Earnings Call Transcript - FOLD Q1 2020

Operator, Operator

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics First Quarter 2020 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, Director of Investor Relations. You may begin.

Andrew Faughnan, Director of Investor Relations

Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics’ first quarter 2020 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; and Daphne Quimi, Chief Financial Officer. Also joining for Q&A are Dr. Jay Barth, Chief Medical Officer; Dr. Hung Do, Chief Scientific Officer; and Dr. Jeff Castelli, Chief Portfolio Officer and Head of Gene Therapy. 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 representations 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 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 disclosure of such forward-looking statements and the risks and uncertainties that may impact them, we refer you to the Forward-Looking Statements and Risk Factors section of our Annual Report on Form 10-K for the year ended December 31, 2019, and the quarterly report on Form 10-Q for the quarter ended March 31, 2020, 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 CEO

Great. Thank you, Andrew. And welcome everyone to our first quarter 2020 results conference call. Let me just begin by stating that I hope everyone and their families are well and safe. I know this is an extraordinary time and it certainly has been for our Amicus team and for our families. Since we first announced that Amicus would be moving to a work-from-home environment back on March 11, the senior leadership team and I, along with Amicus, are focused on two important themes, empathy and performance. The core of empathy has been about protecting the safety, health, and well-being of our global Amicus workforce and, by extension, our families, while emphasizing a range of programs and initiatives to protect and support our workforce. We have also been relentlessly focused on ensuring business continuity and performance against all of our key objectives. With that as background on today’s call, I am pleased to be highlighting the significant progress we have made at Amicus during the first quarter and now into the second quarter of this quite memorable year. While adapting to all of the changes brought about by this world pandemic, 2020 has been a period of growth and execution across our science, clinical and regulatory, and commercial efforts as we continue to build one of the next great global biotechnology companies poised to impact many people around the world living with rare diseases. As we did in this morning’s press release, I would like to highlight four key accomplishments. First, Galafold continues its strong launch performance and remains the cornerstone of our success. With $60.5 million in first quarter revenue, the Galafold launch continues to exceed expectations. First quarter revenue represents the performance across the global business including new patient starts from both switch and treatment-naive patients throughout the quarter in all major regions including those hardest hit by COVID-19. Of course, we will continue to monitor any future impacts from this pandemic, but we are encouraged by the trends that we are seeing. Second, our R&D timelines remain on track. We continue to expect the Phase 3 PROPEL study at AT-GAA in late-onset Pompe disease to read out top-line data on schedule in the first half of 2021. We disclosed also today that the U.S. FDA has now authorized us to proceed with a rolling BLA submission to this program, and our plans remain to start the rolling BLA in the second half of this year. Within our Gene Therapy pipeline, we continued to move forward our lead Batten disease programs in CLN6 and CLN3, as well as our most advanced preclinical gene therapy programs. Third, the Amicus Global Technical Operations team has done a tremendous job maintaining our supply chain during these times, thanks to the extensive contingency planning implemented in advance and the quick work early in this pandemic. We have been able to get drugs to patients where needed and we have taken further measures to ensure that this remains true in the future. We expect to continuously supply all Amicus therapies and potential therapies to patients in all geographies around the world. And fourth, maintaining the strength of our balance sheet remains a core priority for Amicus. We are announcing today that through a recent financial exercise and a series of cost-saving initiatives, our cash runway now leads us well into the second half of 2022. Our team continues to carefully shepherd our financial resources as we continue on the path to profitability. Daphne will discuss this in more detail later in the call. Turning to Slide 4, we are well on track to achieve our five key strategic priorities for 2020. These include: one, Galafold, our precision medicine for Fabry; we will continue to drive Galafold to more patients living with Fabry disease with amenable variants in existing and in new markets. We look to achieve global product revenue of $250 million to $260 million this year. Two, we are increasing the clinical, regulatory manufacturing and preclinical activities surrounding our Pompe program as we advance towards this approval. Three, we are advancing our industry-leading Rare Disease Gene Therapy portfolio stemming from our new global research and Gene Therapy Center of Excellence in Philadelphia. We will be advancing this clinical development manufacturing and regulatory discussions for both our CLN6 and CLN3 Batten programs. Four, additionally, we are progressing our Pompe gene therapy towards IND, and we will disclose up to two additional IND candidates this year. Much work is underway with our manufacturing partners for the manufacturing scale-up of the Pompe Gene Therapy, as well as our other potential IND candidates. We look forward to sharing the additional IND candidates from our Penn collaboration later this year. And again, we continue to maintain a strong financial position as we carefully manage our expenses and investments, remaining fully funded through all major milestones ahead, now with cash well into the second half of 2022. So, with that as an introduction, I will now hand the call over to Bradley Campbell, our President and Chief Operating Officer, to further highlight Galafold performance, our R&D timelines, and the supply chain. Brad, go ahead please.

Bradley Campbell, President and COO

Thanks, John. And as we sit here in what is our first truly virtual earnings call, let me just echo John’s statement, I hope everybody out there is safe and well along with your families and your colleagues. As John said, I will go through in more detail the Galafold performance for the quarter, also our R&D timelines and milestones. But I will provide more detail on the actions we have taken to ensure continuity of the supply chain. But let me start on Slide 6, which goes through the continued growth of Galafold for the first quarter of 2020 and provides a global snapshot of Galafold's commercial progress. For the first quarter, total product revenue was $60.5 million, which is a net increase of 78% from the first quarter in 2019. Galafold revenue has been driven by strong patient demand, as well as continued strong adherence. We’ve had some favorable reimbursement dynamics, and there has been only a minimal contribution from some ordering patterns as healthcare organizations in select countries have adjusted to the impact of the COVID-19 virus. Importantly, global compliance and adherence rates continue to exceed 90%. The geographic breakdown of revenue during the quarter, similar to what we have seen in previous quarters, was $42.8 million or 71% of revenue generated outside the United States, and the remaining $17.7 million or 29% coming from within the U.S. over the quarter. We now have over 40 countries around the world with regulatory approvals, and we have commercial sales in over 30 of those countries now with patients for the first time this quarter beginning to access reimbursed products in Colombia and Romania, with more markets expected later this year. In this expanding global footprint, it is important not just to support the continued growth of patients with access to Galafold, but it lays an incredibly strong foundation, which is highly leverageable to support the anticipated launch of Pompe and our future products as well. Turning now to Slide 7, we have had a very strong Q1, even in the face of the COVID-19 pandemic, and part of that was clearly strong momentum coming into the year after a great finish in 2019. As the COVID crisis has rolled through our various geographies, we have also seen some dynamics that we think could be unique to Galafold as an oral therapy for Fabry in these very unique times. For example, we have seen some patients switch to Galafold or start on Galafold because they’ve been reluctant to go into a hospital or a clinical setting for alternative care. We’ve also seen our first patients prescribed Galafold via telemedicine, a phenomenon that was highlighted in the recently released EURYDICE report, showing that 50% of rare disease patients have now experienced some form of telemedicine, which is up from 20% before the crisis. Finally, our supply chain remains fully intact, allowing our customers to have confidence in their access to Galafold. All of this we believe has led to continued uptake, and we’ve seen new patient starts throughout the crisis, even in the hardest-hit countries including the UK, France, Italy, Spain, Japan, the United States, and many others. We think these dynamics may continue to provide some tailwinds, even as the global pandemic inevitably causes some headwinds, which we will continue to monitor as its full impact remains uncertain. From a numbers perspective, you can see that first quarter sales, as mentioned, increased 78%, which does include a 3% negative impact from foreign exchange. So, if you look at it from an operational performance perspective, sales actually increased by 81% compared to the same quarter last year. On the left-hand side, we can see the quarterly performance over the past several quarters. We continue to expect consistent growth quarter-to-quarter, but again in a relatively non-linear way as we’ve seen in years past. So, on Slide 8, now with several years of performance behind us, we can confidently say that we are on a path to that $500 million sales opportunity in 2023. As I have outlined previously, to reach that $500 million, we would expect that without a five-year compound annual growth rate of 40% from 2018 to 2023, and we are well through that period now. We expect to generate about $1 billion in cumulative revenue between 2020 and 2022 alone, which will go a long way toward funding our R&D and OpEx over that period and we continue to have even further confidence in the billion-dollar revenue opportunity to peak, as we continue to see significant and sustained growth in the Fabry market globally, driven by continued diagnosis from high-risk screening, newborn screening, and other diagnostic initiatives in which we are now investing. We have orphan exclusivity in the U.S. and Europe and other geographies, taking us to the end of the 2020s, in addition to our Orange Book-listed patents that provide us with IP coverage until late 2030. So lots of opportunity and time to provide access to Galafold globally for a long period to come. On Slide 9, I want to highlight all the things we are doing to ensure that continuous supply chain. This isn’t something we’ve focused on before, but we will reiterate that our supply chain has maintained continuous supply during the COVID-19 pandemic for both Galafold and AT-GAA. For Galafold, part of our listing strategy had already been to hold multiple years of inventory in API and drug products. We don’t believe manufacturing will be an issue for us. As the COVID-19 pandemic became increasingly severe, we have successfully pushed inventory as far as possible down the supply chain to the country and even to the pharmacy level. This has been made much easier by the fact that this is a small molecule with long stability and is easy to ship around the world. For AT-GAA, our base plan had already been to build the inventory ahead of our anticipated launch and to move drug products outside of China into the UK, and that plan has been underway. Following the spread and impact of the COVID pandemic, we are taking similar actions to those for Galafold, pushing inventory into the supply chain as far as possible and coordinating on a site-by-site basis for delivery. We would also note here that WuXi, our manufacturing partner for ATB200 the biologics, has not had a single interruption to their manufacturing operations, allowing us to continue producing ATB200 and AT-GAA as we build commercial inventory. So turning now to Slide 11, let me go through some of our R&D updates. AT-GAA for Pompe disease, remember is the first-ever second-generation therapy, as well as the first therapy for Pompe disease to receive breakthrough therapy designation. There is tremendous momentum behind what we believe may be the next standard of care for a broad population of people living with Pompe disease, which represents a $1 billion to $2 billion global opportunity. We are on track, as John mentioned, to complete the Pompe Phase 3 PROPEL study on our original timelines to enroll the pediatric studies and advance manufacturing to support our 2021 BLA and MAA filings. Importantly, we are reiterating that the PROPEL study timelines are on track, even in the face of the COVID crisis, thanks to the incredible efforts of our clinical team, where we have literally created a patient-by-patient plan along with our investigators to protect the integrity of the study. We remind everyone that we did over-enroll the study, which provides further confidence. As of today, we’ve administered 97% of the expected 2,250 infusions up to this point, well within the design study parameters, and we will continue to ensure that patients are receiving their infusions. Today, for the first time, we announced that the US FDA has granted our rolling BLA submission for AT-GAA, and we are on track to initiate that rolling BLA in the second half of this year, supporting full approval under the fast track designation. Additionally, in response to the many requests we receive for compassionate use, particularly for children with Infantile-onset Pompe disease or IOPD, we’ve now initiated the expanded access program that we first discussed on our call for the full year earlier in March. From a manufacturing perspective, we’ve successfully completed all three of the production campaigns, including the bioreactor runs and related downstream purification activities under the PPQ process at WuXi, which will serve as the foundation for the CMC section of the BLA submission. This is a significant step forward in a major de-risking event for this program. We continue to be extremely excited about AT-GAA, as well as our preclinical Pompe Gene Therapy program to build what we believe could be the largest and most valuable franchise in the industry, offering solutions for all patients living with Pompe disease globally. Now on Slide 14, I will briefly highlight our industry-leading portfolio of gene therapies for rare diseases. Thanks to the careful efforts of our science and facilities teams, even during this time, we’ve been able to maintain our critical science and lead program activities across our gene therapy portfolio, including CLN6 and CLN3 Batten disease, as well as the Pompe and Fabry gene therapies in our earlier discovery programs. Starting with our Batten disease franchise and CLN6, we’ve previously reported positive interim data in our clinical study, demonstrating meaningful impact of our AAV gene therapy in this devastating form of Batten disease. This year, we expect additional data from the CLN6 Phase 1, 2 study and we plan to advance our regulatory discussions to finalize the clinical and regulatory paths for that program. We believe the initial CLN6 data provide important read-through for our clinical study in CLN3 Batten disease, which is the most common form of childhood neurodegeneration. Our plans this year include advancing regulatory discussions to finalize that clinical and regulatory path and reporting additional data from the ongoing Phase 1, 2 study in the second half. Importantly, in recognition of its potential to address the significant unmet medical need, our CLN3 gene therapy program has been granted fast track designation by the US FDA, which provides us greater access to the agency for expediting drug development, review, and potential approval. Finally, on Slide 15, let me remind you of all the great things that are happening as part of the research collaboration with the University of Pennsylvania, which will be an important driver of growth for Amicus for decades to come. This collaboration with the Wilson Lab at Penn combines Amicus's protein engineering and glycobiology expertise, along with Penn’s world-class gene transfer technologies to develop novel gene therapies designed for optimal cellular uptake, targeting, dosing, safety, and manufacturability. As part of this collaboration, we have rights to over 50 diseases, including eight that are currently in active preclinical programs. We also announced several Amicus presentations that will be presented at the American Society of Gene and Cell Therapy’s 23rd Annual Meeting, which is being held virtually next week. Our sponsored studies there include one oral presentation and two posters, and we encourage you to take a look at some of the new data provided virtually through that conference. We expect additional preclinical data this year in multiple gene therapy programs and we are continuing to guide to the disclosure of two additional IND candidates by year-end. With that, let me now turn the call over to Daphne, who can go through our financial results, guidance, and outlook.

Daphne Quimi, CFO

Great. Thank you, Bradley, and good morning, everyone. Our financial overview begins on slide 17 with our income statement for the quarter ending March 31, 2020. For Q1, we achieved Galafold revenue of $60.5 million, a 78% increase over the first quarter of 2019. This includes year-over-year operational revenue growth, measured at constant currency exchange rates of 81%, offset by negative currency impact of 3%. Cost of goods sold as a percentage of net sales was 10.8% in the first quarter, as compared to 11.9% for the prior year period. The cost of goods sold as a percentage of revenue was favorable as Galafold revenue continues to grow in the United States, where we do not owe royalties, as well as other countries where we are subject to lower royalties. Total operating expenses were $132 million for Q1 2020, as compared to $111.3 million for Q1 2019. On a non-GAAP basis, total operating expenses were $116.7 million for Q1 2020, compared to $96.2 million in Q1 2019. The increase in research and development costs was primarily due to investments in our gene therapy programs for the initiation of the tech transfer to our contract manufacturers and clinical research within our early-stage programs and within the Pompe program to advance and enroll clinical studies. Our investment in research and development includes the impacts of the implementation of cost reduction measures that were previously announced, as does the decrease in selling, general, and administrative expenses. The non-GAAP quarterly expense is expected to decline throughout 2020. We define non-GAAP operating expense as research and development and selling, general, and administrative expenses, excluding share-based compensation expense, changes in the fair value of contingent consideration, and depreciation. The net loss for Q1 was $88.9 million or $0.35 per share, as compared to a net loss of $120.3 million or $0.56 per share for the prior year period. As of March 31, 2020, we had approximately 257 million shares outstanding. Turning now to slide 18, as John mentioned, we are now fully funded well into the second half of 2022 through major milestones in our portfolio and continued global growth. We have been able to further extend our cash runway by continuing to drive efficiencies, cost savings, and careful expense management. On Slide 19, going forward, we expect total non-GAAP operating expenses in 2020 to remain relatively flat from 2019 as we leverage the commercial infrastructure that is already in place for the AT-GAA launch and other products in our pipeline. We transitioned the costs associated with the development of AT-GAA to multiple gene therapy programs in our pipeline and maintain financial discipline while meeting our objectives. To reiterate, all high-priority research programs in gene therapy are moving ahead on schedule, especially the CLN6, CLN3, Pompe, and Fabry Gene Therapy programs, and we continue to fully support the work with Jim Wilson and patients. A few comments about our current cash position and 2020 financial guidance: Cash, cash equivalents, and marketable securities were $339 million at March 31, 2020, compared to $453 million at December 31, 2019. We are reaffirming our full-year Galafold revenue guidance of $250 million to $260 million and our non-GAAP operating expense guidance of $410 million to $420 million. With that, let me turn the call back over to John for closing remarks.

John Crowley, Chairman and CEO

Great. Thank you, both Daphne and Bradley. As you can see, we have been relentlessly focused on performance across the business despite all of the unprecedented challenges brought about by the global pandemic. We have a great global team of passionate entrepreneurs who have led and will continue to lead us through this, and I am confident that as the world emerges from this crisis, Amicus will emerge even stronger. So, operator, with that, we are happy to take any questions.

Operator, Operator

[Operator Instructions] Your first question comes from the line of Anupam Rama with JPMorgan.

Anupam Rama, Analyst

Hey guys. Thanks for taking the question and congrats on all the progress. I have just a quick question on the cash runway to the guidance which is now second half of 2022. Just wondering what that assumes in terms of sort of reopening again in the U.S. and globally due to the COVID-19 pandemic? And what are the push forward levers we should be thinking about on the expense side if, for whatever reason, there is a second wave of infection and the pandemic lasts longer than what some of us assume? Thanks.

John Crowley, Chairman and CEO

Sure. What we have communicated to all of our employees globally is that we are putting their health, safety, and well-being first and foremost. Thankfully, we have been able to operate, Anup, through this telepresence mode really effectively. The greatest risk to the business, frankly, would be to rush back and put people at risk. We are not going to do that. We have communicated to our employees that June would be the earliest that there would be any reintegration back. We are now undergoing a project carefully evaluating that, looking at all the local guidelines around the world in all the geographies we work in. So, what I would emphasize to our employees is that our return to any of what we were before from a work cadence will be flexible and staggered, with an integration over time. We have taken this time to launch an internal initiative we are calling R3 or R cubed, where I have asked all of our employees to rediscover, reimagine, and reinvent what Amicus looks like on the other side of this, taking into account best practices, efficiencies, and cost-saving efficiencies. Right now, this takes into account how we extend the runway to the second half of 2022. Again, this is a multi-week financial exercise across the entire organization, looking at all of our functional operating expenses, a reprioritization of our earlier stage preclinical programs, and a further measurement of our capital expenditures. Obviously, there are certain evident savings from the crisis itself, including travel and entertainment expenses, which are key parts of the budget, as well as conferences. We have had very limited additional hiring during this period. All of that taken together allows us to extend the runway well into the second half of 2022, bringing us very close to that bridge to profitability ahead. If the crisis were to reemerge and we had to revert to our shelter-in-place routine, we don't expect that that would negatively impact our expense standpoint as well. Did that answer your question, Anupam? Okay, hopefully.

Operator, Operator

Your next question comes from the line of Ellie Merle with Cantor Fitzgerald.

Ellie Merle, Analyst

Hey guys. Thanks so much for taking the questions. Just on the manufacturing front for gene therapy, can you provide us more color on how the scale-up is going for Batten and sort of the latest on your expectation for dosing more patients, and using commercial-scale manufacturing and just any impacts you are seeing in terms of supply or raw materials through the scale-up process due to COVID? Thanks.

John Crowley, Chairman and CEO

Sure. I’m going to let Brad handle that. It seems like your dog is excited as always about the Amicus leads.

Bradley Campbell, President and COO

Yes. Sure. Manufacturing with our gene therapy continues to go very well as you asked in your question. We are in the middle of the tech transfer activities to Brammer, who will be our manufacturer for the CLN6 and CLN3 programs and that is the hyper stack platform being used to manufacture with MCH. That process is going very well, especially as Brammer has a lot of experience with this platform, and we are making good progress. The intention is that the next patients who will be dosed in CLN6 will be using that commercial material from Brammer, which is on track for 2021. Regarding your question around raw materials and inputs into the supply chain for gene therapy, we had purchased study materials, plasmas, etc. ahead of time and secured manufacturing suite time for all of our supply chain for gene therapy. So, we have not experienced interruptions so far and we don’t anticipate any interruptions in the gene therapy manufacturing or supply chain.

Ellie Merle, Analyst

Great. Thank you, Bradley.

Operator, Operator

Your next question comes from the line of Whitney Ijem with Guggenheim.

Whitney Ijem, Analyst

Good morning guys. Thanks for taking the question. I wanted to follow-up on the Galafold, some of the comments around the Galafold trends, I guess, that you are seeing so far in light of COVID regarding patients being hesitant to go into healthcare centers, which could be a tailwind for oral therapies. I’m curious if there are any sort of leading indicators or metrics you’re tracking in terms of patients engaging with you all virtually on the website or anything like that, which could suggest if that trend or potential tailwind for a switch is continuing moving forward?

John Crowley, Chairman and CEO

Great. Thanks, Whitney. I’ll ask Brad to comment in a moment, but just to emphasize we have really strong trends coming into 2020 before the COVID-19 crisis. So this great performance in Q1 was driven by fundamental patient demand. The fact that it’s an oral medicine that can be taken at home is yet another benefit, particularly in this COVID-19 crisis, and we’ve seen that play out in a number of settings across various geographies. Though it’s not the key driver of this great performance, it is a factor. Brad, if you want to add more insight on how we think about it moving forward in the months ahead?

Bradley Campbell, President and COO

Yes. I think that’s well said, John. We have been well on our way in establishing Galafold as an important treatment option in Fabry disease and we think the best treatment option for patients with amenable mutations. The foundation and experience we now have with the physicians and patients set us up for continued success moving forward. To your specific question about those dynamics around the oral product, it’s interesting because outside the U.S., we can’t have direct interactions with patients. It’s really all through physicians, but we do have very close relationships with physicians and have done a good job so far in setting up the ability to interact with them virtually under these circumstances. It has moved from face-to-face meetings to text, phone calls, or video chats, just as we all have. This is how we hear some very encouraging anecdotes of physicians being able to meet some of their patients' needs by moving them to oral options, both for switch patients who may have been receiving replacement therapy and, for the reasons I highlighted, might want to pursue oral options and for new patients. We’ve continued to see new patient starts, even in the hardest-hit countries. So that system is working well. As John noted, we think it provides us some incremental tailwinds, which is exciting. In the U.S., given that we can have a regulatory compliance interaction with patients more directly, we’ve seen an increase in virtual patient meetings, which is really exciting and seems to be a form of engagement that patients appreciate. So, we will continue to explore these avenues as the pandemic plays out.

Operator, Operator

Thank you, sir. Your next question comes from the line of Ritu Baral with Cowen.

Ritu Baral, Analyst

Hi guys. Thanks for taking the question. I want to ask about potential Pompe competition and sort of the commercial landscape coming up. How would you position AT-GAA if the upcoming Genzyme data hits superiority within its naïve patient population? How would that compare to any potential superior six-minute walk data that you might generate? How are you thinking about commercializing a Pompe drug facing the reality of COVID given this patient population's risk to respiratory diseases? Orphan drug launches are usually very high touch, and you have such a great team with patient relationships. How are you thinking about the situation?

John Crowley, Chairman and CEO

Thanks, Ritu. I believe this launch like any other, but particularly in Pompe, will be driven by the strength of the data. The PROPEL study will be incredibly important. We’ve accumulated a unique dataset that I’m not going to review today. We’ve spoken on this at multiple scientific conferences, highlighting how AT-GAA has shown profound impacts on individuals, whether they were treatment-naïve or switching from ERT standard-of-care. While it’s positive that other drugs are in development, we have a unique medicine we developed, and we will let the clinical data speak for itself. Ultimately, the clinical data will drive the success of the launch. Our positioning is strong, importantly, with the PROPEL study having numerous sites in multiple countries and all key opinion leaders gaining experience with AT-GAA, which will also help when we launch this medicine globally. I will not speculate on what any other program may show; ultimately, the success of the launch of AT-GAA will rely on the strength of our data.

Operator, Operator

Your next question comes from the line of Joseph Schwartz with SVB Leerink.

Joseph Schwartz, Analyst

Great. Thanks so much. I was wondering if you could talk about the extent of CLN3 data that you expect to report later this year. Given the slower progression of disease activity in this subtype, how sensitive are the endpoints you are using? And where do you expect to detect the biggest impact versus natural history in CLN3 compared with what you are seeing in CLN6?

John Crowley, Chairman and CEO

Sure, Joe. Thanks. I hope you are well. This will be limited dataset on a handful of patients who were treated at the low end and the high dose with CLN3. I will ask Jeff Castelli, our Head of Gene Therapy, to comment further about the CLN3 program. Again, this will be preliminary data, with most patients beginning treatment in 2021 with the Brammer-produced commercial material that we believe will serve as the basis for approval. Jeff, feel free to add any additional comments.

Jeff Castelli, Chief Portfolio Officer and Head of Gene Therapy

Sure. Thanks, John. We will have the data later this year for CLN3 concerning the first handful of patients we have enrolled. This dataset will reflect one year of dosing data for most of those patients. CLN3 progresses slower than CLN6, but still does progress quite rapidly. The main endpoints we will focus on from an efficacy perspective are the unified Batten disease rating scale, which assesses various aspects of motor function, language, cognition, and vision. It’s a very diversified scale with lots of components. This will just be an early look at one year data, along with an update on safety data in this case.

Operator, Operator

Your next question comes from the line of Mohit Bansal with Citigroup.

Mohit Bansal, Analyst

Great. Thanks for taking my question and very good morning. My question is regarding whether some of your companies in our coverage area have talked about a bit of advanced purchasing, especially for orals in light of COVID. Have you experienced anything like that for Galafold which could potentially impact your Q1 and Q2 results and subsequent quarters? Thank you.

John Crowley, Chairman and CEO

Great. Thanks, Mohit. The $60.5 million results we reported today were driven by fundamental demand and the natural rhythm of sales. There was very minimal impact visible from any advance work in the channel. Bradley, Daphne, if you want to add any specifics to that?

Bradley Campbell, President and COO

Yes, John, I think you said it well. The primary drivers were strong patient demand and strong adherence, which we continue to see from around the world. We did have some favorable reimbursement dynamics as well and fewer bridge programs in the United States. So reauthorization went really well. Those factors have been the underlying drivers of patient demand. So that remains at the forefront.

Operator, Operator

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

Tazeen Ahmad, Analyst

Good morning. Thanks for taking my questions. One for Brad. Can you just walk us through how you are thinking about the Pompe markets, maybe specifics about the range of pricing that we should assume? You do have the benefit of having another drug on the market. I am hoping you could share some of the learnings or observations in terms of how big your sales force might need. Also, just your thoughts on pricing. I have a follow-up question. Thanks.

John Crowley, Chairman and CEO

Yes. Thanks, Tazeen. I’ll turn it over to Brad. We’ll apply the same principle that we did for Galafold, that our products must be fairly priced and broadly accessible. That will be a fundamental principle we apply here. Brad, if you want to broadly share how you’re thinking about ensuring access for patients for AT-GAA once it’s hopefully on a path to launch?

Bradley Campbell, President and COO

Sure. Thanks, John. Thanks, Tazeen, for the question. I believe we’ve learned a lot from Galafold, and our focus on access rather than premium pricing has served us very well. We prioritize providing a great value proposition for patients, physicians, and payers, allowing us to navigate reimbursement and become more agile in our launches across various markets globally. Thus, we will apply those learnings here. The advantage of being a second mover means we don’t have to convince payers that Pompe is a serious disease worthy of reimbursement for a costly rare disease drug; however, we need to demonstrate that our value proposition is superior to existing alternatives. This will hinge on the clinical data we generate, and we feel very confident based on the data we observed in Phase 2. In terms of our infrastructure, we were very successful with our efficient rare disease sales force. We understand the ratio of our team to that of Sanofi, which has the Fabry drug along with a Pompe alternative currently. We anticipate needing a smaller team compared to theirs, serving us well. Ultimately, with more patients on AT-GAA at launch, along with our ability to launch it in key markets like the U.S. early, we believe this sets us up nicely for success. For all these reasons, we feel well prepared and are looking forward to seeing the data.

Operator, Operator

Your next question comes from the line of Debjit Chattopadhyay with H.C. Wainwright.

Debjit Chattopadhyay, Analyst

Hey, good morning and congrats on the execution and thanks for taking the question. I understand the U.S. market is 29% of the revenue base. Given the 33 million unemployment print this morning, since we are all dependent on employer-provided insurance, could you share your current payer mix and how much of this unemployment disruption is built into your $250 million to $260 million full-year guide? Thank you.

John Crowley, Chairman and CEO

Great. Thanks, Debjit. I’ll ask Brad to comment on the payer mix and everything related to that.

Bradley Campbell, President and COO

Yes, it’s a good question. So far, we have had lower exposure to government payers from a mix perspective than we anticipated. But we have great access programs for both private insurers and government payers and, frankly, where patients can’t afford the drug, we provide free access. One of the things we’ve communicated is that we have never had a patient go through the process and ultimately be rejected by a payer. We feel confident that patients are getting access to Galafold, and we haven’t seen disruptions in patient access even due to some of the unemployment factors. Fabry is a sizable population for us, but it’s a small portion of the U.S. population. Those large unemployment numbers feel significant from a U.S. perspective, but exposure in the Fabry population for us has been minimal. Our guidance remains unchanged; we feel very confident in it. However, we will continue monitoring, and should things change, we will provide those updates accordingly. But right now, we feel very secure about where we are.

Operator, Operator

Your next question comes from the line of Mike [Indiscernible].

Unidentified Analyst, Analyst

Hey guys. Thanks for taking the question. And congrats on the strong quarter as well. Just a quick question for you on the PROPEL study. It seems like things are progressing fairly well there despite COVID-19, with 97% of infusions occurring on schedule. Just curious if you are expecting any issues in terms of data collection for that study or are there steps you are taking to help mitigate that potential risk? Thanks.

John Crowley, Chairman and CEO

Yes. No. thanks, Mike. As Bradley indicated, we have a really custom patient-by-patient, site-by-site plan implemented. The areas of focus are twofold: ensuring that infusions occur and that patients can access the drug within the appropriate windows, and secondly, ensuring assessments are carried out. To remind everyone, we conduct assessments at baseline, and also at 3, 6, 9, and then 12 months. We continue to ensure that patients receive those assessments, which is vital to maintaining the study's integrity. In some cases, we've had to go to extraordinary lengths to ensure that those assessments are done; that’s an important part of integrity. We feel good about our position and continue to follow all contingency plans closely to collect the data appropriately. Thankfully, we have not seen any interruption that would impact study integrity.

Operator, Operator

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

Andrea Tan, Analyst

Thanks for taking our questions. This is Andrea on for Salveen. Maybe one question for Brad: with respect to Galafold, could you provide updated metrics on the slip between switch and naïve patients? And how this patient mix is perhaps tracking with your expectations within the context of new and established markets?

Bradley Campbell, President and COO

Yes. Happy to take that. Good question. The dynamics we have are continuing on track. We are about 65:35, two-thirds switch versus one-third naïve. The naïve portion is slowly growing, which we anticipated. Typically, in most markets, the initial uptake is stronger in the switch population, as they are frequently coming into the healthcare system for infusion treatment. Over time, we expect to see more naïve patients join therapy. In mature markets like Europe, the growth driver this year is now around 50:50 between switch and naïve, with some markets starting to tilt towards more naïve patients than switches as we penetrate the existing market. We expect to drive significant growth in the treated market within the Fabry disease setting over the next three to five years. Those trends are ongoing, and as I highlighted on the call, we see new patient starts for both switch and naïve along the proportions we've observed in prior quarters.

Operator, Operator

Thank you. You have a follow-up question from the line of Ritu Baral with Cowen.

Ritu Baral, Analyst

Hey guys. I just wanted to get back in with my commercialization question, just as far as launching into a Pompe population at risk for respiratory diseases when COVID is a risk for the general population. How are you thinking about a virtual orphan drug launch, assuming your data leads to approval in 2021?

John Crowley, Chairman and CEO

Sure, Ritu. I’ll comment and then I’ll ask Brad to provide additional insights. Given the extraordinary demand for this next-generation product in Pompe, I don't expect it to impact our launch. We are looking at a possible launch in late 2021 or early 2022 and hope that the COVID crisis will significantly subside, if not entirely, by that time. With that in mind, I believe the PROPEL and infantile studies will reference our patients seeking better treatment options. The greatest risk is not being on the best available therapy. These physician scientists are extremely motivated, and the key opinion leaders are motivated to ensure patient access to a potential next-generation therapy. Brad, do you want to add color on our global AT-GAA launch strategy?

Bradley Campbell, President and COO

Yes, thanks, John. Just a few additional comments: I think that was well articulated regarding demand and our strong supply chain efforts to keep drug access intact for patients. Moreover, an advantageous factor of AT-GAA’s development program is that we will have many patients on AT-GAA at launch, alongside numerous centers and countries involved—much more than with Galafold. This offers a strong start. We are also exploring other opportunities to engage in what might come after this pandemic. People are adapting to virtual interactions, and we see that physicians and patients will embrace those improvements. For instance, in the U.S., we've been doing more virtual patient meetings, and we regard this as valuable. Our case management and medical affairs teams work closely with our supply chain to ensure that we address all inquiries patients and physicians might have. The experience we gained during the Galafold launch will enhance our support during the upcoming Pompe launch.

Operator, Operator

Your next question is a follow-up from Debjit with H.C. Wainwright.

Debjit Chattopadhyay, Analyst

Hey. Thank you for holding back once again. Regarding the Pompe gene therapy IND, we have little information regarding whether it’s liver-directed or muscle-directed, etc. How should we be factoring in auto expression updates, etc. and, in some negative patients, if it’s not a liver-directed gene therapy, how do you circumnavigate that?

John Crowley, Chairman and CEO

Great. Thanks, Debjit. We have more data coming out at the upcoming FDA Conference next week. I am going to ask Hung to comment broadly about the technology approach we’re taking here. Hung, if you want to field that, and Jeff, feel free to chime in if you have more color.

Hung Do, Chief Scientific Officer

Sure. I think we’ve previously indicated that our Pompe gene therapy program will utilize a broadly distributed tropic type of vector, which allows for transduction not just of the liver, but also muscle and other tissues. This particular vector, designed by his group at Nupen, demonstrates robust transduction capabilities across various delivery sites. We have performed animal studies validating that it effectively transfers the gene. We may have limited data on tolerization, but we are optimistic about its efficacy for all tissues.

Jeff Castelli, Chief Portfolio Officer and Head of Gene Therapy

Sure, Hung. That’s a good overview. I’d like to emphasize that our approach focuses on muscle-tropic promoters which will transduce liver tissue. It allows us to diverge the gene impact across compartments, like muscle, which might have better durability and strength in terms of efficacy. So we anticipate that any tolerization that may arise from hepatic transduction will also provide benefits to other tissues, including muscle.

Operator, Operator

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

John Crowley, Chairman and CEO

Great. Thanks, operator. That was a great conference call. Thank you for all the terrific questions. I hope everybody and your families stay well and healthy. Have a great day.

Operator, Operator

Ladies and gentlemen, this concludes today’s conference. Thank you, and have a great day.