Amicus Therapeutics Inc

NASDAQ:FOLD

Earnings Call Transcript 2021-Q3

[Operator]

Good morning, ladies and gentlemen. Welcome to the Amicus Therapeutics’ Third Quarter 2021 Fiscal Results Conference Call and Webcast. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Faughnan, Head of Investor Relations. You may begin.

[Andrew Faughnan, Head-Investor Relations]

Thank you, operator. And good morning, everyone. Thank you for joining our conference call to discuss Amicus Therapeutics' third quarter 2021 financial results and corporate highlights.

Speaking on today's call, we have John Crowley, Chairman and Chief Executive Officer; Bradley Campbell, President and Chief Operating Officer; Daphne Quimi, Chief Financial Officer; and Dr. Jeff Castelli, Chief Development Officer. Joining for Q&A, we'll have Dr. Mitchell Goldman, Chief Medical Officer; and Sébastien Martel, Chief Business Officer joining.

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 should be according 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 risks and uncertainties that may impact them, we refer you to the forward-looking statements and Risk Factors section on our annual report on Form 10-K for the year ended December 31, 2020, and the quarterly report on Form-10Q for the quarter ended September 30, 2021 to be filed later today with the Securities and Exchange Commission.

At this time, it is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer. John?

[John Crowley, Chairman and Chief Executive Officer]

Great. Thank you, everyone. Good morning. And welcome to our third quarter 2021 results conference call. I am pleased to report that the third quarter reflected broad execution across our business as we remain on track to achieve our key strategic priorities for the year.

As we did in this morning's release, I'd like to go ahead and highlight several key updates. First Galafold continues its strong performance and remains the cornerstone of our success with $79.5 million in third quarter revenue. We are very pleased with the continued momentum of Galafold globally as we added new patients from both switch and naïve populations throughout the quarter.

Second, we continue to make tremendous progress on our global regulatory filings for AT-GAA, our novel next-generation therapy for Pompe disease. In September, the U.S. Food and Drug Administration accepted the review – accepted for review the biologics license application for cipaglucosidase alfa and the new drug application for miglustat, the two components of at AT-GAA. The U.S. review is underway.

The FDA has set a PDUFA action date of May 29, 2022 for the NDA related to the enzyme stabilizer, in July 29, 2022 for the BLA of the biologic. The two regulatory filings, significantly cross reference each other, and we expect that they will be reviewed together.

In the European Union, following our previously announced positive repertoire and co- repertoire meeting, I am pleased to share for the first time this morning that the marketing authorization applications or MAA for AT-GAA have been submitted to the European Medicines Agency. We are now closer to having another potential treatment option for people living with Pompe disease, both in the United States and in Europe with further regulatory applications planned in the months ahead. We are confident in the potential benefits of at AT-GAA and what it can bring to people living with Pompe disease around the world. And we look forward to working with regulators to get this important medicine, to as many patients as quickly as possible.

On the gene therapy front, we continue to make excellent progress in advancing and developing the industry's leading portfolio of next-generation gene therapies for people living with rare genetic diseases. Our teams are preparing and making great progress toward the business combination in which the Amicus gene therapy business will be acquired by ARYA IV, a special purpose acquisition company. Through this business combination, the Amicus gene therapy program technologies, intellectual property, key personnel and relationships will form a new company Caritas Therapeutics. We strongly believe that separating our business into two, highly focused, standalone companies is the best way to unlock value for Amicus shareholders and to properly fund and focus our gene therapy efforts.

In a single stroke, we will have created what will be, what we believe will be one of the world's preeminent, next generation, genetic medicine in gene therapy companies in Caritas, while significantly strengthening Amicus for its future.

Caritas, which is the Latin word for compassion will launched with approximately $400 million in capital. Amicus will become the largest shareholder in Caritas with a 36% ownership, stake and retain co-development and commercialization rights to the Fabry and Pompe gene therapy programs, as well as negotiation rights on select future muscular dystrophy programs.

As we disclosed at the time of announcement, I will lead Caritas as its Chairman and CEO, while remaining the Chairman of Emeritus and Chief Strategic Advisor for Amicus. In this Amicus role, I will work closely with Bradley and the Board of Directors at Amicus to ensure that we get AT-GAA approved and launched in all the major regions around the world, as well as to support major strategic initiatives. Bradley Campbell will succeed me at Amicus as Chief Executive Officer.

The Caritas form S4 is now filed with the Securities and Exchange Commission. And the launch of Caritas Therapeutics is expected in late 2021 or early 2022, dependent on customary closing conditions.

I'd also like to provide an update to our CLN6 Batten gene therapy program. We have very recently learned of the loss of two children with CLN6 in this study, during the long-term follow-up extension period of this study. As deemed by the investigator, the two participants passed away from disease-related complications, unrelated to this study drug. We will, of course continue to assess the data in this extension study. Just as a further reminder of the cruel nature of this disease and the urgency for therapies that may extend and enhance the lives of these children.

And finally, with the current plan, which includes the intended Caritas transaction, the Amicus cash position is sufficient to achieve self-sustainability and profitability in 2023. During the quarter, the balance sheet of Amicus was strengthened through a $200 million private investment from a syndicate of leading biotechnology investors. Our continued revenue growth, prudent expense management and growth potential has allowed us to reach this important milestone as we continue to realize our vision of delivering groundbreaking and new medicines for people living with rare diseases.

So, with that introduction, let me go ahead now and hand the hall over to Bradley Campbell, our President and Chief Operating Officer to further highlight the Galafold performance in the quarter. Bradley?

[Bradley Campbell, President and Chief Operating Officer]

Great. Thanks John. Good morning, everyone. As mentioned, I will walk you through in more detail now our Galafold performance for the quarter.

On Slide 7, we give our typical global snapshot on the Galafold commercial progress. For the quarter total product revenue grew by 18% versus same quarter last year to $79.5 million globally, driven by strong patient demand, new patient starts and business continuity. The geographic breakdown revenue during the quarter was $53.9 million or 8% of revenue generated outside the United States and the remaining $25.6 million or 32% coming within the United States. As we've mentioned before, this is in line with the roughly 70-30 split that we expect as we continue to grow both parts of the business.

Turning now to Slide 8. The third quarter was another strong quarter for our global commercial efforts. The business continues to be incredibly resilient with patients added in all of our major markets and third quarter revenue reflected increased patient demand, it also benefited from continued foreign exchange tailwinds.

We do continue to see some sporadic COVID related slowdowns in new patient starts due to delays at the point of care between patient identification and initiation of treatment. Importantly, though, our customers have confidence they can access Galafold. Our field team has been able to achieve a substantial majority of their pre-COVID touch points. And this is done through a combination of in-person digital telephonic, and other means of interacting with physicians.

So, while COVID is not yet fully abated, as we had hoped, it continues to be a very successful year for Galafold with the first three quarters of 2021 in line with our internal expectations.

As mentioned on past calls, due to a variety of factors, the rate of growth within the year is typically, non-linear. Usually in a given year, we see stronger quarter-to-quarter growth in second quarter and fourth quarter versus first quarter and third quarter. And that pattern has continued. Based on this momentum and assuming the dynamics are just highlighted continue, we're confident we will come within our full year 2021 guidance of $300 million to $315 million in global sales.

Ahead on Slide 9, I do want to recognize the importance of continuing to build the body of evidence for Galafold. And we're pleased to share here that the long-term data were recently published in September, 2021 issue of Molecular Genetics and Metabolism Reports, as well as presented at the recent ASN Kidney Week Conference. These long-term data highlights, stable renal function during treatment up to 8.5 years of Galafold, irrespective of treatment status, gender, or phenotype and inclusive of classic males. So an important part of the growing evidence, supporting the use of Galafold in patients with amenable mutations.

On Slide 10, we've called out several of the drivers and metrics, which will lay the foundation for growth this year and beyond. As we've mentioned, we ended 2020 with more than 1400 patients in Galafold, which was a little under half of the global market share of treated the amenable patients. So, while we're achieving higher market shares in countries where we've been approved the longest, there's clearly still plenty of opportunity to continue to switch patients over to Galafold.

We also note that there are significant numbers of diagnosed, untreated patients who have amenable variance. And while the global mix remains about 60% switch patients and 40% naïve patients. In many geographies, we're starting to see stronger uptake in naïve populations.

As we've said previously, over the next few years, we expect to see the balance of switch of naïve patients to be about fifty-fifty. And in the long-term, we expect to see that percentage reverse in favor of naïve patients.

All of that is underpinned by the impressive compliance and adherence rates that continue to exceed 90% reiterating our belief that those patients who go on Galafold stay on Galafold.

Importantly, the value of Galafold continues to be recognized by payers as well with the vast majority of insurance reauthorizations granted in 2021 by U.S. payers and a very strong track record of successfully negotiating and renegotiating reimbursement outside the United States. Our relentless focus remains on ensuring access to Galafold for anyone who needs it.

And as we touched upon last quarter, we keep exploring ways to expand the label highlighted with the European Commission approval there of the label for Galafold to the adolescent population. We continue to work now closely with government authorities to secure access to Galafold for eligible patients as quickly as possible.

Finally, another important driver of growth for Galafold is the ongoing geographic expansion. In 2021 we've continued to add to the growing list of countries with reimbursement and expect to add more as we look to expand access to those Fabry patients with a manual variance around the globe.

As a reminder, Galafold has received regulatory approval in over 40 countries and commercial sales in over 30 of those today. So, despite the recent COVID-related headwinds and certain geographies demand for Galafold worldwide continues to grow with some queues of potential new Galafold patients building in multiple regions. And again, we're confident we'll end 2021 within our guidance range of $300 million to $350 million in full year global sales.

As a reminder, as part of that guidance, we project net new patients starts this year will be even greater than in 2020. And we're on track for this growth of a number of patients in corresponding revenue to be weighted to the second half of the year as the COVID impact continues to ease.

On Slide 11 with several years of performance and a successful track record we can confidently say we're on a path to achieve the important milestone of $500 million in global revenue in a given year from Galafold within the next few years. The exact timing of this milestone will depend on how quickly things return to normal post-COVID and we'll have a better sense of that depending on how this year ends up.

We continue to expect to generate $1 billion in cumulative revenue over the next three years, which will contribute a significant amount towards funding our R&D and operating expense over that period and we remain confident in the $1 billion peak revenue opportunity for Galafold as we continue to see significant growth in the Fabry market globally driven by diagnosis from high risk screening, newborn screening, and other diagnostic initiatives, which we continue to support and invest in as well.

Finally, we have worked exclusivity in the U.S. and Europe, in addition to our 27 Orange Book listed patents that give us IP coverage into the late 2030s, 13 of which provide protection through 2038. So quite a bit of opportunity to provide access to Galafold globally for long period to come.

And with that, let me now turn the call over to Dr. Jeff Castelli, our Chief Development Officer, who will start on AT-GAA, our next generation therapy for Pompe disease. Jeff?

[Jeff Castelli, Chief Development Officer]

Thank you, Brad and good morning, everyone. Moving on to our R&D updates on Slide 13, we'll start with AT-GAA, our novel next-generation therapy for Pompe disease.

First, it's always important to recognize that Pompe continues to pose a range of health challenges for people affected by the disease and having therapeutic choices and options is crucial. Pompe is a severe and fatal neuromuscular disease and one of the most prevalent lysosomal disorders. In addition to the individual human tragedies we've seen multiple publications and natural history studies of Pompe patients that highlight the initial benefit of treatment that is often followed by continued long-term decline on key measures of disease for many individuals.

As a reminder, this sustaining high unmet means for the ERT experienced population has only ever been studied in a controlled setting in our Phase 3 PROPEL clinical trial. All other controlled lay onset Pompe disease studies to date have been in participants naïve to treatment.

Moving on to Slide 14, we present a summary of the primary key, secondary and biomarker endpoints from our Phase 3 PROPEL study. As a reminder, the study was double blind, randomized, set in the efficacy and safety of AT-GAA in adult treatment naïve and ERT experienced patients against approved therapy alglucosidase alfa.

Here in the slide, grouping these endpoints into domains of motor function, muscle strength, pulmonary function, patient reported outcomes and biomarkers, you can see the majority of endpoints across all of these domains favor AT_GAA alglucosidase alfa in both the overall and ERT experience populations. We believe this consistency of effects across the key disease manifestation illustrates the potential impact of AT_GAA for Pompe patients.

As our reminder on the primary endpoint of six-minute walk distance patients on AT-GAA outperformed those on alglucosidase alfa in the overall population with the difference between groups of 14 meters, which did not quite reach statistical significance for superiority. However, on the first key secondary endpoint of percent predicted for vital capacity or FVC, in the overall population, AT-GAA showed a nominally statistically significant and clinically meaningful difference for superiority versus alglucosidase alfa, with the treatment difference of 3% and a P value 0.023. This is a really important finding as progressive loss of pulmonary function is the leading cause of mortality in Pompe. And this was the main endpoint upon which alglucosidase alfa was initially approved.

We remind everyone that the PROPEL study tested for superiority versus an approved therapy, not versus placebo, and that the [indiscernible] approval of the second product generally requires demonstration only of noninferiority to improve therapy. We're also very pleased to announce that we expect the Phase 3 PROPEL results to be published shortly in a prestigious peer-reviewed medical journal.

Moving on to Slide 15, we have highlighted key updates across the AT-GAA program. First, on the regulatory progress, the U.S. FDA has accepted for review the BLA for cypaglucosidase alpha and the NDA for migalastat, as we previously announced. These two components have PDUFA action date set for May 29, 2022, for the NDA; and July 29, 2022, for the BLA. As John mentioned, we expect these filings to be reviewed together. We are also pleased to share, as John announced, that the MEA has now been submitted to the European Medicines Agency.

In June, we announced also that AT-GAA was granted a positive scientific opinion through the early access to medicine scheme for by the UK's MHRA. This positive opinion recognizes the high unmet medical need faced by the Pompe community and permits eligible adults living with late-onset Pompe who have received alglucosidase alpha for at least two years to switch and to have access to AT-GAA prior to marketing authorization in the UK.

We're really excited to see the significant enthusiasm for AT-GAA under the mechanism with multiple patients and physicians having requested access across the leading Pompe centers. Since that positive scientific opinion in June, the interest and momentum for AT-GAA has grown, and we are pleased to be able to provide access to those who are eligible in the UK.

For the younger Pompe community, we continue to enroll the ongoing open-label study in children up to 18 years of age, living with late-onset Pompe disease and look to expand into patients with infantile onset complete disease as soon as possible. At this point, we're pleased to report that more than 150 patients worldwide are being treated with AT-GAA.

And finally, in response to the many requests for expanded access that we've received, our expanded access programs for both those living with infantile onset and late-onset Pompe disease continue to expand to multiple individuals.

Moving on now to Slide 17, we briefly highlight our industry-leading portfolio of gene therapies for rare diseases and the planned launch for Caritas. To remind everyone, as John noted, the mission of Caritas is to transform the lives of children and adults living with rare genetic diseases through advanced protein engineering and innovative vector technologies. We see significant opportunities for Caritas to overcome current gene therapy challenges around safety, durability, manufacturability and immunogenicity. We have and are developing the tools and technologies to address these challenges and realize the promise of gene therapy.

The Caritas portfolio includes both clinical and preclinical programs. In addition to the two Batten programs in the clinic for CLN6 and CLN3, we expect to see three new INDs over the next 24 months in other rare genetic diseases, including Fabry and Pompe. This also includes the global rights to approximately 50 rare diseases, which include the majority of lysosomal diseases as well as nearly a dozen larger rare diseases and our collaboration with the University of Pennsylvania.

We've disclosed previously that these include Angelman, RET, myotonic dystrophy in addition to multiple muscular dystrophies, including DMD. As part of this transaction, we will continue to jointly work with the University of Pennsylvania as well as with Amicus through an attractive risk and cost sharing partnership.

For manufacturing, we have a fully designed, ready-to-build, state-of-the-art 35,000 square foot clinical manufacturing facility with commercial expansion capabilities planned and together with our continued strong relationship with Thermo Fisher on the CDMO front. All of this is being led by an experienced leadership team with a fully staffed discovery research and development organization of passionate entrepreneurs and problem solvers.

Slide 18 highlights the Fabry and Pompe gene therapy programs, which Amicus and Caritas will share co-development and profit-sharing rights to. These programs utilize a differentiated gene therapy approach for greater potency and optimized cross-correction through protein engineering of stability and targeting. The partnership between Amicus and Caritas will ensure the continued relationship with the leading internal experts in Fabry and Pompe disease working on these programs. The partnership also derisks the funding of those programs and provides a fifty-fifty global profit split. We expect the Fabry IND to be filed first in the second half of next year.

Last and to build on John's earlier comments on our CLN6 Batten gene therapy program, I would like to reiterate the sentiment around the loss of the two children during the long-term follow-up period of the study. As John mentioned, the investigator deemed that both participants passed away from disease-related complication unrelated to the study drug. We continue to assess all available information.

As a reminder, our Phase 1/2 clinical study enrolled 13 participants who all received a single intrathecal injection of ATGCX501 at a dose of 1.5 to the 13 vector genomes. The first children in the study received their gene transport over five years ago. Medical literature indicates that most individuals with CLN6 Batten disease are diagnosed during infancy and early childhood and do not survive beyond childhood or early adolescence.

While extremely news, we recognize the devastating nature of this disorder and continue to see the potential benefits of gene therapy broadly. 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?

[Daphne Quimi, Chief Financial Officer]

Thank you, Jeff. And good morning, everyone.

Our financial overview begins on Slide 20 with our income statement for the third quarter ending September 30, 2021. For the quarter, we achieved Galafold revenue of $79.5 million, which is an 18% increase over the same period last year. This includes a year-over-year operational revenue growth measured at constant currency exchange rates of 17%, further benefited by a positive currency impact of 1%.

Total operating expenses of $110.2 million in the third quarter were down as compared to $111.8 million in the third quarter of 2020. On a non-GAAP basis, total operating expenses were $93.6 million in the third quarter as compared to $92.4 million in the third quarter of 2020. We define non-GAAP operating expense as research and development and SG&A expenses, excluding share-based compensation expense, changes in fair value of contingent consideration and depreciation.

Net loss for the third quarter of 2021 was $50.3 million or $0.19 per share as compared to a net loss of $64 million or $0.25 per share for the prior year period. As of September 30, 2021, we had approximately 279 million shares outstanding.

Turning now to Slide 21, the $200 million private investment in the quarter added to our strong cash position. We are on a path to self-sustainability and profitability in 2023. We have achieved this milestone through our continued revenue growth with Galafold as well as driving efficiencies, cost savings and careful expense management.

For the third straight year, we expect total non-GAAP operating expenses in 2021 to remain relatively flat as we leverage the commercial infrastructure that is already in place for the AT-GAA launch and other products in our pipeline, transition the costs associated with the development of AT-GAA to multiple gene therapy programs in the pipeline and maintain financial discipline while meeting our objectives.

A few comments about our cash position and 2021 financial guidance. Cash, cash equivalents and marketable securities were $557 million at September 30, 2021, as compared to $483.3 million at December 31, 2020. We are reiterating our full year Galafold revenue guidance expecting to be within the $300 million to $315 million and maintaining our non-GAAP operating expense guidance of $410 million to $420 million as we expect the timing of manufacturing batches to impact fourth quarter operating expenses.

And with that, let me turn the call back to John for closing comments.

[John Crowley, Chairman and Chief Executive Officer]

Great. Thank you, Daphne, and Jeff and Bradley as well. As you can see, we've been relentlessly focused on execution and performance across the business, driven by a global team of passionate entrepreneurs who have led and will continue to lead us on our patient-focused mission.

We are immensely excited for what the future of science and biotechnology hold as we continue to advance toward our commitment to people living with rare diseases through not only Amicus, but beginning with the next earnings cycle with Caritas Therapeutics as well.

So with that, operator, we're happy to take any questions.

[Operator]

[Operator Instructions] Thank you. Your first question comes from the line of Ritu Baral with Cowen. Your line is now open.

[Anvita Gupta, Cowen]

Good morning team. This is Anvita on for Ritu today. On the two deaths in the CLN6 study, could you perhaps provide a little more color on how all these patients were when they were diagnosed? And what was the rate of decline on the Hamburg scores in the extension period? And probably more broadly, could we expect a clinical data update at World from the CLN6 and CLN3 programs?

[John Crowley, Chairman and Chief Executive Officer]

Sure. Again, I'll remind everybody the Batten program came to us through the acquisition of Celenex, which was a spinout out of Nationwide Children's Hospital more than three years ago, the 13 children dosed in that study. The first children were dosed about 5.5 years ago. The last patient was dosed more than three years ago. So again, to reiterate, the investigator has deemed this not to be related to study drug. I want to be very clear about that. These children were among the oldest, in fact, one of the two was the oldest in the study upon enrollment and had already advanced in their disease prior to the gene therapy.

With that said, we'll continue to evaluate all of the children who remain in the study, gather data on the children who did pass away. So, I'll turn it to Jeff or Mitch for any additional color or comments.

[Jeff Castelli, Chief Development Officer]

John, this is Jeff. I don't have anything to add to what you noted other than we continue to follow the patients across both studies. We're not noting whether we'll have data at world or not, but we do expect to provide data updates next year as we continue to collect data from those extension studies and continue to work on manufacturing at Thermo Fisher to provide GMP material and to have clinical and regulatory discussions with the agencies to move these forward.

[John Crowley, Chairman and Chief Executive Officer]

Great, thank you.

[Operator]

Your next question comes from the line of Anupam Rama with JPMorgan.

[Anupam Rama, JPMorgan]

Hey guys. Thanks so much for taking the question. I know you probably can't get into a lot of detail here, but how has the engagement been with the FDA on the AT-GAA filing and on the regulatory side? And has there been any indication of a potential ADCOM for the filing? Thanks so much.

[John Crowley, Chairman and Chief Executive Officer]

Yes. Thank you, Anupam. I'll say the FDA has been highly engaged and interactive. The review is continuing. Inspections are underway. So, everything we would expect, we continue to receive information requests. So, it's been a highly engaged review to date, which we're very pleased with. I will note that we have received in writing from the FDA that there will not be an advisory committee. So, while, again, of course, the FDA will always reserve the right to revisit that. As of now, the FDA has confirmed that there will not be an ADCOM.

[Operator]

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

[Joseph Schwartz, SVB Leerink]

Hi, thanks very much. I was wondering if you could give us an update on your plans to study infantile onset Pompe disease and get AT-GAA approved in this population. And how should we think about the size of this opportunity? Is it would compare with the size of the population with late-onset Pompe disease?

[John Crowley, Chairman and Chief Executive Officer]

Great. No, thank you for the question, Joe. Studying the infantile-onset Pompe disease population is incredibly important to us. We expect that study to begin, the formal study to begin very shortly as we finalize the protocols with FDA and the EMA. It is a relatively small portion of the population. We would estimate it to be perhaps 10% or less. And again, in terms of an opportunity, for this product, again, based on weight-based dosing and even smaller proportion there, but obviously incredibly important to be able to treat these children. I will remind everybody that we have treated under our global expanded access or compassionate use program a number of infants, and we're very, very pleased with the results that we've seen in those infants.

[Operator]

Your next question comes from the line of Dae Gon Ha with Stifel.

[Dae Gon Ha, Stifel]

Great. Good morning. Thanks for taking our questions. And John, if this is my last time speaking with you, good luck on your next adventure and look forward to keeping in touch.

[John Crowley, Chairman and Chief Executive Officer]

Thank you, Dae.

[Dae Gon Ha, Stifel]

My one and only one question is with regards to Pompe, as we're gearing up for your progress with AT-GAA. Just wondering if you guys have done any latest market research with regards to Lumizyme and NexViazyme uptake? And how that is faring in the current landscape as you're gearing up your commercial sales force? Thanks.

[John Crowley, Chairman and Chief Executive Officer]

Yes. Let me – Bradley, why don't you go ahead and take that, please. Thanks, Dae.

[Bradley Campbell, President and Chief Operating Officer]

Sure. Obviously, careful not to speak on other products out there in this space, but we can share what's available publicly. We know that the Pompe market continues to grow. We think that reflects the high demand for treatments for Pompe disease. And I would encourage you to look at the latest reports on the uptake around the more released product in the United States, GAA. And I think Sanofi released those numbers recently. So, I think that would be a good indicator of at least their initial uptake there.

I will say that focusing on AT-GAA, we continue to expect high demand for that product. Of course, we have to get – go through the regulatory process. But I do think the color that Jeff provided in particular around the EAMS process in the UK shows that where AT-GAA is now available through a formal mechanism albeit a pre-reimbursement – pre-approval mechanism we're seeing significant demand and interest for use of that product, and we hope that reflects physicians confident that there's an opportunity for AT-GAA to make a major difference here. And clearly for the unmet medical need in the Pompe space.

[Operator]

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

[Yun Zhong, BTIG]

Hi. Good morning. Thanks very much for taking the question. So, a question on your Fabry gene therapy program. And given that you have Galafold in the market and also – excuse me – several other gene therapy programs are already in clinical studies and have reported quite interesting data, so how do you think your program will be able to compete with existing program? What part of the program do you think is most differentiated?

[John Crowley, Chairman and Chief Executive Officer]

Yes, great. Thank you, Yun. So again, we've had great experience working in the Fabry community for really since the day we started Amicus, and we think that will be incredibly important to understand the unmet needs in that community, the nature of clinical studies, regulatory pathways and eventually providing 100% access to everyone living with Fabry disease in the world.

I think for gene therapy, we continue to believe that the greatest unmet need will be in those patients who don't have access to Galafold today, so those patients with non-amenable mutations. And we've got a commitment. It's part of the Amicus promise to patients that we would continue to forever designate a portion, a significant portion of our revenue from Galafold into Fabry disease to not only develop treatments but ultimately until there is a cure. And we think this does have the potential to be that cure.

We've developed something we think is highly differentiated. We think very importantly in the Fabry market, while several firms may be the first to the clinic, we would hope to be the first out of the clinic. And I think that's very important and that gets to the nature of what we've created. Again, the whole notion for what we've developed in gene therapy at Amicus is to look at each disease, understand its unique biology, the unique aspects of the disease, the clinical manifestations and develop a therapeutic gene therapy that can best address that.

Let me turn it to Jeff. Jeff, if you want to just remind everybody the drug candidate that we've developed in conjunction with Dr. Wilson and his team at Penn, and why we think that's differentiated.

[Jeff Castelli, Chief Development Officer]

Sure. Thanks, John. Yes, there is really two main components that we can differentiate our approach. One is this is a ubiquitous promoter with the AAV. So, we get expression not only directly in the cells that are transduced, but also and cost correction that ubiquitous expression can help not only create more of the enzyme out in the circulation, but also could be more durable.

And then secondly, and really what's most differentiated is that we've created a transgene that expresses a GLA that has a stabilized dimer through two engineered disulfide bond. That stabilized enzyme is not only more stable in the blood as it's secreted and then travels to treat other cells and tissues, but even after uptake into tissues and cells, it looks like it is more active. So, we've done studies comparing that stabilized transgene versus the wild-type GLA transgene, and just see that we get much more substrate clearance at a given dose with that more enzyme that's being expressed. So, we're really excited, as John mentioned, about the opportunity here to have what we believe has the potential for a best-in-class AAV gene therapy in Fabry.

And in terms of unmet need, for that nonamenable Fabry portion of the market, which is over half of the patients out there, their only option is every other week ERT infusions, currently. We view there's a real need there for a onetime treatment, which would be a big market expansion from an Amicus perspective.

[Operator]

[Operator Instructions] Our next question comes from the line of Nishant Gandhi with Needham & Co. Your line is now open.

[Nishant Gandhi, Needham & Co]

Hi. This is Nishant. I'm on for Gil. Thank you for taking our questions. I know PDUFA date is six, seven months away, but are you planning in any way, like thinking of commercial launch? Like are you planning for sales force ramp up? Or are you waiting on the decision before proceeding?

[John Crowley, Chairman and Chief Executive Officer]

Yes. Thank you, Bradley, do you want to feel that, please?

[Bradley Campbell, President and Chief Operating Officer]

Sure. Of course, yes, thanks for the question. We are very much geared towards commercial launch and anticipating approvals in the summer of next year, as John articulated. Really excited to do that. Of course, this would be the second product that Amicus has brought from discovery all the way through to launch, and will be an exciting milestone for the company.

Importantly, to your point about building commercial infrastructure, good news is the infrastructure we've built for Galafold is highly leverageable here. Many of the same physicians and centers are responsible for treating Pompe disease as are responsible for treating Fabry disease. What we said previously is that in order to support the launch of AT-GAA, we only anticipate a handful of additional FTEs, maybe in the sort of a dozen or so FTEs to support that launch, primarily in our Amicus Assist team as well as medical affairs and direct marketing.

So that's one of the great things about the team that we've built to support Galafold as they'll also be able to support the launch of AT-GAA. But rest assured, we're eagerly anticipating that opportunity and doing all the things to prepare – to be ready for an exciting launch.

[Operator]

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

[Unidentified Analyst]

Hey, this is Jonny on for Ellie. In terms of the Pope early access in the UK, can you update us on any trends you're seeing there in terms of updating switches from ERT? Thank you.

[John Crowley, Chairman and Chief Executive Officer]

Great. Yes, thank you again to remind everybody, we received the early access approval at the end of May. Since that time, we've been working through all the health authorities, the regional authorities in the United Kingdom to enable the next level of approvals. We've made significant progress there.

Again, we really are seeing significant enthusiasm. We've received for multiple key opinion leaders, multiple centers throughout the United Kingdom request to switch patients from the approved standard of care enzyme therapy to AT-GAA. We've been working through that for all the approvals necessary, the switches. We're starting to see patients now switched, which we're very encouraged by, and we think that, that will continue and that momentum will grow in the months ahead.

Bradley, anything I missed, feel free to add color.

[Bradley Campbell, President and Chief Operating Officer]

No. I think you've captured it, John.

[John Crowley, Chairman and Chief Executive Officer]

Great. Thank you. Yes. It's a very significant commitment for us. And again, something we're very excited about to provide AT-GAA in the United Kingdom to adults eligible to switch.

[Operator]

We have a follow-up question from Ritu Baral from Cowen. Your line is now open.

[Anvita Gupta, Cowen]

Hi, this is Anvita on again for Ritu. Thanks for taking our follow-up question and apologies if I missed this earlier. Of the 150-plus patients on AT-GAA globally, could you provide us a breakdown of open-label extension patients versus early access from the U.S. versus any other compassionate use? Thank you.

[John Crowley, Chairman and Chief Executive Officer]

Yes, we don't provide specific numbers for all of that. Again, the vast majority of those patients are on a long-term extension study following the PROPEL data. We also have all of the Phase 1/2 patients who continue on AT-GAA. We now have patients in the adolescent study. Again, we expect to begin the infantile study very quickly. And we do have an expanded access program that includes a range of patients, including infants as well.

So, it really is a mix in terms of disease onset and characterization within the spectrum of Pompe disease. And importantly, it's also a mix across clinical sites with dozens and dozens of investigators now caring for these patients receiving AT-GAA on now five continents. So geographically quite dispersed.

[Operator]

At this time, I would now like to turn the conference back to Mr. John Crowley, Chairman and CEO, for closing remarks.

[John Crowley, Chairman and Chief Executive Officer]

Great, operator. To all the analysts, thank you for all the great questions today. Again, a good very strong quarter for Amicus on Q3, and we look forward to a very strong closeout to the year. Thanks, everybody. Have a great day. Take care.

[Operator]

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