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

Good morning, thank you Gigi. Thank you for joining our conference call to discuss Amicus Therapeutics first 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; Sébastien 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 of 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 as of 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 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 31, 2022, and the quarterly report on Form 10-Q for the quarter ended March 31, 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?.

number one, 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 amenable variants in existing and new markets; second, securing regulatory approvals of AT-GAA by the FDA, EMA and NHRA this year, as well as continuing to advance preparations for the anticipated launches; number three, continue to judiciously invest in the advancement of our best-in-class next generation Fabry and Pompe genetic medicines and capabilities, as well as our next generation chaperone for Fabry disease; and number four, as always, maintaining a strong financial position as we carefully manage our expenses and investments on our path to non-GAAP profitability.

With that overview, let me now turn the call over to Sébastien Martel, our Chief Business Officer to further highlight the Galafold performance.

Sébastien?.

regulatory, commercial, supply chain, experience with payors, reimbursement and access, in addition and most importantly the key relationships with physicians. We’re confident in our world-class organization that we leverage their experience and relationships and deliver AT-GAA to people living with Pompe disease 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 teams in medical education and publications, 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 are in a very strong position for a second successful launch at Amicus.

With that, let me now hand the call over to Jeff Castelli, our Chief Development Officer, to highlight our AT-GAA program and pipeline updates.

Jeff?.

Thank you Sébastien, and good morning everyone. On Slide 12, we’ll start with our AT-GAA program. Pompe is a severe and fatal neuromuscular disease and one of the most prevalent lysosomal disorders.

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.

Moving onto Slide 13, we remain very excited to be anticipating potential regulatory approvals in three of the largest Pompe markets in the third quarter this year, as Bradley noted. On this slide, we summarize the status of the anticipated regulatory milestones by market. First in the EU, Pombiliti was granted European Commission approval in March.

In April, the CHMP adopted a positive opinion of Opfolda and we expect an EC approval to occur in the third quarter of 2023.

In the U.S., the FDA recently completed the required preapproval 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 close of the inspection are all addressable.

We continue to expect approval of both components of AT-GAA together in the third quarter of this year. Finally, the regulatory submission process for AT-GAA in the U.K.

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 third quarter, so lots of great momentum across the key markets.

Moving onto Slide 14, we highlight our ongoing clinical studies and multiple mechanisms of expanded access that support some of the early demand for AT-GAA. For the younger Pompe community, we continue to enroll the ongoing open label ZIP study in children up to 18 years of age living with late onset Pompe disease.

They have now begun enrolling the open label Rossella 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.S., U.K. Germany, France, Japan, and other countries.

This includes the Early Access to Medicine Scheme, or EAMS in the U.K., where we continue to see significant enthusiasm for AT-GAA 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 AT-GAA 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 AT-GAA across our clinical extension studies and expanded access programs, and experience with AT-GAA 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 therapies to deliver our engineered GLA and GAA transgenes and the next generation Fabry chaperone.

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

Daphne?.

Thank you Jeff, and good morning everyone. Our financial overview begins on Slide 16 with our income statement for the first quarter ending March 31, 2023. For the first quarter, we achieved total revenue of $86.3 million, 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.

Total GAAP operating expenses decreased to $117 million for the first quarter of 2023, as compared to $146.5 million in the first quarter of 2022. On a non-GAAP basis, total operating expenses decreased to $80.6 million for the first quarter of 2023, as compared to $109 million for the first quarter of 2022, reflecting decreased program spend.

We define non-GAAP operating expense 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.

Net loss for the first quarter of 2023 was $52.9 million or $0.18 per share as compared to a net loss of $85.3 million 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.

As of March 31, 2023, we had approximately 283 million 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 2023, 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 million to $360 million.

The expected 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 the global growth of Galafold and pre-launch and launch activities for AT-GAA.

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 second and third quarters of this year.

We also expect to see a larger portion of our operating expense to be allocated to G&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 $267.1 million at March 31, 2023 compared to $293.6 million 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 million to $360 million.

With that, let me turn the call back over to Bradley for our closing remarks..

Great, thank you Daphne, Jeff, Sébastien for the detail there. 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..

Thank you. [Operator instructions] Your first question comes from the line of Anupam Rama from JP Morgan. .

Hi, thanks for taking the question. This is actually Malcolm Kuno on for Anupam.

Looking past the third 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?.

Yes, thanks for the question. Maybe I’ll start. Look, we are laser focused, as we said, at 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 two-component mechanism of action.

We watched Sanofi, both in the United States and other markets around the world, and I think you could see, depending on when they launched, how they’ve progressed. For us, I think it’s much more about continuing to demonstrate the value of AT-GAA to physicians and patients.

I think we look at the expanded access programs and uptake where the product is available through those mechanisms, and we take great confidence from the way we see patients and physicians using the medicine.

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 six-minute walk distance, as well as improvement in forced vital capacity.

We think that differentiation, the only therapy that’s shown in a controlled study that we can show those changes, 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 think about the launch uptake, it will come as no surprise but of course the first focus is to convert our existing clinical and expanded access patients over to commercial medicine, and we’re prepared to do that as quickly as possible.

We’ll also be focusing, of course, on patients who we think we can switch current standard of care over to AT-GAA.

The goal this year, frankly, just given the fact the fact that the approvals happen in the back half of the year, it takes time from a pricing and 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..

Thank you. One moment for our next question. Our next question comes from the line of Ellie Merle from UBS. .

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 what drives your confidence that all of these are addressable, and then just a question procedurally how to think about the time frame from inspection to approval, I guess just in situations like this, like a deferred action.

How should we think about the typical time frame, and maybe also just next steps from here in terms of the timeline for addressing the comments and observations. Thanks..

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 third quarter.

I would say too, there was a high level of engagement and professionalism between the inspectors, WuXi and Amicus, and so all of this together makes us feel very confident in that timeline that we reiterated today.

You’re right - in terms 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 approval, 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 I’d remind folks at least per the regulations, it says that 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 onto approval and launch..

Thank you. One moment for our next question. Our next question comes from the line of Joseph Schwartz from SVB Securities. .

Hi, thanks so much. I was wondering if you could talk a little bit about the market opportunity from Pombiliti 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. .

Yes, maybe Sébastien, do you want to talk a little bit about what we’re seeing and why we’re remaining confident in that market opportunity, to Joe’s question?.

Yes, so Joe, the overall market for Pompe last year reached about $1.2 billion. It’s a market that has been growing at high single digits, low double digits 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.K., in Europe, these are the three largest markets for Pompe disease. We estimate that there is around 1,300 patients treated for Pompe in Europe, another couple of hundred in the U.K., and north of 800 patients in the U.S.

I think we’ve seen repeatedly through all the [indiscernible] 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, naïve patients for Pompe is 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, so we’ll be very much focused on ensuring that we have--we provide easy access to AT-GAA.

As Bradley alluded to, I think that the experience we’re seeing through the early access programs bodes well for the launch of AT-GAA..

Thank you. One moment for our next question. Our next comes from the line of Ritu Baral from TD Cowen..

Hey guys, this is Athena on for Ritu. Thanks for taking the question. Just another follow-up on the WuXi 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? Thank you..

Yes, thanks for the question, Athena. The important part of this inspection, so WuXi has been inspected, 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, and so we feel very confident at this point that we are on track there based on the outcome of the inspection.

I think at this point, we’re sort of all systems go and looking forward to getting through to the finalization of the review and heading towards approval and launch..

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

Hey, good morning guys. Thanks for taking our question. 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 the 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..

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 access more than anything else, so our goal is to get as many patients on therapy as quickly as possible, and we feel like taking price off the table, in other words focusing on the value of the medicine that it brings to patients and physicians versus the cost of the medicine, is the best way to ensure that you move through the reimbursement process as quickly as possible.

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, so we look forward to deploying the same strategy and again focusing on the value of the medicine versus the cost..

Thank you. One moment for our next question. Our next question comes from the line of Kristen Kluska from Cantor Fitzgerald..

Hi everyone, good morning. Thanks for taking my question. Wanted to ask what your latest thoughts are on how you might position AT-GAA relative to the benefits across respiratory outcomes and different measurements there.

I know you presented more data at Worlds this year, and in the past you’ve cited this as a really important end point considering it’s a leading cause of mortality for these patients. Thank you..

Yes, thanks Kristen. 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 Worlds with the extension data..

Yes, thanks Brad, and thanks Kristen for the question.

As we’ve seen in our clinical studies with AT-GAA, we see pulmonary function, which as you pointed out is one of the--basically the leading cause of mortality in Pompe, that patients that had been on standard of care ERTH saw quite a significant decline in their pulmonary function as they remained on treatment, whereas patients that switched to AT-GAA showed good stabilization on that pulmonary function.

Importantly, as we’ve now seen data in our extension studies from Phase I/II out to four years and from our PROPEL study out to two years, that stability seems to be maintained and durable over those additional extended time periods and trials.

We’re very excited about the potential ability to help stabilize that progressive decline in pulmonary function we see, and also importantly the other key arm of Pompe is on the motor function and muscle strength side of things.

There, we’re actually really excited to see some improvements, even, in patients which are not necessarily expected as patients switch to AT-GAA, so across those two key aspects of disease, we’re very excited by both the initial response to treatment, as seen with PROPEL, but then the continued durability of the response in the extension trials..

Thank you. One moment for our next question. Our next question comes from the line of Salveen Richter from Goldman Sachs..

Hi, this is Shrinatra [ph] on for Salveen. We have two questions. The first is how soon post approval can we expect the first patients to be treated with AT-GAA, and I’m referring to patients who are not on the early access program. The second is could you provide some color on what we can expect in the first few months of the launch? Thank you..

Yes, thanks for the question. In terms of first patients on therapy, there’s two different situations. You have the United States, of course, where you get approved and then you’re typically moving quickly to setting up infusions and--you know, getting prescriptions and then 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 first 90 days, and so that’s something we’ll focus on and we were able to execute that very successfully in the Galafold launch. We believe we’ll be able to do that here.

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 Germany, we do have an extended access program ongoing as well, and again that’s within that 30 to 90-day period, and then in the U.K. we will go through the reimbursement process, and again that’s where that focus on access is so important, 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. as well that are on label..

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

Thanks for taking my question. Can you talk about the recent feedback you’ve heard from payors on AT-GAA and what kind of comments do you hear on potential barriers on access or restrictions? Thanks..

Yes, thanks for the question. All of our market research from a payor 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 and if we have a parity or modest discount strategy, payors 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.

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 payors, we think this is going to be a very successful access strategy and we’re just eager to get through the process and provide this medicine to patients..

Thank you..

Thank you. One moment for our next question. Our next question comes from the line of Zhiqiang Shu with Berenberg..

Good morning. Thanks for taking the question. I wanted to just ask your [indiscernible] announced the expansion of the supply and manufacturing agreement with WuXi on its Ireland facility.

Can you talk about the transition plan from the China facility to the Ireland facility, and also do you expect the FDA inspection on the Ireland facility in due course? Thanks very much..

Yes, great question. 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.

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 facility to be licensed, and material 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. Will the FDA 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, a second site of manufacturing come online and one that’s geographically diversified as well. .

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

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

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