Amicus Therapeutics Q1 2023 Earnings Call Transcript

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Provided by Quartr
May 10, 2023

There are 11 speakers on the call.

Operator

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics First Quarter 2023 Financial Results Conference Call and Webcast. At this time, all participants are in a listen only mode. Later, we will conduct a question and answer session and instructions will follow at that time. As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr.

Operator

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

Speaker 1

Good morning. Thank you, Gigi. Thank you for joining our conference call to discuss Amicus Therapeutics' Q1 2023 financial results and corporate highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer Daphne Queeney, Chief Financial Officer Sebastian Mortel, Chief Business Officer and Doctor. Jeff Castelli, Chief Development Officer.

Speaker 1

Joining for Q and A is Doctor. Mitchell Zollman, Chief Medical Officer and Alan Rosenberg, Chief Legal Officer. As referenced on Slide 2, we might make forward looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as well as our plans and prospects. Our forward looking statements should not be regarded as representation by us that any of our plans will be achieved. Any or all the forward looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties.

Speaker 1

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, We undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof. For a full discussion of such forward looking statements and the risks and uncertainties that may impact them, we refer you to the forward looking statements and risk factors section of our annual report on Form 10 ks for the year ended December 31, 2022, and the quarterly report on Form 10 Q for the quarter ended March 31, 20 23 to be filed later today with the Securities and Exchange Commission. 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 Q1 2023 conference call. I'm pleased to report a successful start to the year across our global business. As we did in this morning's press release, let me highlight several key points before I turn it over to the team to provide more detail. First, Galafold continues its strong performance and remains the cornerstone of our success.

Speaker 2

We continue to be very pleased with the commercial uptake of Galafold globally With $86,000,000 in 1st quarter revenue, representing 14% growth from the Q1 last year on a constant currency basis, Strong trends continue across a number of our key performance indicators, including increasing demand through new patient starts from both switch and naive populations, Steady growth of in person visits between our field team and Fabry Treaters and sustained patient compliance and adherence rates of over 90%. We expect to see continued double digit growth in 2023 fueled by significant demand from patients and physicians. And in fact, as Sebastian will highlight in a moment, This was the best Q1 performance for net new patient starts in the past 4 years. 2nd, we continue to make great progress on our global regulatory filings and Our novel 2 component therapy for Pompe disease. Importantly, I'm very happy to report that the U.

Speaker 2

S. FDA has recently completed the required pre approval inspection of the Wuxi Biologics manufacturing site in China. We're very pleased with the outcome of Inspection and believe the comments and observations received by Wuxi at the close of the inspection are all addressable. We therefore continue to expect regulatory approval of ATGA in I'd also like to give a word of thanks to the FDA inspectors, the WuXi team and the Amicus team for the collaborative and professional conduct leading up to and during the course of the inspection. The regulatory reviews in Europe and UK also remain on track.

Speaker 2

In March, the European Commission granted approval of POMBILE for the use in combination with ARFOLDA for adults with late onset Pompe disease. And as we just announced, the CHMP also adopted a positive opinion recommending marketing authorization of OpFolda, so we continue to anticipate the full approval of POMBILITY plassofolda to occur in the Q3 this year. And upon approval, this will be the first two component therapy available in the European Union for the treatment of adults living with late onset Pompe disease. And finally, the U. K.

Speaker 2

Regulatory process for ATGA was initiated December of last year and is following a similar timeline as the U. S. And Europe with expected approval in the Q3 this year as well. As we've observed throughout 2022, we remain We are extremely pleased with the level of interest and participation we're seeing in our expanded access mechanisms for ATGA globally. As we Get through the 1st part of 2023 across all of our ongoing clinical studies and expanded access programs.

Speaker 2

There are now approximately 200 patients on ATGA, which we believe represents more than 5% of the total treated Pompe patients around the world. And we have more patients in the queue, so we expect this number to continue to trickle upward as we In anticipation of the regulatory approvals, our global launch planning progress continues as we eagerly anticipate the final approvals of ATGA. We very much look forward to providing a real choice in this therapeutic area, both in the United States and in Europe, with regulatory applications in additional countries planned in the months ahead. And 3rd, Anakus has maintained a strong financial position as we continue to execute on the global expansion of Galafold and prepare for the global launch of ATGA. Importantly, based on the latest assumptions on approvals and launch of ATGA, our current operating plan and continued careful management of expenses, We remain on track to achieve non GAAP profitability, which we expect in the second half this year.

Speaker 2

And on Slide 4, as we laid out at the beginning of the year, We are laser focused on achieving our key strategic priorities for 2023, including number 1, sustaining that double digit growth revenue of 12% to 17% for Galafold at constant exchange rates, delivering this important medicine to more people living with Fabry disease with a minimal variance in existing and new markets. 2nd, securing regulatory approvals of ATGA by the FDA, EMA and NHRA this year, as well as continuing to advance preparations for the anticipated launches. Number 3, continuing to judiciously invest in the advancement of our best in class next generation Fabry Pompe genetic medicines and capabilities as well as our next generation chaperone for Fabry disease. Number 4, as always, maintaining a With that overview, let me now turn the call over to Sebastien Martel, Our Chief Business Officer to further highlight the Galafold performance. Sebastian?

Speaker 3

Thank you, Bradley, and good morning to everyone on the call. I'll start by providing you with more detail on our Galapold performance for the quarter. As you can see on Slide 6, for the Q1 2023, Galafold reported revenue reached $86,100,000 driven primarily by strong patient demand. Geographic breakdown revenue during the quarter consisted of $57,000,000 or 2 thirds of revenue generated outside of the U. S.

Speaker 3

And the remaining $29,000,000 or 1 third coming from within the U. S. That's in line with the split that we expect as we continue to grow both parts of the business. As Bradley mentioned, we're pleased to see continued patient growth in countries across our leading markets. Now turning to Slide 7, our results for the Q1 highlight the strength of our global commercial efforts.

Speaker 3

The demand for Galafold globally continues to be strong with patients added in all major markets, delivering operational growth rate of 14% over the same period in 2022 at constant exchange rates. From a year over year perspective, The negative impact from foreign currencies was 4% in the period. As a result, Galafold reported revenue growth versus the same period last year was 10% for the Q1. Galafo continues to be the fastest growing treatment for Fabry disease globally. I'm pleased to report that our monthly net new patient trends continues to increase.

Speaker 3

And if you look at the growth in net patients on Galco globally, which is the truest measure of the underlying business, we see a high teen growth rate in patients on Galafold at the end of Q1 this year versus the same period last year. So all indications are of the continued and growing demand for Galafold. When we ended the Q1, we had more than 55% of the global market share of treated amenable patients. And within the global mix, we're starting to see stronger uptake in naive populations. So while we're achieving high market shares in countries where we've been approved the longest, We feel plenty of opportunity to continue to switch patients over to Galafold and continue to grow the market as we penetrate into the diagnosed and treated and newly diagnosed segments.

Speaker 3

All of that is underpinned by impressive compliance and adherence rates that continue to exceed 90%, We recognize by payers as we've got a very strong track record of successfully negotiating and renegotiating Reimbursement outside of the U. S. Our relentless commitment remains on ensuring access to Garafold for anyone who needs it. Based on the performance we've seen here in the Q1 of 2023, Galapol uptake continues to track very well and we're seeing those trends continue with growth across all our major markets as well as most of our smaller markets. Turning to Slide 8.

Speaker 3

GAAP growth remains strong with $86,100,000 in revenue in Q1. As mentioned on past calls due to a variety of factors, Including uneven ordering patterns and FX fluctuations, the rate of growth within the year is typically non linear, and we expect that to continue throughout 2023. In the table that you have on the right hand side of the slide, we've provided a 5 year historical snapshot The percent of GAAP wholesale that occur each quarter during a given year, we would expect a similar trend to occur this year. Now moving on to Slide 9, we know there's significant demand for Galafold and that the segment of the global Fabry market made of patients with amenable mutations has the potential to surpass $1,000,000,000 in annual revenue in around 5 years. We anticipate sustained growth throughout 2023 to be driven by several key core drivers, continuing to penetrate into existing markets, Further uptake into the diagnosed and treated population, expanding into newer geographies and label extensions.

Speaker 3

As we just mentioned, all of these efforts are supported by sterling compliance and adherence rates and positive reimbursement and access mechanisms around the world. I'm pleased to share that we're making continued progress on expanding into new markets and also on extending our label. To name a few examples, we recently received marketing The extension of use to adelisting patients 12 years and above has recently been approved in Canada And we've submitted the marketing authorization application in New Zealand and we've now entered into pricing and reimbursement negotiations for Garafold in Turkey. In the longer term, we continue to see significant growth in the public market globally, driven by diagnosing patients through a variety of measures, including high risk screening, newborn screening and other diagnostic initiatives, which we continue to support and invest in as well. Also important to note here, we have orphan exclusivity in the U.

Speaker 3

S. And Europe. In addition to our now 49 Orange book listed patents, including 8 compositions of master patents and 33 of those give us IP coverage into the late 2030s, which provides us the opportunity to provide access to Carahoe globally for a long time to come. Now moving on to Slide 10. We're happy today to stand less than a quarter from the commercial launch of Mobility and Upholda.

Speaker 3

On that slide, we outlined launch preparations as we're poised for another successful product launch. As you know, Amicus is a presence in over 40 countries around the world Today, including all the major Fabry and Pompe markets, that team that will launch Bombivity and Opoldade largely stayed the same with only a few additional FTEs. We're now fully recruited and trained to support the 1st wave of launch countries. I'm pleased to note The product is being positioned to support the 1st group of patients coming on to commercial supply. We have experienced across all areas that are needed for successful drug launch, Regulatory, commercial, supply chain, experience with payers, reimbursement and access, in addition and most importantly the key relationships with physicians.

Speaker 3

We're confident in our world class organization that we can leverage their experience and relationships and deliver ATGA To people living with competencies around the world. Our educational and promotional materials are ready And training in our initial launch countries is now completed. Market mapping is also complete. From the team, the medical education our ability to market rare disease products successfully, our experience with reimbursement and access around the world, And again, all the strategic planning that we've been doing together with building inventory with our partners at WuXi Biologics, we believe we're in a very strong position for a second With that, let me now hand the call over to Jeff Castelli, our Chief Development Officer, to highlight our ATGA program and pipeline updates. Jeff?

Speaker 4

Thank you, Sebastian, and good morning, everyone. On Slide 12, we'll start with our ATGA program. Pompe is a severe and fatal neuromuscular disease and one of the most prevalent lysosomal disorders. And multiple publications and natural history studies have shown that the initial benefits of treatment are often followed by continued long term decline for many individuals. We recognize that Pompe disease continues to pose a range of health challenges for people affected by the disease and having therapeutic choices is crucial.

Speaker 4

Moving on to Slide 13, we remain very excited to be anticipating potential regulatory approvals in 3 of the largest Pompe markets in the Q3 this year as Bradley noted. On this slide, we summarize the status of the anticipated regulatory milestones by market. First, in the EU, Pumbili was granted European Commission approval in March. In April, the CHMP adopted a positive opinion about FOTA, and we expect an EC approval to occur in the Q3 of 2023. In the U.

Speaker 4

S, the FDA recently completed the Our pre approval inspection of the Wuxi Biologics manufacturing site in China, and we are very pleased with the outcome and believe that the comments and observations received by WuXi at the closing inspection are all addressable. We continue to expect approval of both components of ATGA together in the Q3 of this year. And finally, the regulatory submission process for ATGA in the UK was initiated in December via the recognition procedure based on the CHMP opinion, And we're on track for an expected MHRA approval also in the Q3. So lots of great momentum across the

Speaker 5

key markets.

Speaker 4

Moving on to Slide 14, we highlight our ongoing clinical studies and multiple mechanisms of expanded access that support some of the early demand for ATGA. For the younger Pompe community, we continue to enroll the ongoing open label ZIIP study in children up to 18 years of age living with late onset Pompe disease. We have now begun enrolling the open label RYZELA study for children living with infantile onset Pompe disease. Importantly, in response to the Multiple requests for treatment that we continue to receive for children living with LOPD and IOPD, our expanded access programs continue to enroll those patients. We have multiple expanded access programs in place, including in the U.

Speaker 4

S, U. K, Germany, France, Japan and other countries. This includes the Early Access to Medicine Scheme or EAMS in the UK, where we continue to see significant enthusiasm for ATGA, With physicians having requested access across all of the leading Pompe centers and dozens of patients now on treatment. Since the positive scientific opinions, interest and momentum for ATGA has continued to grow and we are pleased to be able to provide access to those who are eligible. With this growth in our access programs, as Bradley noted, we are pleased to report that there are approximately 200 patients now worldwide Being treated with ATGA across our clinical extension studies and expanded access programs.

Speaker 4

And experience of ATGA is growing worldwide with approximately 75 centers currently participating in those different programs. Finally, as highlighted in the pipeline slide in the appendix For our earlier stage pipeline, we continue to focus on novel approaches for Fabry and Pompe, including gene therapy to deliver our engineered GLA and GAA transgene and the Next Generation Fabrice Chaperone. With that, I would like to now turn the call over to Daphne Queenie, our Chief Financial Officer, to review our financial results, guidance and outlook. Daphne?

Speaker 6

Thank you, Jeff, and good morning, everyone. Our financial overview begins on Slide 16 with our income statement for the Q1 ending March 31, 2023. For the Q1, we achieved total revenue of 86 $300,000 which is a 10% increase over the same period in 2022. This includes year over year operational revenue growth measured at constant currency exchange rates of 14% impacted by a negative currency of 4%. Cost of goods sold as a percentage of net sales was 8% in the year as compared to 9.6% for the prior year period.

Speaker 6

Total GAAP operating expenses decreased to $117,000,000 for the Q1 of 2023 as compared to $146,500,000 in the Q1 of 2022. On a non GAAP basis, total operating expenses decreased to $80,600,000 for the Q1 of 2023 as compared to $109,000,000 for the Q1 of 2022, reflecting decreased program spend. We define non GAAP operating expense as research and development and SG and A expenses, Excluding share based compensation expense, loss on impairment of assets, changes in fair value of contingent consideration and depreciation. Net loss for the Q1 of 2023 was $52,900,000 or $0.18 per share as compared to a net loss of of $85,300,000 or $0.30 per share for the prior year period. Driven by the revenue growth of Galafold and careful expense management, We continue to make progress towards our path to non GAAP profitability in the second half of this year.

Speaker 6

As of March 31, 2023, we had approximately 283,000,000 shares outstanding. Turning now to Slide 17. We continue to operate from a position of financial strength and our goal remains to achieve non GAAP profitability in the second half of twenty twenty three as defined in our press release. Profitability is dependent on a number of factors, including the timing of approvals and launch of AT GAA. Our full year 2023 non GAAP operating expense guidance is $340,000,000 to 360,000,000 The expected decrease in operating expense for 2023 as compared to 2022 will be achieved by continuing to drive efficiencies and prudent Thanks, management, offset by continued investment in the global growth of Galafold and pre launch and launch activities for AT GAA.

Speaker 6

We anticipate operating expenses to be non linear this year due to these pre launch and launch expenses as well as certain non recurring manufacturing costs that we expect will be incurred in the 2nd and third quarters of this year. We also expect to see a larger portion of our operating expense to be allocated to G and A this year as we align our resources to support the launch of AT GAA and the continued growth of Galafold. A few comments about our cash position and 2023 financial guidance. Cash, cash equivalents and marketable securities were 200 and $67,100,000 at March 31, 2023 compared to $293,600,000 at December 31, 2022. Our full year Galafold revenue guidance is revenue growth of 12% to 17% at constant exchange rates in addition to our non GAAP operating expense guidance of $340,000,000 to $360,000,000 And with that, let me turn the call back over to Bradley for our closing remarks.

Speaker 2

Great. Thank you, Daphne, Jeff, Sebastian for the detail there. So as you can all see, we've been relentlessly focused on execution this year across the business and have laid a strong foundation of what we think will be an important year for Amicus. As we approach this next phase of the company, we're confident we can continue to drive sustainable long term value and deliver life changing therapies to people in need. Operator, we can now open the call to questions.

Operator

Thank you. Thank you. One moment for our next question. Our first question comes from the line of Anupam Rama from JPMorgan.

Speaker 5

Hi, thanks for taking the question. This is actually Malcolm Cunha on for Anupam. So looking past the 3rd quarter approval, What observations have you learned from Sanofi's launch in Pompe that you can perhaps apply to your own launch? And then How should we view your launch curve relative to that competition?

Speaker 2

Yes. Thanks for the question. Maybe I'll start. Look, so we are laser focused, as we said, is getting through the approvals and launch. And I think for us, The most important thing is providing a new choice that we think is differentiated in particular with its 2 component mechanism of action.

Speaker 2

We have Watched, I think Sanofi, both in the United States and other markets around the world. And I think you could see depending on when they've launched, How they've progressed. And for us, I think it's much more about continuing to demonstrate the value of ATGA To physicians and patients, I think we look at the expanded access programs and update where the product is available Through those mechanisms and we take great confidence from the way we see patients and physicians using the medicine. And from our perspective, The most important thing is looking at what we've demonstrated in the clinic that in particular in the experienced population, When patients switch from therapy, we see a significant increase in 6 minute walk distance as well as improvement in force vital capacity. We think that Differentiation, the only therapy that's shown in a controlled study that we can show those changes.

Speaker 2

We think that sets us up as a very differentiated therapy when we launch and we're just really eager to get over the finish line. To your second question in terms of how we kind of think about the launch uptake, It will come as no surprise, but of course the first focus is to convert our existing clinical and expanding access patients over to We'll also be focusing, of course, on patients who we think we can switch The current standard of care over to AT GAA. The goal this year, frankly, just given the fact that the approvals happen in the back half of the year or it takes time from a pricing In reimbursement perspective, the goal really is to maximize the number of patients on therapy by the end of the year and that we think gives us a really strong run rate going into 2024.

Operator

Our next question comes from the line of Ellie Merle from UBS.

Speaker 7

Hey guys, thanks so much for taking the question. Just wondering if you could elaborate a little bit on the comments and observations received from the FDA inspection and maybe just kind of What drives your confidence that all of these are addressable? And then just a question proceed early on how to think about the timeframe like from inspection to approval. I guess just in situations like this with a deferred action, how should we think about the typical timeframe and maybe Also just next steps from here, in terms of the timelines for addressing the comments and observations. Thanks.

Speaker 2

Yes. Thanks, Ellie, for the questions. As we said previously, leading up to the inspection, we're not going to provide details of The findings, but I will reiterate, we're very pleased with the outcome. The color I can provide is that we think the feedback was straightforward and addressable, That's shared by WuXi with all their experience with inspections and that's why we feel very confident here reiterating the approval in the Q3. I would say too, there was a high level of engagement, professionalism between the inspectors, WuXi and Amicus.

Speaker 2

And so all of this together makes us feel very confident In that timeline that we reiterated today, you're right, in terms of sort of what happens from here, we are in a little bit of an unusual situation. But typically what happens is, as the FDA finalizes the inspection, which has now happened, then they finalize the inspection report, That takes a period of time. And once the inspection report is finalized and provided to the review division, that's where you get that kind of 30 to 60 day timeline to And so that gives us a lot of comfort that we're on track there based on the fact that we received the deferred action letter. And again, we'll remind folks at least for the regulation, it Those can only be given when no other issues remain and the application otherwise satisfies the requirements for approval. That gives us great confidence that effectively it's really just getting through the final inspection report and then final memos within the agency and then we're on to approvals and launch.

Operator

Our next question comes from the line of Joseph Schwartz from SVB Securities.

Speaker 8

Hi, thanks so much. I was wondering If you could talk a little bit about the market opportunity from Pumbili and whether That has evolved or your strategy for launching it has evolved now that the launch has been delayed and a competitor has been on the market for a while Longer than was initially expected.

Speaker 2

Yes. Maybe Sebastien, do you want to talk a little bit about kind of what we're seeing and why we're remaining confident And that marked opportunity to Joe's question.

Speaker 3

Yes. So Joe, the overall market for Pompe last year Reached about $1,200,000,000 It's a market that has been growing at high single digit, low double digit for a number of years. Still, this is a disease that is under diagnosed. So we expect the overall market growth rate to continue at least at the same Rate, as you look at the various regions and our initial focus in the U. S, In the U.

Speaker 3

K, in Europe, these are the 3 largest markets for PompaDDs. We estimate that there is around 1300 patients treated for Pompe in Europe, Another couple of 100 in the UK and north of 800 patients in the U. S. I think we've seen repeatedly through all the ad comps that we've done and market research assessments that Physicians react very well to the product profile of AT GAA and in particular to the switch data that we've Generated from the PROPEL study. Obviously, the vast majority of the opportunity lies In this switch opportunity, the number of newly diagnosed naive patients for Pompe It's a much smaller number on a global basis, maybe 100 to 200 patients per year compared to an overall market of Actually treated patients somewhere between 3,500 and 4,000.

Speaker 3

So we'll be very much focused on ensuring that we have We provide easy access to ATGA. And as Bradley alluded to, I think that the Experience we're seeing through the early access programs bodes well for the launch of ATGA.

Operator

Our next question comes from the line of Ritu Baral from TD Cowen.

Speaker 5

Hey, guys. This is Athena on for Thanks for taking the question. Just another follow-up on the Wushu inspection. Has every building as part of the entire supply chain been inspected at this point? And do you anticipate that any of the inspection findings could be gating for approval?

Speaker 5

Thank you. Yes.

Speaker 2

Thanks for the question, Athena. So the important part of this inspection, so Wuxi That site in China has been inspected multiple times by multiple regulators for multiple products, all successful, so that's a good thing. This particular inspection was focused on the drug substance and drug product manufacturing facilities for our product in particular. And so I think that's the difference here. Now that that's been complete, there are no other inspections By the United States that we believe are necessary ahead of approval.

Speaker 2

And so we feel very confident at this point that we are on track there based on the outcome of the inspection. So I think at this point, we're Sort of all systems go and looking forward to getting through the finalization of the review and heading towards approval to launch.

Operator

Thank you. One moment for our next question. Our next question comes from the line of Daigouhn from Stifel.

Speaker 5

I'll stick to one. Wanted to get your take on pricing strategy given the parallel review And I understand you guys gave guidance on a 3Q approval for both sides of the Atlantic, but it does seem like EU might Come sooner, considering the processes remaining for the FDA side. So how are you thinking about that overall? And how does that compare to your Fabry strategy? Thanks so much.

Speaker 2

Yes. Thanks, Dae Gon. We have taken the strategy that We will price our medicines at parity or modest discount to standard of care. And that's really because we value That's more than anything else. So our goal is to get as many patients on therapy as quickly as possible.

Speaker 2

And we feel like taking price off the table, in other words, Focusing on the value of the medicine that it brings to patients to physicians versus the cost of the medicine is the best way to ensure that you move through the reimbursement process as quickly as possible. And what we saw with Galafold, to your point, is that we actually moved through that reimbursement process successfully and that we did it in Times that were much faster than industry average. So we'll look to apply the same strategy here. And in some instances, actually those Discussions are already ongoing and so we look forward to deploying the same strategy and again focusing on the value of the medicine versus the cost.

Operator

Our next question comes from the line of Kristin Kluszka from Cantor Fitzgerald.

Speaker 5

Hi, everyone. Good morning. Thanks for taking my questions. Wanted to ask what your latest thoughts are on how you might position ATGAA Relative to the benefits across respiratory outcomes and different measurements there, I know you presented more data at World's this year. And in the past, you've

Speaker 2

Yes. Thanks, Chris. And Jeff, maybe you can talk through the data we've seen both in the initial phase of the study and then what we released at World with the extension data.

Speaker 4

Yes. Thanks, Brad, and thanks, Kristen, for the question. So, as we've seen in our clinical We see on pulmonary function, which as you pointed is one of the basically the leading cause of mortality in Pompe that Patients that had been on state of the care ERT saw quite significant decline in their pulmonary function as they remained on treatment, whereas Patients that switched to ATGA showed good stabilization of that pulmonary function. And importantly, as we've now seen data In our extension studies from Phase onetwo out to 4 years and from our PROPEL study out to 2 years, that stability seems to be maintained and durable over those Additional extended time periods in the trials. So we're very excited about the potential ability to help stabilize that progressive decline in pulmonary function we see.

Speaker 4

And also importantly, the other key arm of Pompe is on the motor function and muscle strength side of things. And there we're actually really excited to see some improvements even in patients That which are not necessarily expected as patients switch to ATGA. So across those two key Aspects of disease, we're very excited by both the initial response to treatment and seen it PROPEL, but then the continued durability of the response in the extension trials.

Operator

Our next question comes from the line of Salveen Richter from Goldman Sachs.

Speaker 9

Hi, this is Srinathra on for Salveen. We have two questions. The first is, how soon post approval can we expect the first patients to be treated with ATGA? And here I'm referring to patients who are not on the early access programs. And the second is, could you provide some color on what we can expect in the 1st few months of the launch?

Speaker 9

Thank you. Yes.

Speaker 2

Thanks for the question. So in terms of first patients on therapy, so So there's 2 different situations. You have the United States, of course, where you get approved and then you're Moving quickly to setting up infusions and getting prescriptions and setting up infusions and getting patients on therapy. What we've seen in our experience with Galafold is our goal is to convert all of our Patients on therapy who are of course on label to commercial drug within the 1st 90 days. And so that's something we'll focus on and we were able to Excuse that very successfully in the Galafold launch, and we believe we'll be able to do that here.

Speaker 2

And then likewise in Europe, of course, you get approval in Germany, you typically have what's kind of colloquially known as that pre pricing period. So again, there Reimbursement will come almost immediately and the goal will be on the back of approval, the goal will be to quickly again convert Patients on existing therapy, I'll note in Europe or sorry, in Germany, we do have an expanded access program ongoing as well. And again, that's within Kind of that 30 to 90 day period. And then in the UK, we do we will go through the reimbursement process. And again, that's where that focus on access is so important.

Speaker 2

So there is a little bit of time to get from MHRA approval to NICE approval and reimbursement. We'll also, of course, while we're converting our existing patients, also be focused on initiating patients who are On existing therapy in the U. S. And all patients outside the U. S.

Speaker 2

As well that are on label.

Operator

Thank you. One moment for our next question. Our next question comes from the line of Jeff Hung from Morgan Stanley.

Speaker 5

Thanks for taking my question. Can you talk about the recent feedback you've heard from payers on AT GAA? And what kinds of comments do you hear on potential barriers on access or restrictions? Thanks.

Speaker 2

Yes. Thanks for the question. All of our market research from a payer perspective has been, I think, very Supportive of the approach that we outlined here in terms of our strategy, which is that as long as it's on label, then and if we have Parity or modest discount strategy, payers I think will be very supportive of this medicine. They've gotten more sophisticated, especially in this disease area. So they do review the data very carefully, And we expect that process to continue.

Speaker 2

And then likewise, outside the U. S. Where you're going through the HCA assessments. Again, I think with the data that we have, with the market research we've done leading into this process and now with the ongoing Negotiations, we've started with some of those payers. We think this is going to be a very successful access strategy and we're just eager to get through the process and provide

Operator

Thank you. One moment for our next question. Our next question comes from the line of Chi Chung Xu from Berenberg.

Speaker 10

Good morning. Thanks for taking the question. I wanted to ask your today announced the expansion of the Supply and manufacturing agreement with Wuxi on its Ireland facility, I guess, can you talk about the transition plan from the China facility to the Ireland And also do you expect the FDA inspection on the Hyalanta study in due course? Thanks very much.

Speaker 2

Yes, great question. So yes, we announced in the last couple of weeks, we did enter into a commercial manufacturing agreement with Wuxi. That was something that was in the works for quite some time and we're really thrilled to solidify that effectively secures Capacity longer term capacity and supply from Wuxi from both their China facility as well as their Ireland facility, The strategy really is to eventually transition so that the majority of supply comes from Ireland. And what we've suggested before is we're on track for that process to conclude that license that facility to be licensed And materials start to enter the commercial supply chain in the back half of next year or early 2025 and that process is going well. In terms of inspection of the facility, of course, you'll have to go through the typical PPQ and validation and comparability Work as we transition to Ireland, all of that is kind of contained in the timeline that I shared with you.

Speaker 2

Will the PA or other regulatory agencies inspect the facility, almost certainly they will at some point, whether that will be required as a pre Licensure inspection or whether it will happen sometime in due course to be determined. But we're very confident In that process, and we're eager to have, of course, the second site of manufacturing come online and one that's geographically diversified as well.

Operator

Thank you. At this time, I would now like to turn the conference back over to Bradley Campbell for closing remarks.

Speaker 2

Great. Thanks, operator. Thanks, everybody, for the participation in the call today, all your great questions. Hope everybody has a great day. Take care.

Operator

Thank you. This concludes today's conference call. Thank you and have a great day.

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Earnings Conference Call
Amicus Therapeutics Q1 2023
00:00 / 00:00
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