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.

[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, Director of Investor Relations. You may begin..

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 Science 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 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 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?.

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’ve first announced that Amicus would be moving to a work from home environment back on March 11 the senior leadership team and I and 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 and 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.

So with that as background on today’s call, I am pleased to be highlighting the significant progress we’ve made at Amicus during the first quarter and now into the second quarter this quite memorable year.

While adapting to all of the changes brought about by this world’s pandemic karmic has started 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 just a tremendous job maintaining our supply chain during these times.

Thanks to the extensive contingency planning in advance in place 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 that remains true into the future.

We expect to be able 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 further financial exercise, and a series of cost savings 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 then 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 – I am sorry, pre-commercial 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, in addition, we are progressing our Pompe gene therapy towards IND and we will disclose up to two additional IND candidates this year. A lot of 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.

And 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 and we remain fully funded through all major milestones ahead, again now with cash well into t he second half of 2022.

So, with that as an introduction, we go ahead now and hand the call over to Bradley Campbell, our President and Chief Operating Officer to further highlight the Galafold performance, our R&D timelines and the supply. Brad, go ahead please..

Thanks, John. And as we sit here and I think 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 strength and 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 we’ll give a global snapshot of the Galafold 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 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. And importantly, global compliance and adherence rates continue to exceed 90%.

The geographic breakdown of revenue during the quarter similar to 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 coming on to reimbursed products in Colombia and Romania and we do have more markets that we expect in the horizon later this year.

In this expanding global footprints, 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 phase of the COVID-19 pandemic and part of that was clearly strong momentum coming into the year after a great finish in 2019.

But as the COVID crisis has rolled through our various geographies, we’ve 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’ve 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 in telemedicine, a phenomenon that actually was highlighted in the recently released EURYDICE report which show that 50% of rare disease patients now have experienced some form of telemedicine which is up from 20% before the crisis.

And then finally, and I will provide more detail later, our supply chain remains fully intact and so our customers have confidence that they can access Galafold.

And all of that, we think has led to continued uptake and we’ve seen new patient starts all through the crisis even in the hardest hit countries including the UK, France, Italy, Spain, Japan United States and many others.

So, we think these dynamics may continue to provide some tailwinds even as the global pandemic inevitably may cause some headwinds, which will continue to close the monitor as the full impact of the pandemic currently remains unknown.

From a numbers perspective you can see that the first quarter sales, as I mentioned increased 78%, which does include a 3% negative impact from foreign exchange. And so, if you look at it from an operational performance perspective, sales actually increased by 81%, compared to the same quarter last year.

And on the left-hand side, here we do show the quarterly performance over the past several quarters and we do 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.

And as I have outlined previously to get to 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 do expect that will generate about $1 billion in cumulative revenue between 2020 and 2022 alone, which of course will go a long way towards 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, which is driven by continued diagnosis from high risk screening, newborn screening, other diagnostic initiatives, which we are now investing in as well.

And we have orphan exclusivity in the U.S. and Europe and other geographies, which alone take us to the end of 2020s in addition to our orange book listed patents that give us IT 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, we do want to highlight all the things that we are doing to ensure that continues supply chain, this isn’t something we’ve focused on before, but we will reiterate again here 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. So we don’t believe manufacturing will be an issue for us.

And as the COVID-19 pandemic became increasingly severe, we have successfully now pushed inventory as far as possible down the supply chain to the country and even to the pharmacy level. And again, this is made much easier by the fact that this is a small molecule with long stability and easy to ship around the world.

And similarly 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. So that – and that we had already been underway.

And now following the spread and impact of the COVID pandemic, we are taking similar actions to Galafold where we have now pushed 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, who is our manufacturing partner for ATB200 the biologics, has not had a single interruption to their manufacturing operations and we continue to successfully manufacture 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 diseases, you remember is the first ever second-generation therapy, as well as the first ever therapy for Pompe disease to receive breakthrough therapy designation.

There is a tremendous momentum behind what we believe maybe the next standard of care for a broad population of people living with Pompe disease, which does represent $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 advanced manufacturing to support by 2021 BLA and MAA filings. And importantly, we are reiterating the PROPEL study timelines are on track even in the phase of the COVID crisis.

And this is really thanks to the incredible efforts of our clinical team where we literally have a patient-by-patient plan that we’ve created along with our investigators to protect the integrity of the study.

We’d remind everyone that we did overenrolled the study which provides further confidence and importantly a little bit more color, as of today, we’ve administered 97% of the expected 2250 infusions up to this point, which is well within the design study parameters and we’ll continue to ensure that patients are getting their infusions.

Today, for the first time, we announced the US FDA has granted our rolling BLA submission of AT-GAA and we are on track to initiate that rolling BLA in the second half of this year, which will support a full approval under the fast track designation.

Additionally, in response to the many requests we are getting for compassionate use that we’ve received in particular 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.

And from a manufacturing perspective, we’ve successfully completed all three of the production campaigns including the Bioreactor Runs and now the related downstream purification activities under the PPQ process at WuXi, which will serve as the foundation for the CMC section for the BLA submission.

And importantly, as you know, this is a significant step forward in a major derisking event for this program.

We do 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 the most valuable franchise of the industry with a potential to offer solutions to all patients living with the Pompe disease globally.

Now on Slide 14, I will briefly highlight here our industry-leading portfolio of gene therapies for rare diseases and I would say, 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 of course, CLN6 and CLN3 Batten disease, as well as the Pompe and Fabry gene therapy in our earlier discovery programs as well again.

And starting with our Batten disease franchise and CLN6, as a reminder, we’ve previously reported positive interim data in our clinical study, as we think demonstrate meaningful impact of our AAV gene therapy in this extremely devastating form of Batten disease.

And this year, we do expect additional data from the CLN6 Phase 1, 2 study and we do plan to advance our regulatory discussions, so we can finalize the clinical and regulatory paths for that program.

We also believe that the initial CLN6 data provide important read-through for our clinical study in CLN3 Batten disease, which as you know is a most common form of childhood neurodegeneration and our plans year is to advance regulatory discussions to finalize that clinical and regulatory path as well as report additional data from the ongoing Phase 1, 2 study in the second half.

And 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. That fast track designation enforce us greater access to the agency for purposes of expediting drug development, review and potential approval.

And then finally, on Slide 15, let me just remind you of all the great things that are going on 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 Protein Engineering and Glycobiology experience, 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 50 plus diseases including eight that are currently in active preclinical programs. Also I’ll note that we announced several Amicus presentations to be presented at the American Society of Gene and Cell Therapy at their 23rd Annual Meeting which is being held virtually next week.

Our sponsored studies there have included one oral presentation and two posters and we would encourage you to take a look at some of the new data that we’ve provided in a virtual way through that conference.

But we do expect additional preclinical data this year in multiple of our gene therapy programs and we are continuing to guide to the disclosure of once two additional 90 candidates by year end. And with that, let me now turn the call over to Daphne, who can go through our financial results, our guidance and our financial outlook.

Daphne?.

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 the first quarter, we achieved Galafold revenue of $60.5 million, which is 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.

Cost of goods sold as a percent 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 the first quarter of 2020, as compared to $111.3 million for the first quarter 2019.

On a non-GAAP basis, total operating expenses were $116.7 million for the first quarter of 2020, as compared to $96.2 million in the first quarter of 2019.

The increase in research and development cost was primarily due to investment 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 the 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 fair value of contingent consideration and depreciation.

Net loss for the first quarter 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, again to emphasize, 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 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, CLN6, CLN3, Pompe and Fabry Gene Therapy programs and we continue to fully support the work with Jim Wilson and patents. 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.

And with that, let me turn the call back over to John closing remarks..

Great. Thank you, both Daphne and Bradley. So, as you can see, we have been relentless focused on performance across the business despite all of the unprecedented things and 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 Instructions] Your first question comes from the line of Anupam Rama with JPMorgan. .

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 is so longer than what some of us assume? Thanks. .

Sure. So, what we have communicated to all of our employees globally on upon this that, we are going to put their health, safety and well-being first and foremost. Thankfully, we’ve been able to operate Anup, is, through this telepresence mode really effectively.

So the greatest risk to the business frankly would be to rush back and to put people at risk. So we are not going to do that. We’ve said 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 the employees is that our return to any – of what we were before from a work cadence will be flexible and will be staggered and it will be an integration over a time.

Frankly, we’ve also 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 to really take the best practices, the best efficiencies including the cost savings efficiencies.

So, right now, that takes into account, as 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, looking at a reprioritization of our earlier stage preclinical programs and a further measuring of our capital expenditures.

Obviously, there is certain very evidence savings from the crisis itself, travel, entertainment expenses, the key part of the budget. Conferences, frankly, hiring as we’ve not – we’ve had very limited additional hiring to this period.

So, all of that taken together allow us to by a couple of quarters now extend the runway well into the second half of 2022, which puts us very close to that bridge to profitability ahead.

And if the crisis were to reemerge and we had to go back to kind of our shelter in place routine, but we don’t expect that that would have any negative bearing from an expense standpoint as well. Did that answer the question, Anupam? Okay, hopefully. .

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

Hey guys. Thanks so much for taking the questions.

Just on the manufacturing front for gene therapy, can you give us more color on how the scale-up is going for Batten and sort of the latest on your expectation for dosing more patients in terms – and using the commercial scale manufacturing and just any impacts you are seeing in terms of sort of supply or raw materials through the scale-up process just due to co-edge.

Thanks..

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

Apologies for that. .

No, no, that’s all part of working from home. It’s fine of course. So, Bradley, I’ll let you take that question and obviously, Jeff as well, please chime in. .

Yes. Sure. No, 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, which was how they are being manufactured with MCH and so that process is going very well with this platform that Brammer has a lot of experience with and we are well underway there.

The intention is that the next patients who will be dosed in CLN6 will be using that commercial material or the commercial platform I should say from Brammer and that’s on track for 2021.

In terms of your question around raw materials and inputs into the supply chain for gene therapy, likewise we had purchased study materials, plasmas, et cetera ahead of time and secured manufacturing suite time for all of our supply chain for gene therapy.

And so, we have had no interruptions so far and we don’t anticipate any interruptions in the gene therapy manufacturing or supply chain. .

Great. Thank you, Bradley. .

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

Good morning guys. Thanks for taking the question. 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 kind of around patients being hesitant to go into healthcare centers and that sort of being a tailwind for oral.

I guess, curious, are there any kind of like, leading indicators or things that in terms of patients engaging with you guys virtually on the website or anything like that that you guys are tracking, that you can talk about to see if that sort of trend or potential tailwind for a switch is continuing going forward?.

Great. Thanks, Whitney. I’ll ask Brad to comment in a moment, but again, 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.

Again, getting back to the natures and characteristics of the medicine, we think the fact that of course, that it’s an oral medicine that can be taken at home is yet one further benefit, particularly in this COVID-19 crisis and we’ve seen that play out now in a number of settings, in a number of geographies.

Though, not the key driver of this great performance but a factor.

Brad if you want to add more color and maybe how we think about it going forward in the months ahead?.

Yes. I think that’s well said John.

I think, you know, look, 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 and that – I mean, that strengths the foundation experienced now with the physicians and patients, it really sets us up for this success and strength going forward.

To your specific question around some of those dynamics around being all product, it’s interesting, outside the U.S., of course, we can’t have direct interaction with patients.

It’s really all through physicians, but we do have very close relationships with physicians and have done a good job ahead of time setting up the ability to interact with them virtually. It’s moved from instead of face-to-face meetings to text or phone call or video chat just as we all have.

And that’s how we hear of some of these really, I mean, encouraging anecdotes of physicians being able to meet some of their patients’ needs by moving them to the oral option and that’s both switch patients who may have been getting in the replacement therapy and again for the reasons I highlighted might want to pursue an oral option and for new patients.

We’ve had – we’ve continued to have new patient starts even in the hardest hit countries. So that system is working well. And again, as John said, that we think it provides us some incremental tailwinds which is exciting. In the U.S., where you can have a regulatory compliance interaction with patients more directly.

What we’ve seen is that, we actually have had more virtual patient meetings, which is really exciting and that seems to be a form in an environment that actually patients are drawn to. So we are going to look to continue to explore that.

And again, in the U.S., with our physicians and with some patients we do know of examples of where specifically patients that wanted to switch or start on Galafold because it’s an oral option. So, again, the important trends we will continue to watch them. We will continue to watch how the pandemic plays out, but so far, again great momentum in Q1. .

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

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 SEC superiority and within its naïve patient population? How would that compare to potential superior six minute walk data that you might generate? How are you thinking about commercializing a Pompe drug in each of COVID, just understanding the risk of this patient population to respiratory diseases.

And orphan drug launches are usually so incredibly high touch and you’ve got such a great team with patient relationships with Jayne and her organization within Amicus.

How are you thinking about the situation?.

Thanks, Ritu. I think, this launch like any other, but particularly in Pompe is going to be driven by the strength of the data. So the PROPEL study will be incredibly important. We’ve got a lot of data that we’ve already accumulated. Again, to remind everybody, the first patient treated with AT-GAA was just over four years ago.

And to remind everybody, we have a very unique dataset that I am not going to review at all here. At the moment, we’ve spoken to it at multiple scientific conferences where for AT-GAA we’ve seen profound impacts on people whether they were treatment-naïve or whether they were switching from the ERT standard-of-care.

So while we think it’s great, but there are other drugs in development. We think we have a very unique medicine that we have developed and we are going to let the clinical data speak for itself and ultimately, the clinical data will have to drive the success of the launch. We are well positioned, importantly with the PROPEL study.

We have dozens of sites in multiple countries around the world, really every key opinion leader is now getting experience with AT-GAA which we think will also help as we hopefully are able to launch this medicine around the world.

So I am not going to speculate as to what any other program may or may not show, ultimately, the success of the launch of AT-GAA will rest on the strength of the AT-GAA data. .

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

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 just given the slower progression of disease activity in this subtype, how sensitive are the endpoints are using? And where do you expect to detect the biggest impact versus natural history in CLN3 versus what you are seeing in CLN6?.

Sure, Joe. Thanks. I hope you are well. Again, it will be a limited dataset on a handful of patients who were treated at the low end and the high dose with CLN3. I am going to ask Jeff Castelli, our Head of Gene Therapy, just to comment a little further about the CLN3 program.

Again, this will be preliminary data with most of the patients to begin being treated in 2021 with the Brammer made commercial material that we think will be the basis of approval. But, Jeff, feel free to add any color..

Sure. Thanks, John. John noted, we will have the data later this year for CLN3 in the first handful of patients that we have enrolled. That would be one year of posting transfer data for most of those patients. CLN3 is slower progressive than CLN6, although it still does progress quite rapidly.

The main endpoints that we’ll be focusing on from an efficacy perspective is the unified Batten disease rating scale, which measures various aspects of motor function, language, cognition, vision. So, it’s a very diversified scale with lots of components. So, with lots of things to look at, but it will just be an early look at one year data.

And then, importantly, it will be an update on the safety data in this case as well. .

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

Great. Thanks for taking my question and very good morning. Hope everyone is staying safe out there. My question is regarding – so some of your – some of the companies in our coverage area have talked about a little bit of advanced purchasing, especially for orals in light of COVID.

Have you experienced anything like that for Galafold which could potentially – you need to keep in mind by year one and second quarter and subsequent quarters? Thank you. .

Great. Thanks, Mohit. Yes, the results, the 6$0.5 million that we reported today was driven by fundamental demand and kind of the natural rhythm of sales. Very, very minimal impacts that we could see from any advance work in the channel.

I’ll – and maybe Bradley and Daphne, if you want to add any more specifics to that?.

Yes. John, I think you said it well and I was careful in my intro comments to highlight that. So, first and foremost, it was strong patient demand and strong clients which we continue to see around the world which is great. We did have some favorable reimbursement dynamics as well, fewer bridge programs in the United States.

So, reauthorization went really well. So that’s all again underlying patient demand. As John said, there was very minimal contribution from some ordering patterns. There were few hospitals here and there and a few countries here and there who ordered a little bit more, but that was a minor contribution.

The fundamental driver of the quarter was lead that momentum, the patient demand and some of those reimbursement dynamics that I mentioned. .

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

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 range of pricing that we should assume. You do have the benefit of having another drug on the market, when that’s been around for a long time.

So, I am hoping you could share some of the learnings of maybe observations about – in terms of how big of a sales force you might need? And again, just your thoughts on pricing. And then I have a follow-up question. Thanks. .

Yes. Thanks, Tazeen. I’ll turn it over to Brad. I’ll just start with – again, we’ll apply the same principle that we did for Galafold as we roll for all of our medicines that they must be fairly priced and broadly accessible and that be a fundamental premise that we apply here.

And with that, Brad, if you want to talk about broadly how you are thinking about ensuring access for patients for AT-GAA once it’s hopefully on a path to launch?.

Sure. Thanks, John, Yes, thanks, Tazeen for the question. Yes, I think, we learned a lot from Galafold and I think the focus on access rather than a premium pricing, I think served us very well.

We focus on providing a great value proposition for both patients and physicians, of course, but also, to payers and that allowed us to go very quickly through the reimbursements, a launch cascade in Europe and really throughout the world. And so, we’ll apply those similar learnings here.

I do think there is some advantage to being kind of a second mover in the sense that, again like with Galafold, in the case of Fabry disease, here in Pompe, we don’t have to convince payers that Pompe is a serious disease that is worth reimbursing for a relatively high cost rare disease drug.

Here we have to show payers and physicians and patients that our value proposition is better than the alternative. And as John mentioned earlier, that will come down to the data, but of course, we feel very confident in our ability to demonstrate that based on the data we saw in Phase 2.

From an infrastructure perspective, there too, we are very successful with our – we think very efficient rare disease sales force. Of course, Sanofi is – has the Fabry drug as well as the Pompe alternative right now. And so, we understand kind of the ratio of our team to their team.

We think we have a smaller team out there in the field and that’s served us very well. And so we are confident that with really only very little additions to our global commercial organization, we will be able to successfully launch and commercialize this drug.

One other thing that I think is important if you think about how we were with Galafold launch versus where we’ll be with the AT-GAA launch, we’ll have a lot more patients on AT-GAA at the time of launch and we hope to be launching in the U.S., which is one of the largest markets early as well as the UK and Europe.

And so, we think that we’ll have an opportunity to access a much higher number of clinical trial patients earlier in the launch cascade than we did with Galafold. So, I think, for all those reasons, we feel well prepared and eagerly looking forward to seeing the data. .

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

Hey, good morning and congrats on the execution and thanks for taking the question. So, I understand the U.S. market is 29% of the revenue base.

But given the 33 million unemployment print this morning, and since we are all dependent on employer provided insurance, wondering what your current payer mix is and how much of the unemployment disruption is built into your $250 million to $260 million full year guide? Thank you. .

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

Yes, it’s a good question. So far, we’ve actually had a lower exposure to government payers from a mix perspective than we had anticipated. But we have great access programs for both private insurers, as well as government payers and frankly where patients can’t afford the drug, we provide free access to the drug.

And so, one of the things we’ve said is, we have never had a patient go through the process and be ultimately rejected by a payer. So we feel very good that patients are getting access to Galafold and have been – we haven’t seen any disruptions so far in patient access, even due to some of the unemployment.

Of course, you have to remember that, Fabry is a big population for us, but it’s a very small portion of the U.S. population. And so, those kinds of numbers that feel quite large from a U.S. unemployment perspective. The exposure in the Fabry population so far for us has been minimal. And so our guidance, I would say, remains the same where we sit here.

We are reiterating and feel very confident in the guidance. Of course, we’ll continue to monitor and should things change, we will provide those updates accordingly. But right now, we feel very good about where we are. .

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

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, you are seeing a 97% of infusions occurring on schedule.

Just curious if you are expecting any issues in terms of the data collection for that study or are there some steps you are taking to help mitigate that potential risk? Thanks. .

Yes. No. thanks, Mike, as Bradley indicated, we have a really custom patient-by-patient, site-by-site plan that we have implemented and the areas of focus are two-fold. One is to ensure that infusions happen and patients were able to access drug within the appropriate windows. And secondly are the assessments.

To remind everybody, we do assessments of course at baseline, but also 3, 6, 9 and then the 12 months assessments as well. So, we continue to ensure that patients are getting those assessments and that’s part of the confidence that we have in the integrity of the study.

In some cases, we’ve had to go to some pretty extraordinary lengths to make sure that those assessments are administered. But that’s an important part of maintaining the integrity of the study. So, we feel good about where we are there as well.

And I think that would continue with all the contingency plans we’ve put in place to ensure that we are able to collect the data appropriately. So we’ve not, thankfully, seen any interruption that would impact the integrity of the study. But it’s something we’ll continue to follow very closely. .

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

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?.

Yes. Happy to take that. Good question. So, the dynamics that we have there in highlighting through most of our calls are continuing kind of on track. And so, right now, we are about 65:35, two-thirds, one-thirds, switch versus naïve. So the naïve portion is slowly growing, which is what we would anticipate.

In most markets, the dynamic we see is that the initial uptake is stronger in the switch population and again, that’s logical because they are the ones that are coming in every other week into the healthcare system to get an infusion and so, they are the ones that are kind of readily available in the system.

And then, typically over time, we see more and more naïve patients come on the therapy. I will say that, in our more mature markets like Europe, the growth driver this year is 50:50 really between switch and naïve and even in some markets, we’ll start to tip towards more naïves than switches as we penetrate the existing market.

And again, that was anticipated that will really grow the market over time. And so, we will expect and we do expect in the three to five year timeline that we are driving significant growth in the treated market in Fabry disease.

So those trends continue and as I highlighted on the call, we are seeing in new patient starts both switch and naïve along the proportions that we’ve seen in previous quarters. .

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

Hey guys. I just wanted get back in with my commercialization question, just as far as launching into an Pompe population at risk or of respiratory diseases when COVID will be risk at the general population. Just how you are thinking about a virtual orphan drug launch assuming your data is got - approval is in 2021. .

Sure, Ritu. I will comment and then I’ll ask Brad to add some color. Again, given the extraordinary demand for a next-generation product in Pompe, I wouldn’t think that it has any impact on the launch ahead.

Of course, again, we are looking at a launch perhaps in late 2021 or early 2022 and hopefully the COVID crisis will have largely if not entirely passed by then. But with that in mind, I would really use the PROPEL study as a reference together with the infantile study that we just launched. Patients are seeking better treatment options.

The greatest risk is do not be on the best available therapy. So, again, these are physician, scientists are extremely motivated. The key opinion leaders across the world.

We’ve seen them go to great lengths just to maintain the integrity of the clinical study and I would think with further data and approvals, they would go to just as greater, even greater lengths to ensure that patients have access to a next – a potential next-generation therapy.

So, Bradley, do you want to maybe add any color or then even a bit more about how we are thinking about a global launch of AT-GAA?.

Yes. Thanks, John. Just a few more comments there. I think that was well said in terms of the demand and our ability to keep the supply chain intact and keep patients getting their infusions.

A couple other things that I would highlight, and one which I mentioned earlier which is, one great thing about the development program for AT-GAA is we will have quite a number of patients on AT-GAA at the time of launch and dozens of centers around the world and even more countries than we have with Galafold.

So if you think about kind of the rate of uptake with Galafold versus with AT-GAA, we have many more sites, many more patients and many more geographies who will be on AT-GAA at the time of launch, which I think gives you a good start.

I also would say, we are looking at a whole host of opportunities to think about what maybe on the other side of this pandemic. It’s very likely that the adoption and acceptance of virtual interactions will go up as we’ve all gotten used to operating in this world.

We expect our physicians and patients will as well, you may have heard in one of the previous questions, in the U.S. as an example we are doing more virtual patient meetings and that seems to be very, very strong and so we’ll continue to look at that. We are interacting with physicians in different ways.

We would never think that you would - that would substitute with direct interactions. But we are going to use this time to see how can augment that and really be a value-add to our customers through this process.

And I didn’t mention it before, but I will here, another important part of this is our case management team and our medical affairs team globally who work very closely with our supply chain to make sure that patients are answering the questions that they have, physicians are answering the questions that they have.

And again, that experience with Galafold which has been very strong will help us any kind of setting, whether it’s in a - this normal or the new normal or whatever it is that really strong support function that we’ve built up through the Galafold launch. We will continue and we think that will serve us well with the launch of Pompe. .

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

Hey. Thank you for holding back and again. So, with regard to the Pompe gene therapy IND, we have a little information regarding the, whether it’s liver directed or muscle directed et cetera.

How should we be thinking about auto – in terms of expression update, et cetera? And also, in some negative patients, if it’s not a liver directed gene therapy, how do you circumvent that? Thank you. .

Great. Thanks, Debjit. Yes, of course, have more data coming out at FDA Conference next week. And I am going to ask Hung to comment broadly about the technology approach that we are taking here. So, Hung, if you want to field that and Jeff, chime in if you have any other color..

Sure. I think that we have described at least in some detail that our Pompe gene therapy program will use a – mostly a broad distribute – tropic type of vector, which allows for transduction of not just liver, but also muscle and other tissues as well.

And so, we know that this particular vector, again this is a vector that was designed by – his group at Nupen and we know that it seems to transduce quite well and we don’t know – we don’t have any data to indicate that autopathy will not be a problem for really to transduce.

We know that that virus is able to deliver the gene and it’s able to get help of the – to basically transfer the gene pretty well and all the animal studies that we’ve done up to this point.

Jeff, do you want to comment anymore on that?.

Sure, Hung. Thanks. I think that’s a really good answer. The only thing I’d reemphasize is that, the approach we are taking with our tabs is in promoters will transduce the liver. It’s just that in addition to that liver transduction we will transduce other tissues like muscle that might have better durability.

So we would expect any tolerization that might come from the liver to also help in the case of – for instance a – negative incident that might have some immunogenicity to the actual GAA protein produced by the cell. .

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

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

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