Q4 2023 Amicus Therapeutics Inc Earnings Call

Okay.

Operator: Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Full Year 2023 Financial Results Conference Call and Webcast. At this time, all participants are in a listen-only mode.

Speaker Change: Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics full year 2023 financial results conference call and webcast.

At this time all participants are in a listen only mode.

Operator: 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.

Speaker Change: Later, we will conduct a question and answer session and instructions will follow at that time.

Speaker Change: As a reminder, this conference call is being recorded I would now like to turn the conference over to your host Mr. Andrew Faughnan, Vice President of Investor Relations you may begin.

Andrew Faughnan: Great. Thank you T D.

Andrew Fonin: Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics' full year 2023 financial results and corporate highlights. Leading today's call is Bradley Campbell, President and Chief Executive Officer, Sebastian Martel, Chief Business Officer, Simon Harford, Chief Financial Officer, and Dr. Jeff Castelli, Chief Development Officer. Also joining for Q&A are Dr. Mitchell Goldman, Chief Medical Officer, and Allen 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. However, our forward-looking statements should not be regarded as representations by us that any of our plans will be achieved.

Andrew Faughnan: Morning. Thank you for everyone. Thank you for joining our conference call to discuss <unk>.

Andrew Faughnan: Full year 2023 financial results and corporate highlights during today's call. We have Bradley Campbell, President and Chief Executive Officer, Sebastien Martel, Chief Business Officer, Simon Harford, Chief Financial Officer, Dr. Jeff Castelli, Chief Development Officer, joining for Q&A is Dr. Mitchell Goldman Chief Medical Officer, and Alan Rosenberg, Chief Legal officer.

Andrew Faughnan: 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 are forward looking statements should not be regarded as representation by us that any of our plans will be achieved and are all the forward looking statements made on this call may turn out to be wrong and can be affected by.

Andrew Fonin: 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 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 Form 10-K for the year ended December 31, 2023, to be filed today with the At this time, it is my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Bradley?

Andrew Faughnan: 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.

Andrew Faughnan: 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.

Andrew Faughnan: For a 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 31, 2023 to be filed 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.

Bradley Lewis Campbell: Executive Officer Bradley.

Bradley Lewis Campbell: Great, thanks, Andrew, and welcome, everybody, to our full year 2023 conference call. This year, our call falls a day before Rare Disease Day. So before I dive into our results, I just want to take a minute to acknowledge this important day for the rare disease community. This year's theme is achieving true health equity for the nearly 300 million people around the world living with a rare condition. Since our inception, Amicus has been dedicated to serving the needs of those in the rare disease community in extraordinary ways, and we are committed to advocating for patient communities and fighting on behalf of those whose voices have not been heard. Rare Disease Day serves as a global call to action to collectively work towards equity and social opportunity, health care, and, perhaps most importantly, access to medicines for people living with rare diseases.

Bradley Lewis Campbell: Great. Thanks, Andrew and welcome everybody to our full year 2023 conference calls this year, our call fault a day before rare disease day, so before I dive into our results I just wanted to take a minute to acknowledge it.

Bradley Lewis Campbell: This important day for the rare disease community. This year's theme is achieving true health equity for the nearly 300 million people around the world living with a rare condition.

Bradley Lewis Campbell: Since our inception amicus has been dedicated to serving the needs of those in the rare disease community in extraordinary ways and we are committed to advocating for patient communities and fighting on behalf of those whose voices have not been heard.

Bradley Lewis Campbell: Rare disease day serves as a global call to action to collectively work towards equity and social opportunity healthcare and perhaps most importantly access to medicines for people living with rare diseases. So I'll just ask it all of you joining me in commemorating this important day for the global rare disease community.

Bradley Lewis Campbell: So I just ask that all of you join me in commemorating this important day for the global rare disease community. With that, let me just shift into our financial results and outlook. And I am very pleased to highlight what tremendous progress we made across our global business last year and into the start of 2024. As you saw in this morning's press release, I'll highlight several key points before I turn it over to the team to go through them in more detail. So first, Galliford continues its strong performance, and it remains the cornerstone of our success. We are very pleased with the continued uptake of Galliford products globally. We now have over 2,400 people living with Fabry disease who take Galliford or will start taking Galliford at the end of 2023. For the full year, that translated to Galliford revenue of $388 million globally, representing 18% growth year over year.

So with that let me just shift into our financial results and outlook and I am very pleased to highlight what tremendous progress we made across our global business last year and into the start of 2024 as you saw in this morning's press release I'll highlight several key points before I turn it over to the team to go through more detail.

Bradley Lewis Campbell: First <unk> continues its strong performance and it remains the cornerstone of our success. We are very pleased with the continued uptake of <unk> globally. We now have over 2400 people living with Fabry disease, who take Galiform, taking Gallup poll at the end of 2023 for the full year that translated to <unk> revenue.

Bradley Lewis Campbell: New Av $388 million globally, representing 18% growth year over year outperformed outperforming our expectations and our initial guidance for the year.

Bradley Lewis Campbell: Outperforming our expectations and our initial guidance for the year, we continue to observe strong trends across our key performance indicators in all of our key geographies, including continued demand through new patient starts from both switch and naive populations in all of our leading markets. We saw steady growth of in-person visits between our field team and Fabry treaters, and we continue to see sustained patient compliance and adherence rates of over 90%. Growth in 2023 was driven primarily by patient demand from net new patient starts, continuing to switch patients in our newer launch markets, and continued penetration into the diagnosed and untreated population, which we expect to continue to be a major driver of growth in 2024 and beyond as the Fabry market continues Just to put that in perspective for a minute, as a reminder, just over eight years ago, we estimated there were 5,000 patients who were treated for Fabry disease and 5,000 patients who were diagnosed but untreated.

Bradley Lewis Campbell: Throughout 2023, we continued to observe strong trends across our key performance indicators and all of our key geographies, including continued demand through new patient starts from both switch and naive populations in all of our leading markets. We saw a steady growth of in person visits between our field team in Fabry Treaters, and we continue to see sustained pace.

Bradley Lewis Campbell: Compliance and adherence adherence rates of over 90%.

Bradley Lewis Campbell: Growth in 2023 was driven primarily by patient demand for net new patient starts continuing to switch patients that are newer launch markets and continued penetration into the diagnosed and untreated population, which we expect to continue to be a major driver of growth in 2024 and beyond as the fabry market continues.

Bradley Lewis Campbell: To see improved diagnosis and medical education, and finding patients just to put that in perspective for a minute. As a reminder, just over eight years ago. We estimated there were 5000 treated fabry patients and 5000 patients who are diagnosed but untreated.

Bradley Lewis Campbell: Today, there are more than 11,000 treated patients, so the treated market has more than doubled in that time, while the untreated market has also increased to almost 6,000 patients. These underlying market dynamics will provide the opportunity to grow Galliford for many, many years to come, including in 2024, when we expect another great year with 11 to 16% projected growth at constant exchange rates. Second, the global commercial launch of Pondbilly and Alpholda is underway, and it's off to a great start in the three largest Pompeii markets.

Bradley Lewis Campbell: There are more than 11000 treated patients. So the treated market has more than doubled in that time, while the untreated market has also increased to almost 6000 patients.

Bradley Lewis Campbell: These underlying market dynamics will provide the opportunity to grow <unk> for many many years to come including in 2024, when we expect another great year with 11% to 16% projected growth at constant exchange rates.

Bradley Lewis Campbell: Second the global commercial launch of <unk> 1 billion up Volta is underway and it's off to a great start in the three largest markets. Our teams have made significant progress in the initial commercial launch in Germany, the UK and the U S and we remain incredibly pleased with the launch so far.

Bradley Lewis Campbell: Our teams have made significant progress in the initial commercial launch in Germany, the U.K., and the U.S., and we remain incredibly pleased with the launch so far. Seven weeks ago, at J.P. Morgan, we shared that 120 patients were on treatment or scheduled for treatment, including 15 new commercial patients. And as we said at the conference, we're not going to give mid-quarter updates, but I can share more color on how incredibly pleased we are with the continued patient and physician demand for this new therapy. The key performance indicators that we talked about last time continue to improve and continue to give us confidence in the strength of this launch. Specifically, the rate of new commercial patients coming on to Pondility and Outfolder continues to increase in all three markets as we progress through the early weeks of the year.

Bradley Lewis Campbell: Seven weeks ago at Jpmorgan, we shared the 120 patients were on treatment or scheduled for treatment, including 15, new commercial patients and as we said at the conference we will not going to give mid quarter updates, but I can share more color on how incredibly pleased we are with the continued patient and physician demand for this new therapy.

Bradley Lewis Campbell: The key performance indicators that we talked about last time continue to improve.

Bradley Lewis Campbell: And continue to give us confidence in the strength of this launch specifically the rate of new commercial patients coming onto possibility in a folder continues to increase in all three markets as we progress through the early weeks of the year.

Bradley Lewis Campbell: We anticipate launches in additional countries will add to that growth through the latter part of the year, and importantly, for our new commercial patients, we continue to see prescriptions proportionally coming from nexviazime and lumizyme, as well as naive patients in ex-U.S. markets. So that means in the U.S., the majority of new patients are switching off of Nexviazime and coming on to Ponvility and Upfolda, whereas in Europe, where Lumizyme has the majority market share, most of our switch patients are coming from that therapy, and we're seeing an increasing number of naive patients as well.

Bradley Lewis Campbell: We anticipate launches in additional countries will add to that growth through the latter part of the year and importantly for our new commercial patients. We continue to see prescriptions proportionately coming from <unk> and <unk> as well as naive patients in ex U S markets. So.

Bradley Lewis Campbell: So that means in the U S. The majority of new patients are switching off of next five design and coming onto upon ability to uphold that whereas in Europe, where <unk> has a majority market share most of our switch patients are coming from that therapy, and we're seeing an increasing number of naive patients as well. So all of the patient segments are performing as we would expect.

Bradley Lewis Campbell: So all the patient segments are performing as we would expect. In a moment, Sebastian will provide further color on the ongoing launch. Key Performance Indicators

In a moment Sebastian will provide further color on the ongoing launch of these key performance indicators, but overall, we're very pleased by the launch and a great momentum, we're seeing across each of our markets.

Bradley Lewis Campbell: But overall, we're very pleased by the launch and the great momentum we're seeing across each of our markets. Throughout 2024, we'll continue to focus on increasing patient access by expanding into additional European countries as we navigate the country-by-country pricing and reimbursement process and focus on additional regulatory submissions as well. We are incredibly pleased to be providing a real choice for patients and challenging therapeutic options for both physicians and people living with pump-aided disease. Third, Amicus has maintained an incredibly strong financial position as we continue to execute on the expansion of Gallifold and advance the global launch of Pumbility and Ufolda.

Bradley Lewis Campbell: Throughout 2024, we will continue to focus on increasing patient access by expanding into additional European countries as we navigate the country by country pricing and reimbursement process and focus on additional regulatory submissions as well.

Bradley Lewis Campbell: We are incredibly pleased to be providing a real choice for patients and challenging therapeutic options for both physicians and people living with Pompe disease.

Bradley Lewis Campbell: Third amicus has maintained incredibly strong financial position as we continue to execute on the expansion of Gulf old and advance the global launch of probability of the fall that we are pleased to share today that as we promised we delivered our first quarter of non-GAAP profitability in the fourth quarter of 2023.

Bradley Lewis Campbell: We are pleased to share today that, as we promised, we delivered our first quarter of non-gap profitability in the fourth quarter of 2023. This is obviously a significant milestone as we now look to deliver our first full year of non-gap profitability in 2024, supported for the first time by well over half a billion in combined product sales, as well as prudent expense management. Two really significant milestones in the evolution of Amicus as a biotechnology company in the rare disease space. Our key strategic priorities for 2024 are laid out in the slide here. As we laid out last month, we're focused on achieving our key strategic priorities for 2024, including, number one, again, sustaining that double-digit Gallifold revenue growth of 11 to 16% at constant exchange rates, continuing our successful launch of Pondbility and Abfold Advancing our ongoing studies to support our medical and scientific leadership in Fabry and Pompeii disease.

Bradley Lewis Campbell: This is obviously a significant milestone as we now look to deliver our first full year of non-GAAP profitability in 2024 supported for the first time by well over half of $1 billion in combined product sales as well as prudent expense management.

Bradley Lewis Campbell: Two really significant milestones in the evolution of and I guess as a biotechnology company in the rare disease space.

Bradley Lewis Campbell: Our key strategic priorities for 2024 are laid out in the slide here for <unk>.

Bradley Lewis Campbell: As we laid out last month, we are focused on achieving our key strategic priorities for 2024, including number one again sustaining that double digit <unk> revenue growth of 11% to 16% at constant exchange rates, continuing our successful launch of possibility on folder and executing multiple successful new commercial launches throughout the year.

Bradley Lewis Campbell: Advancing our ongoing studies to support our medical and scientific leadership in Fabry, and Pompe disease, and finally, maintaining our strong financial position as we carefully manage our expenses and investments in order to achieve our full year non-GAAP profitability.

Bradley Lewis Campbell: With that let me now hand, the call over to Sebastian who can give further highlights on our commercial performance Sebastian.

Sebastian: Thank you Britney good morning, or good afternoon to everyone on the call.

Bradley Lewis Campbell: And finally, maintaining our strong financial position as we carefully manage our expenses and investments in order to achieve a full year of non-gap profitability. With that, let me now hand the call over to Sebastian, who can give further highlights on our commercial.

Sebastian: By providing you with more details on <unk> performance for the year on slide six for the full year 2023, <unk> reported revenue reached $387 8 million.

Sebastian: <unk> growth in 2023 was primarily driven by strong patient demand, particularly from our leading markets.

Sebastian Martel: Thank you, Bradley. Good morning or good afternoon to everyone on the call. I'll start by providing you with more details on our GalaFold performance for the year. On slide 6, for the full year 2023, GalaFold reported revenue reached $387.8 million. The gap of growth in 2023 was primarily driven by strong patient demands, particularly from our leading market. Turning to slide 7, our results for the full year highlight the strength of our global commercial efforts. The demand for Galafil globally continues to be strong, with patients added in all major markets, delivering operational growth of 18% over the same period in 2022, or 17% at constant exchange rates. We finish the year strongly, with fourth-quarter 2023 Galapagos reported revenue of $106.6 million, up 21% or 19% at CEO.

Turning to slide seven our results in the full year highlight the strength of our global commercial efforts. The demand organically globally continues to be strong with patients added in all major markets delivering operational growth of 18% over the same period in 2022 or 17% at constant exchange rates.

Sebastian: We finished the year strongly with fourth quarter 2023, Gala <unk> reported revenue of $106 $6 million up 21% or 19% at CER.

Sebastian: We're pleased to share that 2023 was the strongest year for net new <unk> patients since 2019.

Sebastian: Our leading markets such as the U K the U S. EU five countries and Japan remain the biggest drivers of patient demand.

Sebastian: Great confidence in the growth this product because over the long run.

Sebastian Martel: We're pleased to share that 2023 was the strongest year for net new Galluple patients since 2019. Our leading markets, such as the UK, the US, the EU five countries, and Japan, remain the biggest drivers of patient demand and give great confidence in the growth this product will have over the long run. As Brad mentioned, we ended the year with more than 2,400 patients from Gallup Polls, which is roughly over half of the global market share of treated and medical patients. Gallup Poll has captured 60 to 65% of the global market share of treated and medical patients. But the good news is there are still many more patients to put on to therapy; within the global mix, which is about 43% switch and 57% naive, we're seeing stronger uptake among the naive population.

Sebastian: As Brian mentioned, we ended the year with more than 2400 patients mechanicals.

Sebastian: Which is roughly over half of the global market share of treated amenable patients.

Sebastian: Got it thats captured 60% to 65% of the global market share of treated amenable patients.

Sebastian: The good news is there are still many more patients to put onto therapy.

Sebastian: Within the global mix, which is about 43% switch and 57% naive.

Sebastian: We're seeing stronger uptake in naive populations.

Sebastian: So we continue to achieve high market shares in countries, where <unk> been approved the longest.

Sebastian: There's still plenty of opportunity to continue to switch patients over to <unk>.

Sebastian: And keep on growing the market as we penetrate into the diagnosed untreated newly dangles segments.

Sebastian: All of that is underpinned by sustained compliance and adherence rates that continue to exceed 90% reiterating our belief that those patients who couldnt get a fault for them in the <unk> stay on going forward.

Sebastian Martel: So we continue to achieve 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 keep on growing the market as we penetrate the dino-untreated and newly dino sector. All of that is underpinned by sustained compliance and adherence rates that continue to exceed 90%, reiterating our belief that those patients who go on GALAFOLD predominantly stay on GALAFOLD. As mentioned on past calls, due to a variety of contributing factors and even ordering patterns and ethics fluctuations, the rate of growth within the year is typically non-linear.

Sebastian: As mentioned on past calls due to a variety of different contributing uneven ordering patterns in FX fluctuations the rate of growth within the year is typically non linear.

Sebastian: Additionally, we've historically seen Q1 revenues came in slightly below prior year Q4, due to the timing of orders and the reauthorization process in the U S.

Sebastian: And we expect that to continue into 2024.

Sebastian: So within the table on the right hand side of the slide we have provided a five year historical snapshots of the percentage of <unk> sales that occur each quarter during a given year.

Sebastian Martel: Additionally, we've historically seen Q1 revenues come in slightly below prior Q4 due to the timing of orders and the reauthorization process in the U.S., and we expect that to continue into 2024. So within the table on the right-hand side of the slide, we've provided a five-year historical snapshot of the percentage of Gallup sales that occur each quarter during a given year. Interestingly, the average quarterly sales distribution over the last five years corresponds precisely to what we achieved for the full year 2020.

Sebastian: Interestingly the average quarterly sales distribution over the last five years correspond precisely to what we achieved for the full year 2023.

Sebastian: We would expect that similar trends will occur this year.

Sebastian: So as an example, we expect Q1 sales of the current year to be around 22% of full year sales organically.

On slide eight we.

Sebastian: We know that there is a significant patient demand for <unk> and at the segment of growth of the global Fabry market made of patients with amenable mutation.

Sebastian: The potential to reach up to $1 billion in annual revenue by the end of the decade.

Sebastian Martel: We would expect a similar trend to occur this year. For example, we expect Q1 sales of the current year to be around 22% of two-year sales for Gallup. I'll slide in.

Sebastian: We anticipate sustained growth in 2024 and beyond to be driven by several key growth drivers.

First continuing to increase patient identification through ongoing medical litigation screening and improve diagnosis.

Sebastian Martel: We know that there is significant patient demand for Galapagos and that the segment of the global fabric market made of patients with a minimal mutation has the potential to reach up to a billion dollars in annual revenue by the end of the decade. We anticipate sustained growth in 2024 and beyond, to be driven by several key growth types. First, continuing to increase patient identification through ongoing medical education, screening, and improved diagnosis. As you know, Fabry is one of the most under-diagnosed rare diseases. So the more patients are identified, the more patients can be eligible for GALA.

Sebastian: As you know our company is one of the most underdiagnosed rare disease. So there are more patients are identified that more patients can be eligible for gasoline.

Sebastian: Second.

Sebastian: The other piece is continuing to drive <unk> market share of treated patients through continued commercial execution.

Sebastian: As noted <unk> currently at 60% to 65% of the global market.

Sebastian: We are seeing in our most mature market that we can reach up to about 85 or 90% of market share. So we know that there is a potential to reach those levels in the global market share as well.

Sebastian: Importantly, yes Bradley highlighted earlier this is a robustly growing February market with a significant portion of growth coming from finding new patients and penetrating into the diagnosed untreated population.

Sebastian Martel: The other piece is continuing to drive Gallup's market share of treating patients through continued commercial execution. As noted, Gallup currently has 60 to 65% of the global unable market. But what we're seeing in our most mature market is that we can reach up to about 85 or 90% of market share. So we know that there's a potential to reach those levels in the global market share as well. Importantly, as Bradley highlighted earlier, this is a robustly growing fabric market with a significant portion of growth coming from finding new patients and penetrating the diagnosed untreated population. Just within the 2,400 patients on Galaford at the end of 2023, about 1,400 of those individuals were naive to any treatment before coming on Galaford.

Sebastian: Just within the 2004 hundred patients who've got pulled at the end of 2023 about 1400 of these individuals were naive to any treatments before coming on Garfield.

Sebastian: We've been successful in finding new patients through newborn screening and other diagnostic initiatives.

Sebastian: As well as artificial intelligence through our partnership with <unk>.

Sebastian: And again all of these efforts are supported by solid compliance and adherence rates through physicians and patient education and support programs with.

Sebastian: We continue to make progress on expanding <unk> into new markets and extended the labels.

Sebastian: Still some markets in that time, so I mean, the middle Eastern Asia Pacific regions were GAAP will either newly reimbursed or we expect reimbursements.

Sebastian Martel: We've been successful in finding new patients through newborn screening and other diagnostic initiatives, as well as artificial intelligence through our partnership with OMR. And again, all of these efforts are supported by solid compliance and adherence rates through physicians and patient education and support programs. We'll continue to make progress on expanding Galafold into new markets and expanding the labels. There are still some markets in the TAM, some in the Middle East and Asia-Pacific regions where Galafold is either newly reimbursed, or we expect it to be reimbursed. Also important to note here, we often have exclusivity in the US and Europe, in addition to our 57 orange book-listed patents, 41 of which provide protection into 2038 and beyond, including 11 compositions of batter packs.

Sebastian: Also important to note here we are.

Sebastian: Have orphan exclusivity in the U S and Europe.

Sebastian: In addition throughout 57 Orange book listed patents.

Sebastian: 41 of which provide protection into 2038 and beyond including 11 composition of matter patents.

Sebastian: This provides us with the opportunity to provide access to <unk> globally for a long time to come we intend to continue to protect and enforce our broad intellectual property rights.

Sebastian: Looking ahead, we expect steady double digit growth organically throughout 2024, and we remain confident that our strong IP protection.

Sebastian: We will provide guidance for the long runway well into the next decade.

Sebastian: Turning now on to competitive <unk> on slide 10.

Sebastian Martel: This provides us with the opportunity to provide access to GALIFOR globally for a long time to come. We intend to continue to protect and enforce our broad intellectual property rights. Looking ahead, we expect steady double-digit growth for Galafold throughout 2024, and we remain confident that our strong IP protection will provide Gallup all the long run way well into the next decade. Turning now on to Pompe disease, on slide 10, we outline our global launch progress with the Pombili Channel Program. For the full year 2023, Pombilich and Opolda reported revenue of $11.6 million for the full year and $8.5 million for the fourth quarter.

Sebastian: We outline our global launch progress with commodity general pullback.

Sebastian: So full year 2023 completion of pull down our reported revenue reached $11 6 million.

For the full year and $8 5 million for the fourth quarter.

Sebastian: At the beginning of the year.

Sebastian: 24, 120 patients wound treatments or scheduled for treatment of which approximately 105, where from a clinical trial and early access programs in.

Sebastian: <unk> 15 from competitor ERP domain to treatment with.

Sebastian: We've been pleased with the continued demand for this new therapy.

Sebastian: As Brett said and as anticipated I would say the rate of new commercial patients coming onto <unk> pull that continues to increase.

Sebastian Martel: At the beginning of the year 2024, 120 patients were on treatment or scheduled for treatment, of which approximately 105 were from our clinical trial and early access programs and 15 from competitor ERTs on naive treatment. We've been pleased with the continued demand for this new therapy. As Brad said, and as anticipated, I would say, the rate of new commercial patients coming onto Pombiliti Anapoda continues to increase across all three markets. In the U.S., we continue to see a majority of patients switching from Nexiozyme, roughly about 75% of the switches we've seen, and the remaining 25% coming from Lumizyme.

Sebastian: All three markets.

In the U S. We continue to see a majority of patients switching from Nick just time, roughly about 75% of the switches, we've seen and the remaining 25% coming from <unk>.

Sebastian: Beans, with switching patients proportionately from both products.

Sebastian: Outside of the U S. We're seeing patients switching from all three segments.

Sebastian: <unk> negative dime in the naive population.

Sebastian: Proportional rate exactly what we won't be seeing at this stage.

Sebastian: Our launches leverage our highly experienced cross functional team and we've had great outreach with Kols.

Sebastian Martel: This means we're switching patients proportionally from both partners outside of the US. We're seeing patients switch from all three segments, for Myozyme, Nexiozyme, and the Nave population at a proportional rate. Exactly what we want to be seeing at this stage.

Sebastian: We're seeing an increase in depth as well as breadth of prescribers across all three markets and in particular see a growing number of prescribers, we're not talked about clinical studies or expanded access programs.

Sebastian Martel: Our launch has leveraged our highly experienced cross-functional teams, and we've had great outreach with KOL. We're seeing an increase in depth as well as breadth of prescribers across all three markets, and in particular, we see a growing number of prescribers who are not part of our clinical studies or expanded access program.

Sebastian: All core treating centers have been engaged with and we have had very positive feedback from hcp's and other stakeholders to our business approach support and patient focus.

Sebastian: Finally, we signed an important metric to track is on progress with access and reimbursement.

Sebastian Martel: All core treating centers have been engaged, and we have had very positive feedback from HCPs and other stakeholders as to our business approach, support, and patient focus. Finally, we find an important metric to track is our progress with access and reimbursement. We have a highly experienced team who are engaging in positive conversations with payers to demonstrate the value of mobility and pull-down. In the U.S., and with the start of the new year, many large payers have already put POMBILI-Genopoda on their respective formularies. And we've also seen strong acceptance by Medicare Medicaid as well. Today, we're launched in Germany, the UK, the US, and Austria.

Sebastian: We have a highly experienced team are engaging and positive conversations with payers to demonstrate the value of committed general pull down in.

Sebastian: In the U S and with the stop of the new year. Many of the large payers have already put promptly to general pull that onto their respective formularies.

Sebastian: And we've also seen strong acceptance by Medicare Medicaid as well.

Sebastian: Today were launched in Germany, the UK, the U S and Austria.

Sebastian: Also in active pricing and reimbursement discussions with additional major European markets as we focus on securing broad patient access throughout the EU.

Sebastian: More than 10 reimbursement dossier have been submitted.

Sebastian: Overall, we are starting up that you are strong and we're very pleased with the building momentum on patient demand.

Sebastian Martel: We're also in active pricing and reimbursement discussions with additional major European markets as we focus on securing broad patient access throughout the EU. More than 10 reimbursement dossiers have been submitted. Overall, we're starting off the year strong, and we're very pleased with the building momentum on patient demand. Throughout the course of 2024, our focus will be on maximizing the number of patients on therapy by year-end. So, in summary, we're very pleased with the launches of Pongbili Til Kulda across all the first wave countries.

Over the course of 2024, our focus will be on maximizing the number of patients on therapy by year end.

Sebastian: So in summary, we're very pleased with the launches of completed Youll pulled out across all of the first wave countries. The strength of our clinical data have a depth of experience and talent. We have at amicus gives us great confidence in our ability to make a real difference to people living with comparisons.

Sebastian: We believe that makes this is in a great position for our second successful launch.

Sebastian Martel: The strength of our clinical data, the depth of experience, and talent we have at Amicus gives us great confidence in our ability to make a real difference to people living with Pompidou. We believe Amicus is in a great position for our second successful launch. And with that, I will hand the call over to Jeff Castelli, our Chief Development Officer, to discuss the ongoing clinical studies, as well as regulatory timelines.

Sebastian: And with that I'll hand, the call over to Jessica <unk>, Our Chief development officer to discuss the ongoing technical studies as well as regulatory timelines.

Sebastian: Yes.

Jessica: Thank you Sebastian and good morning, everyone.

Jessica: On slide 11, we remind everyone that we continue to build the body of evidence for homebuilding up Florida through our ongoing clinical studies.

Jessica: We also continued to execute on expanding commercial access through regulatory submissions.

Jessica: As we entered the second phase of launch in addition to the various reimbursement dossiers that we have or that we are in the process of submitting we also have multiple ongoing or planned regulatory submissions for marketing approval in new geographies throughout this year.

Jeffrey P. Castelli: Thank you, Sebastian, and good morning, everyone. On slide 11, we remind everyone that we continue to build the body of evidence for POMBILITY and OPFOLDA through our ongoing clinical studies, as we also continue to execute on expanding commercial access through regulatory submission. As we enter the second phase of launch, in addition to the various reimbursement dossiers that we have or that we are in the process of submitting, we also have multiple ongoing or planned regulatory submissions for marking and approval in new geographies throughout this year. For the younger Pompeii community, we continue to enroll the ongoing open-label ZIP study for children living with late-onset Pompeii disease and the open-label ROSELLA study for children living with infant

Jessica: For the younger content community, we continue to enroll the ongoing open label study for children living with late onset Pompe disease and the open label <unk> study for children living with infantile onset pompe.

Jessica: We see this as an important opportunity to support label expansion into these patient segments and provide access to these kids as much a need in the years ahead.

Jessica: Through ongoing clinical studies and the amicus pumped a registry, we expect to continue generating evidenced on our differentiated mechanism of action and long term data supporting the impacted from building alcoa across endpoints and patient populations.

Jeffrey P. Castelli: We see this as an important opportunity to support label expansions into these patient segments and provide access to these kids much in need in the years ahead. Through ongoing clinical studies and the Amicus Pompei Registry, we expect to continue generating evidence on our differentiated mechanism of action and long-term data supporting the impact of pump building output across endpoints and patient population. I am very excited to announce that we have actually now begun enrolling patients into the Amicus Pompeii Registry Global. And here in February, we once again had a very engaging conference and a significant presence at the 20th Annual World Symposium that was held in San Diego.

Jessica: Very excited to announce that we have actually now begun enrolling patients into the amicus Pompeii registry globally.

Jessica: And here in February we once again had a very engaging conference and a significant presence at the 20th annual World Symposium that was held in San Diego.

Jessica: Amicus had 11 posters in an oral presentation, highlighting our continued work across fabry and Pompe disease.

Jessica: Of note amicus was honored with the World Symposium in 2024, New treatment award for possibility in Florida, which recognizes companies that have made important achievements in advance of treatments for lysosomal diseases.

Jessica: <unk> regulatory approval.

It's an honor to achieve an award based on our scientific innovation, but even more importantly, the meaningful difference we can make in the lives of so many people living with these rare diseases.

Jeffrey P. Castelli: Amicus had 11 posters and an oral presentation highlighting our continued work across Fabry and Pompe disease. Of note, Amicus was honored with the World Symposium 2024 New Treatment Award for Palmdodeon Alpholda, which recognizes companies that have made important achievements in advancing treatments for lysosomal diseases and have obtained regulatory approval. It's an honor to achieve an award based on our scientific innovation, but even more importantly, the meaningful difference we can make in the lives of so many people living with these rare diseases. Finally, as highlighted in the pipeline slide in the appendix, for our earlier stage pipeline, we continue to focus on novel approaches for Fabbrae and Pompeii, including delivery of our engineered GLA and GAA transgenes and a next-generation Fab With that, I would now turn the call over to Simon Harford, our Chief Financial Officer, to review our financial results, guidance, and outlook.

Jessica: 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 Pompe <unk>.

Jessica: Including delivery of our engineered GLA and GAA Trans genes and a next generation Fabry chaperone.

Jessica: With that I would like to now turn the call over to Simon Harford, Our Chief Financial Officer to review, our financial results guidance and outlook Simon.

Simon Harford: Thank you Jeff.

Financial overview begins on slide 13, with our income statement for the full year ending December 31 2023.

Simon Harford: We had a very successful yet full year, achieving total revenue of $399 million, which is a 21% increase over the same period in 2022 at constant exchange rates revenue grew strongly 20%.

Simon Harford: Thank you Jeff. Our financial overview begins on slide 13, with our income statement for the full year ending December 31st, 2023. We had a very successful full year, achieving total revenue of $399 million, which is a 21% increase over the same period in 2022. At constant exchange rates, revenue grew strongly by 20%.

Simon Harford: The global geographic breakdown of total revenue for 2023 consisted of $250 million or 63% of revenue generated outside the United States and the remaining $147 million.

Simon Harford: 37% coming from within the U S.

Simon Harford: Cost of goods sold as a percentage of net sales was nine 3% for the full year 2023, as compared to 11, 7% for the prior year periods.

Simon Harford: The global geographic breakdown of total revenue for 2023 consisted of $250 million, or 63% of revenue generated outside the United States, and the remaining $147 million, or 37%, coming from within the U.S. Cost of goods sold as a percentage of net sales was 9.3% for the full year 2023, as compared to 11.7% for the prior year period. Total Gap Operating Expenses decreased to $439 million in 2023 as compared to $503 million in 2022, a decrease of 13%. On a non-GAAP basis, total operating expenses decreased to $342 million for 2023 as compared to $413 million in the prior period, a decrease of 17 percent. We define non-GAAP operating expense as research and development and SG&A expense, excluding stock-based compensation, loss on impairment of assets, changes in fair value, contingent consideration, and depreciation. Net loss for the full year 2023 was reduced to $152 million, or $0.51 per share, including a $14 million, or $0.05 per share expense related to the extinguishment of prior debt following our Blackstone refinance. That compared to a net loss of $237 million, or 82 cents per share, for 2022.

Simon Harford: Total GAAP operating expenses decreased to 439 million in 2023 as compared to $503 million in 2022, a decrease of 13%.

Simon Harford: On a non-GAAP basis total operating expenses decreased to $342 million for 2023 as compared to $413 million in the prior periods a decrease of 17%.

Simon Harford: We define non-GAAP operating expense as research and development and SG&A expenses.

Simon Harford: Diluting stock based compensation loss on impairment of assets changes in fair value of contingent consideration and depreciation.

Simon Harford: Net loss for the full year 2023 reduced to $152 million or 51 cents per share, including a $14 million or <unk> <unk> per share expense related to the extinguishment of prior debt following our Blackstone refinancing.

Simon Harford: That compared to a net loss of $237 million or <unk> <unk> per share for 2022 in.

Simon Harford: In the fourth quarter as Bradley mentioned, we achieved our goal of non-GAAP profitability, which was $2 $6 million cash.

Simon Harford: In the fourth quarter, as Bradley mentioned, we achieved our goal of non-gap profitability, which was $2.6 million. Cash, Cash Equivalents, and Marketable Securities were $286 million as of December 31, 2023, and that compared with $294 million at the end of 2022. Turning now to slide 14, I'm pleased to share our full-year Garlafold revenue growth guidance of 11 to 16 percent at a constant exchange rate. Our full year 2023 non-GAAP operating expense guidance is $345 to $365 million. We are pleased to share that amidst a launch year, we have kept operating expense growth minimal. As a reminder, we continue to have R&D commitments, including registry studies in both Fabry and Pompeii, the ongoing Pompeii Phase III study in countries not yet reimbursed, as well as next-generation manufacturing for Pombility. There is very minimal investment in preclinical activities, which we expect to continue in 2024.

Simon Harford: Cash cash equivalents and marketable securities with $286 million as of December 31st 2023.

Simon Harford: Compared with $294 million at the end of 2022.

Simon Harford: Turning now to slide 14, I am pleased to share our full year <unk> revenue growth guidance of 11% to 16% at constant exchange rates, our full year 2023, non-GAAP operating expense guidance.

Simon Harford: $345 million to $365 million, we are pleased to share that the midst launch yes, we have kept operating expense growth minimal.

Simon Harford: As a reminder, we continue to have R&D commitments, including registry studies in both Fabry and Pompe pay the ongoing Pompeii phase III study in countries not yet reimbursed as well as next generational manufacturing for possibility.

Simon Harford: There is very minimal investment and preclinical activities, which we expect to continue in 2024.

Simon Harford: Following the achievement of non-GAAP profitability in Q4 of 2023, we anticipate to achieve non-GAAP profitability for the full year of 2024, driven by our first year of well over a half billion dollars in combined revenue, as well as continued prudent expense management. As we think about profitability throughout the year, we anticipate a similar non-gap profit in Q1'24 as we saw in Q4'23, and non-gap profit is expected to build sequentially quarter over quarter after that. And with that, I will turn the call back over to Bradley for our closing remarks. Great, thanks, Simon, Jeff, and Sebastian. And as well, a huge thanks to all of our employees around the world who, I know, continue to work tirelessly for people living with rare diseases. Look, in 2024, we will continue to drive significant top-line revenue growth supported by sustained double-digit Galliford performance. The successful ongoing global commercial launch of Pondility Abfolda, which puts us on track for our first full year of non-gap profitability

Simon Harford: Following the achievement of non-GAAP profitability in Q4 of 2003, we anticipate to achieve non-GAAP profitability for the full year of 2024, driven by our first year of well over half a billion dollars in combined revenue as well as continued prudent.

Simon Harford: <unk> management as we think about profitability throughout the year, we anticipate a similar non-GAAP profit in Q1 'twenty four as we saw in Q4 2003.

Simon Harford: And non-GAAP profit is expected to build sequentially quarter over quarter after that.

Simon Harford: With that let me turn the call back over to Bradley thought closing remarks.

Bradley Lewis Campbell: Great. Thanks, Simon Jeff Sebastian.

Speaker Change: As well a huge thanks to all of our employees around the world too I know continued to work tirelessly for people living with rare diseases.

Bradley Lewis Campbell: Look in 2024, we will continue to drive significant topline revenue growth supported by sustained double digit <unk> performance.

Bradley Lewis Campbell: Successful ongoing global commercial launch upon building up fold, which puts us on track for our first full year of non-GAAP profitability as I said in my opening comments amicus is that a major inflection point, we are strongly positioned to continue to advance our mission of delivering groundbreaking new medicines to thousands of people living with rare diseases around the world.

Bradley Lewis Campbell: As I said in my opening comments, Amicus is at a major inflection point. We are strongly positioned to continue to advance our mission of delivering groundbreaking new medicines to thousands of people living with rare diseases around the world and creating value for our shareholders. With that, operator, we can now open up the call. Thank you. Ladies and gentlemen, if you have a question, please press star one one on your touchtone telephone.

Speaker Change: And creating value for our shareholders with that operator, we can now open up the call to questions.

Speaker Change: Thank you ladies and gentlemen, if you have a question. Please press star one 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.

Operator: 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 first question. And our first question comes from Anupam Rama of J.P. Morgan. Hi, thank you for taking the question. This is actually Malcolm Kuno on for Anupam.

Speaker Change: One moment for your first question.

And our first question comes from <unk> Rama of Jpmorgan.

Rama: Hi, Thank you for taking the question. This is actually now.

Rama: For Auto club.

Operator: So in the U.S., with regard to Pompeii, are you seeing switches from patients that are clearly progressing or patients who are stable but just not gaining a benefit from the current treatment? Yeah, great question. I think the answer is a little bit of both.

Rama: So in the U S with regard to pump PE are you seeing switches for patients that are clearly progressing or patients who are stable, but just not gaining a benefit from their current treatment.

Speaker Change: Yes, Great question I think the answer is a little bit of both so we know that some patients we've heard I should say that some patients.

Bradley Lewis Campbell: So, you know, we know that some patients we've heard, I should say that some patients who were new commercial patients not in clinical studies, heard by word of mouth or through relatives who might be participating in those studies about how well they thought that those patients did in the study. And so those patients, I think, were eager to start Pomvilli and Upfolda kind of regardless of their own status. And then I know that others, I'm sure, were in a declining phase and therefore wanted to switch with the hopes of seeing different outcomes, which, of course, is per the label where the drug is indicated for patients who are not improving on current therapies. So I think we're seeing healthy dynamics from both of those sectors. Great.

Speaker Change: Who were new commercial patients not in clinical studies.

Speaker Change: <unk> heard by word of mouth or through relatives, who might be participating in those studies about how well they thought that those patients in the study and so those patients I think we're eager to start possibility not pulled out kind of regardless regardless of their own status and then I know that others I'm sure. We're in a declining phase and therefore wanted to switch with the hopes of.

Speaker Change: I've seen different outcomes, which of course as per the label.

Speaker Change: The drug is indicated for patients who are not improving on current therapy. So I think we're seeing healthy dynamics from both of those segments.

Bradley Lewis Campbell: Thanks, Brad. Thank you. One moment for our next question. And our next question comes from Ritu Baral of TD Cowan. Good morning, guys. Thanks for taking the question. To drill down a little further on that, Brad, and Sebastian. How are you guys?

Speaker Change: Great. Thanks, Brett.

Speaker Change: Thank you one moment for our next question.

Ritu Baral: And our next question comes from Ritu barrel of TD Cowen.

Ritu Baral: Good morning, guys. Thanks for taking the question.

Ritu Baral: Drill down a little further on that.

Ritu Baral: Brad.

Ritu Baral: Last one how are you guys.

Bradley Lewis Campbell: seeing clinicians in the field define the decliners. You know, the two symposiums at World, both yours and your competitors, I think spent a lot of time defining decliners, defining stable. What do you see in your conversations during the conference on the tools that clinicians are using to define that in their own heads? And have you done any market research on segmenting the population into sort of that definition of decliner, that definition of stable, as you see it now? Sure, yeah, a great question, Ritu. Thanks very much.

Ritu Baral: Clinicians in the field.

Ritu Baral: The decliners.

Ritu Baral: The two symposiums at World, both yours and your competitors.

Ritu Baral: A lot of time on defining decliners defining stable what do you see in your conversations during the conference.

Ritu Baral: The tools that clinicians are using to define that on their own hub.

Ritu Baral: Have you done any market research.

Ritu Baral: On segmentation of the population into sort of definition, a decliner that population of stable.

Sure, Yes, great Great question Ritu, Thanks, very much I'm going to let Jeff talk to the first part of your question, which is sort of what are the tools and the markers that clinicians might use to follow.

Jeffrey P. Castelli: I'm gonna let Jeff talk to the first part of your question, which is sort of what are the tools and the markers that clinicians might use to follow or determine whether a patient is stable or declining? I would remind you, though, that the indication statement is for patients not improving on therapy, which means that either stable or declining patients are eligible for us. From a market research perspective, the work that we did leading into the launch suggests that about 50% of patients would historically be defined as stable, although I think Jeff will talk us through why, in fact, they may be subtly declining as well. About a quarter of patients are clearly declining, and about a quarter of patients are likely in some sort of improvement phase, which is what we would expect based on historical data. But Jeff, maybe talk through how physicians, the obvious ways that they would look for declining patients, and then maybe the not so obvious, more subtle but equally important ways that patients may be declining. Yeah, thanks, Brad.

Jeff: Herman whether a patient is stable or declining I would remind you though that the.

Jeff: The indication statements for patients not improving on therapy, which means that either stable or decline in patients.

Jeff: Are eligible for us from a market research perspective.

Jeff: Work that we did leading into the launch suggests that about 50% of patients would historically be defined as stable. Although I think Jeff will talk us through why in fact, they may be subtly declining as well.

Jeff: About a quarter of patients are clearly declining and about a quarter of patients are likely in some sort of improvement phase, which is what we would expect based on historical data, but Jeff maybe talk through how physicians the obvious ways that they would look for declining patients and then maybe the not so obvious.

Jeff: More subtle, but equally important ways that patients may be declining.

Jeffrey P. Castelli: And thank you for the question, Ritu. So, first of all, just in terms of, you know, follow up, typically, the cadence for Pompe patients is about every six months; they come in and visit with their, you know, primary treating physician and care team. They do measure the quantitative endpoints, you know, on motor function, respiratory function, that are cited in the trials, things like six minute walk distance, force vital capacity, and other measures of motor and respiratory parameters.

Jeff: Yes, Thanks, Brian and thank you for the question Tim.

Jeff: So first of all just in terms of the <unk>.

Jeff: Follow up typically the cadence for Pompe patients is about every six months they come in and visit with their primary trading position and care team.

Brian: To measure the quantitative endpoints on motor function and respiratory function that are cited in the trials things like six minute walk test and supports vital capacity other measures of motor function.

Brian: Respiratory parameters for what we see is that a lot of what they do is it really more qualitative in terms of some of the formal questionnaires, but really just asking the patient how are they doing during activities of daily living.

Jeffrey P. Castelli: What we see is that a lot of what they do is really more qualitative in terms of, you know, some of the formal questionnaires, but really just, you know, asking the patient, how are they doing during activities of daily living. And then, of course, they also do look at biomarkers. Depending on the site, they may put more or less importance on the biomarkers, very much a holistic assessment of different parameters. And what we're learning is that it actually does differ across sites and across countries. And one of the things that we're looking to do is trying to help the community of physicians to try to standardize some of those assessments more moving forward. So in terms of, as Brad mentioned, as we look in the US, for example, it's about 25%.

Brian: And then of course, they also do look at Biomarkers, depending on the site. They may have put more or less importance on the biomarker. So.

Brian: It's very much a holistic assessment of different parameters and what we're learning is it actually does differ across sites and across countries and one of the things that we're looking to do is trying to help the cause.

Brian: <unk> physicians look to try to standardize some of those assessments more moving forward.

So in terms of as Brad mentioned.

Brian: As we look in the U S. For example, it's about 25% we would estimate improving 25% clearly worsening and then that 50% in the middle what we're seeing is physicians now have more treatment options and are really digging into how patients are doing to try to say who is declining who has the bulk of it was improving.

Jeffrey P. Castelli: We would estimate it improving by 25%, clearly worsening. And then that 50% in the middle, what we're seeing as physicians now have more treatment options and are really digging into how patients are doing to try to say who is declining, who is stable, who is improving. We're starting to hear that many of those patients that they might have perceived as stable, as they do that deeper holistic assessment, they're finding out that while the six-minute walk or four-spinal capacity might be generally stable, patients are reporting having So we do think a significant chunk of that 50% sort of stable middle patient segment is actually declining when physicians start to dig in more holistically. So it's definitely an evolving space in terms of how physicians monitor patients, especially related to switching. There's been a lot of different symposiums and meetings on this topic.

Brian: We're starting to hear that many of those patients that they might have perceived as stable as they do do that deeper holistic assessment. They are finding out that while six minute walk or forced vital capacity might be generally stable that patients are reporting having a much harder time climbing stairs are going out on doing chores or their fatigue when there.

Brian: Sleeping so we do think that a significant chunk of that 50% is sort of stable middle patient segment is actually declining.

Brian: Physicians start to dig in more holistically. So it's definitely an evolving space in terms of how physicians monitor patients, especially related to switching theres been a lot of different symposium in meetings on this topic, so something that we'll certainly keep an eye on.

Bradley Lewis Campbell: So something that we'll certainly keep an eye on. But as I said, it's really this holistic approach. It's not one or two parameters that they use to define sort of status and when patients might switch and how they do after the switch. Thanks, Jeff. I would think that's really the exciting opportunity Ritu is for us to challenge the expectations of therapy. Now, as Jeff said, we have more choices for physicians and patients. Great, thanks.

Brian: But as I said, it's really this holistic approach, it's not one or two parameters that they use to define sort of status and when patients might switch and how they do after switch.

Speaker Change: Thanks, Jeff I would think thats really the exciting opportunity with two is for us to challenge the expectations of therapy.

Speaker Change: Now that as Jeff said, we have more choices for physicians and patients.

Jeff: Great. Thanks.

Bradley Lewis Campbell: Thank you. One moment for our next question, and our next question comes from Degan Ha of Stiefel. Good morning, guys.

Speaker Change: Thank you one moment for our next question.

And our next question comes from Jacob <unk> of Stifel.

Bradley Lewis Campbell: Thanks for taking our question. On Pompeii, if you can just remind us of the 15 that were new and not part of the expanded access for clinical trial, did you guys ever break them down in terms of geographies?

Jacob: Good morning, guys. Thanks for taking our question on Pompeii.

Jacob: If you can just remind us on the <unk> that were new and not part of the expanded access to our clinical trial did you guys ever break them down in terms of geographies and I guess, what I'm thinking about is 2020 for how we should think about the cadence of patient onboarding.

Bradley Lewis Campbell: And I guess what I'm thinking about is 2024, and how we should think about the cadence of patient onboarding given that you have these multiple launches going on. Thanks so much. Yeah, thanks, Dagon. What we had said at J.P. Morgan, which we'll remind you here, is that patients came roughly equally from each of the launch countries. Remember, we launched in Germany and the U.K. first, and then the U.S. later in the year, so we thought that was very positive. The other thing we saw, which we highlighted in the call here, is in each of those markets, we're pulling, and this trend continues, kind of proportionally from the patients who are treated on the various medications. So in the U.S., as Sebastian highlighted, the majority of our patients are coming from Nexviazime. That makes sense, because the majority of patients in the U.S. are now treated with Nexviazime, and then in Europe, we're pulling from both Lumizime as well as Nexviazime and increasingly naive patients, which is an important segment there as well.

Speaker Change: Given that you have these multiple largest going on thanks, so much.

Speaker Change: Yes, Thanks, Dae gon, what we had said at Jpmorgan, which we'll remind you here is those patients came roughly equally from each of the launch countries remember, we launched in Germany, and the U K first and then the U S. Later in the year. So we thought that was very positive. The other thing we saw which we highlighted on the call here is in each of those markets.

Speaker Change: We're pulling and this trend continues we're pulling kind of proportionately from.

Speaker Change: The patients who are treated on the various medications. So in the U S is Sebastian highlighted the majority of our patients are coming from <unk> that makes sense because the majority of patients in the U S are now treated with <unk> and then in Europe, we're pulling from both <unk> as well as <unk> and increasingly naive patients, which is an important segment there as well.

Bradley Lewis Campbell: And then to your last point, I think the color we provided here is that we're seeing the rate of new patient starts increasing at an increasing rate. So the first two months of the year have been a faster net new patient acquisition versus the months over the course of the launch last year. So all of those things point to continued building momentum and give great confidence in the launch. But any visibility on the ex-US versus US kind of cadence of growth that you anticipate this year? Yeah, I think you'll see just because the US is such a larger market, you'll start to see, you know, more patients coming on board in the United States. I think that's just a volume point.

Speaker Change: And then to your last point I think.

Speaker Change: The color. We provided here is that we're seeing the rate of new patient starts increasing at an increasing rate. So the first two months of the year and then faster net new patient acquisition versus the months over the course of the launch last year. So all of those things for us point to continued building momentum.

Speaker Change: Give us great confidence in the launch.

But any visibility on the ex U S versus U S kind of cadence of growth that you anticipate this year.

Speaker Change: Yes, I think youll see just because of the U S is such a larger market youll start to see more patients coming on in the United States I think Thats, just a volume point and I think typically what we'll see is that will that will carry us through the various markets the bigger markets add more patients to bring on and Youll start to see that play out.

Bradley Lewis Campbell: And I think, you know, typically, what we'll see is that will power that will carry us through the various markets, the bigger markets have more patients to bring in, and you'll start to see that play out. Excellent. Thank you very much.

Speaker Change: Excellent. Thank you very much.

Bradley Lewis Campbell: Thank you. Please take a moment for our next question. And our next question comes from Joseph Schwartz of Leering Partners. Thanks so much, and congratulations on the progress. It's interesting that you're getting... So, I was wondering if you could talk about why that might be and maybe give us some color in terms of the proportion of each of those subsegments that and MyPT are not improving, and yeah, just give us any color in terms of that dynamic.

Speaker Change: Thank you one moment for our next question.

Speaker Change: And our next question comes from Joseph Schwartz Leerink partners.

Joseph Patrick Schwartz: Thanks, so much and congrats on the progress.

Joseph Patrick Schwartz: Its interesting that youre getting more switches from <unk>.

Joseph Patrick Schwartz: So I was wondering if you could talk about why that might be in.

Joseph Patrick Schwartz: Maybe give us some color in terms of the proportion of each of those sub segments.

Joseph Patrick Schwartz: Do you think might be deemed.

Joseph Patrick Schwartz: To not be improving.

Joseph Patrick Schwartz: And just to give us any color in terms of that.

Bradley Lewis Campbell: That would be helpful. Thank you. Yeah, thanks, Joe. I think, you know, one of the big questions that everybody had was, boy, in the markets where Nexavizine has been launched the longest, well, maybe those patients are going to be too sticky, and you'll have a really hard time penetrating those markets. I think what we've seen, you know, it's still early days, but what we've seen is, no, in fact, as we said, the majority of the patients in the United States, and that has continued into this year, are coming from nexavizine. So I think what it really is, physicians and patients are focused much more on how they're doing, as Jeff described, versus what medication they're on. And so if a patient is stable or declining, they may be looking for better outcomes. And with choice, you have a chance to challenge those outcomes.

Joseph Patrick Schwartz: Dynamic that would be helpful. Thank you.

Speaker Change: Yeah, Thanks, Joe I think.

Joe: One of the big questions that everybody had was boy in the markets, where <unk> has been launched the longest well maybe those patients they're going to be sticky and youll have a really hard time penetrating into those those.

Speaker Change: Those markets I think what we've seen it's still early days, but what we've seen is no in fact.

Speaker Change: We're as we said the majority of the patients in United States and that has continued into this year are coming from <unk>. So I think what it really is.

Physicians and patients are focused much more on how theyre doing as Jeff described versus what medication thereon and so if a patient is stable or declining.

Speaker Change: They may be looking for better outcomes and with choice you have the chance to challenge those outcomes. So I don't think that its.

Bradley Lewis Campbell: So I don't think that it's a question of, you know, which drug I am on. I think it's a question of how am I doing? And what are my expectations for therapy?

A question of which drug on my own and I think it's a question of how am I doing and what are my expectations for therapy, and that's where we're really excited to really challenge those expectations and as Jeff said drive that question for physicians and patients to think a lot more carefully about how they are performing on their current medication. So that's why I think we've seen so far in the.

Bradley Lewis Campbell: And that's where we're really excited to really challenge those expectations and, as Jeff said, drive that question for physicians and patients to think a lot more carefully about how they're performing on their current medication. So that's why I think we've seen, you know, so far in the U.S., again, with the majority of patients on nexviazime, we're switching, you know, the majority of our new patients are switching from nexviazime, although we are getting some lumizyme patients as well. And then outside the U.S., where it's still a majority market for lumizyme, we' So we think all those dynamics are really healthy and will continue. Brett, just, follow up if I could.

Speaker Change: U S again with the majority of.

Speaker Change: Patients on <unk> were switching to the majority of our new patients are switching from <unk>. Although we are getting some windows on patients as well and then outside the U S where it's still a majority luminaire market, where getting a majority of switches from <unk>. Although we are getting an exercise on patients and were getting naive patients as well. So we think all of those dynamics are really healthy.

Speaker Change: And we will continue.

Speaker Change: Brad just to follow up if I could does it indicate or could it indicated all of that.

Bradley Lewis Campbell: Does it indicate, or could it indicate at all, that patients are, that the next slides on patients are essentially, you know, self-selected, more severe, and there might be more people on lumizyme in the United States that are satisfied, and they might be harder to switch? I don't think that's necessarily the case.

Speaker Change: Patients on.

Speaker Change: The next one is on patients who are essentially self selecting to be more severe and there might be more people on Amazon in the United States that are satisfied and they might be harder to switch.

Brad: I don't think that's necessarily the case I think it is.

Bradley Lewis Campbell: I think it's likely that patients who have, you know, just started a medication, i.e., lumizyme or nexomyzyme, are going to be in a, you know, how are you doing mode for a period of time. And that's the kind of 25% of the segment that we talked about when we did the market research. You know, I think physicians say about 25% of their patients are in an improving phase. I don't think patients or physicians would necessarily look to switch those patients initially. It's really more of those stable or declining patients where we have a real opportunity. And again, we estimate that's about 75%. Very helpful. Thank you. Thanks, Joe.

Brad: Likely that patients who have <unk>.

Brad: Just started a magic medication I E <unk>.

Brad: Are going to be DNA.

Brad: How am I doing mode for a period of time and Thats that kind of 25% of the segment that we talked about when we did the market research I think physicians said about 25% of the patients are in an improving phase I don't think patients or physicians would necessarily look to switch those patients. Initially it's really more of those stable or declining patients, where we have a real opportunity in <unk>.

Brad: We estimate that's about 75% of the market.

Speaker Change: Very helpful. Thank you.

Bradley Lewis Campbell: Thank you. Please take a moment for our next question. And our next question comes from Ellie Murrell of UBS. Hey guys, congrats on all the progress and thanks for taking the question. If you could tell us a little bit more about Pompeii in terms of the numbers of centers or positions that you're targeting as the key prescriber base here, and I guess what proportion of these have already written a script for POMbility at Fulda?

Speaker Change: Thanks, Joe.

Speaker Change: One moment for your next question.

Speaker Change: And our next question comes from Ellie Merle of UBS.

Ellie Merle: Hey, guys. Congrats on all the progress and thanks for taking the question.

Ellie Merle: If you could talk a little bit more and pay in terms of the numbers of centers are positions that youre targeting at the key prescriber base here and there.

Ellie Merle: What proportion of these have already written a script for possibility for that.

Bradley Lewis Campbell: Like you mentioned that you're seeing increasing starts at an increasing rate. Are you seeing something similar in terms of the, you know, specific new positions? Or is it, you know, deepening of prescriptions from those sites that were already prescribing or involved in the clinical trials? Thanks. Yeah, great question, Ellie. So as Sebastian mentioned on the call, we're seeing both increasing breadth and depth. So it's kind of both of what you

Ellie Merle: Mike you mentioned that Youre seeing.

Ellie Merle: <unk> starts at an increasing rate are you seeing something similar in terms of.

Ellie Merle: Specific new physicians.

Ellie Merle: Or is it deepening our prescriptions from those sites that were already prescribing are involved in the clinical trial.

Mike: Yeah, Great question, So as Sebastian mentioned on the call, we're seeing both increasing breadth and depth. So it's kind of both of what you asked so.

Bradley Lewis Campbell: So the physicians who have already been writing prescriptions are continuing to write prescriptions. But then we're seeing an increasing number of centers, in particular in the U.S. and Germany, that weren't involved in the clinical trials who are now starting to write prescriptions as well. So both of those segments are improving. I would note that in the U.K., all of the centers participated in the EAMS program or the clinical trial program.

Mike: The physicians, who have already been writing.

Mike: Prescriptions are continuing to write prescriptions, but then we're seeing expanding numbers.

Mike: Centres in particular in the U S and Germany that were involved in the clinical trials, who are now starting to write prescriptions as well. So both of those segments are improving I would note in the U K all of the centers.

Mike: Participated in the <unk> program or the clinical trial program.

Bradley Lewis Campbell: And so they were already writing prescriptions and continue to do so. So I think it's a really healthy, you know, dynamics so far. And then just of the 120 patients that you mentioned at J.P. Morgan, I guess any color, and what proportion of these are already reimbursed now? Yeah, the majority of them are already reimbursed. I would say that, as a reminder, it takes about 90 days to go from prescription to commercial infusion at this point in time.

Mike: And so they all have were already writing prescriptions and continue to do so so I think really healthy dynamic so far.

Mike: And then just 120 patients that you mentioned at Jpmorgan I guess any color on what proportion of these are already reimbursed now.

Mike: Yes, the majority of them are already reimbursed I would say that.

Mike: As a reminder, it takes about 90 days to go from prescription to commercial infusion at this point in time, so that means the people who got their prescriptions in December and January who may still be in that process, but of course as we highlighted in the call. We're continuing to add more patients into the funnel, which is great and the other thing. We're seeing is that the newer commercial patients are going through the process.

Bradley Lewis Campbell: So that means the people who got their prescriptions in December and January may still be in that process. But, of course, as we highlighted in the call, we're continuing to add more patients into the funnel, which is great. And the other thing we're seeing is that the newer commercial patients are going through the process more quickly, as we continue to see Pondility Upfolda added to more and more formularies in the United States. And so we would continue to expect that number to come down as we progress through the launch. That was the same trend we saw, as you might remember, with Gallifold as well, where it sort of started in that 90-day range and then, over time, winnowed down to where we sit today, which is around 30 days. Thanks so much.

Mike: More quickly as we continue to see possibility uphold added to more and more formularies in the United States and so we would continue to expect that number to come down as we progress through the launch that was the same trend. We saw as you might remember with <unk> as well, where it sort of started in that 90 day range and then overtime winnowed down to where we sit today, which is <unk>.

Mike: Round 30 days.

Speaker Change: Great. Thanks, so much.

Bradley Lewis Campbell: Thank you. One moment for our next question. And our next question comes from Jeff Hung of Morgan Stanley. Thanks for taking my question. You talked about the seasonal patterns you've seen in the past with GALFOLDS going from Q4 to Q1. How should we think about the potential seasonal trends for POMBILITY and UPFOLDA? You know, would you expect them to move similarly?

Speaker Change: Thank you one moment for our next question.

Speaker Change: And our next question comes from Jeff Hung of Morgan Stanley.

Jeff Hung: Thanks for taking my question you talked about the seasonal patterns you've seen in the past with <unk> going from Q4 to Q1, how should we think about the potential seasonal trends for <unk> folder would you expect them to move similarly, or do you think that maybe it doesn't apply as much this year given that the launch it's still fairly early.

Bradley Lewis Campbell: Or do you think that maybe it doesn't apply as much this year, given that the launch is still fairly early? Thanks. Yeah, good question. I think probably the answer is too soon to tell. I think, you know, in a launch year, you would expect to continue to build momentum throughout the year, which is, I think, a natural phenomenon as you get more and more physicians and patients with experience as we add countries over the course of the year. So, you know, I guess the answer is too early to tell. As we are able to provide more color there, you know, going forward, we'll share that with you. But I think, in general, you could just expect momentum to build throughout the year, given it's a launch year.

Speaker Change: Yeah.

Speaker Change: Yeah. Good question I think probably the answer is too soon to tell I think in a launch year you would expect to continue to build momentum throughout the year, which is I think a natural.

Speaker Change: Phenomenon as you get more and more physicians and patients with experience as we add countries over the course of the year.

Speaker Change: So.

Speaker Change: I guess the answer is too early to tell as we are able to provide color there going forward of course, we'll share that with you but.

Speaker Change: I think in general you can just expect momentum to build throughout the year, given it's a launch year.

Bradley Lewis Campbell: Thank you. Thank you. One moment for the next question. And our next question comes from Kristen Kluska of Cantor Fitzgerald. Hi, good morning, everybody.

Speaker Change: Thank you.

Speaker Change: Thank you one moment for our next question.

Speaker Change: And our next question comes from Christian <unk> of Cantor Fitzgerald.

Christian: Hi, good morning, everybody.

Bradley Lewis Campbell: The patient diagnosis growth has been really impressive in Fabure, growing 70% from 2015 to 2023. So since part of this growth came from new therapies on the market, including your own, I'd like to know how you're thinking about some of the growth we might see in the Pompei market numbers with the recent launches. Yeah, thanks, Kristen. I think it's a phenomenon you typically see, right, which is when more companies are involved, when more therapeutic options are out there, I think you do tend to see an increased focus on diagnosis and treatment. So I wouldn't at all be surprised if that's part of the driver.

Christian: Patient diagnosis growth has been really impressive fabry growing 70% 2015 to 2023 part of this growth came from new therapies on the market, including Europe, I'm curious, how you're thinking about some of the growth we might.

Christian: The pump market numbers with the recent launch.

Speaker Change: Yeah. Thanks Kristen.

Speaker Change: I think it's a phenomenon you typically see right, which is when more companies are involved with more therapeutic options are out there I think you do tend to see an increased focus in diagnosis and treatment. So.

Speaker Change: Wouldn't that all be surprised if that's part of the driver of growth.

Bradley Lewis Campbell: Although we would also remark, and maybe, Jeff, you can share some of the details here, while not quite as underdiagnosed as Fabry is believed to be, we, in the last sort of 10 years, have seen significant evidence that, in fact, Pompeii is significantly more prevalent than what was originally reported in the literature. So, maybe, Jeff, talk a little bit about the data from newborn screening and from high-risk screening that would lead us to believe that Pompeii should also benefit from increased diagnosis over time. Yeah, thanks, Brad, and thanks for the question, Kristen. You know, there are a few dynamics important leading to better diagnosis for crossbred diseases. One is, you know, better education for physicians.

Speaker Change: Although we would also remark and maybe Jeff you can share some of the details here, while not quite as under diagnosed as fabry is believed to be.

Jeff Hung: The last sort of 10 years Ive seen significant evidence that in fact pompei is significantly more prevalent than what was originally reported in the literature. So maybe just talk a little bit about kind of the data excuse me from newborn screening and from high risk screening.

Jeff Hung: That would lead us to believe that Pompeii should also benefit from increased diagnosis overtime.

Speaker Change: Yes, Thanks, Brian and thanks for the question Kristen.

Speaker Change: There's a few dynamics importantly, leading to better diagnosis across rare diseases. One is.

Speaker Change: Better education of physicians and other importantly is.

Jeffrey P. Castelli: Another important issue is, you know, better diagnostic tools and low-cost genetic testing. There also are newborn screening initiatives in many countries and, importantly, in the U.S. So I think a lot of that is similar in Fabry and Pompe. You know, for Fabry, the extra benefit is the fact it's an excellent dominant disease, and you find one patient, and then you can screen the whole family. Pompe is more of a recessive disease, so you need to find each patient independently of themselves or siblings. But, you know, Pompe, similar to Fabry, when you look classically, it was thought to be one in 40,000, one in 50,000. Screening studies, both in newborns as well as looking at people just with elevated CK levels or limb cortomuscular dystrophy weakness type symptoms. Many of them have Pompe, and the actual estimate of Pompe is more like one in 15,000.

Speaker Change: Better diagnostic tools and low cost genetic testing there.

Speaker Change: They are also our newborn screening initiatives in many countries and importantly in the U S. So I think a lot of that is similar in fabry and Pompe <unk> for fabry. The extra benefit is the fact thats an excellent dominant disease and you find one patient and then you can screen out family Pompei is more of a recessive disease. So you need to find each patient independently in themselves or siblings.

Speaker Change: But.

Speaker Change: Pump a similar to fabry when you look classically it was thought to be 140000 150000.

Speaker Change: Screening studies, both newborn as well as looking at people just with elevated CK levels or limb girdle muscular dystrophy weakness type symptoms. Many of them have palm Bay and the actual estimate of pump a is more like one in 15000. So two to three times more common than what has been thought so a lot of patients still left to be diagnosed.

Jeffrey P. Castelli: So two to three times more common than what has been taught. So a lot of patients still left to be diagnosed in Pompe, a lot of the same underlying factors that are helping us diagnose more patients. I think Fabre is just sort of a little bit ramped up, you know, because of that factor.

Speaker Change: Pump a lot of the same underlying factors that are helping us diagnose more patients I think February is just sort of a little bit ramped up because of that excellent factor, but when you do expect a significant underlying growth of new patients coming on to therapy being diagnosed and definitely having multiple companies helping with.

Bradley Lewis Campbell: But we do expect significant underlying growth of new patients coming on to therapy, being diagnosed, and definitely having multiple companies helping with some of that. You know, diagnostic initiatives and education will certainly help drive that as well. Thank you. Thank you. Please take a moment for our next question. And our next question comes from Salveen Richter of Golden Sac. Hi, this is Srinath Rao and Prasalveen.

Speaker Change: Some of that.

Speaker Change: Diagnostic initiatives in education will certainly help drive that as well.

Speaker Change: Thank you.

Speaker Change: Thank you one moment for our next question.

Speaker Change: And our next question comes from <unk> Richter of Goldman Sachs.

Richter: Hi, This is <unk> on for Jonathan and thank you for taking our question.

Operator: Thank you for taking our question. You mentioned in the slide that your focus in 2024 is to maximize the number of patients on therapy for pulmonary enopphalda. Is there a target number that you have in mind in this aspect? And on payer coverage, particularly for Medicare and Medicaid, when do you expect to see coverage for pulmonary enopphalda there?

Rick Richter: You mentioned that you will focus on claims when he's poised to maximize the number of patients on therapy from the lithium up Florida.

Rick Richter: Is that target number that you have in mind in this aspect.

Rich: And on behalf of rich, particularly for Medicare and Medicaid.

Rick Richter: You expect to see published for public input on that.

Operator: Thank you. And I'm sorry, I just missed the first part of the question. I got the second one.

Speaker Change: Thank you.

Speaker Change: And I'm, sorry, I just missed the first part of the question I caught the second one can you repeat that.

Bradley Lewis Campbell: Can you repeat that? Sure. The first part I meant, given that your focus in 2024 is to maximize the number of patients on therapy and vulnerabilities in a folder, is there a target number that you have in mind that you want to achieve by the end of 2024? Uh, great question.

Speaker Change: Sure.

Speaker Change: First of all Damon.

Speaker Change: Given that you'll focus on training for us to maximize the number of patients on <unk> at the <unk>, Florida is there a target number that you havent, Brian that you want to achieve by the end of 'twenty four.

Damon: Great question as I think we've said before we can't give guidance at this point just given the fact that it's a launch year, but.

Bradley Lewis Campbell: As I think we've said before, we can't give guidance at this point just given the fact that it's a launch year, but, um... But, of course, we have our own internal targets, but I think, at this point, we're still in the early part of the launch, so it wouldn't be prudent to give guidance yet. As soon as we have more color, and I'm sure going into next year, we'll be able to provide, I think, more clarity and more guidance on revenue, in particular. And we will continue, on a quarterly basis, to report patients on therapy, as we have done, and that'll give you a flavor for the ramp. As it relates to your second question, yeah, the good news is, as Sebastian mentioned, in addition to being added to a number of major payer formularies, and that process will continue to increase over the first few weeks of the year here, we have already seen a number of patients accepted through both Medicare and Medicaid, so both government patients and private insurance patients are being accepted and getting through the process.

Brian: But of course, we have our own internal targets, but I think at this point, we're still in the early part of the launch so it would be prudent to give guidance yet.

Brian: Soon as we have more color and I'm sure going into next year, we'll be able to provide I think more clarity and more guidance on revenue in particular.

Speaker Change: We will continue from a quarterly basis to report patients on therapy, as we have done and I'll give you a flavor for the for the ramp.

Speaker Change: As it relates to your second question, Yes. Good news is as Sebastian mentioned in addition to being added added too.

Speaker Change: Number of major payer formularies and that process will continue to increase.

Speaker Change: Over the first few weeks of the year here, we have already seen a number of patients accepted through both Medicare and Medicaid and so both government patients and private insurance patients are being accepted and getting through the process.

Bradley Lewis Campbell: Thank you. Thank you. One moment for our next question. And our next question comes from Gil Blum of Needham. Hi, this is Ethan on behalf of Gil Blum.

Speaker Change: Thank you.

Speaker Change: Thank you one moment for our next question.

Speaker Change: And our next question comes from Gil Blum of Needham.

Speaker Change: Yeah, Hi, this is Ethan on for Gil and thank you for taking our question moving to the Fabry for a second and how important do you think your partnership with <unk>, one and the recent advances in AI and machine learning in general are to identify more patients and further drive end market penetration per gallon sold.

Operator: Thank you for taking our question. Moving to Fabry for a second, how important do you think your partnership with OM1 and the recent advances in AI and machine learning, in general, are to identifying more patients and further driving market penetration for Gallup? Thank you. Yeah, great question. I'll start, but then Jeff can give more color on that, that collaboration.

Bradley Lewis Campbell: Thank you.

Speaker Change: Yes, great question I'll start, but then Jeff can give more color on that that collaboration I think that.

Bradley Lewis Campbell: I think that, you know, machine learning will have a major impact on diagnosing all different sorts of patients. We're already starting to see some of that, but I'm really excited to see the outcome of that partnership, in particular FabRay. But again, I think that'll be an industry-wide and, hopefully, disease-wide phenomenon. Jeff, maybe give a little bit of color on what we're doing with OM1, the partnership, and what the intent is there. Yeah, thanks for the question.

Jeff: Machine learning will have a major impact on diagnosing.

Jeff: All different sorts of patients were already starting to see some of that.

Sebastian Martel: But I'm really excited to see the outcome of that that partnership in <unk>.

Bradley Lewis Campbell: Particular in fabry, but again, I think that'll be an industry wide and hopefully disease wide phenomenon, but Jeff maybe give a little bit of color on what we're doing with on one the partnership and what the intent is there.

Jeff: Yes, thanks for the question.

Jeffrey P. Castelli: So really, the approach with OM1 and other, you know, similar AI electronic medical record initiatives is to create sort of a medical record fingerprint for Fabry disease so that, you know, all of the health care records or people that have given consent within a health system can have their medical records and symptoms kind of scanned by the AI, and you basically flag a bunch of people that very might well have Fabry disease and don't know it. And then, you know, testing could be offered through different programs to those patients. We're very excited about, you know, we published on the development of the algorithm in Fabre. It looks like it can do a very good job, and we're actually just on the cusp here of testing it in a very large academic center here in the States.

Jeff: So it really the approach with <unk> and other similar AI electronic medical record initiatives is to create sort of medical records fingerprint of fabry disease. So that.

Jeff Hung: All of the health care records or people that have given consent within the health system can have their medical records and the symptoms kind of scan by the AI and you basically flying a bunch of people that very may well have fabry disease and don't know it and then.

Jeffrey P. Castelli: Testing could be offered through different programs to those patients.

Jeffrey P. Castelli: We're very excited what we published on the development of the Agra algorithm in February.

Jeff Hung: It looks like it can do a very good job and we're actually just on the cusp here testing it in a very large academic center here in the states and by mid year, we should know how well that.

Jeffrey P. Castelli: And by mid-year, we should know how well that goes, and we could actually see how many people are opting into potentially getting tested through their health provider. So, in Fabre, in particular, we're really excited about this because, you know, with being an X-linked disease. When you find patients within a certain healthcare system, potentially with this type of approach, you could then also help that family find people living around the country or around the globe. You don't have to screen every single healthcare facility. We are going to certainly look to see if it's successful and viable to see if that could be used, you know, in other systems as well. And, you know, this is not only for Fabry; there are actually similar programs and initiatives done already for other diseases like Pompe that have shown a similar approach works.

Jeffrey P. Castelli: Did and we actually could see how many people are opting into potentially getting tested through the health provider. So that in February in particular were really excited about this because with being an excellent disease.

Jeffrey P. Castelli: When you find patients within certain health care system potentially with this type of approach you could then also help that families find people living around the country are aligned that broke around the globe you don't have to kind of screen every single health care system.

Jeffrey P. Castelli: We arent going to certainly look to see if it's successful and valuable to see if that could be used in other systems as well and this is not only for fabry theres actually been similar.

Jeffrey P. Castelli: Programs and initiatives done already for other diseases like Pompeii that have shown similar approach works there as well.

Speaker Change: Thank you.

Speaker Change: Thank you that was your last question. This concludes today's conference call and have a great day.

Jeffrey P. Castelli: Okay.

Jeffrey P. Castelli: Okay.

Operator: Thank you. Thank you. That was your last question. This concludes today's conference call, and have a great day. Thank you for watching!

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