Amicus Therapeutics Q2 2024 Earnings Call Transcript

Quartr
Provided by Quartr
August 8, 2024

There are 10 speakers on the call.

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.

Operator

Andrew Fonin, Vice President of Investor Relations. You may begin.

Speaker 1

Thank you. Good morning, and thank you for joining our conference call to discuss Amicus Therapeutics' Q2 2024 Financial Results and Corporate Highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer Sebastian Martell, Chief Business Officer Doctor. Jeff Castelli, Chief Development Officer and Simon Harford, Chief Financial Officer. Joining for Q and A is Doctor.

Speaker 1

Mitchell Goldman, Chief Medical Officer and Alan Rosenberg, Chief Legal Officer. As referenced on Slide 2, we may make forward looking statements within this meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as well as our plans and prospects. Our forward looking statements should not be regarded as representation by us that any of our plans will be achieved. Any or all the forward looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward looking statements, which speak only to the date hereof.

Speaker 1

All forward looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof. For a full discussion of such forward looking statements and the risks and uncertainties that may impact them, we refer you to the forward looking statements and risk factors sections of our annual report on Form 10 ks 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 Chief Executive Officer. Bradley?

Speaker 2

Great. Thank you, Andrew, and welcome everyone to our Q2 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 1st 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.

Speaker 2

First, we continued our excellent commercial execution and delivered total revenue of $127,000,000 in the quarter, representing 34% growth year over year or 36% on currency basis. 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. At the product level, Galafold continues its strong performance with $111,000,000 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 2nd 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%.

Speaker 2

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 2nd 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%.

Speaker 2

2nd, let me highlight the continued strong global commercial launch of POMBILITY and AFOLDA, our novel therapy for late onset Pompe disease. POMBILITY 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. 1st 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 POMBILE and UFOLDA in 2024 continues to progress exceptionally well.

Speaker 2

In the Q2, 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 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 PONDBILEA and UFVOLDA, which will continue to fuel the momentum throughout the year and beyond. Sebastian 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.

Speaker 2

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've anticipated.

Speaker 2

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 POMBILE and AFOLDA 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 $62,000,000 to $67,000,000 in global POMBILITY and UFVOLDA sales, which will be a significant contributor to our growth and set us on a great course to achieve our ambition for POM UP 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. 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.

Speaker 2

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 the beginning of the year, we're focused on achieving our 4 key strategic priorities for 2024, including: 1st, 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% to 18%. Successfully executing on multiple commercial launches of POMBILE and Opdivo, 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 Sebastien, who'll go through our commercial performance in more detail.

Speaker 2

Sebastien?

Speaker 3

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 Q2 2024, Galafold reported revenue reached $111,000,000 driven by strong patient demand, particularly from our leading markets. We ended the 2nd 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 Q2, we're raising again our full year 2024 revenue growth guidance range to now 14% to 18%.

Speaker 3

Turning to Slide 7, Our results in the Q2 highlight the strength of our global commercial efforts. 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 rates. Our leading markets such as the UK, 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.

Speaker 3

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 stronger uptake in naive populations. We continue to achieve high market shares in countries where we've been approved the longest. 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.

Speaker 3

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 Follow Me 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.

Speaker 3

Data from the study aligns very well with previous observations from clinical trials and extends the available data supporting the real world multi system benefits of Garfield. This publication along with our ongoing medical education at Congress is an important part of the story of this oral precision medicine and help 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 Galaford and that the segment of the global Fabry market made of patients with amenable mutation has the potential to reach up to $1,000,000,000 in annual revenue by the end of the decade. We anticipate sustained growth in 2024 and beyond to be driven by several key drivers. 1st, the Fabry market is growing robustly with a significant portion of growth coming from finding new patients and reaching the diagnosed untreated population.

Speaker 3

As we mentioned previously, at the end of 2023, there were more than 2,400 individuals on Garafold and about 1400 of those were individuals 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 going through treatment through newborn screening, family screening and we continue to increase patient identification capabilities through ongoing medical indication and support of novel screening initiatives. Fabry remains unfortunately one of the most under diagnosed rare diseases. The more patients that can be identified, the more patients may benefit from Galafold.

Speaker 3

We're also seeing many diagnosed untreated patients transition to treatment as the need for earlier treatments, especially in females becomes better appreciated. The other piece is continuing to drive Galafold's market share of treated amenable patients through continued commercial execution. As noted, Galafold currently has more than 60% of the global amenable market. What we're seeing in our most mature markets, it can reach up to 85%, 90% of share. So we know that there's the potential to reach those levels in the global market share as well.

Speaker 3

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 Galaford into new markets and extending the labels. There's still some markets in Latin America, the Middle East and Asia Pacific regions where is either newly reimbursed or we expect reimbursement. Also important to note here, we have orphan drug exclusivity in the U. S.

Speaker 3

And Europe. And in addition to our now 61 orange book listed patents, 45 of which provide protection into 2,038 and beyond, including 14 composition of matter methods. This provides us the opportunity to continue to provide access to Galafo 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 is a long runway well into the next decade.

Speaker 3

Turning now to Pompe disease on Slide 10, we outline our global launch progress with Bombiliity and Opolda. For the Q2 2024, Bombivity and Opolda reported revenue reached $16,000,000 This represents an increase of 44% compared to the Q1 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 Nexvizyme about 68% and the remaining from Lumizyme.

Speaker 3

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 Myozyme, some from Nexvadigm at a proportional rate to the respective market shares and some from the naive populations.

Speaker 3

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 Switzerland Swissmedic has approved Bombilitinoprola as a long term enzyme replacement therapy, an 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 of health technology assessments are seen in this therapy. The price acknowledges the added benefit of formulity clots of Olapodil patients and will be closely aligned with the price we said at launch.

Speaker 3

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 Q1 of launch. We remain very pleased with the uptake of Bombili Tia and Aproda in the U. K, where we've captured greater than a third of market share within the region. With the benefit of beams, Bombilitia and Aproda, I've actually been in the market for 2 years in the UK, and we believe that the current market share is indicative of how this product can perform over 2 year periods, which is a great example of its potential demand and uptake.

Speaker 3

Given Pumbili and Opolda's solid commercial execution thus far, the first half of twenty twenty four, we're well on our way to deliver our full year revenue guidance of $62,000,000 to $67,000,000 for POMpeleti and Opdivo. 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 scheduled to be treated at the end of July. That's about 174 on therapies and the remaining 12 were scheduled. We remain very pleased with the ongoing demand for this therapy as the rate of new commercial patients coming onto Pombili TNFOLDA continues to increase across all three markets.

Speaker 3

Q2 in particular was a very strong quarter having seen the largest number of new demand patients being prescribed Bombili TNFOLDA. These are individuals not from clinical trials choosing to come onto commercial therapy. 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 in new countries in Europe.

Speaker 3

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, 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.

Speaker 3

We have a highly experienced team who are engaging these positive conversations with payers demonstrate the value of pomelite and Opdiv. In the U. S, the largest payers have already put pomelite and Opdiv to their respective formularies and 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. And the last few commercial patients have actually come in significantly quicker.

Speaker 3

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.

Speaker 3

Throughout the second half of twenty twenty four, 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 formulitynofoda 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 2nd 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?

Speaker 4

Thank you, Sebastien, and good morning, everyone. On Slide 12, we outline how we continue to build the body of evidence for PUMBILETA and AMCOLA 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 2nd 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.

Speaker 4

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 RYZELA 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 POMBILE in OpdivoTA 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.

Speaker 4

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 5

Thank you, Jeff. Our financial overview begins on Slide 14 with our income statement for the Q2 ending June 30, 2024. For Q2, we achieved total revenue of $127,000,000 which is a 34% increase over the same period in 20 revenue during the quarter consisted of $77,000,000 or 60% of revenue generated outside the United States and the remaining $50,000,000 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.

Speaker 5

Total GAAP operating expenses decreased to $100,000,000 for the Q2 2024 as compared to $104,000,000 in the same period last year, a decrease of 4%. On a non GAAP basis, total operating expenses decreased to $82,000,000 for the Q2 of this year as compared to $84,000,000 in the same period last year, a decrease of 2%. We define non GAAP operating expenses, research and development and SG and 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 Q2 2024 reduced to $16,000,000 or $0.05 per share compared to a net loss of $43,000,000 or $0.15 per share for the Q2 of 2023. In Q2, non GAAP net income was $18,000,000 or $0.06 per share compared to a loss last year in the same period of $20,000,000 or $0.07 per share.

Speaker 5

Cash, cash equivalents and marketable securities were $260,000,000 at June 30, 2024 compared to $286,000,000 at December 31, 2023. 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%. This is driven by the increase of our full year 2024 Galafold revenue growth guidance, which started the year at 11% to 16%, was most recently 13% to 17% and now is 14% to 18% at constant exchange rates. In addition, we are reiterating the guidance of $62,000,000 to $67,000,000 for PONDBILITY and Oxalder sales for the full year 2024.

Speaker 5

Our full year 2024 non GAAP operating expense guidance has been narrowed from $345,000,000 to 3.65 $1,000,000 to $345,000,000 to $360,000,000 or a $0.05 reduction at the top end. With our commitment to full year non GAAP profitability during the 1st full year of launch for Pumbiliy and OpFolde, 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 and D commitments, including registry studies in both Fabry and Pompe, the ongoing Pompe Phase 3 study in countries which aren't yet reimbursed, as well as next generation manufacturing for POMBILEITY. In the Q2 of 2024, non GAAP profitability was $18,500,000 and for the first half of twenty twenty four, dollars 13,900,000 With our total revenue guidance of 26% to 31% growth, we remain comfortably on track for 2024 to be our 1st 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.

Speaker 5

Bradley?

Speaker 2

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

Operator

Yes. Thank you. Your first question comes from just one moment please. This question comes from the line of Tazeen Ahmad with BofA Securities. Your line is now open.

Speaker 6

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

Speaker 6

Launch is going. The label languages are slightly different. And so 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? Thanks.

Speaker 2

Yes. Thanks, Celine. 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.

Speaker 2

I think you have 2 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 the year. However, in Europe, we're adding new countries as we go.

Speaker 2

We launched in Spain. We just got approval in Switzerland. We're working on 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.

Speaker 2

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 Myozymes and nexviodime as well as new patients, which are on the label there. In the U. S, we're seeing predominantly switches from nexViDyme and some switches from Myozymes 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

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

Speaker 6

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 NEXBizymes, 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 neXtiazine prior to switching to Palm Op? Thanks.

Speaker 2

Yes, thanks. I think essentially we're seeing what we had expected to see, which is the majority of patients switching from Nexvizyme have been on treatment in that kind of year to 2 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 2 years on therapy over the course of this year and into next year. And so I think we've got a large bolus of patients that we can target.

Speaker 2

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 pombilynaFolda. And I think that's another dynamic too that we'll want to continue to see, which is 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 some demand to switch earlier when they realize what Pumbiliant to unfold it can do for them.

Speaker 6

Great. Thanks.

Operator

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

Speaker 1

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.

Speaker 2

Maybe I'll start at a high level and then ask Sebastien 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 Sebastien, 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 3

Yes. Thanks, Brad. So, Anupam, we're seeing very strong demand for naive newly diagnosed patients in the U. S. And have seen that for a number of quarters now.

Speaker 3

That's actually why we've now raised guidance twice this year on Galafold. So a growing proportion of newly diagnosed patients really are put on Galafold as the very first line of treatments if you 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 5 years. So strong, very strong performance in the U. S.

Speaker 3

We continue to see the shift that we've seen for 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 the majority of the growth coming from naive patients. And we're seeing that in our largest markets in the UK. We're seeing great performance in Japan as well. We actually think that we have more growth upside in Japan simply because the Japanese business to us is our 4th largest.

Speaker 3

If you look at the whole family market, Japan is actually the 2nd largest market. So there's no reason why Japan couldn't, at some point in time, become our 2nd largest market as well. So these are some of the key highlights on regional performance.

Operator

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

Speaker 1

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 PAMAAL. 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 Q2 as compared to the Q1.

Speaker 1

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.

Speaker 2

Yes, thanks for the question. I think one of the things we talked about, Sebastian highlighted is the rate of new patients 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, 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 reimbursement and launches will be in the Q4, the rate of new revenue won't change very much, but obviously next year it will 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

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

Speaker 7

Good morning, guys. Thanks for taking the question. Congratulations on the metrics of this quarter. From our tracking survey, our pump up 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 Nexvizyme team over there have some very interesting messaging about Nexvizyme superiority over Myozymes, Lumazyme that seem to be resonating with some of the doctors that we surveyed.

Speaker 7

Can you talk to I mean, if that's the palmoc 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 2 drugs sort of stack up against each other in the field? And then I've got a follow-up on insurance. Thanks.

Speaker 2

Thanks, Ritu. Maybe I'll start and I'll have Jeff add some color. I would just I won't speak to how they're detailing, but what I would say is that we're the only product that has shown in a controlled phase of the Phase 3 an improvement in both 6 minute walkie 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.

Speaker 2

And in fact, you may remember our promotional materials, which is pretty incredible. We can 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. 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.

Speaker 4

Yes. Thanks, Brian. Thanks, 2 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 in that large group of switch patients improvements on 6 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, etcetera.

Speaker 4

So we're still really educating everyone about that data. Obviously, that data is one of the highest levels of evidence that you can have. And then there's still continuing education around mechanism of action and the unique differences in what we think we've built here with Homebuilding Axolta. 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 naive patients outside the U. S, people switching from NyxBionyme, people switching from Lumizyme.

Speaker 4

And we'll continue to report out that data importantly from our registry in other areas. And we're really excited from everything we're hearing anecdotally about the patient physician response to pump ability at FOLTA. 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

Speaker 3

in the future.

Speaker 7

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 Opdivo 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?

Speaker 2

Yes. Thanks, Ritu. It's funny. 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 2 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.

Speaker 2

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 that the staff goes through any new therapy, they have to get used to the paperwork, etcetera. 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.

Speaker 2

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

Speaker 7

Great. Thanks.

Operator

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

Speaker 4

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 add? Just like curious as to why you saw a stronger uptake in naive patients versus switch in 2Q? Thank you.

Speaker 2

Yes. 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, I think, improving diagnosis through the kinds of things that Sebastien talked about. But Jeff, maybe remind us the sort of change in market size from where we started the launch to today and then kind of what has changed in the diagnostic landscape that we think is feeling that?

Speaker 4

Yes. Thanks, Brian. Thanks for the question. As Brad said, it was about 10,000 patients when we launched Galafold 7 or 8 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.

Speaker 4

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. 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.

Speaker 4

Lots of exciting developments on the kind of medical record, use of AI, potential to screen for people that might be suffering with fibro unknowingly. So 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 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.

Speaker 4

And particularly in the U. S, the last thing I'll add is there is newborn screening going on in 5 or 6 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 3 to 5 members that also have Fabry that are adults and likely suffering. So lots of great tailwinds for the community in terms of finding new patients.

Speaker 3

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 Fibrosyme and Replagal, so the 2 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 4

Thanks so much. Appreciate all the context.

Operator

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

Speaker 8

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

Speaker 8

So kind of curious, is that part of your marketingcommercialization 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.

Speaker 2

Thanks, Dae Gon. Yes, 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 2 products that they're looking at. And so it is an element of changing expectations for therapy. We focus on that actually improvement is possible, maybe for the first time.

Speaker 2

So yes, 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 encourage them to look more closely at not just the primary measures, 6 minute walk and force vital capacity, but the other measures that may be changing suddenly 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 PUMPDIVITY and OPFOLTA.

Speaker 3

Thank you. Hey,

Speaker 4

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, the community is all now having to work together on as we have multiple treatments available and patients really need to be 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 in people that switched from ERT to palm up that those patients, many of them saw improvements across different parameters.

Speaker 4

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 from the data we've seen.

Operator

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

Speaker 9

Hi. Thank you so much for taking my question. This is Shunathra on for Salveen. So do you see any trends that suggest that physicians are maybe preferring to switch patients from Myozyme to Nexozyme before and evaluating them on that before going to permeability in both ERT experience patients in the U. S.

Speaker 9

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 early on?

Speaker 2

Yes. Thanks for the questions. I think for the first one, no. I think the dynamic is really that in the U. S.

Speaker 2

As an example, next Mizyme was approved far earlier than us. So the majority of patients were already switched over, although there are some remaining patients from MiZyme. And then outside the U. S, it's much more of a mixed circumstance. So what we've seen is that we're taking switch patients from kind of each segment equally.

Speaker 2

I think that reflects that we're making very good headway with the launch and that their physicians aren't preferencing switching to nexiazem first. I think it's just a reflection that we weren't available before. 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 to 45 days. That's kind of the journey we are on with Galafold. By the end of the 1st full year of launch, we were down to sort of 30 plus days.

Speaker 2

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 2 week kind of infusion window as well. But yes, we're looking to do that by the end of this year.

Operator

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

Speaker 1

Hi, this is Rick Miller on for Kristen. Thanks for taking our question. Just one from us. For POMBILEITY plus OPPOZA, can you comment on the prescriber mix you're seeing? Does this break down across larger academic centers versus community settings?

Speaker 1

And are there any other interesting trends that you're seeing on that front? Thanks.

Speaker 2

Yes, 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 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 adding their second or third or fourth prescriptions.

Speaker 2

So both of those dynamics have 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.

Speaker 2

S, you do have a top prescriber base and we're seeing great penetration into those. But you also have sort of 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. 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.

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Earnings Conference Call
Amicus Therapeutics Q2 2024
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