the United States that we believe are necessary ahead of approval. And so we feel very confident at this point that we are on track there based on the outcome of the inspection. So I think, you know, at this point, we're sort of all systems go and looking forward to getting through the finalization of the review and heading towards approvals and launches.
spk06: Thank you. One moment for our next question. Our next question comes from the line of Daegun Ha from Stifel.
spk08: Hey, good morning, guys. Thanks for taking our questions. I'll stick to one. Wanted to get your take on pricing strategy given the parallel review, and I understand you guys gave guidance on a 3Q approval for both sides of the Atlantic, but It does seem like EU might come sooner, considering the processes remaining for the FDA side. So how are you thinking about that overall? And how does that compare to your Fabry strategy? Thanks so much.
spk13: Yeah, thanks, Dagon. You know, we have taken the strategy that we will price our medicines at parity or modest discount to standard of care. And that's really because we value access more than anything else. So our goal is to get as many patients on therapy as quickly as possible. And we feel like taking price off the table, in other words, focusing on the value of the medicine that it brings to patients and physicians versus the cost of the medicine is the best way to ensure that you move through the reimbursement process as quickly as possible. And what we saw with Gallifold, to your point, is that we actually moved through that reimbursement process successfully and and that we did it in times that were much faster than industry average. So we'll look to apply the same strategy here. And in some instances, actually, those discussions are already ongoing. And so we look forward to deploying the same strategy and, again, focusing on the value of the medicine versus the cost.
spk06: Thank you. One moment for our next question. Our next question goes to the line of Kristin Kluska from Cantor Fitzgerald.
spk00: Hi, everyone. Good morning. Thanks for taking my questions. Wanted to ask what your latest thoughts are on how you might position ATGAA relative to the benefits across respiratory outcomes and different measurements there. And I know you presented more data at Worlds this year. And in the past, you've cited this as a really important endpoint considering it's a leading cause of mortality for these patients. Thank you.
spk13: Yeah, thanks, Kristen. Jeff, maybe you can talk through the data we've seen both in the initial phase of the study and then what we just released at World with the extension data.
spk12: Yeah, thanks, Brian. Thanks, Kristen, for the question. So, you know, as we've seen in our clinical studies with ATGA, We see on pulmonary function, which as you pointed is, you know, one of the basically the leading cause of mortality in Pompe that, you know, patients that had been on state of the care ERT saw quite significant decline in their pulmonary function as they remained on treatment, whereas patients that switched to ATGA showed good stabilization of that pulmonary function. And importantly, as we've now seen data in our extension studies from phase 1-2 up to four years and from our Propel study up to two years, that stability seems to be maintained and durable over those additional extended time periods in the trials. So we're very excited about the potential ability to help stabilize that progressive decline in pulmonary function we see. And also importantly, the other key arm of Pompe is on the motor function and muscle strength side of things. there we're actually really excited to see some improvements even in patients that, which are, you know, not necessarily expected as patients switch to ATGA. So, you know, across those two key aspects of disease, we're very excited by both the initial response to treatment as seen in Propel, but then the continued durability of the response in the extension trials.
spk06: Thank you. One moment for our next question. Our next question comes from the line of Salveen Richter from Goldman Sachs.
spk07: Hi, this is Shranatra on for Salveen. We have two questions. The first is how soon post-approval can we expect the first patients to be treated with APGA? And here I'm referring to patients who are not on the early access programs. And the second is could you provide some color on what we can expect in the first few months of the launch? Thank you.
spk13: Yeah, thanks for the question. So in terms of first patients on therapy, so there's two different situations. You have the United States, of course, where you get approved and then you're typically moving quickly to setting up infusions and getting prescriptions and then setting up infusions and getting patients on therapy. What we've seen in our experience with Gallifold is our goal is to convert all of our patients on therapy who are, of course, on label to commercial drug within the first 90 days. And so that's something we'll focus on. And we were able to execute that very successfully in the Gallifold launch. And we believe we'll be able to do that here. And then likewise in Europe, of course, you get approval. In Germany, you typically have, you know, what's kind of colloquially known as that free pricing period. So, again, their reimbursement will come almost immediately. And the goal will be on the back of approval. The goal will be to quickly, again, convert patients on existing therapy. I'll note in Europe or sorry, in Germany, we do have an expanded access program ongoing as well. And again, that's within, you know, kind of that 30 to 90 day period. And then in the UK, we will go through the reimbursement process. And again, that's where that focus on access is so important. So there is a little bit of time to get from MHRA approval to NICE approval and reimbursement. We'll also, of course, while we're converting our existing patients, also be focused on initiating patients who are on existing therapy in the U.S. and all patients outside the U.S. as well that are on label.
spk06: Thank you. One moment for our next question. Our next question comes from the line of Jeff Hung from Morgan Stanley.
spk11: Thanks for taking my question. Can you talk about the recent feedback you've heard from payers on ATGAA and what kinds of comments do you hear on potential barriers on access or restrictions? Thanks.
spk13: Yeah, thanks for the question. You know, all of our market research from a payer perspective has been, I think, very supportive of the approach that we outlined here in terms of our strategy, which is that as long as it's on label, and if we have a parity or modest discount strategy, payers, I think, will be very supportive of this medicine. You know, they've gotten more sophisticated, especially in this disease area, so they do review the data very carefully, and we expect that process to continue And then likewise, outside the U.S., where you're going through the HCA assessments, again, I think with the data that we have, with the market research we've done leading into this process, and now with the ongoing negotiations we've started with some of those payers, we think this is going to be a very successful access strategy, and we're just eager to get through the process and provide this medicine to patients.
spk05: Thank you.
spk06: Thank you. One moment for our next question. Our next question comes from the line of Chi Chung Shu from Beringberg.
spk14: Good morning. Thanks for taking the question. I wanted to ask your, today announced the expansion of the supply and manufacturing agreement with Wuxi on its Ireland facility. I guess, can you talk about the transition plan from the China facility to the Ireland facility? And also, do you expect the FDA inspection on the Ireland facility in due course? Thanks very much.
spk13: Yeah, great question. So yes, we announced in the last couple of weeks we did enter into a commercial manufacturing agreement with Wuxi. That was something that was in the works for quite some time, and we're really thrilled to solidify that. That effectively secures capacity, longer-term capacity and supply from Wuxi, from both their China facility as well as their Ireland facility. The strategy really is to eventually transition so that the majority of supply comes from Ireland. And what we've suggested before is we're on track for that process to conclude, that facility to be licensed. and materials start to enter the commercial supply chain in the back half of next year or early 2025, and that process is going well. In terms of inspection of the facility, of course, you'll have to go through the typical PPQ and validation and comparability work as you transition to Ireland. All of that is kind of contained in the timeline that I shared with you Will the FDA or other regulatory agencies inspect the facility? Almost certainly they will at some point, whether that will be required as a pre-licensure inspection or whether it will happen sometime in due course, you know, to be determined. But we're very confident in that process, and we're eager to have, of course, a second site of manufacturing come online and one that's geographically diversified as well.
spk06: Thank you. At this time, I would now like to turn the conference back over to Bradley Campbell for closing remarks.
spk13: Great. Thanks, operator. Thanks, everybody, for the participation in the call today. All your great questions. Hope everybody has a great day. Take care.
spk06: Thank you. This concludes today's conference call. Thank you and have a great day.

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