Q2 2024 Amicus Therapeutics Inc Earnings Call

August 8, 2024

Operator: Good morning, ladies and gentlemen, I need to explain the welcome to the Amicus Therapeutics second quarter 2024 financial resource conference call and webcast. At this time, all participants are in a listen-only mode. Later, we'll 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 Faughnan, Vice President of Investor Relations. You may begin.

Operator: Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics second quarter 2024 financial results conference call and webcast. At this time, all participants are in a listen-only mode. Later, we'll 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 Faughnan, Vice President of Investor Relations. You may begin.

Andrew Faughnan: And thank you for joining our conference call to discuss Amicus Therapeutics' second quarter 2024 financial results and corporate highlights. Leading today's call is Bradley Campbell, President and Chief Executive Officer, Sbastien Martel, Chief Business Officer, Dr. Jeff Castelli, Chief Development Officer, and Simon Harford, Chief Financial Officer. Also joining for Q&A are Dr. Mitchell Goldman, Chief Medical Officer, and Ellen Rosenberg, Chief Legal Officer.

Andrew Faughnan: As referenced on slide two, we may make forward-looking statements within the meaning of the Private Security 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 would be achieved. Any or all the forward-looking statements made in 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.

Andrew Faughnan: 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 factor sections of our annual report on Form 10-K for the year-ended December 31, 2023, and the quarterly report on Form 10-Q for the quarter-ended June 30, 2024, to be filed with the Securities and Exchange Commission today. At this time, it is my pleasure to turn the call over to Bradley Campbell, President and Bradley?

Bradley Campbell: Great, thank you, Andrew, and welcome everyone to our second quarter 2024 conference call. I'm pleased to highlight what has been a very successful first half of the year across our global business, and particularly an incredible quarter. In this time, we have continued to build on our top line revenue growth momentum to put us well on our way to our first full year of non-gap profitability, while also advancing our mission of bringing hope to individuals and families affected by rare diseases. As we did in this morning's press release, let me highlight several key takeaways.

Speaker: As we did in this morning's press release, let me highlight several key points in the quarter, representing 34% growth year over year or 36% on a constant currency basis. This strong performance has led us to increase our total revenue guidance for the full year to 26 to 31 percent from 25 to 30 percent previously. We've already made great progress against our key performance indicators, which continue to demonstrate the strength of the program.

Bradley Campbell: First, we continued our excellent commercial execution and delivered total revenue of $127 million in the quarter, representing 34% growth year over year or 36% on a constant currency basis. The strong performance has led us to increase our total revenue guidance for the full year to 26 to 31 percent from 25 to 30 percent previously. At the product level, Galliford continues its strong performance with $111 million in global revenue in the quarter, which represents 19% growth from last year on a constant currency basis. In the first half of the year, operational growth was 17% year over year at constant exchange rates, coming in at the high end of our guidance for the year.

Bradley Campbell: First, we continued our excellent commercial execution and delivered total revenue of $127 million in the quarter, representing 34% growth year over year or 36% on a constant currency basis. This strong performance has led us to increase our total revenue guidance for the full year to 26 to 31 percent from 25 to 30 percent previously. At the product level, Galliford continues its strong performance with $111 million in global revenue in the quarter, which represents 19% growth from last year on a constant currency basis. In the first half of the year, operational growth was 17% year over year at constant exchange rates, coming in at the high end of our guidance for the year.

Bradley Campbell: We continue to observe strong trends across our key performance indicators and all key geographies in the second quarter, including continued demand through new patient starts, both from switch and naive populations in all of our leading markets, and sustained patient compliance and adherence rates of over 90 percent. All of this is against the backdrop of significant growth in diagnosed and treated patients that we've highlighted previously. Eight years now after our first commercial launch, it's remarkable to see what an impact this medicine is having on people living with Fabry disease around the world.

Bradley Campbell: And we continue to gallifold serve as the foundation of our business for the next decade and beyond. Based on this sustained performance, we are increasing our full year 2024 revenue growth guidance range for the second time. We started the year with a growth range of 11% to 16% in May. We raised it to 13% to 17%. And now we're projecting 14 to 18%.

Bradley Campbell: And we continue to gallifold serve as the foundation of our business for the next decade and beyond. Based on this sustained performance, we are increasing our full year 2024 revenue growth guidance range for the second time. We started the year with a growth range of 11% to 16%. In May, we raised it to 13% to 17%. And now we're projecting 14% to 18%.

Bradley Campbell: Second, let me highlight the continued strong global commercial launch of POMPBILITY and APFOLDA, our novel therapy for late-onset POMPADISM. Pondility and Upfolda have been and will continue to be a huge growth driver for us. We've already made great progress against our key performance indicators, which continue to demonstrate the strength of the program.

Bradley Campbell: First and foremost, our number one focus for the year is to maximize the number of patients on therapy by year end. It's great to report that the rate of new commercial patients coming on Pombility and Upfolda in 2024 continues to progress exceptionally well. In the second quarter, we saw the largest number of new commercial patients, meaning patients who were not in our clinical studies, and as of the end of July, we had 186 patients who had been treated or scheduled for treatment.

Speaker: We are incredibly pleased with the demand globally from patients and physicians for this new therapy and consistently hear inspiring anecdotes from healthcare professionals around the world on how their patients are responding to Pondbility and Upfolda, which will continue to fuel the momentum throughout the year and beyond. We're also making significant progress on the reimbursement front globally, and this includes moving patients more quickly through the insurance process in the U.S. as we have been. On the regulatory front, we're pleased to announce that in July, SwissMedic approved POMBILITY and APFOLDA for adults living with late-onset Pompe disease in Switzerland. We're incredibly pleased to be providing a real choice for patients and challenging therapeutic expectations for both physicians and people living with Pompe disease.

Bradley Campbell: We are incredibly pleased with the demand globally from patients and physicians for this new therapy and consistently hear inspiring anecdotes from healthcare professionals around the world on how their patients are responding to Pondbility and Upfolda, which will continue to fuel the momentum throughout the year and beyond. Sbastien will provide more details in a moment, but the switch dynamics in the U.S., Europe, and the UK continue to look strong, and we're seeing great uptake in naive patients and markets outside the U.S. as well.

Bradley Campbell: We're also making significant progress on the reimbursement front globally, and this includes moving patients more quickly through the insurance process in the U.S. as we can. Throughout the remainder of the year, we'll focus on increasing patient access as we gain reimbursement and launch in additional countries throughout Europe. On the regulatory front, we're pleased to announce that in July, SwissMedic approved POMBILITY and APFOLDA for adults living with late-onset Pompe disease in Switzerland.

Bradley Campbell: We're also making significant progress on the reimbursement front globally, and this includes moving patients more quickly through the insurance process in the U.S. as we have been. Throughout the remainder of the year, we'll focus on increasing patient access as we gain reimbursement and launch in additional countries throughout Europe. On the regulatory front, we're pleased to announce that in July, SwissMedic approved POMBILITY and APFOLDA for adults living with late-onset Pompe disease in Switzerland.

Bradley Campbell: For the full year 2024, we're well on track to deliver our guidance of $62 to $67 million in global pommability and uphold sales, which will be a significant contributor to our growth and set us on a great course to achieve our ambition for POMOP to become the new standard of care treatment in this devastating disease. We're incredibly pleased to be providing a real choice for patients and challenging therapeutic expectations for both physicians and people living with Pompe disease.

Bradley Campbell: And finally, as we continue our excellent commercial execution across both therapies and prudently manage our resources, we are excited to share that Amicus was profitable on a non-gap basis in the second quarter, as well as in the first half of the year. We are confident we'll see this number increase as we look to achieve full year non-gap profitability in 2024. On slide four, as we laid out at the beginning of the year, we're focused on achieving our four key strategic priorities for 2024, including First, sustaining double-digit Galliford revenue growth.

Bradley Campbell: And again, on the back of strong sales growth so far, we've raised the expected annual growth rate of Galliford to 14 to 18%. Additionally, successfully executing on multiple commercial launches of POMBILITY and APFOLDA, advancing our ongoing studies to support our medical and scientific leadership in Fabry and Pompe disease, and maintaining a strong financial position as we carefully manage our expenses and investments to achieve non-gap profitability for the full year. With that in mind, let me now hand the call over to Sbastien, who'll go through our commercial performance in more detail.

Sbastien Martel: Thank you, Bradley, and good morning to everyone on the call. As always, I'll start by providing you with more details on our Galliford performance for the quarter.

Sbastien Martel: Thank you, Bradley, and good morning to everyone on the call. As always, I'll start by providing you with more details on our Galliford performance for the quarter. On slide 6, for the second quarter of 2024, Galliford reported revenue of $111 million, driven by strong patient demand, particularly from our leading market. We ended the second quarter with more than 60% of the global market share for treated Fabry patients with amenable mutations. And the good news is that there are still many more potential patients eligible for our therapy. As Bradley highlighted, given the continued strong performance of Galliford throughout the second quarter, we are raising again our full year 2024 revenue growth guidance range to now 14 to 18 percent.

Sbastien Martel: On slide 6, for the second quarter of 2024, Galliford reported revenue of $111 million, driven by strong patient demand, particularly from our leading market. We ended the second quarter with more than 60% of the global market share for treated Fabry patients with amenable mutations. And the good news is that there are still many more potential patients eligible for our therapy. As Bradley highlighted, given the continued strong performance of Gallafold throughout the second quarter, raising again the full year 2024 revenue growth guidance range to now 14 to 18 percent.

Sbastien Martel: Turning to slide seven, our results in the second quarter highlight the strengths of our global commercial efforts. Demand for Galapagos globally continues to be strong, with patients added in all major markets, delivering operational growth of 19% over the same period in 2023 at constant exchange rates. Our leading markets, such as the UK, the US, top new countries, and Japan, remain the biggest drivers of patient demand and give great confidence in the growth this product will achieve over the long term.

Speaker: The U.S. in particular saw impressive patient demand, the highest number of net new patients seen in over five years. And we expect this trend to continue for the full year.

Sbastien Martel: The U.S., in particular, impressed your patients' demand, the highest number of net new patients seen in over five years, and we expect this to continue for the full year. Within the global mix, which is about 42% switch and 58% naive, we're now seeing a stronger uptake in our population.

Sbastien Martel: The U.S. in particular saw impressive patient demand, the highest number of net new patients seen in over five years. And we expect this trend to continue for the full year. Within the global mix, which is about 42% switch and 58% naive, we're now seeing a stronger uptake among the naive population.

Sbastien Martel: We continue to achieve high market shares in countries where we've been approved the longest. There is plenty of opportunity to continue to switch patients over to Garfield and to keep growing the market as we penetrate the diagnosed, untreated, and newly diagnosed segments. 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 GALAFOLG predominantly stay on GALAFOLG. We're very pleased to see that prescribers continue to position Galafold as the treatment of choice for a minimal therapy patient.

Speaker: This leadership position is supported by our continuous investment in medical education and evidence generation. As noted, Galapagos currently has more than 60% of the global amenable market. What we're seeing in our most mature markets is that we can reach up to 85-90% of market share. So we know that there's the potential to reach those levels in the global market share as well. I'm pleased to announce that Switzerland's Swissmedic has approved Pombilichenoproda as a long-term enzyme replacement therapy and enzyme stabilizer for adults with LOG, and all the key regulatory authorities who recognize the benefits of this therapy.

Sbastien Martel: This leadership position is supported by our continuous investment in medical education and evidence generation. I'm very pleased to recognize the first publication of the results from our own Fabry Disease Registry, the Follow Me Registry, in July. These results were published in the Journal of Inherited Metabolic Disorders and constitute a really important milestone for Amicus.

Sbastien Martel: We continue to generate positive data for Gallup. Data from this study aligns very well with previous observations from clinical tribes and extends the available data supporting the real-world multi-system benefits of gas. This publication, along with our ongoing medical education at Congress, is an important part of the story of this oral precision medicine and helps support the long-term growth potential we anticipate Galapagos will have in this growing fabric market. On slide 8, we know that there is significant patient demand for Galafod and that the segment of the global Fabry market made up of patients with a medical mutation has the potential to reach up to $1 billion in annual revenue by the end of the

Sbastien Martel: We anticipate sustained growth in 2024 and beyond to be driven by several key drivers. First, the fabric market is growing robustly, with a significant portion of growth coming from finding new patients and reaching the diagnosed and treated population. As we mentioned previously, at the end of 2023, there were more than 2,400 individuals on GALAFOLD, and about 1,400 of those were individuals who were naive to any treatment before GALAFOLD, and those numbers obviously continue to grow this year, and we'll be pleased to report an update on our year-end results.

Sbastien Martel: As we mentioned previously, at the end of 2023, there were more than 2,400 individuals on GALAFOLD, and about 1,400 of those were individuals who were naive to any treatment before GALAFOLD, and those numbers obviously continue to grow this year, and we'll be pleased to report an update on our year-end results. We've seen many new patients go on to treatment through newborn screening and family screening, and we continue to increase patient identification capability, to provide ongoing medical education and support renewable screening initiatives. But Lee remains, unfortunately, one of the most underdiagnosed rare diseases.

Sbastien Martel: We've seen many new patients go on to treatment through newborn screening, and family screening, and we continue to increase patient identification capability, to ongoing medical education and support renewable screening initiatives. But Lee remains, unfortunately, one of the most underdiagnosed rare diseases.

Sbastien Martel: So the more patients that can be identified, the more patients may benefit from GAP. We're also seeing many diagnosed untreated patients transition to treatment as the need for earlier treatment, especially in females, becomes better appreciated. The other piece is continuing to drive the gap of market share of treated and medical patients to continued commercial execution. As noted, delightful, trying to be as more than 60% of the global eminible markets.

Sbastien Martel: So the more patients that can be identified, the more patients may benefit from GAP. We're also seeing many diagnosed untreated patients transition to treatment as the need for earlier treatment, especially in females, becomes better appreciated. The other piece is continuing to drive the gap of market share of treated and medical patients to continued commercial execution. As noted, Galapagos currently has more than 60% of the global amenable market.

Sbastien Martel: We're saying that in our most mature markets, we can reach up to 85-90% of market share. So we know that there's the potential to reach those levels in the global market share as well. And again, all of these efforts are supported by solid compliance and adherence rates through physician and patient education and support programs.

Sbastien Martel: What we're seeing in our most mature markets is that we can reach up to 85-90% of market share. So we know that there is the potential to reach those levels in the global market share as well. And again, all of these efforts are supported by solid compliance and adherence rates through physician and patient education and support programs.

Sbastien Martel: Additionally, he will continue to make progress on expanding Gallaford into new markets and extending the labels. There are still some markets in Latin America, the Middle East, and Asia-Pacific regions where Gallaford is either newly reimbursed, or we expect it reimbursed. Also important to note here, we often have drug exclusivity in the U.S. and Europe, and in addition to our now 61 orange-book-listed patents, 45 of which provide protection into 2038 and beyond, including 14 composition of matter patents. This provides us with the opportunity to continue to provide access to GALACTO globally for a long time to come. We intend to continue to protect and enforce our broad intellectual property rights.

Sbastien Martel: Additionally, we'll continue to make progress on expanding Galaford into new markets and extending the labels. There are still some markets in Latin America, the Middle East, and Asia Pacific regions where Galaford is either newly reimbursed, or we expect it to be reimbursed. Also important to note here, we often have drug exclusivity in the U.S. and Europe, and in addition to our now 61 orange-book-listed patents, 45 of which provide protection into 2038 and beyond, including 14 composition of matter patents. This provides us with the opportunity to continue to provide access to GALACTO globally for a long time to come. We intend to continue to protect and enforce our broad intellectual property rights.

Sbastien Martel: Looking ahead, we expect steady, double-digit growth for Galliford throughout 2024, and we remain confident that with our strong IP protection, Galliford has a long runway well into the next step. Turning now to Pompe disease, on slide 10, we outline our global launch progress with Pompidou-Tianopolda. For the second quarter of 2024, Mobility and Upholder reported revenue of $16 million.

Sbastien Martel: This represents an increase of 44% compared to the first quarter of 2024 and provides a strong foundation for the remainder of the year in the U.S. Q2 benefited from the remaining clinical trial patients transitioning to commercial treatments in addition to patients switching from other therapies. We continue to see a majority of patients switching from Nexviazine, about 68%, and the remaining from Lumizine. This means we're switching patients proportionally from both products in the U.S. We're also seeing a broadening and deepening of our prescriptions with more sites coming online and multiple new prescriptions from physicians coming in at accelerated and increased rates over Q1. Outside of the U.S., we're seeing patients from all three segments, some switching from Myosin, some from Nexiodine at a proportional rate to the That's exactly what we want to be seeing at this stage in the long run.

Sbastien Martel: A few updates on the launch in Europe. I'm pleased to announce that Switzerland's Swissmedic has approved Pombilichenoproda as a long-term enzyme replacement therapy and enzyme stabilizer for adults with LOG, and all the key regulatory authorities who recognize the benefits of this therapy. In Germany, we have successfully finalized negotiations with the National Association of Statutory Health Funds, and we're very pleased with the outcome, which continues to reflect the value health technology assessments are seeing with this therapy.

Speaker: In Germany, we have successfully finalized the negotiations with the National Association of Statutory Health Funds, and we're very pleased with the outcome. Additionally, we anticipate this to be an important reference point for future pricing and reimbursement discussions. Given Pompidou Tienopolda's solid commercial executions thus far, in the first half of 2024, we're well on our way to deliver our full-year revenue guidance of $62 to $67 million for Pompidou Tienopolda and as we launch new countries in Europe.

Sbastien Martel: The price acknowledges the added benefit of mobility plus a fold-down for LOPD patients, and we'll be closely aligned with the price we set at launch. Additionally, we anticipate this to be an important reference point for future pricing and reimbursement discussions. In Spain, our newest launch country, we've added a significant number of patients and new prescribing accounts, all within the country's first quarter of launch. We remain very pleased with the uptake of Combiti Anapoda in the UK, where we've captured greater than a third of market share within the region.

Sbastien Martel: With the benefit of EAMS, mobility has been upheld, and they have actually been in the market for two years in the UK. And we believe that the current market share is indicative of how this product can perform over two-year periods, which is a great example of Potential Humanity.

Sbastien Martel: Given Pompidou Tienopolda's solid commercial executions thus far, in the first half of 2024, we're well on our way to deliver our full-year revenue guidance of $62 to $67 million for Pompidou Tienopolda. Moving to slide 11. I am very pleased with the launch momentum for the first half of the year. As Bradley mentioned earlier, there are about 186 patients that have been treated or are scheduled to be treated at the end of July. That's about 174 on therapy, and the remaining 12 are scheduled.

Sbastien Martel: We remain very pleased with the ongoing demand for this therapy as the rate of new commercial patients coming on to POMBILITY and EFOLDA continues to increase across all three months. Q2, in particular, was a very strong quarter, having seen the largest number of new demand patients being prescribed pomidigliano products. These are individuals not from clinical trials choosing to come into commercial practice.

Sbastien Martel: This is important because it further supports our thesis that launch momentum will continue to build throughout the year, as we move more quickly through the prescription to treatment process in the U.S. and as we launch new countries in Europe. Our launch has leveraged our highly experienced cross-functional teams, and we've had great outreach with TLP over the years. We're seeing an increase in the depth and breadth of prescribers across all pre-markets. In Q2 alone, the global number of prescribing accounts increased by 50 percent.

Sbastien Martel: All core treating centers have been engaged, and they've provided very positive feedback, feedback from both HCPs and other stakeholders about our business approach, our support, and our 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 POMBILITY and UPHOLDA. In the U.S., the largest payers have already put POMBILITY and UPHOLDA on their respective formularies, and we have also seen strong acceptance by Medicare and Medicaid. The overall time from prescription to infuser is now down to around 65 days, with payer approvals happening in around 30 days, and the last few commercial patients have actually come in significantly quicker.

Speaker: We have a highly experienced team who are engaging in positive conversations with payers to demonstrate the value of POMBILITY and UPHOLDA. In the U.S., the largest payers have already put POMBILITY and UPHOLDA on their respective formularies, and we have also seen strong acceptance by Medicare and Medicaid. Overall, we've had a very strong first half, and we're very pleased with the building momentum on patient outcomes.

Jeff Castelli: We believe this will continue to improve. Today, we're launching in Germany, the UK, the US, Australia, and Spain, but we remain in active pricing and reimbursement discussions with additional major European markets as we focus on securing broad patient access throughout the European market. Overall, we've had a very strong first half, and we're very pleased with the building momentum on patient. Throughout the second half of 2024, our focus will be on maximizing the number of patients on therapy by year-end.

Jeff Castelli: So, in summary, we're very pleased with the launches of the Pompidou Tenor Folder across the first wave of countries. The strength of our clinical data, the depth of our experience, and the talent we have at Amicus gives us great confidence in our ability to make a real difference for people living with Pompidou. We believe Amicus is in a great position with our second commercial launch, and with that, I will hand the call over to Jeff Castelli, our Chief Development Officer, to discuss the ongoing clinical studies and regulatory timeline.

Jeff Castelli: Thank you, Sebastian, and good morning, everyone. In 512, we outline how we continue to build the body of evidence for a probability in Opfoda through our ongoing clinical studies, as well as through our Amicus Pompeii Registrate, as we also continue to execute on expanding commercial access through reimbursement dossiers and regulatory. As we enter this second phase of launch, in addition to the various reimbursement dossiers that we have or are in the process of submitting, we also have multiple ongoing or planned regulatory submissions for marketing approval in new geographies throughout the U.S. We've just mentioned the approval in Switzerland. Additionally, we have our regulatory dossiers being reviewed in Australia and Canada, and we're working towards a submission here in Japan before the end of the year.

Jeff Castelli: Thank you, Sebastian, and good morning, everyone. On slide 12, we outline how we continue to build the body of evidence for pumbility and opphalta through our ongoing clinical studies, as well as through our Amicus Pompeii registration, as we also continue to execute on expanding commercial access through reimbursement dossiers and regulatory. As we enter this second phase of launch, in addition to the various reimbursement dossiers that we have or are in the process of submitting, we also have multiple ongoing or planned regulatory submissions for marketing approval in new geographies throughout the U.S. We've just mentioned the approval in Switzerland. Additionally, we have our regulatory dossiers being reviewed in Australia and Canada, and we're working towards a submission here in Japan before the end of the year.

Speaker: as we also continue to execute on expanding commercial access through reimbursement dossiers and regulatory approvals. We've just mentioned the approval in Switzerland. Additionally, we have our regulatory dossiers being reviewed in Australia and Canada, and we're working towards a submission here in Japan before the end of the year. Finally, as highlighted in the pipeline slide in the appendix,

Simon Harford: 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 Rizela study for children living with infantile-onset Pompeii disease. We see this as an important opportunity to support label expansions into these patient segments in the years ahead and, very importantly, to address the significant unmet needs for these children. Through ongoing clinical studies and the Amicus Pompei Registry, we continue to generate evidence on the differentiated mechanism of action and on the long-term impact of POM, building up FODA across endpoints and patient populations.

Jeff Castelli: 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 Rizela study for children living with infantile-onset Pompeii disease. We see this as an important opportunity to support label expansions into these patient segments in the years ahead and, very importantly, to address the significant unmet needs for these children. Through ongoing clinical studies and the Amicus Pompei Registry, we continue to generate evidence on the differentiated mechanism of action and on the long-term impact of POM, building up FODA across endpoints and patient populations.

Simon Harford: Our medical conference presentations and publications continue to be an important part of our education effort. Finally, as highlighted on the pipeline slide in your 10-Dex, for our earlier stage pipeline, we continue to focus on novel approaches to next-generation therapies in fibroin and pumping. 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.

Jeff Castelli: Our medical conference presentations and publications continue to be an important part of our education effort. Finally, as highlighted in the pipeline slide in the appendix, for our earlier stage pipeline, we continue to focus on novel approaches to next-generation therapies in fibroin and pumping. 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 Harford: Thank you, Jeff. Our financial overview begins on slide 14 with our income statement for the second quarter ending June 30th, 2024. For Q2, we achieved total revenue of $127 million, which is a 34% increase over the same period in 2023. At constant exchange rates, revenue also grew 36%.

Simon Harford: Cost of goods sold as a percentage of net sales was 9% in Q2 2024, as compared to 10% for the prior year period, remaining relatively flat. Total GAAP operating expenses decreased to $100 million for the second quarter 2024 as compared to $104 million in the same period last year, a decrease of 4%. On a non-GAAP basis, total operating expenses decreased to $82 million for the second quarter of this year as compared to $84 million for the same period last year, a decrease of 2%. We define non-GAAP operating expenses as research and development, and SG&A expenses, excluding stock-based compensation. Loss on impairment of assets, changes in fair value of contingent consideration, restructuring charges, and depreciation and amortization.

Simon Harford: The global geographic breakdown of total revenue during the quarter consisted of $77 million, or 60% of revenue generated outside the United States, and the remaining $50 million, or 40% coming from within the U.S. Cost of goods sold as a percentage of net sales was 9% in Q2 2024, as compared to 10% for the prior year period, remaining relatively flat. Total GAAP operating expenses decreased to $100 million for the second quarter of 2024 as compared to $104 million in the same period last year, a decrease of 4%.

Speaker: For Q2, we achieved total revenue of $127 million, which is a 34% increase over the same period in 2023. At constant exchange rates, revenue also grew 36%, which started the year at 11-16%, was most recently 13-17%, and now is 14-18% at constant exchange rates.

Simon Harford: On a non-GAAP basis, total operating expenses decreased to $82 million for the second quarter of this year as compared to $84 million in the same period last year, a decrease of 2%. We define non-GAAP operating expenses, research and development, and SG&A expenses, excluding stock-based compensation, losses on impairment of assets, changes in fair value of contingent consideration, restructuring charges, and depreciation and amortization.

Simon Harford: On a gap basis, net loss for the second quarter of 2024 was $16 million, or $0.05 per share, compared to a net loss of $43 million, or $0.15 per share, for the second quarter of 2023. In Q2, non-GAAP net income was $18 million, or $0.06 per share, compared to a loss last year in the same period of $20 million, or $0.07 per share. Cash, Cash Equivalents, and Marketable Securities were $260 million as of June 30th.

Simon Harford: 2024 compared to $286 million at December 31, 2023. Turning now to slide 15, we are raising our full year 2024 total revenue guidance range to 26 to 31 percent, up from 25 to 30 percent. This is driven by the increase in our full year 2024 Garlafold Revenue Growth Guidance, which started the year at 11-16%, was most recently 13-17%, and now is 14-18% at constant exchange rate. In addition, we are reiterating the guidance.

Simon Harford: $62 to $67 million for Promvility and Oxalda sales for the full year 2024. Our full year 2024 non-GAAP operating expense guidance has been narrowed from $345 to $365 million dollars to $345 to $360 million dollars, or a five percent reduction at the top end. With our commitment to full-year non-GAAP profitability during the first full year of launch for POMbility and OpFolder, we are keeping operating expense growth in low single digits year-over-year at the midpoint of guidance.

Simon Harford: 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 which aren't yet reimbursed, as well as next-generation manufacturing for Pombilities. In the second quarter of 2024, non-gap profitability was $18.5 million, and for the first half of 2024, $13.9 million. With our total revenue guidance of 26 to 31% growth, we remain comfortably on track for 2024 to be our first full year of non-gap profitability, as profitability continues to accelerate in the second half of the year. And with that, I will turn the call back over to Bradley for our closing remarks. Bradley

Bradley Campbell: Thank you, Simon, Jeff, and Sbastien. As you can all see, we've been highly focused this year on our commercial execution, continuing to strengthen our financial profile, and ultimately delivering value for our shareholders. Our first half performance has laid the groundwork for us to continue to achieve our goals and fulfill our mission of delivering life-changing therapy. And with that, Operator, we can now open the call to questions.

Operator: Yes, ladies and gentlemen, if you have a question, please press star 1-1 on your touchtone telephone. At this time, we request that you only ask one question. If you have additional questions, please enter back into the queue. Thank you. Your first question comes from... One moment, please. This question comes from the line of Tazeen Ahmad with BofA Securities.

Operator: Your first question comes from...

Operator: Your line is now open. Hi guys. Thanks for taking my questions.

Tazeen Ahmad: Your line is now open. Hi guys. Thanks for taking my questions.

Bradley Campbell: Yeah, thanks, Tazeen. Thanks for the question. So, as you mentioned, a couple things. First of all, I think, you know, as you saw from the numbers today, we are continuing to see great progress in both markets. I think you have, you know, two different dynamics going on. In the US, it's the largest single market. And so, you know, the rate of new patients will continue to be strong as we go throughout the year.

Bradley Campbell: However, in Europe, we're adding new countries as we go. You know, we launched in Spain, we just got approval in Switzerland, and we're working on other reimbursements. So I think over the course of the year, the balance of new patients will probably start to favor the United States, but that's just a matter of size. But overall, I think both markets are progressing extremely well. In terms of the label, as you said, and as I kind of provided on the call, we and Sebastian provided this detail as well.

Bradley Campbell: In Europe, we're seeing switching patients from both myosin and nexviazine, as well as new patients who are on the label there. In the US, we're seeing, you know, predominantly switching from nexviazine and some switching from myosin. And so, you know, we're seeing that as we go through the year. But overall, I think both markets are progressing extremely well. And so, you know, we're seeing switching patients from both myosin and nexviazine, as well as new patients who are on the label there. Thank you. Your next question comes from Eliana Merle with UB.

Operator: Your next question comes from Eliana Merle with UBS. Your line is now open. Hey guys, thanks so much for taking the question.

Eliana Merle: Your next question comes from Eliana Merle with UBS. Your line is now open. Hey guys, thanks so much for taking the question.

Bradley Campbell: Yeah, thanks. Essentially, we're seeing what we had expected to see, which is the majority of patients switching from next viazine have been on treatment for that kind of year to two years. And that's a dynamic that we've described before. The good news is, of course, there is a huge bolus of patients that are kind of rolling through that year to two years on therapy over the course of this year and into next year.

Bradley Campbell: And so I think we've got a large bolus of patients that we can target. We do see some patients who have switched earlier in their journey. And I would say that probably comes more from word of mouth or from knowing other patients in the community who had a positive experience with Ponvillanabfolda. And I think that's another dynamic, too, that we'll want to continue to see, which is, you know, as we establish this as an important new therapy, as more physicians and more patients get experience, and hopefully, those experiences will be positive, I think that creates, you know, some Thank you. Your next question?

Operator: Your next question comes in the line of Anupam Rama with JPM. Your line is now open.

Anupam Rama: Your next question comes in the line of Anupam Rama with JPM. Your line is now open.

Operator: Your next question.

Bradley Campbell: Maybe I'll start at a high level and then ask Sbastien to add a little color there. So, Anupam, honestly, we're seeing great growth in all of our key markets, which is what you want to be seeing. But Sbastien, maybe provide a little bit of color on some of the unique elements of the U.S. and anything else that you think is relevant there.

Sbastien Martel: Yeah, thanks, Brad. So, Anupam, we're seeing very strong demand for naive patients, newly diagnosed patients in the U.S., and we have seen that, you know, for a number of quarters now. That's actually why we've now raised guidance twice this year on Galafold. So, you know, a growing proportion of newly diagnosed patients really are put on Galafold as the very first line of treatment if they have amenable mutations. The number of PRFs we've recorded in the first half of the year, as I said earlier, is close to the highest we've seen in the last five years.

Sbastien Martel: So, strong, very strong performance in the U.S. We continue to see the shift that we've seen, you know, for ever since launch, actually, that a growing proportion of females with Fabry disease are also being treated, and the tendency of patients who have been diagnosed moving to treatment earlier than they may have in the past.

Sbastien Martel: In Europe, we're very pleased to see a bit of a similar pattern with, you know, the majority of the growth coming from naive patients. And we're seeing that in our largest markets, the U.K. We're seeing great performance in Japan as well. We actually think that we have, you know, more growth upside in Japan simply because the Japanese business is our fourth largest. If you look at the whole Fabry market, Japan is actually the second largest market. So, there's no reason why Japan couldn't at some point in time become our second largest market as well. So, these are sort of the key highlights on regional performance. Thanks.

Operator: Thanks so much for taking our questions. Thank you. Your next question comes from Joseph Schwartz with Lyrinc.

Bradley Campbell: Thanks so much for taking our questions. Thank you. Your next question comes from Joseph Schwartz with Lyrinc.

Operator: Your next question comes from Joseph Schwartz with Lyrinc. Your line is now open. Hi all, this is Will on behalf of Joe. Thanks for taking our question this morning and congratulations on the progress.

Operator: Your line is now open. Hi, all. This is Will on behalf of Joe. Thanks.

Will: Hi, all. This is Will on for Joe. Thanks.

Will: Hi all, this is Will on behalf of Joe. Thanks for taking our question this morning and congratulations on the progress.

Simon Harford: Thank you, Jeff! Our financial overview begins on slide 14 with our income statement for the second quarter ending June 30th, 2024. In Q2, we achieved total revenue of $120,000,000,000, which is a 34% increase over the same period in 2023. At constant exchange rates, revenue also grew 36%. The global geographic breakdown of total revenue during the quarter consisted of $77 million, or 60% of revenue generated outside the United States, and the remaining $50 million, or 40% coming from within the U.S.

Bradley Campbell: Yeah, thanks for the question. I think one of the things we talked about to passion highlight is the rate of new patients, you know, this year continues to be much higher than it was last year. And that's exactly what we expected. In terms of new patient ads, I think we'll continue to build momentum as we go towards the back half of the year, especially as we open up new countries.

Bradley Campbell: That being said, from a revenue perspective, especially because a lot of those reimbursements and launches will be in the fourth quarter, the rate of new revenue, you know, won't change very much. But obviously, next year, it'll be really important. So that's why we focus on sort of maximizing the number of patients on therapy by the end of the year because that, you know, maximizes the run rate going into next year.

Operator: Your next question comes from the line of Ritu Baral with T.D. Cowan. Your line is now open.

Ritu Baral: Your next question comes from the line of Ritu Baral with T.D. Cowan. Your line is now open.

Operator: Good morning, guys. Thanks for taking the time to answer the question. Congratulations on the metrics of this quarter. From our tracking survey, our PUMUP tracking survey that we ran recently, we got some input and some good flavor on the competitive dynamics, which from Genzyme, which seemed to be quite considerable. And we're hearing that at least the Nexviazyme team over there had some very interesting messaging about Nexviazyme superiority over Myozyme, and Lunazyme, and that seemed to be resonating with some of the doctors that we surveyed.

Ritu Baral: Good morning, guys. Thanks for taking the time to answer the question. Congratulations on the metrics of this quarter. From our tracking survey, our PUMUP tracking survey that we ran recently, we got some input and some good flavor on the competitive dynamics, which from Genzyme, which seemed to be quite considerable. And we're hearing that at least the Nexviazyme team over there had some very interesting messaging about Nexviazyme superiority over Myozyme, and Lunazyme, and that seemed to be resonating with some of the doctors that we surveyed.

Operator: Can you talk to, I mean, if that's the PUMUP counter detail, can you talk to your counter counter detail on what's going on messaging wise, on what defines a decliner, and how the two drugs sort of stack up against each other in the field? And then I've got to follow up on insurance. Thank you.

Ritu Baral: Can you talk to, I mean, if that's the PUMUP counter detail, can you talk to your counter counter detail on what's going on messaging wise, on what defines a decliner, and how the two drugs sort of stack up against each other in the field? And then I've got to follow up on insurance. Thank you.

Bradley Campbell: Thanks Ritu. Maybe I'll start it, and I'll have Jeff add some color.

Bradley Campbell: I won't speak to how they're detailing, but what I would say is that, you know, we're the only product that has shown in a controlled phase of phase three and improvements in both the six-minute walk and forced vital capacity versus an active comparator. And for me, the most important thing here is the efficacy of the product. And I think that's a very important part of our label. And, in fact, you may remember our promotional materials, which are pretty incredible.

Bradley Campbell: I won't speak to how they're detailing, but what I would say is that... You know, we're the only product that has shown in a controlled phase of phase three and improvements in both the six-minute walk and forced vital capacity versus an active comparator. And for me, the most important thing here is the efficacy of the product. And I think that's a very important part of our label. And, in fact, you may remember our promotional materials, which are pretty incredible.

Speaker: You know, we're the only product that has shown in a controlled phase of phase three and improvements in

Bradley Campbell: We can effectively say improvement is possible with this therapy, and I think that's a reassuring message that we're very confident in. And I think that describes why we've had such a great launch so far. But Jeff, maybe you could talk a little bit more about the kinds of things that physicians are focusing on and maybe the importance of some of the other data points that they're following. Yeah, thanks, Brian. Thanks for two for the questions.

Jeff Castelli: Brad, I think you hit on the main point, which is, from an amicus perspective, we obviously are still educating people on the data from our phase three trial, which was largely in ERT experienced patients, randomized, blinded, where we showed, you know, in that large group of switch patients, improvements on 6-Minute Walk at PC and the key endpoints. And it'll be presented across all the endpoints in that trial. We see patients when switching have improvements across numerous aspects of muscle strength, quality of life, breathing, etc. So, you know, we're still really educating everyone about that data. Obviously, that data is, you know, one of the highest levels of evidence that you can have.

Jeff Castelli: And then, you know, there's still continuing education around the mechanism of action and the unique differences in what we think we've built here with Pondeli-Napfota. You know, moving forward, as I talked about briefly in the transcript, we are really invested in real-world evidence and the registry, looking to see how people are doing when they're on POMOP across all sorts of parameters, whether that's naive patients outside the U.S., people switching from Nixpiazine, people switching from Lumizyme, and we'll continue to report that data, importantly, from our registry and And we're really excited about everything we're hearing anecdotally about the patient-physician response to Pondeli-Napfota. But, yes, certainly, there are different messages out there, but we're really pleased with where we are today and where we're going.

Ritu Baral: Great, and some of the survey focused on the reimbursement landscape, and there seems to be some sort of continuing griping from some of the respondents about insurance coverage. Not that it's terrible, but we've heard anecdotes about not getting the folder component covered. Can you speak to how that has been going now that you have a full quarter's worth of your new J code? Has this all mostly been resolved?

Bradley Campbell: Great, and some of the survey focused on the reimbursement landscape, and there seems to be some sort of continuing griping from some of the respondents about insurance coverage. Not that it's terrible, but we've heard anecdotes about not getting the folder component covered. Can you speak to how that has been going now that you have a full quarter's worth of your new J code? Has this all mostly been resolved? Yeah, thanks, Ritu. You know, it's funny, I...

Speaker: Yeah, thanks, Ritu. You know, it's funny.

Bradley Campbell: One thing we would remind everybody, I think when the first prescription happens, and in all of these therapies, frankly, the physician or his staff have to go through the pre-approval process, and there are two products here. But I would say that we haven't seen any patients be denied coverage for either the ERT or the small molecule, and we share the trends. You know, we're seeing things proceeding exactly as we've hoped, which is shortening the time from prescription to fusion, which is now down to 65 days, and then shortening the insurance time, too, which is now down to 30 days.

Bradley Campbell: One thing we would remind everybody, I think when the first prescription happens, and in all of these therapies, frankly, the physician or his staff have to go through the pre-approval process. And there are two products here. But I would say that we haven't seen any patients be denied coverage for either the ERT or the small molecule. And we share the results. You know, we're seeing things proceed exactly as we'd hoped, which is shortening the time from prescription to infusion. We're now down to 65 days, and then shortening the insurance time, too, which is now down to 30 days.

Bradley Campbell: Yeah, thanks for that too. You know, it's funny.

Bradley Campbell: Yeah, thanks, Ritu. You know, it's funny.

Jeffrey Castelli: One thing we would remind everybody, I think when the first prescription happens, and in all of these therapies, frankly, the physician or his staff have to go through the pre-approval process. And there are two products here. But I would say that we haven't seen any patients be denied coverage for either the ERT or the small molecule. And we share the results. You know, we're seeing things proceed exactly as we'd hoped, which is shortening the time from prescription to infusion. We're now down to 65 days, and then shortening the insurance time, too, which is now down to 30 days.

Speaker: So, I'm sure that the very first time the staff goes through any new therapy, you know, they have to get used to the paperwork, et cetera. And I'm sure that's what you're seeing in some of your surveys. But everything we're seeing and the facts are that we're getting patients through the system, and we're getting them through it much more quickly than we did at the start of the process, which is what we want to see. And yeah, I'm sure the coding piece helps to some extent there as well. Great, thanks. Thank you.

Jeffrey Hung: So, I'm sure that the very first time the staff goes through any new therapy, you know, they have to get used to the paperwork, et cetera. And I'm sure that's what you're seeing in some of your surveys. But everything we're seeing and the facts are that we're getting patients through the system, and we're getting them through it much more quickly than we did at the start of the process, which is what we want to see. And yeah, I'm sure the coding piece helps to some extent there as well. Great, thanks. Thank you. Your next question comes on the line from Jeffrey Hung with Morgan Stanley.

Bradley Campbell: Yeah, I'll let Jeff maybe talk a little bit more about that. But the reality is that the overall market just continues to grow, and that's fueled by, you know, I think improving diagnosis through the kinds of things that Sbastien talked about. But Jeff, maybe you could remind us the sort of change in market size from where we started, the launch to today, and then, you know, kind of what has changed in the diagnostic landscape that we think. Yeah, thanks, Brian. Thanks for the question.

Jeff Castelli: As Brad said, there were about 10,000 patients when we launched GALFOLD seven or eight years ago. There are now over 18,000 patients diagnosed. When you look at all the numbers out there from various screening programs, whether it's newborn screening across different countries or at-risk screening of patients that likely have Febre, it's clear that there are many, many more people and families living with Febre that are undiagnosed today than the 18,000 that are diagnosed.

Jeff Castelli: So the good news is there's a lot of tailwinds for diagnosis. Medical education continues to improve so physicians know when to suspect Febre. There's a lot more access to screening paradigms, low-cost genetic testing, and a lot more education about when you diagnose a Febre patient, if it's a late-onset male or female, about the burden of treatment and the need to treat earlier. And those are all things that Amicus, along with the other companies in the space and physicians, have really been doing from a medical education perspective.

Sbastien Martel: Lots of exciting developments in the kind of medical record, the use of AI, the potential to screen for people that might be suffering from Godbrain unknowingly. So, you know, as we look moving forward, we think there's going to be lots and lots of people being diagnosed. Many of them will have amenable mutations. That's something we've learned here, too, that we seem to see a higher percentage of amenable patients than the new people being diagnosed than historically, largely because late-onset patients are harder to diagnose.

Jeff Castelli: Lots of exciting developments in the kind of medical record, the use of AI, the potential to screen for people that might be suffering from Godbrain unknowingly. So, you know, as we look moving forward, we think there's going to be lots and lots of people being diagnosed. Many of them will have amenable mutations. That's something we've learned here, too, that we seem to see a higher percentage of amenable patients than the new people being diagnosed than historically, largely because late-onset patients are harder to diagnose.

Sbastien Martel: Late-onset patients tend to have amenable mutations, and more and more, Galliford is a standard of care for people with this mutation. So lots of positive forces are all coming together, I think, that are driving what we're seeing. And particularly in the U.S., the last thing I'll add is that there is newborn screening going on in five or six states. And when they find a newborn, often that is the index patient in a family.

Jeff Castelli: Late-onset patients tend to have amenable mutations, and more and more, Galliford is a standard of care for people with this mutation. So lots of positive forces are all coming together, I think, that are driving what we're seeing. And particularly in the U.S., the last thing I'll add is that there is newborn screening going on in five or six states. And when they find a newborn, often that is the index patient in a family.

Sbastien Martel: And then there's the ability to screen the family for the other three to five members that also have FEDVRA and are adults and likely suffering. So lots of great tailwinds for the community in terms of finding new patients.

Jeff Castelli: And then there's the ability to screen the family for the other three to five members that also have FEDVRA and are adults and likely suffering. So lots of great tailwinds for the community in terms of finding new patients.

Speaker: And I would add that everything that Jeff said is actually highlighted in the growth rate that we see for the quarter, not just for our sales. So we were again the fastest growing brand for Fabri with 19% growth. But when we look at the performance of Fabrizyme and Replica, so the two ERTs, you know, we estimate that the overall Fabri market growth rate from a dollar standpoint was north of 12% in the second quarter. So everything that again, Jeff mentioned, you know, contributes to that overall healthy Fabri market growth.

Sbastien Martel: And I would add that everything that Jeff said is actually highlighted in the growth rate that we see for the quarter, not just for our sales. So we were again the fastest growing brand for Fabri with 19% growth. But when we look at the performance of Fabrizyme and Replica, so the two ERTs, you know, we estimate that the overall Fabri market growth rate from a dollar standpoint was north of 12% in the second quarter. So everything that again, Jeff mentioned, you know, contributes to that overall healthy Fabri market growth.

Operator: Your next question comes to the line about Daikon Ha with Stiefel. Your line is now open.

Daikon Ha: Your next question comes to the line about Daikon Ha with Stiefel. Your line is now open.

Speaker: Hey, good morning, guys. Thanks for squeezing me in here. Maybe going back to, I guess, similar to Ritu's question, you know, our channel checks also indicate that there is a lot of enthusiasm to switch over to POMOP, but I guess what's interesting is, you know, a lot of these physicians say sort of the switching impetus is dependent on progression of disease or next view is I'm not showing any meaningful benefit, yet those same physicians also talk about how the muscle assessment or other functional assessment frequency hasn't really changed since Next View is On launch or POMOP launch.

Operator: Hey, good morning, guys. Thanks for squeezing me in here. Maybe going back to, I guess, similar to Ritu's question, you know, our channel checks also indicate that there is a lot of enthusiasm to switch over to POMOP, but I guess what's interesting is, you know, a lot of these physicians say sort of the switching impetus is dependent on progression of disease or next view is I'm not showing any meaningful benefit, yet those same physicians also talk about how the muscle assessment or other functional assessment frequency hasn't really changed since Next View is On launch or POMOP launch.

Speaker: So kind of curious; is that part of your marketing slash commercialization strategy to maybe induce more of these assessments so that any kind of slowing of efficacy or progression of disease can be picked up quicker and therefore drive more switch? Thanks so much.

Operator: So kind of curious, is that part of your marketing slash commercialization strategy to maybe induce more of these assessments so that any kind of slowing of efficacy or progression of disease can be picked up quicker and therefore drive more switch?

Operator: Thanks so much. Thanks, Dagan. Yeah, you know, it's...

Dae Ha: Thanks so much. Thanks, Dagan. Yeah, you know, it's...

Bradley Campbell: Thanks, Dagon. Yeah, you know, it's, you're hitting a very important issue, which is that this is really the first time physicians have had a real choice between products, and frankly, now they have two products that they're looking at. And so, it is an element of changing expectations for therapy, you know, we focus on, but actually, improvement is possible, you know, maybe for the first time. So, Yeah, it's very much an educational effort, and it's very much... Again, as patients and physicians get experience with the product, I think it will, you know, encourage them to look more closely at, you know, not just the primary measure, the six-minute walk before spinal And if we can change the expectation of those physicians and patients of what they're getting from their therapy, I think that very much favors the possibility of unfolding. Thank you.

Bradley Campbell: Hey, Brad, one other thing I'd add real quickly there. It's definitely something we're hearing from all the physicians about now, kind of the need for more standardization about how often they're monitoring their patients, and what parameters they're able to measure in a given clinical visit. So that's something that Amicus and the community are all now having to work together on as we have multiple treatments available, and patients really need to be armed better with all the information about making informed treatment decisions.

Jeff Castelli: Hey, Brad, one other thing I'd add real quickly there. It's definitely something we're hearing from all the physicians about now, kind of the need for more standardization about how often they're monitoring their patients, and what parameters they're able to measure in a given clinical visit. So that's something that Amicus and the community are all now having to work together on as we have multiple treatments available, and patients really need to be armed better with all the information about making informed treatment decisions.

Speaker: work together on as we have multiple treatments available, and patients really need to be armed better with all the information about making informed treatment decisions. The other side of it from an amicus perspective is that we're out there reminding people about the data that we saw, you know, in people that switched from ERT to POMOP that

Bradley Campbell: The other side of it from an amicus perspective is we're out there reminding people about the data that we saw, you know, in people that switched from ERT to POMOP that many of them saw improvements across different parameters. So in some way, stable is not necessarily the best outcome, that there is potentially an opportunity to regain some function. So that's something that we're reminding people about from the data.

Jeff Castelli: The other side of it from an amicus perspective is we're out there reminding people about the data that we saw, you know, in people that switched from ERT to POMOP that many of them saw improvements across different parameters. So in some way, stable is not necessarily the best outcome, that there is potentially an opportunity to regain some function. So that's something that we're reminding people about from the data.

Operator: Your next question comes with the line of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen Richter: Your next question comes with the line of Salveen Richter with Goldman Sachs. Your line is now open.

Operator: Hi, thank you so much for taking my question. This is Srinath from Salveen.

Speaker: So, do you see any trends that suggest that physicians are maybe preferring to switch patients from myozyme to another myozyme before evaluating them on that before going to pumbility in both ERT-experienced patients in the US as well as naive patients in Europe? And just a quick second part, you mentioned that you're now down to a 65 day lag between prescriptions and infusions. What is your best estimate around when you get to that 30 to 45 day target that you had mentioned early on?

Operator: So, do you see any trends that suggest that physicians are maybe preferring to switch patients from myozyme to another myozyme before evaluating them on that before going to pumbility in both ERT-experienced patients in the US as well as naive patients in Europe? And just a quick second part, you mentioned that you're now down to a 65 day lag between prescriptions and infusions. What is your best estimate around when you get to that 30 to 45 day target that you had mentioned early on?

Bradley Campbell: So, do you see any trends that suggest that physicians are maybe preferring to switch patients from myozyme to another myozyme before evaluating them on that before going to pumbility in both ERT-experienced patients in the US as well as naive patients in Europe? And just a quick second part, you mentioned that you're now down to a 65 day lag between prescriptions and infusions. What is your best estimate around when you get to that 30 to 45 day target that you had mentioned early on?

Bradley Campbell: Yeah, thanks for the questions. I think for the first one, no.

Bradley Campbell: Thanks for the questions. I think for the first one, no, I think the dynamic is really that in the U.S., as an example. Next slide was approved earlier than expected, and the majority of patients were already switched over, although there are some remaining patients at my time. And then outside of the U.S., it's much more of a mix. Circumstance. Excuse me.

Bradley Campbell: Yeah, thanks for the questions. I think for the first one, no. I think what the dynamic is really that, in the U.S., as an example, Nexvizime was approved far earlier than us, so the majority of patients were already switched over, although there are some remaining patients from iZyme, and then outside the U.S., it's much more of a mix. Excuse me.

Speaker: Circumstance. Excuse me. Thank you.

Operator: That was your last question. This concludes today's conference. Have a great day.

Bradley Campbell: So what we've seen is that we're taking switch patients from kind of each segment equally. And I think that reflects that we're making very good headway with the launch and that their physicians aren't preferencing switching to NexViz at first. I think it's just a reflection that we weren't available before, excuse me. And then, as relates to the time to infusion from prescription, our goal is really by the end of this year, we should be down to that 30 to 45 days.

Bradley Campbell: That's kind of the journey we were on with Galafold. By the end of the first full year of launch, we were down to sort of 30 plus days. The one difference here, the reason why we sort of say 30 to 45 days is these patients come in every other week for an infusion. So you probably have a two week kind of infusion window as well. But yeah, we're looking to do that by the end of this year. Thank you. Your next question...

Operator: Your next question comes to the line of Kristen Kluska with Cancer Fitzgerald. Your line is now open. Hi, this is Rick Miller on

Bradley Campbell: Yeah, great question. I think Sbastien highlighted one of the most important metrics, which is that the number of prescribers globally has increased by 50%. And that's really important. We want to see continued breadth, i.e. new prescribers. We also want to see continued depth, which we're seeing as well, which is prescribers, you know, adding their second or third or fourth prescription. So both of those dynamics have been really strong. To your question, in terms of where they are coming from,

Kristen Kluska: Yeah, great question. I think Sbastien highlighted one of the most important metrics, which is that the number of prescribers globally has increased by 50%. And that's really important. We want to see continued breadth, i.e. new prescribers. We also want to see continued depth, which we're seeing as well, which is prescribers, you know, adding their second or third or fourth prescription. So both of those dynamics have been really strong. To your question, in terms of where they are coming from,

Bradley Campbell: Again, I think it depends on the market. In a market like the UK, which is very concentrated, all of the key centers are prescribing. In markets like Germany and the US, you do have a top prescriber base, and we're seeing great penetration into those. But you also have sort of, you know, just because of the size of the geography and the more distribution of physicians, we do, now we're starting to see more and more, I would say, distal physicians that are prescribing as well. And so those are the dynamics we'll continue to speak to. And so far, we're seeing great uptake in both of those segments.

Kristen Kluska: Again, I think it depends on the market. In a market like the UK, which is very concentrated, all of the key centers are prescribing. In markets like Germany and the US, you do have a top prescriber base, and we're seeing great penetration into those. But you also have sort of, you know, just because of the size of the geography and the more distribution of physicians, we do, now we're starting to see more and more, I would say, distal physicians that are prescribing as well. And so those are the dynamics we'll continue to speak to. And so far, we're seeing great uptake in both of those segments.

Operator: Thank you. That was your last question. This concludes today's conference. Have a great day.

All participants are in a listen only mode.

Operator: Later, we will conduct a question 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.

Sbastien Martel: This leadership position is supported by our continuous investment in medical education and evidence generation. I'm very pleased to recognize the first publication of the results from our own Fabry Disease Registry, the Follow Me Registry, in July. These results were published in the Journal of Inherited Metabolic, and constitute a really important milestone for Amicus.

Sebastian Martel: This leadership position is supported by our continuous investment in medical education and evidence generation. I'm very pleased to recognize the first publication of the results from our own Fabry Disease Registry, the Follow Me Registry, in July. These results were published in the Journal of Inherited Metabolic Disorders and constitute a really important milestone for Amicus.

Operator: Thank you.

Speaker Change: Good morning.

Speaker Change: Thank you for joining our conference call to discuss amicus Therapeutics second quarter 2024 financial results and corporate highlights.

Speaker Change: Leading today's call, we have Bradley Campbell, President and Chief Executive Officer, Sebastien Martel, Chief Business Officer, Dr. Jeff Castelli, Chief Development Officer, Simon Harford, Chief Financial Officer, joining for Q&A with Dr. Mitchell Goldman Chief Medical Officer, and Alan Rosenberg, Chief Legal officer as referenced on slide two we may we may.

Sbastien Martel: We continue to generate positive data for Gallup. Data from the study aligns very well with previous observations from clinical tribes and extends the available data supporting the real-world multi-system benefits of gas. This publication, along with our ongoing medical education at Congress, is an important part of the story of this oral precision medicine and helps support the long-term growth potential we envision Galapagos will have in this growing fabric market. On slide 8, we know that there's a significant patient demand for Galafod and that the segment of the global Fabry market made of patients with a medical mutation has the potential to reach up to $1 billion in annual revenue by the end of the decade.

Sebastian Martel: We continue to generate positive data for Gallup. Data from this study aligns very well with previous observations from clinical tribes and extends the available data supporting the real-world multi-system benefits of GAP. This publication, along with our ongoing medical education at Congress, is an important part of the story of this oral precision medicine and helps support the long-term growth potential we anticipate Galapagos will have in this growing fabric market. On slide 8, we know that there is significant patient demand for Galafod and that the segment of the global Fabry market made up of patients with a medical mutation has the potential to reach up to $1 billion in annual revenue by the end of the

Speaker Change: Make forward looking statements within the meaning of the private Securities Litigation Reform Act of 1095 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 forward looking statements made on this call may turn out to be wrong and can be affected by inaccurate.

Speaker Change: <unk>, 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.

Sbastien Martel: We anticipate sustained growth in 2024 and beyond to be driven by several key drivers. First, the fabric market is growing robustly, with a significant portion of growth coming from finding new patients and reaching the diagnosed and treated population. As we mentioned previously, at the end of 2023, there were more than 2400 individuals on GALAFOLD, and about 1400 of those were individuals who were naive to any treatment before GALAFOLD.

Sebastian Martel: We anticipate sustained growth in 2024 and beyond, to be driven by several key drivers. First, the Foundry Market is growing robustly, with a significant portion of growth coming from finding new patients and reaching the diagnosed and treated population. As we mentioned previously, at the end of 2023, there were more than 2,400 individuals on GALATFOLD, and about 1,400 of those were individuals who were naive to any treatment before GALATFOLD.

Speaker Change: 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.

Sbastien Martel: As we mentioned previously, at the end of 2023, there were more than 2,400 individuals on GALAFOLD, and about 1,400 of those were individuals who were naive to any treatment before GALAFOLD, and those numbers obviously continue to grow this year and we'll be pleased to report an update at our year-end results. We've seen many new patients go on to treatment through newborn screening, family screening, and we continue to increase patient identification capability, through ongoing medical education and support of novel screening initiatives. Fabry remains, unfortunately, one of the most underdiagnosed rare diseases.

Speaker Change: The uncertainties that may impact them, we refer you to the forward looking statements and risk factors sections of our annual report on Form 10-K for the year ended December 31, 2023, and the quarterly report on Form 10-Q for the quarter ended June 32024 to be filed with the Securities and Exchange Commission today at this.

Sebastian Martel: And those numbers obviously continue to grow this year, and we will be pleased to report and update that on your end result. We've seen many patients go on to treatment through newborn screening, family screening, and we continue to increase patient identification capability, as well as ongoing Medical Education and Support of Novel Screening Initiatives. Fabry remains, unfortunately, one of the most underdiagnosed rare diseases.

Sbastien Martel: And those numbers obviously continue to grow this year, and we'll be pleased to report an update on our year-end results. We've seen many new patients go on to treatment through newborn screening, family screening, and we continue to increase patient identification capability through ongoing Medical Education and Support of Novel Screening Initiatives. Fabry remains, unfortunately, one of the most underdiagnosed rare diseases.

Operator: Time, it's my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer promptly.

Operator: Great. Thank you Andrew and welcome everyone to our second quarter 2024 conference call I'm pleased to highlight what has been a very successful first half of the year.

Sbastien Martel: So the more patients that can be identified, the more patients may benefit from GAP. We're also seeing many diagnosed untreated patients transition to treatment as the need for earlier treatment, especially in females, becomes better appreciated. The other piece is continuing to drive gap of market share of treated and medical patients through continued commercial execution. As noted, Galapagos currently has more than 60% of the global amenable market.

Sebastian Martel: So the more patients that can be identified, the more patients may benefit from GAP. We're also seeing many diagnosed untreated patients transition to treatment as the need for earlier treatment, especially in females, becomes better appreciated. The other piece is continuing to drive the gap of market share of treated and medical patients through continued commercial execution. As noted, Galapagos currently has more than 60% of the global amenable market.

Sbastien Martel: So the more patients that can be identified, the more patients may benefit from GAP. We're also seeing many diagnosed untreated patients transition to treatment as the need for earlier treatment, especially in females, becomes better appreciated. The other piece is continuing to drive the gap of market share of treated and medical patients through continued commercial execution. As noted, Galapagos currently has more than 60% of the global amenable market.

Speaker Change: Our global business, and particularly an incredible quarter.

Speaker Change: And this time, we have continued to build on our top line revenue growth momentum to put us well on our way to our first full year of non-GAAP profitability. While also advancing our mission of bringing hope to individuals and families affected by rare diseases.

Speaker Change: As we did in this morning's press release, let me highlight several key points.

Sebastian Martel: What we're seeing in our most mature markets is that we can reach up to 85-90% of market share. So we know that there is the potential to reach those levels in the global market share as well. And again, all of these efforts are supported solely by compliance and adherence rates to physician and patient education and support goals.

Speaker Change: First we continued our excellent commercial execution, we delivered total revenue of $127 million.

Speaker Change: In the quarter, representing 34% growth year over year or 36% on a constant currency basis.

Speaker Change: This strong performance has led us to increase our total revenue guidance for the full year to 26% to 31% from 25% to 30% previously.

Sbastien Martel: Additionally, we'll continue to make progress on expanding Galaford into new markets and extending the labels. There's still some markets in Latin America, the Middle East and Asia Pacific regions where Galaford is either newly reimbursed or we expect reimbursed. Also important to note here, we have often drug exclusivity in the U.S. and Europe, and in addition to our now 61 orange-book-listed patents, 45 of which provide protection into 2038 and beyond, including 14 composition of matter patents. This provides us the opportunity, continue to provide access to Galapagos globally for a long time to come. We intend to continue to protect and enforce our broad intellectual property rights.

Sebastian Martel: Additionally, we'll continue to make progress on expanding Galaford into new markets and extending the labels. There are still some markets in Latin America, the Middle East, and Asia Pacific regions where Galaford is either newly reimbursed, or we expect it to be reimbursed. Also important to note here, we often have drug exclusivity in the U.S. and Europe, and in addition to our now 61 orange book-listed patents, 45 of which provide protection into 2038 and beyond, including 14 composition of matter patents. This provides us the opportunity to continue to provide access to God or globally for a long time to come.

Sbastien Martel: Additionally, we'll continue to make progress on expanding Galaford into new markets and extending the labels. There are still some markets in Latin America, the Middle East, and Asia Pacific regions where Galaford is either newly reimbursed, or we expect it to be reimbursed. Also important to note here, we often have drug exclusivity in the U.S. and Europe, and in addition to our now 61 orange book-listed patents, 45 of which provide protection into 2038 and beyond, including 14 composition of matter patents. This provides us with the opportunity to continue to provide access to GALACTO globally for a long time to come. We intend to continue to protect and enforce our broad intellectual property rights.

Speaker Change: Product level <unk> continues its strong performance with $111 million in global revenue in the quarter, which represents 19% growth from last year on a constant currency basis.

Speaker Change: In the first half of the year operational growth was 17% year over year at constant exchange rates coming in at the high end of our guidance for the year.

Speaker Change: We continued to observe strong trends across our key performance indicators in all key geographies in the second quarter, including continued demand through new patient starts both from switch and naive populations in all of our leading markets and sustained patient compliance and adherence rates of over 90%.

Sebastian Martel: We intend to continue to protect and enforce our broad intellectual property rights. Looking ahead, we expect steady, double-digit growth for Galliford through 2024. And we remain confident that with our strong IP protection, Galliford is a long runway well into the next step. Turning now to Pompidou, on slide 10, we outline our global launch progress with Pompidou-Tiannapolda. For the second quarter of 2024, Mobility and Upholder reported revenue of $16 million.

Speaker Change: All of this is against the backdrop of significant growth in diagnosed untreated patients that we've highlighted previously.

Sbastien Martel: Looking ahead, we expect steady, double-digit growth for Galaford throughout 2024, and we remain confident that, with our strong IP protection, Galaford has a long runway well into the next decade. Turning now to Pompedities, on slide 10, we outline our global launch progress with Pompidity and Upholder. For the second quarter of 2024, Humbility and Upholder reported revenue of $16 million.

Sbastien Martel: Looking ahead, we expect steady, double-digit growth for Galaford throughout 2024, and we remain confident that with our strong IP protection, Galaford is a long runway well into the next step. Turning now to Pompe disease on slide 10, we outline our global launch progress with Pompidou-Tianopolda. For the second quarter 2024, Mobility and Upholder reported revenue reached $16 million.

Speaker Change: Eight years now after our first commercial launch it's remarkable to see what an impact. This medicine is having on people living with fabry disease around the world and we continue to <unk>, serving as the foundation of our business for the next decade and beyond.

Sbastien Martel: This represents an increase of 44% compared to the first quarter of 2024 and provides a strong foundation for the remainder of the year, in the U.S. Q2 benefitted from the remaining clinical trial patients transitioning to commercial treatments in addition to patients switching from other therapies. We continue to see a majority of patients switching from Nexviazime, about 68%, and the remaining from Lumizyme. This means we're switching patients proportionally from both products in the U.S. We're also seeing a broadening and deepening of our prescriptions with more sites coming online and multiple new prescriptions from physicians coming in at accelerated and increased rates over Q1.

Speaker Change: Based on the sustained performance, we are increasing our full year 2020 for revenue growth guidance range for the second time.

Sbastien Martel: This represents an increase of 44% compared to the first quarter of 2024 and provides a strong foundation for the remainder of the year. Q2 benefited from the remaining clinical trial patients transitioning to commercial treatments in addition to patients switching from other therapies. We continue to see a majority of patients switching from Nexviazine, about 68%, and the remaining from Lumizine. This means we're switching patients proportionally from both products in the U.S. We're also seeing a broadening and deepening of our prescriptions, with more sites coming online and multiple new prescriptions from physicians coming in at accelerated and increased rates over Q1.

Sebastian Martel: This represents an increase of 44% compared to the first quarter of 2024 and provides a strong foundation for the remainder of the year in the U.S. Q2 benefited from the remaining clinical trial patients transitioning to commercial treatments, in addition to patients switching from other therapies. We continue to see a majority of patients switching from Nexviazime, about 68%, and the remaining from Lumizyme. This means we're switching patients proportionally from both products in the U.S. We're also seeing a broadening and deepening of our prescriptions, with more sites coming online and multiple new prescriptions from physicians coming in at accelerated and increased rates over Q1.

Speaker Change: We started the year with a growth range of 11% to 16% in May we raised it to 13% to 17%.

Speaker Change: And now we are projecting 2014% to 18%.

Speaker Change: Second let me highlight the continued strong global commercial launch of possibility in a folder our novel therapy for late onset Pompe disease.

Speaker Change: Our ability to up Volta has been and will continue to be a huge growth driver for us. This year, we've already made great progress against our key performance indicators, which continue to demonstrate the strength of this launch.

Speaker Change: First and foremost our number one focus for the year is to maximize the number of patients on therapy by year end.

Speaker Change: It's great to report that the rate of new commercial patients coming on possibility in a folder in 2024 continues to progressed exceptionally well in.

Sbastien Martel: Outside of the U.S., we're seeing patients from all three segments, some switching from Myosin, some from Nexiodine at a proportional rate to the respective market shares, and some from naive populations. That's exactly what we want to be seeing at this stage in the long run.

Sbastien Martel: Outside of the U.S., we're seeing patients from all three segments, some switching from myosine, some from nexiodine at a proportional rate to the respective market shares, and some from naive populations. That's exactly what we want to be seeing at this stage in the long run.

Sebastian Martel: Outside of the U.S., we're seeing patients from all three segments, some switching from Myozyme, some from NextGen at a proportional rate to the respective market shares, and some from naive populations. That's exactly what we want to be seeing at this stage in the long term.

Speaker Change: In the second quarter, we saw the largest number of new commercial patients, meaning patients who were not in our clinical studies and as of the end of July we had 186 patients who had been treated or scheduled for treatment we.

Speaker Change: We are incredibly pleased with demand globally from patients and physicians from this new therapy.

Sebastian Martel: A few updates on the launch in Europe. I am pleased to announce that Switzerland's Swissmedic has approved Pombilichinoproda as a long-term enzyme replacement therapy and enzyme stabilizer for adults with LOT, and all the key regulatory authorities who recognize the benefits of this gap. In Germany, we have successfully finalized negotiations with the National Association of Statutory Health Funds, and we're very pleased with the outcome, which continues to reflect the value health technology assessments are seeing with this therapy.

Sbastien Martel: A few updates on the launch in Europe. I'm pleased to announce that Switzerland's Swissmedic has approved Pombilitanoproda as a long-term enzyme replacement therapy and enzyme stabilizer for adults with LOG, and all the key regulatory authorities who recognize the benefits of this gap. In Germany, we have successfully finalized our key negotiations with the National Association of Statutory Health Funds, and we're very pleased with the outcome, which continues to reflect the value health technology assessments are seeing with this therapy.

Sbastien Martel: A few updates on the launch in Europe. I'm pleased to announce that Switzerland's Swissmedic has approved Pombilicinoproda as a long-term enzyme replacement therapy and enzyme stabilizer for adults with LOG, and all the key regulatory authorities who recognizes the benefits of this gap. In Germany, we have successfully finalized the negotiations with the National Association of Statutory Health Funds, and we're very pleased with the outcome, and it continues to reflect the value health technology assessments are seeing with this therapy.

Speaker Change: And consistently hear inspiring anecdotes from health care professionals around the world on how their patients are responding to PON ability in our Florida, which will continue to fuel the momentum throughout the year and beyond.

Sebastian: Sebastian will provide more details in a moment for the switch dynamics in the U S Europe and the U K continue to look strong and we're seeing great uptake in naive patients in markets outside the U S. As well, we're also making significant progress on reimbursement front globally and this includes moving patients more quickly through the insurance process in the U S. As we have anticipated.

Speaker Change: Throughout the remainder of the beer will focus on increasing patient access as we gain reimbursement and launch in additional countries throughout Europe.

Sbastien Martel: The prize acknowledges the added benefit of mobility plus a folder for LOPD patients, and we'll be closely aligned with the price we set at launch. Additionally, we anticipate this to be an important reference point for future pricing and reimbursement discussions. In Spain, our newest launch country, we've added a significant number of patients and new prescribing accounts, all within the country's first quarter of launch. We remain very pleased with the uptake of Combiditi Anopoda in the UK, where we've captured greater than a third of market share within the region. With the benefit of beams, mobility has been pulled up, and they have actually been in the market for two years in the UK.

Sbastien Martel: The price acknowledges the added benefits of POMIDI T-plus or FOLDA for LOPD patients, and we'll be closely aligned with the price we set at launch. Additionally, we anticipate this to be an important reference point for future pricing and reimbursement discussion. In Spain, our newest launch country, we've added a significant number of patients and new prescribing accounts, all within the country's first quarter of launch. We remain very pleased with the uptake of Combinity Anapoda in the UK, where we've captured greater than a third of market share within the region.

Sebastian Martel: The prize acknowledges the added benefits of Combinity Plus or Folda for LOPD patients and will be closely aligned with the price we set at launch. Additionally, we anticipate this to be an important reference point for future pricing and reimbursement. In Spain, our newest launch country, we've added a significant number of patients and new prescribing accounts, all within the country's first quarter of launch. We remain very pleased with the uptake of POMBIDI Tianopoda in the UK, where we've captured greater than a third of the market share within the region.

Speaker Change: On the regulatory front, we're pleased to announce that in July the Swiss medic approve our ability in a folder for adults living with late onset pompe disease in Switzerland.

Speaker Change: For the full year 2024, we're well on track to deliver our guidance of $62 million to $67 million in global possibility in a folder sales, which will be a significant contributor to our growth.

Speaker Change: Set us on a great course to achieve our ambition for <unk> to become the new standard of care treatment in this devastating disease.

Sebastian Martel: With the benefit of EAMS, mobility has been pulled up, and it has actually been in the market for two years in the UK. And we believe that the current market share is indicative of how this product can perform over two-year periods, which is a great example. Potential Human Interface. Given Pompidou-Tianopoulos' solid commercial executions thus far, in the first half of 2024, we're well on our way to deliver our full-year revenue guidance of $62 to $67 million for Pompidou-Tianopoulos. Moving to slide 11.

Sbastien Martel: The Benefit of Inns, Umbilitia, and Apoldab have actually been in the market for two years in the UK, and we believe that the current market share is indicative of how this product can perform over two-year periods, which is a great example of Potential Humanity.

Speaker Change: Incredibly pleased to be providing a real choice for patients and challenging therapeutic expectations for both physicians and people living with Pompe disease.

Sbastien Martel: And we believe that the current market share is indicative of how this product can perform over two-year periods, which is a great example of potential demand and uptake. Given Pompidou-Tianopoulos' solid commercial executions thus far, in the first half of 2024, we're well on our way to deliver our full-year revenue guidance of $62 to $67 million for Pompidou-Tianopoulos. Moving to slide 11.

Speaker Change: And finally, as we continue our excellent commercial execution across both therapies and prudently manage our resources. We are excited to share that amicus was profitable on a non-GAAP basis in the second quarter as well as the first half of the year. We are confident we will see this number increase as we look to achieve our full year non-GAAP profitability in 2024.

Sbastien Martel: Given Pompility and Uphold are solid commercial executions thus far, the first half of 2024, we're well on our way to deliver our full-year revenue guidance of $62 to $67 million for Pompility and Uphold. Moving to slide 11, very pleased with the launch momentum for the first half of the year. As Bradley mentioned earlier, there are about 196 patients that have been treated or are scheduled to be treated at the end of July. That's about 174 on therapy and the remaining 12 are scheduled.

Operator: Okay.

Speaker Change: I had on slide four as we laid out at the beginning of the year, we're focused on achieving our four key strategic priorities for 2024, including.

Sebastian Martel: I'm very pleased with the launch momentum for the first half of the year. As Bradley mentioned earlier, there are about 196 patients that have been treated or are scheduled to be treated at the end of July. That's about 174 on therapy, and the remaining 12 are scheduled.

Sbastien Martel: I'm very pleased with the launch momentum for the first half of the year. As Bradley mentioned earlier, there are about 196 patients that have been treated or are scheduled to be treated at the end of July. That's about 174 on therapy, and the remaining 12 are scheduled.

Speaker Change: First sustaining double digit <unk> revenue growth and again on the back of strong sales growth. So far we've raised the expected annual growth rate of California to 14% to 18%.

Speaker Change: Successfully executing on multiple commercial launches of possibility in a folder advancing our ongoing studies to support our medical and scientific leadership in Fabry and Pompe disease.

Sbastien Martel: We remain very pleased with the ongoing demand for this therapy as the rate of new crematorial patients coming on to POMIDI-Tenofolda continues to increase across all three months. Q2, in particular, was a very strong quarter, having seen the largest number of new demand patients being prescribed Pomegranate Probiotics. These are individuals not from clinical trials choosing to come into commercial practice.

Sebastian Martel: We remain very pleased with the ongoing demand for this therapy, and the rate of new commercial patients coming on to POMBILITY Anaphoda continues to increase across all three months. Q2, in particular, was a very strong quarter, having seen the largest number of new demand patients being prescribed pomidigliano products. These are individuals not from clinical trials choosing to come into commercial practice. It is important that it further supports our thesis that launch momentum will continue to build throughout the year, as we move more quickly through the prescription to treatment process in the U.S. and as we launch new countries in Europe. And as we launch new countries in Europe, we launch new countries in Europe. Our launchers leveraged our highly experienced cross-functional teams, and we've had great outreach with K.O.P. Enigres.

Sbastien Martel: We remain very pleased with the ongoing demand for this therapy, and the rate of new commercial patients coming on to Pombilithia Nephroda continues to increase across all three months. Q2, in particular, was a very strong quarter, having seen the largest number of new demand patients being prescribed POMID. These are individuals not from clinical trials choosing to come onto a commercial platform.

Speaker Change: Maintaining a strong financial position as we carefully manage our expenses and investments to achieve non-GAAP profitability for the full year.

Operator: With that highlight let me now hand, the call over to Sebastian who will go through our commercial performance in more detail Sebastian.

Operator: Thank you Bradley and good morning to everyone on the call.

Sbastien Martel: This is important that it further supports our thesis that launch momentum will continue to build throughout the year, as we move more quickly through the prescription to treatment process in the U.S, and as we launch new countries in Europe. Our launch has leveraged our highly experienced cross-functional teams and we've had great outreach with TLP in the years. We're seeing an increase in the depth and breadth of prescribers across all pre-markets. In Q2 alone, the global number of prescribing accounts increased by 50 percent.

Sbastien Martel: This is important because it further supports our thesis that launch momentum will continue to build throughout the year, as we move more quickly through the prescription to treatment process in the U.S. and as we launch new countries in Europe. Our launch has leveraged our highly experienced cross-functional teams, and we've had great outreach with Ethiopian leaders. We're seeing an increase in the depth and breadth of prescribers across all pre-markets. In Q2 alone, the global number of prescribing accounts increased by 50%.

Speaker Change: As always I'll start by providing you with more details on our get absolute performance for the quarter.

Speaker Change: On slide six for the second quarter of 2024, <unk> reported revenue reached $111 million driven by strong patient demand, particularly from our leading buckets.

Speaker Change: We ended the second quarter with more than 60% of the global market share for treated fabry patients with amenable mutations and the good news is there are still many more potential patients eligible for our therapy.

Sebastian Martel: We're seeing an increase in the depth and breadth of prescribers across the whole pre-market. In Q2 alone, the global number of prescribing accounts increased by 50%. All court-trading centers have been engaged, and they've provided very positive feedback, feedback from both HCPs and other stakeholders about our business approach, our support, and our 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 POMBILITY and UPHOLDA.

Sbastien Martel: All core treating centers have been engaged and they've provided very positive feedback, feedback from both HCPs and other stakeholders to our business approach, our support, and our 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 POMBILITY and UPHOLDA. In the U.S., the largest payers have already put POMBILITY and UPHOLDA onto their respective formularies, and we have also seen strong acceptance by Medicare and Medicaid. The overall time from prescription to infuser is now down to around 65 days with payer approvals happening in around 30 days, and the last few commercial patients have actually come in significantly quicker.

Bradley: As Bradley highlighted given the continued strong performance of <unk> throughout the second quarter.

Speaker Change: Raising again, a full year of 2020 for revenue growth guidance range to now 14% to 18%.

Speaker Change: Turning to slide seven.

Speaker Change: Our results in the second quarter highlights the strength of our global commercial efforts with <unk>.

Speaker Change: Demand for chemicals globally continues to be strong with patients added in all major markets did he bring operational growth of 19% over the same period in 2023 at constant exchange rates.

Sebastian Martel: In the U.S., the largest payers have already put POMBILITY and UPHOLDA on their respective formularies, and we have also seen strong acceptance by Medicare and Medicaid. The overall time from prescription to infuser is now down to around 65 days, with pay or poobles happening in around 30 days, and the last few commercial patients have actually come in significantly quicker.

Speaker Change: Our leading market such as the UK the U S. EU countries and Japan remains the biggest drivers of patient demand and gives great confidence in the growth of this product has over the long term.

Speaker Change: The U S. In particular, so impressive patient demand the highest number of net new patients seen in over five years and we expect this trend to continue for the full year.

Jeff Castelli: We believe this will continue to improve. Today we're launching in Germany, the UK, the US, Austria, and Spain, but we remain in active pricing and reimbursement discussions with additional major European markets as we focus on securing broad patient access throughout the European market. Overall, we've had a very strong first half, and we're very pleased with the building momentum on patients. Throughout the second half of 2024, our focus will be on maximizing the number of patients on therapy by year-end.

Sebastian Martel: We believe this will continue to improve. Today we're launching in Germany, the UK, the US, Austria, and Spain, but we remain in active pricing and reimbursement discussions with additional major European markets as we focus on securing broad patient access throughout the European market. Overall, we've had a very strong first half, and we're very pleased with the building momentum for patient care. Throughout the second half of 2024, our focus will be on maximizing the number of patients on therapy by year-end.

Sbastien Martel: We believe this will continue to improve. Today we're launching Germany, the UK, US, Austria, Spain, but we remain in active pricing and reimbursement discussions with additional major European markets as we focus on securing broad patient access throughout the European market. Overall, we've had a very strong first half and we're very pleased with the building momentum on patient, Throughout the second half of 2024, our focus will be on maximizing the number of patients on therapy by year-end.

Speaker Change: Within the global mix, which is about 42% switch and 58% now you.

Speaker Change: We're now seeing stronger of taking naive populations, we continued to achieve high market shares in countries, where we have been approved the longest.

Speaker Change: <unk> opportunity to continue to switch patients over to <unk> and to keep growing the market as we penetrate the diagnosed untreated annuity dinos segments.

Speaker Change: All of that is underpinned by sustained compliance and adherence rates that continues to exceed 90%.

Sbastien Martel: So in summary, we're very pleased with the launches of Pompidou Tenor Folder across the first wave of countries. The strength of our clinical data, the depth of our experience and talent we have at Amicus gives us great confidence in our ability to make a real difference for people living with Pompidou. We believe Amicus is in a great position with our second commercial launch. And with that, I will hand the call over to Jeff Castelli, our Chief Development Officer, to discuss the ongoing clinical study and regulatory timelines.

Sebastian Martel: So, in summary, we're very pleased with the launches of Combilic and Ophoda across the first wave of countries; the strength of our clinical data, the depth of our experience, and talent we have at Amicus gives us great confidence in our ability to make a real difference for people living with Pompidou. We believe Amicus is in a great position with our second commercial launch. And with that, I will hand the call over to Jeff Castelli, our Chief Development Officer, to discuss the ongoing clinical study and regulatory timelines.

Jeff Castelli: So, in summary, we're very pleased with the launches of the Pompidou Tenor Folder across the first wave of countries. The strength of our clinical data, the depth of our experience, and the talent we have at Amicus gives us great confidence in our ability to make a real difference for people living with Pompidou. I believe Amicus is in a great position with our second commercial launch. And with that, I will hand the call over to Jeff Castelli, our Chief Development Officer, to discuss the ongoing clinical studies and regulatory timeline. Jeff.

Speaker Change: Reiterating our belief that those patients will go and get a cold dominance in sterling.

Speaker Change: We're very pleased to see the prescribers continue to position got pulled.

Speaker Change: The treatment of choice for amenable fabry patients this.

Speaker Change: This leadership position is supported by our continuous investment in medical education and evidence generation.

Speaker Change: I'm very pleased to recognize the first publication of the results from our own Fabry disease registry. The follow me registry in July these.

Jeff Castelli: Thank you, Sebastian. Good morning, everyone.

Jeff Castelli: Thank you, Sebastian, and good morning, everyone. On slide 12, we outline how we continue to build the body of evidence for pumbility and opphalta through our ongoing clinical studies, as well as through our Amicus Pompei registration, as we also continue to execute on expanding commercial access through reimbursement dossiers and regulatory. As we enter this second phase of launch, in addition to the various reimbursement dossiers that we have or are in the process of submitting, we also have multiple ongoing or planned regulatory submissions for marketing approval in new geographies throughout the year. We've just mentioned marketing approval in Switzerland. Additionally, we have our regulatory DASIs being reviewed in Australia and Canada, and we're working towards a submission here in Japan before the end of the year.

Speaker Change: These results were published in the journal of inherited metabolic disease and consider a really important milestone importantly, we continue to generate positive data forgetful.

Jeff Castelli: On slide 12, we outline how we continue to build the body of evidence for pumbility and opphalta through our ongoing clinical studies, as well as through our Amicus Pompei Registry, as we also continue to execute on expanding commercial access through reimbursement dossiers and regulatory. As we enter this second phase of launch, in addition to the various reimbursement dossiers that we have or are in the process of submitting, we also have multiple ongoing or planned regulatory submissions for marketing approval in new geographies throughout the U.S. We've just mentioned approval in Switzerland.

Jeff Castelli: On slide 12, we outline how we continue to build the body of evidence for pumbility and opphalta through our ongoing clinical studies, as well as through our Amicus Pompei registration, as we also continue to execute on expanding commercial access through reimbursement dossiers and regulatory. As we enter this second phase of launch, in addition to the various reimbursement dossiers that we have or are in the process of submitting, we also have multiple ongoing or planned regulatory submissions for marketing approval in new geographies throughout the U.S. We've just mentioned approval in Switzerland.

Speaker Change: Data from the study along very well with previous observations from clinical trials and extends the available data supporting the real world multi system benefits of Gaslog.

Andrew Faughnan: Publication, along with our ongoing medical education of Congress.

Speaker Change: <unk> talked to the story of this oral precision medicine and help support the long term growth potential we envision get off we will have in this growing market.

Speaker Change: On slide eight we know that there is a significant patient demand will get a hold in that segment of the global slightly baskets made of patients with amenable mutations has the potential to reach up to $1 billion in annual revenue by the end of the decade.

Jeff Castelli: Additionally, we have our regulatory dossiers being reviewed in Australia and Canada, and we're working towards a submission here in Japan before the end of the year. For the younger Pompeii community, we continue to enroll the ongoing Open Label ZIP study for children living with laid-on-set Pompeii disease and the Open Label Reseller study for children living with infantile onset. We see this as an important opportunity to support label expansions into these patient segments in the years ahead and, very importantly, to address the significant unmet needs for these children.

Jeff Castelli: Additionally, we have our regulatory DASIs being reviewed in Australia and Canada, and we're working towards a submission here in Japan before the end of the year. For the younger Pompeii community, we continue to enroll the ongoing Open Label Zip study for children.

Andrew Faughnan: We anticipate sustained growth in 2024 and beyond will be driven by several key drivers.

Simon Harford: 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 Rizela study for children living with infantile-onset Pompeii disease. We see this as an important opportunity to support label expansions into these patient segments in the years ahead and, very importantly, to address the significant unmet needs for these children. Through ongoing clinical studies and the Amicus Pompei Registry, we continue to generate evidence on the differentiated mechanism of action and on the long-term impact of pompidoli and opphalta across endpoints in patient populations.

Jeff Castelli: Children living with late-onset Pompe disease.

Speaker Change: First the fabry market is growing robustly the significant portion of growth coming from finding new patients and reaching the diagnosed untreated population.

Jeff Castelli: and the Open Label Rosella Study for Children Living with Infantile Onset Palm Peel. We see this as an important opportunity to support label expansions into these patient segments in the years ahead and, very importantly, to address the significant unmet needs for these children. Through ongoing clinical studies and the Amicus Pompei Registry, we continue to generate evidence on the differentiated mechanism of action and on the long-term impact of POM building up FODA across endpoints and patient populations.

Bradley Campbell: As we mentioned previously at the end of 2023, there were more than 2400 individuals on got pulled in about 1400 of those were individuals.

Bradley Campbell: Were naive to any treatment before gaslog.

Jeff Castelli: Through ongoing clinical studies and the Amicus Pompei Registry, we continue to generate evidence on the differentiated mechanism of action and on the long-term impact of POM building up FODA across endpoints and patient populations. Our medical conference presence and publications continue to be an important part of our education effort. Finally, as highlighted in the pipeline slide in the appendix, for our earlier SAGE pipeline, we continue to focus on novel approaches to next-generation therapies in Fibro and pumping. With that, I would now turn the call over to Simon Harford, our Chief Financial Officer, to review our financial results, guidance, and outlook.

Bradley Campbell: And those numbers, obviously continue to grow this year and we'll be pleased to report.

Bradley Campbell: At our year end results.

Bradley Campbell: <unk> seen many new patients go into treatment through newborn screening family screening and we continue to increase patient identification capabilities.

Jeff Castelli: Our medical conference presence and publications continue to be an important part of our education effort. Finally, as highlighted in the pipeline slide in the appendix, for our earlier SAGE pipeline, we continue to focus on novel approaches to next-generation therapies in Fabry and pumping. 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 Harford: Our medical conference presence and publications continue to be an important part of our education effort. Finally, as highlighted in the pipeline slide in the appendix, for our earlier SAGE pipeline, we continue to focus on novel approaches to next-generation therapies in Fabry and pumping. 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.

Bradley Campbell: Through ongoing medical education, and support renewable screening initiatives.

Bradley Campbell: But he remains unfortunately, one of the most underdiagnosed rare disease. So the more patients that can be identified the more patients may benefit from to Apple.

Bradley Campbell: We're also seeing many diagnosed untreated patients.

Bradley Campbell: <unk> two treatments.

Bradley Campbell: Need for earlier treatments, especially with females becomes better I appreciate it.

Bradley Campbell: The other piece is continuing to drive <unk> market share of treated amenable patients who continued commercial execution.

Simon Harford: The global geographic breakdown of total revenue during the quarter consisted of $77 million, or 60% of revenue generated outside the United States, and the remaining $50 million, or 40% coming from within the U.S. Cost of goods sold as a percentage of net sales was 9% in Q2 2024, as compared to 10% for the prior year period, remaining relatively flat. Total GAAP operating expenses decreased to $100 million for the second quarter of 2024, as compared to $104 million in the same period last year, a decrease of 4%.

Simon Harford: Our financial overview begins on slide 14 with our income statement for the second quarter ending June 30th, 2024. For Q2, we achieved total revenue of $127 million, which is a 34% increase over the same period in 2023. At constant exchange rates, revenue also grew 36%.

Speaker Change: As noted that will currently has more than 60% of the global amenable market, where we are.

Speaker Change: Seeing in our most mature market and reach up to 80, 590% of the market share. So we know that there is the potential to reach those levels in the global market share as well.

Bradley Campbell: And again all of these efforts are supported by solid compliance and adherence rates.

Bradley Campbell: Physician and patient education and support programs.

Simon Harford: The global geographic breakdown of total revenue during the quarter consisted of $77 million, or 60% of revenue generated outside the United States, and the remaining $50 million, or 40% coming from within the U.S. Cost of goods sold as a percentage of net sales was 9% in Q2 2024, as compared to 10% for the prior year period, remaining relatively flat. Total GAAP operating expenses decreased to $100 million for the second quarter of 2024, as compared to $104 million in the same period last year, a decrease of 4%.

Speaker Change: Additionally, we'll continue to make progress on expanding that according to new markets and extending the labels are still some markets in Latin America, the middle East and Asia Pacific regions, where gaslog is either newly reimbursed or we expect reimbursements.

Bradley Campbell: Also important to note here, we have orphan drug exclusivity in the U S and Europe.

Speaker Change: In addition to our now 61 Orange book listed patents for <unk>.

Bradley Campbell: 45 of which provide protection into 2030 and beyond including <unk> composition of matter methods.

Bradley Campbell: This provides us the opportunity to.

Bradley Campbell: Continue 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.

Speaker Change: Looking ahead, we expect steady double digit growth organic hold throughout 2024, and we remain confident that with our strong IP protection Gulfport has a long runway well into the next decade.

Simon Harford: On a non-GATT basis, total operating expenses decreased to $82 million for the second quarter of this year as compared to $84 million in the same period last year, a decrease of 2%. We define non-GAAP operating expenses, research and development, and SG&A expenses, excluding stock-based compensation. Loss on impairment of assets, changes in fair value of contingent consideration, restructuring charges, and depreciation and amortization.

Speaker Change: Turning now to compete these on slide 10, we outline our global launch progress with continued channel pull them over.

Speaker Change: For the second quarter, 2024, 4 million channel pulled our reported revenue.

Speaker Change: <unk> $16 million. This represents an increase of 44% compared to the first quarter of 2024 and provides a strong foundation for the remainder of the year.

Bradley Campbell: In the U S Q2 benefited from the remaining clinical trial patients transitioning to commercial treatments.

Simon Harford: On a gap basis, net loss for the second quarter of 2024 was $16 million, or $0.05 per share, compared to a net loss of $43 million, or $0.15 per share, for the second quarter of 2023. In Q2, non-GAAP net income was $18 million, or $0.06 per share, compared to a loss last year in the same period of $20 million, or $0.07 per share. Cash Equipment's marketable securities were $260 million on June 30th.

Bradley Campbell: Addition to patients switching from other therapies.

Speaker Change: We continue to see a majority of patients switching from mixed design about 68% and the remaining from Luna design.

Bradley Campbell: This means we're switching patients proportionately from both products in the U S. We're also seeing a broadening and deepening of our prescriptions with more sites coming online and multiple new prescriptions from physicians coming in accelerated and increased rates over Q1.

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

Speaker Change: Switching from mine design some per minute next July.

Speaker Change: <unk> rates to the respective market shares and some from the naive populations that's exactly what we want to be seeing at this stage in the launch.

Bradley Campbell: A few updates on the launch in Europe we're.

Speaker Change: We're pleased to announce that Switzerland Swiss medic as accrued competition are pulled out of the long term enzyme replacement therapy and enzymes stabilizer for adults with Italy.

Sbastien: Another key regulatory authority, who recognizes the benefits of this therapy.

Sbastien: In Germany, we have statistically to finalize the negotiations with the National Association of statutory health funds and we're very pleased with the outcome.

Sbastien: Continues to reflect the value of health technology assessments.

Sbastien: Our soon with this therapy.

Sbastien: The price I'm going to just the added benefit of community plus a holdup coelho PD patients and we'll be closely along with the price we sell at launch.

Simon Harford: $62 to $67 million for Pongbility and Oxalda sales for the full year 2024. Our full year 2024 non-GAAP operating expense guidance has been narrowed from $345 to $365 million dollars to $345 to $360 million dollars, or a five percent reduction at the top end. With our commitment to full-year non-gap profitability during the first full year of launch for POMbility and NotFolder, we are keeping operating expense growth in low single digits year-over-year at the midpoint of guidance.

Simon Harford: $62 to $67 million for Pongbility and Oxalda sales for the full year 2024. Our full year 2024 non-GAAP operating expense guidance has been narrowed from $345 to $365 million dollars to $345 to $360 million dollars, or a five percent reduction at the top end. With our commitment to full-year non-gap profitability during the first full year of launch for Pombility and NotFolder, we are keeping operating expense growth in low single digits year-over-year at the midpoint of guidance.

Simon Harford: $62 to $67 million for probability and upsell sales for the full year 2024. Our full year 2024 non-GAAP operating expense guidance has been narrowed from $345 to $365 million dollars to $345 to $360 million dollars, or a five cent reduction at the top end. With our commitment to full-year non-gap profitability during the first full year of launch for POMbility and OpFolder, we are keeping operating expense growth in low single digits year-over-year at the midpoint of guidance.

Bradley Campbell: Additionally, we anticipate this to be an important reference point future pricing and reimbursement discussions.

Bradley Campbell: In Spain, our newest launch country, we've added a significant number of patients in new prescribing accounts all within the country's first quarter of launch.

Speaker Change: We remain very pleased with the uptake of commodity chemical than the U K, where we've captured.

Speaker Change: Greater than third market share within the region.

Speaker Change: With the benefit of beams immediately pulled up.

Speaker Change: Production being in the market for two years in the UK and we believe that the current market share Nikkei two of how this product can perform over two year periods, which is a great example of it.

Bradley Campbell: The potential demand and uptake.

Bradley Campbell: Given competition are pulled a solid commercial execution, thus far the first half of 2024.

Simon Harford: 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 which aren't yet reimbursed, as well as next-generation manufacturing for Pombility. In the second quarter of 2024, non-gap profitability was $18.5 million, and for the first half of 2024, $13.9 million. With our total revenue guidance of 26 to 31% growth, we remain comfortably on track for 2024 to be our first full year of non-gap profitability, as profitability continues to accelerate in the second half of the year. With that, I will turn the call back over to Bradley for our closing remarks.

Simon Harford: 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 which aren't yet reimbursed, as well as next-generation manufacturing for Pombility. In the second quarter of 2024, non-gap profitability was $18.5 million, and for the first half of 2024, $13.9 million. With our total revenue guidance of 26 to 31% growth, we remain comfortably on track for 2024 to be our first full year of non-gap profitability, as profitability continues to accelerate in the second half of the year. And with that, I will turn the call back over to Bradley for our closing remarks. Bradley

Simon Harford: 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 which aren't yet reimbursed, as well as next-generation manufacturing for Pombilities. In the second quarter of 2024, non-GAAP profitability was $18.5 million, and for the first half of 2024, $13.9 million. With our total revenue guidance of 26% to 31% growth, we remain comfortably on track for 2024 to be our first full year of non-gap profitability as profitability continues to accelerate in the second half of the year. And with that, let me turn the call back over to Bradley for our closing remarks.

Bradley Campbell: Well on our way to deliver our full year revenue guidance of $62 million to $67 million or competing center program.

Bradley Campbell: Moving to slide 11.

Bradley Campbell: Very pleased with the launch momentum through the first half of the year.

Bradley Campbell: As Brendan mentioned earlier, there are about 196 patients that had been treated or are scheduled to be treated.

Bradley Campbell: The end of July.

Bradley Campbell: 174 on therapy, and the remaining 12 more scheduled.

Bradley Campbell: We remain very pleased with the ongoing demand for this therapy.

Speaker Change: Eight of new commercial patients coming onto <unk> pulled up.

Sbastien: To increase across all three markets.

Sbastien: Q2 in particular was a very strong quarter, having seen the largest number of new demand patients being prescribed point are you going to pull down.

Sbastien: These are individual not from clinical trials choosing to come onto commercial therapy.

Bradley Campbell: Thank you, Simon, Jeff, and Sebastian. As you can all see, we've been highly focused this year on our commercial execution, continuing to strengthen our financial profile, and ultimately delivering value for our shareholders. Our first half performance has laid the groundwork for us to continue to achieve our goals and fulfill our mission of delivering life-changing therapy. And with that, Operator, we can now open the call.

Sbastien: This is unfortunate as further supports our thesis that launch momentum will continue to build throughout the year.

Sbastien: As we move more quickly through the prescription treatment process in the U S.

Sbastien: And as we launched new countries in Europe.

Sbastien: Our launches leverage our highly experienced cross functional teams and we've had great outreach with key opinion leaders.

Speaker Change: We're seeing an increase in depth and breadth of prescribers across all three markets.

Operator: Yes, ladies and gentlemen, if you have a question, please press star 1-1 on your touch-tone telephone. At this time we request that you only ask one question. If you have additional questions, please enter back into the queue. Thank you. Your first question comes from... One moment, please. This question comes to the line of Tazeen Ahmad with BofA Securities.

Operator: Yes, ladies and gentlemen, if you have a question, please press star 11 on your touchtone telephone. At this time, we request that you only ask one question. If you have additional questions, please enter back into the queue. Thank you. Your first question comes from... One moment, please. This question comes from the line of Tazeen Ahmad with BofA Securities.

Sbastien Martel: In Q2 alone the global number of prescribing accounts increased by 50%.

Speaker Change: All core treating centers have been engaged.

Sbastien Martel: <unk> provides a very positive feedback.

Sbastien Martel: Feedback from both HCP and other stakeholders.

Sbastien Martel: Business approach supports patient focus.

Speaker Change: Finally, we signed an important metric we track is our progress with access and reimbursement.

Operator: Your line is now open. Hi guys. Thanks for taking my questions.

Speaker Change: We have a highly experienced team who are engaging as positive conversations with figures demonstrate the value of Omnichannel program.

Speaker Change: In the U S. The largest payers have already put commodity pull down to their respective formularies and we hope we have also seen strong acceptance by Medicare Medicaid.

Sbastien Martel: Overall time from prescription to infuse room is now down to around 65 days with payer approvals happening in around 30 days.

Bradley Campbell: Yeah, thanks, Tazeen. Thanks for the question. So, as you mentioned, a couple of things. First of all, I think, you know, as you saw from the numbers today, we continue to see great progress in both markets. I think you have, you know, two different dynamics going on.

Bradley Campbell: Yeah, thanks, Tazeen. Thanks for the question. So, as you mentioned, a couple of things. So, first of all, I think, you know, as you saw from the numbers today, continuing to see great progress in both markets. I think you have, you know, two different dynamics going on.

Speaker Change: In the last few commercial patients I'm actually come in soon it's going to be quicker.

Sbastien Martel: We believe this will continue to improve.

Sbastien Martel: Today, we're launching Germany, the U K U S, Australia, Spain, but we remain in active pricing and reimbursement discussions with additional major European markets as we focus on securing broad patient access throughout the European market.

Bradley Campbell: In the U.S., it's the largest single market. And so, you know, the rate of new patients will continue to be strong as we go throughout the year. However, in Europe, we're adding new countries as we go. You know, we launched in Spain. We just got approval in Switzerland, and we're working on other reimbursements. So, I think over the course of the year, the balance of new patients will probably start to favor the United States, but that's just a matter of size.

Bradley Campbell: You know, we launched in Spain, we just got approval in Switzerland, and we're working on other reimbursements. So, I think over the course of the year, the balance of new patients will probably start to favor the United States, but that's just a matter of size. But overall, I think both markets are progressing extremely well. In terms of the label, as you said, and as I kind of provided on the call, we and Sebastian provided this detail as well.

Sbastien Martel: Overall, we've had a very strong first half and we're very pleased with the building momentum in patient demand.

Bradley Campbell: We're working on other reimbursements. So, I think over the course of the year, the balance of new patients will probably start to favor the United States, but that's just a matter of size. But overall, I think both markets are progressing extremely well. In terms of the label, as you said, and as I kind of provided on the call, we and Sebastian provided this detail as well. In Europe, we're seeing switching patients from both myosine and nexviazine, as well as new patients which are on the label there. In the U.S., we're seeing, you know, predominantly switches from nexviazine and some switches from myosine, as that's a much smaller portion of the population.

Sbastien Martel: Throughout the second half of 2024, I'll focus will be on maximizing the number of patients on therapy by year end.

Sbastien Martel: So in summary, we're very pleased with the launches of commodities going to pull down across the first wave of countries. The strength of our clinical data the depth of our experience and talent. We have at amicus gives us great confidence in our ability to make a real difference for people living with Pompe disease.

Bradley Campbell: But overall, I think both markets are progressing extremely well. In terms of the label, as you said, and as I kind of provided on the call, we and Sebastian provided this detail as well. In Europe, we're seeing switching patients from both myosin and nexviadim as well as new patients, which are on the label there. In the U.S., we're seeing, you know, predominantly switches from nexviadim and some switches from myosin as that's a much smaller portion of the population. So, the key performance indicators we're watching are going really well, and we're excited to continue to see the momentum. Thank you. Your next question comes from Eliana Merle with UB.

Bradley Campbell: In Europe, we're seeing switching patients from both myosine and nexiviridine, as well as new patients which are on the label there. In the U.S., we're seeing, you know, predominantly switches from nexiviridine and some switches from myosine, as that's a much smaller portion of the population. So, the key performance indicators we're watching are going really well, and we're excited to continue to see the momentum. Thank you. Your next question comes from Eliana Merle with UB.

Sbastien Martel: Believe me because he is in a great position with our second commercial launch and with that.

Sbastien Martel: And the call over to Jessica <unk>, our Chief development officer to discuss the ongoing clinical studies and regulatory timelines.

Bradley Campbell: So, the key performance indicators we're watching are going really well, and we're excited to continue to see the momentum. Thank you. Your next question comes from Eliana Merle with UB.

Sbastien Martel: Jeff.

Sbastien Martel: Thank you Sebastian and good morning, everyone.

Sbastien Martel: On slide 12, we outline how we continue to build the body of evidence for probability in our Florida to our ongoing clinical studies as well as through our amicus Pompeii registry as.

Operator: Your next question comes from Eliana Merle with UBS. Your line is now open. Hey guys, thanks so much for taking the question.

Sbastien Martel: As we also continue to execute on expanding commercial access to reimbursement dossiers and regulatory submissions.

Sbastien Martel: We entered the second phase of launch.

Sbastien Martel: Addition to the various reimbursement dossiers that we have or are in the process of submitting we also have multiple ongoing or planned regulatory submissions for marketing approval in new geographies throughout the year.

Sbastien Martel: Just mentioned the approval in Switzerland. Additionally, we have our regulatory dossier is being reviewed in Australia, and Canada and we're working on we're working towards a submission here in Japan before the end of the year.

Bradley Campbell: Yeah, thanks. I think essentially, we're seeing what we had expected to see, which is that the majority of patients switching from mexiviazine have been on treatment for that kind of year to two years. And that's a dynamic that we've described before. The good news is, of course, there is a huge bolus of patients that are kind of rolling through that year to two years on therapy over the course of this year and into next year.

Bradley Campbell: Yeah, thanks. I think Essentially, we're seeing what we had expected to see, which is the majority of patients switching from mexifiazine had been on treatment in that kind of year to two years. And that's a dynamic that we've described before. The good news is, of course, there's a huge bolus of patients that are kind of rolling through that year to two years on therapy over the course of this year and into next year.

Sbastien Martel: For the younger pumping 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 disease.

Sbastien Martel: This is an important opportunity to support label expansion into these patient segments in the years ahead, and very importantly to address the significant unmet needs for these children.

Bradley Campbell: And so I think we've got a large bolus of patients that we can target. We do see some patients who have switched earlier in their journey. And I would say that probably comes more from word of mouth or from knowing other patients in the community who had a positive experience with Ponvillanabfolda. And I think that's another dynamic, too, that we'll want to continue to see, which is, you know, as we establish this as an important new therapy, as more physicians and more patients get experience, and hopefully those experiences will be positive, I think that creates, you know, some demand Thank you. Your next question?

Bradley Campbell: And so I think we've got a large bolus of patients that we can target. We do see some patients who have switched earlier in their journey. And I would say that probably comes more from word of mouth or from knowing other patients in the community who've had a positive experience with Ponvillanapifolda. And I think that's another dynamic, too, that we'll want to continue to see, which is, you know, as we establish this as an important new therapy, as more physicians and more patients get experience, and hopefully those experiences will be positive, I think that creates, you know, some Thank you. Your next question?

Bradley Campbell: And so I think we've got a large bolus of patients that we can target. We do see some patients who have switched earlier in their journey. And I would say that probably comes more from word of mouth or from knowing other patients in the community who had a positive experience with Ponvillidab Folda. And I think that's another dynamic, too, that we'll want to continue to see, which is, you know, as we establish this as an important new therapy, as more physicians and more patients get experience, and hopefully those experiences will be positive, I think that creates, you know, some demand Thank you. Your next question?

Sbastien Martel: Through ongoing clinical studies and the amicus pumped a registry.

Sbastien Martel: To generate evidence on the differentiated mechanism of action.

Speaker Change: On the long term impact of possibility in our Florida across endpoints and patient populations are.

Sbastien Martel: Our medical conference presence in publications continue to be an important part of our education efforts.

Sbastien Martel: Finally, as highlighted in the pipeline slide in the appendix.

Sbastien Martel: Our earlier stage pipeline, we continue to focus on novel approaches to next generation therapies in fabry and Pompe diseases.

Simon Harford: 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.

Speaker Change: Thank you, Jeff our financial overview begins on slide 14, with our income statement for the second quarter ending June 30 of 2024.

Operator: Your next question comes in the line of Anupam Rama with JPM. Your line is now open.

Bradley Campbell: Your next question comes to the line of Anupam Rama with JPM. Your line is now open.

Operator: Your next question comes in the line of Anupam Rama with JPM. Your line is now open.

Speaker Change: For Q2, we achieved total revenue of $127 million, which is a 34% increase over the same period in 2023 at constant exchange rates revenue also grew 36%.

Sbastien Martel: The global geographic breakdown of total revenue during the quarter consisted of $77 million or 60% of revenue generated outside the United States and the remaining $50 million of 40% coming from within the U S.

Operator: Maybe I'll start at a high level and then ask Sbastien to add a little color there. So, you know, Anupam, honestly, we're seeing great growth in all of our key markets, which is, you know, what you want to be seeing. But Sbastien, maybe provide a little bit of color on some of the unique elements of the U.S. and anything else that you think is relevant there. Yeah, thanks, Brad. So, Anupam, we're seeing

Bradley Campbell: Maybe I'll start at a high level and then ask Sbastien to add a little color there. So Anupam, honestly, we're seeing great growth in all of our key markets, which is what you want to be seeing. But Sbastien, maybe provide a little bit of color on some of the unique elements of the U.S. and anything else that you think is relevant there.

Speaker Change: Cost of goods sold as a percentage of net sales was 9% in Q2 2024 as compared to 10% for the prior year period staying relatively flat.

Bradley Campbell: Yeah, so thanks, Brad. So, Anupam, we're seeing very strong demand for naive patients, newly diagnosed patients in the U.S., and we have seen that, you know, for a number of quarters now. That's actually why we've now raised guidance twice this year on Galafold. So, you know, a growing proportion of newly diagnosed patients really are put on Galafold as the very first line of treatment if they have amenable mutations. The number of PRFs we've recorded in the first half of the year, as I said earlier, is close to the highest we've seen in the last five years.

Sebastian Martel: Yeah, thanks, Brad. So, Anupam, we're seeing very strong demand for naive patients, newly diagnosed patients in the U.S., and we have seen that, you know, for a number of quarters now. That's actually why we've now raised guidance twice this year on Galafold. So, you know, a growing proportion of newly diagnosed patients really are put on Galafold as the very first line of treatment if they have amenable mutations. The number of PRFs we've recorded in the first half of the year, as I said earlier, is close to the highest we've seen in the last five years.

Sbastien Martel: Total GAAP operating expenses decreased to 100 million for the second quarter 2024, as compared to 104 million in the same period last year, a decrease of 4%.

Sbastien Martel: On a non-GAAP basis total operating expenses decreased to $82 million for the second quarter of this year as compared to $84 million in the same period last year, a decrease of 2% we.

Speaker Change: We define non-GAAP operating expenses research and development and SG&A expenses, excluding stock based compensation.

Bradley Campbell: So, strong, very strong performance in the U.S. We continue to see the shift that we've seen, you know, for ever since launch, actually, that a growing proportion of females with Fabry disease are also being treated, and the tendency of patients who have been diagnosed moving to treatment earlier than they may have in the past.

Sebastian Martel: So, strong, very strong performance in the U.S. We continue to see the shift that we've seen, you know, for ever since launch, actually, that a growing proportion of females with Fabry disease are also being treated, and the tendency of patients who have been diagnosed moving to treatment earlier than they may have in the past.

Speaker Change: Loss on impairment of assets changes in fair value of contingent content subsidiary, <unk> restructuring charges and depreciation and amortization.

Speaker Change: On a GAAP basis net loss for the second quarter of 2020 for reduced to $16 million or five cents per share compared to a net loss of $43 million or <unk> 15 per share for the second quarter of 2023.

Sbastien Martel: In Europe, we're very pleased to see a bit of a similar pattern with, you know, the majority of the growth coming from naive patients. And we're seeing that in our largest market, the U.K. We're seeing great performance in Japan as well. We actually think that we have, you know, more growth upside in Japan simply because the Japanese business is our fourth largest. If you look at the whole Fabry market, Japan is actually the second largest market.

Sebastian Martel: In Europe, we're very pleased to see a bit of a similar pattern with, you know, the majority of the growth coming from naive patients. And we're seeing that in our largest market, the U.K. We're seeing great performance in Japan as well. We actually think that we have, you know, more growth upside in Japan, simply because the Japanese business is our fourth largest. If you look at the whole Fabry market, Japan is actually the second largest market. So, there's no reason why Japan couldn't, at some point in time, become our second largest market as well. So, these are sort of the key highlights on regional performance. Thanks.

Sbastien Martel: In Europe, we're very pleased to see a bit of a similar pattern with, you know, the majority of the growth coming from naive patients. And we're seeing that in our largest markets, the U.K. We're seeing great performance in Japan as well. We actually think that we have, you know, more growth upside in Japan, simply because the Japanese business is our fourth largest. If you look at the whole Fabry market, Japan is actually the second largest market.

Sbastien Martel: In Q2, non-GAAP net income was $18 million or six cents per share compared to a loss last year in the same period of $20 million or 7% plus.

Speaker Change: Cash cash equivalents and marketable securities with $260 million at June 30th two.

Sbastien Martel: <unk> 24, compared to 286 million at December 31, 2023.

Sbastien Martel: So, there's no reason why Japan couldn't at some point in time become our second largest market as well. So, these are sort of the key highlights on regional performance. Thanks so much for taking our questions. Thank you. Your next question comes from Joseph Schwartz with Lyrinc. Your line is now open.

Sbastien Martel: So, there's no reason why Japan couldn't at some point in time become our second largest market as well. So, these are sort of the key highlights on regional performance. Thank you so much for taking our questions. Thank you. Your next question comes from Joseph Schwartz with Lyrinc. Your line is now open.

Operator: Thanks so much for taking our question. Thank you. Your next question comes from Joseph Schwartz with Lyrinc.

Sbastien Martel: Turning now to slide 15, we are raising our full year 2024, total revenue guidance range to 26% to 31% up from 25% to 30%.

Operator: Your line is now open. Hi, all. This is Will on behalf of Joe. Thanks.

Operator: Your next question comes from Joseph Schwartz with Lyrinc. Your line is now open. Hi, all. This is Will Longford Joe. Thanks for taking our question this morning and congratulations on the progress.

Operator: Hi all, this is Will on behalf of Joe. Thanks for taking our question this morning and congratulations on the progress.

Operator: Hi, all. This is Will on for Joe. Thanks for taking our question this morning and congrats on the progress.

Speaker Change: This is driven by the increase of our full year 2024 gala fold revenue growth guidance, which starts at the 11% to 16% was most recently, 13% to 17% and now is 14% to 18% at constant exchange rates.

Sbastien Martel: In addition, we are reiterating the guidance of $62 million to $67 million.

Sbastien Martel: <unk> sales for the full year 2024.

Sbastien Martel: Our full year 2024, non-GAAP operating expense guidance has been narrowed from $345 million to $365 million to $345 million to $360 million or a 5% reduction at the top end.

Bradley Campbell: That being said, from a revenue perspective, especially because a lot of those reimbursements and launches will be in the fourth quarter, the rate of new revenue won't change very much. But obviously, next year, it'll be really important. So that's why we focus on sort of maximizing the number of patients on therapy by the end of the year, because that maximizes the run rate going into next year.

Bradley Campbell: That being said, from a revenue perspective, especially because a lot of those reimbursements and launches will be in the fourth quarter, the rate of new revenue won't change very much, but obviously, next year, it'll be really important. So that's why we focus on sort of maximizing the number of patients on therapy by the end of the year, because that maximizes the run rate going into next year.

Bradley Campbell: That being said, from a revenue perspective, especially because a lot of those, you know, reimbursement and launches will be in the fourth quarter. The rate of new revenue, you know, won't change very much, but obviously next year, it'll be really important. So that's why we focus on sort of maximizing the number of patients on therapy by the end of the year, because that maximizes the run rate going into next year.

Sbastien Martel: With our commitment to full year non-GAAP profitability during the first full year of launch for possibility of not fold.

Sbastien Martel: We are keeping operating expense growth in the low single digits year over year at the midpoint of guidance. As a reminder, we continue to have R&D commitments, including registry studies in both Fabry and Pompe pay the ongoing <unk> phase III study in countries with Trump yet.

Operator: Your next question comes from the line of Ritu Baral with T.D. Cowan. Your line is now open.

Sbastien Martel: As well as next generation manufacturing for comparability.

Operator: Good morning guys. Thanks for taking the time to answer the question. Congratulations on the metrics of this quarter. From our tracking survey, our PUMUP tracking survey that we ran recently, we got some input and some good flavor on the competitive dynamics, which from Genzyme, which seemed to be quite considerable. And we're hearing that at least the Nexviazyme team over there had some very interesting messaging about Nexviazyme superiority over Myozyme, and Lumizyme that seemed to be resonating with some of the doctors that we surveyed.

Operator: Good morning guys, thanks for taking the question. Congratulations on the metrics for this quarter. From our tracking survey, our PUMUP tracking survey that we ran recently, we got some input and some good flavor on the competitive dynamics, which from Genzyme, which seemed to be quite considerable. And we're hearing that at least the Nexviazyme team over there had some very interesting messaging about Nexviazyme superiority over Myozyme, and Lumizyme, and that seemed to be resonating with some of the doctors that we surveyed

Operator: Good morning, guys. Thanks for taking the question. Congratulations on the metrics of this quarter. From our tracking survey, our PUMUP tracking survey that we ran recently, we got some input and some good flavor on the competitive dynamics, which from Genzyme, which seemed to be quite considerable. And we're hearing that at least the Nexviazyme team over there had some very interesting messaging about Nexviazyme superiority over Myozyme, Lumizyme, and that seemed to be resonating with some of the doctors that we surveyed.

Speaker Change: In the second quarter of 2024, non-GAAP profitability was 18 in the half a million and for the first half of 2020 for $13 9 million.

Sbastien Martel: With our total revenue guidance of 26% to 31% growth we remain comfortably on track for 2024 to be our first full year of non-GAAP profitability as profitability continues to accelerate in the second half of the.

Sbastien Martel: And with that let me turn the call back over to Bradley for closing remarks Bradley.

Operator: Can you talk to, I mean, if that's the PUMUP counter detail, can you talk to your counter-counter detail about what's going on messaging-wise, about what defines a decliner, and how the 2 drugs sort of stack up against each other in the field? And then I've got a follow-up on insurance. Thanks.

Operator: Can you talk to, I mean, if that's the PUMUP counter detail, can you talk to your counter-counter detail about what's going on messaging-wise, about what defines a decliner, and how the two drugs sort of stack up against each other in the field? And then I've got a follow-up on insurance. Thanks.

Speaker Change: Thank you Simon Jeff and Sebastian as you can all see we've been highly focused this year on our commercial execution continuing to strengthen our financial profile and ultimately delivering value for our shareholders. Our first half performance has laid the groundwork for us to continue to achieve our goals and fulfill our mission for delivering life changing therapies.

Sbastien Martel: Yeah.

Speaker Change: And with that operator, we can now open the call to questions.

Speaker Change: Yes, 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 additional questions. Please enter back into the queue. Thank you.

Bradley Campbell: Thanks Ritu. Maybe I'll start it, and I'll have Jeff add some color.

Bradley Campbell: Thanks Ritu. Maybe I'll start and I'll have Jeff add some color.

Bradley Campbell: I won't speak to how they're detailing, but what I would say is that... You know, we're the only product that has shown in a controlled phase of phase three an improvement in both the six-minute walk and four-spinal capacity versus an active comparator. And for me, the most important thing here is the efficacy of the product, and I think that's a very important part of our label. And in fact, you may remember our promotional materials, which are pretty incredible.

Bradley Campbell: I won't speak to how they're detailing, but what I would say is that... You know, we're the only product that has shown in a controlled phase of phase three an improvement in both the six-minute walk and four-spinal capacity versus an active comparator. And for me, the most important thing here is the efficacy of the product. And I think that's a very important part of our label. And, in fact, you may remember our promotional materials, which are pretty incredible.

Bradley Campbell: I won't speak to how they're detailing, but what I would say is that... You know, we're the only product that has shown in a controlled phase of a phase three an improvement in both six-minute walking and forced spinal capacity versus an active comparator. And for me, the most important thing here is the efficacy of the product. And I think that's a very important part of our label. And in fact, you may remember our promotional materials, which is pretty incredible.

Speaker Change: Your first question comes from.

Speaker Change: Just one moment please.

<unk> Ahmad: This question comes from the line of <unk> Ahmad with Bofa Securities. Your line is now open.

Sbastien Martel: Hi, guys.

Speaker Change: Thanks for taking my questions.

Sbastien Martel: Morning, Brad I, just wanted to get a sense from you about how we should be thinking about the cadence of the Europe launch versus how the U S launch is going.

Bradley Campbell: We can, you know, effectively say improvement is possible with this therapy. And I think that's a responding message that we're very confident in, and I think that describes why we've had such a great launch so far. But Jeff, maybe talk a little bit more about the kinds of things that physicians are focusing on and maybe the importance of some of the other data points that they're following. Yeah, thanks, Brian. Thanks for two for the questions.

Sbastien Martel: The label language is slightly different.

Speaker Change: As we gain traction in the U S. How should we be thinking about how that could or could not be mirroring what you're seeing in Europe.

Speaker Change: Yes, Thanks, Izzy and thanks for the question. So as you mentioned a couple of things. So first of all I think as you saw from the numbers today continuing to see great progress in both markets. I think you have two different dynamics going on in the U S. It's the largest single market and so the rate of new patients will continue to be strong as we go throughout.

Jeff Castelli: Brad, I think you hit on the main point, which is from an amicus perspective: we obviously are still educating people on the data from our phase three, which was largely in ERT-experienced patients, randomized blinded, where we showed, you know, in that large group of switch patients, improvements on the six-minute walk FEC and the key endpoints. And as we presented across all the endpoints in that trial, we see patients when switching have improvements across numerous aspects of muscle strength, quality of life, breathing, et cetera. So, you know, we're still really educating everyone about that data. Obviously, that data is, you know, one of the highest levels of evidence that you can have.

Jeff Castelli: Brad, I think you hit on the main point, which is from an amicus perspective: we obviously are still educating people on the data from our phase three, which was largely in ERT-experienced patients, randomized, blinded, where we showed, you know, in that large group of switch patients, improvements on the six-minute walk FEC and the key endpoints. And as we presented across all the endpoints in that trial, we see patients when switching have improvements across numerous aspects of muscle strength, quality of life, breathing, et cetera. So, you know, we're still really educating everyone about that data. Obviously, that data is, you know, one of the highest levels of evidence that you can have.

Jeff Castelli: Brad, I think you hit on the main point, which is from an amicus perspective, we obviously are still educating people on the data from our phase three, which was largely in ERQ experienced patients, randomized blinded, where we showed, you know, in that large group of switch patients, improvements on sick moon and walk at BC, and there are key endpoints, and it'd be presented across all the endpoints in that trial. We see patients when switching have improvements across numerous aspects of muscle strength, body, life, breathing, et cetera. So, you know, we're still really educating everyone about that data. Obviously, that data is, you know, one of the highest levels of evidence that you can have.

Sbastien Martel: The year however.

Jeff Castelli: However in Europe, we are adding new countries as we go we launched in Spain, We just got approval in Switzerland, and other reimbursement. So I think over the course of the year the balance of the new patients will probably start to favor the United States, but that's just a matter of size, but overall I think both markets are progressing extremely well in terms of the label as you said and as I.

Speaker Change: I kind of provided on the call.

Sebastian: Sebastian provided this detail as well in Europe, we're seeing switching patients from both <unk> and next five time as well as new patients which are on the label there in the U S. We're seeing.

Jeff Castelli: And then, you know, there's still continuing education around the mechanism of action and the unique differences in what we think we've built here with Pondoli-Napfota. You know, moving forward, as I talked about briefly in the transcript, we are really invested in real-world evidence and the registry, looking to see how people are doing when they're on POMOP across all sorts of parameters, whether that's naive patients outside the U.S., people switching from Nix-Biozyme, people switching from Lumozyme, and we'll continue to report that data, importantly, from our registry and other And we're really excited about everything we're hearing anecdotally about the patient-physician response to Pondoli-Napfota. But, yes, certainly, there are different messages out there, but we're really pleased with where we are today and where we're going.

Jeff Castelli: And then, you know, there's still continuing education around the mechanism of action and the unique differences in what we think we've built here with Pondolino-Folda. You know, moving forward, as I talked about briefly in the transcript, we are really invested in real-world evidence and the registry, looking to see how people are doing when they're on POMOP across all sorts of parameters, whether that's naive patients outside the U.S., people switching from Nix-Biozyme, people switching from Lumozyme, and we'll continue to report out that data, importantly from our registry and other And we're really excited about everything we're hearing anecdotally about the patient-physician response to Pondolino-Folda. But, yes, certainly, there are different messages out there, but we're really pleased with where we are today and where we're going.

Jeff Castelli: Predominantly switches from <unk> and some switches from my time.

Jeff Castelli: A much smaller portion of the population. So the key performance indicators, we're watching are going really well.

Jeff Castelli: And when we're excited to continue to see the momentum.

Jeff Castelli: Thank you.

Speaker Change: Your next question comes from Eliana Merle with UBS. Your line is now open.

Speaker Change: Hey, guys. Thanks, so much for taking my question in terms of some of the dynamics.

Speaker Change: Of the patients are switching.

Speaker Change: I guess, what do you think.

Bradley Campbell: Great, and some of the survey focused on the reimbursement landscape, and there seems to be sort of continuing griping from some of the respondents about insurance coverage. Not that it's terrible, but we've heard anecdotes of not getting the folder component covered. Can you speak to how that has been going now that you got a full quarter's worth of your new J code? Has this all mostly been resolved? Yeah, thanks, Ritu. You know, it's funny, I...

Bradley Campbell: Great, and some of the survey focused on the reimbursement landscape, and there seems to be some sort of continuing griping from some of the respondents about insurance coverage. Not that it's terrible, but we've heard anecdotes about not getting the full cost of the component covered. Can you speak to how that has been going now that you have a full quarter source of your new J-code? Has this all mostly been resolved? Yeah, thanks, Ritu. You know, it's funny, I...

Bradley Campbell: Great. And some of the survey focused on the reimbursement landscape. And there seems to be some sort of continuing griping from some of the respondents about insurance coverage. Not that it's terrible, but we've heard anecdotes about not getting the folder component covered. Can you speak to how that has been going now that you have a full quarter's worth of your new J code? Has this all mostly been resolved? Yeah, thanks, Ritu. You know, it's funny, I...

Jeff Castelli: The types of patients.

Jeff Castelli: Okay.

Speaker Change: And in particular I guess, what are you seeing in terms of like the patients all brands.

Speaker Change: Prior to switching.

Jeff Castelli: Thanks.

Speaker Change: Yes. Thanks.

Speaker Change: I think.

Speaker Change: Essentially we're seeing what we had expected to see which is the majority of patients switching from next five had been on treatment and that kind of year to do to two years and Thats a dynamic that we've described before.

Jeff Castelli: The good news is of course there is.

Bradley Campbell: One thing we would remind everybody, I think when the first prescription happens, and in all of these therapies, frankly, the physician or his staff have to go through the pre-approval process. And there are two products here. But I would say that we haven't seen any patients be denied coverage for either the ERT or the small molecule. And we share the trends, you know; we're seeing things proceed exactly as we'd hoped, which is shortening the time from prescription to infusion; we're now down to 65 days, and then shortening the insurance time, which is now down to 30 days.

Bradley Campbell: One thing we would remind everybody, I think when the first prescription happens, and with all of these therapies, frankly, the physician or their staff have to go through the pre-approval process, and there are two products here. But I would say that we haven't seen any patients be denied coverage for either the ERT or the small molecule. And we share the trends. You know, we're seeing things proceed exactly as we'd hoped, which is shortening the time from prescription to infusion. We're now down to 65 days, and we're shortening the insurance time, too, which is now down to 30 days.

Bradley Campbell: One thing we would remind everybody, I think when the first prescription happens, and in all of these therapies, frankly, the physician or his staff has to go through the pre-approval process. And there are two products here. But I would say that we haven't seen any patients be denied coverage for either the ERT or the small molecule. And we share the results. You know, we're seeing things proceed exactly as we'd hoped, which is shortening the time from prescription to infusion.

Bradley Campbell: Yeah, thanks, Ritu. You know, it's funny.

Bradley Campbell: Yeah, thanks Ritu. You know, it's funny.

Speaker Change: Huge bolus of patients that are kind of rolling through that that year to two years on therapy.

Speaker Change: Course of this year and into next year, and so I think we've got a large bolus of patients that we can target.

Jeff Castelli: We do see some patients who have switched earlier in their journey and I would say that probably comes more from word of mouth or from knowing other patients in the community who had a positive experience with with possibility not falter and I think thats. Another dynamic to that we'll want to continue to see which is as we.

Bradley Campbell: We're now down to 65 days, and we're shortening the insurance time, too, which is now down to 30 days. So I'm sure that the very first time that the staff goes through any new, you know, therapy, you know, they have to get used to the paperwork, et cetera.

Speaker Change: Establish this as an important new therapy as more physicians and more patients get experience and hopefully those experiences will be positive I think that creates some demand to switch earlier.

Bradley Campbell: So I'm sure that the very first time that the staff goes through any new, you know, therapy, you know, they have to get used to the paperwork, etc. And I'm sure that's what you're seeing in some of your surveys, but everything we're seeing and the facts are that we're getting patients through the system. And we're getting them through much more quickly than we did at the start of the process, which is what we want to see. And yeah, I'm sure the coding piece helps to some extent there as well. Great, thanks. Thank you. Your next question comes in the line of Jeffrey Hung with Morgan Stanley.

Bradley Campbell: So I'm sure that the very first time the staff goes through any new therapy, you know, they have to get used to the paperwork, et cetera. And I'm sure that's what you're seeing in some of your surveys. But everything we're seeing and the facts are that we're getting patients through the system, and we're getting them through much more quickly than we did at the start of the process, which is what we want to see. And yeah, I'm sure the coding piece helps to some extent there as well. Great, thanks. Thank you. Your next question comes from the line of Jeffrey Hung with Morgan Stanley.

Operator: And I'm sure that's what you're seeing in some of your surveys. But everything we're seeing, and the facts are, are that we're getting patients through the system. And we're getting them through much more quickly than we did at the start of the process, which is what we want to see. And yeah, I'm sure the coding piece helps to some extent there as well. Great, thanks. Thank you. Your next question comes from the line of Jeffrey Hung with Morgan Stanley.

Speaker Change: They realize what palmdale uphold it can do for them.

Jeff Castelli: Okay.

Simon Harford: Thank you.

Speaker Change: Your next question comes from the line of annual plan.

Rama: <unk> Rama with J P. M. Your line is now open.

Speaker Change: Hey, guys. Thanks, so much for taking the question and congrats on all the progress here.

Operator: Your next question comes in the line of Jeffrey Hung with Morgan Stanley.

Speaker Change: Just on the Gallup poll guidance increases increase should we be thinking about any type of regional variation of where growth is coming from or region or regions that are leading to this guidance increase thanks. So much.

Sebastian: Maybe I'll start at a high level and then asked Sebastian to add a little color there. So.

Sebastian: Honestly, we're seeing great growth in all of our key markets, which is what you want to be seen but Sebastian maybe provide a little bit of color on some of the unique elements of the U S and anything else that you think is relevant there.

Bradley Campbell: Yeah, I'll let Jeff maybe talk a little bit more about that. But the reality is the overall market just continues to grow, and that's fueled by, you know, I think improving diagnosis through the kinds of things that Sbastien talked about. But, Jeff, maybe you could remind us the sort of change in market size from where we started, the launch to today, and then, you know, kind of what has changed in the diagnostic landscape that we think. Yeah, thanks, Brian.

Bradley Campbell: Yeah, I'll let Jeff maybe talk a little bit more about that, but the reality is the overall picture.

Bradley Campbell: Yeah, I'll let Jeff maybe talk a little bit more about that, but the reality is the overall picture. The overall market just continues to grow, and that's fueled by, you know, I think improving diagnosis through the kinds of things that Sbastien talked about, but Jeff, maybe you could remind us the sort of change in market size from where we started, the launch to today, and then, you know, kind of what has changed in the diagnostic landscape, Brian.

Bradley Campbell: The overall market just continues to grow, and that's fueled by, you know, I think improving diagnosis through the kinds of things that Sebastian talked about. But Jeff, maybe you could remind us the sort of change in market size from where we started, the launch to today, and then, you know, kind of what has changed in the diagnostic landscape, Brian. Yeah, thanks, Brian.

Simon Harford: Yeah. Thanks, Brett so on if I'm, where we're seeing very strong.

Speaker Change: Demand for naive patients newly diagnosed patients in the in the U S and have seen that for.

Speaker Change: A number of quarters now that's actually why we've now raised.

Speaker Change: Guidance twice this year on kind of fold.

Simon Harford: No.

Simon Harford: We are.

Speaker Change: A growing proportion of newly diagnosed patients.

Jeff Castelli: Thanks for the question. He had breathed about 10,000 patients when we launched Gallifold seven or eight years ago. It's now over 18,000 patients diagnosed. When you look at all the numbers out there from various screening programs, whether newborn screening across different countries or at-risk screening of patients that likely have Fabre, it's clear that there are many, many more people and families living with Fabre that are undiagnosed today than the 18,000 that are diagnosed.

Jeff Castelli: Thanks for the question. As Brad said, it was about 10,000 patients when we launched GALFOLD seven or eight years ago. It's now over 18,000 patients diagnosed. When you look at all the numbers out there from various screening programs, whether it's newborn screening across different countries or at-risk screening of patients that likely have Fabre, it's clear that there are many, many more people and families living with Fabre that are undiagnosed today than the 18,000 that are diagnosed.

Simon Harford: Really.

Jeff Castelli: Thanks for the question. As Brad said, there were about 10,000 patients when we launched Galafold seven or eight years ago. It's now over 18,000 patients diagnosed. When you look at all the numbers out there from various screening programs, whether it's newborn screening across different countries or at-risk screening of patients that likely have Fabre, it's clear that there are many, many more people and families living with Fabre that are undiagnosed today than the 18,000 that are diagnosed.

Speaker Change: I'll put on guard for the very first line of treatment. If you have amenable mutations.

Speaker Change: The number of Prs.

Simon Harford: Recorded in the first half of the year.

Speaker Change: As I said earlier is close to the highest we've seen in the last five years. So strong very strong performance in the U S.

Simon Harford: Continue to see the shift that we've seen.

Jeff Castelli: So the good news is there's a lot of tailwinds on diagnosis. Medical education continues to improve so physicians know when to suspect Fabre. There's a lot more access to screening paradigms, low-cost genetic testing, a lot more education about when you diagnose a Fabre patient, if it's a late-onset male or female, about the burden of treatment and the need to treat earlier. And those are all things that Amicus, along with the other companies in the space and physicians, have really been doing from a medical education perspective.

Jeff Castelli: So the good news is there's a lot of tailwinds for diagnosis. Medical education continues to improve so physicians know when to suspect Fabre. There's a lot more access to screening paradigms, low-cost genetic testing, and a lot more education about when you diagnose a Fabre patient, if it's a late-onset male or female, about the burden of treatment and the need to treat earlier. And those are all things that Amicus, along with the other companies in the space and physicians, have really been doing from a medical education perspective.

Jeff Castelli: So, the good news is there's a lot of tailwinds for diagnosis. Medical education continues to improve so physicians know when to suspect Fabre. There's a lot more access to screening paradigms, low-cost genetic testing, and a lot more education about when you diagnose a Fabre patient, if it's a late-onset male or female, about the burden of treatment and the need to treat earlier. And those are all things that Amicus, along with the other companies in the space, and physicians have really been doing from a medical education perspective.

Speaker Change: For ever since launch actually.

Speaker Change: That a growing proportion of female with fabry disease also being treated and dependency.

Speaker Change: Patients who had been dying.

Simon Harford: Dinos moving to treatments.

Simon Harford: Earlier than they might have.

Speaker Change: In the past in Europe, we're very pleased to see a bit of a similar pattern with that.

Speaker Change: The majority of the growth coming from naive patients and where you're seeing that in our largest markets in the U K.

Jeff Castelli: Lots of exciting developments in the kind of medical record use of AI potential to screen for people that might be suffering from malaria unknowingly. So, you know, as we look moving forward, we think there's going to be lots and lots of people being diagnosed. Many of them will have amenable mutations. That's something we've learned here too, that we seem to see a higher percentage of amenable patients than the new people being diagnosed than historically, largely because late-onset patients are harder to diagnose.

Jeff Castelli: Lots of exciting developments in the kind of medical record use of AI potential to screen for people that might be suffering from God's brain unknowingly. So, you know, as we look moving forward, we think there will be lots and lots of people being diagnosed. Many of them will have amenable mutations. That's something we've learned here, too, that we seem to see a higher percentage of amenable patients than the new people being diagnosed than historically, largely because late onset patients are harder to diagnose.

Jeff Castelli: Lots of exciting developments on the kind of medical record use of AI potential to screen for people that might be suffering with Godbrain unknowingly. So, you know, as we look moving forward, we think there's going to be lots and lots of people being diagnosed. Many of them will have amenable mutations. That's something we've learned here, too, that we seem to see more higher percentage of amenable patients than the new people being diagnosed than historically, largely because late-onset patients are harder to diagnose.

Speaker Change: <unk> seen great performance in Japan, as well, we actually think that we have more growth.

Speaker Change: Upside in Japan, simply because the Japanese <unk>.

Speaker Change: Business to us is our fourth largest.

Simon Harford: If you look at the whole company market, Japan is actually the second largest market. So there's no reason why Japan couldnt at some point in time become our second largest market as well.

Jeff Castelli: Late-onset patients tend to have a medical mutation, and more and more, Galliford is a standard of care for people with this mutation. So lots of positive forces are all coming together, I think, that are driving what we're seeing.

Jeff Castelli: Late-onset patients tend to have a medical mutation. And more and more, Galliford is the standard of care for people with this mutation. So lots of positive forces are all coming together, I think, that are driving what we're seeing. And particularly in the U.S., the last thing I'll add is that there is newborn screening going on in five or six states. And when they find a newborn, often that is the index patient in a family, and then there's the ability to screen the family for the other three to five members that also have Febrile A and are adults and likely suffering. So lots of great tailwinds for the community in terms of finding newborns. And I would add that, you know,

Speaker Change: So these are some of.

Simon Harford: Other key highlights.

Simon Harford: Highlights on regional performance.

Speaker Change: Thanks, so much for taking our question.

Speaker Change: Thank you.

Jeff Castelli: And particularly in the U.S., the last thing I'll add is, you know, there is newborn screening going on in five or six states. And when they find the newborn, often that is the index patient in a family. And then there's the ability to screen the family for the other three to five members that also have febrile AIDS and are adults and likely suffering. So lots of great tailwinds for the community in terms of finding newborns. [inaudible]

Bradley Campbell: And particularly in the U.S., the last thing I'll add is, you know, there is newborn screening going on in five or six states. And when they find a newborn, often that is the index patient in a family. And then there's the ability to screen the families for the other three to five members that also have Febrile AID and are adults and likely suffering. So lots of great tailwinds for the community in terms of finding newborns. And I would add that, you know,

Simon Harford: Your next question comes from Joseph Schwartz with Leerink. Your line is now open.

Simon Harford: Hi, all this is will on for Joe. Thanks for taking our question. This morning, and congrats on the progress this quarter.

Speaker Change: A question on Palm oil based on the metrics for patients treated or scheduled to be treated it seems like you've added roughly the same number of patients in the second quarter as compared to the first quarter is this within expectations and has to be thinking about the inflection point in the back half of the year as other geographies come online in the EU. Thank you.

Jeff Castelli: And I would add that everything that Jeff said is actually highlighted in the growth rate that we see for the quarter, not just for our sales. So we were, again, the fastest growing brand for Fabri with 19% growth. But when we look at the performance of Fabrizyme and Replica, so the two ERTs, we estimate that the overall Fabri market growth rate from a dollar standpoint was north of 12% in the second quarter. So everything that, again, Jeff mentioned contributes to that overall healthy Fabri market growth.

Operator: And I would add that everything that Jeff said is actually highlighted in the growth rate that we see for the quarter, not just for our sales. So we were again the fastest growing brand for Fabri with 19% growth. But when we look at the performance of Fabrizyme and Replica, so the two ERTs, we estimate that the overall Fabri market growth rate from a dollar standpoint was north of 12% in the second quarter. So everything that, again, Jeff mentioned contributes to that overall healthy Fabri market growth.

Sbastien Martel: And I would add that everything that Jeff said is actually highlighted in the growth rate that we see for the quarter, not just for our sales. So, we were again the fastest growing brand for Fabry with 19% growth. But when we look at the performance of Fabrzyme and Replica, so the two ERTs, we estimate that the overall Fabry market growth rate from a dollar standpoint was north of 12% in the second quarter. So, everything that, again, Jeff mentioned contributes to that overall healthy Fabry market growth.

Speaker Change: Yes. Thanks for the question I think one of the things we talked about the passion highlighted at the rate of new patients. This year continues to be much higher than it was last year and thats exactly what we expected.

Speaker Change: In terms of new patient adds I think we will continue to build momentum as we go towards the back half of the year.

Speaker Change: Specially as we open up new countries that being said from a revenue perspective, especially because a lot of those reimbursement and launches will be in the fourth quarter the rate of new revenue.

Speaker Change: It won't change very much but obviously next year it'll be really important. So that's why we focus on sort of maximizing the number of patients on therapy by the end of the year because that maximizes the run rate going into next year.

Operator: Your next question comes to the line about Daikon Ha with Stiefel. Your line is now open.

Operator: Your next question comes to the line of Daigon Ha with Stiefel. Your line is now open.

Operator: Hey, good morning, guys. Thanks for squeezing me in here.

Bradley Campbell: Maybe going back to, I guess, similar to Ritu's question, you know, our channel checks also indicate that there is a lot of enthusiasm to switch over to PUMOP. But I guess what's interesting is, you know, a lot of these physicians say sort of the switching impetus is dependent on progression of disease or next via is I'm not showing any meaningful benefit. Yet those same physicians also talk about how the muscle assessment or other functional assessment frequency hasn't really changed since next via is I'm launch or PUMOP launch.

Operator: Hey, good morning, guys. Thanks for squeezing me in here. Maybe going back to, I guess, similar to Ritu's question, you know, our channel checks also indicate that there is a lot of enthusiasm to switch over to POMOP, but I guess what's interesting is, you know, a lot of these physicians say sort of the switching impetus is dependent on progression of disease or next via is I'm not showing any meaningful benefit, yet those same physicians also talk about how the muscle assessment or other functional assessment frequency hasn't really changed since next via is I'm launch or POMOP launch.

Simon Harford: Thank you.

Speaker Change: Your next question comes from the line of her two barrel with TD Cowen. Your line is now open.

Bradley Campbell: Good morning, guys. Thanks for taking the question.

Bradley Campbell: Congratulations on the metrics in this quarter.

Simon Harford: Yeah.

Speaker Change: From our tracking survey, our Perm up tracking survey that we ran recently.

Bradley Campbell: So kind of curious, is that part of your marketing slash commercialization strategy to maybe induce more of these assessments so that any kind of slowing of efficacy or progression of disease can be picked up quicker and therefore drive more switch? Thanks so much. Thanks, Degan. Yeah.

Bradley Campbell: We don't.

Speaker Change: Some input and some good flavor on the competitive dynamics, which from genzyme, which seem to be quite considerable.

Speaker Change: And we're hearing that at least in <unk> team over there.

Bradley Campbell: I have some very interesting messaging about next year's on superiority.

Bradley Campbell: Thanks so much. Thanks, Degan. Yeah.

Bradley Campbell: Thanks so much. Thanks, Dagon. Yeah.

Bradley Campbell: Thanks, Dagon. Yeah, you know, it's, it's, you're hitting a very important issue, which is that this is really the first time physicians have had a real choice between products. And frankly, now they have two products that they're looking at. And so it is an element of changing expectations for therapy. You know, we focus on that actual improvement as possible, you know, maybe for the first time. So, Yeah, it's very much an educational effort, and it's very much... Again, as patients and physicians get experience with the product, I think it will, you know, encourage them to look more closely at not just the primary measure, the six-minute walk before spinal capacity, but the other measures that may be changing subtly over time. And if we can change the expectation of those physicians and patients of what they're getting from their therapy, I think that very much favors the possibility of unfolding. Thank you, Brad.

Bradley Campbell: Thanks, Dagon. Yeah, you know, it's, it's, you're, you're hitting a very important issue, which is, this is really the first time physicians have had a real choice between products, and frankly, now they have two products that they're, they're looking at. And so, it is an element of changing expectations for therapy, you know, we focus on, but actually improvement is possible, you know, maybe for the first time. So, Yeah, it's very much an educational effort, and it's very much... Again, as patients and physicians get experience with the product, I think it will, you know, encourage them to look more closely at, you know, not just the primary measure, six-minute walk before a spinal capacity, but the other measures that may be changing subtly over time. And if we can change the expectation of those physicians and patients of what they're getting from their therapy, I think that very much favors ponderability and unfoldability. Thank you. Hey, Brad.

Bradley Campbell: Thanks, Dagon. Yeah, you know, it's, it's, you're, you're hitting a very important issue, which is, this is really the first time physicians have had a real choice between products. And frankly, now they have two products that they're, they're looking at. And so it is an element of changing expectations for therapy. You know, we focus on that actually improvement as possible, you know, maybe for the first time. So, Yeah, it's very much an educational effort, and it's very much... Again, as patients and physicians get experience with the product, I think it will, you know, encourage them to look more closely at, you know, not just the primary measure, six-minute walk before a spinal capacity, but the other measures that may be changing subtly over time. And if we can change the expectation of those physicians and patients of what they're getting from their therapy, I think that very much favors Pondability Outfolding. Thank you. Hey, Brad.

Bradley Campbell: And when the time.

Bradley Campbell: That seem to be resonating with some of the doctors that we surveyed.

Speaker Change: When you talk to I mean, if that's the counter detail can you talk to your car.

Speaker Change: Contract counter detail on whats going on messaging wise on what defines a decliner.

Bradley Campbell: And how the.

Speaker Change: Sort of stack up against each other in the field and then I've got a follow up on insurance.

Speaker Change: Thanks, Ritu, maybe I'll start and I'll.

Jeff Castelli: Jeff add some color.

Speaker Change: I won't speak to how their detailing.

Speaker Change: But what I would say is that.

Speaker Change: We're the only product that is shown in a controlled phase of the phase III and improvement in <unk>.

Bradley Campbell: Six minute walk and forced vital capacity versus.

Speaker Change: An active comparator and for me. The most important thing here is the efficacy of the product and I think thats, a very important part of our label and in fact, you may remember our promotional materials, which is pretty incredible.

Bradley Campbell: One other thing I'd add real quickly there, it's definitely something we're hearing from all the positions about now, kind of the need for more standardization about how often they're monitoring their patients, what parameters they're able to measure, and it at each given clinical visit. So that's something that Amicus and the community are all now having to (inaudible) work together on as we have multiple treatments available, and patients really need to be armed better with all the information about making informed treatment decisions.

Jeff Castelli: One other thing I'd add real quickly. It's definitely something we're hearing from all the physicians about now, the need for more standardization about how often they're monitoring their patients, and what parameters they're able to measure in a given clinical visit. So that's something that Amicus and the community are all now having to work together on as we have multiple treatments available, and patients really need to be armed better with all the information about making informed treatment decisions.

Bradley Campbell: One other thing I'd add real quickly. It's definitely something we're hearing from all the physicians about now, the need for more standardization about how often they're monitoring their patients, and what parameters they're able to measure in a given clinical visit. So that's something that Amicus and the community are all now having to work together on as we have multiple treatments available, and patients really need to be armed better with all the information about making informed treatment decisions.

Speaker Change: Can effectively say improvement is possible with this therapy.

Speaker Change: I think thats resonating message that we're very confident in and I think that describes why we've had such a great launch so far but Jeff maybe talk a little bit more about the kinds of things that physicians.

Speaker Change: Our focusing on.

Speaker Change: And maybe the importance of some of the other data points that they are following.

Bradley Campbell: The other side of it from an amicus perspective is we're out there reminding people about the data that we saw, you know, in people that switched from ERT to POMOP that many of them saw improvements across different parameters. So in some way, stable is not necessarily the best outcome, but there is potentially an opportunity to regain some function. So that's something that we're reminding people about from the data.

Bradley Campbell: The other side of it from an amicus perspective is we're out there reminding people about the data that we saw, you know, in people that switched from ERT to POMOP that many of them saw improvements across different parameters. So in some ways, stable is not necessarily the best outcome, but there is potentially an opportunity to regain some function. So that's something that we're reminding people about from the data.

Jeff Castelli: The other side of it from an amicus perspective is we're out there reminding people about the data that we saw, you know, in people that switched from ERT to POMOP that, Those patients, many of them saw improvements across different parameters. So in some way, stable is not necessarily the best outcome, but there is potentially an opportunity to regain some function. So that's something that we're reminding people from the data.

Speaker Change: Yes, thanks, Brian Thanks for two further questions Brad I think you hit on the main point, which is from an amicus perspective, we obviously are still educating people on the data from our phase III, which was largely an <unk> experienced patients randomized blinded, where we showed in that large group of switch patients.

Speaker Change: Improvements in six minute walk at TC and the key endpoints and as we presented across all of the endpoints in that trial, we see patients when switching to have improvements across numerous aspects of muscle strength quality of life breathing et cetera. So we're still really educating everyone about that data obviously that data as you know one of the highest level of evidence that <unk>.

Operator: Your next question comes with the line of Salveen Richter with Goldman Sachs. Your line is now open.

Jeff Castelli: Your next question comes with the line of Salveen Richter with Goldman Sachs. Your line is now open.

Operator: Your next question comes with the line of Salveen Richter with Goldman Sachs. Your line is now open.

Operator: Hi, thank you so much for taking my question. This is Srinath from Salveen.

Operator: Hi, thank you so much for taking my question. This is Srinath Rao from Salveen.

Speaker Change: You can have and then theres still continuing education around mechanism of action and the unique differences in what we think we built here with homebuilding up Florida.

Operator: So, do you see any trends that suggest that physicians are maybe preferring to switch patients from myozyme to another myozyme before evaluating them on that before going to pumbility in both ERT-experienced patients in the U.S. as well as naive patients in Europe? And just a quick second part, you mentioned that you're now down to a 65-day lag between prescriptions and infusions? What is your best estimate around when you get to that 30 to 45-day target that you had mentioned earlier?

Operator: So, do you see any trends that suggest that physicians are maybe preferring to switch patients from myozyme to another myozyme before evaluating them on that before going to pumbility in both ERT-experienced patients in the US as well as naive patients in Europe? And just a quick second part, you mentioned that you're now down to a 65-day lag between prescriptions and infusions. What is your best estimate around when you get to that 30 to 45-day target that you had mentioned earlier?

Bradley Campbell: So do you see any trends that suggest that physicians are maybe preferring to switch patients from myozyme to next myozyme before and evaluating them on that before going to pumbility in both ERT-experienced patients in the US as well as naive patients in Europe? And just a quick second part, you mentioned that you're now down to a 65-day lag between prescriptions to infusions. What is your best estimate around when you get to that 30 to 45-day target that you had mentioned earlier on?

Speaker Change: So moving forward as I talked about briefly in the transcript we are really invested in real world evidence and the registry.

Sebastian: Looking to see how people are doing when they're on tomo across all sorts of parameters, whether thats naive patients outside the U S people switching from <unk> people switching from the rumors and.

Bradley Campbell: And we will continue to report out that data importantly from a registry and other areas and we're really excited from everything we're hearing anecdotally about the patient physician response to probability of Florida, but yes, certainly it's there's different messages out there, but we're really pleased with where we are today and where we're going in the future.

Bradley Campbell: Yeah, thanks for the questions. I think for the first one, no.

Bradley Campbell: Yeah, thanks for the questions. I think for the first one, no. I think what the dynamic is really that, in the U.S., as an example, Nexvizime was approved far earlier than us, so the majority of patients were already switched over, although there are some remaining patients on Nexvizime, and then outside the U.S., it's much more of a mix. Circumstance. Excuse me.

Bradley Campbell: Yeah, thanks for the questions. I think for the first one, no. I think what the dynamic is really that, in the U.S., as an example, Nexvizyme was approved far earlier than us, so the majority of patients were already switched over, although there are some remaining patients from Myzyme, and then outside the U.S., it's much more of a mix. Circumstance. Excuse me.

Bradley Campbell: Yeah, thanks for the questions. I think for the first one, no, I think what the dynamic is really that in the U.S. as an example, Nexvizyme was approved far earlier than us. So the majority of patients were already switched over, although there are some remaining patients from iZyme. And then outside the U.S., it's much more of a mix. Circumstance. Excuse me.

Eliana Merle: Great and.

Eliana Merle: Some of the survey focused on the reimbursement landscape.

Bradley Campbell: So what we've seen is that we're taking switch patients from kind of each segment equally, and I think that reflects that we're making very good headway with the launch and that their physicians aren't preferencing switching to next liazine first. I think it's just a reflection that we weren't available before, excuse me. And then in the second case, as relates to the time to infusion from prescription, our goal is really by the end of this year, we should be down to that 30 to 45 days.

Bradley Campbell: So what we've seen is that we're taking switch patients from kind of each segment equally. And I think that reflects that we're making very good headway with the launch and that their physicians aren't preferencing switching to NexViz at first. I think it's just a reflection that we weren't available before. And then, in the second case, as relates to the time to infusion from prescription, our goal is really, by the end of this year, we should be down to that 30 to 45 days.

Eliana Merle: There seems to be sort of continuing.

Speaker Change: Griping from some of the respondents about insurance coverage.

Speaker Change: Terrible, but we've heard anecdotes.

Eliana Merle: Getting.

Speaker Change: So the component.

Bradley Campbell: I've covered.

Eliana Merle: Can you speak to that.

Speaker Change: That has been going now that you've got a full quarter's worth of new J code.

Bradley Campbell: Mostly been resolved.

Speaker Change: Yes. Thanks for to you know, it's funny I one thing we would remind everybody I think when the first prescription happens.

Bradley Campbell: And in all of these therapies frankly.

Speaker Change: The physician or their staff has to go through the pre approval process and there are two products here, but I would say that we haven't seen any patients be denied coverage for either the <unk> or those small molecule.

Bradley Campbell: And we shared the trends we're seeing things proceed exactly as we'd hoped which is shortening the time from prescription to infusion. We're now down to 65 days and then shortening the insurance time, too which is now down to 30 days. So I'm sure that the very first time.

Operator: Your next question comes to the line of Kristen Kluska with Cantor Fitzgerald. Your line is now open. Hi, this is Rick Miller on.

Speaker Change: Staff goes through any new therapy.

Speaker Change: They have to get used to the paperwork etcetera and I am sure Thats, what youre seeing in some of your surveys, but everything we're seeing and the facts are that we're getting patients through the system.

Speaker Change: And we're getting through much more quickly than we did started the process, which is where we want to want to see and yes Im sure. The coating piece helps to some extent there as well.

Bradley Campbell: Yeah, great question. I think Sebastian highlighted one of the most important metrics, which is the number of prescribers globally has increased by 50%. And that's, that's really important. We want to see continued breadth, i.e. new prescribers. We also want to see continued depth, which we're seeing as well, which is prescribers, you know, adding their second or third or fourth prescription. So both of those dynamics have been really strong.

Bradley Campbell: Yeah.

Speaker Change: Great. Thanks, Thank you.

Jeffrey Hung: Your next question comes from the line of Jeffrey hung with Morgan Stanley.

Michael: Hi, This is Michael.

Anupam Rama: Hi, This is Michael Rehaut answer Jeff <unk>. Thank you for taking my questions and congrats on the progress on all fronts.

Bradley Campbell: Again, I think it depends on the market. In a market like the UK, which is very concentrated, all of the key centers are prescribing. In markets like Germany and the US, you do have a top prescriber base, and we're seeing great penetration into those. But you also have sort of, you know, just because of the size of the geography and the more distribution of physicians, we are now starting to see more and more, I would say, distal physicians that are prescribing as well. And so those are the dynamics we'll continue to speak to. And so far, we're seeing great uptake in both of those segments.

Bradley Campbell: To your question in terms of where are they coming from, again, I think it depends on the market. So a market like the UK, which is very concentrated, all of the key centers are prescribing. In markets like Germany and the US, you do have a top prescriber base, and we're seeing great penetration into those. But you also have sort of, you know, just because of the size of the geography and the more distribution of physicians, we do, now we're starting to see more and more, I would say, distal physicians that are prescribing as well. And so those are dynamics we'll continue to speak to. And so far, we're seeing great uptake in both of those segments.

Speaker Change: <unk> given the growth you had referenced and diagnosed and treated patients with fabry, how much more room do.

Anupam Rama: We think there is left for new patients add just like I'm curious as to why you saw stronger uptake in naive patients versus switch and <unk>. Thank you.

Speaker Change: Yes, I'll, let Jeff maybe talk a little bit more about that but the reality is the overall.

Sbastien Martel: The overall market just continues to grow and that's fueled by I think.

Bradley Campbell: Improving diagnosis through the kinds of things that Sebastian talked about but Jeff maybe.

Bradley Campbell: Maybe.

Sbastien Martel: Remind us the sort of change in market size from where we started the launch to today and then you know kind of what what has changed in the diagnostic landscape that we think is feeling that.

Operator: Thank you. That was your last question. This concludes today's conference. Have a great day.

Sbastien Martel: Yes, thanks, Brian Thanks for the question.

Anupam Rama: As Brad said is about 10000 patients when we launched Kaufhold seven or eight years ago with now over 18000 patients diagnosed and when you look at all the numbers out there from various screening programs, whether newborn screening across different countries are at risk screening of patients likelihood of February.

Speaker Change: It's clear that there are many many more people and families living with fabry that are undiagnosed today than the 18000 that are diagnosed so.

Speaker Change: The good news is there's a lot of tailwind on diagnosis medical education continuous improves the physicians know lenses suspect cranberry theres a lot more access to screening paradigms low cost genetic testing a lot more education about when you're diagnosed with fabry patients late onset male or female about the burden of treatment and the need to.

Sbastien Martel: Treat earlier and those are all things that amicus along with the other companies in our space and positions has really been doing from a medical education perspective.

Speaker Change: Lots of exciting developments on the kind of medical record use of AI potential to screening for people that might be suffering with cadbury not knowingly.

Speaker Change: So as we look moving forward, we think there is going to be lots and lots of people being diagnosed.

Speaker Change: Many of them will have amenable mutations that's something we've learned here too that we seem to see more a higher percentage of amenable patients and the new people being diagnosed than historically.

Jeff Castelli: Because late onset patients are harder to diagnose late onset patients tend to have a medical mutations and more and more golf world as a standard of care for people with this mutation. So lots of positive forces all coming together I think that are driving what we're seeing and particularly in the U S. The last thing I'll add is you know there is newborn screening going on in five or six states and when they find the newborn often.

Bradley Campbell: As the index patient and family and then there is ability to screen the families for the other three to five members that also have Sudbury are adults and likely suffering so lots of great tailwind for the community in terms of finding new patients.

Operator: Yeah.

Bradley Campbell: And I would add that.

Bradley Campbell: Everything that Jeff said is actually.

Speaker Change: Highlighted in.

Jeff Castelli: The growth rate that we see for the quarter not just for ourselves.

Speaker Change: So we were again the fastest growing brand profitably with 19% growth.

Speaker Change: When we look at the performance of five resign.

Speaker Change: So the two year Ts.

Speaker Change: We estimate that the overall fabry market growth rate from a dollar standpoint was north of 12% in the second quarter.

Speaker Change: So everything that again just mentioned.

Bradley Campbell: Contributes to that overall healthy.

Bradley Campbell: February market growth.

Speaker Change: Thanks, So much I appreciate all the context.

Jeff Castelli: Yeah.

Bradley Campbell: Thank you.

guy <unk>: Your next question comes from the line of Guy <unk> with Stifel. Your line is now open.

Speaker Change: Hey, good morning, guys. Thanks for squeezing me in here, maybe going back to.

Richard: Similar to Richard's question.

Speaker Change: Our channel checks also indicate that there is a lot of enthusiasm to switch over to Palmer, but I guess, what's interesting is a lot of these physicians say sort of the switching impetus is dependent on progression of disease or next year is I am not showing any meaningful benefit.

Speaker Change: Yet those same physicians also talk about how the muscle assessment of our other functional assessment frequency Hasnt really changed since next year launch or pump launch. So kind of curious is that part of your marketing slash commercialization strategy to maybe induce more.

Speaker Change: Of these assessments, so that any kind of slowing of efficacy or progression of disease can be picked up quicker and therefore drive more switch. Thanks, so much.

Speaker Change: Thanks, Dave.

Bradley Campbell: Youre hitting a very important issue, which is this is really the first time physicians have had a real choice between products and frankly now they have two products that they are they are looking at and so it is an element of changing.

Bradley Campbell: Expectations for therapy, we focus on that actually improvement is possible maybe for the first time so.

Ritu Baral:

Bradley Campbell: Yes, it's very much an educational effort and it's very much.

Speaker Change: Again, as as patients and physicians get experience with the product I think it will we'll encourage them to look more closely at not just the the primary measure of six minute walk and forced vital capacity, but the other measures that may be changing suddenly over time and if we can change the expectation of those <unk>.

Speaker Change: <unk> and patients of what they're getting from their therapy.

Speaker Change: I think that very much favors possibility in a folder.

Bradley Campbell: Thank you Brian one other thing I'd add real quickly there.

Speaker Change: It's definitely something we're hearing from all of the physicians about now kind of the need for more standardization about how often they're monitoring their patients what parameters are able to measure in a given clinical visit.

Jeff Castelli: That's something that amicus I think the community is all now having to.

Jeff Castelli: Worked together on as we have multiple treatments available and patients really need to be better with all the information about making informed treatment decisions.

Jeff Castelli: The other side of it from an amicus perspective, as we're out there reminding people about the data that we saw in people that switched from <unk> to palm up that.

Jeff Castelli: Those patients many of them saw improvements across different parameters. So in some way.

Speaker Change: Stable is not necessarily.

Jeff Castelli: You know that the best outcome that there is potentially an opportunity to regain some function. So that's something that we're reminding people from the data we've seen.

Speaker Change: Thank you.

<unk> Richter: Your next question comes from the line of <unk> Richter with Goldman Sachs. Your line is now open.

Speaker Change: Hi, Thank you so much for taking my question John for <unk>.

Speaker Change: So do you see any trends that suggest that physicians may be switching to switch patients from <unk> to <unk> before.

Bradley Campbell: And evaluating them on that before going to mobility invalid.

Speaker Change: Patients in the U S as well as naive patients in yogurt.

Speaker Change: And just a quick second part you mentioned that you're now down to a 65 day lag between prescriptions to infusions.

Speaker Change: What is your best estimate around when you get to that 30 to 45 day target.

Bradley Campbell: As mentioned early on.

Speaker Change: Yes, thanks for the questions I think for the first one no I think what the dynamic is really that in the U S. As an example.

Bradley Campbell: <unk> was approved far earlier than us. So the majority of patients were already switched over although there are some remaining patients for <unk>.

Jeff Castelli: And then outside the U S. It's much more of a mixed circumstance.

Jeff Castelli: Hey.

Bradley Campbell: So what we've seen is that we're taking switch patients from kind of each segment equally and I think that reflects that we're making very good headway with.

Bradley Campbell: With the launch and that physicians aren't preferencing switching to <unk> I think it's just a reflection that we weren't we weren't available before.

Speaker Change: And then in the second case as it relates to the time to to infusion from prescription. Our goal is really by the end of this year, we should be down to that 30% to 45 days, that's kind of the journey, we're on with <unk>.

Speaker Change: The end of the first full year of launch we were down to sort of 30 plus days. The one difference here. The reason why we sort of say, 30% to 45 days.

Speaker Change: These patients come in every other week for an infusion. So you probably have a two week kind of an infusion window as well, but yes.

Bradley Campbell: We're looking to do that by the end of this year.

Bradley Campbell: Thank you.

Speaker Change: Our next question.

Bradley Campbell: Comes from the line of Kristen Costco with Cantor Fitzgerald. Your line is now open.

Rick Miller: Hi, This is Rick Miller on for Kristen. Thanks for taking my question just one from us for Pompe <unk> peso Paula can you comment on the prescriber mix Youre seeing does this breakdown across larger academic centers versus community settings and are there any other interesting trends that youre seeing on that front. Thanks.

Speaker Change: Yeah, Great question, I think Sebastian highlighted one of the most important metrics, which is the number of prescribers globally has increased by 50% and that's that's really important and we want to see continued breath I E. New prescribers. We also want to see continued depth, which we're seeing as well, which is prescribers, adding their second or third or fourth prescription so both.

Speaker Change: Are those dynamics had been really strong to your question in terms of where are they coming from again I think it depends on the market. So a market like the U K, which is very concentrated all of the key centers are prescribing in markets like Germany, and the U S. You do have a top prescriber base and we're seeing great penetration into those but you <unk>.

Speaker Change: Also have sort of.

Bradley Campbell: Just because of the size of the geography, and the more distribution of physicians.

Bradley Campbell: We do have now we're starting to see more and more I would say distal physicians that are prescribing as well and so those are dynamics will continue to speak to an and.

Jeff Castelli: And so far we're seeing great uptake in both of those segments.

Speaker Change: Okay. Thank you.

Speaker Change: That was your last question and this concludes today's conference.

Operator: Great day.

Sebastian Martel: And the good news is that there are still many more potential patients eligible for after. As Bradley highlighted, given the continued strong performance of Galafold throughout the second quarter, we're raising again our full year 2024 revenue growth guidance range to now 14 to 18 percent. Turning to slide 7, our results in the second quarter highlight the strengths of our global commercial efforts.

Bradley Campbell: So I'm sure that the very first time that the staff goes through any new therapy, you know, they have to get used to the paperwork, et cetera, and I'm sure that's what you're seeing in some of your surveys, but everything we're seeing and the facts are that we're getting patients through the system, and we're getting them through much more quickly than we did at the start of the process, which is what And yeah, I'm sure the coding piece helps to some extent there.

Andrew Faughnan: As reference funds fly to, we may make forward-looking statements within this meeting about the meaning of the private security litigation reform act of 1995 relating to our business as well as our plans and prospects. Our forward-looking statements should not be regarded as representations by us that any of our plans would be achieved, and all the forward-looking statements made on this call may turn out to be wrong. It can be expected by inaccurate assumptions we might make, or by no one or unknown risks and uncertainties.

Jeff Castelli: And then, you know, there's still continuing education around the mechanism of action and unique differences in what we think we built here with Pumbola Napoda. You know, moving forward, as I talked about briefly in the transcript, we are really invested in real world evidence and the registry, looking to see how people are doing when they're on palm up across all sorts of parameters, whether that's nae patients outside the US, people switching from next by design, and people switching from loom as I'm.

Operator: Thank you very much. Great, thanks. Thank you. Your next question comes from the line of Jeffrey Hung with Morgan Stanley.

Jeff Castelli: And we'll continue to report out that data, importantly, from a registry in other areas. And we're really excited about everything we're hearing anecdotally about the patient and physician response to Pumbola Napoda. But yeah, certainly there are different messages out there, but we're really pleased with where we are today and where we're going.

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