Q1 2020 Earnings Call

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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. If you should require assistance during the conference, please press star, then zero on your touchtone phone.

Andrew Faughnan: As a reminder, this conference call is being recorded. I would like to turn the conference over to your host, Mr. Andrew Faughnan, Director of Investor Relations. You may begin. Good morning.

Andrew Faughnan: Thank you for joining our conference call to discuss Amicus Therapeutics' First Quarter 2020 Financial Results and Corporate Highlights. Speaking on today's call are 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 Team Therapy.

Andrew Faughnan: 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. However, our forward-looking statements should not be regarded as representations by us that any of our plans will be achieved. Any or all of our 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 that 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.

Andrew Faughnan: 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-Q 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?

Two to four looking statements and risk factor section.

<unk> I know report on form 10, Q. The year ended December 31st 2019, and the quarterly report conform 10 Q.

Quarter ended March 31st 2022, we filed later today with Securities and Exchange Commission.

At this time, it's my pleasure to trying to call over to John probably Chairman and Chief Executive Officer John.

Great. Thank you Andrew and welcome everyone to our first quarter 2020 result conference call, but let me just begin by stating that I hope every running their families are well unsafe I notice it.

And extraordinary time and it certainly has been for our amateurs team and for families.

Since we first announced that amick is would be moving through a work from home environment back on March 11th.

John Crowley: 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 11th, the senior leadership team and I at Amicus have 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 highlighting 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.

The senior leadership team and I had advocate for focus on two important theme.

Empathy and performance.

The core of empathy has been about protecting the safety health and wellbeing of our global Amick us workforce and by extension our families.

While emphasizing arrange your programs and initiative to protect and support our workforce.

We have also been relentlessly focused on inferring business continuity and performance against all of our key objectives.

So with that is background on today's call I'm pleased to be highlighting the significant progress we've made it damages.

During the first quarter and now interpret second quarter of this quite memorable year.

While adapting to all of the changes brought about by this world pandemic chronic is distorted twentytwenty has been a period of growth and execution.

John Crowley: So with that as background for today's call, I'm pleased to be highlighting the significant progress we've 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, CRAMACUS, the start of 2020, has been a period of growth and execution across our science. Clinical, 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'd like to highlight four key accomplishments.

Across our science.

Cynical regulatory in commercial effort.

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 didn't this morning's press release I'd like to highlight for key accomplishments.

Mmm.

Galafold continues it strong loans performance and remains the cornerstone of our success.

With $60.5 million in first quarter revenue the gala hold lawns continues to exceed expectations.

First quarter revenue represents the performance across the global business.

John Crowley: First, Yelapol continues its strong launch performance and remains the cornerstone of our success. Second, with $60.5 million in first quarter revenue, the Gallup Hold launch continues to exceed expectations.

Including new patients starts from both switch and treatment naive patient throughout the quarter in all major regions, including those hardest hit by covert 19.

Of course, we will continue to monitor any future impact from this pandemic, but we <unk>. We are encouraged by the trends that we are seeing.

John Crowley: 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 of 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 of ATGAA in late-onset Pompe disease to read out top-line data on schedule in the first half of 2021.

Second R.R. and D. timelines remain on track.

We continue to expect the phase three propelled study of A.P.T.A. in late onset pump pay disease to read out Puff blind date on schedule in the first half of 2021.

We disclosed also today that the U.S.F.D.A. has now authorized us to proceed with a rolling B.L.A.'s submission to this program.

And our plans remain to start rolling B.L.A. in the second half of this year.

Within our gene therapy pipeline, we continued to move forward or lead Baton disease program in C.L.N. six N.C.L.N. three.

John Crowley: We disclosed today that the U.S. FDA has now authorized us to proceed with a rolling BLA submission for this program, and our plans remain to start the rolling BLA in the second half of this year. In our gene therapy pipeline, we continue to move forward our lead batten disease programs in CLN6 and CLN3, as well as our most advanced preclinical gene therapy program. 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 and advance in place and the quick work early in this pandemic.

As well as are most advanced preclinical gene therapy program.

Third the amick as 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 drug to patients were 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 amick as therapy and potential therapies to patients in all geography or around the world.

John Crowley: We have been able to get the drug 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.

Enforce.

Maintaining the strength of our balance sheet remains a core priority for Amadeus.

We are announcing today that through a recent further financial exercise in a series of cost savings initiatives.

Or cast runway now leads us well into the second half of 2022.

Our team continues to carefully Shepherd, our financial resources as we continue on a path to profitability.

John Crowley: We are announcing today that through a recent further financial exercise and a series of cost savings initiatives, our CAST runway now leads us well into the second half of 2022. Our team continues to carefully shepherd our financial resources as we continue on a path to profitability. Daphne will discuss this in more detail later in the call.

Naval discuss this in more detail later in the call.

Turning dislike for we are well on track then to achieve our five key strategic priorities <unk>.

For 2020.

These include one.

<unk> precision medicine Prosep right.

We will continue to drive galafold to more patience living with separate disease with an animal variance in existing and didn't new markets.

John Crowley: Turning to slide four, we are well on track then to achieve our five key strategic priorities for 2020. These include one, Gallifold, our precision medicine for February. We will continue to drive Galliford to more patients living with rare disease with a minimal variance in existing and in new markets. We look to achieve global product revenue of $250 million to $260 million this year.

We look to achieve global product revenue with 250 million to $260 million this year.

Two we are increasing the clinical regulatory manufacturing and preclinical I'm I'm, sorry pre commercial activities.

Surrounding our palm pay program as we advance toward disapproval.

Three we are advancing our industry, leading rare disease gene therapy portfolio.

John Crowley: Two, we are increasing the clinical regulatory manufacturing and pre-clinical, I'm sorry, pre-commercial activities surrounding our Pompe program as we advance toward 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 the clinical development, manufacturing, and regulatory discussions for both our CLN-6 and CLN-3 BATN programs. Furthermore, in addition, we are progressing our pump-paging therapy toward 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 manufacture and scale up of the pump-aid gene therapy, as well as our other potential IND candidates, and we look forward to sharing the additional IND candidate from our Penn collaboration later this year.

Stemming from our new Global research and Gene therapy Center of excellence in Philadelphia, we will be advancing the clinical development manufacturing and regulatory discussions for both are Zealand six and C.L.N. three baton programs.

For in addition, we are progressing are pumping therapy towards I.N.D., and we will disclose up to two additional I.N.D. candidates this year.

A lot of work is underway with our manufacturing partners for the manufacturing scale up to the palm paid gene therapy.

As well as our other potential I.M.D. candidate.

And we look forward to sharing the additional anti candidates.

From our 10 collaboration later this year.

And again, we continue to maintain a strong financial position.

We carefully manage our expenses and investments and we remain fully funded through all major milestones ahead again now with cash well into the second half 2022. So.

So with that as an introduction, let me go hit now and and the call over to Bradley Campbell or President and Chief operator.

John Crowley: 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 with cash well into the second half of 2022. So with that as an introduction, let me 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 chain. So, Brad, please.

Operating officer to for the highlight the Galafold performance.

R.R. and D. time lines and the supply so <unk>.

Thanks, John and as we sit here I think what is our first truly virtual earnings call I'll. Let me just echo John statement I hope everybody out there is safe and well along with their families and your colleagues.

As jumps that I will go through a more detailed the galafold performance for the corner also r. and D. timelines in milestones and I will provide more detail actually we've taken to ensure the strengthen continuity with supply chains.

Bradley L. Campbell: Thanks, John. And as we sit here, in what I think 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.

But let me start on on slides six.

Which goes through the continued growth of Galafold for the first quarter and 2020, and we'll give a global snapshot both the Galafold commercial progress for the first quarter total product revenue was $60.5 million, which is an increase of 78% from the first quarter in 2019.

Bradley L. Campbell: As John said, I will go through in more detail the Gallup poll performance for the quarter, as well as R&D timelines and milestones, but I will provide more detailed actions we've taken to ensure the strength and continuity of the supply chain. But let me start on slide six, which shows the continued growth of Gallifold for the first quarter of 2020. And we'll give a global snapshot of the Gallifold commercial progress. For the first quarter, total product revenue was $60.5 million, which is an increase of 78% from the first quarter of 2019.

Well hold revenue has been driven by strong patients demand as well as continued strong adherence we've had some favorable reimbursement dynamics and there's been only minimal contribution from some ordering patterns as helped organizations like countries have adjusted to the impact of coping 19 virus.

Bradley L. Campbell: Household revenue has been driven by strong patient demand, as well as continued strong adherence. We've had some favorable reimbursement dynamics, and there's been only a minimal contribution from some ordering patterns as health care 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 what we've 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.

And importantly, global compliance and adherence rates continue to exceed 90%.

Did you graphic breakdown of revenue during the quarter similar to we've 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 corner. 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 patience for the first time this quarter coming on to reimburse product in Columbia in Romania, and we do have more markets that we expect them horizon later this year.

And this expanded global footprint is important not just to support the continued growth of patience with access to galafold, but it lays an incredibly strong foundation, which is highly leveraged able to support the anticipated launch upon pay and our future products as well.

Bradley L. Campbell: 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 reimburse the product in Colombia and Romania, and we do have more markets than we expect on the horizon later this year. And this expanding global footprint is important not just to support the continued growth of patients with access to Gallifold, but it lays an incredibly strong foundation that is highly leverageable to support the anticipated launch of Pompeii and our future products as well. Turning now to slide 7, we 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.

During notice like seven we have had a very strong Q1, even in the face of the covert 19 pandemic and part of that was clearly strong momentum coming into the year. After a great finish in 2019, but as the code crisis has rolled through or various geography is we've also seen some dynamics that we think could be unique.

Galafold as an oral therapy for SAP Ray in these very unique times. For example, we've seen some patients switched to galafold or start on galafold, because they've been reluctant to go into a hospital or clinical setting for alternative care that we've also seen our first patients prescribed galafold tell medicine phenomenon that actually was high.

Bradley L. Campbell: But as the COVID crisis has rolled through our various geographies, we've also seen some dynamics that we think could be unique to Gallup Hold as an oral therapy for Fabre in these very unique times. For example, we've seen some patients switch to Gallup Hold or start on Gallup Hold because they've been reluctant to go into a hospital or clinical setting for alternative care.

In the recently released Gerardus report, which showed it's 50 per cent of rare disease patients now have experienced some form of telling medicine, which is up from 20 per cent before the crisis and then finally and I'll provide more detail later, our supply chain remains fully intact and so our customers have confidence that they can access galafold.

Bradley L. Campbell: We've also seen our first patients prescribed Gallup Hold via telemedicine, a phenomenon that was highlighted in the recently released Urotis report, which showed that 50% of rare disease patients now have experienced some form of telemedicine, which is up from 20% before the crisis. And then, finally, our supply chain remains fully intact, and so our customers have confidence that they can access Gallup Hold. And all 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, the 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 we'll continue to closely 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 all that we think is legit competed uptake and we've seen new patient starts all through the crisis, even in the hardest hit countries, including the U.K., France, Italy, Spain, Japan, the United States and many others.

So we think these dynamics may continues to provide some tailwinds, even as the global pandemic inevitably may cause some headwinds, which will continue to closely 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 increase 78%, which does include a three per cent negative impacts from foreign exchange and so if you look at it from an operational performance perspective sales actually increased by 81 percentage 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.

Bradley L. Campbell: 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 nonlinear way, as we've seen in years past. So on slide eight, 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've outlined previously, to get to $500 million, we would expect about a five-year compound annual growth rate of 40% from 2018 to 2023, and we are well through that period now.

So slide eight now with several years or performance behind us. We can confidently say that we are on a path to that $500 million sales opportunity.

In 2023.

And as I've outlined previously to get to that 500 million, we would expect about a five year compound annual growth rate of 40 per cent from 2018 to 2023, and we're well through that period now we do expect that will generate about doing in dollars in cumulative revenue between 2020 and 2022 alone.

Which of course will go a long way towards funding, our r. and d. and objects over that period.

And we continue to have even further confidence in the billion dollar revenue opportunities peak as we continue to see significant <unk> sustained growth and the February March globally, which is driven by continue diagnosis from high risk screening newborn screening other diagnostic initiatives, which were now investing in as well and.

Bradley L. Campbell: We do expect that we'll generate about a billion dollars 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, and other diagnostic initiatives, which we're 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 the 2020s, in addition to our Orange Book listed patents that give us IT coverage into the late 2030s. So lots of opportunity and time to provide access to Galliford globally for a long period.

We have orphaned exclusivity in the U.S. in Europe, and other geography, which alone take us to the end of the 20 twenties. In addition to our Orange book listed patents that give us I.T. coverage into like 2030, so lots of opportunity in time to provide access to go home globally for a long period to come.

I'm slide nine to be doing a highly all the things that we're doing to ensure that continues supply chain <unk> isn't something we've focused on before but we will reiterate again here that are supply chain has maintained continue supplied during the cold in 19 pandemic from both Galafold and H.P.G.A. <unk>.

Bradley L. Campbell: On slide nine, we do want to highlight all the things that we're doing to ensure that continuous supply chain. This isn't something we've focused on before, but we will reiterate again here that our supply chain has maintained a continuous supply during the COVID-19 pandemic for both Galliford and ATGAA. For Galafold, part of our existing strategy had already been to hold multiple years of inventory and API and direct product, so we don't believe manufacturing will be an issue for us. And 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. And again, this is made much easier by the fact that this is a small molecule, so it has long stability and is easy to ship around the world.

Fold part of our existing strategy had already been hold multiple years of inventory A.P.I.N. drug product.

We don't believe manufacturing will be an issue for us.

And as a covert 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 was long stability and easy to ship around the world.

And similarly for H.P.G.A. or base clan had already been to build inventory ahead of our anticipated launch and to move drug product outside of China into the U.K.. So that endeavour had already been underway and now following the spread it impacted the code pandemic, we're taking similar actions to Galafold, where we've now pushed inventory.

Bradley L. Campbell: And similarly, for APGAA, our base plan had already been to build inventory ahead of our anticipated launch and to move drug products outside of China into the UK. So that endeavor had already been underway. And now, following the spread and impact of the COVID pandemic, we're taking similar actions with Galafold, where we've 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 ATP200, the biological, has not had a single interruption to their manufacturing operation, and we continue to successfully manufacture A In summary, we expect to be able to continue to supply Gallifold and ATGA in all of our global geographies for all of our patients living with Fabry and Pompe disease. Additionally, we do have sufficient inventory now of ATGA to complete all of our ongoing clinical studies in the POMPEI program with a high degree of confidence that the studies, including the Faith Street Health Study, will continue uninterrupted and be completed on schedule. And I'll go through that in more detail here in a moment.

Through the supply chain as far as possible and coordinating on a site by sight basis for delivery.

We would also note here that mushy, who was our manufacturing partner for a T.V. 200. The biological has not had a single interruption to their manufacturing operations and we continue to successfully manufacture 80 280, G.A. as we build commercial inventory.

In summary, we expect to be able to continues to supply galafold and A.T.J. and all of our global geography is for all of our patients living with <unk> disease.

And Additionally, we do have sufficient inventory now with V.T.G.A. to complete all of our ongoing clinical studies in the Palm pay program with a high degree of confidence that the studies, including the States Health study importantly will continue uninterrupted Andy completed on schedule and I'll go through that in more detail here in a moment.

So train out a slide 11, let me go through some of our R. and D. updates H.P.G.A. front palm pay disease, you'll remember is the first ever second generation therapy as well as the first ever therapy for pumping disease to receive break through therapy designation.

There's a tremendous momentum behind what we believe maybe next standard of care for abroad population of people living with pump day disease, which does represent a billion to $2 billion global opportunity.

Bradley L. Campbell: So turning now to slide 11, let me go through some of our R&D. ATGAA for Pompe disease, you'll remember, is the first ever second generation therapy as well as the first ever therapy for Pompe disease to receive breakthrough therapy designation. There's 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 billion to $2 billion global opportunity. We're on track, as John mentioned, to complete the Pompeii Phase III Propel Study on our original timelines to enroll the pediatric studies and advanced manufacturing to support a 2021 BLA and MAA filing. And importantly, we're reiterating that Propel study timelines are on track, even in the face 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 created, along with our investigators, to protect the integrity of the study. We'd remind everyone that we did over-enroll the study, which provides further confidence and, importantly, a little bit more color.

We're on track as John mentioned to complete the pump they phase three propel study.

On our original timelines two enrolled a pediatric studies and advanced manufacturing to support a 2021 delay in any a silence.

And importantly were reiterating that propelled study time lines ontrack, even if in the face of the Kobe crisis and this is really thanks being credible efforts of our clinical team, where we literally have a patients by patient plan that we've created along with our investigators to protect the integrity of the study.

We remind everyone that we did over the whole study provides further confidence and importantly, a little bit more color as up 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 there and <unk>.

Today for the first time, we announced that the U.S.F.D.A.'s granted are rolling B.L.A. submission the V.T.G.A. and we're on track to initiate that rolling delay and the second half of this year, which will support a full approval and fast track designation.

Bradley L. Campbell: As of today, we've administered 97% of the expected 2,250 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 that the U.S. FDA has granted our rolling VLA submission for ATGAA, and we're on track to initiate that rolling VLA 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're getting for compassionate use, that we received in particular for children with Infantile Octet 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.

<unk> in response to the many requests were getting for compassionate use now that we receive for in particular for children with infants highlights it upon pay disease or I.O.P.D.. 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 I, we've successfully completed all three of the production campaigns, including to buy rector runs and now the related downstream purification activities under the P.B.Q. process at machine, which will serve as the foundation for the C.M.C. section. So the B.L.A. submission and importantly, as you know.

Bradley L. Campbell: 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 WUSHI, which will serve as the foundation for the CMC section of the BLA submission. And importantly, as you know, this is a significant step forward in a major de-risking event for this program.

Oh this is a significant steps forward in a major d. risking eventually this program.

We do continue to be extremely excited about eight P.G.A. as well as are pretty clinical pumping gene therapy program to build that we believe could be the largest and the most valuable franchise in the industry with the potential to offer solutions to all patients living with pumped data is equally.

No one slide 14, I'll briefly highlight here our industry, leading portfolio gene therapies.

Bradley L. Campbell: We do continue to be extremely excited about APGAA, as well as our preclinical Pompe gene therapy program to build what we believe could be the largest and the most valuable franchise in the industry, with the potential to offer solutions to all patients living with Pompe disease globally. Now on slide 14, I'll 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 Pompeii and Fabry gene therapy in our earlier discovery programs as well at Penn.

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 are critical science and lead program activities across our gene therapy portfolio, including of course, Ceylon Six and Zealand three baton disease as well as the Palm paid in February gene therapy in earlier discovery.

Grams of well it as well at Penn.

Starting with their baton with these franchises dealing six as a reminder, we've previously reported positive interim data in or clinical study, we think demonstrates meaningful impacted or a gene therapy emus extremely devastating form of that disease and this year. We do expect additional data from the ceiling six days one to study and we do plan to.

Bradley L. Campbell: Starting with our BAT disease franchise in CLN6, as a reminder, we've previously reported positive interim data in our clinical study that we think demonstrates meaningful impact of our AAV gene therapy in this extremely devastating form of BAT disease. 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 CLM6 data provide important read-through for our clinical study in CLM3 batten disease, which, as you know, is the most common form of childhood neurodegeneration.

Dance or regulatory discussions so we can finalize clinical or regulatory path so that program.

We also believe that the initial ceiling six data provide important read through for a clinical study and ceiling three band disease, which as you know is the most common form of childhood Neurodegeneration and our plans this year or two advanced regulatory discussions to finalize that clinical and regulatory path as wells report additional data from the ongoing.

Phase one to study in the second half.

And importantly in recognition of its potential to address is significant unmet medical need our seal in three gene therapy program has been granted fast track designation by the U.S.F.D.A.

Bradley L. Campbell: And our plans this year are to advance regulatory discussions to finalize that clinical and regulatory path, as well as report initial data from the ongoing Phase 1-2 study in the second half. And importantly, in recognition of its potential to address a significant unmet medical need, our CLN3 gene therapy program has been granted fast-track designation by the U.S. FDA. That Fast Track designation affords us greater access to the agency for purposes of expediting the drug's development, review, and potential approval. And then, 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.

That fast track designation affords is greater access to the agency for purposes expediting the drums developments review and potential approval.

And then finally on 515, 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 growth for anarchist for decades to come.

This collaboration with the Wilson lab and combines advocates protein engineering and microbiology experience along with 10 World Class Gene transfer technologies to develop novel gene therapies designed for optimal cellular uptake targeting dosing safety and Manufacturability.

Bradley L. Campbell: 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 progress. Also, I'll note that we announced several Amicus presentations to be presented at the American Society of Gene and Cell Therapy, their 23rd annual meeting, which is being held virtually next week. Our sponsored studies there included one oral presentation and two posters, and we would encourage you to take a look at some of the new data that will be provided in a virtual way through that conference. We do expect additional preclinical data this year in multiple of our gene therapy programs, and we're continuing to guide to the disclosure of one to two additional IND candidates by year end. And with that, I now turn the call over to Daphne, who can go through our financial results, our guidance, and our financial outlook. Daphne?

As part of this collaboration we have rights to 50, plus diseases, including eight that are currently in active preclinical programs.

Also note that we announced several anikas presentations to be presented at the American Society of gene and sell therapy, there 23rd annual meeting, which is being held virtually next week or.

Are sponsored studies there have included one oral presentation into posters and would encourage you to take a look at something new data.

Slide it in a virtual way through that conference. We do expect additional pre clinical data this year and multiple of our gene therapy programs and we're continuing to guide to the disclosure of wants to additional 90 candidates by year end.

And with that let me know turn the called over to Daphne, who can go through our financial results or guidance and our financial outlook ethics.

Great. Thank you bribery and good morning, everyone. Our financial overview began on Friday 17, with our income statement for the quarter ending March 31st 2020.

First quarter, we achieved Galafold revenue 60.5 million.

78% increase over the first quarter of 2019.

Daphne E. Quimi: 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 Galliford 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 a 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 Percent of Revenue was favorable as Galliford's 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.

I think with year over year operational revenue growth measured at constant currency exchange rate of 81% offset by negative parents impact 3%.

<unk> the percentage of next sales with 10.8% in the first quarter as compared to 11.9% of the prior year period.

Costco gets old per cent of revenue with favorable as Galafold revenue continues to grow in the United States, where we do not own 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 of 2019.

On a now I got basic total operating expenses 416.7 million.

Quarter of 2020, as compared to 96.2 million in the first quarter of 2019 <unk>.

Daphne E. Quimi: Total operating expenses were $132 million for the first quarter of 2020 as compared to $111.3 million for the first quarter of 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 costs was primarily due to investment in our gene therapy programs for the initiation of the technology transfer to our contract manufacturers and clinical research within our early stage programs and within the POMPEI program to advance and enroll clinical studies. Our investment in research and development includes the impact of the implementation of the cost reduction measures that were previously announced, as well as the decrease in selling, general, and administrative expenses.

The increase in research and development costs with <unk> investment in our gene therapy program for the initiation at the tech transfer to our contract manufacturers.

And clinical research within our early stage program and within the Palm pay program to them in a rural critical studies.

Our investment in research and development increase the impact of the implementation of the cost reduction measures that were previously announced as guys to decrease in selling general and administrative expenses.

The non gap quarterly expense is expected to decline throughout 2020.

If I nine gap operating expense as research and development and selling generally administrative <unk>, excluding sharebased compensation extend.

Changes in fair value, a contingent consideration and appreciation.

Daphne E. Quimi: 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. The 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.

Net loss for the first player with 88.9 million or 35 cents per share as compared to the net loss of the hiring 20.3 million or 56 cents per share for the prior year period.

March 31st 2020, we had approximately 257 million shares outstanding.

Turning out to slide 18, I've 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 efficiency costs skating and careful extends management.

Daphne E. Quimi: 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 ATGAA launch and other products in our pipeline. We transition the costs associated with the development of ATGAA 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, Pompeii, and Fabry gene therapy programs.

I'm slide 19 going forward again, she emphasized we expect total non gap operating expenses and 2022 remain relatively flat from 2019 ugly leverage the commercial infrastructure that is already in place for the A.P.G.A. launch and other product in our pipeline.

We transition the costs associated with the development of H.P.G.A. to multiple gene therapy.

Programmed in our <unk> in our pipeline and maintain financial discipline, while meeting our objective.

To reiterate all high priority research programs in gene therapy are moving your head on schedule, especially C.O.N.

C.L.M. three <unk> therapy program.

And we continue to fully support to work with <unk>.

A few comments about our current cash position and 2020 financial data.

Cash equivalent in marketable securities for 339 million at March 31st 2020, compared to 453 million at December 31st 2019.

Daphne E. Quimi: And we continue to fully support the work with Jim Wilson and Penn. Here are a few comments about our current cash position and 2020 financial guidance. Cash, Cash Equivalents, and Marketable Securities of $339 million at March 31st, 2020, compared to $453 million at December 31st, 2019. We are reaffirming our full-year Galliford revenue guidance of $250 million to $260 million and our non-GAAP operating expense guidance of $410 million to $420 million. And with that, I will turn the call back to John for his closing remarks.

We are reaffirming our full year Galafold revenue guidance at 250 million to 260 million.

<unk> are non gap operating expense guidance, a 410 million to 420 million.

And with that let me trying to call back to John for closing remarks.

Great. Thank you, both Daphne and Bradley so as we can see we <unk> relentlessly focused on performance across the business. Despite all of the unprecedented teens and challenges brought about by the global pandemic.

We have a great global team that passionate entrepreneur, who have led and will continue to lead us through this and am competent that as the world emerges from this crisis unofficial emerged even stronger so operated with that we're happy to take any questions.

John Crowley: Great, thank you both Daphne and Bradley. So, as you can see, we have been relentlessly focused on performance across the business, despite all of the unprecedented change 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'm confident that as the world emerges from this crisis, Amicus will emerge even stronger. So, operator, with that, we're happy to take any questions.

<unk> Oh, let isn't doing them. If you have questions. Please for star and then the number one.

You know tests on phone at this time, we ask such we ask that you only ask one question. If you have additional questions. Please enter back into the queue.

If you a question has been answered or you wish to remove yourself from the queue.

Operator: Thank you, ladies and gentlemen. If you have questions, please press the star and then the number 1. At this time, we ask that you only ask one question. If you have additional questions, please enter back into the... If your question has been answered or you wish to remove yourself, Preston Pound, your first question comes to the line from Anupam Rama with J.P. Hey guys, thanks for taking the question and congrats on all the progress.

<unk>.

The first question comes on line of no more Rama what J.P. Morgan.

Hey, guys. Thanks for taking the question and congrats on all the progress.

I'm just a quick question on the cash only good guidance, which is now second half of 2022 I'm just wondering what's that assumes in terms of sort of.

Reopening here in the U.S. in globally due to the cold in 1910.

And making what are the full lovers, we should be thinking about on the expense I did it for whatever reason, there's a second wave infection independent make is allowing longer than.

Anupam Rama: Just a quick question on the cash runway guidance, which is now second half of 2022. I'm just wondering what that assumes in terms of sort of reopening here in the U.S. and globally due to the COVID-19 pandemic. What are the push-pull levers we should be thinking about on the expense side if, for whatever reason, there's a second wave of infection and the pandemic lasts longer, longer than expected? What some of us.

Some of us and soon thanks.

Sure. So what we've communicated to all of our employees globally on upon is that you never going to put their health safety and wellbeing first and foremost thankfully we've been able to operate anarchists through this tele presence load really effectively.

So there you know the greatest [noise] the greatest refers to the business frankly would be to rush back to put people at risk. So we're not going to do that you know we've said to our employees that June would be the earliest that they would be any reintegration back. We are now undergoing a project carefully evaluating that looking at all the local.

John Crowley: Sure, so what we've communicated to all of our employees globally on Apom is that, you know, we're going to put their health, safety, and well-being first and foremost. Thankfully, we've been able to operate Amicus through this telepresence mode really effectively. So, you know, the greatest...

John Crowley: And the greatest risk to the business would be...

Guidelines around the world in all the geography, as we work and so what I've emphasized to the employees is that our return to any semblance of what we were before.

John Crowley: So we're not going to do that. You know, 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've emphasized to the employees is that our return to any semblance of what we were before, working from a work cadence. We'll be flexible, and we'll be staffed, and It Will Be an Integration Over Time. Frankly, we've also taken this time to launch an internal initiative we're calling R3, or R3, where I've 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.

From a work cadence.

We'll be flexible and we'll be step.

<unk>.

And we'll be an integration over time.

Frankly, we've also taken this time to launch an internal initiative, recalling or three or cube.

I've asked all of our employees to rediscover Reimagine and reinvent <unk> 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 you know as we extend the runway to the second half or 2022 again. This was a multi week financial exercise across the entire organization looking at all of our functional operating expenses.

Looking at a reprioritization ever earlier stage preclinical programs and a further measuring ever capital expenditures.

John Crowley: So, right now, that takes into account, you know, as we extend the runway to the second half of 2022. Again, this was 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 measurement of our capital expenditures. Obviously, there are certain, you know, very evident savings from the crisis itself. Travel, entertainment expenses, the T&E part of the budget, conferences, and, frankly, hiring as we've not, we've had very limited additional hiring through this period. So, all of that taken together allows 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, you know, bridge to profitability ahead. And, you know, if the crisis were to re-emerge, and we had to go back to kind of our shelter-in-place routine, we don't expect that that would have any— any negative bearing from an expense Does that answer the question, Anupam? Okay, hopefully.

See there's certain you know very evident savings from the crisis itself travel entertainment expenses. The t. any part of the budget conferences frankly hiring as we've not we had very limited additional hiring through this period. So all of that taken together is allow us to buy a couple of quarters now.

Extend the runway well into the second half of 2022.

Which puts us very close to that you know bridge to profitability ahead.

And you know if the crisis where to re emerge.

And and we had to go back to kind of our shelter in place routine. We don't expect that that would have any.

<unk> any negative bearing from an expense standpoint as well.

That answer the question on upon.

Okay hopefully.

The next question comes a line of L.A. Merril with cats are fixed Gerald.

[laughter]. Thanks for taking the question from our manufacturing right for gene therapy can you give us more tolerant and you have the scanner, it's going for battles and sort of you know the list on your expectations I forgot to thing our patients <unk> anything be commercial scale manufacturing and and just any impact your c.

Operator: Your next question comes from the line of Ellie Merle with Cancer Fixed General. Hey guys, thanks so much for taking the time to answer our question. Just on the manufacturing front for gene therapy, can you give us more color on how the scale-up is going for batons and sort of, you know, the latest on your expectations for dosing more patients and using commercial scale manufacturing, and just any impact you're seeing in terms of sort of supply, buy, or raw materials through the scale-up process just due to COVID?

Being in terms of sort of stuff.

Hi, our raw materials for the scale that process just get a co ed. Thanks.

Sure I'm Gonna, let Brad handle that it seems like your dogs as excited as ours are about the out at this meeting.

It's about that but no no. That's all all part of working from home. It's fine of course, so badly I'll. Let you take that question, then obviously, Jeff as well please China.

Yes, or no so.

Bradley L. Campbell: Sure, I'm gonna let Brad handle that. It seems like your dogs are as excited as ours are about the Amicus News Series. I apologize about that.

Manufactured for gene therapy continues to go very well as you.

Asking your question I work in the middle of the Tech transfer activities to Brammer, who beat our manufacturer for a they see on six and see on three programs.

John Crowley: 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.

Bradley L. Campbell: Yes, sure. No, so... Manufacturing for gene therapy continues to go very well. As you asked in your question, we're in the middle of the tech transfer activities to Brammer, who will be our manufacturer for the CLM-6 and CLM-3 programs. And that is on the HyperStack platform, which is how they're being manufactured with NCH. And so that process is going very well. It's a platform that Brammer has a lot of experience with, and we're well underway there. The intention is that the next patients who will be dosed in CLM-6 will be using that, commercial material or 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 have purchased starting materials, plasmids, et cetera, ahead of time and secured manufacturing suite time for all of our supply And so we have had no interruptions so far, and we don't anticipate any interruptions in the gene therapy manufacturing or supply chain.

And that is in the hyper stack platform, which was where the out of there being manufactured with N.C.H. and so that process is going very well. It's a platform that bremmer has a lot of experience with and we're well underway there.

The intention is that the next patients who will be docent ceiling six will be using that.

Commercial material and the commercial platform I should say from grammar and that's on track for 2021 and in terms of your your question around raw materials and inputs into the supply chain for for gene therapy.

Wise, we had purchased starting materials plasma et cetera hadn't time and secured manufacturing sweet 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 in the gene therapy manufacturing or supply chain.

Yeah.

Great. Thank you Bradley.

Sure next question comes line of Whitney I, Jim what Guggenheim.

The mind guys. Thanks for taking a question wanted to put up on the galafold through the comments around the Galafold trends I guess that you're seeing so far in light of co then I'm kind of around patient being hesitant to go go into health care centres and that sort of being a talent for oral.

Bradley L. Campbell: Great. Thank you, Bradley.

Operator: Your next question comes from the line Whitney, IGN with Guggenheim. Good morning, guys. Thanks for taking the question.

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

Operator: I wanted to follow up on some of the comments around the Galliford trends that you're seeing so far in light of COVID.

Great. Thanks, Whitney I'll have spread to comment in a moment, but again just to emphasize we have really strong trends coming into 2020 before the curve at 19 crisis. So this great performance in Q1 was driven by fundamental patient demand again getting back to the nature and characteristics of the medicine.

Operator: , , , , , , ,

And we think the fact that of course that it's an oral medicine that can be taken at home is yet one further benefits, particularly in this coded 19 crisis and we've seen that play out now in a number of settings. The number of geography is so not the key driver of is great performance, but a factor Brad if you want to add.

Color and and maybe how we think about it going forward in the months ahead.

Yeah, I think that's well said John I think you know look we have been well it away and establishing gal folders in a courtroom treatment option in separate disease, and we think the s. treatment option for patients with amenable mutations and that I think that strength foundation experience now with the positions and patients really set 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 we can't have direct interaction with patients is really all through physicians, but we do have very close relationships with the with positions and have done a good job ahead of time setting up the the ability to interact with them virtually you know it's moved from instead of face to face meetings to text.

Phone call or video chat just as we all have and that's how we hear of some of these really you know <unk>.

Purging anecdotes of positions being able to meet some of their patients needs by moving them to the oral option and that's boats, which patients who may have been getting and that replaced with therapy and again for the reasons I highlighted.

Operator: Virtually or 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.

Operator: is continuing going forward.

Might want to pursue an oral option and for a new patients. We've had we've continued to have new patients starts even in the hardest hit countries. So that that system is working well and again as John said that we think provides us some incremental tailwinds, which was exciting in the U.S. you know 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 form in an environment that that actually patients are are drawn to so we're going to look to continue to explore that and again in the U.S. with our positions and with.

Some patients we do know of examples of where specifically patients at wanted to switch for star Galafold, because it's immoral option. So again important trends will continue to watch them will continue to watch how the the pandemic plays out, but but so far getting great and went up and coupons.

Thank you Sir there next question comes a lot of red too, but wrong with Cohen.

I don't thanks for taking the question I will ask about potential.

Pay a competition in sort of the commercial landscape coming up how well cheap wishing 18, <unk>. If you know the upcoming charms our own data.

John Crowley: Great. Thanks, Whitney. I'll ask Brad to comment in a moment.

P.C. superiority and and within it when you patient population holiday.

Are too.

Feel superior six minute walk they don't think you might generate how are you thinking about commercializing pumping draws in Egypt called they just understanding a real patient preparation tools respiratory diseases and you know orphan drug lunches are usually so incredibly high touching.

That's a great team with like patient relationship with fun game on her organization or with them on okay.

How are you thinking about the situation.

Thanks for two you know I think [noise]. This launch like any other but it could really and pumped pay is going to be driven by the strength that the data. So that propelled study will be incredibly important we've got a lot of data that we've already accumulated a again to remind everybody. The first patient treated with A.T.G.A. was just over four years ago.

Bradley L. Campbell: But again, just to emphasize, we had 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 nature 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 and geographies. So 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.

Bradley L. Campbell: Yeah, I think that's well said, John. I think, you know, we have been well underway in establishing Galifold as an important treatment option in febrile disease and we think the best treatment option for patients with amenable mutations, and I think that our strong foundation experience now with physicians and patients really sets us up for this success and strength going forward. To your specific question around some of those dynamics around being an oil product, you know, it's interesting outside the U.S., 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.

And you know to remind everybody we have a very unique data set that I'm not gonna review at all here at the moment, we've spoken to it it multiple scientific conferences rear you know for A.T.G.A., we've seen profound impacts on people, whether they were treatment naive or whether they restrict switching from the ear.

Bradley L. Campbell: You know, 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, you know, emerging anecdotes of physicians being able to meet some of their patients' needs by moving them to the oral option. And that's both for switch patients who may have been getting enzyme replacement therapy and, again, for the reasons I highlighted, might want to pursue an oral option, and for new patients.

Bradley L. Campbell: 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 provides us with some incremental tailwinds, which is exciting. In the U.S., you know, where you can have a regulatory-compliant 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 forum and an environment that patients are actually drawn to. So we're 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 where specifically patients have wanted to switch or start on Galifold because it's an oral option.

Operator: So, again, important trends. We'll continue to watch them. We'll continue to watch how the pandemic plays out. But so far, again, great momentum in Q1.

Standard of care, so well, we think it's great that there are other drugs in development. We think we have a very unique medicine that we have developed and we're 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 their <unk>.

Study, yeah dozens of sites in multiple countries around the world really every key opinion later now getting experience with a T.G.A., which we think will also help as we hopefully are able to launch this medicine around the world. So I'm not going to speculate as to what any other program may or may not show ultimately.

The success of the launch.

Do they T.G.A. will rest on the strength of the A.T.G.A. data.

Your next question comes a lot of Joseph source with S.V. Lee rink.

Oh, great. Thanks, so much I was wondering if you could talk about the extent of Ceylon three data.

Expect to report later this year.

Just given the slower progression of disease activity in this subtype, how sensitive on them quencher, using and where do you expect to detect the biggest impact versus natural history and see on three versus what you've seen and see on six.

Ritu Subhalaksmi Baral: Thank you, sir. Your next question comes on the line from Ritu Baral with Cohen. Hi guys.

Sure a Joe things I hope, you're well again, it'll be a limited data set on a handful of patients who retreated at the low end and the high goes with C.L.N. three I'm Gonna asked Jeff gets Kelly or head of gene therapy, just a comment a little further about the the L.N. three program again this'll be preliminary data.

With most of the patients to begin being treated in 2021 with the grammar made commercial material that we think will be the basis of approval, but just feel free to add any color.

Short things drawn you know John noted we will have the data later this year for sale and three in the first handful patients that we've been road that would be one year coasting transfer data for most of those spaces.

Ceiling three is lower progressive in Salem, six although it's still does progress quite rapidly.

And and point that will be focusing on from an advocacy perspective unified baton 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 a lot the different things to look at but it will just be an early look at one year data and then importantly, it'll be an update on the safety data in as good as well.

The next question comes a line of <unk>.

With city group.

Great. Thanks for taking my question ready good morning, cope have even distinctive Oh dead. My question is <unk>. So some of you have some of the company than us coverage area.

John Crowley: 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 ATGAA if, you know, the upcoming Genzyme data hits FBC superiority within its naive patient population? How would that compare? potential superior six-minute walk data that you might generate. How are you thinking about commercializing a Pompe drug in the age of COVID, just understanding the risk of this patient population for respiratory diseases? And, you know, orphan drug lunches are usually so incredibly high-touch, and you've got such a great team with, like, patient relationships with Jane and her organization within Amicus. How are you thinking about this situation?

Talked about.

It also advised by chasing specially foot or in the light of cool <unk> have you experienced anything like that <unk>.

<unk> you need to keep keep in mind by you have money checking platoon subsequent quadras. Thank you.

Great thing smell it yeah. The results that 60.5 million that would result reported today was driven by fundamental demand and kind of the natural rhythm sales very very minimal impact that we could see from any advanced work in the channel all maybe Bradley in Daphne if you want to add any more.

Depicts to that.

Yeah, John at that 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 compliance, which would continue to see around the world, which is great. We did have some favorable reimbursement dynamics as well fewer bridge programs the United States.

Reauthorization went really well so that's all again underlying patients demand as John said, there was very minimal contribution from some ordering patterns. There were a few hospitals here and there and a few countries here and there who who ordered a little bit more but that was a minor contribution the fundamental a driver of a quarter was really that momentum.

The patient demand in some of those reimbursement dynamics that I mentioned.

Thank you go next question comes a lot of Tenzing Ahmad with Bank of America.

Good morning.

And one for fraud.

That's true.

About the pumpkin market, maybe specifics about ranges type thing that we should yeah. You do have the benefit of of having another truck on the market and when that's been around for a long time. So I'm, hoping you can share some of the learning.

The observations about about my design in terms of how big of a of a self worth you might need and again just your thoughts on pricing and then I've the question.

Yeah, things Dizzy and I'll turn it over to Brad I'll, just start with like again will apply the same principle that we did four galafold as we will for all of our medicines that they must be fairly priced and broadly accessible and that would be a fundamental premise that we apply here in with that Brad if you want to talk about broadly.

Operator: Thanks, Ritu. You know, I think this launch, like any other, but particularly in Pompeii, 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 gathered. Again, to remind everybody, the first patient treated with ATGAA was just over four years ago. And, you know, to remind everybody, we have a very unique data set. I'm not going to review it all here.

How we're thinking about ensuring access for patients.

John Crowley: At the moment, we've spoken about it at multiple scientific conferences where, you know, for ATGAA, we've seen profound impacts on people, whether they were treatment naive or whether they were switching from the ERT standard of care. So while we think it's great that there are other drugs in development, we think we have a very unique medicine that we have developed. And we're 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 ATGAA, which we think will also help as we, hopefully, are able to launch this medicine around the world. So I'm not going to speculate as to what any other program may or may not show. Ultimately, the success of the launch will rest on the strength of the ATGAA data.

For a T.G.A. wants it to hopefully on a path to launch.

Yeah. Thanks to the answer the question Yeah. I think you know we learned a lot from Galafold and I think the the focus on access you know rather than a premium pricing having served us very well, we focused on providing a great value proposition for both patients in positions of course, but also to payers.

And that allowed us to go very quickly through the reimbursements.

Launch Cascade in Europe in really throughout the world and so will apply those similar learnings here I do think there's some advantage to being kind of a second mover in the sense that you know again like with Galafold indicates a separate disease here in palm pay we don't have to convince payers that palm pays a serious disease.

That is worse reimbursing for you know relatively high cost a rare disease drug here, we have to show payers and physicians and patients that are value proposition is is you know better than the alternative in as John mentioned earlier, you know that's that would come down to the data but of course, we feel very confident.

In our ability to to demonstrate that based on the data we saw in in phase two from an infrastructure perspective, you know there too we are very successful with our we think very efficient a rare disease sales force of course Sanofi is has the the.

Right drug as well as the palm pay alternative right now and so we understand kind of the ratio heart team to their team. We think we have a smaller team out there in the field and that served us very well and so we're confident that with really only very little additions to our global commercial organization, we will be able.

To successfully launched commercialize this drug one other thing that I think is important if you think about you know how we were with Galafold launch versus where we'll be with the A.T.G.A. launch we'll have a lot more patients on H.T.G.A. at the time of launch and we hope to be you know.

Launching in the U.S., which is one of the largest markets early as well you K. in 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 with Galafold. So I think for all those reasons, we feel well prepared and eagerly looking for to seeing the data.

The next question comes a line of digits.

Chopped up with.

<unk> Wainwright.

[noise] pay good money and talking about some execution and thanks for taking the question. So I understand the U.S. market, it's 29% of the revenue base, but given d. 23 million unemployment print this morning, and since fit all dependent on unemployed provide an insurance wondering what's your current affair mix is and how much of the unemployed.

I'm in disruption is embedded into you up to 50 to 60 million for your guide. Thank you.

Great things Debjit I'll ask bred to comment on the pay or mix and how we're thinking about that.

Yeah. It's a good question so far we've actually had a lower exposure to government paid pairs from a mixed perspective than we had anticipated, but we have great access programs for both private insurers as well as government payers and.

Ugly where patients can't afford the drug we provide free access to the drugs and so one of things we've sat as you've never adaptation go through the process and be ultimately rejected.

By a pair and so we feel very good that patients are getting access to galafold and have been we haven't seen any disruption so far in in a patient access even due to somebody and unemployment of course, you have to remember that.

Separate is big population for us, but it's a very small portion of the the U.S. population and so those kinds of numbers that you know seal quite large from from the U.S. unemployment perspective, the exposure in the fabric population so far for us as and you know minimal and so are.

Are guidance and I would say remain the same where we sit here. We are reiterating you feel very competent the guidance of course will continue to monitor and and should things change. We'll we'll provide those those updates accordingly, but right now we still very good about where we are.

Your next question comes a lot of Mike also put bird.

You guys think sort of taken the question then congrats on the strong quarter as well just a quick question for you on the propel study seems like things are progressing fairly well there. Despite covert 19. Your thing you know 97% of infusions occurring on schedule just carry.

Joseph Patrick Schwartz: Your next question comes to the line of Joseph Schwartz with SVB-Lead. 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 you're using, and where do you expect to detect the biggest impact versus natural history in CLN3 versus what you've seen in CLN6?

John Crowley: Sure, Joe, thanks, I hope you're well. Again, it'll be a limited data set on a handful of patients who were treated at the low and then the high doses of CLN3. I'm gonna 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.

Jeffrey P. Castelli: Sure. Thanks, John.

If you're expecting any issues in terms of the data collection for that study or there's some steps you're you're taking the help mitigate potential risk. Thanks.

Jeffrey P. Castelli: You know, as John noted, we will have data later this year for CLN3 and the first handful of patients that we've enrolled. That would be one year post-gene transfer data for most of those patients. CLN3 is slower progressive than CLN6, although it still does progress quite rapidly.

Jeffrey P. Castelli: The main endpoint 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, and vision. So it's a very, you know, diversified scale with lots of components. So we'll have lots of different things to look at, but it will just be an early look at one-year data. And, importantly, it'll be an update on the safety data in this case as well. Your next question comes on the line from Mohit Bosnalt with Citi. Great. Thanks for taking my question, and very good morning. I hope everyone is staying safe out there. My question is regarding, so some of your companies and some of the companies in our coverage area have talked about a little bit of advanced purchasing, especially for orals in the light of COVID. Have you experienced anything like that for Galliford, which could potentially, we need to keep in mind while we are modeling second quarter and subsequent quarters? Thank you.

Mohit Bosnalt: Great. Thanks, Mohit. Yeah, the results, the $60.5 million that we reported today was driven by fundamental demand and kind of the natural rhythm of sales. Very, very minimal impact that we could see from any advanced work in the channel, maybe Bradley and Daphne, if you want to add any more specifics to that.

John Crowley: Yeah, John, I think you said it well, and I was careful in my intro comments to highlight that.

Bradley L. Campbell: So first and foremost, it was strong patient demand and strong compliance, 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.

Bradley L. Campbell: The fundamental driver of the quarter was really that momentum, patient demand, and some of those reimbursement dynamics that I mentioned.

Yeah no. Thanks, Mike is Bradley indicated we have a really custom patient by patients side by side plan that we've implemented in the areas of focus are twofold. One is to ensure that infusions happen in patients were able to access drug within the appropriate windows and secondly, or the assessments.

To remind everybody we do assessments of course at baseline, but also the three six [noise].

Benign and then the 12 month assessments as well. So we continued to ensure that patients are getting those assessments and that's part of the competence that we have in in the integrity of the study in some cases, we've had to go to some pretty extraordinary lengths to make sure that those [noise].

Excuse me 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 to be put in place to ensure that we're 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 will continue to follow very close.

Yeah.

Thank you.

Next question <unk> Richter with Goldman Sachs.

Thanks for taking a question <unk>, maybe one question <unk> old catchy provide updated that check on the flip between such a nice patients on how this patient next day, perhaps tracking with your expectations, but then the context doesn't even established my guess.

Operator: Thank you. Good morning. Thanks for taking my questions. One for Brad.

Tazeen Ahmad: Can you just walk us through how you're thinking about the Pompei market, maybe specifics about the range of pricing that we should assume?

Yeah.

Yeah happy to take that and good question. So the dynamics that we've been highlighting through most of the recalls are continuing kind of on track and so right. Now we're about 65 35, two thirds, one third switch versus naive. So the naive portion is slowly growing which is what we what we would.

Anticipate in 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're the ones that are coming in every other week into the healthcare system to get an infusion and so they're they're the ones that are kind of readily available in the system and then typical.

The overtime, we see more and more naive patients come under therapy.

I will say that in our more mature markets like Europe. The growth driver. This year is is 50 50 really between switch and I, even and even in some markets will start to tip towards more naive sense witches as we'd penetrate the the existing market and again that was anticipated that will really grow the market over time and so.

We will expect than we do expect you know in the three to five year timeline that we are driving significant broken treated market in separated so.

So those <unk> trends continue and as I highly on the call. We are seeing and new patients starts both switch and naive along the the proportions that that we've seen in previous quarters.

Thank you you have a full of of course I'm from the line of Red to borrow with Cohen.

Hey, guys I just wanted to get back in with my my commercialization question, just as far as launching in launching <unk> pump population opera Wow <unk>.

Bradley L. Campbell: You do have the benefit of having another drug on the market and one that's been around for a long time. So I'm hoping you could share some of the learnings or maybe observations about lumizyme.

Something of Restful General population, just hung up until about a virtual or from truck lunch, assuming that approval isn't 21.

Bradley L. Campbell: 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.

Sure It to all comment and then I'll ask bred to add some color again, given the extraordinary demand for a next generation product in <unk>, Hey, I wouldn't think they didn't have any impact on the launch ahead of course again, we're looking at the launch perhaps in late 21 or early twenties.

Two and hopefully the the covert crisis will have largely if not entirely passed by then I, but with that in mind I would really use their propel study as a reference together with the infant tell study that we just launched patients are seeking better treatment option. The greatest risk is to not beyond the best available therapy.

So you know again these are positioned 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 a clinical study and I would think with further data and approvals. They would go to justice greater even greater length to ensure that patients have access to next.

Potential next generation therapy. So badly if you want up maybe at any color or where then even a bit more about how we're thinking about a global lawn chair or they T.G.A.

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

A couple other things that I would I would highlight one of which I mentioned earlier, which is you know one great thing about the development program for for a C.G.A.'s, we will have quite a number of patients on eight T.J. at the time of launched and dozens of centers around the world and even more countries than we have with Kielholz. So if you think about.

Bradley L. Campbell: Yeah, thanks, Tazeen. I'll turn it over to Brad. I'll just start with, again, we will apply the same principles that we did for Gallifold, as we will for all of our medicines, that they must be fairly priced and broadly accessible. And that would be a fundamental premise that we apply here. And with that, Brad, if you want to talk about broadly how we're thinking about ensuring access for patients to ATGAA once it's, hopefully, on a path to launch. Thanks, Jeff.

Bradley L. Campbell: and I think the focus on access, you know, rather than premium pricing, served us very well. We focused 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 reimbursement and launch cascade in Europe and really throughout the world. And so we'll apply those similar learnings here.

Bradley L. Campbell: I do think there's some advantage to being kind of a second mover in the sense that, you know, again, like with Galliford in the case of Fabry disease, here in Pompeii, we don't have to convince payers that Pompeii is a serious disease that is worth reimbursed for a relatively high-cost rare disease drug. Here, we have to show payers, physicians, and patients that our value proposition is, you know, better than the alternatives. And as John mentioned earlier, you know, that will come down to the data.

The rate of uptake with Galafold versus with A.T.G.A. I think we have many more sites. Many more patients in many more geography is who will be on a T.J. at the time at launch, which I think gives you gives you a good start I I also would say we are looking at a whole host of opportunities to think about what may.

Maybe on the other side of this pandemic you know, it's very likely that the adoption and acceptance of virtual interactions. We'll go up a you know as we've all gotten used to to operating this world, we expect our physicians and patients will as well.

Bradley L. Campbell: But, of course, we feel very confident in our ability to demonstrate that based on the data we saw in phase two. From an infrastructure perspective, you know, there, too, we are very successful with our, we think, very efficient rare disease sales force. Of course, Sanofi has the Fabry drug as well as the Pompeii alternative right now, and so we understand kind of the ratio of our team to theirs. We think we have a smaller team out there in the field, and that served us very well.

Bradley L. Campbell: And so we're confident that with really only very few 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 Galliford launch versus where we'll be with the APGA launch, we'll have a lot more patients on APGA 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 U.K. 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 Galliford. So I think for all those reasons, we feel.

Operator: 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, I'm 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.

You may have heard on one of the previous questions in the U.S. as an example, we're doing more virtual patient meetings and that seems to be very very strong and so will continue to look at that we're we're interacting with physicians in different ways. We would never think that you would that would substitute with direct interaction, but we're going to use this time to see how we can.

Augment that and really be a value add to our customers through this process and I didn't mention it before but I, but I will hear another important part of this is our case management team and our medical fairs team globally, who work very closely with our supply chain to make sure that patients are answering the questions. They have.

Positions are answering the questions they have and again that experience with Galafold, which has been very strong will help us in any kind of setting whether it's in you know you know this normal or the new normal or whatever it is that you know really strong support function that we built up through the Galafold launch will continue and we think.

That will serve as well with with the launch of <unk>.

Your next question is a follow up from that job with H.C. Wainwright.

[noise]. They thank you for living back in again, so with regard to the bumping into the I.N.D.

A little information regarding the you know whether it's limited about their muscles cetera, I shouldn't be thinking how should we be thinking about all buffet g. in terms of expression uptake et cetera, and also you know income negative <unk>, if it's not a liver deducted gene therapy, how do you circumvent that thank you.

Great. Thank <unk>, Yeah of course have more data coming out at the conference next week them and ask hung to comment a broadly about the technology <unk>. Your so hung if you're going to feel that and and gesture.

Any other color.

Sure you know I think that we have described at least in some detail that are 18 therapy program will use a.

Deb: Great. Thanks, Deb. I'll ask Brad to comment on the payer mix and how we're thinking about that.

Bradley L. Campbell: Yeah, it's a good question. So far, we've actually had a lower exposure to government payers from a mixed 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 that we've never had a patient go through the process and be ultimately rejected by a payer. And so, we feel very good that patients are getting access to GALFOLD and have been. We haven't seen any disruption so far in patient access, even due to some of the unemployment. Of course, you have to remember that FABRAE 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 FABRAE population so far for us has been minimal, and so our guidance, I Where we sit here, we're reiterating and feel very confident in the guidance. Of course, we'll continue to monitor, and should things change, we'll provide those updates accordingly, but right now, we feel very good about where we are.

Operator: Your next question comes from the line of Mike Oldsford-Baird. Hey guys, thanks for taking the question and congratulations on the strong quarter as well.

Mike Oldsford-Baird: Just a quick question for you on the PROPEL study. Seems like things are progressing fairly well there. Despite COVID-19, you're seeing 97% of infusions occurring on schedule. Just curious if you're expecting any issues in terms of the data collection for that study, or are there some steps you're taking to help mitigate that potential risk? Thanks.

John Crowley: As Bradley indicated, we have a really custom patient-by-patient, site-by-site plan that we've implemented. And the areas of focus are twofold.

John Crowley: One is to ensure that infusions happen and patients are able to access the drug within the appropriate windows. And secondly, there are the assessments. To remind everybody, we do assessments, of course, at baseline, but also 3, 6, 9, and then the 12-month 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 will continue with all the contingency plans we've put in place to ensure that we're 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.

Salveen Jaswal Richter: Yeah, happy to take that. Good question.

Operator: Thank you. Your next question comes from the line of Salveen Richter with Goldman Sachs. Thanks for taking our questions. This is Andrea on behalf of Salveen. Maybe one question for Brad. With respect to GALFOLD, could you provide updated metrics on the split between the switch and naive patients and how this patient mix is perhaps tracking with your expectations within the context of new and established markets?

Bradley L. Campbell: The dynamic that we've

Mostly abroad, you know distribute tropic type of sector, which allows for transcription of not just liver, but also bustle with other.

Bradley L. Campbell: I've been highlighting through most of our calls are continuing kind of on track, and so right now, we're about 65, 35, or two-thirds, one-third switch versus naive. So the naive 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're the ones that are coming into the healthcare system every other week to get an infusion, and so they're the ones that are kind of readily available in the system. And then, typically, over time, we see more and more naive patients come on to therapy. I will say that in our more mature markets, like Europe, the growth driver this year is really 50-50, really, between switch and naive.

Bradley L. Campbell: And even in some markets, we'll start to tip towards more naives than switches as we penetrate the existing market. And again, that was anticipated; we'll really grow the market over time. And so we will expect, and we do expect, you know, in the three- to five-year timeline that we are driving significant growth in the treated market for febrile disease. So those trends continue, and as I highlighted on the call, we are seeing in new patient starts both switch and naive along the proportions that we've seen in previous quarters.

Tissues as well.

And so we know that this particular vector you know again. This is the better it that was designed by a journalist scoop at U. Penn and we know that it seems to do was quite well and we don't know no.

And we don't have data indicate that you know a topic will be a problem for <unk> no that that viruses able to deliberate scheme and be able to get out of the in the so too.

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Yep, you what kind of comment the movement.

Sure hung thing Yeah, I think those are really good answer the only thing I'd I'd Reemphasizes that you know the approach, we're taking with our cops isn't promoter as well certainly trends do sliver. It's just that in addition to that live or trying to talk some more trends to other tissues like muscle that might have better durability. So we would expect any you know.

<unk> that might come from the labor to also help in the case for instance, a crumb negative and that might have some I mean engineer today to the actual G.A. protein produced by itself.

Thank you.

At this time I like this on a conference <unk>, chairman and C.E.O. for closing remarks.

Great. Thanks, operator that was a great conference call. Thanks for all the terrific questions I hope everybody in your family it stays well unhealthy integrate that.

Ladies and gentlemen. This US include today's conference. Thank you and have a great Dane.

Bradley L. Campbell: Thank you. You have a follow-up question from the line of Ritu Baral on Cohen. Hey guys, I just wanted to get back in with my commercialization question, just as far as launching into an at-risk Pompei population at risk for respiratory diseases when COVID could still be a risk in the general population, just how you're thinking about a virtual orphan drug launch, assuming your data is good and approval is in 2021.

Ritu Subhalaksmi Baral: Sure, Ritu, I'll comment, and then I'll ask Brad to add some color. Again, given the extraordinary demand for a next-generation product in Pompeii, I wouldn't think that it would have any impact on the launch ahead. Of course, again, we're looking at a launch perhaps in late 21 or early 22, and hopefully, the COVID crisis will have largely, if not entirely, passed by then. With that in mind, I would really use the PURPEL study as a reference. Together with the infantile study that we just launched, patients are seeking better treatment options. The greatest risk is to not be on the best available therapy. Again, these are physician scientists that are extremely motivated, the key opinion leaders across the world. We've seen them go to great lengths just to maintain the integrity of a clinical study, and I would think with further data and approvals, they would go to just as great or even greater lengths to ensure that patients have access to a potential next-generation therapy. Bradley, do you want to maybe add some color or even talk a bit more about how we're thinking about a global launch of ATGAA?

John Crowley: Yeah, thanks, John. Just a few more comments there.

Bradley L. Campbell: 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, one of which I mentioned earlier, which is, you know, one great thing about the development program for ATGAA is that we will have quite a number of patients on ATGAA at the time of launch and dozens of centers around the world and even more countries than we had with Gallup Hold. So if you think about the kind of rate of uptake with Gallup Hold versus ATGAA, I think we have many more sites, many more patients in many more geographies who will be on ATGAA at the time of launch, which I think gives you a good start.

Bradley L. Campbell: I also would say we are looking at a whole host of opportunities to think about what may be on the other side of this pandemic. You know, it's very likely that the adoption and acceptance of virtual interactions will go up. You know, as we've all gotten used to operating in this world, we expect our physicians and patients will as well. As you may have heard in one of the previous questions, in the U.S., as an example, we're doing more virtual patient meetings, and that seems to be very strong. And so we'll continue to look at that. We interact with physicians in different ways. We would never think that that would substitute for direct interactions, but we're going to use this time to see how we can augment that and really be a value add to our customers through this process.

Bradley L. Campbell: 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 they have, and physicians are answering the questions they have. And again, that experience with Gallifold, which has been very strong, will help us in any kind of setting, whether it's in a, you know, this normal or the new normal or whatever it is that, you know, really strong support function that we've built up through the Gallifold launch will continue. And we think that'll serve us well with the launch of Pumpet.

[noise].

Deb: This question is a follow-up from Deb. Thank you for holding back in again. So with regard to the Pompe gene therapy IND, we have very little information regarding whether it's a liver director, muscle director, et cetera. How should we be thinking about autophagy in terms of expression, uptake, et cetera, and also, you know, in crew negative patients, if it's not a liver-directed gene therapy, how do you circumvent that?

John Crowley: Great. Thanks, Deb.

Jeff Hung: We, of course, have more data coming out at the conference next week. I'm going to ask Hung to comment broadly about the technology approach that we're taking here. So, Hung, if you want to field that, and Jeff, Simon, if you have any other color.

Jeff Hung: Sure. You know, I think that we have described, at least in some detail, that our pump aging therapy program will use an [inaudible] It seems to transduce quite well, and we don't know, you know, if... At least we don't have any data to indicate that autophagy will be a problem for its ability to transduce. We know that that virus is able to deliver the gene, and it's able to get out of the endosome to basically transfer the gene pretty well in all the animal studies that we've done up to this point. Jeff, do you want to comment any more on that? Sure, Hung, thanks.

Jeffrey P. Castelli: Yeah, I think that's a really good answer. The only thing I'd re-emphasize is that, you know, the approach we're taking with our CAHPS and promoters will certainly transduce liver. It's just that in addition to that liver transduce...

Jeffrey P. Castelli: https://www.globalonenessproject.org

Operator: in the case of

Operator: For instance, a CRIB negative.

John Crowley: that might have some immunogenicity to the actual GAA protein produced by the cells. 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: Great. Thanks, Operator. That was a great conference call.

Operator: 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 conclude today's conference. Thank you, and have a great day.

Operator: Bye! We have an individual who's losing his call; I just didn't want to...

[noise] [laughter].

[laughter].

[laughter].