Amicus Therapeutics Q2 2024 Earnings Report

Amicus Therapeutics EPS Results

• Actual EPS: -$0.05
• Consensus EPS: -$0.05
• Beat/Miss: Met Expectations
• One Year Ago EPS: -$0.15

Amicus Therapeutics Revenue Results

• Actual Revenue: $126.67 million
• Expected Revenue: $121.21 million
• Beat/Miss: Beat by +$5.46 million
• YoY Revenue Growth: +34.00%

Amicus Therapeutics Announcement Details

• Quarter: Q2 2024
• Date: 8/8/2024
• Time: Before Market Opens
• Conference Call Date: Thursday, August 8, 2024
• Conference Call Time: 8:30AM ET

Amicus Therapeutics (NASDAQ:FOLD) (NASDAQ:FOLD) is a biopharmaceutical company focused on the discovery, development and commercialization of treatments for rare and orphan diseases. The company specializes in pharmacological chaperones and gene therapy approaches designed to address the underlying causes of lysosomal storage disorders. Its proprietary technology platform integrates structure‐based drug design with precision medicine to identify small molecules that stabilize misfolded proteins and restore cellular function.

The company’s lead marketed product, Galafold (migalastat), is an oral pharmacological chaperone approved in the United States, European Union and other territories for the treatment of Fabry disease in patients with amenable genetic variants. Beyond Fabry disease, Amicus is advancing a pipeline of investigational gene therapies and combination therapies, including programs targeting Pompe disease and Batten disease, with several candidates in late‐stage and mid‐stage clinical development.

Founded in 2005 and headquartered in Cranbury, New Jersey, Amicus Therapeutics completed its initial public offering on the NASDAQ in 2015. The company maintains research and development operations in multiple locations across the United States and Europe, leveraging global manufacturing and regulatory expertise to support its clinical trials and commercial supply chain.

Leadership at Amicus includes Chief Executive Officer Chirfi Guindo, who brings extensive experience in rare disease drug development, and John Crowley, co‐founder and Chairman of the Board. The company collaborates with leading pharmaceutical and academic partners to expand its platform and accelerate the development of novel therapies for underserved patient communities.

Amicus Therapeutics Q2 2024 Earnings Call Transcript

Key Takeaways

• Amicus delivered $127 million total revenue in Q2 2024, up 34% year-over-year, and raised its full-year revenue guidance to 26–31% growth.
• Galafold global sales reached $111 million in Q2 (+19% constant currency), prompting a raise of its full-year growth guidance to 14–18%.
• POMBILITY & Opfolda achieved $16 million in Q2 (44% quarter-over-quarter growth), treated or scheduled 186 patients by end-July, secured Swiss approval, and remains on track for $62–67 million 2024 sales.
• Amicus attained non-GAAP profitability in Q2 and H1 2024 and narrowed full-year non-GAAP operating expense guidance to target its first full-year non-GAAP profitability.
• The company continues to advance its pipeline with ongoing pediatric ZIP and RYZELA studies in Pompe disease, a global Pompe registry, and regulatory submissions in Australia, Canada, and Japan.

Presentation

[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, VP of Investor Relations at Amicus Therapeutics]

Thank you. Good morning, and thank you for joining our conference call to discuss Amicus Therapeutics' second quarter 2024 financial results and corporate highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer, Sébastien Martel, Chief Business Officer, Dr. Jeff Castelli, Chief Development Officer, and Simon Harford, Chief Financial Officer. Joining for Q&A is Dr. Mitchell Goldman, Chief Medical Officer, and Ellen Rosenberg, Chief Legal Officer. As referenced on slide 2, we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as well as our plans and prospects.

[Andrew Faughnan, VP of Investor Relations at Amicus Therapeutics]

Our forward-looking statements should not be regarded as representation by us that any of our plans will be achieved, and they are all forward-looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward-looking statements, which speak only to the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof.

[Andrew Faughnan, VP of Investor Relations at Amicus Therapeutics]

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's my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Bradley?

[Bradley Campbell, President and CEO at Amicus Therapeutics]

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-GAAP 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 points. 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.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

This strong performance has led us to increase our total revenue guidance for the full year to 26%-31%, from 25%-30% previously. At the product level, Galafold 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. 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%.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

All of this is against the backdrop of significant growth in diagnosed and treated patients that we've highlighted previously. 8 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 see Galafold serving as the foundation of our business for the next decade and beyond. Based on the 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%-16%. In May, we raised it to 13%-17%, and now we're projecting 14%-18%. Second, let me highlight the continued strong global commercial launch of Pombiliti and Opfolda, our novel therapy for late-onset Pompe disease.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

Pombiliti and Opfolda 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. 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 Pombiliti and Opfolda 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.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

We are incredibly pleased with the demand globally from patients and physicians from this new therapy and consistently hear inspiring anecdotes from healthcare professionals around the world on how their patients are responding to Pombiliti and Opfolda, which will continue to fuel the momentum throughout the year and beyond. Sébastien will provide more details in a moment, but 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 and markets outside the U.S. as well. 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've anticipated.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

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, the Swissmedic approved Pombiliti and Opfolda for adults living with late-onset Pompe disease in Switzerland. For the full year 2024, we're well on track to deliver our guidance of $62 million-$67 million in global Pombiliti and Opfolda sales, which will be a significant contributor to our growth and set us on a great course to achieve our ambition for Pombiliti, 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, President and CEO at Amicus Therapeutics]

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'll see this number increase as we look to achieve our full-year non-GAAP profitability in 2024. Ahead on slide 4, as we laid out at the beginning of the year, we're focused on achieving our 4 key strategic priorities for 2024, including, first, sustaining double-digit Galafold revenue growth. And again, on the back of strong sales growth so far, we've raised the expected annual growth rate of Galafold to 14%-18%.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

Successfully executing on multiple commercial launches of Pombiliti and Opfolda, 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-GAAP profitability for the full year. With that highlight, let me now hand the call over to Sébastien, who'll go through our commercial performance in more detail. Sébastien?

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

Thank you, Bradley, and good morning to everyone on the call. As always, I'll start by providing you with more details on our Galafold performance for the quarter. On slide 6, for the second quarter 2024, Galafold reported revenue reached $111 million, driven by strong patient demand, particularly from our leading markets. 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 Galafold throughout the second quarter, we're raising again our full year 2024 revenue growth guidance range to now 14%-18%. Turning to slide 7, our results in the second quarter highlight the strengths of our global commercial efforts.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

The demand for Galafold 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 rate. Our leading markets, such as the U.K., the U.S., top new countries and Japan, remain the biggest drivers of patient demand and gives great confidence in the growth this product has over the long term. The U.S., in particular, saw impressive patient demand, the highest number of net new patients seen in over 5 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 in naive populations. We continue to achieve high market shares in countries where we've been approved the longest.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

There's plenty of opportunity to continue to switch patients over to Galafold 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 Galafold predominantly stay on Galafold. We're very pleased to see that prescribers continue to position Galafold as the treatment of choice for amenable Fabry patients. 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 FollowME Registry, in July. These results were published in the Journal of Inherited Metabolic Disease and considered a really important milestone for Amicus as we continue to generate positive data for Galafold.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

Data from the study aligns very well with previous observations from clinical trials and extends the available data supporting the real-world multisystem benefits of Galafold. This publication, along with our ongoing medical education at congresses, is an important part of the story of this oral precision medicine and helps support the long-term growth potential we envision Galafold will have in this growing Fabry market. On slide 8, we know that there's a significant patient demand for Galafold and that the segment of the global Fabry market, made of patients with amenable mutation, has the potential to reach up to $1 billion in annual revenue by the end of the decade. We anticipate sustained growth in 2024 and beyond to be driven by several key drivers.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

First, the Fabry 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, were naive to any treatment before Galafold. 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 capabilities through ongoing medical education and support of novel screening initiatives. Fabry remains, unfortunately, one of the most underdiagnosed rare diseases, so the more patients that can be identified, the more patients may benefit from Galafold.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

We're also seeing many diagnosed and treated patients transition to treatment as the need for earlier treatment, especially in females, becomes better appreciated. The other piece is continuing to drive Galafold market share of treated amenable patients through continued commercial execution. As noted, Galafold currently has more than 60% of the global amenable market, but we're seeing in our most mature markets, it 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. Additionally, we'll continue to make progress on expanding Galafold into new markets and extending the labels.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

There's still some markets in Latin America, the Middle East, and Asia Pacific region, where Galafold is either newly reimbursed or we expect reimbursement. Also important to note here, we have orphan drug exclusivity in the U.S. and Europe, and in addition to our now 61 Orange Book-listed patents, 41 of 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 Galafold globally for a long time to come. We intend to continue to protect and enforce our broad intellectual property rights. Looking ahead, we expect steady double-digit growth for Galafold throughout 2024, and we remain confident that with our strong IP protection, Galafold has a long runway well into the next decade. Turning now to Pompe disease. On slide 10, we outlined our global launch progress with Pombiliti and Opfolda.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

For the second quarter, 2024, Pombiliti and Opfolda reported revenue reached $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. In the U.S., Q2 benefited from the remaining clinical trial patients transitioning to commercial treatment, in addition to patients switching from other therapies. We continue to see a majority of patients switching from Nexviazyme, 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.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

Outside of the U.S., we're seeing patients from all three segments, some switching from Myozyme, some from Nexviazyme at a proportional rate 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. A few updates on the launch in Europe. We're pleased to announce that Swissmedic in Switzerland has approved Pombiliti and Opfolda as a long-term enzyme replacement therapy and enzyme stabilizer for adults with LOPD, another key regulatory authority who recognizes the benefits of this therapy. 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, as it continues to reflect the value health technology assessments are seeing with this therapy.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

The price acknowledges the added benefit of Pombiliti plus Opfolda 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 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 Pombiliti and Opfolda in the U.K., where we've captured greater than a third of market share within the region. With the benefit of EAMS, Pombiliti and Opfolda have actually been in the market for two years in the U.K., and we believe that the current market share is indicative of how this product can perform over a two-year period, which is a great example of its potential demand and uptake.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

Given Pombiliti and Opfolda solid commercial execution thus far, the first half of 2024, we're well on our way to deliver our full-year revenue guidance of $62 million-$67 million for Pombiliti and Opfolda. Moving to slide 11. 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 a remaining 12 are scheduled. We remain very pleased with the ongoing demand for this therapy, as the rate of new commercial patients coming onto Pombiliti and Opfolda continues to increase across all three markets. Q2, in particular, was a very strong quarter, having seen the largest number of new demand patients being prescribed Pombiliti and Opfolda.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

These are individuals not from clinical trials, choosing to come onto commercial therapy. This is important, as 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 in new countries in Europe. Our launch has leveraged our highly experienced cross-functional teams, and we've had great outreach with key opinion leaders. We're seeing an increase in depth and breadth of prescribers across all three markets. In Q2 alone, the global number of prescribing accounts increased by 50%. All core treating centers have been engaged, and they've provided very positive feedback from both HCPs and other stakeholders as 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.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

We have a highly experienced team who are engaging in positive conversations with payers, demonstrate the value of Pombiliti and Opfolda. In the U.S., the largest payers have already put Pombiliti and Opfolda down to their respective formularies, and we've—we have also seen strong acceptance by Medicare and Medicaid. The overall time from prescription to infusion is now down to around 65 days, with payer approvals happening in around 30 days. The last few commercial patients have actually come in significantly quicker. We believe this will continue to improve. 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. Overall, we've had a very strong first half, and we're very pleased with the building momentum on patient demand.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

Throughout the second half of 2024, our focus will be on maximizing the number of patients on therapy by year-end. So in summary, we're very pleased with the launches of Pombiliti and Opfolda 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. 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?

[Jeff Castelli, Chief Development Officer at Amicus Therapeutics]

Thank you, Sébastien, and good morning, everyone. On slide 12, we outline how we continue to build the body of evidence for Pombiliti and Opfolda through our ongoing clinical studies, as well as through our Amicus Pompe Registry, as we also continue to execute on expanding commercial access through reimbursement dossiers and regulatory submissions. 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 the approval in Switzerland. Additionally, we have our regulatory dossiers being reviewed in Australia and Canada, and we're working on working towards a submission here in Japan before the end of the year.

[Jeff Castelli, Chief Development Officer at Amicus Therapeutics]

For the younger Pompe community, we continue to enroll the ongoing open-label ZIP study for children living with late-onset Pompe disease, and the open-label Rossella study for children living with infantile-onset Pompe 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 Pompe Registry, we continue to generate evidence on the differentiated mechanism of action and on the long-term impact of Pombiliti and Opfolda across endpoints and patient populations. Our medical conference presence and publications continue to be an important part of our education efforts. 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 Fabry and Pompe diseases.

[Jeff Castelli, Chief Development Officer at Amicus Therapeutics]

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

[Simon Harford, CFO at Amicus Therapeutics]

Thank you, Jeff. Our financial overview begins on slide 14 with our income statement for the second quarter, ending June 30, 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%. 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, staying relatively flat.

[Simon Harford, CFO at Amicus Therapeutics]

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 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. On a GAAP basis, net loss for the second quarter 2024 reduced to $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.

[Simon Harford, CFO at Amicus Therapeutics]

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 at June 30th, 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%-31%, up from 25%-30%. This is driven by the increase of our full year 2024 Galafold revenue growth guidance, which started the year at 11%-16%, was most recently 13%-17%, and now is 14%-18% at constant exchange rates.

[Simon Harford, CFO at Amicus Therapeutics]

In addition, we are reiterating the guidance of $62 million-$67 million for Pombiliti and Opfolda sales for the full year 2024. Our full year 2024 non-GAAP operating expense guidance has been narrowed from $345 million-$365 million to $345 million-$360 million, or a $0.05 reduction at the top end. With our commitment to full-year non-GAAP profitability during the first full year of launch for Pombiliti and Opfolda, we are keeping operating expense growth in 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, the ongoing Pompe phase III study in countries which aren't yet reimbursed, as well as next-generation manufacturing for Pombiliti.

[Simon Harford, CFO at Amicus Therapeutics]

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%-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 year. And with that, let me turn the call back over to Bradley for our closing remarks. Bradley?

[Bradley Campbell, President and CEO at Amicus Therapeutics]

Thank you, Simon, Jeff, and Sébastien. 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. With that, operator, we can now open the call to questions.

[Operator]

Yes. Ladies and gentlemen, if you have a question, please press star one 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. Your first question comes from. Just one moment, please. This question comes to the line of Tazeen Ahmad with BofA Securities. Your line is now open.

[Tazeen Ahmad, Analyst at BofA Securities]

Hi, guys. Thanks for taking my questions. Good morning.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

Good morning.

[Tazeen Ahmad, Analyst at BofA Securities]

Maybe, 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. The label languages are slightly different. And so-

[Bradley Campbell, President and CEO at Amicus Therapeutics]

Yeah

[Tazeen Ahmad, Analyst at BofA Securities]

... as you 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? Thanks.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

Yeah, thanks, Tazeen. Thanks for the question. So, as you mentioned, a couple 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. In the U.S., it's the largest single market, and so the, 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, we're working on other reimbursements. So I, I think over the course of the year, the balance of the new patients will probably start to favor the United States, but, but that's just a matter of size. But overall, I think both markets are, are progressing extremely well.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

In terms of the label, as you said, and as I kind of provided on the call, we and Sébastien provided this detail as well. In Europe, we're seeing switching patients from both Myozyme and Nexviazyme, as well as new patients, which are on the label there. In the U.S., we're seeing, you know, predominantly switches from Nexviazyme and some switches from Myozyme, as that's the 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.

[Operator]

Thank you. Your next question comes from Eliana Merle with UBS. Your line is now open.

[Eliana Merle, Senior Biotech Equity Research Analyst at UBS]

Hey, guys. Thanks so much for taking the question. In terms of some of the dynamics in the U.S., I guess of the patients that are switching from Nexviazyme, I guess, what are you seeing just in terms of trends of the types of patients that are switching? And in particular, I guess, what are you seeing in terms of, like, the patient average length on Nexviazyme prior to switching to Pombiliti? Thanks.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

Yeah, thanks. I think essentially we're seeing what we had expected to see, which is the majority of patients switching from Nexviazyme have been on treatment in that kind of year or two to two years, and that's, you know, a dynamic that we've described before. The good news is, of course, you know, there's a huge bolus of patients that are kind of rolling through that year to two years on therapy, you know, over the course of this year and into next year. So I think, you know, we've got a large bolus of patients that we can target.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

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 Pombiliti and Opfolda. And I think that's another dynamic too that we'll wanna 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 to switch earlier when they realize, you know, what Pombiliti and Opfolda can do for them.

[Eliana Merle, Senior Biotech Equity Research Analyst at UBS]

Great, thanks.

[Operator]

Thank you. Your next question comes to the line of Anupam Rama with JPM. Your line is now open.

[Anupam Rama, Senior Biotech Analyst at JPM]

Hey, guys. Thanks so much for taking the question, and congrats on all the progress here. Just on the Galafold guidance 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.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

Maybe I'll start at a high level and then ask Sébastien to add a little color there. So, you know, Anupam, honestly, we're, we're seeing great growth in all of our key markets, which is, you know, what you want to be seeing. But Sébastien, 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.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

Yeah. So thanks, Brad. So Anupam, we're seeing very strong demand for naive patients, newly diagnosed patients in the U.S. and have seen that, you know, for a number of quarters now. That's actually why we've now raised, you know, 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 you have amenable mutations. The number of PSFs 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. So strong, very strong performance in the U.S..

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

We continue to see the shift that we've seen, you know, ever since launch, actually, that a growing proportion of female 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. 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 in 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 to us is our fourth largest.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

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 some of the key highlights on regional performance.

[Anupam Rama, Senior Biotech Analyst at JPM]

Thanks so much for taking our question.

[Operator]

Thank you. Your next question comes from Joseph Schwartz with Leerink. Your line is now open.

[Analyst at Leerink]

Hi, all. This is Will on for Joe. Thanks for taking our question this morning, and congrats on the progress this quarter. A question on Pombiliti. 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 third quarter. Is this within expectations, and how should we think about the inflection point in the back half of the year as other geographies come online in the EU? Thank you.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

Yeah, thanks for the question. I think one of the things we talked about, Sébastien highlighted, 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 adds, you know, 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. 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.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

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]

Thank you. Your next question comes from the line of Ritu Baral with TD Cowen. Your line is now open.

[Ritu Baral, Managing Director and Senior Biotechnology Analyst at TD Cowen]

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

[Ritu Baral, Managing Director and Senior Biotechnology Analyst at TD Cowen]

Can you talk to, I mean, if that's the Pompe 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 a follow-up on insurance. Thanks.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

Thanks, Ritu. Maybe I'll start, and I'll have Jeff add some color. You know, I would just—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 3, an improvement in both 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 is pretty incredible. We can, you know, effectively say improvement is possible with this therapy. And I think that's a resonating message that we're very confident in, and I think that describes why we've had such a great launch so far.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

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.

[Jeff Castelli, Chief Development Officer at Amicus Therapeutics]

Yeah, thanks, Brad. Thanks, Ritu, for the 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 3, which was largely in ERT-experienced patients, randomized, blinded, where we showed, you know, in that large group of switch patients, you know, improvements on six-minute walk FVC 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, Chief Development Officer at Amicus Therapeutics]

And then, you know, there's still continuing education around mechanism of action and the unique differences in what we think we've built here with Pombiliti Opfolda. 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 Pombiliti across all sorts of parameters, whether that's naive patients outside the U.S., people switching from Nexviazyme, people switching from Lumizyme, and we'll continue to report out that data, importantly, from our registry and other areas. And we're really excited from everything we're hearing anecdotally about the patient-physician response to Pombiliti Opfolda. But, yeah, 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.

[Ritu Baral, Managing Director and Senior Biotechnology Analyst at TD Cowen]

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 Opfolda 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?

[Bradley Campbell, President and CEO at Amicus Therapeutics]

Yeah. Thanks, Ritu. You know, it's funny, I... One thing we would remind everybody, I think when the first prescription happens, and in all of these therapies, frankly, 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 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, too, which is now down to 30 days.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

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, 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 wanna see. And yeah, I'm sure the coding piece helps to some extent there as well.

[Ritu Baral, Managing Director and Senior Biotechnology Analyst at TD Cowen]

Great. Thanks.

[Operator]

Thank you. Your next question comes to the line of Jeffrey Hung with Morgan Stanley.

[Michael Riad, Equity Research Associate at Morgan Stanley]

Hi, this is Michael Riad on for Jeff Hung. Thank you for taking our questions, and congrats on the progress on all fronts. For Galafold, given the growth you had referenced in diagnosed and treated patients with Fabry, how much more room do you think there is left for new patients at just, like, curious as to why you saw a stronger uptake in naive patients versus switch in 2Q. Thank you.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

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 Sebastian talked about. But, Jeff, maybe 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 is fueling that?

[Jeff Castelli, Chief Development Officer at Amicus Therapeutics]

Yeah. Thanks, Brad. Thanks for the question. Yeah, as Brad said, it was about 10,000 patients when we launched Galafold seven or eight years ago. It's now over 18,000 patients diagnosed. And 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 Fabry. It's clear that there are many, many more people and families living with Fabry that are undiagnosed today than the 18,000 that are diagnosed. So, the good news is there's a lot of tailwinds on diagnosis. Medical education continues to improve, so physicians know when to suspect Fabry.

[Jeff Castelli, Chief Development Officer at Amicus Therapeutics]

There's a lot more access to screening paradigms, low-cost genetic testing, a lot more education about when you diagnose a Fabry patient, if it's a late-onset male or female, about the burden of treatment and the need to treat earlier. 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. Lots of exciting developments on the kind of medical record use of AI, potential to screen for people that might be suffering with Fabry unknowingly. So, you know, as we look moving forward, we think there's gonna be lots and lots of people being diagnosed. Many of them will have amenable mutations.

[Jeff Castelli, Chief Development Officer at Amicus Therapeutics]

That's something we've learned here, too, that we seem to see more higher percentage of amenable patients in the new people being diagnosed than historically, largely because late-onset patients are harder to diagnose. Late-onset patients tend to have amenable mutations, and more and more Galafold is 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 that is the index patient in a family, and then there's ability to screen the families for the other three to five members that also have Fabry that are adults and likely suffering.

[Jeff Castelli, Chief Development Officer at Amicus Therapeutics]

So lots of great tailwinds for the community in terms of finding new patients.

[Sébastien Martel, Chief Business Officer at Amicus Therapeutics]

I would add that, you know, everything that Jeff said is actually, you know, highlighted in the growth rate that we see for the quarter, not just for ourselves. So we were again the fastest growing brand for Fabry with 19% growth. But when we look at the performance of Fabrazyme and Replagal, so the two ERTs, you know, 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, you know, contributes to that overall healthy Fabry market growth. Thanks so much. Appreciate all the context.

[Operator]

Thank you. Your next question comes to the line of Dae Gon Ha with Stifel. Your line is now open.

[Dae Gon Ha, Director of Biotechnology Equity Research at Stifel]

Hey, good morning, guys. Thanks for squeezing me in here. Maybe going back to, I guess, similar to Ritu's question, you know, our, our channel checks also indicate that there is a lot of enthusiasm to switch over to Pombiliti. But I guess what's interesting is, you know, a lot of these physicians say sort of the, the switching impetus is dependent on progression of disease or Nexviazyme 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 Nexviazyme launch or Pombiliti launch. 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.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

Thanks, Dae Gon. Yeah, you know, it's 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 looking at. And so it is an element of changing expectations for therapy. You know, we focus on that 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 experienced 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 and forced vital capacity, but the other measures that may be changing subtly over time.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

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 Pombiliti and Opfolda.

[Operator]

Thank you.

[Jeff Castelli, Chief Development Officer at Amicus Therapeutics]

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, what parameters they're able to measure in a given clinical visit. So that's something that Amicus, I think, you know, the community is 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. 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 Pombiliti, that those patients, many of them, saw improvements across different parameters.

[Jeff Castelli, Chief Development Officer at Amicus Therapeutics]

In some way, you know, stable is not necessarily the best outcome, that there is potentially an opportunity to regain some function. That's something that we're reminding people from the data we've seen.

[Operator]

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

[Analyst at Goldman Sachs]

Hi, thank you so much for taking my question. This is Sunnath on for Salveen. So do you see any trends that suggest that physicians are maybe preferring to switch patients from Myozyme to Nexviazyme before, and evaluating them on that before going to Pombiliti in both ERT experienced patients in the U.S. as well as naive patients in Europe?

[Analyst at Goldman Sachs]

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'll get to that 30- to 45-day target that you had mentioned early on?

[Bradley Campbell, President and CEO at Amicus Therapeutics]

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, Nexviazyme was approved far earlier than us, so the majority of patients were already switched over, although there are some remaining patients from Myozyme. And, and, and then outside the U.S., it's much more of a mixed circumstance. Excuse me. 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 Nexviazyme first. I think it's just a reflection that we weren't available before. Excuse me.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

And then in the second case, as it relates to the time to infusion from prescription, our goal is really by the end of this year, we should be down to that 30-45 days. 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+ days. The one difference here, the reason why we sort of say 30-45 days is, you know, 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, we're looking to do that by the end of this year.

[Operator]

Thank you. Your next question comes from the line of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

[Rick Miller, VP of Biotech Equity Research at Cantor Fitzgerald]

Hi, this is Rick Miller on for Kristen. Thanks for taking our question, just one from us. For Pombiliti plus Opfolda, can you comment on the prescriber mix you're seeing? Does this break down across larger academic centers versus community settings, and are there any other interesting trends that you're seeing on that front? Thanks.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

Yeah, great question. I think Sébastien 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. To your question in terms of where are they coming from, again, I, 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.

[Bradley Campbell, President and CEO at Amicus Therapeutics]

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 that those are 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.

Participants

Executives

• Andrew Faughnan, VP of Investor Relations
• Bradley Campbell, President and CEO
• Jeff Castelli, Chief Development Officer
• Simon Harford, CFO
• Sébastien Martel, Chief Business Officer

Analysts

• Anupam Rama, Senior Biotech Analyst at JPM
• Dae Gon Ha, Director of Biotechnology Equity Research at Stifel
• Eliana Merle, Senior Biotech Equity Research Analyst at UBS
• Michael Riad, Equity Research Associate at Morgan Stanley
• Rick Miller, VP of Biotech Equity Research at Cantor Fitzgerald
• Ritu Baral, Managing Director and Senior Biotechnology Analyst at TD Cowen
• Tazeen Ahmad, Analyst at BofA Securities
• Analyst at Goldman Sachs
• Analyst at Leerink