Amicus Therapeutics Inc

NASDAQ:FOLD

Earnings Call Transcript 2022-Q3

[Operator]

Good morning ladies and gentlemen and welcome to the Amicus Therapeutics third quarter 2022 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, Executive Director of Investor Relations. You may begin.

[Andrew Faughnan, Executive Director, Investor Relations]

Thank you Shannon. Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics third quarter 2022 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 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 quarterly report on Form 10-Q for the quarter ended September 30, 2022 filed this morning 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?

[Bradley Campbell, President, Chief Executive Officer]

Great, thank you Andrew, and welcome everybody to our third quarter 2022 results conference call. I’m really pleased today to review the continued progress made across our business this year, and as we did in this morning’s press release, let me highlight several key accomplishments for the quarter.

First and foremost, Galafold continues its strong performance and remains a cornerstone of our success. We continue to be very pleased with the uptake of Galafold globally despite significant foreign currency headwinds we’ve seen this year, and the first three quarters of the year represented 8% reported revenue growth or 16% on an operational basis.

In the third quarter, our growth and key performance indicators are meeting our objectives in all geographies, and as Sébastien will highlight, we continue to see strong trends in a number of our metrics, including new patient starts and in-person visits between our field team and our physicians, so we are reiterating our guidance of 15% to 20% growth in Galafold revenues at constant exchange rates.

We’re also pleased to announce that the Galafold intellectual property estate continues to grow this quarter. This year, we’ve strengthened our IP estate through the issuance of 19 new patents. There are now 46 orange book-listed issued patents related to Galafold in the United States, 30 of which provide protection to 2038 and beyond, importantly includes five composition of matter patents. We see this patent portfolio along with our orphan drug exclusivities providing broad and long-term intellectual property rights well into the late 2030s with this novel precision medicine.

As we have discussed throughout the year and as anticipated, we saw three abbreviated new drug application, or ANDA filers and have received their Paragraph 4 certifications. Given our strong and innovative intellectual property estate, we believe we are well positioned to address any potential generic challenges to our patents, and we announced this morning that later today, we intend to file infringement lawsuits against three ANDA filers who are requesting approval to market a generic version of Galafold. We intend to continue to protect and enforce our broad intellectual property rights and looking ahead, we expect continued growth for Galafold this year and remain confident that with our strong IP protection, it has a long runway well into the next decade.

Second, we continue to make progress on our global regulatory filings and commercial planning for AT-GAA, our novel next-generation therapy for Pompe disease. As we just announced last week, the U.S. Food and Drug Administration recently deferred action on the biologics licensing application, or BLA for cipaglucosidase alfa, the biological component of AT-GAA which we also call ATB200. The Agency cited that due to restrictions on travel related to COVID-19, it was unable to conduct the required inspection of the WuXi Biologics manufacturing site in China during the review cycle and is deferring action on the application until the inspection is complete.

That being said, the good news is that per FDA guidance, a deferral in such circumstances can only be given, quote, provided that no deficiencies have been identified and the application otherwise satisfies the requirements for approval, end quote, so as we’ve been saying, we believe this is a matter of when, not if AT-GAA is approved. Additionally, the company is now actively engaged in the FDA on this step and at the Agency’s direction, the company has requested a Type A meeting with the Office of Pharmaceutical Quality to discuss and agree upon plans and logistics for the pre-approval inspection.

We at Amicus, along with our manufacturing partner WuXi Biologics are ready for the inspection of the manufacturing facility in China, and as soon as we have more clarity, we will provide an update on when we might expect an approval in the United States. As a reminder, we continue to expect the two components of AT-GAA will be approved together.

Importantly, in Europe we are very far along now in the review by the EMA on the marketing authorization application for AT-GAA. To provide a little more color there, we are announcing today that we are included now on the agenda for an oral explanation in November and thus the CHMP opinion is expected at the December meeting, setting us up for a commercial launch in 2023. As a reminder, the EMA has indicated that it does not require an inspection of the WuXi manufacturing site as a condition of their approval.

We’re also extremely pleased with the level of interest and participation we’re seeing in our expanded access programs globally. We now have programs in place in the United States, United Kingdom, France, Germany and Japan, with a growing number of patients participating in each. In fact, across all of our ongoing clinical studies and access programs, there are now nearly 190 patients on AT-GAA today which we believe represents more than 5% of the total treated Pompe patients around the world. We expect this number to continue to grow as we approach approvals in the United States and Europe.

In anticipation of these approvals in the near term, our global launch plans continue to move ahead, including our pre-launch activities, targeted investments, and handful of additional personnel to support the launch, and our ongoing investments in building launch inventory. We are close now to reaching this much anticipated milestone of AT-GAA getting across the regulatory finish line and providing another treatment option for people living with Pompe disease both in the United States and in Europe, and with further regulatory applications planned in the months ahead.

Third, Amicus has maintained a strong financial position as we continue to execute on the global expansion of Galafold and prepare for the global launch of AT-GAA. Despite all the headwinds with FX and the delays with AT-GAA approval in the United States, we will continue on our path to profitability. Our goal is to achieve non-GAAP profitability in the second half of 2023. This is dependent on a number of factors, including the timing of approval and launch of AT-GAA, but based on the current operating plans and projections, we believe this will happen in the second half next year.

Additionally, today we entered into an at-the-market, or ATM equity offering of up to $250 million. We see this as a good financial housekeeping measure and we have no present intention to use the ATM this year. In 2023 and beyond, we will be judicious about any use of the ATM and any proceeds will go towards ensuring continued commercial expansion of Galafold and maximizing anticipated launches of AT-GAA.

I’ll emphasize that we’re sharply focused on our three primary objectives: number one, continuing to advance Galafold to as many patients in as many geographies as possible; number two, securing approvals for and launch of AT-GAA globally; and number three, ensuring the financial strength of Amicus.

On Slide 5, we see that we’re well on our way towards achieving our key strategic priorities for this year, including, number one, continuing to drive Galafold to more people living with Fabry disease with amenable variants in existing and new markets. As I mentioned, we’re on track to achieve our guidance of double-digit global product revenue growth of 15% to 20% at constant exchange rates. This reflects the strong momentum and demand behind this precision medicine globally.

We remain steadfast in our commitment to advancing AT-GAA regulatory approvals and the anticipated launch of AT-GAA, leveraging our seasonal global commercial, medical and market access teams and our experience across all areas needed for a successful and effective drug launch. We are fully prepared for and anticipate a successful launch of AT-GAA.

We continue to judiciously invest in the advancement of our best-in-class next generation genetic medicines and capabilities as well as our next generation chaperone for Fabry disease, and again we’ll continue to maintain a strong financial position as we carefully manage our expenses and our investments in the business.

With that, let me now hand the call over to Sébastien Martel, our Chief Business Officer, who will give further highlights on the Galafold performance for the quarter. Sébastien?

[Sébastien Martel, Chief Business Officer]

Thank you Bradley. Good morning to everyone on the call. I will start by providing you with more details on our Galafold performance for the quarter.

On Slide 7, for the third quarter of 2022, Galafold reported revenue reached $81.6 million driven by strong new patient accruals partly offset by significant foreign currency headwinds. The geographic breakdown of revenue during the quarter consisted of $51 million or 63% of revenue generated outside of the U.S., and the remaining $30 million or 37% coming from within the U.S. When ignoring FX impacts, this is in line with the two-thirds to one-third split that we expect as we continue to grow both parts of the business. We’re pleased to see continued strong patient growth in countries like the USA, Japan, Canada, Spain, Portugal, and Poland, just to name a few.

Turning to Slide 8, our results in the first nine months of the year highlights the strength of our global commercial efforts. The business continues to be incredibly resilient with patients added in all major markets and an operational growth rate of 16.2% over the same period in 2021 at constant exchange rates. The negative impact from foreign currency was 8.3% in the period. As a result, Galafold reported revenue growth was 7.9% in the first three quarters of the year.

To add a bit of color on our geographical performance, in the first nine months of the year our sales in the U.S. grew 16.8% while our ex-U.S. sales grew 16% at constant exchange rates. On a year-to-year basis, Galafold continues to be the fastest growing product in 2022 for Fabry disease globally and the greatest contributor to the global Fabry market growth. I’m pleased to report that our monthly net patient trends continue to show positive signals; indeed, the three-month trend is the highest in the last two years, and if you look at the growth in net patients on Galafold globally, which is perhaps the truest measure of the underlying business, we see greater than 19% growth in patients on Galafold at the end of Q3 this year versus the same period last year, all indications of the continued and growing demand for Galafold.

We ended the third quarter with over half of the global market share of treated amenable patients, and while the global mix remains about 55% switch and 45% naïve in many geographies, we’re seeing a stronger uptake in naïve populations, so while we are achieving high market shares in countries where we’ve been approved the longest, there’s plenty of opportunity still to continue to switch patients over to Galafold and continue to grow the market as we penetrate into the diagnosed untreated as well as the newly diagnosed segments. All of that is underpinned by the impressive compliance and adherence rates that we continue to see exceeding 90%, reiterating our belief that those patients who go on Galafold generally stay on Galafold.

We continue to expect non-linear quarterly growth due to uneven ordering patterns and FX fluctuations. Importantly, the value of Galafold continues to be recognized by payors as we have a very strong track record of successfully negotiating and renegotiating reimbursement outside of the U.S. Our relentless commitment remains on ensuring access to Galafold for anyone who needs it.

On Slide 9, what we’ve seen so far this year is that Galafold uptake continues to track very well and we’re seeing growth across all our major markets as well as most of our smaller markets while on track to achieve our full year revenue guidance of 15% to 20% growth at constant exchange rates. Altogether, we view this as a great place to be three quarters of the way through the year.

Moving to Slide 10, we know that Galafold has the potential to surpass $500 million in annual revenue over the next few years through three key growth drivers: first, continuing to penetrate into existing markets; second, expanding into new geographies; and third, broadening the label. I’m pleased to share that we’re making continued progress on expanding into new markets. Just to name a few examples, we recently received marketing authorization in Turkey. We submitted a marketing authorization application in Hong Kong earlier this year and we’re about to submit in New Zealand. We’ve also successful renegotiated pricing and reimbursement agreements recently for Galafold in Poland and Spain.

In the longer term, we continue to see significant growth in the Fabry market globally driven by diagnosis of patients through a variety of measures, including higher screenings, newborn screenings, and other diagnostic initiatives which we continue to support and invest in as well.

Finally, we have orphan exclusivity in the U.S. and Europe in addition to our now 45 orange book-listed patents that give us IP coverage into the late 2030s, 30 of which provide protection through 2038 and beyond, including five composition of matter patents, all of which gives us opportunity to provide access to Galafold globally for a long time to come.

Moving onto AT-GAA on Slide 11, we outline launch preparations as we are poised for another successful product launch. Unlike when we launched Galafold, when we were hiring and building the commercial infrastructure from scratch, we now have a presence in over 40 countries around the world, including all the major markets. That same team will be largely the one involved in launching AT-GAA with only a handful of new FTEs needed. We have experience across all areas that are needed for a successful drug launch in regulatory, commercial, supply chain, experience with payors reimbursement and access, and in addition and perhaps most importantly, we have key relationships with physicians. We’re very confident in our world-class organization and we can leverage their experience and relationships to deliver AT-GAA to people living with Pompe disease around the world.

From the team, the medical education, the published Phase III data in the highly regarded Lancet Neurology Journal, our experience with reimbursement and access around the world, and again all the strategy planning that we’re doing together with building inventory with our partners at WuXi Biologics, we believe we’re in a very strong position for a second successful launch for Amicus.

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

[Jeff Castelli, Chief Development Officer]

Thank you Sébastien, and good morning everyone. On Slide 13, we’ll start with our AT-GAA program.

Pompe is a severe and fatal neuromuscular disease and one of the most prevalent lysosomal disorders, and we recognize that Pompe poses a range of health challenges for people affected by the disease and having therapeutic choices is crucial. Multiple publications and natural history studies highlight the initial benefits of treatment generally being followed by continued long term decline for many individuals.

On Slide 14, we present a summary of the primary and key secondary end points from our Phase III study. As a reminder, PROPEL was a double-blind randomized study assessing the efficacy and safety of AT-GAA in adult treatment naïve and ERT-experienced participants with late onset Pompe disease, or LOPD, against the approved therapy, avalglucosidase alfa. PROPEL is the only controlled clinical trial to date that included both ERT-experienced patients and ERT-naïve, with the experience patients representing one of the sets of patients with the greatest clinical unmet need. End points across motor function, muscle strength, pulmonary function, patient reported outcomes and biomarkers, including the two most recognized end points in Pompe - six-minute walk distance and FVC, shown here on the slide, favored AT-GAA over avalglucosidase alfa in the overall population. We believe this consistency of effect across the key disease manifestations of Pompe illustrates the potential impact of AT-GAA for patients.

Additionally, in the ERT-experienced population, where 95 participants were on the standard of care for more than 7.5 years on average, generally associated with continued progression for most patients at this point in treatment, we actually saw an increase in six-minute walk distance and stabilization in FVC after switching to AT-GAA, which achieved nominal statistical superiority on both end points versus the current treatment and showed a clinically meaningful outcome never before seen in this population.

Moving to Slide 15, as part of the growing body of evidence supporting AT-GAA, the Amicus team presented additional positive long-term data from the Phase I/II study of AT-GAA at the 2022 World Muscle Society Conference. As seen here on this slide, these latest data continue to represent very meaningful and durable improvements in functional outcomes as well as persistent reductions in key biomarkers of muscle damage and disease substrate. Compared with what is known about the natural history of both untreated and ERT-experienced Pompe patients, the observed durable improvements give great hope that AT-GAA has the potential to become the new global standard of care for people living with LOPD.

On Slide 16, we show key results from an indirect treatment comparison of Pompe ERTs that was also recently presented at the World Muscle Society Conference. This analysis used published data from the Phase I/II and Phase III studies for the three ERTs currently available or under regulatory review for early onset Pompe. A multi-level of network meta regression accounting for effects of study-level covariance was performed using individual patient-level data from the PROPEL study and available aggregate data from the other studies. The four plots shown here on the slide estimated relative effects and 95% credible intervals of each treatment comparison and the base case analysis in which all covariance were set for the target population of the PROPEL trial. These results suggest cipaglucosidase plus miglustat may potentially have a differentiated clinical profile versus the other ERTs, particularly for individuals with some level of previous ERT treatment.

On Slide 17, we have highlighted key updates on the AT-GAA program. First on the regulatory progress, as shared previously, last year the U.S. FDA accepted for review the BLA for cipaglucosidase alfa and the NDA for miglustat, the two components of AT-GAA. As Bradley summarized earlier, following the recent FDA deferred action which was due solely to COVID-related inspection delays, the company is now actively engaged with the FDA and at the Agency’s direction has requested a Type A meeting with the Office of Pharmaceutical Quality to develop plans and logistics for the pre-approval inspection. We continue to expect that the two components of AT-GAA will be approved together and once we have more clarity, we’ll be able to give more color on estimated approval timing.

We have also shared previously that the MAA has been submitted to the European Medicines Agency and is now in the later stages of review. Following an upcoming oral explanation in November, the CHMP opinion is expected at the December meeting. Of note, the EMA has indicated in writing that based on the extensive and prior manufacturing inspections of the WuXi facility, that an inspection is not required prior to AT-GAA approval.

We now have multiple expanded access programs in place, including in the U.S., the U.K., Germany, France, Japan, and other countries. This includes the EAMS framework, of which we had previously announced that AT-GAA was granted a positive scientific opinion through the Early Access to Medicine Scheme by the U.K.’s MHRA. We are seeing significant enthusiasm for AT-GAA under the EAMS mechanism with multiple physicians having requested access across the leading Pompe centers in the U.K. and dozens of patients now receiving AT-GAA through this program. With this growth in our access programs, as Bradley noted, we are pleased to report that approximately 190 patients worldwide are now being treated with AT-GAA across our clinical extension study and expanded access programs.

For the younger Pompe community, we continue to enroll the ongoing open label study in children up to 18 years of age living with LOPD and expect to expand into patients with infantile onset Pompe disease later this year. Importantly, in response to the many requests for treatment that we continue to receive for children living with LOPD and IOPD, our expanded access programs, continue to increase.

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

[Daphne Quimi, Chief Financial Officer]

Thank you Jeff, and good morning everyone. Our financial overview begins on Slide 20 with an overview of our third quarter revenue performance and FX impacts.

For the third quarter, we achieved total revenue of $81.7 million, which is a 3% increase over the same period in 2021. This includes operational revenue growth of 14% offset by a negative currency impact of 11%. Given a majority of Galafold revenue is generated outside the U.S., we see significant FX exposure to our reported revenue numbers. The euro, British pound, and Japanese yen are the currencies we are most exposed to, and on a year-to-date basis these have declined 14%, 18% and 20% respectively. Applying average October 2022 exchange rates, the FX impact on 2022 full year Galafold reported sales would be a negative impact of approximately 9% or $28.5 million.

Slide 21 outlines our income statement for the third quarter ending September 30, 2022. Cost of goods sold as a percentage of net sales was 16% in the quarter as compared to 15% for the prior year period. Total GAAP operating expenses were $102.1 million in the third quarter as compared to $110.2 million in the third quarter of 2021. The decrease reflects the reprioritization of the gene therapy portfolio.

On a non-GAAP basis, total operating expenses were $85.5 million in the third quarter as compared to $93.6 million in the third quarter of 2021. We define non-GAAP operating expense as research and development, 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 third quarter of 2022 was $33.3 million or $0.12 per share as compared to a net loss of $50.3 million or $0.19 per share for the prior year period. Driven by the revenue growth of Galafold and expense management, we continue to make progress towards our path to profitability in the second half of next year. At September 30, 2022, we had approximately 281 million shares outstanding.

We are updating our full year 2022 non-GAAP operating expense guidance from $470 million to $485 million to $430 million to $440 million, driven by prudent expense management while maintaining AT-GAA manufacturing and pre-launch activities. Importantly, in 2023 and beyond we continue to expect non-GAAP operating expense levels to decline below levels we saw in 2021.

Turning now to Slide 22, 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 approval and launch of AT-GAA. We will focus the majority of our investments on our core value driving franchises in Fabry disease and Pompe disease by continuing to deliver on the global growth of Galafold, securing approval and launching AT-GAA globally, as well as driving efficiencies, cost savings and careful expense management.

As a good financial housekeeping measure, today we entered into an at-the-market, or ATM equity program of up to $250 million. We have no current intention to use the ATM this year. In 2023 and beyond, we will be strategic and judicious about any use of the ATM.

A few comments about our cash position and 2022 financial guidance. Cash, cash equivalents and marketable securities were $354.7 million at September 30, 2022 compared to $482.5 million at December 31, 2021. Our full year Galafold revenue guidance is $350 million to $365 million at constant foreign currency exchange rates, in addition to our non-GAAP operating expense guidance of $430 million to $440 million.

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

[Bradley Campbell, President, Chief Executive Officer]

Great, thanks Daphne, Jeff, Sébastien for highlighting all the great progress of the quarter. Thanks to everybody at Amicus and all of our employees around the world, who work so tirelessly for people living with rare diseases.

With that, Operator, we can now open the call to questions.

[Operator]

[Operator instructions]

Your first question comes from the line of Ritu Baral with Cowen. Your line is now open.

[Ritu Baral, Cowen]

Good morning guys. Thanks for taking the question.

[Bradley Campbell, President, Chief Executive Officer]

Good morning Ritu.

[Ritu Baral, Cowen]

Good morning. It’s basically on the timeline of AT-GAA approval. I guess you outlined today that you’re going to request a Type A meeting.

[Bradley Campbell, President, Chief Executive Officer]

Correct.

[Ritu Baral, Cowen]

Can you tell us when you plan on requesting the Type A meeting? Do you need to put a briefing book like usual together in advance, or can this be done more ad hoc, and what those timelines might be? Then I have a quick follow-up.

[Bradley Campbell, President, Chief Executive Officer]

Sure, great questions. The request actually has already gone in, so that’s already been submitted, and that was at the direction of the Agency. I think that’s important--this is really the appropriate vehicle for us to have formal conversations, and we were careful to highlight it’s with the Office of Pharmaceutical Quality. We had talked when we put the press release out last week about why we felt confident this was really an active step to get to a solution around conducting the inspection. I think having this formal vehicle really helps put a fine point on that.

In terms of when we might have an update, and then I’ll come to your next question, in terms of when we might have an update, we still hope to have an update in the coming weeks. As I mentioned last week, as soon as we have more clarity, we can provide some expectations on when we might see an approval; but again, we think this is a very important step that we’re having this discussion, and the request indeed already went into the agency.

[Ritu Baral, Cowen]

Got it, and my quick follow-up is on Europe. Have you responded to the 180 day questions, and were there any surprises or differential content from U.S. review issues for the EMA review? Thanks.

[Bradley Campbell, President, Chief Executive Officer]

Yes, thanks Ritu. Jeff, do you want to talk to the progress we’re making in Europe and where we are with the oral explanation and the expectation for December?

[Jeff Castelli, Chief Development Officer]

Yes, thanks Brad, and thanks Ritu.

We are in very late stage review with CHMP. There have been no, I would say, surprises in terms of the remaining topics being discussed. Well have explanation coming up here shortly and then we still have a lot of confidence that we have an approvable filing, and we expect that opinion at the December meeting, but very consistent so far with a lot of the U.S. reviewing questions.

[Ritu Baral, Cowen]

Great, thanks.

[Operator]

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

[Anupam Rama, JP Morgan]

Hey guys, thanks so much for taking the questions. If I remember correctly, you guys did provide sort of forward year guidance at an investor conference in January. With the CHMP decision on the horizon, would you be in a place to give some guidance and metrics around AT-GAA, or should we just be thinking it will be focused on Galafold and expenses? Thanks so much.

[Bradley Campbell, President, Chief Executive Officer]

Yes, thanks Anupam. It’s a great question. I think we will be, as you said, able to give some color on Galafold and expenses. Let us get a little bit more certainty around the timing of the approvals, which as you said, we should have more clarity here relatively soon, especially in Europe and hopefully in the U.S. as well. We’ll certainly give some metrics to follow in terms of the launch and the launch progress, but stay tuned on exactly what those look like. We’ll do our best to provide the street with some sense for how to make sure we’re tracking the progress of the launch.

[Anupam Rama, JP Morgan]

Thanks so much for taking our question.

[Bradley Campbell, President, Chief Executive Officer]

Thanks Anupam.

[Operator]

Thank you. Our next question comes from the line of Joseph Schwartz with SVB Securities. Your line is now open.

[Joseph Schwartz, SVB Securities]

Hi, thanks so much. I guess I’ll ask on the inspection situation as well. What is your understanding for why the FDA has not been receptive to a hybrid inspection to this point? Is an in-person inspection the only path forward, and what plans and logistics will you propose to the FDA in order to get WuXi, the process there inspected? Do you have any reason to believe that they’ll be receptive to any particular solutions?

[Bradley Campbell, President, Chief Executive Officer]

Yes, thanks Joe. A couple things. First of all, I can’t speak on behalf of the Agency, but we have said all along that despite what we believe are multiple types of inspections the Agency could use to satisfy this PLI for AT-GAA, they have always signaled to us that they would like to inspect in person. Now, whether that’s in person from Maryland or in person from a hybrid team, I don’t know, but they have always said that they’d like to inspect in person.

Specifically, what we’re working on now is exactly that, which is coming up with logistics and a plan to enable the agency to inspect the WuXi facility in China in person, and I think the biggest difference here versus some of the commentary we’ve given over the course of the summer as we approached this latest PDUFA date is that now we have active dialog and now formal dialog through this Type A meeting request, and again that was at the Agency’s direction.

I think what’s changed here is, number one, we are actively discussing the logistics, in particular how to navigate the COVID situation on the ground in China, and that it was at the Agency’s request that we have this formal meeting and they are now actively engaged in that dialog, so we feel a high degree of confidence that we now have an engaged discussion, that we have specifics of a plan and logistics that are being discussed, and our hope here again is that in the coming weeks, we can finalize that and then we can provide more clarity externally on timing for when an approval might happen.

[Joseph Schwartz, SVB Securities]

Okay, thanks. Then what are you able to do during this time while you’re waiting for FDA approval to get in front of Pompe physicians and patients in a compliant manner, in order to ensure that you can really hit the ground running and not lose too much ground to competition once you’re approved in the U.S.?

[Bradley Campbell, President, Chief Executive Officer]

Yes, I think what Jeff highlighted on the call is really the most important way to do that is really through medical education at ongoing medical conferences. We have a great presence at the World Muscle Society, presented a host of new data there.

I don’t know, Jeff, do you want to remind us just at a high level, the Pompe data in particular that was presented there, and then kind of a general medical education publication posted abstract, etc.?

[Jeff Castelli, Chief Development Officer]

Yes, thanks Brad, and hi, Joe. As I summarized briefly on the call, we had a very active presence at World Muscle, we presented four year long term data from the Phase I/II trial. We presented some new indirect treatment comparisons from available data across different ERT products, and we continue to keep planning for upcoming conferences - you know, World LVN and then other meetings early next year, to continue to have lots of new evidence presented and data presented on AT-GAA.

Of course, we’re having appropriate disease education. Our teams, both the sales team and medical team are all ready to go with materials and messages, so we can do everything that you typically would do prior to an approval, and we’re going to continue to get ready for the launch. We are ready in many ways, but we’ll be even more ready with the extra time.

[Bradley Campbell, President, Chief Executive Officer]

The only other thing I would add there, Joe, is I think this is a highly anticipated development program and regulatory process, and so the good news here is I think the community - physicians, patients, etc. are well aware that AT-GAA is going through this process, and so I think there’s a lot of anticipation as well in addition to the medical education activities that Jeff highlighted.

[Joseph Schwartz, SVB Securities]

Thanks for taking my questions.

[Bradley Campbell, President, Chief Executive Officer]

Thanks Joe.

[Operator]

Thank you. Our next question comes from the line of Tazeen Ahmad with Bank of America. Your line is now open.

[Tazeen Ahmad, Bank of America]

Good morning, thanks so much for taking my question.

Just want to get your thoughts. I know you’re not talking about the specifics of what a label language could look like, but in the event that you do get the specification about the switch data patients, how do you think that would impact your ability to gain traction against your key competitor, who’s been marketing really without that language, and longer term, how should we think about the split between U.S. and ex-U.S. sales for AT-GAA? Thank you.

[Bradley Campbell, President, Chief Executive Officer]

Sure. Maybe I’ll turn to Sébastien in a moment on the split between the two--between the geographies in terms of the sales, but in terms of the first question around the ability for us to be successful with AT-GAA and what’s some of the label scenarios might suggest, I think the most important thing is the differentiated data set that Jeff highlighted on the call. We are the only manufacturer to have studied in an active control setting that switch population, and as we’ve said before, that makes up probably 90% of the opportunity over the next few years, so I think that differentiated data set and, hopefully over time, the long term data that we saw from the Phase I/II study, as we build that body of evidence, I think that gives us a really differentiated product especially in that switch population for us to build on.

The other thing I would remind people is that while I know we’ve spent a lot of time around what could be scenarios in the United States, obviously we have multiple jurisdiction that we’re seeking approval in, and just because one jurisdiction has one label, doesn’t mean all jurisdictions will have that label, so I think it will be really important to see how that evolves over the--as we see some of these anticipated approvals in the coming months, so I think people should watch out for that as well.

Then the last thing I’ll add and then turn it over to Sébastien, again we do intend to continue to build the body of evidence in many Pompe populations, and so there may be an opportunity down the road, of course, if for some reason you have one specific population in your label to add to that over time and potentially expand those labels.

With that, maybe Sébastien, just talk a little bit about the distribution of the opportunity between the U.S. and some of the other major markets around the world.

[Sébastien Martel, Chief Business Officer]

Yes, thank you Bradley. There are a couple of ways to look at that. You can either look at it from a patient standpoint or look at it from a sales standpoint. I’ll start with the breakdown of sales across the main geographies. The U.S. represents about 40% of sales, Europe about 35% of sales, and rest of world roughly 25%.

Now, the picture is slightly different when you look at actual patients, mostly because of differences in pricing across the [indiscernible]. Europe accounts for more than 40% of patients currently treated with [indiscernible] while the U.S. represented around 25% of patients only, so Europe represents a very large opportunity from a patient percentage standpoint.

[Bradley Campbell, President, Chief Executive Officer]

Great, thanks Sébastien.

[Operator]

Thank you. Our next question comes from the line of Ellie Merle with UBS. Your line is now open.

[Sarah, UBS]

Hi, this is Sarah on for Ellie. Thanks for taking our question.

I think two quick ones from us. In terms of the Type A meeting with the FDA, I guess anything we know historically about--you know, now that the request is submitted, how long it may take to start conversations and the duration, maybe, of that process? Then looking at ex-U.S. growth in Galafold, what are the key regions that you see driving growth in ’23 and sort of maybe cadence of expanding geographically over 2023 as well? Thanks.

[Bradley Campbell, President, Chief Executive Officer]

Sure, thanks Sarah. Your first question as it relates to the Type A meeting, as I mentioned, the request has already gone in, so that process has already started. Our hope is that--you know, technically there’s a formal clock that we can meet with them as soon as possible, and again, as soon as we have more color there, we’ll provide that update. In terms of how long the process will take, again our hope is that it can be fairly expedient. Let us get through those discussions and, as soon as we can, we’ll provide more color.

Then on the ex-U.S. growth opportunities for Galafold, one thing I’ll just say at a high level, then maybe Sébastien, you can provide some specific color, Sébastien talked on the call how Galafold is actually the largest contributor to Fabry revenue growth around the world, which I think is pretty incredible given the fact that we’re restricted to the amendable population. The other thing I’ll say is that as we look at the numbers and as you look at the metrics we’ve given, what we’re seeing is really a continued building of momentum coming out of what was probably the depths of the COVID impact, and so as Sébastien highlighted on the call, seeing the three-month net new patient numbers being the largest they’ve been for the last two years, I think that gives us great confidence that we are seeing resumed growth and momentum, which is so important for that key franchise.

But Sébastien, maybe talk a little bit about what are the geographic growth drivers for next year for Galafold.

[Sébastien Martel, Chief Business Officer]

Yes, so as I highlighted earlier, what we’ve seen this year is actually pretty much the same kind of growth rates in the U.S. and ex-U.S. We were at 16.8% in the U.S. and 16% ex-U.S. When you start looking at specific countries, we continue to see a double-digit growth rate in countries where we have been launched for the longest, and that’s explaining--in those markets where we’ve been longest in the market, that’s explained by the growing participation we have to put naïve patients on treatment. There’s also countries where we’ve launched more recently, and there you see a much larger proportion of switches initially, but overall we continue to see double growth rates in most markets where we’re currently launched.

We are seeing very strong growth in some rest of world markets, so if I take some LatAm markets, for example - Colombia, Brazil, in eastern Europe the Polish market has also seen great expansion for Galafold and as I highlighted, we continue to launch in, or to register at least in markets where Galafold has not been launched yet, so I mentioned specifically Turkey. Turkey is quite a sizeable market from a total number of patients. The actual number of treated patients in Turkey is close to any of each of the European top five markets. There are still a small number of markets where we’ve got approval and are working through the pricing and reimbursement process, so these will be new launches in 2023. But again, the core markets, and even I would say the top 10 markets continue to show very significant growth.

[Bradley Campbell, President, Chief Executive Officer]

Great, thanks Sébastien.

[Operator]

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

[Dae Gon Ha, Stifel]

Hey, good morning guys. Thanks for taking the question. I’ll stick with one.

Just going back to AT-GAA, there have been some rumblings on unverified reports of China maybe reopening, given the impact of the tech sector and that economy. I guess if you can speak to two different scenarios, one is what’s your guess as to the timeline towards them reopening, and two, in a bear case where they don’t, what’s the real hypothetical here given that the U.S. Chamber of Commerce was still able to inspect WuXi in the whole zero COVID policy era, what should we be thinking about in that case? Thanks so much.

[Bradley Campbell, President, Chief Executive Officer]

Yes, thanks Dae Gon. Two things, so first of all, I think we’re hearing the same thing you’re hearing, which is perhaps at the margins, things are getting better, or there’s still some hope that there might be a very different or more permissive policy around COVID. That being said, the plan we’re working on right now actually does not require any change to the official COVID policy, so we’re working on plans with the Agency that would allow them to conduct the inspection in the current environment. Maybe that is the bear case, but I think we’re being conservative there. Again, the intent is that this is something that can happen within a finite amount of time.

I know that the key question is exactly what that amount of time looks like, and I wish we had more color there, but we are actively working on it and, again, we feel like in the coming weeks, we’ll have some more line of sight there and then we can provide a better update. But again, this is assuming that the current COVID travel and quarantine restrictions remain in place and we’re working to execute on a plan that will allow the Agency to inspect in those conditions.

[Dae Gon Ha, Stifel]

Great, thanks so much.

[Bradley Campbell, President, Chief Executive Officer]

Thanks.

[Operator]

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

[Salveen Richter, Goldman Sachs]

Good morning, thanks for taking my question. I know with regard to the launch around Pompe, there was a question about cadence, but could you speak to how you think the launch will play out in terms of the competitive dynamics with Sanofi and how you think patients will be triaged between the drugs?

[Bradley Campbell, President, Chief Executive Officer]

Yes, so first and foremost, I think from a European perspective, as Sanofi went through the review process and the appeal process around the new active substance, I think that significantly shortened the time between their launch and ours, so I think that gives us, I think, good confidence that effectively we’ll be launching roughly contemporaneously based on the timelines that we’re on, so that, I think is a very different scenario.

As it relates to the U.S., as we’ve mentioned, I think what’s important about AT-GAA is we have a very differentiated data set, and if you look at a number of the market research polls and a number of the sell side research that’s been published out there, I think you do see an alignment around the indicated--or sorry, the populations that were studied, and so some alignment around AT-GAA in the switch population and the other products in the naïve population, so I think that data set that Jeff highlighted on the call today is really the key for us, that we can show an improvement in patients who switch from enzyme replacement therapy on both six-minute walk and forced vital capacity, and we think those are the data that are really going to be compelling for physicians and look forward to having the opportunity to get out there and promote the product on the other side of an approval.

[Salveen Richter, Goldman Sachs]

Thank you.

[Operator]

Thank you. Our next question comes from the line of Yun Zhong with BTIG. Your line is now open.

Yun Zhong, your line is open. Please check your mute button.

[Yun Zhong, BTIG]

Hi, thank you. Sorry, I didn’t hear my name. Good morning, thank you very much for taking the question.

My question is on AT-GAA. Can you remind us if the launch in Europe is going to be supported by the Ireland site solely, or is that going to be supported the WuXi site as well? Also, what was your plan in terms of how you were going to coordinate between the two sites, and given that you had this issue on inspection, has that changed your plan in terms of how you’re going to coordinate between the two sites going forward?

[Bradley Campbell, President, Chief Executive Officer]

Yes, great question, so just as a reminder, as you suggested, we do have the current manufacturing facility in WuXi City in China, and that’s where we’ve been manufacturing ATP200, which is the biologic, throughout the course of the development. That will support the initial launch in both the United States and Europe; however, WuXi has also now completed the build-out and we’re now actually in engineering runs for manufacturing of ATP200 at a site in Dundalk, Ireland. That site has actually more capacity than the site in China, and so our anticipation is that material from that site will enter into the commercial supply chain sometime in the ’24 timeframe, and that was always the intention. But once that site comes onboard, it’s likely that it will supply the majority of product for both Europe and the U.S., and rest of world for that matter, with likely a minority of product coming out of China.

We are not too far away here from having dual site manufacturing, which is critically important, but also increasing the capacity, and the intention was for that capacity to come online into the commercial supply chain when you’re starting to really get into the meat of the global launch expansion, which would again be in kind of that ’24 timeline. Hopefully that gives some clarity in terms of the plan there, and it hasn’t really changed in the context of the situation right now with the Agency and the inspection.

[Yun Zhong, BTIG]

Great, thank you.

[Operator]

Thank you. Our next question comes from the line of Gil Blum with Needham & Company. Your line is now open.

[Gil Blum, Needham & Co.]

Hi, good morning everyone. Congrats on your progress.

I have a quick one. Do you think that any of those 190 patients on AT-GAA could potentially convert to commercial product once it’s approved? Thank you.

[Bradley Campbell, President, Chief Executive Officer]

Yes, it’s a great question, and the answer very much is yes. As soon as we get regulatory approvals, then we look to convert our clinical trial or expanded access patients as quickly as possible. What we’ve given is just kind of rough numbers in terms of the breakout of those patients, so probably about half of the 190 are in Europe with a big portion of those coming from the U.K., Germany, and the EU5. The U.K. with the EAMS program is now the largest number of patients on AT-GAA today, and then likewise in the United States we have said that 20 or so patients in the U.S. are in our clinical trials and other ongoing access programs, and so again there too, that would be a population we would look to switch very quickly.

With Galafold, the goal was to be able to switch patients from either development drug or expanded access drug within 90 days, and we would have a similar goal here.

[Gil Blum, Needham & Co.]

Thank you.

[Operator]

Thank you. That was your last question. This concludes today’s conference call. Thank you and have a great day.