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Good morning, ladies and gentlemen and welcome to the Amicus Therapeutics Second Quarter 2023 Financial Results Conference Call and webcast. [Operator Instructions] As a reminder, this conference 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.

Thank you, Tana. Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics' second quarter 2023 financial results and corporate highlights. Leading today's call we have Bradley Campbell, President and Chief Executive Officer; Daphne Quimi, Chief Financial Officer; Sebastien Martel, Chief Business Officer and Dr. Jeff Castelli, Chief Development 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. 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. All forward-looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof. For a full discussion of such forward-looking statements and the risks and uncertainties that may impact them, we refer you to the forward-looking statements and risk factors section of our Annual Report on Form 10-K for the year ended December 31st, 2022 and the quarterly report on Form 10-Q for the quarter ended June 30, 2023 to be filed later today with the Securities and Exchange Commission. At this time, it's my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Bradley?

Thank you, Sebastian and good morning, everyone. Starting on Slide 10, we remind everyone that late Onset Pompe is a severe and fatal neuromuscular disease and one of the most prevalent lysosomal disorders. Within LOPD individuals may experience impaired motor function and respiratory difficulties as the disease progresses, but these potentially debilitating symptoms tending to become more serious and problematic over time and orphan respiratory failure being a major cause of mortality in LOPD. Multiple publications and natural history studies continue to show 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 options is crucial. Moving on to Slide 11, we briefly outline the current regulatory status of AT-GAA by key markets. First, as noted in June, Pombiliti and Opfolda both were approved in EU and the commercial launch is underway, as Sebastien will detail in more in the next section. And in the U.S. as Bradley noted, we're pleased to see the increased level of engagement from the agency in recent weeks and remain confident we are on track for approval in the third quarter for both components of AT-GAA. And finally just this morning, we were very excited to receive the final full approval and orphan designations for both Pombiliti and Opfolda by MHRA and now the team can begin launching this important new therapy for people living in the U.K. with Pompe disease leveraging that positive reimbursement appraisal from NICE. Moving on to Slide 12, we remind everyone of our ongoing clinical studies and multiple mechanisms of expanded access that support much of the early demand for AT-GAA. For the younger Pompe community, we continue to enroll the ongoing open label

Thank you, Sebastian and good morning or afternoon, everyone. Our financial overview begins on Slide 16 with our income statement for the second quarter ending June 30, 2023. For the second quarter, we achieved total revenue of $94.5 million, which is a 17% increase over the same prior year period in 2022. This includes year-over-year operational revenue growth measured at constant currency exchange rate of 17% and a negligible currency impact at 0%. Cost of goods sold as a percentage of net sales was 9.6% as compared to 10.2% for the prior year period. Total GAAP operating expenses decreased to $104.2 million for the second quarter of 2023, as compared to $133.1 million in the second quarter of 2022. On a non-GAAP basis, total operating expenses decreased to $84 million for the second quarter of 2023 as compared to $119.2 million in the second quarter of 2022, primarily reflecting decreased program spend. We define non-GAAP operating expenses as research and development and SG&A expenses, excluding share-based compensation expense, loss on impairment of assets, changes in fair value of contingent consideration and depreciation. Within other expenses in the income statement in the second quarter, we recorded approximately $11 million in unrealized foreign exchange losses. This compares to a $7 million gain in the second quarter of 2022. We are currently taking steps to mitigate our future exposure. Net loss for the second quarter of 2023 was $43.2 million or $0.15 per share as compared to a net loss of $62.2 million or $0.21 per share for the prior year period, driven by the revenue growth in Galafold and careful expense management, we continue to make progress towards our path to non-GAAP profitability in the second half of this year. As of June 30, 2023, we had approximately 287 million shares outstanding. Turning now to Slide 17. With a focus on achieving non-GAAP profitability, I'm pleased to share that we have revised both our revenue and operating expense guidance for the year. We are raising full year Galafold revenue growth guidance to 14% to 18% at constant exchange rates, driven by patient demand. We are also reducing our full-year 2023 non-GAAP operating expense guidance to $330 million to $350 million. The decrease in operating expense for 2023 as compared to 2022 will be achieved by continuing to drive efficiencies and prudent expense management, offset by continued investment in Galafold, AT-GAA clinical studies, nonrecurring costs for manufacturing, as well as global launch activities. We anticipate operating expenses to be nonlinear this year due to the pre launch and launch expenses. We also expect to see a larger portion of our operating expenses allocated to G&A this year, as we align our resources to support the launch of AT-GAA and the continued growth of Galafold. Cash, cash equivalents and marketable securities were $265.6 million as of June 30, 2023, compared to $293.6 million as of December 31, 2022. And with that, let me turn the call back over to Bradley for our closing remarks.

Great. Thanks, Daphne, Jeff, Sebastian. As you can see, we've been relentlessly focused on execution across our global business and we look forward to embarking on this next phase of Amicus as a company with 2 commercial therapies. Thank you to all of our employees who've enabled us to achieve these recent corporate milestones and we're always committed to our patient-focused mission of delivering life-changing therapies to people in need. Before I hand the call over to Q&A, as you may have also seen in this morning's filing after 15 incredible years at Amicus, Daphne, our long-time Chief Financial Officer, leader and friend has decided to retire. She'll remain in her current role as CFO until we appoint her successor at which point she'll remain on with Amicus through the end of the year in order to support a smooth transition. A search is underway and we've already identified a number of highly qualified candidates. I also just want to add that I'm personally grateful to you, Daphne, for your years of exemplary service here at Amicus and in particular for helping lead us on our course to achieve non-GAAP profitability later this year. On behalf of the Amicus Board of Directors and the entire executive team, I'd like to take this opportunity to formally thank you. You've been a significant part of the growth at Amicus and instrumental in guiding us to the sound financial position we're in today. With that, operator, we can now open up the call to questions.

Certainly. [Operator Instructions] And our first question comes from Tazeen Ahmad of Bank of America.

Brad, just wanted to clarify on the time line that you've stated forward for GAA is still expecting approval in 3Q. Do you get a sense that the responses that you gave FDA post the WuXi inspection facility have been sufficient for the agency, if not, what do you think are the remaining gating factors regarding questions around the facility in order to get approval?

Just as a reminder, we were very pleased with the outcome of the pre approval inspection at WuXi. We do feel like our responses were all adequate and the color we provided in the call here today, I think is important, which is in the last few weeks, we’ve seen an increased level of engagement from the agency and so that gives us great confidence, we’re on track for Q3 approval.

And our next question will come from Anupam Rama of JPM.

And Daphne I'll miss you at the conference this coming year. On the Galafold guidance increase, maybe from a regional perspective, where is that coming from? Is it coming from deeper penetration in core countries or progress in some of the new emerging countries?

Yes, maybe on the other things I’d highlight, sorry, I didn’t mean to cut you off, I was just going to say the other thing I’d highlight is and Sebastian mentioned it on the call, not only are we seeing great penetration rates, we’re opening up new geographies. So we have some important countries coming on board this year. It’s also in the backdrop of a very healthy and growing fabric market. So diagnosis continues to be strong and as we find those patients, as a reminder, typically, you find one undiagnosed Fabry patient, newly diagnosed Fabry patient because it’s an X-linked disease that typically leads to 4 to 5 additional family members who are diagnosed. So in addition to, I think, executing incredibly well, which we’ve continued to do, we’re also seeing just a healthy growth of market and as Sebastian highlighted, we’re actually the fastest growing of the Fabry treatments for patients with mutations.

Our next question will come from Ritu Baral of TD Cowen.

I want to go back to the WuXi inspection and follow-up on Tazeen's question. Brad, can you give any additional detail on the points of the increased level of engagement that the agency has had just wrapping all of this up? Do you think that there's any possibility of requiring the re-inspection? Or do you feel that the outstanding items are addressable over written or verbal interactions. Any additional detail would be helpful?

No, we do not believe that there'll be any additional inspections needed at WuXi, we are very confident and we're very pleased with the outcome of the inspection. And again, just based on the kind of level of reengagement we've seen, the increased level of engagement we've seen in the last couple of weeks, it feels to us as you said, that they're wrapping things up and we feel like we're getting close to an approval here as we suggested on track for Q3. So we're really excited. The team is ready. We're really pleased to see the U.K. news this morning, so I think the momentum is building and really eager to see the remaining months of the year play out.

If I can squeeze one more in about how much supply do you -- thanks -- how much supply from the open label are German and U.K. patients sort of sitting with before they start on the path to commercial conversion?

Yes, we have -- our target is about 2 years of go-forward supply. Remember, we've made quite a bit...

I'm sorry, I meant like how much supply clinic -- how much drug supply do they have sitting from the open label...

That they’re working through, right, right. Got it. Sorry. Thanks for the clarity. Yes, the good news is, unlike Galafold, where you would typically – because it was a long shelf life, et cetera, you could then be sitting on kind of 3 months of supply of Galafold as we were winding down the clinical studies and transitioning over to commercial. In this case, it’s a much tighter supply situation and the clinical operations team at Amicus, who, of course, has already done this once before with Galafold works very closely with the medical affairs team, with the sites themselves. And so it’s a really fine and precise level of supply and that’s what allows us to very carefully manage and have that goal of 90-day transition. And in Germany, so far, as I’ve said, even in the first kind of 30-days of launch now, we are making those transitions and we’re confident we’ll be able to execute that within the first 90-days. And I think likewise in the U.K. and then the U.S. when we see the approval.

And our next question will be coming from Eliana Merle of UBS.

Just if you could provide us a little bit more color on how you're thinking about new patient starts in Germany and the U.K. beyond the patients that are on expanded access. So maybe just of the 200, I think you said Pompe patients total in the U.K. and I think you said 45 were already on AT-GAA. How should we think about the other 150 patients? And maybe what proportion might be on Nexviazyme already and just the time lines for when you expect the NICE reimbursement to kick in from a revenue perspective? And then in Germany, I know you mentioned you have 20 patients already on AT-GAA, but just if you could give us a sense of maybe how many Pompe patients there are in Germany? And how you think about the landscape there relative to Nexviazyme?

And I think you had maybe one more question, Eli, on the reimbursement process in the U.K. So as I mentioned in the call, once you have MHRA approval, what's remarkable here is we've already had the initial recommendation for reimbursement by NICE, which we think is one of the fastest ever to get to that answer prior to MHRA approval. The process from here should take a few days to a week to get to the final appraisal document by NICE, the draft is already posted, so that should come here relatively shortly. And then it takes around 30 days for NHS to provide funding to the local centers and that's when we'll start actually converting those expanded access in clinical trial patients. But in the meantime, with the approval, our sales team can go out into the field, visit the physicians. And again, along with medical affairs and clinical operations team can begin scheduling those infusions ahead of time. So effectively, launch is underway and we're waiting about 30-days for that reimbursement process to kick in at the local centers.

And our next question will be coming from Joseph Schwartz of Leerink Partners.

I was wondering beyond the EAP conversion of those patients to commercial status, how should we be thinking about the cadence of uptake for AT-GAA? And what metrics will you be providing us in order to gauge your progress with that? And are there any analogues that you can think of that might be helpful for us when envisioning the launch curve? And then can you remind us of your most recent thoughts around pricing and how this relates to the current price of Sanofi's products?

All good questions. So as it relates – I’ll start with the last one first, as a reminder, our pricing philosophy is parity or modest discount to standard of care. And we have shown with Galafold that, that is an incredibly successful strategy. We went through the pricing and reimbursement process with Galafold much faster than industry average. And I think it’s because the healthcare systems are recognizing that we’re bringing significant value, but we’re pricing at roughly parity, which takes the pricing decision off the table and focuses on the value of the product and we saw is that way you ca’ maximize access to therapy and maximize the number of patients on therapy as quickly as possible versus taking time to negotiate for some extra dollars on the price. And I think the nice appraisal that we saw here, which again I think is one of the fastest ever in our industry is a great reflection of that value that the payers are seeing. So we’re confident that strategy will be very successful here and we’ll roll that out as we go through the process in Europe, in the U.S. and in other markets. On the first one in terms of key performance indicators that we share, I think what you’ve heard on the call today is some of the qualitative things we’ll talk about in terms of getting out to see the key centers in terms of having patient started infusions and convert from clinical trials to commercial product. We haven’t given any forward-looking guidance for this year just because it’s such a stub year, but I think you’ll hear on the November call that we’ll provide an update on the number of commercial patients on drug, so that will be a nice milestone to wait for. In terms of kind of analog, it’s so unusual to have a second-generation product launched into the rare disease space. I don’t know if there are great analogs. The one thing I would point to though is kind of a thought experiment, perhaps is remember, we were – we qualified for that IMS program in the U.K. about a year ago. And within a first year, remember that you can’t promote on that product, it’s simply offering it and it’s purely demand-driven. And we were able to get to a 15% to 20% market share through that program alone, again, with no promotion. So for me, that gives you a great perspective on imagine what we can do once we’re out there promoting the product. So we’ll do our best to give good color on how the launch is going and hopefully that gives you a flavor of how we can talk to that over the coming days and weeks. And again, November, I think, will be a really important update there.

And our next question will be coming from Dae Gon Ha of Stifel.

One clarification, the slides as first patients as [en plural] has been infused with LP, I guess, AT-GAA product. Just wanted to clarify that it was more than one and then my actual question was the physician feedback and engagement, what are you guys hearing in terms of the line-up that physicians will take once next year, I guess, AT-GAA becomes available as a third line, I mean does it get kind of placed at a third line? Or do you see it being used sort of in the -- in between Myozyme, Nexviazyme or Nexviazyme on to AT-GAA? Any color on that would be helpful.

And the last thing I'll say there is, remember, the real promise of this medicine is that we're the only product in a well-controlled clinical study that shows that patients who are on an existing ERT can switch to AT-GAA and see improvement in 6-minute walk and forced vital capacity. So we think that's a truly differentiated component of this therapy and we're just so pleased and eager to get this out to as many patients as possible as quickly as possible.

And Daphne, thanks so much for your service.

And our next question will be coming from Jeff Hung of Morgan Stanley.

Congratulations on the MHRA approval of Opfolda and congrats to Daphne on her upcoming retirement. You've reduced expenses to support your path to non-GAAP profitability. Can you just talk about the importance of that relative to further bolstering your pipeline and advancing your next-gen pipeline programs?

Yes, so the vast majority of our expenses go towards both the ongoing clinical studies to support post-marketing commitments and remaining clinical study extensions in Pompe disease, also manufacturing until recently when now we have commercial product, so that ends up showing up in cost of goods, but that's the vast majority. And then of course, the G&A that goes along with that. We do have some modest spend towards the pipeline and we are making, I think, good progress there. I think you should expect to hear us talk more about the pipeline as we come into next year, but you should know that this laser focus on non-GAAP profitability does include a modest amount of investment in the pipeline, that's an important part of the future of Amicus and again we'll talk more about that probably coming into next year.

And our next question will be coming from Salveen Richter of Goldman Sachs.

This is [Sinatra] on for Salveen. First, on Galafold, in your view, how much of the growth that you need to get to your prior guidance of $1 billion peak sales for Galafold will be driven by new patient diagnostics like genetic screening, et cetera, versus the naive patients who are already diagnosed and switched patients. And on AT-GAA, if you could provide some more color on the reimbursement process in Germany?

And our next question will be coming from Kristen Kluska of Cantor Fitzgerald.

And congrats on the additional approval this morning and also sending my best to Daphne. I know it's early days in Europe, but can you talk about what have been the biggest selling points in terms of generating interest over other therapies now that you have an approval in place beyond the prep work. So for example, is the mechanism, the full data set across the different patient populations, open label, a certain endpoint that they're focusing on? Or is it really just a combination of this?

I'll hit the top -- start with that, I'll hit the top line and then maybe Jeff talk about the broader data that we've showed in previous calls. So from a label perspective, clearly, the most important two data points are the 6-minute walk test and the forced vital capacity, which as I mentioned before, we've shown significant differentiation. We're the only therapy that's shown improvement in those measures in patients who switch from existing ERT to AT-GAA and so I think that's a really powerful story and one that physicians will clearly focus on. But I do think another important part of this is the really broad data set that we've published now in various places. It won't necessarily show up in the label, but as we continue our medical education strategy, we'll be able to share those. So maybe Jeff, talk about what we've already published across those other endpoints and any other color you might want to provide?

As Brad noted, the key there is showing the improvement on 6-minute walk in [FDC] and the switch patients in a controlled trial. But it’s really that consistency across endpoints. We’ve shown some of that previously at a high level. We’re going to continue to really focus on that here in upcoming conferences just to show the range of improvements that we’re seeing across endpoints for these patients. And I’d say the other thing is the emerging durability data as well. We’ve now shown out to 4 years from our Phase ½ that the initial improvements observed across I patients, switch patients in those trials have been maintained long-term. So we think that, that’s going to be a continued focus of physicians and a strength so far of what we’ve seen with AT-GAA. But what’s really strong core of data, I think the MoA is also something that gives people a reason to believe in some of those clinical observations that there is a very differentiated MoA and that’s also something that I think we’re going to continue to provide publications and more information there as we continue to learn about the MoA.

And our next question will be coming from Ethan Markowski of Needham & Company.

Just a quick one on the current SG&A spend. So -- you're currently relying somewhat on the existing sales force for Galafold, but we've definitely seen an uptick in SG&A. Just to give us an idea now that you're rolling out Pombiliti and looking at a U.S. launch, how much more of the SG&A spend increase should we expect in the near-term?

Daphne, I'll let you talk to the accounting treatment that I think is describing a lot of that change. From an infrastructure perspective, what we've said before is, we literally have hired less than a dozen employees globally to support the launch of AT-GAA that includes some Amicus Assist, which is our take management hub in the United States, so patient education liaisons, some direct marketing and medical affairs people, but hugely leverageable, existing commercial infrastructure. But maybe Daphne talk to the trends that the question was speaking to.

Yes, sure. So I would remind everyone, in prior years, we had a portion of the support for both Galafold and now AT-GAA in the research and development line. But now as we are aligning resources to support two commercial products, some of that spend is now shifting from the R&D line to the SG&A line. So it is a bit of a re -- it's a classification issue when you look at current year versus prior year.

Yes, so it's not actually...

...part of the true increase [indiscernible].

Exactly, yes. Thanks, Daphne. So you should not expect to see significant additional increases in G&A, it was really more of a change in the classification for some of those employees who support the global business.

And our next question will be coming from Tazeen Ahmad of Bank of America.

Brad, just to maybe put people minds at ease. In terms of the -- going back to the GAA question about your time line for 3Q, just because PDUFA has officially passed, how are you getting your confidence that they will -- the FDA will provide an answer this quarter? And also as we head into the later part of August, I think the assumption is that the folks at FDA kind of shut down for holiday and so if it's slipped into September, is there anything to read into that?

Thanks, Tazeen, we’ll provide as much color as we can here. So the confidence really comes from 2 places. The first is, if you remember back when we announced the inspection, we estimated that if you look on average, even though we don’t have a specific PDUFA date on average, it takes about 30-days from the inspection to finish the inspection report and then it’s about 50 to 90 days for the agency to complete their review and so we’re kind of right in that zone. So we feel very comfortable that it’s kind of in the time line that we’ve seen when you look on average. And then the other piece that gives us good confidence to your point about whether the FDA is round or not as we have seen in the last couple of weeks, an increase in the engagement by the review division and that gives us good comfort ’hat they're wrapping up their work. And again, we expect the decision here in Q3 and we’re very confident in that.

And that was our last question. I'd like to turn the call back to Bradley Campbell for closing remarks.

Great. Thank you, everybody for tuning in today and hope everybody has a great day. Take care.